Data from international public/private partnership leads to the first therapy for Duchenne muscular dystrophy approved by both EMA and FDA.

Data from interna,onal public/private partnership leads to the first therapy for Duchenne muscular dystrophy approved by both EMA and FDA. - AGAMREE®(vamorolone) oral suspension is the first pharmacological interven;on in Duchenne muscular dystrophy (DMD) to demonstrate robust evidence of efficacy via a randomized, double-blind, placebo-controlled clinical trial. - The robust and compelling data suppor;ng approval of AGAMREE in both the EU (EMA) and US (FDA) resulted from a broad interna;onal public/private par;cipa;on model, involving government agencies, over a dozen non-profit founda;ons, an established interna;onal academic clinical trial network (CINRG; TRiNDS), and ReveraGen BioPharma Inc spanning over 13 years. Rockville, MD The EMA approved AGAMREE (vamorolone) for Duchenne muscular dystrophy (DMD), ages 4 years and older on 18 December 2023. The US FDA previously approved AGAMREE for DMD (26 October 2023). This makes AGAMREE the first pharmacological intervenOon poised to receive full approval for DMD populaOons in both the US and EU. AGAMREE was developed uOlizing an innovaOve public and private parOcipaOon model with central governments, non-profit foundaOons and commercial stage research and development organizaOons working in coordinaOon to advance the development program from discovery through clinical trials all the way to drug approval. PaOent-focused foundaOons including the Muscular Dystrophy AssociaOon (USA), Parent Project Muscular Dystrophy foundaOon (USA), Joining Jack (UK), DRF (UK) and Duchenne Children’s Trust (UK)), under venture philanthropy models (shared risk, shared benefit) provided financial support for specific development acOviOes. Further investments in the form of licensing and milestone payments by Actelion, Idorsia, and Santhera provided key addiOonal resources to complete the AGAMREE development program. The US Department of Defense through the Congressionally Directed Medical Research Programs (CDMRP) funded research for early drug discovery, clinical outcomes research, and natural history studies by the CooperaOve InternaOonal Neuromuscular Research Group. The NaOonal InsOtutes of Health helped de-risk the non-clinical program by compleOng robust confirmatory efficacy studies and implemenOng an improved syntheOc process for drug manufacturing (NaOonal Center for Advancing TranslaOonal Sciences – TRND program) and supported clinical trials (NaOonal InsOtute of Neurological Disorders and Stroke – SBIR program). The European Commission provided a Horizons 2020 grant for the clinical trial program through Newcastle University in the UK. “The overall AGAMREE program leveraged infrastructure for drug development and clinical trials in DMD, with extensive peer review and feedback at each step of the development pipeline,” said Dr . Eric Hoffman, CEO, and co-founder of ReveraGen. “A broad swath of the internaOonal stake holders and the academic research community has been inOmately involved with bringing AGAMREE to approval,” Dr. Hoffman conOnued. “The CooperaOve InternaOonal Neuromuscular Research Group (CINRG) and the TRiNDS coordinaOng center brought extensive disease-specific experOse to the clinical trial program,” said Dr . Paula Clemens, Professor of Neurology at the University of Pibsburgh, and Study Chair of the Phase 2a and Phase 2b clinical trials. “Without the extensive outcomes research and collaboraOve structure of the CINRG group, obtaining the robust data from the AGAMREE trials would likely not have been possible,” Dr . Clemens noted. A key aspect of de-risking of the AGAMREE program was de-risking in the early pre-clinical space. "One reason for the program's success is robust foundaOonal preclinical in vitro, and in vivo data using well- standardized outcome measures that our laboratories have developed over the last two decades in collaboraOon with the internaOonal TREAT-NMD network,” said Dr. Kanneboyina Nagaraju co-founder of ReveraGen, and Dean of the School of Pharmacy and PharmaceuOcal Sciences, Binghamton University – State University of New York. Dr . Jesse Damsker, COO of ReveraGen said, “I believe the compelling data of vamorolone efficacy and safety through the clinical trials, and extensive and successful inspecOons by both FDA and EMA of our clinical trial program, is a testament to the highly collaboraOve team and supporOng expert CROs.” The flexibility of the vamorolone clinical team to respond to the COVID-19 pandemic was aided by the very small central group coordinaOng the studies, and rapid and effecOve communicaOons with the FDA. “I’ve worked in major pharma for many decades, and working with this small ReveraGen team and their collaboraOve network is one of the best examples of efficiency and adaptability I’ve seen in the drug development space,” said Dr . John McCall, VP for Chemistry and co-founder of ReveraGen. “The FoundaOon to Eradicate Duchenne has been a parOcipant in the AGAMREE project since the beginning, and I am delighted to see AGAMREE be approved,” said Joel Wood, President, and CEO of the foundaOon. “Children's NaOonal Hospital was the iniOal home for early vamorolone research, and ReveraGen the first spin-off company,” said Mark Batshaw, M.D., disOnguished invesOgator in the Center for GeneOc Medicine Research at Children’s NaOonal. “The EMA and FDA approval underscores the importance of supporOng clinicians and researchers who are developing soluOons to advance healthcare for children. “We are delighted that proceeds from the future sales of AGAMREE will be shared with over a dozen non-profit foundaOons, conOnuing the shared risk – shared benefit agreements between ReveraGen and these foundaOons,” noted Dr . Hoffman. Significant porOons of the proceeds from sales-based milestone payments and dividends that are anOcipated to be coming to ReveraGen as a result of licensing the product will be distributed to non-profits based on sales of AGAMREE. About Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammaOon which is present at birth or shortly thereaker . InflammaOon leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneraOon and weakness. Major milestones in the disease are the loss of ambulaOon, the loss of self- feeding, the start of assisted venOlaOon, and the development of cardiomyopathy. CorOcosteroids are the current standard of care for the treatment of DMD. About ReveraGen BioPharma Inc. ReveraGen was founded in 2008 to develop first-in-class dissociaOve 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 foundaOons worldwide, including Muscular Dystrophy AssociaOon USA, Parent Project Muscular Dystrophy, FoundaOon to Eradicate Duchenne, Save Our Sons, JoiningJack, AcOon Duchenne, CureDuchenne, Ryan’s Quest, Alex’s Wish, DuchenneUK, Pietro’s Fight, Michael’s Cause, Duchenne Research Fund, and Defeat Duchenne Canada. ReveraGen has also received generous support from the US Department of Defense CDMRP , NaOonal InsOtutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020). www.reveragen.com This work was supported by the Office of the Assistant Secretary of Defense for Health Affairs and the Defense Health Agency J9, Research and Development Directorate, or the U.S. Army Medical Research AcquisiOon AcOvity at the U.S. Army Medical Research and Development Command. Opinions, interpretaOons, conclusions and recommendaOons are those of the author and are not necessarily endorsed by the Department of Defense.

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