BNTC Stock Price Increased Over 80% Pre-Market: Why It Happened – Pulse 2.0

The stock price of Benitec Biopharma Inc (NASDAQ: BNTC) a development-stage biotechnology company that focuses on the development of novel genetic medicines increased by over 80% pre-market. Investors are responding to a Schedule 13G filing showing that Morgan Stanley now has 249,174 shares in the company.

Last month, Benitec Biopharma had announced the successful results of the interim analysis of the BB-301 Pilot Dosing Study. The proprietary DNA-directed RNA interference (ddRNAi) platform combines RNA interference (RNAi) with classical AAV-based gene therapy.

And through the use of the ddRNAi platform Benitecs 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. 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.

And 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 genetic medicine employing the Silence and Replace approach for the treatment of OPMD.

OPMD is known as a chronic life-threatening genetic disorder affecting approximately 15,000 patients in the United States, Canada, Western Europe, and Israel. And 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.

There are no therapeutic agents approved for the treatment of OPMD. And there is not any surgical interventions capable of altering the long-term natural history of OPMD are available. BB-301 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 had scheduled a Scientific Advice Meeting in France in May 2021 to review the interim data and the Phase 1 clinical trial design. And the company continues to plan for the initiation of the first-in-human clinical study of BB-301 in OPMD patients in 2022. The interim data validated the promise of the Silence and Replace approach to disease management and Benitec plans to provide additional pipeline updates in the second half of 2021.

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BNTC Stock Price Increased Over 80% Pre-Market: Why It Happened - Pulse 2.0