5 of 8 patients (62.5%) achieved Undetectable MRD (uMRD) LEXINGTON, Mass., Dec. 9, 2025 /PRNewswire/ -- Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 and FLT3 inhibitor, yesterday provided updated clinical data from the ongoing frontline Acute Myeloid Leukemia (AML) triplet study (CA-4948-104) in a poster presentation at the 67th ASH Annual Meeting (ASH). The AML triplet study is evaluating the addition of ...

Ziftomenib + Ven/Aza in Newly Diagnosed NPM1-m AML - In newly diagnosed NPM1-m AML patients, the combination of ziftomenib 600 mg QD with Ven/Aza resulted in a composite complete remission (CRc) rate of 86% (32/37), with a complete remission (CR) rate of 73% (27/37)[35] - Among CRc responders in newly diagnosed NPM1-m AML, 68% (17/25) achieved MRD negativity at a threshold of ≤01% and 44% (11/25) at a threshold of ≤001%[36] - In newly diagnosed NPM1-m AML, 68% (27/40) of patients remained alive and continued on study after a median follow-up of 261 weeks[40] Ziftomenib + Ven/Aza in R/R NPM1-m or KMT2A-r AML - In R/R NPM1-m AML patients, the combination of ziftomenib 600 mg with Ven/Aza resulted in an overall response rate (ORR) of 65% (31/48) and a CRc rate of 48% (23/48)[57] - In R/R KMT2A-r AML patients, the combination of ziftomenib 600 mg with Ven/Aza resulted in an ORR of 41% (13/32) and a CRc rate of 28% (9/32)[57] - Among R/R NPM1-m AML patients without prior venetoclax exposure, the CRc rate was 70% (16/23) and the ORR was 83% (19/23)[58] - Among R/R KMT2A-r AML patients without prior venetoclax exposure, the CRc rate was 60% (6/10) and the ORR was 70% (7/10)[58] - For R/R NPM1-m AML, the median duration of CRc was 399 weeks after a median follow-up of 274 weeks[61] - For R/R KMT2A-r AML, the median duration of CRc was 124 weeks after a median follow-up of 169 weeks[64]

Core Insights - The combination of ziftomenib with venetoclax and azacitidine shows promising clinical activity in treating acute myeloid leukemia (AML) with NPM1 mutations and KMT2A rearrangements, with high rates of complete remission and molecular negativity [2][3][8] Group 1: Clinical Efficacy - In newly diagnosed NPM1-m AML, 86% achieved composite complete remission (CRc) and 73% achieved complete remission (CR), with 68% of CRc responders attaining molecular minimal residual disease (MRD) negativity [1][2] - In relapsed/refractory (R/R) NPM1-m AML, the overall response rate (ORR) was 65%, and in venetoclax-naïve patients, the ORR was 83% [1][2] - For R/R KMT2A-r AML, the ORR was 41%, with 70% in venetoclax-naïve patients [1][2] Group 2: Safety Profile - The triplet combination of ziftomenib, venetoclax, and azacitidine was well tolerated, with low rates of ziftomenib-related myelosuppression and no increase in toxicity beyond venetoclax/azacitidine alone [1][4][7] - Adverse reactions included differentiation syndrome and QTc prolongation, but these were managed without treatment discontinuation [4][7] Group 3: Ongoing Development - Kura Oncology is conducting registrational trials for ziftomenib in both intensive chemotherapy-eligible and -ineligible patients [1][2][8] - The ongoing KOMET-007 Phase 1a/1b trial is evaluating ziftomenib's efficacy across multiple AML subtypes, reinforcing its potential as a foundational treatment option [1][8][12] Group 4: Company Information - Kura Oncology is focused on precision medicines for cancer treatment, with KOMZIFTI (ziftomenib) being the first oral menin inhibitor approved for adult patients with R/R AML harboring NPM1 mutations [12][28] - Kyowa Kirin collaborates with Kura Oncology, emphasizing their commitment to innovative treatments for high unmet medical needs [29]

Core Insights - Galecto, Inc. is presenting preclinical data and clinical development plans for GB3226, a novel dual ENL-YEATS and FLT3 inhibitor for acute myeloid leukemia (AML), at the ASH Annual Meeting in December 2025 [1][2] Group 1: Product Overview - GB3226 is a first-in-class, orally bioavailable small-molecule inhibitor targeting ENL-YEATS and FLT3, which is mutated in approximately 30% of adult AML patients [2][4] - The dual inhibition of ENL-YEATS and FLT3 may provide greater therapeutic benefits compared to existing treatments [2] - Preclinical studies show GB3226's potent activity across diverse AML patient-derived samples, leading to rapid tumor regression and significantly prolonged survival in xenograft models [2][4] Group 2: Clinical Development - Two poster presentations will detail the Phase 1 study of GB3226 in patients with relapsed/refractory AML, scheduled for December 6 and December 8, 2025 [3] - The presentations will be held at the Orange County Convention Center in Orlando, Florida, during the ASH Annual Meeting [3] Group 3: Efficacy and Safety - GB3226 demonstrated favorable pharmacokinetics and tolerability in animal studies, with potential additive or synergistic effects when combined with current standard-of-care agents [2][5] - The investigational candidate showed superior efficacy compared to both FLT3 and menin inhibitors in preclinical models [4] Group 4: Company Background - Galecto is a clinical-stage biopharmaceutical company focused on developing novel treatments for cancer and liver diseases, with a pipeline that includes GB3226 and other first-in-class small molecule drug candidates [6]

Core Insights - AB Science SA has provided initial Phase 1 data for the combination of AB8939 with Venetoclax for treating refractory or relapsed acute myeloid leukemia (AML) [1] Group 1: Clinical Data and Efficacy - Early data indicates that AB8939, either as monotherapy or in combination, shows significant activity in high-risk subtypes of AML [2] - The combination of AB8939 and Venetoclax has demonstrated a disease control rate of 100% (3/3) and a partial response rate of 100% (3/3), including one patient achieving complete remission after the first treatment cycle [5][9] - AB8939 has shown activity in MECOM, with long overall survival benefits, and is effective in cell lines resistant to standard treatments [10][8] Group 2: Mechanism of Action - AB8939 operates through a dual mechanism: disrupting microtubules to block leukemia cell proliferation and targeting leukemia stem cells by inhibiting ALDH [10][11] - The combination with Venetoclax is expected to enhance apoptosis in cancer cells, as AB8939 destabilizes microtubules while Venetoclax inhibits the BCL2 pathway, which is crucial for AML resistance [11] Group 3: Market Potential - The addressable market for AB8939 in relapsed/refractory AML is estimated to exceed EUR 2 billion annually [13] - The total incidence of AML cases is approximately 90,200 globally, with significant relapse rates, indicating a persistent unmet medical need [14] Group 4: Next Steps and Development Plans - The next steps include completing Phase 1 in combination therapy and launching an expansion study involving around 15 AML patients eligible for AB8939 + Venetoclax [12] - Discussions with regulatory bodies such as the FDA and EMA are ongoing regarding potential registrational studies for AB8939 [13] Group 5: Intellectual Property - AB8939's intellectual property rights in AML are secured until 2036, with potential extensions until 2044 for specific genetic abnormalities [18]

Core Viewpoint - Omeros Corporation has established the Omeros Oncology Clinical Steering Committee to advance its OncotoX biologics program targeting acute myeloid leukemia (AML), which is a highly fatal form of leukemia accounting for about 80% of acute leukemias in adults and one-third of all blood/bone marrow cancers [1][4]. Group 1: OncotoX Program Development - The OncotoX program consists of proprietary targeted, engineered molecules that deliver a toxic payload directly into cancer cells, effectively killing them [2]. - The steering committee will aid in developing the OncotoX-AML program, designing clinical trials, and interacting with institutional review boards [3]. Group 2: Efficacy and Safety - In vivo and ex vivo studies indicate that OncotoX-AML is highly effective at low doses, providing significant survival benefits compared to existing therapies like AbbVie’s Venclexta and Bristol Myers Squibb’s Vidaza [4]. - OncotoX-AML targets both AML blasts and leukemia stem cells (LSCs), which are often resistant to chemotherapy, thus addressing potential relapse [5]. - Preliminary studies show that OncotoX is well tolerated at doses significantly higher than effective doses without causing neutropenia or major changes in blood chemistry [5]. Group 3: Market Potential and Analyst Insights - Omeros is initiating IND-enabling work for OncotoX-AML, which is crucial given the limited therapeutic options for AML, especially for elderly patients or those with high-risk mutations [6]. - Analyst Jason Kolbert maintains a Buy rating on Omeros with a price target of $36, highlighting the potential of OncotoX-AML to fill gaps in AML treatment [7]. - The company plans to submit a European marketing authorization application for its lead therapy, narsoplimab, in the first half of 2025 [8].