Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Corporate Update (November 2025)
31 slides from Benitec Biopharma's corporate update, Nov 3, 2025
Slide 1: Positive Clinical Data Supporting Advancement of BB-301 in OPMD
Positive Clinical Data Supporting Advancement of BB-301 in OPMD WEBCAST: November 3, 2025 ©2025 Benitec Biopharma. All rights reserved. OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) A Debilitating, Progressive Disease With No Approved Therapies NASDAQ BNTC
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: Positive Interim Data and Regulatory Milestones Position BB-301
SAFETY NO TREATMENT-RELATED SEVERE ADVERSE EVENTS REGULATORY FAST TRACK DESIGNATION (FDA) Granted following review of positive interim clinical results and proprietary OPMD responder analysis ORPHAN DRUG DESIGNATION (FDA & EMA) Recognizes BB-301’s potential to address a rare, life-threatening diseas
Slide 4: Oculopharyngeal Muscular Dystrophy BB-301: A Novel Disease-Modifying Genetic Medicine
Oculopharyngeal Muscular Dystrophy BB-301: A Novel Disease-Modifying Genetic Medicine
Slide 5: 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 Eyelid drooping (ptosis) which can be asymmetric at onset Difficulty swallowing (dysphagia) and choking during meals Proximal limb weakness PABPN1 is a ubiqui
Slide 6: Swallowing Overview and Rationale for BB-301 Evaluation in OPMD
Swallowing Overview and the Rationale for BB-301 Evaluation in Oculopharyngeal Muscular Dystrophy (OPMD) 1. https://www.mayoclinic.org/diseases-conditions/dysphagia/symptoms-causes/syc-20372028 2. Rudney et al. (1995) Anatomical Structures of the Pharynx and BB-301 Injection Sites BB-301 Middle phar
Slide 7: BB-301: A Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia
* Strings-Ufombah, et al., Molecular Therapy: Nucleic Acids, Vol. 24, 67-78, June 2021 BB-301: A Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia NOVEL “SILENCE AND REPLACE” PLATFORM BB-301 uses DNA-directed RNA interference (ddRNAi) to simultaneously silence the mutant gene causing OPM
Slide 8: Study Design and Key Efficacy Endpoints of the OPMD Natural History Study and BB-301 Phase 1b/2a
Study Design and Key Efficacy Endpoints of the OPMD Natural History Study and BB-301 Phase 1b/2a Clinical Study
Slide 9: BB-301: Clinical Development Program
5 Clinical follow-up visits during which pre-treatment baseline assessments are carried out for each patient Natural History Study patients enroll into the Phase 1b/2a Study 4 Clinical follow-up visits during which post-treatment assessments are carried out for each patient BB-301: Clinical Developm
Slide 10: 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 The total dysphagic symptom burden experienced by OPMD patients has several known underlying contributors The serial assessments of dysphagia facilitated the cr
Slide 11: BB-301: Cohort 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 dat
Slide 12: 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 Worsening Improving Total SSQ Score Improves Over Time Following BB-301 Admin
Slide 13: Improvement in Pharyngeal Area at Maximum Pharyngeal Constriction (PhAMPC)
Worsening Improving Improving Worsening 40% decline in PhAMPC post-BB-301 treatment 42% decline in PhAMPC post-BB-301 treatment BB-301: Cohort 1 Demonstrated a Highly Clinically Meaningful Improvement in Pharyngeal Area at Maximum Pharyngeal Constriction (PhAMPC) PhAMPC Improves Over Time Following
Slide 14: Improvement in Total Pharyngeal Residue (TPR)
BB-301: Cohort 1 Demonstrated a Highly Clinically Meaningful Improvement in Total Pharyngeal Residue (TPR) Worsening Improving Improving Worsening TPR Improves Over Time Following BB-301 Administration and the Statistical Requirement for Improvement Has Been Achieved 45% decline in TPR post-BB-301 t
Slide 15: 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) 69% decline in NRRSv post-BB-301 treatment 63% decline in NRRSv post-BB-301 treatment NRRSv Improves Over Time Following BB-301 Administration and the Statistical Req
Slide 16: 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 Worsening Improving BB-301: Cohort 1 Demonstrated a Clinically Meaningful Improvement in Cold Water Timed Drinking Test (CWDT) CWDT Improves Over Time Following BB-30
Slide 17: Patients Serve as Their Own Control; Pre- and Post-Treatment Results
Without intervention, the functional elements of the swallowing process worsen over time for OPMD patients, as shown in the pre-treatment results for Cohort 1 patients. The progressive loss of swallowing function drives the clinically significant symptom burden experienced by the OPMD patients. Pre-
Slide 18: BB-301 Responder Analyses: A Framework for Evaluation of Efficacy
BB-301 Responder Analyses: A Framework for Evaluation of Efficacy The total dysphagic symptom burden experienced by OPMD patients has several known underlying contributors Development of a multi-component composite endpoint to evaluate the potential treatment effects of BB-301 allows for incorporati
Slide 19: Responder Analysis Requirements and Scoring
Assessment Requirement for a Responder Responder Scoring Patient-Reported Oral-Pharyngeal Dysphagia determined by SSQ Total Score SSQ must not worsen over the post-treatment period (if this criteria is not met, then no further analyses are done; the patient is not a responder). Maximum score of 1 fo
Slide 20: BB-301: 100% of Patients in Cohort 1 (n = 6) Were Responders
Patient Follow-Up at the time of Fast Track Submission Response Criteria Achieved Total Responder Score Updated Follow-Up post Fast Track Submission* Updated Response Criteria Achieved* Updated Total Responder Score* Responder? Patient 1 12- months SSQ, PhAMPC, Pharyngeal residue, CWDT 4 Patient 2 1
Slide 21: Summary of Clinical and Regulatory Results
Distinct causes of their respective total dysphagic symptom burdens were safely treated with BB-301 Fast Track Designation was granted by the FDA FDA and EMA Orphan Drug Designation First patient in Cohort 2 successfully treated with BB-301 The company plans to engage the FDA in 2026 to confirm deta
Slide 22: Thank You
www.benitec.com ©2025 Benitec Biopharma. All rights reserved. THANK YOU! NASDAQ BNTC
Slide 23: Appendix
Appendix
Slide 24: Natural History Study Data Informs Pivotal Study Design and Responder Analysis
OPMD-Related Dysphagia Natural History Study Data Informs Putative Pivotal Study Design and a Potential Proprietary Responder Analysis Transformative BB-301 Phase 1b/2a Clinical Study Results High Unmet Need and Significant Market Opportunity There are no therapeutic interventions approved for OPMD
Slide 25: Robust, Blinded Methods are Utilized to Evaluate Swallowing
Swallowing is an involuntary process: The standardized swallowing tasks employed during the VFSS are not impacted by the effort of the patient, as the autonomic nervous system controls the involuntary muscle movements comprising the pharyngeal phase of swallowing Blinded assessments: All Study patie
Slide 26: Patient-Reported Oral-Pharyngeal Dysphagia: As Assessed by the SSQ
Szczesniak, M., et al, Dysphagia, 2014 Patient-Reported Oral-Pharyngeal Dysphagia: As Assessed by the SSQ Patient-reported oral-pharyngeal dysphagia as assessed by the SSQ: The SSQ is a 17-item self-report inventory assessing subjective symptoms of oral-pharyngeal dysphagia The questionnaire uses a
Slide 27: Pharyngeal Constrictor Muscle Function: As Measured by PhAMPC
Steele, C., et al. ASHA 2019 Convention Session on the ASPEKT C Method of Videofluoroscopy Analysis 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) oc
Slide 28: Swallowing Inefficiency: As Measured by Post-Swallow TPR
Steele, C., et al. ASHA 2019 Convention Session on the ASPEKT C Method of Videofluoroscopy Analysis 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 bo
Slide 29: Swallowing Inefficiency: As Measured by NRRSv
1. Pearson W.G. et. al., Dysphagia, 2013; 2. Steele, C.M. et. al., Journal of Speech, Language and Hearing Research, 2019; 3. Molfenter S.M. et. al., Dysphagia, 2013; 4. Kroon, R.H.M.J.M. et. al., Neurology, 2021 Swallowing Inefficiency: As Measured by NRRSv, a Measure of Vallecular Residue NRRS inc
Slide 30: Swallowing Ineffectiveness: Frequency of Pathologic Sequential Swallows (SEQ)
1. Ambrocio, K. R., et al., Dysphagia (2023) 38:1497–1510; 2. Steele, C., et al., Journal of Speech, Language, and Hearing Research, Vol. 62, 1338–1363, May 2019 Swallowing Ineffectiveness: As Measured by the Frequency of Pathologic Sequential Swallows (SEQ) Sequential swallowing is defined as the c
Slide 31: Functional Swallowing Capacity: As Measured by the CWDT
Functional Swallowing Capacity: As Measured by the CWDT 1. Nathardwarawala, K.M. et. al. Journal of Neurology, Neurosurgery, and Psychiatry, 1992; 2. Codère, F. et. al., Orbit, 2001; 3. Côté, C. et. al., Dysphagia, 2024; 4. Périé, S. et. al., Molecular Therapy, 2013 The CWDT measures the time requir