bempikibart (ADX-914) / Q32 Bio, BMS - LARVOL DELTA

Completed enrollment in Part B of the SIGNAL-AA Phase 2a clinical trial and increased the trial size to 33 patients based on patient demand, with 36-week topline data readout expected in mid-2026. (PRNewswire) - "In addition, based on preliminary pharmacokinetic data available to date in the Part B portion of SIGNAL-AA, steady state concentration of drug in patients is achieved at least nine weeks earlier as compared to Part A of the clinical trial due to the inclusion of the loading regimen, which may have the potential to induce earlier responsiveness. The trial is intended to support advancement into pivotal trials upon completion, pending review of the results....Part A OLE of the SIGNAL-AA Phase 2a clinical trial is ongoing."

P2a data • Trial status • Alopecia

Q32 Bio Announces $10.5 Million Registered Direct Offering (PRNewswire) - "The gross proceeds to Q32 Bio from this offering are expected to be approximately $10.5 million, before deducting certain offering expenses. Q32 Bio intends to use the net proceeds from the offering, together with its existing cash, cash equivalents and marketable securities, for working capital purposes including expenses related to research, clinical development and commercialization efforts including for supporting the advancement of bempikibart into future clinical trials for the treatment of alopecia areata."

Financing • Alopecia

Bempikibart, a Novel Human IL-7Rα Antagonist Inhibiting IL-7 and TSLP Signaling, Demonstrated Robust Impact on T-cell Maintenance and Th2 Responses in T-cell Driven Skin Disease (ISDS 2025) - "These results highlight bempikibart as a novel and potent IL-7Rα inhibitor with a favorable PK/PD profile, demonstrating dual activity in reducing Th2 biomarkers and T-cell counts. These findings support its continued development for T-cell-driven autoimmune diseases."

Clinical • Atopic Dermatitis • Dermatitis • Dermatology • Immunology • CRLF2 • IL7 • IL7R • TSLP

Dosing of patients in Part A OLE of the SIGNAL-AA Phase 2a clinical trial is ongoing. (PRNewswire) - "Based on the continued emergence of bempikibart data suggesting a remittive effect and durable responses in long-term follow-up from SIGNAL-AA Part A, as well as re-consent rates and strong patient demand for continued dosing, Q32 Bio initiated an OLE in April 2025 for eligible patients that completed Part A to enable longer-term follow-up of patients."

Enrollment status • Alopecia

A Phase 2a Proof-of-Concept Trial of Bempikibart (ADX-914) for the Treatment of Severe Alopecia Areata (SIGNAL-AA) (clinicaltrials.gov) - P2 | N=75 | Active, not recruiting | Sponsor: Q32 Bio Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Alopecia • Dermatology • Immunology

Q32 Bio Announces Completion of Enrollment in Part B of SIGNAL-AA Phase 2a Clinical Trial of Bempikibart for Alopecia Areata (PRNewswire) - "Total enrollment of 33 patients exceeded the initial target of approximately 20 patients due to demand from patients and their healthcare providers. As a result of the over-enrollment, Q32 Bio expects to report topline data from SIGNAL-AA Part B in mid-2026."

P2a data • Trial status • Alopecia

Q32 Bio Reports Second Quarter 2025 Financial Results and Provides Corporate Update (PRNewswire) - "Dosing of patients in Part B of the SIGNAL-AA Phase 2a clinical trial is ongoing, with topline data readout on-track for the first half of 2026....Dosing of patients in Part A OLE of the SIGNAL-AA Phase 2a clinical trial is ongoing....Cash and cash equivalents were $54.8 million as of June 30, 2025. Q32 Bio believes its cash and cash equivalents are sufficient to fund operations into 2027, through the SIGNAL-AA OLE and topline results of the SIGNAL-AA Part B trial evaluating bempikibart in patients with AA expected in the first half of 2026."

Commercial • P2a data • Trial status • Alopecia

Q32 Bio Announces FDA Fast Track Designation Granted to Bempikibart (ADX-914) for the Treatment of Alopecia Areata (PRNewswire) - "Q32 Bio Inc...announced that the United States Food and Drug Administration ('FDA') has granted Fast Track designation (FTD) to Q32 Bio's bempikibart (ADX-914) for the treatment of AA....'This Fast Track designation follows the encouraging clinical activity observed in Part A of our SIGNAL-AA clinical trial and the recent initiation of dosing in the Part A open-label extension and Part B portions of the SIGNAL-AA trial'."

Fast track • Alopecia

Q32 Bio Reports First Quarter 2025 Financial Results and Provides Corporate Update (PRNewswire) - "Cash and cash equivalents were $65.5 million as of March 31, 2025. Q32 Bio believes its cash and cash equivalents are sufficient to fund operations into the second half of 2026, through the SIGNAL-AA OLE and topline results of the SIGNAL-AA Part B trial evaluating bempikibart in patients with AA...Research and development expenses were $7.1 million for the three months ended March 31, 2025, compared to $9.8 million for the three months ended March 31, 2024. The decrease in expense of $2.7 million was primarily due to higher clinical development expenses for bempikibart Phase 2 clinical trials in AA and atopic dermatitis in 2024."

Commercial • Alopecia • Atopic Dermatitis

A Phase 2a Proof-of-Concept Trial of Bempikibart (ADX-914) for the Treatment of Severe Alopecia Areata (SIGNAL-AA) (clinicaltrials.gov) - P2 | N=75 | Recruiting | Sponsor: Q32 Bio Inc. | Active, not recruiting ➔ Recruiting | N=40 ➔ 75 | Trial completion date: Dec 2024 ➔ Sep 2026 | Trial primary completion date: Dec 2024 ➔ Jun 2026

Enrollment change • Enrollment open • Trial completion date • Trial primary completion date • Alopecia • Dermatology • Immunology

Q32 Bio Doses First Patients in Both Part A Open-Label Extension and Part B of SIGNAL-AA Phase 2a Trial Evaluating Bempikibart in Alopecia Areata (PRNewswire) - "Q32 Bio Inc...announced that the Company has dosed the first patients in both the Part A open-label extension (OLE) and Part B of the SIGNAL-AA Phase 2a clinical trial evaluating bempikibart in patients with alopecia areata (AA)....Q32 Bio expects to report topline data from SIGNAL-AA Part B in the first half of 2026."

P2a data • Trial status • Alopecia

Q32 Bio Reports Fourth Quarter 2024 Financial Results and Provides Corporate Update (PRNewswire) - "Cash and cash equivalents were $78.0 million as of December 31, 2024. The Company believes its cash and cash equivalents are sufficient to fund operations into the second half of 2026, through the SIGNAL-AA OLE and topline results of the SIGNAL-AA Part B trial evaluating bempikibart in patients with AA. Research and development expenses were $10.5 million for the three months ended December 31, 2024 compared to $8.3 million for the three months ended December 31, 2023. The increase in expense of $2.2 million was primarily due to higher clinical trial and manufacturing costs associated with the Phase 2 clinical trials evaluating the use of bempikibart."

Commercial • Alopecia

Q32 Bio Presents Results from SIGNAL-AA Part A Clinical Trial Evaluating Bempikibart in Patients with Alopecia Areata at the 2025 American Academy of Dermatology Meeting (PRNewswire) - P2a | N=40 | SIGNAL-AA (NCT06018428) | Sponsor: Q32 Bio Inc. | "Results presented in AAD late-breaker demonstrate bempikibart's encouraging improvement on SALT reduction at week 24 and continued effects after dosing cessation in patients with severe and very severe alopecia areata (AA); Durable, ongoing responses in multiple patients through week 36 follow-up period and beyond to week 55, despite only 24 weeks of dosing; In the Phase 2a clinical trial, bempikibart was observed to be safe and well-tolerated, with PK supporting subcutaneous dosing and receptor occupancy data demonstrating desired target engagement; clinical biomarkers showed changes in Th2 biomarkers, and expected on-mechanism changes in T-cells, indicative of potent IL-7 and TSLP inhibition; Bempikibart development program remains on track with open-label extension study to initiate in 1H'25; SIGNAL-AA Part B remains on track for initiation of dosing in 1H'25, with topline data expected in 1H'26."

P2a data • Trial status • Alopecia • Immunology

Initial Results From The SIGNAL-AA Study: Randomized Placebo Controlled Phase 2a Trial of a Bempikibart, Novel IL-7/TSLP Bifunctional Receptor Antagonist in Patients with Severe or Very Severe... (AAD 2025) - No abstract available

Clinical • Late-breaking abstract • P2a data • IL7 • TSLP

Q32 Bio Announces Late-Breaking Presentation of Results from SIGNAL-AA Part A Clinical Trial of Bempikibart in Alopecia Areata at the 2025 American Academy of Dermatology Annual Meeting (PRNewswire) - "Q32 Bio Inc...today announced that results from Part A of its SIGNAL-AA Phase 2a clinical trial of bempikibart in patients with alopecia areata (AA) will be presented in a late-breaking oral session at the 2025 American Academy of Dermatology (AAD) Annual Meeting, taking place March 7-11 in Orlando, FL. Bempikibart is a fully human anti-IL-7Rα antibody that re-regulates adaptive immune function by blocking IL-7 and TSLP signaling for the treatment of AA currently being evaluated in a Phase 2 program."

Late-breaking abstract • P2a data • Alopecia

Bempikibart Phase 2 Development Program (PRNewswire) - P2a | N=40 | SIGNAL-AA (NCT06018428) | Sponsor: Q32 Bio Inc. | "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....Q32 Bio expects to initiate dosing in SIGNAL-AA Part B in the first half of 2025....Q32 Bio expects to report initial data from SIGNAL-AA Part B in the first half of 2026."

P2a data • Trial status • Alopecia • Immunology

Q32 Bio Provides Bempikibart Program Update, Including Next Steps for Advancing Alopecia Areata Development Program (Q32 Bio) - P2a | N=102 | SIGNAL-AD (NCT05509023) | P2a | N=40 | SIGNAL-AA (NCT06018428) | Sponsor: Q32 Bio Inc. | "At week 24: patients treated with bempikibart showed a mean reduction in SALT score of 16% in the bempikibart group vs a reduction of 2% in the placebo group. A Wilcoxon Rank Sum test yielded a p-value of 0.045....In Part A, at week 14, improvement in average EASI score from baseline was 58% in patients treated with 2mg/kg Q2W SC and 84% in patients treated at 3mg/kg Q2W SC, and 72% on a pooled basis, compared to 38% in patients treated with placebo.... At week 14, data from Part B demonstrated that patients treated with bempikibart showed a 74% improvement in average EASI from baseline, compared to 76% for the placebo group (p= not statistically significant). Results of the primary endpoint were generally consistent when stratified for pre-specified baseline entry criteria. Bempikibart was observed to be safe and well tolerated in the SIGNAL-AD trial."

P2a data • Alopecia • Atopic Dermatitis • Immunology

Q32 Bio Reports Third Quarter 2024 Financial Results and Provides Corporate Update (PRNewswire) - "Research and development expenses were $14.3 million for the three months ended September 30, 2024, compared to $7.5 million for the three months ended September 30, 2023. The increase in expense of $6.8 million was primarily due to higher clinical trial and manufacturing costs associated with the Phase 2 clinical trials evaluating the use of bempikibart to treat AA and AD and included a development milestone payment of $4.0 million to Bristol-Myers Squibb under our license agreement."

Commercial • Alopecia • Atopic Dermatitis • Immunology

Evaluating Safety and Efficacy of ADX-914 in Patients With Moderate to Severe Atopic Dermatitis (SIGNAL-AD) (clinicaltrials.gov) - P2 | N=102 | Active, not recruiting | Sponsor: Q32 Bio Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Atopic Dermatitis • Dermatitis • Dermatology • Immunology

Q32 Bio Announces Completion of Enrollment in the SIGNAL-AD Phase 2 Clinical Trial of Bempikibart for Atopic Dermatitis (PRNewswire) - "Q32 Bio...announced that it has completed enrollment in the SIGNAL-AD Phase 2 clinical trial of bempikibart (ADX-914) for the treatment of persistent, moderate-to-severe atopic dermatitis (AD)....SIGNAL-AD (NCT05509023) is a two-part Phase 2, randomized, double-blind, placebo-controlled, multi-center clinical trial evaluating bempikibart in adult patients with persistent, moderate-to-severe AD....A total of 121 patients were enrolled, including 15 patients in Part A. Total enrollment exceeded the initial target of approximately 100 patients due to Part B patient enrollment demand. Topline data from Parts A and B are expected in the fourth quarter of 2024."

Enrollment closed • P2 data • Atopic Dermatitis

Bempikibart: Data from P2 SIGNAL-AD trial (NCT05509023) for atopic dermatitis in Q4 2024 (Q32 Bio) - Corporate Presentation

P2 data • Atopic Dermatitis • Dermatology

Q32 Bio Reports First Quarter 2024 Financial Results and Provides Corporate Update (PRNewswire) - "Recent Business Highlights...Enrollment was completed in the ongoing bempikibart Phase 2 clinical trial in AA, with topline results expected in the fourth quarter of 2024...The Phase 2 clinical trial is ongoing in approximately 40 patients with severe AA treated over 24 weeks...Enrollment remains on-track in the bempikibart Phase 2 clinical trial in AD, with topline results expected in the fourth quarter of 2024. The Phase 2 clinical trial in AD is approximately 100 patients and consists of two parts....Research and development expenses were $9.8 million for the three months ended March 31, 2024, compared to $7.9 million for the three months ended March 31, 2023. The increase in expense of $1.9 million was primarily due to higher clinical trial and manufacturing costs associated with the Phase 2 clinical trials evaluating the use of bempikibart to treat AA and AD."

Commercial • Enrollment closed • P2 data • Alopecia • Atopic Dermatitis • Dermatitis • Dermatology • Immunology

SIGNAL-AA: Randomized, Double-Blind, Placebo-Controlled Phase 2a, Proof-of-Concept Trial of ADX-914 Phase 2a Trial for the Treatment of Severe Alopecia Areata (clinicaltrials.gov) - P2 | N=40 | Active, not recruiting | Sponsor: Q32 Bio Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Alopecia • Dermatology • Immunology

SIGNAL-AA: Randomized, Double-Blind, Placebo-Controlled Phase 2a, Proof-of-Concept Trial of ADX-914 Phase 2a Trial for the Treatment of Severe Alopecia Areata (clinicaltrials.gov) - P2 | N=40 | Recruiting | Sponsor: Q32 Bio Inc. | Trial completion date: Sep 2024 ➔ Dec 2024 | Trial primary completion date: Jun 2024 ➔ Dec 2024

Trial completion date • Trial primary completion date • Alopecia • Dermatology • Immunology

Evaluating Safety and Efficacy of ADX-914 in Patients With Moderate to Severe Atopic Dermatitis (SIGNAL-AD) (clinicaltrials.gov) - P2 | N=102 | Recruiting | Sponsor: Q32 Bio Inc. | Trial completion date: May 2024 ➔ Dec 2024 | Trial primary completion date: Feb 2024 ➔ Dec 2024

Trial completion date • Trial primary completion date • Atopic Dermatitis • Dermatitis • Dermatology • Immunology