Biophytis has decided to concentrate its research efforts on the degeneration of the muscle and the degeneration of the retina, pathologies for which the selected molecules seem to have the most potential. Sarcopenia (age-related muscular dystrophy) and AMD (age-related macular degeneration) have been given priority as the major causes of disability in the over-65 age group and no treatment is currently available for them. Since 2017, Biophytis has also invested in adapting its programmes to genetic degenerative diseases: Duchenne muscular dystrophy and Stargardt disease.
SARCONEOS in Sarcopenia : SARA clinical program
Sarconeos is the first representative of a new class of drug candidates formulated to activate the MAS receptor (which plays a major role in the renin–angiotensin system), stimulate muscle anabolism, inhibit myostatin, and promote the development of muscle mass in animal models of muscular dystrophies. Sarconeos is being developed for the treatment of Sarcopenia, an age-related muscular dystrophy characterized by a loss of muscle mass and strength, leading to a loss of mobility among seniors. This new pathological condition, for which no drug treatment is available, was first described in 1993 and was recently listed under code M62.84 in ICD-10-CM, (International Classification of Diseases, Tenth Revision, Clinical Modification, based on the WHO classification used in the United States for patient follow-up). It affects up to 10% of the elderly persons in the world.
The SARA program is a multi-centre clinical program carried out in 4 countries (USA, France, Belgium and Italy) and aimed at determining the effective therapeutic dose of Sarconeos in a Phase 2b clinical study performed on 334 patients with sarcopenia. The program includes a six-month observational study (SARA-OBS) performed on patients with sarcopenia in 11 clinical centres. After obtaining their consent, patients who participated in SARA-OBS may enter in the SARA-INT phase 2b study. The objective of SARA-INT is to assess the safety and efficacy of two doses of Sarconeos (175 mg bid and 350 mg bid) administered orally for 26 weeks vs a placebo in a population of men and women over the age of 65 at risk of motor disability, and to estimate the effect of the treatment as concerns the improvement of the physical function and the reduction of the risk of motor disability.
SARCONEOS in DUCHENNE MUSCULAR DYSTROPHY : MYODA clinical program
Sarconeos has obtained Orphan Drug Disease (ODD) designation from the FDA (Food and Drug Administration) and the EMA (European Medicines Agency), to treat Duchenne Muscular Dystrophy (DMD). As there are very few drug treatments available, Sarconeos is a drug candidate that has the potential to significantly slow the progression of the disease and could be used alone or in combination with genetic therapy, when the latter is available, in children with DMD. The status of ODD or orphan medication could give Sarconeos the advantages associated with orphan drug status, in particular the possibility of early registration (on the basis of a conditional authorization) and protection for 7 to10 years after receiving the marketing authorization.
Sarconeos’ clinical development plan for Duchenne muscular dystrophy consists of two main clinical studies: a phase 1/2 pharmacokinetic study (MYODA-PK) expected to begin in 2019, and a phase 2/3 efficacy study (MYODA-INT) that could start in 2020.
Macuneos in AGE-RELATED MACULAR DEGENERATION : MACA clinical program
Macuneos is the first representative of a new class of drug candidates intended as PPAR (nuclear receptor) agonists. Macuneos protects the retinal pigment epithelium. Biophytis has demonstrated in animal models that Macuneos protects the cells of the retina from the phototoxic effects of A2E in the presence of blue light (photo-oxidative stress), reduces the accumulation of this phototoxic molecule and thus slows down the retinal degeneration process. Macuneos is a drug candidate for dry AMD in its intermediate stage. AMD affects the central part of the retina, known as the macula, leading to serious visual impairment and irreversible loss of central vision after the age of 60. Macuneos comes in capsule form (taken once a day).
The MACA program is a multi-centre clinical program carried out in several countries (USA, Europe) and aimed at determining the effective therapeutic dose of Macuneos to slow the progression of the disease in patients with AMD in a Phase 2b clinical study.
The first study, MACA-PK, set to begin in 2019, a pharmacokinetic and safety study on healthy volunteers and elderly patients with intermediate AMD.
The first patient has been incorporated into the study in May 2018.