Santhera and ReveraGen to Present Phase 2a/2b Efficacy and New
Safety Data with Vamorolone at Forthcoming Conferences
Pratteln, Switzerland,
and Rockville, MD, USA,
September 23,
2021 – Santhera Pharmaceuticals
(SIX: SANN) and ReveraGen BioPharma, Inc (US:
private) announce presentations
of the positive topline
results from the pivotal
VISION-DMD study and new safety analyses
from long-term treatment with vamorolone at
the forthcoming World Muscle
Society (WMS)
2021 Virtual Annual Conference and
The American Society for Bone and Mineral Research (ASBMR)
2021 Annual Meeting.
Recently, Santhera and ReveraGen announced
statistically highly significant and clinically relevant data from
the pivotal Phase 2b VISION-DMD study in patients with Duchenne
muscular dystrophy (DMD) [1-3]. In the first 24-week phase of this
trial, vamorolone demonstrated statistically significant and
clinically relevant improvements versus placebo across five
functional outcome measures and over a three-fold dose range from 2
to 6 mg/kg/day. The efficacy data confirm earlier results from
a Phase 2a long-term extension trial, where vamorolone demonstrated
similar efficacy to corticoid-treated DMD-patients after 30 months
of treatment [4]. Based on clinical trial results, including
long-term safety data up to 30 months, vamorolone at doses up to 6
mg/kg/day was generally well-tolerated. Vamorolone treatment has
been shown to preserve height trajectory and had a significantly
lower adverse impact on measures of bone health and behavior
changes compared to prednisone [3-6]. This unique benefit risk
profile over a wide dose range may represent a promising
therapeutic approach for the chronic therapy of DMD patients,
allowing for an individualized and optimized treatment regimen.
Efficacy data and recent safety analyses from
studies with vamorolone will be presented as follows:
World Muscle Society (WMS) 2021 Virtual Annual
Conference (September 20-24, 2021)
“Vamorolone versus
placebo and prednisone in Duchenne muscular dystrophy: Results from
a 24-week double-blind randomized trial” Late-breaking
poster (LBP.11/EP 524, September 23, 16:30-18:30 BST)
“Vamorolone
versus corticosteroid real-world
experience: Comparisons of
2-year
treatment period with NorthStar UK Network
and CINRG Duchenne natural history
study”Late-breaking poster (LBP.08/EP
521, September 23, 16:30-18:30 BST)
“2.5-years of vamorolone treatment in
Duchenne muscular dystrophy: Results of an open label long-term
extension”Oral presentation (O.3a, September 23,
14:00-15:00 BST) and poster presentation (EP.147, September 23,
16:30-18:30 BST)
The American Society for Bone and Mineral
Research (ASBMR) 2021 Annual Meeting (October 1-4, 2021)
“Vamorolone, a first-in-class
dissociative steroid, is associated with improved muscle strength
and favorable bone and growth plate profiles in young boys with
ambulatory Duchenne Muscular Dystrophy”Oral presentation
(LB-1113, October 4, 11:30-11:45 PT)
Vamorolone is being investigated as a
first-in-class dissociative steroid with lower incidence of
corticosteroid-associated adverse effects. The pivotal VISION-DMD
study met its primary endpoint of superiority in change of time to
stand from supine positioning to standing (TTSTAND) velocity with
vamorolone 6 mg/kg/day versus placebo (p=0.002) with a
treatment difference of 0.06 [95% CI: 0.02–0.10] rises/second from
baseline at 24 weeks. The study also demonstrated superiority of
both vamorolone dose levels (2 and 6 mg/kg/day) versus placebo
across multiple secondary endpoints. Importantly, vamorolone showed
a favorable safety and tolerability profile compared to prednisone.
Vamorolone did not stunt growth, as validated in the current
24-week study, in which vamorolone 6 mg/kg/day versus
prednisone 0.75 mg/kg/day showed a significant difference in
growth velocity (p=0.02). Furthermore, statistically significant
differences between vamorolone (2 and 6 mg/kg/day) and
prednisone groups were seen at week 24 in biomarkers assessing bone
health: osteocalcin, Procollagen 1 N-Terminal Propeptide (P1NP) and
Type I Collagen C-Telopeptides (CTX) (p<0.001 for vamorolone
both doses vs prednisone for all three parameters). On the basis of
the available data, vamorolone could emerge as a promising
alternative to existing corticosteroids, the current standard of
care in children and adolescent patients with DMD.
References:[1] ClinicalTrials.gov
Identifier: NCT03439670
[2] Press release
“Santhera and ReveraGen Announce Positive and Statistically Highly
Significant Topline Results with Vamorolone in Pivotal VISION-DMD
Study”, June 1, 2021,
link[3] Hoffman E.
Presented at virtual PPMD Annual Conference, June 22–26,
2021.[4] Mah JK, et
al. 2021. [Manuscript in
progress][5] Hoffman
E, et al. Neurology. 2019 Sep 24; 93(13):
e1312–e1323[6] Data
on File. ReveraGen Biopharma, Rockville, MD
About Vamorolone Vamorolone is
a first-in-class dissociative steroid which retains the
anti-inflammatory activity of corticosteroids while decreasing the
deleterious side effects. As such, vamorolone could emerge as a
promising alternative to existing corticosteroids, the current
standard of care in children and adolescent patients with DMD.
There is substantial unmet medical need in this patient group as
high-dose corticosteroids have significant systemic side effects
that diminish patient quality of life. 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.
About SantheraSanthera
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 has an exclusive license for
all indications worldwide to vamorolone, a first-in-class
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 clinical stage
pipeline also includes lonodelestat (POL6014) to treat cystic
fibrosis (CF) and other neutrophilic pulmonary diseases as well as
an exploratory gene therapy approach targeting congenital muscular
dystrophies. Santhera out-licensed rights to its first approved
product, Raxone® (idebenone), outside North America and 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
BioPharmaReveraGen 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, Duchenne Research
Fund, and Jesse’s Journey. 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
Prattelnpublic-relations@santhera.com orEva Kalias, Head External
CommunicationsPhone: +41 79 875 27 80eva.kalias@santhera.com
ReveraGen BioPharmaEric
Hoffman, PhD, President and CEO Phone: + 1
240-672-0295eric.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 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
these statements, particularly not in connection with any contract
or investment decision. The Company disclaims any obligation to
update these forward-looking statements.
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- 2021 09 23_WMS_ASBMR_e_final
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