Benitec Biopharma Announces Positive Interim Phase 1b/2a Results for High Dose BB-301 and Continued Durable Improvements for Low Dose BB-301 

Benitec is advancing toward a pivotal BB-301 study in mid-2027.

Rhea-AI Summary

Benitec Biopharma (NASDAQ: BNTC) reported interim BB-301 trial results showing a 100% response rate among treated patients with OPMD-related swallowing difficulties.

The Phase 1b/2a study includes six low-dose and three high-dose patients. All low-dose patients had 12-month follow-up; one high-dose patient had nine-month data and two had three-month data. At three months, throat-emptying response rates were 67% for low dose versus 100% for high dose; functional swallowing response rates on a timed water-drinking test were 17% versus 67%. Both groups had 100% response rates on the Sydney Swallow Questionnaire and 67% for throat closing at three months. All nine patients qualified as formal responders overall. Benitec reported continued durable swallowing improvements in the low-dose group. No serious adverse events have occurred in any BB-301-treated patient to date.

8 points · 0 major

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0 major · 2 points

Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate point100% of treated patients qualified as formal responders across six low-dose and three high-dose participants.
• Minor pointThroat-emptying response at three months reached 100% with high dose versus 67% with low dose.
• Minor pointTimed water-drinking response at three months reached 67% with high dose versus 17% with low dose.
• Minor pointSwallowing questionnaire response at three months reached 100% in both dose groups.
• Minor pointThroat-closing response at three months reached 67% in both dose groups.

3 minor points

• Minor pointLow-dose swallowing improvements remained durable, Benitec reported, with all six patients reaching 12-month follow-up.
• Minor pointNo serious adverse events have occurred in any BB-301-treated patient to date.
• Minor point. Forward-looking: it has not happened yet and may not happen.Pivotal study is planned for mid-2027, as Benitec advances the program.

Negative

• Minor pointInterim high-dose results cover only three patients; full-cohort dose comparisons were limited to three months.
• Minor pointHigh-dose follow-up reached nine months for one patient but only three months for the other two.

Key Figures

Cohort 1 sample: 6 patients Cohort 2 sample: 3 patients Formal responder rate: 100% +5 more

Cohort 1 sample

6 patients

Low-dose cohort; 12-month follow-up results available

Cohort 2 sample

3 patients

High-dose cohort; interim follow-up results available

Formal responder rate

100%

Patients treated across both dose cohorts

Throat emptying response

67% vs. 100%

Cohort 1 vs. Cohort 2 at Month 3

Functional swallowing capacity response

17% vs. 67%

Cohort 1 vs. Cohort 2 at Month 3

Total SSQ score response

100% in both cohorts

Month 3

Throat closing response

67% in both cohorts

Month 3

Serious adverse events

None reported

In patients treated with BB-301 to date

Previous Clinical trial Reports

3 past events · Latest: Jan 11

Same Type 3 events

1. Jan 11

   clinical results

   24h Move

   +2.5%

   First Cohort 1 patient showed durable, deepening swallowing improvements at 24-month follow-up.

2. Nov 03

   clinical results

   24h Move

   -3.1%

   All six Cohort 1 patients met formal response criteria; FDA granted Fast Track designation.

3. Mar 19

   clinical results

   24h Move

   +0.9%

   Three treated subjects reported improved swallowing measures, including durable 12-month outcomes.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

opmd, dysphagia, aav9 capsid, sirnas, +1 more

5 terms

opmd medical

"Patients with Oculopharyngeal Muscular Dystrophy (OPMD)"

Oculopharyngeal muscular dystrophy (OPMD) is an inherited, progressive muscle disorder caused most often by a short repeat expansion in the PABPN1 gene; it typically appears in mid-adulthood. The condition primarily weakens the muscles that lift the eyelids and those used for swallowing, producing drooping eyelids (ptosis), difficulty swallowing (dysphagia), and later weakness in limb and neck muscles; progression is gradual and can increase risk of choking or aspiration. Most cases follow an autosomal dominant pattern of inheritance and diagnosis is made by clinical exam and genetic testing, distinguishing OPMD from other neuromuscular diseases by its characteristic age of onset, pattern of muscle involvement, and genetic finding.

dysphagia medical

"swallowing function for dysphagic Patients"

Dysphagia is difficulty or discomfort swallowing solids, liquids, or saliva due to problems in the mouth, throat, or esophagus. Investors care because it creates clear patient need and influences the size and urgency of markets for drugs, devices, and therapies: severe or persistent swallowing problems can lead to weight loss, poor nutrition, and lung infections, which affect clinical trial endpoints, regulatory decisions, reimbursement, and long‑term demand—much like a recurring plumbing blockage drives demand for repairs.

aav9 capsid technical

"a novel, modified AAV9 capsid"

AAV9 capsid is the protein shell of adeno-associated virus serotype 9 used to package and deliver genetic material in gene therapy. The capsid controls which tissues the vector naturally targets, how the immune system recognizes it, and how easy it is to manufacture and scale. For investors, those properties affect a therapy’s potential market reach, development complexity, safety profile, and regulatory considerations—like choosing the right delivery truck for a specific cargo and route.

sirnas technical

"two small inhibitory RNAs (siRNAs)"

Small interfering RNAs (siRNAs) are short strands of genetic material designed to bind and silence specific genes, like sending a targeted "mute" command to a particular instruction in a cell. Investors care because siRNA-based therapies can precisely treat diseases by turning off harmful genes, offering potential for strong clinical benefits and revenue but also carrying scientific, regulatory and manufacturing risks that affect a biotech company’s value.

rna interference technical

"combines RNA interference, or RNAi, with gene therapy"

RNA interference is a natural cellular process that acts like a mute switch to reduce or silence the activity of a specific gene by targeting its messenger RNA, the blueprint used to make a protein. For investors, it matters because therapies and diagnostics that harness this mechanism can precisely block disease-causing proteins, creating new drug classes with high potential reward but also scientific, manufacturing and regulatory risks that affect company value.

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- Both low dose and high dose BB-301 continue to drive robust improvements in swallowing function for dysphagic Patients with Oculopharyngeal Muscular Dystrophy (OPMD)

- 100% of Patients treated with BB-301 across both doses are formal Responders to therapy

- Interim clinical results for Patients treated with high dose BB-301 demonstrate greater relative improvements at the 3-month post-treatment time-point in throat emptying ability (assessed by videofluoroscopic evaluation of post-swallow food and liquid residue) and functional swallowing capacity (assessed by the Cold-Water Timed Drinking Test)

HAYWARD, Calif., Oct. 28, 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 positive interim clinical results from the ongoing BB-301 Phase 1b/2a first-in-human study (NCT06185673) for Patients treated with high dose BB-301 and continued durable improvements for Patients treated with the low dose of BB-301. The clinical results observed across both dose cohorts of the BB-301 first-in-human study continue to demonstrate robust and durable improvements in swallowing function in dysphagic Patients with OPMD, with a 100% response rate observed across all Patients treated to date.  

“Progressive loss of swallowing function has a devastating impact on OPMD Patients, yet there are no approved therapies to address this critical unmet medical need,” said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. “The progress we continue to see regarding both the safety and efficacy of BB-301 reinforces our conviction in the program and the potential to establish a new treatment paradigm for Patients suffering from OPMD-related dysphagia. As we advance toward our pivotal study in mid-2027, we remain focused on the promise of delivering a potentially transformative therapy to the OPMD community.”

Key Updated Clinical Study Results

The updated analyses include 12-month post-treatment follow-up results for all six Patients enrolled in Cohort 1 (low dose BB-301) and the available post-treatment follow-up results for all three Patients enrolled in Cohort 2 (high dose BB-301).

Clinical and fluoroscopic results were available for the final 12-month post-treatment follow-up time-point of the study for all Cohort 1 Patients. Clinical and fluoroscopic results were available for the 9-month post-treatment follow-up time-point for the first Patient enrolled in Cohort 2 and for the 3-month post-treatment follow-up time-point for the second and third Patients enrolled in Cohort 2.

• For Cohort 1, all six Patients are formal Responders to BB-301.
• For Cohort 2, interim clinical results are available, and all three Patients are formal Responders to BB-301.
• Evaluations of dose-related improvements were carried out.
  • Full Cohort comparisons (Cohort 1 vs. Cohort 2) were carried out at the 3-month post-treatment time-point, as complete clinical and fluoroscopic results were available for the total Patient populations of the respective cohorts at this post-treatment time-point.
• A larger proportion of Cohort 2 Patients exhibited improvements in throat emptying ability (assessed by videofluoroscopic evaluation of post-swallow food and liquid residue) and functional swallowing capacity (assessed by the Cold-Water Timed Drinking test) at Month 3 post-BB-301 administration.
  • 67% of Cohort 1 Patients and 100% of Cohort 2 Patients met the statistical criteria for response for improved throat emptying ability (assessed by videofluoroscopic evaluation of post-swallow food and liquid residue) at the 3-month post-treatment time-point.
  • 17% of Cohort 1 Patients and 67% of Cohort 2 Patients met the statistical criteria for response for improved functional swallowing capacity as assessed by Cold-Water Timed Drinking Test (CWDT) at the 3-month post-treatment time-point.
    
    
• Improvements in total Sydney Swallow Questionnaire (SSQ) score and throat closing ability for Cohort 1 and Cohort 2 Patients were equivalent at Month 3 post-BB-301 administration.
  
  • 100% of Patients in Cohort 1 and Cohort 2 met the statistical criteria for response for improved total SSQ score at the 3-month post-treatment time-point.
  • 67% of Patients in Cohort 1 and Cohort 2 met the statistical criteria for response for improved throat closing ability (assessed by videofluoroscopic evaluation) at the 3-month post-treatment time-point.
    
    

There have been no Serious Adverse Events in any patient treated with BB-301 to date.

The full poster, accepted as a late-breaking abstract at the Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), is available here.

About OPMD

There are currently no approved therapies for OPMD, a rare autosomal-dominant degenerative muscle disorder, that impacts an estimated 15,000 patients in North America, Europe and Israel. OPMD is caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene; PABPN1 is a ubiquitous protein that controls the length of mRNA poly(A) tails, mRNA export from the nucleus and alternative poly(A) site usage. OPMD is a debilitating progressive disease that weakens the pharyngeal muscles, causing severe swallowing difficulties (dysphagia).¹ Progressive dysphagia impacts 97% of OPMD patients and is a severe, life-threatening complication of OPMD which can lead to chronic choking, malnutrition, aspiration pneumonia and death.

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. BB-301 is administered locally, in a one-time procedure, to the muscles in the throat that drive the swallowing process, an approach designed to maximize local benefit and minimize systemic exposure. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by slowing or halting mutant PABPN1 expression while simultaneously 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 and is currently being evaluated in a Phase 1b/2a, first-in-human, open-label dose escalation study to evaluate the safety and clinical activity of intramuscular doses of BB-301 administered to subjects with OPMD (NCT06185673).

About Benitec Biopharma Inc.

Benitec Biopharma Inc. (NASDAQ: BNTC) is a clinical-stage biotechnology company developing disease-modifying genetic medicines designed to improve the lives of people with life-threatening, genetically defined diseases. The Company’s proprietary “Silence and Replace” DNA-directed RNA interference (ddRNAi) platform combines RNA interference, or RNAi, with gene therapy to create medicines designed to facilitate sustained silencing of disease-causing genes and simultaneous delivery of replacement genes that restore normal cellular function following a single administration. The Company’s lead investigational candidate, BB-301, is the first and only disease-modifying genetic medicine in clinical development for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD)-related dysphagia. For additional information, visit 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, within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding Benitec’s plans to develop and commercialize its product candidates, whether the interim clinical results described in this press release, including the durability of treatment effects observed to date, will be maintained or confirmed with additional follow-up or in later-stage clinical trials, the timing of the completion of preclinical and clinical trials, the anticipated timing of initiation and the design of our planned pivotal study of BB-301, 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, the potential for BB-301 to establish a new treatment paradigm for, and to be a transformative therapy for, patients with OPMD-related dysphagia, and the clinical utility and potential attributes and benefits of ddRNAi and Benitec’s product candidates, and other forward-looking statements. Words such as “anticipate”, “believe”, “continue”, “could”, “designed”, “estimate”, “expect”, “intend”, “may”, “plan”, “potential”, “should”, “will” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

These forward-looking statements are based on the Company’s current expectations and assumptions and are 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 ongoing and 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; regulatory developments in the United States of America; 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 and market competition; reliance on third party manufacturers and suppliers, and the risks associated with manufacturing complexity, supply interruptions, and compliance with current good manufacturing practices, sales, marketing and distribution requirements for our product candidates; greater than expected expenses; including expenses relating to litigation or strategic activities; our incurrence of significant losses and the uncertainty of our ability to achieve or sustain profitability or generate any revenue; the Company’s ability to satisfy its capital needs through increasing revenue and obtaining additional financing; the impact of local, regional, national and international economic conditions and events; including geopolitical instability, and risks associated with conducting business and seeking regulatory approvals in international markets; our ability to attract and retain key management, scientific, and technical personnel; cyber-security threats and vulnerabilities in our and our third parties' information technology systems; the impact of legislative and regulatory reforms affecting drug pricing and reimbursement, including Medicare drug-price negotiation and government pricing frameworks; potential product liability claims arising from the use of our product candidates in clinical trials or following any future marketing approval; physicians, patients, third-party payers, or others in the medical community may not be receptive to our product candidates, and we may not generate any future revenue from the sale or licensing of our product candidates; disruptions to U.S. government agency operations, including FDA staffing changes; and other risks detailed from time to time in the Company’s reports filed with the Securities and Exchange Commission (the “SEC”), including under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the fiscal year ended June 30, 2026 and subsequent Quarterly Reports on Form 10-Q filed with the SEC. Forward-looking statements speak only as of the date of this press release, and readers are cautioned not to place undue reliance on them. The Company disclaims any intent or obligation to update these forward-looking statements, except as required by law.

References:

1. https://www.mayoclinic.org/diseases-conditions/dysphagia/symptoms-causes/syc-20372028
   
   

Media Contact:
Audra Friis
Sam Brown Healthcare Communications
(917) 519-9577
audrafriis@sambrown.com

Investor Relations Contact: 
Candice Masse
Astr Partners
(978) 879-7273
candice.masse@astrpartners.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did Benitec's BB-301 Phase 1b/2a trial show?

All six low-dose and all three high-dose patients qualified as formal responders. At three months, high-dose patients had higher response rates for throat emptying and timed water drinking, while response rates for the swallowing questionnaire and throat closing were equal between groups.

Were serious adverse events reported in Benitec's BB-301 trial?

No serious adverse events have occurred in any patient treated with BB-301 to date.

How is Benitec's BB-301 Phase 1b/2a study designed?

The BB-301 study is a first-in-human, open-label dose-escalation trial, meaning treatment is known and different dose levels are evaluated. It assesses safety and clinical activity in patients with OPMD. BB-301 is administered directly into throat muscles in a one-time procedure.