This segment focuses on the research, development, and commercialization of therapies for rare and life-threatening diseases. Research and development activities include clinical trials for EDSIVO (celiprolol) for vascular Ehlers-Danlos Syndrome and ACER-001 (sodium phenylbutyrate) for urea cycle disorders and maple syrup urine disease. The company utilizes small molecule drug development and clinical trial methodologies. The patient impact is significant, aiming to improve the quality of life and address unmet medical needs in rare disease populations. Market positioning is focused on niche markets with high unmet needs. Future opportunities include expanding the pipeline and securing regulatory approvals. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials. Partnerships include collaborations with Relief Therapeutics for ACER-001.

This segment is dedicated to the development of ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms. Research and development activities include Phase 2a clinical trials to evaluate the efficacy and safety of osanetant. The company utilizes small molecule drug development and clinical trial methodologies. The therapeutic area focuses on addressing the symptoms associated with menopause. The patient impact is aimed at improving the quality of life for women experiencing moderate to severe vasomotor symptoms. Market positioning involves targeting a specific patient population with a high unmet need. Future opportunities include securing regulatory approvals and expanding the therapeutic applications of osanetant. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials. The company has a license agreement with Sanofi to acquire worldwide rights to osanetant.

This segment focuses on the development of ACER-2820 (emetine) as a host-directed therapy against a variety of infectious diseases, including COVID-19. Research and development activities include exploring the potential of emetine hydrochloride as a treatment for COVID-19. The company utilizes drug discovery and clinical trial methodologies. The therapeutic area covers infectious diseases. The patient impact aims to provide effective treatments for infectious diseases. Market positioning involves targeting infectious diseases with high unmet needs. Future opportunities include expanding the therapeutic applications of emetine. Regulatory and clinical aspects involve navigating FDA approval processes and conducting clinical trials. The company has a research collaboration agreement with the National Center for Advancing Translational Sciences (NCATS) to develop emetine hydrochloride.