Biophytis SA announced the publication of promising results from its Phase II SARA-INT clinical trial for BIO101, a potential first-in-class treatment for sarcopenia, in the prestigious Journal of Cachexia, Sarcopenia and Muscle (JCSM). The clinical-stage biotechnology company is now preparing to advance to Phase III trials, positioning itself as the leader in addressing this significant unmet medical need.
Significant Clinical Improvements Demonstrated
The SARA-INT trial showed that BIO101 at a dose of 350mg twice daily demonstrated clinically meaningful improvement in the 400-meter walk test (400MWT), which served as the primary endpoint of the study. The drug candidate exhibited an excellent safety profile across all tested doses, with no serious adverse events related to the product.
Notably, the treatment showed nominally significant effects versus placebo in specific high-risk subpopulations, including slow walkers and patients with sarcopenic obesity. These results are particularly important as these groups often experience more severe functional limitations and health consequences.
"These results represent a world premiere in the field of sarcopenia treatment," said a Biophytis representative. "The publication in JCSM, the key reference journal for sarcopenia research, validates the scientific rigor of our approach and the potential clinical significance of BIO101."
Addressing a Major Global Health Challenge
Sarcopenia, characterized by age-related loss of muscle mass and function, affects between 121 and 194 million people worldwide. This prevalence is expected to increase dramatically in coming decades as the global population ages. Currently, no approved therapeutic solutions exist for treating this condition, which significantly impacts mobility, increases fall risk, and reduces quality of life in older adults.
The condition is associated with substantial healthcare costs and contributes to increased hospitalization rates and loss of independence among affected individuals. BIO101's potential to improve physical function could represent a significant advancement in geriatric medicine.
Mechanism of Action and Development Program
BIO101 (20-hydroxyecdysone) is a small molecule that appears to target the biology of muscle aging. While the company has not disclosed the complete mechanism of action in these announcements, previous research suggests it may activate pathways involved in muscle protein synthesis and mitochondrial function.
The drug is being developed as an oral therapy, which could offer advantages in terms of patient compliance and ease of administration compared to other approaches such as physical therapy interventions alone.
Regulatory Progress and Partnership Opportunities
Biophytis reports making significant progress in obtaining regulatory approvals for its planned Phase III trial in sarcopenia. The company is simultaneously accelerating discussions with potential pharmaceutical partners, including a major international pharmaceutical company in China and other industrial partners across Asia.
These partnerships could provide Biophytis with additional resources and expertise to support the Phase III program and eventual commercialization efforts. The company's leadership position in sarcopenia treatment development makes it an attractive partner for pharmaceutical companies looking to expand their age-related disease portfolios.
Broader Pipeline Development
Beyond sarcopenia, Biophytis is developing BIO101 for other indications including Duchenne muscular dystrophy (planning to start Phase 1-2), respiratory diseases (completed Phase 2-3 for COVID-19), and metabolic disorders (Phase 2 for obesity to be initiated).
This multi-indication strategy leverages the company's understanding of BIO101's mechanism of action across different age-related and metabolic conditions, potentially creating multiple paths to market and diversifying development risk.
Company Background
Biophytis SA is headquartered in Paris, France, with subsidiaries in Cambridge, Massachusetts, USA, and Brazil. The company's ordinary shares are listed on Euronext Growth Paris (ALBPS), and its American Depositary Shares are listed on the OTC market (BPTSY).
With a current market capitalization of approximately €4.44 million, the company has experienced a year-to-date price performance of -16.29%. Despite this market performance, the advancement of BIO101 into Phase III for sarcopenia represents a significant milestone that could potentially transform the company's prospects.
Looking Forward to Phase III
As Biophytis prepares for the pivotal Phase III program, the company will need to confirm the optimal dosing regimen, refine patient selection criteria based on Phase II subgroup analyses, and design a trial with sufficient statistical power to demonstrate efficacy for regulatory approval.
The Phase III program will likely need to demonstrate durable improvements in physical function and potentially show benefits in secondary endpoints such as muscle mass, strength, and quality of life measures to support a comprehensive label for clinical use.
If successful, BIO101 could become the first approved pharmacological treatment for sarcopenia, addressing a significant unmet need in the aging global population.
