Pipeline

We are developing a portfolio of programs targeting biological resilience pathways that slow the degenerative processes associated with aging and improve functional outcomes for patients suffering from age-related diseases.

 

SARCOPENIA (SARA CLINCAL PROGRAM)

Sarcopenia is an age-related degeneration of skeletal muscle characterized by a loss of muscle mass strength, balance and the ability to stand and/or walk. Sarcopenia leadsto mobility disability in the elderly (³65 years), resulting in a loss of independence, increased risk of adverse health events and hospitalization, and potential death stemming from falls, fractures, and physical disability.

Estimated prevalence ranks between 6-22% in the elderly (≥ 65 years), resulting in a total patient population between 20 and 73 millions worldwide.

Sarcopenia was first defined in 1989 and was classified as a disease in 2016 based on the World Health Organization (WHO) ICD-10-CM code M62.84.

There is currently no approved medication and no widely accepted standard of care for sarcopenia. Current non-medicinal treatment recommendations primarily focus on moderate physical activity and nutritional intervention.

We are testing the safety and efficacy of Sarconeos (BIO101) in an ongoing, global, randomized, multicenter, double-blinded, placebo-controlled Phase 2b clinical trial (SARA-INT) with sarcopenic patients at risk of mobility disability. We believe SARA-INT is one of the largest and most advanced clinical studies worldwide for the treatment of elderly patients with sarcopenia at risk of mobility disability.

DUCHENNE MUSCULAR DYSTROPHY (MYODA PROGRAM)

Duchenne Muscular Dystrophy (DMD) is a rare, genetic neuromuscular disease in male children characterized by accelerated degeneration of muscles and is responsible for a loss of mobility, respiratory failure and cardiomyopathy, leading to premature death. It is the most common form of muscular dystrophy in children (first clinical symptoms are usually reported before the age of 5 years), affecting approximately one in 5,000 new born boys (approximately 20,000 new cases annually worldwide).

DMD is caused by a certain number of mutations in the dystrophin gene that results in the absence or very low levels of functional dystrophin, a cytoskeletal protein that protects muscle cells.

The absence of dystrophin in muscle severely weakens the structural and membrane stability of the muscle fibers, resulting in the loss of muscle strength and mobility, impaired respiratory function and cardiac defects. DMD evolves according to a very well understood progression with symptoms that are similar to those associated with accelerated aging across all stages.

The progression of DMD follows a highly predictable course, which is usually described in 5 consecutive stages: pre-symptomatic, early ambulatory, late ambulatory, early non-ambulatory, late non-ambulatory

Currently there is no cure for DMD and only limited treatment options primarily consisting of corticosteroids and two targeted therapies (targeting specific dystrophin mutations either by exon skipping or with stop codons) available on the market (one in the United States and one in Europe). While these treatments aim to address the root cause of DMD, by allowing a partial expression of the dystrophin gene, they do not allow full restoration of muscle cell structure as the resulting gene product is a truncated dystrophin.

In preclinical animal studies, we observed a positive effect on muscle function, mobility, and respiratory function (a major disability in later stage DMD disease progression) in mdx mice models of DMD that were treated with Sarconeos (BIO101).

We received orphan drug designation from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for Sarconeos (BIO101) in DMD in 2018. The company was granted FDA IND approval in December 2019 to initiate a Phase 1/2 clinical proof of concept trial (MYODA-INT), utilizing a ‘seamless’ clinical trial design with composite clinical endpoints.

DRY AGE-RELATED MACULAR DEGENERATION (MACA PROGRAM)

Age-related Macular Degeneration (AMD) is an age-related degeneration of the macula, the central part of the retina. It is one of the leading causes of irreversible vision loss and blindness in people over the age of 50 worldwide. The dry (atrophic) form of AMD affects central vision and impairs many functions affecting quality of life and independent living such as reading, driving, and facial recognition. Currently there are no approved therapies available for dry AMD.

We have completed chronic and acute animal toxicology studies to support IND and clinical trial applications. The clinical development of Macuneos (BIO201) is Biophytis’ next asset for value creation. It starts with a Phase 1 clinical trial (MACA-PK) to assess the safety, pharmacokinetic (PK) and pharmacodynamic (PD) effects of Macuneos (BIO201) in healthy volunteers, followed by a Phase 2a/b interventional study (MACA-INT) in patients with geographic atrophy (GA) in one eye and iAMD in the fellow eye.