Santhera and ReveraGen Start Rolling NDA Submission to the FDA for
Vamorolone for the Treatment of Duchenne Muscular Dystrophy
Pratteln, Switzerland, March 29, 2022 – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen
BioPharma, Inc (US: private) announce the initiation of a rolling new drug application (NDA)
submission to the U.S. Food and Drug Administration (FDA) for vamorolone for the treatment ofDuchenne muscular dystrophy (DMD). Vamorolone for DMD has been granted Fast Track Designationby the FDA.
Santhera and ReveraGen have now commenced the NDA filing as a rolling submission following a
successful pre-NDA meeting with the FDA in November 2021. In its conclusions from this meeting, theFDA considered the proposed clinical efficacy and safety data sufficient to support an NDA filing ofvamorolone for the treatment of DMD. Acceptance of the NDA will be subject to FDA’s review of thecomplete filing.
“The NDA filing for vamorolone marks a tremendous milestone for Santhera and an important next stepfor the Duchenne community. The potential benefits of vamorolone could address significant unmet
needs that represent a burden to DMD patients and their families,” said Dario Eklund, Chief Executive
Officer of Santhera. “We are fully focused on working closely with the FDA and completing thesubmission as soon as possible.”“We are delighted about the initiation of the filing for approval for vamorolone as it represents aculmination of over a decade of scientific research for the benefit of patients with DMD. In having granted
Fast Track Designation, the FDA has acknowledged the significant innovation vamorolone could bring inaddressing a high unmet medical need of patients with DMD,” said Eric Hoffman, PhD, President andCEO of ReveraGen BioPharma. “We thank study participants, their families and caregivers, as well asinvestigators and study personnel, for their commitment to the vamorolone program.”
Santhera and ReveraGen expect to complete the NDA filing in the second quarter of 2022. Based on FDA
review timelines, notification from the FDA on the acceptance of the filing for review is expected inAugust 2022. In its assessment, the FDA will also determine eligibility of vamorolone for priority review
which Santhera and ReveraGen will request upon completion of the rolling NDA submission. If granted,
this would shorten review time and set an anticipated approval date for as early as the first quarter of2023. Subject to FDA approval, Santhera plans to launch vamorolone in the U.S., the first country, shortly
thereafter with its own organization.
In Europe, Santhera plans to submit a marketing authorization application (MAA) for vamorolone for thetreatment of DMD to the European Medicines Agency (EMA) in Q3-2022. Assuming a review time ofabout one year, this could pave the way for approval and launch in H2-2023. Santhera expects the firstEuropean launch country to be Germany.

Santhera and ReveraGen Start Rolling NDA Submission to the FDA forVamorolone for the Treatment of Duchenne Muscular Dystrophy
March 29, 2022 / Page 2 of 3
Vamorolone has been granted Orphan Drug status in the US and in Europe for DMD, and has received
Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine(PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently notapproved for use by any health authority.
Vamorolone was discovered by US-based ReveraGen and is being developed in collaboration withSanthera who owns worldwide rights to the drug candidate for all indications. In January 2022, Santhera
licensed rights to vamorolone in rare diseases for the Greater China region to Sperogenix Therapeutics.
About Vamorolone and the VISION-DMD study
Vamorolone is a drug candidate with a novel mode of action that binds to the same receptor as
corticosteroids but modifies its downstream activity and as such is a dissociative agonist [2-4]. Thismechanism has the potential to ‘dissociate’ efficacy from typical steroid safety concerns.
VISION-DMD was a Phase 2b study comprising a (1) pivotal double-blind 24-week period to demonstrateefficacy and safety of vamorolone (2 and 6 mg/kg/day) versus placebo and prednisone (0.75 mg/kg/day),followed by a (2) 24-week period to evaluate the maintenance of efficacy and collect additional longerterm safety and tolerability data. 121 ambulant boys aged 4 to <7 years with Duchenne muscular
dystrophy (DMD) were included in the study. The trial met its primary endpoint of superiority in changeof time to stand from supine position (TTSTAND) velocity with vamorolone 6 mg/kg/day versus placebo(p=0.002) at 24 weeks (period 1). Vamorolone 6 mg/kg/day also met its secondary efficacy endpoints
and no statistically significant differences were observed between vamorolone and prednisone. During
the second 24-week period of this 48-week study, all participants received vamorolone. Participants fromthe placebo and prednisone arms were randomized to either the 2 or 6 mg/kg/day dose of vamorolone
and the vamorolone arms continued on their existing dose. Efficacy observed at 24 weeks forvamorolone 6 mg/kg/day was maintained across multiple endpoints over 48 weeks. In addition toefficacy, the study aimed to confirm the favorable tolerability profile of vamorolone with the potential
to offer an alternative to current standard of care.
In clinical studies, vamorolone was generally well tolerated. The most commonly reported adverse
events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin Ddeficiency. Adverse events were generally of mild to moderate severity.
References:[1] ClinicalTrials.gov Identifier: NCT03439670, link[2] Heier CR at al. (2013). EMBO Mol Med 5: 1569–1585.
[3] Reeves EKM, et al (2013). Bioorg Med Chem 21(8):2241-2249.
[4] Liu X, et al. (2020). Proc Natl Acad Sci USA 117:24285-24293.

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 inflammation which is present at birth or shortly
thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle
degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of selffeeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life
expectancy to before the fourth decade due to respiratory and/or cardiac failure.

Santhera and ReveraGen Start Rolling NDA Submission to the FDA for
Vamorolone for the Treatment of Duchenne Muscular Dystrophy
March 29, 2022 / Page 3 of 3About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on thedevelopment and commercialization of innovative medicines for rare neuromuscular and pulmonary
diseases with high unmet medical need. Santhera has an exclusive license for all indications worldwide
to vamorolone, a dissociative steroid with novel mode of action, which was investigated in a pivotal study
in patients with DMD as an alternative to standard corticosteroids. The Company plans to complete therolling submission of its filing for approval for vamorolone with the US FDA in Q2-2022. The clinical stage
pipeline also includes lonodelestat to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases.
Santhera out-licensed rights to its first approved product, Raxone® (idebenone), outside North Americaand France for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further
information, please visit www.santhera.com.
Raxone® is a trademark of Santhera Pharmaceuticals.
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 leadcompound, vamorolone, has been supported through partnerships with foundations worldwide,including Muscular Dystrophy Association USA, Parent Project Muscular Dystrophy, Foundation toEradicate Duchenne, Save Our Sons, JoiningJack, Action Duchenne, CureDuchenne, Ryan’s Quest, Alex’sWish, 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,National Institutes of Health (NCATS, NINDS, NIAMS), and European Commission (Horizons 2020).
www.reveragen.com
For further information please contact:
SantheraSanthera Pharmaceuticals Holding AG, Hohenrainstrasse 24, CH-4133 Pratteln
public-relations@santhera.com orEva Kalias, Head External Communications
Phone: +41 79 875 27 80
eva.kalias@santhera.com
ReveraGen BioPharma
Eric Hoffman, PhD, President and CEO
Phone: + 1 240-672-0295
eric.hoffman@reveragen.com
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities
of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking
statements concerning the Company and its business. Such statements involve certain risks,uncertainties and other factors which could cause the actual results, financial condition, performance orachievements of the Company to be materially different from those expressed or implied by suchstatements. Readers should therefore not place undue reliance on these statements, particularly not inconnection with any contract or investment decision. The Company disclaims any obligation to updatethese forward-looking statements.
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