Acer Therapeutics Closes $4.1 Million Series A Financing, Forms Board of Directors and Advances Lead Development Programs.
CAMBRIDGE, MA, July 1, 2015 – Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, closed its Series A round with $4.1 million. Proceeds from the financing will be used to advance the development of a proprietary, taste-masked, immediate-release formulation of sodium phenylbutyrate, ACER-001, for maple syrup urine disease (MSUD), a rare, devastating genetic disease which prevents the proper metabolism of three essential, branched-chain amino acids (BCAAs) – leucine, isoleucine and valine. The Series A financing was led by TVM Capital Life Science. Acer has also formed its Board of Directors. Cynthia Lavoie, Ph.D., General Partner at TVM, joined the Board in March 2015, and two other independent members joined in October 2015: Stephen Aselage, CEO of Retrophin and former Executive Vice President and Chief Business Officer of BioMarin; and John Dunn, former Managing Director of Biogen-Idec Ventures, and General Counsel, IDEC Pharmaceuticals. Beyond ACER-001, Acer is also advancing ACER-002 (celiprolol hydrochloride), a new chemical entity (NCE), for the treatment of vascular Ehlers-Danlos Syndrome (vEDS). In people with vEDS, mutations in the COL3A1 gene for type III procollagen lead to a deficit of collagen synthesis. This rare and lethal form of EDS causes catastrophic, vascular complications, and is associated with ruptured blood vessels, aneurysms, dissections, fistulas and spontaneous ruptures of the uterus and gastrointestinal structures. There are currently approximately 2,000 people in the U.S. diagnosed with vEDS, though experts estimate as many as 5,000 patients may be affected.1
References:
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.
Acer Therapeutics Appoints Benjamin Dewees as Vice President, Regulatory Affairs and Manufacturing
Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, appointed Benjamin Dewees as Vice President, Regulatory Affairs and Manufacturing. In this role, Mr. Dewees brings extensive experience in regulatory processes for, and approvals of, therapies for rare diseases. “Ben brings critically important expertise in regulatory affairs and manufacturing to Acer as we move forward with our programs in Maple Syrup Urine Disease, Urea Cycle Disorders and vascular Ehlers-Danlos Syndrome,” said Chris Schelling, COO and Founder of Acer Therapeutics. “We are looking forward to working closely with the FDA as we advance ACER-001 and ACER-002 through clinical studies to potentially bring the first approved therapies to patients suffering from these serious, rare diseases.” Prior to Acer, Mr. Dewees spent 16 years at BioMarin and was instrumental in the approval of the first three products that BioMarin developed. He coordinated the Chemistry, Manufacturing and Controls portion of the Aldurazyme marketing applications in the US, EU, Japan, Canada, and various other countries. He led the approval process for Naglazyme in the EU, was the regulatory lead for the Kuvan program starting with the Phase 3 stage of development through marketing approval in the U.S., and continued to support Kuvan in the post-marketing setting including collaborating with corporate partners for approvals in the EU and ROW. Mr. Dewees’s initial experience in industry was at IDEC Pharmaceuticals, where he served in manufacturing with an emphasis on facility and process design start-up. He led key aspects of a clinical stage Good Manufacturing Practice (GMP) facility at IDEC and one clinical and one commercial scale GMP manufacturing facility at BioMarin.
The U.S. Food and Drug Administration Grants Orphan Drug Designation to ACER-002 for the Potential Treatment of Vascular Ehlers-Danlos Syndrome
In January 2015, Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) to celiprolol hydrochloride (ACER-002), a new chemical entity (NCE), for the treatment of vascular Ehlers-Danlos Syndrome (vEDS). The disease, also known as Ehlers-Danlos Syndrome type IV, is a rare, autosomal dominant, connective tissue disorder with no FDA-approved therapy for the disease. In people with vEDS, mutations in the COL3A1 gene for type III procollagen lead to a deficit of collagen synthesis. This rare and lethal form of Ehlers-Danlos Syndrome (EDS) is associated with ruptured blood vessels, aneurysms, dissections, fistulas and spontaneous ruptures of the uterus and gastrointestinal structures. The median age of survival for people with vEDS is 51 years. 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.1 Orphan drug designation is provided to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.
Reference:
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.
The U.S. Food and Drug Administration Grants Orphan Drug Designation to ACER-001 for the Potential Treatment of Maple Syrup Urine Disease
In August 2014, Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) to sodium phenylbutyrate (ACER-001) for the treatment of Maple Syrup Urine Disease (MSUD). Approximately 1,000 patients suffer from MSUD in the U.S. and 3,000 patients are affected worldwide. There is no FDA-approved therapy for MSUD, and the condition is sub-optimally managed via a BCAA-restricted diet alone. Despite this diet, patients still experience poor neurological outcomes and social impairment.
Orphan drug designation, which qualifies Acer for various development incentives under the Orphan Drug Act, is provided to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.
Acer Therapeutics Licenses Exclusive Worldwide Rights from Baylor College of Medicine for Development of Sodium Phenylbutyrate for Maple Syrup Urine Disease
In April 2014, Acer Therapeutics Inc. entered into an exclusive worldwide license agreement with Baylor College of Medicine to develop and commercialize formulations and prodrugs of sodium phenylbutyrate for the treatment of Maple Syrup Urine Disease (MSUD). MSUD is a rare, devastating genetic disease which prevents the proper metabolism of three essential, branched-chain amino acids (BCAAs) – leucine, isoleucine and valine. There is no FDA-approved therapy for MSUD, and the condition is sub-optimally managed via a BCAA-restricted diet alone. Despite this diet, patients still experience poor neurological outcomes and social impairment.
“The research data generated by the team at Baylor College of Medicine supports phenylbutyrate, when used in conjunction with a BCAA-restricted diet, as a promising therapy to lowering levels of BCAAs – and in particular leucine – for patients affected by MSUD,” said Chris Schelling, COO and Founder of Acer Therapeutics. “We are looking forward to advancing our proprietary taste-masked, immediate-release formulation of sodium phenylbutyrate, ACER-001, through clinical studies and potentially bring the first therapeutic option to patients with this serious disease.”
