Wednesday, June 20, 2012

Competitors Form Partnership to Develop Autism Drugs



Two of the front-runners in the race to develop drugs to treat mental retardation and autism are joining forces, hoping to save money and get to the market sooner.
A deal, expected to be announced on Tuesday, will pool the resources of Roche, the Swiss pharmaceutical giant, and Seaside Therapeutics, a private 30-employee company based in Cambridge, Mass.
“This deal will establish the biggest effort to date” in autism drugs, Luca Santarelli, head of neuroscience for Roche, said before the announcement. Financial terms are not being disclosed.
There is rising excitement that drugs might be able to relieve some of the behavioral problems associated with autism and in particular a cause of autism and mental retardation known as fragile X syndrome. About 100,000 Americans have fragile X syndrome.
Some parents of children being treated with new drugs in clinical trials have said they see positive changes in behavior.
Becky Zorovic of Sharon, Mass., said that when she used to take her son Anders, who has fragile X, to the dentist, she would have to lie in the chair and hold him on top of her as he screamed.
But after Anders starting taking Seaside’s drug, arbaclofen, in a clinical trial, she said, “He sat in the chair by himself and he opened his mouth and let the dentist polish his teeth and even scrape his teeth.” Anders has also has gone to birthday parties, which he once refused to do, she said.
Still, Ms. Zorovic said that until the clinical trial comparing the drug to placebo was completed, it was difficult to say whether the effects were from the drugs or merely a result of parents’ perceptions.
The mechanism that has perhaps shown the most promise, at least in mice, is to damp signaling in the brain by blocking a receptor called mGluR5.
Novartis is considered ahead in developing such a drug. Roche is next, having entered the midstage of clinical trials. Seaside is further back but has patents covering the use of mGluR5 antagonists to treat neurodevelopmental disorders.
Under the deal, Roche will get exclusive rights to those patents from Seaside. That might provide some leverage that Roche can use against Novartis, although Dr. Santarelli said Roche had no intention of keeping a rival’s drug off the market.
Roche also gets the option to license commercial rights to Seaside’s arbaclofen. That drug, which works somewhat differently, is in late-stage testing for fragile X and in midstage testing for autism.
Roche will provide money to help Seaside complete its clinical trials of arbaclofen. Seaside will halt development of its own mGluR5 antagonist, which it licensed from Merck, and will instead receive royalties on sales of Roche’s drug.
The alliance could pose a challenge to Novartis. “This is No. 2 and No. 3 ganging up on No. 1,” said Dr. Michael Tranfaglia, medical director of the Fraxa Research Foundation, which sponsors research into treatments for fragile X syndrome.
Dr. Randall L. Carpenter, chief executive of Seaside, said the money from Roche was a needed diversification of the company’s funding. Virtually all of the $90 million Seaside has raised has come from the Barony Trust, which is run by Peter Whipp, a British investment manager.
“They would like to see new treatments,” Dr. Carpenter said, referring to Mr. Whipp and his family. “I think they carried us a long way, longer than they wanted to.”

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