By Mark Terry
Financial details were not disclosed. Acer plans to start development of osanetant for specific but unannounced neuroendocrine-related disorders.
Acer focuses on buying, developing and commercializing drugs for serious rare and ultra-rare diseases. It currently has two late-stage pipeline candidates. Edsivo (celiprolol) is being developed for vascular Ehlers-Danlos syndrome (vEDS) and ACER-001, which is a fully taste-masked, immediate release formulation of sodium phenylbutyrate. ACER-001 is being developed for urea cycle disorders (UCD) and Maple Syrup Urine Disease (MSUD).
In the summer of 2017, Acer went public via a reverse merger with Opexa Therapeutics, based in Texas. Opexa laid off all but two of its 20 staffers after its lead compound for multiple sclerosis failed a mid-stage clinical trial in October 2016. Under that deal, Acer received a $15.7 million financing round led by TVM Capital.
The company submitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) on October 29, 2018 for Edsivo for vEDS. The company also requested Priority Review.
eVDA is the most severe form of a group of diseases of the connective tissue called Ehlers-Danlos Syndrome (EDS). Patients with vEDS suffer from life-threatening arterial dissections and ruptures, in addition to ruptures of the intestines and uterus. The average mortality is 51 years of age.
On December 26, 2018, the FDA accepted the NDA for review and gave it a target action date of June 25, 2019.
“The acceptance of our NDA for Edsivo is an important step in our efforts to help patients with vEDS, who suffer with a devastating disease that currently has no approved treatment,” stated William Andrews, Acer’s chief medical officer. “We have had the honor of learning about the significant challenges of living with vEDS directly from patients and their families. This has in large part driven the hard work, passion and complete dedication that our small team has given to this effort, and we will continue to do so as the FDA reviews our NDA for Edsivo. We are excited about the possibility of making Edsivo available in the U.S. for patients in the near future.”
Sanofi originally tried to develop osanentant for schizophrenia, but failed in the attempt. It abandoned the efforts in August 2005. Other possible applications were for drug addiction, because it blocked the effects of cocaine in animal models. Other possible indications include depression and visceral pain.
Chris Schelling, Acer’s founder and chief executive officer, stated, “We are thrilled to expand our pipeline of product candidates by in-licensing the global rights to osanetant. The drug perfectly fits Acer’s acquisition and development model of de-risked assets—it already has robust non-clinical, pharmacokinetic and human safety data, and based on recent studies involving antagonism of the NK3 receptor, we believe it can be successfully repurposed to treat a variety of neuroendocrine disorders. We very much appreciate the opportunity to expand our relationship with Sanofi.”
In related news, MyoKardia announced it had regained global rights to all the programs it had inked with Sanofi. The research deal ended on December 31, 2018, and will effectively conclude in its entirety on April 1, 2019.
MyoKardia gains all rights to programs in the portfolio, including to mavacamten and MYK-491. They launched the collaboration in 2014 to advance up to three programs through discovery and into the clinic for the treatment of hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM).
During that period, Sanofi paid MyoKardia about $230 million in funding, and mavacamten advanced from preclinical studies into a late-stage trial for HCM, and MYK-491 from discovery to a Phase II proof-of-concept trial in DCM.
“We are grateful for Sanofi’s support over the past four years in what was a highly productive relationship,” stated Tassos Gianakakos, MyoKardia’s chief executive officer. “Since entering into the collaboration, MyoKardia has become a leading research and development organization with a robust pipeline of targeted cardiovascular therapeutics. Regaining worldwide rights enables us to capture the full value of the data being generated in the next 12-24 month as we prepare for the potential registration of mavacamten in obstructive hypertrophic cardiomyopathy and obtain proof-of-concept for MYK-491 in patients with dilated cardiomyopathy. Importantly, consolidated control over our entire portfolio allows us to make decisions about how we advance each of our therapeutic candidates in alignment with our precision medicine approach.”