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Last update 19 Jul 2025

Pembrolizumab

Last update 19 Jul 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Monoclonal antibody

Synonyms

Lambrolizumab, Pembrolizumab (Genetical Recombination), Pembrolizumab (genetical recombination) (JAN)

+ [10]

Target

PD-1

Action

inhibitors

Mechanism

PD-1 inhibitors(Programmed cell death protein 1 inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesInfectious Diseases+ [13]

Active Indication

Unresectable Hepatocellular CarcinomaMalignant Pleural MesotheliomaUnresectable Urothelial Carcinoma+ [347]

Inactive Indication

Adenocarcinoma, metastaticAdvanced Colorectal AdenocarcinomaAdvanced Urothelial Carcinoma+ [84]

Originator Organization

Merck & Co., Inc.

Active Organization

Merck Sharp & Dohme BV

MSD R&D (China) Co. Ltd.

Merck Sharp & Dohme Corp.

+ [10]

Inactive Organization-

License Organization

Nektar Therapeutics

NeoImmuneTech, Inc.

Canada Pension Plan Investment Board

+ [74]

Drug Highest PhaseApproved

First Approval Date

United States (04 Sep 2014),

Melanoma

RegulationPriority Review (United States), Breakthrough Therapy (United States), Accelerated Approval (United States), Orphan Drug (United States), PRIME (European Union), Conditional marketing approval (China), Orphan Drug (Japan), Orphan Drug (Australia), Priority Review (Australia), Priority Review (China)

Structure/Sequence

Sequence Code 93660H

Source: *****

Sequence Code 93670L

Source: *****

2,420

Clinical Trials associated with Pembrolizumab

NCT07037004

/ Not yet recruitingPhase 2IIT

Impact of Microbiome Modulation With CBM588 in Combination With Pembrolizumab for Adjuvant Therapy of High-Risk, Resected Renal Cell Carcinoma (RCC)

This phase II trial compares the effect of adding a probiotic called CBM588 to pembrolizumab versus pembrolizumab alone in preventing return of disease (recurrence) after surgery for patients with renal cell cancer. Pembrolizumab is an immune checkpoint inhibitor. Immunotherapy with monoclonal antibodies such as pembrolizumab may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Pembrolizumab is approved for the treatment of renal cell cancer after surgery. Research has shown that changes to the composition of the healthy bacteria in the body (the microbiome), may improve a patient's response to treatment with immunotherapy. CBM588, a probiotic supplement containing a bacteria called Clostridium butyricum, has been shown to improve outcomes in patients treated with immunotherapy for other types of cancer. Adding CBM588 to treatment with pembrolizumab after surgery may cause changes in the microbiome that improve patient response to treatment and reduce disease recurrence, compared to pembrolizumab alone.

Start Date04 May 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT06543576

/ Not yet recruitingPhase 1/2IIT

A Prospective Cohort Study of Integrating Radiotherapy Into Chemotherapy With Pembrolizumab and Bevacizumab in Newly Diagnosed Stage IVB Cervical Cancer

This phase I/II trial tests the safety and effectiveness of receiving external beam radiation therapy (EBRT) and brachytherapy along with chemotherapy, consisting of cisplatin and paclitaxel, and immunotherapy, consisting of bevacizumab and pembrolizumab, for the treatment of patients with stage IVB cervical cancer. EBRT is type of radiation therapy that uses a machine to aim high-energy rays at the cancer from outside of the body. Brachytherapy, also known as internal radiation therapy, uses radioactive material placed directly into or near a tumor to kill tumor cells. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of tumor cells. Paclitaxel is in a class of medications called antimicrotubule agents. It stops tumor cells from growing and dividing and may kill them. Bevacizumab is in a class of medications called antiangiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to tumor. This may slow the growth and spread of tumor. A monoclonal antibody, such as pembrolizumab, is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Giving EBRT and brachytherapy along with chemotherapy and immunotherapy may be a safe and effective way to treat patients with stage IVB cervical cancer.

Start Date31 Jan 2026

Sponsor / Collaborator

Jonsson Comprehensive Cancer Center

[+1]

NCT06669572

/ Not yet recruitingPhase 2IIT

A Phase II, Multi-center, Single Arm Trial of Lenvatinib Plus Pembrolizumab in Patients With Unresectable Locally Advanced and/or Metastatic Anorectal Squamous Cell Carcinoma (ASCC) After Progression on First Line Chemotherapy.

The purpose of this study is to gather information on the safety and effectiveness of lenvatinib combined with pembrolizumab in anal/rectal cancer that has spread to other parts of the body and will not respond to standard care.

Start Date13 Jan 2026

Sponsor / Collaborator

The University of Chicago

100 Clinical Results associated with Pembrolizumab

100 Translational Medicine associated with Pembrolizumab

100 Patents (Medical) associated with Pembrolizumab

10,887

Literatures (Medical) associated with Pembrolizumab

01 Jan 2026·TALANTA

Biosimilarity and advanced structural characterization of monoclonal antibodies charge variants using capillary zone electrophoresis and mass spectrometry

Article

Author: Joomun, Rania ; Gahoual, Rabah ; Alez-Martin, Lola ; François, Yannis-Nicolas ; Mignet, Nathalie ; Houzé, Pascal

Monoclonal antibodies (mAbs) structural complexity arises from their macromolecular nature and their propensity to undergo post-translational modifications (PTM), potentially leading to the formation of charge variants. Capillary zone electrophoresis (CZE) showed to be particularly relevant for their analysis, however the selectivity provided by CZE separation is not completely elucidated. In this work, CZE-UV analysis was used to characterize charge variants for biosimilars products corresponding to infliximab. Results demonstrated the possibility to identify faint variations between the different products showing its applicability to contribute to mAbs biosimilarity assessment. Enzymatic treatment allowed to attribute the origin of infliximab charge variants. CZE-UV analysis of pembrolizumab showed that none of the five charge variants separated were originating from C-terminal lysine residues and/or N-glycans. To enable further identification, an analytical strategy was developed to achieve CZE-UV fraction collection and enrichment of mAb charge variants followed by systematic offline characterization in CE coupled to tandem mass spectrometry (MS/MS). CE-MS/MS experiments allowed the identification of different types of PTMs such as N-terminal pyroglutamic acid formation and asparagine deamidation for charge variant fractions correlated with decreased mobility. In addition, for the first time succinimide intermediate formation could be successfully characterized using CE-MS/MS data, which could be correlated to increased mobility. Thus, the CZE-UV separation resulted from the synergistic effect of several simultaneous PTMs affecting the apparent mobilities of the charge variants. As a consequence, experiments illustrated the relevance and potential of intact mAbs analysis using CZE-UV to provide an overview of the structural diversity of therapeutic mAbs.

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

The cost-effectiveness of treatment for high-risk, early-stage, triple-negative breast cancer in Egypt: an analysis of neoadjuvant pembrolizumab plus chemotherapy followed by adjuvant single-agent pembrolizumab

Article

Author: Haiderali, Amin ; Huang, Min ; Abdelaziz, Ahmed H. ; Pöllinger, Bernadette ; Kassem, Loay ; Akyol Ersoy, Burcu ; Elsisi, Gihan Hamdy

OBJECTIVE:

The cost-effectiveness of neoadjuvant pembrolizumab + chemotherapy followed by adjuvant pembrolizumab compared to neoadjuvant chemotherapy plus placebo followed by adjuvant placebo was assessed in high-risk, early-stage, triple-negative breast cancer patients from an Egyptian societal perspective over a lifetime horizon.

METHODS:

A 4-state Markov cohort model was developed to compare the cost-effectiveness of pembrolizumab + chemotherapy/pembrolizumab vs chemotherapy alone for the treatment of high-risk, early-stage, triple-negative breast cancer. The model simulated the clinical course of high-risk, early-stage, triple-negative breast cancer across four health states: event-free survival, locoregional recurrence, distant metastasis, and death. Clinical inputs for the simulation were derived from modeling of efficacy and safety data collected in the KEYNOTE-522 trial. Direct medical costs and indirect costs were reported in 2022 Egyptian pounds (EGP) and converted to US dollars ($). Probabilistic and deterministic sensitivity analyses were conducted to assess the robustness of model results.

RESULTS:

Compared with chemotherapy alone, pembrolizumab + chemotherapy/pembrolizumab led to expected gains of 2.92 life years and 2.25 quality-adjusted life years, respectively, while increasing overall treatment costs by EGP 491,695 ($102,436). Incremental costs per year gained were EGP 218,285 ($45,476) per quality-adjusted life year and EGP 168,223 ($35,046) per life year, both of which were lower than the 2022 Egyptian cost-effectiveness threshold of EGP 398,439 ($83,008). The findings of sensitivity analyses indicated that the model was robust across a range of inputs and assumptions.

CONCLUSIONS:

In Egypt, pembrolizumab + chemotherapy/pembrolizumab is a cost-effective treatment for high-risk, early-stage, triple-negative breast cancer when considering health-related quality-of-life and years of life gained.

31 Dec 2025·OncoImmunology

Clinical impact of cancer cachexia on the outcome of patients with non-small cell lung cancer with PD-L1 tumor proportion scores of ≥50% receiving pembrolizumab monotherapy versus immune checkpoint inhibitor with chemotherapy

Article

Author: Katayama, Yuki ; Hasegawa, Isao ; Kijima, Takashi ; Iwasaku, Masahiro ; Kawachi, Hayato ; Date, Koji ; Yamada, Tadaaki ; Shimose, Takayuki ; Nakao, Akira ; Harada, Taishi ; Okada, Asuka ; Tokuda, Shinsaku ; Nishioka, Naoya ; Morimoto, Kenji ; Tamiya, Motohiro ; Tanimura, Keiko ; Shiotsu, Shinsuke ; Goto, Yasuhiro ; Takayama, Koichi ; Negi, Yoshiki ; Tamiya, Nobuyo ; Chihara, Yusuke ; Takeda, Takayuki

This retrospective, multicenter cohort study aimed to determine whether cancer cachexia serves as a biomarker for determining the most effective treatment for patients having non-small-cell lung cancer (NSCLC) with high programmed death ligand 1 (PD-L1) expression treated with immune checkpoint inhibitors (ICIs) alone or combined with chemotherapy (ICI/chemotherapy). We included 411 patients with advanced NSCLC with a PD-L1 tumor proportion score of ≥50%. The patients were treated with pembrolizumab monotherapy or ICI/chemotherapy. Cancer cachexia was defined as a weight loss of >5% of the total body weight or a body mass index of <20 kg/m² coupled with an additional weight loss of >2% within 6 months before starting treatment. Eighty-five (21%) patients met the cancer cachexia criteria. Overall survival (OS) was significantly shorter in patients with cachexia than in those without cachexia in both the pembrolizumab monotherapy group (17.2 vs. 35.8 months, p < 0.001) and the ICI/chemotherapy group (27.0 months vs. not reached, p = 0.044). However, after stratifying by cancer cachexia status, no significant difference in OS was observed between the pembrolizumab monotherapy and chemoimmunotherapy groups, regardless of cachexia. In conclusion, ICI/chemotherapy offers limited benefits for NSCLC patients with high PD-L1 expression and concurrent cancer cachexia. Considering the frailty associated with cachexia, ICI monotherapy may be preferred to ICI/chemotherapy for these patients. New interventions that can better address the negative prognostic impact of cachexia in patients treated using ICIs with or without chemotherapy remain warranted.

5,593

News (Medical) associated with Pembrolizumab

16 Jul 2025

·www.prnewswire.com

Cancer Pipeline Milestones Coming Fast as Regulators Revisit Research Priorities

Equity Insider News Commentary Issued on behalf of Oncolytics Biotech Inc. VANCOUVER. BC, July 16, 2025 /PRNewswire/ -- As lawmakers weigh potential cuts to scientific research funding, the outlook for developing cancer cures faces growing uncertainty. The situation is further complicated by renewed regulatory scrutiny of mRNA vaccines at both federal and state levels, casting a shadow over ongoing cancer research efforts. Meanwhile, the incidence of cancers—especially gastrointestinal and colorectal cancers among younger populations—is on the rise, heightening the need for new therapeutic approaches. In response, a new generation of biotech companies is stepping forward with promising clinical milestones on the horizon, including Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), Zai Lab Limited (NASDAQ: ZLAB), GeoVax Labs, Inc. (NASDAQ: GOVX), OS Therapies (NYSE-American: OSTX), and SELLAS Life Sciences Group, Inc. (NASDAQ: SLS). Industry forecasts suggest the global oncology drug market could climb past US$900 billion by 2034. Within that, next-generation cancer treatments—powered by advances in personalized and precision medicine—are expected to reach US$175.2 billion, according to Precedence Research, growing at a compound annual rate of 7.35%. Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC) just rolled out an expanded translational data review package that tightens the scientific case for pelareorep, its intravenously delivered oncolytic virus. Fresh analyses from the GOBLET gastrointestinal‑cancer study and the AWARE‑1 breast‑cancer study shows pelareorep actively replicating inside tumors, switching on interferon signaling in the immune system, and drawing tumor‑infiltrating lymphocytes into the cancer microenvironment. "This robust data set, amassed from several studies in cancers that have historically resisted immunotherapeutic approaches, provides definitive validation of pelareorep's immune-mediated mechanism of action," said Dr. Thomas Heineman, Chief Medical Officer of Oncolytics. "We observed tumor biopsy-confirmed virus replication, immune cell activation, and the recruitment of cytotoxic T cells into the TME – all consistent with the durable responses observed in patients with metastatic PDAC and HR+/HER2- breast cancer who were treated with pelareorep." Investigators also recorded a clear increase in PD‑L1 expression, a checkpoint marker that helps immune cells recognize tumors, and tracked newly expanded T‑cell clones in the blood that matched those inside shrinking lesions. Together, these findings suggest pelareorep can convert "cold" tumors into "hot" ones that respond better to modern immunotherapies. "The collection of data here show that pelareorep works how a cancer immunotherapy should work," said Jared Kelly, CEO of Oncolytics. "Pelareorep is a versatile product candidate with strong platform potential to enhance immunological responses in multiple indications, including hard-to-treat cancers. Such compelling findings should be exciting to strategic partners focused on finding a platform immunotherapy in large indications with high unmet medical needs." Two GOBLET cohorts—metastatic pancreatic ductal adenocarcinoma (mPDAC) and anal cancer—remain open. Management plans to outline the next clinical milestones before the end of the third quarter. Clinical outcomes already hint at real‑world benefit. In more than 100 first‑line mPDAC patients, pelareorep‑based regimens achieved a two‑year overall‑survival rate of 21.9%, compared with the historical benchmark of 9.2%. A separate single‑arm study that paired pelareorep with chemotherapy and a checkpoint blocker produced a 62% objective response rate. No immune checkpoint therapy is approved in this cancer today, which makes the signal especially noteworthy. Progress extends to hormone‑receptor‑positive, HER2‑negative metastatic breast cancer (HR+/HER2‑ mBC). Across two randomized trials, pelareorep added more than ten months of median overall survival. In BRACELET‑1, the drug nearly doubled median progression‑free survival to 12.1 months versus 6.4 months in the control arm, suggesting durable disease control. Next week, Oncolytics will host a KOL webinar on July 22 featuring leading GI and immuno-oncology experts to discuss pelareorep's data and positioning in pancreatic and gastrointestinal cancers. Participating physicians include the GOBLET trial's primary investigator as well as global leaders in immunotherapy and clinical trial design, underscoring the growing interest in pelareorep's mechanism and outcomes. To steer these data toward value‑creating deals and late‑stage trials, Oncolytics Biotech strengthened its leadership earlier this year. The board tapped industry veteran Jared Kelly for the CEO seat and named Andrew Aromando Chief Business Officer. Both executives helped guide Ambrx Biopharma into a $2‑billion sale to Johnson & Johnson, giving them a playbook for capital‑efficient development and strategic partnering. "Pelareorep's clinical data across multiple tumors is striking and represents the potential for a true backbone immunotherapy to address many in-need indications," said Kelly. "With a renewed focus and sharpened clinical development plan, we believe we will move pelareorep forward effectively and efficiently to a place where potential partners will see the value of a de-risked immunotherapy." As CBO, Aromando is now leading global business development and helping shape the company's corporate, clinical, and regulatory strategies. The leadership tandem is expected to prioritize partnering and expansion opportunities while preserving capital efficiency—a strategy well-suited for pelareorep's growing clinical profile. "I'm thrilled to join Oncolytics at such a pivotal moment in its evolution," said Aromando. "With promising data in difficult-to-treat cancers and a compelling body of clinical evidence in over 1,100 patients, I believe the Company is uniquely positioned to deliver meaningful value to patients and other stakeholders in the near term." Pelareorep currently holds FDA Fast Track designation in both mPDAC (pancreatic cancer) and HR+/HER2- mBC (breast cancer), with Orphan Drug status for pancreatic cancer in the U.S. and Europe. CONTINUED… Read this and more news for Oncolytics Biotech at: In other recent industry developments and happenings in the market include: Zai Lab Limited (NASDAQ: ZLAB) recently reported that its Phase 3 FORTITUDE‑101 study found bemarituzumab plus mFOLFOX6 significantly improved overall survival over chemotherapy alone in first‑line fibroblast growth factor receptor 2b (FGFR2b)‑positive gastric and gastroesophageal junction cancers. The antibody becomes the first FGFR2b inhibitor to post a statistically and clinically meaningful survival benefit, and the company plans a rapid regulatory filing in China under its Breakthrough Therapy designation. "Bemarituzumab is the first FGFR2b inhibitor to demonstrate a statistically and clinically significant overall survival benefit in a randomized Phase 3 trial for the first-line treatment of FGFR2b-positive gastric cancer," said Dr. Rafael Amado, M.D., President, Head of Global Research and Development at Zai Lab. "The success of the global Phase 3 FORTITUDE-101 study highlights the potential of bemarituzumab to redefine the standard of care for a patient population that has faced poor outcomes with existing therapies. We are proud to have contributed meaningfully to this pivotal trial, including a substantial number of patients enrolled in China. Based on these results, and the regulatory Breakthrough Designation, we plan to move rapidly toward regulatory submission in China to bring this transformative therapy to patients as quickly as possible." GeoVax Labs, Inc. (NASDAQ: GOVX) recently stated that the FDA's curative‑intent approval of Keytruda for head and neck cancer strengthens the rationale for its planned Phase 2 trial combining Gedeptin and pembrolizumab. "We believe Gedeptin's tumor-targeted cytotoxicity can enhance immunotherapy efficacy, particularly in the perioperative window where anti-tumor immunity can be primed," added Dr. Kelly McKee, GeoVax's Chief Medical Officer. "We are excited to embark on the next phase of Gedeptin development as we attempt to build on the important advances being made in this disease." The study, expected to launch in 2026, will give Gedeptin intratumorally before surgery to trigger local cytotoxicity, then add checkpoint inhibition to boost systemic immunity. Endpoints include pathologic response, recurrence rates, and immune biomarkers, aligning with guidance in a recent New England Journal of Medicine editorial. OS Therapies (NYSE-American: OSTX) has locked in an End‑of‑Phase 2 FDA meeting for 27 August 2025 to discuss a rolling Biologics License Application for OST‑HER2 in recurrent, lung‑metastatic osteosarcoma, while parallel regulatory consultations are set with the EMA and the UK MHRA. "We are making significant progress towards our primary objective of obtaining regulatory approval for OST-HER2 in recurrent, pulmonary metastatic osteosarcoma prior to the sunsetting of the rare pediatric disease priority review voucher ("PRV") program," said Paul Romness, MPH, Chairman & CEO of OS Therapies. "We strongly believe in the promise of the listeria immunotherapy platform to help prevent and treat cancer, and intend to judiciously deploy our capital to focus on the OST-HER2 approval while advancing our other clinical programs without deploying significant capital or running other clinical studies while we wait for the OST-HER2 approval and related PRV sale." The company is also advancing a Phase 1 prostate‑cancer immunotherapy, OST‑504, with full data expected later in 2025. OST‑HER2 already carries Rare Pediatric Disease, Fast Track, and Orphan Drug designations, positioning the program for a potential Priority Review Voucher and accelerated approvals in multiple markets. SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) met every primary endpoint in its Phase 2 trial of SLS009 in relapsed/refractory acute myeloid leukemia-myelodysplasia-related changes (AML MR), delivering a 44% overall response rate at the optimal 30 mg twice‑weekly dose and a median overall survival of 8.9 months—far above the 2.4 month historical benchmark. The CDK9 inhibitor also showed a 50% response rate in high‑risk ASXL1‑mutated and M4/M5 subgroups, with no dose‑limiting toxicities reported. "We are excited to report that our Phase 2 trial met all key endpoints, with clinical responses and survival outcomes that exceed targeted expectations and historical benchmarks," said Angelos Stergiou, MD, ScD h.c., President and CEO of SELLAS. "AML remains an area of urgent unmet medical need, particularly for patients with relapsed or refractory disease, where standard treatments are often ineffective and poorly tolerated. What sets SLS009 apart is its consistent efficacy across a broad range of molecular subtypes." Following FDA guidance, the company will launch an 80‑patient randomized study in newly diagnosed first‑line AML by Q1 2026 to support a potential New Drug Application. Source: CONTACT: Equity Insider [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. Equity Insider is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Oncolytics Biotech Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Oncolytics Biotech Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Oncolytics Biotech Inc. which were purchased in the open market, and reserve the right to buy and sell, and will buy and sell shares of Oncolytics Biotech Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved by Oncolytics Biotech Inc.; this is a paid advertisement, we currently own shares of Oncolytics Biotech Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between the any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment. Logo - SOURCE Equity Insider WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultImmunotherapyPhase 2Phase 3Fast Track

16 Jul 2025

·pipelinereview.com

Genetic Leap receives FDA IND clearance for first-in-class, oral natural IL-2 modulator

– GL-IL2-138 is a small molecule RNA agent that modulates natural IL-2, allowing for downregulating or upregulating the immune system to fight devastating diseases – Potential to unite Humira-grade inflammation control and Keytruda-class anti-cancer power in a single pill, without the drawbacks NEW YORK, NY, USA I July 16, 2025 I Genetic Leap, a clinical stage, AI-native biotech pioneering RNA genetic therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for GL-IL2-138, the company’s small-molecule modulator of natural interleukin-2 (IL-2). IL-2 is a pleiotropic cytokine that regulates the careful homeostatic balance between immune tolerance and activation. The tremendous potential of IL-2 to address multiple major diseases is universally recognized. Recombinant IL-2 protein (rIL-2) was the first FDA-approved immunotherapy proven effective against cancer at high dose, though toxicity has historically limited its use. And low dose rIL-2 is clinically effective in autoimmune and neurodegeneration diseases. But numerous attempts by pharma companies to remove toxicity from rIL-2 muteins have flopped, earning IL-2 its reputation as an impossible target. Genetic Leap addressed this herculean challenge by developing a small molecule drug that binds endogenous IL-2 mRNA to drive controlled, systemic production of natural IL-2 protein by immune cells, which has many advantages over rIL-2 including: “We created the Genetic Leap AI platform to deliver breakthroughs like GL-IL2-138,” said Dr. Bertrand Adanve, Founder and CEO of Genetic Leap. “Innovative medicine that defies known scientific limitations, like a sci-fi drug made real. GL-IL2-138 is like combining Humira (leading autoimmune biologic) and Keytruda (leading cancer biologic) into one oral pill without the downsides and applicable to even more diseases. GL-IL2-138 is poised to be a giant leap for human health, and Genetic Leap is the company making it happen.” The drug will now be tested in a randomized, double-blind, placebo-controlled phase 1 clinical trial to evaluate its tolerability, pharmacokinetics and pharmacodynamics in humans. About Genetic Leap Genetic Leap is innovating at the cutting edge of AI and RNA genetic therapeutics to redefine drug development and more quickly address the health needs of millions of people. For more information, please visit www.genetic-leap.com or our LinkedIn . SOURCE: Genetic Leap

Phase 1INDImmunotherapy

16 Jul 2025

·www.globenewswire.com

Cue Biopharma Reports New Complete Response and Confirmed 50% Overall Response Rate in Ongoing Phase 1 Trial of CUE-101 and Pembrolizumab in Recurrent/Metastatic HPV+ Head and Neck Cancer

Additional complete response (CR) observed in patient with multiple tumorsConfirmed overall response rate (ORR) of 50% in patients with combined positive score (CPS) ≥1, including 50% ORR in patients with low CPS (1-19)12-month overall survival of 88% and median overall survival (mOS) of 32 months BOSTON, July 16, 2025 (GLOBE NEWSWIRE) -- Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of autoimmune disease and cancer, today provided a clinical update on its most advanced asset, CUE-101, representative of the CUE-100 series. “We are excited to report an additional CR in a patient that had recurrent metastatic HPV+ head and neck squamous cell carcinoma (HNSCC) treated with CUE-101 in combination with pembrolizumab (KEYTRUDA®),” said Matteo Levisetti, chief medical officer at Cue Biopharma. “This patient had durable stable disease for close to two years and more recently demonstrated significant tumor reductions and now a complete response. Notably, the patient had multiple sites of disease, including the lungs that cleared prior to the complete response observed in the target lesion. We believe the kinetics of tumor reduction and disease eradication in this patient is due to the repeated stimulation and expansion of tumor-specific T cells given the mechanism of action of CUE-101. It also serves as a clear example of the differences in timing of the clinical activity often observed with immunotherapy compared to traditional cytotoxic therapies and supports the prolonged mOS observed in this trial.” Key data highlights from the expansion portion of the trial evaluating CUE-101 at the recommended Phase 2 dose (RP2D) of 4mg/kg in combination with pembrolizumab in 1L patients (data cutoff of July 14, 2025) include: ORR of 50% (2 CR and 10 partial responses (PR) in patients with CPS ≥1, compared to an ORR of 19% observed with pembrolizumab alone in the historical third-party KEYNOTE-048 trial).Survival metrics have continued to mature favorably: 12-month overall survival (OS) of 88% compared to 57% with pembrolizumab alone in the historical KEYNOTE-048 trial, representing a reduction in the risk of death (HR 0.23) compared to historical data.mOS of 32 months compared to 12.3 months in the historical KEYNOTE-048 trial.ORR of 50% in patients, including 50% with low PD-L1 expression CPS (1-19). Dan Passeri, chief executive officer at Cue Biopharma, added, "The culmination of maturing data further support our conviction that CUE-101, representative of our approach with the CUE-100 series, demonstrates a potential breakthrough therapeutic approach for establishing a new standard of care. With this maturing data, we are further emboldened in our conviction that our Immuno-STAT® platform represents transformative potential for selectively modulating the patient’s immune system.” About CUE-101 and the Phase 1 TrialCUE-101 is Cue Biopharma’s most advanced clinical stage drug candidate from the CUE-100 series of interleukin 2 (IL-2)-based biologics. It is designed to activate and expand HPV16 tumor-specific T cells by presenting two signals or “cues” to T cells. Signal #1 incorporates the HPV E7 protein, harbored by HPV-induced cancer cells, to provide selectivity through interaction with the HPV-specific T cell receptor. Signal #2 consists of an engineered IL-2 variant to stimulate the activity of T cells. CUE-101 is currently being evaluated in a fully enrolled Phase 1 open-label, dose escalation and expansion study, for the treatment of HPV16+ driven recurrent/metastatic head and neck squamous cell carcinoma in second line (2L) and beyond patients as a monotherapy, and as a first line (1L) therapy in combination with pembrolizumab (KEYTRUDA®). About Cue BiopharmaCue Biopharma, a clinical-stage biopharmaceutical company, is developing a novel class of injectable biologics to selectively engage and modulate disease-specific T cells directly within the patient’s body. The company’s proprietary platform, Immuno-STAT® (Selective Targeting and Alteration of T cells), and biologics are designed to harness the curative potential of the body’s intrinsic immune system without the adverse effects of broad systemic immune modulation. Headquartered in Boston, Massachusetts, we are led by an experienced management team with deep expertise in immunology and immuno-oncology as well as the design and clinical development of protein biologics. For more information please visit www.cuebiopharma.com and follow us on X and LinkedIn. Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding: the company’s belief regarding the potential benefits and applications of its drug candidates and programs, including the CUE-100 series and that CUE-101 represents a potential breakthrough therapeutic approach for establishing a new standard of care for treating patients battling HNSCC and that the Immuno-STAT® platform represents transformative potential for selectively modulating the patient’s immune system; and the company’s business strategies, plans and prospects. Forward-looking statements, which are based on certain assumptions and describe the company’s future plans, strategies and expectations, can generally be identified by the use of forward-looking terms such as “believe,” “expect,” “may,” “will,” “should,” “would,” “could,” “seek,” “intend,” “plan,” “goal,” “project,” “estimate,” “anticipate,” “strategy,” “future,” “likely,” “promise” or other comparable terms, although not all forward-looking statements contain these identifying words. All statements other than statements of historical facts included in this press release regarding the company’s strategies, prospects, financial condition, operations, costs, plans and objectives are forward-looking statements. Important factors that could cause the company’s actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the company’s limited operating history, limited cash and a history of losses; the company’s ability to obtain adequate financing to fund its business operations in the near term and successfully remediate its current “going concern” determination that it does not have sufficient capital on hand to continue operations beyond the next twelve months; the company’s ability to achieve profitability; potential setbacks in the company’s research and development efforts including negative or inconclusive results from its preclinical studies or clinical trials or the company’s ability to replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; serious and unexpected drug-related side effects or other safety issues experienced by participants in clinical trials; its ability to secure required U.S. Food and Drug Administration (“FDA”) or other governmental approvals for its product candidates and the breadth of any approved indication; adverse effects caused by public health pandemics, including possible effects on the company’s operations and clinical trials; delays and changes in regulatory requirements, policy and guidelines including potential delays in submitting required regulatory applications to the FDA; the company’s reliance on licensors, collaborators, contract research organizations, suppliers and other business partners; the company’s ability to obtain adequate financing to fund its business operations in the future and ability to continue as a going concern; the company’s ability to maintain and enforce necessary patent and other intellectual property protection; competitive factors; general economic and market conditions and the other risks and uncertainties described in the Risk Factors and Management's Discussion and Analysis of Financial Condition and Results of Operations sections of the company’s most recently filed Annual Report on Form 10-K and any subsequently filed Quarterly Report(s) on Form 10-Q. Any forward-looking statement made by the company in this press release is based only on information currently available to the company and speaks only as of the date on which it is made. The company undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. Investor Contact Marie CampinellSenior Director, Corporate CommunicationsCue Biopharma, Inc.mcampinell@cuebio.com Media ContactJonathan PappasLifeSci Communicationsjpappas@lifescicomms.com

ImmunotherapyClinical ResultPhase 1Phase 2

100 Deals associated with Pembrolizumab

External Link

KEGG	Wiki	ATC	Drug Bank
D10574	Pembrolizumab	L01XC18	DB09037

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Unresectable Hepatocellular Carcinoma	China	Merck Sharp & Dohme LLC	10 Jun 2025
Malignant Pleural Mesothelioma	United States	Merck & Co., Inc.	17 Sep 2024
Malignant Pleural Mesothelioma	United States	Merck Sharp & Dohme LLC	17 Sep 2024
Unresectable Urothelial Carcinoma	European Union	Merck Sharp & Dohme BV	25 Jul 2024
Unresectable Urothelial Carcinoma	Iceland	Merck Sharp & Dohme BV	25 Jul 2024
Unresectable Urothelial Carcinoma	Liechtenstein	Merck Sharp & Dohme BV	25 Jul 2024
Unresectable Urothelial Carcinoma	Norway	Merck Sharp & Dohme BV	25 Jul 2024
Advanced Endometrial Carcinoma	United States	Merck Sharp & Dohme LLC	17 Jun 2024
Microsatellite instability-high Endometrial Carcinoma	United States	Merck Sharp & Dohme LLC	17 Jun 2024
Mismatch repair-deficient Endometrial Carcinoma	United States	Merck Sharp & Dohme LLC	17 Jun 2024
Advanced gastric carcinoma	Japan	MSD KK (Tokyo)	17 May 2024
recurrent gastric cancer	Japan	MSD KK (Tokyo)	17 May 2024
Unresectable Biliary Tract Carcinoma	Canada	Merck Sharp & Dohme Corp.	09 May 2024
Advanced biliary tract cancer	China	Merck Sharp & Dohme LLC	30 Jan 2024
Locally Advanced Cholangiocarcinoma	China	Merck Sharp & Dohme LLC	30 Jan 2024
stomach adenocarcinoma	Norway	Merck Sharp & Dohme BV	20 Dec 2023
HER2 negative Gastric Cancer	United States	Merck Sharp & Dohme LLC	16 Nov 2023
Resectable Lung Non-Small Cell Carcinoma	United States	Merck Sharp & Dohme Corp.	16 Oct 2023
HER2 Positive Stomach Adenocarcinoma	European Union	Merck Sharp & Dohme BV	06 Sep 2023
HER2 Positive Stomach Adenocarcinoma	Iceland	Merck Sharp & Dohme BV	06 Sep 2023

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Locally Advanced Head and Neck Squamous Cell Carcinoma	NDA/BLA	United States	Merck Sharp & Dohme LLC	25 Feb 2025
Small intestine carcinoma	NDA/BLA	European Union	Merck Sharp & Dohme Corp.	25 Mar 2022
Ovarian Epithelial Carcinoma	Phase 3	United States	Merck Sharp & Dohme Corp.	13 Dec 2021
Ovarian Epithelial Carcinoma	Phase 3	Japan	Merck Sharp & Dohme Corp.	13 Dec 2021
Ovarian Epithelial Carcinoma	Phase 3	Belgium	Merck Sharp & Dohme Corp.	13 Dec 2021
Ovarian Epithelial Carcinoma	Phase 3	Canada	Merck Sharp & Dohme Corp.	13 Dec 2021
Ovarian Epithelial Carcinoma	Phase 3	Colombia	Merck Sharp & Dohme Corp.	13 Dec 2021
Ovarian Epithelial Carcinoma	Phase 3	Denmark	Merck Sharp & Dohme Corp.	13 Dec 2021
Ovarian Epithelial Carcinoma	Phase 3	Germany	Merck Sharp & Dohme Corp.	13 Dec 2021
Ovarian Epithelial Carcinoma	Phase 3	Israel	Merck Sharp & Dohme Corp.	13 Dec 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02940496 (CTgov)	Phase 2	Non-small cell lung cancer stage IIIA \| Advanced Hepatocellular Carcinoma \| Hepatitis C ... View more	15	Pembrolizumab ("ARM A Pembrolizumab (Q3W)")	pehgcklzkm(bexabxbpir) = tdmnnvwaxq wjgnbstgks (reonayekgw, uaxovlitum - vaabxgvejt) View more	-	15 Jul 2025
				Elbasvir+Grazoprevir+Pembrolizumab ("ARM B Pembrolizumab (Q3W)+Grazoprevir (qd x 12 Weeks)+Elbasvir (QD x 12 Weeks")	hijtkombtf(camwvaqden) = zicnvuezhq dgivkeapll (zzpoezaaly, vnjmscliak - foifypyoke) View more
NCT04120454 (CTgov)	Phase 2	metastatic non-small cell lung cancer \| Recurrent Non-Small Cell Lung Cancer \| EGFR-mutated non-small Cell Lung Cancer	6	Pembrolizumab+Ramucirumab	wioltvhdgk = hpmpsvotpu dvtdgqvfmv (rfmmhhnfcj, gpdbdrnngq - bjiddzksxb) View more	-	11 Jul 2025
NCT04306367 (Pubmed \| NPJ Precis Oncol)	Phase 2	Advanced biliary tract cancer Second line IDH1/IDH2 mutations	13	Pembrolizumab and Olaparib Combination	hjwpssfquu(mmrmqujhan) = dbdyqmyhus yushtzccda (mouaospstx ) View more	Positive	08 Jul 2025
ESMO_GI2025	Not Applicable	Stomach Cancer	24	Pembrolizumab (Automated Segmentation using DL model)	rkrdmumdsj(rxghblhmtf) = zmeriekrhg gnucnvridp (gglkmvtrfv )	-	03 Jul 2025
				Pembrolizumab (Manual Segmentation by Radiology Resident)	rkrdmumdsj(rxghblhmtf) = ezkwbvpesk gnucnvridp (gglkmvtrfv )
ESMO_GI2025	Not Applicable	Microsatellite instability-high colorectal cancer First line dMMR \| MSI-H	-	Pembrolizumab	phprzwhrzu(uqnpwerjjp) = 17.9% eltomskjcz (uyttolcbml ) View more	Positive	03 Jul 2025
NCT03765918 (KEYNOTE-689, Pubmed \| N Engl J Med)	Phase 3	Head and Neck Neoplasms Neoadjuvant \| Adjuvant programmed death ligand 1 (PD-L1)	-	Pembrolizumab + Standard Care	hungaqmkos(okizedkxqe) = 10.0% of the participants in the pembrolizumab group ybufnnxwqn (pkjqgbljqh ) View more	Positive	03 Jul 2025
				Pembrolizumab (Standard Care Alone)
NCT05131919 (PUMA, ESMO_GI2025)	Phase 2	Microsatellite instability-high colorectal cancer Neoadjuvant dMMR	25	Pembrolizumab 200mg Q3W	dywtnmfqpe(lzsxumeofz) = jhvdcwpcbb siurkjghzt (thnverfivo ) View more	Positive	03 Jul 2025
ESMO_GI2025	Phase 3	Advanced biliary tract cancer	36	Pembrolizumab + Gemcitabine + Cisplatin	rjzfxwtdhf(oablnmrpuw) = qspekeplmg tzzuhjpdia (qtydhkhifa ) View more	Positive	03 Jul 2025
NCT02635360 (CTgov)	Phase 2	Locally Advanced Malignant Neoplasm \| Advanced Cervical Carcinoma	94	Brachytherapy+cisplatin+Pembrolizumab (Following Chemoradiation)	jvdphelsmt(rxfrcbopls) = lhqzsnxnxn bauzzigmqy (fnedrcqgyt, 124.77) View more	-	03 Jul 2025
				Brachytherapy+cisplatin+Pembrolizumab (Concurrent to Chemoradiation)	jvdphelsmt(rxfrcbopls) = cnfgptesym bauzzigmqy (fnedrcqgyt, 812.53) View more
NCT05075122 (FOCUS, Pubmed \| Med)	Phase 2	Head and Neck Neoplasms First line \| Add-on PD-L1	75	Pembrolizumab	jnguvdcnfe(bwsjwbquph) = a reversible allergic reaction that appeared in one patient xhoecaxzof (ouclstthrj )	Negative	01 Jul 2025
				Pembrolizumab + UV1

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