Amicus Therapeutics Inc. has suspended a midstage study of its developing Pompe disease treatment after two patients suffered what were described as serious side effects. Amicus’ stock dropped nearly 1/3, to just over $8, after the announcement was made on Friday, February 27.

Two of the children of Amicus CEO John Crowley suffer from the rare fatal disease. Pompe, a progressive disease, affects respiratory function and muscles, including the heart. Crowley has devoted himself to finding better treatments for the disease since soon after his children, Patrick and Megan, now pre-teens, were diagnosed with the disease. Megan was just 15-months-old and Patrick was 5-months-old. Doctors held out no hope, and the Crowleys soon learned that not only was there no cure, but that the disease was so rare that there was little research being directed toward finding one.

Crowley quit his job as a financial consultant and invested himself and his life savings in a biotechnology start-up company. In just over a year, Novazyme Pharmaceuticals, went from an endowment of $37,000 to $27 million, and was sold to Genzyme soon thereafter for $137.5 million. Crowley’s children began receiving treatment developed by Genzyme in 2003.

Crowley went on to found Amicus, which has grown to 120 employees, and is focused on finding cures for rare genetic diseases. The treatment Crowley’s company has developed for Pompe would be a step forward, says COO Matthew Patterson, in that it would be an oral dose rather than the multi-hour infusion that is now administered to Pompe patients, and that causes serious side effects. The company hopes that it would also be more effective.

Patterson says that the two patients whose conditions caused the Phase II study of the treatment to be halted suffered muscle weakness. The next step, he says, will be “to work with the doctor caring for the patients.” The company’s science team will then “think about what we’ve learned” and will most likely propose an amended treatment to the FDA, the agency that regulates pharmaceutical trials. That amendment most likely would be a decreased dose, he says.

Patterson says that there is not yet a timeline for resuming the trials. “The whole situation is preliminary,” he says. “It takes time. But we hope to get the trail re-started soon.”

Asked how disappointed Crowley is in the trial interruption, Patterson says “John has learned to balance his personal situation with the needs of the company. He’s disappointed, as we all are, but he understands it is what happens.”

Patterson emphasizes that the Pompe treatment is just one of three Amicus products, and is the one that is in the most preliminary stages. The company is working on treatments for two other rare diseases. They are Amigal, for Fabry disease, and Plicera, for Gaucher disease. Amigal is soon to start Phase III testing, and Plicera is in the middle of Phase II.

The story of Crowley’s quest to find a cure for his children is the subject of a book, “The Cure: How a Father Raised $100 million and Bucked the Medical Establishment in a Quest to Save His Children”, by Wall Street Journal reporter Geeta Anand. The book is being made into a movie with the working title “Crowley”. According to IMbD, the Internet movie database, casting is largely complete. Actors who have signed on include Harrison Ford, Brendan Fraser, and Keri Russell. Filming is to begin in early-April. Patterson says that Crowley and his wife, Aileen Crowley, have been involved in shaping the movie.

Amicus Therapeutics, 5 Cedar Brook Drive, Cranbury 08512; 609-662-2000; fax, 609-662-2001. John F. Crowley, CEO. www.amicustherapeutics.com.

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