Biophytis SA American Depositary Share (0.01 Euro) Stock Price, News & Analysis

Biophytis is a clinical-stage biotechnology company focused on developing therapeutics for age-related diseases and neuromuscular disorders. The company originated as a spin-off from Université Pierre et Marie Curie in Paris and maintains dual headquarters in Paris, France and Cambridge, Massachusetts. Biophytis trades on the OTC Pink market in the United States and maintains its primary listing on Euronext Growth Paris.

Important Trading Information: Biophytis American Depositary Shares were delisted from NASDAQ in April 2024 due to non-compliance with stockholders' equity requirements. The shares now trade on the OTC Pink market under the symbol BPTS. As an OTC Pink stock, Biophytis has limited reporting requirements compared to major exchange-listed companies, which may result in less publicly available information and reduced liquidity. Investors should be aware of the increased risks associated with OTC Pink securities.

Lead Drug Candidate: BIO101

The cornerstone of Biophytis' pipeline is BIO101, also known by the brand names Sarconeos and Ruvembri. BIO101 is a pharmaceutical-grade formulation of 20-hydroxyecdysone, a polyhydroxylated plant steroid that activates the MAS receptor. This receptor plays a critical role in the protective arm of the renin-angiotensin system, a biological pathway involved in regulating protein synthesis and muscle metabolism.

BIO101 represents the first oral daily MAS receptor activator to enter clinical development. The compound works by enhancing protein synthesis in muscle tissue while potentially reducing muscle degradation, making it particularly relevant for conditions characterized by muscle wasting and weakness. The oral formulation offers convenient administration compared to injectable alternatives, which could improve patient compliance if the drug reaches market approval.

Sarcopenia Program

Sarcopenia, the progressive loss of skeletal muscle mass and function associated with aging, represents one of Biophytis' primary therapeutic targets. This condition affects a substantial portion of the elderly population and is associated with increased risk of falls, fractures, disability, and mortality. Despite the clinical significance of sarcopenia, no pharmacological treatments have been approved specifically for this indication, with current recommendations focusing primarily on nutrition and exercise interventions.

Biophytis completed a Phase 2 clinical trial known as SARA-INT, which evaluated BIO101 in sarcopenic patients at risk of mobility disability. The trial used the 400-meter walk test as its primary endpoint, a validated measure of functional capacity in elderly populations. Results from this study showed clinically meaningful improvements in walking speed at the highest dose tested, accompanied by a favorable safety profile with no serious adverse events attributed to the treatment.

Building on these Phase 2 results, Biophytis has outlined plans for a Phase 3 clinical trial targeting age-related sarcopenia. This would represent the first Phase 3 trial ever conducted specifically for sarcopenia treatment. The planned study aims to recruit patients primarily in Asia and Europe, regions selected for both clinical need and commercial potential. The company has secured financing arrangements with strategic partners and investors to support the advancement of this pivotal trial, with clinical operations expected to commence in the coming years.

Duchenne Muscular Dystrophy

Biophytis is developing BIO101 for Duchenne Muscular Dystrophy, a rare genetic neuromuscular disease that primarily affects male children. DMD is characterized by accelerated muscle degeneration leading to progressive loss of mobility, respiratory complications, and cardiomyopathy. The disease is caused by mutations in the dystrophin gene and represents the most common form of muscular dystrophy in children.

BIO101 has received orphan drug designation from both the U.S. Food and Drug Administration and the European Medicines Agency for the treatment of DMD. These designations provide regulatory and commercial incentives for developing therapies for rare diseases, including extended market exclusivity periods and potential fee reductions. Preclinical studies in mdx mouse models of DMD demonstrated positive effects on muscle function, mobility, and respiratory function, key aspects of disease progression in DMD patients.

The company is developing a pediatric formulation of BIO101 specifically for DMD patients, recognizing the need for age-appropriate dosage forms in this patient population. The DMD program is currently in early clinical development phases, with the company working to advance the compound toward efficacy studies in affected patients.

Obesity and Muscle Preservation

Biophytis has initiated a clinical development program exploring BIO101 in obesity, with particular focus on preserving muscle mass and function in patients receiving weight loss treatments. The emergence of highly effective GLP-1 receptor agonists for obesity has created a new medical need, as these therapies can induce significant weight loss but may also lead to unintended loss of muscle mass. This muscle loss can compromise patients' strength, mobility, and overall quality of life.

The company's OBA clinical program investigates whether BIO101 can protect against muscle loss while patients undergo obesity treatment. Regulatory authorities in Europe have provided favorable assessments for initiating Phase 2 clinical trials in this indication. By potentially preserving muscle function during weight loss, BIO101 could address an important limitation of current obesity pharmacotherapies and improve long-term outcomes for patients.

Additional Pipeline Programs

Beyond its primary development programs, Biophytis has explored BIO101's potential in several other neuromuscular and age-related conditions. Preclinical research has investigated the compound in spinal muscular atrophy, another rare neuromuscular disease characterized by motor neuron loss and progressive muscle weakness. The company has also evaluated potential applications in age-related macular degeneration, a leading cause of vision loss in older adults.

These exploratory programs reflect the broad potential applicability of MAS receptor activation across multiple conditions involving tissue degeneration and impaired regenerative capacity. While these indications remain at earlier stages of development compared to sarcopenia and DMD, they represent potential future expansion opportunities for the BIO101 platform.

Scientific Foundation and Mechanism

The scientific rationale for BIO101 is grounded in research demonstrating that 20-hydroxyecdysone activates the MAS receptor, a component of the renin-angiotensin system traditionally known for regulating blood pressure and fluid balance. More recent research has revealed that this system also plays important roles in tissue metabolism and regeneration. The MAS receptor activation represents a "protective arm" that can enhance protein synthesis and muscle cell differentiation, potentially counteracting the muscle degradation processes that occur in various disease states.

Preclinical studies have shown that BIO101 stimulates myoblast differentiation and improves muscle function in both adult and aged animal models. The compound increases calcium influx and stimulates protein synthesis pathways in muscle cells, contributing to its anabolic effects. These mechanisms provide a biological rationale for targeting conditions characterized by impaired muscle protein balance and compromised muscle regenerative capacity.

Business Model and Strategy

As a clinical-stage biotechnology company, Biophytis focuses its resources on advancing BIO101 through clinical development and regulatory pathways. The company pursues strategic partnerships and licensing arrangements to support its development programs and expand market reach. Recent strategic initiatives include joint ventures with Asian pharmaceutical companies and co-development agreements for specific indications such as amyotrophic lateral sclerosis.

The company employs a geographical strategy that leverages both European scientific expertise and North American clinical development infrastructure. By maintaining operations in both regions, Biophytis aims to optimize its clinical trial execution and regulatory interactions across multiple jurisdictions. The company also pursues development opportunities in Asian markets, recognizing the significant patient populations and commercial potential in countries like China and Japan.

Investment Considerations

Investors should understand that Biophytis operates in the high-risk biotechnology sector, where clinical development outcomes are inherently uncertain. The company's transition from NASDAQ to the OTC Pink market reflects financial challenges common among clinical-stage biotechnology companies that have not yet achieved product commercialization. The OTC Pink listing means reduced regulatory oversight, potentially less financial disclosure, and lower trading liquidity compared to major exchange listings.

The company's success depends on successfully advancing BIO101 through expensive and time-consuming clinical trials, obtaining regulatory approvals, and eventually commercializing products in competitive markets. Clinical trials may fail to meet their endpoints, regulatory agencies may not approve the drug candidate, and even approved products may face commercial challenges. Biophytis will likely require additional capital to fund its development programs, which could result in dilution to existing shareholders.

On the positive side, the company is addressing significant unmet medical needs in areas where few or no approved therapies exist. Success in developing effective treatments for sarcopenia or DMD could represent substantial commercial opportunities given the large patient populations and lack of current pharmacological options. The orphan drug designations for DMD provide regulatory advantages and potential market exclusivity that could benefit the company if development is successful.