Therapy Areas: Respiratory
Apic Bio Receives FDA Fast Track Designation for APB-102 for the Treatment of Patients with SOD1 ALS
29 July 2021 - - The US Food and Drug Administration has granted Fast Track designation to APB-102, US-based gene therapy company Apic Bio, Inc's lead gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis, the company said.

Apic plans 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.

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 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.

Amyotrophic lateral sclerosis is a fatal neurodegenerative 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 % 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 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 US Food and Drug Administration.

Apic Bio is an innovative gene therapy company focused on developing first-in-class treatment options for rare, undertreated neurological and liver diseases. The company's lead program is an adeno-associated -based gene therapy for the treatment of SOD1 ALS.

Preclinical studies of additional genetic forms of ALS (C9orf72) and Alpha-1 Antitrypsin Deficiency (Alpha-1) are ongoing.

The company is also advancing discovery programs for two undisclosed CNS indications that leverage its proprietary silence and replace THRIVE platform. The company is backed by leading and disease-centric investors, including Morningside Ventures, ALS Investment Fund, and The Alpha-1 Project.
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