FDA Events for Aptose Biosciences (APTO)
This section highlights FDA-related milestones and regulatory updates for drugs developed by Aptose Biosciences (APTO).
Over the past two years, Aptose Biosciences has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
tuspetinib. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
tuspetinib - FDA Regulatory Timeline and Events
tuspetinib is a drug developed by Aptose Biosciences for the following indication: Tuspetinib is a potent inhibitor of FLT3, SYK, cKITMUT, JAK, and other kinases.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- tuspetinib
- Announced Date:
- February 20, 2025
- Indication:
- Tuspetinib is a potent inhibitor of FLT3, SYK, cKITMUT, JAK, and other kinases
Announcement
Aptose Biosciences Inc announced that the Cohort Safety Review Committee (CSRC) monitoring Aptose's Phase 1/2 TUSCANY trial of tuspetinib in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) has unanimously approved escalating from 40 mg TUS to 80 mg TUS based on its favorable review of data from the first four patients in the trial.
AI Summary
Aptose Biosciences recently announced an important update from its Phase 1/2 TUSCANY trial evaluating tuspetinib combined with venetoclax and azacitidine for newly diagnosed AML patients. The trial’s Cohort Safety Review Committee unanimously approved increasing the dose of tuspetinib from 40 mg to 80 mg after reviewing encouraging data from the first four patients. These early results showed no significant safety concerns or dose-limiting toxicities, with patients experiencing no prolonged myelosuppression. The decision paves the way for enrolling more patients at the higher dosage level, bolstering efforts to develop a frontline therapy that effectively treats difficult-to-treat AML, including cases with TP53 mutations and FLT3-wildtype profiles. This safety approval represents a significant milestone in the TUSCANY trial and reinforces hope that the TUS+VEN+AZA triplet regimen may become a game changer in AML treatment.
Read Announcement- Drug:
- tuspetinib
- Announced Date:
- February 12, 2025
- Indication:
- Tuspetinib is a potent inhibitor of FLT3, SYK, cKITMUT, JAK, and other kinases
Announcement
Aptose Biosciences Inc today reported promising early safety and response results from newly diagnosed acute myeloid leukemia (AML) patients dosed in Aptose's Phase 1/2 TUSCANY trial with a 40 mg dose of tuspetinib in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet).
AI Summary
Aptose Biosciences Inc. announced promising early safety and response results in its Phase 1/2 TUSCANY trial for newly diagnosed acute myeloid leukemia (AML) patients. In the trial, patients received a 40 mg dose of tuspetinib combined with standard dosing of the drugs venetoclax and azacitidine. This TUS+VEN+AZA triplet is aimed at treating a wide range of AML patients who cannot undergo standard induction chemotherapy.
Initial results showed that three patients with FLT3-wildtype AML completed Cycle 1 without dose-limiting side effects, with two achieving complete remissions by the end of the cycle. Notably, one patient with a challenging TP53 mutation and complex karyotype also achieved complete remission in Cycle 1. Pharmacokinetic studies indicate that adding azacitidine did not affect tuspetinib levels, supporting a predictable safety and dosing profile. These early findings suggest that the triplet therapy could offer a new frontline treatment option for diverse AML populations.
Read Announcement- Drug:
- tuspetinib
- Announced Date:
- December 12, 2024
- Indication:
- Tuspetinib is a potent inhibitor of FLT3, SYK, cKITMUT, JAK, and other kinases
Announcement
Aptose Biosciences Inc. announced the publication of preclinical data for Aptose's lead hematology compound tuspetinib (TUS) in Cancer Research Communications
AI Summary
Aptose Biosciences has published new preclinical research on its lead hematology compound, tuspetinib (TUS), in Cancer Research Communications. The study shows that TUS, a well-tolerated, once-daily oral kinase inhibitor, targets several key oncogenic kinases involved in acute myeloid leukemia (AML), including SYK, JAK1/2, and FLT3. The data indicate that TUS not only effectively kills AML cells but also improves survival in various preclinical AML models.
The findings also reveal that TUS works exceptionally well when combined with venetoclax (VEN), creating a synthetic lethality effect that overcomes drug resistance. This combination approach enhances TUS's activity against difficult-to-treat AML cases and supports further clinical development, including trials using a triplet regimen with TUS, VEN, and azacitidine. These promising results could pave the way for a broader antileukemic treatment strategy in AML patients.
Read Announcement- Drug:
- tuspetinib
- Announced Date:
- December 9, 2024
- Indication:
- Tuspetinib is a potent inhibitor of FLT3, SYK, cKITMUT, JAK, and other kinases
Announcement
Aptose Biosciences Inc. today featured a wealth of clinical data for Aptose's lead compound tuspetinib (TUS) in a poster presentation at the 66th American Society of Hematology (ASH) Annual Meeting in San Diego.
AI Summary
Aptose Biosciences Inc. shared new clinical data for its lead compound, tuspetinib (TUS), at the 66th American Society of Hematology Annual Meeting in San Diego. The poster presentation highlighted results from Phase 1/2 trials that demonstrated promising safety and effectiveness of TUS, both on its own and in combination with venetoclax (VEN). Clinical findings showed that TUS-based therapies are active across diverse acute myeloid leukemia (AML) populations, including patients with FLT3 wildtype, which makes up about 70% of AML cases, and those with prior VEN treatment failure. The data suggest that TUS may help prevent resistance to VEN, supporting the advancement of a triplet regimen combining tuspetinib, venetoclax, and azacitidine (AZA) for frontline treatment in newly diagnosed AML patients ineligible for intensive chemotherapy.
Read Announcement- Drug:
- tuspetinib
- Announced Date:
- November 20, 2024
- Indication:
- Tuspetinib is a potent inhibitor of FLT3, SYK, cKITMUT, JAK, and other kinases
Announcement
Aptose Biosciences Inc nnounced initiation of the TUSCANY study, tuspetinib (TUS) in combination therapy with azacitidine (AZA) and venetoclax (VEN) as a frontline triplet combination therapy for patients newly diagnosed with acute myeloid leukemia, or AML. The trial is being conducted at multiple U.S. clinical sites.
AI Summary
Aptose Biosciences Inc. has launched the TUSCANY study to investigate a new frontline treatment for patients newly diagnosed with acute myeloid leukemia (AML). The trial will evaluate a triplet combination of tuspetinib (TUS), azacitidine (AZA), and venetoclax (VEN) in patients ineligible for intensive chemotherapy. Tuspetinib, a once-daily oral agent, targets multiple kinases to potentially reduce the toxicity concerns seen with other treatments while broadening its use to treat a larger AML population.
The study is being conducted at multiple clinical sites across the United States. It will involve varying doses of TUS administered in 28-day cycles, starting from 40mg, with planned dose escalations following a safety review. Approximately 18-24 patients are expected to be enrolled at 12 U.S. sites, with further study updates anticipated at the ASH 2024 meeting.
Read Announcement
Aptose Biosciences FDA Events - Frequently Asked Questions
As of now, Aptose Biosciences (APTO) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Aptose Biosciences (APTO) has reported FDA regulatory activity for tuspetinib.
The most recent FDA-related event for Aptose Biosciences occurred on February 20, 2025, involving tuspetinib. The update was categorized as "Approved," with the company reporting: "Aptose Biosciences Inc announced that the Cohort Safety Review Committee (CSRC) monitoring Aptose's Phase 1/2 TUSCANY trial of tuspetinib in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) has unanimously approved escalating from 40 mg TUS to 80 mg TUS based on its favorable review of data from the first four patients in the trial."
Currently, Aptose Biosciences has one therapy (tuspetinib) targeting the following condition: Tuspetinib is a potent inhibitor of FLT3, SYK, cKITMUT, JAK, and other kinases.
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:APTO) was last updated on 7/9/2025 by MarketBeat.com Staff
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