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Abstract Details

Long-term Follow-up of Cipaglucosidase Alfa/Miglustat in Ambulatory Patients with Pompe Disease: An Open-label Phase I/II Study (ATB200-02)
Neuromuscular and Clinical Neurophysiology (EMG)
S48 - Therapeutics for Muscle Disease (4:42 PM-4:54 PM)
007

The ongoing ATB200-02 (NCT02675465), open-label, Phase I/II clinical trial aims to evaluate the long-term efficacy and safety of cipaglucosidase alfa/miglustat in adults with Pompe disease.

Cipaglucosidase alfa/miglustat is an investigational, two-component therapy for Pompe disease. We report data up to 48 months for 6-minute walk distance (6MWD) and % predicted sitting forced vital capacity (FVC) for ATB200-02.

Three adult ambulatory cohorts - two enzyme replacement therapy (ERT)-experienced (2–6 years [n=11] and ≥7 years [n=6]) and one ERT-naïve (n=6) - received 20 mg/kg intravenous-infused cipaglucosidase alfa plus 260 mg miglustat orally biweekly in an ongoing study. Changes from baseline (CFBL) in multiple endpoints were assessed at intervals. We report data from 6, 12, 24, 36 and 48 months.

Baseline characteristics represented the Pompe disease population. Durable improvements occurred at 6, 12, 24, 36 and 48 months in 6MWD (m): pooled analyses of ERT-experienced cohorts, mean(±SD) CFBL: 23.1(±44.75), n=16; 33.5(±49.62), n=16; 25.2(±63.30), n=13; 9.8(±85.98), n=12; 20.7(±101.84), n=9, respectively; ERT-naïve cohort: 36.7(±29.08), n=6; 57.0(±29.96), n=6; 54.4(±36.18), n=6; 43.5(±45.19), n=5; 52.2(±46.59), n=4, respectively. FVC (%) was stable or improved in ERT-experienced cohorts, mean(±SD) CFBL: −0.9(±8.59), n=16; −1.2(±5.95), n=16; 1.0(±7.96), n=13; −0.3(±6.68), n=10; 1.0(±6.42), n=6, respectively, and improved in the ERT-naïve cohort: 4.2(±5.04), n=6; 3.2(±8.42), n=6; 4.7(±5.09), n=6; 6.2(±3.35), n=5; 8.3(±4.50), n=4, respectively. Over 48 months, serum CK and urine Hex4 biomarkers improved in ERT-experienced and ERT-naïve cohorts.

In ATB200-02, ERT-experienced cohorts had durable mean improvements (CFBL) in motor function that were sustained up to 48 months of follow-up; respiratory function was stable and maintained over the same period. The ERT-naïve cohort showed durable mean improvements (CFBL) in motor and respiratory function that were sustained up to 48 months of follow-up. Biomarker outcomes were consistent with other efficacy results. The overall safety profile for ambulatory cohorts was similar to approved ERT. Supported by Amicus Therapeutics, Inc.
Authors/Disclosures
Tahseen Mozaffar, MD, FAAN (University of California Irvine)
PRESENTER 		Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi-Genzyme. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas Gene Therapy. Dr. Mozaffar has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer. Dr. Mozaffar has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Applied Therapeutics. The institution of Dr. Mozaffar has received research support from NIH. The institution of Dr. Mozaffar has received research support from Muscular Dystrophy Association. The institution of Dr. Mozaffar has received research support from Sanofi. The institution of Dr. Mozaffar has received research support from Argenx. The institution of Dr. Mozaffar has received research support from Amicus Therapeutics. The institution of Dr. Mozaffar has received research support from Astellas Gene Therpay. The institution of Dr. Mozaffar has received research support from Cartesian. The institution of Dr. Mozaffar has received research support from Cabaletta. Dr. Mozaffar has received personal compensation in the range of $500-$4,999 for serving as a Study Section Member with NIH.
Benedikt Schoser Benedikt Schoser has received personal compensation for serving as an employee of Alexion. Benedikt Schoser has received personal compensation for serving as an employee of Argenx. Benedikt Schoser has received personal compensation for serving as an employee of Amicus. Benedikt Schoser has received personal compensation for serving as an employee of Astellas. Benedikt Schoser has received personal compensation for serving as an employee of Denali. Benedikt Schoser has received personal compensation for serving as an employee of Avidity . Benedikt Schoser has received personal compensation for serving as an employee of Sanofi. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Taysha. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astellas. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded. Benedikt Schoser has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Kedrion.
Priya Kishnani, MD (Duke University) Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Maze Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for JCR Pharmaceutical. Priya Kishnani, MD has received personal compensation in the range of $500,000-$999,999 for serving as a Consultant for Asklepios Biopharmaceutical Inc. (AskBio). Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi/Genzyme. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus Therapeutics. Priya Kishnani, MD has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Baebies, Inc.. The institution of Priya Kishnani, MD has received research support from Sanofi/Genzyme. Priya Kishnani, MD has received research support from Amicus Therapeutics. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care. Priya Kishnani, MD has received intellectual property interests from a discovery or technology relating to health care.
Drago Bratkovic No disclosure on file
Paula R. Clemens, MD (Univ of Pittsburgh/ Dept of Neurology) The institution of Dr. Clemens has received research support from NS Pharma. The institution of Dr. Clemens has received research support from ReveraGen. The institution of Dr. Clemens has received research support from Amicus. The institution of Dr. Clemens has received research support from Sanofi. The institution of Dr. Clemens has received research support from Spark. The institution of Dr. Clemens has received research support from NIH. The institution of Dr. Clemens has received research support from MDA. The institution of Dr. Clemens has received research support from FDA.
No disclosure on file
Xue Ming, MD (Dept of Neurosciences) Dr. Ming has nothing to disclose.
Mark Roberts Mark Roberts has nothing to disclose.
Kumaraswamy Sivakumar, MD, MRCP Dr. Sivakumar has nothing to disclose.
Ans Van Der Ploeg (Metabolic Disease and Genetics, Erasmus Medic) The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi Genzyme . The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amicus. The institution of Ans Van Der Ploeg has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics . The institution of Ans Van Der Ploeg has received research support from Sanofi Genzyme.
No disclosure on file
No disclosure on file
Frederick Holdbrook Frederick Holdbrook has received personal compensation for serving as an employee of Amicus Therapeutics, Inc. Frederick Holdbrook has stock in Amicus Therapeutics, Inc.
No disclosure on file
No disclosure on file
Yasmine Wasfi No disclosure on file
Barry Byrne Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rocket Pharma. Barry Byrne has received personal compensation in the range of $5,000-$9,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association.