Repligen forms partnership on spinal muscle drug

NEW YORK (AP) -- Repligen Corp. said Thursday it will partner with Pfizer Inc. to pursue development of a drug designed to treat spinal muscular atrophy, a rare genetic disorder that is one of the leading causes of infant mortality.

The Waltham, Mass., company said it will get $70 million up-front from Pfizer, and it could receive an additional $65 million in future milestone payments. Repligen would also receive royalty payments on any approved products. The deal gives Pfizer one drug in clinical testing and other compounds and technologies.

The drug, designated RG309, is being tested on healthy people in an early-stage clinical trial. Repligen said it will continue that study and complete safety testing the drug on some groups of volunteers by the end of March. Pfizer will then take over development of the product.

Spinal muscular atrophy causes progressive damage to motor neurons, and Repligen said it is a leading cause of infant mortality. Symptoms typically appear before age 2, and the disease often causes early death because of respiratory problems. Repligen says RG309 is the first drug developed specifically to treat spinal muscular atrophy.

RG309 has received fast track status from the Food and Drug Administration. Fast track status is designed to speed up the approval of drugs that treat serious or life-threatening diseases for which there are few other therapies. The drug has also received orphan drug designation in the U.S. and the European Union, which means that similar products will be barred from the market for up to seven years in the U.S. and 10 years in Europe.

Shares of Repligen rose 4 cents to $6.67 in morning trading. Shares of Pfizer, which is based in New York, lost 16 cents to $25.75.