-- Funds will support NDA preparatory activities for anticipated filing 
in 2021 -- 
 
   November 17, 2020, 08:30 AM Eastern Time (press release provided in 
english only) 
 
   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; 
https://www.globenewswire.com/Tracker?data=k3b7oUGnX0CHwd9ercnCyHyrz_SnCx6S7oTRZzFgOe0yRurVaruxQSaIbWKLzssS4W-BB60AzKsppPgNqJ-eUz2W9cXSm0KCXsVstGt9e3kVOLViLlFntP-rExR9pKNg 
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 
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24-weeks and 
https://www.globenewswire.com/Tracker?data=k4vqJgXcOZw5ANQnsuZVZIQtpfCTyTghQFiEXyxcdwscljCEfTpiWDniGyAydqkFeHqNV9sn43UVGzedxDiifTdROibJ0jwpImNQ5K26iAnuBjzztdaERjkdlmp_wL4CPbDptw3Oa5kHjY5hNRdORA== 
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. 
 
   https://www.globenewswire.com/Tracker?data=5ya3gGCfHh9VasOXkCtffUQauDs2zL2YCAk3TH0MwNaIfWem0wPNNxOyrB2Hz_c1Kwa8tIuef_VuRtWIDezeO1feomNKsBVTS6up4IYw2tTmNBldQmJebnnP9LL8dsL7 
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. 
 
   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://www.globenewswire.com/Tracker?data=ZT8EAmc2TahWjcyu1eo1M-fQkxiVYtOVFyBnoLtHfugmqri8nXteCTlurEowoz30n60PEUIyX3DJPnZ70ByeQNpWovHu2rB9n1fc5XSXCUYbe5dmVzY8DM-sWRnLMKJk95uu9lwi_SYSvuxLgNBVgQ== 
https://vision-dmd.info/2b-trialinformation ) is currently being 
conducted at study sites across North America, Europe, Israel and 
Australia and topline 6-month data are expected in Q2-2021, paving the 
way for a US NDA submission in Q4-2021. Vamorolone has been granted 
Orphan Drug status in the US and in Europe, and has received Fast Track 
and Rare Pediatric Disease designations by the US FDA and Promising 
Innovative Medicine (PIM) status from the UK MHRA. Vamorolone was 
discovered by US-based ReveraGen BioPharma, Inc. and is being developed 
in collaboration with Santhera, which owns worldwide rights to the drug 
candidate in all indications. The vamorolone development program has 
received funding from several international non-profit foundations and 
patient organizations, the US National Institutes of Health, the US 
Department of Defense and the European Commission's Horizon 2020 
program. See 
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www.reveragen.com; 
https://www.globenewswire.com/Tracker?data=ZT8EAmc2TahWjcyu1eo1M-fQkxiVYtOVFyBnoLtHfuj0yqji3rMAb3Ii4SPaXZf9lJOYJG7jbz8UBGl40bYpc0SG2WzLqHrBCAMXBnb3inc= 
https://vision-dmd.info. 
 
   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 Muscular Dystrophy Association USA, Parent Project 
Muscular Dystrophy, Foundation to Eradicate Duchenne, Save Our Sons, 
JoiningJack, Action Duchenne, CureDuchenne, Ryan's Quest, Alex's Wish, 
DuchenneUK, Pietro's Fight, Michael's Cause, and Duchenne Research Fund. 
ReveraGen has also received generous support from the US Department of 
Defense CDMRP, National Institutes of Health (NCATS, NINDS, NIAMS), and 
European Commission (Horizons 2020). 
https://www.globenewswire.com/Tracker?data=33QUPqeXJ43C3vEF7yDx_-iKPrG-bssCW3k3MckgRjxrhCACn0OBKCRDibRKvKkbQ6csJ7MI-lvPaua7zRYQKvQYKB08MN-cyntXp8TJAIs= 
www.reveragen.com. 
 
   Contact 
 
   ReveraGen BioPharma 
 
   Eric Hoffman, PhD, Vice President of Research 
 
   Phone: + 1 240-672-0295 
 
   eric.hoffman@reveragen.com 
 
 
 
   Anhang 
 
 
   -- ReveraGen Receives CRP-final 
      https://ml-eu.globenewswire.com/Resource/Download/665167cb-ee2d-472a-be0e-0b43cff5513c

(END) Dow Jones Newswires

November 17, 2020 12:45 ET (17:45 GMT)