Our clinical and discovery pipeline programs are centered on restoring immune homeostasis in patients with severe inflammatory and autoimmune diseases.
Q32 Bio has developed a novel discovery platform that is enabling the generation of first-in-class complement inhibitors that target diseased tissues. From this platform, a strong pipeline of promising candidates, with potential therapeutic activity across the numerous diseases in which C3 complement fragments are deposited in the diseased tissue, is being advanced through research and into clinical development.
In preclinical studies, our lead clinical candidate, ADX-097, distributed to affected tissues/organs and demonstrated durable tissue pharmacokinetics and pharmacodynamics. Based on the compelling evidence from these studies, ADX-097 may potently and durably block complement activity in humans, while preserving the body’s ability to fight infection effectively, thereby avoiding the potentially damaging effects of systemic complement inhibition.
ADX-097 is currently being evaluated in a single ascending dose/multiple ascending dose and proof-of-mechanism study, ADX-097-101.
IL-7Rα Antagonist Program
Q32 Bio is collaborating with Horizon Therapeutics to clinically evaluate ADX-914 (bempikibart), a potent IL-7Rα antagonist with the potential to be a best-in-class modulator of IL-7 signaling.
Q32 Bio recently completed a biomarker-enabled first-in-human study that characterized the safety, pharmacokinetics, pharmacodynamics of ADX-914; the study also demonstrated that ADX-914 had a pharmacological effect on T cells in healthy volunteers.
ADX-914 (bempikibart) is currently being evaluated in SIGNAL-AD, a Phase 2 study in patients with atopic dermatitis [ADX-914-202] and SIGNAL-AA, a Phase 2 study in patients with alopecia areata [ADX-914-203].