DUCHENNE MUSCULAR DYSTROPHY: MYODA CLINICAL PROGRAMME
1. A rare genetic disease with limited treatment options
Duchenne muscular dystrophy (DMD) is a rare genetic disease affecting 1 in 5,000 boys at birth, or 220,000 people worldwide. It is characterised by accelerated muscle degeneration, leading to loss of mobility, respiratory failure and cardiomyopathy, with premature death generally between the ages of 25 and 30.
Despite advances in research over the last few years, no treatment has been able to cure or effectively control the progression of the disease. The solutions currently used, whether genetic therapies or corticosteroids, are controversial and fail to halt the development of this neuromuscular disease. When the disease reaches its most advanced stage, respiratory assistance is the only recommended solution, but it considerably impairs patients’ quality of life.
2. MYODA clinical programme aims to develop a first-in-class drug
Biophytis has received IND (Investigational New Drug) approval from the U.S. Food and Drug Administration (FDA) in December 2019 for the clinical development of BIO101 (20-hydroxyecdysone) in a pediatric formulation for Duchenne muscular dystrophy. BIO101 (20-hydroxyecdysone) aims to improve the respiratory capacity of patients in the advanced (non-ambulatory) stages of the disease. The positive effects of BIO101 have been demonstrated in animal models, leading to an improvement in respiratory capacity.
3. An accelerated development plan
BIO101 already has orphan drug designation in Europe and the United States in this indication, which means that clinical development and time to market authorisation can be accelerated.
Over the last few months the protocol has been refined for the regulatory agencies (FDA, EMA). Biophytis now aims to start a phase 1-2 clinical trial in non-ambulatory DMD patients suffering from respiratory failure. The aim of this clinical trial will be to assess the pharmacokinetics, safety and clinical efficacy of BIO101 in this indication. Biophytis is looking for partners to support this development.