Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Investor Presentation (January 2026)
43 slides from Benitec Biopharma's investor presentation, January 2026
Slide 1: BB-301: Potential First-and-Only Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia
BB-301: Potential First-and-Only Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia Novel combination of gene replacement and permanent gene silencing revolutionizes treatment paradigms for human disease. January 2026 NASDAQ BNTC ©2026 Benitec Biopharma. All rights reserved.
Slide 2: Safe Harbor Statement
Safe Harbor Statement 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-clinic
Slide 3: Redefining the Future of Treatment
REDEFINING THE FUTURE OF TREATMENT Oculopharyngeal Muscular Dystrophy (OPMD) has long been a story of suffering and stalled science. Benitec’s BB-301 is changing the narrative, delivering results never seen before.
Slide 4: Benitec: Transforming Rare Disease Therapy with One-Time Genetic Medicine
Benitec: Transforming Rare Disease Therapy with One-Time Genetic Medicine DIFFERENTIATED LEAD ASSET: WHAT SETS PLATFORM BB-301 BENITEC APART SILENCE + REPLACE First and only clinical-stage therapy ü First-mover advantage in a a single-vector approach to for OPMD-related dysphagia defined rare diseas
Slide 5: Oculopharyngeal Muscular Dystrophy (OPMD)
Oculopharyngeal Muscular Dystrophy (OPMD) A rare autosomal-dominant degenerative muscle disorder
Slide 6: OPMD is a Debilitating Progressive Disease With No Approved Therapies
Oculopharyngeal Muscular Dystrophy (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 degenerative muscle disorder, caused (dysphagia) and choking by a mutation in
Slide 7: Swallowing Overview and the Rationale for BB-301 Evaluation in OPMD
Swallowing Overview and the Rationale for BB-301 Evaluation in Oculopharyngeal Muscular Dystrophy (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
Slide 8: BB-301: A Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia
BB-301: C A Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia
Slide 9: What Sets BB-301 Apart
What Sets BB-301 Apart ADDRESSING UNMET NEEDS The ddRNAi approach can tackle diseases that are not treatable by gene silencing or gene therapy alone BB-301uses DNA-directed RNA interference (ddRNAi) to DELIVERING A TARGETED simultaneously GENE THERAPY BB-301 is designed to block production of the ha
Slide 10: BB-301: Mechanism of Action
BB-301: Mechanism of Action BB-301 AAV9-PL Muscle ITR ITR Specific coPABPN1 shmiR-17 shmiR-13 Promoter Replaces Silences with a healthy gene mutant gene coPABPN1 is insensitive to shRNA Restoration of functional PABPN1 Strings-Ufombah, et al., Molecular Therapy: Nucleic Acids, Vol. 24, 67-78, June 2
Slide 11: Positive Interim Clinical Study Results and Regulatory Milestones Position BB-301
Positive Interim Clinical Study Results and Regulatory Milestones Position BB-301 as the First Potential Disease-Modifying Therapy for OPMD BB-301 PHASE 1b/2a CLINICAL EFFICACY R E G U L AT O R Y 100% Significant All patients safely Cohort 2 dosing Fast Track (FDA) responder rate improvements in tre
Slide 12: Gene Therapy/RNA Interference Competitive Landscape Overview
Gene Therapy/RNA Interference Completive Landscape Overview Approach Focus Modality Differentiation vs. ddRNAi Gene silencing and replacement ddRNAi No major competitor is delivering a single-vector, dual-function therapy with Gene silencing RNAi (siRNA) Requires repeat dosing both: • Silencing of m
Slide 13: Study Design and Key Efficacy Endpoints of the OPMD Natural History Study and BB-301
Study Design and Key Efficacy Endpoints C of the OPMD Natural History Study and BB-301 Phase 1b/2a Clinical Study
Slide 14: 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 Natural History Study patients enroll into the 4 Clinical follow-up visits during which post
Slide 15: 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-based methods used to asse
Slide 16: BB-301: Cohort 1 Pre-Treatment Trends and Post-Treatment Trends
BB-301: Cohort 1 Pre-Treatment Trends and Post-Treatment Trends • The pre-treatment period includes the first six months of natural history study follow-up and the screening visit for enrollment in the Phase 1b/2a study • The interim post-treatment period includes all clinical assessments accrued to
Slide 17: Improvement in Sydney Swallow Questionnaire (SSQ) Total Score
BB-301: Cohort 1 Demonstrated a Highly Clinically Meaningful Improvement in Sydney Swallow Questionnaire (SSQ) Total Score • Pre-Treatment = 586 • Post-Treatment = 258 • 328-point decline in SSQ Total Score post-BB-301 treatment Pre-Treatment SSQ Total Score Post-Treatment SSQ Total Score 800 800 70
Slide 18: Improvement in Pharyngeal Area at Maximum Pharyngeal Constriction (PhAMPC)
BB-301: Cohort 1 Demonstrated a Highly Clinically Meaningful Improvement in Pharyngeal Area at Maximum Pharyngeal Constriction (PhAMPC) Pre-Treatment PhAMPC, Liquids Post-Treatment PhAMPC, Liquids 30 30 25 25 40% decline in PhAMPC Improving PhAMPC (%) PhAMPC (%) Worsening 20 20 15 R² = 0.9 15 post-B
Slide 19: Improvement in Total Pharyngeal Residue (TPR)
BB-301: Cohort 1 Demonstrated a Highly Clinically Meaningful Improvement in Total Pharyngeal Residue (TPR) Pre-Treatment TPR, Liquids Post-Treatment TPR, Liquids 20 20 16 16 Improving R² = 1.0 Worsening TPR (%) 12 TPR (%) 12 R² = 0.8 45% decline in TPR 8 8 post-BB-301 treatment 4 4 0 0 NH-S creening
Slide 20: Improvement in Normalized Residue Ratio Scale for the Valleculae (NRRSv)
BB-301: Cohort 1 Demonstrated a Highly Clinically Meaningful Improvement in Normalized Residue Ratio Scale for the Valleculae (NRRSv) 0.5 Pre-Treatment NRRSv Liquids 0.5 Post-Treatment NRRSv Liquids 0.4 0.4 Improving Worsening 0.3 0.3 NRRSv NRRSv R² = 0.9 R² = 0.6 0.2 0.2 69% decline in NRRSv 0.1 0.
Slide 21: Improvement in Cold Water Timed Drinking Test (CWDT)
BB-301: Cohort 1 Demonstrated a Clinically Meaningful Improvement in Cold Water Timed Drinking Test (CWDT) • Pre-Treatment = 18.1 seconds • Post-Treatment = 11.0 seconds • Post-treatment scores were reduced as compared to the pre-treatment period Pre-Treatment CWDT Post-Treatment CWDT 25 20 20 15 Im
Slide 22: BB-301 Demonstrated Clinically Meaningful Improvements In Cohort 1
BB-301 Demonstrated Clinically Meaningful Improvements In Cohort 1 BEFORE TREATMENT AFTER BB-301 TREATMENT • OPMD patients showed progressive • Each patient serves as their own control, enabling clear worsening of swallowing function and measurement of change (FDA highlighted this approach in the sy
Slide 23: BB-301 Responder Analyses: A Framework for Evaluation of Efficacy
BB-301 Responder Analyses: A Framework for Evaluation of Efficacy WHAT IS IT? HOW DOES IT WORK? WHY DOES IT MATTER? A simple, multi-part scoring Each patient is evaluated across a combination of efficacy The total dysphagic symptom burden experienced system that allows for assessments which were der
Slide 24: Scoring Clinical Success: BB-301's Responder Framework
Scoring Clinical Success: BB-301’s Responder Framework 1. Patient-Reported Oral-Pharyngeal Dysphagia (SSQ) Criteria: SSQ score must not worsen after treatment TOTAL 2. Pharyngeal Constrictor Muscle Function (PhAMPC) RESPONDER Criteria: Statistical criteria* met in at least 1 of 4 swallowing tasks SC
Slide 25: BB-301: 100% of Patients in Cohort 1 (n=6) Were Responders
BB-301: 100% of Patients in Cohort 1 (n=6) Were Responders Follow-Up at the Time of Fast Total Updated Follow-Up Updated Total Track Response Criteria Responder Post Fast Track Updated Response Responder Patient Submission Achieved Score Submission* Criteria Achieved* Score* Responder Total Responde
Slide 26: BB-301: Transformative Potential for Patients with OPMD
BB-301: Transformative Potential for Patients with Oculopharyngeal Muscular Dystrophy (OPMD) COHORT 1 COHORT 2 REGULATORY MILESTONES All 6 patients with All 6 patients First patient in Fast Track The company plans distinct causes of experienced Cohort 2 successfully Designation was to engage the FDA
Slide 27: If approved, BB-301's OPMD rare disease market is ~15K addressable patients
Addressable OPMD Market If approved, BB-301’s • There are currently no treatment options OPMD rare disease approved for OPMD market is ~15K • OPMD is diagnosed in ~35 countries addressable patients • ~15,000 patients total across U.S., Canada, Europe across North America, and Israel Europe and Israe
Slide 28: Positive Interim Clinical Results and Regulatory Milestones Position BB-301
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 NO TREATMENT-RELATED SEVERE 100% RESPONDER RATE IN COHORT 1 ADVERSE EVENTS
Slide 29: Benitec: Driving the Future of Genetic Medicine
C Benitec: Driving the Future of Genetic Medicine
Slide 30: Distinguished Leadership Team
Distinguished Leadership Team M A N A G E M E N T B O A R D O F D I R E C T O R S Jerel Banks, Jerel Banks, M.D., Ph.D. M.D., Ph.D. Peter Edward Francis Smith Chief Executive Officer & Chief Executive Officer & Chairman of the Board Chairman of the Board Director Director Megan Megan Dr. Sharon Bost
Slide 31: Strong Financial Position and Clear Milestones Driving Benitec's Path
Strong Financial Position and Clear Milestones Driving Benitec’s Path To Pivotal Data and Commercialization BB-301 Key Milestones On track for 2026 FDA meeting Pivotal Study Planning Financial Snapshot Initiated Q4 2025 Cash and Cash $188.6 M* Cohort 2 Dosing Equivalents: Fast Track (FDA) and Orphan
Slide 32: SILENCE + REPLACE
SILENCE + REPLACE One Platform. One Dose. Potentially Transformative Outcomes. NASDAQ BNTC
Slide 33: Thank You!
THANK YOU! w w w. b e n i t e c . c o m NASDAQ BNTC ©2026 Benitec Biopharma. All rights reserved.
Slide 34: Appendix
Appendix
Slide 35: BB-301: Potential One-Time Treatment Designed to Address the Root Cause of OPMD
BB-301: Potential One-Time Treatment Designed to Address the Root Cause of Oculopharyngeal Muscular Dystrophy (OPMD)-Related Dysphagia • There are no therapeutic interventions approved for OPMD High Unmet Need and • BB-301 is the only clinical-stage therapeutic agent in development designed to treat
Slide 36: Robust, Blinded Methods are Utilized to Evaluate the Dysphagic Symptom Burden
Robust, Blinded Methods are Utilized to Evaluate the Dysphagic Symptom Burden of each Study patient • Patient-Reported Outcome Assessments: The SSQ is • Swallowing is an involuntary process: The standardized employed to serially evaluate the severity of dysphagia swallowing tasks employed during the
Slide 37: Patient-Reported Oral-Pharyngeal Dysphagia: As Assessed by the SSQ
Patient-Reported Oral-Pharyngeal Dysphagia: As Assessed by the SSQ Patient-reported oral-pharyngeal dysphagia as assessed by the SSQ Example Questions SSQ: • The SSQ is a 17-item self-report inventory assessing subjective symptoms of oral-pharyngeal dysphagia • The questionnaire uses a 100-mm long v
Slide 38: An Estimate of Pharyngeal Constrictor Muscle Function: As Measured by PhAMPC
An Estimate of Pharyngeal Constrictor Muscle Function: As Measured by PhAMPC • PhAMPC characterizes the area of the pharynx at the point of maximum constriction during swallowing • Measurement (yellow) occurs on the videofluoroscopy frame with the smallest amount of unobliterated air space and bariu
Slide 39: Swallowing Inefficiency: As Measured by Post-Swallow TPR
Swallowing Inefficiency: As Measured by Post-Swallow TPR • Total Pharyngeal Residue measurement comprises the amount of food or liquid material remaining in the pharynx after the first swallow of the bolus • Normal Total Pharyngeal Residue values should be close to zero C2-C4 length squared serves a
Slide 40: Swallowing Inefficiency: As Measured by NRRSv
Swallowing Inefficiency: As Measured by NRRSv, a Measure of Vallecular Residue • NRRS incorporates the ratio of residue occupying a pharyngeal space and a proportion expressing the area of observed residue normalized against the size of an individual1 • Pharyngeal residue constitutes a risk for post
Slide 41: Swallowing Ineffectiveness: Frequency of Pathologic Sequential Swallows (SEQ)
Swallowing Ineffectiveness: As Measured by the Frequency of Pathologic Sequential Swallows (SEQ) Sequential swallowing is defined as the completion of two or more consecutive swallows in rapid succession where the hyolaryngeal complex (HLC) does not return to rest and the pharynx lacks complete pate
Slide 42: Functional Swallowing Capacity: As Measured by the CWDT
Functional Swallowing Capacity: As Measured by the CWDT • The CWDT measures the time required for a patient to swallow 80 mL of ice-cold water1 • The CWDT (dysphagia documented by the inability to swallow 80 mL of cold water in less than 8 seconds), alongside family history of the disease and the pr
Slide 43: Proprietary OPMD Responder Analysis Description
Proprietary OPMD Responder Analysis Description Assessment Requirement for a Responder Responder Scoring Patient-Reported Oral-Pharyngeal Dysphagia SSQ must not worsen over the post-treatment period (if this Maximum score of 1 for this component determined by SSQ Total Score criteria is not met, the
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