On June 15, 2025 Genmab A/S (Nasdaq: GMAB) reported new results from the Phase 1b/2 EPCORE NHL-2 trial Arm 10 (NCT04663347), evaluating epcoritamab, a T-cell engaging bispecific antibody administered subcutaneously, in combination with rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) in adult patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) who are eligible for autologous stem cell transplantation (ASCT) (Press release, Genmab, JUN 15, 2025, View Source [SID1234653895]). Results demonstrated an overall response rate (ORR) of 87 percent, a complete response (CR) rate of 65 percent and a partial response (PR) of 23 percent. The majority of patients (65 percent) proceeded to ASCT. At six months, an estimated 81 percent of responses were ongoing, 74 percent of patients were progression free, and 100 percent of patients were alive. These results were shared today during an oral presentation at the 30th European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress.

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The safety profile of this combination therapy showed cytokine release syndrome (CRS) being low grade and no discontinuations due to treatment-emergent adverse events (TEAEs). The most common TEAEs were neutropenia (74 percent), anemia (68 percent), and thrombocytopenia (68 percent). CRS occurred in 52 percent; all were low grade (1/2) and resolved. One patient had immune effector cell-associated neurotoxicity syndrome (ICANS; grade 1), which resolved. No clinical tumor lysis syndrome was observed. Infections occurred in 18 patients (58 percent); five (16 percent) had serious infections. There were no Grade 5 TEAEs.

"These results are particularly encouraging because many of the patients in this study had high-risk disease, having progressed rapidly after initial treatment," said Raul Cordoba, MD, PhD, Head of the Lymphoma Unit at the Fundacion Jimenez Diaz University Hospital, Madrid, Spain. "This combination therapy of epcoritamab plus rituximab, ifosfamide, carboplatin, and etoposide (R-ICE) offers a potential new treatment option for patients with relapsed/refractory diffuse large B-cell lymphoma, providing high response rates and a bridge to potentially curative autologous stem cell transplantation."

Among patients in the study who progressed within 12 months after first-line treatment (n=20), epcoritamab in combination with R-ICE demonstrated an 85 percent ORR and 55 percent CR. Patients in the study who progressed after 12 months from first-line therapy experienced a 91 percent ORR and 82 percent CR. Additionally, patients with one prior line of therapy experienced an 88 percent ORR and 68 percent CR, and patients who were treated with more than one prior line of therapy experienced an 83 percent ORR and 50 percent CR.

"The results from this trial highlight the potential of this investigational epcoritamab containing regimen, especially in patients who progress quickly after initial treatment, and reinforce our joint efforts with AbbVie to develop epcoritamab as a core therapy for B-cell lymphomas, especially as we develop epcoritamab in earlier lines of therapy and a broader patient population," said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. "Our comprehensive EPCORE clinical trial program is dedicated to advancing epcoritamab as both monotherapy and in combination to address the significant unmet need in relapsed/refractory diffuse large B-cell lymphoma and other hematologic malignancies."

Use of epcoritamab + R-ICE in patients with R/R DLBCL eligible for ASCT is not approved and the safety and efficacy of epcoritamab for use as a combination therapy in DLBCL have not been established.

About Diffuse Large B-Cell Lymphoma
DLBCL is the most common type of non-Hodgkin’s lymphoma (NHL) worldwide, accounting for approximately 25-30 percent of all NHL cases. In the U.S., there are approximately 25,000 new cases of DLBCL diagnosed each year. DLBCL can arise in lymph nodes as well as in organs outside of the lymphatic system, occurs more commonly in the elderly and is slightly more prevalent in men. DLBCL is a fast-growing type of NHL, a cancer that develops in the lymphatic system and affects B-cell lymphocytes, a type of white blood cell. For many people living with DLBCL, their cancer either relapses, which means it may return after treatment, or becomes refractory, meaning it does not respond to treatment. Although new therapies have become available, treatment management can remain a challenge.

About the EPCORE NHL-2 Trial
EPCORE NHL-2 is a Phase 1b/2 open-label interventional trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics/biomarkers, immunogenicity, and preliminary efficacy of epcoritamab as a monotherapy and in combination with other standard of care agents in patients with B-cell non-Hodgkin’s lymphoma (B-NHL). The trial consists of two parts: Part 1 (Dose Escalation) and Part 2 (Dose Expansion). The primary objective of Part 1 is safety, and the primary goal of Part 2 is preliminary efficacy. The primary endpoint was overall response rate (ORR) based on best overall response per Lugano criteria. MRD negativity was assessed as a secondary endpoint.

Arm 10 of the EPCORE NHL-2 study enrolled 31 patients with R/R DLBCL, who were eligible for R-ICE and ASCT, and had received ≥1 prior line of treatment. At the time of data cutoff (December 18, 2024), median follow-up was 11 months (range, 6−15). Among the 31 patients treated with epcoritamab 48 mg + R-ICE, 61 percent were Ann Arbor stage III/IV, 42 percent had bulky disease ≥7 cm, 81 percent had one prior LOT (range, 1−3), and 65 percent had progressed within 12 months of first-line treatment. More information on this trial can be found at View Source (NCT: 04663347).

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.i

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 several territories. Where approved, epcoritamab is available as a readily accessible therapy without the need for reducing tumor burden ("debulking"). Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies will 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 R/R FL indication and additional approvals for the R/R 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 five ongoing Phase 3, open-label, randomized trials including a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL compared to investigators choice chemotherapy (NCT04628494), a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) in patients with R/R FL (NCT05409066), a trial evaluating epcoritamab in combination with rituximab and lenalidomide (R2) compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744), and a trial evaluating epcoritamab in combination with R2 compared to chemotherapy infusion in patients with R/R DLBCL (NCT06508658). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.

On June 13, 2025 Valar Labs, a pioneer in AI-powered diagnostics for oncology, reported the publication of its latest research in European Urology, the highest-impact journal in the field of urology (Press release, Valar Labs, JUN 13, 2025, View Source [SID1234653892]). The peer-reviewed study highlights the performance and clinical impact of Valar Labs’ Vesta platform in prognosticating outcomes for patients with high grade Ta non muscle invasive bladder cancer, a population frequently on the borderline of guideline-based risk stratification who may receive different treatment depending on which guideline their provider uses.

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Bladder cancer treatment remains highly variable in its outcomes, with limited tools available to tailor therapies to individual patients. Valar Labs’ Vesta platform, built using advanced artificial intelligence, analyzes pathology to provide critical clinical insights. The newly published study demonstrates that Vesta Risk Stratify biomarkers accurately predicts recurrence and disease progression and outperforms existing guideline-based risk stratification, offering a transformative step forward in precision oncology for bladder cancer. Vesta allows for tailored management based on a patient’s individual risk.

"HGTa tumors carry differing risks as current guidelines attempt to address, and clinical factors such as tumor focality and size can help delineate the true nature of these tumors. These clinical factors, however, are somewhat crude and can be misleading at times. This study demonstrated that CHAI biomarkers can offer improved and additional risk stratification that can go beyond traditional risk factors to better prognosticate outcomes in this population." – Dr. Sam S. Chang, Chief Surgical Officer, Vanderbilt Ingram Cancer Center

"The ability to derive clinically meaningful predictive and prognostic information from routinely-collected pathology is a paradigm shift for the risk stratification of bladder cancer patients." – Dr. Siamak Daneshmand, Professor of Urology and Medicine (Oncology), and Director of Urologic Oncology, University of Southern California

The multi-center validation study was conducted in collaboration with leading academic centers and underscores both the reproducibility and robustness of the CHAI biomarkers. These findings further establish Vesta as a leading precision oncology platform in the management of non-muscle-invasive bladder cancer.

"This publication is a testament to the clinical significance and scientific rigor of our work," said Anirudh Joshi, CEO of Valar Labs. "Vesta’s prognostic and predictive tests represent a leap forward in precision medicine for bladder cancer—providing physicians and patients with data-driven insights to guide treatment decisions more effectively."

"This impactful work continues to build on Vesta’s evidence base as the leading predictive and prognostic biomarker portfolio in bladder cancer. We are excited for patients and providers to benefit from this technology," said Viswesh Krishna, CTO of Valar Labs.

Valar Labs continues to expand its suite of AI-driven diagnostic tests under the Vesta portfolio and beyond, aiming to improve outcomes across a range of cancers. For more information or to access the full study, please visit View Source

On June 13, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported that Professor Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University, will present updated data for Xervyteg (MaaT013) in treating acute Graft-versus-Host Disease (aGvHD) under the Early Access Program (EAP) at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress 2025 (Press release, MaaT Pharma, JUN 13, 2025, View Source [SID1234653891]). This independent EAP dataset further supports the efficacy and safety profile of Xervyteg previously shown in the pivotal ARES trial. It also confirms the breakthrough potential of Xervyteg for aGvHD patients with limited treatment options and it also serves as supportive data within the Marketing Authorization Application (MAA) recently submitted to the European Medicines Agency (EMA).

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Key highlights:

aGvHD is a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. The patients (N=173) treated in EAP previously failed 1 to 6 aGvHD systemic treatment lines and most had grade III (49%) or IV (38%) aGvHD. The real-world data presented underscores the favorable safety profile of Xervyteg the strong and durable responses, translating into increased overall survival:
Gastrointestinal Overall Response Rate (GI-ORR) of 53% at D28, with Complete Response (CR) observed in 30% of patients; all-organ Overall Response Rate (ORR) was 50% with 26% CR.
Response is maintained at D56 indicating a long-term disease control with a GI-ORR of 47% and an ORR considering all organs of 46%.
Overall Survival (OS) in all patients was 55% at 6 months, 48% at 12 months, 44% at 24 months.
Xervyteg displayed a good overall safety profile in the EAP population.
OS was significantly higher in patients who responded to Xervyteg (MaaT013) compared to non-responders (69% versus 25% at 12 months, and 61% versus 25% at 24 months).
Median survival in all patients was 312 days. In responder patients, median survival was 834 days vs 69 days in non-responders.
A subset of patients (n=70) failing both steroid resistant (SR) and ruxolitinib resistant (RR) and thus resembling the cohort enrolled in the pivotal Phase 3 ARES trial (NCT04769895), exhibited a significant and consistent efficacy profile:

At both Day 28 and Day 56, Xervyteg demonstrated durable efficacy in SR/RR aGvHD patients, with GI-ORRs of 57% and Complete Response (CR) observed in 44% of patients at D28 and 51% at D56. All-organs ORR was 54% with 41% CR at D28, and 55% with 48% CR at D56.
OS was 55% at 6 months, 51% at 12 months, 40% at 24 months.
OS was significantly higher in patients who responded to Xervyteg compared to non-responders (77% versus 14% at 12 months, and 59% versus 14% at 24 months).
Median survival in all 70 patients was 445 days. In responder patients, median survival was 834 days vs 53 days in non-responders.
The complete data may be found here.

"The consistency between the real-world Early Access Program data and our pivotal ARES trial underscores Xervyteg’s clinical benefit for patients with severe, treatment-resistant aGvHD," said Dr. Gianfranco Pittari, PhD, Chief Medical Officer at MaaT Pharma. "This is particularly meaningful for clinicians and patients, as it confirms the potential of microbiome therapies to deliver long-term survival benefits in a population with historically poor outcomes."

In comparison, historical data from Abedin et al. 2021 demonstrated that in a similar population of patients, i.e. third-line aGvHD patients receiving additional treatment after ruxolitinib failure, the median survival was only 28 days.

"Among patients who responded by Day 28, the majority achieved a complete resolution of aGvHD symptoms — a strong predictor of sustained disease control over time. The overall safety profile is favorable in this high-risk patient population," outlines Professor Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University.

Details of the Oral Presentation:

Title: Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe
Abstract number: S260
Presenting Author: Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University
Session title: s424 Stem cell transplantation – Session 2
Date & Time: 13/06/2025 (17:00 – 17:15 CEST) – Brown Hall 3
MaaT Pharma also presented a poster on the design of its ongoing Phase 2b trial (PHOEBUS) evaluating MaaT033 to enhance overall survival in allo-HSCT. This international, multi-center trial (NCT05762211) is the largest randomized controlled study to date of a microbiome-based therapy in oncology, enrolling up to 387 patients across 60 sites.

On June 13, 2025 City of Hope, one of the largest and most advanced cancer research and treatment organizations in the U.S. with its National Medical Center named top 5 in the nation for cancer by U.S. News & World Report, reported the first study to demonstrate that characterizing genetic material near chromosomes forecasts how mutated, cancer-causing genes reengineer DNA and alter the tumor microenvironment (Press release, City of Hope, JUN 13, 2025, View Source [SID1234653890]). The leading-edge brain cancer research provides foundational knowledge that one day will improve the practice of precision medicine and allow oncologists to deliver more personalized therapies to cancer patients.

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Tiny DNA molecules outside of chromosomes were once disregarded, but in the past decade research has revealed that these circles called extrachromosomal DNA, or ecDNA, fuel cancer by breaking the laws of biology.

"Our study offers new insights into the interplay between different ecDNA. Importantly, when there is a prevalence of ecDNA and cancer-causing ingredients like the EGFR protein or tumor protein p53, the tumor microenvironment becomes hypoxic. It falls into a state of reduced oxygen, which has been linked to cancer progression, resistance to therapy and poor clinical outcomes," said David Craig, Ph.D., professor and chair of the Department of Integrative Translational Sciences at City of Hope and co-corresponding author of a study published today in Nature Communications.

Spatial transcriptomics (measuring and mapping of DNA activity) combined with genomic data can help identify groups of cells within a tumor that share a common ancestor but have acquired additional mutations. How they are distributed spatially informs the understanding of tumor evolution. The new translational science data underpins City of Hope’s precision medicine research, which applies innovation to create better outcomes with fewer side effects for more patients. City of Hope aspires to cure more cancer patients.

City of Hope researchers led a team that performed bulk RNA sequencing, tumor/normal DNA sequencing and spatial transcriptomics in a small sample of gliomas — tumors that develop in the brain or spinal cord. Through varied experiments and validation cohorts, they were able to identify common and distinct characteristics of the tumor microenvironment, developing an integrated analysis framework that can be leveraged by others.

"While our paper solely evaluated different types of brain cancers, the spatial transcriptomic principles and genome sequencing techniques we outlined will one day enable physicians to provide more personalized therapies for cancer patients," said Gabriel Zada, M.D., a neurosurgeon with Keck Medicine of USC, professor of neurological surgery and physiology and neuroscience at the Keck School of Medicine of USC, co-director of the USC Brain Tumor Center and co-corresponding study author. "Cancer and its treatment is not one-size-fits-all. Understanding the molecular activity of ecDNA near inheritable and non-inheritable cells provides deep insights into potential therapeutic targets and risk of cancer recurrence."

The researchers demonstrated that ecDNA drives rapid cancer cell (oncogene) proliferation outside of chromosomes, the thread-like structures inside the cell nucleus that houses DNA and RNA. EcDNA contributes to the development of gliomas, genetic instability and distinct tumor cell populations within a single tumor, making cancer more difficult to eliminate.

The dynamic nature of ecDNA may be what enables cancer cells to adapt and reprogram their genomes, driving tumor progression in response to changes in their microenvironment, including changes resulting from therapies.

"We have now demonstrated how cancer cells dynamically reprogram their own genome to control and respond to the tumor microenvironment. By uncovering these mechanisms, we are paving the way for more precise and effective treatments tailored to the unique biology of each patient," Dr. Craig said.

The Nature Communications study entitled "Resolving Subclonal Genomic Heterogeneity in Gliomas by Integrating Spatial Transcriptomics with Loss of Heterozygosity and Extrachromosomal DNA Analysis" was supported by the National Center for Advancing Translational Science of the U.S. National Institutes of Health (KL2TR001854).

On June 13, 2025 Enliven Therapeutics, Inc. (Enliven or the Company) (Nasdaq: ELVN), a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapeutics, reported updated, positive data from the Phase 1 ENABLE clinical trial evaluating ELVN-001 in patients with chronic myeloid leukemia (CML) in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress taking place June 12-15 in Milan, Italy, and virtually (Press release, Enliven Therapeutics, JUN 13, 2025, View Source [SID1234653888]).

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"Thanks to the success of tyrosine kinase inhibitors (TKIs), patients with CML now have a near-normal life expectancy. As a result, treatment goals have evolved beyond response and survival to also prioritize quality of life and tolerability," said Andreas Hochhaus, Professor of Internal Medicine, Hematology and Oncology and Head of the Department of Hematology and Medical Oncology at the Jena University Hospital, Germany. "However, significant unmet needs remain, particularly related to treatment resistance and intolerance, across all lines of therapy. The data from ELVN-001 are encouraging, showing an efficacy, safety and tolerability profile that compare favorably to approved BCR::ABL1 inhibitors, despite being studied in a more heavily pretreated population. I look forward to future data, which could support ELVN-001 as a promising new option for patients who need better long-term disease management."

"We are highly encouraged by the ELVN-001 data, specifically as it relates to the consistency of the cumulative and achieved MMR rates as the Phase 1 trial progresses, with more evaluable patients and longer duration of treatment," said Helen Collins, M.D., Chief Medical Officer of Enliven. "While MMR is the efficacy endpoint in CML, safety and tolerability are equally critical given the chronic nature of the disease. ELVN-001 was reported to be well tolerated across all evaluated doses and had low levels of dose reductions and discontinuations, which we believe is the key sign of a favorable safety and tolerability profile. We believe ELVN-001 has the potential to offer best-in-class efficacy and tolerability, which are key attributes for people living with CML. We look forward to sharing additional data in the future."

ELVN-001 is a potent, highly selective, potentially best-in-class small molecule kinase inhibitor designed to specifically target the BCR::ABL gene fusion, the oncogenic driver for patients with CML.

ELVN-001 Data Highlights

Patient Demographics

As of the cutoff date of April 28, 2025, 90 patients have been enrolled in the ongoing Phase 1 trial across dose levels ranging from 10 mg once a day (QD) to 80 mg twice a day (BID).
The vast majority of patients (80%) remain on study with a median treatment duration of ~29 weeks.
Patients enrolled were heavily pretreated:
67% of patients received three or more unique prior TKIs, including 58% of patients who received prior asciminib and 43% of patients who received prior ponatinib.
72% of patients had discontinued their prior TKI due to lack of efficacy.
Encouraging ELVN-001 Efficacy Data by 24 Weeks

Of the enrolled patients, 53 with typical BCR::ABL1 transcripts and without T315I mutations were evaluable for major molecular response (MMR) by 24 weeks.
25 of 53 (47%) evaluable patients were in MMR by 24 weeks, with 13 of 41 (32%) achieving and 12 of 12 (100%) maintaining MMR.
Of those resistant to their last TKI, 14 of 34 (41%) were in MMR by 24 weeks.
Of those previously treated with asciminib or ponatinib, 12 of 34 (35%) were in MMR by 24 weeks.
All patients who achieved or maintained MMR were still in MMR at the time of data cutoff.
These data continued to compare favorably to precedent Phase 1 MMRs for approved BCR::ABL1 TKIs, particularly given the more heavily pretreated patient population in the ELVN-001 clinical trial.
Specifically, the achieved MMR rate by 24 weeks of 32% compares favorably with historical data from less heavily pretreated patients receiving asciminib, which showed achieved MMR rates of 24% in the Phase 1 trial and 25% in the ASCEMBL Phase 3 trial.
ELVN-001’s Safety Profile Consistent with High Selectivity for ABL1

ELVN-001 remains well-tolerated across all evaluated doses.
Only 3.4% (3 of 87) of patients had dose reductions due to treatment-emergent adverse events (TEAEs) and 4.6% (4 of 87) of patients discontinued due to TEAEs.
The majority of TEAEs were low frequency and low grade, and the hematologic TEAE profile was similar to or better than the approved TKIs.
Only 2.3% (2 of 87) of patients experienced ≥ Grade 3 non-hematologic treatment-related AEs.
No evidence to date of enhanced cardiovascular toxicity and no treatment-related arterial occlusive events (AOEs).
The maximum tolerated dose was not reached, and no exposure-toxicity relationship was observed.
ELVN-001 Pharmacokinetic (PK) Profile

The PK profile supports once-daily dosing with flexible administration requirements, including the ability to take with or without food.
There is low potential for drug-drug interactions, an important advantage given that the average CML patient takes approximately five concurrent medications.
"We believe there remains significant opportunity to improve upon existing therapies," said Sam Kintz, Co-founder and Chief Executive Officer of Enliven. "Based on today’s encouraging Phase 1 update, we believe ELVN-001 has the potential to compete across all lines of therapy. We believe that precedent registrational trials in CML provide a roadmap for the regulatory pathway for ELVN-001, and the use of biomarker-based endpoints, like MMR, enables smaller, faster studies. Importantly, historical Phase 1 data in late-line CML trials have predicted success in subsequent pivotal trials. Building off this exciting update, we expect to initiate our first head-to-head Phase 3 pivotal trial in 2026 and remain confident in ELVN-001 and its potential positioning in the future in the CML treatment paradigm."

The oral presentation titled: "ENABLE: A Phase 1a/1b Study of ELVN-001, a selective active site inhibitor of BCR::ABL1, in patients with previously treated CML" will be presented by Andreas Hochhaus, Professor of Internal Medicine, Hematology and Oncology and Head of the Department of Hematology and Medical Oncology at the Jena University Hospital, Germany later today. A copy of the presentation will be available on the "Program Presentations & Publications" section of the Company’s website at www.enliventherapeutics.com.

Webcast and Conference Call Information
Enliven will host a live webcast and conference call today at 1:30 p.m. ET / 7:30 p.m. CEST. To participate in the live event, please register using this link. Following registration, participants will have access to dial in numbers and a unique passcode should they prefer to participate by phone. The event and accompanying slides can also be accessed by visiting the investor relations section of the Company’s website at View Source An archived webcast will be available on the Company’s website following the event.

About the ENABLE Trial
The ENABLE study (NCT05304377) is a Phase 1 study of ELVN-001 in patients with previously treated CML. The trial is currently in Phase 1a/1b development and is a dose escalation and expansion trial designed to evaluate safety and tolerability and to determine the recommended dose for further clinical evaluation of ELVN-001 in patients with CML with and without T315I mutations that is relapsed, refractory or intolerant to TKIs. Secondary endpoints include pharmacokinetics, MMR by central quantitative reverse transcriptase polymerase chain reaction, duration of MMR, BCR::ABL1 transcript levels and complete hematologic response. Enliven is preparing for the potential start of a pivotal trial for ELVN-001 in 2026.

About ELVN-001
ELVN-001 is a potent, highly selective, potentially best-in-class small molecule kinase inhibitor designed to specifically target the BCR::ABL gene fusion, the oncogenic driver for patients with chronic myeloid leukemia. As a highly selective active site inhibitor, ELVN-001 has a mechanism of action that is complementary to allosteric BCR::ABL1 inhibitors, which may play an increasingly important role in the standard of care. ELVN-001 was also designed to have activity against the T315I mutation, the most common BCR::ABL1 mutation, which confers resistance to nearly all approved TKIs, as well as activity against mutations known to confer resistance to allosteric BCR::ABL1 inhibitors.