Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — American Society of Cell & Gene Therapy 2026
20 slides from Benitec Biopharma's conference presentation, May 15, 2026
Slide 1: Durable Responses to Low Dose Investigational Gene Therapy BB-301 in OPMD
Durable Responses to Low Dose Investigational Gene Therapy BB-301 in Patients with Oculopharyngeal Muscular Dystrophy with Dysphagia at 12-Months and 24- Months and Improved Depth of Response to High Dose BB- 301 American Society of Cell & Gene Therapy May 15, 2026 NASDAQ BNTC ©2026 Benitec Biopharm
Slide 2: Safe Harbor Statement
Safe Harbor Statement Except for the historical information set forth herein, the matters set forth in this presentation include forward-looking statements, including statements regarding Benitec’s plans to develop and commercialize its product candidates, the timing of the completion of pre-clinica
Slide 3: Oculopharyngeal Muscular Dystrophy (OPMD)
Oculopharyngeal Muscular Dystrophy (OPMD) A rare autosomal-dominant degenerative muscle disorder
Slide 4: OPMD is a Debilitating Progressive Disease With No Approved Therapies
OPMD is a Debilitating Progressive Disease With No Approved Therapies OPMD ONSET: TYPICAL AGE IS 40s-50s OPMD is a rare autosomal-dominant Eyelid drooping (ptosis) Difficulty swallowing (dysphagia) degenerative muscle disorder, caused by a and choking during meals mutation in the poly(A)-binding pro
Slide 5: Swallowing Overview and the Rationale for BB-301 Evaluation in OPMD
Swallowing Overview and the Rationale for BB-301 Evaluation in OPMD Anatomical Structures of the Pharynx and BB-301 Injection Sites OPMD weakens pharyngeal muscles, causing severe swallowing difficulties (dysphagia) BB-301 is designed to increase muscle mass and function, reducing dysphagia and impr
Slide 6: BB-301: A Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia
C BB-301: A Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia
Slide 7: BB-301: Mechanism of Action
BB-301: Mechanism of Action BB-301 AAV9-PL BB-301 uses DNA-directed RNA interference (ddRNAi) to simultaneously SILENCE + REPLACE T HE WIT H A Muscle ITR ITR mutant gene functional gene Specific coPABPN1 shmiR-17 shmiR-13 Promoter potentially providing a permanent solution with a Replaces Silences w
Slide 8: Clinical Study Design and Key Efficacy Endpoints
Clinical Study Design and Key Efficacy Endpoints C of the OPMD Natural History Study and the BB-301 Phase 1b/2a First-in-Human Study
Slide 9: BB-301: Clinical Development Program
BB-301: Clinical Development Program BB-301 Phase 1b/2a OPMD Natural History Study Dosing Open-Label Dose (26 weeks) (1 Day) Escalation Study (52 weeks) 5 Clinical follow-up visits during which pre-treatment baseline Natural History Study patients enroll into the 4 Clinical follow-up visits during w
Slide 10: BB-301: Serial Characterization of Dysphagia Severity Informs Efficacy Assessment
BB-301: Serial Characterization of Dysphagia Severity Informs Efficacy Assessment • The Natural History Study and BB-301 Phase 1b/2a Clinical Study include comprehensive assessments of dysphagia approximately every 3 months Efficacy assessments were derived from literature- • The total dysphagic sym
Slide 11: Scoring Clinical Success: BB-301's Responder Framework
Scoring Clinical Success: BB-301’s Responder Framework 1. Patient-Reported Oral-Pharyngeal Dysphagia (SSQ) TOTAL Criteria: SSQ score must not worsen after treatment RESPONDER 2. Pharyngeal Constrictor Muscle Function (PhAMPC) Criteria: Statistical criteria* met in at least 1 of 4 swallowing tasks SC
Slide 12: BB-301 Phase 1b Study: Cohort 1 Study Completers
BB-301 Phase 1b Study: Cohort 1 Study C Completers
Slide 13: All Cohort 1 Study Completers are Formal Responders
All Cohort 1 Study Completers are Formal Responders Completers are Patients that have reached the 12-month post-BB-301-treatment assessment time-point May 2026 Benitec Corporate Overview | Non-Confidential 13
Slide 14: BB-301 Phase 1b Study: Long Term Efficacy Trends for Cohort 1
BB-301 Phase 1b Study: Long Term Efficacy C Trends for Cohort 1
Slide 15: Long-Term Efficacy Trends: 24-months Post BB-301 Treatment
Long-Term Efficacy Trends: 24-months Post BB-301 Treatment, Patient 1 of Cohort 1 Experienced Continued Robust, Disease-Modifying, Outcomes Patient 1 of Cohort 1 experienced deepening improvements in both post-swallow pharyngeal residue and total dysphagic symptom burden 24-months post-BB-301 treatm
Slide 16: BB-301 Phase 1b Study: Cohort 1 Low Dose vs. Cohort 2 High Dose
BB-301 Phase 1b Study: Cohort 1 Dose C vs. Cohort 2 Dose Preliminary comparisons of efficacy trends
Slide 17: Significant Improvements in Throat Closure, Pharyngeal Residue, and Dysphagic Symptom Burden
Significant Improvements in Throat Closure, Post-Swallow Pharyngeal Residue, and Total Dysphagic Symptom Burden Observed Following High Dose BB-301 Treatment • Patient A (Cohort 1) and Patient B (Cohort 2) had comparable baseline functional and anatomical deficits. • Patient B (Cohort 2) was safely
Slide 18: BB-301 Responder Status: Interim Clinical Results (3-Months)
BB-301 Responder Status: Interim Clinical Results (3-Months Post-BB-301 Treatment) Indicate the First Patient in Cohort 2 is a Responder to BB-301 May 2026 Benitec Corporate Overview | Non-Confidential 18
Slide 19: Positive Interim Clinical Results and Regulatory Milestones
Positive Interim Clinical Results and Regulatory Milestones Position BB-301 as the First Potential Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia P H A S E 1 b / 2 a C L I N I C A L E F F I C A C Y S A F E T Y ROBUST RESPONSES TO BB-301 IN COHORT 1 AND COHORT 2 NO TREATMENT-RELATED SE
Slide 20: THANK YOU!
SILENCE + REPLACE One Platform. One Dose. Potentially Transformative Outcomes. THANK YOU! w w w. b e n i t e c . c o m
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