Press Release: Santhera: ReveraGen Receives $3.3 Million NIH Commercialization Readiness Pilot Grant for NDA Preparations for Vamorolone in Duchenne Muscular...

-- 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;

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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

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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.

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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,

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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;

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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).

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www.reveragen.com.

Contact

ReveraGen BioPharma

Eric Hoffman, PhD, Vice President of Research

Phone: + 1 240-672-0295

eric.hoffman@reveragen.com

Attachment

-- ReveraGen Receives CRP-final

https://ml-eu.globenewswire.com/Resource/Download/45f5d64c-1a55-4bf0-b9d4-2bf05b6e637f

(END) Dow Jones Newswires

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