Analyzing a Novel Therapy for CDKL5

Student: Daniel Hogan, 2017-2018

Sponsor: Amicus Therapeutics, Cranbury, NJ

Cyclin-Dependent Kinase-Like 5 (CDKL5) is a rare X-linked genetic disorder that results in early onset, treatment-resistant seizures, gastrointestinal disturbances, cortical vision impairment and mental retardation. An estimated 4,000 patients suffer from CDKL5 in the United States. The CDKL5 protein acts as a kinase, governing neuronal structure, connectivity and signal transmission. No targeted, FDA-approved therapy or cure exists for this devastating disease. In 2016, Amicus Therapeutics expanded its biologics pipeline with a preclinical program for CDKL5. Amicus technology in development, a modified enzyme replacement therapy, presents a novel biologics treatment for an unmet clinical need with the potential for breakthrough designation. The patent and early preclinical success of this therapy, especially in demonstrating dendrite growth, also present the potential opportunity to expand the technology to other enzymes. The commercialization of this technology, including an analysis of its market potential and financial feasibility, will be thoroughly analyzed in order to formulate strategies for efficient clinical implementation.