Apic Bio, Inc., announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for APB-102, the Company’s lead gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis (ALS) – a common cause of familial ALS.
“FDA IND clearance for our drug candidate APB-102 to treat SOD1 ALS is a significant milestone for Apic Bio and takes us one step closer to bringing potentially disease-modifying therapies to patients with genetic diseases,” said John Reilly, Chief Executive Officer and Co-founder of Apic Bio.
APB-102 is designed to slow or reverse progression of SOD1 ALS through a next generation recombinant adeno associated virus (AAV) capsid and micro ribonucleic acid (miRNA) vector construct which has been shown in preclinical proof of concept studies to suppress activity of the mutated SOD1 gene.
The Phase 1/2 clinical trial which will be initiated in late 2021/early 2022 is a multi-center, three-part study to evaluate the safety, tolerability, and efficacy of intrathecally administered APB-102 in patients with SOD1 ALS mutations: part I, single ascending dose; part II, randomized, double-blind, placebo-controlled; and part III, extended follow-up.
