A Phase 1/2 Open Label, Multicenter, Dose Escalation and Expansion Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of HM43239 in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)

The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that is relapsed or refractory after at least one line of prior therapy, or in study participants with newly diagnosed AML. Tuspetinib will be administered as a single agent or in combination with other drugs (venetoclax or venetoclax plus azacitidine), as specified for each part of the study.

开始日期2019-03-11

申办/合作机构

Aptose Biosciences, Inc.

100 项与 Tuspetinib 相关的临床结果

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100 项与 Tuspetinib 相关的转化医学

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100 项与 Tuspetinib 相关的专利（医药）

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项与 Tuspetinib 相关的文献（医药）

2025-05-01·Brain Stimulation

Biophysical effects and neuromodulatory dose of transcranial ultrasonic stimulation

Article

作者: Verhagen, Lennart ; Stagg, Charlotte J ; Naftchi-Ardebili, Kasra ; Nandi, Tulika ; Kop, Benjamin R ; Pauly, Kim Butts

Transcranial ultrasonic stimulation (TUS) has the potential to usher in a new era for human neuroscience by allowing spatially precise and high-resolution non-invasive targeting of both deep and superficial brain regions. Currently, fundamental research on the mechanisms of interaction between ultrasound and neural tissues is progressing in parallel with application-focused research. However, a major hurdle in the wider use of TUS is the selection of optimal parameters to enable safe and effective neuromodulation in humans. In this paper, we will discuss the major factors that determine the efficacy of TUS. We will discuss the thermal and mechanical biophysical effects of ultrasound, which underlie its biological effects, in the context of their relationships with tunable parameters. Based on this knowledge of biophysical effects, and drawing on concepts from radiotherapy, we propose a framework for conceptualising TUS dose.

2025-04-01·Journal of Cosmetic Dermatology

A Novel Technology to Boost Natural Production of Hyaluronic Acid in the Skin Tissue: Human Histology Study

Article

作者: Goldberg, David J.

ABSTRACT:

Introduction:

Combining monopolar radiofrequency (RF) with targeted ultrasound (TUS), this study investigated whether these modalities promote facial rejuvenation through the production of hyaluronic acid (HA) in human skin.

Methods:

Seven subjects (51–64 years, BMI 21.1–29.8 kg/m2) were enrolled and divided into three treatment groups in this single‐center study; Group A (n = 3, simultaneous RF + TUS), Group B (n = 3, stand‐alone RF), and control (n = 1, no treatment). Both treated groups underwent four (4) 60‐min treatments on the face delivered 7–14 days apart. Punch biopsies (3 mm in diameter) were collected from the infra‐auricular area at baseline and both follow‐up visits and stained for HA by using hyaluronic acid binding protein. Digital photographs were taken to document changes in visual appearance. Finally, the subjects' comfort and satisfaction were assessed.

Results:

There was a statistically significant (p < 0.05) average increase at 1 month in the HA‐stained area of +112 358.7 μm2 in group A (RF + TUS) representing an increase of 48.65%. The treatment effect peaked at 3 months with an increase of +156 345.2 μm2, corresponding to a 67.69% increase in the HA‐stained area. In Group B, there was no significant difference in the average increase of the HA‐stained area between 1 month (+14 830 μm2) and 3 months (+20 995 μm2) corresponding to a 6.76% and 9.56% increase, respectively. The control samples did not indicate any changes throughout the study. Digital photographs of the RF + TUS group showed both a decrease in rhytids and tighter skin. Therapies were comfortable with no adverse events.

Conclusion:

Overall, this study has shown that the combination treatment of RF + TUS has a more pronounced and sustained effect on facial rejuvenation compared to RF alone. The measurable increase in the production of HA with simultaneous use of RF + TUS peaked at a 3‐month follow‐up, suggesting the gradual advancement of the treatment effect and overall improvement in facial appearance.

Trial Registration:

ClinicalTrials.gov identifier: NCT05987917

2025-04-01·JOURNAL OF DAIRY SCIENCE

The association of lung consolidation and respiratory pathogens identified at weaning on the growth performance of beef-on-dairy calves

Article

作者: Felix, T L ; Renaud, D L ; Fernandes, I L B ; Sockett, D ; Cantor, M C ; Welk, A

This observational study evaluated the relationship between lung consolidation (LC) observed at weaning and calf ADG, and the association of pathogen shedding at weaning on ADG in beef × dairy calves up to 238 d. Beef × Holstein calves (n = 143) were sourced from 2 dairies. Calves were managed in 3 cohorts and fed milk replacer and calf starter before weaning. Calves were transported to another facility after weaning and raised in one group, where they were fed calf starter with oat hay and transitioned to a corn silage-based TMR diet. Calf ADG was calculated from arrival to weaning at 61 ± 14 d (period 1), from weaning to 83 ± 21 d (period 2), and from 83 d to 238 ± 21 d (period 3). Thoracic ultrasonography (TUS) was performed at weaning to evaluate if a calf had LC (characterized as TUS+ if ≥1 cm² in one lobe) and to categorize the degree of LC found (none [TUS-], 1-2 cm², or = 3 cm²). Nasopharyngeal swabs were taken from the TUS+ calves and from pair-matched TUS- calves (n = 35 pairs) for pathogen identification by culture at a diagnostic laboratory. A mixed linear regression model assessed the association of LC with calf ADG with LC, period, period × LC, and sire breed as fixed effects; arrival weight as a covariate; and calf nested within the cohort as a random effect. Another mixed linear regression model assessed the association of pathogen shedding with calf ADG from weaning to 238 d with period and sire breed as fixed effects, and pair was nested within cohort as a random effect. A logistic regression model was used to evaluate the likelihood of TUS+ calves shedding a pathogen with pair as a fixed effect. We found an LC × period interaction affecting ADG over period 2 where TUS- calves had increased ADG (1.18 ± 0.02 kg/d) compared with calves with LC = 3 cm² (1.03 ± 0.04 kg/d). However, TUS- calves had similar ADG to calves with LC = 1 to 2 cm² in period 2. Calf ADG was not associated with LC in period 3, and calves weighed 324 ± 37 kg (mean ± SD) at 238 d. In addition, 57% (20/35) of TUS+ calves and 26% (9/35) of TUS- calves shed Pasteurella multocida. We found no association of pathogen shedding with calf ADG, but TUS+ calves were more likely to shed a pathogen. These findings suggest that calves with pneumonia experienced poor growth up to 20 d postweaning, but compensatory gain occurred by 238 d. Furthermore, P. multocida was not associated with growth performance up to 238 d in beef × dairy calves.

项与 Tuspetinib 相关的新闻（医药）

2025-05-14

·aptose.com

Aptose Selected for Prestigious Oral Presentation of Data from TUSCANY Phase 1/2 Clinical Trial of Tuspetinib Triplet Therapy in Newly Diagnosed AML at the 2025 EHA Congress

Aptose is developing TUS+VEN+AZA as a one-of-a-kind safe and mutation agnostic frontline triple drug therapy for newly diagnosed AML Oral presentation at EHA will include updated data at the 40 mg and 80 mg dose levels and longer duration SAN DIEGO and TORONTO, May 14, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (TSX: APS; OTC: APTOF), a clinical-stage precision oncology company, today announced that data from its Phase 1/2 TUSCANY trial in newly diagnosed patients treated with tuspetinib (TUS) in combination with standard of care dosing venetoclax and azacitidine (TUS+VEN+AZA triplet) has been selected for oral presentation at the European Hematology Association Congress (EHA 2025), being held June 12-15, 2025, in Milan, Italy. The TUS+VEN+AZA triplet is being developed as the only safe and mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. As reported prior, the first two dose cohorts at 40 mg of TUS or 80 mg of TUS in the TUS+VEN+AZA triplet, have demonstrated safety, complete remissions, and MRD negativity across patients with diverse mutations, including TP53-mutated/CK AML and FLT3-wildtype AML patients. The oral presentation at EHA will include updated safety, complete remission, minimal residual disease (MRD) clinical findings, and longer duration of follow-up. Details of the presentation are as follows: Title: 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 Session: Oral Presentations: Acute Myeloid Leukemia – Clinical Session Date and Time: Thursday, June 12, 2025, 5:00 – 6:15 pm CEST Presenter: Dr. Gabriel Mannis, Associate Professor of Medicine, Stanford University School of Medicine Abstract #: S139 Abstracts are available on the EHA2025 website 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 clinical study 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 in R/R AML patients with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes. The TUSCANY 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 mid-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 Released May 14, 2025

临床研究临床结果

2025-05-11

·药明康德

使“癌症之王”患者总生存期优于历史对照的小分子疗法；使多名患者肿瘤完全消失的组合疗法…… | 一周盘点

本期看点1. 在一项早期临床试验中，tuspabetinib（TUS）联用标准剂量的venetoclax和azacitidine的三联疗法使多名急性髓系白血病（AML）患者的肿瘤细胞完全消失。2. 放射增敏剂JNJ-1900用于局部晚期或临界可切除胰腺癌患者的1期临床试验结果亮眼，患者的中位总生存期（OS）优于历史对照组，并有两名患者成功实现R0根治性手术切除。3. 大环MEK抑制剂PAS-004的药效学（PD）数据积极，对评估MEK抑制剂活性的金标准PD生物标志物的抑制率高达91%。药明康德内容团队整理Tuspetinib：公布1/2期联合治疗试验的新数据Aptose Biosciences公司公布了其针对新确诊的AML患者开展的1/2期TUSCANY试验的新数据。这些患者分别接受了40毫克或80毫克剂量的tuspabetinib联用标准剂量的venetoclax和azacitidine（TUS+VEN+AZA三联疗法）。该三联疗法正在被开作发为一种安全且不依赖于特定突变的一线治疗方案，用于治疗那些无法接受诱导化疗的新诊断AML患者，这些患者具有多样的突变。在两种剂量下，均有多名携带不同突变（包括TP53突变/复杂核型、FLT3野生型）的患者达到了完全缓解（CR）或伴有血液学不完全恢复的完全缓解（CRi），40毫克剂量组中达到CR/CRi的3名患者的最小残留病（MRD）状态均为阴性。安全性方面，该三联疗法的安全性良好，在两个剂量水平下均未观察到剂量限制性毒性。JNJ-1900（NBTXR3）：公布1期临床试验数据Nanobiotix公司公布了其潜在“first-in-class”的新型放射增敏剂JNJ-1900（NBTXR3）用于局部晚期或临界可切除胰腺癌患者的1期临床试验数据。NBTXR3由功能化二氧化铪（HfO2）纳米颗粒组成，经由一次性瘤内注射给药并通过放射疗法激活。它的物理作用机制为：通过放疗激活，诱导被注射肿瘤内大量的肿瘤细胞死亡，随后触发适应性免疫反应和长期的抗癌记忆。得益于该物理作用机制，Nanobiotix公司认为NBTXR3可扩展到任何可以通过放疗治疗的实体肿瘤和任何联合治疗方案中，特别是与免疫检查点抑制剂联合。2023年，Nanobiotix宣布与强生旗下杨森公司（现名为强生创新制药）达成全球共同开发和商业化NBTXR3的许可协议。此次公布的研究结果显示，22名接受JNJ-1900治疗的患者的中位OS达到23个月（从诊断日期开始计算），优于历史对照组（19.2个月）；中位局部无进展生存期（PFS）为13.3个月（从放疗结束时开始计算）；两名患者成功实现R0根治性手术切除。探索性生物标志物分析显示，循环肿瘤突变负荷（cTMB）升高与更长的局部PFS和OS相关；CA19-9肿瘤标志物在59%的患者中恢复正常，同样与更长的OS相关。这些结果支持在随机对照试验中进一步评估JNJ-1900。PAS-004：公布1期临床试验数据Pasithea Therapeutics公司公布了其大环MEK抑制剂PAS-004治疗MAPK通路驱动的晚期实体肿瘤患者的1期临床研究的PD数据。与目前FDA批准的MEK抑制剂不同，PAS-004是大环化合物，环化可增强药物与靶受体的结合，被认为有望改善药代动力学和安全性。ERK磷酸化（pERK）的抑制被广泛认为是评估MEK抑制剂活性的金标准PD生物标志物。为了评估PAS-004的靶点作用，研究人员在基线和第22天稳态时检测了患者外周血单核细胞（PBMCs）中的pERK水平。结果显示，PAS-004对pERK的抑制率高达91%，证实其具有强大的靶点作用。该结果得到了初步临床观察的支持，表现为多名患者实现了疾病稳定（SD）和肿瘤缩小。其中，一名IV期携带KRAS G12R突变的胰腺癌患者已获得超过5个月的SD，肿瘤体积缩小了9.8%。REC-4881：公布1b/2期临床试验数据Recursion公司公布了其正在进行的1b/2期TUPELO试验中REC-4881的初步安全性和疗效结果。REC-4881是一种正在被开发用于治疗家族性腺瘤性息肉病（FAP）的选择性变构MEK1/2抑制剂。FAP是一种由APC基因突变引起的罕见遗传性疾病，患者若不治疗，几乎100%会发展为结直肠癌。目前，该病尚无获得美国FDA批准的治疗方法。REC-4881已获得FDA和欧洲药品管理局授予的孤儿药资格及FDA的快速通道资格。截至2025年3月17日的数据，在接受4 mg每日一次治疗的6例可评估患者中，第13周时息肉负荷的中位降幅为43%，其中83%的患者（5人）的降幅范围为31%至82%，1例患者的息肉负荷较基线大幅增加。50%的患者实现了Spigelman分期（衡量上消化道疾病严重程度的指标）改善≥1分。REC-4881的早期安全性特征与以往的MEK1/2抑制剂相似。在1b期和2期研究里的19名患者中，大多数治疗相关不良事件为1/2级，16%的患者发生了3级不良事件，目前未报告任何4级及以上的治疗相关不良事件。JANX007：启动1b期扩展研究Janux Therapeutics公司宣布在未接受过紫杉烷类药物的转移性去势抵抗性前列腺癌（mCRPC）患者中启动1b期扩展研究，该研究的启动得到了此前公布的1a期剂量递增研究数据的支持。JANX007是一款靶向前列腺特异性膜抗原（PSMA）的肿瘤激活T细胞接合器，分别靶向PSMA和T细胞表面表达的CD3受体。它的设计让JANX007在肿瘤微环境中经过蛋白酶切割而被激活，从而在降低了其潜在毒性的同时，最大化抗肿瘤免疫反应。截至2025年4月21日的数据，16名mCRPC患者的中位影像学PFS为7.5个月，6个月时的影像学无进展生存率为65%；接受6 mg和9 mg治疗剂量的9名患者的中位影像学PFS为7.9个月，6个月时的影像学无进展生存率为78%。安全性数据与2024年12月披露的数据保持一致。SPY001：公布1期临床试验的新数据Spyre Therapeutics公司公布了其抗体疗法SPY001的1期临床试验的新数据。SPY001是一种高效、具有选择性的抗α4β7单克隆抗体，采用工程化改造延长半衰期，正在被开发用于治疗炎症性肠病（IBD）。长达8个月的随访结果显示，SPY001具有良好的耐受性，药代动力学（PK）数据显示，其半衰期长达约80天，是现有标准治疗之一的vedolizumab的三倍以上。PD数据显示，在预期的2期谷浓度，单次给药SPY001仍能维持对靶点的持续作用，导致α4β7受体快速、持续饱和。该数据进一步支持SPY001在更高暴露量下可能实现更优的诱导治疗反应，以及具有每季度或每半年给药一次的潜力。参考资料：[1] Perfuse Therapeutics Announces Positive Results from the Completed Phase 1/2a Clinical Trial of PER-001 Intravitreal Implant in Patients with Glaucoma. Retrieved May 9, 2025, from https://www.prnewswire.com/news-releases/perfuse-therapeutics-announces-positive-results-from-the-completed-phase-12a-clinical-trial-of-per-001-intravitreal-implant-in-patients-with-glaucoma-302446082.html[2] IDEAYA Biosciences Announces US FDA IND-Clearance for IDE849, a Potential First-in-Class DLL3 TOP1 ADC, for a Phase 1 Study in Solid Tumors. Retrieved May 9, 2025, from https://www.prnewswire.com/news-releases/ideaya-biosciences-announces-us-fda-ind-clearance-for-ide849-a-potential-first-in-class-dll3-top1-adc-for-a-phase-1-study-in-solid-tumors-302446498.html[3] Cohen, R.B., Jimeno, A., Hreno, J. et al. Safety, tolerability, and preliminary efficacy of nadunolimab, an anti-IL- 1 receptor accessory protein monoclonal antibody, in combination with pembrolizumab in patients with solid tumors. Invest New Drugs (2025). https://doi.org/10.1007/s10637-025-01538-3[4] Opus Genetics Announces Presentation of OPGX-LCA5 Gene Therapy Data at ARVO; 12 Month Phase 1/2 Results Support Potential to Restore to Meaningful Vision. Retrieved May 9, 2025, from https://ir.opusgtx.com/press-releases/detail/484/opus-genetics-announces-presentation-of-opgx-lca5-gene-therapy-data-at-arvo-12-month-phase-12-results-support-potential-to-restore-to-meaningful-vision[5] Janux Therapeutics Initiates Phase 1b Expansion Studies with JANX007 in Patients with Prostate Cancer and Provides Program Updates. Retrieved May 9, 2025, from https://investors.januxrx.com/investor-media/news/news-details/2025/Janux-Therapeutics-Initiates-Phase-1b-Expansion-Studies-with-JANX007-in-Patients-with-Prostate-Cancer-and-Provides-Program-Updates/default.aspx[6] Lantern Advances Drug Candidate LP-184 with IND Clearance for Phase 1b/2 Clinical Trial in Triple Negative Breast Cancer (TNBC). Retrieved May 9, 2025, from https://ir.lanternpharma.com/news-events/press-releases/detail/177/lantern-advances-drug-candidate-lp-184-with-ind-clearance[7] Spyre Therapeutics Announces Poster Presentations at Digestive Disease Week (DDW) 2025 Including Up to Eight months of Follow-up from an Ongoing Phase 1 Trial of SPY001. Retrieved May 9, 2025, from https://www.prnewswire.com/news-releases/spyre-therapeutics-announces-poster-presentations-at-digestive-disease-week-ddw-2025-including-up-to-eight-months-of-follow-up-from-an-ongoing-phase-1-trial-of-spy001-302445258.html[8] Aptose Provides Clinical Update for the Tuspetinib-based Triple Drug Frontline Therapy in Newly Diagnosed AML Patients from the Phase 1/2 TUSCANY Trial. Retrieved May 9, 2025, from https://www.aptose.com/news-media/press-releases/detail/317/aptose-provides-clinical-update-for-the-tuspetinib-based[9] Regeneration Biomedical to Present Updated Phase 1 Trial Data on Autologous Stem Cell Therapy Injected Directly into the Brain for Alzheimer’s Disease in Podium Presentation at the ISCT 2025 Scientific Annual Meeting. Retrieved May 9, 2025, from https://www.globenewswire.com/news-release/2025/5/5/3073944/0/en/Regeneration-Biomedical-to-Present-Updated-Phase-1-Trial-Data-on-Autologous-Stem-Cell-Therapy-Injected-Directly-into-the-Brain-for-Alzheimer-s-Disease-in-Podium-Presentation-at-the.html[10] Nanobiotix Announces Full Results From Completed Phase 1 Study Evaluating JNJ-1900 (NBTXR3) in Pancreatic Cancer. Retrieved May 9, 2025, from https://ir.nanobiotix.com/news-releases/news-release-details/nanobiotix-announces-full-results-completed-phase-1-study[11] Preliminary Phase 1b/2 Data for REC-4881 in Familial Adenomatous Polyposis (FAP) Demonstrates Reduced Polyp Burden. Retrieved May 9, 2025, from https://ir.recursion.com/news-releases/news-release-details/preliminary-phase-1b2-data-rec-4881-familial-adenomatous[12] TransCode Therapeutics Reports Further Progress on Phase 1a Clinical Trial with No Dose Limiting Toxicities Reported in Patients with Metastatic Cancer. Retrieved May 9, 2025, from https://www.prnewswire.com/news-releases/transcode-therapeutics-reports-further-progress-on-phase-1a-clinical-trial-with-no-dose-limiting-toxicities-reported-in-patients-with-metastatic-cancer-302443677.html[13] Design Therapeutics Announces Favorable Phase 1 Data for DT-168 Supporting Advancement into Phase 2 Biomarker Trial for Patients with Fuchs Endothelial Corneal Dystrophy. Retrieved May 9, 2025, from https://investors.designtx.com/news-releases/news-release-details/design-therapeutics-announces-favorable-phase-1-data-dt-168[14] Pasithea Therapeutics Reports Positive Pharmacodynamic Results Demonstrating Robust Target Engagement from its Ongoing Phase 1 Clinical Trial of PAS-004. Retrieved May 9, 2025, from https://www.globenewswire.com/news-release/2025/05/06/3074902/0/en/Pasithea-Therapeutics-Reports-Positive-Pharmacodynamic-Results-Demonstrating-Robust-Target-Engagement-from-its-Ongoing-Phase-1-Clinical-Trial-of-PAS-004.html[15] Aspen Neuroscience Announces 6-Month ASPIRO Phase 1/2a Clinical Trial Results of Personalized Cell Therapy for Parkinson's Disease. Retrieved May 9, 2025, from https://www.prnewswire.com/news-releases/aspen-neuroscience-announces-6-month-aspiro-phase-12a-clinical-trial-results-of-personalized-cell-therapy-for-parkinsons-disease-302448009.html[16] Arbutus Presents Clinical Trial Data from its Two HBV Assets, Imdusiran and AB-101, at the European Association for the Study of the Liver (EASL) Congress 2025. Retrieved May 9, 2025, from https://investor.arbutusbio.com/news-releases/news-release-details/arbutus-presents-clinical-trial-data-its-two-hbv-assets[17] CRISPR Therapeutics Provides First Quarter 2025 Financial Results and Announces Positive Top-Line Data from Phase 1 Clinical Trial of CTX310™ Targeting ANGPTL3. Retrieved May 7, 2025 from https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-provides-first-quarter-2025-financial[18] Marengo Doses First Patient in STARt-002 Clinical Trial Evaluating First-in-Class Dual T Cell Agonist, Invikafusp Alfa in Combination with TROP2-directed ADC Trodelvy® in Metastatic Breast Cancer. Retrieved May 7, 2025 from https://www.prnewswire.com/news-releases/marengo-doses-first-patient-in-start-002-clinical-trial-evaluating-first-in-class-dual-t-cell-agonist-invikafusp-alfa-in-combination-with-trop2-directed-adc-trodelvy-in-metastatic-breast-cancer-302449274.html[19] Phio Pharmaceuticals Announces Positive Pathology Results in Third Cohort in INTASYL PH-762 Skin Cancer Clinical Trial. Retrieved May 9, 2025, from https://phiopharma.com/phio-pharmaceuticals-announces-positive-pathology-results-in-third-cohort-in-intasyl-ph-762-skin-cancer-clinical-trial/免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新2

放射疗法临床1期临床结果临床2期

2025-05-08

·GlobeNewswire-Health Care

Aptose Reports First Quarter 2025 Results

Highlights Progress in TUSCANY Clinical Trial of Tuspetinib in AML Triple Frontline TherapySAN DIEGO and TORONTO, May 08, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (TSX: APS and OTC: APTOF), a clinical-stage precision oncology company developing a tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed acute myeloid leukemia (AML), today announced financial results for the first quarter ended March 31, 2025, and provided a corporate update. “Our TUSCANY clinical trial of tuspetinib in combination with venetoclax (VEN) and azacitidine (AZA) for frontline treatment of newly diagnosed acute myeloid leukemia (AML) continues to deliver robust safety and response data, with support and enthusiasm from our clinical investigators,” said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. “We recently escalated the TUS dose to 80 mg from the initial dose of 40 mg in the TUS+VEN+AZA triplet therapy. Three patients receiving the initial dose of 40 mg and three patients receiving the 80 mg dose all have achieved complete remissions (CRs*) and continue on treatment. We are especially encouraged that two patients having highly adverse TP53 mutations have achieved objective responses and remain on treatment.” Key Corporate Highlights Tuspetinib Data Reported from First Two Cohorts of TUSCANY Phase 1/2 Trial - Tuspetinib based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating a one-of-a-kind frontline therapy for newly diagnosed AML patients that is safe and active across diverse AML populations (mutation agnostic triplet frontline therapy), including patients without FLT3 mutations (wildtype FLT3). Data from the first two cohorts, with a 40 mg or 80 mg dose of tuspetinib in the TUS+VEN+AZA combination, reveal promising clinical safety and antileukemic activity (press release here). In the first cohort, our newly diagnosed AML patients received the initial 40 mg dose of TUS as part of the TUS+VEN+AZA combination. No safety concerns or dose limiting toxicities (DLTs) were observed. Three patients experienced rapid bone marrow blast reductions to achieve CRs; one having biallelic mutated TP53, complex karyotype (CK) chromosomal abnormalities, and wildtype FLT3; one having an IDH2 mutation wildtype FLT3; and one having FLT3-ITD and mutated NPM1. Clinical sites or central labs reported all these patients also achieved MRD-negative status. The fourth patient at the 40 mg dose of TUS, did not achieve a complete remission and discontinued the study. The first three patients continue on treatment. In the second cohort, three newly diagnosed AML patients having diverse mutation profiles have received the 80 mg dose of TUS, as part of the TUS+VEN+AZA combination. To date, no safety concerns or DLTs have been reported. All patients at the 80 mg dose of TUS rapidly achieved CRs; one patient with biallelic TP53 mutations and a complex karyotype and wildtype FLT3 status, a second patient having mutated DDX41 and wildtype FLT3 status, and a third patient having FLT3-ITD and NPM1 mutations. All three patients at the 80 mg dose of TUS are early in their course of treatment, are expected to achieve normal blood count recovery as their leukemia is resolved, and MRD status will be monitored as the patients move through their courses of therapy. OTC Exchange - Aptose common shares (“the Common Shares”) are now listed for trading on the OTC Markets (OTC) under the ticker “APTOF,” in addition to the Company’s continued listing on the Toronto Stock Exchange (TSX) under the symbol “APS”. This significantly expands Aptose accessibility for U.S.-based investors and enhances overall share liquidity by enabling trading through U.S. broker-dealers. Listing on the OTC Markets is part of Aptose’s strategy to align with TSX capital markets while increasing visibility and participation from the U.S. investment community. Aptose’s Common Shares were delisted from The Nasdaq Stock Market effective April 2, 2025 because of non-compliance with the Exchange’s equity requirements. Completed and Planned Value-Creating Milestones 2025: 1H Reported safety and efficacy with 40mg TUS+VEN+AZAReported safety and efficacy with 80mg TUS+VEN+AZA 2025: European Hematology Association (EHA) Report maturing data from TUS+VEN+AZA triplet study 2025: American Society of Hematology (ASH) Report response rate and durability of TUS+VEN+AZA tripletSelect TUS dose for TUS+VEN+HMA triplet Ph 2/3 PIVOTAL trialsPrepare for initiation of Ph 2/3 PIVOTAL program FINANCIAL RESULTS OF OPERATIONSAptose Biosciences Inc.Statements of Operations Data(unaudited)($ in thousands, except for share and per share data) Quarter endedMarch 31, 2025 2024 Expenses: Research and development$2,364 $6,445 General and administrative 3,097 3,315 Operating expenses 5,461 9,760 Other (loss) income, net (82) 120 Net loss$(5,543) $(9,640) Net loss per share, basic and diluted$(2.61) $(22.02) Weighted average number ofcommon shares outstanding used incomputing net loss per share, basicand diluted 2,126,287 437,750 Net loss for the quarter ended March 31, 2025 decreased by $4.1 million to $5.5 million, as compared to $9.6 million for the comparable period in 2024. Aptose Biosciences Inc.Balance Sheet Data(unaudited)($ in thousands) March 31,2025 December 31,2024 Cash, cash equivalents and restricted cashequivalents$4,743 $6,707 Working capital 651 5,053 Total assets 7,467 10,127 Long-term liabilities 8,542 10,193 Accumulated deficit (546,510) (540,967) Shareholders’ deficit (7,393) (4,543) Total cash, cash equivalents and restricted cash equivalents as of March 31, 2025 were $4.7 million. Based on current operations, the Company expects that cash on hand and available capital provides the Company with sufficient resources to fund planned Company operations including research and development until the end of May 2025. The Company is proactively implementing financing and cost reduction efforts to extend its cash runway. As of May 1, 2025, we had 2,552,429 Common Shares issued and outstanding. In addition, there were 38,736 Common Shares issuable upon the exercise of outstanding stock options and there were 1,267,585 Common Shares issuable upon the exercise of the outstanding warrants. RESEARCH AND DEVELOPMENT EXPENSES Research and development expenses for the three months ended March 31, 2025 and 2024 were as follows: Quarter ended (in thousands) March 31,2025 March 31,2024 Program costs – Tuspetinib$1,479 $3,923 Program costs – Luxeptinib 98 208 Program costs – APTO-253 - 22 Personnel related expenses 646 1,924 Stock-based compensation 141 328 Depreciation of equipment - 10 Total$2,364 $6,445 Research and development expenses decreased by $4.1 million to $2.3 million for the quarter ended March 31, 2025, as compared to $6.4 million for the comparable period in 2024. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events: Program costs for tuspetinib were $1.5 million for the quarter ended March 31, 2025, compared with $3.9 million for the comparable period in 2024. The lower program costs for tuspetinib in the current period are attributable to reduced activity in our APTIVATE clinical trial, reduced manufacturing activity, and related expenses. Program costs for luxeptinib decreased by approximately $0.1 million primarily due to lower clinical trial and manufacturing activities. Program costs for APTO-253 were zero. This was due to the Company’s decision to discontinue further development of APTO-253. Personnel-related expenses decreased by $1.3 million due to lower headcount for research and development personnel in the current quarter. Stock-based compensation decreased by approximately $0.2 million in the quarter ended March 31, 2025, primarily due stock options forfeited and/or vested in prior periods that are no longer being expensed resulting in lower expense in the current period. 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 small molecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. The Company’s lead clinical-stage compound tuspetinib (TUS), is an oral kinase inhibitor that 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 contains forward-looking statements within the meaning of Canadian and U.S. securities laws, including, but not limited to, statements regarding the Company’s clinical development plans, the clinical potential, anti-cancer activity, therapeutic potential and applications and safety profile of tuspetinib, clinical trials, upcoming milestones, financing activities, expectations regarding capital available to the Company to fund planned Company operations, the Company’s cash runway, use of proceeds from financings, and statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “hope”, “should”, “would”, “may”, “potential” 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; 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 and economic conditions; unexpected manufacturing defects, the evolving regulatory and political landscape and the funding of government programs and other risks detailed from time-to-time in our ongoing current reports, quarterly filings, annual information forms, annual reports and 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 PietropaoloCorporate Communications & Investor Relations201-923-2049spietropaolo@aptose.com

临床结果财报ASH会议

100 项与 Tuspetinib 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15343

研发状态

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适应症	最高研发状态	国家/地区	公司	日期
成人急性髓性白血病	临床2期	美国	Aptose Biosciences, Inc.	2019-03-11
成人急性髓性白血病	临床2期	澳大利亚	Aptose Biosciences, Inc.	2019-03-11
成人急性髓性白血病	临床2期	德国	Aptose Biosciences, Inc.	2019-03-11
成人急性髓性白血病	临床2期	新西兰	Aptose Biosciences, Inc.	2019-03-11
成人急性髓性白血病	临床2期	西班牙	Aptose Biosciences, Inc.	2019-03-11
慢性粒单核细胞白血病	临床2期	美国	Aptose Biosciences, Inc.	2019-03-11
慢性粒单核细胞白血病	临床2期	澳大利亚	Aptose Biosciences, Inc.	2019-03-11
慢性粒单核细胞白血病	临床2期	德国	Aptose Biosciences, Inc.	2019-03-11
慢性粒单核细胞白血病	临床2期	新西兰	Aptose Biosciences, Inc.	2019-03-11
慢性粒单核细胞白血病	临床2期	西班牙	Aptose Biosciences, Inc.	2019-03-11

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03850574 (APTIVATE \| TUSCANY, NEWS) 人工标引	临床1/2期	急性髓性白血病	-	tuspabetinib+venetoclax+azacitidine	鬱夢餘醖積願構襯構簾(構鑰網構願製製鑰簾淵) = 在两种剂量下，均有多名携带不同突变（包括TP53突变/复杂核型、FLT3野生型）的患者达到了完全缓解（CR）或伴有血液学不完全恢复的完全缓解（CRi），40毫克剂量组中达到CR/CRi的3名患者的最小残留病（MRD）状态均为阴性。網窪艱獵鏇製築鬱構鹹 (網獵壓鏇窪艱艱繭夢顧 ) 更多	积极	2025-05-12
NCT03850574 (TUSCANY, Company_Website 1 \| Company_Website 2) 人工标引	临床1/2期	急性髓性白血病一线 TP53 Mutation	4	tuspetinib+venetoclax+azacitidine	鹽醖壓選鑰製夢簾餘醖(願獵鹹繭壓憲齋製積鑰) = 齋襯觸構積顧繭網積鬱憲願糧膚衊顧膚顧鹽膚 (醖膚築鏇願顧範壓窪顧 )	积极	2025-02-12
ASH2024 人工标引	临床1期	急性髓性白血病 RAS Mutation (Activating) \| FLT3 Mutation \| TP53 Mutation	172	Tuspetinib 80mg	壓網鏇淵選憲艱膚壓鹽(簾積壓簾衊蓋憲衊選窪) = 醖糧獵積艱廠衊遞網選糧選築膚鬱壓壓網憲餘 (範簾壓繭醖糧獵蓋構範 )	积极	2024-12-09
				Tuspetinib (VEN-naïve)	壓網鏇淵選憲艱膚壓鹽(簾積壓簾衊蓋憲衊選窪) = 製製艱衊顧製鬱網願願糧選築膚鬱壓壓網憲餘 (範簾壓繭醖糧獵蓋構範 )
NCT03850574 (APTIVATE, GlobeNewswire) 人工标引	临床1期	急性髓性白血病 FLT3 Mutation	172	Tuspetinib 80 mg (FLT3 mutated)	襯窪鑰淵築繭糧艱鬱衊(獵構構構構鑰繭選鬱廠) = 鑰製餘製窪鏇艱夢觸獵鬱網構襯艱選獵齋積窪 (觸蓋遞積遞構鏇糧衊蓋 ) 更多	积极	2024-12-09
				TuspetinibTuspetinib 80 mg (all-comer VEN-naïve)	襯窪鑰淵築繭糧艱鬱衊(獵構構構構鑰繭選鬱廠) = 顧願顧鏇壓選鑰淵夢齋鬱網構襯艱選獵齋積窪 (觸蓋遞積遞構鏇糧衊蓋 )
NCT03850574 (EHA2024) 人工标引	临床1/2期	急性髓性白血病	170	Tuspetinib (TUS) 20-200mg	蓋淵淵觸餘鏇窪壓壓蓋(壓鹽淵糧簾糧製艱選糧) = 蓋顧襯壓齋淵鏇憲壓製築夢齋鬱膚鏇壓膚築淵 (鑰繭艱簾鹹艱選獵鹽淵 ) 更多	积极	2024-05-14
				Tuspetinib Venetoclax/Venetoclax (VEN) 400mg	遞糧齋膚糧壓餘構鹹積(鹹醖憲醖衊壓積鏇窪選) = 壓窪繭鬱窪齋構襯觸糧餘餘積築簾糧艱鹹糧廠 (壓鹽積願網簾範觸鑰艱 ) 更多
NCT03850574 (APTIVATE, ASH2023) 人工标引	临床1/2期	急性髓性白血病	100	Tuspetinib 20mg to 200mg (Parts A/B)	艱淵鬱網鹽網網遞製構(鏇鏇糧壓範壓製鑰觸願) = One patient experienced a DLT (Grade 3 muscular weakness) at 200 mg which demonstrated partial improvement at the time of treatment discontinuation (9 days after AE onset). 觸餘襯艱範範範簾鏇襯 (鬱蓋鑰簾鹹壓餘鏇鹽蓋 ) 更多	积极	2023-12-09
				Tuspetinib 120 mg (Part C)
NCT03850574 (APTIVATE, GlobeNewswire) 人工标引	临床1/2期	急性髓性白血病 FLT3 Mutation	117	Tuspetinib	築願鏇蓋壓廠憲鏇壓鹽(鬱衊醖鏇構襯範觸鑰醖) = 醖膚憲淵獵醖構繭餘鑰鑰鏇鏇淵膚範醖選膚淵 (獵廠膚範膚襯淵鏇窪簾 ) 更多	积极	2023-12-09
				Tuspetinib 80 mg + Venetoclax 200 mg	築願鏇蓋壓廠憲鏇壓鹽(鬱衊醖鏇構襯範觸鑰醖) = 願鹽膚繭願壓糧網觸鏇鑰鏇鏇淵膚範醖選膚淵 (獵廠膚範膚襯淵鏇窪簾 )
NCT03850574 (EHA2022)	临床1/2期	急性髓性白血病	-	HM43239	窪築齋築夢鑰蓋糧範網(積觸觸廠鹹壓顧憲艱醖) = 獵鏇範築餘製糧顧襯艱淵廠範壓醖鬱膚簾壓鹽 (觸廠選壓淵壓膚願淵齋 ) 更多	-	2022-05-12
NCT03850574 (ASH 12021 \| ASH 22021) 人工标引	临床1/2期	FLT3阳性急性髓性白血病二线 FLT3	28	HM43239	衊夢範觸範餘醖膚齋艱(艱積醖鏇鏇鏇網醖範夢) = diarrhea (14%), nausea (7%), vomiting (7%) and alanine aminotransferase increased (7%), and no drug related > Grade 3 TEAEs were observed to date 襯糧鬱蓋築餘鏇壓壓膚 (壓積簾齋網糧製蓋艱襯 )	积极	2021-11-05
				HM43239 (80 mg dose)