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The outside of the FDA headquarters is seen in White Oak, Maryland. © Al Drago

FDA Expediting Accelerated Development of Novel Therapies for COVID-19

News Commentary

Published on April 28, 2020

The U.S. Federal government is working closely with companies and researchers to find treatments and vaccines to overcome the global health crisis. The FDA has been cutting red tape and has launched new programs that relax certain time-consuming regulations while still assuring the efficacy and safety of the treatments. The FDA recently issued an update describing its most recent programs. The update stated that as part of the administration’s: “… all-hands-on-deck approach across public, academic, and private sectors to combat the COVID-19 pandemic, the U.S. Food and Drug Administration stood up a new program to expedite the development of potentially safe and effective life-saving treatments. The program, known as the Coronavirus Treatment Acceleration Program (CTAP), is using every tool at the agency’s disposal to bring new therapies to sick patients as quickly as possible, while at the same time supporting research to further evaluate whether these medical countermeasures are safe and effective for treating patients infected with this novel virus.   Active healthcare stocks in news today include Citius Pharmaceuticals, Inc. (NASDAQ: CTXR), Mesoblast Limited (NASDAQ: MESO), Inovio Pharmaceuticals, Inc. (NASDAQ: INO), Gilead Sciences, Inc. (NASDAQ: GILD), Aytu BioScience, Inc. (NASDAQ: AYTU).

“The FDA is announcing a new, comprehensive public-private approach to bring coronavirus treatments to market as fast as possible,” said HHS Secretary Alex Azar. “As part of this new program, the FDA is cutting red tape, redeploying staff, and working day and night to review requests from companies, scientists, and doctors who are working toward therapies. We are grateful to the men and women of the FDA who have been working in concert with industry and other parts of HHS to support potential coronavirus treatments for weeks now. Each day, President Trump’s all-of-America approach is making progress and providing new hope in our fight against the coronavirus.”

There are a large number of companies and researchers developing and evaluating COVID-19 related therapies. Given the urgent nature of the pandemic, under the FDA’s accelerator program, staff from the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research are providing regulatory advice, guidance, and technical assistance as quickly as possible. As part of this work, the FDA is triaging requests from developers and scientists seeking to develop a new drug and biologic therapies, getting the relevant FDA staff in touch with them, and providing rapid, interactive input to get studies underway quickly.

Citius Pharmaceuticals, Inc. (NASDAQ: CTXR) BREAKING NEWSCitius Announces Pre-IND Submission to FDA Under the Coronavirus Treatment Acceleration Program for a Novel Stem Cell Therapy for Acute Respiratory Distress Syndrome (ARDS) in COVID-19  – Citius Pharmaceuticals (“Citius” or the “Company”), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, this week announced that it submitted a pre-IND meeting request and supporting briefing documents to the Center for Biologics Evaluation and Research (“CBER”) of the FDA under the Coronavirus Treatment Acceleration Program (CTAP) on April 24. The Company has requested the Division’s feedback to support the most expeditious pathway into the clinic to evaluate a novel cell therapy in patients suffering from COVID-19-related ARDS.

The cells, called NoveCite Cells or NC-MSCs, are made by Novellus, Inc. (“Novellus”), a Cambridge-based biotechnology company, using its patented mRNA-based cell-reprogramming process. NC-MSCs are mesenchymal stem cells derived from a single donor’s fibroblasts that have been dedifferentiated into an induced pluripotent stem cell (iPSC) master cell bank, thereby avoiding the need to source additional donor cells. The iPSCs are then further differentiated into a mesenchymal stem cell (MSC) therapy. Citius and Novellus plan to develop NC-MSCs for the treatment of ARDS, and last month the companies signed an exclusive option agreement.

The Company plans a multi-center randomized placebo-controlled dose-finding study followed by an expansion phase to assess the safety, tolerability, and efficacy of NC-MSCs in patients with moderate to severe ARDS due to COVID-19. The proposed trial, a Phase 1b/2 clinical trial, is titled “A Randomized Placebo-Controlled Dose-Finding Study Followed by a Dose Level Expansion to Assess the Safety and Efficacy of NoveCite MSCs in Subjects with Acute Respiratory Distress Syndrome (ARDS) Due to SARS-CoV-2 Disease (COVID-19),” or “MARCO”. The primary objectives of this study are to evaluate the safety and efficacy of NoveCite cells as a treatment for subjects with moderate-to-severe ARDS due to COVID-19 and to identify therapeutic doses.

“MSCs have an established track record of clinical safety, and have shown promise in the treatment of inflammatory lung disease,” said Matt Angel, Ph.D., co-founder and Chief Science Officer at Novellus, Inc. “Our research has shown that the NoveCite cells, being derived from mRNA-reprogrammed iPSCs, secrete higher levels of immunomodulatory proteins than donor-derived MSCs, and have unique manufacturing advantages.”

“We believe we have the key elements in place from a clinical design and manufacturing point of view to evaluate this novel cell therapy approach to deal with the current pandemic,” said Myron Holubiak, Chief Executive Officer of Citius. “ARDS is a very serious complication for many patients suffering from COVID-19 and is believed to account for about 80% of the deaths in ventilated patients. There is no proven or FDA-approved treatment for it, other than oxygen therapy, including the use of mechanical ventilation, and fluid management. Literature from previous investigational studies with MSCs in the treatment of lung injuries support the idea that MSCs could prove effective in treating COVID-19-related ARDS. We look forward to our FDA discussions and are excited to be at the cusp of what could be a novel and effective therapy for ARDS.” Read this full release here.

In other healthcare news of note:

Mesoblast Limited (NASDAQ: MESO) recently announced 83% survival in ventilator-dependent COVID-19 patients (10/12) with moderate/severe acute respiratory distress syndrome (ARDS) treated during the period March-April 2020 with two intravenous infusions of Mesoblast’s allogeneic mesenchymal stem cell product candidate remestemcel-L within the first five days. 75% (9/12) have successfully come off ventilator support at a median of 10 days. At this time, seven have been discharged from the hospital. Patients received a variety of experimental agents prior to remestemcel-L. All patients were treated under an emergency Investigational New Drug (IND) application or expanded access protocol at New York City’s Mt Sinai hospital.

In contrast, only 9% (38/445) of ventilator-dependent COVID-19 patients at a major referral hospital network in New York City were able to come off ventilator support when treated with standard of care during March/April 2020.

Inovio Pharmaceuticals, Inc. (NASDAQ: INO) announced this week that first-quarter 2020 financial results will be released after the market close on May 11, 2020. Following the release, the Company will host a live conference call and webcast at 4:30 p.m. ET to discuss financial results and provide a general business update, including the company’s ongoing vaccine developments for COVID-19.  A live and archived version of the audio presentation will be available online. This is a listen-only event but will include a live Q&A with analysts.  Telephone replay will be available approximately one hour after the call at 877-344-7529 (US toll-free) or 412-317-0088 (international toll) using replay access code 10143530.

Gilead Sciences, Inc. (NASDAQ: GILD) Kite, a Gilead Company, and oNKo-innate recently announced the companies have entered into a three-year cancer immunotherapy research collaboration to support discovery and development of next-generation drug and engineered cell therapies focused on natural killer (NK) cells.

Current cancer immunotherapy approaches primarily focus on T cell-mediated anti-tumor immunity, including checkpoint inhibition and chimeric antigen receptor (CAR) T cell therapy. Like T cells, NK cells are a class of lymphocytes (white blood cells) that play a critical surveillance and effector role in the immune system. NK cells and T cells each have the potential to attack cancer cells but have different mechanisms for tumor cell killing. Thus, appropriately activated and targeted NK cells may represent a differentiated approach that would be potentially complementary and synergistic with T cell-mediated anti-tumor strategies.

Aytu BioScience, Inc. (NASDAQ: AYTU) recently announced that it has signed an agreement with Sterling Medical Devices (“Sterling”) to finalize the development of Healight, a novel endotracheal catheter, as a potential treatment for coronavirus.

The company announced last week that it licensed exclusive worldwide rights to the Healight technology from Cedars-Sinai for all endotracheal and nasopharyngeal indications. The patent-pending Healight Platform has been in development since 2016 by the Medically Associated Science and Technology (MAST) team at Cedars-Sinai. Following their pre-clinical findings that Healight may be a safe and effective antiviral and antibacterial treatment, the team engaged Sterling to rapidly develop a novel endotracheal device to help combat coronavirus.

Associate Writer