French DMD-Focused Biotech, Anagenesis Biotherapies, Sets Up Shop in Boston

Published: Apr 20, 2016

French DMD-Focused Biotech, Anagenesis Biotherapies, Sets Up Shop in Boston
April 19, 2016
By Mark Terry, Breaking News Staff

Anagenesis Biotherapies, headquartered in Strasbourg, France, announced yesterday that it was opening a U.S. subsidiary in Boston, Mass. The offices will be located at the Cambridge Innovation Center’s 50 Milk Street facility and will focus on Duchenne Muscular Dystrophy (DMD).

“We are very excited to become part of this exceptional biotechnology and academic cluster,” said Jean-Yves Bonnefoy, president and chief executive officer of Anagenesis, in a statement. “Massachusetts is the ideal location for us to advance our DMD cell therapy product candidate, which has unique advantages over other therapies currently under development. Indeed, the local strength in stem cell research will greatly benefit the cell therapy focus of our U.S. subsidiary and also will help us expand our product candidate portfolio to brown fat or cartilage-based therapies to address other diseases druggable by our technology.”

DMD is a muscle wasting disease caused by mutations in the dystrophin gene. The disease is progressive and generally causes death in early childhood. Complications include serious heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 to 5,000 male children.

It hasn’t been a particularly good year for companies focused on DMD. On Jan. 25, Cambridge, Mass.-based Akashi Therapeutics halted its DMD trial for HT-100 after one of its patients developed serious, life-threatening health problems. San Rafael, Calif.-based BioMarin Pharmaceutical ’s application for its DMD drug Kyndrisa (drisapersen) was turned down by the U.S. Food and Drug Administration (FDA) on Jan. 15, with the FDA arguing the drug didn’t show enough benefit.

Cambridge, Mass.-based Sarepta Therapeutics has had a more complicated road for its DMD drug eteplirsen. Originally the FDA’s Peripheral and Central Nervous System Advisory Committee was scheduled on Jan. 21 to make a decision on the company’s New Drug Application (NDA). But, because of an impending snowstorm on the east coast, the meeting was postponed until April 25.

Last month, 36 DMD experts signed a letter to the FDA urging it to approve eteplirsen. Penned by two co-directors of the Center for Duchenne Muscular Dystrophy at UCLA, M. Carrie Miceli and Stanley Nelson, the letter noted the difficulty of conducting trials on DMD patients, saying, “These three-year data alone are clearly supportive of accelerated approval, however the new four-year data regarding age at loss of ambulation now provide even more compelling data for accelerated approval based on an irreversible morbidity. … We suggest that the most scientifically robust way forward and the most ethnical choice for the Duchenne community is in the context of an accelerated approval followed by a confirmatory trial.”

In February, 109 members of Congress also sent a letter to the FDA urging it to accelerate approval of a DMD drug—any DMD drug.

Anagenesis is a private company that focuses on muscle diseases, which include DMD, as well as sarcopenia and cachexia. Anagenesis’ approach is on stem cells and reprogrammed cell-based technologies. Based on the research of Olivier Pourquie, Paraxial Mesoderm Multipotent Cells (P2MCs) are shared precursors of skeletal muscle, ribs, vertebra, dermis, endothelium and brown fat. Its technology platform essentially allows it to engineer stem cells to produce muscle cells, as well as other cell types.

It also utilizes high throughput and high content screening in customized in vitro assays to identify lead compounds that act on muscle progenitors for sarcopenia and cachexia, which are muscle disorders related to aging and cancer, respectively.

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