Amicus Therapeutics revealed its success in a small midstage study of its drug to enhance enzyme therapy in patients with the rare genetic disorder Pompe disease. On the back of the upbeat data, the biotech company's shares recovered some of the lost ground from news last month of a Phase III failure for its lead candidate.
Its shares had jumped 17.88% to $3.89 as of 9:34 a.m. on Friday.
The Cranbury, NJ-based company ($FOLD) has a pipeline of chaperone therapies for patients with rare diseases, and its Pompe drug is one of two Phase II programs under the hood that follow its Phase III contender for Fabry disease that is partnered with GlaxoSmithKline ($GSK). The new results today showed how the Pompe drug, AT2220, used with enzyme replacement therapies (ERT) from Sanofi's ($SNY) Genzyme, boosted levels of the enzyme in the blood and activity in muscle tissue compared to treatment on enzyme replacements alone.
Amicus aims to launch a repeat-dose Phase II study of AT2220 in the third quarter of 2013 to further weigh the impact of the candidate's use with the enzyme drugs. Patients with Pompe, which affects 1 in 40,000 births, have mutations in a gene for an enzyme call GAA that lead to buildups of glycogen in tissues around the body. Genzyme has the only approved therapies for the disease, Myozyme and Lumizyme, which are pricy drugs that replace the lacking GAA enzyme in patients.
In the results revealed today, the biotech showed increases in GAA levels in all four groups of patients on its drug. There was no change in enzyme activity in the muscle of patients on the lowest (50 mg) dose of AT2220, yet the study showed a 133% increase in patients on the highest (600 mg) dose.
"We believe that co-administration may deliver significant benefits compared to ERT alone and become an important therapy for people with Pompe disease," Amicus CEO John Crowley said in statement.
Still, the major driver at Amicus remains its late-stage candidate Amigal in another enzyme-related disorder called Fabry disease. The company and partner GSK are awaiting 12-month data on the treatment later this year from the first Phase III study, with results of a second late-stage trial due in 2014.
- here's the release
Related conversation on 
Twitter :
-
Amicus announces positive Phase II data for Pompe disease. But was the data leaked early?
With input from @RyanMFierce