Benitec Biopharma Announces Acceptance of Late- Breaking Abstract for the BB-301 Phase 1b/2a Clinical Treatment Study at the Muscular Dystrophy Association Clinical & Scientific Conference
Rhea-AI Summary
Benitec Biopharma (NASDAQ: BNTC) announced acceptance of a late-breaking poster for the BB-301 Phase 1b/2a treatment study in oculopharyngeal muscular dystrophy (OPMD) at the Muscular Dystrophy Association Clinical & Scientific Conference on March 9, 2026.
The poster (501 LB) will report interim clinical results including 12-month follow-up for four Cohort 1 completers, 24-month results for the first Cohort 1 patient, and interim data for the first Cohort 2 patient, with multiple poster session times listed.
Positive
- 12-month follow-up reported for four Cohort 1 completers
- 24-month clinical results available for first Cohort 1 patient
- Interim Cohort 2 data reported for first treated patient
- Late-breaking poster acceptance at a major MDA conference on March 9, 2026
Negative
- Data are interim and from very small cohorts (four and single patients)
- Results presented as a poster; not a peer-reviewed full publication
Key Figures
Market Reality Check
Peers on Argus
BNTC was down 1.85% while only one tracked peer (AUTL) appeared in the momentum scanner, moving up 1.2000000104308128%. With no broad peer moves or same-day peer news, trading looked stock-specific rather than sector-driven.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| 2026-02-12 | Earnings and BB-301 update | Positive | -2.7% | Reported Q2 FY2026 results and durable BB-301 responder data with strong cash. |
| 2025-11-14 | Earnings and financing | Positive | +2.8% | Q1 FY2026 results, 100% Cohort 1 response, Fast Track, and ~$100M equity raise. |
| 2025-11-05 | Equity offering announcement | Negative | +0.5% | Proposed underwritten offering and registered direct deal using S-3 registration. |
| 2025-11-03 | Clinical trial update | Positive | -3.1% | Positive BB-301 interim data and FDA Fast Track designation for OPMD dysphagia. |
| 2025-11-03 | Board appointment | Positive | -3.1% | Appointment of Sharon Mates, ex-CEO linked to a $14.6B acquisition by J&J. |
BNTC has frequently traded down or muted on fundamentally positive clinical and corporate updates, with only one of the last five news events showing aligned positive price action.
Over the last few months, Benitec has focused on advancing BB-301 for OPMD-related dysphagia while strengthening its balance sheet. On Nov 3, 2025, it reported positive Phase 1b/2a interim data and FDA Fast Track, yet shares fell 3.06%. A proposed equity offering on Nov 5, 2025 saw only a 0.45% move. Q1 FY2026 results on Nov 14, 2025 with a ~$100M raise led to a 2.81% gain, but Q2 FY2026 results on Feb 12, 2026 again saw a 2.66% decline. Today’s conference-focused announcement fits this BB-301–centric trajectory.
Regulatory & Risk Context
An active Form S-3 resale registration dated 2026-01-30 covers 1,481,481 shares of common stock held by existing investors from a prior registered direct offering. Benitec will not receive any proceeds from these sales but will bear registration expenses, and filings highlight risks from concentrated ownership and potential resale-related stock price pressure.
Market Pulse Summary
This announcement highlights that additional BB-301 Phase 1b/2a data, including 12- and 24-month follow-up and early Cohort 2 results, will be featured in a late-breaking poster at a major muscular dystrophy conference on March 9, 2026. It extends a series of updates centered on BB-301’s development path. Investors may watch for durability of response, differences between dose cohorts, and forthcoming FDA discussions on pivotal study design, while also tracking any impact from ongoing registered resale activity.
Key Terms
phase 1b/2a medical
dysphagia medical
gene therapy medical
dna-directed rna interference medical
late-breaking abstract technical
AI-generated analysis. Not financial advice.
-Interim clinical study results for the BB-301 Phase 1b/2a Treatment Study including 12-month follow-up results for the first four Cohort 1 completers, 24-month clinical study results for the first Cohort 1 Patient, and interim clinical study results for the first Cohort 2 Patient will be reported in a late-breaking poster presentation at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference on March 9, 2026-
HAYWARD, Calif., Feb. 23, 2026 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference ("ddRNAi") platform, today announced the acceptance of a late-breaking abstract for the BB-301 Phase 1b/2a Clinical Treatment Study ongoing in Oculopharyngeal Muscular Dystrophy Patients (OPMD) with moderate dysphagia. Interim clinical study results for Patients enrolled into Cohort 1 and Cohort 2 will be discussed in a poster presentation at the Muscular Dystrophy Association Clinical and Scientific Conference, being held in Orlando, Florida on March 9, 2026.
“We are pleased to present long-term clinical study results for Patients treated with BB-301 in Cohort 1 and interim clinical study results for the first Patient treated with BB-301 in Cohort 2 at the Muscular Dystrophy Association Clinical & Scientific Conference,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “We are grateful to the OPMD community and investigators, and we look forward to providing updates on the ongoing clinical study and future discussions with the FDA as we work toward confirming the pivotal study path for BB-301.”
Late-Breaking Poster Presentation:
An interim clinical study update for the Phase 1b/2a Clinical Treatment Study of BB-301 in OPMD subjects with moderate dysphagia will be provided in a late-breaking poster presentation, (poster number 501 LB) entitled “Durable Responses to Low-Dose BB-301 in Oculopharyngeal Muscular Dystrophy at 12- and 24-months and Improved Depth of Response to High-Dose BB-301” during poster sessions from 10:15-10:45 am, 12:00-1:30 pm, 3:30-4:00 pm and 6:00-8:00 pm Eastern Time on March 9th in the Exhibit Hall at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference.
About BB-301
BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1 (the causative gene for OPMD). The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein. BB-301 has received Orphan Drug Designation from the EMA and Orphan Drug and Fast Track Designations from the FDA.
About Benitec Biopharma Inc.
Benitec Biopharma Inc. (“Benitec” or the “Company”) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary “Silence and Replace” DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitec’s website at www.benitec.com.
Forward Looking Statements
Except for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings and other regulatory steps, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements.
These forward-looking statements are based on the Company’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: the success of our plans to develop and potentially commercialize our product candidates; the timing of the completion of preclinical studies and clinical trials; the timing and sufficiency of patient enrollment and dosing in any future clinical trials; the timing of the availability of data from our clinical trials; the timing and outcome of regulatory filings and approvals; the development of novel AAV vectors; our potential future out-licenses and collaborations; the plans of licensees of our technology; the clinical utility and potential attributes and benefits of ddRNAi and our product candidates, including the potential duration of treatment effects and the potential for a “one shot” cure; our intellectual property position and the duration of our patent portfolio; expenses, ongoing losses, future revenue, capital needs and needs for additional financing, and our ability to access additional financing given market conditions and other factors; the length of time over which we expect our cash and cash equivalents to be sufficient to execute on our business plan; unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities and other regulatory developments; the Company’s ability to protect and enforce its patents and other intellectual property rights; the Company’s dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Company’s products and the products of the Company’s collaboration partners; the acceptance of the Company’s products and the products of the Company’s collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the impact of, and our ability to remediate, the identified material weakness in our internal controls over financial reporting; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements.
Investor Relations Contact:
Irina Koffler
LifeSci Advisors, LLC
(917) 734-7387
ikoffler@lifesciadvisors.com
FAQ
What BB-301 data will Benitec (BNTC) present on March 9, 2026?
Where and when is Benitec’s BB-301 poster (501 LB) scheduled at the MDA conference?
Which patient cohorts are included in the BB-301 Phase 1b/2a interim results for BNTC?
Does the BB-301 announcement indicate the study is moving toward a pivotal path for BNTC?
What endpoints or clinical improvements does Benitec report for BB-301 at 12 and 24 months?
