Our Pipeline
Our clinical and discovery pipeline programs are centered on restoring immune homeostasis in patients with severe inflammatory and autoimmune diseases.
For pipeline inquiries, please contact: BD@q32bio.com.
IL-7/TSLP Program
Promising data from a completed Phase 1 clinical trial showed bempikibart (ADX-914) was well tolerated and exhibited a pharmacokinetic and pharmacodynamic profile supporting dosing of no more frequently than once every two weeks. Q32 Bio is currently evaluating bempikibart in two ongoing Phase 2 clinical trials: SIGNAL-AD, a Phase 2 study in patients with atopic dermatitis (AD) [ADX-914-202] and SIGNAL-AA, a Phase 2 study in patients with alopecia areata (AA) [ADX-914-203].
Bempikibart is a fully human antibody anticipated to block IL-7- and TSLP-mediated signaling via their cognate receptors. IL-7 and TSLP signaling have been biologically linked to numerous inflammatory and autoimmune diseases including Q32 Bio’s initial target diseases of AD and AA.
Accumulating evidence suggests that TSLP and IL-7 may act as sequential mediators of AD initiation (via Th2 pathways) and progression (via TH1 and/or TH17). This emerging view supports the belief that novel therapeutics, such as bempikibart, which more specifically address the underlying immune-phenotypic progression of the disease, are needed. TH1 has long been implicated in the pathogenesis of AA supporting the potential for bempikibart to directly address the underlying driver of follicle damage and hair loss. In addition, given that AA is a disease often diagnosed in young adults, there is a critical need for effective novel treatments with a safety profile suitable for long-term, chronic treatment.
Complement Program
Q32 Bio has developed a novel discovery platform that is enabling the advancement of tissue-targeted complement inhibitors which have potential therapeutic activity across the numerous diseases in which C3 complement fragments are deposited in diseased tissue. From this platform, we are advancing ADX-097, our lead clinical candidate, into Phase 2 clinical development. Q32 Bio is initially developing ADX-097 for the treatment of renal and other complement-mediated diseases of high unmet need, including lupus nephritis (LN), IgA nephropathy (IgAN), C3 glomerulopathy (C3G) and ANCA-associated vasculitis (AAV).
In preclinical studies, ADX-097 distributed to affected tissues/organs and demonstrated durable tissue pharmacokinetics and pharmacodynamics. Additionally, Q32 Bio has evaluated ADX-097 in a Phase 1 clinical trial in healthy volunteers where Q32 Bio observed circulating PK/PD consistent with preclinical studies, which established in vivo ADX-097 integrity and informed Q32 Bio’s dosing strategy for next stage clinical testing. ADX-097 was also shown to be well tolerated.
Based on the compelling evidence from these studies, Q32 believes ADX-097 has the potential to address the limitations of the currently available systemic approaches to complement inhibition, including infection risk and the need for high drug doses and frequent administration, to achieve therapeutic levels of inhibition. Q32 Bio plans to evaluate ADX-097 in an open-label Phase 2 renal basket clinical trial and a Phase 2 clinical trial in AAV.