Q32 Bio (QTTB) TD Cowen 45th Annual Healthcare Conference summary

Strategic focus and clinical development

• Prioritizing bempikibart for alopecia areata (AA) due to strong, durable clinical responses observed up to seven months post-treatment.

• Phase 2a completed; adaptive Part B and open-label extension (OLE) added to address patient demand and further validate efficacy.

• Recruitment for Part B and OLE underway, with data expected in the first half of next year.

• Plans to advance to pivotal trials in AA and expand to other Th1/Th2-mediated diseases as capital allows.

• Strategic alternatives sought for complement inhibitor program (ADX097, ADX096) to focus resources on AA.

Clinical data and patient outcomes

• Bempikibart, a bifunctional antibody targeting IL-7 and TSLP, demonstrated favorable PK/PD, minimal ADA, and robust biomarker engagement.

• Durable hair regrowth observed in severe, long-duration AA patients, with some showing near-complete recovery months after last dose.

• Both per protocol and modified intent-to-treat analyses showed clear separation from placebo at 24 weeks, with responses deepening beyond week 36.

• Open-label extension initiated after patients reported continued improvement post-study, including those with prior access to JAK inhibitors.

• Safety profile remains favorable across 130 patients, supporting competitive positioning.

Market opportunity and competitive landscape

• AA market in the US includes 700,000 patients, with only 30,000 currently treated with JAK inhibitors due to safety concerns.

• JAK inhibitors carry black box warnings and require indefinite use; bempikibart offers a differentiated, durable alternative.

• No biologic competitors currently in AA; potential for bempikibart to become the first-line biologic therapy.

• Market projected to reach $2.6 billion by 2030, with high demand for safer, less frequent dosing options.

• Physician and patient feedback highlights strong interest in biologics with remittive effects and subcutaneous administration.