CRANBURY, N.J., June 25, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today disclosed a preclinical Chaperone-Advanced Replacement Therapy (CHART™) program for Mucopolysaccharidosis Type I (MPS I), a lysosomal storage disease caused by missing or deficient alpha-L-iduronidase (IDUA) enzyme. Amicus is developing a proprietary human recombinant IDUA (rhIDUA) enzyme co-formulated with a novel pharmacological chaperone as a next-generation therapy for MPS I.
The pharmacological chaperone is designed to improve tissue uptake and reduce the immunogenicity of rhIDUA by stabilizing the enzyme in its properly folded and active form. In addition, in support of its development of a proprietary rhIDUA enzyme, Amicus has received a grant of up to approximately $250,000 from a private U.S.-based donor that provides medical research grants to find better treatments and cures for rare genetic disorders, including lysosomal storage diseases.
David J. Lockhart, Ph.D., Chief Scientific Officer of Amicus Therapeutics, said, "This grant expands our preclinical work and supports our development of a next-generation therapy for MPS I. Our goal is to engineer a proprietary, potentially bio-better rhIDUA enzyme that may be further improved by the addition of a pharmacological chaperone stabilizer. First-generation ERT for MPS I has helped to improve certain manifestations of the disease, however, rhIDUA is highly immunogenic. Directly co-formulating rhIDUA with a chaperone has the potential to increase enzyme stability in the infusion bag and in circulation, enhance uptake of active enzyme in disease-relevant tissues, and potentially reduce immunogenicity."
In people with MPS I, missing or deficient IDUA leads to the accumulation of complex carbohydrates that can affect physical abilities, organ and system functioning, as well as mental and skeletal development. First-generation ERT (laronidase) with rhIDUA has been shown to improve walking capacity and pulmonary function in people with MPS I. However, most patients who receive laronidase develop anti-rhIDUA antibodies, and the laronidase label carries a black-box warning that cites life-threatening anaphylactic reactions in some patients during infusion.1 Amicus believes it may be able to improve upon the properties of the rhIDUA enzyme itself, while also incorporating a pharmacological chaperone stabilizer.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, "Our MPS I program is an additional step forward for our CHART platform technology to combine proprietary enzymes with novel pharmacological chaperones across the lysosomal storage diseases. Importantly, the grant funding supports our internal biologics capabilities at Amicus, which we are leveraging alongside our expertise with chaperones to develop next-generation therapies for people living with lysosomal storage diseases."
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: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 include the small molecule pharmacological chaperones migalastat HCl as a monotherapy and in combination with enzyme replacement therapy (ERT) for Fabry disease; and AT2220 (duvoglustat HCl) in combination with ERT for Pompe disease.
About Chaperone-Advanced Replacement Therapy (CHART™)
The Chaperone-Advanced Replacement Therapy (CHART™) platform combines unique pharmacological chaperones with enzyme replacement therapies (ERTs) for lysosomal storage diseases (LSDs). In a chaperone-advanced replacement therapy, a unique pharmacological chaperone is designed to bind to and stabilize a specific therapeutic enzyme in its properly folded and active form. This proposed CHART mechanism may allow for enhanced tissue uptake of active enzyme, greater lysosomal activity, more reduction of substrate, and lower immunogenicity compared to ERT alone. Improvements in enzyme stability may also enable more convenient delivery of next-generation therapies. Amicus is leveraging the CHART platform to develop pharmacological chaperones co-administered with currently marketed ERTs as well as proprietary next-generation therapies that consist of lysosomal enzymes co-formulated with pharmacological chaperones.
About Mucopolysaccharidosis Type I (MPS I)
Mucopolysaccharidosis Type I (MPS I) is a severe, fatal, multi-system lysosomal storage disease which can affect physical abilities, organ and system functioning, as well as mental and skeletal development. It is caused by a deficiency of the lysosomal enzyme alpha-L-iduronidase, which leads to the accumulation of complex carbohydrates known as glycosaminoglycans (GAGs).
The incidence of MPS I is estimated to be at about 1 in 100,000 births. MPS I is typically classified into three categories: 1) Hurler Syndrome, the most severe form involving progressive developmental delay, physical problems, and death within the first decade of life, 2) Hurler-Scheie Syndrome, an intermediate form characterized by physical symptoms and normal or near-normal cognitive ability, and 3) Scheie Syndrome, an attenuated form in which individuals may have normal cognitive ability, average height, skeletal deformities and an average life span.
1Aldurazyme prescribing information, www.aldurazyme.com
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to clinical development of Amicus' candidate drug products. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "plan," "targets," "likely," "will," "may", "potential", "would," "should" and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Amicus might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the potential goals, progress, timing and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in the business of Amicus, including, without limitation: the potential that results of clinical or pre-clinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we will need additional funding to complete all of our studies and, our dependence on third parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. In addition, all forward looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2012. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and Amicus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.