100% of Patients Achieve Remission Within 30 Days in Cohort 1 of Bispecific Mipletamig Frontline AML Trial
Aptevo Therapeutics (APVO) announced remarkable results from Cohort 1 of its RAINIER frontline acute myeloid leukemia (AML) Phase 1b trial. All patients achieved remission within 30 days, with two out of three patients reaching complete remission with minimal residual disease (MRD)-negative status, indicating 100% elimination of cancer cells.
The company's bispecific antibody, mipletamig, being tested in combination with venetoclax and azacitidine, demonstrated consistent success across trials, with 86% of patients achieving remission within 30 days of first treatment. Notably, one MRD-negative patient had a TP53 mutation, typically associated with poor prognosis. The drug continues to show favorable safety and tolerability profiles, with Cohort 2 enrollment now beginning.
Aptevo Therapeutics (APVO) ha annunciato risultati straordinari dal Gruppo 1 del suo studio clinico di Fase 1b RAINIER per la leucemia mieloide acuta (AML) in prima linea. Tutti i pazienti hanno raggiunto la remissione entro 30 giorni, con due pazienti su tre che hanno raggiunto la remissione completa con stato negativo alla malattia residua minima (MRD), indicando l'eliminazione del 100% delle cellule tumorali.
L'anticorpo bispecifico dell'azienda, mipletamig, testato in combinazione con venetoclax e azacitidina, ha dimostrato un successo costante nei trial, con l'86% dei pazienti che ha raggiunto la remissione entro 30 giorni dal primo trattamento. È da notare che un paziente MRD-negativo presentava una mutazione TP53, tipicamente associata a una prognosi sfavorevole. Il farmaco continua a mostrare profili di sicurezza e tollerabilità favorevoli, con l'arruolamento per il Gruppo 2 che è ora iniziato.
Aptevo Therapeutics (APVO) anunció resultados notables del Cohorte 1 de su ensayo de Fase 1b RAINIER para la leucemia mieloide aguda (LMA) en línea frontal. Todos los pacientes lograron remisión dentro de los 30 días, con dos de cada tres pacientes alcanzando remisión completa con estado negativo para enfermedad residual mínima (MRD), lo que indica la eliminación del 100% de las células cancerosas.
El anticuerpo bispecífico de la compañía, mipletamig, que se está probando en combinación con venetoclax y azacitidina, demostró un éxito constante en los ensayos, con el 86% de los pacientes alcanzando remisión dentro de los 30 días posteriores al primer tratamiento. Notablemente, un paciente negativo para MRD tenía una mutación TP53, típicamente asociada con un mal pronóstico. El medicamento continúa mostrando perfiles de seguridad y tolerabilidad favorables, y la inscripción para el Cohorte 2 ya ha comenzado.
Aptevo Therapeutics (APVO)는 RAINIER 1상 b 임상시험의 1코호트에서 놀라운 결과를 발표했습니다. 모든 환자가 30일 이내에 완전 관해에 도달했습니다, 세 명 중 두 명이 최소 잔여 질병(MRD) 음성 상태로 완전 관해에 도달했으며, 이는 암세포가 100% 제거되었음을 나타냅니다.
회사의 이중 특이성 항체인 mipletamig는 venetoclax 및 azacitidine과의 병용 투여에서 일관된 성공을 보였으며, 첫 치료 30일 이내에 86%의 환자가 완전 관해를 달성했습니다. 특히, MRD 음성인 한 환자는 일반적으로 나쁜 예후와 연관된 TP53 변이를 보유하고 있었습니다. 이 약물은 계속해서 유리한 안전성과 용인 프로필을 나타내며, 이제 2코호트 등록이 시작되었습니다.
Aptevo Therapeutics (APVO) a annoncé des résultats remarquables pour la Cohorte 1 de son essai clinique de Phase 1b RAINIER sur la leucémie myéloïde aiguë (LMA) en première ligne. Tous les patients ont atteint la rémission dans les 30 jours, avec deux des trois patients atteignant une rémission complète avec un statut négatif pour la maladie résiduelle minimale (MRD), ce qui indique une élimination à 100% des cellules cancéreuses.
L'anticorps bispécifique de la société, mipletamig, testé en combinaison avec le vénotoclax et l'azacitidine, a démontré un succès constant au cours des essais, avec 86% des patients atteignant la rémission dans les 30 jours suivant le premier traitement. Notamment, un patient MRD-négatif avait une mutation TP53, généralement associée à un pronostic défavorable. Le médicament continue de montrer des profils de sécurité et de tolérance favorables, avec le début de l'inscription pour la Cohorte 2.
Aptevo Therapeutics (APVO) hat bemerkenswerte Ergebnisse aus Kohorte 1 seiner Phase 1b-Studie RAINIER zur akuten myeloischen Leukämie (AML) bekannt gegeben. Alle Patienten erreichten innerhalb von 30 Tagen Remission, wobei zwei von drei Patienten eine vollständige Remission mit negativem Status für minimale Restkrankheit (MRD) erreichten, was die 100%ige Eliminierung von Krebszellen anzeigt.
Der bispezifische Antikörper des Unternehmens, mipletamig, der in Kombination mit Venetoclax und Azacitidin getestet wird, zeigte in allen Studien konstanten Erfolg, wobei 86% der Patienten innerhalb von 30 Tagen nach der ersten Behandlung Remission erreichten. Bemerkenswert ist, dass ein MRD-negativer Patient eine TP53-Mutation hatte, die typischerweise mit einer schlechten Prognose assoziiert wird. Das Medikament zeigt weiterhin günstige Sicherheits- und Verträglichkeitsprofile, und die Rekrutierung für Kohorte 2 hat nun begonnen.
- 100% remission rate achieved in Cohort 1 within 30 days
- Two out of three patients reached MRD-negative status (complete cancer cell elimination)
- 86% of patients across trials achieved remission within 30 days of first treatment
- Demonstrated efficacy in difficult-to-treat TP53 mutation cases
- Favorable safety and tolerability profile maintained
- Small patient sample size in Cohort 1 (only three patients)
Insights
The early clinical results for mipletamig in frontline AML treatment are remarkably strong. The 100% remission rate within 30 days in Cohort 1, with two out of three patients achieving MRD-negative status, suggests exceptional efficacy. Particularly notable is the complete response in a patient with TP53 mutation, typically associated with poor outcomes. The consistency of results between this cohort and previous trial data (100% remission in frontline patients) strengthens confidence in mipletamig's therapeutic potential.
The combination with venetoclax and azacitidine appears synergistic, while maintaining a favorable safety profile. The rapid onset of action (86% remission within 30 days across trials) and deep responses (MRD negativity) are clinically meaningful advantages that could position mipletamig as a significant advancement in frontline AML treatment.
These clinical results represent a significant value driver for Aptevo, particularly given the company's small
While early-stage, the robust efficacy signals and clean safety profile reduce development risk. The commencement of Cohort 2 enrollment maintains momentum in the clinical program. Given the strong data package, mipletamig could attract partnership interest from larger pharmaceutical companies, providing potential near-term catalysts.
Two of three patients achieved both complete remission and MRD-negative status
High response rates observed in earlier studies continue in ongoing mipletamig trial
Cohort 2 enrollment commencing
SEATTLE, WA / ACCESSWIRE / December 12, 2024 / Aptevo Therapeutics ("Aptevo") (Nasdaq:APVO), a clinical-stage biotechnology company focused on developing novel bispecific immune-oncology therapeutics based on its proprietary ADAPTIR® and ADAPTIR-FLEX® platform technologies, today announced
"We are very excited by these Cohort 1 results as they add to an already compelling story that highlights mipletamig's potential to elevate the frontline AML treatment paradigm. We now have a
Mipletamig, a CD3 x CD123 bispecific antibody, is being investigated as frontline therapy in combination with venetoclax and azacitidine, a current standard of care for AML. These latest results further reinforce mipletamig's potential as a transformative treatment, supported by impressive efficacy, safety, and tolerability data from two prior clinical trials involving 90 patients.
"Achieving remission in all three Cohort 1 patients is highly encouraging, particularly when viewed alongside prior trial results. Notably, two of these three patients achieved MRD-negative status, a critical outcome indicating that even the most sensitive diagnostic methods detect no remaining cancer cells. This result is strongly associated with longer-lasting remissions and improved survival rates," said Dirk Huebner, MD, Chief Medical Officer at Aptevo. "What makes these outcomes even more compelling is that one of the MRD-negative patients had a TP53 mutation, a subgroup known for its poor prognosis due to chemotherapy resistance, genetic instability, and overall treatment challenges. This growing body of data underscores mipletamig's potential to address some of the most difficult hurdles in AML treatment, delivering deep and durable responses for patients with the greatest need."
About RAINIER
RAINIER, a frontline AML study, is a Phase 1b/2 dose optimization, multi-center, multi-cohort, open label study of up to 39 patients who are being treated across five dose levels ranging from 9 mcg - 140 mcg in combination with venetoclax and azacitidine (ven/aza). Subjects will be adults aged 18 or older, newly diagnosed with AML who are not eligible for intensive induction chemotherapy. Phase 1b consists of 28-day cycles of treatment in five sequential cohorts. Aptevo has partnered with Prometrika (https://www.prometrika.com/), a premier contract research organization for the trial. RAINIER will be conducted in two parts. First, a Phase 1b dose optimization study in frontline AML patients followed by a Phase 2 study.
About Mipletamig
Aptevo's wholly owned lead proprietary drug candidate, mipletamig, targeting AML, MDS and other leukemias, is differentiated by design to redirect the immune system of the patient to destroy leukemic cells and leukemic stem cells expressing the target antigen CD123, which is a compelling target for AML due to its overexpression on leukemic stem cells and AML blasts. This antibody-like recombinant protein therapeutic is designed to engage both leukemic cells and T cells of the immune system and bring them closely together to trigger the destruction of leukemic cells. Mipletamig is purposefully designed to reduce the likelihood and severity of CRS by use of a unique CD3 derived from CRIS-7 vs. the CD3 used by other competitors. Mipletamig has received orphan drug designation ("orphan status") for AML according to the Orphan Drug Act. Mipletamig has been evaluated in 90 patients over two trials to date. RAINIER, Aptevo's Phase 1b/2 frontline AML program, was initiated in 3Q24.
About Aptevo Therapeutics
Aptevo Therapeutics Inc. (Nasdaq:APVO) is a clinical-stage biotechnology company focused on developing novel bispecific immunotherapies for the treatment of cancer. The company has two clinical candidates. Mipletamig is currently being evaluated in RAINIER, a Phase 1b/2 trial for the treatment of frontline acute myeloid leukemia in combination with standard of care venetoclax + azacitidine. Mipletamig has orphan status for AML according to the Orphan Drug Act. ALG.APV-527, a bispecific conditional 4-1BB agonist that is only active upon simultaneous binding to 4-1BB and 5T4, is being co-developed with Alligator Bioscience and is being evaluated in a Phase 1 clinical trial for the treatment of multiple solid tumor types likely to express 5T4. Aptevo has three pre-clinical candidates with different mechanisms of action designed to target a range of solid tumors. All pipeline candidates were created from two proprietary platforms, ADAPTIR® and ADAPTIR-FLEX®. The Aptevo mission is to improve treatment outcomes and transform the lives of cancer patients. For more information, please visit www.aptevotherapeutics.com.
Safe Harbor Statement
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical fact, including, without limitation, Aptevo's expectations about the activity, efficacy, safety, tolerability and durability of its therapeutic candidates and potential use of any such candidates, including in combination with other drugs, as therapeutics for treatment of disease, its expectations regarding the effectiveness of its ADAPTIR and ADAPTIR-FLEX platforms, statements related to the progress of Aptevo's clinical programs, including statements related to anticipated clinical and regulatory milestones, whether further study of mipletamig in a Phase 1b dose optimization trial focusing on multiple doses of mipletamig in combination with venetoclax + azacitidine on a targeted patient population will continue to show remissions, let alone at a rate of
There are several important factors that could cause Aptevo's actual results to differ materially from those indicated by such forward-looking statements, including a deterioration in Aptevo's business or prospects; further assessment of preliminary or interim data or different results from later clinical trials; adverse events and unanticipated problems, adverse developments in clinical development, including unexpected safety issues observed during a clinical trial; and changes in regulatory, social, macroeconomic and political conditions. For instance, actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the uncertainties inherent in the results of preliminary or interim data and preclinical studies being predictive of the results of later-stage clinical trials, initiation, enrollment and maintenance of patients, and the completion of clinical trials, the availability and timing of data from ongoing clinical trials, the trial design includes combination therapies that may make it difficult to accurately ascertain the benefits of mipletamig, expectations for the timing and steps required in the regulatory review process, expectations for regulatory approvals, the impact of competitive products, our ability to enter into agreements with strategic partners or raise funds on acceptable terms or at all and other matters that could affect the availability or commercial potential of Aptevo's product candidates, business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises such as the coronavirus (referred to as COVID-19), geopolitical risks, including the current war between Russia and Ukraine, war between Israel and Hamas, and macroeconomic conditions such as economic uncertainty, rising inflation and interest rates, continued market volatility and decreased consumer confidence. These risks are not exhaustive, Aptevo faces known and unknown risks. Additional risks and factors that may affect results are set forth in Aptevo's filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the fiscal year ended December 31, 2023, and its subsequent reports on Form 10-Q and current reports on Form 8-K. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from Aptevo's expectations in any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, Aptevo does not assume any obligation to update any forward-looking statement to reflect new information, events, or circumstances.
CONTACT:
Miriam Weber Miller
Head, Investor Relations & Corporate Communications
Aptevo Therapeutics
Email: IR@apvo.com or Millerm@apvo.com
Phone: 206-859-6628
SOURCE: Aptevo Therapeutics
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