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A life-saving biotech with a personal mission

Amicus Therapeutics CEO: Driving science to a cure
Amicus Therapeutics CEO: Driving science to a cure

Jim Cramer has been on a mission to find the hottest biotechs of 2015. While reviewing all of the top performers of last year, he has found one that not only doubled in value last year but has a personal mission to find a life-saving cure.

"That's some incredible performance, and I think there could be even more upside here if you're willing to take the risk that's inherent in owning a company this small," Cramer said.

John Crowley is CEO of Amicus Therapeutics, a father and a Navy reservist who fought to raise money to become a biotech entrepreneur. He didn't do this just to make money; he did it in an effort to save his two children from a rare condition called Pompe disease.

John Crowley, Amicus CEO
Scott Mlyn | CNBC

Wednesday celebrates the five-year anniversary of the movie "Extraordinary Measures," starring Brendan Frasier and Harrison Ford, which chronicled Crowley's devotion to saving his children's lives.

Pompe is a neuromuscular disease that can ultimately be fatal, even with enzyme replacement therapy. Amicus has a drug in Phase 2 trials that leverages their advanced enzyme replacement chaperone platform.

To find out more, Cramer spoke with Crowley on how his personal passion has fed into the passion of the company culture and stock.

"We got involved in biotech in the late 90s, when two of our children were diagnosed with a rare genetic disease…at the time there was nothing," said Crowley "So we really focused as parents initially on what could we do to help drive science to a cure. That was our initial effort, and that led to the development to a first generation treatment that our kids have been on for more than a decade. It saved their lives."

Amicus is a company that develops treatments for rare orphan diseases that need good medications. Biotechs that specialize in orphan diseases have been on the radar of big biotech recently, such as the acquisition of NPS Pharma by Shire, which indicates to Cramer that these companies could be headed higher in the future.

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The company is specifically working on treatments for lysosomal storage disorders, a genetic condition. The current standard of care is done with enzyme replacement, and Amicus has a drug that stabilizes the patient's enzymes that naturally occur in the body.

"We are always working in devastating disorders where there are tremendous burdens on the people living with the disease, the family members and the people that care for them. What we want to do is develop these life-saving therapies," Crowley added.

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