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

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

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 • Trial primary completion date • Alopecia • 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. | Phase classification: P2a ➔ P2

Phase classification • Alopecia • 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. | Phase classification: P2a ➔ P2

Phase classification • Atopic Dermatitis • Dermatitis • Dermatology • Immunology

Q32 Bio Regains Worldwide Rights to Bempikibart (ADX-914) from Amgen (PRNewswire) - "Q32 Bio...today announced that it will regain full development and commercial rights to bempikibart (previously known as ADX-914) from Amgen....All data from the Phase 2 trials remain blinded and Q32 Bio remains on track to report topline Phase 2 results in the second half of 2024....Under the terms of the re-acquisition agreement with Amgen, Q32 Bio retains the initial consideration and all development funding received. Q32 Bio will pay Amgen a milestone upon the first regulatory approval and subsequent downstream commercial milestones based on annual net sales thresholds."

Licensing / partnership • P2 data • Alopecia • Atopic Dermatitis • Immunology

Q32 Bio and Horizon Therapeutics plc Announce Dosing of First Patient in Phase 2 Trial of Bempikibart (formerly ADX-914) for Severe Alopecia Areata (Businesswire) - "Q32 Bio...and Horizon Therapeutics plc...today announced that the first patient has been dosed in a randomized, double-blind, placebo-controlled, Phase 2 multicenter, proof-of-concept study to evaluate bempikibart in adult patients with severe alopecia areata."

Trial status • Alopecia • 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) - P2a | N=40 | Recruiting | Sponsor: Q32 Bio Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Alopecia

SIGNAL-AA: Randomized, Double-Blind, Placebo-Controlled Phase 2a, Proof-of-Concept Trial of ADX-Phase 2a Trial for the Treatment of Severe Alopecia Areata (clinicaltrials.gov) - P2a | N=40 | Not yet recruiting | Sponsor: Q32 Bio Inc.

New P2a trial • Alopecia