- Optimized for Efficient Targeting to Multiple Key Muscle Tissues of Disease
- Significantly Better Than Approved ERT in Reducing Disease Substrate (Glycogen)
- Addition of Chaperone Further Improves Glycogen Reduction
ORLANDO, FL and CRANBURY, NJ, USA I February 10, 2015 I Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today highlighted results from preclinical studies of its next-generation Pompe ERT at WORLDSymposium(TM) 2015 in Orlando, Florida.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Our goal is to develop a fundamentally better therapy for patients living with Pompe disease. The results presented at WORLDSymposium suggest that we have made significant progress with our Pompe program as we look toward addressing some of the major challenges with the current approved therapy including enzyme activity and stability; targeting and uptake; and tolerability and immunogenicity. These results also validate our internal biologics capabilities to develop and manufacture our own cell line at clinical scale. We look forward to moving our next-generation ERT into the clinic later this year.”
Amicus is leveraging its biologics capabilities and CHART(TM) (Chaperone-Advanced Replacement Therapy) platform to develop a next-generation Pompe ERT. This ERT consists of a uniquely engineered recombinant human acid alpha-glucosidase (rhGAA) enzyme (designated ATB200) with an optimized carbohydrate structure to enhance uptake, administered in combination with a pharmacological chaperone to improve activity and stability. In earlier preclinical studies, ATB200 demonstrated greater tissue enzyme levels and further substrate reduction compared to the current approved ERT for Pompe disease (alglucosidase alfa). Clinical studies1,2 of pharmacological chaperones in combination with currently marketed ERTs have established initial human proof-of-concept that a chaperone can stabilize enzyme activity and potentially improve ERT tolerability.
An oral presentation3 and posters3,4 at WORLDSymposium describe updated preclinical results that support clinical development of ATB200 in combination with a chaperone:
- A proprietary cell line and manufacturing processes have been developed to produce ATB200 with optimized carbohydrate structures for efficient cellular uptake and lysosomal targeting
- ATB200 was significantly better than the approved ERT for reducing disease substrate in multiple key muscle tissues in a Pompe animal model (Gaa knock-out mice)
- The addition of a pharmacological chaperone further improved glycogen reduction by ATB200
- ATB200 has been successfully scaled up to 250L production, and is on track to enter the clinic in the second half of 2015.
About Amicus Therapeutics
Amicus Therapeutics (FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as next-generation enzyme replacement therapy (ERT) products for Fabry disease, Pompe disease, and MPS-1.
- Kishnani, et al., LDN WORLD 2013
- Doerfler, et al., LDN WORLD 2014
- Gotschall, et al., WORLDSymposium 2015
- Lun, et al., WORLDSymposium 2015
SOURCE: Amicus Therapeutics
Post Views: 109
- Optimized for Efficient Targeting to Multiple Key Muscle Tissues of Disease
- Significantly Better Than Approved ERT in Reducing Disease Substrate (Glycogen)
- Addition of Chaperone Further Improves Glycogen Reduction
ORLANDO, FL and CRANBURY, NJ, USA I February 10, 2015 I Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today highlighted results from preclinical studies of its next-generation Pompe ERT at WORLDSymposium(TM) 2015 in Orlando, Florida.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Our goal is to develop a fundamentally better therapy for patients living with Pompe disease. The results presented at WORLDSymposium suggest that we have made significant progress with our Pompe program as we look toward addressing some of the major challenges with the current approved therapy including enzyme activity and stability; targeting and uptake; and tolerability and immunogenicity. These results also validate our internal biologics capabilities to develop and manufacture our own cell line at clinical scale. We look forward to moving our next-generation ERT into the clinic later this year.”
Amicus is leveraging its biologics capabilities and CHART(TM) (Chaperone-Advanced Replacement Therapy) platform to develop a next-generation Pompe ERT. This ERT consists of a uniquely engineered recombinant human acid alpha-glucosidase (rhGAA) enzyme (designated ATB200) with an optimized carbohydrate structure to enhance uptake, administered in combination with a pharmacological chaperone to improve activity and stability. In earlier preclinical studies, ATB200 demonstrated greater tissue enzyme levels and further substrate reduction compared to the current approved ERT for Pompe disease (alglucosidase alfa). Clinical studies1,2 of pharmacological chaperones in combination with currently marketed ERTs have established initial human proof-of-concept that a chaperone can stabilize enzyme activity and potentially improve ERT tolerability.
An oral presentation3 and posters3,4 at WORLDSymposium describe updated preclinical results that support clinical development of ATB200 in combination with a chaperone:
- A proprietary cell line and manufacturing processes have been developed to produce ATB200 with optimized carbohydrate structures for efficient cellular uptake and lysosomal targeting
- ATB200 was significantly better than the approved ERT for reducing disease substrate in multiple key muscle tissues in a Pompe animal model (Gaa knock-out mice)
- The addition of a pharmacological chaperone further improved glycogen reduction by ATB200
- ATB200 has been successfully scaled up to 250L production, and is on track to enter the clinic in the second half of 2015.
About Amicus Therapeutics
Amicus Therapeutics (FOLD) is a biopharmaceutical company at the forefront of therapies for rare and orphan diseases. The Company is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as next-generation enzyme replacement therapy (ERT) products for Fabry disease, Pompe disease, and MPS-1.
- Kishnani, et al., LDN WORLD 2013
- Doerfler, et al., LDN WORLD 2014
- Gotschall, et al., WORLDSymposium 2015
- Lun, et al., WORLDSymposium 2015
SOURCE: Amicus Therapeutics
Post Views: 109
