Q32 Bio to Present Tissue-Targeted Complement Inhibitor ADX-097 Positive Phase 1 Clinical Trial Results at the American Society of Nephrology Kidney Week 2024
Q32 Bio Inc. (Nasdaq: QTTB) announced it will present clinical data for ADX-097, its lead product candidate from its tissue-targeted complement inhibitor platform, at the American Society of Nephrology (ASN) Kidney Week 2024. The presentation will highlight data from the completed first-in-human, Phase 1 ascending dose clinical trial of ADX-097 in healthy volunteers.
Key points:
- ADX-097 is a humanized anti-C3d mAb fusion protein being evaluated for renal diseases associated with increased complement activation.
- The Phase 1 trial showed ADX-097 to be generally well-tolerated with desirable pharmacokinetics and pharmacodynamics properties.
- Results support Phase 2 dose selection and continued clinical advancement of ADX-097.
- Q32 Bio aims to achieve relevant complement inhibition directly in the tissue while minimizing systemic activity.
- The company is currently advancing ADX-097 in two Phase 2 clinical trials.
Q32 Bio Inc. (Nasdaq: QTTB) ha annunciato che presenterà dati clinici per ADX-097, il suo principale candidato farmaceutico proveniente dalla piattaforma di inibitore del complemento mirato ai tessuti, alla American Society of Nephrology (ASN) Kidney Week 2024. La presentazione evidenzierà i dati della sperimentazione clinica di fase 1, in dose ascendente, completata su soggetti sani.
Punti chiave:
- ADX-097 è una proteina di fusione anticorpale umanizzata anti-C3d in fase di valutazione per le malattie renali associate a un aumentato attivazione del complemento.
- La sperimentazione di fase 1 ha mostrato che ADX-097 è generalmente ben tollerato con proprietà farmacocinetiche e farmacodinamiche desiderabili.
- I risultati supportano la selezione della dose per la fase 2 e il continuo avanzamento clinico di ADX-097.
- Q32 Bio mira a ottenere un'inibizione del complemento rilevante direttamente nel tessuto, riducendo al minimo l'attività sistemica.
- L'azienda attualmente sta portando avanti ADX-097 in due sperimentazioni cliniche di fase 2.
Q32 Bio Inc. (Nasdaq: QTTB) anunció que presentará datos clínicos de ADX-097, su principal candidato a producto de su plataforma de inhibidores del complemento dirigidos a tejidos, en la American Society of Nephrology (ASN) Kidney Week 2024. La presentación destacará datos del ensayo clínico de fase 1, en dosis ascendentes, completado en voluntarios sanos.
Puntos clave:
- ADX-097 es una proteína de fusión anti-C3d humanizada que se está evaluando para enfermedades renales asociadas con una mayor activación del complemento.
- El ensayo de fase 1 mostró que ADX-097 se tolera generalmente bien con propiedades farmacocinéticas y farmacodinámicas deseables.
- Los resultados respaldan la selección de dosis para la fase 2 y el avance clínico continuo de ADX-097.
- Q32 Bio tiene como objetivo lograr una inhibición del complemento relevante directamente en el tejido, minimizando la actividad sistémica.
- La empresa está avanzando actualmente con ADX-097 en dos ensayos clínicos de fase 2.
Q32 Bio Inc. (Nasdaq: QTTB)는 조직 표적 보체 억제 플랫폼의 주요 제품 후보인 ADX-097에 대한 임상 데이터를 American Society of Nephrology (ASN) Kidney Week 2024에서 발표할 것이라고 발표했습니다. 발표는 건강한 자원 봉사자를 대상으로 한 최초의 인체 단계를 포함한 1상 상승 용량 임상 시험의 데이터를 강조할 것입니다.
주요 사항:
- ADX-097는 증가된 보체 활성화와 연관된 신장 질환을 평가 중인 인간화된 항-C3d 단일클론 항체 융합 단백질입니다.
- 1상 시험은 ADX-097이 일반적으로 잘 견디며 바람직한 약물동태학 및 약리학적 특성을 가지고 있음을 보여주었습니다.
- 결과는 2상 용량 선택과 ADX-097의 지속적인 임상 발전을 지원합니다.
- Q32 Bio는 시스템 활동을 최소화하면서 조직 내에서 직접적으로 관련된 보체 억제를 달성하는 것을 목표로 합니다.
- 회사는 현재 2상 임상 시험에서 ADX-097를 진행하고 있습니다.
Q32 Bio Inc. (Nasdaq: QTTB) a annoncé qu'il présentera des données cliniques sur l'ADX-097, son principal candidat médicament issu de sa plateforme d'inhibiteurs du complément ciblés sur les tissus, lors de la American Society of Nephrology (ASN) Kidney Week 2024. La présentation mettra en avant des données de l'essai clinique de phase 1, à dose ascendante, terminé sur des volontaires en bonne santé.
Points clés :
- ADX-097 est une protéine de fusion anticorps humanisée anti-C3d, en cours d'évaluation pour les maladies rénales associées à une activation accrue du complément.
- L'essai de phase 1 a montré qu'ADX-097 est généralement bien toléré avec des propriétés pharmacocinétiques et pharmacodynamiques souhaitables.
- Les résultats soutiennent la sélection de dose pour la phase 2 et l'avancement clinique continu de l'ADX-097.
- Q32 Bio vise à obtenir une inhibition pertinente du complément directement dans le tissu tout en minimisant l'activité systémique.
- L'entreprise fait actuellement progresser l'ADX-097 dans deux essais cliniques de phase 2.
Q32 Bio Inc. (Nasdaq: QTTB) gab bekannt, dass es klinische Daten für ADX-097, den führenden Produktkandidaten seiner gewebegezielten Komplementinhibitor-Plattform, auf der American Society of Nephrology (ASN) Kidney Week 2024 präsentieren wird. Die Präsentation wird Daten aus der abgeschlossenen ersten menschlichen Phase-1-Studie mit aufsteigenden Dosen von ADX-097 bei gesunden Probanden hervorheben.
Wichtige Punkte:
- ADX-097 ist ein humanisierter anti-C3d mAb-Fusionsprotein, das für Nierenerkrankungen, die mit einer erhöhten Komplementaktivierung verbunden sind, evaluiert wird.
- Die Phase-1-Studie zeigte, dass ADX-097 im Allgemeinen gut vertragen wird und wünschenswerte pharmakokinetische sowie pharmakodynamische Eigenschaften aufweist.
- Die Ergebnisse unterstützen die Dosiswahl der Phase 2 und den fortgesetzten klinischen Fortschritt von ADX-097.
- Q32 Bio zielt darauf ab, relevante Komplementhemmung direkt im Gewebe zu erreichen und gleichzeitig die systemische Aktivität zu minimieren.
- Das Unternehmen führt derzeit ADX-097 in zwei klinischen Phase-2-Studien weiter.
- ADX-097 demonstrated a favorable safety profile in Phase 1 clinical trial
- Desirable pharmacokinetics and pharmacodynamics properties observed in Phase 1 trial
- Results support Phase 2 dose selection and continued clinical advancement
- Potential to achieve targeted complement inhibition while minimizing systemic effects
- Two Phase 2 clinical trials for ADX-097 are currently ongoing
- None.
Insights
The Phase 1 clinical trial results for ADX-097 represent a significant milestone for Q32 Bio. This novel tissue-targeted complement inhibitor shows promise for treating renal diseases associated with increased complement activation. Key takeaways include:
- ADX-097 demonstrated a favorable safety profile and desired pharmacokinetics/pharmacodynamics in healthy volunteers
- The data supports dose selection for Phase 2 trials, a critical step in clinical development
- The drug's mechanism aims to inhibit complement locally in diseased tissue while minimizing systemic effects
- This approach could potentially address limitations of current systemic therapies, including infection risk and dosing challenges
While promising, investors should note that Phase 1 results in healthy volunteers don't guarantee efficacy in patients. The upcoming Phase 2 trials will be important in demonstrating ADX-097's therapeutic potential in renal disease patients. Overall, this news indicates positive progress in Q32 Bio's pipeline, but further clinical validation is needed to assess the drug's market potential.
-- Poster presentation to highlight data from completed first-in-human, Phase 1 ascending dose clinical trial of ADX-097 in healthy volunteers; results support Phase 2 dose selection and continued clinical advancement of ADX-097 --
"We look forward to presenting our promising clinical data at ASN Kidney Week 2024 that supports the continued advancement of ADX-097, our novel tissue-targeted inhibitor of complement activation designed to inhibit complement in the diseased tissue and minimize systemic complement blockade," said Shelia Violette, Ph.D., Chief Scientific Officer of Q32 Bio. "In our Phase 1 clinical trial, ADX-097 was observed to be generally well-tolerated with desirable pharmacokinetics and pharmacodynamics (PK/PD) properties. We believe ADX-097 has the potential to achieve relevant complement inhibition directly in the tissue while sparing systemic activity, with the goal of addressing limitations of the currently available systemic approaches, including infection risk and the need for high drug doses and frequent administration to achieve therapeutic levels of complement inhibition."
"These first-in-human data for ADX-097 that we will share at Kidney Week demonstrate the potential of our novel and differentiated approach to inhibiting the complement system, a validated biologic target," said Jodie Morrison, Chief Executive Officer of Q32 Bio. "We remain focused on advancing our pipeline of programs targeting the immune system for the treatment of autoimmune and inflammatory diseases, including ADX-097 in two Phase 2 clinical trials."
Presentation Details:
Abstract Title: ADX-097, a Tissue-Targeted Complement Inhibitor for the Treatment of Renal Diseases: Phase 1 Results in Healthy Participants and Model-Informed Phase 2 Dose Selection
Presenter: Dr. Bernd Jilma, M.D., Professor, Department of Clinical Pharmacology, Medical University of
Date & Time: Saturday, October 26, 2024, from 10:00 a.m. to 12:00 p.m. PT
Session Type: Poster
Session Title: C3G, TMA, MGRS, Amyloidosis, and More
Abstract ID: 4123429
Summary:
- ADX-097, a C3d mAb – fH fusion protein, was designed to inhibit complement in the diseased kidney while avoiding systemic blockade, providing the potential for an enhanced activity and safety profile. In a Phase 1 ascending dose clinical study of ADX-097 in healthy volunteers, ADX-097 demonstrated a favorable safety profile and desired PK/PD properties, supporting a Phase 2 dose that is predicted to provide tissue inhibition of complement in glomerular diseases while sparing systemic complement activity.
About ADX-097
ADX-097, the lead product candidate from Q32 Bio's tissue-targeted complement inhibitor platform, is a humanized anti-C3d mAb fusion protein. ADX-097 is designed to restore complement regulation—an integral part of the innate immune system—through a novel, tissue-targeted mechanism. Q32 Bio is currently evaluating ADX-097 in an open-label Phase 2 renal basket clinical trial and is planning to evaluate ADX-097 in a Phase 2 clinical trial in ANCA-Associated Vasculitis (AAV).
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. It is being evaluated in two Phase 2 trials for the treatment of atopic dermatitis and alopecia areata. 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, 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. Q32 Bio has completed a first-in-human, Phase 1 ascending dose clinical study of ADX-097 in healthy volunteers.
For more information, visit www.Q32Bio.com.
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Forward-Looking Statements
This communication contains forward-looking statements within the meaning of the
Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," and other similar expressions among others. Statements that are not historical facts are forward-looking statements. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; data generated from our preclinical and clinical studies such as our Phase 2 clinical trials of ADX-097, may not meet our expectations; our product candidates may not provide the intended therapeutic benefits; our product candidates may cause serious adverse side effects; the ability to integrate our business with our merger partner successfully and to achieve anticipated synergies; the possibility that other anticipated benefits of the merger will not be realized, including without limitation, anticipated revenues, expenses, earnings and other financial results, and growth and expansion of our operations, and the anticipated tax treatment of the merger; our ability to retain, attract and hire key personnel; potential adverse reactions or changes to relationships with employees, suppliers or other parties resulting from the completion of the merger; potential business uncertainty, including changes to existing business relationships that could affect our financial performance; the need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the regulatory approval process; the inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties, including for the manufacture of materials for our research programs, preclinical and clinical studies; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; securities class action litigation; the impact of global pandemics and general economic conditions on our business and operations, including the our preclinical studies and clinical trials; the possibility of system failures or security breaches; risks relating to intellectual property; significant costs incurred as a result of operating as a public company; and such other factors as are set forth in Q32 Bio's periodic public filings with the SEC, including but not limited to those described under the heading "Risk Factors" in our Form 10-Q filed with the Securities and Exchange Commission on August 8, 2024 and any subsequent filings made with the Securities and Exchange Commission. Except as required by applicable law, we undertake no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Contacts:
Investors: Brendan Burns
Media: Sarah Sutton
Argot Partners
212.600.1902
Q32Bio@argotpartners.com
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FAQ
What is the purpose of ADX-097 being developed by Q32 Bio (QTTB)?
What were the key findings from the Phase 1 clinical trial of ADX-097 presented at ASN Kidney Week 2024?
How does Q32 Bio (QTTB) plan to advance ADX-097 following the Phase 1 results?
What potential advantages does ADX-097 offer compared to current complement inhibitors?
