Great Novel Therapeutics Biotech & Medicals Corporation (GNTbm) Presented Preclinical Data on GNTbm-38, an Novel Epigenetic Immune Activator, at the 2025 ASCO Annual Meeting

On May 22, 2025 GNTbm (stock code: 7427, Taiwan) reported the preclinical data on GNTbm-38, an novel epigenetic immune activator for cancer immunotherapy. GNTbm-38 was presented as posters at the 2025 American Association of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, which is held in Chicago, USA, from May 30 to Jun 04, 2025 (Press release, GNT Biotech & Medicals, MAY 22, 2025, View Source;medicals-corporation-gntbm-presented-preclinical-data-on-gntnm-38-an-novel-epigenetic-immune-activator-at-the-2025-asco-annual-meeting-302461449.html [SID1234653327]).

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Abstract: 2574

Title: Preclinical development of GNTbm-38, a novel class I histone deacetylase inhibitor, while combined with anti-VEGFR TKI or anti-PD-1 Ab: Assessment of immune activation and immune memory in cancer immunotherapy.

Session Date/Time: 6/2/2025, 1:30 PM-4:30 PM CT

Poster Board Number: 221

At present, there is no ICI-based drug combination for the treatment of advanced MSS colorectal cancer, which is a cold tumor. Studies have shown that epigenetic regulators such as class I HDAC inhibitors are an emerging and important drug component for combination therapy that can greatly increase the anti-cancer benefits of cancer immunotherapy. Therefore, rational drug combinations, containing class I HDACi, may provide opportunities in cancer immunotherapy.

GNTbm-38 is a new drug candidate independently developed by GNTbm. GNTbm-38 acts as a TME reprogramming regulator in immunotherapy. When combined with TKI, GNTbm-38 significantly improved tumor response rate and survival rate through synergistic effect by normalizing tumor vessels, increasing tumor antigen presentation, increasing activated CD8+ T cell infiltration into tumors, inducing memory T cell persistence, and inhibiting mobilization of immunosuppressive cells into tumors. On the other hand, treatment with GNTbm-38 plus anti-PD-1 antibody in the CT-26 model showed greatly improved tumor response rate and survival rate with a strong synergistic effect. Furthermore, in B-hPD-1/hPD-L1 mice (humanized model) subcutaneously injected with B-hPD-L1 CT-26 cells, treatment of pembrolizumab and GNTbm-38 resulted in a 46.5% inhibition on tumor growth. Therefore, our data provided a strong rationale to explore the combination of GNTbm-38 with anti-VEGF TKI with or without ICI. From these data GNTm-38 has been shown to display powerful induction of immune activation and immune memory in combination therapy with TKI/ICI against colon CT-26 cold tumor. Based on the in vitro, in vivo and preclinical studies, these data show that GNTbm-38 exhibits markedly superior pharmacokinetics, tolerability, and efficacy in animal models. It is expected to complete the US IND application by the end of 2025 and enter the clinical study.

About GNTbm-38

GNTbm-38 is a new chemical entity with potential as a pivotal drug component of a new generation of cancer immunotherapy independently developed by GNTbm. It is a drug candidate selected by an immuno-competent tumor-bearing animal testing platform, and has undergone many preclinical research studies to confirm that it has very outstanding anti-cancer activity in tumor microenvironment. GNTbm-38 is an oral drug with dual effects of epigenetic regulation of gene expression and immune activation, which is unique when compared to the mechanism of other epigenetic drugs. GNTbm-38 can remodel the tumor microenvironment (TME) through a unique epigenetic regulatory mechanism, including the cell composition and gene expression that affect the TME, so that "cold tumors" can be transformed into "hot tumors", which can attract CTL to infiltrate into the TME, and at the same time, it can also reduce the attraction of immunosuppressive cells (such as : TAM, Treg, and MDSCs) into the TME, so as to achieve the remodeling of the TME, which is more conducive to obtaining the therapeutic benefits of cancer immunotherapy. GNTbm-38 monotherapy can be used in the treatment of hematological tumors, and GNTbm-38 can also be combined with a unique multi-kinase inhibitor or immune checkpoint inhibitor for treatment of a variety of solid tumors, mainly through a unique anti-cancer immune-regulating mechanism to achieve anti-cancer treatment goals.

Eisai to Present E7386, Co-created by PRISM BioLab and Eisai, at the ASCO (American Society of Clinical Oncology) Annual Meeting

On May 22, 2025 PRISM BioLab, Co. Ltd. ("PRISM"), a leading discovery and development biotechnology company designing small molecule inhibitors of protein-protein interaction (PPI) targets, reported that the analysis of a combination study of E7386(*1), created through collaboration research with Eisai Co., Ltd. ("Eisai"), and Lenvatinib mesylate ("lenvatinib") (*2) will be presented by Eisai at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Congress 2025, held in Chicago, USA from May 30 to June 3, 2025 (Press release, Eisai, MAY 22, 2025, View Source [SID1234653326]). The abstract of the study has been released today.

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To determine the optimal dose of E7386 in combination with Lenvatinib in the open-label Phase Ib study (NCT04008797(*3)), expansion cohort of advanced endometrial cancer patients progressed following platinum-based chemotherapy and anti-PD-(L)1 immunotherapy have been implemented by Eisai and the enrollment of 30 patients was completed. By data cutoff (Oct 22, 2024), with 9 patients remaining on treatment, 30% (9 patients) showed the confirmed response (decrease of tumor size > 30%) for an overall response rate of 30.0%. Furthermore, among patients without prior Lenvatinib treatment, the overall response rate was 42.9%.

Completing the enrollment of dose expansion cohort (n=30), the results confirmed promising preliminary antitumor activity of E7386 + Lenvatinib with a manageable safety profile. For the subsequent dose-optimization part for E7386 + LEN in advanced endometrial cancer, enrollment of patients had been initiated (NCT04008797).

(*1) E7386

E7386 is an orally available small molecule CBP/ β-catenin inhibitor that inhibits protein-protein interactions between the transcription factor CBP and β-catenin, and regulates the Wnt signaling. E7386 achieved clinical POC (Proof of concept) in October 2021 and following clinical studies are ongoing including phase I for solid tumors as monotherapy, Phase Ib for solid tumors in combination with tyrosine kinase inhibitor Lenvatinib, Phase Ib/II for solid tumors in combination with pembrolizumab, the anti-PD-1 antibody from Merck & Co., Inc., Rahway, NJ, USA.

(*2) Lenvatinib

Lenvatinib is a multi-kinase inhibitor, discovered by Eisai and being co-developed and co-commercialized under a collaboration agreement with Merck & Co., Inc., Rahway, NJ, USA, which inhibits vascular endothelial growth factor receptors (VEGFRs),VEGFR1, VEGR2, VEGFR3 and fibroblast growth factor receptors (FGFRs), FGFR1, FGFR2, FGFR3, FGFR4, and other receptor tyrosine kinases, PDGFR-alpha, KIT, RET. Lenvatinib have been approved for thyroid cancer, hepatocellular carcinoma, thymic caner and renal cell carcinoma (in combination with Everolimus or pembrolizumab, the anti-PD-1 antibody from Merck & Co., Inc., Rahway, NJ, USA.). Lenvatinib is also approved for endometrium cancer in combination with pembrolizumab

(*3) NCT04008797

NCT04008797 is an open-label Phase Ib study of E7386 in combination with other anticancer drug, Lenvatinib for the patients with solid tumors. The study has been implemented by Eisai in Japan, Korea, Taiwan region, US, and France determine the safety and the recommended phase 2 dose (RP2D) and also to see the pharmacokinetics and efficacy of E7386 + Lenvatinib. Enrolment of each cohort of hepatic, colon, endometrial cancers are ongoing.

Transgene to Present 24-month Disease-Free Survival Data from All Patients in Phase I Part of Trial of Individualized Cancer Vaccine, TG4050, at ASCO 2025

On May 22, 2025 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported that it will present two-year disease-free survival (DFS) data from all patients in the randomized Phase I part of the trial of its individualized neoantigen therapeutic cancer vaccine TG4050 (Press release, Transgene, MAY 22, 2025, View Source [SID1234653325]). The data will be shared during a rapid oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place in Chicago from May 30 to June 3, 2025.

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TG4050 is a viral vector-based immunotherapy based on Transgene’s myvac platform and powered by NEC’s cutting-edge AI capabilities designed to optimize antigen selection.
TG4050 is being evaluated in a randomized multicenter Phase I/II trial as a single agent in the adjuvant treatment of HPV-negative head and neck cancers (NCT04183166). Based on promising Phase I data, Transgene and partner NEC have advanced the joint development of TG4050 in this indication with a Phase II extension of the trial, which is currently enrolling patients.

Key data to be presented include:

Safety in locally advanced HPV-negative resectable head and neck squamous cell carcinoma (HNSCC),
Disease free survival after 2-year follow-up,
Updated immunogenicity data.
Prof. Le Tourneau, MD, PhD, Head of the Department of Drug Development and Innovation (D3i) at Institut Curie, and Principal Investigator commented: "It is a privilege to present these study data which are based on the highly promising myvac platform for individualized cancer vaccines. This oral presentation at ASCO (Free ASCO Whitepaper) offers an opportunity to highlight both the strength of the clinical data and the collaborative efforts of everyone involved in the trial."

Dr. Emmanuelle Dochy, MD, Chief Medical Officer of Transgene, added: "We are pleased to have our latest findings on TG4050 selected for presentation among so many high-quality submissions to ASCO (Free ASCO Whitepaper). This recognition reflects the dedication of our teams and the significance of our work in advancing medical science and patient care for those suffering from cancer. The Phase II part of our trial is currently enrolling patients internationally, as we continue to evaluate TG4050’s potential in this setting."

The rapid oral presentation will take place on June 1st at 1:30 p.m. CDT during ASCO (Free ASCO Whitepaper) 2025 and will also be available to view on Transgene’s website.

In addition, Transgene will present a poster at ASCO (Free ASCO Whitepaper) on TG4001, presenting randomized Phase II data in combination with avelumab in a cervical cancer subgroup. Following the previously reported topline results from this study in the overall patient population (see press release here), Transgene is currently evaluating potential partnership opportunities to determine the best path forward for the program.

BeiGene Presents Pioneering Cancer Research at ASCO 2025 – Redefining Treatment Across Hematology and Solid Tumors

On May 22, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that will change its name to BeOne Medicines Ltd., reported it will share 23 abstracts featuring new data across its hematology and solid tumor portfolio at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL, May 30 – June 3, 2025 (Press release, BeiGene, MAY 22, 2025, View Source [SID1234653324]). With two abstracts selected for rapid oral presentation, these data reflect the Company’s vision to address cancer across multiple fronts and provide innovative medicines to as many patients as possible worldwide.

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"ASCO is a powerful platform for highlighting progress in cancer care, and we’re proud to contribute 23 accepted abstracts that reflect our mission to improve outcomes for more patients around the world," said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. "From long-term follow-up results for BRUKINSA in CLL to first-time clinical data for two promising breast cancer assets, our presentations this year speak to the depth and momentum of our oncology portfolio — and our commitment to delivering transformative medicines across a range of cancers."

Presentations feature the impressive clinical profile of BRUKINSA (zanubrutinib) across broad patient populations; notable highlights include:

Long-term data from SEQUOIA Arm C, which evaluated BRUKINSA in patients with treatment naïve (TN) chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) with del(17p) mutations.
First results from the full population from Arm D of the SEQUOIA study, which evaluated BRUKINSA plus venetoclax in patients with TN CLL/SLL with and without del (17p) and/or TP53 mutation.
Robust analyses across clinical trials and real-world evidence that deepen understanding of treatment patterns, safety, and outcomes in CLL and mantle cell lymphoma (MCL).
Highlights include new comparative efficacy data for BRUKINSA versus fixed-duration regimens based on a network meta-analysis, as well as real-world studies evaluating BTK inhibitor use, treatment disparities, and clinical outcomes across diverse patient populations.
Early phase data includes never-before-presented clinical data from BeiGene’s emerging breast cancer pipeline; notable highlights include:

Preliminary results of the dose escalation study for BG-C9074, a topoisomerase inhibitor antibody-drug conjugate (ADC) targeting the B7-H4 protein, in patients with advanced solid tumors, including breast cancer.
Early clinical activity for BG-68501, a cyclin-dependent kinase-2 inhibitor (CDK2i), in HR+/HER2- breast cancer patients with prior CDK4/6i exposure, supporting its development as a next-line option for tumors with CDK2 dependency.
Results from the final analysis of the RATIONALE-213 study demonstrate that, using a PET-guided approach, TEVIMBRA plus chemotherapy or chemoradiotherapy showed promising efficacy and a tolerable safety profile in the neoadjuvant setting for resectable esophageal squamous cell carcinoma (ESCC), in both patients who responded and did not respond to preoperative chemotherapy. This adds further evidence to the PD-1 inhibitor’s established ability to deliver clinically meaningful efficacy benefits as well as its consistent safety profile.

BeiGene Presentations at 2025 ASCO (Free ASCO Whitepaper) Annual Meeting

Abstract Title

Presentation Details (CDT)

Lead Author

Hematology

BRUKINSA

SEQUOIA 5-year follow-up of Arm C: Frontline zanubrutinib monotherapy in del(17p) patients with treatment-naïve chronic lymphocytic leukemia/small lymphocytic lymphoma

Rapid Oral Presentation: 7011

Session Title: Hematologic Malignancies – Lymphoma and Chronic Lymphocytic Leukemia

Session Date/Time: May 31, 2025, 8:00-9:30 AM

C.S. Tam

Combination of zanubrutinib + venetoclax for treatment-naive CLL/SLL: Results from SEQUOIA Arm D

Rapid Oral Presentation: 7009

Session Title: Hematologic Malignancies – Lymphoma and Chronic Lymphocytic Leukemia

Session Date/Time: May 31, 2025, 8:00-9:30 AM

M. Shadman

Solid Tumor

TEVIMBRA

Tislelizumab (BGB-A317) plus chemotherapy/chemoradiotherapy as positron emission tomography-guided neoadjuvant treatment for resectable esophageal squamous cell carcinoma: RATIONALE-213 final analysis

Poster #: 317

Poster Presentation

Session Title: Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary

Session Date/Time: May 31, 2025, 9:00 AM-12:00 PM

L. Chen

Final analysis of multicenter, open-label, phase 2 study evaluating the efficacy and safety of tislelizumab in combination with fruquintinib in patients with selected solid tumors

Poster #: 251

Poster Presentation

Session Title: Developmental Therapeutics – Immunotherapy

Session Date/Time: June 2, 2025, 1:30 PM-4:30 PM

K. Lee

Pipeline

BG-68501 (CDK2i)

A first-in-human, phase 1a/b, dose-escalation/expansion study of BG-68501, a selective CDK2 inhibitor, as monotherapy or in combination with fulvestrant for patients with HR+/HER2- breast cancer and other advanced solid tumors: First disclosure of clinical data

Poster#: 430

Poster Presentation

Session Title: Development Therapeutics – Molecularly Targeted Agents and Tumor Biology

Session Date/Time: June 2, 2025, 1:30-4:30 PM

R. Joshi

BG-C9074

First-in-human study of BG-C9074, a B7-H4-targeting ADC in patients with advanced solid tumors: Preliminary results of the dose-escalation phase

Poster #: 348

Poster Presentation

Session Title: Development Therapeutics – Molecularly Targeted Agents and Tumor Biology

Session Date/Time: June 2, 2025, 1:30-4:30 PM

C.A. Perez

BGB-A445 (OX40)

A phase 1 study of the OX40 agonist, BGB-A445, with or without tislelizumab, an anti-PD-1 monoclonal antibody, in patients with advanced NSCLC, HNSCC or NPC

Poster #: 172

Poster Presentation

Session Title: Development Therapeutics – Molecularly Targeted Agents and Tumor Biology

Session Date/Time: June 2, 2025, 1:30-4:30 PM

M. Hee Hong

A phase 2 study of the OX40 agonist BGB-A445, in combination with docetaxel or BGB-15025, an HPK1 inhibitor, in patients with NSCLC pretreated by anti-PD-(L)1 antibodies

Abstract #: e14513

Online Abstract

T. Min Kim

Additional Abstracts

Clinical Trial Diversity

Lung cancer enrollment of demographic subgroups in US clinical trial sites

Poster #: 216

Poster Presentation

Session Title: Lung Cancer – Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Session Date/Time: May 31, 2025, 1:30-4:30 PM

C. Nigoghossian

Integrative Evidence Generation and Health Economics Related to Zanubrutinib

Preference Survey

Treatment preferences of patients, caregivers, and physicians in follicular lymphoma: A global discrete-choice experiment study

Poster #: 448

Poster Presentation

Session Title: Quality Care/Health Services Research

Session Date/Time: May 31, 2025, 1:30-4:30 PM

M. Smith

Matching-Adjusted Indirect Comparison

Adverse events of interest of zanubrutinib vs. fixed-duration combination of venetoclax and obinutuzumab in treatment-naïve chronic lymphocytic leukemia

Abstract #: e19028

Online Abstract

W. Aldairy

Efficacy of continuous zanubrutinib vs. fixed duration venetoclax in combination with obinutuzumab in treatment-naïve chronic lymphocytic leukemia: A matching-adjusted indirect comparison

Abstract #: e19027

Online Abstract

T. Munir

Comparative efficacy of zanubrutinib versus fixed-duration acalabrutinib plus venetoclax for first-line treatment of chronic lymphocytic leukemia: A matching-adjusted indirect comparison

Abstract #: e91032

Online Abstract

T. Munir

Network Meta-Analysis

A network meta-analysis of efficacy of zanubrutinib versus fixed-duration acalabrutinib plus venetoclax in treatment-naïve chronic lymphocytic leukemia

Abstract #: e19031

Online Abstract

M. Shadman

Real-World Evidence

Real-world comparative effectiveness of first-line Bruton tyrosine kinase inhibitors in patients with chronic lymphocytic leukemia

Abstract #: e23264

Online Abstract

R. Jacobs

Evaluating uptake of targeted agents by race/ethnicity in patients receiving first-line treatment for chronic lymphocytic leukemia

Abstract #: e13741

Online Abstract

A.S. Kittai

Real-world Bruton tyrosine kinase inhibitor use and clinical outcomes among patients with chronic lymphocytic leukemia/small lymphocytic lymphoma

Abstract #: e23271

Online Abstract

J. Hou

Real-world zanubrutinib treatment patterns in chronic lymphocytic leukemia/small lymphocytic lymphoma among US community oncology patients with prior acalabrutinib therapy

Abstract #: e23265

Online Abstract

J. Hou

Real-world zanubrutinib treatment patterns in mantle cell lymphoma among US community oncology patients with prior Bruton tyrosine kinase inhibitor therapy

Abstract #: e23270

Online Abstract

R. Choksi

Risk of hypertension in patients newly diagnosed with chronic lymphocytic leukemia/small lymphocytic lymphoma and treated with covalent Bruton tyrosine kinase inhibitors: A real-world study

Abstract #: e23334

Online Abstract

A.K. Ali

Real-world treatment utilization patterns, discontinuation and healthcare resource utilization of first-line Bruton tyrosine kinase inhibitor therapy in chronic lymphocytic leukemia: Age-related disparity

Abstract #: e19033

Online Abstract

K. Yang

Serious infections in patients with CLL/SLL treated with combination venetoclax and obinutuzumab compared to those treated with zanubrutinib: A real-world study

Abstract #: e19026

Online Abstract

J. Colasurdo

Comparing real-world treatment patterns and outcomes of zanubrutinib and acalabrutinib in CLL/SLL at University of California academic health centers

Abstract #: e23263

Online Abstract

A. Ayati

For additional information about our presence at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting, please visit our meeting hub: congress.beonemedicines.com.

About BRUKINSA (zanubrutinib)

BRUKINSA is an orally available, small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.

BRUKINSA has the broadest label globally of any BTK inhibitor and is the only BTK inhibitor to provide the flexibility of once or twice daily dosing. Additionally, BRUKINSA is also the only BTK inhibitor to demonstrate superiority to another BTK inhibitor in a Phase 3 study of patients with relapsed / refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

The global BRUKINSA clinical development program includes about 7,100 patients enrolled in 30 countries and regions across more than 35 trials. BRUKINSA is approved in more than 75 markets, and more than 200,000 patients have been treated globally.

About TEVIMBRA (tislelizumab-jsgr)

TEVIMBRA is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1 (PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping the body’s immune cells detect and fight tumors.

TEVIMBRA is the foundational asset of BeiGene’s solid tumor portfolio and has shown potential across multiple tumor types and disease settings. The global TEVIMBRA clinical development program includes almost 14,000 patients enrolled to date in 35 countries and regions across 70 trials, including 21 registration-enabling studies. TEVIMBRA is approved in 46 markets, and more than 1.5 million patients have been treated globally.

U.S. Indications and Important Safety Information for BRUKINSA (zanubrutinib)

INDICATIONS

BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with:

Chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
Waldenström’s macroglobulinemia (WM).
Mantle cell lymphoma (MCL) who have received at least one prior therapy.
Relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen.
Relapsed or refractory follicular lymphoma (FL), in combination with obinutuzumab, after two or more lines of systemic therapy.
The MCL, MZL and FL indications are approved under accelerated approval based on overall response rate and durability of response. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.

IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Hemorrhage

Fatal and serious hemorrhage has occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher hemorrhage including intracranial and gastrointestinal hemorrhage, hematuria, and hemothorax was reported in 3.8% of patients treated with BRUKINSA in clinical trials, with fatalities occurring in 0.2% of patients. Bleeding of any grade, excluding purpura and petechiae, occurred in 32% of patients.

Bleeding has occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Coadministration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage.

Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days before and after surgery depending upon the type of surgery and the risk of bleeding.

Infections

Fatal and serious infections (including bacterial, viral, or fungal infections) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA. Grade 3 or higher infections occurred in 26% of patients, most commonly pneumonia (7.9%), with fatal infections occurring in 3.2% of patients. Infections due to hepatitis B virus (HBV) reactivation have occurred.

Consider prophylaxis for herpes simplex virus, pneumocystis jirovecii pneumonia, and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately.

Cytopenias

Grade 3 or 4 cytopenias, including neutropenia (21%), thrombocytopenia (8%) and anemia (8%) based on laboratory measurements, developed in patients treated with BRUKINSA. Grade 4 neutropenia occurred in 10% of patients, and Grade 4 thrombocytopenia occurred in 2.5% of patients.

Monitor complete blood counts regularly during treatment and interrupt treatment, reduce the dose, or discontinue treatment as warranted. Treat using growth factor or transfusions, as needed.

Second Primary Malignancies

Second primary malignancies, including non-skin carcinoma, have occurred in 14% of patients treated with BRUKINSA. The most frequent second primary malignancy was non-melanoma skin cancers (8%), followed by other solid tumors in 7% of the patients (including melanoma in 1% of patients) and hematologic malignancies (0.7%). Advise patients to use sun protection and monitor patients for the development of second primary malignancies.

Cardiac Arrhythmias

Serious cardiac arrhythmias have occurred in patients treated with BRUKINSA. Atrial fibrillation and atrial flutter were reported in 4.4% patients treated with BRUKINSA, including Grade 3 or higher cases in 1.9% of patients. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher ventricular arrhythmias were reported in 0.3% of patients.

Monitor for signs and symptoms of cardiac arrhythmias (e.g., palpitations, dizziness, syncope, dyspnea, chest discomfort), manage appropriately, and consider the risks and benefits of continued BRUKINSA treatment.

Hepatotoxicity, Including Drug-Induced Liver Injury

Hepatotoxicity, including severe, life-threatening, and potentially fatal cases of drug-induced liver injury (DILI), has occurred in patients treated with Bruton tyrosine kinase inhibitors, including BRUKINSA.

Evaluate bilirubin and transaminases at baseline and throughout treatment with BRUKINSA. For patients who develop abnormal liver tests after BRUKINSA, monitor more frequently for liver test abnormalities and clinical signs and symptoms of hepatic toxicity. If DILI is suspected, withhold BRUKINSA. Upon confirmation of DILI, discontinue BRUKINSA.

Embryo-Fetal Toxicity

Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity, including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for 1 week after the last dose. Advise men to avoid fathering a child during treatment and for 1 week after the last dose. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

Adverse Reactions

The most common adverse reactions (≥30%), including laboratory abnormalities, in patients who received BRUKINSA (N=1729) are decreased neutrophil count (51%), decreased platelet count (41%), upper respiratory tract infection (38%), hemorrhage (32%), and musculoskeletal pain (31%).

Drug Interactions

CYP3A Inhibitors: When BRUKINSA is co-administered with a strong CYP3A inhibitor, reduce BRUKINSA dose to 80 mg once daily. For coadministration with a moderate CYP3A inhibitor, reduce BRUKINSA dose to 80 mg twice daily.

CYP3A Inducers: Avoid coadministration with strong or moderate CYP3A inducers. Dose adjustment may be recommended with moderate CYP3A inducers.

Specific Populations

Hepatic Impairment: The recommended dose of BRUKINSA for patients with severe hepatic impairment is 80 mg orally twice daily.

Please see full U.S. Prescribing Information including U.S. Patient Information.

U.S. Indication and Important Safety Information for TEVIMBRA (tislelizumab-jsgr) injection

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Severe and Fatal Immune-Mediated Adverse Reactions

TEVIMBRA is a monoclonal antibody that belongs to a class of drugs that bind to either the programmed death receptor-1 (PD-1) or PD-ligand 1 (PD-L1), blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions.

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue. Immune-mediated adverse reactions can occur at any time after starting treatment with a PD-1/PD-L1 blocking antibody. While immune-mediated adverse reactions usually manifest during treatment with PD-1/PD-L1 blocking antibodies, immune-mediated adverse reactions can also manifest after discontinuation of PD-1/PD-L1 blocking antibodies. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated reactions.

Early identification and management of immune-mediated adverse reactions are essential to ensure safe use of PD-1/PD-L1 blocking antibodies. Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue TEVIMBRA depending on severity. In general, if TEVIMBRA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose immune-mediated adverse reactions are not controlled with corticosteroids.

Immune-Mediated Pneumonitis

TEVIMBRA can cause immune-mediated pneumonitis, which can be fatal. In patients treated with other PD-1/PD-L1 blocking antibodies, the incidence of pneumonitis is higher in patients who have received prior thoracic radiation.

Immune-mediated pneumonitis occurred in 4.9% (96/1972) of patients receiving TEVIMBRA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (1.6%) and Grade 2 (1.9%) adverse reactions. Pneumonitis led to permanent discontinuation of TEVIMBRA in 38 (1.9%) patients and withholding of TEVIMBRA in 32 (1.6%) patients.

Seventy-four (77.1%) of the 96 patients received systemic corticosteroids. Sixty-five (67.7%) of the 96 patients received high-dose systemic corticosteroids. Immune-mediated pneumonitis resolved in 50% of the 96 patients. Of the 32 patients in whom TEVIMBRA was withheld for pneumonitis, 20 (62.5%) reinitiated TEVIMBRA after symptom improvement; of these, 2 (10%) patients had recurrence of pneumonitis.

Immune-Mediated Colitis

TEVIMBRA can cause immune-mediated colitis, which can be fatal. Cytomegalovirus (CMV) infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis treated with PD-1/PD-L1 blocking antibodies. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies.

Immune-mediated colitis occurred in 0.8% (16/1972) of patients receiving TEVIMBRA, including Grade 3 (0.3%) and Grade 2 (0.4%) adverse reactions. Colitis led to permanent discontinuation of TEVIMBRA in 4 (0.2%) patients and withholding of TEVIMBRA in 5 (0.3%) patients. Twelve (75%) of the 16 patients received systemic corticosteroids. Eight (50%) of the 16 patients received high-dose systemic corticosteroids. Two (12.5%) of the 16 patients received immunosuppressive treatment. Immune-mediated colitis resolved in 93.8% of the 16 patients. All 5 patients in whom TEVIMBRA was withheld for colitis reinitiated TEVIMBRA after symptom improvement; of these, none of the patients had recurrence of colitis.

Immune-Mediated Hepatitis

TEVIMBRA can cause immune-mediated hepatitis, which can be fatal.

Immune-mediated hepatitis occurred in 1.2% (24/1972) of patients receiving TEVIMBRA, including fatal (0.1%), Grade 4 (0.2%), Grade 3 (0.5%) and Grade 2 (0.4%) adverse reactions. Immune-mediated hepatitis led to permanent discontinuation in 3 (0.2%) patients and withholding of TEVIMBRA in 13 (0.7%) patients. Eighteen (75%) of the 24 patients received systemic corticosteroids. Thirteen (54.2%) of the 24 patients received high-dose systemic corticosteroids. Two patients (8.3%) of the 24 patients received immunosuppressive treatment. Immune-mediated hepatitis resolved in 70.8% of the 24 patients. Of the 13 patients in whom TEVIMBRA was withheld for hepatitis, 7 (53.8%) reinitiated TEVIMBRA after symptom improvement; of these, none of the patients had recurrence of hepatitis.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

TEVIMBRA can cause immune-mediated adrenal insufficiency. For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold TEVIMBRA depending on severity.

Immune-mediated adrenal insufficiency occurred in 0.4% (8/1972) of patients receiving TEVIMBRA, including Grade 4 (0.1%), Grade 3 (0.1%) and Grade 2 (0.3%) adverse reactions. Adrenal insufficiency did not lead to permanent discontinuation of TEVIMBRA. TEVIMBRA was withheld in 7 (0.4%) patients. All 8 patients received systemic corticosteroids. Three (37.5%) of the 8 patients received high-dose systemic corticosteroids. Adrenal insufficiency resolved in 25% of the 8 patients. Of the 7 patients in whom TEVIMBRA was withheld for adrenal insufficiency, 5 (71.4%) reinitiated TEVIMBRA after symptom improvement; of these, none of the patients had recurrence of adrenal insufficiency.

Hypophysitis

TEVIMBRA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as clinically indicated. Withhold or permanently discontinue TEVIMBRA depending on severity.

Hypophysitis/hypopituitarism occurred in 0.2% (4/1972) of patients receiving TEVIMBRA, including a Grade 2 (0.2%) adverse reaction. No TEVIMBRA treatment discontinuation was required, while treatment was withheld in 1 (0.1%) patient. Three (75%) of the 4 patients received systemic corticosteroids. One (25%) of the 4 patients received high-dose systemic corticosteroids. Hypophysitis/hypopituitarism did not resolve in the 4 patients. For the 1 patient where TEVIMBRA was withheld for hypophysitis/hypopituitarism, there was no recurrence of hypophysitis/hypopituitarism.

Thyroid Disorders

TEVIMBRA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue TEVIMBRA depending on severity.

Thyroiditis: Immune-mediated thyroiditis occurred in 1.2% (24/1972) of patients receiving TEVIMBRA, including Grade 2 (0.5%) adverse reactions. Thyroiditis did not lead to permanent discontinuation of TEVIMBRA. TEVIMBRA was withheld in 3 (0.2%) patients. Two (8.3%) of the 24 patients received systemic corticosteroids. Thyroiditis resolved in 41.7% of the 24 patients. All three patients in whom TEVIMBRA was withheld for thyroiditis reinitiated TEVIMBRA after symptom improvement; of these, none of the patients had recurrence of thyroiditis.

Hyperthyroidism: Immune-mediated hyperthyroidism occurred in 4.8% (95/1972) of patients receiving TEVIMBRA, including Grade 3 (0.1%) and Grade 2 (0.9%) adverse reactions. Hyperthyroidism led to the permanent discontinuation of TEVIMBRA in 1 (0.1%) patient and withholding of TEVIMBRA in 4 (0.2%) patients. One (1.1%) of the 95 patients received systemic corticosteroids. Hyperthyroidism resolved in 75.8% of the 95 patients. Of the 4 patients in whom TEVIMBRA was withheld for hyperthyroidism, 3 (75%) reinitiated TEVIMBRA after symptom improvement; of these, none of the patients had recurrence of hyperthyroidism.

Hypothyroidism: Immune-mediated hypothyroidism occurred in 12.7% (250/1972) of patients receiving TEVIMBRA, including Grade 4 (0.1%) and Grade 2 (6.8%) adverse reactions. TEVIMBRA was not permanently discontinued in any patient, while treatment was withheld in 7 (0.4%) patients. Two (0.8%) of the 250 patients received systemic corticosteroids and 158 patients (63.2%) received hormone replacement therapy. Hypothyroidism resolved in 31.6% of the 250 patients. The majority (51.6%) of patients with hypothyroidism required long-term thyroid hormone replacement. Of the 7 patients in whom TEVIMBRA was withheld for hypothyroidism, 6 (85.7%) reinitiated TEVIMBRA after symptom improvement; of these, none of the patients had recurrence of hypothyroidism.

Type 1 Diabetes Mellitus, which can present with Diabetic Ketoacidosis

Diabetes mellitus has been reported with PD-1/PD-L1 blocking antibodies. Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Initiate treatment with insulin as clinically indicated. Withhold or permanently discontinue TEVIMBRA depending on severity.

Diabetes mellitus occurred in 0.9% (18/1972) of patients receiving TEVIMBRA, including Grade 4 (0.1%), Grade 3 (0.4%) and Grade 2 (0.4%) adverse reactions. TEVIMBRA was permanently discontinued in 3 (0.2%) patients and TEVIMBRA treatment was withheld in 3 (0.2%) patients. Twelve (66.7%) patients received insulin therapy for diabetes mellitus. Diabetes mellitus resolved in 27.8% of the 18 patients. Of the 3 patients in whom TEVIMBRA was withheld for diabetes mellitus, none of the patients reinitiated TEVIMBRA after symptom improvement.

Immune-Mediated Nephritis with Renal Dysfunction

TEVIMBRA can cause immune-mediated nephritis, which can be fatal.

Immune-mediated nephritis with renal dysfunction occurred in 0.3% (5/1972) of patients receiving TEVIMBRA, including Grade 3 (0.1%) and Grade 2 (0.2%) adverse reactions. TEVIMBRA was permanently discontinued in 1 (0.1%) patient and treatment was withheld in 3 (0.2%) patients. Three (60%) of the 5 patients received systemic corticosteroids. All 3 (60%) of the 5 patients received high-dose systemic corticosteroids. Nephritis with renal dysfunction resolved in 40.0% of the 5 patients. Of the 3 patients in whom TEVIMBRA was withheld for nephritis, 2 (66.7%) reinitiated TEVIMBRA after symptom improvement and one (50%) patient had recurrence of nephritis.

Immune-Mediated Dermatologic Adverse Reactions

TEVIMBRA can cause immune-mediated rash or dermatitis. Cases of severe cutaneous adverse reactions (SCARs), including exfoliative dermatitis, Stevens-Johnson Syndrome (SJS), and toxic epidermal necrolysis (TEN), have been reported, some with fatal outcome. Topical emollients and/or topical corticosteroids may be adequate to treat mild to moderate non-exfoliative rashes. Withhold or permanently discontinue TEVIMBRA depending on severity.

Immune-mediated dermatologic adverse reactions occurred in 15.3% (301/1972) of patients receiving TEVIMBRA, including Grade 4 (0.1%), Grade 3 (0.9%) and Grade 2 (3.5%) adverse reactions. Dermatologic adverse reactions led to permanent discontinuation of TEVIMBRA in 2 (0.1%) patients and withholding of TEVIMBRA in 18 (0.9%) patients. Thirty (10.0%) of the 301 patients received systemic corticosteroids. Thirteen (4.3%) of the 301 patients received high-dose systemic corticosteroids. Immune-mediated skin reactions resolved in 190 (63.1%) of the 301 patients. Of the 18 patients in whom TEVIMBRA was withheld for dermatologic adverse reactions, 15 (83.3%) reinitiated TEVIMBRA after symptom improvement; of these, 1 (6.7%) patient had recurrence of immune-mediated dermatologic adverse reactions.

Other Immune-Mediated Adverse Reactions

The following clinically significant immune-mediated adverse reactions occurred at an incidence of less than 1% each in 1972 patients who received TEVIMBRA: myositis, myocarditis, arthritis, polymyalgia rheumatica, and pericarditis.

The following additional clinically significant immune-mediated adverse reactions have been reported with other PD-1/PD-L1 blocking antibodies, including severe or fatal cases.

Cardiac/Vascular: Vasculitis.

Nervous System: Meningitis, encephalitis, myelitis and demyelination, myasthenic syndrome/myasthenia gravis (including exacerbation), Guillain-Barré syndrome, nerve paresis, autoimmune neuropathy.

Ocular: Uveitis, iritis, and other ocular inflammatory toxicities can occur. Some cases can be associated with retinal detachment. Various grades of visual impairment, including blindness, can occur. If uveitis occurs in combination with other immune-mediated adverse reactions, consider a Vogt-Koyanagi-Harada-like syndrome, as this may require treatment with systemic steroids to reduce the risk of permanent vision loss.

Gastrointestinal: Pancreatitis including increases in serum amylase and lipase levels, gastritis, duodenitis.

Musculoskeletal and Connective Tissue: Polymyositis, rhabdomyolysis and associated sequelae including renal failure.

Endocrine: Hypoparathyroidism.

Other (Hematologic/Immune): Hemolytic anemia, aplastic anemia, hemophagocytic lymphohistiocytosis, systemic inflammatory response syndrome, histiocytic necrotizing lymphadenitis (Kikuchi lymphadenitis), sarcoidosis, immune thrombocytopenic purpura, solid organ transplant rejection, other transplant (including corneal graft) rejection.

Infusion-Related Reactions

TEVIMBRA can cause severe or life-threatening infusion-related reactions. Infusion-related reactions occurred in 5% (99/1972) patients receiving TEVIMBRA, including Grade 3 or higher (0.2%) reactions. Monitor patients for signs and symptoms of infusion-related reactions.

Slow the rate of infusion for mild (Grade 1) and interrupt the infusion for moderate (Grade 2) infusion-related reactions. For severe (Grade 3) or life-threatening (Grade 4) infusion-related reactions, stop infusion and permanently discontinue TEVIMBRA.

Complications of Allogeneic HSCT

Fatal and other serious complications can occur in patients who receive allogeneic hematopoietic stem cell transplantation (HSCT) before or after being treated with a PD-1/PD-L1 blocking antibody. Transplant-related complications include hyperacute graft-versus-host disease (GVHD), acute GVHD, chronic GVHD, hepatic veno-occlusive disease after reduced intensity conditioning, and steroid-requiring febrile syndrome (without an identified infectious cause). These complications may occur despite intervening therapy between PD-1/PD-L1 blockade and allogeneic HSCT.

Follow patients closely for evidence of transplant-related complications and intervene promptly. Consider the benefit versus risks of treatment with a PD-1/PD-L1 blocking antibody prior to or after an allogeneic HSCT.

Embryo-Fetal Toxicity

Based on its mechanism of action, TEVIMBRA can cause fetal harm when administered to a pregnant woman. Animal studies have demonstrated that inhibition of the PD-1/PD-L1 pathway can lead to increased risk of immune-mediated rejection of the developing fetus resulting in fetal death. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with TEVIMBRA and for 4 months after the last dose.

ADVERSE REACTIONS

First-line Treatment of Unresectable Advanced or Metastatic Esophageal Carcinoma (ESCC)

Permanent discontinuation of TEVIMBRA due to adverse reactions occurred in 13% of patients. The adverse reaction which resulted in discontinuation in ≥2% of patients was pneumonitis (2.2%).

Dosage interruptions of TEVIMBRA due to adverse reactions occurred in 52% of patients. Adverse reactions which required dosage interruption in ≥2% of patients were neutrophil count decreased (7%), fatigue (6%), pneumonia (6%), anemia (4.3%), neutropenia (4.3%), white blood cell count decreased (4.3%), rash (3.7%), dysphagia (2.8%), platelet count decreased (2.8%), pyrexia (2.8%), and diarrhea (2.2%).

The most common (≥20%) adverse reactions, including laboratory abnormalities were decreased neutrophil count, decreased sodium, increased glucose, anemia, fatigue, decreased appetite, increased AST, decreased potassium, increased serum creatinine, decreased calcium, increased ALT, diarrhea, stomatitis, and vomiting.

Previously Treated Unresectable Advanced or Metastatic ESCC

Permanent discontinuation of TEVIMBRA due to an adverse reaction occurred in 19% of patients. Adverse reactions which resulted in permanent discontinuation in ≥1% of patients were hemorrhage, pneumonitis (including pneumonitis and immune-mediated pneumonitis), and pneumonia.

Dosage interruptions of TEVIMBRA due to an adverse reaction occurred in 23% of patients. Adverse reactions which required dosage interruptions in ≥2% of patients were pneumonia, pneumonitis, and fatigue.

The most common (≥20%) adverse reactions, including laboratory abnormalities, were increased glucose, decreased hemoglobin, decreased lymphocytes, decreased sodium, decreased albumin, increased alkaline phosphatase, anemia, fatigue, increased AST, musculoskeletal pain, decreased weight, increased ALT, and cough.

Treatment of Previously Untreated Unresectable or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma (G/GEJ)

Permanent discontinuation of TEVIMBRA due to an adverse drug reaction occurred in 16% of patients. Adverse drug reactions which resulted in permanent discontinuation in ≥1% of patients were death, fatigue, and pneumonitis.

Dosage interruption of TEVIMBRA in the TEVIMBRA plus chemotherapy arm due to an adverse drug reaction occurred in 49% of patients. Adverse drug reactions which required dosage modifications in ≥2% of patients were, platelet count decreased (12%), neutrophil count decreased (10%), neutropenia (6%), white blood cell count decreased (6%), increased AST (4.8%), increased ALT (3.8%), increased blood bilirubin (3%), COVID-19 (3%), thrombocytopenia (2.8%), leukopenia (2.6%), pneumonitis (2.2%), and pneumonia (2%).

The most common (≥20%) adverse reactions, including laboratory abnormalities, for TEVIMBRA in combination with chemotherapy were nausea, fatigue, decreased appetite, anemia, peripheral sensory neuropathy, vomiting, decreased platelet count, decreased neutrophil count, increased aspartate aminotransferase, diarrhea, abdominal pain, increased alanine aminotransferase, decreased white blood cell count, decreased weight, and pyrexia.

INDICATIONS

TEVIMBRA is a programmed death receptor-1 (PD-1)-blocking antibody indicated for:

Esophageal Cancer

in combination with platinum-containing chemotherapy for the first-line treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1 (≥1).
as a single-agent, for the treatment of adults with unresectable or metastatic ESCC after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.
Gastric Cancer

in combination with platinum and fluoropyrimidine-based chemotherapy for the first-line treatment of adults with unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma whose tumors express PD-L1 (≥1).
Please see full U.S. Prescribing Information including the U.S. Medication Guide.

Verastem Oncology Announces Positive Updated Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in Frontline Metastatic Pancreatic Ductal Adenocarcinoma

On May 22, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported positive updated safety and efficacy results from the RAMP 205 Phase 1/2 trial evaluating avutometinib plus defactinib in combination with gemcitabine and Nab-paclitaxel in the front-line for patients with metastatic pancreatic ductal adenocarcinoma (PDAC) (Press release, Verastem, MAY 22, 2025, View Source [SID1234653323]). As of April 25, 2025, patients in the dose level 1 cohort, which was selected as the recommended Phase 2 dose (RP2D), achieved an overall response rate (ORR) of 83% (10/12).

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The Company will host a research and development (R&D) investor webcast on Monday, June 2 at 11:00 am CDT to review the full updated data from RAMP 205 and the updated data from the Phase 1/2 study in China of VS-7375 (also known as GFH375), an oral KRAS G12D (ON/OFF) inhibitor, by partner GenFleet Therapeutics that will be presented in a rapid oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL on June 2, 2025.

"The exciting results from the RAMP 205 trial reinforce our commitment to maximizing the synergistic potential of the avutometinib plus defactinib combination in other advanced solid tumors for market expansion opportunities beyond KRAS-mutated recurrent low-grade serous ovarian cancer, for which the combination recently became the first-ever FDA-approved treatment for this disease," said Dan Paterson, president and chief executive officer of Verastem Oncology. "We look forward to the mature data from the Phase 1 portion of the VS-7375 study in China being presented at ASCO (Free ASCO Whitepaper) and dosing the first patient in our Phase 1/2a trial in the U.S. and in solid tumor cohorts including advanced pancreatic, lung, and colorectal cancers."

Updated Data from RAMP 205 Phase 1/2 Clinical Trial in Frontline Metastatic PDAC

As of April 25, 2025, 60 patients (12 per cohort) had been treated in one of five dose regimens with the combination of avutometinib and defactinib with gemcitabine and Nab-paclitaxel in frontline metastatic PDAC. In the dose level 1 cohort, 12 patients received 2.4 mg of avutometinib twice a week (BIW), 200 mg of defactinib twice a day (BID) for 3 weeks out of every 4 and 800 mg/m2 of gemcitabine and 125 mg/m2 of Nab-paclitaxel on a schedule of day 1, day 8 and day 15. In dose level 1, 83% (10/12) of patients achieved partial responses (8 confirmed and 2 unconfirmed who remain on treatment). Given these strong and consistent results, the Company has selected dose level 1 as the RP2D, has met the pre-defined criteria to advance beyond the first stage of the expansion study, and is now enrolling up to 29 patients at this dose level.

In evaluating all the dose cohorts, dose level 1 demonstrated the highest response rate and across all five dose cohorts, 92% (48/52) of efficacy evaluable patients showed tumor reduction as best response. Adverse events across all dose cohorts remained generally consistent with the previously announced safety and tolerability profile, and no new safety signals have emerged. While anticipating results from the study expansion cohort, the Company is now developing plans for a registrational Phase 3 front-line metastatic PDAC trial to begin in 2026.

Webcast Information

Verastem will hold an investor webcast on Monday, June 2, at 11:00 am CDT, to review the RAMP 205 updated data and the VS-7375 program including updated data from the study in China. The event will feature members of Verastem’s management team and key opinion leaders. A live audio webcast of the call, along with accompanying slides, will be accessible here.

About AVMAPKI and FAKZYNJA Combination Therapy

AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.

The U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is also evaluating avutometinib in combination with defactinib and other agents as a potential treatment for patients with advanced pancreatic cancer (RAMP 205; NCT05669482) and advanced KRAS G12C mutant non-small cell lung cancer (RAMP 203; NCT05074810). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.

AVMAPKI FAKZYNJA CO-PACK U.S. Indication

Indication

AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

Warnings and Precautions

Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions

The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.

Drug Interactions

Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations

Lactation: Advise not to breastfeed.
Fertility: May impair fertility in males and females.
Click here for full Prescribing Information.

About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor

VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem announced in April 2025 that the U.S. Investigational New Drug (IND) application for VS-7375 was cleared and plans to initiate a Phase 1/2a clinical trial in mid-2025. GenFleet’s IND for VS-7375 (known as GFH375 in China) was approved in China in June 2024, and the first patient was dosed in a Phase 1/2 study in July 2024.