Valerion Reports Preclinical Potential for Pompe Treatment

Valerion Reports Preclinical Potential for Pompe Treatment

CONCORD, Mass. – (April, 2017) – Valerion Therapeutics, part of the Alopexx Enterprises portfolio of companies, has announced the development of VAL-1221, a fusion protein that combines its technology with recombinant human acid alpha-glucosidase (rhGAA) to improve delivery of rhGAA to affected tissues in patients with Pompe disease.

Valerion Reports Preclinical Potential for Pompe Treatment

Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease

Concord, MA – (March 3, 2017) – Valerion Therapeutics announced today that it has developed a fusion protein, VAL-1221, which combines its proprietary antibody delivery technology with recombinant human acid alpha-glucosidase (rhGAA) to improve the delivery of rhGAA into affected tissues of patients with Pompe disease (Glycogen Storage Disease, Type II; GSDII).

Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium™ 2017

Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium™ 2017

CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today presented new scientific findings and preclinical data on functional outcomes in an oral presentation and poster1 at the 13thAnnual WORLDSymposium™ in San Diego, CA. 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 in muscles, co-administered with AT2221, a pharmacological chaperone designed to stabilize ERT in circulation.

Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium™ 2017

Amicus Therapeutics Presents Additional Positive Preliminary Phase 1/2 Data at WORLDSymposium™ 2017

CRANBURY, N.J. and SAN DIEGO, Feb. 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today presented additional positive preliminary data from a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in a poster1 at the 13th Annual WORLDSymposium™ in San Diego, CA. 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.

BioMarin Update for the Pompe Community

BioMarin Update for the Pompe Community

BioMarin greatly appreciates the many patients, families, and physicians who have participated in our Pompe program, as well as your ongoing support of the entire patient community. After much deliberation, BioMarin has decided to discontinue the clinical development of the BMN 701 (reveglucosidase alfa) Pompe program.

Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium™ 2017

BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J.P. Morgan Annual Healthcare Conference

SAN RAFAEL, Calif., Jan. 11, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced interim results from INSPIRE, a Phase 2 trial for reveglucosidase alfa, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) being studied for the treatment of late-onset Pompe disease (LOPD).

Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium™ 2017

Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that its investigational new drug (IND) application, submitted to U.S. Food and Drug Administration (FDA), is now effective which allows Amicus to begin site initiation and enrollment of a Phase 1/2 study of ATB200 in patients with Pompe disease.