| • | | The data derived from the interim analysis validate the promise of the “Silence-and-Replace” approach to disease management, and Benitec plans to provide additional pipeline updates in 2H 2021 |
HAYWARD, Calif., February 24, 2021 — Benitec Biopharma Inc., a development-stage biotechnology company focused on the advancement of novel genetic medicines, today announced the successful results of the interim analysis of the BB-301 Pilot Dosing Study. In addition to the data summarized below, please see the accompanying slide presentation available at www.benitec.com and accessible via the following link.
The proprietary DNA-directed RNA interference (ddRNAi) platform combines RNA interference (RNAi) with classical AAV-based gene therapy. Through the use of the ddRNAi platform Benitec’s goal is to create genetic medicines that, following a single administration, will enable target tissues to perpetually produce siRNA molecules which facilitate the sustained silencing of disease-causing genes. Importantly, the ddRNAi platform also allows for concomitant delivery of wild type replacement genes, and these distinct genetic elements work in concert to silence the expression of disease-causing mutant genes and to simultaneously replace the mutant genes with normal (wild type) genes to restore the natural underlying physiology of the diseased tissues. BB-301, the most advanced genetic medicine currently under development by Benitec, employs the proprietary platform, which allows for a “Silence and Replace” approach to the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).
BB-301 is a first-in-class genetic medicine employing the “Silence and Replace” approach for the treatment of 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. BB-301 has received Orphan Drug Designation in the United States and the European Union which provides commercial exclusivity (independent of intellectual property protection) and opportunities for efficient pathways for regulatory review and approval. While OPMD is a rare (Orphan) disorder, the commercial opportunity for a safe and efficacious therapeutic agent in this indication exceeds $1 billion over the course of the commercial life of the product.
Benitec has previously outlined the core IND-enabling studies required by global regulatory agencies to support the initiation of BB-301 clinical trials in OPMD patients, and these IND-enabling studies include a BB-301 Pilot Dosing Study in large animals and a classical 12-week GLP Toxicology and Biodistribution Study. BB-301 is directly injected into the pharyngeal muscles known to underlie the morbidity and mortality characterizing the natural history of OPMD. Against this backdrop, the BB-301 Pilot Dosing Study in large animal subjects was conducted to demonstrate that direct intramuscular injection of BB-301 via the use of a proprietary dosing device in an open surgical procedure could safely achieve the following goals:
| • | | Biologically significant, highly-consistent, dose-dependent levels of BB-301 tissue transduction (i.e., delivery of the multi-functional genetic construct into the target pharyngeal muscle cells) |