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BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease

Posted on: January 11, 2016

Reveglucosidase Alfa Trends Positive in Respiratory Muscle Strength and Endurance Endpoints in Patients Previously Treated With Enzyme Replacement Therapy

SAN RAFAEL, Calif., Jan. 11, 2016 (GLOBE NEWSWIRE)—BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced interim results from INSPIRE, a Phase 2 trial for reveglucosidase alfa, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) being studied for the treatment of late-onset Pompe disease (LOPD). The interim efficacy and safety analysis is based on 24 patients who previously had been on treatment with the enzyme replacement therapy, alglucosidase alfa, and were switched to reveglucosidase alfa.

Investigators indicated that, while on treatment with alglucosidase alfa, the majority of the patient population were considered to have worsening of their Pompe disease over the last 12 months. At week 24, the 18 patients on treatment with reveglucosidase alfa and who completed the study demonstrated respiratory muscle improvements with a mean increase of 2.2 points from baseline in percent predicted Maximal Inspiratory Pressure (MIP) and a mean increase of 3.1 points from baseline in percent predicted Maximal Expiratory Pressure (MEP). Patients completing the study also experienced a mean improvement of 26.1 meters in 6 Minute Walk Test (6MWT). In the 14 patients who met eligibility at both screening and baseline and completed the study, a mean increase of 3.8 points from baseline in percent predicted MIP also was observed. The 18 patients completing the study showed a mean decrease of 3.7 points from baseline in percent predicted Forced Vital Capacity (FVC), but were considered relatively unchanged from screening at -0.7 points in percent predicted. BioMarin will present these data at an upcoming medical meeting.

“We are encouraged by the positive trends in respiratory muscle strength as measured by pressures, which may indicate a possible halt in decline or improvement in lung capacity and endurance in late-onset Pompe disease,” said Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer at BioMarin. “Reveglucosidase alfa has the potential to be an additional choice for Pompe patients, and we look forward to working with health authorities and the patient community to advance this experimental therapy to the next stage of clinical development.”

“Pompe is a progressive and debilitating disease, and patients need additional treatment options. The reveglucosidase alfa data appears promising and could be a potential new enzyme replacement therapy that could make a meaningful difference to late-onset Pompe patients,” said Professor Benedikt Schoser of the Friederich-Baur Institute and speaker of the German working group for Pompe disease.

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