Q32 Bio (QTTB) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Company overview and pipeline

• Founded seven years ago, focusing on complement inhibitors and immune modulation.

• Lead asset benpicobart (ADX-914) targets IL-7/TSLP pathways, with two phase II trials in atopic dermatitis and alopecia areata, both reading out in Q4 2024.

• Complement program includes ADX-097, a cell-targeted bifunctional fusion protein in a phase II renal basket study for IgA nephropathy, lupus nephritis, and C3G.

• Benpicobart is now wholly owned after reacquisition from Amgen following its acquisition of Horizon.

Benpicobart mechanism and competitive landscape

• Benpicobart is a bifunctional antibody antagonizing both IL-7 and TSLP pathways, providing broad Th2 and Th1 immunophenotype coverage.

• No other IL-7 pathway antibodies are currently being tested in atopic dermatitis.

• Competing antibodies (OSE, Zura, GSK) differ in potency, formulation, and clinical focus; benpicobart is subcutaneous and highly potent at low doses.

• Preclinical and genetic evidence supports dual pathway inhibition for efficacy in atopic dermatitis and alopecia.

Clinical trial design and benchmarks

• Atopic dermatitis phase II trial has two parts: Part A (dose-ranging, 15 patients, completed) and Part B (efficacy, 106 patients, ongoing), with a 14-week primary endpoint (mean change in EASI score).

• Monotherapy trial excludes topical corticosteroids and calcineurin inhibitors; allows prior biologic exposure with washout.

• Efficacy benchmark is a placebo-adjusted EASI change of ~30% (as seen in OX40 ligand trials); EASI 50, 75, 90, and IGA response will be disclosed.

• Alopecia phase II trial enrolls severe and very severe patients (SALT 50-100), with a 24-week endpoint; efficacy benchmark is >20% placebo-adjusted SALT change.

• Both atopic dermatitis and alopecia trials use the same dosing and are expected to report results simultaneously in Q4 2024.