ReveraGen BioPharma

What ReveraGen BioPharma does

ReveraGen BioPharma is a small clinical-stage biopharmaceutical company founded in 2008 and headquartered in the Rockville, Maryland / Vienna, Virginia area. The company developed vamorolone (commercially branded AGAMREE), a first-in-class dissociative steroidal anti-inflammatory drug originally known as VBP15. Vamorolone is engineered by removing a single oxygen moiety from the corticosteroid backbone at the 11β position, retaining the anti-inflammatory efficacy of traditional corticosteroids (NF-κB transrepression) while substantially reducing the side-effect profile associated with transactivation (bone loss, growth suppression, cataracts). The molecule additionally functions as a mineralocorticoid receptor antagonist and is not a substrate for HSD11B1/HSD11B2 enzymes, distinguishing it mechanistically from prednisone and deflazacort. The US FDA approved AGAMREE for Duchenne muscular dystrophy in October 2023 and the EMA followed in December 2023, making it the first DMD therapy approved by both regulatory agencies.

Name

ReveraGen BioPharma

Legal name

ReveraGen BioPharma, Inc.

Website

https://reveragen.com

Company type

Private

Founded year

2008

Operating status

Operating

Headcount range

11–50 employees

Short description

ReveraGen BioPharma is a small clinical-stage biopharmaceutical company that developed vamorolone (AGAMREE), a first-in-class dissociative steroid approved by the FDA and EMA for Duchenne muscular dystrophy. The company operates a venture philanthropy model and licensed worldwide commercialization rights to Santhera Pharmaceuticals.

Ownership category

akta.pro rank

Product category

Orphan Disease Biopharmaceuticals

NAICS

Pharmaceutical Preparation Manufacturing (325412), Research and Development in Biotechnology (except Nanobiotechnology) (541714), Pharmaceutical and Medicine Manufacturing (3254)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

akta.pro secondary industry

Specialty Care Pharmaceuticals (HLAIAAAB)

Keywords

• Orphan drug development
• Duchenne muscular dystrophy
• Dissociative steroid therapy
• Venture philanthropy biotech
• Rare disease therapeutics

Headquarters

HQ city

Rockville

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel

Revenue model

1. Licensing Revenue: ReveraGen developed vamorolone and licensed commercialization rights to Santhera Pharmaceuticals. In September 2020, Santhera exercised its option to obtain worldwide rights to vamorolone for DMD and all other indications. ReveraGen receives licensing fees and royalties from drug sales.
2. Venture Philanthropy Returns: ReveraGen's unique business model commits to returning 2/3 of all income from sales and marketing of vamorolone to the international non-profit sector, enabling foundations to further invest in promising research - a 'pay it forward' model.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

ReveraGen BioPharma develops vamorolone (AGAMREE), a first-in-class dissociative steroidal anti-inflammatory drug originally called VBP15, as a safer alternative to traditional corticosteroids for treating Duchenne muscular dystrophy (DMD) and other inflammatory disorders. The company operates under a venture philanthropy model, having licensed worldwide commercialization rights to Santhera Pharmaceuticals in September 2020. AGAMREE received FDA approval in October 2023 and EMA approval in December 2023 for DMD patients ages 4 and older, becoming the first therapy approved by both agencies for this indication.

Product overview

ReveraGen BioPharma is a biopharmaceutical company that developed vamorolone (commercially named AGAMREE), a first-in-class dissociative steroidal anti-inflammatory drug. The core product is an oral daily medication for Duchenne muscular dystrophy that retains the anti-inflammatory efficacy of corticosteroids while reducing their serious side effects. Vamorolone acts through multiple mechanisms: inhibiting NF-κB inflammatory pathways, providing membrane stabilization effects, and functioning as a mineralocorticoid receptor antagonist. The company operates under a venture philanthropy model and licensed commercialization rights to Santhera Pharmaceuticals. The product portfolio includes the approved DMD treatment plus pipeline candidates for Becker muscular dystrophy and other inflammatory conditions.

Differentiator

Problem solved

Functional benefit

Products and services

• AGAMREE (vamorolone) for Duchenne Muscular Dystrophy AGAMREE is the first-in-class dissociative steroidal anti-inflammatory drug (vamorolone, originally VBP15) developed as an oral once-daily treatment for boys with Duchenne muscular dystrophy ages 4 and older. It retains anti-inflammatory efficacy comparable to traditional corticosteroids while significantly reducing the side effects such as vertebral fractures, growth suppression, and cataracts. Commercialization is licensed to Santhera Pharmaceuticals worldwide.
• Vamorolone for Becker Muscular Dystrophy Pipeline candidate investigating vamorolone treatment in adult patients with Becker muscular dystrophy (BMD) in an FDA-funded Phase 2 pilot study. The product leverages vamorolone's anti-inflammatory properties along with potential dystrophin-increasing effects for the BMD patient population.
• Vamorolone for Other Inflammatory Indications Preclinical and early clinical investigation of vamorolone for potential use in asthma, inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis, ANCA-associated vasculitis (AAV), juvenile dermatomyositis (JDM), LGMD2A (Calpain3 Deficiency), and LGMD2B (dysferlin-deficient limb girdle muscular dystrophy), where glucocorticoids are standard of care but side effects limit long-term use.

Quantifiable outcome

• 80% reduction in vertebral fractures (8.1% vs 41.9% on traditional corticosteroids) over up to 8 years of treatment
• +4 more outcomes

Named customers2 records

Segments3 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Three partnerships are on record, tiered flagship and core.

• Santhera PharmaceuticalsflagshipOEM/ Whitelabel/ Licensing Partner · 2 September 2020In September 2020, Santhera Pharmaceuticals exercised its option to obtain worldwide rights to vamorolone for DMD and all other indications, becoming the direct license holder. Previously, Santhera had licensed from Idorsia and ReveraGen. Santhera handles all commercialization, regulatory submissions, and marketing of AGAMREE.
• Actelion (acquired by Idorsia)coreOEM/ Whitelabel/ Licensing Partner · 3 November 2016Actelion obtained an exclusive option to in-license vamorolone for the treatment of Duchenne Muscular Dystrophy at two different stages in its development. Following Actelion's acquisition by Johnson & Johnson in 2017, Idorsia was spun out and held the rights before Santhera exercised the option.
• NIH Therapeutics for Rare and Neglected Diseases (TRND)coreStrategic or Co-development Partner · 15 August 2011ReveraGen was one of three initial drugs accepted into the NIH TRND program in 2011. NIH program officers provided independent validation of key vamorolone properties and extensive collaborative work on chemistry, manufacturing, controls, and formulation. The partnership ended in mid-2014 after de-risking the program.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Sarepta Therapeutics: Sarepta develops DMD therapies including Elevidys gene therapy and EXONDYS 51, making it a direct competitor to ReveraGen in the same rare pediatric muscular dystrophy market. Sarepta represents the most significant competitive threat to AGAMREE's commercial trajectory.
• PTC Therapeutics: PTC Therapeutics markets Translarna (ataluren) for nonsense mutation DMD and Emflaza (deflazacort) — the latter being a direct corticosteroid competitor to vamorolone. Direct overlap in DMD steroid treatment market.
• Santhera Pharmaceuticals: Santhera holds worldwide commercialization rights to vamorolone (AGAMREE) and is ReveraGen's license partner. Santhera also develops other rare disease therapies (e.g., DMD, mitochondrial disease), making it both a commercial channel and adjacent competitor in DMD.
• Italfarmaco: Italfarmaco developed and markets deflazacort (Emflaza in the US), the corticosteroid whose side effect profile vamorolone was designed to improve. Direct comparator product in DMD steroid treatment.
• Catabasis Pharmaceuticals: Catabasis developed edasalonexent, an oral NF-κB inhibitor for DMD with a similar mechanistic approach to vamorolone's anti-inflammatory pathway. Comparable small-cap DMD drug developer focused on safer alternatives to corticosteroids.

Emerging players

• Capricor Therapeutics: Capricor is developing CAP-1002 (cell therapy) for DMD and other rare diseases. Emerging DMD player with a different modality (cell therapy) but same orphan pediatric indication overlap.
• Solid Biosciences: Solid Biosciences is developing SGT-001 and other gene therapies for Duchenne muscular dystrophy. Emerging DMD gene therapy player competing for the same patient population with a curative-intent approach.

Broad incumbents

• FibroGen: FibroGen developed pamrevlumab for DMD and other rare diseases, including Duchenne. Broad rare disease biopharma incumbent with overlapping DMD pipeline exposure.
• BioMarin Pharmaceutical: BioMarin is a large rare disease biopharma with a portfolio including enzyme replacement and gene therapies for genetic conditions. Broad incumbent in the orphan drug space with muscular dystrophy-adjacent expertise.

Others

• Parent Project Muscular Dystrophy: PPMD is a nonprofit venture philanthropy partner that has funded ReveraGen's vamorolone program through the Xcelerate grant program. Represents the foundation ecosystem that both funds ReveraGen and shares in upside via the "pay it forward" model.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/reveragen

Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds1 record

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about ReveraGen BioPharma