Month: December 2016

Inaugural team of experts to provide valuable insights and guidance on NDA filing of the company’s lead product, VASEBRA™ (celiprolol), for the treatment of vascular Ehlers-Danlos Syndrome, and pivotal clinical studies of ACER-001 in MSUD and UCD

CAMBRIDGE, Mass., December 20, 2016 – Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, today announced the formation of its Scientific Advisory Board (SAB). The five inaugural members of Acer’s SAB are:

• Gerard Berry, M.D.: Harvey Levy Chair in Metabolism; Director, Metabolism Program, Division of Genetics and Genomics, Boston Children’s Hospital; Professor of Pediatrics, Harvard Medical School Thomas Fleming, Ph.D.: Professor, Biostatistics and Professor, Statistics, University of Washington; member of the Fred Hutchinson Cancer Research Center; former Director of the Statistical Center for HIV/AIDS National Institutes of Allergy and Infectious Diseases Prevention Trial Network Marlene Haffner, M.D., M.P.H.: Founder, Haffner Associates; former Director of the Office of Orphan Products Development at the Food and Drug Administration (FDA) Brendan Lee, M.D., Ph.D.: Chairman, Molecular and Human Genetics, Baylor College of Medicine; Robert and Janice McNair Endowed Chair and Professor of Molecular and Human Genetics, Baylor College of Medicine Stuart Swiedler, M.D., Ph.D.: Biotechnology consultant and former senior executive at BioMarin Pharmaceutical

Visit www.www.acertx.com for full bios of SAB members. “We are excited to welcome this team of experts to our SAB, as they will provide important guidance as we progress our late-stage therapeutic candidates for serious rare diseases,” said Chris Schelling, CEO and Founder of Acer. “We look forward to working closely with our advisors as we rapidly advance our lead candidate VASEBRA™ for vascular Ehlers-Danlos Syndrome towards a New Drug Application (NDA) filing, and initiate pivotal clinical studies of ACER-001, which we are developing for Maple Syrup Urine Disease (MSUD) and Urea Cycle Disorder (UCD), devastating genetic diseases which impact amino acid and ammonia metabolism, respectively.”

About Acer Therapeutics Acer Therapeutics, headquartered in Cambridge, MA, is developing therapies with established clinical proof-of-concept for the treatment of serious, ultra-rare diseases with critical unmet medical need. The company’s late-stage clinical pipeline includes two candidates for severe genetic disorders for which there are currently no FDA-approved treatments: VASEBRA™ for vascular Ehlers-Danlos Syndrome (vEDS), and ACER-001 for Maple Syrup Urine Disease (MSUD) and Urea Cycle Disorders (UCD).

Media Contact:

Ann Stanesa

Ten Bridge Communications

617-230-0347

[email protected]

Company obtains exclusive rights to NDA-enabling clinical data of the company’s lead product, VASEBRA™ (celiprolol), for the treatment of vascular Ehlers-Danlos Syndrome (vEDS)

CAMBRIDGE, Mass., December 13, 2016 – Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, today announced the signing of an agreement with the Greater Paris University Hospitals AP-HP (via its Department of Clinical Research and Development*) granting the company exclusive rights to access and use data from a randomized controlled clinical study of celiprolol.1 The company will use this pivotal clinical data to support a New Drug Application (NDA) regulatory filing for its lead product, VASEBRA™ (celiprolol), for the treatment of vascular Ehlers-Danlos Syndrome (vEDS). “We have studied celiprolol for nearly two decades in vEDS patients and this is the only drug ever to demonstrate a clinical benefit in a randomized, controlled clinical study,” said Pierre Boutouyrie M.D., Ph.D., co-director of the clinical pharmacology service at the Georges-Pompidou European Hospital AP-HP and Principal Investigator for the celiprolol study. “Having established celiprolol as a standard of care in France for vEDS patients, we are excited to partner with Acer to help bring celiprolol to members of the U.S. patient community who are suffering from this devastating, life-threatening disease.” “We are committed to bringing VASEBRA™ to vEDS patients who currently do not have access to this treatment,” said Robert D. Steiner, M.D., Chief Medical Officer of Acer. “This pivotal clinical data from AP-HP will represent a critical element of the clinical module in our NDA, which we are diligently building along with manufacturing, non-clinical and other components of the regulatory package.” “This collaboration between AP-HP and Acer is a fantastic example of academic-industry partnership,” said Chris Schelling, CEO and Founder of Acer. “The signing of this agreement marks an important corporate milestone as it will enable us to continue to rapidly advance our lead candidate VASEBRA™, a potential life-saving therapy for patients with vEDS, towards an NDA filing.”

About VASEBRA™ and Vascular Ehlers-Danlos Syndrome (vEDS) Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. Vascular EDS (vEDS) is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The average mortality is 51 years of age. There are approximately 2,000 people in the U.S. diagnosed with vEDS, though experts estimate as many as 5,000 patients may be affected. There are currently no FDA-approved therapies for vEDS.2 Acer is advancing VASEBRA™ (celiprolol), a new chemical entity (NCE), for the treatment of vEDS and plans to file an NDA based on a randomized controlled clinical study of celiprolol.1 In 2015, the U.S. Food and Drug Administration (FDA) granted VASEBRA™ orphan drug designation for the potential treatment of vEDS. *The Office for Technology Transfer & Industrial Ventures of the AP-HP’s Department of Clinical Research and Development, protects and enhances the innovations and clinical expertise of medical personnel by setting up privileged partnerships with healthcare companies. Nearly half of the patented innovations are transferred to companies all over the world and, in particular, the creation of nearly 70 young companies. The AP-HP organizes each year the APinnov Technology Transfer Meetings.

About Greater Paris university hospitals AP-HP: AP-HP (Greater Paris University Hospitals) is a European world-renowned university hospital. Its 39 hospitals treat 8 million people every year: in consultation, emergency, during scheduled or home hospitalizations. The AP-HP provides a public health service for everyone, 24 hours a day. This mission is a duty as well as a great source of pride. AP-HP is the leading employer in the Greater Paris area: 100,000 staff members – doctors, researchers, paramedical staff, administrative personnel and workers – work there.

About Acer Therapeutics Acer Therapeutics, headquartered in Cambridge, MA, is developing therapies with established clinical proof-of-concept for the treatment of serious, ultra-rare diseases with critical unmet medical need. The company’s late-stage clinical pipeline includes two candidates for severe genetic disorders for which there are currently no FDA-approved treatments: VASEBRA™ for vascular Ehlers-Danlos Syndrome (vEDS), and ACER-001 for Maple Syrup Urine Disease (MSUD) and Urea Cycle Disorders (UCD). For more information, visit www.www.acertx.com.

References:

1. Ong KT, et al. Effect of celiprolol on prevention of cardiovascular events in vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial. Lancet. 2010; 376: 1476–84.

2. Pepin MG, et al. Survival is affected by mutation type and molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type IV) Genet Med. 16: 881-888.

Media Contact:

Ann Stanesa

Ten Bridge Communications

617-230-0347

[email protected]