Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Apic Bio’s APB-102, the company’s lead gene therapy candidate designed to treat the SOD1-linked rare neurodegenerative condition amyotrophic lateral sclerosis.

Amyotrophic lateral sclerosis (ALS) is a fatal disorder characterized by loss of motor neurons, leading to muscle weakness and eventual paralysis. Most patients face mortality within five years of disease onset due to respiratory failure. ALS can be caused by multiple genetic mutations and can be sporadic (spontaneous mutations) or familial (inherited mutations). Familial mutations account for approximately ten percent of ALS cases, and of these, approximately twenty percent are linked to a mutation in the SOD1 gene that codes for the enzyme superoxide dismutase 1. SOD1-linked ALS is most likely caused by toxic mutant forms of the superoxide dismutase 1 (SOD1) protein (a gain-of-function mutation). Current approved ALS treatments only delay disease progression without addressing the underlying genetic causes of the disease.

APB-102 is a gene therapy candidate being evaluated for the potential treatment of SOD1 ALS. 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. APB-102 has received Orphan Drug and Fast Track designation from the U.S. Food and Drug Administration.

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. Drugs that receive a Fast Track designation are eligible for more frequent meetings and written communication with the FDA to discuss development plans and clinical trial design.

The company plans to commence a phase 1/2 clinical trial in early 2022. The 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.

“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 Jorge Quiroz, executive vice president 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.”

Photo: Jorge Quiroz, executive vice president and chief medical officer of Apic Bio

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