Financing will support the regulatory filing for the company’s lead product, VASEBRA™, for the treatment of Vascular Ehlers – Danlos Syndrome, as well as pivotal clinical studies for ACER-001 in MSUD and UCD

CAMBRIDGE, MA, May 9, 2016 – Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, today announced the company has closed its Series B round with $8.15 million in new investment. Proceeds from the financing will primarily be used to prepare a new drug application (NDA) for the company’s lead candidate, VASEBRA™ (celiprolol hydrochloride), which is being developed for the treatment of vascular Ehlers-Danlos Syndrome (vEDS), a rare, life- threatening genetic connective tissue disorder. Acer also plans to use the funds to support manufacturing and clinical development of ACER-001, a proprietary taste-masked, immediate-release formulation of sodium phenylbutyrate (NaPBA) for Maple Syrup Urine Disease (MSUD) and Urea Cycle Disorder (UCD), serious genetic diseases which impact amino acid and ammonia metabolism, respectively.

With a lead investment from TVM Life Science Venture VII, Dr. Luc Marengere, Managing Partner of TVM Life Science Capital,joins the board. He joins Dr. Cynthia Lavoie, TVM General Partner, who has led investments by TVM Life Science Venture VI into the Series A and B rounds. “TVM is pleased to support an experienced team and the development of late- stage products for orphan indications with a high unmet need,” says Dr. Lavoie. “This latest investment is representative of our commitment to the team and to the patients suffering from
these disorders,” adds Dr. Marengere. Investors include Bukwang Pharm. Co., which has supported the company since its Series A round.

“With this latest financing, Acer is well-positioned to progress our late-stage therapeutic candidates for serious rare diseases, particularly as we rapidly advance our lead candidate VASEBRA™ for vascular Ehlers-Danlos Syndrome toward key regulatory milestones,” said Chris Schelling, CEO and founder of Acer. “We are gratified that our investors have demonstrated confidence in Acer’s approach through their participation in both rounds of the company’s financing efforts.”

Both VASEBRA™ and ACER-001 received orphan drug designation from the U.S. Food and Drug Administration (FDA), which 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.

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.1

Acer is advancing ACER-002 (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.2 In 2015, the U.S. Food and Drug Administration (FDA) granted ACER-002 orphan drug designation for the potential treatment of vEDS.

About ACER-001
Maple Syrup Urine Disease (MSUD) is a rare, devastating genetic disease which prevents the proper metabolism of three essential amino acids – leucine, isoleucine and valine. There is no FDA-approved therapy for MSUD, and the condition is sub-optimally managed by a highly-restricted diet alone. Despite this diet, patients experience poor neurological outcomes and social impairment. Approximately 1,000 people suffer from MSUD in the U.S. and 3,000 are affected worldwide.

A Urea Cycle Disorder (UCD) is a genetic disorder caused by a mutation that results in a deficiency of one of the six enzymes in the urea cycle. These enzymes are responsible for removing ammonia from the blood stream. In UCDs, nitrogen accumulates in the form of ammonia, a highly toxic substance, resulting in hyperammonemia (elevated blood ammonia).Ammonia then reaches the brain through the blood, where it can cause irreversible brain damage, coma and/or death. Approximately 2,000 patients have been diagnosed with a UCD in the U.S.

Acer is planning to initiate pivotal clinical studies of ACER-001 for the treatment of MSUD and UCD. In 2014, the U.S. Food and Drug Administration (FDA) granted ACER -001 orphan drug designation for the potential treatment of MSUD.

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 : ACER-002 for vascular Ehlers-Danlos Syndrome (vEDS), and ACER-001 for Maple Syrup Urine Disease (MSUD). To date, Acer has raised $12.25 million in Series A and Series B rounds.
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1.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.
2.Ong  KT,  et  al.  Effect  of  celiprolol  on  prevention  of  cardiovascular  events  in  vascular Ehlers-Danlos syndrome: a prospective randomised, open, blinded-endpoints trial.
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Media Contact:
Ann Stanesa
Ten Bridge Communications