Amicus Therapeutics Receives Positive Results in Pompe Disease Study

Andrew Klips  |

After missing earlier this month on achieving a statistically significant response in a phase 3 study with partner GlaxoSmithKline (GSK) for migalastat HCl against Fabry disease that chopped their market cap in half, Amicus Therapeutics, Inc. (FOLD) is recovering a portion of the lost value.  Shares bounced of lows of $2.47 on December 27 to close on Thursday at $3.30.  Friday morning, the Cranbury, New Jersey-based biotech said that it has received positive preliminary results from all four dose cohorts in an open-label, multi-center Phase 2 study (Study 010) evaluating the pharmacological chaperone AT2220 (duvoglustat HCl) for Pompe disease.

Study 010 is evaluating the safety and pharmacokinetic effects of four increasing oral doses of AT2220 (50 mg, 100 mg, 250 mg, or 600 mg) co-administered with enzyme replacement therapies (ERT) Myozyme® or Lumizyme® versus ERT alone in males and females with Pompe disease.

Myozyme or Lumizyme are the only two FDA approved treatments for Pompe disease, also called glycogen storage disease type II.  Pompe disease is a neuromuscular disorder affecting people of all ages causing muscle weakness as the result of deficient GAA enzymes leading to accumulation of glycogen in tissue.

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Pompe disease affects between 5,000 and 10,000 people worldwide.

Preclinical data by Amicus in a mouse model of Pompe disease testing the combination of AT2220 and ERT enhanced rhGAA enzyme activity, reduces glycogen accumulation, and showed the potential to mitigate ERT-related immunogenicity. In Study 010, co-administration of AT2220 to Pompe patients increased rhGAA enzyme activity and enhanced rhGAA enzyme uptake into muscle tissue compared to ERT alone, according to today’s corporate statement.

With the study establishing human proof-of-concept that the combination treatment increases GAA enzyme activity, Amicus plans to initiate a repeat-dose clinical study analyzing the treatment on additional clinical measures.  “We believe that co-administration may deliver significant benefits compared to ERT alone and become an important therapy for people with Pompe disease,” said John F. Crowley, Chairman and Chief Executive Officer at Amicus Therapeutics.

Shares of FOLD are tracking higher by 11 percent in pre-market activity on Friday with the positive report.  Trading near $3.70 per share, the company has risen about 50 percent from lows five days ago, but is still far below the $6 per share mark that it was trading near when the Fabry disease data was released on December 19.  Apropos, at this point Amicus is still committed to advancing migalastat as a potential new therapy for Fabry disease.

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