Q32 Bio Announces Strategic Restructuring to Focus on Advancement of Bempikibart in Patients with Alopecia Areata
Q32 Bio (NASDAQ: QTTB) announced a strategic restructuring to focus on advancing bempikibart for alopecia areata (AA) treatment. The company is discontinuing its Phase 2 renal basket trial of ADX-097 and evaluating strategic options for its complement inhibitor platform. The restructuring, including personnel reductions, is expected to extend cash runway to 2H'26.
Based on encouraging results from SIGNAL-AA Part A trial, showing improvement in SALT scores and continued response through week 55, Q32 Bio plans to initiate an open-label extension in 1H'25. The company will also begin SIGNAL-AA Part B dosing in 1H'25, with topline data expected in 1H'26. Part B will evaluate approximately 20 patients with severe AA over 36 weeks of treatment.
Q32 Bio (NASDAQ: QTTB) ha annunciato una ristrutturazione strategica per concentrarsi sull'avanzamento del bempikibart per il trattamento dell'alopecia areata (AA). L'azienda sta interrompendo il suo trial di Fase 2 per il trattamento renale di ADX-097 e sta valutando opzioni strategiche per la sua piattaforma di inibitori del complemento. La ristrutturazione, che include riduzioni del personale, dovrebbe estendere il periodo di liquidità fino al secondo semestre del '26.
Sulla base di risultati incoraggianti dal trial SIGNAL-AA Parte A, che mostrano un miglioramento nei punteggi SALT e una risposta continua fino alla settimana 55, Q32 Bio prevede di avviare un'estensione in aperto nella prima metà del '25. L'azienda inizierà anche la somministrazione della Parte B di SIGNAL-AA nella prima metà del '25, con dati preliminari attesi nella prima metà del '26. La Parte B valuterà circa 20 pazienti con AA severa per un trattamento della durata di 36 settimane.
Q32 Bio (NASDAQ: QTTB) anunció una reestructuración estratégica para centrarse en el avance del bempikibart para el tratamiento de la alopecia areata (AA). La empresa está descontinuando su ensayo de fase 2 para el tratamiento renal de ADX-097 y está evaluando opciones estratégicas para su plataforma de inhibidores del complemento. Se espera que la reestructuración, que incluye reducciones de personal, extienda el período de liquidez hasta la segunda mitad de '26.
Basado en resultados alentadores del ensayo SIGNAL-AA Parte A, que muestra una mejora en los puntajes SALT y una respuesta continua hasta la semana 55, Q32 Bio planea iniciar una extensión abierta en la primera mitad de '25. La empresa también comenzará la dosificación de la Parte B de SIGNAL-AA en la primera mitad de '25, con datos preliminares esperados en la primera mitad de '26. La Parte B evaluará aproximadamente 20 pacientes con AA severa durante 36 semanas de tratamiento.
Q32 Bio (NASDAQ: QTTB)는 탈모증(AA) 치료를 위한 벰피키바트의 개발에 집중하기 위해 전략적 구조조정을 발표했습니다. 이 회사는 ADX-097에 대한 2상 신장 임상 시험을 중단하고 보완제 억제제 플랫폼에 대한 전략적 옵션을 평가하고 있습니다. 인력 감축을 포함한 이 구조조정은 '26년 하반기까지 자금 운용 기간을 연장할 것으로 예상됩니다.
SIGNAL-AA A 파트의 고무적인 결과를 바탕으로 SALT 점수가 개선되고 55주차까지 지속적인 반응을 보인 Q32 Bio는 '25년 상반기에 개방형 연장을 시작할 계획입니다. 이 회사는 또한 '25년 상반기에는 SIGNAL-AA B 파트의 투여를 시작할 예정이며, '26년 상반기에는 topline 데이터가 나올 것으로 예상됩니다. B 파트는 심각한 AA 환자 20명을 36주 동안 평가할 것입니다.
Q32 Bio (NASDAQ: QTTB) a annoncé une restructuration stratégique visant à se concentrer sur l'avancement du bempikibart pour le traitement de l'alopecia areata (AA). L'entreprise interrompt son essai clinique de Phase 2 concernant l'ADX-097 et évalue des options stratégiques pour sa plateforme d'inhibiteurs du complément. Cette restructuration, qui inclut des réductions de personnel, devrait prolonger la période de liquidité jusqu'au deuxième semestre de '26.
Sur la base des résultats encourageants de l'essai SIGNAL-AA Partie A, montrant une amélioration des scores SALT et une réponse continue jusqu'à la semaine 55, Q32 Bio prévoit de lancer une extension ouverte au cours de la première moitié de '25. L'entreprise commencera également la administration de la Partie B de SIGNAL-AA au cours de la première moitié de '25, avec des données préliminaires attendues pour la première moitié de '26. La Partie B évaluera environ 20 patients souffrant d'AA sévère pendant 36 semaines de traitement.
Q32 Bio (NASDAQ: QTTB) hat eine strategische Umstrukturierung angekündigt, um sich auf die Weiterentwicklung von Bempikibart zur Behandlung von Alopecia areata (AA) zu konzentrieren. Das Unternehmen stellt seine Phase-2-Nierenstudie zu ADX-097 ein und überprüft strategische Optionen für seine Plattform von Komplementinhibitoren. Es wird erwartet, dass die Umstrukturierung, einschließlich Personalabbau, die finanzielle Laufzeit bis zur zweiten Hälfte von '26 verlängert.
Basierend auf ermutigenden Ergebnissen der SIGNAL-AA Teil A-Studie, die eine Verbesserung der SALT-Werte und eine anhaltende Reaktion bis zur Woche 55 zeigt, plant Q32 Bio, in der ersten Hälfte von '25 eine offene Verlängerung zu initiieren. Das Unternehmen wird auch in der ersten Hälfte von '25 mit der Dosierung der SIGNAL-AA Teil B beginnen, wobei die Hauptdaten in der ersten Hälfte von '26 erwartet werden. Teil B wird ungefähr 20 Patienten mit schwerem AA über 36 Wochen behandeln.
- Extended cash runway to second half of 2026
- Positive clinical data from SIGNAL-AA Part A with continued patient response through week 55
- Strong patient re-consent rates indicating treatment demand
- Well-tolerated safety profile for bempikibart
- Discontinuation of Phase 2 renal basket trial for ADX-097
- Workforce reduction as part of restructuring
- Potential loss of value from complement inhibitor platform assets
Insights
Q32 Bio's strategic pivot represents a calculated but significant transformation of their business model. The restructuring decision to focus on bempikibart while discontinuing the ADX-097 renal program marks a important inflection point for the company.
The emerging data from SIGNAL-AA Part A demonstrates several compelling advantages for bempikibart:
- Sustained SALT score improvements through week 55 (7 months post-dosing)
- Potential remittive effect and durability of response, differentiating it from current therapies
- Strong safety profile with robust pharmacologic activity
- High patient re-consent rates indicating treatment satisfaction
The extension of cash runway to 2H'26 through cost-cutting provides adequate financial cushion to reach multiple value-inflection points, including the critical SIGNAL-AA Part B topline data in 1H'26. However, the discontinuation of the ADX-097 program, while fiscally prudent, eliminates near-term diversification and increases dependency on bempikibart's success.
The alopecia areata market represents a significant commercial opportunity, with current treatments having limitations in durability and remission. Bempikibart's differentiated profile, particularly its potential remittive effect, could position it favorably against existing therapies if the Phase 2 data continues to be positive.
This strategic realignment essentially transforms Q32 from a multi-platform company to a more focused entity betting primarily on bempikibart's success. While this increases concentration risk, it also allows for more efficient capital allocation and clearer value drivers for investors to evaluate.
-- 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 --
"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
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.
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 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
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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