Date: 2016-07-06
Type of information: Initiation of preclinical development
phase: preclinical
Announcement: initiation of preclinical development
Company: Amicus Therapeutics (USA - NJ)
Product: CDKL5 program
Action mechanism:
Disease: cyclin-dependent kinase-like 5 (CDKL5) deficiency
Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases
Country:
Trial details:
Latest
news: * On July 6, 2016, Amicus Therapeutics announced that the company has expanded its biologics pipeline with a new preclinical program for cyclin-dependent kinase-like 5 (CDKL5) deficiency, a rare and devastating genetic neurological disease for which there is no currently approved treatment. Signs and symptoms typically begin with persistent, spontaneous seizures in infancy followed by severe delays in neurological development. Amicus has obtained the rights and related intellectual property to a preclinical CDKL5 program through the acquisition of MiaMed, Inc. Under the terms of the acquisition agreement with MiaMed, at closing, Amicus paid approximately $1.8 million in cash and approximately $4.7 million in Amicus common stock to the former shareholders of MiaMed. In addition, the former shareholders of MiaMed are eligible to receive up to $18 million upon the achievement of clinical and regulatory milestones and up to $65 million upon achievement of commercial milestones. The acquisition of MiaMed does not impact previous full-year 2016 net cash spend guidance of $135 million to $155 million.
CDKL5 (cyclin-dependent kinase-like 5) is a gene on the X-chromosome encoding the CDKL5 protein that regulates the expression of several essential proteins for normal brain development. Genetic mutations in the CDKL5 gene result in CDKL5 protein deficiency. The disorder manifests clinically as persistent seizures starting in infancy, followed by severe impairment in neurological development. Most children affected by CDKL5 deficiency cannot walk or care for themselves and may also suffer from scoliosis, visual impairment, sensory issues, and gastrointestinal complications. CDKL5 mutations have been found in children diagnosed with cerebral palsy, infantile epilepsies, and autism, among other conditions, and the disorder was previously classified as atypical Rett Syndrome, an early seizure variant of Rett Syndrome. There are more than 1,200 documented cases of CDKL5 deficiency worldwide, with the number of identified patients increasing as genetic testing for the disorder becomes more common.
