First Patient Dosed in Aptevo's Ongoing RAINIER Trial Achieves 90% Reduction in Leukemic Blasts Within the First 30 Days of Treatment, Continues Overall Efficacy Trend Seen in Prior Mipletamig AML Studies
Aptevo Therapeutics (NASDAQ: APVO) reports promising early results from its RAINIER trial, where the first patient treated with mipletamig achieved a 90% reduction in leukemic blasts within 30 days. The trial evaluates mipletamig as a frontline therapy for Acute Myeloid Leukemia (AML) in combination with venetoclax and azacitidine. The triplet combination therapy targets AML through multiple mechanisms, potentially improving patient outcomes, particularly in elderly patients with treatment options. Previous trials demonstrated favorable safety profiles with manageable cytokine release syndrome levels, along with compelling response and durability of remission.
Aptevo Therapeutics (NASDAQ: APVO) riporta risultati iniziali promettenti dal suo trial RAINIER, dove il primo paziente trattato con mipletamig ha ottenuto una riduzione del 90% delle cellule leucemiche entro 30 giorni. Il trial valuta mipletamig come terapia di prima linea per Leucemia Mieloide Acuta (LMA) in combinazione con venetoclax e azacitidina. La terapia combinata tripla mira a colpire l'LMA attraverso molteplici meccanismi, migliorando potenzialmente gli esiti per i pazienti, in particolare gli anziani con opzioni di trattamento limitate. I trial precedenti hanno dimostrato profili di sicurezza favorevoli con livelli gestibili di sindrome da rilascio di citochine, insieme a risposte convincenti e durata della remissione.
Aptevo Therapeutics (NASDAQ: APVO) informa sobre resultados iniciales prometedores de su ensayo RAINIER, donde el primer paciente tratado con mipletamig logró una reducción del 90% en los blastos leucémicos en 30 días. El ensayo evalúa mipletamig como terapia de primera línea para Leucemia Mieloide Aguda (LMA) en combinación con venetoclax y azacitidina. La terapia de combinación triple ataca la LMA a través de múltiples mecanismos, mejorando potencialmente los resultados en los pacientes, especialmente en pacientes ancianos con opciones de tratamiento limitadas. Ensayos anteriores demostraron perfiles de seguridad favorables con niveles manejables de síndrome de liberación de citoquinas, además de respuestas contundentes y durabilidad de la remisión.
Aptevo Therapeutics (NASDAQ: APVO)는 RAINIER 임상시험에서 유망한 초기 결과를 보고하며, 여기서 mipletamig로 치료된 첫 번째 환자가 30일 이내에 백혈병 세포가 90% 감소했습니다. 이 시험은 mipletamig를 급성 골수성 백혈병 (AML)의 1차 요법으로, venetoclax와 azacitidine과 함께 평가합니다. 세 가지 약물의 조합 치료는 다양한 메커니즘을 통해 AML을 표적으로 하여, 특히 치료 옵션이 제한된 노인 환자들의 결과를 개선할 가능성이 있습니다. 이전 시험들은 관리 가능한 사이토카인 방출 증후군 수준과 함께 유리한 안전성 프로필 및 강력한 반응과 지속적인 관해를 입증했습니다.
Aptevo Therapeutics (NASDAQ: APVO) rapporte des résultats initiaux prometteurs de son essai RAINIER, où le premier patient traité avec mipletamig a atteint une réduction de 90 % des blasts leucémiques en 30 jours. L'essai évalue mipletamig en tant que thérapie de première ligne pour leucémie myéloïde aiguë (LMA) en combinaison avec venetoclax et azacitidine. La thérapie combinée en triplet cible la LMA grâce à plusieurs mécanismes, améliorant potentiellement les résultats pour les patients, notamment chez les personnes âgées avec des options de traitement limitées. Des essais précédents ont démontré des profils de sécurité favorables avec des niveaux gérables de syndrome de libération de cytokines, ainsi qu'une réponse convaincante et une durabilité de la rémission.
Aptevo Therapeutics (NASDAQ: APVO) berichtet von vielversprechenden frühen Ergebnissen aus seiner RAINIER-Studie, in der der erste Patient, der mit mipletamig behandelt wurde, innerhalb von 30 Tagen eine 90%ige Reduktion der leukämischen Blasten erreichte. Die Studie bewertet mipletamig als Erstlinientherapie für Akute Myeloische Leukämie (AML) in Kombination mit Venetoclax und Azacitidin. Die Dreifachkombinationstherapie zielt auf AML durch mehrere Mechanismen ab und könnte die Behandlungsergebnisse, insbesondere bei älteren Patienten mit eingeschränkten Therapieoptionen, verbessern. Frühere Studien zeigten günstige Sicherheitsprofile mit handhabbaren Zytokinfreisetzungssyndromniveaus sowie überzeugende Antworten und Langlebigkeit der Remission.
- First patient in RAINIER trial showed 90% reduction in leukemic blasts within 30 days
- Strong safety profile with manageable cytokine release syndrome levels
- FDA approval to proceed with frontline treatment evaluation
- Demonstrated compelling response and durability of remission in previous trials
- None.
Insights
The early clinical data from the RAINIER trial presents a significant milestone in AML treatment development. A 90% reduction in leukemic blasts within 30 days in the first patient is particularly noteworthy, as blast reduction is a key indicator of treatment efficacy. The triplet combination strategy of mipletamig with venetoclax and azacitidine represents a novel approach targeting multiple pathways in AML.
The drug's demonstrated safety profile, particularly the manageable CRS levels compared to similar therapeutics, is important for its potential adoption in frontline therapy. Moving to frontline treatment evaluation is significant, as successful outcomes in this setting could position mipletamig as a standard first-line option. The focus on elderly patients, who typically have treatment options, addresses an important market gap.
However, while these early results are promising, investors should note that this data represents just one patient and broader trial results will be necessary to confirm efficacy across a larger population.
Potential to redefine frontline AML treatment being evaluated in clinic: Mipletamig, CD3 x CD123 bispecific, in combination with standard of care, offers a multi-mechanism strategy for potential improved patient outcomes
Favorable early safety, efficacy, tolerability and durability of remission data informed Aptevo's ongoing RAINIER Phase 1b/2 trial
SEATTLE, WA / ACCESSWIRE / November 20, 2024 / Aptevo Therapeutics ("Aptevo") (Nasdaq:APVO), a clinical-stage biotechnology company focused on developing novel immune-oncology therapeutics based on its proprietary ADAPTIR™ and ADAPTIR-FLEX™ platform technologies, today expanded on the potential of mipletamig, currently being evaluated as frontline therapy for the treatment of Acute Myeloid Leukemia (AML), in combination with standard of care venetoclax and azacitidine, noting the first patient dosed in the ongoing RAINIER trial achieved a
The Company's move to evaluate mipletamig as frontline combination therapy is informed by favorable data from earlier trials, which demonstrated strong safety, efficacy, and tolerability profiles, alongside evidence of durable remission. These early findings have fueled excitement around RAINIER among the group of investigators at premier cancer treatment institutions who have been working with the drug and members of the biotechnology industry who have been following its progress.
RAINIER seeks to further demonstrate the impact of mipletamig's unique, dual targeted mechanism of action by combining it with standard of care therapies venetoclax and azacitidine. The triplet combination targets AML from multiple angles and has the potential to improve patient outcomes, especially among the elderly who have few treatment options available.
"The FDA's decision to allow the RAINIER trial to proceed in a frontline setting allows us to explore mipletamig and the potential synergistic effect with standard of care in a well-defined patient population in need of improved therapy options. Safety data from our prior studies support our approach to conduct a study that is designed for a statistically defined quantitative efficacy analysis. RAINIER represents a clear development path that allows us to evaluate mipletamig in newly diagnosed patients and represents a pivotal opportunity to make a difference right at the start of treatment, where outcomes can have the greatest impact," said Dirk Huebner, MD, Chief Medical Officer at Aptevo.
Dr. Huebner continued, "To date, mipletamig has demonstrated a strong safety profile with manageable incidence and severity of the most common therapeutic side effect, cytokine release syndrome (CRS), below levels seen with other treatment modalities of similar therapeutic mechanisms of action. Strong support for the development of a triplet combination that includes mipletamig is driven by efficacy data from prior mipletamig studies, showing compelling response and durability of remission compared to benchmarks from literature*." (*Aldoss 2019, Maiti 2021, Morsia 2020, Garciaz 2022, Feld 2021).
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 will be 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 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, 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. The Company 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, statements regarding advancement of Aptevo's therapeutic candidates in clinical trials, including the initiation of the Phase 1b/2 dose optimization trial to further evaluate mipletamig in combination with venetoclax and azacitidine , whether the Phase 1b/2 protocol will be successful, whether further study of mipletamig in Phase 1b/2 trial focusing on a targeted patient population will continue to show clinical benefit, whether Aptevo's strategy will translate into an improved overall survival rate in acute myeloid leukemia, statements related to the durability of mipletamig and whether its duration of remission results will be indicative of later stage clinical trials, whether the mipletamig data in combination therapy and monotherapy will be indicative of later stage clinical trials, statements related to mipletamig's mechanism of action and whether such mechanism of action will improve patient outcomes, the timing for its expected data readouts and whether this trial will establish a recommended Phase 2 dose, the potential use of any such candidate as therapeutics for treatment of disease, expectations about the safety, tolerability, efficacy and durability of its therapeutic candidate, statements regarding preclinical and clinical results and any suggestion that those results will be replicated in clinical development, the effectiveness of its ADAPTIR and ADAPTIR-FLEX platforms, and any other statements containing the words "may," "believes," "expects," "potential," "designed," "engineered," "innovative," "initiate," "allow," "promise," "plans," "will" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Aptevo's current intentions, beliefs, and expectations regarding future events. Aptevo cannot guarantee that any forward-looking statement will be accurate. Investors should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from Aptevo's expectations. Investors are, therefore, cautioned not to place undue reliance on any forward-looking statement.
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; adverse developments in the U.S. or global capital markets, credit markets or economies generally; 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 initiation, enrollment and maintenance of patients in clinical trials, uncertainties inherent in the results of preliminary or interim data and preclinical and clinical studies being predictive of the results of later-stage 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 the Company's product candidates, business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises such as the novel coronavirus (referred to as COVID-19), geopolitical risks, including the current war between Russia and Ukraine as well as the war between Israel and Hamas, and macroeconomic conditions such as rising inflation and interests rates, increased 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.
Aptevo Therapeutics
Miriam Weber Miller
Email: IR@apvo.com or millerm@apvo.com
Phone: 206-859-6629
SOURCE: Aptevo Therapeutics
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