WE ARE A CLINICAL-STAGE BIOTECHNOLOGY COMPANY FOCUSED ON THE DEVELOPMENT OF THERAPEUTICS THAT SLOW THE DEGENERATIVE PROCESSES ASSOCIATED WITH AGING AND IMPROVE FUNCTIONAL OUTCOMES FOR PATIENTS SUFFERING FROM AGE-RELATED DISEASES, WITH A PRIMARY FOCUS ON NEUROMUSCULAR DISEASES.
Our goal is to become a leader in the emerging field of aging science by delivering life-changing therapies to the growing number of patients in need that have limited or no treatment options.
To accomplish this goal, we have assembled an experienced and skilled group of industry professionals, scientists, clinicians and key opinion leaders from leading industry and academic institutions from around the world.
INCREASING PREVALENCE OF AGE-RELATED DISEASES
In the last century, a number of degenerative diseases associated with aging have been characterized, including sarcopenia and AMD. However, effective treatment solutions are lacking and the prevalence of age-related diseases continues to increase. The global population of people over the age of 60 is expected to double from approximately 962 million in 2017 to 2.1 billion by 2050 (source: United Nations’ World Population Prospects: the 2017 Revision). In addition, healthcare costs, including costs associated with treatments and long-term care for age-related diseases associated with this demographic shift, are expected to rise. We believe that developing treatments to slow disease progression and reduce the risk of severe disability associated with age-related diseases is of the utmost importance.
FILLING A THERAPEUTIC GAP
Our lead drug candidate, Sarconeos (BIO101), is an orally administered small molecule in development for the treatment of neuromuscular diseases, including sarcopenia and Duchenne muscular dystrophy (DMD). Sarconeos (BIO101) is currently in a Phase 2b clinical trial for sarcopenia (SARA-INT) in the U.S. and Europe.
A pediatric formulation of Sarconeos (BIO101) is being developed for the treatment of DMD, for which the company was granted FDA IND approval in December 2019.
Our second drug candidate, Macuneos (BIO201), is an orally administered small molecule in development for the treatment of retinal diseases, including dry age-related macular degeneration (AMD) and Stargardt disease.