The Story of the Foundation to Eradicate Duchenne

Media Release
June 2, 2020

ReveraGen BioPharma Completes 2.5 Years Vamorolone Treatment of 41 Duchenne Muscular Dystrophy Boys

Rockville, MD

Vamorolone is a first-in-class daily oral drug being developed to improve muscle function in Duchenne muscular dystrophy. Vamorolone has multiple mechanisms of action shown by published pre-clinical studies, including potent anti-inflammatory activities, cardioprotective activity, promotion of membrane repair, and synchronization of cell repair. While a steroidal drug, pre-clinical and clinical data has shown that vamorolone may lack multiple safety concerns of corticosteroidal anti-inflammatories, such as deflazacort and prednisone, while adding novel aspects of potential efficacy such as mineralocorticoid antagonism.

In 2016-2017, 48 DMD boys (age 4 to <7 years) entered a series of pharmacokinetics, safety and dose-finding efficacy studies (VBP15-002; VBP15-003). After 6-months of treatment, the DMD participants and their families were given the option to transition to standard of care (deflazacort or prednisone), or remain on vamorolone via a 2-year long-term extension study (VBP15-LTE). Of the 46 DMD boys completing the 6-month dose-ranging study, all (100%) requested to continue vamorolone treatment in the long-term extension, rather than transition to corticosteroids. The last participant, last visit of VBP15-LTE occurred in April 2020, with 41 of 46 DMD boys completing the full 2-year treatment period. The large majority of the 41 DMD boys completing the 2-year LTE have transitioned to Expanded Access Program (USA, Canada, Israel), or compassionate use programs (UK, Sweden, Australia).

“Parents and their physicians seem to be satisfied with vamorolone, as nearly all wish to continue vamorolone treatment,” said Paula Clemens, MD, Professor at the University of Pittsburgh School of Medicine, and Study Chair. The long-term extension study enabled dose escalation and de-escalation at the preference of the physician and family (suggested range 2.0 to 6.0 mg/kg/day). Of those 41 participants completing the 2-year end-of-study visit, 27 ended at 6.0 mg/kg/day (66%), 11 at 2.0 mg/kg/day (27%), and 3 at 4.0 mg/kg/day (7%). Thus, the majority (2/3) of physicians/families chose treatment at the highest tested dose of vamorolone by the end of the LTE study (6.0 mg/kg/day).

“With most participants continuing treatment with vamorolone long-term, we have assembled a strong safety database, with 106 patient-years of vamorolone exposure in DMD boys, with no serious adverse events attributable to vamorolone to date,” said Eric Hoffman, PhD, Vice President of Research at ReveraGen BioPharma.

A registration trial, VBP15-004, is ongoing with 103 of 128 DMD participants enrolled. Enrollment is expected to complete soon, with 6-month read-out for FDA NDA submission in 4Q2020 or 1Q2021. Information on the VBP15-004 trial can be obtained from Suzanne Gaglianone ( or Andrea D’Alessandro (


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About Duchenne muscular dystrophy
Duchenne muscular dystrophy is a rare genetic disease that predominantly affects young boys. Loss of the large protein, dystrophin, in muscle leads to persistent damage to muscle. DMD is a progressive disease, with gradual loss of muscle and weakness over 20 years leading to loss of walking abilities, and shortened lifespan.


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).


About vamorolone
Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors [1,2]. This has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore could replace existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is significant unmet medical need in this patient group as high dose corticosteroids have severe systemic side effects that detract from patient quality of life. Phase 1 studies in adult volunteers [3], and Phase 2a studies in 48 DMD boys [4] showed biomarker studies consistent with a partial agonist mechanism of action, with dose-responsive improvements in both efficacy and safety biomarkers. Dose-finding studies with 24-weeks of vamorolone treatment over a dose range of 0.25 to 6.0 mg/kg/day showed dose-related improvements in multiple measures of muscle strength and endurance [5]. Vamorolone has been granted Orphan Drug status by both FDA and EMA, Fast Track designation by the FDA, and Priority Innovative Medicine designation by the UK MHRA. In November 2018, Santhera acquired from Idorsia Pharmaceuticals Ltd (SIX: IDIA), who has an option to an exclusive, worldwide license to vamorolone, the option to an exclusive sub-license to vamorolone in all indications and all countries worldwide, except Japan and South Korea.


[1] Heier CR at al. (2013). VBP15, a novel anti‐inflammatory and membrane‐stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med 5: 1569–1585

[2] Heier CR et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Science Alliance DOI 10.26508/lsa.201800186

[3] Hoffman EP et al. (2018). Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids 134: 43-52.

[4] Conklin LS et al. (2018). Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first in-class dissociative steroidal anti-inflammatory drug. Pharmacological Research 136:140-150.

[5] Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 93: e1312-e1323


About the Cooperative International Neuromuscular Research Group (CINRG)
CINRG was founded in 2000 as an international academic clinical trial network, with a focus on pediatric neuromuscular disease. CINRG has enrolled over 1,500 patients into clinical research studies. Recent studies include the CINRG Duchenne Natural History Study (DNHS) with 440 DMD patients and over 100 healthy peers followed by expert neuromuscular physicians in 20 sites in 10 countries. See

Media Release
May 26, 2020

NS Pharma Announces Publication of Clinical Trial Data for Viltolarsen in DMD Patients in JAMA Neurology


NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka), a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; President Shigenobu Maekawa), announced today that JAMA Neurology has published results from a clinical trial of viltolarsen, an investigational agent being evaluated in Duchenne muscular dystrophy (DMD) patients who are amenable to exon 53 skipping therapy.

“In this study, 100% of patients were shown to have more dystrophin after treatment with viltolarsen and 88% achieved dystrophin levels of greater than 3%,” said lead study author and investigator Paula Clemens, MD, University of Pittsburgh School of Medicine. “These increases were seen in patients as young as four years of age and after six months or less of treatment, which underscores the impressive results seen in this study.”

This Phase 2, two-period, dose-finding study enrolled 16 DMD patients, from four to less than 10 years of age, who were amenable to exon 53 skipping therapy. Study participants were randomized to two doses of viltolarsen (40 mg/kg/wk and 80 mg/kg/wk) for 20 to 24 weeks. At the end of the study, treatment with viltolarsen was associated with statistically significant increases in mean dystrophin expression (40 mg/kg/wk: p<0.001; 80 mg/kg/wk p=0.012). Mean dystrophin levels of 5.7% and 5.9% were observed in comparison to mean baseline levels of 0.3% and 0.6% in the 40 mg/kg/wk and 80 mg/kg/wk groups, respectively. Fourteen out of 16 patients (88%) reached dystrophin levels greater than 3%.

The most common treatment emergent adverse events occurring in greater than one patient were: cold, cough, nasal congestion, bruising, joint pain, diarrhea and vomiting. No serious adverse events were observed in the study.

“Lack of functional dystrophin is recognized as the singular underlying cause of the devastating impact of DMD,” said study author and investigator Vamshi Rao, MD, Ann & Robert H. Lurie Children’s Hospital of Chicago. “As a pediatric neurologist who specializes in the treatment of DMD, I am encouraged by the dystrophin increases observed in this study and the potential of viltolarsen to address the underlying cause of DMD.”

“This is one of the first studies of exon skipping therapies in DMD to generate rigorous data on the primary biomarker of dystrophin protein,” said study author Eric Hoffman, PhD, Associate Dean for Research and Professor of Pharmaceutical Sciences at Binghamton University. “Having assisted in the development of viltolarsen for many years, and decades more researching the genetics and genomics of DMD, I am pleased by the results of this study and the implications for families facing DMD.”

Viltolarsen has not yet been approved in the U.S. and its New Drug Application was recently granted Priority Review by the FDA with an anticipated action date in the third quarter of 2020. In March 2020, viltolarsen was approved in Japan for the treatment of DMD patients amenable to exon 53 skipping therapy.

“We are deeply committed to the development of viltolarsen and offering healthier futures to DMD patients and families,” said Tsugio Tanaka, President, NS Pharma, Inc. “Based on these results we are optimistic that, if it is approved, viltolarsen will become an important new treatment option for DMD patients and healthcare providers.”

NS Pharma continues to study the safety and efficacy of viltolarsen in the confirmatory Phase 3 RACER53 trial. This study was initiated in October 2019 and is currently enrolling. The purpose of this Phase 3 trial is to confirm the clinical findings that were submitted under the Accelerated Approval pathway.


About Duchenne Muscular Dystrophy (DMD)
DMD is a progressive form of muscular dystrophy that occurs primarily in males. DMD causes progressive weakness and loss of skeletal, cardiac, and pulmonary muscles. Early signs of DMD may include delayed ability to sit, stand or walk. There is a progressive loss of mobility, and by adolescence, patients with DMD may require the use of a wheelchair. Cardiac and respiratory muscle problems begin in the teenage years and lead to serious, life-threatening complications.


About Viltolarsen
Viltolarsen has been granted Rare Pediatric Disease, Orphan Drug and Fast Track Designations in the U.S. The viltolarsen New Drug Application was granted Priority Review by the FDA with an anticipated action date in the third quarter of 2020. In March 2020, viltolarsen was approved in Japan for the treatment of patients with DMD who are amenable to exon 53 skipping therapy. Prior to its approval in Japan, viltolarsen was granted with the SAKIGAKE designation, Orphan drug designation, and designation of Conditional Early Approval System.


About NS Pharma, Inc.
NS Pharma, Inc., is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd. For more information, please visit NS Pharma is a registered trademark of the Nippon Shinyaku group of companies.


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Media Release
October 17th, 2019

Vamorolone Designated Promising Innovative Medicine (PIM) for treatment in Duchenne muscular dystrophy

Rockville, MD

In the UK the Early Access to Medicines Scheme (EAMS) is a regulatory path by the MRHA that aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.  The initial step in this process is Promising Innovative Medicine (PIM) designation.


Co-founders of Duchenne UK Alex Johnson and Emily Crossley said, “We are delighted that MHRA has given PIM status to Vamorolone as a treatment for Duchenne Muscular Dystrophy. A PIM designation is the first step of a process that could allow patients earlier access to a new medicine. This is part of the Early Access to Medicines Scheme which Duchenne UK and Joining Jack lobbied for in 2014. We are pleased to see that the scheme may be used for Vamorolone.”


UK foundations that have aided the development of vamorolone for DMD include Joining Jack, Duchenne Children’s Trust, ActionDuchenne, Alex’s Wish Foundation, and Duchenne Research Fund.


Vamorolone is a first-in-class drug that targets multiple biochemical pathways in DMD patient muscle simultaneously, and in initial open label studies has shown improvements of patient muscle function.  A pivotal trial that may lead to drug approval is currently enrolling patients age 4 to 7 years at 6 sites in the United Kingdom (Newcastle University, Royal Hospital for Children [Glasgow], Alder Hey Children’s Hospital [Liverpool], Leeds Teaching Hospital Trust, Great Ormond Street Institute of Child Health [London] and University Hospitals Birmingham). Information on the currently recruiting vamorolone clinical trial with contact information for UK recruitment sites can be found at


About the UK Early Access to Medicines Scheme (EAMS)

The UK’s industry-sponsored EAMS aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. The EAMS is a two-step process:

Step I is the Designation as a Promising Innovation Medicine (PIM). The PIM designation is an early indication that a medicinal product is a promising candidate for EAMS and gives reassurance that its clinical development is on track by having an early review of its data by the medicines regulator.

Step II is the Scientific Opinion by the Medicines and Healthcare products Regulatory Agency (MHRA, UK regulatory agency). The Scientific Opinion describes the benefits and risks of the medicine and supports the prescriber and patient to make a decision on using the medicine before its license is approved.

What is Duchenne Muscular Dystrophy?