Acer Therapeutics

The company's lead product, Olpruva (sodium phenylbutyrate) is approved in the USA for the treatment of urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

Acer is also advancing a pipeline of investigational product candidates for rare and life-threatening diseases, including: Olpruva for treatment of various disorders, including Maple Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of Vasomotor Symptoms (VMS), post-traumatic stress disorder (PTSD) and prostate cancer; and Edsivo (celiprolol) for treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation.

In March 2023, Acer paused the ACER-801 program following dissapointing Phase IIa results until it conducts a thorough review of the full data set.

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