-- Based on continued emergence of bempikibart data from SIGNAL-AA Part A, including strong re-consent rates and patient demand for continued dosing, Company initiating open-label extension in 1H'25; SIGNAL-AA Part B remains on track for initiation of dosing in 1H'25, with topline data expected in 1H'26 --
-- Company to discontinue renal basket trial for ADX-097 to conserve resources and evaluate strategic options for its tissue-targeted complement inhibitor platform --
-- Cash runway expected to extend to 2H'26 --
WALTHAM, Mass., Feb. 10, 2025 /PRNewswire/ -- Q32 Bio Inc. (Nasdaq: QTTB) ("Q32 Bio"), a clinical stage biotechnology company focused on developing biologic therapeutics to restore immune homeostasis, today announced a corporate restructuring to focus on the advancement of its bempikibart clinical development program for the treatment of patients with alopecia areata (AA). As part of the restructuring, the Company is discontinuing the Phase 2 renal basket clinical trial of ADX-097 and is evaluating strategic options for its tissue-targeted complement inhibitor platform, inclusive of ADX-097 and early-stage assets. In combination with other cost-saving measures including a reduction in personnel and related expenses, the strategic restructuring is expected to extend cash runway to the second half of 2026.
Q32 Bio Logo (PRNewsfoto/Q32 Bio)
"We have conviction that bempikibart is differentiated from existing AA therapies and has the potential to transform the treatment paradigm for this disease. This is based on the continued emergence of bempikibart data in alopecia areata patients with longer-term follow-up from Part A of the SIGNAL-AA Phase 2a clinical trial, the robust pharmacologic data, and a well-tolerated safety profile," said Jodie Morrison, Chief Executive Officer of Q32 Bio. "Further, based on the clinical characteristics of bempikibart, we continue to believe there is potential utility across additional autoimmune conditions. Our immediate next steps will be to extend dosing of eligible patients from SIGNAL-AA Part A in an open-label extension arm and to initiate dosing of patients in the SIGNAL-AA Part B clinical trial in the first half of 2025, with Part B topline data expected in the first half of 2026."
Morrison added, "ADX-097, our Phase 2 complement inhibitor, is a clinical asset with strong potential to treat patients with complement disorders across multiple indications through its novel tissue-targeted approach. While this restructuring is a very difficult decision, it is a necessary step in the context of the evolving complement-mediated renal disease landscape and as we prioritize our focus and capital to maximize the potential of bempikibart. Our restructuring necessitates parting with some valued colleagues, and I want to personally thank them for their dedication to our mission, commitment to patients, and their many contributions to the Company."
Bempikibart Phase 2 Development Program
Results observed to date from Part A of the SIGNAL-AA Phase 2a clinical trial of bempikibart in patients with AA demonstrated encouraging clinical activity, including improvement from baseline on Severity of Alopecia Tool (SALT) score and meaningful achievement of SALT-20 (SALT score less than or equal to 20) response through week 36, and continued response in multiple patients through week 55, approximately 7 months post last dose. Results from this longer-term follow-up show continued mean SALT reductions, despite dosing through only 24 weeks, which may be suggestive of a remittive effect, durability of response, and a key differentiation from currently approved therapies. Across clinical trials, including SIGNAL-AA, bempikibart was observed to be well-tolerated, with robust pharmacologic activity through desired target engagement, as demonstrated by receptor occupancy, robust changes in Th2 biomarkers, and expected changes in T-cells, indicative of potent IL-7 and TSLP inhibition.
Data continues to be collected from a portion of patients who re-consented to the trial, with patient data available through 55 weeks and longer follow-up expected. Based on re-consent rates and strong patient demand for continued dosing, Q32 Bio is initiating an open-label extension (OLE) following the same bempikibart dosing regimen leveraged in Part A to enable longer-term follow-up of patients. In addition to the initiation of the OLE, Q32 Bio expects to initiate dosing in SIGNAL-AA Part B in the first half of 2025.
SIGNAL-AA Part B is an open-label clinical trial, dosing patients with bempikibart for 36 weeks, with follow-up out to 52 weeks, in approximately 20 evaluable patients with severe or very severe AA. Dosing will include an initial loading regimen of 200mg of bempikibart dosed weekly over four weeks, followed by a maintenance dose of 200mg every-other-week over a 32-week period for a total of 36 weeks. The primary efficacy endpoints include the proportion of patients achieving a 30% or greater reduction in SALT score and proportion of patients achieving a SALT-20 at week 36, with follow-up through week 52. The trial is intended to enable advancement into pivotal trials upon completion, pending review of the results. Q32 Bio expects to report initial data from SIGNAL-AA Part B in the first half of 2026.
Complement Inhibitor Platform
ADX-097, a Phase 2 asset, is designed to be a tissue-targeted inhibitor of complement activation while minimizing systemic complement blockade with potential in a broad range of indications associated with C3d deposition including renal, autoimmune, vascular, and skin diseases. In preclinical studies, ADX-097 distributed to affected tissues/organs and demonstrated durable tissue pharmacokinetics (PK) and pharmacodynamics (PD). Q32 Bio has evaluated ADX-097 in a Phase 1 clinical trial in healthy volunteers where it observed circulating PK/PD consistent with preclinical studies, which established in vivo integrity of ADX-097. ADX-097 was also shown to be well-tolerated and demonstrated minimal anti-drug antibodies (ADA).
ADX-097 was discovered by Q32 Bio and is the most advanced program of its tissue-targeted complement inhibitor platform. Additional discovery and earlier development efforts include ADX-096, a C3d mAb – CR1 fusion protein which demonstrated preclinical data supportive of its use in ophthalmologic indications, as well as other C3d mAb fusions and nanobodies designed for tissue-targeted complement inhibition.
About Q32 Bio
Q32 Bio is a clinical stage biotechnology company whose science targets potent regulators of the adaptive immune system to re-balance immunity in autoimmune and inflammatory diseases.
Q32 Bio is advancing bempikibart (ADX-914), a fully human anti-IL-7Rα antibody that re-regulates adaptive immune function for the treatment of autoimmune diseases being evaluated in a Phase 2 program. The IL-7 and TSLP pathways have been genetically and biologically implicated in driving several T cell-mediated pathological processes in numerous autoimmune diseases.
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Forward-Looking Statements
This communication contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements contained herein which do not describe historical facts, including, among others, our beliefs, observations, expectations and assumptions regarding the topline data from the SIGNAL-AA Phase 2a and the safety, tolerability, clinical activity including biomarker data, potential efficacy and potential benefits of bempikibart; plans and expectations for Part B of the SIGNAL-AA Phase 2a clinical trial; and plans and expectations in connection with the evaluation and execution of strategic options for our tissue-targeted complement inhibitor platform are forward-looking statements, which involve risks and uncertainties that could cause actual results to differ materially from those discussed in such forward-looking statements.
Forward-looking statements are based on management's current beliefs and assumptions, which are subject to risks and uncertainties and are not guarantees of future performance. Such risks and uncertainties include, among others, the risk that additional data, or the results of ongoing data analyses, may not support our current beliefs and expectations for bempikibart, future clinical studies, including that Part B of the SIGNAL-AA Phase 2a clinical trial, may not be completed by the first half of 2026 or at all, might be more costly than expected or might not yield anticipated results, we may not be successful in evaluating and executing strategic options for our tissue-targeted complement inhibitor platform; our expectations regarding the sufficiency of our cash and cash equivalents to provide financial runway through clinical milestones and into the second half of 2026, and that we may need additional funding to complete clinical studies, which may not be available on favorable terms or at all, and such other risks and uncertainties identified in the Company's periodic, current and other filings with the U.S. Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and any subsequent filings with the Commission, which are available at the SEC's website at www.sec.gov. Any such risks and uncertainties could materially and adversely affect the Company's results of operations and its cash flows, which would, in turn, have a significant and adverse impact on the Company's stock price. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. The Company disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
