ACER20230421C5
ACER Apr 21 2023 5.00 Call (ACER230421C00005000)
option OPRA

acer therapeutics is a biotech company, headquartered in cambridge, ma, that develops repurposed and reformulated medicines for the treatment of ultra-orphan diseases with significant unmet medical needs. acer's lead candidate, edviso™ (celiprolol), is the first pharmaceutical therapy seeking approval for patients with vascular-type ehlers-danlos syndrome (veds). ehlers-danlos syndrome is a group of hereditary disorders of connective tissue with no ethnic predisposition. veds is a subtype characterized by severe arterial dissections, ruptures and early death. complications are rare in childhood, but affect 25% of patients before the age of 20, and 80% by the age of 40. median age of death is estimated to be around 50 years. patients are diagnosed by clinical symptoms and confirmed by presence of mutations in the col3a1 gene. there are approximately 2,000 documented patients, but prevalence could be as high as 5,000 patients in the u.s. (pepin 2014). there are no specific pharmacologica