Benitec Biopharma Inc. · NASDAQ:BNTC
Benitec Biopharma — Investor Presentation (September 2025)
34 slides from Benitec Biopharma's investor presentation, September 2025
Slide 1: Corporate Presentation September 2025
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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: OPMD is a Debilitating Progressive Disease With No Approved Therapies
OPMD is a Debilitating Progressive Disease With No Approved Therapies • Oculopharyn geal Mus c ular Dys trophy (OP MD) is a rare (15,000 patients in North America, Europe, and Israel), autosomal-dominant degenerative muscle disorder, caused by a mutation in the poly(A)-binding protein nuclear 1 (PA
Slide 4: ddRNAi 'Silence and Replace' Platform Enables Both Permanent Silencing and Replacement
ddR NAi “S ilence and R eplace” P latform E nables B oth P ermanent S ilencing AND R eplacement of Mutated G enes in the T arget T is sue LIMITATIONS OF CURRENT siRNA TECHNOLOGIES: • Requires repeated administration • Enables only transient silencing of mutated gene • Silencing capacity restricted t
Slide 5: OPMD: Overview of the Clinical Presentation
OPMD: Overview of the Clinical Presentation • OPMD typically presents with: • Ptosis (eyelid drooping) which can be asymmetric at onset • Difficulty swallowing (dysphagia) and choking during meals • A retrospective chart review was conducted at the Saguenay Neuromuscular Clinic (Quebec, Canada) to s
Slide 6: Current Clinical Management Strategies for Dysphagia in OPMD Do Not Prevent Disease Progression
Current Clinical Management Strategies for Dysphagia in OPMD Patients Do Not Prevent Disease Progression • Nutritional recommendations including dietary texture/consistency modifications • Surgical interventions for moderate to severe dysphagia have temporary effects and may require repeated adminis
Slide 7: Swallowing Overview and the Rationale for BB-301 in OPMD
S wallowing O verview and the R ationale for B B -301 in O P MD OPMD Drives Disordered Swallowing Anatomical Structures of the Pharynx and BB-301 Injection Sites • In OP MD, th e ph aryn geal con s trictor m u s c les are weakened and atrophic and u n able to s upport th e propu ls ion of food or li
Slide 8: BB-301 Clinical Development Program
BB-301 Clinical Development Program OPMD Natural BB-301 Phase 1b/2a Open-Label Dose History Study Dosing Escalation Study (NCT06185673) (26wk) (1 Day) (52wk)* 23 Subjects Natural History Study Study can enroll up to 30 Subjects enrolled as of Subjects enroll into the Six Subjectshave been safely tre
Slide 9: Key Assessments: Subject-Reported and Videofluoroscopic Endpoints
Key Assessments: Subject-Reported and Videofluoroscopic Endpoints The key secondary endpoints of the OPMD Natural History Study and the BB-301 Phase 1b/ 2a Clinical Study (NCT06185673) facilitate serial characterization of: • Subject-Reported Oral-Pharyngeal Dysphagia (via use of the 17-question pat
Slide 10: Clinical Utility, Sensitivity, and Specificity of Key Assessment Methods
Clinical Utility, Sensitivity, and Specificity of Key Assessment Methods • In th e B B -301 P h as e 1b/2a C lin ic al T reatm en t S tu dy, R adiologis ts an d S peec h L an gu age P ath ologis ts e m ploy s erial VF S S to objec tively c h arac terize th e n atu re an d s everity of an atom ic al
Slide 11: Distinct Drivers of Dysphagia: Inefficient Swallowing and Ineffective Swallowing
Distinct Drivers of Dysphagia Observed in the OPMD Study Population: Inefficient Swallowing and Ineffective Swallowing • OPMD Subjects enrolled into the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Treatment Study can be impacted by the post swallow accumulation of food and liquid
Slide 12: Post-Swallow Pharyngeal Residue and Dysphagic Symptom Burden
Post-Swallow Pharyngeal Residue and Dysphagic Symptom Burden • It is not known which of the abnormal consistency post-swallow residue value(s) contribute most to the dysphagic symptom burden in the pre-treatment period • Additionally, it is not clear if there exists a specific magnitude of post-swal
Slide 13: Interim Cohort 1 Phase 1b Clinical Data
Interim Cohort 1 Phase 1b Clinical Data Demonstrate Clinically Meaningful Improvements in Swallowing Function for the Two Discrete Causes of Dysphagia 13
Slide 14: 12-Months Post BB-301: VFSS TPR Results for Subject 1
12-Months Post BB-301 Treatment: VFSS TPR Assessments Demonstrate Significant, Clinically Meaningful Reductions of Post Swallow Residue Across all Consistencies for Subject 1 Total Pharyngeal Residue 30 29% improvement vs. Pre-dose Average TPR 25 18% improvement vs. Pre-dose Average TPR 15.1 Total P
Slide 15: 12-Months Post BB-301: Total SSQ Scores for Subject 1
12-Months Post BB-301 Treatment: Total SSQ Scores Demonstrate Significant, Clinically Meaningful Reductions in the Total Dysphagic Symptom Burden Experienced by Subject 1 • The 12-Month Average Post-Dose SSQ Score Showed a 41% Improvement vs. the Average Pre-Dose SSQ Score Sydney Swallow Questionnai
Slide 16: 12-Months Post BB-301: VFSS TPR Results for Subject 2
12-Months Post BB-301 Treatment: VFSS TPR Assessments Demonstrate Stabilization of Post Swallow Residue for Thin Liquid and Reduction of Post Swallow Residue for Solid Food for Subject 2 Total Pharyngeal Residue 30 25 Total Pharyngeal Residue (%) 20 6.5-point increase vs. Pre-dose Average TPR 11% im
Slide 17: 12-Months Post BB-301: Pathologic Sequential Swallows for Subject 2
12-Months Post BB-301 Treatment: VFSS Assessments Demonstrate Significant, Clinically Meaningful Reductions in the Frequency of Pathologic Sequential Swallows for Subject 2 For Subject 2, pathologic sequential swallows for Thin Liquid were observed: • during 12 of the 15 swallowing tasks (frequency
Slide 18: 12-Months Post BB-301: Total SSQ Scores for Subject 2
12-Months Post BB-301 Treatment: Total SSQ Scores Demonstrate Significant, Clinically Meaningful Reductions in the Total Dysphagic Symptom Burden Experienced by Subject 2, with Subject 2 Achieving Scores Representing Clinically Normal Swallowing • The 12-Month Average Post-Dose SSQ Score Showed a 91
Slide 19: 3-Months Post BB-301: VFSS TPR Results for Subject 3
3-Months Post BB-301 Treatment: VFSS TPR Assessments Demonstrate Significant, Clinically Meaningful Reductions of Post Swallow Residue for Thick Liquid and Stabilization of Post Swallow Residue for Solid Food for Subject 3 Total Pharyngeal Residue 30 22.6 20% improvement vs. 25 Pre-dose Average TPR
Slide 20: 3-Months Post BB-301: Pathologic Sequential Swallows for Subject 3
3-Months Post BB-301 Treatment: VFSS Assessments Demonstrate Significant, Clinically Meaningful Reductions in the Frequency of Pathologic Sequential Swallows for Subject 3 For Subject 3, pathologic sequential swallows for Thin and Thick liquids were observed: VFSS Thin Liquid Tasks and Thick Liquid
Slide 21: 3-Months Post BB-301: Total SSQ Scores for Subject 3
3-Months Post BB-301 Treatment: Total SSQ Scores Demonstrate Significant, Clinically Meaningful Reductions in the Total Dysphagic Symptom Burden Experienced by Subject 3, with Subject 3 Achieving Scores Representing Clinically Normal Swallowing • The 3-Month Post-Dose SSQ Score Showed a 68% Improvem
Slide 22: Summary and Conclusions
Summary and Conclusions • Three Subjects with distinct causes of their respective total dysphagic symptom burdens were safely treated with BB-301 (1.2e13 vg/Subject) and experienced significant, clinically meaningful improvements in swallowing function • There were no Treatment-Related Severe Advers
Slide 23: Financial Summary
Financial Summary Nasdaq Listed: BNTC Shares Outstanding Cash & Cash Equivalents • Exclusively Nasdaq listed; re- • 26,250,469 as of 8/31/25 • $96.25mm as of 8/31/25 domiciled in April 2020 23
Slide 24: Appendix
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Slide 25: OPMD: A Debilitating, Progressive Disease With No Approved Therapies
OPMD: A Debilitating, Progressive Disease With No Approved Therapies • Oculopharyngeal muscular dystrophy (OPMD) is an autosomal dominant, chronic, myopathic disorder with a typical age of onset in the 40s-50s • OPMD results from a trinucleotide repeat expansion within exon 1 of the polyadenylate bi
Slide 26: Models for OPMD Pathogenesis
Models for OPMD Pathogenesis • 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 • Currently two general models are used to explain how alanine-expanded PABPN1 confers muscle pathology in autosomal dominant
Slide 27: Subject-Reported Oral-Pharyngeal Dysphagia: Sydney Swallow Questionnaire Scoring
S ubject-R eported O ral-P haryngeal Dysphagia: S ydney S wallow Questionnaire (S S Q) S coring SSQ Example Questions Subject-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 quest
Slide 28: Swallowing Inefficiency: Post Swallow Total Pharyngeal Residue
Swallowing Inefficiency: As Measured by Post Swallow Total Pharyngeal Residue • C2-C4 length serves as an anatomical scalar • Total Pharyngeal Residue measurement comprises the amount of food or liquid material remaining in the pharynx after the first swallow of the bolus • Measurement (yellow, gree
Slide 29: Swallowing Ineffectiveness: Frequency of Pathologic Sequential Swallows
Swallowing Ineffectiveness: As Measured by the Frequency of Pathologic Sequential Swallows • 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
Slide 30: IND-Enabling Studies for BB-301
IND-Enabling Studies for BB-301 Pilot Dosing Study in Beagle Dogs Toxicology Study in Beagle Dogs • 8-week study to confirm the transduction 12-week GLP Toxicology and Biodistribution efficiency of BB-301 following direct intramuscular study in Beagle dogs injection into the pharyngeal muscles via t
Slide 31: Dose-Dependent BB-301 Tissue Transduction in Beagle Dogs
Dose-Dependent BB-301 Tissue Transduction Observed 8 Weeks Following Direct Intramuscular Injection in Beagle Dogs Copies of BB-301 (average copies per cell) BB-301 Dose Hypopharyngeal Thyropharyngeal 5.66 (vg/mL) Muscle 5.12 Muscle 3.00 x 1013 vg/ml 5.12 5.66 High Volume 3.00 x 1013 vg/ml 3.15 2.70
Slide 32: Dose-Dependent Expression of coPABPN1 in Beagle Dogs
Dose-Dependent Expression of coPABPN1 Observed 8 Weeks Following Direct Intramuscular Injection of BB-301 in Beagle Dogs Copies of coPABPN1 (average copies per cell) BB-301 Dose Hypopharyngeal Thyropharyngeal 5.66 (vg/mL) Muscle 5.12 Muscle 3.00 x 1013 vg/ml 61.69 77.26 High Volume 3.00 x 1013 vg/ml
Slide 33: Dose-Dependent Expression of siRNA13, siRNA17 in Beagle Dogs
Dose-Dependent Expression of siRNA13, siRNA17 Observed 8 Weeks Following Direct Intramuscular Injection of BB-301 in Beagle Dogs siRNA13 siRNA17 Hypopharyngeal Thyropharyngeal Hypopharyngeal Thyropharyngeal Muscle Muscle Muscle Muscle BB-301 Dose average copies per cell average copies per cell avera
Slide 34: Dose-Dependent Inhibition of PABPN1 in Beagle Dogs
Dose-Dependent Inhibition of PABPN1 Observed 8 Weeks Following Direct Intramuscular Injection of BB-301 in Beagle Dogs Average Reported % Inhibition of wtPABPN1 BB-301 Dose Hypopharyngeal Thyropharyngeal 5.66 (vg/mL) Muscle Muscle 5.12 3.00 x 1013 vg/ml 83% 82% High Volume 3.00 x 1013 vg/ml 74% 64%
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