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Last update 27 Jul 2026

Pexastimogene devacirepvec

Last update 27 Jul 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Oncolytic virus

Synonyms

Pexa-Vec, Pexastimogene devacirepvec (USAN), JX-594

+ [1]

Target

CSF-2R

Action

stimulants

Mechanism

CSF-2R stimulants(Granulocyte-macrophage colony-stimulating factor receptor stimulants)

Therapeutic Areas

NeoplasmsDigestive System DisordersUrogenital Diseases+ [1]

Active Indication

Liver CancerProstatic CancerRenal Cell Carcinoma+ [1]

Inactive Indication

Advanced breast cancerAdvanced cancerAdvanced Hepatocellular Carcinoma+ [20]

Originator Organization

SillaJen, Inc.

Active Organization

SillaJen, Inc.

SillaJen Biotherapeutics, Inc.

GC Corp.

Inactive Organization

Zhaoke Pharmaceutical (Hefei) Co., Ltd.

Transgene SA

Lee's Pharmaceutical Holdings Ltd.

+ [1]

License Organization

Transgene SA

SillaJen Biotherapeutics, Inc.Lee's Pharmaceutical (HK) Ltd.

Drug Highest PhasePhase 3

First Approval Date-

RegulationOrphan Drug (United States)

Structure/Sequence

Sequence Code 1390932638

Clinical Trials associated with Pexastimogene devacirepvec

ACTRN12620001286932

/ RecruitingPhase 2IIT

Neoadjuvant Study of the effect of Intratumoural and Intravenous Pexastimogene Devacirepvec (Pexa-Vec) on CD8-based anti-tumour response in Prostate Cancer Prior to Radical Prostatectomy

Start Date05 May 2023

Sponsor / Collaborator

Royal Melbourne Hospital

NCT04849260

/ Unknown statusPhase 1/2

Phase Ib/II, Open Label, Controlled Clinical Study of Pexa-Vec (Vaccinia GM CSF / Thymidine Kinase-Deactivated Virus) Combined With Recombinant Whole Human Anti-PD-L1 Monoclonal Antibody (ZKAB001) in Metastatic Melanoma After First-line Treatment Failure

The study will be divided into two stages: phase Ib and phase II. The safety and phase II recommended dose (RP2D) of Pexa-Vec combined with ZKAB001 in patients with local progression of failed first-line treatment or metastatic melanoma will be evaluated in phase Ib.
Objective response rate (ORR) and progression-free survival (PFS) of ZKAB001 combined with RP2D's Pexa-Vec or ZKAB001 monotherapy in patients with local progression or metastatic melanoma will be evaluated in phase II.

Start Date26 Aug 2021

Sponsor / Collaborator

Lee's Pharmaceutical Holdings Ltd.

NCT03294083

/ CompletedPhase 1/2

A Phase 1b/2a Dose-escalation and Safety/Efficacy Evaluation Study of Pexa-Vec (Thymidine Kinase-Deactivated Vaccinia Virus Plus GM-CSF) in Combination With Cemiplimab (REGN2810; Anti-PD-1) in Patients With Metastatic or Unresectable Renal Cell Carcinoma (RCC)

This is a Phase 1b/2a, open-label, multi-center, dose-escalation and safety/efficacy evaluation trial of Pexa-Vec plus Cemiplimab in patients with metastatic or unresectable renal cell carcinoma (RCC). The trial consists of a dose-escalation stage, where the maximum feasible dose of Pexa-Vec in combination with Cemiplimab will be determined, followed by an expansion stage. During the expansion patients will receive Cemiplimab alone or in combination with Pexa-Vec, which will be administered either through intravenous (IV) or intratumoral (IT) injection.

Start Date19 Jun 2018

Sponsor / Collaborator

SillaJen, Inc.

[+1]

100 Clinical Results associated with Pexastimogene devacirepvec

100 Translational Medicine associated with Pexastimogene devacirepvec

100 Patents (Medical) associated with Pexastimogene devacirepvec

Literatures (Medical) associated with Pexastimogene devacirepvec

01 Jan 2025·Critical reviews in oncogenesis

Review Article: Intratumoral Immunotherapy: Old Discoveries and Recent Developments in Sarcomas

Review

Author: Crompton, Joseph G ; Navasardyan, Inesa ; Lim, Sin Yee Amelie

Intratumoral immunotherapy represents a promising frontier in the treatment of sarcomas, aiming to transform immunologically "cold" tumors into "hot" ones capable of generating systemic antitumor responses. Building on William Coley’s early work with bacterial infections, modern intratumoral therapies induce the cancer immunity cycle by delivering immune-activating agents directly into tumors to stimulate local and systemic immunity while minimizing systemic toxicity. This review summarizes key strategies under investigation for sarcomas, including cancer vaccines (e.g., LTX-315), cytokines and immunomodulators (e.g., STING agonists), pattern recognition receptor agonists (e.g., TLR4/9 agonists), oncolytic virotherapy (e.g., T-VEC, JX-594), cytotoxic agents (e.g., INT230-6), and cell-based therapies (e.g., dendritic cell injections). These agents are also being evaluated in combination with systemic immunotherapies and local therapies such as radiation. Early clinical trials and preclinical studies demonstrate feasibility, safety, and signs of immune activation, though consistent clinical efficacy remains to be established. Since sarcomas typically exhibit poor immunogenicity due to low tumor mutational burden and immunosuppressive tumor microenvironments, future research is needed to optimize delivery strategies, identify predictive biomarkers, and integrate intratumoral approaches with other emerging immunotherapies to induce the cancer immunity cycle. Collectively, intratumoral immunotherapy may play a transformative role in overcoming resistance and enhancing outcomes for patients with sarcoma.

01 Jan 2024·Liver Cancer

PHOCUS: A Phase 3, Randomized, Open-Label Study of Sequential Treatment with Pexa-Vec (JX-594) and Sorafenib in Patients with Advanced Hepatocellular Carcinoma

Article

Author: Lusky, Monika ; Gane, Edward ; Qin, Shukui ; Borad, Mitesh J ; Pelusio, Adina ; Luca, Angelo ; Burke, James ; Chao, Yee ; Breitbach, Caroline ; Agathon, Delphine ; Heo, Jeong ; Erinjeri, Joseph ; Abou-Alfa, Ghassan K ; Galle, Peter R

Introduction: Intratumoral administration of pexa-vec (pexastimogene devacirepvec), an oncolytic and immunotherapeutic vaccinia virus, given to patients with hepatocellular carcinoma (HCC), is associated with both local and distant tumor responses. We hypothesized subsequent treatment with sorafenib could demonstrate superior efficacy. Methods: This random phase III open-label study evaluated the sequential treatment with pexa-vec followed by sorafenib compared to sorafenib in patients with advanced HCC and no prior systemic treatment. The primary endpoint is overall survival (OS). Key secondary endpoints included time to progression (TTP), progression-free survival, overall response rate (ORR), and disease control rate (DCR). Safety was assessed in all patients who received ≥1 dose of study treatment. Results: The study was conducted at 142 sites in 16 countries. From December 30, 2015, to the interim analysis on August 2, 2019, 459 patients were randomly assigned (pexa-vec plus sorafenib: 234, sorafenib: 225). At the interim analysis, the median OS was 12.7 months (95% CI: 9.89, 14.95) in the pexa-vec plus sorafenib arm and 14.0 months (95% CI: 11.01, 18.00) in the sorafenib arm. This led to the early termination of the study. The median TTP was 2.0 months (95% CI: 1.77, 2.96) and 4.2 months (95% CI: 2.92, 4.63); ORR was 19.2% (45 patients) and 20.9% (47 patients); and DCR was 50.0% (117 patients) and 57.3% (129 patients) in the pexa-vec plus sorafenib and sorafenib arms, respectively. Serious adverse events were reported in 117 (53.7%) patients in the pexa-vec plus sorafenib and 77 (35.5%) patients in the sorafenib arm. Liver failure was the most frequently reported in both groups. Conclusion: Sequential pexa-vec plus sorafenib treatment did not demonstrate increased clinical benefit in advanced HCC and fared worse compared to sorafenib alone. The advent of the added value of checkpoint inhibitors should direct any further development of oncolytic virus therapy strategies.

03 Oct 2023·Expert opinion on biological therapy

Oncolytic virotherapies for pediatric tumors

Review

Author: Nicholas MB Laskay ; James M Markert ; Atchley, Travis J ; Johnston, James M ; Laskay, Nicholas Mb ; Gross, Evan G ; Markert, James M ; Hamo, Mohammad A ; Travis J Atchley ; Mohammad A Hamo ; James M Johnston ; Evan G Gross ; Estevez-Ordonez, Dagoberto ; Dagoberto Estevez-Ordonez

INTRODUCTION:

Many pediatric patients with malignant tumors continue to suffer poor outcomes. The current standard of care includes maximum safe surgical resection followed by chemotherapy and radiation which may be associated with considerable long-term morbidity. The emergence of oncolytic virotherapy (OVT) may provide an alternative or adjuvant treatment for pediatric oncology patients.

AREAS COVERED:

We reviewed seven virus types that have been investigated in past or ongoing pediatric tumor clinical trials: adenovirus (AdV-tk, Celyvir, DNX-2401, VCN-01, Ad-TD-nsIL-12), herpes simplex virus (G207, HSV-1716), vaccinia (JX-594), reovirus (pelareorep), poliovirus (PVSRIPO), measles virus (MV-NIS), and Senecavirus A (SVV-001). For each virus, we discuss the mechanism of tumor-specific replication and cytotoxicity as well as key findings of preclinical and clinical studies.

EXPERT OPINION:

Substantial progress has been made in the past 10 years regarding the clinical use of OVT. From our review, OVT has favorable safety profiles compared to chemotherapy and radiation treatment. However, the antitumor effects of OVT remain variable depending on tumor type and viral agent used. Although the widespread adoption of OVT faces many challenges, we are optimistic that OVT will play an important role alongside standard chemotherapy and radiotherapy for the treatment of malignant pediatric solid tumors in the future.

News (Medical) associated with Pexastimogene devacirepvec

04 Feb 2026

·www.prnewswire.com

Oncolytic Virus Clinical Trial Landscape Gains Momentum: 100+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight

The oncolytic virus therapy landscape has expanded steadily over the past decade, with over 100 companies worldwide pursuing clinical or preclinical programs. A few leaders, such as Candel Therapeutics, Binhui Biopharmaceutical, Oncolytics Biotech, EpicentRx, Hangzhou Converd, and others, anchor the mid and late-stage pipeline. LAS VEGAS, Feb. 4, 2026 /PRNewswire/ -- DelveInsight's Oncolytic Virus Competitive Landscape 2026 report provides comprehensive global coverage of pipeline oncolytic virus therapies across various stages of clinical development. The report offers an in-depth analysis of key trends, emerging therapies, and the competitive landscape, highlighting the strategies of major pharmaceutical companies to advance their pipelines and capitalize on future growth opportunities. In addition, it provides critical insights into clinical trial benchmarking, partnering and licensing, and regulatory pathways with the FDA and EMA, enabling stakeholders to make informed decisions and optimize development strategies in the oncolytic virus domain. Oncolytic Virus Clinical Trial Analysis Summary DelveInsight's oncolytic virus pipeline report highlights a robust landscape, with 100+ active players developing 110+ oncolytic virus drugs. Key oncolytic virus companies such as Oncolytics Biotech, SillaJen, Candel Therapeutics, Binhui Biopharmaceutical, Oryx GmbH, Oncolys Biopharma, EpicentRx, Theriva Biologics, Lokon Pharma, DNAtrix, Surv BioPharma Inc., Treovir, Immvira Pharma, Virogin Biotech, OrienGene Biotechnology, Apices Soluciones, Vyriad, Transgene, BioEclipse Therapeutics, Imugene, CG Oncology, Istari Oncology, Akamis Bio, Elicera Therapeutics, and others are evaluating new oncolytic virus drugs to improve the treatment landscape. Promising pipeline oncolytic virus therapies, such as Pelareorep, Pexa-Vec, CAN-2409, BS001, ParvOryx, Telomelysin, AdAPT-001, VCN-01, LOAd 703, DNX-2401, Surv.m-CRA-1, G207, MVR-T3011, VG161, OrienX010, AloCelyvir, Voyager-V1, TG6002, CRX-100, CF33-hNIS, CG0070, PVS-RIPO, NG-350A, ELC-201, and others, are in different phases of oncolytic virus clinical trials. Request a sample and discover the recent advances in oncolytic virus drugs @ Key Factors Driving the Oncolytic Virus Therapy Market Growing Cancer Incidence: The global rise in cancer prevalence, especially in hard-to-treat solid tumors, has intensified demand for novel, effective treatment modalities. Conventional therapies such as chemotherapy and radiation often have significant side effects and limited long-term efficacy, motivating the adoption of immunotherapeutic alternatives like oncolytic virus therapies. Increasing Understanding of Cancer Biology: Growing knowledge of cancer biology has enabled the development of oncolytic virus therapies that selectively target and destroy cancer cells while sparing healthy cells. Advancements in Viral Engineering: These advances are enabling more precise, targeted oncolytic virus therapies, significantly expanding the global oncolytic virus therapy market. Rising Adoption of Personalized Medicine: Oncolytic virus therapies align closely with personalized treatment approaches by enabling tailored viral constructs and immunomodulatory payloads based on tumor genomics. What is Oncolytic Virus Therapy? Oncolytic virus therapy is an innovative form of cancer treatment that uses genetically engineered or naturally occurring viruses to selectively infect and destroy cancer cells. These viruses replicate inside tumor cells, causing them to burst while leaving healthy cells largely unharmed. As the cancer cells break apart, they release tumor antigens that help activate the body's immune system, enhancing its ability to recognize and attack remaining cancer cells. This dual action, direct tumor destruction and immune stimulation, makes oncolytic virus therapy a promising approach in modern oncology, often used alongside other treatments such as immunotherapy or chemotherapy. Applications of Oncolytic Virus Therapy Oncolytic virus (OV) cancer therapies can be used for both diagnosis and treatment of cancer. OVs can function as cancer vaccines by boosting tumor-specific T-cell responses. Oncolytic viruses can be engineered with immunostimulatory molecules to enhance immune activation. OVs show strong synergy when combined with other immunotherapies, such as checkpoint inhibitors. Oncolytic Viruses in Combination with Chemotherapy Chemotherapy works systemically, unlike surgical resection or radiotherapy. It remains essential for treating patients with disseminated (widespread) cancer. Because chemotherapy is systemic, it is often combined with oncolytic viruses (OVs), which typically require localized delivery to achieve a strong antitumor effect. Systemically administered OVs have shown limited therapeutic benefit in several clinical trials. Delivering OVs effectively to metastatic sites remains a major challenge. Therefore, using chemotherapy as a systemic adjuvant to localized OV therapy is of significant clinical interest and is currently being actively investigated. Oncolytic Virus Market Analysis: Drug Profile IMLYGIC: Amgen IMLYGIC (talimogene laherparepvec), developed by Amgen, is a first-of-its-kind oncolytic viral therapy for melanoma that cannot be surgically removed. It combines direct tumors destruction with immune system stimulation. The therapy is based on a genetically engineered herpesvirus engineered to preferentially infect and destroy cancer cells, releasing GM-CSF to trigger a broader anti-tumor immune response. In the Phase III OPTiM trial, IMLYGIC produced durable responses in about 16% of patients, compared with 2% with GM-CSF alone, and overall responses in roughly 26%. Patients at earlier disease stages achieved better outcomes, with some achieving long-term control. The treatment is usually well tolerated, with most adverse effects being mild and resembling flu symptoms. Although its independent effect on survival and deep organ metastases is modest, its value is most evident when combined with checkpoint inhibitors. Early data show response rates approaching 56%, and ongoing studies aim to further define these synergistic benefits. As the first FDA-approved oncolytic virus in the West, IMLYGIC has validated the broader concept of viral immunotherapy and is now being explored as a component of combination immuno-oncology approaches. Oncorine: Shanghai Sunway Biotech Oncorine, also known as H101, is a genetically engineered adenovirus with an E1B gene deletion developed by Shanghai Sunway Biotech in China. It is administered alongside chemotherapy to treat nasopharyngeal carcinoma and head and neck cancers. Its approval and effective clinical use have significantly advanced oncolytic virus–based cancer immunotherapy. Marketed as Recombinant Human Adenovirus Type 5 Injection, Oncorine became the first oncolytic virus approved for cancer treatment by the Chinese State Food and Drug Administration (CFDA) in November 2005. Since its introduction, it has demonstrated a favorable safety profile and notable antitumor activity across various cancers and malignant pleural effusions. Exploratory clinical studies have also evaluated Oncorine for treating liver cancers, pancreatic cancer, cervical cancer, malignant melanoma, and metastatic liver tumors. Find out more about oncolytic virus cancer drugs @ Oncolytic Virus Analysis A snapshot of the Pipeline Oncolytic Virus Drugs mentioned in the report: Learn more about the emerging oncolytic virus therapies @ Oncolytic Virus Clinical Trials Recent Developments in Oncolytic Virus Treatment Space In November 2025, Imugene Ltd and JW Therapeutics announced a co-development collaboration to evaluate the combination of Imugene's oncolytic virus CF33-CD19 (onCARlytics) and JW's Carteyva®, a CD19-directed autologous CAR-T cell therapy, for patients with advanced solid tumors. This collaboration includes preclinical in vitro and in vivo studies, followed by a Phase I investigator-initiated trial to be conducted exclusively in China at premier CAR-T clinical centers. The approach utilizes Imugene's CF33-CD19 virus to induce CD19 expression on tumor cells, rendering them susceptible to targeting by CD19 CAR-T therapies, a transformative "mark and kill" strategy. In July 2025, Calidi Biotherapeutics, Inc., a clinical-stage biotechnology company pioneering the development of targeted therapies with the potential to deliver genetic medicines to distal sites of disease, announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for CLD-201 (SuperNova), the company's allogeneic adipose stem-cell loaded oncolytic virus for the treatment of soft tissue sarcoma. In June 2025, Oncolytics Biotech presented a poster demonstrating the role of pelareorep in activating anti-tumor immunity in PDAC. In May 2025, Candel Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) had granted Regenerative Medicine Advanced Therapy (RMAT) designation to CAN-2409 (aglatimagene besadenovec), the Company's biological immunotherapy lead candidate, for the treatment of newly diagnosed localized prostate cancer in patients with intermediate-to-high-risk disease. CAN-2409 was also previously granted FDA Fast Track designation for the same indication. In May 2025, Elicera Therapeutics AB announced that it had entered into a Material Transfer Agreement (MTA) with University Hospital Tübingen in Germany. Under the agreement, the company's oncolytic virus candidates ELC-100 and ELC-201 will be included in tests aimed at developing a new type of companion diagnostic to predict the potential success of a novel neuroendocrine neoplasm (NEN) therapy, immuno-virotherapy. In April 2025, Hangzhou ConVerd Co., Ltd. announced that the first Phase IIa clinical study of hV01, ConVerd's first oncolytic vaccinia virus product, had successfully completed first patient dosing, with the subject in good condition. This phase IIa clinical study investigates the administration of a single dose of recombinant human IL-21 oncolytic poxvirus injection (hV01) per cycle for the treatment of patients diagnosed with advanced sarcoma and cervical cancer. It is important to note that the indication is for advanced sarcoma and recurrent or metastatic cervical cancer for which there is no standard of care available at this stage of the treatment process, or where standard of care has failed. Scope of the Oncolytic Virus Pipeline Report Coverage: Global Key Oncolytic Virus Companies: Oncolytics Biotech, SillaJen, Candel Therapeutics, Binhui Biopharmaceutical, Oryx GmbH, Oncolys Biopharma, EpicentRx, Theriva Biologics, Lokon Pharma, DNAtrix, Surv BioPharma Inc., Treovir, Immvira Pharma, Virogin Biotech, OrienGene Biotechnology, Apices Soluciones, Vyriad, Transgene, BioEclipse Therapeutics, Imugene, CG Oncology, Istari Oncology, Akamis Bio, Elicera Therapeutics, and others Key Oncolytic Virus Pipeline Therapies: Pelareorep, Pexa-Vec, CAN-2409, BS001, ParvOryx, Telomelysin, AdAPT-001, VCN-01, LOAd 703, DNX-2401, Surv.m-CRA-1, G207, MVR-T3011, VG161, OrienX010, AloCelyvir, Voyager-V1, TG6002, CRX-100, CF33-hNIS, CG0070, PVS-RIPO, NG-350A, ELC-201, and others Dive deep into rich insights for new oncolytic Virus treatments, visit @ Oncolytic Virus Drugs Table of Contents Related Reports Oncolytic Virus Therapies Market Oncolytic Virus Therapies Market Size, Target Population, Competitive Landscape, and Market Forecast – 2034 report deliver an in-depth understanding of the market trends, market drivers, market barriers, and key oncolytic virus therapies companies including Targovax, Genelux, Imugene, EpicentRx, Replimune, Genelux Corporation, Candel Therapeutics, DNAtrix, SillaJen, Treovir, Lokon Pharma AB, Istari Oncology, CG Oncology, Amgen, Daiichi Sankyo, and others. Melanoma Market Melanoma Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key melanoma companies including IO Biotech, Merck Sharp & Dohme, Eisai, Regeneron Pharmaceuticals, Iovance Biotherapeutics, Highlight Therapeutics, Linnaeus Therapeutics, Spring Bank Pharmaceuticals, Taiga Biotechnologies, Inc., Aivita Biomedical, Inc., Genentech, Inc., Dana-Farber Cancer Institute, BioMed Valley Discoveries, Inc, Amgen, Checkmate Pharmaceuticals, Astellas Pharma Inc, Galectin Therapeutics Inc., Eucure (Beijing) Biopharma Co., Ltd, Mologen AG, Ultimovacs ASA, Seagen Inc., MedImmune LLC, OnKure, Inc., Syntrix Biosystems, Inc., Anaveon AG, iOx Therapeutics, Portage Biotech, ModernaTX, Inc., Nektar Therapeutics, BioNTech SE, Sapience Therapeutics, HUYABIO International, LLC., Provectus Biopharmaceuticals, Inc., Cantargia AB, Turnstone Biologics, Corp., Aura Biosciences, F-star Therapeutics, Inc., Day One Biopharmaceuticals, Inc., Kinnate Biopharma, Xencor, Inc., HiFiBiO Therapeutics, and others. Colorectal Cancer Market Colorectal Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key colorectal cancer companies, including Mirati Therapeutics, Exelixis, Enterome, Arcus Biosciences, Lyell Immunopharma, AstraZeneca, Novartis Pharmaceuticals, Surgimab, Numab Therapeutics, SOTIO Biotech, Amgen, Sichuan Baili Pharmaceutical, Qilu Pharmaceutical, Bristol-Myers Squibb, NGM Biopharmaceuticals, Takeda, PureTech, Pfizer, Kezar Life Sciences, Salubris, Biotherapeutics , and others. Pancreatic Cancer Market Pancreatic Cancer Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key pancreatic cancer companies including FibroGen, OSE Immunotherapeutics, Lokon Pharma, Oncotelic Inc., Tesaro, Inc., Helix BioPharma Corporation, Theradex, Tvardi Therapeutics, Incorporated, AIM ImmunoTech Inc., BioNTech SE, Prestige Biopharma Limited, HCW Biologics, Cantex Pharmaceuticals, XBiotech, Inc., Lumicell, Inc., CARsgen Therapeutics Co., Ltd., Panbela Therapeutics, Inc. , and others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP 21% more press release views with Request a Demo

Clinical ResultPhase 2ImmunotherapyFast TrackPhase 3

15 Oct 2024

·www.pharmaceutical-technology.com

Transgene’s stock drops after Phase II cancer jab trial misses primary endpoint

Transgene’s Phase II trial of viral-based vaccine TG4001 failed to meet its primary endpoint. Credit: Kitsawet Saethao via Shutterstock. Transgene ’s stock has dropped after a Phase II trial of its therapeutic vaccine candidate TG4001 (tipapkinogene sovacivec) failed to meet its primary endpoint of improvement in progression-free survival (PFS) in solid tumours. Transgene’s share price fell 18.8% from €1.17 at close on 11 October to a low of €0.95 on Monday morning (14 October) after the announcement, settling at around €1 per share. The trial (NCT03260023) aimed to assess the impact of TG4001 in combination with Merck KgaA’s Bavencio (avelumab), a PD-L1 checkpoint inhibitor, in 150 patients with recurrent or metastatic HPV16-positive cervical and anogenital cancers who were either treatment-naïve or had one prior systemic chemotherapy, but no earlier immunotherapy. The addition of TG4001 did not produce statistically significant improvements in PFS across the study population, however, Transgene said it remains optimistic. In a subgroup of patients with cervical cancer – comprising about 54% of the trial population – the data suggested a potential trend towards improved PFS. The French biotech is planning further analysis, including examining the data by PD-L1 status, before deciding on the next steps for the candidate’s development. PD-L1 status refers to the level of expression of the PD-L1 protein on tumour cells, which can influence how well a patient’s cancer responds to Bavencio. See Also: Bayer applies for EU approval of menopause drug elinzanetant ENHERTU gains conditional approval in China to treat NSCLC TG4001, a viral-based vaccine, works by inducing an immune response against cells expressing the HPV16 E6 and E7 antigens, aided by the inclusion of human IL-2 to strengthen this immune reaction. In the announcement accompanying the data, Transgene’s CEO Alessandro Riva said: “Failure to meet the primary objective in our Phase II study with TG4001 is disappointing. Nevertheless, we are encouraged by the positive efficacy trend in favour of the combination regimen in cervical cancer patients.” Transgene said that it remains focused on its lead asset TG4050 , an individualised cancer vaccine that is in a Phase I/II trial (NCT04183166) in patients with head and neck cancer. TG4050 is based on Transgene’s myvac platform, which generates personalised therapies, based on patient-specific mutations (neoantigens), that are designed to induce a T cell response to tumour cells. The company expects to share 24-month data on Phase I patients in November at the Society for Immunotherapy of Cancer (SITC) conference. According to a GlobalData report , cancer vaccines are a key immuno-oncology development trend. TG4050 is a personalised vaccine, whereas TG4001 is an off-the-shelf standardised treatment. According to over half of high-prescribing key opinion leaders who responded to a GlobalData survey in 2023, personalised mRNA vaccines are the most promising cancer vaccine type. GlobalData is the parent company of Clinical Trials Arena. The company is also developing an oncolytic virus therapy, BT-001, for treating solid tumours in partnership with BioInvent International . The therapy is being evaluated in a Phase I/IIa clinical trial (NCT04725331) as a single agent and in combination with MSD’s PD-1 checkpoint inhibitor Keytruda (pembrolizumab). Transgene’s journey with oncolytic virus therapies has been challenging. In May 2023, AstraZeneca ended its four-year partnership with the company, a collaboration formed in 2019 with a $10m upfront payment to Transgene to develop oncolytic virus candidates. The partnership’s troubles started early, as one of Transgene’s candidates Pexa-Vec failed a Phase III trial (NCT02562755) just months after the deal was signed.

Phase 2VaccineDrug ApprovalImmunotherapyPhase 3

22 Apr 2024

·www.prnewswire.com

OSR Holdings and SillaJen have entered into a Memorandum of Understanding to collaboratively pursue the development of innovative therapeutics and enter the U.S. market

SEOUL, South Korea, April 22, 2024 /PRNewswire/ -- OSR Holdings, a global healthcare holding company, announced that it has signed a memorandum of understanding (MOU) with SillaJen, a Kosdaq-listed biotech firm with a pipeline of immuno-oncology drug candidates, to share technology and clinical development resources and launch a strategic collaboration between the two companies. Through the MOU, the two companies will explore various ways to collaborate to create synergies between current platform technologies and new drug candidates. OSR Holdings was established in 2019 in South Korea as a "hub-and-spoke" business model, which has become more visible in the biopharma space over the past decade. The "hub-and-spoke" model, exemplified by companies like Roivant Sciences, PureTech Health and BridgeBio Pharma, enables centralized management of drug development strategies, clinical plans, and resource allocation by the holding company. The model allows the holding companies' subsidiaries to concentrate on the advancement of new drug assets and technological innovations. The model's scalability and resource efficiency are notably enhanced, especially as clinical phases of drug development progresses. OSR Holdings' current portfolio includes Vaximm AG, a Swiss entity developing immuno-oncology therapeutics; Darnatein, a South Korean drug developer of osteoarthritis therapeutics; and RMC, a distributor of specialized medical devices. Vaximm AG, a venture spun out from Merck KGaA, has completed Phase 2a clinical trials for glioblastoma. Darnatein, established by Dr. Seung-Hyun Choi, a former researcher at one of the leading research institutes in the U.S., Salk Institute for Biological Studies, boasts a robust platform for cartilage and bone regeneration and is developing new drugs for other diverse disease indications based on this foundational technology. SillaJen boasts a diverse pipeline of new drugs, including the SJ-600 series and BAL0891, and is leading the way in developing anti-cancer viruses based on its GEEV® platform. Known for its immune-evasion capabilities, GEEV® enables anti-cancer viruses to efficiently target affected areas. The SJ-600 series is an anti-cancer pipeline based on SillaJen's GEEV® platform. BAL0891, a mitotic checkpoint inhibitor, has advanced to Phase 1 clinical trials targeting solid tumors and will be conducted in the United States. Sung Jae "Alex" Yu, COO of OSR Holdings, stated, "the signing of this MOU marks the two companies transitioning into strategic partners for the development of innovative drug technologies in the global immuno-oncology market. We eagerly anticipate exploring a variety of opportunities to further our relationship and collaborations with SillaJen, particularly in the U.S., the largest pharmaceutical market in the world." A representative of SillaJen stated, "We can anticipate synergies by collaborating between a company that possesses original anti-cancer drug development technology and a company with global business expertise," and added, "Aside from our cooperation with existing partners, we will have the opportunity to present SillaJen's exceptional pipeline in the U.S. market." About OSR Holdings OSR Holdings, headquartered in Korea, is a global healthcare holding company with biopharma and medical device subsidiaries in areas with high unmet medical needs such as certain cancer indications. The biopharma subsidiaries are developing novel drug candidates based on differentiated technologies. The medical device subsidiary operates as an exclusive distributor of neuro-intervention devices to nationwide hospitals in Korea. In November 2023, OSR Holdings announced a business combination with Bellevue Life Sciences Acquisition Corp (Nasdaq: BLAC), a SPAC that completed a $69M IPO in February 2023. About SillaJen SillaJen, Inc. (KOSDAQ: 215600) is a publicly held biotech company developing anti-cancer therapies, with its clinical-stage products, Pexa-Vec, an oncolytic viral immunotherapy, and BAL0891, a mitotic checkpoint inhibitor. The company is also developing a unique CD-55 expressing vaccinia virus platform, GEEV®, which can be delivered to tumors via intravenous injection while evading the complement system and neutralizing antibodies against oncolytic viruses. Additional information about SillaJen is available at . Disclaimer Language: This press release contains certain forward-looking statements regarding, among other things, statements relating to goals, plans and projections regarding the Company's financial position, results of operations, market position, product development and business strategy. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statements can be guaranteed, and actual results may differ materially from such statements. The information in this release is provided only as of the date of this release, and OSR Holdings undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law. SOURCE OSR Holdings

Phase 1Phase 2IPOAcquisition

100 Deals associated with Pexastimogene devacirepvec

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KEGG	Wiki	ATC	Drug Bank
D10485	Pexastimogene devacirepvec	-	DB15069

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Advanced Hepatocellular Carcinoma	Phase 3	United States	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	China	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	Australia	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	Canada	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	France	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	Germany	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	Hong Kong	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	Israel	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	Italy	SillaJen, Inc.	01 Oct 2015
Advanced Hepatocellular Carcinoma	Phase 3	New Zealand	SillaJen, Inc.	01 Oct 2015

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00429312 (CTgov)	Phase 1/2	Unresectable Melanoma	10	JX-594	fvffpmntgy = qlrqkvxjbf fxyeqanjto (lguxeymypp, sskniuvarm - iwwmvjomqt) View more	-	27 Jul 2026
NCT01380600 (CTgov)	Phase 1	Refractory Colorectal Carcinoma	15	Pexa-Vec (Cohort 1: Pexa-Vec 1 × 10^6 Pfu/kg)	fwybvlahli = ictmvxsvtf tbnegqytiq (pxegrcvzyq, yptokqbxto - gvivpsugcx) View more	-	08 Jul 2026
				Pexa-Vec (Cohort 2: Pexa-Vec 1 × 10^7 Pfu/kg)	fwybvlahli = gksvtrispi tbnegqytiq (pxegrcvzyq, fuusrtalwh - dysvnafjcb) View more
NCT03294083 (CTgov)	Phase 1/2	Unresectable Renal Cell Carcinoma	95	Cemiplimab+Pexastimogene Devacirepvec (Part 2-Arm A, Pexa-Vec (IT) and Cemiplimab)	dyjjgymmjz = rhgxmghvcc britaihsqb (xgycllmsyr, ginfcuamaf - cjuttrdbqh) View more	-	18 Jun 2026
				Cemiplimab+Pexastimogene Devacirepvec (Part 2-Arm B, Cemiplimab)	dyjjgymmjz = jvbgxrdtqu britaihsqb (xgycllmsyr, taydvpdmxu - nhdtkikdca) View more
NCT03206073 (Pubmed)	Phase 1/2	Colorectal Cancer	34	PexaVec	nhqjmkxecl(olwsjlosnt) = wnfdipqeyt fawpvdzgbg (cxacehydcp )	-	01 Feb 2023
NCT02630368 (METROmaJX, Pubmed) Manual	Phase 2	Advanced breast cancer	10	Cyclophosphamide+Pexastimogene devacirepvec	khbgeqvxsu(qgslqdixpi) = hrqjnviahs fwnfwldiiq (xmwadczfal, 1.1 - 1.9) View more	Positive	06 Dec 2022
NCT03071094 (CTgov)	Phase 1/2	Advanced Hepatocellular Carcinoma	14	Nivolumab+Pexa Vec (Pexa-Vec Combined With Nivolumab - Phase I)	lthdgqzxpc = lukibyzavh khfpgwxoqj (utbcqqpwfn, rsssyqoxgr - yxwlefbfkm) View more	-	19 Nov 2021
				Nivolumab+Pexa Vec (Pexa-Vec Combined With Nivolumab - Phase IIa)	wtsspicleq(ixguaevxoy) = czahhbaipx bxjosqhvtd (klhxfppbdw, wpsquzfcvq - suaykwmxwi) View more
NCT03206073 (CTgov)	Phase 1/2	Adenocarcinoma of large intestine \| Refractory Colorectal Carcinoma \| Refractory Cancer	34	Pexa-Vec+Durvalumab (1/Arm A1 Pexa-Vec 3 x 10E^8 Plaque-forming Unit (Pfu) + Durvalumab 1500 mg)	uwlmdnlfho = tufcaokosq gxugpjriug (zyxzpazhhn, jxzurelknp - xllsrakqfi) View more	-	26 Oct 2021
				Pexa-Vec+Durvalumab (2/Arm A2 Pexa-Vec 1 x 10E^9 Plaque-forming Unit (Pfu) +Durvalumab 1500 mg)	uwlmdnlfho = vthasyrisr gxugpjriug (zyxzpazhhn, ivysmzaczx - wjeeucjsda) View more
NCT01394939 (CTgov)	Phase 1/2	Refractory Colorectal Carcinoma	52	JX-594 (Single Agent_ Cohort 1)	yjdbjjuqle = leshzosjow sbgyvumwwo (nfcvhtpuvh, pxgbguwadb - hhjzwhthyp) View more	-	08 Jan 2021
				JX-594 (Single Agent_Cohort 2)	yjdbjjuqle = fadhfrkhoe sbgyvumwwo (nfcvhtpuvh, rmdmfegfxq - peazpjuzfb) View more
NCT02562755 (PHOCUS, CTgov)	Phase 3	Advanced Hepatocellular Carcinoma	459	Pexa Vec+Sorafenib (Pexa-Vec Followed by Sorafenib)	dtkzwnredj = evslwzxcgo mzlpsleeas (xzrvwuqzvs, smczkleanf - cbouyhumoa) View more	-	16 Dec 2020
				Sorafenib (Sorafenib)	dtkzwnredj = kwvokkiirj mzlpsleeas (xzrvwuqzvs, wzaetarsxe - fuuudluzez) View more
NCT01636284 (FLASH, CTgov)	Phase 2	Advanced Hepatocellular Carcinoma	16	JX-594 recombinant vaccina GM-CSF	uwkfpjzyvp = pgzlhaukfb xdarruzqhs (pdskvjvyyg, leiiyqkxtl - scwaigzfus) View more	-	24 Nov 2020

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