On June 11, 2026 Kyntra Bio (Nasdaq: KYNB) reported additional data from the Phase 3 MATTERHORN trial showing improvements in transfusion independence in patients with anemia associated with lower-risk myelodysplastic syndromes (LR-MDS) treated with roxadustat will be presented as a poster at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress 2026, taking place June 11-14, 2026 in Stockholm, Sweden.

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"In addition to roxadustat demonstrating clinically meaningful efficacy in patients with lower-risk MDS and high transfusion burden, improvements in transfusion independence in both RS+ and RS- disease were also observed in this post-hoc analysis, which is an important finding given the limited effectiveness of currently available treatment options for patients with RS- disease," said Thane Wettig, Chief Executive Officer of Kyntra Bio. "These findings underscore the potential of roxadustat to elevate the standard of care for patients with lower-risk MDS who are in need of additional treatment options. We are finalizing the protocol for the pivotal Phase 3 trial, which we expect to initiate in the second half of 2026, with the aim to build upon and confirm these findings in patients with lower-risk MDS and high transfusion burden, including both RS+ and RS- disease."

"Through this novel MOA, stabilizing HIF1-⍺, thereby normalizing erythroid precursor development and improving hemoglobin production, these findings highlight the potential for roxadustat to address a significant unmet need, providing a convenient, well-tolerated and effective treatment option for anemia in patients with LR-MDS independent of RS histology," said Amer Zeidan, MD, Professor of Medicine at Yale School of Medicine and Chief of the Division of Hematologic Malignancies at Yale Cancer Center. "This post-hoc analysis from the MATTERHORN trial shows clinically meaningful RBC transfusion independence among high transfusion burden patients, as well as clear evidence of hemoglobin increase among patients who received roxadustat compared to placebo. I am excited to be able to share this data with the MDS community at the EHA (Free EHA Whitepaper) meeting and believe they provide strong rationale for the planned randomized Phase 3 trial in anemic patients with LR-MDS and high RBC transfusion burden," concluded Dr. Zeidan, who is also the global principal investigator of the planned randomized Phase 3 trial.

As previously disclosed, the initial analysis with all of the patients who participated in the Phase 3 MATTERHORN trial showed that more patients receiving roxadustat achieved transfusion independence vs. placebo (48% vs. 33%; p=0.22). The presentation highlights data from a post hoc analysis of the entire trial population, demonstrating that roxadustat led to similar rates of transfusion independence in both RS+ and RS- patients. In RS- patients, which comprised 84 of the 140 patients enrolled in the trial, treatment with roxadustat led to transfusion independence for ≥8 weeks over 28 weeks in 48% of patients vs. 28% for placebo.

The presentation at EHA (Free EHA Whitepaper) also provides additional details on the subgroup of patients (n=37) who met the criteria of HTB (≥ 4 units pRBCs per 8-week period for 2 consecutive 8-week periods) per IWG-2018, where roxadustat achieved clinically meaningful efficacy in patients with LR-MDS and HTB with higher rates of ≥8-, 12-, 16-week RBC TI vs placebo. TEAEs were generally lower grade and managed medically with no new safety signals.

The poster presentation, titled "Roxadustat improves transfusion independence in LR-MDS patients with anemia and high transfusion burden and in ring sideroblast positive and negative disease: post-hoc analysis of MATTERHORN study" is scheduled for the poster session taking place on June 12, 2026 at 18:45 CEST.

The pivotal Phase 3 trial protocol of roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden is being finalized based on feedback received from the FDA.

About Myelodysplastic Syndromes Anemia
Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S., thereof 77% are considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease. Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin.

Roxadustat is approved in Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Kyntra Bio has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and Kyntra Bio are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa.

(Press release, Kyntra Bio, JUN 11, 2026, View Source [SID1234666578])

On June 11, 2026 Genmab A/S (Nasdaq: GMAB) reported new data from a post-hoc subgroup analysis from the pivotal Phase 3 EPCORE FL-1 trial, evaluating epcoritamab, a subcutaneous T-cell engaging bispecific antibody, in combination with rituximab and lenalidomide (epcoritamab + R2) in adult patients with relapsed or refractory (R/R) follicular lymphoma (FL), which showed that epcoritamab + R2 delivered consistent and sustained efficacy benefits across clinically relevant subgroups, including Follicular Lymphoma International Prognostic Index (FLIPI) score (0–2 vs 3–5), progression of disease less than or equal to two years from the date of initial frontline therapy (POD24) (POD24 vs non-POD24), and patient fitness (non-Hodgkin lymphoma 5 score). These results were presented during an oral presentation (abstract S229) at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress held in Stockholm, Sweden, June 11 -14, 2026.

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"The EPCORE FL-1 trial, bolstered by this subgroup analysis, established fixed-duration epcoritamab in combination with R2 for relapsed or refractory follicular lymphoma," said Benoit Tessoulin, M.D., Ph.D., Nantes University School of Medicine & University Hospital. "It delivers consistent efficacy and a manageable safety profile, regardless of comorbidity burden."

The EPCORE FL-1 trial randomized a total of 481 patients, with 243 receiving epcoritamab + R2 and 238 receiving R2 alone. The subgroup analysis of the Phase 3 EPCORE FL-1 trial was performed to assess the benefit and tolerability of epcoritamab + R2 across clinically relevant subgroups, including patients with higher- and lower-risk disease features, compared with standard of care R2.

The data demonstrated that progression-free survival (PFS) benefits continued to favor epcoritamab + R2, with hazard ratios (HR) consistently below 0.3 across FLIPI 0–2 (0.18 [0.10–0.33]) and FLIPI 3–5 (0.25 [0.15–0.42]), POD24 (HR 0.22 [95% CI 0.13–0.37]), and non-Hodgkin lymphoma 5 (NHL-5) subgroups (low: HR 0.27 [0.17–0.42]; H+I, high and intermediate: HR 0.14 [0.06–0.29]), indicating a substantially reduced risk of disease progression or death.

Additionally, overall response rates (ORR) were higher with the combination of epcoritamab and R² compared to R² alone across different FLIPI risk groups. For patients with FLIPI scores of 0–2, the ORR was 96.5% with the combination versus 84.8% for R2 alone. In patients with FLIPI scores of 3–5, the ORR was 93.0% with the combination compared to 72.6% with R2 alone. Moreover, complete response rates (CRR) were consistently higher with epcoritamab and R² across all analyzed subgroups. In patients with lower FLIPI scores (0–2), the CRR was 86.6% with the combination, compared to 62.1% for R2 alone. Among those with higher FLIPI scores (3–5), the CRR was 77.0% for the combination versus 35.4% for R² alone. Similar improvements in CRR were noted among non-POD24 patients (85.5% vs. 57.6%) and across various patient fitness categories, including NHL-5 low-risk patients (81.3% vs. 50.3%) and H+I patients (85.7% vs. 49.0%).

The safety profile of epcoritamab + R2 was manageable across all patient subgroups and consistent with that observed in the overall trial population, with no new safety signals identified. Although adverse events such as neutropenia and infections were more frequent among patients receiving lower lenalidomide doses, the consistent and sustained efficacy of epcoritamab + R2 compared with R² alone was maintained in this subgroup. These findings are consistent with standard clinical practice, in which lenalidomide dose reductions are routinely implemented to manage adverse events while preserving treatment benefit in combination with epcoritamab.

"The EPCORE FL-1 subgroup analysis demonstrated consistent and deep responses with a manageable safety profile across all patient characteristics, including varying risk profiles and lenalidomide dosing schedule, which validate the potential of the combination therapy," said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. "These data strongly reinforce our belief that epcoritamab, combined with rituximab and lenalidomide, is poised to transform the treatment paradigm, offering a highly effective and broadly accessible option for relapsed or refractory follicular lymphoma."

About the EPCORE FL-1 Trial
EPCORE FL-1 (NCT05409066) is a Phase 3 open-label interventional trial to evaluate the safety and efficacy of epcoritamab plus rituximab and lenalidomide (R2) versus R2 alone in patients with relapsed/refractory (R/R) follicular lymphoma (FL). Patients were randomized to receive EPKINLY in combination with rituximab and lenalidomide (n=243) or rituximab and lenalidomide alone (n=245). Patients received EPKINLY in 28-day cycles for a total of 12 cycles or until disease progression or unacceptable toxicity, whichever occurred first. Efficacy was established based on the dual primary endpoints of progression free survival (PFS) and overall response rate (ORR) determined by Lugano 2014 criteria as assessed by Independent Review Committee (IRC). Additional efficacy outcome measures include complete response (CR) and duration of response (DOR).

More information on this trial can be found at www.clinicaltrials.gov/.

About Follicular Lymphoma (FL)
Follicular lymphoma (FL) is typically an indolent, or slow-growing, form of non-Hodgkin lymphoma (NHL), that arises from B-lymphocytes. The second most common form of NHL, FL accounts for 20-30% of all NHL cases.i FL is considered incurable.ii Patients often relapse, and with each relapse the remission and time to next treatment shorten.iii Over time, transformation to diffuse large B-cell lymphoma (DLBCL), an aggressive form of NHL associated with poor survival outcomes, can occur in more than 25% of FL patients.iii,iv

About Epcoritamab
Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.v

Epcoritamab (approved under the brand name EPKINLY in the U.S. and Japan, and TEPKINLY in the EU) has received regulatory approval in certain lymphoma indications in more than 65 territories. Where approved, epcoritamab is a readily accessible therapy. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational relapsed or refractory (R/R) follicular lymphoma (FL) indication and additional approvals for the R/R diffuse large B-cell lymphoma (DLBCL) indication.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes several Phase 3, open-label, randomized trials, including a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with lenalidomide compared to chemotherapy infusion in patients with R/R DLBCL (NCT06508658), and a trial evaluating epcoritamab in combination with lenalidomide and rituximab (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.

(Press release, Genmab, JUN 11, 2026, View Source [SID1234666577])

On June 11, 2026 Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") reported encouraging long-term results from the Phase 1/2 KOMET-007 single-arm trial (NCT05735184) evaluating ziftomenib in combination with intensive chemotherapy, 7+3, in newly diagnosed NPM1-m or KMT2A-r AML. These results will be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress.

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These data compare favorably to historical standard-of-care data with 7+3 alone:

NPM1-m Patients KOMET-0071 Historical 7+3 Benchmark
CR

Age ≤ 65 years
Age > 65 years

91% (31/34)
100% (15/15)

88%2
56%2
CRc 96% 56-89%2,3,4
CR MRD- (bone marrow) 56% 44%5
12-month OS rate 94% ~ 70-80% in younger fit patients3,4,5
~ 45-55% in patients > 65 years old2,6

1KOMET-007 (N=49) at 600 mg ziftomenib; MRD neg < 10-4; 2Lachowiez et al., Blood Adv. 2020; 4(7): 1311–1320; 3Hernández-Sánchez et al., Leukemia. 2026; 40(2): 418-428; 4Othus et al. Leukemia. 2019; 33(2):371-378; 5Othman et al., Blood. 2024; 144(7):714-728, including Supplemental Material; 6Recher et al., Leukemia. 2022; 36(4): 913-922.

Overall Survival (OS) for NPM1-m Patient Subset in Single-Arm KOMET-007 Trial: Median OS Not Reached

Kura Oncology

KOMZIFTI (ziftomenib) is approved by the U.S. Food and Drug Administration (FDA) as monotherapy for adult patients with relapsed or refractory AML with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. The use of ziftomenib in combination with 7+3 is investigational and has not been approved by any health authority.

"The updated results from the KOMET-007 trial provide important evidence supporting the safety and clinical activity of adding ziftomenib to intensive chemotherapy for patients with newly diagnosed NPM1-m and KMT2A-r AML," said Amer Zeidan, M.B.B.S., M.H.S., Chief, Division of Hematologic Malignancies at Yale Cancer Center and Professor of Medicine at Yale School of Medicine, and the lead investigator for the registrational KOMET-017 program. "Across nearly 100 patients treated to date, composite remission rates reaching 90-96%, high rates of MRD negativity and encouraging durability are especially meaningful in a disease where depth of response can inform long-term treatment decisions. The 12-month survival estimate of 94% for the NPM1-m patient cohort is particularly impressive. Based on the results observed to date, this regimen could represent a transformative therapeutic approach and may allow some patients to avoid allogeneic hematopoietic cell transplantation, a procedure that carries a significant risk of mortality and morbidity. We will continue to follow patients to assess long-term safety and clinical activity, including outcomes for those who do not undergo transplantation, and these results continue to strongly support the registrational Phase 3 KOMET-017 trials."

As of the data cut-off on April 10, 2026:

High remission rates across both molecular subtypes

96% CRc and 98% ORR in NPM1-m AML, 90% CRc and 92% ORR in KMT2A-r AML
Deep molecular responses, including marrow central MRD assessment

Local CRc MRD-negativity rates were 85% in NPM1-m AML and 82% in KMT2A-r AML
In NPM1-m AML, marrow central MRD negativity (10-4, NGS) among CRc responders was 79% (31/39) at the <0.1% threshold and 56% (22/39) at the <0.01% threshold, with all CRc responders who achieved central MRD negativity doing so by Cycle 2
Durable responses and encouraging durability with extended follow-up

After median follow-up of nearly 18 months (range 1.0-23.5) in NPM1-m AML and 11.0 months (range 0.9-21.9) in KMT2A-r AML, median duration of complete response was not reached for the NPM1-m AML cohort and was 12 months for the KMT2A-r AML cohort
Median OS was not reached, with median follow-up of 17.6 months in NPM1-m and 11.0 in KMT2A-r, respectively
NPM1-m: 94% OS rate at 12 months (range 1.0-23.5)
KMT2A-r: 71% OS rate at 12 months (range 0.9-21.9)
The majority of patients remained alive and continued on study at time of data cut-off:
NPM1-m: 90% (44/49)
KMT2A-r: 62% (31/50)
Consistent and manageable safety profile

Ziftomenib 600 mg once-daily plus 7+3 was generally well tolerated, with no new or unexpected safety signals observed with longer follow-up
Low rates of ziftomenib-related cytopenias and minimal additive myelosuppression were observed with this combination
Ziftomenib 600 mg once-daily did not delay neutrophil or platelet count recovery
No Grade 4 differentiation syndrome or QTc prolongation events were reported
Four patients (4%) experienced Grade 3 differentiation syndrome; all cases successfully resolved with protocol-specified mitigation and three continued on ziftomenib treatment
Three patients (3%) experienced Grade 3 investigator-assessed QTc prolongation (all three on azole antifungals, fluoroquinolones, or other medications at time of assessment; one with ongoing hypokalemia and hypomagnesemia); none were assessed as ziftomenib-related and all QTc events successfully resolved with all patients continuing on ziftomenib treatment
60-day mortality rate of 2% (1/49) in NPM1-m patients
"KOMET-007 has meaningfully strengthened the scientific and clinical foundation for KOMET-017 after ziftomenib was successfully integrated into intensive frontline therapy resulting in high remission rates, deep molecular clearance, encouraging durability and a favorable tolerability profile," said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. "These data increase our confidence in the ongoing registrational program and support the potential for ziftomenib to serve as a foundational menin inhibitor backbone in frontline AML. Importantly, as more patients in clinical trials receive ziftomenib earlier in the treatment course and remain on therapy for longer periods, we believe there may be an opportunity to extend the benefit of menin inhibition beyond induction and deepen its impact across the AML treatment continuum."

"These data strongly support the continued study of ziftomenib as part of a frontline regime in newly diagnosed AML," said Yoshifumi Torii, Ph.D., Chief Medical Officer of Kyowa Kirin. "We view the high remission rates, along with deep MRD negativity and encouraging durability, as particularly meaningful. Despite advances in treatment, AML remains associated with a high risk of relapse, underscoring the continued need for improved long-term treatment strategies. These results suggest that ziftomenib, when combined with standard therapy, has the potential to advance the current treatment paradigm. We look forward to further evaluating its clinical value through the ongoing Phase 3 KOMET-017 trial."

The companies plan to publish these data in a peer-reviewed publication in the second half of 2026.

Copies of the presentation will be available on Kura’s website at www.kuraoncology.com/pipeline/publications following presentation at the meeting.

Virtual Investor Event
Kura will host a webcast and conference call on June 12, 2026, at 8:00 am ET / 5:00 am PT, featuring management and Amer Zeidan, M.B.B.S., M.H.S., Chief, Division of Hematologic Malignancies and Professor of Medicine at Yale School of Medicine, and the lead investigator for the registrational KOMET-017 study. The live webcast and replay will be available on the Company’s website at www.kuraoncology.com under the Investors tab in the Events and Presentations section.

Abbreviations
7+3 (cytarabine plus daunorubicin), AML (acute myeloid leukemia), CR (complete response), CRc (composite complete remission), KMT2A-r (KMT2A-rearranged), MRD (measurable residual disease), NGS (next-generation sequencing), NPM1-m (NPM1-mutant), ORR (objective response rate), OS (overall survival), QTc (corrected QT interval)

(Press release, Kura Oncology, JUN 11, 2026, View Source [SID1234666576])

On June 11, 2026 Theriva Biologics, Inc. (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported that clinical and translational results from VCN-01’s Phase 1 clinical trial in Head & Neck Squamous Cell Carcinoma (HNSCC) were recently published on-line first in the journal Clinical Cancer Research. The article, titled "Phase I trial of intravenous VCN-01 oncolytic adenovirus and durvalumab in patients with head and neck metastatic squamous cell carcinoma refractory to immunotherapy", can be read here.

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"We are very excited to see Clinical Cancer Research share the VCN-01 results in immunotherapy-refractory metastatic HNSCC patients with the broad oncology community," said Ricard Mesia (Catalan Institute of Oncology, ICO), expert on HNSCC and coordinating investigator in this study. "The trial demonstrates the ability of VCN-01 to resensitize tumors from these heavily pretreated patients to the immune checkpoint inhibitor durvalumab. Pharmacokinetic, tissue biopsy, radiomic and transcriptomic results from the study all support the VCN-01 stroma-degrading mode-of-action, being investigated to enhance tumor penetration by VCN-01 and coadministered therapies and enable/enhance an anti-tumor immune response. There are very few treatment options available to immunotherapy-refractory metastatic HNSCC patients, and the clinically impactful findings from this report encourage further clinical development of VCN-01 with immune checkpoint inhibitors or other immune modulating anticancer therapies."

Data summary – VCN-01 Phase 1 clinical trial (NCT03799744) in metastatic, immunotherapy-refractory Head & Neck Squamous Cell Carcinoma (HNSCC)

The trial enrolled 20 adult patients with refractory or metastatic HNSCC, whose disease progressed despite previous therapies, including anti-PD-(L)1 immune checkpoint inhibitors. Six patients were enrolled into the concomitant Arm I LD of the study and were administered IV low dose VCN-01 (3.3E12 virus particles; LD) four hours prior to a fixed IV dose of durvalumab (1500 mg/q4w). Eight patients were enrolled into the sequential Arm II LD of the study, receiving low dose IV VCN-01 14 days prior to IV durvalumab administration. An additional six patients were entered into Arm II HD, receiving high dose IV VCN-01 (1.0E13 virus particles; HD) 14 days prior to IV durvalumab administration.

Median progression-free survival (PFS) was 1.6 months in Arm I LD, 3.7 months in Arm II LD, and 2.1 months in Arm II HD.
Median overall survival (OS) was 10.3 months in Arm I LD, 15.5 months in Arm II LD, and 17.3 months in Arm II HD.
Circulating levels of the stroma-degrading hyaluronidase enzyme PH20 (expressed during selective VCN-01 intratumoral replication) increased significantly after VCN-01 administration in all tested patients, peaking on day 3-8 for most patients and detectable until day 28 in 11 of 12 patients.
Similarly, VCN-01 viral genome levels detected in patient blood exhibited an initial peak immediately following administration and a secondary peak on day 3-8, consistent with continued viral replication in tumors followed by a return of virus to circulation.
Upregulation of CD8 and IDO was observed in tumor biopsy samples, consistent with increased tumor infiltration by activated cytotoxic T cells – historically associated with increased HNSCC patient survival. Diminished levels of FoxP3, CD25, and CTLA4 were also observed, consistent with a reduction in tumor Tregs and inhibition of tumor immunosuppression.
Tumor biopsies revealed upregulation of PD-1 and PD-L1 in most patients following VCN-01 administration that correlated with patient survival, suggesting that immune system activity and heightened PD-L1 expression in tumors contributed to the improved outcomes from VCN-01 and durvalumab combination.

(Press release, Theriva Biologics, JUN 11, 2026, View Source [SID1234666575])

On June 11, 2026 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported that data from the Phase 1/2 SAVE trial of an all-oral regimen of Revuforj (revumenib), decitabine/cedazuridine, and venetoclax in relapsed or refractory (R/R) NPM1 mutated (NPM1m), KMT2A-rearranged (KMT2Ar), or NUP98-rearranged (NUP98r) acute myeloid leukemia (AML) were published in the Journal of Clinical Oncology and simultaneously presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2026 Congress in Stockholm, Sweden.

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Revuforj is the first and only menin inhibitor that is FDA approved for patients one year and older with R/R AML with a susceptible NPM1 mutation who have no satisfactory alternative treatment options or R/R acute leukemia with a KMT2A translocation as determined by an FDA-authorized test.

"The deep and durable remissions observed among patients with R/R NPM1m, KMT2Ar, or NUP98r AML who received an all-oral combination of revumenib, decitabine/cedazuridine, and venetoclax, highlight the potential for revumenib combinations to advance the standard of care treatment for menin-dependent acute leukemias," said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. "The SAVE data provide strong support for further studying revumenib with venetoclax and a hypomethylating agent in multiple settings, including among newly diagnosed patients who are unfit for intensive chemotherapy in the ongoing pivotal EVOLVE-2 trial and among fit patients in the RAVEN trial."

"The results observed with the all-oral SAVE regimen in heavily pretreated patients with R/R NPM1m, KMT2Ar, or NUP98r AML are very encouraging," said Ghayas C. Issa, M.D., Associate Professor of Leukemia at The University of Texas MD Anderson Cancer Center and Principal Investigator of the SAVE trial. "Notably, 88% of patients achieved a response with the majority achieving MRD negativity, 45% proceeded to a potentially curative stem cell transplant, and we observed a 14-month median overall survival. We also saw an impressive 50% CR/CRh rate and approximately 12-month median overall survival in patients with prior venetoclax exposure, a population that has historically experienced poor outcomes with a median survival of less than three months."

Summary of Key Results from the SAVE Trial in R/R NPM1m, KMT2Ar, and NUP98r AML

The publication entitled, "All-Oral Combination of Revumenib, Decitabine, and Venetoclax for Relapsed or Refractory AML (SAVE)" reports results from the R/R cohort of patients in the Phase 1/2, single-center, open-label SAVE trial. The primary endpoint of Phase 1 was the recommended Phase 2 dose of revumenib in combination therapy, which was identified as dose level 1 (the FDA-approved monotherapy dose of revumenib). The primary endpoint of Phase 2 was the composite complete remission1 (CRc) rate.

As of January 2026, 42 patients with R/R AML were enrolled in the SAVE trial, including five adolescents. 38% (16/42) had NPM1m, 40% (17/42) had KMT2Ar, and 21% (9/42) had NUP98r. The median age was 40 years (range: 12-82). Patients were heavily pretreated, with a median of two prior lines of therapy (range: 1-5); 52% (22/42) had received prior venetoclax and 33% (14/42) a prior hematopoietic stem cell transplant (HSCT).

The overall response rate (ORR) was 88% (37/42), the CRc rate was 71% (30/42), and the complete remission plus complete remission with partial hematological recovery (CR/CRh) rate was 60% (25/42) for the entire population. Response rates were similar across genotypes, including patients with NPM1m, KMT2Ar, or NUP98r. Overall, 45% (19/42) of patients proceeded to HSCT following treatment with the regimen, including 38% (6/16) of NPM1m patients, 65% (11/17) of KMT2Ar patients, and 22% (2/9) of NUP98r patients. Of the 19 patients that proceeded to HSCT, 63% (12/19) resumed revumenib after HSCT.

The rates of measurable residual disease (MRD) negativity by flow cytometry were 68% (25/37) among evaluable responders, 80% (24/30) among those with CRc, and 80% (20/25) among those with CR/CRh. Among evaluable patients with CRc, 67% (8/12) were MRD negative by NPM1 NGS (<0.01% threshold), with the vast majority of those patients (7/8) below the limit of detection of the assay (5×10-5), highlighting the depth of MRD clearance.

With a median follow-up of 22 months, the median duration of response among patients with CR/CRh was 10.5 months for the entire cohort, 10.7 months among NPM1m patients, not reached among KMT2Ar patients, and 5.9 months among NUP98r patients. The observed 1-year overall survival (OS) rate was 56% for the entire cohort, 63% among NPM1m patients, 47% among KMT2Ar patients, and 67% among NUP98r patients. Median OS after HSCT was not reached. Patients who were MRD negative by flow cytometry had a longer median duration of response (20 months vs. 2.9 months) and OS (not reached vs. 8.4 months) compared to those who were MRD positive.

Notably, clinical activity was observed among patients with prior exposure to venetoclax, a population in whom outcomes are typically poor, with a historical estimated median survival of 2.4 months. In this trial, the CR/CRh rate was 50% (11/22) in patients with venetoclax exposure versus 70% (14/20) in those without. The median OS observed was similar between the two groups (at least 12 months in both groups), based on a Kaplan-Meier estimate. This observation supports a potential biologic synergy between BCL2 inhibition and menin inhibition and the possibility that menin inhibition may restore sensitivity to BCL2 inhibition after resistance has developed.

Revumenib was generally well-tolerated in combination with decitabine/cedazuridine and venetoclax. The most common treatment-emergent adverse events (TEAEs) included elevations in aspartate aminotransferase or alanine aminotransferase (71%), nausea (52%), and vomiting (48%). The most common Grade ≥3 TEAEs were febrile neutropenia (36%), lung infection (21%), thrombocytopenia (21%), and elevations in aspartate aminotransferase or alanine aminotransferase (21%). Rates of Grade ≥3 differentiation syndrome (5%) and QTc prolongation (5%) were both low.

About Revuforj (revumenib)

Revuforj (revumenib) is the first and only menin inhibitor that is FDA approved for the treatment of adult and pediatric patients one year and older with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options or R/R acute leukemia with a KMT2A translocation as determined by an FDA-authorized test.

Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.

Revuforj (revumenib)

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME, QTc PROLONGATION, and TORSADES DE POINTES

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

QTc prolongation and Torsades de Pointes have occurred in patients receiving Revuforj. Correct hypokalemia and hypomagnesemia prior to and during treatment. Do not initiate Revuforj in patients with QTcF > 450 msec. If QTc interval prolongation occurs, interrupt, reduce, or permanently discontinue Revuforj.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, rash, and/or hypotension.

In clinical trials, DS occurred in 60 (25%) of 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia. Among those with a KMT2A translocation, DS occurred in 33% of patients with acute myeloid leukemia (AML), 33% of patients with mixed-phenotype acute leukemia (MPAL), and 9% of patients with acute lymphoblastic leukemia (ALL); DS occurred in 18% of patients with NPM1m AML. DS was Grade 3 or 4 in 12% of patients and fatal in 2 patients. The median time to initial onset was 9 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.

Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours in adults or dexamethasone 0.25 mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.

QTc Interval Prolongation and Torsades de Pointes: Revuforj can cause QT (QTc) interval prolongation and Torsades de Pointes.

Of the 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia in clinical trials, QTc interval prolongation was reported as an adverse reaction in 86 (36%) patients. QTc interval prolongation was Grade 3 in 15% and Grade 4 in 2%. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 10%, and the increase from baseline QTcF was greater than 60 msec in 24%. Revuforj dose reduction was required for 7% due to QTc interval prolongation. QTc prolongation occurred in 21% of the 34 patients less than 17 years old, 35% of the 146 patients 17 years to less than 65 years old, and 46% of the 61 patients 65 years or older. One patient had a fatal outcome of cardiac arrest, and one patient had non-sustained Torsades de Pointes.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to and throughout treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia
Embryo-Fetal Toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 9 (4%) patients who received Revuforj, including 4 with sudden death, 2 with differentiation syndrome, 2 with hemorrhage, and 1 with cardiac arrest.

Serious adverse reactions were reported in 184 (76%) patients. The most frequent serious adverse reactions (≥10%) were infection (29%), febrile neutropenia (20%), bacterial infection (15%), differentiation syndrome (13%), and hemorrhage (11%).

The most common adverse reactions (≥20%) including laboratory abnormalities, were phosphate increased (51%), hemorrhage (48%), nausea (48%), infection without identified pathogen (46%), aspartate aminotransferase increased (44%), alanine aminotransferase increased (40%), creatinine increased (38%), musculoskeletal pain (37%), febrile neutropenia (37%), electrocardiogram QT prolonged (36%), potassium decreased (34%), parathyroid hormone intact increased (34%), alkaline phosphatase increased (33%), diarrhea (29%), bacterial infection (27%), triglycerides increased (27%), phosphate decreased (25%), differentiation syndrome (25%), fatigue (24%), edema (24%), viral infection (23%), decreased appetite (20%), and constipation (20%).

DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

Strong CYP3A4 inhibitors: reduce Revuforj dose
Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec
SPECIFIC POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.

Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.

Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.

Pediatric: monitor bone growth and development in pediatric patients.

Geriatric: no overall differences were observed in the effectiveness of Revuforj between patients who were 65 years and older, and younger patients. Compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.

To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information, including BOXED WARNINGS.

(Press release, Syndax, JUN 11, 2026, View Source [SID1234666574])