On December 8, 2025 Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, reported new data from the company-sponsored ASTX030-01 and ASTX727-03 studies evaluating all-oral regimens of azacitidine and cedazuridine or decitabine and cedazuridine in patients with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) or acute myeloid leukemia (AML). Data from the studies will be shared in two oral presentations at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held on December 6-9, 2025, in Orlando, Florida. Collectively, new data from 15 company-sponsored and company-funded externally led studies will be presented, demonstrating an increasing commitment to understanding novel oral regimens in hematology.

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An oral presentation will highlight results from the Phase 2 ASTX030-01 trial, a multicenter, randomized, open-label, crossover trial comparing the all-oral combination of azacitidine and cedazuridine to subcutaneous azacitidine among patients with MDS, CMML or AML.

A second oral presentation will share findings from the Phase 2 ASTX727-03 trial of low-dose oral decitabine and cedazuridine versus an attenuated course of standard-dose decitabine in patients with lower-risk MDS.

Azacitidine and decitabine belong to a class of antineoplastic agents known as DNA methyltransferase inhibitors (DMTIs). Each is paired with cedazuridine, a cytidine deaminase inhibitor, to help the agent stay active in the body without degrading.

"We are pleased to present positive data suggesting that oral azacitidine and cedazuridine in patients with MDS, CMML and AML, and decitabine and cedazuridine in patients with lower-risk MDS may be comparable in safety and effectiveness to frequently used parenteral therapies for those populations," said Harold Keer, MD, PhD, Chief Medical Officer of Taiho Oncology. "While standard hypomethylating agents are administered via infusion in the clinic, these novel treatments are designed to be taken orally at home, potentially improving flexibility and lowering the treatment burden for patients."

Authors will report results from the ASTX030-01 study of oral azacitidine and cedazuridine versus subcutaneous azacitidine in patients with MDS, CMML or AML: 1

Investigator Summary of Results

As of the May 2025 data cutoff, 30 patients received treatment, including 22 individuals with MDS, five with CMML, two with AML and one with MDS/myeloproliferative neoplasms (MPN); all were eligible for single-agent azacitidine. The patients were randomly assigned in a 1:1 ratio to receive ongoing cycles of therapy. One group received ASTX030 except during cycle 2, when subcutaneous azacitidine was administered, while the other group received subcutaneous azacitidine in cycle 1 followed by ASTX030 in all subsequent cycles.

As of the data cutoff, ASTX030 Phase 2 results demonstrated the following:

The primary endpoint was the geometric mean ratio (GMR) of azacitidine total cycle AUC 0–24 exposures after oral ASTX030 over subcutaneous azacitidine, and the result for that endpoint was 0.913 (90% confidence interval [CI]: 0.78, 1.07).
In patients with MDS (n=22), the complete response (CR) rate was 22.7% and overall response rate was 50%.
Among patients who were dependent on red blood cell (RBC) transfusions at baseline (n=13), 30.8% achieved independence from RBC transfusions for 56 days or more.
When stratifying by body surface area (BSA), patients with intermediate BSA had a GMR of 0.980 (90% CI: 0.85, 1.13), whereas the subset of patients with a high BSA had a GMR of 0.700 (90% CI: 0.55, 0.89) and further simulations suggested that BSA-adjusted dosing will help ensure the PK exposure of the oral combination approximates that of subcutaneous azacitidine.

Summary of Preliminary Safety and Tolerability

Adverse events (AEs) were reported in 100% of trial participants, with 83.3% of those AEs classified as grade 3 or higher.
The most common treatment-emergent adverse events (TEAEs), the majority of which were grade 1 or 2, were nausea (70%), constipation (66.7%) and fatigue (60%).
The most common TEAEs of grade 3 or higher were thrombocytopenia (43.3%), neutropenia (33.3%) and anemia (30%).
AEs leading to treatment withdrawal or dose reduction occurred in two (6.7%) and four (13.3%) patients, respectively, and there were 12 (40%) AEs that led to treatment interruption or delay.
Authors will report results from the ASTX727-03 study of low-dose oral decitabine and cedazuridine versus standard-dose decitabine and cedazuridine in patients with lower-risk MDS2

Investigator Summary of Results

As of the October 2024 data cutoff, 81 patients with low-risk or intermediate-1 MDS and either one or more cytopenias or dependence on red blood cell transfusions were treated in the Phase 2 ASTX727-03 study, comparing a low-dose (LD), all-oral regimen of decitabine and cedazuridine with an attenuated course of standard dose (SD) decitabine and cedazuridine (DEC-C). Patients received 10 mg of oral decitabine and 100 mg cedazuridine for five days or 35 mg decitabine and 100 mg cedazuridine for three days. Patients were treated a median of 8.8 months, with patients in the LD arm receiving a median of 10 cycles and those in the SD arm receiving a median of 9 cycles.

As of the data cutoff, ASTX727-03 Phase 2 results demonstrated the following:

Median overall survival (OS) was 23.9 months in the LD arm versus 26.0 months in the SD arm.
Median leukemia-free survival (LFS) was 23.8 months in the LD arm versus 25.7 months in the SD arm.
Hematologic improvement per International Working Group 2006 criteria was achieved in 27.5% LD patients and 26.8% SD patients.
Among patients dependent on RBC transfusions, 52.4% of LD patients and 37.5% SD patients achieved RBC transfusion independence.
Pharmacokinetic exposure AUC in the LD arm was approximately half that in the SD arm.
Summary of Preliminary Safety and Tolerability

Delayed cycles occurred in 72.5% of patients in the LD arm versus 82.9% of those in the SD arm. Dose reductions occurred in 40% of patients in the LD cohort versus 46.3% of patients in the SD group.
Both treatment regimens caused a decrease in blood counts. Neutropenia was more pronounced in early cycles and was more severe in the SD arm. Blood counts across all lineages remained stable or improved through 12 or more cycles with the LD arm, suggesting a more favorable safety and tolerability profile.
AEs of grade 3 or higher occurred in 85% of patients in the LD arm and 90.2% of patients in the SD arm. Treatment discontinuation due to AEs occurred in 2.5% of patients in the LD arm versus 17.1% of patients in the SD arm.
The most commonly reported treatment-emergent AEs were anemia (42.5% LD versus 39% SD), fatigue (32.5% LD versus 43.9% SD) and thrombocytopenia (37.5% LD versus 39% SD).
3 deaths occurred in the trial: 2 in the LD arm, both unrelated to study treatment, and 1 in the SD arm due to pseudomonal bacteremia in cycle 1, which was treatment-related.

(Press release, Taiho, DEC 8, 2025, View Source [SID1234661283])

On December 8, 2025 Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported positive new data from its pipeline of allogeneic T-cell immunotherapies at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

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Orca-Q with and Without the Use of GvHD Prophylaxis

A subset of the multi-center Phase 1 clinical trial evaluated Orca-Q, Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy, in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML), myelofibrosis (MF), myelodysplastic syndrome (MDS) and mixed phenotype acute leukemia (MPAL) with HLA-matched related and unrelated donors. New data evaluating Orca-Q with and without the use of pharmacological graft versus host disease (GvHD) prophylaxis showed encouraging outcomes including rapid neutrophil recovery and low rates of acute and chronic GvHD, relapse and non-relapse mortality (NRM).

"Controlling alloreactivity and reducing GvHD following a conventional stem cell transplant typically requires multi-agent immunosuppression. However, this can impair immune reconstitution and increase the risk of organ toxicity, infection and relapse," said presenting author Samer Srour, M.D., Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center. "In these new findings, Orca-Q demonstrated encouraging outcomes even without the use of any pharmacological GvHD prophylaxis. While ongoing enrollment is important to further validate these results, the data suggest that Orca-Q may be able to control alloreactivity and potentially offer a platform to improve transplant outcomes through controlling GvHD and infection and reducing non-relapse mortality without increasing the risk of relapse."

Patients on Arm A received Orca-Q and single-agent tacrolimus (tac, n=18) while patients on Arm C received Orca-Q and no immune suppression (n=26). Median time to neutrophil engraftment was 15 days for patients who received tac and 11 days for patients who did not. Orca-Q was well-tolerated with 94% overall survival (OS) at one year for patients with tac and 87% for patients without tac. At one year, GvHD-and-relapse-free survival (GRFS) was 77% with tac and 79% without tac, and NRM was 6% and 0% with tac and without tac, respectively. Relapse-free survival (RFS) at one year was 88% with tac and 87% without tac. At Day 180, moderate-to-severe chronic GvHD was 12% with tac and 0% without tac. Grade 3-4 acute GvHD was 8% and 6% with and without tac, respectively.

Importantly, Orca-Q patients treated without GvHD prophylaxis demonstrated more rapid immune reconstitution and improved control of infections. Specifically, BMT CTN Grade 2+ infections at one year were 33% with tac and 17% without tac.

Orca-T and CAR-T Combination Treatment Versus Autologous CAR-T

A second oral presentation compared the results from two separate Phase 1 trials in adults with high-risk relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL). The first trial evaluated OrCAR-T (n=16), a treatment that combines Orca-T and allogeneic CD19/CD22 CAR-T cells, and the second trial evaluated an autologous CD19/22 (n=17). At 18 months, progression-free survival (PFS) and OS were 100% and 100% with OrCAR-T, and 38.5% and 77% in the autologous cohort. Toxicities were mild across both cohorts, with no grade 3-4 CAR-related toxicities reported. At a median follow-up of 2.5 years, there were no relapses or patient deaths with OrCAR-T and seven deaths with autologous, six from refractory B-ALL.

"Among adults with B-ALL, CAR-T therapy is often followed by a consolidative allogeneic transplant to achieve long-term remission," said Lori Muffly, M.D., associate professor in the Division of Blood and Marrow Transplantation-Cellular Therapies at Stanford Health Care. "Emerging data showing improved progression-free survival in patients with a prior transplant has prompted interest in exploring whether combining a high-precision allogeneic therapy like Orca-T with CAR-T cells could provide a feasible, all-in-one treatment. While these findings are early, they suggest this approach has the potential to benefit patients across a range of hematologic diseases."

"Our team is encouraged by these new findings from our broader pipeline programs, including results that highlight the potential to eliminate the need for GvHD prophylaxis while preserving immune reconstitution with Orca-Q," said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. "The durability of Orca-T when combined with CAR-T therapy exemplifies how our high-precision approach may be applied to expand treatment options both within and beyond hematologic malignancies. Overall, these results reinforce our continued focus on advancing our pipeline to bring this high- precision platform to more patients who could potentially benefit."

About Orca-Q

Orca-Q is Orca Bio’s second-generation investigational allogeneic T-cell immunotherapy under evaluation in clinical trials for the treatment of multiple hematologic malignancies, including in patients with haploidentical and mismatched donors. Orca-Q is a proprietary composition of stem cells combined with specific T-cell subsets derived from healthy donors and engineered by Orca Bio’s high-precision platform.

(Press release, Orca Bio, DEC 8, 2025, View Source [SID1234661299])

On December 8, 2025 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported the presentation of encouraging updated data of patients treated in the Phase 1 NATHALI-01 clinical trial with eti-cel, at the 67th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, FL.

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Eti-cel product candidate is the first allogeneic dual CAR-T targeting CD20 and CD22 simultaneously, being developed in Phase 1 of the NATHALI-01 clinical trial, for patients with relapsed/refractory non-Hodgkin lymphoma (r/r NHL), following at least two lines of therapy.

Cellectis presented preliminary results on eti-cel, which demonstrated an encouraging overall response rate (ORR) of 88% and a complete response (CR) rate of 63% (n=8) at the current dose level.

Additional in vivo data presented suggest that exogenous low dose Interleukin-2 (IL-2) support can significantly enhance the expansion and persistence of CAR-T cells to boost CAR-T efficacy without exacerbating toxicity.

"Cellectis believes that, with the addition of low dose IL-2 support, it is possible to further deepen the already high response rates seen with eti-cel in these patients who have relapsed following multiple prior lines of therapy including, in most cases, a CD19 CAR-T" said Adrian Kilcoyne, MD, MPH, MBA, Chief Medical Officer at Cellectis. "The trial will now investigate any potential impact of low dose IL-2 support in these difficult to treat patients. We look forward to sharing the full Phase 1 dataset expected in 2026."

Next Steps

Overall, these preliminary data underscore the potential of this innovative approach to transform outcomes for r/r NHL patients. The Company will now investigate the potential impact of low dose IL-2 support and will start recruitment of patients in the IL-2 support cohort in Q1 2026. Cellectis expects to present the full Phase 1 dataset in 2026.

(Press release, Cellectis, DEC 8, 2025, View Source [SID1234661251])

On December 8, 2025 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines in oncology and autoimmune disease, reported new clinical data from patients with relapsed or refractory Waldenström macroglobulinemia (WM) treated in the Phase 1 clinical trial of its Bruton’s tyrosine kinase (BTK) degrader bexobrutideg (NX-5948). These data will be presented by Scott Huntington M.D., MPH, Associate Professor of Internal Medicine (Hematology), Yale School of Medicine, and a clinical investigator on the trial, on December 8, 2025, at 6 p.m. ET at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held in Orlando, FL.

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"The data presented at ASH (Free ASH Whitepaper) in this older and heavily pre-treated WM population that includes patients with MYD88 and CXCR4 mutations continue to demonstrate encouraging activity of bexobrutideg with durable and deepening responses with longer time on treatment," said Paula G. O’Connor, M.D., chief medical officer of Nurix. "Bexobrutideg was well tolerated, consistent with the overall study population and previous disclosures."

"Collectively, these clinical data and recent data highlighting the unique properties of our potent and highly selective BTK degrader contribute to a growing body of evidence that support bexobrutideg’s potential to be the best-in-class and an important new therapeutic option for patients," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "We believe bexobrutideg is an innovative therapy with the potential to transform care in CLL, WM, and additional NHL indications, while supporting long-term value creation as its development expands into inflammatory and autoimmune settings."

The data presented at the 2025 ASH (Free ASH Whitepaper) Annual Meeting include patients with WM (n=31) treated with bexobrutideg at doses ranging from 200 mg to 600 mg once daily by oral administration from both the Phase 1a dose escalation and Phase 1b cohort expansions. Among the 31 WM patients, the median age was 71.0 years (range 49–88 years), and the median number of prior lines of therapy was 3 (range 1-7). All 31 patients previously had been treated with a BTK inhibitor (100%), 28 had received prior chemotherapy/chemo-immunotherapy (90.3%), four had received prior non-covalent BTK inhibitor (12.9%), and four patients had received prior treatment with a BCL2 inhibitor (12.9%). Twenty-four patients (77.4%) had mutations in MYD88, and six patients (19.4%) had mutations in CXCR4. Three patients (9.7%) had central nervous system (CNS) involvement at baseline.

Bexobrutideg was well tolerated in patients with WM, consistent with the overall study population and previous disclosures. Adverse events (AEs) were predominantly low grade with the most common being neutropenia (29.0%), petechiae (29.0%), diarrhea (25.8%), anemia (22.6%), purpura/contusion (22.6%), and thrombocytopenia (19.4%). There were no dose limiting toxicities observed and no grade 5 AEs. Two treatment emergent AEs led to drug discontinuation. No new onset atrial fibrillation was observed.​

As of the September 19, 2025 data cut, 28 patients were evaluable for response. Bexobrutideg demonstrated an objective response rate (ORR) of 75.0%, including very good partial responses (VGPR) in three patients (10.7%), partial responses (PR) in 14 patients (50.0%), and minor responses (MR) in four patients (14.3%). Six patients (21.4%) had a best response of stable disease (SD). In a subgroup analysis of patients with 2 or more disease assessments (n=23), ORR was 82.6% and disease control rate (DCR) was 100.0%.

Responses were observed in patients regardless of their baseline mutations in MYD88 and CXCR4. Out of three patients with CNS involvement (2 with systemic disease), two have responded and none progressed. Overall, responses were durable. With a median follow up of 8.1 months, median duration of response and median progression-free survival were not reached. As of the September 19, 2025 data cut, fourteen patients had continued on treatment for more than six months, and six patients had remained on treatment for more than one year.

Nurix Webcast Details
Date and time: Monday, December 8, 2025, 8:15 p.m. ET
Access Details: The live webcast and subsequent archived replay will be available in the Events section of the Investor page of the Nurix website at ir.nurixtx.com.

About Bexobrutideg (NX-5948)
Bexobrutideg is an investigational, orally bioavailable, brain penetrant, highly selective small molecule degrader of BTK currently being evaluated in the DAYBreak CLL-201 clinical trial (NCT07221500), a pivotal single-arm Phase 2 study of bexobrutideg in patients with relapsed or refractory chronic lymphocytic leukemia. Nurix also continues enrollment in the NX-5948-301 Phase 1a/1b clinical trial (NCT05131022) of bexobrutideg in patients with relapsed or refractory B cell malignancies.

(Press release, Nurix Therapeutics, DEC 8, 2025, View Source [SID1234661267])

On December 8, 2025 Pillar Biosciences and AstraZeneca reported an expansion of their existing laboratory access program for NGS-based kitted liquid biopsy tumor profiling to include China.

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This expanded collaboration aims to deliver rapid, cost-effective, and clinically actionable genomic insights through liquid biopsy testing. By increasing the local availability of plasma-based tumor profiling at leading clinical laboratories in China, the partnership seeks to accelerate diagnostic turnaround times and improve access to precision oncology solutions.

China faces one of the world’s highest cancer burdens, accounting for approximately 24% of new global cancer cases and 30% of cancer-related deaths, according to the World Health Organization’s GLOBOCAN 2022 report. Lung, colorectal, and liver cancers remain among the most prevalent, and access to early detection and molecular diagnostics continues to be a significant healthcare challenge. By expanding localized liquid biopsy testing, this collaboration supports China’s ongoing initiatives to enhance early cancer detection, precision diagnostics, and equitable access to targeted therapies.

As part of the initiative, AstraZeneca, Pillar Biosciences, and Shanghai Zhengu Biological Technology Co., Ltd. (Zhengu) will collaborate to support assay validation in local hospital laboratories and facilitate the implementation of Pillar’s liquid biopsy panels to enable localized tumor profiling.

"Expanding access to decentralized, high-quality molecular testing is critical to improving outcomes for cancer patients," said Dan Harma, Chief Commercial Officer, Pillar Biosciences. "By enabling local laboratories to perform in-house next-generation sequencing, we can reduce turnaround times, lower costs, and ensure that oncologists have faster access to actionable insights that guide personalized treatment decisions."

(Press release, Pillar Biosciences, DEC 8, 2025, View Source [SID1234661284])