BIOPHYTIS (EPA:ALBPS) - Biophytis receives favourable opinion from EMA for the Orphan Drug designation of Sarconeos in Duchenne Muscular Dystrophy (DMD)

Transparency directive : regulatory news

28/05/2018 18:07

Press release

Biophytis receives favourable opinion from EMA for the Orphan Drug designation
of Sarconeos in Duchenne Muscular Dystrophy (DMD)

Paris (France), May 28, 2018, 6pm - BIOPHYTIS (Euronext Growth Paris: ALBPS), a
biotechnology company specialized in the development of drug candidates to
treat age-related degenerative diseases, announces that the EMA (European
Medicines Agency) has given a favorable opinion for Sarconeos to be designated
as an Orphan Drug, its drug candidate in Muscular Dystrophy or Duchenne
Muscular Dystrophy (DMD).

Stanislas Veillet, CEO of BIOPHYTIS, said: "The positive opinion given by the
EMA (which follows the favourable opinion of the FDA) is a significant
regulatory step for the progress of this project, in order to provide a
therapeutic response to patients with Duchenne muscular dystrophy. The next
step is the submission of the Pediatric Investigation Plan, for scientific
advice, to the EMA, and the finalization of the clinical development program.
Consisting of two studies, this program includes a phase 1/2a MYODA-PK
pharmacokinetic study which could begin in 2018 and a phase 2/3 MYODA-INT
efficacy study which could start in 2019."

Duchenne muscular dystrophy (DMD) is an orphan pediatric disease linked to a
genetic abnormality. The young patients affected by this dystrophy see their
muscles become damaged at each contraction, going as far as their total
destruction. The respiratory, cardiac and digestive muscles in particular are
affected in a progressive and irreversible way, thus affecting very
significantly the life expectancy of the patients.

In the reference animal model, Sarconeos significantly improved exercise
tolerance and muscle strength, and reduced fibrosis. While there are currently
few effective treatment options, Sarconeos 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.

The Committee for Orphan Medicinal Products (COMP) has given a favourable
opinion that Sarconeos be designated an orphan drug in the treatment of
Duchenne muscular dystrophy. This opinion is sent to the European Commission
for approval, validating the orphan drug status of Sarconeos. This status will
allow Biophytis to benefit from numerous incentives for the development of its
drug candidate, including: assistance in the development of protocols,
allocation of credits for the development of the drug candidate, access to a
centralized marketing authorization procedure in Europe, and a 10-year
marketing exclusivity in the European Community.

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 favourable opinion from EMA for the Orphan Drug designation of Sarconeos in Duchenne Muscular Dystrophy (DMD)

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