On December 8, 2025 TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA"), a Phase 3 immune-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, reported that its research on the potential role of the Delta Opioid Receptor (DOR) in controlling the immunosuppressive capabilities of MDSCs was presented in an oral presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition that took place on Sunday, December 7 in Orlando, FL. The Company presented these updated results, along with a poster presentation highlighting the effects of DOR inhibition on TAMs, another immunosuppressive cellular component critical to the tumorigenic microenvironment.

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In the oral presentation, entitled: Delta Opioid Receptor (DOR) Expression on Myeloid-Derived Suppressor Cells (MDSCs) Represents a Novel Target to Overcome Resistance to Immune Checkpoint Inhibitors (ICIs), Dr. Michael Turner, Vice President Immunology at TuHURA Biosciences presented updated data validating DOR expression on MDSCs and further showing that the pharmacological antagonism of DOR reduced the suppressive activity of MDSCs. MDSCs are a heterogeneous population of immature myeloid cells that contribute to creating an immunosuppressive tumor microenvironment (TME) by suppressing anti-tumor immune responses. The study showed that antagonism of the DOR with a specific inhibitor modulated a variety of direct and indirect MDSC-mediated immunosuppressive factors and reversed T cell suppression, suggesting the DOR may be a novel target to reprogram MDSC induced immunosuppression in the TME.

In the poster, entitled: Delta Opioid Receptor (DOR): A Novel Target for Reprogramming Tumor-Associated Macrophage (TAM) Immunosuppressive Phenotype to Overcome Acquired Resistance and Enhance the Effectiveness of Cancer Immunotherapies, Dr. Krit Ritthipichai, Director of Immunology at TuHURA Biosciences, presented the results of a study that showed how the DOR is highly expressed in tumor-infiltrating myeloid cells, particularly TAMs, indicating that the TME induces DOR upregulation relative to peripheral macrophages, and that targeting the DOR provides a promising strategy to reprogram suppressive TAMs and MDSCs, alleviate T-cell dysfunction, and potentially overcome resistance to checkpoint blockade and other immunotherapies.

Dr. James Bianco, President and Chief Executive Officer of TuHURA Biosciences, said "Innate immune cells, MDSCs, TAMs, and adaptive regulatory T cells (Tregs) are among the most important cellular components of the body’s immune system that provide the ability to regulate inflammation, autoimmunity and immune tolerance. Our discovery of the expression of the DOR on MDSCs and TAMs, and that its activation is coupled to mechanisms by which these cells contribute to immunosuppression, makes the DOR a compelling target for pharmacologic intervention to overcome acquired resistance to cancer immunotherapy. Data demonstrating that the DOR is also expressed on T regs and controls the expression of FOXP3, a critical immunosuppressive gene, provides a shared mechanism by which endogenous opioids, via the DOR, control the immunosuppressive tone of both innate and adaptive immune responses critical in the pathology associated with cancer and autoimmune disease. TuHURA is the first to demonstrate that this single target shares control of the immune suppressive capabilities of these cells and provides us a unique position to exploit pharmacologic modulation of the DOR to overcome resistance to cancer immunotherapy and the treatment of autoimmune and inflammatory diseases."

"The Company has developed a library of highly selective (>1,200 fold) and potent (<1.0 ng/ml) DOR antagonists and is in position to advance our first-in-class immune modulating bi-functional, bi-specific antibody drug conjugates (ADCs). We anticipate our lead ADC candidate to consist of a DOR inhibitor conjugated to our VISTA inhibiting antibody. TuHURA’s updates at this ASH (Free ASH Whitepaper) meeting demonstrate that elucidating the role of VISTA on macrophages and MDSCs in promoting the progression of myelodysplasia (MDS) to acute leukemia, as well as VISTA’s documented role in being central to how leukemia escapes immune recognition, makes it an ideal candidate to link to a DOR inhibitor. Our ADCs have the potential to not only remove the immunosuppressive tone of the TME but to also checkpoint release resting T cells, allowing them to recognize and kill leukemic cells." Dr. Bianco concluded, "We are excited to be at the forefront of these discoveries and look forward to working on the development of a whole new class of ADCs that could meaningfully change the treatment of cancer."

(Press release, TuHURA Biosciences, DEC 8, 2025, View Source [SID1234661286])

On December 8, 2025 Pierre Fabre Pharmaceuticals Inc., reported updated results presented at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting of the pivotal Phase 3 ALLELE study of tabelecleucel in adults and children with R/R EBV+ PTLD following HCT or SOT. The two abstracts included sub-analyses of pediatric patients and by prior therapy.

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"These two sub-analyses highlight the potential of tabelecleucel to improve outcomes for patients, both pediatric and adult, with R/R EBV+ PTLD who after undergoing a potentially life-saving solid organ or hematopoietic cell transplant suddenly face yet another life-threatening illness," said presenter and clinical investigator, Dr. Sarah Nikiforow, Assistant Professor, Stem Cell Transplantation, Dana-Farber Cancer Institute. "The updated data support the potential of tabelecleucel as an important advancement in addressing the significant unmet need for patients with EBV+ PTLD, who currently have no FDA-approved treatment options and may experience poor overall survival of only weeks to a few months following the failure of standard treatment."

ALLELE is an ongoing multicenter, open-label Phase 3 study. This analysis encompassed a larger cohort of 86 patients (29 HCT, 57 SOT). In the study, patients received a median of two treatment cycles. Each cycle of tabelecleucel consisted of three infusions given on days 1, 8 and 15 with an imaging assessment of efficacy on day 28.

The updated findings showed patients receiving tabelecleucel had an objective response rate (ORR) of 47.7% with the HCT cohort achieving an 48.3% ORR and SOT cohort achieving a 47.4% ORR. The median (95% CI) overall survival (OS) from Kaplan-Meier survival estimates for the HCT cohort was 18.6 months. For the SOT cohort, median OS was not estimable as more than half of the patients remained in follow-up. In a sub-analysis of treatment response by prior therapy in SOT patients, ORR was 52.4% for those receiving rituximab and 44.4% for those receiving rituximab and chemotherapy.

Safety findings presented were consistent with previously published data. Serious adverse events (SAEs) were reported in 58.6% of HCT and 66.7% of SOT patients. These events were related to study treatment in 1 HCT patient and 7 SOT patients. Fatal SAEs were reported in 5 HCT and 9 SOT patients, and none were reported by investigators to be treatment related. Pyrexia was reported in 1 SOT patient and considered as a potential sign of cytokine release syndrome possibly related to treatment. In the updated ALLELE study data, there were no reports of tumor flare or infusion reactions, immune effector cell-associated neurotoxicity syndrome, or transmission of infectious diseases. No events of graft vs host disease or organ rejection were reported as tabelecleucel related.

A new sub-analysis of 12 pediatric patients less than 17 years of age found the ORR was 50.0% with complete response reported in 4 of 12 patients, and partial response observed in 2 of 12 patients. Efficacy and safety observed in the pediatric sub-group was consistent with that reported in the overall population. Six patients developed treatment emergent adverse events (TESAEs); 4 TESAEs in 2 patients were considered treatment related. Fatal SAEs were reported in 2 patients, neither considered related to treatment. There were no reports of tumor flare, infusion reactions, immune effector cell-associated neurotoxicity syndrome, graft vs. host disease, transmission of infection or bone marrow/solid organ transplant rejection.

"The growing body of evidence supporting the efficacy and safety of tabelecleucel in treatment of people living with EBV+PTLD enhances our confidence in the potential of this innovative cell therapy. These patients have undergone a difficult journey to receive a life-saving transplant only to be diagnosed with this ultra rare form of cancer and they deserve an effective treatment option following failure of standard-of-care therapy," said Adriana Herrera, Chief Executive Officer of Pierre Fabre Pharmaceuticals Inc., the Pierre Fabre Laboratories pharmaceutical subsidiary in the United States. "We look forward to our FDA target action date in January 2026 and if approved, the opportunity to address the significant unmet medical need of these patients who urgently need new treatment options."

A BLA for tabelecleucel is currently under FDA review with a target action date of January 10, 2026. The application is granted priority review for tabelecleucel indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with EBV+ PTLD who have received at least one prior therapy including an anti-CD20 containing regimen. The BLA is supported by pivotal study data from the ALLELE study and supportive data covering more than 430 patients treated with tabelecleucel.

About Tabelecleucel
Tabelecleucel is an allogeneic, off-the-shelf, EBV-specific T-cell immunotherapy designed to selectively target and eliminate EBV-infected cells. Unlike autologous CAR-T therapies, allogeneic T-cells are derived from third-party donors and are not genetically modified. T-cells are collected from the blood of healthy donors and activated by exposure to Epstein-Barr virus antigens, enriching for a population of T-cells that recognize EBV. These EBV-specific T-cells are then expanded, characterized, and cryopreserved for future use to treat patients.

About EBV+PTLD
EBV+ PTLD is an ultra-rare, acute, and potentially deadly blood malignancy that occurs after transplantation when patient T-cell immune responses are compromised by immunosuppression. It can impact patients who have undergone allogeneic HCT or SOT. Poor median survival of 3 weeks and 4.1 months for HCT and SOT, respectively, is reported in EBV+ PTLD patients for whom standard of care failed, underscoring the significant need for new therapeutic options.

(Press release, Pierre Fabre, DEC 8, 2025, View Source [SID1234661285])

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])

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 Sumitomo Pharma America, Inc. (SMPA) reported new clinical data supporting further development of enzomenib, an investigational, oral selective menin inhibitor being researched for the treatment of relapsed or refractory acute leukemia, and nuvisertib, an oral investigational highly selective small molecule PIM1 kinase inhibitor, being researched for the treatment of relapsed or refractory myelofibrosis (MF), at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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Updated Data Shared with the Use of Enzomenib in Acute Myeloid Leukemia
Updated preliminary data were presented from the ongoing Phase 1/2 study of enzomenib monotherapy, which as of October 4, 2025, included 116 total patients with acute leukemia, most of whom (93.1%, 108/116) had acute myeloid leukemia (AML) with a median of two prior regimens. Genomic abnormalities of leukemia subtypes including KMT2A rearrangement (KMT2Ar) were documented in 61 patients (52.6%), NPM1 mutation (NPM1m) in 34 patients (29.3%), and other HOXA9/MEIS1-driven abnormalities in 21 patients (17.7%).

Enzomenib was escalated from 40 mg twice a day (BID) to 400 mg BID with no dose-limiting toxicities (DLTs). Treatment-related adverse events (TRAEs) observed in at least 10% of patients were nausea (16.4%), and vomiting (11.2%). Differentiation syndrome (DS) was reported in 12.9% of patients and did not result in patient deaths, study discontinuations, or dose reductions of enzomenib. No treatment-related deaths were observed in the study. Dose-dependent increases in exposure were observed, particularly at doses greater than 140 mg BID. Little to no drug accumulation was observed, and CYP3A4 inhibitor azoles did not have a significant impact on exposure.

In patients with KMT2Ar, dose optimization of 200, 300, and 400 mg BID is complete, and the RP2D has been determined as 300 mg BID. At RP2D, in patients with KMT2Ar who had not received prior treatment with a menin inhibitor (n = 15), the objective response rate (ORR) was 73.3% and CR+CRh was 40%. Across the optimization dose levels, the duration of CR+CRh (n = 11) was 12.5 months and in all optimization patients (n = 39) median overall survival (mOS) was 11.8 months.

For patients with NPM1m AML, dose optimization is ongoing at 200, 300, and 400 mg BID with a focus on 200 and 300 mg BID in patients who have not received a prior menin inhibitor. In the NPM1m dose optimization population (n=25, pts who received ≥ 200 mg BID), ORR is 52% and CR+CR is 44% with a duration of CR+CRh of 5.7 months. The mOS was 8.5 months.

"Despite improved understanding of the genetic factors of certain high-risk subtypes in acute leukemias, poor prognosis for patients remains a significant unmet need," said Naval G. Daver, M.D., professor and director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center in Houston. "The data from this ongoing Phase 1/2 study continue to show that enzomenib exhibits promising clinical activity, with encouraging overall and complete response rates, duration of response, and no significant drug interactions with azoles in patients with relapsed or refractory KMT2Ar or NPM1m AML. As an intentionally designed oral therapy to inhibit menin and KMT2A protein interaction, these encouraging clinical results combined with a promising safety profile support the potential of enzomenib as a therapeutic option for relapsed or refractory acute leukemia patients with KMT2A-rearranged or NPM1-mutated subtypes of the disease."

Also presented were preliminary results of a Phase 1 study of enzomenib at dose levels of 140 mg, 200 mg, and 300 mg BID in combination with azacitidine and venetoclax (VEN/AZA) in patients with relapsed or refractory AML with KMT2Ar or NPM1m. VEN was administered on days 1-14 of a 28-day study cycle, AZA on days 1-7, and enzomenib was administered on days 1-28 with and without azole antifungal agents.

A total of 40 patients were enrolled, of which 18 had KMT2Ar (45%) and 22 (55%) had NPM1m. The median number of prior regimens was 2, with 15 patients having received prior venetoclax (37.5%) and 11 patients (27.5%) a prior menin inhibitor.

There were no DLTs observed in the 40 patients enrolled. Hematologic TRAEs related to any regimen component observed in at least 15% of patients included thrombocytopenia (45%), leukopenia (35%), neutropenia (30%), anemia (22.2%), and lymphopenia (15%). Non-hematologic TRAEs were nausea (25%), diarrhea (20%), and AST increased and constipation (15% each). Any-cause QT interval prolongation was reported in 4 patients (10%) with no grade 3 or higher events; no cases were considered related to enzomenib. There were also 4 events of non-serious DS with 0 events grade 3 or higher. Pharmacokinetic data indicated that there were no significant drug-drug interactions between enzomenib and VEN.

As of the clinical data cutoff on October 4, 2025, clinical activity data is available for 26 of the 40 total patients as 11 patients were still in Cycle 1 (n = 9) or Cycle 2 (n = 2), and 3 patients had cutaneous leukemia without measurable disease in the bone marrow (bone marrow blasts <5%).

Promising preliminary clinical activity has been observed, particularly in patients without prior VEN or menin inhibitor exposure (N=13). The ORR is 85% (11/13) and the composite complete remission (CRc) rate is 62% (8/13). Local MRD assessments were available in 9 patients and 7/9 (78%) achieved MRD negativity as of the cut-off.

These data support evaluating enzomenib with VEN/AZA in patients with newly diagnosed AML. Study arms are being added to investigate the combination regimen for patients with newly diagnosed disease. Enzomenib will be administered at 200 mg or 300 mg BID using VEN/AZA administered according to the FDA label. Enrollment of newly diagnosed patients with KMT2Ar or NPM1m AML will begin in early 2026.

Nuvisertib in Patients with Relapsed or Refractory (R/R) MF
For the first time, clinical data were presented from the ongoing global Phase 1/2 study evaluating the safety and efficacy of nuvisertib in combination with momelotinib (MMB) in 18 patients with R/R MF with anemia. All enrolled patients in the study had previously been treated with a JAK inhibitor, the current standard of care for patients with MF, and 61% of patients had high molecular risk mutation. Preliminary data showed that the treatment with nuvisertib and MMB combination appeared well tolerated, with early clinical activity observed, including ³50% total symptom score reduction (TSS50 response) in 58% of patients with an absolute reduction in all individual symptoms, a spleen volume reduction ³ 25% (SVR25 response) in 50% of patients, anemia improvement, and cytokine modulation in patients with relapsed or refractory MF with anemia. These preliminary data, collected as of October 15, 2025, support further development of nuvisertib in combination with MMB as a potential treatment option for patients with MF.

Additionally, data presented from the ongoing global Phase 1/2 study of nuvisertib in patients with relapsed or refractory MF (N=77) showed that nuvisertib monotherapy continued to be well tolerated with no DLTs and limited myelosuppression. The results showed that treatment with nuvisertib monotherapy led to SVR25 response in 20% of patients, TSS50 response in 45% of patients with absolute reduction in all individual symptoms. Data also showed that treatment with nuvisertib resulted in significant cytokine modulation over time, correlating with spleen and symptom responses and one-year overall survival rate of 81%.

"Patients living with myelofibrosis with anemia usually have a dismal prognosis, still continue to face limited treatment options," said John Mascarenhas, M.D., Director of the Center of Excellence for Blood Cancers and Myeloid Disorders, Icahn School of Medicine at Mount Sinai, New York. "The promising preliminary results from the combination of nuvisertib and momelotinib underscore the urgent need for new therapeutic approaches that may offer meaningful clinical benefits to a difficult to treat disease."

"We are excited to present the first-ever myelofibrosis data with a momelotinib-based combination, specifically nuvisertib in combination with momelotinib. The development of innovative therapies—both alone and in combination with other treatments—are critical for physicians and patients with blood cancers such as AML or MF who are in urgent need of new effective therapies," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "Based on these updated preliminary data presented at ASH (Free ASH Whitepaper), which continue to show promising clinical activity and safety profiles for both enzomenib and nuvisertib, we remain committed to accelerate the clinical development in these programs with the ultimate goal of improving patient outcomes."

About Leukemia
Leukemia is a type of cancer that forms in blood-forming tissue, characterized by the uncontrolled growth of blood cells, usually white blood cells, in the bone marrow. Acute leukemia, a form of leukemia, requires immediate treatment as blood cells multiply rapidly leading to a sudden onset of symptoms.1 Approximately 30% of patients with AML have NPM1 mutations,2 and 5%-10% of patients with AML have KMT2A rearrangements.3

About Myelofibrosis (MF)
MF is a rare type of blood cancer that is characterized by the buildup of fibrous tissue in the bone marrow, which can affect the production of blood cells. This buildup is caused by dysregulation in the JAK signaling pathway. MF is a serious and rare disease with 0.7 new cases per 100,000 people worldwide each year.4

About Enzomenib (DSP-5336)
Enzomenib is an investigational, oral, small molecule inhibitor of the menin and Lysine (K)-specific methyltransferase 2A (KMT2A) protein interaction, a key interaction in acute leukemia and other tumor cell proliferation and growth. Menin is a scaffold nuclear protein, which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.5,6 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A rearrangements or NPM1 mutations.5,7 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1 and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with KMT2A rearrangements and NPM1 mutation.8,9 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose-escalation/dose-expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). Additionally, the registrational Phase 2 Horizen-1 R/R mono AML/ALL (KMT2Ar + NPM1m) study is now open for enrollment. The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in June 2024. Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in September 2024.

About Nuvisertib (TP-3654)
Nuvisertib is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.10,11 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.10 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization and reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.11 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate- and high-risk myelofibrosis (NCT04176198). The FDA granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024. The FDA granted Fast Track Designation to nuvisertib for the indication of myelofibrosis in July 2025.

(Press release, Sumitomo Pharmaceuticals, DEC 8, 2025, View Source [SID1234661282])