Aptevo Therapeutics Reports 2024 Financial Results And Provides A Business Update
Aptevo Therapeutics (NASDAQ:APVO) reported significant clinical progress for 2024. In the RAINIER trial, testing mipletamig for frontline AML treatment, 100% of Cohort 1 patients achieved remission within 30 days, with one patient reaching MRD-negative status. The drug showed a favorable safety profile with cytokine release syndrome.
The company's ALG.APV-527 Phase 1 trial for solid tumors demonstrated promising results, with a 59% stable disease rate (10 out of 17 evaluable patients). Notable outcomes included a breast cancer patient maintaining stable disease for >12 months and a colon cancer patient with stable disease for >6 months. The treatment showed liver toxicity, a common side effect in similar therapies.
These results build upon previous successful trials, including a Phase 1b combination therapy trial where 100% of frontline patients achieved remission, and a Phase 1a monotherapy trial showing 36% of evaluable patients experienced significant leukemic blast reduction.
Aptevo Therapeutics (NASDAQ:APVO) ha riportato progressi clinici significativi per il 2024. Nella sperimentazione RAINIER, che testa il mipletamig per il trattamento di prima linea dell'AML, il 100% dei pazienti del Coorte 1 ha raggiunto la remissione entro 30 giorni, con un paziente che ha raggiunto lo stato MRD-negativo. Il farmaco ha mostrato un profilo di sicurezza favorevole con sindrome da rilascio di citochine.
La sperimentazione di Fase 1 dell'ALG.APV-527 per tumori solidi ha dimostrato risultati promettenti, con un 59% di tasso di malattia stabile (10 su 17 pazienti valutabili). Risultati notevoli includevano una paziente con cancro al seno che ha mantenuto la malattia stabile per oltre 12 mesi e un paziente con cancro al colon con malattia stabile per oltre 6 mesi. Il trattamento ha mostrato tossicità epatica, un effetto collaterale comune in terapie simili.
Questi risultati si basano su precedenti sperimentazioni di successo, inclusa una sperimentazione di terapia combinata di Fase 1b in cui il 100% dei pazienti di prima linea ha raggiunto la remissione, e una sperimentazione di monoterapia di Fase 1a che ha mostrato che il 36% dei pazienti valutabili ha sperimentato una significativa riduzione dei blast leucemici.
Aptevo Therapeutics (NASDAQ:APVO) reportó avances clínicos significativos para 2024. En el ensayo RAINIER, que prueba el mipletamig para el tratamiento de primera línea de la AML, el 100% de los pacientes del Cohorte 1 alcanzaron remisión en 30 días, con un paciente alcanzando el estado MRD-negativo. El fármaco mostró un perfil de seguridad favorable con síndrome de liberación de citoquinas.
El ensayo de Fase 1 de ALG.APV-527 para tumores sólidos demostró resultados prometedores, con un 59% de tasa de enfermedad estable (10 de 17 pacientes evaluables). Resultados notables incluyeron una paciente con cáncer de mama que mantuvo enfermedad estable durante más de 12 meses y un paciente con cáncer de colon con enfermedad estable durante más de 6 meses. El tratamiento mostró toxicidad hepática, un efecto secundario común en terapias similares.
Estos resultados se basan en ensayos anteriores exitosos, incluida una prueba de terapia combinada de Fase 1b en la que el 100% de los pacientes de primera línea alcanzaron remisión, y un ensayo de monoterapia de Fase 1a que mostró que el 36% de los pacientes evaluables experimentaron una reducción significativa de los blastos leucémicos.
Aptevo Therapeutics (NASDAQ:APVO)는 2024년을 위한 중요한 임상 진전을 보고했습니다. RAINIER 시험에서, AML 치료를 위한 1차 치료제로 mipletamig을 테스트한 결과, 1군 환자의 100%가 30일 이내에 완전 관해에 도달했습니다, 한 환자는 MRD 음성 상태에 도달했습니다. 이 약물은 사이토카인 방출 증후군과 함께 유리한 안전성 프로필을 보였습니다.
ALG.APV-527의 고형 종양에 대한 1상 시험은 59%의 안정적인 질병 비율 (17명의 평가 가능한 환자 중 10명)로 유망한 결과를 보여주었습니다. 특히 주목할 만한 결과로는 유방암 환자가 12개월 이상 안정적인 질병을 유지하였고, 대장암 환자가 6개월 이상 안정적인 질병을 유지한 사례가 있습니다. 이 치료는 유사한 요법에서 일반적인 부작용인 간 독성을 보였습니다.
이 결과는 1상 결합 요법 시험을 포함한 이전의 성공적인 시험에 기반하고 있으며, 이 시험에서 1차 환자의 100%가 완전 관해에 도달하였고, 1상 단독 요법 시험에서는 평가 가능한 환자의 36%가 유의미한 백혈병 세포 감소를 경험하였습니다.
Aptevo Therapeutics (NASDAQ:APVO) a rapporté des progrès cliniques significatifs pour 2024. Dans l'essai RAINIER, testant le mipletamig pour le traitement de première ligne de l'AML, 100% des patients du Cohorte 1 ont atteint la rémission en 30 jours, un patient ayant atteint le statut MRD-négatif. Le médicament a montré un profil de sécurité favorable avec syndrome de libération de cytokines.
L'essai de Phase 1 de l'ALG.APV-527 pour les tumeurs solides a démontré des résultats prometteurs, avec un taux de maladie stable de 59% (10 sur 17 patients évaluables). Parmi les résultats notables, une patiente atteinte de cancer du sein a maintenu une maladie stable pendant plus de 12 mois et un patient atteint de cancer du côlon a eu une maladie stable pendant plus de 6 mois. Le traitement a montré une toxicité hépatique, un effet secondaire courant dans des thérapies similaires.
Ces résultats s'appuient sur des essais précédents réussis, y compris un essai de thérapie combinée de Phase 1b où 100% des patients en première ligne ont atteint la rémission, et un essai de monothérapie de Phase 1a montrant que 36% des patients évaluables ont connu une réduction significative des blasts leucémiques.
Aptevo Therapeutics (NASDAQ:APVO) berichtete über bedeutende klinische Fortschritte für 2024. In der RAINIER-Studie, die mipletamig als Erstlinientherapie für AML testet, erreichten 100% der Patienten der Kohorte 1 innerhalb von 30 Tagen Remission, wobei ein Patient den MRD-negativen Status erreichte. Das Medikament zeigte ein günstiges Sicherheitsprofil mit Zytokinfreisetzungssyndrom.
Die Phase-1-Studie des Unternehmens ALG.APV-527 für solide Tumoren zeigte vielversprechende Ergebnisse, mit einer 59% stabilen Krankheitsrate (10 von 17 evaluierten Patienten). Bemerkenswerte Ergebnisse umfassten eine Brustkrebspatientin, die ihre stabile Erkrankung über >12 Monate aufrechterhielt, und einen Patienten mit Dickdarmkrebs, der seine stabile Erkrankung über >6 Monate behielt. Die Behandlung zeigte Lebertoxizität, eine häufige Nebenwirkung ähnlicher Therapien.
Diese Ergebnisse bauen auf früheren erfolgreichen Studien auf, einschließlich einer Phase-1b-Kombinationstherapiestudie, bei der 100% der Erstlinientherapie-Patienten Remission erreichten, und einer Phase-1a-Monotherapie-Studie, die zeigte, dass 36% der evaluierten Patienten eine signifikante Reduktion der leukämischen Blasten erfahren haben.
- 100% remission rate within 30 days in RAINIER trial Cohort 1
- 59% stable disease rate in ALG.APV-527 Phase 1 solid tumor trial
- Favorable safety profile with cytokine release syndrome
- liver toxicity in ALG.APV-527 treatment compared to competitors
- None.
Insights
The clinical data presented by Aptevo Therapeutics demonstrates remarkable efficacy signals that warrant careful attention. In the RAINIER trial, mipletamig's 100% remission rate within 30 days in Cohort 1 is particularly noteworthy, as typical remission rates for standard venetoclax/azacitidine therapy in AML range from 65-70%. The achievement of MRD negativity in one patient is especially significant, as MRD status is a important predictor of long-term outcomes in AML.
The incidence of cytokine release syndrome (CRS) with mipletamig is a important differentiator. Many bispecific antibodies in AML treatment require complex step-up dosing and hospitalization due to CRS risk, making a favorable safety profile particularly valuable for both patients and healthcare providers.
For ALG.APV-527, the 59% stable disease rate across multiple solid tumor types suggests broad therapeutic potential. The durability of response, particularly in breast cancer (>12 months) and colorectal cancer (>6 months) patients, is encouraging for a Phase 1 trial. The liver toxicity profile could provide a significant competitive advantage, as hepatotoxicity often restricts dosing and patient eligibility for similar therapeutics.
However, these results should be viewed with appropriate context: the RAINIER trial data comes from just the first cohort, and the true potential of both programs will depend on maintaining these promising efficacy and safety profiles in larger patient populations. The solid tumor program's focus on 5T4-expressing tumors represents a targeted approach, but successful commercialization will require careful patient selection and biomarker strategy.
Promising Data, including a
SEATTLE, WA / ACCESS Newswire / February 14, 2025 / 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 year ended December 31, 2024 and provided a business update.
Business Update
"2024 was a successful year for Aptevo, as we've demonstrated the power of our science and our commitment to advancing oncology care. From the remarkable progress of mipletamig in AML, where
Mipletamig AML bispecific highlights:
Launched RAINIER, a dose optimization trial evaluating mipletamig in combination with venetoclax and azacitidine, the standard of care in frontline acute myeloid leukemia (AML) patients who are unfit to receive intensive high dose chemotherapy. RAINIER results reported to date include:
100% of patients in Cohort 1 of the trial achieved remission within 30 daysOne patient experienced complete remission with minimal residual disease (MRD)-negative status (
100% elimination of cancer cells)Favorable safety profile consistent with prior trials, showing limited incidences of cytokine release syndrome (CRS), a common and often dose limiting side effect seen in similar therapies
These results build on data from prior results, including:
Phase 1b dose expansion combination therapy trial in which
100% of frontline patients also achieved remission (Remission = complete remission (CR) and, complete remission with blood markers that have not yet recovered (CRi))Phase 1a dose escalation monotherapy trial in which
36% of evaluable patients experienced substantial leukemic blast reduction to a clinical meaningful degree compared to baseline (range of17% to88% reduction), providing evidence of the pharmacodynamic effect of the drug
The Company anticipates providing multiple data readouts in 2025 and to present results at the American Society of Hematology meeting in the fourth quarter.
ALG.APV-527 solid tumor bispecific highlights:
Completed a readout from our ALG.APV-527 Phase 1 dose escalation study evaluating the drug for the treatment of multiple solid tumor types likely to express tumor antigen 5T4. ALG.APV-527 is being developed in partnership with Alligator Bioscience.
Key trial data:
10 of 17 efficacy evaluable patients (
59% ) achieved stable diseaseThe longest duration of stable disease was in a breast cancer patient who entered the study with progressive disease, achieved stable disease and remained on study for >12 months. This patient successfully transitioned to a higher dose level twice
One colon cancer patient achieved stable disease for more than six months
One prostate cancer patient has been on study for at least four months and remains in stable disease
Safety results include limited incidence, and no severe cases of liver toxicity, a common and often dose limiting side effect seen in similar treatments
The data was presented at both the European Society for Medical Oncology Congress and the Society for Immunotherapy of Cancer Conference in 2024
"We are thrilled with the Cohort 1 RAINIER trial results that, taken together with data from our previously completed trials, demonstrate mipletamig's potential as a transformative therapy for the treatment of frontline AML in combination with standard of care venetoclax and azacitidine. Seeing all Cohort 1 patients achieve remission within 30 days, including a patient that reached MRD-negative status, is a testament to the strength of our science and commitment to addressing this devastating disease. These outcomes build on favorable data from our earlier trials, including monotherapy results where
About Mipletamig and RAINIER
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 binding domain derived from CRIS-7 vs. the CD3 binding domain used by competitors (which is derived from SP34). Mipletamig has received orphan drug designation ("orphan status") for AML according to the Orphan Drug Act. Mipletamig has been evaluated in over 100 patients to date. RAINIER, Aptevo's Phase 1b/2 frontline AML program, was initiated in 3Q24 and is ongoing.
RAINIER, a frontline AML study, is a two part Phase 1b/2 trial. Part one is an ongoing 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 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. 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 ALG.APV-527 and The Phase 1 Dose Escalation Trial
ALG.APV-527 is a bispecific conditional 4-1BB agonist, only active upon simultaneous binding to 4-1BB and 5T4. This has the potential to be clinically important because 4-1BB can stimulate the immune cells (antitumor-specific T cells and NK cells) involved in tumor control, making 4-1BB a particularly compelling target for cancer immunotherapy. 5T4 is an oncofetal tumor associated antigen overexpressed on numerous solid tumors including non-small-cell lung carcinoma (NSCLC), breast, head and neck, cervical, renal, gastric, and colorectal cancer.
The ALG.APV-527 Phase 1 trial was a multi-center, multi-cohort, open-label dose-escalation trial that involved the administration of ALG.APV-527 in up to six escalating dose levels. The trial enrolled adult patients with multiple solid tumor types/histologies likely to express the 5T4 antigen. ALG.APV-527 will be given intravenously once every two weeks. The trial is assessing the safety and tolerability, pharmacokinetics, pharmacodynamics and preliminary anti-tumor activity of ALG.APV-527.
2024 Summary Financial Results
Cash Position: Aptevo had cash and cash equivalents as of December 31, 2024, totaling
Research and Development Expenses: Research and development expenses decreased by
General and Administrative Expenses: General and administrative expenses decreased by
Other Income (Expense) Net:
Other Income from Continuing Operations, Net consists of other income, net of
Gain Related to Sale of Non-FinancialAsset consists of a
Discontinued Operations: Income from discontinued operations was
Net Income (Loss): Aptevo had a net loss of
Aptevo Therapeutics Inc.
CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share amounts)
| December 31, 2024 |
|
| December 31, 2023 |
| |||
ASSETS |
|
|
|
|
|
| ||
Current assets: |
|
|
|
|
|
| ||
Cash and cash equivalents |
| $ | 8,714 |
|
| $ | 16,904 |
|
Prepaid expenses |
|
| 1,689 |
|
|
| 1,473 |
|
Other current assets |
|
| 256 |
|
|
| 689 |
|
Total current assets |
|
| 10,659 |
|
|
| 19,066 |
|
Property and equipment, net |
|
| 543 |
|
|
| 895 |
|
Operating lease right-of-use asset |
|
| 4,389 |
|
|
| 4,881 |
|
Total assets |
| $ | 15,591 |
|
| $ | 24,842 |
|
LIABILITIES AND STOCKHOLDERS' EQUITY |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable and other accrued liabilities |
| $ | 3,053 |
|
| $ | 3,984 |
|
Accrued compensation |
|
| 1,856 |
|
|
| 2,098 |
|
Other current liabilities |
|
| 1,298 |
|
|
| 1,142 |
|
Total current liabilities |
|
| 6,207 |
|
|
| 7,224 |
|
Other long-term liabilities |
|
| - |
|
|
| - |
|
Operating lease liability |
|
| 4,629 |
|
|
| 5,397 |
|
Total liabilities |
|
| 10,836 |
|
|
| 12,621 |
|
|
|
|
|
|
|
|
| |
Stockholders' equity: |
|
|
|
|
|
|
|
|
Preferred stock: issued or outstanding |
|
| - |
|
|
| - |
|
Common stock: and 11,958 shares issued and outstanding at December 31, 2024 and December 31, 2023, respectively |
|
| 84 |
|
|
| 61 |
|
Additional paid-in capital |
|
| 252,248 |
|
|
| 235,607 |
|
Accumulated deficit |
|
| (247,577 | ) |
|
| (223,447 | ) |
Total stockholders' equity |
|
| 4,755 |
|
|
| 12,221 |
|
Total liabilities and stockholders' equity |
| $ | 15,591 |
|
| $ | 24,842 |
|
Aptevo Therapeutics Inc.
CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
| For the Year Ended December 31, |
| ||||||
| 2024 |
|
| 2023 |
| |||
Operating expenses: |
|
|
|
|
|
| ||
Research and development |
| $ | (14,378 | ) |
| $ | (17,107 | ) |
General and administrative |
|
| (10,224 | ) |
|
| (11,771 | ) |
Loss from operations |
|
| (24,602 | ) |
|
| (28,878 | ) |
Other income: |
|
|
|
|
|
|
|
|
Other income from continuing operations, net |
|
| 472 |
|
|
| 578 |
|
Gain related to sale of non-financial asset |
|
| - |
|
|
| 9,650 |
|
Net loss from continuing operations |
| $ | (24,130 | ) |
| $ | (18,650 | ) |
Discontinued operations: |
|
|
|
|
|
|
|
|
Income from discontinued operations |
| $ | - |
|
| $ | 1,239 |
|
Net loss |
| $ | (24,130 | ) |
| $ | (17,411 | ) |
|
|
|
|
|
|
|
| |
Basic and diluted net loss per share from continuing operations: |
|
|
|
|
|
|
|
|
Basic |
| $ | (87.38 | ) |
| $ | (2,481.70 | ) |
Diluted |
| $ | (87.38 | ) |
| $ | (2,481.70 | ) |
Basic and diluted net loss per share: |
|
|
|
|
|
|
|
|
Basic |
| $ | (87.38 | ) |
| $ | (2,316.83 | ) |
Diluted |
| $ | (87.38 | ) |
| $ | (2,316.83 | ) |
Shares used in calculation: |
|
|
|
|
|
|
|
|
Basic |
|
| 276,137 |
|
|
| 7,515 |
|
Diluted |
|
| 276,137 |
|
|
| 7,515 |
|
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, 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 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
What were the key results from Aptevo's (APVO) RAINIER trial in 2024?
How effective was APVO's ALG.APV-527 in the Phase 1 solid tumor trial?
What distinguishes APVO's ALG.APV-527 from competitor treatments?
What were the results of APVO's previous mipletamig trials before RAINIER?
