Skip to main content

Market Overview

Benitec Biopharma's Gene Therapy For Rare Muscle Disorder Shows Encouraging Action In Animal Studies

Share:
  • Benitec Biopharma Inc (NASDAQ: BNTCreports an interim analysis of the BB-301 Pilot Dosing study conducted in large animal subjects.
  • BB-301 a genetic medicine directly injected into the pharyngeal muscles. It employs the proprietary platform, which allows for a "Silence and Replace" approach to the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).
  • OPMD is caused by a mutation in the gene encoding poly(A) binding protein nuclear 1 (PABPN1), wherein patients lose the ability to swallow liquids and solids.
  • The interim analysis showed biologically significant, highly-consistent, dose-dependent levels of BB-301 tissue transduction, with BB-301 copy numbers ranging from 1.7 copies per cell up to 8.6 copies per cell achieved after a single administration of increasing doses of BB-301.
  • Durable and biologically significant levels of target gene knockdown (inhibition of the expression of the gene encoding PABPN1) were observed.
  • Low-dose, intermediate-dose, and high-dose BB-301 administration achieved similar inhibition levels, with an average of 74% inhibition of PABPN1 expression observed across all doses.
  • Following an optimized route and method of administration, Benitec demonstrated a 248-fold improvement and a 111-fold improvement in BB-301 transduction of the two key pharyngeal muscles relative to the levels of BB-301 transduction observed in the analogous Beagle dog study conducted by the previous BB-301 licensee.
  • The company has scheduled a Scientific Advice Meeting in France in May to review the interim data and the Phase 1 clinical trial design.
  • The company will provide additional pipeline updates in 2H2021. Plans to initiate human trials of BB-301 in OPMD patients in 2022.
  • Price Action: BNTC gained 5.8% at $3.97 in premarket trading on the last check Wednesday.
 

Related Articles (BNTC)

View Comments and Join the Discussion!

Posted-In: gene therapy Phase 1 TrialBiotech News Penny Stocks FDA General

Don't Miss Any Updates!
News Directly in Your Inbox
Subscribe to:
Benzinga Premarket Activity
Get pre-market outlook, mid-day update and after-market roundup emails in your inbox.
Market in 5 Minutes
Everything you need to know about the market - quick & easy.
Fintech Focus
A daily collection of all things fintech, interesting developments and market updates.
SPAC
Everything you need to know about the latest SPAC news.
Thank You

Thank you for subscribing! If you have any questions feel free to call us at 1-877-440-ZING or email us at vipaccounts@benzinga.com