The National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH) has awarded a $3.3 million grant to ReveraGen Inc. to support the advancement of vamorolone towards FDA submission and commercialization.

The two-year Small Business Innovation Research (SBIR) / Small Business Technology Transfer (STTR) Commercialization Readiness Pilot Program award will provide additional funding towards the new drug application (NDA) preparation, enabling timely filing after the read-out of the fully-enrolled registration study anticipated in the second quarter of 2021.

The SBIR and STTR program is a government funded program aimed at supporting and stimulating small U.S. based businesses. Known as ‘America’s Seed Fund’, funding is provided by 11 federal agencies to increase U.S. private-sector commercialization of innovate technologies.

Read the Announcement from ReveraGen

ReveraGen Receives $3.3 Million NIH Commercialization Readiness Pilot Grant for NDA Preparations for Vamorolone in Duchenne Muscular Dystrophy

 — Funds will support NDA preparatory activities for anticipated filing in 2021 —

November 17, 2020, 08:30 AM Eastern Time

Rockville, Maryland—ReveraGen, Inc., a privately held company developing vamorolone (VBP15) as a potential safer alternative for corticosteroid treatment in Duchenne muscular dystrophy and other disorders, today announced an award of a $3.3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) at National Institutes of Health (NIH). The two-year SBIR/STTR Commercialization Readiness Pilot (CRP) Program award will provide additional funding towards the new drug application (NDA) preparation, enabling timely filing after the read-out of the fully-enrolled registration study (VBP15-004; NCT03439670) anticipated in Q2 2021.

The Commercialization Readiness Pilot (CRP) program is designed to aid companies with previously funded SBIR/STTR Phase II/IIB projects as they advance to commercialization. ReveraGen has held a NINDS SBIR Phase II grant that enabled completion of Phase 2a clinical trials that showed dose-responsive improvement of vamorolone-treated DMD participants at both 24-weeks and 18-months treatment. A subsequent NIH SBIR Phase IIB award has supported the ongoing pivotal trial in 121 DMD boys recruited at 30 sites in 11 countries.

“This generous support from NIH is enabling the execution of the global regulatory and global intellectual property strategy of vamorolone, which we are developing jointly with Santhera,” said Dr. Eric Hoffman, VP Research and principal investigator at ReveraGen on the award.

“Upfront of the CRP application, ReveraGen had carried out a formal gap analysis to identify pending items required for the upcoming NDA submission, and the CRP award will be beneficial in addressing these,” noted Dr. Jesse Damsker, VP Operations at ReveraGen.

“We congratulate our partner ReveraGen to this grant which not only provides support in advancing vamorolone towards FDA submission and commercialization but also stands for recognition of the drug’s promising potential,” added Dario Eklund, Chief Executive Officer of Santhera.

ReveraGen has open INDs for development of vamorolone in both Duchenne muscular dystrophy and Becker muscular dystrophy. Studies of vamorolone in animal models of multiple chronic inflammatory diseases (multiple sclerosis, asthma, inflammatory bowel disease, arthritis and others) have shown efficacy similar to corticosteroids, and an improved safety profile. Studies of vamorolone in DMD boys have shown loss of the stunting of growth seen with treatment with corticosteroid standard of care (deflazacort, prednisone), with less physician-reported safety concerns.

Structure/activity studies comparing the active metabolites of glucocorticoid receptor ligands (vamorolone, deflazacort, prednisone) have shown differential activity in binding co-activators and co-repressors, leading to vamorolone showing unique mechanisms of action. Specifically, vamorolone binding leads to less positive gene transcription activity associated with safety concerns, while retaining potent anti-inflammatory activity.

Recently, Santhera Pharmaceuticals has acquired a world-wide license to vamorolone in all indications. ReveraGen continues to manage the clinical programs and carry out NDA preparations.