Accessibility StatementSkip Navigation -- Asset sale further enables Company's strategic focus on advancing bempikibart for alopecia areata -- -- $12 million in upfront and guaranteed near-term milestone payments expected to extend cash runway into the second half of 2027 -- -- Eligible to receive up to a total of $592 million including the $12 million in upfront and near-term payments upon achievement of certain development, regulatory and commercial milestones and eligible for tiered royalties up to a mid ...

Core Insights - Q32 Bio has completed enrollment in Part B of the SIGNAL-AA Phase 2a clinical trial, increasing the trial size to 33 patients due to high patient demand, with topline data expected in mid-2026 [1][5][2] - The company reported cash and cash equivalents of $49.0 million as of September 30, 2025, which is anticipated to fund operations into 2027 [1][4][11] - The third quarter of 2025 showed a significant reduction in research and development expenses, totaling $3.6 million compared to $14.3 million in the same quarter of 2024 [4][6][11] Clinical Development - The SIGNAL-AA Phase 2a clinical trial is evaluating bempikibart, a fully human anti-IL-7R antibody, in patients with severe alopecia areata (AA) [5][7] - The trial includes a loading regimen of 200mg bempikibart weekly for four doses, followed by a maintenance dose every other week for a total of 36 weeks [5] - Preliminary pharmacokinetic data indicates that steady state drug concentration is achieved at least nine weeks earlier in Part B compared to Part A, suggesting potential for earlier clinical responsiveness [5][2] Financial Performance - Q32 Bio reported a net loss of $7.4 million for the quarter ended September 30, 2025, a decrease from a net loss of $17.6 million in the same quarter of 2024 [11][13] - General and administrative expenses decreased to $4.0 million from $4.5 million year-over-year, primarily due to lower personnel-related costs [11][4] - Total operating expenses for the third quarter of 2025 were $7.6 million, down from $18.8 million in the same period of 2024 [11][4] Market Context - Approximately 700,000 individuals in the United States are affected by alopecia areata, highlighting a significant market need for effective treatments [7] - Q32 Bio aims to provide a differentiated therapeutic option for AA patients, addressing the demand for safer and more durable treatment alternatives [2][5]

Core Insights - Q32 Bio has completed enrollment in Part B of the SIGNAL-AA Phase 2a clinical trial for bempikibart, exceeding the initial target of approximately 20 patients, with a total of 33 patients enrolled due to high demand [1][3] - Bempikibart is a fully human anti-IL-7R antibody aimed at treating severe alopecia areata (AA) by re-regulating adaptive immune function [1][3] - The trial is designed to evaluate the efficacy of bempikibart over a 36-week treatment period, with follow-up extending to 52 weeks, and aims to support advancement into pivotal trials based on the results [2][3] Company Overview - Q32 Bio is a clinical stage biotechnology company focused on developing therapies for alopecia areata and other autoimmune diseases, targeting the adaptive immune system [3] - Approximately 700,000 individuals in the United States are affected by alopecia areata, highlighting the need for new treatment options [3] Clinical Trial Details - Part B of the SIGNAL-AA trial includes a loading regimen of 200mg of bempikibart administered weekly for four doses, followed by a maintenance dose every other week for a total of 36 weeks [2] - Efficacy will be assessed using the Severity of Alopecia Tool (SALT) scores, with the goal of observing significant improvements in patients' conditions [2] Future Expectations - Preliminary data from early-enrolling patients in Part B indicate emerging signs of clinical activity, with a faster achievement of steady-state drug concentration compared to Part A [2] - Q32 Bio anticipates reporting topline data from Part B in mid-2026, which could lead to further development of bempikibart pending positive results [3]

Financial Data and Key Metrics Changes - The company has extended its cash runway into Q1 2027, providing sufficient margin to allow ongoing trials to complete [47][48] - The company reported a 20% reduction in SALT score at week 36 in part A, with expectations to improve upon this in part B [28][29] Business Line Data and Key Metrics Changes - The company is advancing bempikibart, focusing on alopecia areata, with ongoing enrollment in phase 2 part B after positive results from part A [5][36] - In part A, statistically significant hair improvement was observed at week 24, with no grade 3 or higher related events reported [8][12] Market Data and Key Metrics Changes - The competitive landscape includes other IL-7 receptor alpha programs, with the company believing it has a best-in-class asset due to its pharmacology and dosing advantages [43][44] Company Strategy and Development Direction - The company is focusing on immunomodulating agents rather than immunosuppressants, aiming for clinically meaningful efficacy with durable responses [31][32] - Future trials may include registrational studies, with potential for seamless phase 2 part B/3 trials [36] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the differentiated profile of bempikibart, emphasizing its safety and potential for durable effects [49][51] - Early signs in part B indicate improved responsiveness compared to part A, suggesting that changes made to the trial design are effective [52][53] Other Important Information - The company has made significant changes to trial design in part B, including a loading dose regimen and modified inclusion criteria to enhance patient responsiveness [9][25] - Management noted that the atopic dermatitis trial did not yield expected results, but it provided valuable insights for future indications [42] Q&A Session Summary Question: What are the next steps for bempikibart? - The next steps could include registrational trials, with various options being considered for the trial design [36] Question: How does the company plan to fund operations moving forward? - The company has sufficient cash runway into 2027 and is exploring strategic options for further funding [47][48] Question: What differentiates bempikibart from competitors? - The company believes its pharmacology and dosing regimen provide a competitive edge over other IL-7 receptor alpha programs [43][44] Question: What are the expectations for part B results? - Management hopes to see improved efficacy and responsiveness in part B compared to part A, with a focus on clinically meaningful outcomes [52][53]

Core Viewpoint - Q32 Bio reported a significantly reduced GAAP net loss of $0.78 per share for Q2 2025, with no revenue, reflecting its pre-commercial status and a focus on its lead drug candidate, bempikibart [1][5][10] Financial Performance - The net loss (GAAP) decreased to $9.5 million in Q2 2025 from $17.0 million in Q2 2024, primarily due to an $8.2 million reduction in research and development expenses [5] - Research and development expenses were $5.2 million in Q2 2025, down 61.2% from $13.4 million in Q2 2024 [2] - General and administrative expenses decreased to $4.0 million in Q2 2025 from $4.5 million in Q2 2024, reflecting lower staffing and legal costs [2][5] - Cash and cash equivalents at the end of the period were $54.8 million, expected to fund operations into 2027 [2][7] Business Focus and Strategy - Q32 Bio is concentrating on bempikibart, an experimental therapy for autoimmune diseases, and has halted development on its ADX-097 project [3][4] - The company underwent a restructuring in February 2025 to conserve resources and accelerate progress on bempikibart [4] Clinical Development - The bempikibart program is currently in the SIGNAL-AA Phase 2a trial, with ongoing enrollment and topline results expected in the first half of 2026 [6][10] - Bempikibart received Fast Track designation from the U.S. FDA for alopecia areata, which may expedite its development and review process [8] Future Outlook - Management does not anticipate revenue in the near term and has prioritized completing the SIGNAL-AA trial [10]

Clinical Trial Results - The SIGNAL-AA Phase 2a trial studied bempikibart in patients with severe or very severe Alopecia Areata [1] - The study included patients with SALT scores between 50 and 100 [15] - At Week 24, patients with baseline SALT 50-100 showed a mean SALT score change of 163% with bempikibart treatment [20] - In patients with baseline SALT 50-95, the mean SALT score change at Week 24 was 245% with bempikibart [20] - Some patients experienced continued response even 7 months post last dose [23, 24] Safety and Tolerability - In the bempikibart group, 70% of participants experienced at least one treatment-emergent adverse event (TEAE) [27] - In the placebo group, 38% of participants experienced at least one TEAE [27] - No Grade 3 or higher related adverse events were reported in the bempikibart group [27] Study Design and Demographics - The study randomized patients in a 3:1 ratio to bempikibart (n=33) or placebo (n=81) [15] - The mean baseline SALT score in the bempikibart group was 749, while in the placebo group it was 819 [16]

Core Insights - Q32 Bio has made significant progress in its clinical trials for bempikibart, receiving Fast Track designation and initiating dosing in both parts of the SIGNAL-AA Phase 2a clinical trial [2][3][4] - The company reported a net loss of $11.0 million for Q1 2025, compared to a net income of $1.0 million in Q1 2024, indicating a shift in financial performance [9][13] - Cash and cash equivalents stood at $65.5 million as of March 31, 2025, which is expected to fund operations into the second half of 2026 [9][12] Clinical Development - The first patient has been dosed in Part B of the SIGNAL-AA Phase 2a clinical trial, with topline data readout anticipated in the first half of 2026 [3][4] - Part B will evaluate bempikibart in approximately 20 patients with severe or very severe alopecia areata (AA) over a 36-week treatment period [3][4] - An open-label extension (OLE) for Part A has been initiated to allow for longer-term follow-up of patients who completed the initial trial [3][4] Fast Track Designation - Bempikibart has received Fast Track designation from the FDA, which aims to expedite the development and review of drugs for serious diseases [3][4] - This designation may allow for more frequent communication with the FDA and eligibility for Accelerated Approval and Priority Review [3] Financial Performance - Research and development expenses decreased to $7.1 million in Q1 2025 from $9.8 million in Q1 2024, reflecting a focus on clinical development [9][13] - General and administrative expenses remained stable at $5.1 million for Q1 2025, consistent with the previous year [9][13] - The company reported a basic and diluted net loss per share of $(0.90) for Q1 2025, compared to a net income per share of $1.03 in Q1 2024 [9][13] Market Context - Approximately 700,000 individuals in the United States are affected by alopecia areata, highlighting the significant unmet medical need in this area [6] - Q32 Bio is focused on developing therapies that target the adaptive immune system, particularly for autoimmune and inflammatory diseases [6]