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BIOPHYTIS Receives Orphan Drug Designation in the United States for Sarconeos in Duchenne Muscular Dystrophy (DMD)
PARIS, May 15, 2018 (GLOBE NEWSWIRE) -- BIOPHYTIS (Euronext Growth Paris:ALBPS), a biotechnology company specializing 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 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 our team and the development of Sarconeos in Duchenne Myopathy, a particularly severe pediatric indication. We hope to obtain this designation in Europe as well in the coming weeks. We will then be able to conduct the MYODA clinical program. This program will 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 grant Sarconeos with the benefits associated with the status, in particular, a 7 years protection after marketing authorization, possible co-financing of clinical trials with the FDA and the possibility of an accelerated registration procedure. BIOPHYTIS has also filed an application for an orphan drug designation for Sarconeos in Duchenne myopathy 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 Dystrophy 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. Orphan drug designation is a 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 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. There are very few effective treatment options and 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 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 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
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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.
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