Veracyte Announces Expanded Availability of Decipher Prostate Test to Patients with Metastatic Prostate Cancer

On April 25, 2025 Veracyte, Inc. (Nasdaq: VCYT), a leading genomic diagnostics company, reported the launch of its Decipher Prostate Metastatic Genomic Classifier for use in patients whose prostate cancer has spread beyond the primary tumor (Press release, Veracyte, APR 25, 2025, View Source [SID1234652150]). The Decipher Prostate test, already widely used for patients with localized disease, is now the only gene expression test available and covered by Medicare to inform treatment decisions for patients across the full continuum of prostate cancer risk.

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Veracyte has begun making the Decipher Prostate Metastatic test available to select clinical sites through an early access program and will begin taking orders for the test more broadly in June 2025.

Prostate cancer is the second-leading cause of cancer deaths among men in the United States and the rate of men diagnosed with advanced disease has been growing in recent years.1 Veracyte estimates that approximately 10% (or about 30,000) of all prostate cancers diagnosed annually in the United States are metastatic.2

"A number of treatment options are now available to increase survival for patients whose prostate cancer has metastasized," said Elai Davicioni, Ph.D., Veracyte’s medical director for Urology. "Until now, however, clinicians had limited ways to determine which of these patients will likely benefit from these therapies and which will not and may thus avoid their toxic side effects. We believe the Decipher Prostate Metastatic test will provide an important new tool to help clinicians make more-informed treatment recommendations for their patients with metastatic prostate cancer."

The Decipher Prostate test’s clinical validity and clinical utility for use in patients with metastatic prostate cancer have been demonstrated in multiple, prospective, Phase 3 clinical studies.3-6 These studies have shown that such patients with high Decipher scores are likely to have more-aggressive tumor biology compared to those with lower scores, informing the absolute benefit from treatment intensification. These findings build upon extensive data already established for the Decipher Prostate test’s use in patients with localized prostate cancer, where it is the only gene expression test to achieve "Level I" evidence status in the most recent NCCN Guidelines* for prostate cancer.

"Our expansion into metastatic prostate cancer underscores the power of the Veracyte Diagnostics Platform to uncover novel insights that can enable us to further help patients," said Philip Febbo, M.D., Veracyte’s chief scientific officer and chief medical officer.

About Decipher Prostate

The Decipher Prostate Genomic Classifier is a 22-gene test, developed using RNA whole-transcriptome analysis and machine learning, that helps inform treatment decisions for patients across the full spectrum of prostate cancer. The test is performed on biopsy or surgically resected samples and conveys the aggressiveness of the cancer. For patients with localized or regional prostate cancer, the Decipher score indicates a patient’s risk of metastasis, helping to determine treatment timing and intensity. For patients with metastatic prostate cancer, the Decipher score indicates the likelihood of cancer progression and survival benefit with treatment intensification. Armed with this information, physicians can better personalize their patients’ care. The Decipher Prostate test’s performance and clinical utility has been demonstrated in over 85 studies involving more than 200,000 patients. It is the only gene expression test to achieve "Level I" evidence status and inclusion in the risk-stratification table in the most recent NCCN Guidelines* for prostate cancer.

City of Hope Scientists Present Leading-Edge Research at American Association for Cancer Research (AACR) Annual Meeting

On April 25, 2025 Researchers with City of Hope, one of the largest and most advanced cancer research and treatment organizations in the U.S. with its National Medical Center named top 5 in the nation for cancer by U.S. News & World Report, reported that it will present more than 74 chaired, plenary, educational, minisymposium, poster and other sessions on innovative clinical trial results, breakthrough diagnostic techniques and advances in treatment options at the AACR (Free AACR Whitepaper) Annual Meeting, which started April 25 and ends April 30 in Chicago (Press release, City of Hope, APR 25, 2025, View Source [SID1234652167]).

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In addition to City of Hope’s innovative research being presented throughout the meeting, David W. Craig, Ph.D., professor and founding chair of the Department of Integrative Translational Sciences within Beckman Research Institute of City of Hope, is chairing the final plenary session of the conference, "Opportunities in Predictive Oncology," on Wednesday from 8 to 10 a.m. CT. He will also present an educational session on using a biological analysis approach called multiomics to investigate the unique genetic makeup of different cell populations in solid tumors on Friday from 4:46 to 5:06 p.m. CT.

On Saturday from 10:00 to 11:30 a.m. CT, Michael A. Caligiuri, M.D., former president of City of Hope National Medical Center and professor in the Department of Hematology & Hematopoietic Cell Transplantation, will chair a session on advances in the application of natural killer (NK) cells and present on "Innate immune lymphocytes, including NK cells." He will also chair "Academic Entrepreneurship: Getting Your Discovery to Patients, Part 1—Liftoff" on Saturday from 8:00 to 9:30 a.m. CT, which will help define the steps required for translating research from the bench to the bedside.

As part of a session on advances in diagnostics and therapeutics, Hope Rugo, M.D., who recently joined City of Hope as director of its Women’s Cancers Program, will talk about new findings in managing toxicities from antibody-drug conjugates on Monday from 1:25 to 1:45 p.m. CT. Dr. Rugo will also serve as a discussant at the Clinical Trials Plenary Session on Biologics and T-cell Engagers on Tuesday from 10:15 a.m. to 12:15 p.m. CT.

Highlights of City of Hope research presented at the AACR (Free AACR Whitepaper) conference include:

Phase 3 clinical trial shows promising results for novel immunotherapy

Cancer of the nasopharynx, or the upper part of the throat that plays a crucial role in breathing and swallowing, is relatively rare. However, in certain regions like China and North Africa, the disease is much more common. To combat recurrent or metastatic nasopharyngeal carcinoma, a new immunotherapy drug has been tested in combination with standard chemotherapy.

Data from a phase 3 clinical trial of the medication called penpulimab resulted in the Food and Drug Administration (FDA) approving its use this past week in combination with cisplatin or carboplatin and gemcitabine for the first-line treatment of adult patients with recurrent or metastatic nasopharyngeal carcinoma. The FDA also approved penpulimab as a single agent for similar patients with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy.

Medical oncologist Aditya Shreenivas, M.D., M.S. will present the supporting data from "Penpulimab versus placebo in combination with chemotherapy as first-line treatment for recurrent or metastatic nasopharyngeal carcinoma: A global, multicenter, randomized, double-blind, phase 3 trial (AK105-304)" during a clinical trials minisymposium session on Sunday from 3:50 to 4:00 p.m. CT.

Penpulimab is an anti-PD-1 inhibitor, a type of treatment that helps the immune system recognize and attack cancer. Building on previous studies that have shown that the combination of PD-1 inhibitors with chemotherapy exhibits promising efficacy as a first-line treatment for Asian patients, phase 3 of the trial included 291 patients from both Asian and non-Asian countries.

Patients received either a combination of penpulimab and standard chemotherapy or a placebo plus chemotherapy. The study’s findings showed that patients who had penpulimab added to their treatment plan had their cancer controlled for 9.63 months on average, compared to just 7.00 months for those who did not. This represents a 55% reduction in the risk of disease progression. The researchers also found that the combination of penpulimab with chemotherapy had a manageable safety profile with tolerable side effects.

"What makes penpulimab unique is that it is an anti-PD-1 antibody with a modified structure designed to potentially improve efficacy while reducing immune-related side effects," said Dr. Shreenivas, who led at the study at City of Hope, one of 46 trial sites worldwide. "This research supports use of penpulimab plus chemotherapy as a new first-line treatment option for patients with recurrent or metastatic nasopharyngeal carcinoma."

He also says that since penpulimab has already been approved in China for some other cancers, like relapsed or refractory classic Hodgkin’s lymphoma and metastatic squamous non-small-cell lung cancer, this study might lead to expanded approvals for penpulimab in additional countries.

According to Dr. Shreenivas, future findings from the clinical trial could include explorations of how to identify which patients benefit most from this treatment, as the researchers analyzed various subgroups including those with liver metastases, different protein expression levels, and different DNA levels of Epstein-Barr virus, which is considered a strong risk factor for nasopharyngeal carcinoma.

The clinical trial was sponsored by Akeso, a Chinese biopharmaceutical company that developed penpulimab.

Researchers use innovative technologies to learn more about treatment responses on ovarian, prostate and breast cancer patients

Ovarian cancer

Not all patients with aggressive, high-grade serous ovarian cancer — the most common type of the disease — respond the same way to immunotherapy. To elucidate differences that might help tailor immunotherapy strategies for individual patients, a group of researchers from City of Hope and USC led by Jing Qian, a doctoral student in the lab of John D. Carpten, Ph.D., City of Hope’s chief scientific officer, Irell & Manella Cancer Center Director’s Distinguished Chair and Morgan & Helen Chu Director’s Chair of the Beckman Research Institute, sought to characterize the cancer and immune cells within the tumor environments of patients with different responses to treatment.

By using spatial transcriptomic technologies to map interactions between cancer and immune cells, the team was able to reveal differing immune cell behavior and composition in tumors with varied responses to immunotherapy. Spatial transcriptomic methods can provide valuable insights into gene expression within tissue and the technology is an area of rapid development in medical research.

"In the future, this approach could help identify patients who are more likely to benefit from immunotherapy and uncover new targets to improve treatment for those who don’t respond," said Qian. "Ultimately, it pushes the field closer to precision immuno-oncology in high-grade serous ovarian cancer."

Next, the team plans to use cell models and additional patient cohorts to explore whether targeting specific immune cell types can improve responses to immunotherapy in ovarian cancer.

Qian will present "Spatial transcriptomics reveals differences in the tumor and immune microenvironment of high-grade serous ovarian cancers with differing responses to immune checkpoint inhibitors" as a late breaking poster session on Sunday from 2:00 to 5:00 p.m. CT.

Prostate cancer

Recent studies suggest that a complex interplay between ethnicity and disease biology could influence how metastatic hormone sensitive prostate cancer (mHSPC) behaves in different patients. To investigate differences in tumor tissue from patients of Hispanic background compared to those not of Hispanic background, a team of City of Hope researchers led by Tanya Barauskas Dorff, M.D., professor in the Department of Medical Oncology & Therapeutics Research, borrowed pre-treatment prostate biopsy specimens from an existing clinical trial.

Using digital spatial profiling, a technology used to study gene and protein expression in tissues, the researchers were able to interrogate the tumor microenvironment from different patients with mHSPC with greater granularity and depth. They found a difference in expression of certain proteins like Foxp3, PARP and STING, all of which are targets of certain cancer medications, between Hispanic and non-Hispanic patients.

"This type of exploration in tissue samples from patients of diverse backgrounds may help uncover factors that could account for differences in treatment response," said Dr. Dorff. "As we complete the testing on additional tissue samples, we hope to compare results against treatment outcomes, to see a clearer signal emerge identifying candidate proteins for future validation."

She said the team hopes to increase the variety of samples being tested and compare their findings to clinical outcomes from the trial, which should become available early 2026.

Peter Zang, M.D., a hematology & oncology fellow and first author on "Digital spatial profiling with GeoMx to identify differential protein expression in Non-Hispanic/Latino and Hispanic/Latino Patients with metastatic hormone sensitive prostate cancer," will present the team’s poster abstract on Tuesday from 2:00 to 5:00 p.m. CT.

Breast cancer

A poster presentation by Sydney Grant, a postdoctoral fellow, and Aritro Nath, Ph.D., assistant professor in the Department of Medical Oncology & Therapeutics Research, on Tuesday from 9:00 a.m. to 12:00 p.m. CT, "Integrating multimodal data with survival-based variational autoencoders to predict recurrence-free survival in breast cancer," will highlight a powerful new AI-based approach the team developed to predict recurrence-free survival in breast cancer patients.

By enhancing existing generative AI models to process a broader range of real-world patient data, which extend far beyond traditional biomarkers currently used in the clinic, Grant and Nath established an approach that enables more accurate and personalized predictions about cancer patient outcomes.

"This work could lead to new clinical tests used at the time of diagnosis to guide treatment decisions and bring us closer to truly personalized care," said Dr. Nath. "For example, these tools would help doctors identify which patients need more aggressive therapy and which patients could avoid overtreatment and unnecessary side effects."

The team is now working to extend their models to predict which types of treatment or drug classes will work best for individual patients, with the goal of guiding therapy selection in the clinic using AI.

New technology identifies unique genetic changes in early-onset colorectal cancer among Hispanic and Latino patients

Colorectal cancer is the second leading cause of cancer-related deaths and cases among younger patients are rising, particularly among Hispanic and Latino populations. A new study from the lab of Enrique Velazquez Villarreal, M.D., Ph.D., M.P.H., M.S., assistant professor in the Department of Integrative Translational Sciences, has found that early-onset colorectal cancer in Hispanic and Latino patients has unique genetic changes that help explain how the cancer grows and spreads.

"This is the first study to look closely at the genetics of colorectal cancer in Hispanic and Latino patients from the Los Angeles area, a group that’s often left out of cancer research," said postdoctoral fellow Francisco (Paco) Carranza, who is first author on the study. "By understanding how colorectal cancer affects different populations, especially those who are often overlooked, we can help create better and more targeted treatments."

To find genetic changes, the research team used DNA and RNA sequencing plus a powerful new technology called 10x Genomics Visium that let them see which genes are turned on or off in specific parts of a tumor. This helped them better understand how cancer cells interact with the immune system and how the disease behaves in this patient population.

"This kind of research brings us closer to making sure all patients—no matter their background—have access to the best possible care and the same chance at successful outcomes," said Dr. Velazquez Villarreal, whose research is focused on addressing colorectal cancer health disparities in Hispanic and Latino communities.

Next, Carranza and his collaborators plan to move this research even closer to patient care by using an advanced version of the 10x Genomics Visium platform that allows researchers to look at tumors one cell at a time to give even more detailed information that could lead to better tailored therapies and help design new clinical trials.

Carranza will discuss the team’s work during a minisymposium session called "Multi-omics analysis of MYC gene and WNT signaling pathway alterations in early-onset colorectal cancer in Hispanic/Latino patients, enhanced with spatial transcriptomics approaches" on Monday from 2:35 to 2:50 p.m. CT.

AI tool developed at City of Hope makes precision medicine more accessible and inclusive

Dr. Velazquez Villarreal will also present a poster on his lab’s development of a new precision medicine AI tool that addresses key challenges in the integration of different data sets to promote better equity in cancer research.

The new tool, called the Precision Medicine Artificial Intelligence Agent (PM-AI), is a conversational AI system that can understand plain-language questions and automatically run complex data analyses. It combines clinical information, genetic data, and social factors like income or access to care to make it easier for scientists to study cancer in a more complete and inclusive way.

"By making it easier to analyze large and complex datasets, PM-AI can help researchers and doctors discover which treatments work best for different groups of people," said Dr. Velazquez Villarreal. This means patients could one day receive more personalized care based on their genetics, health history, and social conditions—leading to better outcomes for everyone, especially underserved communities."

He said the research team plans to keep expanding PM-AI’s capabilities by applying it to more types of cancer and integrating even more kinds of data. The goal is to support clinical decisions, help design new studies and ultimately make precision medicine more accessible for all patients.

The study, "PM-AI agent: A conversational artificial intelligence system for precision medicine and advancing health equity through integrative clinical, genomic and social determinants of health data analysis," is the first accepted by AACR (Free AACR Whitepaper) to use a cutting-edge technology known as an AI-driven conversational agent and will be presented during a poster session on Sunday from 2:00 to 5:00 p.m. CT.

Combination treatment found to overcome therapy resistance in ER+ breast cancer

In patients with estrogen receptor-positive (ER+) breast cancer — the most common type of the disease — 30-50% eventually develop resistance to primary endocrine therapy and progress to advance disease. While cell cycle inhibitor therapies help slow down metastatic ER+ breast cancer, patients often become resistant to these treatments, too.

Now, a new study by City of Hope researchers has revealed that ER+ breast cancers resistant to cell cycle inhibitors undergo dynamical rewiring of both apoptosis (or cell death) pathways and proliferative pathways, which regulate cell division and growth, to survive. To overcome this resistance, the team looked for add-on therapies that target growth factor receptors. Their goal was to find a combination that provides a blockade of proliferative pathways with sustained upregulation of apoptosis pathways to maintain treatment effectiveness.

Using leading-edge integration of short- and long-term biological experimentation cell models with mathematical and computational analysis, they discover a new proposed combination therapy of ribociclib (a cell cycle inhibitor) plus afatinib (a growth factor inhibitor) that can durably control breast cancer cell growth over time.

"Although cell growth-targeted drugs have improved outcomes for hormone-dependent breast cancer patients, resistance remains a major clinical challenge limiting their long-term benefit," said Andrea Bild, Ph.D., professor in the Department of Medical Oncology & Therapeutics Research and senior author of the study. "Our research presents a novel strategy to enhance the durability and effectiveness of current treatments."

Next, the research team aims to translate their findings for patient benefit in the clinic. To do so, Dr. Bild said they will expand their use of temporal computational and mathematical modeling to monitor molecular changes over time and identify dynamic biomarkers that can guide future treatment strategies.

Kimya Karimi, a doctoral student in Dr. Bild’s lab, will present the team’s study "Overcoming intrinsic mechanisms of cell cycle inhibitor resistance in estrogen receptor-positive (ER+) breast cancer" during a minisymposium on Tuesday from 3:05 to 3:20 p.m. CT.

Onc.AI Announces Presentation of Breakthrough-Designated AI Model Evaluated in Clinical Trial Data at AACR 2025

On April 25, 2025 Onc.AI, a digital health company developing advanced AI-driven clinical management solutions for oncology, reported that findings from a recent collaboration with global biopharma company GSK using Onc.AI’s FDA breakthrough-designated Serial CTRS AI model will be presented at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting (Press release, Onc AI, APR 25, 2025, View Source [SID1234652184]). The study externally evaluated Serial CTRS in GSK’s GARNET Phase I clinical trial (NCT02715284) Cohort E, that enrolled patients with advanced non-small cell lung cancer (NSCLC) treated with dostarlimab, GSK’s anti-PD-1 checkpoint inhibitor.

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Results, highlighted in a poster presentation during the Predictive Biomarkers 2 session (poster #21, April 27, 2025, 2-5pm), demonstrated that the Serial CTRS biomarker, leveraging routine CT imaging without manual annotations, improved prediction of overall survival (OS) compared to traditional surrogates including RECIST 1.1 response criteria and tumor volume. The analysis was conducted through an independent and blinded retrospective validation study carried out by GSK. In particular, Serial CTRS showed the ability to distinguish patients with intermediate versus high probabilities of 12-month OS (HR: 2.91, 95% CI: 1.16–7.31), outperforming RECIST 1.1 (HR: 1.34, 95% CI: 0.57–3.13) and tumor volume change assessments (HR: 1.00, 95% CI: 0.43–2.34). This enhanced predictive performance supports Onc.AI’s commitment to establishing Serial CTRS as a new standard for automated, AI-based imaging endpoints in the early assessment of treatment response, seamlessly integrating into standard imaging workflows across diverse therapeutic regimens.

Key findings include:

Serial CTRS improved discrimination between intermediate and high probabilities of overall survival compared to RECIST 1.1 and tumor volume changes.
Serial CTRS remained a significant predictor of overall survival after adjusting for known prognostic factors, such as age, baseline tumor volume, and PD-L1 Tumor Proportion Score (TPS).
"This important milestone for Serial CTRS builds on a recent breakthrough-designation from FDA," said Akshay Nanduri, CEO of Onc.AI. "The success of this validation study and ongoing continued collaboration with GSK reflects the strength and robustness of model performance. This can only be achieved using advanced Deep Learning combined with methods for harmonizing diverse imaging data and the breadth of our training data. Onc.AI’s high-quality data on thousands of patients has been sourced from dozens of healthcare systems representing hundreds of clinics across the United States and other international cancer centers, ensuring unparalleled diversity in training, test and validation."

"As the past head of multiple Phase I clinics at top cancer centers, I believe that Onc.AI’s innovation with Serial CTRS could transform the pharma clinical development process from Phase I to Phase III studies," said George R. Simon, MD, FACP, FCCP, Vice President of Oncology at OhioHealth.

ALX Oncology Reports ASPEN-03 and ASPEN-04 Phase 2 Trials Evaluating Evorpacept with a Checkpoint Inhibitor for the Treatment of Head and Neck Cancers Did Not Meet Primary Endpoints

On April 25, 2025 ALX Oncology Holdings Inc., ("ALX Oncology" or the "Company") (Nasdaq: ALXO), a clinical-stage biotechnology company advancing a pipeline of novel therapies designed to treat cancer and extend patients’ lives, reported topline data from its Phase 2 ASPEN-03 and ASPEN-04 clinical trials (Press release, ALX Oncology, APR 25, 2025, View Source [SID1234652135]). The company’s investigational CD47-blocker evorpacept, when added to Merck’s (known as MSD outside of the US and Canada) anti-PD-1 therapy, KEYTRUDA (pembrolizumab) with or without chemotherapy, did not meet the primary endpoints in the ASPEN-03 and ASPEN-04 trials of improved objective response rates (ORR) as compared to historical controls of pembrolizumab alone and pembrolizumab with chemotherapy, respectively, as a first-line treatment in patients with advanced head and neck squamous cell carcinoma (HNSCC). The combination of evorpacept and pembrolizumab with or without chemotherapy in ASPEN-03 and ASPEN-04 demonstrated a manageable safety profile and was consistent with what has been previously reported for pembrolizumab and chemotherapy in this setting. Although the company will no longer pursue evorpacept in combination with pembrolizumab in HNSCC, multiple clinical trials of evorpacept in combination with anti-cancer antibodies will continue based on established proof-of-concept.

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Evorpacept blocks the ‘don’t eat me’ signal transmitted by CD47 on the surface of cancer cells that these cells use to evade detection by the immune system. As its primary mechanism of action, evorpacept is uniquely designed to stimulate macrophages to selectively attack cancer cells and not healthy cells, when combined with active anti-cancer antibodies. This mechanism has translated into durable clinical responses and a well-tolerated safety profile in HER2-positive gastric and HER2-positive breast cancer clinical trials. To further explore the benefit in this setting, evorpacept is currently being evaluated in combination with various anti-cancer antibodies in colorectal cancer, breast cancer, non-Hodgkin lymphoma and multiple myeloma.

In the ASPEN-03 and ASPEN-04 clinical trials, evorpacept was combined with pembrolizumab with or without chemotherapy to investigate a second and distinct mechanism of action. This discrete approach explored the concept that evorpacept may enhance T-cell priming by activating dendritic cells and stimulating the adaptive immune system. The trial outcomes were not sufficiently supportive of advancing evorpacept in combination with pembrolizumab in HNSCC into a registrational trial.

"While there were encouraging trends in ASPEN-03 in ORR versus the historical and internal control, we’ve decided not to pursue evorpacept and pembrolizumab in head and neck cancer in light of our prioritization of the more established anti-cancer antibody combination program based on multiple positive studies," said Alan Sandler, M.D., Chief Medical Officer at ALX Oncology. "We are disappointed that these studies did not meet their primary endpoints, most importantly for the patients for whom current standard-of-care treatment approaches fall short, and we thank all who participated in the trials."

"Moving forward, we are continuing to rapidly advance our clinical program combining evorpacept with anti-cancer antibodies supported by robust clinical data across trials in multiple tumor types. Evorpacept has demonstrated response rates and durability beyond what is expected from standard of care across several studies when combined with HERCEPTIN, zanidatamab and RITUXAN," said Jason Lettmann, Chief Executive Officer at ALX Oncology. "Based on the positive data and strong mechanistic rationale, we maintain our confidence in the evorpacept clinical development program and intend to deliver on that promise with additional clinical data in breast cancer and colorectal cancer in the near-term. With evorpacept and ALX2004, our novel EGFR-targeted antibody-drug conjugate, we continue our commitment to bringing forth meaningful therapies for patients living with cancer."

Detailed findings from the ASPEN-03 and ASPEN-04 trials will be submitted to a future medical meeting.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About ASPEN-03 and ASPEN-04 Clinical Trials
ASPEN-03 and ASPEN-04 are randomized, multi-center, international Phase 2 trials evaluating evorpacept, ALX Oncology’s investigational CD47-blocking therapeutic that uniquely combines a high-affinity CD47-binding domain with an inactivated proprietary Fc domain, in patients with metastatic or unresectable, recurrent HNSCC who have not yet been treated for their advanced disease. The ASPEN-03 trial (NCT04675294) is evaluating evorpacept in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) against pembrolizumab alone for the treatment of patients whose HNSCC is PD-L1 positive. The ASPEN-04 trial (NCT04675333) is evaluating evorpacept in combination with pembrolizumab and chemotherapy against pembrolizumab and chemotherapy alone in patients with HNSCC, regardless of PD-L1 status. Patient characteristics in the trials (N=346) were generally well-balanced across arms. The primary endpoints for the ASPEN-03 and ASPEN-04 trials is ORR compared to historical control. Key secondary endpoints for both trials are safety, duration of response (DOR), progression-free survival (PFS) and overall survival (OS).

About Head and Neck Squamous Cell Carcinoma (HNSCC)
HNSCC is a serious and life-threatening disease that originates in the squamous cells lining the mucosal surfaces of the head and neck, including the mouth, throat, voice box, sinuses and nasal cavity. HNSCC is the seventh most common cancer worldwide and the incidence of HNSCC is expected to increase 30% by 2030. Despite current standard-of-care therapies and recent advancements in diagnosis and treatment, people with HNSCC face a poor prognosis, particularly when diagnosed at advanced stages. Further, the survival rate for HNSCC has only modestly improved in recent years, even with the availability of new treatment modalities, underscoring a need for improved therapeutics.

About Evorpacept
ALX Oncology’s lead therapeutic candidate, evorpacept, is a highly differentiated potential best- and first-in-class CD47 checkpoint inhibitor and one of the most advanced checkpoint inhibitors to target and activate the innate immune system. Evorpacept was intentionally designed to maximize the clinical potential of blocking CD47, while reducing the toxicities associated with previous approaches to CD47 blockade. In clinical studies across a wide spectrum of tumor types in more than 700 patients to date, evorpacept has demonstrated potential to enhance the therapeutic activity of many of the most important cancer therapies available today, contributing an additional, differentiated immuno-oncology mechanism. Based on this potential, ALX Oncology is advancing a robust clinical program evaluating evorpacept in a wide range of cancer indications, prioritizing its combination with anti-cancer antibodies in breast, gastric and colon cancers. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to evorpacept for the second-line treatment of patients with HER2-positive gastric or GEJ carcinoma. Additionally, both the FDA and European Commission have granted Orphan Drug Designation for this indication.

Verastem Oncology Announces $75 million Private Placement

On April 25, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported that it has entered into a securities purchase agreement with certain institutional and accredited investors for a private placement of approximately $24 million of shares of its common stock at a price of $7.00 per share and, in lieu of common stock to certain investors, $51 million of pre-funded warrants to purchase shares of its common stock at a price of $6.9999 per pre-funded warrant (Press release, Verastem, APR 25, 2025, View Source [SID1234652151]). The exercise price of each pre-funded warrant will equal $0.0001 per share. Verastem expects to receive gross proceeds from the offering of approximately $75 million, before deducting placement agent fees and other offering expenses.

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The private placement was led by RTW Investments, with participation from other new and existing investors, including BVF Partners, Nantahala Capital, Octagon Capital, OrbiMed and Stonepine Capital Management.

The private placement is expected to close on or about April 28, 2025, subject to the satisfaction of customary closing conditions.

Proceeds from the financing are expected to fund the potential launch of avutometinib and defactinib in recurrent low-grade serous ovarian cancer, continued clinical research and development of product candidates including VS-7375, and for working capital and other general corporate purposes.

Guggenheim Securities is acting as the lead placement agent for the private placement. RBC Capital Markets, BTIG, Mizuho and B. Riley Securities are acting as co-placement agents for the private placement (together with Guggenheim Securities, the "Placement Agents"). The Company has agreed to pay customary placement fees and reimburse certain expenses of the Placement Agents.

The securities to be sold in the private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other applicable jurisdictions’ securities laws, and may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. Verastem has agreed to file a registration statement with the United States Securities and Exchange Commission (the "SEC") registering the resale of the shares of common stock issued in the private placement and the shares of common stock issuable upon the exercise of the pre-funded warrants issued in the private placement, no later than 30 days after the closing of the private placement.

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 offer, solicitation or sale of these securities in any jurisdiction in which such offer, solicitation or sale would be unlawful. Any offering of the securities under the resale registration statement will only be made by means of a prospectus.