DUCHENNE MUSCULAR DYSTROPHY
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.
MYODA CLINICAL PROGRAM
Biophytis was granted IND approval in December 2019 from the FDA for the clinical development of Ruvembri using an oral pediatric formulation for the Duchenne Muscular Dystrophy. Due to the COVID-19 crisis and its consequences, the MYODA study has been postponed and the protocol has been thoroughly revised.
We intend to use a ‘seamless’ clinical trial design that will encompass two phases of clinical development (Phases 1 2), with key functional measurements such as the respiratory function, strength and parameters related to Quality of Life. The MYODA clinical trial aims to encompass two distinct stages: (i) a phase of dose finding (Part 1), followed by (ii) a clinical proof of concept stage aiming at showing the efficacy of Ruvembri (Part 2).
Biophytis will propose this Phase 1-2 study for non-ambulatory DMD patients with signs of deteriorating lung function to competent authorities in order to start the clinical study in 2024.