Q32 Bio Inc. · NASDAQ:QTTB
Q32 Bio — Investor Presentation (January 2025)
34 slides from Q32 Bio's investor presentation, Jan 13, 2025
Slide 1: Building The Future of Immune Therapeutics: Company Overview
Exhibit 99.1 Building The Future of Immune Therapeutics Company Overview January 2025
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation has been prepared by Q32 Bio Inc. (“we”, “us,” “our,” “Q32” or the “Company”) and is made for informational purposes only. The information set forth herein does not purport to be complete or contain all of the information you may desire. Statements contai
Slide 3: Q32 Mission: Building The Future of Immune Therapeutics
Q32 Mission: Building The Future of Immune Therapeutics • Proof of concept (POC) demonstrated in alopecia areata (AA) in Phase 2a Part A; high unmet need where treatments Bempikibart (ADX-914) include JAK inhibitors which have class-wide safety warnings, a significant limitation for the AA patient d
Slide 4: Q32 Pipeline: Poised to Deliver Multiple Near-term Clinical Readouts
Q32 Pipeline: Poised to Deliver Multiple Near-term Clinical Readouts Discovery/ Anticipated Program Indication Phase 1 Phase 2 Preclinical Milestones IL-7/TSLP PROGRAM Bempikibart Alopecia Initiate Part B enrollment 1H’25; 1 (ADX-914) Areata Topline data expected 1H’26 COMPLEMENT INHIBITOR PLATFORM
Slide 5: Bempikibart (ADX-914) (IL-7 / TSLP Receptor Inhibitor)
Bempikibart (ADX-914) (IL-7 / TSLP Receptor Inhibitor)
Slide 6: Bempikibart: Bifunctional Antibody for T-cell Mediated I&I Diseases With Demonstrated Activity in AA
Bempikibart: Bifunctional Antibody for T-cell Mediated I&I Diseases With Demonstrated Activity in AA Bempikibart IL-7Rα antagonist antibody: Biomarker changes and clinical activity in Phase 2a: Blocks IL-7 and TSLP signaling Supports IL-7Rα antagonist approach Biomarker Median Changes Over Time IL-7
Slide 7: SIGNAL-AA Case Study - Severe AA with 4.5 Year Episode Showing SALT-10 at Week 36
SIGNAL-AA Case Study - Severe AA with 4.5 Year Episode Showing SALT-10 at Week 36: Supports Potential for Durable Hair Regrowth with Bempikibart Treatment Baseline Week 24 Week 36 SALT Score % Change from Baseline 0 -20 -40 -60 -80 -100 111-0010 ❖ 52-year-old female ❖ Duration of episode: 4.5 years
Slide 8: SIGNAL-AA: Part A SALT Data Through Week 36 Follow-up Showed Continued Benefit
SIGNAL-AA: Part A SALT Data Through Week 36 Follow-up Showed Continued Benefit Over Time Supporting Potential for Remittive Effect Following Dosing Cessation SALT Percentage Change Over Time SALT Percentage Change Over Time SALT Percentage Change Over Time 1 1 1 (SIGNAL AA, Per Protocol ) (SIGNAL AA
Slide 9: SIGNAL-AA Case Study - Very Severe AA with SALT-10 Response After Dosing Cessation
SIGNAL-AA Case Study- Very Severe AA with SALT-10 Response After Dosing Cessation: Supports Potential for Remittive Effect with Bempikibart Treatment Baseline Week 24 Week 36 Week 57 7 months after last dose No additional concomitant treatments ❖ 61-year-old female ❖ Duration of Episode: 3.1 years ❖
Slide 10: SIGNAL-AA First-in-Patient Observations of Durable Response Supported by Literature
SIGNAL-AA First-in-Patient Observations of Durable Response Supported by Broad Literature Describing IL-7 Mechanistic Modulation of T cells eff/mem 10
Slide 11: Preclinical Evidence of Long-Term Durable Effects Following IL-7Rα Antibody Treatment
Preclinical Evidence of Long-Term Durable Effects Following IL-7Rα Antibody Treatment: Models Suggest a Mechanism for Rebalancing T and T function eff/mem reg Type 1 Diabetes Model Collagen Induced Arthritis Model Tuberculin Challenge Model Belarif 2018 Nat. Comm. Days post immunization BMS report B
Slide 12: Maturing SIGNAL-AA Data Package Supports Potentially Differentiated Profile in AA
Maturing SIGNAL-AA Data Package Supports Potentially Differentiated Profile in AA • Durable hair regrowth observed supports potential for transformative paradigm • Mean SALT scores continued to improve from Week 24 to Week 36 • Multiple reports received of post 36-week hair growth; data collection u
Slide 13: SIGNAL-AA Phase 2a: Part A Complete With Ongoing Expansion for Additional Follow-up
SIGNAL-AA Phase 2a: Part A Complete Through Week 36 Follow-up Period With Ongoing Expansion for Additional Follow-up Week 24 Part A ✓ Design Elements Primary Endpoint Week 36 Part A Percent change from baseline in SALT score at Week 24 • First exposure in AA patients Bempikibart (n=30) Efficacy and
Slide 14: AA Has Life-Altering Impact and Limited Treatment Options Including JAK Inhibitors
AA Has Life-Altering Impact and Limited Treatment Options Including JAK Inhibitors Carrying Black Box Warnings 1 700K people living with AA in the U.S. Doctors and patients seek alternatives to currently 2 approved agents including JAK inhibitors which Often manifesting before age 50 carry significa
Slide 15: AA is Prevalent, Stigmatizing and Psychologically Burdensome; Unmet Need for New Options
AA is Prevalent, Stigmatizing and Psychologically Burdensome; Unmet Need for New Options with Better Safety and Ability to Provide Durable, Long-Term Remittance Total AA Market Forecast 3 ($ Billions) 1 700K $3.0 US Alopecia Areata (AA) Patients $2.6B $2.5 $2.0 1 300K $1.5 Severe (SALT > 50) $1.0B $
Slide 16: A Product With Remittive Properties Could Transform the AA Treatment Paradigm
A Product With Remittive Properties Could Transform the AA Treatment Paradigm: Potential to Differentiate on Safety and Inducing Long-Term Durable Remission [T]he potential to have a side effect profile perhaps closer to biologics we use in atopic dermatitis…is certainly a compelling value propositi
Slide 17: Opportunities Beyond AA: Potential to Expand into Th1 and Th2 Mediated Diseases
Opportunities Beyond AA: Potential to Expand into a Broad Range of Th1 and Th2 Mediated Diseases SIGNAL Phase 2a SIGNAL Phase 2a Meaningful effect on Results support the Th2 biomarkers potential for long observed term, durable responses Eosiniphils, IgE, TARC Suggestive of T /T impact eff mem Confid
Slide 18: Tissue-Targeted Complement Platform Lead Asset: ADX-097
Tissue-Targeted Complement Platform Lead Asset: ADX-097
Slide 19: Q32 Tissue-targeted Platform Value Proposition
Q32 Tissue-targeted Platform Value Proposition: Designed to Enable Clinical Profile Superior to Systemic Complement Inhibitors The Unmet Need The Opportunity • Limited activity: • Enhanced activity through tissue targeting: Reliant on systemic blockade for impact on Differentiated approach to drivin
Slide 20: ADX-097 Lead Bivalent Fusion Protein: Unique MOA for Localized Complement Re-regulation
ADX-097 Lead Bivalent Fusion Protein: Designed with Unique MOA to Drive Localized, Complement Re-regulation for Enhanced Activity and Tolerability ADX-097 Design: C3d antibody – fH 1-5 Designed to be held at site of tissue complement Inactivation of alternative pathway convertases activity allowing
Slide 21: Phase 1 Study: Complete with Primary Goals Achieved
Phase 1 Study: Complete with Primary Goals Achieved Phase 1 SAD/MAD (n= 56 Healthy Volunteers) Explored Single Doses of 0.1 - 30 mg/kg IV and/or SC and 450 mg SC Multiple Dose Cohort (~6 mg/kg) Primary Goals Achieved Results • Attained expected dose-dependent PK/PD Confirm planned Phase 2 • Once wee
Slide 22: ADX-097 SPOTLIGHT Phase 2 Renal Basket Clinical Trial: Enrollment Ongoing
ADX-097 SPOTLIGHT Phase 2 Renal Basket Clinical Trial: Enrollment Ongoing Details and Timeline Renal Basket (n= up to 30) • Open-label trial designed to assess safety, tissue 26-week treatment pharmacology and magnitude/timing of treatment effect • Key assessments: Drug localization and impact in ti
Slide 23: ADX-097: Significant Market Opportunity Including Initial Focus on Renal Diseases
ADX-097: Significant Market Opportunity Significant Market Opportunity including in Initial Focus on Renal Diseases (LN, IgAN, C3G) Lupus Nephritis (LN) 1,2 • 6-fold mortality risk increase vs general population • Up to 30% develop kidney failure requiring dialysis or 3,4 kidney transplant within 15
Slide 24: Summary: Financial Overview and Anticipated Milestones
Summary: Financial Overview and Anticipated Milestones
Slide 25: Q32 Bio Has Significant Potential to Unlock Near-term Value Creation
Q32 Bio Has Significant Potential to Unlock Near-term Value Creation Runway through multiple Phase 2 clinical readouts and into mid-2026 • Q3 cash balance of $89.1M, providing expected cash runway to mid 2026 Financial Overview • Approximately 12.1M shares outstanding • 1H’25 – Bempikibart: Initiate
Slide 26: Appendix
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Slide 27: Bempikibart in AA: IL-7 Inhibition is Believed to Block CD8+ T cell-Driven Inflammation
Bempikibart in AA: IL-7 Inhibition is Believed to Block CD8+ T cell-Driven Inflammation Hair Follicle Immune Dysregulation in Alopecia Curr Res Immunol. 2021 27
Slide 28: Bempikibart Shows Potential to be Best in Class IL-7Rα Antibody
Bempikibart Shows Potential to be Best in Class IL-7Rα Antibody Bempikibart Lusvertikimab (OSE-127) ZB-168 GSK-2618960 Q32 Bio OSE Zura Bio (formerly Pfizer) GSK (IgG1 Effector-less) (IgG4) (IgG1) (IgG1) 1 Active in development Not currently funded ✓✓û Antagonist -- ✓✓û PK/PD supports current single
Slide 29: SIGNAL-AA Part A: Favorable PK and Receptor Occupancy Achieved
SIGNAL-AA Part A: Favorable PK and Receptor Occupancy (RO) Achieved 2 1 SIGNAL-AA: PK SIGNAL-AA: RO 100 100 10 10 5 μg/mL 5 μg/mL 1 1 0.1 0.1 Nominal Time After Dose (Days) Nominal Time After Dose (Days) 200 mg 200 mg Bempikibart Phase 2a achieved expected PK and RO, supporting current subcutaneous
Slide 30: SIGNAL-AA Part A: Baseline Characteristics
SIGNAL-AA Part A: Baseline Characteristics 1 mITT (n=44) Revised Per Protocol (n=27) Bempikibart 200 mg (n=33) Placebo (n=11) Bempikibart 200 mg (n=23) Placebo (n=4) Gender (n, %) Female (27, 81.8%) Female (7, 63.6%) Gender (n, %) Female (18, 78.3%) Female (2, 50.0%) Age 48.8 ± 10.2 47.1 ± 14.2 Age
Slide 31: SIGNAL-AA: Topline Data, as Presented December 10, 2024
SIGNAL-AA: Topline Data, as Presented December 10, 2024 KEY FINDINGS: Clinical Activity Observed in SIGNAL-AA • Mean change in SALT 16% vs 2% for placebo (Week 24) Post 24 Week Treatment • Mean change in SALT 18% vs 2% for placebo (Week 26) Updated for Complete • SALT-20 achieved in 9% at Week 24, 1
Slide 32: SIGNAL Phase 2a: Favorable Safety and Tolerability Profile
SIGNAL Phase 2a: Favorable Safety and Tolerability Profile in Phase 2a with No Grade 3 or Higher Related AEs Adverse Events (SIGNAL Trials Through Week 24) Bempikibart 200 mg Placebo (N=96) (N=69) n (%) [E] n (%) [E] Participants with at least one TEAE 55 (57.2) [154] 30 (43.5) [76] Participants wit
Slide 33: Substantial Activity on Biomarkers Observed in Clinical Trials
Substantial Activity on Biomarkers Observed in Clinical Trials Supports Potent Inhibition of TSLP and IL-7 Mediated Signaling 1 2 Th2 Biomarkers: SIGNAL Phase 2a Th1 Biomarkers: SIGNAL Phase 2a Median Changes Over Time (95% CI) Mean Changes Over Time (±SD) + TARC IgE Eosinophils CD3 T cells Placebo
Slide 34: ADX-097 Preclinical Data: Desired PK/PD, Favorable Tolerability and Immunogenicity
ADX-097 Preclinical Data: Desired PK/PD, Favorable Tolerability and Immunogenicity Preclinical data supports ADX-097 as a tissue-targeted complement inhibitor • Durable (>7 days) tissue PK/PD after SC dosing • Reduction in key proof of mechanism/proof of concept biomarkers including proteinuria and