Biophytis receives Orphan Drug Designation from the FDA for Sarconeos in
Duchenne Muscular Dystrophy (DMD)
Paris (France), May 15, 2018, 7.30 pm - BIOPHYTIS (Euronext Growth Paris:
ALBPS), a biotechnology company specialized in the development of drug
candidates to treat age-related degenerative diseases, announces that the FDA
(Food and drug administration) has granted Orphan Drug Designation (ODD) to its
drug candidate Sarconeos in Duchenne Muscular Dystrophy (DMD).
Stanislas Veillet, CEO of BIOPHYTIS, said: "The Orphan Drug Designation that
has just been granted by the FDA is a significant milestone for the development
of Sarconeos in DMD, a particularly severe pediatric indication. We hope to
obtain the ODD in Europe in the coming weeks. This will allow us to advance the
clinical development program in DMD, entitled MYODA. This program would consist
of two studies: a pharmacokinetic phase 1/2a MYODA-PK study, which could begin
in 2018, and an efficacy phase 2/3 MYODA-INT study, which could start in
The Orphan Drug Designation by the FDA will provide Sarconeos the following
benefits in the USA in DMD indication: a 7 years exclusivity after marketing
authorization, possible co-financing of clinical trials by the FDA, and the
possibility of a fast track registration procedure. Biophytis has also filed an
application for an Orphan Drug Designation for Sarconeos in DMD in Europe with
the EMA (European Medicines Agency). The Agency's response is expected in the
Sarconeos' proof-of-concept in the treatment of Duchenne Muscular Distrophy has
been presented at the World Muscle Society congress in 2017. It was
demonstrated, in the reference animal model of Duchenne Muscular Dystrophy,
that Sarconeos had significantly improved exercise tolerance and muscle
strength, and reduced muscle fibrosis. There are very few effective treatment
options and Sarconeos is a new class of drug candidate that has the potential
to significantly slow down the progression of the disease. It could be used as
a standalone treatment, or in combination with gene therapy when it will be
available for children with DMD.
MYODA is the name of drug candidate Sarconeos' new clinical development program
in Muscular Dystrophy or Duchenne myopathy (DMD). Sarconeos is a drug candidate
that activates the MAS receptor, stimulates muscle anabolism and reduces the
appearance of muscle fibrosis, with the potential to suspend the disease's
progression, particularly to delay the loss of mobility. The clinical
development program will include a phase I/II pharmacokinetic study (MYODA-PK),
which is expected to begin in 2018, and a phase II/III study (MYODA-INT), which
is expected to start in 2019.
About Duchenne muscular dystrophy:
Duchenne muscular dystrophy (DMD), is an X-linked inherited muscular disease,
which concerns 1 in 3,500 male births, and characterized by progressive muscle
weakness and cardiomyopathy, leading to premature death. Muscles undergo
repeated cycles of necrosis/regeneration and are replaced by connective and
adipose tissues. Glucocorticoids and supportive therapy are the current
standard of care leaving many patients with an unmet medical need.
Sarconeos is a first-in-class drug candidate based on the activation of the MAS
receptor (major player of the renin-angiotensin system) restoring muscular
anabolism, inhibiting myostatin, and that had demonstrated meaningful activity
in animal models of muscular dystrophies. Sarconeos is developed in the
treatment of sarcopenia, an age-related degeneration of skeletal muscle,
leading to loss of mobility in elderly people. This condition, for which no
medical treatment currently exists, was first described in 1993 and has entered
the International Classification of Diseases (M62.84) in 2016. It affects more
than 50 million people worldwide.
Biophytis SA (www.biophytis.com), founded in 2006, develops drug candidates
targeting diseases of aging. Using its technology and know-how, Biophytis has
begun clinical development of innovative therapeutics to restore the muscular
and visual functions in diseases with significant unmet medical needs.
Specifically, the company is advancing two lead products into mid-stage
clinical testing this year: Sarconeos (BIO101) to treat sarcopenic obesity and
Macuneos (BIO201) to treat dry age-related macular degeneration (AMD).
The business model of BIOPHYTIS is to ensure the conduct of the project until
clinical activity in the patient is proven, then to license the technologies in
order to continue the development in partnership with a pharmaceutical
Based on the Sorbonne Université campus, Biophytis collaborates with expert
scientists from several Sorbonne Université institutes such as the Paris Seine
Biology Institute, the Institute of Myology, and the Vision Institute.
BIOPHYTIS is listed on the Euronext Growth market of Euronext Paris
(ALBPS; ISIN: FR0012816825).
For more information: http://www.biophytis.com
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