Amicus Therapeutics ($FOLD) plans to quickly ramp up a new Phase II study of its experimental therapy for Pompe disease now that the FDA has decided to lift a clinical hold on the program. The Cranbury, NJ-based biotech says that it expects to have Phase II data available before the end of the year on AT2220.
Amicus's drug ran afoul of regulators after researchers reported that two patients in the initial Phase II study--which used a high dose as a monotherapy for patients with Pompe disease--experienced serious adverse effects that were likely linked to the therapy. In this new study, researchers plan to use a combo treatment using an enzyme replacement therapy.
"The addition of a pharmacological chaperone has been shown to prevent the loss of activity of ERT in the circulation, increase tissue uptake and increase substrate reduction in multiple disease-relevant tissues," the company said in its release. "Preclinical proof of concept has been established for Fabry disease and Pompe disease."
Amicus Therapeutics CEO John Crowley has been an enthusiastic cheerleader for the company's prospects in the rare disease field. He has vowed to follow in the footsteps of Genzyme, which helped illustrate the huge rewards that could be obtained from orphan drugs.
- here's the Amicus release