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Ascentage Pharma Receives Approvals for Two Phase Ib/II Clinical Studies of the Bcl-2 Inhibitor APG-2575 for the Treatment of Waldenstrm…

Posted: November 23, 2020 at 7:05 am

SUZHOU, China and ROCKVILLE, Md., Nov.23, 2020 /PRNewswire/ -- Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has approved two Phase Ib/II clinical studies of its novel Bcl-2 inhibitor APG-2575; one for APG-2575 as a single agent or in combination with ibrutinib/rituximab for the treatment of patients with Waldenstrm macroglobulinemia (WM), and the other one for APG-2575 as a single agent or in combination with lenalidomide/dexamethasone for the treatment of patients with multiple myeloma (MM).

APG-2575 is a novel, orally administered Bcl-2selective inhibitor being developed by Ascentage Pharma. APG-2575 is designed to treat several hematologic malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells. APG-2575 is the first China-developed Bcl-2 inhibitor having entered clinical development in China. APG-2575 has received clearances and approvals for multiple Phase Ib/II clinical studies in China, Australia, and the US, and is currently being developed in a range of hematologic malignancies globally. Of those studies, the Phase Ib/II study of APG-2575 as a single agent or in combination with ibrutinib/rituximab for the treatment of WM is a global multicenter trial with centers in Australia, China, and the US.

This global multicenter, open-label Phase Ib/II dose-expansion study is designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of APG-2575 as a single agent or in combination with ibrutinib/rituximab for the treatment of patients with WM.

WM is a rare indolent B-cell lymphoma, accounting for <2% of all non-Hodgkin's lymphoma (NHL) cases. Treatment recommendations for WM from current guidelines suggest an objective response rate (ORR) of about 80% with contemporary therapies, but they deliver a very low rate of very good partial response (VGPR) or deeper responses (20% or lower), with most patients eventually relapsing or experiencing further disease progression. Furthermore, patients are diagnosed with WM at a median age of 70, when many individuals are intolerant of aggressive therapies because of poor health conditions, hence presenting an urgent clinical need for more effective therapies[1].

Preclinical study data of APG-2575 have shown responses generated in WM models resistant or insensitive to ibrutinib, as well as the synergistic effect with ibrutinib in various models of NHL, including follicular lymphoma, diffuse large B-cell lymphoma, and WM.

This multicenter, open-label Phase Ib/II dose-escalation study to be carried out in China is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of APG-2575 as a single agent or in combination with lenalidomide/dexamethasone in patients with relapsed/refractory MM.

MM is a plasma cell proliferative disorder with manifestations like hypercalcemia, anemia, renal failure, and bone disease. MM remains incurable. As reported, MM accounts for about 1.8% of all malignant tumors and 18.2% of all hematopoietic neoplasms. MM is the second most common hematological malignancy[2]. The age-standardized incidence rate of MM in the US is approximately 6.9 per 100,000[3]. The incidence rate of MM in China has increased significantly in recent years, and the mortality rate increased with age, especially for patients over 60 years. The median age at diagnosis in China is 59 years, much younger than US (~69 years). The incidence also increases with age. With an aging population and advanced diagnostic capabilities, the prevalence of MM is anticipated to keep growing in China[4].

In the preclinical studies of Ascentage Pharma, APG-2575 demonstrated potent antiproliferative activity in MM cell lines bearing the chromosomal t (11;14). I And in MM cell lines without t (11;14), the combinations with lenalidomide or pomalidomide and dexamethasone greatly enhanced APG-2575 cell sensitivity and triggered more potent cell death.

"APG-2575 is a key drug candidate in our apoptosis-targeted pipeline, and the first China-developed selective Bcl-2 small-molecule inhibitor, with great therapeutic potential as a single agent or in combinations in a range of hematologic malignancies including WM and MM," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "There is a growing emphasis on combination therapy in cancer treatments. We will accelerate these studies of APG-2575 and strive to develop a new treatment option for patients in need."


[1] NCCN Clinical Practice Guidelines in Oncology for Waldenstrm Macroglobulinemia, Version 1.2020-December 6,2019

[2] Siegel, R. L., Miller, K. D., & Jemal, A. (2019). Cancer statistics, 2019. CA: a cancer journal for clinicians, 69(1), 7-34.

[3] Cancer Stat Facts: Myeloma; Surveillance, Epidemiology, and End Results Program, US National Cancer Institute. https://seer.cancer.gov/statfacts/html/mulmy.html Accessed on 2020.3.18

[4] Liu, J., Liu, W., Zeng, X., Ma, J., et al. Incidence and Mortality of Multiple Myeloma in China, 2006-2016: An Analysis of the Global Burden of Disease Study 2016. J Hematol Oncol. 2019; 12: 136.

About APG-2575

APG-2575 is a novel, orally administered Bcl-2selective inhibitor being developed by Ascentage Pharma. APG-2575 is designed to treat a variety of hematologic malignancies by selectively blocking Bcl-2 to restore the normal apoptosis process in cancer cells. Ascentage Pharma has previously commenced Phase I studies of APG-2575 single agent in China, Australia, and the United States. Since March 2020, the company has received approvals and clearances for several Phase Ib/II studies of APG-2575 in China, Australia, and the US, and is advancing clinical development of APG-2575 for a variety of hematologic malignancy indications, including relapsed/refractory chronic lymphocytic leukemia or small lymphocytic lymphoma, Waldenstrm macroglobulinemia, relapsed/refractory multiple myeloma, and relapsed/refractory acute myeloid leukemia. APG-2575 was recently granted two orphan drug designations by the US Food and Drug Administration in the treatment of Waldenstrm Macroglobulinemia and Chronic Lymphocytic Leukemia.

About Ascentage Pharma

Ascentage Pharma (6855.HK) is a globally, clinical-stage biotechnology company engaged in developing novel therapies for cancers, CHB, and age-related diseases. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 6855.HK.

Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis or programmed cell death. The company has built a pipeline of eight clinical drug candidates, including novel, highly potent Bcl-2, and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors. Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company is conducting more than 40 Phase I/II clinical trials in the US, Australia, and China. HQP1351, the company's core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia has been granted an Orphan Drug Designation (ODD) and a Fast Track designation by the US Food and Drug Administration (FDA), and a New Drug Application for the drug candidate has been submitted in China. To date, Ascentage Pharma has obtained a total of six ODDs from the FDA for four of the company's investigational drug candidates.

Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events, or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

SOURCE Ascentage Pharma


Ascentage Pharma Receives Approvals for Two Phase Ib/II Clinical Studies of the Bcl-2 Inhibitor APG-2575 for the Treatment of Waldenstrm...

CytoDyn Reaches Enrollment Target of 293 Patients for 2nd DSMC Interim Analysis of Phase 3 COVID-19 Trial and Expects to Enroll the Remaining 97…

Posted: at 7:05 am

Concurrently, CytoDyn is working diligently with the FDA to initiate its Phase 2 COVID-19 Long Hauler Trial, with more than 100 volunteers wanting to enroll

VANCOUVER, Washington, Nov. 23, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing Vyrologix (leronlimab-PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today it has reached enrollment of 293 patients in its Phase 3 trial for COVID-19 patients with severe-to-critical symptoms, thereby meeting the requested criteria for a second interim efficacy analysis by the Data Safety Monitoring Committee (DSMC).

After the first interim analysis, the DSMC requested a second interim analysis of all data after enrollment had reached 293 patients or 75% of the total patients for the trial. Approximately five weeks ago, the DSMC completed the first interim analysis on 195 patients (or 50% of the 390 planned patients) and recommended the trial continue without modification to achieve the primary endpoint and requested another interim analysis when enrollment reached 75% level (or 293 patients) to review patient mortality and other clinical outcome data.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn, commented, In addition to filing our biologics license applications in Canada and the U.K. for HIV, the Company is in full swing to obtain full enrollment in the Phase 3 COVID-19 trial before year end and initiate our Phase 2 trial for COVID-19 patients with multiple long-hauler symptoms and perhaps complete enrollment in 4-6 weeks. On another front, CytoDyn is also about to enroll its first patient in the NASH trial this month. We are very appreciative of the all-out effort by our clinical operations team (especially Mr. Brian Brothen, the Companys SVP of Global Oncology, and Dr. Kush Dhody from Amarex) and the clinical sites to expedite enrollment in this important trial and are hopeful the DSMC can complete their second interim analysis as quickly as possible. We continue to advance enrollment, without any pause to achieve the trials planned 390 patients and we are currently evaluating the ability to conduct an interim analysis as soon as possible. In the meantime, if the pace of enrollment we have experienced in the last two weeks continues, we will have the CD12 enrollment completed before the end of the year. These are exciting times for the Company and I am honored to be working alongside such dedicated co-workers.

About Coronavirus Disease 2019 CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a double-blinded, randomized clinical trial for mild-to-moderate patients in the U.S. which produced statistically significant results for NEWS2. Enrollment continues in its Phase 2b/3 randomized clinical trial for the severe-to-critically ill COVID-19 population in several hospitals and clinics throughout the U.S., which are identified on the Companys website under the Clinical Trial Enrollment section of the homepage; an interim analysis on the first 195 patients was conducted mid-October and is expected to occur again now that the Company has reached enrollment of 293 patients.

About Leronlimab (PRO 140) The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses. The first indication is a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells.CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDyn CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA met telephonically with Company key personnel and its clinical research organization and provided written responses to the Companys questions concerning its recent Biologics License Application (BLA) for this HIV combination therapy in order to expedite the resubmission of its BLA filing for this indication.

CytoDyn has completed a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than six years.

CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking StatementsThis press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Company's cash position, (ii) the Company's ability to raise additional capital to fund its operations, (iii) the Company's ability to meet its debt obligations, if any, (iv) the Company's ability to enter into partnership or licensing arrangements with third parties, (v) the Company's ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Company's ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Company's clinical trials, (viii) the results of the Company's clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Company's products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Company's control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSInvestors: Michael MulhollandOffice: 360.980.8524, ext. 102mmulholland@cytodyn.com

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CytoDyn Reaches Enrollment Target of 293 Patients for 2nd DSMC Interim Analysis of Phase 3 COVID-19 Trial and Expects to Enroll the Remaining 97...

Can-Fite to Conduct Investor Call to Review Q3 Results and Provide Business Update on Monday, November 30, 2020 at 9:15 a.m. ET – Business Wire

Posted: at 7:05 am

PETACH TIKVA, Israel--(BUSINESS WIRE)--Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address inflammatory, cancer and liver diseases, today announced it will conduct a conference call with investors to review financial results for its third quarter ended September 30, 2020, and provide an update on clinical and corporate developments, including its advanced stage drug candidates Piclidenoson and Namodenoson.

Call Information

Date: Monday, November 30, 2020

Time: 9:15 a.m. ET

Dial-in U.S.: 1-877-423-9813

Dial-in Israel: 1-201-689-8573

Conference ID: 13713545

Audio Webcast: http://public.viavid.com/index.php?id=142533

A press release reviewing the third quarter results and clinical updates will be issued prior to the call. A replay of the webcast will be archived on Can-Fites website for a period of time.

About Can-Fite BioPharma Ltd.

Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CFBI) is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion dollar markets in the treatment of cancer, liver, inflammatory disease and COVID-19. The Company's lead drug candidate, Piclidenoson, is currently in a Phase III trial for psoriasis and a Phase II study in the treatment of moderate COVID-19. Can-Fite's liver drug, Namodenoson, is headed into a Phase III trial for hepatocellular carcinoma (HCC), the most common form of liver cancer, and successfully achieved its primary endpoint in a Phase II trial for the treatment of non-alcoholic steatohepatitis (NASH). Namodenoson has been granted Orphan Drug Designation in the U.S. and Europe and Fast Track Designation as a second line treatment for HCC by the U.S. Food and Drug Administration. Namodenoson has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma. CF602, the Company's third drug candidate, has shown efficacy in the treatment of erectile dysfunction. These drugs have an excellent safety profile with experience in over 1,500 patients in clinical studies to date. For more information please visit: http://www.can-fite.com.

Forward-Looking Statements

This press release may contain forward-looking statements, about Can-Fites expectations, beliefs or intentions regarding, among other things, market risks and uncertainties, its product development efforts, business, financial condition, results of operations, strategies or prospects. In addition, from time to time, Can-Fite or its representatives have made or may make forward-looking statements, orally or in writing. Forward-looking statements can be identified by the use of forward-looking words such as believe, expect, intend, plan, may, should or anticipate or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. These forward-looking statements may be included in, but are not limited to, various filings made by Can-Fite with the U.S. Securities and Exchange Commission, press releases or oral statements made by or with the approval of one of Can-Fites authorized executive officers. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to risks and uncertainties that could cause Can-Fites actual results to differ materially from any future results expressed or implied by the forward-looking statements. Many factors could cause Can-Fites actual activities or results to differ materially from the activities and results anticipated in such forward-looking statements. Factors that could cause our actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: our history of losses and needs for additional capital to fund our operations and our inability to obtain additional capital on acceptable terms, or at all; uncertainties of cash flows and inability to meet working capital needs; the impact of the COVID-19 pandemic; the initiation, timing, progress and results of our preclinical studies, clinical trials and other product candidate development efforts; our ability to advance our product candidates into clinical trials or to successfully complete our preclinical studies or clinical trials; our receipt of regulatory approvals for our product candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of our product candidates; our ability to establish and maintain strategic partnerships and other corporate collaborations; the implementation of our business model and strategic plans for our business and product candidates; the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and our ability to operate our business without infringing the intellectual property rights of others; competitive companies, technologies and our industry; statements as to the impact of the political and security situation in Israel on our business; and risks and other risk factors detailed in Can-Fites filings with the SEC and in its periodic filings with the TASE. In addition, Can-Fite operates in an industry sector where securities values are highly volatile and may be influenced by economic and other factors beyond its control. Can-Fite does not undertake any obligation to publicly update these forward-looking statements, whether as a result of new information, future events or otherwise.

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Can-Fite to Conduct Investor Call to Review Q3 Results and Provide Business Update on Monday, November 30, 2020 at 9:15 a.m. ET - Business Wire

BBB says scammers using texts about phony clinical studies in effort to steal – The Advocate

Posted: November 20, 2020 at 8:57 pm

The Better Business Bureau of South Central Louisiana warns that scammers are sending out text messages promoting phony clinical studies that purportedly target the coronavirus. Those who fall for their ruse are tricked into giving their bank account information or downloading malicious software.

While scientists have sought volunteers to help in their research, none of them require people to pay to be a part of a study. Those behind real studies may ask medical-related questions and for demographic information, but not bank account numbers.

The unsolicited texts often promise pay for participation in a "Local Covid19 Study," the BBB warns.

"Its a scam! The phony message includes a link to see whether or not you qualify for the study," the agency said. Clicking the link could ultimately give scammers access to usernames, passwords or other personal information.

At other times, a link could take you to an official-looking site that request for personal information like a government ID or bank account number.

The BBB says the National Institutes of Health (NIH) and the National Library of Medicine (NLM) maintain ClinicalTrials.gov, a free searchable database of clinical studies on a wide range of diseases. If there is no government agency, university, or hospital mentioned, its likely a scam.

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BBB says scammers using texts about phony clinical studies in effort to steal - The Advocate

Meeting the Needs of Rare Disease Patients in Clinical Trials During the COVID-19 Pandemic, Upcoming Webinar Hosted by Xtalks – PR Web

Posted: at 8:57 pm

Rare diseases are an emerging global public health priority.

TORONTO (PRWEB) November 18, 2020

This webinar will discuss how the COVID-19 pandemic has impacted clinical studies for rare diseases and how patient-centric trial design with regulatory consideration can be utilized to mitigate and manage potential delays and support patient recruitment in rare and orphan clinical trials.

COVID-19 is a serious, life-threatening and fast-spreading viral infection that has had an unexpected global impact. As the world is learning to adapt to a new normal, so have clinical trials.

Rare diseases are an emerging global public health priority. There are more than 7,000 identified and classified rare diseases, 71.9% of which are genetic and 69.9% which are exclusively pediatric onset. Following the European definition of the incidence of a rare disease (no more than 5 in 10,000 individuals), it is estimated that there are over 440 million individuals globally who are affected by a rare disease at any point in time, excluding rare cancers, infectious diseases and poisonings. Rare diseases are numerous, heterogeneous in nature and geographically disparate. Few of them are preventable or curable, most are chronic, degenerative and many lead to early death. The inherent challenges are derived from their low prevalence, poor scientific knowledge and the scarcity of expertise, as well as their chronic, degenerative and life-threatening nature.

Patient groups, physicians and regulators are eager to support the delivery of rare and orphan clinical trials, allowing flexibility while maintaining the standards necessary to deliver high-quality data. Orphan drugs companies are looking for support and guidance on how to navigate this new challenging clinical landscape and CROs are working to deliver solutions that meet the needs of patients and drug developers.

Join experts from Simbec-Orion, Dr. Carlos Camozzi, Chair, Rare and Orphan Advisory Board; and Dr. Chirag Patel, Director of Regulatory Affairs, in a live webinar on Tuesday, December 8, 2020 at 9am EST (2pm GMT/UK).

For more information, or to register for this event, visit Meeting the Needs of Rare Disease Patients in Clinical Trials During the COVID-19 Pandemic.


Xtalks, powered by Honeycomb Worldwide Inc., is a leading provider of educational webinars to the global life science, food and medical device community. Every year, thousands of industry practitioners (from life science, food and medical device companies, private & academic research institutions, healthcare centers, etc.) turn to Xtalks for access to quality content. Xtalks helps Life Science professionals stay current with industry developments, trends and regulations. Xtalks webinars also provide perspectives on key issues from top industry thought leaders and service providers.

To learn more about Xtalks visit http://xtalks.comFor information about hosting a webinar visit http://xtalks.com/why-host-a-webinar/

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Meeting the Needs of Rare Disease Patients in Clinical Trials During the COVID-19 Pandemic, Upcoming Webinar Hosted by Xtalks - PR Web

INOVIO Presents Clinical Results of its DNA Medicines INO-5401 + INO-9012 in Novel Combination with PD-1 Inhibitor Libtayo (cemiplimab) in the…

Posted: at 8:57 pm

PLYMOUTH MEETING, Pa., Nov. 20, 2020 /PRNewswire/ -- INOVIO (NASDAQ: INO), a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases and cancer,announced today that data from the company's novel combination trial of DNA medicines INO-5401 and INO-9012 in combination with PD-1 inhibitor Libtayo (cemiplimab) in the treatment of newly diagnosed glioblastoma (GBM), will be presented by Dr. David Reardon in the plenary session at the Society for Neuro-Oncology (SNO) 2020 Annual Meeting. The study demonstrated that INO-5401 + INO-9012 with Libtayo, radiation (RT) and temozolomide (TMZ) are tolerable, immunogenic, and may improve median survival for patients with newly diagnosed GBM. Survival data at 18 months showed that 70% (14/20) of MGMT promoter methylated GBM patients were alive, and 50% (16/32) of MGMT promoter unmethylated patients, which are the more difficult to treat group, were alive after 18 months. Median overall survival in the unmethylated GBM patients was 17.9 months, which compares favorably to historical controls; Median OS for methylated patients has not yet been reached and the study is ongoing.

Dr. David Reardon, Clinical Director of the Center for Neuro-Oncology at the Dana-Farber Cancer Institute and coordinating principal investigator of GBM-001 said, "This is a landmark combination trial in which a novel DNA vaccine is combined with a checkpoint inhibitor and radiation and chemotherapy.We look forward to continuing to review these data, with an eye towards those patients who are most likely to benefit from this innovative approach and to see whether, over time, there is an extension of survival in these very hard-to-treat patients.Coupling immune response with clinical outcome may prove insightful."

Interim data demonstrated that in the MGMT promoter unmethylated cohort, 19/22 (86%) subjects to date had an IFN-gamma T cell response that increased over baseline to one or more of the antigens encoded by INO-5401. In the MGMT promoter methylated cohort, 16/17 (94%) subjects to date had an IFN-gamma response that increased over baseline to one or more of the antigens encoded by INO-5401. The novel combination of INO-5401 + INO-9012 continues to demonstrate a well-tolerated safety profile when given not only with radiation and TMZ, but also with PD-1 blockade by Libtayo, which is being jointly developed by Regeneron and Sanofi.

Dr. Jeffrey Skolnik, INOVIO's senior vice president, clinical development, said, "INO-5401 + INO-9012, with Libtayo and RT/TMZ, generates cancer antigen-specific T cells that may be able to attack GBM and provide a survival advantage. We are using our knowledge of immunology to define a patient population for which this novel DNA medicine plus checkpoint inhibitor combination may offer a survival advantage, by continuing to assess all of our data: efficacy, safety and most important, immunogenicity and tissue expression data."

Additional data will be provided in the coming months, including correlative immunology and tissue data, as well as total study drug exposure and concomitant medication use.

INO-5401, INO-9012 and Libtayo, and the combination of these products have not been approved or evaluated by any Regulatory Authority worldwide for the treatment of newly diagnosed GBM.

Presentation Details

Abstract: LTBK-01

Title: "INO-5401 and INO-9012 delivered intramuscularly (IM) with electroporation (EP) in combination with cemiplimab (REGN2810) in newly diagnosed glioblastoma"

Presenting Author: Dr. David ReardonPlenary Session Date and Time: 2020 SNO Annual Meeting, Plenary 1A, Friday, November 20, 2020 beginning at 11 a.m. EST

Study Design

The trial was designed to evaluate safety, immunogenicity and efficacy of INO-5401 and INO-9012 in combination with Libtayo, with radiation and chemotherapy, in subjects with newly diagnosed glioblastoma (GBM). This is a Phase 1/2, open-label, multi-center trial conducted in 52 evaluable patients with GBM. There are two cohorts in this trial. Cohort A includes 32 participants with a tumor with an unmethylated O6-methylguanine-deoxyribonucleic acid (DNA) methyltransferase (MGMT) promoter. Cohort B includes 20 participants with a tumor with a MGMT methylated promoter. Both cohorts received INO-5401 and INO-9012 and Libtayo at the same doses and on the same dosing schedule, and both cohorts received radiation and TMZ. For more information of the clinical study, seewww.clinicaltrials.gov, identifier NCT03491683.

About Glioblastoma Multiforme (GBM)

GBM is the most common and aggressive type of brain cancer and remains a devastating disease for both patients and caregivers. Its prognosis is extremely poor, despite a limited number of new therapies approved over the last 10 years. The median overall survival for patients receiving standard of care therapy is approximately 15 to 22 months and the median progression-free survival is approximately 7 months. In the U.S., the estimated annual incidence of GBM is 11,362 cases or 3.21 cases per 100,000 persons and the median age at diagnosis is 65 years.

About INO-5401 and INO-9012

INO-5401 encodes for INOVIO's SynCon antigens for hTERT, WT1, and PSMA, and has the potential to be a powerful cancer immunotherapy in combination with checkpoint inhibitors. The National Cancer Institute previously highlighted hTERT, WT1, and PSMA among a list of important cancer antigens, designating them as high priorities for cancer immunotherapy development. These three antigens were reported to be over-expressed, and often mutated, in a variety of human cancers, and targeting these antigens may prove efficacious in the treatment of patients with cancer. INO-9012 encodes for IL-12, which is a T cell immune activator.

About INOVIO's DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including coronaviruses associated with MERS and COVID-19 diseases being developed under grants from the Coalition for Epidemic Preparedness Innovations (CEPI) and the U.S. Department of Defense. DNA medicines are composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

INOVIO's DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using INOVIO's proprietary hand-held smart device called CELLECTRA. The CELLECTRA device uses a brief electrical pulse to reversibly open small pores in the cell to allow the plasmids to enter, overcoming a key limitation of other DNA and other nucleic acid approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the cell to produce the targeted antigen. The antigen is processed naturally in the cell and triggers the desired T cell and antibody-mediated immune responses. Administration with the CELLECTRA device ensures that the DNA medicine is efficiently delivered directly into the body's cells, where it can go to work to drive an immune response. INOVIO's DNA medicines do not interfere with or change in any way an individual's own DNA. The advantages of INOVIO's DNA medicine platform are how fast DNA medicines can be designed and manufactured; the stability of the products, which do not require freezing in storage and transport; and the robust immune response, safety profile, and tolerability that have been observed in clinical trials.

With more than 2,000 patients receiving INOVIO investigational DNA medicines in more than 7,000 applications across a range of clinical trials, INOVIO has a strong track record of rapidly generating DNA medicine candidates with potential to meet urgent global health needs.


INOVIO is a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases, cancer, and diseases associated with HPV. INOVIO is the first and only company to have clinically demonstrated that a DNA medicine can be delivered directly into cells in the body via a proprietary smart device to produce a robust and tolerable immune response. Specifically, INOVIO's lead candidate VGX-3100, currently in Phase 3 trials for precancerous cervical dysplasia, destroyed and cleared high-risk HPV 16 and 18 in a Phase 2b clinical trial. High-risk HPV is responsible for 70% of cervical cancer, 91% of anal cancer, and 69% of vulvar cancer. Also in development are programs targeting HPV-related cancers and a rare HPV-related disease, recurrent respiratory papillomatosis (RRP); non-HPV-related cancers glioblastoma multiforme (GBM) and prostate cancer; as well as externally funded infectious disease DNA vaccine development programs in Zika, Lassa fever, Ebola, HIV, and coronaviruses associated with MERS and COVID-19 diseases. Partners and collaborators include Advaccine, ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for Epidemic Preparedness Innovations (CEPI), Defense Advanced Research Projects Agency (DARPA)/Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND)/Department of Defense (DOD), HIV Vaccines Trial Network, International Vaccine Institute (IVI), Medical CBRN Defense Consortium (MCDC), National Cancer Institute, National Institutes of Health, National Institute of Allergy and Infectious Diseases, Ology Bioservices, the Parker Institute for Cancer Immunotherapy, Plumbline Life Sciences, Regeneron, Richter-Helm BioLogics, Thermo Fisher Scientific, University of Pennsylvania, Walter Reed Army Institute of Research, and The Wistar Institute. INOVIO also is a proud recipient of 2020 Women on Boards "W" designation recognizing companies with more than 20% women on their board of directors. For more information, visit http://www.inovio.com.

CONTACTS:Investors: Ben Matone, 484-362-0076, ben.matone@inovio.comMedia: Jeff Richardson, 267-440-4211, jrichardson@inovio.com

This press release contains certain forward-looking statements relating to our business, including our plans to develop DNA medicines, our expectations regarding our research and development programs, including the planned initiation and conduct of preclinical studies and clinical trials and the availability and timing of data from those studies and trials, and our ability to successfully manufacture and produce large quantities of our product candidates if they receive regulatory approval. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials, product development programs and commercialization activities and outcomes, our ability to secure sufficient manufacturing capacity to mass produce our product candidates, the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable DNA medicines, our ability to support our pipeline of DNA medicine products, the ability of our collaborators to attain development and commercial milestones for products we license and product sales that will enable us to receive future payments and royalties, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by us or our collaborators, including alternatives that may be more efficacious or cost effective than any therapy or treatment that we and our collaborators hope to develop, issues involving product liability, issues involving patents and whether they or licenses to them will provide us with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether we can finance or devote other significant resources that may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of our technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 and other filings we make from time to time with the Securities and Exchange Commission. There can be no assurance that any product candidate in our pipeline will be successfully developed, manufactured or commercialized, that final results of clinical trials will be supportive of regulatory approvals required to market products, or that any of the forward-looking information provided herein will be proven accurate. Forward-looking statements speak only as of the date of this release, and we undertake no obligation to update or revise these statements, except as may be required by law.

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INOVIO Presents Clinical Results of its DNA Medicines INO-5401 + INO-9012 in Novel Combination with PD-1 Inhibitor Libtayo (cemiplimab) in the...

NorthSea Therapeutics doses first patient in Phase 1 trial of SEFA-1024 in dyslipidemia and further expands its clinical pipeline – BioSpace

Posted: November 19, 2020 at 3:58 pm

Nov. 19, 2020 09:00 UTC

NAARDEN, The Netherlands--(BUSINESS WIRE)-- NorthSea Therapeutics B.V, (NST) a Dutch biotech company developing novel and innovative therapies for NASH (Non-alcoholic Steatohepatitis) and other metabolic, inflammatory, and fibrotic diseases based on its SEFA platform, today announces the dosing of the first patient with SEFA-1024 in a Phase 1 study. SEFA stands for Structurally Engineered Fatty Acid, i.e. chemically engineered fatty acids, to generate compounds with differentiated physiochemical properties.

This Phase 1 trial will evaluate the safety, tolerability, and pharmacokinetics of SEFA-1024 in 96 otherwise healthy volunteers with elevated plasma triglyceride levels. Hypertriglyceridemia may cause acute pancreatitis and is often associated with other conditions that increase the risk of heart disease and stroke, including type 2 diabetes, obesity, and metabolic syndrome.

SEFA-1024 is a novel, orally-administered, highly potent, chemically modified fatty acid derived from naturally occurring fatty acid EPA. SEFA-1024 was designed to enhance its pharmacological effects on metabolic markers in the intestine and liver.

In pre-clinical dyslipidemia models, SEFA-1024 exhibited broad and marked beneficial effects, significantly reducing both non-HDL cholesterol and triglycerides in conjunction with an increase in HDL cholesterol. The cholesterol-lowering activity of SEFA-1024 was additive to the lowering obtained with statin treatment, suggesting that SEFA-1024 could be used in combination with these important standard-of-care cardiovascular therapies. Further, it was shown that dosing with SEFA-1024 leads to marked improvements in glycemic control, indicating that SEFA-1024 has the potential to improve overall glycemic control in patients with diabetes.

Professor John Kastelein, NST scientific advisory board and supervisory board member commented: SEFA-1024 has shown, in a range of pre-clinical models, a broad effect on major metabolic markers and the availability of the oral, once-daily therapy could optimize the management of multiple dyslipidemia diseases in patients with high triglycerides or mixed dyslipidemia.

Rob de Ree, NSTs CEO said: The commencement of the SEFA-1024 trial is a significant landmark in our move to become a multi-asset company, expanding beyond the success so far of our existing program in liver disease, and delivering on our promise to produce multiple clinical candidates with the SEFA technology. Our move to focus on dyslipidemia enables us to target new disease areas with products where the potential to help patients reach their therapeutic targets with a convenient oral therapy is substantial.

SEFA-1024 is one of NSTs three SEFA programs. NSTs lead program, icosabutate, is currently in a phase 2b ICONA study for the treatment of NASH. The Companys third SEFA, 6179, is expected to enter the clinic in the first half of 2021, targeting IFALD, for which no treatment is currently available.

1 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6068776/


Notes to Editors

About NorthSea Therapeutics

NorthSea Therapeutics B.V.(NST) is a Dutch biotech company focused on developing structurally engineered fatty acids ('SEFAs') for the treatment of inflammatory, metabolic, and liver diseases. NST licensed the rights to its lead compound icosabutate and a library of discovery- and pre-clinical-stage SEFAs from Pronova BioPharma Norge AS, which developed Omacor, a blockbuster cardiovascular drug. Icosabutate was been found to be safe and effective in two prior Phase 2 clinical studies for the treatment of hypertriglyceridemia and is currently in clinical development for non-alcoholic steatohepatitis (NASH). A Phase 2b study was initiated in July 2019 (ICONA) to study the efficacy of icosabutate in NASH. NST is headquartered in the Netherlands with a presence in the UK and Norway and is supported by several investors including Forbion, Novo Seeds, BGV, NSV venBio Partners, and Sofinnova Investments. Find out more about us online at http://www.northseatherapeutics.com

View source version on businesswire.com: https://www.businesswire.com/news/home/20201119005111/en/

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NorthSea Therapeutics doses first patient in Phase 1 trial of SEFA-1024 in dyslipidemia and further expands its clinical pipeline - BioSpace

Freeline to present at the 3rd Annual Evercore ISI Health CONx Conference – GlobeNewswire

Posted: at 3:58 pm

LONDON, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline), a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases, today announced that management will participate in a fireside chat at the 3rd Annual Evercore ISI Health CONx Conference on 3 December, 2020 from 08.00 8.20 am ET.

A live audio webcast of the fireside chat will be available on the events section of Freelines website. An archived replay will be available on the Companys website for a period of 90 days after the conference.

About FreelineFreeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the UK and has operations in Germany and the US.

Forward-Looking StatementsThis press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Companys opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include discussion of the Companys strategies, financing plans, and clinical trial plans. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work; the Companys ability to design and implement successful clinical trials for its product candidates; the potential for a pandemic, epidemic or outbreak of infectious diseases in the U.S., U.K. or EU, including the COVID-19 pandemic, to disrupt the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience which could result in delays in the development or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law.

For further information, please reference the Companys reports and documents filed with theU.S. Securities and Exchange Commission. You may get these documents by visiting EDGAR on theSECwebsite atwww.sec.gov.

Further information:

United StatesLifeSci AdvisorsDan Ferry+1 (617) 430 7576daniel@lifesciadvisors.com

EuropeJW CommunicationsJulia Wilson+44 (0) 7818 430877juliawilsonuk@gmail.com

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Freeline to present at the 3rd Annual Evercore ISI Health CONx Conference - GlobeNewswire

Clinical Trials Market Analysis, COVID-19 Impact,Outlook, Opportunities, Size, Share Forecast and Supply Demand 2021-2027|Trusted Business Insights -…

Posted: at 3:58 pm

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Clinical Trials market.

Trusted Business Insights presents an updated and Latest Study on Clinical Trials Market 2020-2029. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Clinical Trials market during the forecast period (2020-2029).It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

Get Sample Copy of this Report @ Clinical Trials Market 2020 and Forecast 2021-2027 Includes Business Impact Analysis of COVID-19

Report Overview: Clinical Trials Market

The global clinical trials market size was valued at USD 35.7 billion in 2020 and looks set to grow at a compound annual growth rate (CAGR) of 4.9% from 2021 to 2027. Increasing prevalence of chronic disease and growing demand for clinical trials in developing countries is fueling market growth. Rising number of biologics, need for personalized medicines and orphan drugs, and demand for advanced technologies are other factors projected to fuel growth. Factors such as globalization of clinical trials, technological evolution, and demand for Contract Research Organizations (CROs) to conduct clinical trials are further projected to drive growth.

A growing demand of CROs for conducting clinical trials in the pharmaceutical sector due to the diversified expertise of CROs and adoption of advanced technologies in clinical trials is supporting the market growth. Digitization in biomedical research is also paving the way for the market growth. Incorporating advanced technologies such as electronic data capture (EDC) aid market participants in managing patient data that ultimately reduces monitoring costs. Digitization also helps in meeting stringent regulations by maintaining patient data records that reduces trial process errors through adoption of software such as electronic clinical outcome assessment (e-COA).The market is also driven by the emergence of global pandemic caused by coronavirus. The rapidly evolving threat due to the COVID-19 outbreak is impacting lives, communities, businesses, and industries around the world. The pandemic has also negatively impacted the current ecosystem of clinical trials. It has affected many ongoing trials for various therapeutic areas. However, to overcome this, researchers are rapidly trying to develop innovative therapeutics and vaccines against COVID-19, which is supporting the market growth.

The current pandemic is changing the way of conducting ongoing or upcoming clinical trials. Regulatory agencies such as the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), National Institutes of Health (NIH), and Chinas National Medical Products Administration and several others have issued guidelines related to the conduct of trials during the outbreak of coronavirus, which are in complete support of incorporating virtual services.The current scenario across the globe and the need to come up with treatment options has also led to the fast-track of clinical trials. The favorable government support is boosting the market growth. The World Health Organization (WHO) launched Solidarity, an international clinical trial launched, to find effective treatment against COVID-19. It includes comparing four treatment options against the standard of care to evaluate their effectiveness against Coronavirus. WHO also announced in May 2020; an international alliance for simultaneously developing numerous candidatevaccinesto prevent the spread for coronavirus disease, calling this effort theSolidarity trial for vaccines.

Phase Insights: Clinical Trials Market

The Phase III segment lead the global clinical trials market with a share of 53.0% in 2019. This is attributed to the fact that Phase III trials are the most expensive ones and involves huge subjects. The median cost for single Phase III trials is around USD 19.0 million with 59 new therapeutic agents approved by the FDA from 2015 to 2016. Also, Phase III requires higher number of patients and often a longer treatment period.

The Phase II segment followed Phase III in terms of market share accounting for 19.8% in 2019. It is also the second most expensive stage after Phase III. This study is performed in two parts. The first part includes exploring a range of doses along with efficacy studies, and the second part includes finalizing the dose. Phase II plays a crucial role, especially in oncology-related studies. The FDA estimates that about 33.0% of medication goes into Phase III trial. Besides, there are various therapeutic and vaccines currently in Phase II for the treatment of coronavirus, thereby, boosting the market growth.

For instance, there are currently 43 therapeutics in Phase II for COVID-19. Companies engaged in the development are Astrazeneca plc; Arch Biopartners Inc.; Applied Therapeutics Inc.; Apeiron Biologics GmbH; 4D Pharma plc; AB Science SA; and others. Companies are collaborating with other firms to accelerate the development of therapeutics and vaccines. Examples include Eli Lilly partnered with AbCellera for the development of vaccines; GSK, Novartis & MSD working with the Bill & Melinda Gates Foundation. GSK & Sanofi are working together to develop an adjuvanted COVID-19 vaccine.

Study Design Insights: Clinical Trials Market

The interventional design segment led the market and accounted for the largest revenue share with 45.6% in 2019. It is one of the most prominent methods used in the clinical trial. The interventional studies comprise 79.0% of total registered studies as of May 2020, out of which the majority of studies are for drug or biologics, followed by behavioral, clinical procedure, and device intervention studies. Intervention studies contribute to 94.0% of total studies with posted results out of which drug or biologics contribute the most, followed by behavioral, devices, and clinical procedure intervention studies.

Expanded access trials, also referred to as compassionate use trials, are anticipated to register the highest CAGR of 4.1% during the forecast period. It is a potential pathway for patients with serious disease conditions to carry out treatment outside the trial, when no satisfactory therapies are available. Increasing innovation in clinical trial methods is projected to drive the growth of the expanded access trials segment. For example, numerous oncology drugs are regularly administered to patients before their approval by the U.S. FDA and are considered as a part of expanded access trial. Also, there are currently 20 therapeutics for COVID-19 in expanded access trials/compassionate use in Phase II/III. Firms currently engaged in expanded access include Incyte Corp.; Novartis AG; Capricor Therapeutics Inc.; Alexion Pharmaceuticals Inc.; Bellerophon Therapeutics Inc.; Algernon Pharmaceuticals Inc. and subsidiary Nash Pharmaceuticals, and Ansun Biopharma Inc.

Indication Insights: Clinical Trials Market

Oncology segment accounted for the largest revenue share of 23.2% in 2019. The segment is also anticipated to witness the fastest CAGR of 5.4% over the forecast period 2021-2027. As per the U.S. FDA and various other sources, more than USD 38.0 billion is currently spent by the pharmaceutical industry towards preclinical and clinical development of oncology therapy products.

Cardiovascular condition segment is also anticipated to witness lucrative growth of 4.3% over the forecast period. The growing prevalence and increased demand for cost-effective medication across the world led to significant investment in R&D in this segment with more than 190 drugs in pipeline. Majority of drugs in the pipeline are for heart failure, lipid disorders, vascular diseases, and stroke. Growing demand for cost-effective medicine in low and middle-income countries is expected to increase the R&D investment by the government in this segment, thereby strengthening the market growth.

However, the current pandemic is creating a threat and acting as an obstacle in the clinical trials for finding effective treatments and cures for a myriad of diseases. At least 18 pharma or biotech companies have reported disruption to a clinical trial due to this pandemic. In March, there was around 65.0% global average decrease in the enrollment of new patients year-over-year. 84.0% reduction in India and a 43.0% decrease in Japan were also observed. The U.S. is down by an average of 67.0%.

Regional Insights: Clinical Trials Market

North America accounted for 51.2% of the global market in 2019 and is expected to continue its dominance over the forecast period. This can be attributed to increasing R&D and increasing adoption of new technologies in clinical research. For instance, incorporation of virtual services in the clinical trial protocol by market players such as IQVIA, and PRA Health Sciences is anticipated to further fuel the market growth.

Moreover, the favorable government support in the U.S. regarding the clinical trials is anticipated to boost the demand. For instance, in March 2020, the FDA launched a Coronavirus Treatment Acceleration Program (CTAP) for possible therapies to speed-up the development of treatment for the global disease caused by the coronavirus. The program employs every available way to provide novel treatment to patients as rapidly as possible, simultaneously finding out whether they are harmful or helpful.

Asia Pacific region is anticipated to expand at the fastest CAGR of 5.9% over the forecast period owing to the increasing availability of large patient pool facilitating easy recruitment of candidates. The global pandemic is also the main contributing factor for market growth. As per Asia Pacifics largest expertized biotech CRO Novotech, the company is observing an increase in the demand from biotechnology sponsors for studies in the region due to the availability of quality and speed. An increasing number of biotechnology firms are looking at Asia Pacific for their COVID-19 trials to take advantage of the large patient pool and fast-track procedures.

Key Companies & Market Share Insights: Clinical Trials Market

The global market is highly competitive. Some of the players operating in the market include IQVIA, PAREXEL International Corporation, Pharmaceutical Product Development, LLC; PAREXEL International Corporation, and Charles River Laboratory. Significant factors affecting competitive nature are the quick adoption of advanced technology for improved healthcare. Also, to retain share and expand the product portfolio, major players are often involved in mergers and acquisitions along with new product launches.

For instance, in January 2020, Wuxi AppTec announced offering a fully integrated adeno-associated virus Vector Suspension Platform to speed up the cell and gene therapy development, manufacturing and launch, thus, expanding its service capabilities. Another instance occurred in June 2018, where Acurian and Synexus, a part of PPD, launched SynexusPlus. SynexusPlus is a site solution for patient enrollment in clinical studies. This initiative is anticipated to improve the clinical trial productivity. Besides, the solution is also helpful in the current pandemic of COVID-19 as it helps reduce the site footprints. Some of the prominent players in the clinical trials market include:

Key companies Profiled: Clinical Trials Market Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2016 to 2027. For the purpose of this study, Trusted Business Insights has segmented the global clinical trials market report on the basis of phase, study design, indication, and region:

Phase Outlook (Revenue, USD Million, 2016 2027)

Study Design Outlook (Revenue, USD Million, 2016 2027)

Indication Outlook (Revenue, USD Million, 2016 2027)

Looking for more? Check out our repository for all available reports on Clinical Trials in related sectors.

Quick Read Table of Contents of this Report @ Clinical Trials Market 2020 and Forecast 2021-2027 Includes Business Impact Analysis of COVID-19

Trusted Business InsightsShelly ArnoldMedia & Marketing ExecutiveEmail Me For Any ClarificationsConnect on LinkedInClick to follow Trusted Business Insights LinkedIn for Market Data and Updates.US: +1 646 568 9797UK: +44 330 808 0580

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Artelo Biosciences Announces Clinical Trial Authorization to Commence Cancer Appetite Recovery Study for the Treatment of Cancer-Related Anorexia and…

Posted: at 3:58 pm

First patients on track for enrollment this year

Targeting multi-billion market with no approved therapies for cancer-related anorexia

LA JOLLA, Calif., Nov. 16, 2020 (GLOBE NEWSWIRE) -- Artelo Biosciences, Inc. (NASDAQ: ARTL), a clinical stage biopharmaceutical company focused on the development of therapeutics that modulate endogenous signaling pathways, including the endocannabinoid system, today announced receipt of the Clinical Trial Authorization (CTA) in the UK for the Companys Cancer Appetite Recovery Study (CAReS). The Medicines and Healthcare products Regulatory Agency (MHRA) authorized the initiation of the study entitled A Phase 1/2 Trial of Synthetic Cannabinoid ART27.13 in Patients with Cancer Anorexia and Weight Loss. Artelo expects the study to initiate enrollment before year end.

Receiving our Clinical Trial Authorization clears the path to commence our CAReS trial and we are excited about the prospect of enrolling patients this year, stated Andrew Yates, PhD., Program Leader for ART27.13. We will now proceed to open up sites throughout the UK with an overall recruitment goal of 43 patients, while maintaining safe and efficient operations during the Covid-19 pandemic.

Steven D. Reich, M.D., Artelos Chief Medical Officer, added, Cancer-related anorexia is a dramatically underserved market, with no approved therapies. Cancer-related anorexia affects greater than 60% of advanced stage cancer patients and it is characterized by loss of appetite, weight loss, poor quality of life and often precedes a patients death. Reich continued, The Phase 1/2 CAReS trial is designed to determine the most effective and safest dose and to evaluate activity using criteria such as lean body mass, weight gain, and improvement of anorexia.

About Artelo Biosciences

Artelo Biosciences, Inc. is a San Diego-based biopharmaceutical company dedicated to the development and commercialization of proprietary therapeutics that modulate endogenous signaling pathways, including the endocannabinoid system. Artelo is rapidly advancing a portfolio of broadly applicable product candidates designed to address significant unmet needs in multiple diseases and conditions, including anorexia, cancer, pain, inflammation, and anxiety. Led by proven biopharmaceutical executives collaborating with highly respected researchers and technology experts, the company applies leading edge scientific, regulatory, and commercial discipline to develop high-impact therapies. More information is available at http://www.artelobio.com and Twitter: @ArteloBio.

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About ART27.13

ART27.13 is a highly potent, peripherally restricted synthetic, dual G protein-coupled receptor agonist believed to target peripheral CB1/CB2 receptors, which has the potential to increase appetite and food intake. Originally developed by AstraZeneca plc, ART27.13 has been in five Phase 1 clinical studies including over 200 subjects where it demonstrated a statistically significant and dose-dependent increase in body weight in healthy subjects. Importantly, the changes in body weight were not associated with fluid retention and the distribution of the drug enables systemic metabolic effects while minimizing central nervous system mediated toxicity. Artelo plans to advance ART27.13 as a supportive care therapy for cancer patients suffering from anorexia and weight loss where the current annual global market is estimated to be valued in excess of $2 billion.

Forward Looking StatementsThis press release contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and Private Securities Litigation Reform Act, as amended, including those relating to the Companys product development, clinical and regulatory timelines, market opportunity, competitive position, possible or assumed future results of operations, business strategies, potential growth opportunities and other statement that are predictive in nature. These forward-looking statements are based on current expectations, estimates, forecasts and projections about the industry and markets in which we operate and managements current beliefs and assumptions. These statements may be identified by the use of forward-looking expressions, including, but not limited to, expect, anticipate, intend, plan, believe, estimate, potential, predict, project, should, would and similar expressions and the negatives of those terms. These statements relate to future events or our financial performance and involve known and unknown risks, uncertainties, and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such factors include those set forth in the Companys filings with the Securities and Exchange Commission, including our ability to raise additional capital in the future. Prospective investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this press release. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise, except to the extent required by applicable securities laws.

Investor Relations Contact:Crescendo Communications, LLCTel: 212-671-1020Email: ARTL@crescendo-ir.com

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Artelo Biosciences Announces Clinical Trial Authorization to Commence Cancer Appetite Recovery Study for the Treatment of Cancer-Related Anorexia and...

Arbutus Announces Presentation of Phase 1a/1b Clinical Trial Results for AB-729 in Chronic Hepatitis B Subjects at The Liver Meeting Digital…

Posted: at 3:58 pm

Across all single-dose cohorts, mean HBsAg concentrations continuously declined up to week 12 before reaching a plateau, suggesting dosing of AB-729 less frequently than every 4 weeks may be warranted

In the 60 mg every 4 weeks multi-dose cohort, HBsAg concentrations continued to decline steadily beyond week 12 with no plateau in response observed to date

Both HBV RNA and HBcrAg concentrations declined after single- and multi-dose administration of AB-729

AB-729 was generally safe and well tolerated

Conference Call and Webcast Scheduled for Monday, November 16, 2020 at 8:00 am ET

WARMINSTER, Pa., Nov. 15, 2020 (GLOBE NEWSWIRE) -- Arbutus Biopharma Corporation(Nasdaq: ABUS), a clinical-stage biopharmaceutical company primarily focused on developing a cure for people with chronic hepatitis B virus (HBV) infection as well as therapies to treat coronaviruses (including COVID-19), today announced the presentation of updated clinical data from an ongoing Phase 1a/1b clinical trial (AB-729-001) with AB-729, its proprietary GalNAc delivered RNAi compound. The presentation, entitled Safety and pharmacodynamics of the GalNAc-siRNA AB-729 in subjects with chronic hepatitis B infection, was presented by Professor Man-Fung Yuen, D.Sc., M.D., Ph.D., Chief of Division of Gastroenterology and Hepatology, Department of Medicine, The University of Hong Kong, Hong Kong, during a virtual oral session: Hepatitis B: Therapeutics (New) at The Liver Meeting Digital ExperienceTM, The American Association for the Study of Liver Diseases Meeting.

Summary of presented data

Single-doses of AB-729 studied to date, 60 mg, 90 mg and 180 mg, resulted in comparable mean HBsAg declines at week 12, followed by a sustained plateau phase. During the multiple-dose portion of the trial, 60 mg of AB-729 dosed every 4 weeks resulted in continuous declines in HBsAg, reaching a mean of 1.44 log10 IU/ML at week 16. Data beyond week 16 demonstrate further declines in HBsAg with no plateau seen to date. AB-729 also resulted in meaningful decreases in both HBV RNA and HBcrAg. AB-729 was generally safe and well tolerated. The presentation can be accessed through the Investors section under Events & Presentations of Arbutus' website at http://www.arbutusbio.com.

Repeat dosing of AB-729 60 mg every 4 weeks results in continuous HBsAg declines beyond week 12

Professor Yuen stated These are the first multi-dose data for AB-729 and show continuous decline of HBsAg throughout the dosing period. Importantly, AB-729 was generally safe and well tolerated. These encouraging data support the continued development of AB-729 as a potential cornerstone of future combination regimens for the treatment of chronic hepatitis B infection.

Summary of clinical trial design

AB-729-001 is an ongoing first-in-human clinical trial consisting of three parts:

In Part 1, three cohorts of healthy subjects were randomized 4:2 to receive single-doses (60 mg, 180 mg or 360 mg) of AB-729 or placebo.

In Part 2, non-cirrhotic, HBeAg positive or negative, chronic HBV subjects (N=6) on a background of nucleos(t)ide therapy with HBV DNA below the limit of quantitation received single-doses (60 mg to 180 mg) of AB-729. An additional cohort in Part 2 included 90 mg single-dose of AB-729 in HBV DNA positive chronic HBV subjects.

In Part 3, chronic HBV subjects, HBV DNA negative first and HBV DNA positive later, are receiving multi-doses of AB-729 for up to six months.

About AB-729

AB-729 is an RNA interference (RNAi) therapeutic targeted to hepatocytes using Arbutus novel covalently conjugated N-acetylgalactosamine (GalNAc) delivery technology that enables subcutaneous delivery. AB-729 inhibits viral replication and reduces all HBV antigens, including hepatitis B surface antigen in preclinical models. Reducing hepatitis B surface antigen is thought to be a key prerequisite to enable reawakening of a patients immune system to respond to the virus. In an ongoing single- and multi-dose Phase 1a/1b clinical trial, AB-729 demonstrated positive safety and tolerability data and meaningful reductions in hepatitis B surface antigen.

About HBV

Chronic hepatitis B virus (HBV) infection is a debilitating disease of the liver that afflicts over 250 million people worldwide with up to 90 million people inChina, as estimated by theWorld Health Organization. HBV is a global epidemic that affects more people than hepatitis C virus (HCV) and HIV infection combinedwith a higher morbidity and mortality rate. HBV is a leading cause of chronic liver disease and need for liver transplantation, and up to one million people worldwide die every year from HBV-related causes.

The current standard of care for patients with chronic HBV infection is life-long suppressive treatment with medications that reduce, but do not eliminate, the virus, resulting in very low cure rates. There is a significant unmet need for new therapies to treat HBV.

Conference Call and Webcast

Arbutus will hold a conference call and webcast on Monday, November 16, 2020 at 8:00 am Eastern Time to provide an AB-729 clinical update. You can access a live webcast of the call, which will include presentation slides, through the Investors section of Arbutus website at http://www.arbutusbio.com or directly at Live Webcast. Alternatively, you can dial (866) 393-1607 or (914) 495-8556 and reference conference ID 7791835.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (855) 859-2056 or (404) 537-3406, and reference conference ID 7791835.

About Arbutus

Arbutus Biopharma Corporationis a publicly traded (Nasdaq: ABUS) biopharmaceutical company primarily dedicated to discovering, developing and commercializing a cure for people with chronic hepatitis B virus (HBV) infection. The Company is advancing multiple drug product candidates that may be combined into a potentially curative regimen for chronic HBV infection. Arbutus has also initiated a drug discovery and development effort for treating coronaviruses (including COVID-19). For more information, please visitwww.arbutusbio.com.

Forward-Looking Statements and Information

This press release contains forward-looking statements within the meaning of the Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, and forward-looking information within the meaning of Canadian securities laws (collectively, forward-looking statements). Forward-looking statements in this press release include statements regarding the Companys expectation that AB-729 could be the cornerstone of future combination regimens for the treatment of chronic hepatitis B infection.

With respect to the forward-looking statements contained in this press release, Arbutus has made numerous assumptions regarding, among other things: the effectiveness and timeliness of preclinical studies and clinical trials, and the usefulness of the data; the timeliness of regulatory approvals; the continued demand for Arbutus assets; and the stability of economic and market conditions. While Arbutus considers these assumptions to be reasonable, these assumptions are inherently subject to significant business, economic, competitive, market and social uncertainties and contingencies, including uncertainties and contingencies related to the ongoing COVID-19 pandemic.

Additionally, there are known and unknown risk factors which could cause Arbutus actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements contained herein. Known risk factors include, among others: anticipated pre-clinical studies and clinical trials may be more costly or take longer to complete than anticipated, and may never be initiated or completed, or may not generate results that warrant future development of the tested drug candidate; Arbutus may elect to change its strategy regarding its product candidates and clinical development activities; Arbutus may not receive the necessary regulatory approvals for the clinical development of Arbutus products; economic and market conditions may worsen; market shifts may require a change in strategic focus; and the ongoing COVID-19 pandemic could significantly disrupt Arbutus clinical development programs.

A more complete discussion of the risks and uncertainties facing Arbutus appears in Arbutus Annual Report on Form 10-K, Arbutus Quarterly Reports on Form 10-Q and Arbutus continuous and periodic disclosure filings, which are available at http://www.sedar.comand at http://www.sec.gov. All forward-looking statements herein are qualified in their entirety by this cautionary statement, and Arbutus disclaims any obligation to revise or update any such forward-looking statements or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, except as required by law.

Contact Information

Investors and Media

William H. CollierPresident and CEO Phone: 267-469-0914Email: ir@arbutusbio.com

Pam MurphyInvestor Relations ConsultantPhone: 267-469-0914Email: ir@arbutusbio.com

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Arbutus Announces Presentation of Phase 1a/1b Clinical Trial Results for AB-729 in Chronic Hepatitis B Subjects at The Liver Meeting Digital...

Basilea announces Clinical Trial Collaboration and Supply Agreement with Eli Lilly and Company for ramucirumab in the ongoing FIDES-03 study with…

Posted: October 28, 2020 at 11:54 am

Basel, Switzerland, October 28, 2020

Basilea Pharmaceutica Ltd. (SIX: BSLN) announced today that it has entered into a clinical trial collaboration and supply agreement with Eli Lilly and Company for the use of the anti-VEGFR2 antibody ramucirumab (CYRAMZA)1 in the ongoing multi-cohort phase 1/2 study FIDES-03 with the FGFR inhibitor derazantinib in advanced gastric (stomach) cancer patients with FGFR genetic aberrations. Basilea is the sponsor of the study and Lilly will collaborate on clinical aspects and provide clinical supply of ramucirumab.

Ramucirumab is an anti-angiogenic therapy approved for the treatment of various cancers, including the second-line treatment of advanced gastric cancer as a single agent or in combination with paclitaxel. The FIDES-03 study will assess the efficacy and safety of derazantinib as monotherapy and combination therapy with ramucirumab and paclitaxel or with Roches PD-L1 checkpoint inhibitor atezolizumab.

Dr. Marc Engelhardt, Chief Medical Officer, said: The agreement with Lilly allows us to investigate the potential of derazantinib combined with ramucirumab and paclitaxel, which is the standard of care in the second-line treatment of gastric cancer. Derazantinib inhibits FGFR1-3 kinases but also inhibits CSF1R and VEGFR2. Its unique kinase inhibition profile may complement the anti-angiogenic effects of ramucirumab in the treatment of patients with advanced gastric cancer. Exploring combination therapies in order to strengthen the clinical evidence on the differentiation of derazantinib versus other FGFR inhibitors is one important element of our development strategy for the compound.

Gastric cancer is the fifth most common cancer worldwide and the third most lethal cancer type.2 Median survival rarely exceeds twelve months and the five-year survival is less than 10%.3 Basilea estimates that there are approximately 190,000 new cases of gastric cancer per year in total across the top 5 EU countries, Japan and the U.S. FGFR genetic aberrations have been observed in about 10% of gastric cancers.4

About derazantinib

Derazantinib is an investigational orally administered small-molecule FGFR inhibitor with strong activity against FGFR1, 2, and 3.5 FGFR kinases are key drivers of cell proliferation, differentiation and migration. FGFR genetic aberrations, e.g. gene fusions, mutations or amplifications, have been identified as potentially important therapeutic targets for various cancers, including intrahepatic cholangiocarcinoma (iCCA), urothelial, breast, gastric and lung cancers.6 In these cancers, FGFR genetic aberrations are found in a range of 5% to 30%.7Derazantinib also inhibits the colony-stimulating-factor-1-receptor kinase (CSF1R).5, 8 CSF1R-mediated signaling is important for the maintenance of tumor-promoting macrophages and therefore has been identified as a potential target for anti-cancer drugs.9 Pre-clinical data has shown that tumor macrophage depletion through CSF1R blockade renders tumors more responsive to T-cell checkpoint immunotherapy, including approaches targeting PD-L1/PD-1.10,11Derazantinib has demonstrated antitumor activity and a manageable safety profile in a previous biomarker-driven phase 1/2 study in iCCA patients,12 and has received U.S. and EU orphan drug designation for iCCA. Basilea is currently conducting three clinical studies with derazantinib. The first study, FIDES-01, is a registrational phase 2 study in patients with inoperable or advanced iCCA. It comprises one cohort of patients with FGFR2 gene fusions and another cohort of patients with mutations or amplifications.13 The second study, FIDES-02, is a phase 1/2 study evaluating derazantinib alone and in combination with Roche's PD-L1-blocking immune-checkpoint inhibitor, atezolizumab, in patients with advanced urothelial cancer, including metastatic, or recurrent surgically unresectable disease, expressing FGFR genetic aberrations.14 The third study, FIDES-03, is a phase 1/2 study evaluating derazantinib alone and in combination with Lillys anti-VEGFR2 antibody ramucirumab and paclitaxel or with Roches PD-L1 checkpoint inhibitor atezolizumab in patients with advanced gastric cancer with FGFR genetic aberrations.15 Basilea in-licensed derazantinib from ArQule Inc., a wholly-owned subsidiary of Merck & Co., Inc., Kenilworth, N.J., U.S.A.

About Basilea

Basilea Pharmaceutica Ltd. is a commercial-stage biopharmaceutical company, focused on the development of products that address the medical challenges in the therapeutic areas of oncology and infectious diseases. With two commercialized drugs, the company is committed to discovering, developing and commercializing innovative pharmaceutical products to meet the medical needs of patients with serious and life-threatening conditions. Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland and listed on the SIX Swiss Exchange (SIX: BSLN). Additional information can be found at Basilea's website http://www.basilea.com.


This communication expressly or implicitly contains certain forward-looking statements, such as "believe", "assume", "expect", "forecast", "project", "may", "could", "might", "will" or similar expressions concerning Basilea Pharmaceutica Ltd. and its business, including with respect to the progress, timing and completion of research, development and clinical studies for product candidates. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Basilea Pharmaceutica Ltd. to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Basilea Pharmaceutica Ltd. is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise. Derazantinib and its uses are investigational and have not been approved by a regulatory authority for any use. Efficacy and safety have not been established. The information presented should not be construed as a recommendation for use. The relevance of findings in nonclinical/preclinical studies to humans is currently being evaluated

For further information, please contact:

This press release can be downloaded from http://www.basilea.com.


1. CYRAMZA is a registered trademark owned by or licensed to Eli Lilly and Company its subsidiaries, or affiliates.2. F.M Johnston, M.Beckman. Updates on management of gastric cancer. Current Oncology Reports 2019 (21), 673. M.Orditura, G.Galizia, V.Sforza et al. Treatment of gastric cancer, World Journal of Gastroenterology 2014 (20), 1635-16494. A.Bass, V.Thorsson, I.Shmulevich et al. Comprehensive molecular characterization of gastric adenocarcinoma. Nature 2014 (513), 202-2095. T.G.Hall, Y.Yu, S.Eathiraj et al. Preclinical activity of ARQ 087, a novel inhibitor targeting FGFR dysregulation. PLoS ONE 2016, 11 (9), e01625946. R.Porta, R.Borea, A.Coelho et al. FGFR a promising druggable target in cancer: Molecular biology and new drugs. Critical Reviews in Oncology/Hematology 2017 (113), 256-2677. T.Helsten, S.Elkin, E.Arthur et al. The FGFR landscape in cancer: Analysis of 4,853 tumors by next-generation sequencing. Clinical Cancer Research 2016 (22), 259-2678. P.McSheehy, F.Bachmann, N.Forster-Gross et al. Derazantinib (DZB): A dual FGFR/CSF1R-inhibitor active in PDX-models of urothelial cancer. Molecular Cancer Therapeutics 2019 (18), 12 supplement, pp. LB-C129. M.A.Cannarile, M.Weisser, W.Jacob et al. Colony-stimulating factor 1 receptor (CSF1R) inhibitors in cancer therapy. Journal for ImmunoTherapy of Cancer 2017, 5:5310. Y.Zhu, B.L.Knolhoff, M.A.Meyer et al. CSF1/CSF1R Blockade reprograms tumor-infiltrating macrophages and improves response to T cell checkpoint immunotherapy in pancreatic cancer models. Cancer Research 2014 (74), 5057-506911. E.Peranzoni, J.Lemoine, L.Vimeux et al. Macrophages impede CD8 T cells from reaching tumor cells and limit the efficacy of antiPD-1 treatment. Proceedings of the National Academy of Science of the United States of America 2018 (115), E4041-E405012. V.Mazzaferro, B.F.El-Rayes, M.Droz dit Busset et al. Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. British Journal of Cancer 2019 (120), 165-171. ClinicalTrials.gov identifier: NCT0175292013. FIDES-01: ClinicalTrials.gov identifier: NCT0323031814. FIDES-02: ClinicalTrials.gov identifier: NCT0404561315. FIDES-03: ClinicalTrials.gov identifier: NCT04604132


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Basilea announces Clinical Trial Collaboration and Supply Agreement with Eli Lilly and Company for ramucirumab in the ongoing FIDES-03 study with...

CHNC Aims to Be the Global Leader in Cannabis Clinical Trials Through a Merger with Pharmacology University – Yahoo Finance

Posted: at 11:54 am

Houston, Texas--(Newsfile Corp. - October 28, 2020) - CHNC (OTC Pink: CHNC) announces its merger with Pharmacology University, Inc., a global leader in the field of medical cannabis education that markets its services under the brand Pharmacology University. More to the point, CHNC joining forces with Pharmacology University Inc. and Precision Research Institute has created one of the most complete companies in the cannabis industry. COO Elizabeth Hernandez explains the reasons behind the decision: "While pursuing my dream, I had wanted to unite forces with a company in the Cannabis Industry and that is when I found Pharmacology University. The synergy between the two companies has been superb and we are positioning ourselves to become the high-end authority of the Cannabis Research Industry. Now this journey finally feels complete. Riding this new wave of inspiration, we are deeply committed to produce revenue generating models and building shareholder value."

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The merger of CHNC, which is publicly traded on OTC markets under the ticker symbol CHNC, provides the framework for Pharmacology University to expand its focus into cannabis clinical trials and bolstering its education offerings.

Founded in 2010, Pharmacology University offers educational products and consulting services in the United States, Puerto Rico, Latin and South America; to train doctors, dispensary owners, growers, lawyers, and other professionals on the palliative and myriad health benefits of cannabis. The company also has partnered with private accredited universities to offer an intensive master's certification program in cannabis science and is now the top international provider of medical cannabis education.

In addition to its classroom education, Pharmacology University owns and operates Canna Law Magazine, which is a digital informational piece that provides cultural enrichment to the cannabis entrepreneur. The magazine has biweekly editions and informs the public about the most recent legal cases in the cannabis industry worldwide, also providing strategies by which its readers can avoid finding themselves in legal situations for lack of knowledge. Canna Law Magazine is currently available in English, Spanish, Portuguese, Italian and Arabic, and it is being developed to be published in Chinese and Hindi. To view the magazine, visit http://www.cannalawmagazine.com.

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"We want to be pioneers in the search for the truth with actual clinical trials, in regards to how cannabis can be utilized," says Pharmacology University In-House Legal Counsel, Anne Graham. "Predominantly because the number one problem for people that are set against using cannabis as medicine is simply due to the fact that the cannabis industry has not produced sufficient medical studies," she says.

The global market for medical cannabis (also known as medical marijuana), is expected to reach more than $150 billion annually by 2027, according to a 2019 report by ResearchAndMarkets.com. The anticipated growth is driven in large part by the legalization of cannabis for medical use in Europe, as well as the passage in the U.S. of the Hemp Farming Act of 2018, which legalized hemp, a variety of the Cannabis Sativa species that has less than 0.3% concentration of tetrahydrocannabinol (THC). And although many companies are looking to capitalize on the industry's projected growth by opening dispensaries, cannabis farms, or manufacturing facilities, Pharmacology University sees limitless opportunities in the education space.

Says Dr. Jose Torres, International Medical Director for Pharmacology University, "The endocannabinoid system is represented in every organ and system in the body. That means that at times, we could have deficiencies, just like now and again, our bodies present insufficiencies of minerals and vitamins; accordingly, the human body could also lack endocannabinoids." Preliminary research indicates that the scarcity of endocannabinoids may be the root of various illnesses, such as epilepsy and even certain types of cancer, he adds. By becoming a pioneer in clinical trials to explore more than 120 cannabis molecules, the newly formed public company aims to equip healthcare professionals and others in the industry with the information and knowledge to help millions of people through the use of cannabis in medical treatments.

"We had a vision when we started ten years ago, and 2021 will be our dream becoming a reality," says Katerin Osuna, International Director of Marketing and Public Relations of Pharmacology University, Magister in Audiovisual Communications and Advertising Content, from the Autnoma University of Barcelona, Spain. "We look forward to being the world leader in cannabis clinical studies."

About Pharmacology University

Pharmacology University, which markets its educational products under the brand Pharmacology University, aims to eliminate outmoded taboos and political obstacles surrounding medical cannabis to help alleviate the physical pain and suffering; whereby improving the quality of life for millions of people worldwide. The company is determined to vastly increase the number of patients who will benefit from cannabis medication through clinical trials, education, and lobbying efforts that appeal to the compassion of legislators in the United States and around the world. For more information please visit http://www.pharmacologyuniversity.com and view us here https://www.youtube.com/watch?v=yThdpf-WluI.

Safe Harbor Statement

The information posted in this release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. You can identify these statements by use of the words "may," "will," "should," "plans," "explores," "expects," "anticipates," "continue," "estimate," "projects," "intends," and similar phrases. Forward-looking statements involve risks and uncertainties that could cause actual results to differ from those projected or anticipated. These risks and uncertainties include, but are not limited to, general economic and business conditions, effects of geopolitical conditions, competition, changes in technology and methods of marketing, and various other factors beyond the company's control.

Media Contact:

Henry Levinski(817) 528-2475info@pharmacologyuniversity.com

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/66985

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CHNC Aims to Be the Global Leader in Cannabis Clinical Trials Through a Merger with Pharmacology University - Yahoo Finance

Regulus Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant…

Posted: October 16, 2020 at 7:52 pm

DetailsCategory: DNA RNA and CellsPublished on Friday, 16 October 2020 14:11Hits: 183

LA JOLLA, CA, USA I October 15, 2020 I Regulus Therapeutics Inc.(Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced initiation of dosing in its Phase 1b clinical study of RGLS4326 in patients with ADPKD.

The Phase 1b is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD and will evaluate administration of RGLS4326 for safety, pharmacokinetics, and changes in levels of polycystin 1 (PC1) and polycystin 2 (PC2). Patients with ADPKD, due to a mutation in the PKD genes, have been reported to have low levels of PC1 and PC2, the proteins encoded by the PKD1 and PKD2 genes, respectively. This study is designed to assess whether different dose levels of RGLS4326 can increase levels of PC1 and PC2 in ADPKD patients. The first cohort is expected to enroll up to nine patients who will receive RGLS4326 every two weeks over a six week period. The Company anticipates availability of results from the first cohort by the end of Q1 2021.

The Company plans to use the data from this first cohort of patients with ADPKD, together with the data from the multiple ascending dose and the single ascending dose studies in healthy volunteers as well as the recently completed nonclinical studies, to obtain feedback from the U.S. Food & Drug Administration ("FDA") on the acceptability of the Company's approach to addressing the second set of FDA requirements to support studies of extended duration in patients.

"We are excited to evaluate this potentially disease modifying investigational drug in patients with ADPKD," saidJay Hagan, CEO of Regulus. "This Phase 1b study will provide important data on safety, pharmacokinetics, and biomarkers of ADPKD which we plan to use in our approach to address the remaining partial clinical hold requirements."

For more information about the clinical trial design, please visit http://www.clinicaltrials.gov (NCT04536688).


ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and designed to preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD.The RGLS4326 IND is currently on a Partial Clinical Hold for treatment of extended duration by FDAuntil the second set of requirements outlined by the agency have been satisfactorily addressed. Information from the Phase 1 clinical studies, together with information from the recently completed additional nonclinical studies, will be used to address the second set of requirements to support studies of extended duration. RGLS4326 has received orphan drug designation from FDA in July 2020.

About Regulus

Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs.Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field.Regulus maintains its corporate headquarters in La Jolla, CA.

SOURCE: Regulus Therapeutics

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Regulus Therapeutics Announces First Patient Dosed in Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients with Autosomal Dominant...

Rocket Pharmaceuticals Presents Positive LAD-l Clinical Update and Comprehensive IMO Preclinical Review at the European Society for Immunodeficiencies…

Posted: at 7:52 pm

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data at the European Society for Immunodeficiencies (ESID) 2020 Meeting being held virtually October 14-17, 2020. An oral presentation provides positive longer-term follow-up data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster highlights preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO) supporting clinical development of the trial.

Today, Rocket presents positive results from our LAD-I gene therapy trial demonstrating further clinical benefit in this severely affected patient population, said Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket. Patients with LAD-I have markedly diminished expression of the integrin CD18 and suffer from life-threatening bacterial and fungal infections. Natural history studies indicate that an increase in CD18 expression to 4-10% is associated with survival into adulthood. The two patients enrolled in our Phase 1 trial demonstrated restored CD18 expression substantially exceeding this threshold. In addition, we continue to observe a durable treatment effect in the patient followed through one year, with improvement of multiple disease-related skin lesions after therapy and no further requirements for prophylactic anti-infectives.

Dr. Schwartz continued, In addition, preclinical results in Infantile Malignant Osteopetrosis represent an early positive signal of in vivo efficacy that support evaluation of RP-L401 in a Phase 1 trial. IMO is a devastating bone resorption disorder resulting in skeletal deformities, neurologic abnormalities and bone marrow failure. Rocket has developed RP-L401 as a potential treatment option to prevent the devastating morbidity and childhood mortality associated with IMO. Preclinical data indicate that even a modest level of engraftment can correct the disease phenotype, with increased long-term survival, growth, and normalized bone and tooth development.

Preliminary Data Highlights from Rockets Phase 1/2 Study of RP-L201 in LAD-I

The data presented in the oral presentation are from two pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. Both patients were treated with RP-L201, Rockets ex vivo lentiviral gene therapy candidate. Patient L201-003-1001 was 9-years of age at treatment and has been followed for 12-months and Patient L201-003-1004 was 3-years of age at treatment and has been followed for four months. Key highlights from the presentation include:

A copy of the oral presentation and e-poster can be accessed by visiting: https://www.rocketpharma.com/ESID/

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Presents Positive LAD-l Clinical Update and Comprehensive IMO Preclinical Review at the European Society for Immunodeficiencies...

New NIH clinical trial run by the U of M School of Public Health tests a combination of drugs to treat COVID-19 – UMN News

Posted: October 15, 2020 at 6:01 pm

A new National Institutes of Health (NIH) clinical trial, called ITAC, is being conducted by the University of Minnesota School of Public Health. The trial will test the safety, tolerability and efficacy of a highly concentrated solution of antibodies called hIVIG (hyperimmune intravenous immunoglobulin) that neutralizes SARS-CoV-2, the virus that causes COVID-19, when given with remdesivir compared to remdesivir alone. Remdesivir is a broad-spectrum antiviral that is currently recommended for treating certain hospitalized patients with COVID-19. An earlier trial showed that those who received remdesivir had a statistically significant shorter time to recovery compared to patients who received a placebo.

The solution being tested contains antibodies from plasma (the liquid portion of blood) donated by healthy people who have recovered from COVID-19. The antibodies in the solution are highly purified and concentrated so that it contains several times more neutralizing antibodies than typically found in the plasma of people who have recovered from the illness.

Study investigators hypothesize that giving people anti-coronavirus hIVIG before the body makes a protective immune response on its own could augment the natural antibody response to the virus, thereby reducing the risk of progression of COVID-19.

ITAC enrolled its first patient on October 8 and will eventually enroll 500 hospitalized adults age18 or older who provide informed consent, have had COVID-19 symptoms for 12 days or fewer, and do not have life-threatening organ dysfunction or organ failure. Enrollment will occur in 50 to 75 sites in Africa, Asia, Europe, North America and South America. In Minnesota, Hennepin Healthcare and the Minneapolis VA Health Care System will be part of the trial.

Study participants will be assigned at random to receive infusions of either anti-coronavirus hIVIG and remdesivir or a placebo and remdesivir. Neither the participants nor the study team will know who is receiving which treatment regimen. The ITAC study participants will be followed for 28 days.

An independent data and safety monitoring board (DSMB) will review interim safety and efficacy data to ensure patient well-being and safety as well as study integrity.

The trial is being conducted by the NIAID-funded International Network for Strategic Initiatives in Global HIV Trials (INSIGHT) at the University of Minnesota School of Public Health (SPH). NIAID is the National Institute of Allergy and Infectious Diseases, which is part of NIH. While INSIGHT was established to conduct large, global clinical studies on HIV, ithas been involved in clinical trials of treatments for other infectious diseases since 2009.

ITAC is a global collaboration involving five INSIGHT international coordinating centers in Bethesda, Copenhagen, London, Sydney, and Washington D.C., and four manufacturers of hIVIG: CSL Behring, Emergent BioSolutions, Grifols Therapeutics, Inc., and Takeda Pharmaceuticals, said Jim Neaton, leader of INSIGHT and a professor in SPH. The protocol will provide evidence on the efficacy of anti-coronavirus hIVIG and potentially identify a treatment to slow the progression of COVID-19.

The trial is also known as INSIGHT 013, and the protocol chair is Mark Polizzotto, head of the Therapeutic and Vaccine Research Program at The Kirby Institute at the University of New South Wales, in Sydney, Australia.The INSIGHT leadership group and statistical and data management center is housed at the Coordinating Centers for Biometric Research (CCBR) in the School of Public Health Division of Biostatistics. CCBR is also conducting several other COVID-19 studies for NIAID. Over the last 30 years, CCBR, through INSIGHT and other NIAID-funded work, has investigated treatments for HIV and influenza, and vaccines and treatments for Ebola, changing the way these diseases are managed across the globe.


New NIH clinical trial run by the U of M School of Public Health tests a combination of drugs to treat COVID-19 - UMN News

Zhittya Genesis Medicine Announces It Has Been Approved to Initiate Clinical Trials in Mexico for the Treatment of Amyotrophic Lateral Sclerosis (ALS)…

Posted: at 6:01 pm

LAS VEGAS, Oct. 15, 2020 (GLOBE NEWSWIRE) -- Zhittya Genesis Medicine, Inc. (Zhittya), a private company, announces that it will initiate clinical trials in Mexico to test a medical hypothesis that has been advanced over the last five years that ALS may be caused by vascular disruption in the areas of the brain which house motor neurons, those neurons which become dysfunctional in patients suffering from ALS. The hypothesis is simply that the micro-vascularization in that area of the brain is blocked or narrowed, restricting the flow of blood needed to nourish the motor neurons. Just as with heart disease, where blockage of coronary arteries can lead to angina and heart attacks, that same process is now thought to underlie the development of ALS.

Dr. Jack Jacobs, Zhittyas President and Chief Science Officer will be giving a free Zoom webinar on this topic entitled: Amyotrophic Lateral Sclerosis (ALS): Is Therapeutic Angiogenesis a Potential Treatment to Reverse this Disease?This webinar will broadcast on Thursday, October 29, 2020 at 9:00 am Pacific time, 12 noon Eastern time.

Click Here to Register for this Webinar

Zhittya is developing a biological drug which in previous US FDA-authorized clinical trials has demonstrated it can trigger therapeutic angiogenesis or the growth of new blood vessels in ischemic tissues. Zhittya has prepared a White Paper entitled: Human FGF-1 as a Potential Treatment for Amyotrophic Lateral Sclerosis (ALS), which is available to all, free of charge, by emailing: dan@zhittyamedicine.com

Daniel C. Montano, CEO of Zhittya stated, I believe we are truly on to something here. Over the last three years, we have continually uncovered data which enhances our belief that therapeutic angiogenesis might be a viable breakthrough treatment for patients that suffer from ALS. If, as we believe, ALS is initiated by micro-vascular disruption in the brain, we hope our molecule can do in the brain, what it has already demonstrated it can do in the US FDA-cleared heart trial, namely, grow new blood vessels.

Dr. Jack Jacobs added, ALS is a progressive and fatal neuromuscular disease and the majority of ALS patients die within 25 years of receiving a diagnosis. There is no known definitive cause of ALS and hereditary forms of the disease only account for 5%10% of cases. No cure has been identified and the lack of proven and effective therapies for ALS is an ongoing challenge. There are now multiple lines of evidence that have established that angiogenesis is deficient in this disease, leading directly to the death of motor neurons. I believe therapeutic angiogenesis may be a potentially novel way to halt the progression of ALS and we are pleased that the Mexican regulatory authorities have given us permission to test our biological drug candidate in subjects with ALS.

About Zhittya Genesis Medicine

Zhittyas management has been working to advance these medicines for over 21 years and many tens of millions of dollars have been expended in preclinical and clinical studies. Zhittyas medicine initiates a biological process in the human body referred to as therapeutic angiogenesis and this process will only occur in diseased tissues that become ischemic due to a lack of blood flow. In those areas with insufficient blood flow, the drug stimulates the growth of new blood vessels, providing nourishment and removing metabolic waste products, thereby re-establishing normal cellular functions. Heart disease, stroke, peripheral artery disease (PAD) and diabetic foot ulcers are just some of the more obvious disorders the drugs can treat, but in fact, over 75 human diseases, including neurodegenerative diseases such as ALS and Parkinsons disease, are thought to be caused by lack of blood flow to a specific area of the brain.

Contact information:Daniel C. Montano, CEOZhittya Genesis Medicine, Inc.Phone: (1) 702-790-9980 email: dan@zhittyamedicine.comwebsite:zgm.care

Zhittya Genesis Medicine Announces It Has Been Approved to Initiate Clinical Trials in Mexico for the Treatment of Amyotrophic Lateral Sclerosis (ALS)...

FDA Authorizes Phase 1 Trial of ImmunityBio’s Novel COVID-19 Vaccine Candidate hAd5; Dual Construct is Designed to Drive Both T Cell and Antibody…

Posted: at 6:01 pm

Oct. 15, 2020 10:00 UTC

CULVER CITY, Calif.--(BUSINESS WIRE)-- ImmunityBio, Inc., a privately-held immunotherapy company, today announced it has received authorization from the U.S. Food and Drug Administration (FDA) to begin a Phase I clinical trial of hAd5-COVID-19, the companys novel COVID-19 vaccine candidate that targets both the inner nucleocapsid (N), engineered to activate T cells, and outer spike (S) protein, engineered to activate antibodies against the coronavirus (SARS-CoV-2). These dual constructs (bivalent sequences) of SARS-CoV-2 offer the potential for the hAd5 vaccine to provide recipients with durable, long-term cell-mediated immunity with potent antibody stimulation against both the S and N proteins.

The company anticipates launching a Phase I trial at Irvine, Calif. based Hoag Hospital this month with adult subjects up to age 55.

While there are a number of vaccine candidates in development, we believe most are limited by their sole focus on antibody responses to the monovalent spike protein, which may be insufficient to activate the full potential of the immune system to fight the coronavirus, said Dr. Patrick Soon-Shiong, Chairman and CEO of ImmunityBio. By targeting the nucleocapsid protein on the interior of the virus particle as well as the spike protein on the viruss surface, we believe this vaccine can stimulate both T-cell-mediated and antibody-mediated immunity to SARS-CoV-2.

hAd5 Vaccine Seeks to Develop Immune Memory The hAd5-COVID-19 vaccine candidate is a novel, engineered, second-generation human adenovirus serotype 5 vaccine. Because it delivers both the S and N proteins, the vaccine has the potential to enable recipients to develop immune memory, promoting long-lasting immunity to SARS-CoV-2. The N protein has been engineered with a novel signaling domain to enhance T cell activation. The hAd5 vaccine with this Enhanced T cell Signaling Domain (ETSD) has generated potent CD4+ and CD8+ COVID specific T cell activation recognizing S and N following hAd5 vaccination in pre-clinical studies.

ImmunityBios approach differs from first-generation adenoviral platforms, including one based on the chimpanzee adenovirus and the first-generation human adenovirus vaccines being tested in Europe, China, Russia and the United States. All first-generation COVID-19 vaccines in late-stage clinical trials deliver only the monovalent spike protein on the surface of the virus and therefore focus primarily on antibody protection as their primary endpoints.

Based on our studies in cancer, we recognized that potent T-cell activation can kill the affected cells and requires multiple antigen targets to be maximally activated, Soon-Shiong said. Our preclinical studies showed that the N protein was highly immunogenic and generated potent CD4+ and CD8+ T cells. Furthermore, studies have shown that patients with SARS-CoV infection have long-term T cell memory to the N protein.

On the basis of these findings, continued Soon-Shiong, we are targeting both the N and S proteins in order to drive T cell response, with the goal of generating B and T cell memory to the COVID-19 antigens, and long-term immunity to the virus.

hAd5 Vaccine Circumvents Pre-Existing Adenoviral Immunity Current first-generation adenovirus platforms suffer from the disadvantage of generating neutralizing antibodies which occur after repeated administration, resulting in the attack of the adenovirus vector itself and reduce the effectiveness of the vaccine. ImmunityBios hAd5 second generation adenovirus platform has four deletions which overcome pre-existing adenoviral immunity allowing for multiple administrations. This ability to circumvent pre-existing adenoviral immunity has been demonstrated in multiple Phase I / II clinical trials of hAd5 showing tumor specific CD4+ and CD8+ T cells following repeated administrations in the presence of pre-existing adenovirus immunity.i,ii,iii,iv

ImmunityBio plans to administer the hAd5-COVID-19 vaccine both as a prime and boost to sustain protection against SARS-CoV-2 since multiple re-immunizations using the same vector platform are now possible with the novel hAd5 platform. The company is also pursuing preclinical development for oral, inhalational and intranasal administration to provide mucosal immunity in addition to durable humoral and cell-mediated immunity against COVID-19.

Given the need for global distribution in both developing and developed countries, the enormous challenge of cold-chain requirements could be mitigated by a room temperature stable formulation of hAd5 that can be administered without the need for needles, Soon-Shiong said.

COVID-19 Vaccine Program Status:

ImmunityBio and NantKwest Joint Collaboration Agreement Under the terms of a definitive agreement announced on August 24, 2020, ImmunityBio, Inc. and its affiliate NantKwest, Inc. agreed to share equally the costs of development, manufacturing, marketing and commercialization of the products each is developing related to COVID-19, including the hAd5 vaccine candidate. Should a product be commercialized successfully, the companies have agreed to a 60-40 percentage split of net profits, with the larger share going to the company that developed the product. The agreement also details the structure of shared governance of the joint collaboration.

In addition to the vaccine candidate, the agreement currently covers a mesenchymal stem cell therapeutic from NantKwest, whose goal is to reduce the time a critically ill patient spends on a ventilator.

By working together and combining the novel technologies of the two companies, as well as their resources, we believe we have been able to more rapidly develop products that will address the needs of COVID-19 patients, said Soon-Shiong, who also serves as Chairman and CEO of NantKwest. We also will be better able to contribute to the global fight being waged against this deadly pandemic.

About ImmunityBio ImmunityBio, Inc. is a late-clinical-stage immunotherapy company developing next-generation therapies that drive immunogenic mechanisms for defeating cancers and infectious diseases. The companys immunotherapy platform activates both the innate (natural killer cell and macrophage) and adaptive (T cell) immune systems to create long-term immunological memory. This novel approach is designed to eliminate the need for high-dose chemotherapy, improve upon the outcomes of current CAR T-cell therapies, and extend beyond checkpoint inhibitors.

ImmunityBios platform is based on the foundation of three separate modalities: antibody cytokine fusion proteins, synthetic immunomodulators, and second-generation human adenovirus (hAd5) vaccine technologies.

Anktiva (ImmunityBios lead cytokine infusion protein) is a novel interleukin-15 (IL-15) superagonist complex and has received Breakthrough Therapy and Fast Track Designations from the U.S. Food and Drug Administration (FDA) for BCG-unresponsive CIS non-muscle invasive bladder cancer (NMIBC). The company is also in Phase 2 or 3 trials for indications such as first- and second-line lung cancer, triple-negative breast cancer, metastatic pancreatic cancer, recurrent glioblastoma, and soft tissue sarcoma in combination with the companys synthetic immune modulator (aldoxorubicin).

ImmunityBio is also developing therapies, including vaccines, for the prevention and treatment of HIV, influenza, and the coronavirus SARS-CoV-2 with its second-generation human adenovirus (hAd5) vaccine technologies.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning or implying that ImmunityBio will be successful in improving the treatment of various diseases, including, but not limited to the novel coronavirus and cancer. Risks and uncertainties related to this endeavor include, but are not limited to, the companys beliefs regarding the success, cost, and timing of its development activities and clinical trials.

Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

About NantKwest NantKwest (NASDAQ: NK) is an innovative, clinical-stage, immunotherapy company focused on harnessing the power of the innate immune system to treat cancer and infectious diseases. NantKwest is the leading producer of clinical dose forms of off-the-shelf natural killer (NK) cell therapies. The activated NK cell platform is designed to destroy cancer and virally-infected cells. The safety of these optimized, activated NK cellsas well as their activity against a broad range of cancershas been tested in phase I clinical trials in Canada and Europe, as well as in multiple phase I and II clinical trials in the United States. By leveraging an integrated and extensive genomics and transcriptomics discovery and development engine, together with a pipeline of multiple, clinical-stage, immuno-oncology programs, NantKwests goal is to transform medicine by bringing novel NK cell-based therapies to routine clinical care. NantKwest is a member of the NantWorks ecosystem of companies. For more information, please visit http://www.nantkwest.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning or implying that NantKwest will be successful in improving the treatment of cancer or other critical illnesses, including COVID-19. Risks and uncertainties related to these endeavors include, but are not limited to, obtaining FDA approval of NantKwests NK cells and MSC as well as other therapeutics and manufacturing challenges.

Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements.

These and other risks regarding NantKwests business are described in detail in its Securities and Exchange Commission filings, including in NantKwests Quarterly Report on Form 10-Q for the quarter ended June 30, 2020. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

iGattiMays, et al. A Phase I Trial Using a Multitargeted Recombinant Adenovirus 5 (CEA/MUC1/Brachyury)Based Immunotherapy Vaccine Regimen in Patients with Advanced Cancer. The Oncol, 25: 479-e899. doi:10.1634/theoncologist.2019-0608

iiGabitzsch ES, et al. Anti-tumor immunotherapy despite immunity to adenovirus using a novel adenoviral vector Ad5 [E1-, E2b-]-CEA. Cancer Immunol Immunother. 2010 Jul;59(7):1131-5. doi: 10.1007/s00262-010-0847-8. Epub 2010 Apr 2. PMID: 20361185.

iiiMorse MA, et al. Novel adenoviral vector induces T-cell responses despite anti-adenoviral neutralizing antibodies in colorectal cancer patients. Cancer Immunol Immunother. 2013 Aug;62(8):1293-301. doi: 10.1007/s00262-013-1400-3. Epub 2013 Apr 30. PMID: 23624851; PMCID: PMC3732790.

ivOsada T, et al. Optimization of vaccine responses with an E1, E2b and E3-deleted Ad5 vector circumvents pre-existing anti-vector immunity. Cancer Gene Ther. 2009 Sep;16(9):673-82. doi: 10.1038/cgt.2009.17. Epub 2009 Feb 20. PMID: 19229288; PMCID: PMC3800002.

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FDA Authorizes Phase 1 Trial of ImmunityBio's Novel COVID-19 Vaccine Candidate hAd5; Dual Construct is Designed to Drive Both T Cell and Antibody...

Fate Therapeutics Announces Presentations at the 2020 Society for Immunotherapy of Cancer Annual Meeting – GlobeNewswire

Posted: at 6:01 pm

SAN DIEGO, Oct. 15, 2020 (GLOBE NEWSWIRE) -- FateTherapeutics, Inc. (NASDAQ:FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, today announced that five abstracts for the Companys induced pluripotent stem cell (iPSC) product platform were accepted for presentation at the Society for Immunotherapy of Cancer (SITC) annual meeting being held virtually from November 9-14, 2020.

Accepted abstracts include clinical data from 15 patients in the dose-escalation stage of the Companys Phase 1 clinical trial of FT500 in advanced solid tumors (NCT03841110), which includes nine patients in Regimen A (three once-weekly doses of FT500 for up to two 30-day cycles as monotherapy) and six patients in Regimen B (three once-weekly doses of FT500 for up to two 30-day cycles in combination with checkpoint inhibitor therapy). The Company is currently enrolling the dose-expansion stage of the Phase 1 clinical trial for patients with non-small cell lung cancer or classical Hodgkin lymphoma who are refractory to, or have relapsed on, checkpoint inhibitor therapy. Each patient in the dose-expansion stage is to receive three once-weekly doses of FT500 at 300 million cells per dose, each with IL-2 cytokine support, for up to two 30-day cycles in combination with the same checkpoint inhibitor on which the patient failed or relapsed.

Oral Presentation

Poster Presentations

All abstracts are scheduled to be available on the SITC website on November 9, 2020.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

About FT500FT500 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial for the treatment of advanced solid tumors (NCT03841110). The study is designed to assess the safety and tolerability of FT500 as a monotherapy and in combination with one of three FDA-approved immune checkpoint inhibitor (ICI) therapies nivolumab, pembrolizumab or atezolizumab in patients that have failed prior ICI therapy. Despite the clinical benefit conferred by approved ICI therapy against a variety of tumor types, these therapies are not curative and, in most cases, patients either fail to respond or their disease progresses on these agents. One common mechanism of resistance to ICI therapy is associated with loss-of-function mutations in genes critical for antigen presentation. A potential strategy to overcome resistance is through the administration of allogeneic NK cells, which have the inherent capability to recognize and directly kill tumor cells with these mutations.

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Companys immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the advancement of, plans related to, and the therapeutic potential of the Company's product candidates, the Companys clinical development strategy and plans for the clinical investigation of its product candidates, and the Companys preclinical research and development programs. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk of difficulties or delay in the initiation of any planned clinical studies, or in the enrollment or evaluation of subjects in any future clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that results observed in preclinical studies of the Companys product candidates may not be replicated in ongoing or future clinical trials or studies, and the risk that the Companys product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

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Fate Therapeutics Announces Presentations at the 2020 Society for Immunotherapy of Cancer Annual Meeting - GlobeNewswire

Use virtual visits to enhance the patient centricity of clinical trials – MedCity News

Posted: October 13, 2020 at 9:58 am

After a concerted effort by our industry over the last several years, sponsors, clinical research organizations (CROs) and clinical trial suppliers have added patient centricity to our vocabulary and study planning.

The shift to not just think about the patient but think as the patient is a critical effort to advance clinical research for and with patients. Patient-focused strategies such as protocol design insights, focus groups, satisfaction surveys, indication-specific content and study results sharing continue to evolve as potential standard approaches in research studies. These strategies enable more patient-centric approaches and are important but are not the only solutions required to be patient centric.

Participants travel to specific research locations (sometimes an hour or more away) at specific times (which often means taking time off of work) to conduct assessments that in many cases can be conducted remotely. In between study visits, it can be challenging for patients to provide clinically relevant information and quickly connect with their sites to complete study activities. 15% to 40% of patients enrolled in clinical studies drop out before they are completed, delaying study timelines and new product launches. These delays can cost $600,000 per day in lost revenue opportunities for a niche drug and as much as $8 million for a blockbuster drug, underscoring the importance of patient retention in clinical trials.

Fortunately, there is an additional way to improve engagement, retention and timelines that has been brought to the forefront as a result of the Covid-19 pandemic. Telehealth virtual visits have been accelerating as an option available for sponsors throughout the global clinical research industry. Rather than requiring participants to go to sites for every check-in or visit, many clinical trial leaders have turned to telehealth, not only to keep operations steady during challenging times but also to adopt and offer an additional patient-centric approach to studies.

Bringing the study visits to patientsThe pandemic gave a new sense of urgency to patient-centric solutions that extended the site into a patients home safely. In the midst of the rampant spread of Covid-19 and fears about contracting it, many patients were reluctant to travel to a centralized location and some patients had concerns with home visits. As unemployment numbers increased due to the shutdown, participants (and their loved ones) were also more reluctant to take time off work and travel outside of their community. Yet it was critical that these life-changing, and in some cases life-saving, clinical trials continue.

Advances in telehealth offered an ideal solution. Rather than bringing patients to the clinic, sponsors and CROs could bring the study visits to patients. This approach enabled visits to continue (albeit remotely) to capture as many of the protocol-required assessments as possible without in-person interaction. Throughout the pandemic, our study stakeholders have been creating new habits around virtual meetings, video teleconferencing and conducting activities outside of brick-and-mortar locations.

This does not infer that in-person visits should be eliminated but does establish that patients, sites and sponsors are adopting virtual visits as an option to provide flexible approaches for study activities. This hybrid omni-channel approach that includes a mix of on-site, telehealth, phone, home health and mobile app creates a new level of convenience, flexibility and simplicity for patients, encouraging them to remain with the clinical trial.

Telehealth creates patient-centric benefitsIncorporating the use of telehealth offers many patient-centric benefits that can be used to mitigate dropout rates and help bring clinical trials to close on-time and on-budget:

Here to stayAs we continue to manage through the pandemic and plan for studies post-pandemic, it is clear that telehealth as a patient-centric approach has the ability to become a standard option for studies. By enabling a more hybrid approach to clinical trials with the added flexibility of telehealth, studies can improve patient-centricity, which will ultimately improve recruitment, engagement, and retention.

Photo: ronnachaipark, Getty Images

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Use virtual visits to enhance the patient centricity of clinical trials - MedCity News

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