Acer Therapeutics Inc.
Acer Therapeutics is a pharmaceutical company focused on the acquisition, development, and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs. They have a pipeline of investigational product candidates including OLPRUVA™ for urea cycle disorders and Maple Syrup Urine Disease, and EDSIVO™ for vascular Ehlers-Danlos syndrome. The company is committed to supporting patients and families in need, engaging with patient and clinician communities, and advancing therapies through regulatory pathways.
Industries
Nr. of Employees
small (1-50)
Acer Therapeutics Inc.
Products
Sodium phenylbutyrate oral suspension (U.S. approved)
An oral suspension formulation of sodium phenylbutyrate approved in the U.S. for treatment of certain urea cycle disorders involving specific enzyme deficiencies.
Taste‑masked multiparticulate sodium phenylbutyrate formulation (investigational)
A taste‑masked, multi‑particulate immediate‑release formulation of sodium phenylbutyrate designed to improve palatability and allow potential administration under fasted conditions; subject to bioequivalence and regulatory studies.
Celiprolol program for vascular Ehlers‑Danlos syndrome (investigational)
Clinical development program for celiprolol as a treatment candidate for vascular Ehlers‑Danlos syndrome in patients with confirmed COL3A1 mutation; program includes observational and cost/utilization analyses and referenced clinical literature.
Osanetant program for vasomotor symptoms and investigational prostate cancer applications (paused)
An investigational neurokinin 3 receptor antagonist evaluated for vasomotor symptoms and being explored in pilot trials in prostate cancer; development was paused following review of Phase 2a topline data.
Sodium phenylbutyrate oral suspension (U.S. approved)
An oral suspension formulation of sodium phenylbutyrate approved in the U.S. for treatment of certain urea cycle disorders involving specific enzyme deficiencies.
Taste‑masked multiparticulate sodium phenylbutyrate formulation (investigational)
A taste‑masked, multi‑particulate immediate‑release formulation of sodium phenylbutyrate designed to improve palatability and allow potential administration under fasted conditions; subject to bioequivalence and regulatory studies.
Celiprolol program for vascular Ehlers‑Danlos syndrome (investigational)
Clinical development program for celiprolol as a treatment candidate for vascular Ehlers‑Danlos syndrome in patients with confirmed COL3A1 mutation; program includes observational and cost/utilization analyses and referenced clinical literature.
Osanetant program for vasomotor symptoms and investigational prostate cancer applications (paused)
An investigational neurokinin 3 receptor antagonist evaluated for vasomotor symptoms and being explored in pilot trials in prostate cancer; development was paused following review of Phase 2a topline data.
Services
Licensing and collaboration for therapeutic development
Structured collaborations and license agreements to develop and commercialize clinical‑stage therapeutic candidates globally.
Sponsor and support of clinical trials (including investigator‑sponsored studies)
Sponsorship and operational support for early‑phase and investigator‑sponsored clinical studies in rare diseases.
Regulatory planning and submission support
Preparation for regulatory interactions and planned NDA submissions, including pre‑NDA meetings and pathway selection.
Pharmacovigilance and medical information services
Mechanisms for reporting adverse events and providing medical information for marketed and investigational products.
Licensing and collaboration for therapeutic development
Structured collaborations and license agreements to develop and commercialize clinical‑stage therapeutic candidates globally.
Sponsor and support of clinical trials (including investigator‑sponsored studies)
Sponsorship and operational support for early‑phase and investigator‑sponsored clinical studies in rare diseases.
Regulatory planning and submission support
Preparation for regulatory interactions and planned NDA submissions, including pre‑NDA meetings and pathway selection.
Pharmacovigilance and medical information services
Mechanisms for reporting adverse events and providing medical information for marketed and investigational products.
Expertise Areas
- Rare disease drug development
- Clinical development and trial execution
- Formulation development for oral small molecules
- Pharmacokinetics and bioequivalence studies
Key Technologies
- Multiparticulate taste‑masked oral formulations
- Bioequivalence study design
- Clinical pharmacokinetic (PK) analysis
- Physiologically‑based PK/PD (in silico) modeling