BIOPHYTIS (EPA:ALBPS) - Biophytis files for Orphan Drug Designation for Sarconeos in Duchenne Muscular Dystrophy (DMD)

Transparency directive : regulatory news

20/03/2018 18:58

Press release

Biophytis files for Orphan Drug Designation for Sarconeos in Duchenne Muscular
                                Dystrophy (DMD)

Presentation of MYODA: a new clinical program in the treatment of Duchenne
                              Muscular Dystrophy

Paris (France), March 20, 2018, 6.45pm - BIOPHYTIS (Euronext Growth Paris:
ALBPS), a biotechnology company specializing in the development of drug
candidates to treat age-related degenerative diseases, announces that it has
filed for orphan drug designation with the European Medicines Agency (EMA) and
presents MYODA, the new clinical program of Sarconeos in Duchenne Muscular
Dystrophy (DMD).

Stanislas Veillet, CEO of BIOPHYTIS, comments: "The filing for orphan drug
designation with the EMA, and soon with the FDA, marks an important step in the
development of Sarconeos in Duchenne Myopathy. The proofs of concept in
animals, presented in 2017 at the World Muscle Society Congress, as well as the
discussions with clinicians and patients' associations, prompted us to
investigate the potential of Sarconeos in the treatment of this particularly
severe orphan paediatric indication. We were able to specify the main elements
of Sarconeos' clinical development plan in this muscular dystrophy, which will
consist of two studies: a pharmacokinetic phase I/II MYODA-PK study, which is
expected to begin in 2018 and an efficacy phase II/III MYODA-INT study, which
could start in 2019. While there are very few effective treatment options,
Sarconeos is a new class of drug candidate with the potential to significantly
slow down the evolution of the disease and could be used as a standalone
treatment or in combination with gene therapy when it will be made available
for children with Duchenne Myopathy."

* The Orphan Drug Designation for Sarconeos in Duchenne Myopathy

Biophytis has filed an application for an orphan drug designation for Sarconeos
in Duchenne myopathy with the EMA and will file the same application with the
FDA in the coming days. The agencies' answer is expected in the coming months
and could give Sarconeos the benefits associated with the orphan drug status,
in particular an accelerated registration procedure, and an 8 or 10 years
protection after marketing authorization.
This application follows the proof-of-concept studies presented at the World
Muscle Society congress in 2017, which demonstrated in the reference animal
model of Duchenne Muscular Dystrophy, that Sarconeos had significantly improved
exercise tolerance and muscle strength, and reduced muscle fibrosis. It is the
first regulatory step to obtain authorizations to develop Sarconeos in Duchenne
Muscular Dystrophy, in parallel with its development in Sarcopenia. It marks
the implementation of a dual development strategy of Biophytis' drug
candidates, not only in chronic geriatric diseases, but also in genetic
degenerative diseases.

* Sarconeos' clinical development plan in Duchenne myopathy

Sarconeos' clinical development plan in Duchenne myopathy consists of two main
clinical studies: a pharmacokinetic phase I/II MYODA-PK study to be initiated
in 2018, and a phase II/III efficacy study, MYODA-INT, that could start in
2019. While there are very few effective treatment options, Sarconeos is a new
class of drug candidate that has the potential to significantly delay
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
Duchenne myopathy.

MYODA-PK
The MYODA-PK phase I/II study aims at validating Sarconeos' safety as well as
its pharmacokinetics and pharmacodynamic profile in paediatric patients with
Duchenne myopathy. For a duration of 4 weeks, the double-blind
placebo-controlled study will be conducted in approximately 24 boys with
Duchenne myopathy aged 2 to 18 years. It will be carried out in 2 phases:
single-ascending dose (SAD) and multiple-ascending dose (MAD). Collected data
will be used to assess dosage for the phase II/III clinical trial. MYODA-PK
will take place in several paediatric investigation centers in Europe.

MYODA-INT

Multicentric international phase II/III MYODA-INT study will evaluate the
safety and efficacy of a Sarconeos dose in about 60 ambulant boys with Duchenne
myopathy. Of a minimal treatment duration of 6 months, the study will be
randomized, double-blind, placebo-controlled. The primary endpoint will be the
North Star Ambulatory Assessment (NSAA), used to measure functional motor
abilities in ambulant children with DMD. Conducted in Europe and the United
States, the results of this study should allow to file for a conditional
marketing authorization.

                                   ****

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.
The company was founded in partnership with researchers at the UPMC (Pierre and
Marie Curie University) and also collaborates with scientists at 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 41 83 66 00

Citigate Dewe Rogerson
International media & Investors
Laurence BAULT/Antoine DENRY
Laurence.bault@citigatedewerogerson.com
antoine.denry@citigatedewerogerson.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 files for Orphan Drug Designation for Sarconeos in Duchenne Muscular Dystrophy (DMD)

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