ReveraGen and Santhera Announce FDA Orphan Grant Funding for
Clinical Trial with Vamorolone in Becker Muscular Dystrophy
Rockville, MD,
USA, and Pratteln, Switzerland,
September 27,
2021 – ReveraGen
Biopharma and Santhera Pharmaceuticals (SIX:
SANN) announce that ReveraGen has
received a USD 1.2
million grant from the FDA under their
“Clinical Studies of Orphan Products Addressing Unmet Needs of Rare
Diseases (R01)” grants program. The grant
adds to existing grants from the National Institutes of
Health, NIAMS, and the Foundation to Eradicate Duchenne to initiate
a clinical trial of vamorolone in adults and
children with Becker muscular dystrophy, a
progressive muscle wasting disease similar to
Duchenne muscular dystrophy, but usually
milder.
Vamorolone, a dissociative steroid drug, that
has shown retention of efficacy and reduction of safety concerns
typically associated with corticosteroids in Duchenne muscular
dystrophy (DMD) will now be tested in a 24-week clinical
exploratory trial in Becker muscular dystrophy (BMD). BMD is caused
by mutations of the DMD gene but shows residual dystrophin protein
in muscle, and variable onset and progression of muscle weakness.
The double-blind trial will test efficacy and safety of daily
vamorolone on motor outcomes and established biomarker outcomes,
with participants randomized 2:1 vamorolone or placebo. The
clinical trial plans to enroll at sites in Padova (Italy) and
Pittsburgh (USA).
“There are currently no approved drugs for BMD
in any country, and there is a high unmet need,” said Paula
Clemens, MD, Professor of Neurology
at the University of Pittsburgh School of
Medicine, and co-principal investigator on the FDA, NIH
and Foundation awards.
The mechanisms of actions, providing basis for
vamorolone efficacy as demonstrated in the pivotal VISION-DMD study
in the more severe DMD, are felt to be highly relevant to BMD too.
In addition, vamorolone is hypothesized to increase dystrophin
protein levels in BMD via inhibition of microRNAs that
deleteriously target dystrophin, and this may further complement
the mechanism of action specifically in BMD [1-3].
“While the drug development pipeline has greatly
expanded for DMD in recent years, there are very few clinical
investigational efforts underway for BMD,” said Elena
Pegoraro,
MD, PhD, Professor of
Neurology at the University of Padova in
Italy. “Corticosteroids are often not tolerated by
patients with BMD due to their side effects. Therefore, the
lessened side effect burden of vamorolone seen in DMD trials may
prove important to the underserved BMD patient community,” she
continued.
“The Foundation to Eradicate Duchenne is pleased
to provide support of the vamorolone program to include BMD
patients,” said Joel Wood, President of Foundation to
Eradicate Duchenne.
Vamorolone was discovered by US-based ReveraGen
BioPharma, Inc. and is being developed in collaboration with
Santhera who owns worldwide rights to the drug candidate for all
indications.
About Vamorolone Vamorolone is
a first-in-class drug candidate that binds to the same receptor as
corticosteroids but modifies its downstream activity and as such is
a dissociative partial agonist [4-6]. This mechanism has the
potential to ‘dissociate’ efficacy from typical steroid safety
concerns and therefore 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 many patient groups where
corticosteroids are standard of care or hold therapeutic promise,
but side effects limit prescription and patient adherence. US NDA
submission for DMD is anticipated in Q1-2022. 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.
References:[1]
Hoffman EP. Causes
of clinical variability in Duchenne and Becker muscular dystrophies
and implications for exon skipping therapies. Acta Myol. 2020 Dec
1;39(4):179-186. doi: 10.36185/2532-1900-020. PMID: 33458572;
PMCID: PMC7783439.[2]
Kinder TB, Heier
CR, Tully CB, Van der Muelen JH, Hoffman EP, Nagaraju K, Fiorillo
AA. Muscle Weakness in Myositis: MicroRNA-Mediated Dystrophin
Reduction in a Myositis Mouse Model and Human Muscle Biopsies.
Arthritis Rheumatol. 2020 Jul;72(7):1170-1183. doi:
10.1002/art.41215. Epub 2020 May 31. PMID: 32009304; PMCID:
PMC7384101.[3] Fiorillo
AA, Heier CR, Novak JS, Tully CB, Brown KJ, Uaesoontrachoon K, Vila
MC, Ngheim PP, Bello L, Kornegay JN, Angelini C, Partridge TA,
Nagaraju K, Hoffman EP. TNF-α-Induced microRNAs Control Dystrophin
Expression in Becker Muscular Dystrophy. Cell Rep. 2015 Sep
8;12(10):1678-90. doi: 10.1016/j.celrep.2015.07.066. Epub 2015 Aug
28. PMID: 26321630; PMCID: PMC4757433.
[6] Heier CR at al.
(2013). EMBO Mol Med 5:
1569–1585.[7] Reeves
EKM, et al (2013). Bioorg Med Chem
21(8):2241-2249.[8] 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 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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