ATB200/AT2221 Safety Data Show No Infusion-Associated Reactions Following 100+ Infusions
Clinical Pharmacokinetic (PK) Profile as Predicted Based on Previously Reported Preclinical Data
Biomarkers of Muscle Damage are Generally Stable or Trending Towards Improvement
Conference Call at 8:30am ET
CRANBURY, NJ, USA I December 08, 2016 I Amicus Therapeutics (FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/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.
ATB200-02 Study – Design and Objectives
- Primary objectives: to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221
- Study duration: 18-week primary treatment period with all patients eligible for a long-term extension
- Patient cohorts: ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2) and ERT-naïve patients (Cohort 3). Enrolling up to ~20 total patients across all cohorts.
- Preliminary data now available:
- Safety data for nine patients through interim data analysis (maximum 24 weeks)
- PK and PD (muscle biomarker) data through Week 14 for four patients in Cohort 1
- Safety data for nine patients through interim data analysis (maximum 24 weeks)
- PK and PD (muscle biomarker) data through Week 14 for four patients in Cohort 1
ATB200-02 Study – Preliminary Data Highlights in Initial ERT-Switch Patients
- ATB200/AT2221 safety measures (n=9) showed:
- No serious adverse events (SAEs)
- AEs were generally mild and transient
- No serious adverse events (SAEs)
- AEs were generally mild and transient
- To date, ATB200/AT2221 has shown no infusion-associated reactions following 100+ infusions
- Clinical PK profile through Week 14 was as predicted based on previously reported preclinical data (n=4).
- ATB200 plasma clearance suggests optimized carbohydrate structure provides efficient uptake into tissues
- ATB200 alone showed greater than dose-proportional increases in exposure, which was further enhanced with the addition of the chaperone AT2221
- ATB200 plasma clearance suggests optimized carbohydrate structure provides efficient uptake into tissues
- ATB200 alone showed greater than dose-proportional increases in exposure, which was further enhanced with the addition of the chaperone AT2221
- Biomarkers of muscle damage were trending toward improvement or stable through Week 14 (n=4)
- Biomarkers of muscle damage (creatine kinase (CK) enzyme, alanine aminotransferase (ALT), and aspartate aminotransferase (AST)) in the initial four ERT-switch patients showed early trends toward improvement in two patients and were stable in the other two patients.
- Biomarkers of muscle damage (creatine kinase (CK) enzyme, alanine aminotransferase (ALT), and aspartate aminotransferase (AST)) in the initial four ERT-switch patients showed early trends toward improvement in two patients and were stable in the other two patients.
“We set out on this journey in Pompe disease at Amicus with the intent to develop a new treatment paradigm,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “These preliminary results, while still early, give us greater confidence that we are developing a drug regimen that may be highly differentiated from any other approach. Even at this interim analysis, it is very encouraging to see no infusion-associated reactions in these initial patients, as well as the desired PK profile and improvements in key muscle damage biomarkers in two of four patients. We have much more work to do to generate additional important data in the months ahead but this is an excellent foundation for this program and for persons living with this devastating disorder.”
Barry Byrne, M.D., Ph.D., Professor, Pediatrics and Molecular Genetics & Microbiology and Director, University of Florida Powell Center, stated, “As a lead investigator in the ATB200-02 study, I have had a positive initial experience with the novel Amicus treatment regimen for Pompe. Elevated levels of CK, ALT and AST indicate damage to muscle tissue in patients with Pompe disease. A reduction in markers of muscle damage, such as CK, ALT, and AST in patients with Pompe disease is very encouraging, especially with favorable safety and tolerability. If these positive trends continue, ATB200/AT2221 may help to address the significant unmet need among Pompe patients. I look forward to seeing additional data from this clinical study.”
Conference Call and Webcast
Amicus Therapeutics will host a conference call and webcast today, December 8, 2016 at 8:30 a.m. ET. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international). The slide presentation to accompany this conference call and webcast will be available at http://ir.amicusrx.com/events.cfm.
An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/events.cfm, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 34348280.
About ATB200/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. Amicus Therapeutics is currently conducting a global Phase 1/2 study (ATB200-02) to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of ATB200/AT2221.
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.
About Amicus Therapeutics
Amicus Therapeutics (FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.
SOURCE: Amicus Therapeutics
Post Views: 49
ATB200/AT2221 Safety Data Show No Infusion-Associated Reactions Following 100+ Infusions
Clinical Pharmacokinetic (PK) Profile as Predicted Based on Previously Reported Preclinical Data
Biomarkers of Muscle Damage are Generally Stable or Trending Towards Improvement
Conference Call at 8:30am ET
CRANBURY, NJ, USA I December 08, 2016 I Amicus Therapeutics (FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/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.
ATB200-02 Study – Design and Objectives
- Primary objectives: to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221
- Study duration: 18-week primary treatment period with all patients eligible for a long-term extension
- Patient cohorts: ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2) and ERT-naïve patients (Cohort 3). Enrolling up to ~20 total patients across all cohorts.
- Preliminary data now available:
- Safety data for nine patients through interim data analysis (maximum 24 weeks)
- PK and PD (muscle biomarker) data through Week 14 for four patients in Cohort 1
- Safety data for nine patients through interim data analysis (maximum 24 weeks)
- PK and PD (muscle biomarker) data through Week 14 for four patients in Cohort 1
ATB200-02 Study – Preliminary Data Highlights in Initial ERT-Switch Patients
- ATB200/AT2221 safety measures (n=9) showed:
- No serious adverse events (SAEs)
- AEs were generally mild and transient
- No serious adverse events (SAEs)
- AEs were generally mild and transient
- To date, ATB200/AT2221 has shown no infusion-associated reactions following 100+ infusions
- Clinical PK profile through Week 14 was as predicted based on previously reported preclinical data (n=4).
- ATB200 plasma clearance suggests optimized carbohydrate structure provides efficient uptake into tissues
- ATB200 alone showed greater than dose-proportional increases in exposure, which was further enhanced with the addition of the chaperone AT2221
- ATB200 plasma clearance suggests optimized carbohydrate structure provides efficient uptake into tissues
- ATB200 alone showed greater than dose-proportional increases in exposure, which was further enhanced with the addition of the chaperone AT2221
- Biomarkers of muscle damage were trending toward improvement or stable through Week 14 (n=4)
- Biomarkers of muscle damage (creatine kinase (CK) enzyme, alanine aminotransferase (ALT), and aspartate aminotransferase (AST)) in the initial four ERT-switch patients showed early trends toward improvement in two patients and were stable in the other two patients.
- Biomarkers of muscle damage (creatine kinase (CK) enzyme, alanine aminotransferase (ALT), and aspartate aminotransferase (AST)) in the initial four ERT-switch patients showed early trends toward improvement in two patients and were stable in the other two patients.
“We set out on this journey in Pompe disease at Amicus with the intent to develop a new treatment paradigm,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “These preliminary results, while still early, give us greater confidence that we are developing a drug regimen that may be highly differentiated from any other approach. Even at this interim analysis, it is very encouraging to see no infusion-associated reactions in these initial patients, as well as the desired PK profile and improvements in key muscle damage biomarkers in two of four patients. We have much more work to do to generate additional important data in the months ahead but this is an excellent foundation for this program and for persons living with this devastating disorder.”
Barry Byrne, M.D., Ph.D., Professor, Pediatrics and Molecular Genetics & Microbiology and Director, University of Florida Powell Center, stated, “As a lead investigator in the ATB200-02 study, I have had a positive initial experience with the novel Amicus treatment regimen for Pompe. Elevated levels of CK, ALT and AST indicate damage to muscle tissue in patients with Pompe disease. A reduction in markers of muscle damage, such as CK, ALT, and AST in patients with Pompe disease is very encouraging, especially with favorable safety and tolerability. If these positive trends continue, ATB200/AT2221 may help to address the significant unmet need among Pompe patients. I look forward to seeing additional data from this clinical study.”
Conference Call and Webcast
Amicus Therapeutics will host a conference call and webcast today, December 8, 2016 at 8:30 a.m. ET. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international). The slide presentation to accompany this conference call and webcast will be available at http://ir.amicusrx.com/events.cfm.
An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/events.cfm, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 34348280.
About ATB200/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. Amicus Therapeutics is currently conducting a global Phase 1/2 study (ATB200-02) to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of ATB200/AT2221.
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.
About Amicus Therapeutics
Amicus Therapeutics (FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.
SOURCE: Amicus Therapeutics
Post Views: 49
