Apic Bio receives FDA Fast Track Designation for APB-102 for treatment of patients with SOD1 ALS
The US FDA has granted Fast Track designation to APB-102, Apic Bio’s lead gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis (ALS). Apic said they plan to commence a Phase 1/2 clinical trial in early 2022.
The FDA’s Fast Track program facilitates the development and expedites the review of drugs to treat serious conditions and fill an unmet medical need, allowing important new drugs to reach the patient earlier.
“We are pleased that the FDA recognizes the significant unmet need for treatments for SOD1 ALS, an always fatal neurogenerative disorder, where mutations in the SOD1 gene account for approximately one-fifth of all inherited forms of the disease,” said Dr. Jorge Quiroz, EVP and Chief Medical Officer of Apic Bio. “We believe in the therapeutic potential of our gene therapy candidate APB-102 that targets the underlying pathophysiology of the disease, and we remain on track to initiate our Phase 1/2 study of APB-102 in early 2022.”
APB-102 is a recombinant AAVrh10 vector that expresses an anti-SOD1 artificial microRNA. The microRNA binds to SOD1 mRNA thereby reducing production of the mutant protein in patients with this form of the disease. Reducing mutant SOD1 protein levels may improve survival and function of motor neurons and potentially provide a therapeutic benefit to people with SOD1-linked ALS.
The Phase 1/2 clinical trial 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.
