BIOPHYTIS (EPA:ALBPS) - Biophytis receives Orphan Drug Designation from the FDA for Sarconeos in Duchenne Muscular Dystrophy (DMD)

Transparency directive : regulatory news

15/05/2018 20:28

Press release

   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
2019."

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
coming weeks.

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.

About MYODA:
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.

About SARCONEOS:
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.

About BIOPHYTIS
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
laboratory.
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

Follow us on Twitter @biophytis

BIOPHYTIS is eligible for the SMEs scheme

Disclaimer
This press release contains certain forward-looking statements. Although the
Company believes its expectations are based on reasonable assumptions, these
forward-looking statements are subject to numerous risks and uncertainties,
which could cause actual results to differ materially from those anticipated.
For a discussion of risks and uncertainties which could cause the Company's
actual results, financial condition, performance or achievements to differ from
those contained in the forward looking statements, please refer to the Risk
Factors ("Facteurs de Risque") section of the Listing Prospectus upon the
admission of Company's shares for trading on the regulated market Euronext
Growth of Euronext Paris filed with the AMF, which is available on the AMF
website (www.amf- france.org) or on BIOPHYTIS' website (www.biophytis.com).

This press release and the information contained herein do not constitute an
offer to sell or a solicitation of an offer to buy or subscribe to shares in
BIOPHYTIS in any country. Items in this press release may contain
forward-looking statements involving risks and uncertainties. The Company's
actual results could differ substantially from those anticipated in these
statements owing to various risk factors which are described in the Company's
prospectus. This press release has been prepared in both French and English. In
the event of any differences between the two texts, the French language version
shall prevail.

BIOPHYTIS
Stanislas VEILLET
CEO
contact@biophytis.com
Tel: +33 (0) 1 44 27 23 00

Citigate Dewe Rogerson
International media & Investors
Laurence BAULT/Antoine DENRY
Laurence.bault@citigatedewerogerson.com
antoine.denry@citigatedewerogreson.com
Tel: +33 (0)1 53 32 84 78
Mob: +33(0)6 64 12 53 61

LifeSci Advisors
Chris MAGGOS
Managing Director, Europe
chris@lifesciadvisors.com
Tel: +41 79 367 6254

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BIOPHYTIS (EPA:ALBPS) - Biophytis receives Orphan Drug Designation from the FDA for Sarconeos in Duchenne Muscular Dystrophy (DMD)

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