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Aptose’s Tuspetinib Exceeds Expectations When Combined with Standard of Care Treatment Across Diverse Populations of Newly Diagnosed AML

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Aptose (OTC: APTOF) reported Phase 1/2 TUSCANY data showing the tuspetinib (TUS) triplet with venetoclax and azacitidine (TUS+VEN+AZA) produced high remission and MRD results in newly diagnosed AML patients ineligible for induction chemotherapy.

Key data: CR/CRh 9/10 (90%) overall, 100% CR/CRh (6/6) at 80/120 mg TUS, 88% CR/CRh (7/8) in FLT3 wildtype, and MRD-negativity 7/9 (78%) of responders. No DLTs, no treatment-related deaths, no QTc prolongation, no differentiation syndrome, and no prolonged myelosuppression reported to date. Dosing at 160 mg TUS has begun; enrollment of 18–24 patients is anticipated by end-2025.

Aptose (OTC: APTOF) ha riportato dati di fase 1/2 di TUSCANY che mostrano che il triplo TUS (TUS) con venetoclax e azacitidina (TUS+VEN+AZA) ha prodotto alti tassi di remissione e MRD in pazienti AML recentemente diagnosticati non eleggibili alla chemioterapia di induzione.

Dati chiave: CR/CRh 9/10 (90%) complessivo, 100% CR/CRh (6/6) a 80/120 mg TUS, 88% CR/CRh (7/8) in FLT3 wildtype, e MRD-negatività 7/9 (78%) dei responder. Nessuna DLT, nessun decesso correlato al trattamento, nessuna prolungata QTc, nessuna sindrome da differenziazione, e nessuna mielosoppresione prolungata riportata a oggi. L'inizio della somministrazione a 160 mg TUS è iniziato; si prevede l'arruolamento di 18–24 pazienti entro la fine del 2025.

Aptose (OTC: APTOF) presentó datos de Fase 1/2 de TUSCANY que muestran que el triplete de tuspetinib (TUS) con venetoclax y azacitidina (TUS+VEN+AZA) produjo altas tasas de remisión y MRD en pacientes con AML recién diagnosticada no elegibles para la quimioterapia de inducción.

Datos clave: CR/CRh 9/10 (90%) en total, CR/CRh al 100% (6/6) a 80/120 mg de TUS, CR/CRh 88% (7/8) en FLT3 wild-type, y MRD-negatividad 7/9 (78%) de los respondedores. Sin DLT, sin muertes por tratamiento, sin prolongación del QTc, sin síndrome de diferenciación y sin mielosopresión prolongada reportada hasta la fecha. Se ha iniciado la dosis a 160 mg de TUS; se espera reclutar de 18 a 24 pacientes para fines de 2025.

Aptose (OTC: APTOF)는 TUSCANY의 1상/2상 데이터를 발표했으며, 테스페틴(TUS, tuspetinib) 트리플렛이 벤토클락스와 아자시티딘(TUS+VEN+AZA)과 함께 신규 진단 AML 환자 중 유도 화학요법 대상이 아닌 환자에서 높은 관해 및 MRD 결과를 보여주었다고 발표했습니다.

주요 데이터: CR/CRh 9/10(90%) 전체, 80/120 mg TUS에서 100% CR/CRh(6/6), FLT3 와일드타입에서 88% CR/CRh(7/8), 그리고 응답자 중 MRD 음성 7/9(78%). DLT 없음, 치료 관련 사망 없음, QTc 연장 없음, 분화 증후군 없음, 그리고 현재까지 지속적인 골수억제 보고 없음. 160 mg TUS의 용량이 시작되었고 2025년 말까지 18–24명의 환자 모집이 예상됩니다.

Aptose (OTC: APTOF) a publié des données de phase 1/2 de TUSCANY montrant que le triplet tuspetinib (TUS) avec venetoclax et azacitidine (TUS+VEN+AZA) a produit des taux élevés de rémission et de MRD chez les patients AML nouvellement diagnostiqués non éligibles à la chimiothérapie d’induction.

Donnees clés: CR/CRh 9/10 (90%) au total, CR/CRh 100% (6/6) à 80/120 mg de TUS, CR/CRh 88% (7/8) chez FLT3 sauvage, et MRD-négativité 7/9 (78%) des répondants. Aucune DLT, aucune mort liée au traitement, aucune prolongation du QTc, aucun syndrome de différenciation et aucune myélosuppression prolongée rapportée à ce jour. Le dosage à 160 mg de TUS a commencé; le recrutement de 18–24 patients est prévu d’ici la fin 2025.

Aptose (OTC: APTOF) hat Phase-1/2-Daten von TUSCANY gemeldet, die zeigen, dass das Triplet aus Tuspetinib (TUS) mit Venetoclax und Azacitidin (TUS+VEN+AZA) bei neu diagnostizierten AML-Patienten, die nicht für eine Induktionschemotherapie geeignet sind, hohe Remissions- und MRD-Ergebnisse erzielt.

Wesentliche Daten: CR/CRh 9/10 (90%) insgesamt, CR/CRh 100% (6/6) bei 80/120 mg TUS, CR/CRh 88% (7/8) bei FLT3-Wildtyp, und MRD-Negativität 7/9 (78%) der Respondenten. Keine DLTs, keine behandlungsbedingten Todesfälle, keine QTc-Verlängerung, kein Differenzierungs-Syndrom und keine prolongierte Myelosuppression berichtet. Die Dosierung mit 160 mg TUS hat begonnen; die Einschreibung von 18–24 Patienten wird bis Ende 2025 erwartet.

Aptose (OTC: APTOF) أصدرت بيانات المرحلة 1/2 من TUSCANY تُظهر أن الثلاثي tuspetinib (TUS) مع venetoclax و azacitidine (TUS+VEN+AZA) حقق نتائج عالية في مرضى AML الذين تم تشخيصهم حديثاً وغير المؤهلين للعلاج الكيميائي التحضيري.

البيانات الرئيسية: CR/CRh 9/10 (90%) بشكل عام، CR/CRh 100% (6/6) عند جرعة TUS 80/120 mg، CR/CRh 88% (7/8) في FLT3 Wild-type، و MRD-negativity 7/9 (78%) من المستجيبين. لا توجد DLTs، ولا وفيات مرتبطة بالعلاج، ولا طول QTc، ولا متلازمة التمايز، ولا نقص نخاع العظام المستمر المبلغ عنه حتى الآن. بدأ إعطاء جرعة 160 mg من TUS؛ ومن المتوقع تسجيل 18–24 مريضاً بحلول نهاية 2025.

Aptose (OTC: APTOF) 报告了 TUSCANY 的 I/II 期数据,显示 tuspetinib (TUS) 与 venetoclax 和 azacitidine(TUS+VEN+AZA)的三联治疗在新诊断的 AML、不适合诱导化疗的患者中取得了高水平的缓解和 MRD 结果。

关键数据:CR/CRh 9/10 (90%) 总体80/120 mg TUS 时 CR/CRh 100% (6/6)FLT3 野生型中 CR/CRh 88% (7/8),以及 应答者中 MRD 阴性 7/9 (78%)。尚无 DLT、无治疗相关死亡、无 QTc 延长、无分化综合征,且迄今未报告持续性骨髓抑制。开始使用 160 mg TUS;预计到 2025 年底将入组 18–24 名患者。

Positive
  • CR/CRh 9/10 (90%) overall
  • 100% CR/CRh (6/6) at 80 mg and 120 mg TUS
  • MRD-negativity 7/9 (78%) of responders
  • No DLTs, no treatment-related deaths reported
Negative
  • Dataset limited to 10 patients to date
  • No long-term durability or survival data reported yet

Data from Phase 1/2 TUSCANY trial presented at the European School of Haematology (ESH) 7th International Conference

  • Addition of TUS to VEN+AZA achieves CR/CRh responses in all (6/6, 100%) patients treated at the higher dose levels of 80 mg and 120 mg TUS, exceeding the 66% rate expected from VEN+AZA alone
  • CR/CRh responses in 7/8 (88%) FLT3 wildtype AML, representing 70% of AML population
  • TUS+VEN+AZA achieves CR/CRh and MRD-negativity in TP53-mutated (2/2), RAS-mutated (1/1) and FLT3-ITD (2/2) AML patients to date
  • TUS+VEN+AZA is well tolerated with no DLT, differentiation syndrome, QTc prolongation, or prolonged myelosuppression at any dose level to date in newly diagnosed AML patients
  • TUS+VEN+AZA is being developed as a safe and mutation agnostic frontline therapy for AML
  • Dosing with 160 mg TUS is now ongoing

SAN DIEGO and TORONTO, Oct. 16, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, today announced that data from the ongoing TUSCANY trial of tuspetinib in combination with venetoclax and azacitidine (TUS+VEN+AZA) are being presented in a poster presentation, “TUSCANY Study of Safety and Efficacy of Tuspetinib plus Standard of Care Venetoclax and Azacitidine in Study Participants with Newly Diagnosed AML Ineligible for Induction Chemotherapy,” at the European School of Haematology (ESH) 7th International Conference on Acute Myeloid Leukemia “Molecular and Translational”: Advances in Biology and Treatment, being held from October 16-18, 2025 in Estoril, Portugal. Data to date from 10 patients in the TUSCANY trial across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, reveal promising clinical safety and antileukemic activity and support the use of TUS with standard of care treatment across a broad range of AML populations, including those carrying adverse mutations regardless of FLT3 mutation status.

The TUS+VEN+AZA triplet is being developed as a safe and well-tolerated, mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. Across all dose cohorts to date, no significant safety concerns or dose limiting toxicities (DLTs) have been observed in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission, no reports of drug-related QTc prolongation or differentiation syndrome (DS), no CPK elevation and no treatment-related deaths. Dosing has begun at the 160 mg TUS dose level.

“We have observed that TUS can be safely added to a backbone VEN+AZA without needing to reduce the dose of these standard-of-care drugs. The activity we have observed with the TUS triplet in the first 10 patients has exceeded our expectations with 9 achieving complete remissions and 7 demonstrating MRD-negativity by central flow cytometry,” said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. “In addition, these remissions are happening in diverse genetic subtypes including those with unmutated FLT3, FLT3-ITD, NPM1c, biallelic TP53 with complex karyotype, RAS, or myelodysplasia related mutations, making this a truly mutation agnostic therapy.”

Data highlights:

  • TUS in combination with standard dosing of VEN+AZA has been well tolerated with no DLT, no treatment-related deaths, no differentiation syndrome, no QTc prolongation, no prolonged myelosuppression after remission in Cycle 1, and no CPK elevations reported at any dose levels to date in these newly diagnosed AML patients.
  • Addition of TUS to VEN+AZA achieved CR/CRh responses in 6/6 (100%) patients treated at the higher dose levels of 80 mg and 120 mg TUS, exceeding the 66% rate expected from VEN+AZA alone.
  • Overall, TUS+VEN+AZA CR/CRh responses were observed in 9/10 (90%) patients.
  • 7 of 8 (88%) CR/CRh responses in FLT3 wildtype AML, representing 70% of AML population.
  • TUS+VEN+AZA MRD-negativity noted in 7/9 (78%) responding patients by central flow cytometry.
  • CR/CRh responses achieved across diverse mutational subtypes including: unmutated FLT3, FLT3-ITD, NPM1c, biallelic TP53 with complex karyotype, RAS, and myelodysplasia related mutations.
  • Dosing at the TUS 160 mg dose level is now ongoing.

See the ESH poster presentation here.

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The tuspetinib-based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated.

The TUSCANY triplet Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by the end of 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here).

About Aptose

Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company’s lead clinical-stage, oral kinase inhibitor tuspetinib (TUS) has demonstrated activity as a monotherapy and in combination therapy in patients with relapsed or refractory acute myeloid leukemia (AML) and is being developed as a frontline triplet therapy in newly diagnosed AML. For more information, please visit www.aptose.com.

Forward Looking Statements

This press release may contain forward-looking statements within the meaning of Canadian and U.S. securities laws, including, but not limited to, statements relating to the therapeutic potential and safety profile of tuspetinib (including the triplet therapy) and its clinical development, goals, the anticipated enrollment rate in the TUSCANY trial and the timing thereof, as well as statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “should”, “would”, “may”, and other similar expressions. Such statements reflect our current views with respect to future events and are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by us are inherently subject to significant business, economic, competitive, political and social uncertainties and contingencies. Many factors could cause our actual results, performance or achievements to be materially different from any future results, performance or achievements described in this press release. Such factors could include, among others: our ability to obtain the capital required for research and operations and to continue as a going concern; the inherent risks in early stage drug development including demonstrating efficacy; development time/cost and the regulatory approval process; the progress of our clinical trials; our ability to find and enter into agreements with potential partners; our ability to attract and retain key personnel; changing market conditions; inability of new manufacturers to produce acceptable batches of GMP in sufficient quantities; unexpected manufacturing defects; and other risks detailed from time-to-time in our ongoing quarterly filings, annual information forms, annual reports and annual filings with Canadian securities regulators and the United States Securities and Exchange Commission.

Should one or more of these risks or uncertainties materialize, or should the assumptions set out in the section entitled “Risk Factors” in our filings with Canadian securities regulators and the United States Securities and Exchange Commission underlying those forward-looking statements prove incorrect, actual results may vary materially from those described herein. These forward-looking statements are made as of the date of this press release and we do not intend, and do not assume any obligation, to update these forward-looking statements, except as required by law. We cannot assure you that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Investors are cautioned that forward-looking statements are not guarantees of future performance and accordingly investors are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty therein.

For further information, please contact:

Aptose Biosciences Inc.
Susan Pietropaolo
Corporate Communications & Investor Relations
201-923-2049
spietropaolo@aptose.com


FAQ

What were Aptose (APTOF) TUSCANY trial remission rates announced on October 16, 2025?

Aptose reported CR/CRh 9/10 (90%) overall with 100% (6/6) CR/CRh at 80 mg and 120 mg TUS dose levels.

How many patients in the APTOF TUSCANY study achieved MRD-negativity by central flow cytometry?

7 of 9 responding patients (78%) were MRD-negative by central flow cytometry.

Did the Aptose APTOF TUS+VEN+AZA triplet show safety concerns in the TUSCANY data?

No significant safety signals were reported: no DLTs, no QTc prolongation, no differentiation syndrome, and no treatment-related deaths to date.

Which mutational subtypes responded to TUS+VEN+AZA in the APTOF data?

Responses were observed across diverse subtypes including FLT3-ITD, FLT3 wildtype, NPM1c, biallelic TP53 with complex karyotype, RAS, and myelodysplasia-related mutations.

Is Aptose dosing a higher tuspetinib dose after the October 16, 2025 update?

Yes. Dosing has begun at the 160 mg TUS dose level.

How many patients does the APTOF TUSCANY study expect to enroll by end of 2025?

The company anticipates enrolling 18–24 patients in the TUSCANY study by the end of 2025.
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