Resource Data

Santhera and ReveraGen will present new data with Vamorolone


Pratteln, Switzerland,
and Rockville, MD, USA, June 92022 –Santhera Pharmaceuticals (SIX:SANN) and ReveraGen BioPharma, Inc. announce presentations of New Data with vamorolone in Duchenne muscular dystrophy (DMD).

Cross-study comparisons of long-term efficacy, impact on growth velocity, frequency and severity of vertebral fractures for vamorolone versus current standard of care from the phase 2b study VISION-DMD and the FOR-DMD study will be featured during the poster session at the Parent Project Muscular Dystrophy (PPMD) 2022 Annual Conference June 23-26, 2022 in Scottsdale, AZ, USA .

“The data presented by our academic collaborators at PPMD ​​this year will provide further insight into our understanding of the potential of a dissociative steroid, such as vamorolone, in maintaining muscle strength while addressing important safety concerns. such as preventing growth retardation and reducing the frequency and severity of bone-related side effects such as vertebral fractures in the treatment of Duchenne muscular dystrophy,” said Dr. Shabir Hasham, Chief Medical Officer Officer and Global Medical Director Business of Santhera.

The propensity score matched the populations of the pivotal Phase 2b VISION-DMD study and the FOR-DMD study compared the efficacy of vamorolone with that of prednisone and deflazacort over 48 weeks. Safety analyzes focused on comparing long-term endpoints of height and vertebral fractures, with vamorolone data collected in the VBP15-LTE study (long-term extension) [1] versus prednisone and deflazacort data from the FOR-DMD study [2] over 2.5 years.

Abstracts will be available on the PPMD ​​website.

Santhera will be participating in the PPMD ​​Resource Fair (June 23-24) during the conference where medical representatives from the company will be on hand to provide scientific information and the latest updates.

Santhera expects to complete the New Drug Application (NDA) filing for vamorolone in DMD with the US FDA in June 2022. Vamorolone has been granted orphan drug status in the US and Europe for DMD , and has received Fast Track and Rare Pediatric Disease designations from the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational drug and is not currently approved by any health authority.

About Vamorone
Vamorolone is a drug candidate with a novel mode of action that binds to the same receptor as corticosteroids but alters its downstream activity and as such is a dissociative agonist [2-4]. This mechanism has the potential to ‘decouple’ efficacy from typical steroid safety issues and therefore vamorolone could emerge as a promising alternative to existing corticosteroids, the current standard of care in children and adolescents with DMD. . In the pivotal VISION-DMD study, vamorolone achieved the primary endpoint Withdrawal Time (TTSTAND) speed compared to placebo (p=0.002) at 24 weeks of treatment and showed a good safety profile and tolerability. In clinical studies, vamorolone was generally well tolerated. The most frequently reported adverse events compared to placebo in the VISION-DMD study were Cushings-like features, vomiting and vitamin D deficiency. Adverse events were generally mild to moderate in intensity.

About the FOR-DMD study
Finding the Optimum Regimen for DMD (FOR-DMD; ClinicalTrials.gov identifier NCT01603407 / EudraCT 2010-023744-33) is a randomized, prospective, multicenter, double-blind, comparative efficacy and safety study of 3 corticosteroid regimens (prednisone daily, intermittent prednisone, or deflazacort daily) in boys aged ≥ 4 years and

References:
[1] Mah JK et al (2022). JAMA Netw Open. 2022;5(1):e2144178. doi:10.1001/jamanetworkopen.2021.44178
[2] Guglieri, et al (2022) JAMA. doi:10.1001/jama.2022.4315
[3] Heier CR et al (2019). Life Sciences Alliance DOI: 10.26508
[4] Liu X, et al (2020). Proc Natl Acad Sci USA 117:24285-24293

About Santhera
Santhera Pharmaceuticals (SIX:SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. Santhera holds an exclusive license for all indications worldwide for vamorolone, a dissociative steroid with a novel mode of action, which has been studied in a pivotal study in patients with DMD as an alternative to standard corticosteroids. The company expects to complete the continued submission of its approval dossier for vamorolone to the U.S. FDA in June 2022. The clinical-stage pipeline also includes lonodelestat to treat cystic fibrosis (CF) and other lung diseases neutrophils. Santhera has licensed the rights to its first approved product, Raxone® (idebenone), outside of North America and France for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) to the Chiesi Group. For more information, please visit www.santhera.com.

raxon® is a registered trademark of Santhera Pharmaceuticals.

About ReveraGen BioPharma
ReveraGen was founded in 2008 to develop first-class dissociative steroid drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has been supported by partnerships with foundations around the world, 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, Duchenne Research Fund and Defeat Duchenne Canada. ReveraGen has also received generous support from the US Department of Defense CDMRP, the National Institutes of Health (NCATS, NINDS, NIAMS), and the European Commission (Horizons 2020). www.reveragen.com

For more information, please contact:

Santhera
Santhera Pharmaceuticals Holding AG, Hohenrainstrasse 24, CH-4133 Pratteln
[email protected] or
Eva Kalias, Head of Investor Relations and Communications
Telephone: +41 79 875 27 80
[email protected]

ReveraGen BioPharma
Eric Hoffman, PhD, President and CEO
Telephone: + 1 240-672-0295
[email protected]

Disclaimer / Forward-Looking Statements
This communication does not constitute an offer or an invitation to subscribe or purchase securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements regarding the Company and its business. These statements involve certain risks, uncertainties and other factors that could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on such statements, particularly in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

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