Biogen Idec and Atlas Venture have agreed to invest a combined $17 million of Series A financing in a nearly-year-old drug developer focused on hereditary ataxias.

Biogen Idec is separately providing R&D and other funding to the company, called Ataxion. The biotech giant has the option to acquire Ataxion to continue development of the program upon completion of a Phase I multiple ascending dose (MAD) study at pre-negotiated terms, including undisclosed upfront and milestone payments.

“Our investment in Ataxion fits perfectly into our mission to bring innovative therapies to people who have severe neurodegenerative diseases and few treatment options,” Steven Holtzman, evp of corporate development at Biogen Idec, said in a statement. “Ataxion's early research in the area of ataxias is compelling, and we look forward to working with them as this research progresses.” 

Biogen Idec and Atlas said the drug candidates being developed for several genetic forms of ataxia may also be able to treat other forms of the orphan genetic disorder, such as those associated with multiple sclerosis and Huntington's disease.

By focusing on treating a group of ataxias, Ataxion distinguishes itself from several other companies focused on particular forms of the disease marked by dysfunction or degeneration of the cerebellum. Shire in January completed its $4.2 billion acquisition of ViroPharma, whose product candidates included the preclinical drug candidate VP20629 for Friedrich’s ataxia.

Also in January, BioMarin Pharmaceutical agreed to purchase Repligen's histone deacetylase inhibitor (HDACi) library and related intellectual property for $2 million upfront plus additional undisclosed milestone and royalty payments. BioMarin said the HDACi portfolio’s potential applications included treatments for Friedreich's ataxia and other neurological disorders.

Earlier this month, Edison Pharmaceuticals won FDA “fast-track” designation for its own Fredrich's ataxia drug, the company's lead drug candidate EPI-743, now in Phase II trials. And on February 12, the developer of a preclinical gene therapy for Friedrich’s ataxia, Voyager Therapeutics, was launched by Third Rock Ventures with $45 million in Series A financing.

Ataxion was launched in April 2013 from Atlas Venture’s seed program by Joshua Resnick, M.D., and David Grayzel, M.D. Dr. Resnick is Ataxion’s co-founder and CEO, as well as a venture partner at Atlas. Dr. Grayzel is managing director of Atlas Venture Development Corp. and a member of Ataxion’s board.

Three months later in July 2013, Ataxion acquired the ataxia development program from Aniona, a Danish biotech that has since been renamed Saniona and focuses on ion-channel drug discovery. Saniona retains responsibility for key program discovery activities through Ataxion CSO Martin Jefson, Ph.D., former head of Neuroscience Research at Pfizer.

As part of the Series A financing, Drs. Resnick and Grayzel will be joined on the Ataxion Board of Directors by Holtzman; Kevin Koch, Ph.D., Biogen Idec svp of R&D; Atlas Partner Jean-Francois Formela, M.D., and Claus Braestrup, former CEO of Lundbeck Pharmaceuticals and chairman of Saniona. 

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