Aptevo Therapeutics Reports 1Q25 Financial Results And Provides an Update on Mipletamig
- 5 out of 6 patients in RAINIER trial achieved remission within 30 days - No cytokine release syndrome (CRS) observed in current trial - One patient achieved complete remission with MRD-negative status - Cohort 2 enrollment completed, Cohort 3 now enrolling
The drug has been tested in nearly 100 patients across three clinical studies, showing promising results in both combination and monotherapy settings. In monotherapy trials, 36% of evaluable patients who failed prior treatments showed significant leukemic blast reduction. The company plans multiple data readouts in 2025.
- 5 pazienti su 6 nello studio RAINIER hanno ottenuto la remissione entro 30 giorni - Nessuna sindrome da rilascio di citochine (CRS) osservata nello studio attuale - Un paziente ha raggiunto la remissione completa con stato MRD-negativo - Completato il reclutamento del Gruppo 2, in corso quello del Gruppo 3
Il farmaco è stato testato in quasi 100 pazienti in tre studi clinici, mostrando risultati promettenti sia in combinazione che in monoterapia. Nei trial in monoterapia, il 36% dei pazienti valutabili che avevano fallito trattamenti precedenti ha mostrato una significativa riduzione dei blasti leucemici. L'azienda prevede molteplici aggiornamenti sui dati nel 2025.
- 5 de 6 pacientes en el ensayo RAINIER lograron la remisión en 30 días - No se observó síndrome de liberación de citocinas (CRS) en el ensayo actual - Un paciente logró remisión completa con estado MRD-negativo - Finalizada la inscripción del Grupo 2, el Grupo 3 está en proceso de inscripción
El medicamento ha sido probado en casi 100 pacientes en tres estudios clínicos, mostrando resultados prometedores tanto en combinación como en monoterapia. En los ensayos de monoterapia, el 36% de los pacientes evaluables que no respondieron a tratamientos previos mostraron una reducción significativa de blastos leucémicos. La compañía planea múltiples presentaciones de datos en 2025.
- RAINIER 시험에서 6명 중 5명이 30일 이내에 완화 달성 - 현재 시험에서 사이토카인 방출 증후군(CRS) 관찰되지 않음 - 한 명의 환자가 MRD 음성 상태로 완전 관해 달성 - 2차 코호트 등록 완료, 3차 코호트 등록 중
이 약물은 세 건의 임상연구에서 약 100명의 환자에게 투여되어 병용요법과 단독요법 모두에서 유망한 결과를 보였습니다. 단독요법 임상에서는 이전 치료에 실패한 평가 가능한 환자의 36%가 백혈병 세포 감소를 보였습니다. 회사는 2025년에 여러 차례 데이터 발표를 계획하고 있습니다.
- 5 patients sur 6 dans l'essai RAINIER ont obtenu une rémission en 30 jours - Aucun syndrome de libération de cytokines (CRS) observé dans l'essai actuel - Un patient a obtenu une rémission complète avec statut MRD négatif - Le recrutement du groupe 2 est terminé, celui du groupe 3 est en cours
Le médicament a été testé chez près de 100 patients dans trois études cliniques, montrant des résultats prometteurs en association et en monothérapie. Dans les essais en monothérapie, 36 % des patients évaluables ayant échoué aux traitements précédents ont montré une réduction significative des blastes leucémiques. La société prévoit plusieurs publications de données en 2025.
- 5 von 6 Patienten in der RAINIER-Studie erreichten innerhalb von 30 Tagen eine Remission - Kein Zytokinfreisetzungssyndrom (CRS) im aktuellen Versuch beobachtet - Ein Patient erreichte vollständige Remission mit MRD-negativem Status - Kohorte 2 abgeschlossen, Kohorte 3 in der Rekrutierung
Das Medikament wurde bei fast 100 Patienten in drei klinischen Studien getestet und zeigte vielversprechende Ergebnisse sowohl in Kombination als auch als Monotherapie. In Monotherapiestudien zeigten 36 % der auswertbaren Patienten, die auf vorherige Behandlungen nicht angesprochen hatten, eine deutliche Reduktion der leukämischen Blasten. Das Unternehmen plant mehrere Datenveröffentlichungen im Jahr 2025.
- 90% remission rate (9 out of 10) in frontline AML patients across two trials
- Rapid remission achievement within 30 days of treatment
- No cytokine release syndrome (CRS) observed in RAINIER trial, demonstrating favorable safety profile
- One patient achieved complete remission with MRD-negative status (100% cancer cell elimination)
- Trial progression on track with Cohort 2 completed and Cohort 3 enrolling
- One patient progressed after first cycle and died (unrelated to study drug)
- Still in early clinical trial phases (Phase 1b/2)
Insights
Aptevo's mipletamig shows exceptional 90% remission rate in frontline AML with no cytokine release syndrome, significantly outperforming standard care.
The data from Aptevo's mipletamig trials represents a potentially significant advance in AML treatment. The reported 90% remission rate (9 out of 10 patients) across two trials in frontline AML patients is substantially higher than the typical 66-70% remission rates observed with the current standard of care (venetoclax + azacitidine) alone. What's particularly noteworthy is the absence of cytokine release syndrome (CRS) in the RAINIER trial—a common dose-limiting toxicity with T-cell engagers that often restricts dosing and efficacy.
The achievement of an MRD-negative complete remission in one patient is especially promising. MRD negativity correlates strongly with improved long-term outcomes and potential durability of response. Additionally, the rapid time to response (within 30 days) could translate to shortened hospital stays and reduced treatment-related complications.
The drug's apparent efficacy across three different clinical contexts—as a triplet combination in frontline patients, in the expansion study with the same triplet, and even as monotherapy in relapsed/refractory patients (17-88% blast reduction)—suggests consistent biological activity across different disease stages.
The ADAPTIR platform's engineered CD3 binding domain (CRIS-7) appears to be successfully balancing T-cell activation with reduced cytokine release, addressing a critical safety concern that has limited the utility of other T-cell engagers in AML. If these early results hold up in larger cohorts, mipletamig could potentially address the significant unmet need in AML treatment, particularly for elderly or unfit patients who cannot tolerate intensive chemotherapy.
9 of 10 frontline AML patients achieve remission with mipletamig + standard of care across two trials
Mipletamig triplet combination continues to outperform standard doublet benchmark
No cytokine release syndrome observed in the ongoing RAINIER trial: favorable safety profile supports role in frontline AML
Cohort 2 enrollment is complete, and Cohort 3 is now enrolling
Aptevo Therapeutics Inc. (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 reported financial results for the quarter ended March 31, 2025, and provided an update on the ongoing mipletamig clinical trial.
Business Update
"The latest outcomes from the RAINIER trial-remissions that were achieved rapidly and with no reported cytokine release syndrome-underscore the strength of our approach and the potential of mipletamig to redefine frontline AML treatment. With each milestone, we are not only validating the safety and efficacy of our drug, but moving closer to changing the standard of care for patients who have long had limited options. We remain focused on advancing the trial and look forward to sharing further clinical updates in the months ahead. This is more than progress-it's momentum toward a new era in frontline AML care," said Marvin White, President and CEO of Aptevo.
Mipletamig Data Highlights
Enrollment continues in the RAINIER dose optimization trial. This is a Phase 1b/2 study evaluating mipletamig in combination with venetoclax and azacitidine, the standard of care for frontline acute myeloid leukemia (AML) patients who are unfit to receive intensive high dose chemotherapy. Cohort 2 enrollment is now complete, and Cohort 3 is now open and enrolling.
Mipletamig has been evaluated in almost 100 patients across three clinical studies, thus far, in both combination and monotherapy settings. Outcomes have consistently demonstrated a growing body of evidence that supports its potential role in the treatment of frontline AML.
Data Highlights include:
Efficacy Results Reported on March 20, 2025
Data from Six frontline AML patients has been reported in the ongoing RAINIER Phase 1b/2 trial evaluating mipletamig in combination with venetoclax and azacitidine. Of these, five of six (5/6) patients have achieved remission within 30 days of being dosed
One patient progressed after the first cycle and passed away due to reasons unrelated to the study drug
These results build on data from a prior expansion study, in which four additional frontlinepatients treated with the same triplet combination also achieved remission*
In total, 9 of 10 (
90% ) frontline patients across two trials have achieved remission with mipletamig in combination with standard of care therapy
Safety
Mipletamig continues to demonstrate a highly favorable safety profile, particularly with respect to cytokine release syndrome (CRS)-a common and often dose-limiting side effect seen in other T-cell engagers and bispecific antibodies.
No CRS has been observed in the ongoing RAINIER trial to date
This favorable profile is consistent with earlier trials, where CRS was generally minimal and manageable
The company attributes this safety profile, a key differentiator, to the engineered CD3 binding domain (CRIS-7) and its ADAPTIR® format, which are designed to reduce cytokine release without compromising T-cell activation or anti-leukemic activity
Additional Data
RAINIER Cohort 1 data (included in the total patient outcomes above) in which
100% of patients achieved remission within the first 30 daysOne patient experienced complete remission with minimal residual disease (MRD)-negative status (
100% elimination of cancer cells)MRD-negative status is 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
Phase 1b dose expansion combination therapy (included in the total patient outcomes above) trial in which
100% of frontline patients achieved remissionPhase 1a dose escalation monotherapy trial in which
36% of evaluable patients who failed prior regimens experienced substantial leukemic blast reduction to a clinically meaningful degree compared to baseline (range of17% to88% reduction), providing evidence of the pharmacodynamic effect of the drug - and efficacy contribution to the triplet combination
*Remission = complete remission (CR) and, complete remission with blood markers that have not yet recovered CRi.
The Company anticipates providing multiple data readouts in 2025 and to present results at the American Society of Hematology meeting in the fourth quarter.
About Mipletamig
Mipletamig, a differentiated by design CD123xCD3 bispecific antibody built on Aptevo's ADAPTIR platform and driven by a unique CRIS-7-derived CD3 binding domain, is being investigated as frontline therapy in combination with venetoclax and azacitidine, the current standard of care for AML patients who are unfit for intensive chemotherapy. 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 almost 100 patients.
2025 Summary Financial Results
Cash Position: Aptevo had cash and cash equivalents as of March 31, 2025, totaling
Research and Development Expenses: Research and development expenses decreased by
General and Administrative Expenses: General and administrative expenses decreased by
Other Income, Net: Other income, net consists primarily of interest income from our cash equivalents and short-term rental income. Other income, net was
Net Income (Loss): Aptevo had a net loss of
Aptevo Therapeutics Inc.
CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share amounts)
March 31, 2025 | December 31, 2024 | |||||||
ASSETS | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 2,139 | $ | 8,714 | ||||
Prepaid expenses | 1,348 | 1,689 | ||||||
Other current assets | 120 | 256 | ||||||
Total current assets | 3,607 | 10,659 | ||||||
Property and equipment, net | 482 | 543 | ||||||
Operating lease right-of-use asset | 4,253 | 4,389 | ||||||
Total assets | $ | 8,342 | $ | 15,591 | ||||
LIABILITIES AND STOCKHOLDERS' (DEFICIT) EQUITY | ||||||||
Current liabilities: | ||||||||
Accounts payable and other accrued liabilities | $ | 3,789 | $ | 3,053 | ||||
Accrued compensation | 472 | 1,856 | ||||||
Other current liabilities | 1,132 | 1,298 | ||||||
Total current liabilities | 5,393 | 6,207 | ||||||
Operating lease liability | 4,422 | 4,629 | ||||||
Total liabilities | 9,815 | 10,836 | ||||||
Stockholders' (deficit) equity: | ||||||||
Preferred stock: issued or outstanding | - | - | ||||||
Common stock: and 1,458,443 shares issued and outstanding at March 31, 2025, and December 31, 2024, respectively | 84 | 84 | ||||||
Additional paid-in capital | 252,428 | 252,248 | ||||||
Accumulated deficit | (253,985 | ) | (247,577 | ) | ||||
Total stockholders' (deficit) equity | (1,473 | ) | 4,755 | |||||
Total liabilities and stockholders' (deficit) equity | $ | 8,342 | $ | 15,591 | ||||
Aptevo Therapeutics Inc.
CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
For the Three Months Ended March 31, | ||||||||
2025 | 2024 | |||||||
Operating expenses: | ||||||||
Research and development | $ | (3,633 | ) | $ | (3,752 | ) | ||
General and administrative | (2,847 | ) | (3,231 | ) | ||||
Loss from operations | (6,480 | ) | (6,983 | ) | ||||
Other income: | ||||||||
Other income from continuing operations, net | 72 | 149 | ||||||
Net loss | $ | (6,408 | ) | $ | (6,834 | ) | ||
Basic and diluted net loss per share: | $ | (4.39 | ) | $ | (368.21 | ) | ||
Shares used in calculation: | 1,458,458 | 18,560 | ||||||
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 two-part Phase 1b/2 trial for the treatment of frontline acute myeloid leukemia in combination with standard-of-care venetoclax + azacitidine. Mipletamig has received orphan drug designation ("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, ADAPTIRand 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, whether Aptevo's final trial results will vary from its earlier assessment, whether Aptevo's strategy will translate into an improved overall survival in AML, especially among patient subgroups with poor prognosis, whether further study of ALG.APV-527 across multiple tumor types will continue to show clinical benefit, the possibility and timing of interim data readouts for ALG.APV-527, statements related to Aptevo's cash position and balance sheet, statements related to Aptevo's ability to generate stockholder value, whether Aptevo will continue to have momentum in its business in the future, and any other statements containing the words "may," "continue to," "believes," "knows," "expects," "optimism," "potential," "designed," "promising," "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; 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 and the 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, 2024, 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
View the original press release on ACCESS Newswire
FAQ
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