Aptose Announces First AML Patients Dosed with Tuspetinib Triplet Frontline Therapy in TUSCANY Trial
Aptose Biosciences (NASDAQ: APTO, TSX: APS) has initiated dosing of the first patients in its TUSCANY Phase 1/2 study, testing tuspetinib (TUS) in combination with venetoclax (VEN) and azacitidine (AZA) as a frontline triple therapy for newly diagnosed acute myeloid leukemia (AML) patients.
The TUS+VEN+AZA triplet therapy aims to provide an improved frontline treatment option that is effective across diverse AML populations. Earlier APTIVATE trials showed that TUS, both as a single agent and combined with VEN, demonstrated favorable safety and broad activity in relapsed or refractory AML patients, including those with prior-VEN and prior-FLT3 inhibitor therapies.
The TUSCANY study will evaluate various doses and schedules of TUS with standard dosing of azacitidine and venetoclax in AML patients ineligible for induction chemotherapy. Starting at 40mg once daily, the trial plans to enroll 18-24 patients by mid-late 2025 across multiple U.S. sites.
Aptose Biosciences (NASDAQ: APTO, TSX: APS) ha avviato la somministrazione ai primi pazienti nello studio TUSCANY fase 1/2, che testa il tuspetinib (TUS) in combinazione con venetoclax (VEN) e azacitidina (AZA) come trattamento triplo di prima linea per pazienti con leucemia mieloide acuta (LMA) diagnosticati recentemente.
La terapia TUS+VEN+AZA mira a fornire un'opzione di trattamento di prima linea migliorata, efficace in diverse popolazioni di LMA. Precedenti studi APTIVATE hanno dimostrato che TUS, sia come agente singolo che in combinazione con VEN, ha mostrato una sicurezza favorevole e una vasta attività in pazienti con LMA recidivata o refrattaria, inclusi quelli con terapie precedenti con VEN e inibitori FLT3.
Lo studio TUSCANY valuterà varie dosi e schemi di TUS con dosaggio standard di azacitidina e venetoclax in pazienti con LMA non idonei alla chemioterapia di induzione. Partendo da 40mg una volta al giorno, lo studio prevede di arruolare 18-24 pazienti entro la metà-fine del 2025 in diversi centri negli Stati Uniti.
Aptose Biosciences (NASDAQ: APTO, TSX: APS) ha iniciado la dosificación de los primeros pacientes en su estudio TUSCANY de fase 1/2, que prueba el tuspetinib (TUS) en combinación con venetoclax (VEN) y azacitidina (AZA) como terapia triple de primera línea para pacientes recién diagnosticados con leucemia mieloide aguda (LMA).
La terapia triple TUS+VEN+AZA tiene como objetivo proporcionar una opción de tratamiento de primera línea mejorada que sea efectiva en diversas poblaciones de LMA. Los ensayos APTIVATE anteriores mostraron que TUS, tanto como agente único como en combinación con VEN, demostró una seguridad favorable y una amplia actividad en pacientes con LMA que habían recaído o eran refractarios, incluidos aquellos con terapias previas de VEN e inhibidores de FLT3.
El estudio TUSCANY evaluará varias dosis y horarios de TUS con dosificación estándar de azacitidina y venetoclax en pacientes con LMA no elegibles para quimioterapia de inducción. Comenzando con 40 mg una vez al día, el ensayo planea inscribir de 18 a 24 pacientes para mediados o finales de 2025 en múltiples sitios en EE.UU.
APTOS 바이오사이언스 (NASDAQ: APTO, TSX: APS)는 첫 환자에게 TUSCANY 1/2 단계 연구의 투약을 시작했으며, 여기서 tuspetinib (TUS)를 venetoclax (VEN) 및 azacitidine (AZA)과 조합하여 새롭게 진단된 급성 골수성 백혈병 (AML) 환자를 위한 1차 삼중 요법으로 테스트하고 있습니다.
TUS+VEN+AZA의 삼중 요법은 다양한 AML 집단에서 효과적인 개선된 1차 치료 옵션을 제공하는 것을 목표로 하고 있습니다. 이전 APTIVATE 시험에서는 TUS가 단독 요법으로서 또는 VEN과 결합하여 재발 또는 난치성 AML 환자에서 안전성이 우수하고 폭넓은 활성을 보였음을 보여주었습니다. 여기에는 이전에 VEN 및 FLT3 억제제로 치료를 받은 환자도 포함됩니다.
TUSCANY 연구는 유도 화학요법에 적합하지 않은 AML 환자에서 azacitidine 및 venetoclax의 표준 투약과 함께 TUS의 다양한 용량 및 일정을 평가할 것입니다. 하루 40mg로 시작하여, 연구는 2025년 중반에서 말까지 미국의 여러 장소에서 18-24명의 환자를 등록할 계획입니다.
Aptose Biosciences (NASDAQ: APTO, TSX: APS) a lancé l'administration aux premiers patients dans son étude TUSCANY de phase 1/2, testant le tuspetinib (TUS) en combinaison avec le venetoclax (VEN) et l'azacitidine (AZA) en tant que thérapie triple de première ligne pour les patients nouvellement diagnostiqués avec une leucémie myéloïde aiguë (LMA).
La thérapie TUS+VEN+AZA vise à fournir une option de traitement de première ligne améliorée qui soit efficace dans diverses populations de LMA. Des essais précédents APTIVATE ont montré que TUS, tant en tant qu'agent unique qu'en combinaison avec VEN, a démontré une sécurité favorable et une large activité chez les patients atteints de LMA en rechute ou réfractaire, y compris ceux ayant déjà reçu des thérapies avec VEN et des inhibiteurs de FLT3.
L'étude TUSCANY évaluera différentes doses et horaires de TUS avec des dosages standard d'azacitidine et de venetoclax chez des patients atteints de LMA non éligibles à une chimiothérapie d'induction. Commençant à 40 mg une fois par jour, l'essai prévoit d'enrôler 18 à 24 patients d'ici le milieu ou la fin de 2025 dans plusieurs sites aux États-Unis.
Aptose Biosciences (NASDAQ: APTO, TSX: APS) hat mit der Dosisvergabe an die ersten Patienten in der TUSCANY Phase 1/2 Studie begonnen, in der tuspetinib (TUS) in Kombination mit venetoclax (VEN) und azacitidine (AZA) als 3-fach-Therapie der ersten Wahl für neu diagnostizierte Patienten mit akuter myeloischer Leukämie (AML) getestet wird.
Die TUS+VEN+AZA Dreifachtherapie zielt darauf ab, eine verbesserte Erstlinienbehandlungsoption anzubieten, die in verschiedenen AML-Populationen effektiv ist. Frühere APTIVATE-Studien haben gezeigt, dass TUS, sowohl als Einzelmittel als auch in Kombination mit VEN, eine günstige Sicherheit und eine breite Wirksamkeit bei Patienten mit rezidiverender oder refraktärer AML aufweist, einschließlich solcher mit vorheriger VEN- und FLT3-Hemmertherapie.
Die TUSCANY-Studie wird verschiedene Dosen und Zeitpläne von TUS in Kombination mit der Standarddosierung von azacitidine und venetoclax bei AML-Patienten evaluieren, die nicht für eine Induktionschemotherapie geeignet sind. Beginnend mit 40 mg einmal täglich plant die Studie, bis Mitte bis Ende 2025 in mehreren US-Standorten 18-24 Patienten einzuschreiben.
- Expansion into frontline therapy market with triple combination treatment
- Previous trials showed favorable safety and broad activity across diverse AML populations
- Potential to treat larger AML population regardless of mutation status
- Trial completion not expected until mid-late 2025
- initial enrollment of only 18-24 patients
Insights
The initiation of the TUSCANY Phase 1/2 trial represents a significant milestone in AML treatment development. The triplet combination of tuspetinib (TUS), venetoclax (VEN) and azacitidine (AZA) is particularly noteworthy for several reasons: First, the trial targets newly diagnosed AML patients ineligible for standard induction chemotherapy - a population with treatment options. Second, previous APTIVATE trials showed TUS's effectiveness across diverse AML mutations, including challenging cases like TP53 and RAS mutations, which typically have poor prognoses. The planned enrollment of 18-24 patients by mid-late 2025 suggests a focused, well-controlled study design.
For simpler understanding: Think of this like testing a new "master key" (tuspetinib) that, when combined with two existing "keys" (venetoclax and azacitidine), might unlock treatment options for many different types of AML, rather than just working on specific subtypes. This is especially important because current treatments often work only for specific mutations.
The market potential for this triplet therapy is compelling. Current frontline AML treatments often face limitations due to mutation specificity or toxicity concerns. Tuspetinib's demonstrated activity across multiple mutation types, including resistant populations (prior-VEN and prior-FLT3i failures), positions it as a potentially valuable addition to the AML treatment landscape. The oral administration route adds convenience and could improve patient compliance. For Aptose, with a market cap of
In simple terms: Most current AML drugs are like specialized tools that only work on specific types of leukemia. This new combination therapy is showing promise as a more versatile treatment that could work for many different types of AML patients, which could translate to broader market adoption and better commercial success if approved.
TUS+VEN+AZA triplet has potential as frontline therapy to treat large, mutationally diverse populations of AML
SAN DIEGO and TORONTO, Jan. 09, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company, today announced dosing the first set of patients in the TUSCANY Phase 1/2 study with tuspetinib (TUS) in combination with venetoclax (VEN) and azacitidine (AZA) as a frontline triple drug combination (triplet) therapy for patients newly diagnosed with acute myeloid leukemia, or AML.
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. Responses to TUS were also observed in those with prior-VEN and prior-FLT3 inhibitor (FLT3i) therapies, those with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes. Tuspetinib is a convenient once daily oral agent, and the TUS+VEN+AZA triplet has the potential to treat the larger AML population in a mutation agnostic manner, not just narrow subpopulations.
“We’re excited that our first several patients on the TUSCANY trial have received TUS+VEN+AZA,” said Rafael Bejar, MD, PhD, Aptose’s Chief Medical Officer. “TUS+VEN+AZA triplet therapy holds the promise of delivering high response rates and longer survival to newly diagnosed AML patients, while avoiding toxicities seen with other agents, thereby broadening the application of triplet therapy to more AML patients, including those with adverse disease features.”
TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study
The TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of azacitidine and venetoclax for patients with AML who are ineligible to receive induction chemotherapy. TUS will be administered in 28-day cycles, beginning at 40mg once daily, with dose escalations planned after a safety review of each dose level. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025.
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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