Acer Therapeutics is developing therapeutics for the treatment of (ultra) orphan diseases with significant unmet medical need
We are a venture-backed specialty orphan pharmaceutical company, headquartered in Cambridge, MA. Our business strategy is to repurpose and reformulate existing drugs for (ultra) orphan diseases with significant unmet medical need.
Our current pipeline consists of two late-stage clinical products: ACER-002 for vascular Ehlers-Danlos Syndrome (vEDS), and ACER-001 for Maple Syrup Urine Disease (MSUD). There are no FDA-approved drugs for vEDS and MSUD diseases, which collectively impact approximately 5,000 patients in the U.S. Our products have clinical proof-of-concept, mechanistic differentiation, and we intend to seek approval by using the fast and cost-effective 505(b)(2) regulatory pathway in the U.S. We are also pursuing other lifecycle and in licensing of additional pipeline opportunities.
We are focused on orphan drugs, because: (1) patients are in desperate need of them, (2) orphan drugs are commercially viable, and (3) regulatory approvals can be based on clinical surrogates.