Amicus Therapeutics (FOLD) is purchasing a portfolio of ten gene therapies for $100 million and the promise to pay up to $352 million if the treatments are approved and hit undisclosed sales milestones. The deal arrives just a month after the approval of
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, Amicus’ first drug.
“This is really the next step in putting us in a place where I always hoped Amicus could be,” says John Crowley, Amicus’ chairman and chief executive. But there’s more to this story than most business deals. Both Crowley and one of the executives at the company Amicus is purchasing have children with an extremely rare disease.
Amicus has continued to be a company surrounded by excitement. Crowley’s quest, at a previous company, to create a treatment for his two children, Megan and Patrick, was documented in a number of Wall Street Journal stories, a book, and a movie starring Harrison Ford.
At that time, Amicus’ drug, Galafold, for another extremely rare disease called Fabry disease, was facing questions from the FDA even as it was approved in Europe, Canada, Australia, and Japan to treat patients as long as their illness was stemming from specific mutations. The treatment isn’t cheap however as Galafold costs $315,000 per patient per year in the U.S., which is actually a normal price for a drug for an ultrarare disease.
The Galafold approval allowed Crowley to go forward to identify new assets. He identified them in Celenex, the company Amicus is buying. Its story closely mimics Crowley’s own. Celenex was created by a Hollywood producer, Gordon Gray, whose two children had an awful and rare disease called Batten’s disease, which results in profound and lethal neurological decline.
“John, I’m very aware of your family’s journey,” Gray said the first time they talked. “Twelve years ago I was one of the producers considering bidding on your life rights.”
Crowley says Gray, who had produced films including Secretariat and Invincible, exhibited “superhuman effort” to create a drug company. “He put all of that aside to find the science that could save his daughters.” He found the technology he was looking for in gene therapies created at Nationwide Children’s Hospital Center for Gene Therapy that use a virus, called adeno-associated virus, to insert genetic code into cells. The result was a ten-patient study that tested a gene therapy against the CLN6 version of Batten that Gray’s girls have. The girls were among those 10 patients.
Gray and Crowley first connected over the summer. After a long conversation, Crowley brought a team to Ohio State. After a full day, Crowley asked Gordon and his partner if they could return with him to New Jersey. They flew there and went to Crowley’s house, where Meghan was getting ready to go back to Notre Dame. They spent the next day together at Amicus’ headquarters in Cranbury, New Jersey. Across the negotiating table, Crowley says he made a promise.