GSK lassos rare disease program in Angiochem tie-up

GlaxoSmithKline ($GSK) has grabbed rights to biotech startup Angiochem's early-stage program for treating a lysosomal storage disease. The deal comes as the London-based drug giant and its large pharma peers plunge headlong into the rare diseases field.

Angiochem, headquartered in Montreal, expects to get up to $31.5 million in upfront, research and additional fees from GSK if the drugmaker decides to secure rights to more of the small company's rare disease treatments. Depending on a lot of things going its way in its rare diseases program, and the partnership expanding, Angiochem said that it could bring in more than $300 million from its work with GSK.

GSK, which develops orphan disease drugs internally and via several partnerships, has made treating rare diseases in the brain a main research priority. With Angiochem's technology, GSK hopes to be able to develop at least one enzyme therapy for a lysosomal storage disease that can be smuggled through the blood-brain barrier into the central nervous system--where no current treatments against such rare diseases can reach.  

Developers of rare disease drugs are trying to figure out how to get their protein treatments into the brain, including Shire's ($SHPGY) Human Genetic Disease unit, which is working on a device that administers the treatments into the cerebrospinal fluid. Angiochem develops drugs that are linked to a peptide that homes in on the LRP-1 receptor to sneak drugs into the brain, and last year the company began testing an enzyme-replacement drug that is linked to its brain barrier-penetrating peptide, company CEO Dr. Jean-Paul Castaigne told FierceBiotech. His company's research has gone as far as to show that its tech can deliver enzyme drugs for lysosomal storage diseases into the brains of mice.

Venture-backed Angiochem had some early success in using its tech to carry cancer drugs into the brain to attack tumors, and Geron ($GERN) snagged rights to its Phase II brain cancer candidate in 2010 for $35 million. GSK is the small company's first major partner in the rare diseases arena, and Angiochem hopes that success in the partnership will lead to deals with other drugmakers. The CEO said that the company's enzyme replacements for rare diseases are intended to treat tissues in the brain and the rest of the body. 

"[The deal] means a lot," Castaigne said. He added that "the fact that we may bring drugs to the market with Glaxo that address the CNS symptoms of these diseases makes all the people in the lab very proud."