Mean Six-Minute Walk Distance at Month Six Improved in
ERT-Naïve Patients (+52 Meters) and ERT-Switch Patients (+38 Meters)
Muscle Function Improved in 9 out of 10 Patients
Pulmonary Function Improved in a Majority of Patients
No Infusion-Associated Reactions Following 200+ Infusions
Conference Call at
"We are very pleased to see improvements in six minute walk distance and other measures of motor function in both naïve and ERT-switch patients, as well as stability or improvements in forced vital capacity. The consistency and magnitude of improvements exceeded our expectations and follow the initial improvements seen on key biomarkers of muscle damage and disease substrate," said
ATB200-02 Study - Updated Data Highlights in Initial ERT-Switch and Naive Patients
Safety, Tolerability & Pharmacokinetics (PK)
Safety and tolerability data are currently available for all 20 patients enrolled in the study (maximum 48 weeks). To date, adverse events have been generally mild and transient. Importantly, ATB200/AT2221 has also shown no infusion-associated reactions following 200+ infusions. As previously reported, the clinical PK profile has been consistent with previously reported preclinical data.
Pharmacodynamic (PD) Data on Muscle Damage and Disease Substrate Biomarkers (n=16)
PD data are currently available for 11 ERT-switch patients and five ERT-naïve patients. Improvements in key biomarkers of muscle damage and disease substrate continue to suggest a positive effect of ATB200/AT2221 on muscle cells after up to 34 weeks of treatment.
Functional Outcomes at Month 6 (n=10)
Functional outcomes data from baseline to Month 6 are currently available for 10 patients (seven ambulatory ERT-switch, two ERT-naïve and one non-ambulatory ERT-switch). Motor function improved and pulmonary function was stable in ambulatory ERT-switch patients; motor and pulmonary function both improved in ERT-naïve patients. Muscle strength data are available from the first non-ambulatory ERT-switch patient and showed improvement.
- Motor function: Six-minute walk test (6MWT) distance, a primary measure of motor function in Pompe patients, increased in both ERT-switch patients (mean +38 meters; improvement in 6/7 patients) and ERT-naïve patients (mean +52 meters; improvement in 2/2 patients). Other motor function tests also showed mean improvements, consistent with 6MWT distance.
- Muscle Strength: In the first non-ambulatory ERT-switch patient, improvements in four out of four muscle groups on the quantitative muscle testing (QMT) and two of three muscle groups on the manual muscle testing (MMT) were observed.
Summary of Functional Outcomes from Baseline to Month 6
|Cohort 1 ERT-Switch Patients (n=7): Functional Outcomes on ATB200/AT2221 from Baseline to Month 6|
|Motor Function Tests (n=7)||Pulmonary Function Tests (n=6-7)|
|6MWT (m)||4 Stair |
|Timed up |
and go (sec)
|10m walk |
|383 (103)||4.4 (3.1)||11.0 (7.7)||7.5 (3.5)||51 (17)||35.4 (11.3)||69.5 (21.2)|
|Change from |
|+38 (43)||-1.1 (1.3)||-1.9 (2.8)||-0.04 (1.6)||+0.3 (3)||+1.0 (5.2)||+15.5 (25.4)|
|Cohort 3 ERT-Naïve Patients (n=2): Functional Outcomes on ATB200/AT2221 from Baseline to Month 6|
|Motor Function Tests (n=2)||Pulmonary Function Tests (n=2)|
|6MWT (m)||4 Stair |
|Timed up |
and go (sec)
|10m walk |
|432 (68)||3.9 (0.6)||8.9 (0.9)||6.9 (0.8)||51 (27)||45.5 (27.6)||57.5 (9.2)|
|Change from |
|+52 (15)||-0.3 (0.0)||-1.4 (0.4)||-0.5 (0.2)||+3 (0)||+8.5 (3.5)||-4.5 (17.7)|
Conference Call and Webcast
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About ATB200-02 Clinical Study
The primary objectives of the open-label ATB200-02 clinical study are to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221 over an 18-week primary treatment period followed by a long-term extension. The study enrolled a total of 20 patients across three patient cohorts: ambulatory ERT-switch (Cohort 1, n=11), non-ambulatory ERT-switch (Cohort 2, n=4) and ERT-naïve (Cohort 3, n=5). Patients in Cohort 1 received escalating doses of ATB200 (5, 10, 20 mg/kg), followed by 3 doses of 20 mg/kg ATB200 plus low dose AT2221, followed by ongoing doses of 20 mg/kg ATB200 plus high dose AT2221. Patients in Cohort 2 and 3 patients have all received 20 mg/kg ATB200 plus high dose AT2221.
ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced glycogen levels in muscle, which was further improved when AT2221 was co-administered with ATB200.
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Progressive accumulation of glycogen is believed to lead to the morbidity and mortality associated with Pompe disease, including muscle weakness and respiratory insufficiency.
1Johnson, et. al, WORLDSymposium 2017, First-in-human preliminary pharmacokinetic and safety data on a novel recombinant acid-α-glucosidase, ATB200, co-administered with the pharmacological chaperone, AT2221, in ERT-experienced Pompe patients
This press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to encouraging preliminary data from a global Phase 1/2 study to investigate ATB200/AT2221 for the treatment of Pompe and the potential implications on these data for the future advancement and development of ATB200/AT2221. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "confidence," "encouraged," "potential," "plan," "targets," "likely," "may," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the preliminary data based on a small patient sample and reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the preliminary data presented, that the Company will be not able to demonstrate the safety and efficacy of ATB200/AT2221, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize ATB200/AT2221. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended
Amicus Therapeutics Sara PellegrinoSenior Director, Investor Relations firstname.lastname@example.org (609) 662-5044 Media: MWW PR Sid Dinsaysdinsay@mww.com (646) 381-9017
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