Rare-disease powerhouse Alexion grabs an option on muscular dystrophy buyout

Martin Mackay, Alexion's head of R&D--Courtesy of Alexion

Alexion is looking to beef up its pipeline of drugs for ultra-rare diseases. The biotech ($ALXN) has struck a deal with little Prothelia and the University of Nevada, Reno, where it will step in and conduct development research on a protein replacement therapy for merosin-deficient congenital muscular dystrophy, or MDC1A. Alexion has an option to buy the company and directly in-license laminin-111 from the university, provided the research pans out.

The idea here is that laminin-111 can be used to substitute for laminin-211, a glycoprotein that forms an extracellular matrix necessary for healthy muscles. The rare patients who contract the disease suffer from muscle weakness and a gradual degradation of their respiratory system. Milford, MA-based Prothelia, which appears to have been funded primarily through patient groups and the NIH, won orphan status for MDC1A as well as Duchenne muscular dystrophy, a disease field that has recently seen a series of bitter setbacks for companies like Prosensa ($RNA), Sarepta ($SRPT) and PTC Therapeutics ($PTCT).

Alexion's success with treating rare diseases, demonstrating how to earn big revenue from small patient populations, has helped popularize the field for a slate of biotechs working on new drugs. The company quickly turned Soliris, the world's most expensive therapy, into a blockbuster. And it has bagged two coveted breakthrough drug designations from the FDA, putting it in line for rapid-fire development programs. 

None of the companies disclosed the terms in the option deal.

"MDC1A is a disease that aligns very well with our passion to develop and deliver first-in-class drug candidates that have the potential to transform the lives of people suffering with devastating disorders that currently have no treatment options," said Martin Mackay, the former R&D chief at AstraZeneca ($AZN) who now runs research at Alexion. "Laminin protein replacement is a promising experimental therapy and addition to our research initiatives as we seek to further expand our product portfolio."

- here's the press release

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