BIOPHYTIS Receives Orphan Drug Designation in the United States for Sarconeos in Duchenne Muscular Dystrophy (DMD)
May. 15, 2018
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.
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 BIOPHYTISBIOPHYTIS 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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Citigate Dewe Rogerson BIOPHYTIS International media & Investors LifeSci Advisors Stanislas VEILLET Laurence BAULT/Antoine DENRY Chris MAGGOS CEO Laurence.firstname.lastname@example.org Managing Director, Europe email@example.com firstname.lastname@example.org Tel: +33 (0) 1 44 27 23 00 email@example.com Tel: +41 79 367 6254 Tel: +33 (0)1 53 32 84 78 Mob: +33(0)6 64 12 53 61