FDA Puts APB-102 Gene Therapy for SOD1 ALS on Fast Track

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by Vanda Pinto, PhD |

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APB-102 gene therapy for SOD1 ALS/alsnewstoday.com/FDA grants fast track status


The U.S. Food and Drug Administration (FDA) has granted fast track status to Apic Bio’s experimental therapy APB-102, designed for amyotrophic lateral sclerosis (ALS) patients who carry mutations in the SOD1 gene that lead to misfolded proteins in cells.

This designation accelerates the development of investigational therapies that address unmet medical needs in serious or life-threatening conditions. It makes Apic Bio eligible for more frequent meetings with the FDA and discussions about APB-102’s development plan.

“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,” Jorge Quiroz, MD, executive vice president and chief medical officer of Apic Bio, said in a press release.

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Phase 1/2 Trial of APB-102, Gene Therapy for Familial ALS, Cleared to Open

About 15–20% of people with familial ALS and 1–2% of those with sporadic ALS carry mutations in the SOD1 gene. These mutations result in the production of neurotoxic forms of the SOD1 protein, an enzyme responsible for the removal of free radicals, which is important for cellular health.

APB-102 is a gene therapy designed to address ALS cases caused by SOD1 mutations. It consists of a microRNA — a small RNA molecule that latches onto intermediate RNAs carrying the genetic instructions for protein production — that binds to a certain portion of the SOD1 RNA sequence and prevents the SOD1 protein from being produced.

This microRNA molecule is contained inside a harmless adeno-associated virus and delivered into the spinal canal via a direct (intrathecal) injection. In a proof-of-concept study involving two ALS patients, APB-102 lowered SOD1 levels in their brain and spinal cord.

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The FDA recently cleared a Phase 1/2 clinical trial to investigate APB-102’s safety, tolerability, and efficacy in ALS patients with SOD1 mutations. The study is scheduled to begin early next year, and will be conducted in three parts.

In part 1, study participants will receive single but ascending doses of APB-102 to determine an optimal treatment dose. In the second part, patients will be randomly assigned to receive either a placebo or APB-102, at the dose determined in part 1. Part 3 will consist of an extended follow-up.

APB-102 received orphan drug status from the FDA in July 2019, a designation intended to promote the development of investigational therapies for rare and serious diseases. It offers several benefits, including exemption from FDA application fees and seven years of market exclusivity upon approval.

“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,” said Quiroz.