Amicus Therapeutics Announces Positive 18-Month Data in Pompe Disease Phase 1/2 Study

Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society

CRANBURY, N.J., Oct. 04, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) today announced additional positive results from a global Phase 1/2 clinical study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen (disease substrate) in cells.

Amicus Therapeutics Announces Positive 18-Month Data in Pompe Disease Phase 1/2 Study

Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease

CONCORD, Mass., July 11, 2017 /PRNewswire/ — Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced that it has initiated dosing in a Phase 1/2 clinical trial evaluating VAL-1221 in patients with late-onset Pompe disease.

Amicus Therapeutics Announces Positive 18-Month Data in Pompe Disease Phase 1/2 Study

Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study

CRANBURY, N.J., (May 15, 2017) (GLOBE NEWSWIRE) – Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC).

Amicus Therapeutics Announces Positive 18-Month Data in Pompe Disease Phase 1/2 Study

Valerion Reports Preclinical Potential for Pompe Treatment

CONCORD, Mass. – (April, 2017) – Valerion Therapeutics, part of the Alopexx Enterprises portfolio of companies, has announced the development of VAL-1221, a fusion protein that combines its technology with recombinant human acid alpha-glucosidase (rhGAA) to improve delivery of rhGAA to affected tissues in patients with Pompe disease.