A biotech startup formed by Atlas Venture is collaborating with Biogen Idec to develop a treatment for inherited forms of Ataxia– a degenerative neurological condition that leads to physical disabilities, according to a company statement. They include the loss of coordination of hands, fingers, arms, legs but it can also cause a curvature of the spine and heart problems such as arrhythmias.
Ataxion has raised $17 million in a Series A round to develop treatments for inherited versions of the disease from Atlas Venture and Biogen. Friedreich’s ataxia is the most common version of the orphan disease, which affects under 200,000 people. The condition, which tends to show symptoms in adolescence, has no known cure and has been the subject of increasing attention from the life sciences industry and research institutions.
It reflects a broader life sciences industry trend of developing treatments for orphan diseases because there’s a higher barrier to entry and it allows companies to keep exclusivity for longer periods of time than conditions with larger patient populations.
Atlas Venture formed Ataxion last year when it licensed the technology from Danish biotech company Aniona, since renamed Saniona. The treatments under development focused on using potassium channel activators to protect Purkinje cells, the neurons in the cerebellum that control movement.
The hope is that these treatments have the potential to treat other forms ataxia, such as those associated with degenerative neurologic disorders including multiple sclerosis and Huntington’s disease.
In addition to the Series A funding, Biogen Idec is providing research and development and other funding to Ataxion. As part of the agreement, Biogen will have the option to acquire Ataxion to continue development of the program after the completion of a Phase 1 multiple ascending dose study to better understand pharmacokinetics and pharmacodynamics of multiple doses of the drug.
Saniona is responsible for key program discovery activities under the direction of Ataxion Chief Scientific Office Martin Jefson, former head of neuroscience research at Pfizer.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
A couple of other life science industry companies are developing treatments for the orphan disease. RepliGen has a group of compounds in Phase 1 development called Histone Deacetylase inhibitors designed to override the “silencing effect” of the gene’s expression that causes the genetic mutation behind Friedreich’s ataxia.
Edison Pharmaceuticals drug, vatiquinone, is in Phase 2 clinical trials.
Earlier this month Penn Medicine, The Children’s Hospital of Philadelphia, and FARA, a nonprofit organization dedicated to curing FA opened the Penn Medicine/CHOP Friedreich’s Ataxia Center of Excellence with a $3.25 million gift from Friedreich’s Ataxia Research Alliance, in partnership with the Hamilton and Finneran families.
