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News from Vamorolone in Duchenne Muscular Dystrophy

Santhera Announces Completion of First 6-Month Period of Pivotal VISION-DMD Trial with Vamorolone in Duchenne Muscular Dystrophy

Original Press release > Pratteln, Switzerland, March 3, 2021

Santhera Pharmaceuticals announces that the last patient has completed the last visit for the first period of the placebo-controlled pivotal VISION-DMD study with vamorolone in patients with Duchenne muscular dystrophy (DMD), conducted by partner ReveraGen Biopharma Inc. Subject to a positive 6-month topline data readout of this first study phase, this could allow for regulatory submission to the US FDA in Q1-2022 with the potential to offer an alternative to the current standard of care in DMD.

The 48-week Phase 2b VISION-DMD study is designed as a pivotal trial to demonstrate the efficacy and safety of vamorolone administered orally versus prednisone and placebo in ambulant boys aged 4 to <7 years with DMD. Efficacy outcome measures are motor function and strength outcomes with the Time to Stand test (TTSTAND) as the primary study endpoint. Additional analyses compare safety and tolerability between the vamorolone dose groups, placebo and prednisone. In the now completed 24-week, placebo- and active-controlled treatment period, patients were randomized to receive vamorolone 2.0 mg/kg/day, vamorolone 6.0 mg/kg/day, prednisone 0.75 mg/kg/day or matching placebo. For the second treatment period of another 24 weeks of continued study conduct, patients who previously received prednisone or placebo have been randomized and will be switched to one of two doses of vamorolone. This treatment period where all patients receive vamorolone is evaluating the persistence of effect in the longer term. In addition to efficacy, the study aims to confirm the differentiated safety and favourable tolerability profile of vamorolone with the potential to offer an alternative to the current standard of care. Although glucocorticoids are part of DMD’s current care recommendations, their adverse effect profile limits their use as chronic therapy.

 

Eric Hoffman, Ph.D., President and CEO at ReveraGen BioPharma – “We are delighted about having achieved this important milestone and are looking forward to announcing the topline 6-month results of this pivotal study together with Santhera. The use of glucocorticoids, despite having proven benefits in the treatment of DMD, is severely limited due to side effects and poor tolerability. Our expectation is that vamorolone will have the benefits but avoids many of the tolerability issues that limit the use of this standard of care. Our thanks go out to the study participants, their families and healthcare professionals who, in the midst of the COVID-19 pandemic, are enabling us to advance this pivotal study as intended.”

 

Dario Eklund, CEO of Santhera – “Based on previously established data, we believe that vamorolone has the potential to become a foundational therapy in DMD for patients irrespective of the underlying gene mutation and a promising alternative to existing corticosteroids. Our organization is wholeheartedly dedicated to bringing this novel therapy to patients who are hoping for a DMD therapy with fewer treatment-limiting side effects, making it suitable for longer-term administration and also improving quality of life.”

 

 

About Vamorolone – first-in-class dissociative steroid

Vamorolone is a first-in-class drug candidate that binds to the same receptors as corticosteroids but modifies the receptors’ downstream activity. 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 a significant unmet medical need in this patient group as high-dose corticosteroids have severe systemic side effects that detract from treatment compliance and patient quality of life.

In the pivotal Phase 2b VISION-DMD trial, the last patient has completed the last visit of the 24-week, placebo- and active-controlled treatment period. Topline 6-month data are expected in Q2-2021, paving the way for a US NDA submission in Q1-2022. Vamorolone has been granted Orphan Drug status in the US and Europe and has received Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status 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.

Clinical trial in Canada

 

About Santhera

Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing and commercializing innovative medicines for rare neuromuscular and pulmonary diseases with a high unmet medical need. Santhera is building Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa® (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone, a first-in-class dissociative steroid presently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. Santhera out-licensed ex-North American rights to its first approved product; for further information, please visit www.santhera.com.

 

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 USAParent Project Muscular DystrophyFoundation to Eradicate DuchenneSave Our SonsJoiningJackAction DuchenneCureDuchenneRyan’s QuestAlex’s WishDuchenneUKPietro’s FightMichael’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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