CAMBRIDGE, Mass.--(BUSINESS WIRE)--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, ACER-002 (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 ACER-002 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 ACER-002 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 ACER-002 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.
For more information, visit www.acertx.com.
References:
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. Lancet. 2010; 376: 1476–84.