Benzinga | Sep 8, 2021 08:17AM EDT

Benitec Biopharma Provides Regulatory Updates for the BB-301 Development Program; BB-301 Phase 1b/2a Clinical Trial to Begin in 2022

Benitec Biopharma Inc. (NASDAQ:BNTC), a development-stage biotechnology company focused on the advancement of novel genetic medicines, today announced significant progress regarding the regulatory interactions

Benitec Biopharma Inc. (NASDAQ:BNTC), a development-stage biotechnology company focused on the advancement of novel genetic medicines, today announced significant progress regarding the regulatory interactions in Europe required to support advancement of the BB-301 development program towards the Phase 1b/2a clinical study in 2022. Benitec also confirmed the scheduling of an upcoming regulatory interaction with the Food and Drug Administration in the United States.

BB-301 is a novel investigational gene therapy under development for the treatment of patients with Oculopharyngeal Muscular Dystrophy (OPMD). OPMD is a chronic, life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel. OPMD is caused by a mutation in the gene encoding poly(A) binding protein nuclear 1 (PABPN1). Patients with OPMD lose the ability to swallow liquids and solids, and the natural history of the disorder is characterized by chronic malnutrition, aspiration, and fatal episodes of aspiration pneumonia. Currently, no therapeutic agents are approved for the treatment of OPMD. Additionally, no surgical interventions capable of altering the long-term natural history of OPMD are available.

Benitec has previously disclosed key data-points related to the completed non-clinical studies and the planned non-clinical studies for BB-301 that were anticipated to support the filing of Clinical Trial Applications in Europe and in Canada and, similarly, to facilitate the filing of an Investigational New Drug Application in the United States. In addition to the non-clinical proof-of-concept studies carried out in murine models of OPMD (Strings-Ufombah, V., et al., Molecular Therapy: Nucleic Acids, 2021; Malerba, A., et al. Nature Communications, 2017), Benitec recently completed a BB-301 Pilot Dosing Study in large animals (Beagle dogs). The BB-301 Pilot Dosing Study yielded favorable interim data related to BB-301 target tissue transduction, gene expression, and target protein knock-down, all of which were disclosed in February 2021 via press release and presentation. Additionally, Benitec initiated a 12-week GLP Biodistribution and Toxicology Study for BB-301 in Beagle dogs during the first half of 2021.

Following the disclosure of the positive BB-301 Pilot Dosing Study data, Benitec completed a Scientific Advice meeting with The National Agency for the Safety of Medicines and Health Products in France (L'Agence nationale de s?curit? du m?dicament et des produits de sant? or "ANSM") in the first half of 2021.