ReveraGen BioPharma has been awarded a $3.3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) to support further development of vamorolone as a safer alternative to corticosteroids in people with Duchenne muscular dystrophy (DMD) and other disorders.
The two-year SBIR/STTR Commercialization Readiness Pilot (CRP) Program award provides additional funding toward a new drug application (NDA) seeking regulatory approval of vamorolone in the U.S. According to ReveraGen, the funding will enable timely filing after the release of top-line results from the ongoing VISION-DMD Phase 2b clinical trial (NCT03439670), expected in the second quarter of 2021.
“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,” Jesse Damsker, PhD, vice president of operations at ReveraGen, said in a press release.
Vamolorone (VBP15) is an anti-inflammatory steroid designed to slow DMD progression while causing fewer and less-severe side effects than currently available corticosteroids. ReveraGen is developing the therapy along with partner Santhera Pharmaceuticals, which holds a worldwide license for vamorolone.
“This generous support from NIH is enabling the execution of the global regulatory and global intellectual property strategy of vamorolone,” said Eric Hoffman, PhD, vice president of research at ReveraGen.
The CRP program is designed to help companies with previously funded SBIR/STTR projects move toward commercialization.
ReveraGen was awarded a NINDS SBIR Phase 2 grant that supported the completion of Phase 2a trials (NCT02760277 and NCT02760264) showing improved motor function with vamorolone over 18 months in boys with DMD. Such benefit also was seen in a two-year extension study (NCT03038399). Data from boys who completed 18 months of treatment showed that vamorolone led to clinically meaningful improvements in all motor outcomes analyzed — without the stunted growth seen in those treated with conventional corticosteroids in the CINRG Duchenne Natural History Study (NCT00468832).
A subsequent NIH SBIR award supported the international VISION-DMD trial to assess the effectiveness and safety of vamorolone, compared to prednisone or a placebo, in a group of boys with DMD, ages 4 to 7, who can walk on their own.
The effect of vamorolone, prednisone, or placebo on a patient’s muscle strength and motor function is the primary goal of the study, first assessed after nearly six months of treatment by the time-to-stand test and other measurements of motor function.
Positive results may support submitting an application to the U.S. Food and Drug Administration (FDA) requesting vamorolone’s approval by the end of next year.
The therapy was granted orphan drug status in the U.S. and Europe, and received fast track and rare pediatric disease designations by the FDA. Its development has been supported by international non-profits and patient organizations, as well as the NIH, the U.S. Department of Defense, and the European Commission’s Horizon 2020 program.
“We congratulate our partner ReveraGen [on] 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,” said Dario Eklund, CEO of Santhera.
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