Biophytis has received authorization from the FDA to launch SARA-31, the first-ever Phase 3 clinical trial in sarcopenia, marking a significant milestone in addressing an age-related condition that affects 10-16% of elderly populations globally. The authorization complements a positive opinion obtained from Belgian authorities earlier this year, positioning the company to advance treatment for a condition that currently lacks effective pharmaceutical interventions.
Study Design and Patient Population
The SARA-31 study will evaluate the efficacy and safety of Sarconeos (BIO101) in approximately 900 patients over 65 years of age with severe sarcopenia. Eligible participants must have a Short Physical Performance Battery (SPPB) score between 3 and 7, low walking speed (4-meter walking speed ≤ 0.8 m/s), and reduced hand grip strength (less than 20kg for women and less than 35.5kg for men).
Patients will receive treatment for a minimum of 12 months and a maximum of 36 months, receiving either placebo or 350mg of Sarconeos twice daily. The primary efficacy parameter will measure the time to onset of Major Mobility Disability (MMD), defined as the inability to walk 400 meters in less than 15 minutes without sitting, assistance from another person, or use of a walker.
Clinical Development Foundation
The Phase 3 authorization follows promising results from a Phase IIb study where patients receiving Sarconeos at the highest dose (350mg twice daily) showed meaningful improvement in the 400-meter walk test, meeting the study's primary endpoint. These results, combined with discussions with health authorities in 2022, provided the foundation for advancing to Phase 3 development.
Roger A. Fielding, PhD, a sarcopenia expert and laboratory director at Tufts University in Boston, will serve as the principal investigator for the SARA-31 study, continuing his contribution to the Sarconeos clinical development program.
Regulatory Pathway and Timeline
The FDA authorization follows the company's submission to the European Medicines Agency (EMA) in mid-May, with Biophytis expecting a response during the third quarter of 2023. However, the company still requires authorization from ethics committees in participating countries before study initiation.
The effective start of the study is scheduled for 2024, contingent upon finalizing partnership agreements and securing adequate financial resources. Stanislas Veillet, Chief Executive Officer of Biophytis, noted that "FDA approval suggests authorities may be increasingly aware of the growing need for effective therapeutics against a major disease in an aging society."
Market Context and Unmet Need
According to GlobalData's Pharmaceutical Intelligence Centre, no competing drugs have been marketed or are currently in Phase 3 trials for sarcopenia, with only six Phase II and two Phase I competing drugs in development. This positions Biophytis as a pioneer in the field, with the potential to be the first company to launch a Phase 3 clinical development program in this indication.
The primary treatment for sarcopenia currently consists of exercise and resistance or strength training, highlighting the significant unmet medical need for pharmaceutical interventions. Veillet emphasized the company's pioneering role, stating they "intend to be the first company to launch, in partnership with global or regional pharmaceutical companies, a Phase III clinical development program in this indication."
Drug Mechanism and Development Pipeline
Sarconeos (BIO101) is a small molecule drug candidate classified as a phytoecdysone that targets the Mas-related G protein-coupled receptor. The compound was developed based on Biophytis' SARCOB platform and is also being investigated for Duchenne muscular dystrophy and acute respiratory distress syndrome (ARDS) associated with COVID-19 pneumonia and COVID-19-induced respiratory failure.
Secondary endpoints in the SARA-31 study will include walking speed measured by the 4-meter walking speed from the SPPB test, hand grip strength, and patient-reported quality of life using the SarQol questionnaire, which was specifically developed for sarcopenia assessment.
