Benitec Biopharma Inc. · NASDAQ:BNTC

Benitec Biopharma — Investor Day (April 2024)

71 slides from Benitec Biopharma's investor day presentation, Apr 18, 2024

Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements within the meaning of section 27A of the US Securities Act of 1933 and section 21E of the US Securities Exchange Act of 1934. Benitec has tried to identify such forward-looking statements by use of such words as expects, int
Slide 3: Research and Development Day Agenda
Research and Development 01 Introduction to S peakers Day Agenda Corporate Overview 02 03 OP MD Clinical and P athophys iological Overview 04 BB -301: Mechanis m of Action and Clinical Developm ent P rogram VF S S and S ubject-R eported Outcom e Meas ures 05 06 P relim inary B B -301 P has e 1b Clin
Slide 4: Professional Biography: Bernard Brais, MDCM, PhD
Professional Biography: Bernard Brais, MDCM, PhD, Professor, Department of Neurology and Neurosurgery at Montreal Neurological Institute (MNI), McGill University Health Centre (MGH, MNH) is Director of the Rare Neurological Diseases Group. He completed his MDCM, neurology residency and PhD at McGill
Slide 5: Professional Biography: Emily Plowman, PhD, CCC-SLP, FASHA
Professional Biography: Emily Plowman, PhD, CCC-SLP, FASHA, Professor, Department of Otolaryngology – Head and Neck Surgery, The Ohio State University College of Medicine is Director of the Aerodigestive Research Core across its two sites at the Ohio State University and University of Florida and Di
Slide 6: Benitec Corporate Overview
Benitec Corporate Overview Jerel A. Banks, MD, PhD 6 ©2024 B enitec B iopharm a | All R ights R es erved
Slide 7: Corporate Highlights
Corporate Highlights Novel Technology Platform • Benitec’s DNA-directed RNA interference (ddRNAi) platform combines gene therapy with RNA interference (RNAi) to simultaneously silence & replace disease-causing genes permanently, following a single administration • Platform has application in disease
Slide 8: Experienced and Efficient Management
E xperienced and E fficient Management Jerel A. Banks, MD, PhD CEO & Executive Chairman Healthcare investment professional with over 15 years of experience Former Vice president & co-portfolio manager at Franklin Templeton Investments M.D., Ph.D. Brown University; A.B. Princeton University Megan Bos
Slide 9: Oculopharyngeal Muscular Dystrophy: Clinical and Pathophysiological Overview
Oculopharyngeal Muscular Dystrophy: Clinical and Pathophysiological Overview Bernard Brais, MDCM, PhD 9 ©2024 Benitec Biopharma | All Rights Reserved
Slide 10: OPMD: A Chronic, Progressive Disease With No Approved Therapeutics
OPMD: A Chronic, Progressive Disease With No Approved Therapeutics • Oculopharyngeal muscular dystrophy (OPMD) is an autosomal dominant, chronic, myopathic disorder characterized by ptosis (drooping of the upper eyelid) and progressive dysphagia (loss of the ability to swallow) due to impairment of
Slide 11: Models for OPMD Pathogenesis
Models for OPMD Pathogenesis • Currently two general models are used to explain how alanine-expanded PABPN1 confers muscle pathology in autosomal dominant OPMD where patients have one normal and one mutant allele of PABPN1 • One model suggests that nuclear aggregates cause disease (right column, out
Slide 12: Key Epidemiological Estimates for OPMD
K ey E pidemiological E stimates for OP MD: At Least 15k adults in the United States, Canada, Europe and Israel • OPMD has a global distribution and has been diagnosed in at least 33 countries • The estimated prevalence in Western countries is 1:100,000 • Large patient cohorts exist in the United St
Slide 13: Swallowing Overview: Normal vs. Disordered Swallowing
S wallowing Overview Normal vs. Disordered Swallowing Anatomical Structures of the Pharynx • Under normal conditions, a food bolus leaves the oral cavity and is able to traverse the length of the pharynx, en route to the esophagus, via the propulsive activity of the coordinated constriction of the s
Slide 14: OPMD: Onset and Progression
OPMD: Onset and Progression • More frequent choking • Prolonged mealtime • Avoidance of specific foods (e.g., rice, chicken, toast, etc.) • Drooping eyelids that are often asymmetrical at disease onset • Absence of limb weakness at disease onset 14 ©2024 B enitec B iopharm a | All R ights R es erved
Slide 15: OPMD: Clinical Presentation
OPMD: Clinical Presentation • A retrospective chart review was conducted at the Saguenay Neuromuscular Clinic (Quebec, Canada) • All health record of patients with an OPMD diagnosis were screened to identify patients who met inclusion criteria • Patients were excluded if another neurological or musc
Slide 16: OPMD: Clinical Presentation (continued)
OPMD: Clinical Presentation (continued) Median age at symptom onset as reported by the subject: • Dysphagia onset at 54 years of age • Pharyngeal pooling of thickened secretions onset at 66 years of age Brisson, JD, Muscle & Nerve, 2020 16 ©2024 B enitec B iopharm a | All R ights R es erved
Slide 17: OPMD: Clinical Presentation (continued)
OPMD: Clinical Presentation (continued) Subjects may present with: • Ptosis (drooping of the upper eyelid) and dysphagia (difficulty swallowing) together • Ptosis, followed by dysphagia (median of 3.0 years following initial presentation of ptosis) • Dysphagia, followed by ptosis (median of 5.0 year
Slide 18: OPMD: Clinical Complications and Causes of Death
OPMD: Clinical Complications and Causes of Death Approximately one third of subjects had respiratory diagnoses listed as the cause of death: • Impaired swallowing (dysphagia) can lead to aspiration pneumonia • Pneumonia can result from the aspiration of food and/or liquid • Pneumonia can also result
Slide 19: Clinical Management of Dysphagia
Clinical Management of Dysphagia • Nutritional recommendations including adaptations • Surgical Interventions for moderate to severe dysphagia: – Cricopharyngeal muscle paralysis with botulinum toxin injection (temporary effect in some patients, requires repeated administration) – Cricopharyngeal mu
Slide 20: BB-301: Mechanism of Action and Clinical Development Program
BB-301: Mechanism of Action and Clinical Development Program Jerel A. Banks, MD, PhD 20 ©2024 Benitec Biopharma | All Rights Reserved
Slide 22: BB-301 Simultaneously Silences Mutant PABPN1 & Delivers Wildtype PABPN1
BB -301 S imultaneously S ilences Mutant P ABP N1 & Delivers Wildtype P ABP N1 To R estore Normal Myocyte F unction Design of L ead C andidate BB-301 * PABPN1 in OPMD • P AB P N1 is a ubiquitous protein that controls the length of m R NA poly(A) tails , m R NA export from the nucleus & alternative p
Slide 23: ddRNAi Platform Enables Both Permanent Silencing AND Replacement
ddR NAi P latform E nables Both P ermanent S ilencing AND R eplacement of Mutated G enes in the Target Tissue LIMITATIONS OF CURRENT siRNA TECHNOLOGIES: Requires repeated administration Enables only transient silencing of mutated gene Silencing capacity restricted to a single gene ADVANTAGES OF THE
Slide 24: BB-301 Silenced and Replaced PABPN1 in the A17 Mouse Model
BB-301 Silenced and Replaced PABPN1 Over a Broad Range of Doses in the A17 Mouse Model of OPMD “Silence” “Replace” BB-301 Dose (vg) Inhibition of PABPN1 coPABPN1 Expression Varying levels of inhibition of PABPN1 Phase 1b 11 63% 7.50 x 10 86% expres s ion, when coupled with partial Cohort 3 Analog Ph
Slide 25: BB-301 Restored Muscle Strength to Wildtype Levels in A17 Model
BB-301 Restored Muscle Strength to Wildtype Levels in A17 Model At 14-weeks post intramuscular administration of BB-301, statistically significant improvements in muscle strength and complete phenotypic correction were achieved at doses ≥ 5.00 x 1010 vg Muscle Response Muscle Response (10- (150 Hz)
Slide 26: Dose-Dependent Resolution of INIs in A17 Mouse Model
Dose-Dependent Resolution of INIs in Injected Muscles of A17 Mouse Model of OPMD Quantitative Analysis of INIs Immunofluorescence Staining of INIs in Mouse Muscle Tissue in Mouse Muscle Tissue Heavy accumulation of Absence of INIs in Very Few PABPN1 PABPN1 INIs in A17 Normal Mouse Muscle INIs in BB-
Slide 27: The Rationale for BB-301 in OPMD
The Rationale for BB-301 in OPMD • In OPMD, the pharyngeal constrictor muscles are weakened and atrophic and, as a result, are unable to consistently exert the level of force required to support the propulsion of the food and liquid bolus • In the preclinical efficacy studies for BB-301 carried out
Slide 29: BB-301 Clinical Development Program and Key Efficacy Assessments
BB-301 Clinical Development Program and Key Efficacy Assessments E ndpoints Characterization of OPMD Natural BB-301 Phase 1b/2a Open- Dosing OPMD subject disposition History Study label Dose Escalation Study • Primary: (1 Day) at baseline assessing: (26wk) (NCT06185673) S afety and tolerability (52w
Slide 30: Outcome Measures for the OPMD Natural History Study and BB-301 Phase 1b/2a Study
Outcome Measures for the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Study (NCT06185673) Videofluoroscopic Swallowing Study Assessments Clinical Assessments Global Swallowing Pharyngeal Constrictor Swallowing Efficiency Other Assessments Function Muscle Function Total Pharyngeal R
Slide 31: Videofluoroscopic Swallowing Studies and Subject-Reported Outcome Measures
Videofluoroscopic Swallowing Studies and Subject-Reported Outcome Measures: Clinical and Methodological Overview Emily Plowman, PhD, CCC-SLP, FASHA 31 ©2024 Benitec Biopharma | All Rights Reserved
Slide 32: Key Videofluoroscopic and Subject-Reported Endpoints
K ey Videofluoroscopic and S ubject-R eported E ndpoints The primary and secondary outcome measures for the OPMD Natural History Study and the BB-301 Phase 1b/2a Clinical Study (NCT06185673) facilitate serial characterization of: • Pharyngeal Constrictor Muscle Function • Swallowing Efficiency • Sub
Slide 33: Pharyngeal Constrictor Muscle Function: PhAMPC
P haryngeal C onstrictor Muscle F unction: P hAMP C Pharyngeal constrictor muscle function as estimated by PhAMPC: • C2-C4 length act as an anatomical scalar • “Pharyngeal Area” measurement comprises the area of visible airspace or bolus at maximal constriction • Measurement (yellow) occurs on the f
Slide 34: Swallowing Efficiency: Total Pharyngeal Residue
S wallowing E fficiency: Total P haryngeal R esidue Swallowing Efficiency as measured by Total Pharyngeal Residue: • C2-C4 length act as an anatomical scalar • “Total Pharyngeal Residue” measurement comprises the amount of material remaining in the pharynx after the first swallow of the bolus • Meas
Slide 35: Subject-Reported Oropharyngeal Dysphagia: Sydney Swallow Questionnaire
S ubject-R eported Oropharyngeal Dysphagia: S ydney S wallow Questionnaire (SSQ) Subject-reported oropharyngeal dysphagia as assessed by the SSQ: • The SSQ is a self-report inventory assessing subjective symptoms of oropharyngeal dysphagia with strong content, construct, discriminant, and predictive
Slide 36: Clinically Meaningful Improvements
Clinically Meaningful Improvements Clinically meaningful improvement over the course of the BB-301 clinical development program will be defined by: • Improvements in Subject-Reported Outcome assessments (i.e., reductions in the Sydney Swallow Questionnaire [”SSQ”] Scores) post BB-301 dose and Reduct
Slide 37: Preliminary BB-301 Phase 1b Clinical Data Summary for the First Subject (Day 90)
Preliminary BB-301 Phase 1b Clinical Data Summary for the First Subject (Day 90) Jerel A. Banks, MD, PhD 37 ©2024 Benitec Biopharma | All Rights Reserved
Slide 38: Key Questions for Subject of the Phase 1b Study
Key Questions for Subject 1 of the Phase 1b Study Subject 1 experienced disease progression during their enrollment in the Natural History Study In this regard, several critical questions emerged with respect to the potential impact of BB-301: • Would BB-301 slow progression, halt, or improve dyspha
Slide 40: PhAMPC: Solid Food
PhAMPC: Solid Food (Declines Below SD and NH Study Screening Visit at Day 0) BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose VFSS: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose PhAMPC: Improved 8.3 Units, Declined -43.2% Relative
Slide 41: PhAMPC: Thin Liquid
PhAMPC: Thin Liquid, 5 mL (Declines Below SD Post-Dose) BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose VFSS: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose PhAMPC: Improved 11.9 Units, Declined -75.3% Relative to the Average Pre-D
Slide 42: PhAMPC: Moderately Thick Liquid
PhAMPC: Moderately Thick Liquid BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose VFSS: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose PhAMPC: Improved 7.5 Units, Declined -33.6% BB-301 Relative to the Average Pre-Dose PhAMPC: Improv
Slide 43: PhAMPC: Extremely Thick Liquid
PhAMPC: Extremely Thick Liquid BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose VFSS: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose PhAMPC: Improved 5.7 Units, Declined -23.6% BB-301 Relative to the Average Pre-Dose PhAMPC: Improve
Slide 44: Improvement was Observed Across all PhAMPC Assessments
Improvement was Observed Across all P hAMP C Assessments Average Pre-Dose PhAMPC Values vs. Day 90 PhAMPC Values Final Pre-Dose PhAMPC Values vs. Day 90 PhAMPC Values 44 ©2024 Benitec Biopharma | All Rights Reserved
Slide 46: Total Pharyngeal Residue: Solid Food
Total Pharyngeal Residue: Solid Food (Declines Below NH Study Screening Visit at Day 0) BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose VFSS: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose Total PR: Improved 5.0 Units, Declined -28
Slide 47: Total Pharyngeal Residue: Thin Liquid
Total Pharyngeal Residue: Thin Liquid, 5 mL (Declines Below SD and NH Study Screening Visit at Day 0) BB-301 administered on Day 296 (as designated by the yellow bolt) Post-Dose VFSS: Day 287 (Average) to Day 389 P re-Dose VFS S : Day 0 to Day 287 Relative to Final Pre-Dose Total PR: Improved 6.8 Un
Slide 48: Total Pharyngeal Residue: Moderately Thick Liquid
Total Pharyngeal Residue: Moderately Thick Liquid BB-301 administered on Day 296 (as designated by the yellow bolt) P re-Dose VFS S : Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose Total PR: Improved 0.4 Units, Declined -3.5% Relative to the Average Pre-Dose
Slide 49: Total Pharyngeal Residue: Extremely Thick Liquid
Total Pharyngeal Residue: Extremely Thick Liquid (Declines Below SD and NH Study Screening Visit at Day 0) BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose VFSS: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose Total PR: Improved 10.6
Slide 50: Improvement was Observed Across all Total Pharyngeal Residue Assessments
Improvement was Observed Across all Total P haryngeal R esidue Assessments Average Pre-Dose Total Pharyngeal Residue Values vs. Day 90 Total Pharyngeal Residue Values Final Pre-Dose Total Pharyngeal Residue Values vs. Day 90 Total Pharyngeal Residue Values 50 ©2024 Benitec Biopharma | All Rights Res
Slide 52: Timed Drinking Test: CWDT
Timed Drinking Test: CWDT BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose Results: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose Time: Improved 2.0 Seconds, Declined -7.1% BB-301 Relative to the Average Pre-Dose Time: Improved 4.0
Slide 53: Improvement was Observed for the CWDT Assessment
Improvement was Observed for the C WDT Assessment Average Pre-Dose CWDT Values vs. Day 90 CWDT Values Final Pre-Dose CWDT Values vs. Day 90 CWDT Values 53 ©2024 Benitec Biopharma | All Rights Reserved
Slide 55: Subject Reported Outcomes: SSQ
Subject Reported Outcomes: SSQ (Declines Below SD and NH Study Screening Visit at Day 0) BB-301 administered on Day 296 (as designated by the yellow bolt) Pre-Dose Scores: Day 0 to Day 287 Post-Dose VFSS: Day 287 (Average) to Day 389 Relative to Final Pre-Dose Score: Improved 329 Points, Declined -2
Slide 56: Improvement was Observed for the SSQ Assessment
Improvement was Observed for the S S Q Assessment Average Pre-Dose SSQ Scores vs. Day 90 SSQ Scores Final Pre-Dose SSQ Scores vs. Day 90 SSQ Scores 56 ©2024 Benitec Biopharma | All Rights Reserved
Slide 57: Clinically Meaningful Improvements
C linically Meaningful Improvements Clinically meaningful improvement over the course of the BB-301 clinical development program will be defined by: • Improvements in Subject-Reported Outcome assessments (i.e., reductions in the Sydney Swallow Questionnaire [”SSQ”] Scores) post BB-301 dose and Reduc
Slide 58: Our Learnings for Subject of the Phase 1b Study
Our Learnings for Subject 1 of the Phase 1b Study Subject 1 experienced disease progression during their enrollment in the Natural History Study After dosing: • BB-301 slowed improved dysphagia in this study subject • The current, low dose of BB-301 was sufficiently biologically active to facilitate
Slide 61: Preliminary Observations and Conclusions for Subject
Preliminary Observations and Conclusions for Subject 1 • Subject 1 experienced progressive worsening of dysphagia during the pre-dose period as demonstrated by the videofluoroscopic swallowing study (VFSS) results, the cold water timed drink test results, and the subject-reported outcome results • F
Slide 62: Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study
Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study (NCT06185673) • The benign safety profile for Subject 1 (the first Study Subject dosed with BB-301) remains unchanged from that which has been reviewed by the Data Safety Monitoring Board • During the first week following the administr
Slide 63: Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study, Continued
Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study (NCT06185673), Continued • During the first week following the administration of BB-301, Subject 2 (the second Study Subject dosed with BB-301) experienced heartburn • The heartburn was attributed to the prophylactic corticosteroids th
Slide 64: Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study, Continued
Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study (NCT06185673), Continued • According to the Phase 1b/2a Study Protocol, any Grade 2 Adverse Event not resolving within 14 days, assessed to be possibly related to the investigational product (see Figure 2) is characterized as a Dose Li
Slide 65: Upcoming Milestones
Upcoming Milestones • Updates on additional subjects are anticipated later in 2024 • Updates on additional subjects at higher doses, and with longer durations of follow-up, are anticipated in 2025 • By year-end 2025, Benitec would potentially have clinical follow-up data for multiple study subjects
Slide 67: PhAMPC Natural History Assessments for Other Enrolled Subjects
PhAMPC Natural History Assessments for Other Enrolled Subjects Moderately Thick PhAMPC 5mL Thin PhAMPC 25 30 25 20 20 15 15 10 10 5 5 0 0 0 50 100 150 200 250 300 0 50 100 150 200 250 300 Study Day Study Day Subject A Subject B Subject A Subject B 67 ©2024 B enitec B iopharm a | All R ights R es erv
Slide 68: PhAMPC Natural History Assessments for Other Enrolled Subjects
PhAMPC Natural History Assessments for Other Enrolled Subjects Extremely Thick PhAMPC Cracker PhAMPC 25 30 25 20 20 15 15 10 10 5 5 0 0 0 50 100 150 200 250 300 0 50 100 150 200 250 300 Study Day Study Day Subject A Subject B Subject A Subject B 68 ©2024 B enitec B iopharm a | All R ights R es erved
Slide 69: Sydney Swallow Questionnaire Natural History Assessments for Other Enrolled Subjects
Sydney Swallow Questionnaire Natural History Assessments for Other Enrolled Subjects SSQ 800 700 600 500 400 300 200 100 0 0 50 100 150 200 250 300 Study Day Subject A Subject B 69 ©2024 B enitec B iopharm a | All R ights R es erved SSQ total score
Slide 70: Cold Water Timed Drinking Test Natural History Assessments for Other Enrolled Subjects
Cold Water Timed Drinking Test Natural History Assessments for Other Enrolled Subjects CWDT 20 18 16 14 12 10 8 6 4 2 0 0 50 100 150 200 250 300 Study Day Subject A Subject B 70 ©2024 B enitec B iopharm a | All R ights R es erved Time (s) to swallow 80ml of ice cold water
Slide 71: For additional information contact
For additional information contact: Irina Koffler LifeSci Advisors, LLC 917-734-7387 ikoffler@lifesciadvisors.com 71 ©2024 Benitec Biopharma | All Rights Reserved

Benitec Biopharma — Investor Day (April 2024) on 6ix

Benitec Biopharma — Investor Day (April 2024)

Slide 1: Research and Development Day Presentation April 2024

Slide 2: Safe Harbor Statement

Slide 3: Research and Development Day Agenda

Slide 4: Professional Biography: Bernard Brais, MDCM, PhD

Slide 5: Professional Biography: Emily Plowman, PhD, CCC-SLP, FASHA

Slide 6: Benitec Corporate Overview

Slide 7: Corporate Highlights

Slide 8: Experienced and Efficient Management

Slide 9: Oculopharyngeal Muscular Dystrophy: Clinical and Pathophysiological Overview

Slide 10: OPMD: A Chronic, Progressive Disease With No Approved Therapeutics

Slide 11: Models for OPMD Pathogenesis

Slide 12: Key Epidemiological Estimates for OPMD

Slide 13: Swallowing Overview: Normal vs. Disordered Swallowing

Slide 14: OPMD: Onset and Progression

Slide 15: OPMD: Clinical Presentation

Slide 16: OPMD: Clinical Presentation (continued)

Slide 17: OPMD: Clinical Presentation (continued)

Slide 18: OPMD: Clinical Complications and Causes of Death

Slide 19: Clinical Management of Dysphagia

Slide 20: BB-301: Mechanism of Action and Clinical Development Program

Slide 21: BB-301 Construct Design and Mechanism of Action

Slide 22: BB-301 Simultaneously Silences Mutant PABPN1 & Delivers Wildtype PABPN1

Slide 23: ddRNAi Platform Enables Both Permanent Silencing AND Replacement

Slide 24: BB-301 Silenced and Replaced PABPN1 in the A17 Mouse Model

Slide 25: BB-301 Restored Muscle Strength to Wildtype Levels in A17 Model

Slide 26: Dose-Dependent Resolution of INIs in A17 Mouse Model

Slide 27: The Rationale for BB-301 in OPMD

Slide 28: BB-301 Clinical Development Program

Slide 29: BB-301 Clinical Development Program and Key Efficacy Assessments

Slide 30: Outcome Measures for the OPMD Natural History Study and BB-301 Phase 1b/2a Study

Slide 31: Videofluoroscopic Swallowing Studies and Subject-Reported Outcome Measures

Slide 32: Key Videofluoroscopic and Subject-Reported Endpoints

Slide 33: Pharyngeal Constrictor Muscle Function: PhAMPC

Slide 34: Swallowing Efficiency: Total Pharyngeal Residue

Slide 35: Subject-Reported Oropharyngeal Dysphagia: Sydney Swallow Questionnaire

Slide 36: Clinically Meaningful Improvements

Slide 37: Preliminary BB-301 Phase 1b Clinical Data Summary for the First Subject (Day 90)

Slide 38: Key Questions for Subject of the Phase 1b Study

Slide 39: VFSS: PhAMPC Results

Slide 40: PhAMPC: Solid Food

Slide 41: PhAMPC: Thin Liquid

Slide 42: PhAMPC: Moderately Thick Liquid

Slide 43: PhAMPC: Extremely Thick Liquid

Slide 44: Improvement was Observed Across all PhAMPC Assessments

Slide 45: VFSS: Total Pharyngeal Residue Results

Slide 46: Total Pharyngeal Residue: Solid Food

Slide 47: Total Pharyngeal Residue: Thin Liquid

Slide 48: Total Pharyngeal Residue: Moderately Thick Liquid

Slide 49: Total Pharyngeal Residue: Extremely Thick Liquid

Slide 50: Improvement was Observed Across all Total Pharyngeal Residue Assessments

Slide 51: Cold Water Timed Drinking Test (CWDT) Results

Slide 52: Timed Drinking Test: CWDT

Slide 53: Improvement was Observed for the CWDT Assessment

Slide 54: SSQ Results

Slide 55: Subject Reported Outcomes: SSQ

Slide 56: Improvement was Observed for the SSQ Assessment

Slide 57: Clinically Meaningful Improvements

Slide 58: Our Learnings for Subject of the Phase 1b Study

Slide 59: Improvement was Observed Across All Assessments

Slide 60: Improvement was Observed Across all Assessments

Slide 61: Preliminary Observations and Conclusions for Subject

Slide 62: Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study

Slide 63: Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study, Continued

Slide 64: Clinical Safety Update for the BB-301 Phase 1b/2a Clinical Study, Continued

Slide 65: Upcoming Milestones

Slide 66: Appendix

Slide 67: PhAMPC Natural History Assessments for Other Enrolled Subjects

Slide 68: PhAMPC Natural History Assessments for Other Enrolled Subjects

Slide 69: Sydney Swallow Questionnaire Natural History Assessments for Other Enrolled Subjects

Slide 70: Cold Water Timed Drinking Test Natural History Assessments for Other Enrolled Subjects

Slide 71: For additional information contact