AMICUS THERAPEUTICS, INC. (NASDAQ:FOLD) Files An 8-K Other Events
Item 8.01. Other Events

On January 3, 2019, Amicus Therapeutics, Inc. issued a press release announcing the initiation of a Phase 1/2 clinical study to evaluate the safety and efficacy of a single intrathecal administration of adeno-associated virus serotype 9 AAV9-CLN3 gene therapy in children with CLN3 Batten disease. A copy of this press release is attached hereto as Exhibit 99.1 and is incorporated herein by reference.

Item 9.01 Financial Statements and Exhibits

Exhibits:

AMICUS THERAPEUTICS, INC. Exhibit
EX-99.1 2 exhibit991.htm EXHIBIT 99.1 exhibit991 Amicus Therapeutics Announces Phase 1/2 Study of Gene Therapy for CLN3 Batten Disease Second Gene Therapy to Enter Clinic at Nationwide Children’s Hospital for Neurologic Lysosomal Storage Disorders Initial Safety is Encouraging with No Serious Adverse Events at One Month CRANBURY,…
To view the full exhibit click here

About AMICUS THERAPEUTICS, INC. (NASDAQ:FOLD)

Amicus Therapeutics, Inc. is a biopharmaceutical company. The Company is focused on the discovery and development of therapies to treat a range of rare and orphan diseases. Its lead product candidate, migalastat HCl (Galafold), is an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease, which is a type of lysosomal storage disorder (LSD). It has completed two Phase III global registration studies (Study 011 and Study 012) of migalastat monotherapy. It is also engaged in Phase III clinical development of a topical cream, SD-101, for the treatment of the genetic connective tissue disorder Epidermolysis Bullosa. It has also initiated a clinical study in patients with Pompe disease, a LSD to investigate its treatment paradigm that consists of ATB200, which is an engineered recombinant human acid alpha-glucosidase enzyme with an optimized carbohydrate structure to enhance uptake, co-administered with AT2221, to improve activity and stability.