Q32 Bio Provides Bempikibart Program Update, Including Next Steps for Advancing Alopecia Areata Development Program
Q32 Bio announced topline results from two Phase 2a clinical trials for bempikibart. The SIGNAL-AA trial in alopecia areata showed encouraging results, with patients experiencing a 16% mean reduction in SALT score versus 2% for placebo at week 24. Additionally, 13% of bempikibart patients achieved SALT-20 by week 26 compared to 0% for placebo.
However, the SIGNAL-AD trial in atopic dermatitis did not meet its primary endpoint in Part B, despite promising findings in Part A. The drug demonstrated safety and tolerability across both trials, with significant biomarker evidence showing potent IL-7 and TSLP inhibition. Based on these results, Q32 Bio plans to advance bempikibart specifically for alopecia areata treatment, expanding the SIGNAL-AA trial with approximately 20 additional patients.
Q32 Bio ha annunciato i risultati preliminari di due studi clinici di fase 2a per bempikibart. Il trial SIGNAL-AA sull'alopecia areata ha mostrato risultati promettenti, con i pazienti che hanno registrato una riduzione media del punteggio SALT del 16% rispetto al 2% del placebo alla settimana 24. Inoltre, il 13% dei pazienti trattati con bempikibart ha raggiunto il SALT-20 entro la settimana 26, rispetto allo 0% del placebo.
Tuttavia, il trial SIGNAL-AD per la dermatite atopica non ha raggiunto il suo obiettivo primario nella Parte B, nonostante i risultati promettenti nella Parte A. Il farmaco ha dimostrato sicurezza e tollerabilità in entrambi gli studi, con evidenze significative di biomarker che mostrano una potente inibizione di IL-7 e TSLP. Sulla base di questi risultati, Q32 Bio prevede di sviluppare bempikibart specificamente per il trattamento dell'alopecia areata, ampliando il trial SIGNAL-AA con circa 20 pazienti aggiuntivi.
Q32 Bio anunció los resultados preliminares de dos ensayos clínicos de fase 2a para bempikibart. El ensayo SIGNAL-AA en alopecia areata mostró resultados alentadores, con los pacientes experimentando una reducción media del 16% en el puntaje SALT frente al 2% del placebo en la semana 24. Además, el 13% de los pacientes tratados con bempikibart alcanzaron SALT-20 en la semana 26 en comparación con el 0% del placebo.
No obstante, el ensayo SIGNAL-AD en dermatitis atópica no alcanzó su objetivo principal en la Parte B, a pesar de los hallazgos prometedores en la Parte A. El fármaco demostró seguridad y tolerabilidad en ambos ensayos, con evidencia significativa de biomarcadores que muestran una potente inhibición de IL-7 y TSLP. Basado en estos resultados, Q32 Bio planea avanzar con bempikibart específicamente para el tratamiento de la alopecia areata, ampliando el ensayo SIGNAL-AA con aproximadamente 20 pacientes adicionales.
Q32 Bio는 bempikibart에 대한 두 개의 2a 단계 임상 시험에서 topline 결과를 발표했습니다. SIGNAL-AA 시험은 원형 탈모증에 대한 것으로, 환자가 24주차에 플라시보 대비 SALT 점수에서 평균 16% 감소를 경험하여 고무적인 결과를 보였습니다. 또한, bempikibart 치료를 받은 환자의 13%가 26주차에 SALT-20에 도달했으며, 이는 플라시보의 0%에 해당합니다.
그러나 SIGNAL-AD 시험은 아토피성 피부염에 대해 B파트에서 주요 목표를 달성하지 못했으며, A파트에서는 유망한 결과를 보였습니다. 이 약물은 두 번의 시험에서 모두 안전성과 내약성을 보여주었고, IL-7과 TSLP의 강력한 억제를 보여주는 중요한 바이오마커가 발견되었습니다. 이러한 결과를 바탕으로 Q32 Bio는 alopecia areata 치료를 위해 bempikibart를 개발할 계획이며, SIGNAL-AA 시험을 약 20명의 추가 환자로 확장할 예정입니다.
Q32 Bio a annoncé les résultats préliminaires de deux essais cliniques de phase 2a pour bempikibart. L'
Cependant, l'
Q32 Bio gab die vorläufigen Ergebnisse aus zwei Phase 2a klinischen Studien zu bempikibart bekannt. Die SIGNAL-AA-Studie bei Alopecia areata zeigte ermutigende Ergebnisse, wobei die Patienten eine durchschnittliche Reduzierung des SALT-Scores um 16% im Vergleich zu 2% für das Placebo in Woche 24 erfuhren. Darüber hinaus erreichten 13% der bempikibart-Patienten bis Woche 26 SALT-20 im Vergleich zu 0% für das Placebo.
Das SIGNAL-AD-Studie zur atopischen Dermatitis erreichte jedoch nicht das primäre Ziel in Teil B, trotz vielversprechender Ergebnisse in Teil A. Das Medikament zeigte Sicherheit und Verträglichkeit über beide Studien hinweg, mit erheblichen Biomarker-Nachweisen, die eine starke Hemmung von IL-7 und TSLP belegen. Basierend auf diesen Ergebnissen plant Q32 Bio, bempikibart speziell für die Behandlung von Alopecia areata weiterzuentwickeln und die SIGNAL-AA-Studie um etwa 20 zusätzliche Patienten zu erweitern.
- SIGNAL-AA trial showed 16% reduction in SALT score vs 2% for placebo
- 13% of patients achieved SALT-20 response by week 26 vs 0% for placebo
- Drug demonstrated safety with no serious adverse events
- Strong biomarker data showing target engagement
- SIGNAL-AD trial failed to meet primary endpoint in Part B
- Company deferring planned Phase 2 trial of ADX-097 in ANCA-Associated Vasculitis
- One trial site excluded from efficacy analysis due to protocol violations
Insights
The SIGNAL-AA trial shows encouraging but mixed results for bempikibart in alopecia areata treatment. The
However, the SIGNAL-AD trial results in atopic dermatitis were disappointing, with the drug showing no significant advantage over placebo (
The biomarker data showing
Q32 Bio's strategic pivot to focus on alopecia areata while deferring other programs reflects a pragmatic approach to resource allocation. The global alopecia treatment market presents a significant opportunity, with current options and safety concerns around existing treatments. The company's
However, the failure in atopic dermatitis - a larger market with established competitors - may limit the drug's commercial potential. The decision to defer the AAV program also indicates financial constraints. Investors should note that while the AA results are encouraging, the modest efficacy seen so far may require enhanced dosing strategies or longer treatment duration to achieve competitive clinical benefits.
-- SIGNAL-AA demonstrated encouraging clinical activity of bempikibart in patients with alopecia areata (AA), including improvement from baseline on SALT score and meaningful achievement of SALT-20 response --
-- SIGNAL-AD Phase 2a clinical trial in atopic dermatitis demonstrated promising findings in Part A but did not meet primary endpoint in Part B --
-- Across both trials, bempikibart was observed to be safe and well tolerated; demonstrated potent IL-7 and TSLP inhibition via changes in both Th2 biomarkers and T-cells, and desired target engagement --
-- Based on these results, Company plans to advance bempikibart in patients with AA --
"We are pleased with the emerging signals observed in the SIGNAL-AA Phase 2a clinical trial and based upon these, the positive biomarker data and well tolerated safety profile observed across both trials, we plan to enroll additional patients into the SIGNAL-AA clinical trial to further explore the clinical effects of bempikibart in this patient population. We believe bempikibart has the potential to be an important new treatment option in a disease needing new and safer alternatives to currently approved agents," said Jodie Morrison, Chief Executive Officer of Q32 Bio. "We are disappointed that the SIGNAL-AD trial did not achieve its primary endpoint. Based upon the findings, including the high placebo rate, we plan to conduct a review to better understand the results."
"Results from our analysis of SIGNAL-AA showed clinically meaningful activity and a safety profile that we believe is differentiated from the currently approved therapies for AA. We are encouraged by our findings from this clinical trial, and we look forward to advancing bempikibart as a potential treatment for AA," said Jason Campagna, M.D., Ph.D., Chief Medical Officer of Q32 Bio. "On behalf of Q32 Bio, I want to express my gratitude to the patients, their caregivers, and clinical trial sites that participated across both our bempikibart Phase 2a trials."
The Company is also providing an update on the SIGNAL-AD clinical trial in patients with atopic dermatitis (AD). Although the Company is reporting promising findings in Part A, the trial did not meet its primary endpoint in Part B. Q32 Bio plans to conduct a review of the results.
Topline Results from SIGNAL-AA Phase 2a Clinical Trial:
SIGNAL-AA is a Phase 2a, randomized, double-blind, placebo-controlled, multi-center clinical trial evaluating bempikibart in adult patients with severe and very severe AA (baseline Severity of Alopecia Tool (SALT) scores of 50-100) treated over 24 weeks, with follow-up through 36 weeks. The study is being conducted to evaluate the efficacy and safety of bempikibart 200 mg administered subcutaneously (SC), every-other-week (Q2W) compared to placebo. A total of 44 patients were enrolled in the trial with a primary endpoint of the mean relative percent change in SALT score at 24 weeks compared with baseline.
Following database lock, one site was excluded from the efficacy analysis based on marked protocol violations of entry criteria. This resulted in the removal of three placebo patients, rendering the planned statistical analyses for the primary endpoint inappropriate due to the reduced sample size. On a post-hoc analysis of the remaining per-protocol population of patients with AA (n=27), bempikibart demonstrated an improvement in hair re-growth compared to placebo:
- At week 24: patients treated with bempikibart showed a mean reduction in SALT score of
16% in the bempikibart group vs a reduction of2% in the placebo group. A Wilcoxon Rank Sum test yielded a p-value of 0.045. - At week 24:
9% of bempikibart patients in the trial achieved a SALT-20 (SALT score less than or equal to 20) compared to0% in placebo. - At week 26:
13% of bempikibart patients achieved SALT-20 compared0% in placebo.
Bempikibart was observed to be safe and well tolerated in the SIGNAL-AA trial. There were no serious adverse events (SAE) or Grade 3 or higher adverse events related to treatment.
"Advancing bempikibart in AA is supported by preclinical data demonstrating the potential of IL-7Rα inhibition in this disease, and now the resulting data from SIGNAL-AA demonstrated the clinical potential of an IL-7Rα inhibitor in AA. I am encouraged by the biomarker data that provide evidence of biological activity, the safety profile of bempikibart, and the clinical signal of hair regrowth in patients," said Brett King, M.D., Ph.D., of Dermatology Physicians of
Q32 Bio plans to enroll approximately 20 additional patients in a Part B expansion of the SIGNAL-AA Phase 2a clinical trial to further evaluate bempikibart in AA, including a loading regimen. The Company will defer enrollment into the planned Phase 2 clinical trial of ADX-097 in ANCA-Associated Vasculitis (AAV), previously expected to begin in 2025, to focus efforts on continued enrollment in the ongoing bempikibart AA and ADX-097 renal basket Phase 2 clinical trials.
Topline Results from SIGNAL-AD Phase 2a Clinical Trial:
SIGNAL-AD is a two-part Phase 2a, randomized, double-blind, placebo-controlled, multi-center clinical trial evaluating bempikibart in adult patients with persistent, moderate-to-severe AD. Part A (n=15) was conducted to evaluate safety, pharmacokinetics (PK), and to enable dose selection for Part B of the clinical trial. Part A was randomized 2:1 between bempikibart and placebo in each of two dose cohorts of 2mg/kg or 3mg/kg Q2W SC for 12 weeks.
In Part A, at week 14, improvement in average EASI score from baseline was
In Part B, which evaluated both efficacy and safety of bempikibart compared to placebo, patients were enrolled 1:1 in the bempikibart 200 mg Q2W SC (n=52) and placebo (n=54) arms for 12 weeks of treatment. The primary endpoint is the mean percent change from baseline to week 14 in the Eczema Area and Severity Index (EASI) score. At week 14, data from Part B demonstrated that patients treated with bempikibart showed a
Biomarker Results in SIGNAL-AD and SIGNAL-AA:
Across SIGNAL-AD and SIGNAL-AA, bempikibart at 200mg Q2W SC demonstrated favorable PK and target engagement as demonstrated by substantial reductions in biomarkers of Th2 and Th1. These results include:
- A reduction in Th2 biomarkers, including TARC, IgE and eosinophils, which was consistent with the type of biomarker impact previously observed with other agents that have demonstrated utility in atopic dermatitis, such as IL-4Rα, IL-13 and OX40 ligand-targeted agents.
- An expected modulation of T-cells, with a 20
-30% reduction, consistent with target engagement and IL-7Rα blockade.
Q32 Bio believes these results demonstrate that bempikibart is a potent inhibitor of both TSLP and IL-7 signaling as evidenced by robust changes in both Th2 biomarkers and T-cells. The Company believes the mechanism of action of bempikibart has the potential to be active in other Th2 and Th1 driven diseases, including asthma, COPD, ulcerative colitis (UC), rheumatoid arthritis (RA), celiac disease, multiple sclerosis (MS) and others.
"These impressive biomarker data represent a meaningful advancement in the clinical understanding of how inhibition of IL-7Rα can be leveraged to treat autoimmune and inflammatory diseases," said Shelia Violette, Ph.D., Co-Founder and Chief Scientific Officer of Q32 Bio. "Based upon its observed mechanism of action, bempikibart continues to show strong potential as an IL-7Rα inhibitor to treat AA and other diseases."
The Company has published an updated investor presentation with additional details regarding the bempikibart update for review by interested parties. The updated presentation can be found on the company website at www.Q32Bio.com under Investors & Media.
About Q32 Bio
Q32 Bio is a clinical stage biotechnology company developing biologic therapeutics targeting potent regulators of the innate and adaptive immune systems to re-balance immunity in autoimmune and inflammatory diseases. Q32 Bio's lead programs, focused on the IL-7 / TSLP receptor pathways and complement system, address immune dysregulation to help patients take back control of their lives.
Q32 Bio's program for adaptive immunity, bempikibart (ADX-914), is 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. Q32 Bio's program for innate immunity, ADX-097, being evaluated in a Phase 2 program, is based on a novel platform enabling tissue-targeted regulation of the complement system without long-term systemic blockade – a key differentiator versus current complement therapeutics.
For more information, visit www.Q32Bio.com.
Availability of Other Information About Q32 Bio
Investors and others should note that we communicate with our investors and the public using our company website www.Q32Bio.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly Twitter) and LinkedIn. The information that we post on our website or on X or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
Forward-Looking Statements
This communication contains forward-looking statements within the meaning of the
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 the Part B of the SIGNAL-AA Phase 2a clinical trial, may not be completed in a timely manner or at all, might be more costly than expected or might not yield anticipated results, the Company may need additional funding to complete its 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
Contacts:
Investors: Brendan Burns
Media: Sarah Sutton
Argot Partners
212.600.1902
Q32Bio@argotpartners.com
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SOURCE Q32 Bio
FAQ
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