Aptose Enrollment is Open for 160 mg Dosing Cohort of Tuspetinib in Phase 1/2 TUSCANY Trial of Frontline Triple Drug Therapy
Aptose Biosciences (OTC: APTOF) has received approval to escalate dosing to 160 mg in its Phase 1/2 TUSCANY trial, evaluating tuspetinib (TUS) in combination with venetoclax and azacitidine for newly diagnosed AML patients. The trial has successfully completed three dosing cohorts (40 mg, 80 mg, and 120 mg) with no dose-limiting toxicities.
The TUS+VEN+AZA triplet therapy has demonstrated complete remissions (CRs) and minimal residual disease (MRD) negativity in difficult-to-treat AML populations, including patients with adverse biallelic TP53 or FLT3-ITD mutations. The company also received a US$1.1M advance from Hanmi Pharmaceutical, bringing total advances under their loan agreement to US$5.6M.
The TUSCANY trial, conducted at 10 leading U.S. clinical sites, aims to enroll 18-24 patients by late 2025, targeting AML patients ineligible for induction chemotherapy.
Aptose Biosciences (OTC: APTOF) ha ottenuto l'approvazione per aumentare la dose a 160 mg nel suo studio di fase 1/2 TUSCANY, che valuta tuspetinib (TUS) in combinazione con venetoclax e azacitidina per pazienti con AML di nuova diagnosi. Lo studio ha completato con successo tre coorti di dosaggio (40 mg, 80 mg e 120 mg) senza tossicità dose-limitanti.
La terapia tripla TUS+VEN+AZA ha dimostrato remissioni complete (CR) e negatività per malattia residua minima (MRD) in popolazioni di AML difficili da trattare, inclusi pazienti con mutazioni bialleliche avverse TP53 o FLT3-ITD. L'azienda ha inoltre ricevuto un anticipo di 1,1 milioni di dollari USA da Hanmi Pharmaceutical, portando il totale degli anticipi previsti dal loro accordo di prestito a 5,6 milioni di dollari USA.
Lo studio TUSCANY, condotto in 10 principali centri clinici statunitensi, punta a reclutare 18-24 pazienti entro la fine del 2025, con l'obiettivo di trattare pazienti AML non idonei alla chemioterapia di induzione.
Aptose Biosciences (OTC: APTOF) ha recibido la aprobación para aumentar la dosis a 160 mg en su ensayo de fase 1/2 TUSCANY, que evalúa tuspetinib (TUS) en combinación con venetoclax y azacitidina para pacientes con LMA recién diagnosticada. El ensayo ha completado con éxito tres cohortes de dosis (40 mg, 80 mg y 120 mg) sin toxicidades limitantes de dosis.
La terapia triple TUS+VEN+AZA ha demostrado remisiones completas (CR) y negatividad para enfermedad residual mínima (MRD) en poblaciones difíciles de tratar con LMA, incluyendo pacientes con mutaciones bialélicas adversas TP53 o FLT3-ITD. La compañía también recibió un anticipo de 1,1 millones de dólares estadounidenses de Hanmi Pharmaceutical, elevando el total de anticipos bajo su acuerdo de préstamo a 5,6 millones de dólares.
El ensayo TUSCANY, realizado en 10 principales sitios clínicos de EE.UU., tiene como objetivo inscribir entre 18 y 24 pacientes para finales de 2025, enfocándose en pacientes con LMA no elegibles para quimioterapia de inducción.
Aptose Biosciences (OTC: APTOF)는 새로 진단받은 AML 환자를 대상으로 tuspetinib(TUS)와 venetoclax, azacitidine 병용 요법을 평가하는 1/2상 TUSCANY 임상시험에서 160mg까지 투여량을 증량하는 승인을 받았습니다. 이 임상시험은 40mg, 80mg, 120mg 세 개의 투여군을 성공적으로 완료했으며 투여 제한 독성은 없었습니다.
TUS+VEN+AZA 3제 병용 요법은 불리한 양대성 TP53 또는 FLT3-ITD 돌연변이를 가진 난치성 AML 환자군에서 완전 관해(CR)와 최소 잔류 질환(MRD) 음성을 입증했습니다. 또한 회사는 Hanmi Pharmaceutical로부터 110만 달러 선급금을 받았으며, 대출 계약에 따른 총 선급금은 560만 달러에 이릅니다.
미국 내 10개 주요 임상 기관에서 진행 중인 TUSCANY 임상시험은 2025년 말까지 18~24명의 환자를 등록할 계획이며, 유도 화학요법에 적합하지 않은 AML 환자를 대상으로 합니다.
Aptose Biosciences (OTC : APTOF) a obtenu l'autorisation d'augmenter la dose à 160 mg dans son essai de phase 1/2 TUSCANY, évaluant le tuspetinib (TUS) en combinaison avec le venetoclax et l'azacitidine chez des patients atteints de LMA nouvellement diagnostiquée. L'essai a réussi à compléter trois cohortes de dosage (40 mg, 80 mg et 120 mg) sans toxicités limitantes de dose.
La thérapie triplette TUS+VEN+AZA a démontré des rémissions complètes (RC) et une négativité de la maladie résiduelle minimale (MRD) chez des populations de LMA difficiles à traiter, y compris des patients porteurs de mutations bialléliques défavorables TP53 ou FLT3-ITD. La société a également reçu une avance de 1,1 million de dollars US de Hanmi Pharmaceutical, portant le total des avances dans le cadre de leur accord de prêt à 5,6 millions de dollars US.
L'essai TUSCANY, mené dans 10 principaux centres cliniques américains, vise à recruter 18 à 24 patients d'ici fin 2025, ciblant les patients atteints de LMA non éligibles à la chimiothérapie d'induction.
Aptose Biosciences (OTC: APTOF) hat die Zulassung erhalten, die Dosierung in der Phase-1/2-Studie TUSCANY auf 160 mg zu erhöhen. Diese Studie bewertet Tuspetinib (TUS) in Kombination mit Venetoclax und Azacitidin bei neu diagnostizierten AML-Patienten. Die Studie hat erfolgreich drei Dosierungsgruppen (40 mg, 80 mg und 120 mg) ohne dosislimitierende Toxizitäten abgeschlossen.
Die Dreifachtherapie TUS+VEN+AZA zeigte komplette Remissionen (CRs) und minimale Resterkrankung (MRD) Negativität bei schwer behandelbaren AML-Patienten, einschließlich Patienten mit ungünstigen biallelischen TP53- oder FLT3-ITD-Mutationen. Das Unternehmen erhielt zudem eine Vorauszahlung von 1,1 Mio. USD von Hanmi Pharmaceutical, womit sich die Gesamtvorauszahlungen im Rahmen ihres Darlehensvertrags auf 5,6 Mio. USD erhöhen.
Die TUSCANY-Studie, die an 10 führenden US-Klinikzentren durchgeführt wird, plant die Einschreibung von 18-24 Patienten bis Ende 2025 und richtet sich an AML-Patienten, die für eine Induktionschemotherapie nicht infrage kommen.
- Successful completion of three dosing cohorts with no dose-limiting toxicities
- Achievement of complete remissions and MRD-negativity in difficult-to-treat AML populations
- Received additional US$1.1M funding advance from Hanmi Pharmaceutical
- No safety concerns or prolonged myelosuppression reported in trial patients
- None.
- Safety Review Committee endorses escalation to 160 mg TUS dosing
- Cohorts with 120 mg, 80 mg, 40 mg TUS dosing completed with no dose-limiting toxicities
- Excellent safety and complete remissions (CRs) in some of the most difficult-to-treat AML populations
- No dose reductions required to the standard-of-care VEN/AZA with TUS dose cohorts
- TUS+VEN+AZA triplet continues to achieve CRs and minimal residual disease (MRD)-negativity with favorable safety in newly diagnosed AML patients
- Aptose receives third advance under the Loan Agreement with Hanmi Pharmaceutical
SAN DIEGO and TORONTO, Aug. 06, 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 the Cohort Safety Review Committee (CSRC) monitoring Aptose’s Phase 1/2 TUSCANY trial of TUS in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) has approved escalating from 120 mg TUS dose to 160 mg TUS dose based on its favorable review of safety and efficacy data from patients in the first three cohorts (40 mg, 80 mg, and 120 mg TUS dose levels) of the trial. Enrollment is open for dosing subjects at the 160 mg TUS dose level.
Aptose also announced that it has received an additional advance of US
The TUS+VEN+AZA triplet is being developed as a one-of-a-kind, safe and mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. Aptose reports that at the 120 mg TUS dose level, as with the prior reported 40 mg and 80 mg dose cohorts, no significant safety concerns or dose limiting toxicities (DLTs) have been reported in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission.
All patients treated in the 120 mg dose cohort remain on study while enrollment is open for the 160 mg dose cohort. Aptose previously reported that the first two dose cohorts have demonstrated safety, CRs, and minimal residual disease (MRD) negativity across patients with diverse mutations in an oral presentation at the European Hematology Association Congress (EHA 2025) in June (press release here).
“Data from three cohorts, with a 40, 80 or 120 mg dose of TUS in the TUS+VEN+AZA triplet, continue to build a strong case for TUS as a therapeutic option for some of the most difficult to treat AML populations,” said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. “In particular, patients with adverse biallelic TP53 or FLT3-ITD mutations, and those without FLT3 mutations, were able to safely achieve complete remissions with MRD negativity. After review of the most recent safety and efficacy data, our CSRC recommended that we continue to escalate dosing.”
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. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Indeed, responses were also noted in R/R AML patients with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes.
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 late 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, 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
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