-- Funds will support NDA preparatory activities for anticipated filing in 2021 --
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
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
“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.
“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
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,
About Vamorolone
Vamorolone is a first-in-class drug candidate that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative partial agonist. This mechanism has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore vamorolone could emerge as a promising alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is substantial unmet medical need in this patient group as high-dose corticosteroids have significant systemic side effects that diminish patient quality of life. The fully enrolled, pivotal Phase 2b VISION-DMD trial (VBP15-004, https://vision-dmd.info/2b-trialinformation ) is currently being conducted at study sites across
About ReveraGen BioPharma
ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including
Contact
ReveraGen BioPharma
Phone: + 1 240-672-0295
eric.hoffman@reveragen.com
Attachment
- ReveraGen Receives CRP-final
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