On December 7, 2024 Merus N.V. (Nasdaq: MRUS) (Merus, the Company, we, or our), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported interim clinical data as of a July 5, 2024 data cutoff from the ongoing phase 1/2 trial of petosemtamab, a Biclonics targeting EGFR and LGR5, in previously treated (2L+) patients (pts) with recurrent/metastatic (r/m) head and neck squamous cell carcinoma (HNSCC) (Press release, Merus, DEC 7, 2024, View Source [SID1234648857]). These data were presented by Christophe Le Tourneau MD, Ph.D., Institut Curie, Paris, France at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Asia Congress on Saturday, Dec. 7 in Singapore.

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"Petosemtamab clinical data in r/m HNSCC continues to demonstrate potentially practice changing efficacy and safety, both as monotherapy in 2L+ and in combination with pembrolizumab in 1L PD-L1 expressing HNSCC," said Fabian Zohren, M.D., Ph.D., Chief Medical Officer of Merus. "Further, the monotherapy durability of petosemtamab thus far compares favorably to current standard of care, which we believe is another positive indicator for the likelihood of success of our phase 3 investigation of petosemtamab and pembrolizumab in 1L PD-L1 expressing HNSCC."

"Head and neck squamous cell carcinoma remains a deadly disease with limited treatment options," added Dr. Le Tourneau. "With its strong clinical outcomes across a large dataset of patients, regardless of HPV status and EGFR expression, petosemtamab has the potential to become a new standard of care for patients with recurrent/metastatic head and neck cancer."

Presentation title: Petosemtamab (MCLA-158) monotherapy in previously treated (2L+) recurrent/metastatic (r/m) head and neck squamous cell carcinoma (HNSCC): Phase 2 trial
Observations in the presentation include:

As of a July 5, 2024 data cutoff date, 82 pts were treated with petosemtamab 1500 mg Q2W
The efficacy population consists of 75 pts who had the opportunity for 4 or more months follow up and ≥1 post-baseline tumor assessment; or who discontinued early due to disease progression or death
Seven pts were not efficacy evaluable: 6 pts were previously described at AACR (Free AACR Whitepaper) 2023 and one additional patient withdrew due to infusion related reaction (IRRs) on Day 1
Confirmed overall response rate (ORR): 36% (90% CI: 27–46; 27/75) by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. per investigator assessment, including 4 complete responses (CRs), with one CR continuing on treatment for more than 3 years as of the data cutoff; and 13% (2/15) ORR in HPV associated cancer with another 5 patients achieving stable disease
At the time of data cutoff, 10 pts remain on treatment including 8 responders and 2 pts with stable disease
Median duration of response (DOR), progression free survival (PFS) and overall survival (OS) were 6.2, 4.9 and 11.4 months
For the most mature data set, the single arm cohort previously presented at AACR (Free AACR Whitepaper) 2023, as of a July 5, 2024 data cutoff, for all 54 patients, the median DOR, PFS and OS were 6.7, 5.1, and 12.0 months, respectively; among the 48 treatment evaluable subset, they were 6.7, 5.2, and 12.5 months, respectively
Petosemtamab 1500 mg Q2W continues to be well tolerated with a manageable safety profile with no new safety signals observed (82 pts)
Infusion related reactions (IRRs) were predominantly seen on day 1 of cycle 1; a clinically meaningful reduction in the incidence and severity of IRR was observed with an updated administration regimen
As of a July 5, 2024 data cutoff date, 28 pts were treated with petosemtamab 1100 mg Q2W
The efficacy population consists of 27 pts who had the opportunity for 4 or more months follow up and ≥1 post-baseline tumor assessment; or who discontinued early due to disease progression or death
One pt was not evaluable for efficacy due to withdrawing consent with <2 months treatment
ORR: 19% (90% CI: 8–35; 5/27), including 2 CRs, by RECIST v1.1. per investigator assessment
The full presentation is available on the Merus website.

Petosemtamab Clinical Development
r/m HNSCC: LiGeR-HN1 phase 3 trial in 1L and LiGeR-HN2 phase 3 trial in 2/3L enrolling; phase 2 trial of petosemtamab in combination with pembrolizumab in PD-L1+ 1L HNSCC ongoing with a clinical data update planned for 2025

mCRC: Phase 2 trial of petosemtamab in combination with standard chemotherapy in 2L metastatic colorectal cancer (mCRC) enrolling; phase 2 trial in 1L mCRC in combination with standard chemotherapy planned to initiate in 2025, and phase 2 trial in 3L+ monotherapy planned to initiate in 2025; mCRC initial clinical data planned for 2025

Company Conference Call and Webcast Information
Merus will hold a conference call and webcast for investors on December 7, 2024 at 9:00 a.m. ET. A replay will be available after the completion of the call in the Investors and Media section of our website for a limited time. 

Date & Time: Dec. 07, 2024 at 9:00 a.m. ET
Webcast link: Available on our website
Dial-in: Toll Free: 1 (800) 715-9871/ International: 1 (646) 307-1963
Conference ID: 1978503

About Head and Neck Cancer
Head and neck squamous cell carcinoma (HNSCC) describes a group of cancers that develop in the squamous cells that line the mucosal surfaces of the mouth, throat, and larynx. These cancers begin when healthy cells change and grow in an unchecked manner, ultimately forming tumors. HNSCC is generally associated with tobacco consumption, alcohol use and/or HPV infections, depending on where they develop geographically. HNSCC is the sixth most common cancer worldwide and it is estimated that there were more than 930,000 new cases and over 465,000 deaths from HNSCC globally in 2020.1 The incidence of HNSCC continues to rise and is anticipated to increase by 30% to more than 1 million new cases annually by 2030.2 HNSCC is a serious and life-threatening disease with poor prognosis despite currently available standard of care therapies.

1 Sung et al. CA Cancer J Clin, 71:209-49, 2021; 2 Johnson, D.E., Burtness, B., Leemans, C.R. et al. Head and neck squamous cell carcinoma. Nat Rev Dis Primers 6, 92 (2020)

About Petosemtamab
Petosemtamab, or MCLA-158, is a Biclonics low-fucose human full-length IgG1 antibody targeting the epidermal growth factor receptor (EGFR) and the leucine-rich repeat containing G-protein-coupled receptor 5 (LGR5). Petosemtamab is designed to exhibit three independent mechanisms of action including inhibition of EGFR-dependent signaling, LGR5 binding leading to EGFR internalization and degradation in cancer cells, and enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP) activity.

On December 6, 2024 BostonGene, a leading provider of AI-driven molecular and immune profiling solutions, reported the selection of 10 abstracts for poster presentations and two abstracts for online publication at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, which will be held December 7-10, 2024, in San Diego, California. BostonGene will exhibit in booth #1955 (Press release, BostonGene, DEC 6, 2024, View Source [SID1234648860]).

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"We are excited to present our research findings at ASH (Free ASH Whitepaper) that underscores the transformative potential of integrating molecular and immune profiling with advanced analytics. This approach is pivotal in advancing precision medicine and improving outcomes for individuals battling cancer," said Nathan Fowler, MD, Chief Medical Officer at BostonGene.

Details of the presentations are below:

Abstract: 1593
Title: Machine Learning-Based Approach to Improve Classification and Diagnostics of Peripheral T-Cell Lymphomas
Date & time: Saturday, December 7 | 5:30 PM -7:30 PM
Presenter: Anastasia Sobol, MS, BostonGene

BostonGene developed a machine learning-based classifier to aid in the diagnoses of peripheral T-cell lymphoma not otherwise specified (PTCL-NOS) cases. Using this transcriptomic classifier to examine over 400 cases, approximately 50% of PTCL-NOS cases were reclassified into specific subtypes based on the gene expression pattern of each sample. These findings may enhance the understanding of PTCL pathogenesis and lead to improved diagnostic methods.

Research conducted in collaboration with Weill Cornell Medicine

Abstract: 3610
Title: Novel Plasma Cell-Free RNA-Based Liquid Biopsy Approach for CLL
Date & time: Sunday, December 8 | 6:00 PM – 8:00 PM
Presenter: Andrey Shubin, PhD, BostonGene

To address gaps in disease monitoring for chronic lymphocytic leukemia (CLL) patients, BostonGene developed a novel liquid biopsy approach based on plasma cell-free RNA (cfRNA) sequencing. From a simple blood draw, the cfDNA assay revealed malignant B-cell fractions, B-cell receptor repertoires and clinically significant mutations from cfRNA transcriptomes, as well as associations between treatment and immune- or tissue-specific processes. This liquid biopsy platform may support longitudinal monitoring CLL dynamics and minimal residual disease with further validation.

Research conducted in collaboration with Massachusetts General Hospital and Harvard Medical School

Abstract: 2981
Title: Gene Expression-Based Classifier Reclassifies Burkitt Lymphoma, HGBL NOS, DLBCL NOS, and Double/Triple Hit Lymphomas into Subtypes with More Uniform Mutational and Microenvironment Landscapes and Treatment Response
Date & time: Sunday, December 8 | 6:00 PM – 8:00 PM
Presenter: Juan Pablo Alderuccio, MD, Sylvester Comprehensive Cancer Center at the University of Miami

A machine learning-driven, expression-based classifier was applied to high-grade B-cell lymphomas, identifying subgroups based on unique genomic and transcriptomic features. Validated using large datasets, this classifier could accurately and repeatedly divide aggressive lymphoma cases into diffuse large B-cell and Burkitt lymphoma-like groups based on gene expression. These findings illustrate the clinical applications of a gene expression-based classifier in identifying subsets of patients with aggressive lymphomas.

Research conducted in collaboration with the Sylvester Comprehensive Cancer Center at the University of Miami

Abstract: 3038
Title: Acalabrutinib with Rituximab is Highly Effective as a First Line Treatment for Older Patients with Mantle Cell Lymphoma
Date & time: Sunday, December 8 | 6:00 PM – 8:00 PM
Presenter: Preetesh Jain, MD, MBBS, PhD, DM, The University of Texas MD Anderson Cancer Center

A phase II clinical trial for mantle cell lymphoma patients receiving acalabrutinib and rituximab demonstrated a 94% overall response rate, with 90% of patients achieving complete remission. Correlative analyses with next-generation sequencing and immunoprofiling techniques revealed key features of the immune system’s dynamics and adaptive response following treatment with the combination.

Research conducted in collaboration with the MD Anderson Cancer Center

Abstract: 2982
Title: Use of Molecular Immune Signatures for Frontline Treatment Selection in Patients with Advanced Stage Follicular Lymphoma
Date & time: Sunday, December 8 | 6:00 PM – 8:00 PM
Presenter: Tony Zhuang, MD, MD Anderson Cancer Center

Transcriptomic analysis of pretreatment biopsies from advanced follicular lymphoma (FL) patients revealed that B-cell-associated gene signatures were linked with survival in FL patients receiving chemoimmunotherapy, while T-cell signatures correlated with improved outcomes in patients treated with lenalidomide and rituximab. These findings support the clinical application of transcriptomic profiling for FL patients.

Research conducted in collaboration with the MD Anderson Cancer Center

Abstract: 2958
Title: Circulating Tumor DNA Predicts Outcomes in Follicular Lymphoma: Analysis from a Prospective Study
Date & time: Sunday, December 8 | 6:00 PM – 8:00 PM
Presenter: Rahul Lakhotia, MBBS, National Institutes of Health

An ongoing prospective clinical trial examined the utility of serial circulating tumor DNA (ctDNA) for monitoring disease progression in follicular lymphoma patients. Detectable in over 90% of patients, baseline ctDNA levels correlated with FLIPI prognostic scores, total metabolic tumor volume and spontaneous regression. Serial ctDNA analysis accurately reflected tumor dynamics, demonstrating its value as a minimally invasive tool for tracking disease progression.

Research conducted in collaboration with the National Cancer Institute, University of South Florida, National Institutes of Health, Medical College of Georgia, University of Virginia, Adaptive Biotechnologies

Abstract: 4341
Title: Comprehensive Analysis of Malignant B-Cell Receptors Provides Insights into B-Cell Lymphoma Pathogenesis
Date & time: Monday, December 9 | 6:00 PM – 8:00 PM
Presenter: Evgeniia Alekseeva, PhD, BostonGene

Aimed at characterizing B-cell receptor (BCR) repertoires, BostonGene leveraged a blood-based approach to discover biomarkers capable of distinguishing B-cell lymphoma subtypes. Several features, including BCR isotype, IGVH somatic hypermutation rate and chain structure, were associated with specific subtypes, highlighting the potential for diagnostic biomarkers within BCR repertoires.

Abstract: 4377
Title: Radiation Therapy and Monocyte Activation in Large B-cell Lymphoma Patients Treated with CAR T-Cell Therapy
Date & time: Monday, December 9 | 6:00 PM – 8:00 PM
Presenter: Penny Fang, MD Anderson Cancer Center

A study evaluating CART-treated large B-cell lymphoma (LBCL) patients revealed radiation therapy reshapes the immune landscape, particularly by increasing monocyte levels, proliferation and cytotoxicity. This shift suggested radiation therapy is a viable bridging therapy for CART LBCL patients, promoting a suppressive immunotype linked with decreased toxicity and increased treatment response.

Research conducted in collaboration with the MD Anderson Cancer Center

Abstract: 4348
Title: Genomic and Spatial Proteomic Characterization of the Microenvironment of Diffuse Large B-Cell Lymphoma in African American Patients
Date & time: Monday, December 9 | 6:00 PM – 8:00 PM
Presenter: Michelle Lee, MD, PhD, Winship Cancer Institute of Emory University

A flagship multiomics study characterizing diffuse large B-cell lymphoma (DLBCL) in African American patients revealed the prevalence of immune-inflamed microenvironments, molecular alterations and distinct spatial cellular communities. These findings suggest tumor-specific factors may drive the disparity in survival outcomes and underscore the need for larger, more diverse clinical cohorts.

Research conducted in collaboration with the Winship Cancer Institute of Emory University, MD Anderson Cancer Center and Georgia Institute of Technology

Abstract: 4364
Title: Evaluating the clinical utility of comprehensive whole exome sequencing (WES) and RNA-seq for patients with lymphoma
Date & time: Monday, December 9 | 6:00 PM – 8:00 PM
Presenter: Dai Chihara, MD, PhD, MD Anderson Cancer Center

The BostonGene Tumor PortraitTM test, featuring integrated genomic and transcriptomic profiling, was used to generate clinically relevant findings, treatment recommendations and matched clinical trials for lymphoma patients. With a median turnaround time of just 8 days, the Tumor PortraitTM test’s robust findings and rapid turnaround time demonstrated the utility of integrated whole exome and transcriptome sequencing for lymphoma patients.

Research conducted in collaboration with the MD Anderson Cancer Center

Online only
Title: Tumor Genomics and Microenvironment Characterization of Diffuse Large B-Cell Lymphoma in Asian and Pacific Islander Patients

Integrated whole exome and transcriptome sequencing was performed on diffuse large B-cell lymphoma (DLBCL) tumors from the Hawaii Surveillance, Epidemiology, and End Results residual tissue repository, constituting an initial characterization of the DLBCL microenvironment in Asian and Pacific Islander patients, an under-represented population in prior studies. The findings suggest existing classification systems may not represent DLBCL heterogeneity in non-European populations, illustrating the need for studies with more diverse patient populations to reveal how ancestry, socioeconomic elements and environmental factors contribute to DLBCL pathobiology and outcomes.

Research conducted in collaboration with the Winship Cancer Institute of Emory University and MD Anderson Cancer Center

Title: Comprehensive Molecular Characterization of Monomorphic Post-Transplant Lymphoproliferative Disorder (PTLD)

Comparative analysis of Epstein-Barr virus (EBV)-positive and -negative post-transplant lymphoproliferative disorder (PTLD) patients with diffuse large B-cell lymphoma (DLBCL) uncovered distinct immune signatures and a higher prevalence of protumor elements among EBV-positive PTLD patients. When compared with immunocompetent-DLBCL, PTLD samples contained a higher fraction of plasma B-cells. While EBV-negative PTLDs largely resembled de novo DLBCLs, EBV-positive PTLDs were enriched with unique molecular features. These findings emphasize the role of EBV status in PTLD biology and associated clinical outcomes.

Research conducted in collaboration with the MD Anderson Cancer Center

In addition to the poster presentations, the abstracts have been published online in the November supplemental issue of "Blood."

On December 6, 2024 Indapta Therapeutics, Inc., a privately held clinical stage biotechnology company developing next-generation differentiated cell therapies for the treatment of cancer and autoimmune diseases, reported a collaboration with Sanofi to explore the combination of its allogeneic g-NK cell therapy, IDP-023, with Sanofi’s CD38-targeting monoclonal antibody, Sarclisa (isatuximab) (Press release, Indapta Therapeutics, DEC 6, 2024, View Source [SID1234648859]).

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Indapta has amended its ongoing phase 1 study of IDP-023, to add a cohort of IDP-023 in combination with Sarclisa for patients with relapsed/refractory multiple myeloma. Up to three dose levels of IDP-023 will be explored in the combination.

Under the agreement, Indapta will sponsor the clinical trial, Sanofi will supply Sarclisa, and the Parties will co-fund the trial. As presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Meeting, IDP-023 administered with or without interleukin-2 achieved a mean maximum reduction in serum M-protein or light chain of 73% in eight relapsed/refractory myeloma patients. This appears to be superior to prior trials of natural killer cells in myeloma.

"With its deep experience in multiple myeloma and pipeline of products in hematologic malignancies and immunologic disorders, Sanofi is an excellent partner to help Indapta develop our differentiated natural killer cell product," said Robert Sikorski, Indapta’s Chief Medical Officer. "We look forward to a close collaborative relationship and leveraging Sanofi’s expertise in the design of potential subsequent studies."

Indapta’s Proprietary g-NK Cell Therapy

Indapta’s universal, allogeneic NK cell therapy platform consists of a potent subset of naturally occurring NK cells, known as "g minus" NK cells, or "g-NK" cells. g-NK cells arise from epigenetic changes resulting from exposure to cytomegalovirus (CMV). To generate IDP-023, Indapta preferentially expands g-NK cells from healthy donors, with low donor to donor variability. IDP-023 has several differentiated mechanisms of killing target cells without the need for genetic engineering, including highly robust antibody-dependent cell mediated cytotoxicity (ADCC), the targeting of HLA-E expressing cells via the NKG2C receptor, and the inherent anti-viral activity of g-NK cells.

Indapta’s g-NK can release dramatically more immune activating cytokines and cell-killing compounds than conventional NK cells. In preclinical studies, IDP-023 has demonstrated more potent and durable antitumor activity when combined with cancer targeting monoclonal antibodies as compared to conventional NK cells. (Bigley et al., Blood Advances 2021, View Source). g-NK cells in combination with a B cell targeting antibody can also deplete normal B cells from healthy donors or patients with autoimmune disease.

On December 6, 2024 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a clinical-stage biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, reported the closing of its previously announced underwritten public offering of 6,150,793 shares of its common stock, which includes the exercise in full by the underwriters of their option to purchase up to 833,333 additional shares of common stock, at a public offering price of $63.00 per share and pre-funded warrants to purchase 238,095 shares of common stock at a price of $62.999 per pre-funded warrant, which represents the per share price for the common stock less the $0.001 per share exercise price for each such pre-funded warrant (Press release, Janux Therapeutics, DEC 6, 2024, View Source [SID1234648858]). The aggregate gross proceeds to Janux from the offering, before deducting underwriting discounts and commissions and other estimated offering expenses and excluding the exercise of any pre-funded warrants, were approximately $402.5 million.

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BofA Securities, TD Cowen, Stifel, Cantor and William Blair acted as joint book-running managers for the offering. Wedbush PacGrow, LifeSci Capital, BTIG and Jones acted as co-managers for the offering.

The Company intends to use the net proceeds from the offering to advance clinical development of its internal product pipeline and for general corporate purposes.

The securities were offered by the company pursuant to a Registration Statement on Form S-3 filed with the Securities and Exchange Commission (SEC) that became automatically effective upon filing. A final prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC and are available on the SEC’s website, located at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained from BofA Securities, NC1-022-02-25, 201 North Tryon Street, Charlotte, North Carolina 28255-0001, Attention: Prospectus Department, or by email at [email protected]; TD Securities (USA) LLC, 1 Vanderbilt Avenue, New York, New York 10017, by telephone at (855) 495-9846 or by email at [email protected]; Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at (415) 364-2720, or by email at [email protected]; Cantor Fitzgerald & Co., Attention: Capital Markets, 110 East 59th Street, 6th Floor, New York, New York 10022, or by email at [email protected]; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, Illinois 60606, by telephone at (800) 621-0687 or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On December 6, 2024 UP Oncolytics, Inc’s reported that program to develop Zika Virus strains to treat glioblastoma has achieved two major milestones (Press release, UP Oncolytics, DEC 6, 2024, View Source [SID1234648856]).

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SBIR Fast Track Grant from the NINDS
Orphan Drug Designation from the FDA
"In the nearly 40 years I have been caring for glioblastoma patients, prognosis hasn’t changed. By using multiple resistance mechanisms, including the blood brain barrier, glioblastoma can defeat traditional chemotherapy, radiation, targeted therapy, and immunotherapy. Something needed to change," commented Richard A Rovin, MD, CEO and co-founder of UP Oncolytics. That change comes in the form of an oncolytic virus–a naturally occurring or genetically modified virus that can preferentially enter and kill cancer cells.

According to Parvez Akhtar, PhD, CSO and co-founder, "The Zika Virus strains we are testing have the unique combination of safety, efficacy, and the ability to cross the blood brain barrier."

The SBIR award and Orphan Drug Designation come after years of extensive pre-clinical testing led by Dr Akhtar. "Both of these validate the scientific work we have done and the company we have built," observed Dr Akhtar.

Dr Rovin: "I think the SBIR and Orphan Designation show that the NINDS and FDA understand the urgent and unmet need to develop novel, effective treatments for GBM. Parvez and I are humbled and excited to bring Zika Virus treatment to clinical trial."

About the SBIR Fast Track Grant

The Small Business Innovation Research (SBIR) program through the NIH provides seed funding to early-stage small businesses so that they can bring their innovation from "bench to bedside". The Fast-Track process allows companies to submit both Phase I and Phase II in one application for review. The Fast-Track mechanism minimizes the funding gap between phases but requires a fully developed Phase II application/plan at the time of submission. In 2023, fewer than 20% of applicants received funding.

About Orphan Drug Designation

The FDA Office of Orphan Products Development (OOPD) "supports and advances the development and evaluation of new treatments for rare diseases." A rare disease affects fewer than 200,000 people in the United States. The OOPD can grant Orphan Status to a drug or biologic for the treatment of a rare disease. An Orphan Drug Designation provides benefits to the sponsor including tax benefits, waiver of fees, and seven-year market exclusivity after approval.

About Gliomas

Gliomas are considered primary tumors as they originate in the brain. They account for 23% of all primary brain tumors and 81% of malignant primary brain tumors. Glioblastoma (GBM), the most aggressive brain cancer, accounts for 61% of gliomas. (Price et al., 2024a)

There are approximately 13,000 new cases of GBM each year (Price et al., 2024b) and about 25,000 people in the United States are living with GBM. (Neff et al., 2023)

In the United States, the median survival for patients with GBM is 9 months and the 5-year survival rate is 7.1%. (Price et al., 2024a)