Abstract Details

Title 90-month Muscle Function and Biomarker Outcomes With Cipaglucosidase Alfa Plus Miglustat (Cipa+Mig) in Adults With Pompe Disease in ATB200-02, an Open-label Phase I/II Study

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S7 - Updates on Muscle Disorders (4:30 PM-4:42 PM)

Poster/Presentation
Number 006

Objective

ATB200-02 (NCT02675465) evaluated cipa+mig in adults with Pompe disease. We report muscle function and biomarker outcomes up to month 90.

Background

Pompe disease is a progressive disease that can lead to irreversible muscle damage.

Design/Methods

Four patient cohorts were included: three ambulatory (two enzyme replacement therapy [ERT] experienced [2–6 and ≥7 years] and one ERT naïve) and one non-ambulatory, ERT-experienced cohort. All patients received cipa+mig (20mg/kg intravenous + 260mg oral) biweekly. Assessments included 6-minute walk distance (6MWD); Gait, Stairs, Gowers’ maneuver, Chair (GSGC); creatine kinase (CK); hexose tetrasaccharide (Hex4); safety.

Results

Twenty-nine patients enrolled (ERT experienced, ambulatory: n=17; ERT naïve, ambulatory: n=6; ERT experienced, non-ambulatory: n=6); 24 completed the study, with five discontinuations. In ambulatory patients, mean (standard deviation [SD]) baseline % predicted 6MWD was 60.2 (16.20)% and 67.8 (12.61)% for ERT-experienced and ERT-naïve groups, respectively. Mean (SD) change from baseline (CFBL) to month 60 was +4.8 (9.08)% and −1.5 (19.71)%, and +2.7 (9.20)% and +0.9 (15.00)% to month 90. Mean (SD) GSGC score improved from 14.3 (5.23) and 12.8 (3.55) at baseline in ERT-experienced and ERT-naïve ambulatory patients to months 60 (mean [SD] CFBL −1.2 [5.41] and −0.8 [2.32]) and 90 (−1.2 [4.49] and −0.5 [2.12]). ERT-experienced and ERT-naïve ambulatory patients had improvements in mean (SD) percent CFBL in CK (−46.5 [12.13]% and −53.8 [10.03]%) and Hex4 (−20.3 [47.28]% and −2.3 [133.95]%) at month 90. Non-ambulatory, ERT-experienced patients had improvements in CK and Hex4 at month 60 (mean [SD] percent CFBL −12.3 [43.91]% and −2.9 [25.32]%); 90-month data not available.

Conclusions

Cipa+mig was generally well tolerated up to month 90. Across patient populations, long-term treatment with cipa+mig demonstrated durable stability or improvements in muscle function and biomarker outcomes over 7.5 years, with consistent trends despite the inherent variability of small patient cohorts, especially at later time points. Supported by Amicus Therapeutics, Inc.

Authors/Disclosures
Alberto Di Ronza, PhD (Amicus Therapeutics) PRESENTER	Dr. Di Ronza has received personal compensation for serving as an employee of Amicus Therapeutics. Dr. Di Ronza has or had stock in Amicus Therapeutics.
Drago Bratkovic	Drago Bratkovic has nothing to disclose.
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.
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.
Ozlem Goker-Alpan, MD	Dr. Goker-Alpan has received personal compensation for serving as an employee of LDRTC. Dr. Goker-Alpan has received personal compensation for serving as an employee of LDRTC. Dr. Goker-Alpan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Goker-Alpan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Goker-Alpan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sanofi. Dr. Goker-Alpan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Chiesi. The institution of Dr. Goker-Alpan has received research support from Sanofi. The institution of Dr. Goker-Alpan has received research support from AMICUS. The institution of Dr. Goker-Alpan has received research support from ASTELLAS.
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.
Tahseen Mozaffar, MD, FAAN (University of California Irvine)	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.
Mark Roberts	Mark Roberts has nothing to disclose.
Nadine V. Van Der Beek, MD, PhD (Erasmus MC)	The institution of Dr. Van Der Beek has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. The institution of Dr. Van Der Beek has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bayer. The institution of Dr. Van Der Beek has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus Therapeutics.
Farah Amon, MS	Mrs. Amon has received personal compensation for serving as an employee of Amicus Therapeutics. Mrs. Amon has stock in Amicus Therapeutics.
Brian Fox, PhD (Amicus Therapeutics)	Dr. Fox has received personal compensation for serving as an employee of Amicus Therapeutics. Dr. Fox has stock in Amicus Therapeutics.
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.
Vipul Jain, Programmer	Mr. Jain has nothing to disclose.
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.

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