Need: There are currently no safe and effective therapeutics for the treatment of muscle wasting associated with a number of serious chronic and acute medical conditions such as surgical and severe burn recovery, end-stage renal disease and cancer- and AIDS-related cachexia. The shortage of existing treatments results in extended hospital stays and recovery time as well as diminished quality of life.
Potential: The SARM program has the potential to address a significant unmet medical need, muscle wasting, across a number of diseases and disorders where treatments to current therapy are needed. Market sizes for these diseases range from $400 million to in excess of $1 billion.
Therapeutic Target: Endogenous or natural androgens are hormones that play an important role in several systems in the body while also stimulating the growth of the prostate in men. As individuals age, the level of these hormones gradually declines, potentially causing the loss of muscle mass and osteoporosis. This loss of muscle mass and bone becomes accelerated in situations of extreme stress and immobility, such as occurs in patients with end-stage renal disease or severe burns. SARM agonists are small molecules which have been shown to activate the androgen receptor selectively in certain tissues. In doing so, SARMs may, through oral administration, supplement the lack of endogenous androgens without causing undesirable effects such as prostate growth.
Program Status: The SARM program was licensed from Bristol-Myers Squibb. The lead compound in Pharmacopeia’s SARM program (PS178990), which the company believes to be one of the most potent SARM agonists currently identified, is presently in Phase 1 clinical development. PS178990 has been well characterized both pre-clinically and in a Phase 1 single ascending dose (SAD) study. Results for the SAD study demonstrated PS178990 to be safe and well tolerated at doses anticipated to be clinically efficacious. Pharmacopeia expects to complete additional Phase 1 studies for PS178990 in 2008 and initiate a Phase 2 study of the compound in early 2009.
