Duchenne Muscular Dystrophy (DMD) is a rare, genetic neuromuscular disease in male children characterized by accelerated degeneration of muscles, responsible for loss of mobility, respiratory failure and cardiomyopathy, leading to premature death. There is no known cure and limited treatment options.


Biophytis was granted FDA IND approval in December 2019 to initiate a Phase 1/2 clinical proof of concept trial (MYODA-INT) of an oral pediatric formulation of Sarconeos (BIO101), utilizing a ‘seamless’ clinical trial design with composite clinical endpoints.

We intend to use a ‘seamless’ clinical trial design that will encompass all three phases of clinical development (Phase 1 to Phase 3), allowing patients to participate in one or more phases, with composite clinical endpoints comprised of key functional measurements, including mobility, strength and respiratory function. The MYODA clinical trial aims to encompass two distinct stages: (i) a Phase 1/2 clinical proof of concept stage (MYODA-INT), followed by (ii) Phase 3 confirmatory stage.


The global, double-blind, randomized, placebo-controlled MYODA-INT Phase 1-2-3 clinical trial will assess safety and efficacy of two doses of Sarconeos (BIO101) in non-ambulatory DMD young patients, administered orally with a meal over 26 weeks, as compared to placebo. The first week of dosing will be mainly for assessment of safety, tolerability, and pharmacokinetic (PK) objectives in 18 patients (Phase 1). Dosing will continue seamlessly into Phase 2, provided that no serious safety issues arise during the first seven days of the treatment. An interim analysis will take place after all 48 subjects have finished 12 weeks of dosing, and thereafter for every 12-week period until either success or futility is achieved. Upon completion of the MYODA-INT stage of the seamless trial (up to 52 weeks), participants may be enrolled in an open-label extension period. Regulatory approval will be required before progressing into and commencing the confirmatory stage, expected to recruit up to 200 patients and measure respiratory function after dosing for 52 weeks (Phase 3).