On May 30, 2025 Onc.AI, a digital health company developing AI-powered oncology clinical management solutions, reported that new validation study results from research collaborations with Pfizer, Baylor Scott & White and the University of Rochester Medical Center will be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, held May 30–June 3, 2025, in Chicago, IL (Press release, Onc AI, MAY 30, 2025, View Source [SID1234653537]).

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Onc.AI’s poster presentation showcases its FDA-breakthrough designated deep learning radiomics model, Serial CTRS, which evaluates changes across routine CT scans over time to predict overall survival in late-stage non–small cell lung cancer (NSCLC) and other solid tumor types. In collaboration with Baylor Scott & White and Pfizer, Serial CTRS has demonstrated:

Superior prediction of overall survival (OS): Hazard ratios (HRs) for OS improvement and stratification exceed those of the conventional imaging approach (RECIST 1.1).
Generalizability across real-world and clinical trial cohorts: Robust performance in both routine real-world datasets and a Pfizer-sponsored PD-1 checkpoint inhibitor trial.
Actionable insights for early treatment adaptation: Dynamic monitoring identifies non-responders months before conventional criteria would signal poor prognosis.
At the ASCO (Free ASCO Whitepaper) Innovation Hub (IH13), Onc.AI will share latest results from its pipeline of deep learning radiomic models to customers and partners spanning medical oncologist investigators and biopharma companies looking to accelerate oncology clinical development.

Program Highlights

Poster Presentation:

Abstract #253138: Validation of Serial CTRS for Early Immunotherapy Response Prediction in Metastatic NSCLC – View Source

Presenter: Ronan Kelly, MD, Baylor Scott & White
Date & Time: June 1, 2025; 9:00 am–12:00 pm CDT
Location: Hall A, Poster Board 325
Abstract #251996: Retrospective Single-Institution Application of a Deep Learning–Based Radiomic Score in Metastatic NSCLC: Potential Impact on First-Line Treatment Decisions – View Source

Lead Author: Nicholas Love, MD, University of Rochester
Abstract #245837: Image Harmonization for PD-(L)1 Immune Checkpoint Inhibitor Response Prediction Using Deep Learning Radiomic Features in Advanced NSCLC – View Source

Lead Author: Taly Gilat-Schmidt, PhD, Onc.AI
"These strong validation study results spanned both RWD and a pharma-sponsored clinical trial. Serial CTRS could represent a high-potential tool for medical oncologists and for optimizing pharma clinical development," said Dr. Ronan Kelly, MD, Director of Oncology at the Charles A. Sammons Cancer Center, Baylor University Medical Center, Dallas Texas

"Our retrospective study highlights how Onc.AI’s Deep Learning Radiomic baseline score can be extremely helpful to medical oncologists as a prognostic marker for first line mutation negative NSCLC patients," added Arpan Patel, MD and Associate Professor of Medical Oncology at the University of Rochester Medical Center.

On May 30, 2025 Pfizer Inc. (NYSE: PFE) reported statistically significant and clinically meaningful survival results from the Phase 3 BREAKWATER trial evaluating BRAFTOVI (encorafenib) in combination with cetuximab (marketed as ERBITUX) and mFOLFOX6 (fluorouracil, leucovorin, and oxaliplatin) in patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation (Press release, Pfizer, MAY 30, 2025, View Source [SID1234653536]). These data will be presented today in an oral presentation (Abstract LBA3500) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and have been simultaneously published in the New England Journal of Medicine.

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In a second interim analysis of overall survival (OS), a key secondary endpoint, the BRAFTOVI combination regimen reduced the risk of death by 51% compared to standard-of-care chemotherapy with or without bevacizumab (Hazard Ratio [HR] 0.49; 95% Confidence Interval [CI], 0.38, 0.63, p<0.0001). Median OS was 30.3 months (95% CI, 21.7, Not Estimated) with BRAFTOVI in combination with cetuximab and mFOLFOX6 compared to 15.1 months with chemotherapy with or without bevacizumab (95% CI, 13.7, 17.7). In the primary analysis of progression-free survival (PFS), the BRAFTOVI combination regimen reduced the risk of disease progression or death by 47% compared to standard-of-care chemotherapy with or without bevacizumab (HR 0.53; 95% CI, 0.41, 0.68, p<0.0001) as assessed by blinded independent central review (BICR). Median PFS was 12.8 months (95% CI, 11.2, 15.9) with the BRAFTOVI combination regimen compared to 7.1 months (95% CI, 6.8, 8.5).

"Patients with metastatic colorectal cancer whose tumors harbor a BRAF V600E mutation generally face a daunting prognosis, as this aggressive tumor often does not respond well to standard-of-care chemotherapy," said Elena Élez, M.D., Ph.D., senior investigator at Vall d’Hebron Institute of Oncology in Barcelona, Spain, and co-principal investigator of the BREAKWATER trial. "The BREAKWATER results are the first promising survival outcomes ever reported for BRAF-mutant metastatic colorectal cancer in the first-line setting, representing a practice-changing breakthrough for patients."

CRC is the third most common type of cancer in the world1 BRAF mutations are estimated to occur in 8-12% of people with mCRC and represent a poor prognosis.2 The BRAF V600E mutation is the most common BRAF mutation and the risk of mortality in patients with CRC harboring this mutation is more than double that of patients with no known BRAF mutation present.2-4

"The BRAFTOVI combination helped significantly reduce the risk of disease progression or death, potentially altering the course of disease for people with metastatic colorectal cancer with a BRAF V600E mutation," said Johanna Bendell, M.D., Chief Oncology Development Officer, Pfizer. "These unprecedented results from the BREAKWATER trial further establish the benefit of the BRAFTOVI combination regimen and its potential to become a new standard-of-care, building on Pfizer’s legacy in precision medicine and commitment to delivering breakthrough medicines that help people with cancer live better and longer lives."

The updated objective response rate (ORR) by BICR confirmed the improvement previously observed with the BRAFTOVI combination regimen compared to patients receiving chemotherapy with or without bevacizumab (65.7%; 95% CI, 59.4, 71.4 and 37.4%; 95% CI, 31.6, 43.7, respectively). The estimated median duration of response and median time to response were also maintained from the prior primary analysis. Results from the primary analysis of ORR were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancer Symposium (ASCO GI) and were simultaneously published in Nature Medicine in January 2025. Additional data from a separate arm of the BREAKWATER study evaluating BRAFTOVI in combination with cetuximab will also be presented at ASCO (Free ASCO Whitepaper).

"The risk of death for patients with BRAF V600E-mutant metastatic colorectal cancer is more than double compared to those with no known mutation," said Michael Sapienza, Chief Executive Officer, Colorectal Cancer Alliance. "These survival outcomes from the BREAKWATER study bring renewed hope to patients and their loved ones, providing the possibility of more time together. We are thrilled to see important cancer research propel us closer to our goal of ending this disease."

At the time of this analysis, the safety profile of BRAFTOVI in combination with cetuximab and mFOLFOX6 continued to be consistent with the known safety profile of each respective agent. No new safety signals were identified. The most common side effects (≥30%) were nausea, anemia, diarrhea, decreased appetite, vomiting, neutrophil count decrease, arthralgia, and rash. Among patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6, 13.8% experienced an adverse reaction that resulted in permanent discontinuation of BRAFTOVI.

The BRAFTOVI combination regimen received accelerated approval by the U.S. Food and Drug Administration (FDA) in December 2024 for patients with BRAF V600E -mutant mCRC based on a clinically meaningful and statistically significant improvement in confirmed ORR in treatment-naïve patients, the study’s other dual primary endpoint.5 Continued approval for this indication is contingent upon verification of clinical benefit. The approval was among the first in the industry to be conducted under the FDA’s Project FrontRunner, which seeks to support the development and approval of new cancer drugs for advanced or metastatic disease. The BREAKWATER survival data are being discussed with the U.S. FDA to support potential conversion to full approval in 2025.

Pfizer is continuing its commitment to help non-scientists understand the latest findings with the development of abstract plain language summaries (APLS) for company-sponsored research being presented, which are written in non-technical language. Those interested in learning more can visit www.Pfizer.com/apls to access the summaries.

About BREAKWATER
BREAKWATER is a Phase 3, randomized, active-controlled, open-label, multicenter trial of BRAFTOVI with cetuximab, alone or in combination with mFOLFOX6 in participants with previously untreated BRAF V600E-mutant metastatic CRC. Patients were randomized to receive BRAFTOVI 300 mg orally once daily in combination with cetuximab (discontinued after randomization of 158 patients), BRAFTOVI 300 mg orally once daily in combination with cetuximab and mFOLFOX6 (n=236) or mFOLFOX6, FOLFOXIRI, or CAPOX each with or without bevacizumab (control-arm) (n=243). The dual primary endpoints are ORR and progression-free survival (PFS) as assessed by blinded independent central review (BICR). Overall survival is a key secondary endpoint.

About Colorectal Cancer (CRC)
CRC is the third most common type of cancer in the world, with approximately 1.8 million new diagnoses in 2022.1 It is the second leading cause of cancer-related deaths.6 Overall, the lifetime risk of developing CRC is about 1 in 24 for men and 1 in 26 for women.6 In the U.S. alone, an estimated 154,270 people will be diagnosed with cancer of the colon or rectum in 2025, and approximately 53,000 are estimated to die from the disease each year.7 For 20% of those diagnosed with CRC, the disease has metastasized, or spread, making it harder to treat, and up to 50% of patients with localized disease eventually develop metastases.8

BRAF mutations are estimated to occur in 8-12% of people with mCRC and represent a poor prognosis for these patients.2 The BRAF V600E mutation is the most common BRAF mutation and the risk of mortality in patients with CRC with the BRAF V600E mutation is more than double that of patients with no known BRAF mutation present.2-4 Despite the high unmet need in BRAF V600E-mutant mCRC, prior to December 20, 2024, there were no approved biomarker-driven therapies specifically indicated for people with previously untreated BRAF V600E-mutant mCRC.9,10

About BRAFTOVI (encorafenib)
BRAFTOVI is an oral small molecule kinase inhibitor that targets BRAF V600E. Inappropriate activation of proteins in the MAPK signaling pathway (RAS-RAF-MEK-ERK) has been shown to occur in certain cancers, including CRC.

Pfizer has exclusive rights to BRAFTOVI in the U.S., Canada, Latin America, Middle East, and Africa. Ono Pharmaceutical Co., Ltd. has exclusive rights to commercialize the product in Japan and South Korea, Medison has exclusive rights to commercialize the product in Israel and Pierre Fabre Laboratories has exclusive rights to commercialize the product in all other countries, including Europe and Asia (excluding Japan and South Korea).

INDICATION AND USAGE
BRAFTOVI (encorafenib) is indicated, in combination with cetuximab and mFOLFOX6, for the treatment of patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-approved test. This indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

BRAFTOVI is also indicated, in combination with cetuximab, for the treatment of adult patients with mCRC with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy.

Limitations of Use: BRAFTOVI is not indicated for treatment of patients with wild-type BRAF CRC.

IMPORTANT SAFETY INFORMATION

Refer to the prescribing information for cetuximab and individual product components of mFOLFOX6 for recommended dosing and additional safety information.

WARNINGS AND PRECAUTIONS

New Primary Malignancies: New primary malignancies, cutaneous and non-cutaneous, can occur. In BEACON CRC (previously treated BRAF V600E mutation-positive mCRC), cutaneous squamous cell carcinoma (cuSCC), including keratoacanthoma (KA), occurred in 1.4% of patients with CRC, and a new primary melanoma occurred in 1.4% of patients who received BRAFTOVI in combination with cetuximab. In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC) skin papilloma was reported in 2.6%, basal cell carcinoma in 1.3%, squamous cell carcinoma of skin in 0.9%, keratoacanthoma in 0.4% and malignant melanoma in situ in 0.4% of patients who received BRAFTOVI in combination with cetuximab and mFOLFOX6. Perform dermatologic evaluations prior to initiating treatment, every 2 months during treatment, and for up to 6 months following discontinuation of treatment. Manage suspicious skin lesions with excision and dermatopathologic evaluation. Dose modification is not recommended for new primary cutaneous malignancies. Based on its mechanism of action, BRAFTOVI may promote malignancies associated with activation of RAS through mutation or other mechanisms. Monitor patients receiving BRAFTOVI for signs and symptoms of non-cutaneous malignancies. Discontinue BRAFTOVI for RAS mutation-positive non-cutaneous malignancies. Monitor patients for new malignancies prior to initiation of treatment, while on treatment, and after discontinuation of treatment.

Tumor Promotion in BRAF Wild-Type Tumors: In vitro experiments have demonstrated paradoxical activation of MAP-kinase signaling and increased cell proliferation in BRAF wild-type cells exposed to BRAF inhibitors. Confirm evidence of BRAF V600E or V600K mutation using an FDA-approved test prior to initiating BRAFTOVI.

Cardiomyopathy: Cardiomyopathy manifesting as left ventricular dysfunction associated with symptomatic or asymptomatic decreases in ejection fraction, has been reported in patients. Assess left ventricular ejection fraction (LVEF) by echocardiogram or multi-gated acquisition (MUGA) scan prior to initiating treatment, 1 month after initiating treatment, and then every 2 to 3 months during treatment. Safety has not been established in patients with a baseline ejection fraction that is either below 50% or below the institutional lower limit of normal (LLN). Patients with cardiovascular risk factors should be monitored closely. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

Hepatotoxicity: Hepatotoxicity can occur. In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC), the incidence of Grade 3 or 4 increases in liver function laboratory tests in patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6 was 2.2% for alkaline phosphatase, 1.3% for ALT, and 0.9% for AST. Monitor liver laboratory tests before initiation of BRAFTOVI, monthly during treatment, and as clinically indicated. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

Hemorrhage: In BEACON CRC (previously treated BRAF V600E mutation-positive mCRC), hemorrhage occurred in 19% of patients receiving BRAFTOVI in combination with cetuximab; Grade 3 or higher hemorrhage occurred in 1.9% of patients, including fatal gastrointestinal hemorrhage in 0.5% of patients. The most frequent hemorrhagic events were epistaxis (6.9%), hematochezia (2.3%), and rectal hemorrhage (2.3%). In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC), hemorrhage occurred in 30% of patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6; Grade 3 or 4 hemorrhage occurred in 3% of patients. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

Uveitis: Uveitis, including iritis and iridocyclitis, has been reported in patients treated with BRAFTOVI. Assess for visual symptoms at each visit. Perform an ophthalmological evaluation at regular intervals and for new or worsening visual disturbances, and to follow new or persistent ophthalmologic findings. Withhold, reduce dose, or permanently discontinue based on severity of adverse reaction.

QT Prolongation: BRAFTOVI is associated with dose-dependent QTc interval prolongation in some patients. In BREAKWATER (previously untreated BRAF V600E mutation-positive mCRC), an increase of QTcF >500 ms was measured in 3.6% (8/222) of patients receiving BRAFTOVI in combination with cetuximab and mFOLFOX6. Monitor patients who already have or who are at significant risk of developing QTc prolongation, including patients with known long QT syndromes, clinically significant bradyarrhythmias, severe or uncontrolled heart failure and those taking other medicinal products associated with QT prolongation. Correct hypokalemia and hypomagnesemia prior to and during BRAFTOVI administration. Withhold, reduce dose, or permanently discontinue for QTc >500 ms.

Embryo-Fetal Toxicity: BRAFTOVI can cause fetal harm when administered to pregnant women. BRAFTOVI can render hormonal contraceptives ineffective. Advise females of reproductive potential to use effective nonhormonal contraception during treatment with BRAFTOVI and for 2 weeks after the final dose.

Risks Associated with Combination Treatment: BRAFTOVI is indicated for use as part of a regimen in combination with cetuximab, or in combination with cetuximab and mFOLFOX6. Refer to the prescribing information for cetuximab and individual product components of mFOLFOX6 for additional risk information.

Lactation: Advise women not to breastfeed during treatment with BRAFTOVI and for 2 weeks after the final dose.

Infertility: Advise males of reproductive potential that BRAFTOVI may impair fertility.

ADVERSE REACTIONS

BREAKWATER Trial (previously untreated BRAF V600E mutation-positive mCRC)

Serious adverse reactions occurred in 38% of patients who received BRAFTOVI in combination with cetuximab and mFOLFOX6. Serious adverse reactions in >3% of patients included intestinal obstruction (3.5%) and pyrexia (3.5%).
Fatal gastrointestinal perforation occurred in 0.9% of patients who received BRAFTOVI in combination with cetuximab and mFOLFOX6.
Most common adverse reactions (≥25%, all grades) in the BRAFTOVI with cetuximab and mFOLFOX6 arm compared to the control arm (mFOLFOX6 ± bevacizumab or FOLFOXIRI ± bevacizumab or CAPOX ± bevacizumab) were peripheral neuropathy (62% vs 53%), nausea (51% vs 48%), fatigue (49% vs 38%), rash (31% vs 4%), diarrhea (34% vs 47%), decreased appetite (33% vs 25%), vomiting (33% vs 21%), hemorrhage (30% vs 18%), abdominal pain (26% vs 27%), and pyrexia (26% vs 14%).
Most common laboratory abnormalities (≥10%, grade 3 or 4) in the BRAFTOVI with cetuximab and mFOLFOX6 arm compared to the control arm (mFOLFOX6 ± bevacizumab or FOLFOXIRI ± bevacizumab or CAPOX ± bevacizumab) were: increased lipase (51% vs 25%), decreased neutrophil count (36% vs 34%), decreased hemoglobin (13% vs 5%), decreased white blood cell count (12% vs 7%), and increased glucose (11% vs 2%).
BEACON CRC Trial (previously treated BRAF V600E mutation-positive mCRC)

Most common adverse reactions (≥25%, all grades) in the BRAFTOVI with cetuximab arm compared to irinotecan with cetuximab or FOLFIRI with cetuximab (control) were: fatigue (51% vs 50%), nausea (34% vs 41%), diarrhea (33% vs 48%), dermatitis acneiform (32% vs 43%), abdominal pain (30% vs 32%), decreased appetite (27% vs 27%), arthralgia (27% vs 3%), and rash (26% vs 26%).
Other clinically important adverse reactions occurring in <10% of patients who received BRAFTOVI in combination with cetuximab was pancreatitis.
Most common laboratory abnormalities (all grades) (≥20%) in the BRAFTOVI with cetuximab arm compared to irinotecan with cetuximab or FOLFIRI with cetuximab (control) were: anemia (34% vs 48%) and lymphopenia (24% vs 35%).
DRUG INTERACTIONS

Strong or moderate CYP3A4 inhibitors: Avoid coadministration of BRAFTOVI with strong or moderate CYP3A4 inhibitors, including grapefruit juice. If coadministration is unavoidable, reduce the BRAFTOVI dose.

Strong CYP3A4 inducers: Avoid coadministration of BRAFTOVI with strong CYP3A4 inducers.

Sensitive CYP3A4 substrates: Avoid the coadministration of BRAFTOVI with CYP3A4 substrates (including hormonal contraceptives) for which a decrease in plasma concentration may lead to reduced efficacy of the substrate. If the coadministration cannot be avoided, see the CYP3A4 substrate product labeling for recommendations.

Dose reductions of drugs that are substrates of OATP1B1, OATP1B3, or BCRP may be required when used concomitantly with BRAFTOVI.

Avoid coadministration of BRAFTOVI with drugs known to prolong QT/QTc interval.

View the full Prescribing Information.

On May 30, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported that ten abstracts have been accepted for presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Tempus, MAY 30, 2025, View Source [SID1234653535]). The event will take place May 30 – June 3 in Chicago, Illinois. Tempus will present its cutting-edge clinical findings and innovative technologies, highlighting the company’s use of AI-driven solutions to advance precision oncology.

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"At ASCO (Free ASCO Whitepaper) 2025, we are proud to showcase a diverse portfolio of studies that highlight how our cutting-edge technologies, including tumor-naive and tumor-informed MRD monitoring, whole genomic sequencing for hematologic malignancies, and multi-omic biomarker profiling, are providing clinicians and researchers with actionable insights to personalize treatment and improve outcomes for patients," said Ezra Cohen, MD, Chief Medical Officer of Oncology at Tempus. "We are excited to share these advancements and collaborate with the oncology community to shape the future of precision medicine."

Research highlights include:

Oral Presentation (1506): A technology-enabled clinical trial program’s impact on patient screening and trial enrollment in 2024
Date/Time: Monday, June 2 from 3:00 PM–6:00 PM CDT
Overview: The TIME network utilized algorithmic screening technology combined with nurse match review to screen over 1.28 million patients for clinical trials in 2024, resulting in 573 total consents – an average of 1.57 consents per day. The network’s programmatic screening at scale, Tempus nurse review, and rapid activation processes helps increase patient access to trials.
Poster Presentation (213/3544): Circulating tumor DNA (ctDNA) dynamics in liver-limited metastatic colorectal cancer (mCRC) patients resected after first-line systemic treatment
Date/Time: Saturday, May 31 from 9:00 AM–12:00 PM CDT
Overview: Tempus xM, a tumor-naive test, serves as a prognostic tool for monitoring disease recurrence in resected liver-limited mCRC patients treated with upfront systemic therapy. xM demonstrates strong performance in predicting clinical recurrence and relapse-free survival following surgery.
Poster Presentation (499/11160): Evaluation of large language model (LLM)-based clinical abstraction of Electronic Health Records (EHRs) for Non-Small Cell Lung Cancer (NSCLC) patients
Date/Time: Saturday, May 31 from 1:30 PM–4:30 PM CDT
Overview: LLMs show promise for improving abstraction efficiency, converting clinical data from unstructured EHRs into a structured format suitable for analysis. Tempus utilized a two-stage LLM system to abstract critical clinical data of NSCLC patients, achieving high agreement with human abstractors across various data domains.
Poster Presentation (148/6532): Detection of KMT2A Partial Tandem Duplication (PTD) in AML by Whole Genome Sequencing (WGS): Addressing Limitations of Traditional Techniques in the Era of Revumenib Approval
Date/Time: Sunday, June 1 at 9:00 AM–12:00 PM CDT
Overview: Tempus xH, a whole genome sequencing assay for hematologic malignancies, enables highly sensitive detection of KMT2A-PTDs in AML—unlocking access to targeted therapies like revumenib. xH helps identify patients who may benefit from emerging treatments and equips clinicians with the insights they need.
Poster Presentation (20/2558): A molecular biomarker for longitudinal monitoring of therapeutic efficacy in a real-world cohort of advanced solid tumors treated with immune checkpoint inhibitors
Date/Time: Sunday, June 1 at 1:30 PM–4:30 PM CDT
Overview: Tempus xM, a tumor-naive test, monitors treatment response by tracking ctDNA dynamics over time. Patients classified as a molecular non-responder at at least one timepoint while on immunotherapy had worse overall survival than molecular responders or patients with no ctDNA detected, highlighting the value of xM molecular response monitoring as a tool to guide ICI treatment decisions.

On May 30, 2025 Artera, the developer of multimodal artificial intelligence (MMAI)-based prognostic and predictive cancer tests, reported the presentation of two new abstracts at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, including an oral presentation selected for Best of ASCO (Free ASCO Whitepaper) 2025, an honor reserved for studies with the greatest potential to shape the future of cancer care (Press release, Artera, MAY 30, 2025, View Source [SID1234653534]).

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The featured oral presentation showcases the first validated MMAI algorithm—used in the ArteraAI Prostate Test—to identify high-risk, non-metastatic prostate cancer patients who are likely to benefit from adding androgen receptor pathway inhibitors (ARPIs) to standard therapy. The STAMPEDE trial helped to establish ARPIs as the standard of care treatment for high-risk patients, but adoption of ARPIs has been uneven, likely due to concerns over side effects and follow-up care.

The study, conducted as part of the STAMPEDE trial, evaluated the addition of ARPIs—specifically abiraterone acetate + prednisolone—to standard androgen deprivation therapy (ADT) and radiation. Artera’s model identified that only 25% of high-risk patients derived meaningful benefit from ARPI intensification, suggesting the opportunity to spare up to 75% of this cohort from unnecessary toxicities.

"This data helps answer one of the most critical questions in cancer care: which patients will benefit from added treatment, and which will not," said Nick James, MD, PhD, Professor of Prostate and Bladder Cancer Research at The Institute of Cancer Research, London, and Consultant Clinical Oncologist at The Royal Marsden NHS Foundation Trust. While traditional tests flag patients at risk of poor outcomes, they don’t personalize treatment decisions. Our collaboration with Artera allows us to uncover patterns invisible to the human eye and optimize treatments like never before. The AI tool allows us to connect beneficial treatments to the patient, while sparing those who may suffer unnecessary side effects, or even premature death, if they receive ARPIs they don’t need."

In addition to the oral presentation, Artera will also present a poster featuring external validation of its MMAI platform in men who have undergone radical prostatectomy (RP) for localized prostate cancer. The study demonstrates that the RP MMAI model is an independent prognostic tool for predicting biochemical recurrence (BCR) and long-term outcomes, even when controlling for clinical risk models. Artera’s solution works with routine pathology and clinical data and does not require extra tissue or complex molecular testing, making it broadly scalable, cost-effective, and faster to implement.

"We are proud to see Artera’s MMAI platform recognized with two abstracts at ASCO (Free ASCO Whitepaper), including an oral presentation selected for Best of ASCO (Free ASCO Whitepaper)," said Timothy Showalter, Chief Medical Officer of Artera. "These studies reinforce our commitment to the rigorous clinical validation of the ArteraAI Prostate Test and our broader MMAI platform. Together, they reflect our mission to empower clinicians and patients with personalized, actionable insights that support confident, shared decision-making in prostate cancer care."

The studies presented by Artera add to the growing body of evidence that its MMAI platform can inform real-time clinical decisions and bring personalized cancer care to broader patient populations. For more information on Artera, visit Artera.ai.

Presentations at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting include:

Oral Presentation: Multimodal artificial intelligence (MMAI) model to identify benefit from 2nd-generation androgen receptor pathway inhibitors (ARPI) in high-risk non-metastatic prostate cancer patients from STAMPEDE.
Abstract Number: 5001
Session Type and Title: Oral Session – Genitourinary Cancer—Prostate, Testicular, and Penile
Date and Time: Tuesday, June 3rd at 9:45 a.m. CT

Poster Presentation: External validation of a pathology-based multimodal artificial intelligence biomarker for predicting prostate cancer outcomes after prostatectomy.
Abstract Number: 5106
Session Type and Title: Poster Session – Genitourinary Cancer—Prostate, Testicular, and Penile
Date and Time: Monday, June 2nd at 9:00 a.m. CT

On May 30, 2025 Annals of Oncology reported full results from the pivotal investigational Phase III PEACE III trial, evaluating XOFIGO (radium-223 dichloride) in combination with enzalutamide, an AR pathway inhibitor (ARPI), versus enzalutamide alone in the first-line treatment of patients with metastatic castration-resistant prostate cancer (mCRPC) with bone metastases (Press release, Bayer, MAY 30, 2025, View Source [SID1234653533]).

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XOFIGO is indicated for the treatment of patients with castration-resistant prostate cancer, symptomatic bone metastases and no known visceral metastatic disease.2

Initially presented as a late-breaking abstract during the Presidential Symposium at ESMO (Free ESMO Whitepaper) 2024, results demonstrated that the addition of XOFIGO to enzalutamide significantly increased radiological progression-free survival (rPFS) for patients with mCRPC with bone metastases, with a 31% reduction in the risk of progression or death (HR 0.69; 95% CI, 0.54-0.87; p=0.0009) compared to enzalutamide alone.1,3 Patients receiving XOFIGO in combination with enzalutamide had a median rPFS of 19.4 months (95% CI, 17.1-25.3 months) compared to 16.4 months (95% CI, 13.8-19.2 months) with enzalutamide, a 3-month difference in median rPFS.1,3 The trial was a collaboration between the European Organization for Research and Treatment of Cancer (EORTC), Clinical Trial Ireland (CTI), the Canadian Urological Oncology Group (CUOG), the Latin American Cooperative Oncology Group (LACOG), and French UNICANCER Urogenital Tumor Study Group (GETUG).

Additionally, at a preplanned interim analysis the results demonstrated statistically significant overall survival (OS), with a 31% reduction in the risk of death (HR=0.69; 95% CI 0.52-0.90; p=0.0031), with a median OS of 35.0 months (95% CI 28.8-38.9) in the enzalutamide arm compared to 42.3 months (95% CI 36.8-49.1) for XOFIGO plus enzalutamide.1 The study will continue to the final OS analysis. XOFIGO is the first and only alpha emitting radiopharmaceutical that treats bone metastases in mCRPC approved by the U.S. Food and Drug Administration (FDA).

"PEACE III is the first major Phase III trial to combine an ARPI with radiopharmaceutical that showed statistical significance in meeting the primary endpoint," said Denis Lacombe, Chief Executive Officer, EORTC. "The EORTC is proud to be at the forefront of this groundbreaking trial, helping to redefine the development of clinical trials and supporting patient care for difficult to treat diseases."

The results were consistent with the established safety profile of XOFIGO, although authors noted the importance of administering bone protective agents (BPAs) to avoid fractures. Following the release of the ERA-223 results, the PEACE III study was amended in March 2018 making BPAs mandatory at the monthly skeletal-related-event dose. The observed reduction in fractures following this amendment underlined the importance of using a BPA in patients with metastatic castration-resistant prostate cancer (mCRPC) and bone metastasis also in the era of ARPI’s.4 Grade 3 or higher treatment emergent adverse events (TEAE) were recorded in 65.6% of patients in the combination arm compared to 55.8% of patients who received enzalutamide alone.1 The most frequent Grade 3 or higher TEAEs were hypertension (34%), fatigue (6%), fracture (5%), anemia (5%), and neutropenia (5%).1 Fractures (either treatment-emergent or post-treatment, symptomatic or pathologic, or with or without BPA use) were reported in 24.3% of patients in the combination arm and 13.4% of patients in the enzalutamide arm.1 These results demonstrate the potential for this combination to be a new treatment option for patients with mCRPC and bone metastases who have experienced disease progression on androgen deprivation therapy (ADT).

"There remains an unmet patient need for people living with metastatic castration-resistant prostate cancer who have bone metastases," said Christine Roth, Global Head of Product Strategy and Commercialization at Bayer’s Pharmaceuticals Division. "The PEACE III trial underscores our dedication to advancing therapies for patients with prostate cancer and exploring the full potential of XOFIGO."

The trial is supported by Astellas and Pfizer who manufacture enzalutamide (Xtandi) in collaboration with Bayer.

About PEACE III (EORTC GUCG-1333)

The PEACE III trial is an international, randomized, open-label, Phase III trial designed to investigate the efficacy and safety of XOFIGO in combination with enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC) and bone metastases. A total of 446 patients were randomized 1:1 to receive either XOFIGO 55 kBq/kg as an intravenous injection monthly for six cycles in combination with enzalutamide 160mg orally daily or enzalutamide 160mg orally daily.

The primary endpoint was radiological progression-free survival (rPFS) by investigator assessment. Key secondary endpoints included overall survival (OS), time to subsequent systemic treatment, pain progression, and symptomatic skeletal event.

This trial was a collaboration with several cancer cooperative groups: EORTC, CTI, CUOG, LACOG, and GETUG.

About Xofigo (radium-223 dichloride) Injection2

Xofigo is indicated for the treatment of patients with castration-resistant prostate cancer, symptomatic bone metastases and no known visceral metastatic disease.

Important Safety Information for Xofigo (radium-223 dichloride) Injection

Warnings and Precautions:

Bone Marrow Suppression: In the phase 3 ALSYMPCA trial, 2% of patients in the
Xofigo arm experienced bone marrow failure or ongoing pancytopenia, compared to no patients treated with placebo. There were two deaths due to bone marrow failure. For 7 of 13 patients treated with Xofigo bone marrow failure was ongoing at the time of death. Among the 13 patients who experienced bone marrow failure, 54% required blood transfusions. Four percent (4%) of patients in the Xofigo arm and 2% in the placebo arm permanently discontinued therapy due to bone marrow suppression. In the randomized trial, deaths related to vascular hemorrhage in association with myelosuppression were observed in 1% of Xofigo-treated patients compared to 0.3% of patients treated with placebo. The incidence of infection-related deaths (2%), serious infections (10%), and febrile neutropenia (<1%) was similar for patients treated with Xofigo and placebo. Myelosuppression–notably thrombocytopenia, neutropenia, pancytopenia, and leukopenia–has been reported in patients treated with Xofigo.

Monitor patients with evidence of compromised bone marrow reserve closely and provide supportive care measures when clinically indicated. Discontinue Xofigo in patients who experience life-threatening complications despite supportive care for bone marrow failure
Hematological Evaluation: Monitor blood counts at baseline and prior to every dose of Xofigo. Prior to first administering Xofigo, the absolute neutrophil count (ANC) should be ≥1.5 × 109/L, the platelet count ≥100 × 109/L, and hemoglobin ≥10 g/dL. Prior to subsequent administrations, the ANC should be ≥1 × 109/L and the platelet count ≥50 × 109/L. Discontinue Xofigo if hematologic values do not recover within 6 to 8 weeks after the last administration despite receiving supportive care
Concomitant Use With Chemotherapy: Safety and efficacy of concomitant
chemotherapy with Xofigo have not been established. Outside of a clinical trial,
concomitant use of Xofigo in patients on chemotherapy is not recommended due to the potential for additive myelosuppression. If chemotherapy, other systemic radioisotopes, or hemibody external radiotherapy are administered during the treatment period, Xofigo should be discontinued
Increased Fractures and Mortality in Combination With Abiraterone Plus Prednisone/Prednisolone: Xofigo is not recommended for use in combination with abiraterone acetate plus prednisone/prednisolone outside of clinical trials. At the primary analysis of the Phase 3 ERA-223 study that evaluated concurrent initiation of Xofigo in combination with abiraterone acetate plus prednisone/prednisolone in 806 asymptomatic or mildly symptomatic mCRPC patients, an increased incidence of fractures (28.6% vs 11.4%) and deaths (38.5% vs 35.5%) have been observed in patients who received Xofigo in combination with abiraterone acetate plus prednisone/prednisolone compared to patients who received placebo in combination with abiraterone acetate plus prednisone/prednisolone. Safety and efficacy with the combination of Xofigo and agents other than gonadotropin-releasing hormone analogues have not been established
Embryo-Fetal Toxicity: The safety and efficacy of Xofigo have not been established in females. Xofigo can cause fetal harm when administered to a pregnant female. Advise pregnant females and females of reproductive potential of the potential risk to a fetus. Advise male patients to use condoms and their female partners of reproductive potential to use effective contraception during and for 6 months after completing treatment with Xofigo
Administration and Radiation Protection: Xofigo should be received, used, and administered only by authorized persons in designated clinical settings. The administration of Xofigo is associated with potential risks to other persons from radiation or contamination from spills of bodily fluids such as urine, feces, or vomit. Therefore, radiation protection precautions must be taken in accordance with national and local regulations

Fluid Status: Dehydration occurred in 3% of patients on Xofigo and 1% of patients on

placebo. Xofigo increases adverse reactions such as diarrhea, nausea, and vomiting, which may result in dehydration. Monitor patients’ oral intake and fluid status carefully and promptly treat patients who display signs or symptoms of dehydration or hypovolemia

Injection Site Reactions: Erythema, pain, and edema at the injection site were reported in 1% of patients on Xofigo

Secondary Malignant Neoplasms: Xofigo contributes to a patient’s overall long-term cumulative radiation exposure. Long-term cumulative radiation exposure may be associated with an increased risk of cancer and hereditary defects. Due to its mechanism of action and neoplastic changes, including osteosarcomas, in rats following administration of radium-223 dichloride, Xofigo may increase the risk of osteosarcoma or other secondary malignant neoplasms. However, the overall incidence of new malignancies in the randomized trial was lower on the Xofigo arm compared to placebo (<1% vs 2%; respectively), but the expected latency period for the development of secondary malignancies exceeds the duration of follow-up for patients on the trial

Subsequent Treatment With Cytotoxic Chemotherapy: In the randomized clinical trial, 16% of patients in the Xofigo group and 18% of patients in the placebo group received cytotoxic chemotherapy after completion of study treatments. Adequate safety monitoring and laboratory testing was not performed to assess how patients treated with Xofigo will tolerate subsequent cytotoxic chemotherapy

Adverse Reactions: The most common adverse reactions (≥10%) in the Xofigo arm vs the placebo arm, respectively, were nausea (36% vs 35%), diarrhea (25% vs 15%), vomiting (19% vs 14%), and peripheral edema (13% vs 10%). Grade 3 and 4 adverse events were reported in 57% of Xofigo-treated patients and 63% of placebo-treated patients. The most common hematologic laboratory abnormalities in the Xofigo arm (≥10%) vs the placebo arm, respectively, were anemia (93% vs 88%), lymphocytopenia (72% vs 53%), leukopenia (35% vs 10%), thrombocytopenia (31% vs 22%), and neutropenia (18% vs 5%).

Please see the full Prescribing Information for Xofigo (radium Ra 223 dichloride).