BioWorld International Correspondent

Santhera Pharmaceuticals AG entered its first agreement since its formation, through a German-Swiss merger last month between Graffinity Pharmaceuticals AG and MyoContract AG.

The company entered a two-pronged agreement on Duchenne muscular dystrophy with Genzyme Corp., in which the latter will have access to its proprietary cell-based assays to screen for lead molecules and also will take an option on a preclinical small-molecule drug discovery program for Duchenne and related diseases.

Terms were not disclosed, but Cambridge, Mass.-based Genzyme is paying Santhera a technology license fee for gaining nonexclusive access to its screening platform. Genzyme will assess the program before deciding whether it will proceed further with a licensing deal, said Santhera's vice president for business development in the U.S., Matt Woker.

"It's a market in which, in a way, you have to collaborate with Genzyme if you want to go with a big partner," he said. Few companies are working in the area.

Duchenne, an X-linked inherited disorder, is caused by mutations in the gene encoding dystrophin, which plays a structural role in muscle cells. It Ieads to muscle weakness, progressive loss of function, severe physical disability and early mortality. Some 20,000 individuals in industrialized countries are diagnosed with the condition every year. "It's the genetic disease most frequently diagnosed in children today," Woker said.

Santhera, now headquartered in Liestal, Switzerland, has not publicly disclosed the target associated with the program Genzyme has optioned. It has a second preclinical program in Duchenne, based on finding small-molecule inhibitors of calpain, a protease that functions in the presence of high levels of calcium, which, in turn, are caused by cell membrane lesions. That activity is thought to contribute to progressive muscle wasting.

PTC Therapeutics Inc., of South Plainfield, N.J., is taking an alternative approach. It hopes to correct nonsense mutations in a subset of Duchenne cases through the use of an orally available small molecule, PTC124, which promotes translational read-through and restoration of protein function. The program has entered a Phase I trial in Duchenne and cystic fibrosis. The calpain inhibition program is one to two years from the clinic, Woker said. "I think there is space [in the market] for orphan players. Whoever arrives there first will do well," he said.