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©2026 BenitecBiopharma. All rights reserved. NASDAQBNTC BB-301: Potential First-and-Only Disease-Modifying Genetic Medicine for OPMD-Related DysphagiaNovel combination of gene replacement and permanent gene silencing revolutionizes treatment paradigms for human disease. January 2026
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Safe Harbor StatementExcept for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitec’splans 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 the clinical utility and potential attributes and benefits of ddRNAiand Benitec’sproduct 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 maycause 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 regulatoryfilings 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 ddRNAiand 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 needsand 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 andthe 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 impactof local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Company’s reports filed withthe Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements. January 2026Benitec Corporate Overview | Non-Confidential2
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REDEFINING THE FUTURE OF TREATMENTOculopharyngeal 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.
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Benitec: Transforming Rare Disease Therapy with One-Time Genetic Medicine January 2026Benitec Corporate Overview | Non-Confidential4 DIFFERENTIATED PLATFORMSILENCE + REPLACEa single-vector approach to simultaneously silence the mutant gene causing OPMD and deliver a healthy, functional gene LEAD ASSET: BB-301First and only clinical-stage therapy for OPMD-related dysphagia100% responder rate in Cohort 1Pivotal study planning underwayFast Track from FDA and Orphan Drug Designations from FDA/EMA validate clinical and regulatory differentiation WHAT SETS BENITEC APARTüFirst-mover advantage in a defined rare diseaseüProprietary OPMD Natural History dataüProprietary, local delivery method to target tissues One Platform. One Dose. Potentially Transformative Outcomes.
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Oculopharyngeal Muscular Dystrophy (OPMD)A rare autosomal-dominant degenerative muscle disorder
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Oculopharyngeal Muscular Dystrophy (OPMD) is a Debilitating Progressive Disease With No Approved TherapiesOPMD ONSET: TYPICAL AGE IS 40s-50sEyelid drooping (ptosis)Difficulty swallowing (dysphagia) and choking during mealsProximal limb weaknessPABPN1 is a ubiquitous protein that controls the length of mRNA poly(A) tails, mRNA export from the nucleus and alternative poly(A) site usage 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 BB-301 is the only clinical-stage therapeutic in development designed to treat dysphagia in patients with OPMD OPMD is a rare autosomal-dominant degenerative muscle disorder, caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene Patients in North America, Europe and Israel~15KJanuary 2026Benitec Corporate Overview | Non-Confidential6
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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-301Middle pharyngeal constrictor Inferior pharyngeal constrictor OPMD weakens pharyngeal muscles, causing severe swallowing difficulties (dysphagia) Dysphagia leads to choking, malnutrition, aspiration pneumonia1 times per hour a healthy human will spontaneously swallow218-400 BB-301 is designed to increase muscle mass and function, reducing dysphagia and improving patient outcomes BB-301 is delivered to the pharyngeal constrictor muscles via direct intramuscular injection in the operating room January 2026Benitec Corporate Overview | Non-Confidential7
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C BB-301: A Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia
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What Sets BB-301 Apart January 2026Benitec Corporate Overview | Non-Confidential9 The ddRNAi approach can tackle diseases that are not treatable by gene silencing or gene therapy aloneADDRESSING UNMET NEEDS BB-301 is designed to block production of the harmful PABPN1 protein and restore normal muscle function by supplying a new, functional version of the protein DELIVERING A TARGETED GENE THERAPY Administered directly into the pharyngeal constrictor muscles via intramuscular injection in the operating room, BB-301 maximizes local benefit and minimizes systemic exposure MAXIMIZING LOCAL IMPACT WITH PRECISE DELIVERY Evaluated at doses of 1.2e13 vg/patient (Cohort 1) and 1.8e13 vg/patient (Cohort 2)OPTIMIZING DOSE FOR SAFETY AND EFFICACY THE mutant gene BB-301uses DNA-directed RNA interference (ddRNAi) to simultaneously WITH A functional geneSILENCE + REPLACEpotentially providing a permanent solution with a single administration
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Strings-Ufombah, et al., Molecular Therapy: Nucleic Acids, Vol. 24, 67-78, June 2021 BB-301: Mechanism of Action January 2026Benitec Corporate Overview | Non-Confidential10 AAV9-PL BB-301 ITR ITRMuscle Specific PromotercoPABPN1shmiR-17shmiR-13 Replaceswith a healthy geneSilencesmutant gene coPABPN1 is insensitive to shRNA Restoration of functional PABPN1
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Positive Interim Clinical Study Results and Regulatory Milestones Position BB-301 as the First Potential Disease-Modifying Therapy for OPMD January 2026Benitec Corporate Overview | Non-Confidential11 100% responder rate in Cohort 1 (proprietary analysis) Cohort 2 dosing initiated, pivotal study planning underway All patients safely treated with BB-301No treatment-related severe adverse events Fast Track (FDA) and Orphan Drug Designations (FDA/EMA) Significant improvements in swallowing function and quality of life BB-301 PHASE 1b/2a CLINICAL EFFICACYREGULATORY patient reported outcome measures over timethroat closure over timefunctional swallowing capacity over timepharyngeal residue over timeIMPROVEDIMPROVEDREDUCEDIMPROVED Benitec plans to meet with the FDA in 2026 to confirm all key elements of the anticipated BB-301 Pivotal Study design
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Gene Therapy/RNA Interference Completive Landscape Overview January 2026Benitec Corporate Overview | Non-Confidential12 Approach FocusModalityDifferentiation vs. ddRNAiGene silencing and replacementddRNAi Gene silencingRNAi (siRNA)Requires repeat dosing Gene silencingASOsFrequent injectionsNo gene replacementGene replacementAAV Gene TherapyNo silencing of mutant gene Gene editingCRISPR/EditingOff-target riskComplex deliveryDelivery technologyCapsid EngineeringNot a therapeutic modality No major competitor is delivering a single-vector, dual-function therapy with both:•Silencing of mutant gene,•Simultaneous replacement with a healthy geneUsing a single administration with the potential for long-term durability.This represents a clear point of differentiation vs. RNAi, ASOs, traditional AAV, or CRISPR approach
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C Study Design and Key Efficacy Endpoints of the OPMD Natural History Study and BB-301 Phase 1b/2a Clinical Study
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BB-301 Phase 1b/2a Open-Label Dose Escalation Study(52 weeks)5 Clinical follow-up visits during which pre-treatment baseline assessments are carried out for each patientNatural 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 Development Program •23 patients have been enrolled in the OPMD Natural History Study to date•Natural History Study patients may be eligible for the pivotal study•All patients are blinded to their Total SSQ Scores and VFSS (TPR and Sequential Swallowing) assessment results, and the Central Reader for the VFSS assessments is blinded to the SSQ Scores for all patients•The BB-301 Phase 1b/2a Open-Label Dose Escalation Study can enroll up to 30 patients across 3 dosesoAll 6 Cohort 1 patients have been treated with BB-301oThe first Cohort 2 patient has been successfully treated with BB-301oPrimary endpoint is safety, secondary endpoints include a range of efficacy assessments Dosing (1 Day)OPMD Natural History Study (26 weeks) January 2026Benitec Corporate Overview | Non-Confidential14
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•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 creation of a multi-component responder analysis which incorporates multiple discrete assessments that holistically assess disease progression and treatment benefit of BB-301Patient-Reported Oral-Pharyngeal Dysphagia via use of a clinically validated 17-question patient reported outcome instrument, Sydney Swallow Questionnaire (SSQ) BB-301: Serial Characterization of Dysphagia Severity Informs Efficacy AssessmentEfficacy assessments were derived from literature-based methods used to assess OPMD dysphagic symptom burden and include patient-reported outcomes, objective anatomic assessments, and functional parameters Videofluoroscopic Swallowing Studies (VFSS) of liquids and solids for anatomic and functional assessments of:•Pharyngeal constrictor muscle function via assessment of Pharyngeal Area at Maximum Pharyngeal Constriction (PhAMPC)•Swallowing Efficiency via assessment of post-swallow accumulation of food/liquid material•Total Pharyngeal Residue (TPR) •Normalized Residue Ratio Scale, Valleculae (NRRSv)•Swallowing Effectiveness via assessment of frequency of pathologic sequential swallows (SEQ) Functional swallowing capacity as measured by the Cold Water Timed Drinking Test (CWDT) January 2026Benitec Corporate Overview | Non-Confidential15
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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 date for Cohort 1 patients:•12-months of post-BB-301-treatment follow-up for Patient 1 and Patient 2•9-months of post-BB-301-treatment follow-up for Patient 3•6-months of post-BB-301-treatment follow-up for Patient 4 and Patient 5 •3-months of post-BB-301-treatment follow-up for Patient 6•Swallowing tasks comprised liquids (thin, moderately thick and extremely thick) and solid food SSQPhAMPCTPRNRRSVLiquidsAll ConsistenciesLiquidsAll ConsistenciesLiquidsAll ConsistenciesPost-treatment Improvement328-point decline40% decline42%decline45% decline49% decline69% decline63% decline The following assessments met the statistical requirement for response: January 2026Benitec Corporate Overview | Non-Confidential16
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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 R² = 0.2 0100200300400500600700800 Vis it 1Vis it 2Vis it 3Vis it 4Ph1-Screening SSQ Total Score Pre-Treatment SSQ Total Score R² = 0.7 0100200300400500600700800 Month 3Month 6Month 9Month 12 SSQ Total Score Post-Treatment SSQ Total Score Worsening Improving Total SSQ Score Improves Over Time Following BB-301 Administration and the Statistical Requirement for Improvement Has Been AchievedJanuary 2026Benitec Corporate Overview | Non-Confidential17 SSQ Cut-Off Score forNormal Swallowing1,2 SSQ Cut-Off Score forNormal Swallowing1,2 1. Bua, B.A. and Bülow, M., BMC Research Notes (2014) 7:742, 2. Audag N., et al., Dysphagia (2019) 34:556-566
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R² = 0.9 051015202530 NH-ScreeningNH-Day 1NH-Month 3NH-Month 6Ph1-Screening PhAMPC (%) Pre-Treatment PhAMPC, Liquids R² = 0.7 051015202530 Month 3Month 6Month 9Month 12 PhAMPC (%) Post-Treatment PhAMPC, Liquids R² = 0.9 051015202530 NH-ScreeningNH-Day 1NH-Month 3NH-Month 6Ph1-Screening PhAMPC (%) Pre-Treatment PhAMPC, All Consistencies R² = 0.7 051015202530 Month 3Month 6Month 9Month 12 PhAMPC (%) Post-Treatment PhAMPC, All Consistencies Worsening ImprovingImproving 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 BB-301 Administration and the Statistical Requirement for Improvement Has Been AchievedJanuary 2026Benitec Corporate Overview | Non-Confidential18
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R² = 0.8 048121620 NH-ScreeningNH-Day 1NH-Month 3NH-Month 6Ph1-Screening TPR (%) Pre-Treatment TPR, Liquids R² = 1.0 048121620 Month 3Month 6Month 9Month 12 TPR (%) Post-Treatment TPR, Liquids R² = 0.7 048121620 NH-ScreeningNH-Day 1NH-Month 3NH-Month 6Ph1-Screening TPR (%) Pre-Treatment TPR, All Consistencies R² = 0.9 048121620 Month 3Month 6Month 9Month 12 TPR (%) Post-Treatment TPR, All Consistencies BB-301: Cohort 1 Demonstrated a Highly Clinically Meaningful Improvement in Total Pharyngeal Residue (TPR)Worsening ImprovingImproving 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 treatment 49% decline in TPR post-BB-301 treatment January 2026Benitec Corporate Overview | Non-Confidential19
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R² = 0.6 0.00.10.20.30.40.5 NH-ScreeningNH-Day 1NH-Month 3NH-Month 6Ph1-Screening NRRSv Pre-Treatment NRRSv Liquids R² = 0.9 0.00.10.20.30.40.5 Month 3Month 6Month 9Month 12 NRRSv Post-Treatment NRRSv Liquids R² = 0.5 0.00.10.20.30.40.5 NH-ScreeningNH-Day 1NH-Month 3NH-Month 6Ph1-Screening NRRSv Pre-Treatment NRRSv All Consistencies R² = 0.9 0.00.10.20.30.40.5 Month 3Month 6Month 9Month 12 NRRSv Post-Treatment NRRSv All Consistencies 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 Requirement for Improvement Has Been Achieved Worsening ImprovingImproving Worsening January 2026Benitec Corporate Overview | Non-Confidential20
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CWDT Improves Over Time Following BB-301 Administration, However, at the Time of this Interim Analysis, Statistical Criteria for Improvement Have Not Yet Been Met •Pre-Treatment = 18.1 seconds•Post-Treatment = 11.0 seconds•Post-treatment scores were reduced as compared to the pre-treatment period R² = 0.4 0 5 10 15 20 25 NH-ScreeningNH-Day 1NH-Month 3NH-Month 6Ph1-Screening Time (sec) Pre-Treatment CWDT R² = 0.6 0 5 10 15 20 Month 3Month 6Month 9Month 12 Time (sec) Post-Treatment CWDT Worsening Improving BB-301: Cohort 1 Demonstrated a Clinically Meaningful Improvement in Cold Water Timed Drinking Test (CWDT) January 2026Benitec Corporate Overview | Non-Confidential21
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BEFORE TREATMENT•OPMD patients showed progressive worsening of swallowing function and symptom burden•Key measures (pharyngeal residue post-swallow, pharyngeal constrictor muscle function, patient-reported outcome scores, timed drinking test) all indicated significant disease impact AFTER BB-301 TREATMENT•Each patient serves as their own control, enabling clear measurement of change (FDA highlighted this approach in the Sept 2025 Draft Guidance for Industry “Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations”)•Post-treatment, pharyngeal residue post-swallow, pharyngeal constrictor muscle function, and patient-reported outcome scores showed significant improvement•Timed drinking test results were reduced compared to those pre-treatment, however statistical criteria for improvement were not yet met at this interim timepoint BB-301 Demonstrated Clinically Meaningful Improvements In Cohort 1 January 2026Benitec Corporate Overview | Non-Confidential22 Natural history trends of Cohort 1 patients inform potential pivotal study design and the development of a proprietary responder analysisKEY POINT
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BB-301 Responder Analyses: A Framework for Evaluation of Efficacy January 2026Benitec Corporate Overview | Non-Confidential23 WHAT IS IT?HOW DOES IT WORK?WHY DOES IT MATTER?A simple, multi-part scoring system that allows for holistic assessment of disease progression and treatment benefit of BB-301 Each patient is evaluated across a combination of efficacy assessments which were derived from literature-based methods used to assess OPMD dysphagic symptom burden and include: 1.Patient-reported outcome measures of swallowing difficulty (SSQ)2.Pharyngeal constrictor muscle function (PhAMPC)3.Swallowing efficiency (NRRSv/TPR)4.Frequency of pathologic sequential swallows (SEQ)5.Time taken to drink cold water (CWDT)If a patient achieves the statistical criteria* for improvement in at least 2 out of 5 measures, they are considered a “responder.” 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 incorporation of multiple discrete assessments that, in total, assess disease progression and treatment benefit of BB-301.The straightforward scoring system also facilitates clear communication of the magnitude of treatment benefit to patients treated with BB-301. Interim clinical results using this analysis were submitted to the FDA over the course of an interactive process that supported the grant of Fast Track Designation for BB-301KEY POINT *Formal statistical criteria were submitted to FDA over the course of the interactive process that supported the grant of Fast Track Designation for BB-301
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Scoring Clinical Success: BB-301’s Responder Framework 1.Patient-Reported Oral-Pharyngeal Dysphagia (SSQ)Criteria: SSQ score must not worsen after treatment2.Pharyngeal Constrictor Muscle Function (PhAMPC)Criteria: Statistical criteria* met in at least 1 of 4 swallowing tasks3.Frequency of Pathologic Sequential Swallows (SEQ)Criteria: At least a 30% decrease in sequential swallows post-treatment4.Swallowing Efficiency (NRRSv or TPR)Criteria: Statistical criteria* met in at least 1 of 8 swallowing tasks5.Functional Swallowing Capacity (CWDT)Criteria: Statistical criteria* met for the cold-water timed drinking test January 2026Benitec Corporate Overview | Non-Confidential24 TOTAL RESPONDER SCORINGMAXIMUM SCORE5 “Yes” values (one per measure)RESPONDER STATUSAchieving ≥2 “Yes” values (≥40%) qualifies a patient as a responder *Formal statistical criteria were submitted to FDA over the course of the interactive process that supported the grant of Fast Track Designation for BB-301
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Patient Follow-Up at the Time of Fast Track SubmissionResponse Criteria AchievedTotal Responder ScoreUpdated Follow-Up Post Fast Track Submission*Updated Response Criteria Achieved*Updated Total Responder Score*Responder112- monthsSSQ, PhAMPC, Pharyngeal residue, CWDT 4 212- monthsSSQ, PhAMPC, SEQ, CWDT 4 39- monthsSSQ, SEQ, Pharyngeal residue312- monthsSSQ, PhAMPC, SEQ, Pharyngeal residue4 46- monthsSSQ, Pharyngeal residue29- monthsSSQ, PhAMPC, Pharyngeal residue3 56- monthsSSQ, Pharyngeal residue2 63-monthsSSQ, Pharyngeal residue2 Total Responder Score Increases Over Time *Data pending source data verification BB-301: 100% of Patients in Cohort 1 (n=6) Were Responders January 2026Benitec Corporate Overview | Non-Confidential25
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All 6 patients with distinct causes of their respective total dysphagic symptom burdens were safely treated with BB-301 Fast Track Designation was granted by the FDAFDA 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 details of the planned OPMD pivotal trial All 6 patients experienced significant, clinically meaningful improvements in swallowing function and quality of life COHORT 1REGULATORY MILESTONES Patients experienced significant reductions in their total dysphagic symptom burdens as assessed by the proprietary OPMD responder analysis There have been no deaths and no treatment-related Severe Adverse Events for any patient enrolled in Cohort 1 to date 100% of patients in Cohort 1 (including those who have completed the statistical follow-up period as well as those with only interim clinical results) were determined to be responders to BB-301 treatment BB-301: Transformative Potential for Patients with Oculopharyngeal Muscular Dystrophy (OPMD) COHORT 2 January 2026Benitec Corporate Overview | Non-Confidential26
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•There are currently no treatment options approved for OPMD•OPMD is diagnosed in ~35 countries•~15,000 patients total across U.S., Canada, Europe and Israel•Up to 90% of OPMD patients may be eligible for treatment with BB-301 •Approximately 10% of patients expected to have pre-existing anti-AAV9 antibody levels that are sufficiently elevated to restrict use of BB-301 If approved, BB-301’s OPMD rare disease market is ~15K addressable patients across North America, Europe and Israel, translating into a high-value, durable rare disease opportunity. Addressable OPMD Market January 2026Benitec Corporate Overview | Non-Confidential27
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SAFETYNO TREATMENT-RELATED SEVERE ADVERSE EVENTSREGULATORYFAST TRACK DESIGNATION (FDA)ORPHAN DRUG DESIGNATION (FDA & EMA) Positive Interim Clinical Results and Regulatory Milestones Position BB-301 as the First Potential Disease-Modifying Genetic Medicine for OPMD-Related Dysphagia January 2026Benitec Corporate Overview | Non-Confidential28 PHASE 1b/2a CLINICAL EFFICACY100% RESPONDER RATE IN COHORT 1MEANINGFUL IMPROVEMENTS POST-BB-301 ADMINISTRATION ACROSS KEY ENDPOINTS•Improved patient reported outcome measures over time•Improved throat closure over time•Reduced pharyngeal residue over time•Improved functional swallowing capacity over timeFIRST PATIENT IN COHORT 2 SUCCESSFULLY TREATED If approved, BB-301’s OPMD rare disease market is ~15K addressable patients across North America, Europe and Israel, translating into a high-value, durable rare disease opportunity.15K
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C Benitec: Driving the Future of Genetic Medicine
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BOARD OF DIRECTORS Distinguished Leadership Team January 2026Benitec Corporate Overview | Non-Confidential30 MANAGEMENT Megan BostonChief Financial Officer & Director Jerel Banks, M.D., Ph.D.Chief Executive Officer & Chairman of the Board Sophie MukadamChief Operating Officer Peter FrancisDirector Dr. Sharon MatesDirector Kishen MehtaDirector Edward SmithDirector Jerel Banks, M.D., Ph.D.Chief Executive Officer & Chairman of the Board Megan BostonChief Financial Officer & Director J. Kevin BuchiDirector
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* December 31, 2025 Strong Financial Position and Clear Milestones Driving Benitec’s Path To Pivotal Data and Commercialization January 2026Benitec Corporate Overview | Non-Confidential31 Cohort 2 DosingInitiated Q4 2025 Regulatory ProgressFast Track (FDA) and Orphan Drug Designations (FDA/EMA) ManufacturingGMP-compliant, commercial-scale batches produced BB-301 Key Milestones Financial SnapshotCash and Cash Equivalents:$188.6 M* Pivotal Study PlanningOn track for 2026 FDA meeting Outstanding Shares:34.25 M*
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SILENCE + REPLACEOne Platform. One Dose. Potentially Transformative Outcomes. NASDAQBNTC
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www.benitec.com©2026 Benitec Biopharma. All rights reserved. THANK YOU! NASDAQBNTC
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Appendix
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Benitec continues to plan for a formal meeting with FDA in 2026 to confirm all key elements of the anticipated BB-301 Pivotal Study design 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 •BB-301 is the only clinical-stage therapeutic agent in development designed to treat dysphagia (loss of the ability to swallow) in patients with OPMD•15,000 patients in the U.S., Europe, Canada, and Israel •BB-301 is delivered via direct intramuscular (IM) injection to pharyngeal constrictor muscles resulting in minimal systemic exposure; 6 patients have been safely treated with the low dose of BB-301 and there have been no treatment related severe adverse events•Following the administration of BB-301, Cohort 1 patients experienced significant reductions in dysphagic symptom burden, reductions in post-swallow residue accumulation, reductions in the time required to consume fixed volumes of liquid, and improvements in pharyngeal closure during swallowing•All 6 patients enrolled into Cohort 1 met the formal statistical criteria for Response to BB-301, representing a 100% response rate •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•Without intervention, the functional elements of the swallowing process worsened over time for OPMD patients prior to their enrollment into Cohort 1 of the BB-301 Phase 1b/2a Clinical Treatment Study•The Natural History Study results facilitated the development of a putative proprietary Responder Analysis and potential pivotal study design, both of which were presented to the FDA in support of Fast Track Designation for BB-301 BB-301: Potential One-Time Treatment Designed to Address the Root Cause of Oculopharyngeal Muscular Dystrophy (OPMD)-Related Dysphagia January 2026Benitec Corporate Overview | Non-Confidential35
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•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 patients are blinded to their Total SSQ Scores and VFSS Assessment Results, and the Central Reader for the VFSS assessments is blinded to the SSQ Scores for all Study patients •Patient-Reported Outcome Assessments: The SSQ is employed to serially evaluate the severity of dysphagia as reported by the patient at each clinical site visit•X-ray Based Swallowing Assessments: VFSS are employed to serially analyze Swallowing Efficiency and Swallowing Effectiveness at each clinical site visit, with imaging results reviewed and rated via a standardized process Robust, Blinded Methods are Utilized to Evaluate the Dysphagic Symptom Burden of each Study patient January 2026Benitec Corporate Overview | Non-Confidential36
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Szczesniak, M., et al, Dysphagia, 2014 Patient-Reported Oral-Pharyngeal Dysphagia: As Assessed by the SSQPatient-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 100-mm long visual analogue scale for all but 1 question•Possible scores range from 0 to 1700, with higher scores indicating greater swallowing difficulty•The SSQ has demonstrated strong content, construct, discriminant, and predictive validity and test-retest reliability in a range of patient populations •The SSQ evaluates a range of domains, including: difficulty swallowing specific food types/consistencies, frequency of choking during ingestion of specific food types/consistencies, and the requirement for multiple swallows during ingestion of food and liquid SSQ Example Questions January 2026Benitec Corporate Overview | Non-Confidential37
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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) occurs on the videofluoroscopy frame with the smallest amount of unobliterated air space and barium-containing bolus visible in the pharynx •Normal PhAMPC values span the range of 0% to 2.2% for the characteristic food and liquids that are evaluated in the swallowing tasks in the ongoing clinical studies C2-C4 length serves as an anatomical scalar January 2026Benitec Corporate Overview | Non-Confidential38
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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 bolus•Normal Total Pharyngeal Residue values should be close to zero C2-C4 length squared serves as an anatomical scalar January 2026Benitec Corporate Overview | Non-Confidential39
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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, 2012; 4. Kroon, R.H.M.J.M. et. al., Neurology, 2021 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-swallow aspiration, with clinically significant residue determined by NRRSv values ≥ 0.063 •Residue accumulation in the valleculae (NRRSv ≥ 0.09) has also shown to be correlated with an increased risk of penetration-aspiration•In a 2021 Dutch OPMD Natural History Study of 43-patients, a statistically significant decline in swallowing efficiency was captured via the use of longitudinal NRRSv assessments (2 assessments, occurring 20-months apart). The amount of abnormal pharyngeal residue in the valleculae increased significantly (mean NRRSv ratio 0.24 vs 0.13, p = 0.007) over this time period.4 •Valleculae are the bilateral spaces between the base of the tongue and the epiglottisC2-C4 length squared (dashed red line) serves as an anatomical scalar and spatial housing areas are shown in white2Measurement of residue (yellow) occurs on the first frame showing pyriform sinuses at lowest position2 January 2026Benitec Corporate Overview | Non-Confidential40
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1. Ambrocio, K. R., et al., Dysphagia (2023) 38:1497–1510; 2Steele, 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) January 2026Benitec Corporate Overview | Non-Confidential41 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 patency between swallows1•Under normal physiologic conditions, sequential swallowing occurs during continuous drinking of large volumes (90 mL or greater) of thin liquids via straw, cup, or bottle (for reference, one can of a soft drink has a volume of approximately 355 mL of thin liquid) For the BB-301 Phase 1b/2a Study, the return of the HLC to its resting position (i.e., achievement of pharyngeal muscle relaxation) between swallows is characterized on VFSS via the identification of the “Swallow Rest Frame”•Swallow Rest is the terminal event of each discrete swallow, and the Swallow Rest Frame is identified as the first VFSS frame showing the pyriform sinuses at their lowest position, relative to the spine, prior to any hyoid burst or laryngeal elevation for a subsequent subswallow2 In dysphagic disorders (e.g., OPMD), during the consumption of small volumes of liquids (e.g., less than 15 mL, as employed in the BB-301 Phase 1b/2a Study), pharyngeal muscle relaxation may not be achieved consistently between swallows (i.e., the HLC does not return to rest and the pharynx lacks complete patency); instead, a patient may experience pathologic sequential swallows when consuming small volumes of liquidsPathologic sequential swallows occur during the consumption of small volumes of liquid and are detected during VFSS as a series of involuntary contractions of the pharyngeal muscles without restoration of the resting pharyngeal diameter between contractions
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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, 2009; 3. Côté, C. et. al., Dysphagia, 2024; 4. Périé, S. et. al., Molecular Therapy, 2013 •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 presence of at least one ptotic eyelid with a fissure of less than 8 mm, can be used to make a clinical diagnosis of OPMD2•Failure to drink 80 mL of water in less than 8 seconds suggests the presence of dysphagia in OPMD individuals (sensitivity = 87% and specificity = 77%)3 •The timed swallowing of 80 mL of cold water represents a global functional evaluation of swallowing and has been used as one of the endpoints to assess the outcome of an autologous myoblast transplant Phase 1/2a clinical study in OPMD patients4 January 2026Benitec Corporate Overview | Non-Confidential42
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AssessmentRequirement for a ResponderResponder ScoringPatient-Reported Oral-Pharyngeal Dysphagia determined by SSQ Total ScoreSSQ 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 for this component if requirement is met Pharyngeal Constrictor Muscle Function as measured by PhAMPCA patient is considered a responder if statistical criteria* are achieved for at least 1 out of the 4 consistencies evaluated Maximum score of 1 for this component if PhAMPC for at least 1 consistency assessment meets responder criteriaFrequency of Pathologic Sequential Swallows (SEQ): Number of sequential swallows (detected during VFSS as a series of involuntary contractions of the pharyngeal muscles without restoration of the resting pharyngeal diameter between contractions) A patient is considered a responder if they experience a 30% decrease in the total frequency of sequential swallows post-treatment compared to pre-treatmentMaximum score of 1 for this component if the total frequency of sequential swallows decreases by ≥ 30% post-treatment with BB-301 Swallowing Efficiency as measured by either NRRSv or TPR A patient is considered a responder if statistical criteria* are achieved for at least 1 out of the 8 consistency assessments evaluated Maximum score of 1 for this component if at least 1 consistency assessment meets responder criteriaFunctional Swallowing Capacity as measured by the CWDT A patient is considered a responder if statistical criteria* are achieved Maximum score of 1 for this component if requirement is met A maximum total score of 5 “Yes” values can be achieved if all criteria above are met. If a score of ≥ 2 “Yes” values (≥ 40%) is achieved, then the patient is a Responder.*Formal statistical criteria were submitted to FDA over the course of the interactive process that supported the grant of Fast Track Designation for BB-301 Proprietary OPMD Responder Analysis Description January 2026Benitec Corporate Overview | Non-Confidential43