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 is an age-related degeneration of skeletal muscle characterized by a loss of muscle mass and strength leading to mobility disability in the elderly (³65 years). 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-blind, placebo-controlled Phase 2b clinical trial (SARA-INT) of 334 elderly patients with sarcopenia 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 (DMD) is rare genetic neuromuscular disease in male children characterized by accelerated degeneration of muscle and is responsible for a loss of mobility, respiratory failure and cardiomyopathy, leading to premature death. 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).

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. We are preparing to submit an Investigation New Drug (IND) application to the FDA and clinical trial applications to the applicable regulatory agencies in Europe in the second half of 2019 to initiate our Phase 1/2 clinical proof of concept trial (MYODA-INT), utilizing a ‘seamless’ clinical trial design with composite clinical endpoints, as early as 2020, subject to regulatory approval of our study plan and clinical trial design, and study endpoints.


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. We plan to hold scientific advice meetings with the applicable regulatory agencies in Europe in the second half of 2019 regarding our clinical development plan in dry AMD, commencing with a Phase 1 clinical trial (MACA-PK) to assess the safety, pharmacokinetic (PK) and pharmacodynamic (PD) effects of Macuneos (BIO201) in healthy volunteers.