Genialis Highlights Power of Its Supermodel to Predict and Explain Therapeutic Response at AACR 2025

On April 23, 2025 Genialis, the RNA biomarker company, reported two new studies demonstrating how predictive algorithms can drive better outcomes for patients, providers, and drug developers (Press release, Genialis, APR 23, 2025, View Source [SID1234652080]). In poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, Genialis showed its AI foundation model of cancer—the Genialis Supermodel—can predict how a patient will respond to a specific therapy (Abstract 3313 / 16). The Supermodel can also help explain how drugs work in the body and how the tumor learns to resist treatment, as well as propose potential new drug combinations to improve outcomes. The AACR (Free AACR Whitepaper) presentations share these insights from Genialis’ own work with KRAS G12C NSCLC patient data and its collaboration with Debiopharm on the Debio 0123 WEE1 inhibitor in various cancer types. (Abstract 3659 / 21)

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Most drug candidates fail in clinical trials, and of the ones that are successful, most cancer patients do not benefit, a problem that continues to plague drug developers, physicians, patients, and their families. Predictive biomarkers are known to improve these results considerably. The Genialis Supermodel is a large molecular model trained on over one billion RNA-seq-derived data points that enables rapid development and validation of accurate and information-rich biomarker algorithms across a wide range of cancer drug targets to act as a recommendation engine for every cancer target, drug, and patient.

"A shortcoming in precision medicine is we need a deeper understanding of individual patient biology and the molecular context in which a drug is acting, as this can provide crucial translational and clinical intelligence," said Rafael Rosengarten, PhD, CEO of Genialis. "We built the Genialis Supermodel to embrace the complexity and learn the underlying biology of cancer so we can guide therapy decisions and derisk drug development. At AACR (Free AACR Whitepaper), we’re showing that the same framework applies across very different drug targets and tumor types – and most importantly, that it works."

Genialis Supermodel for KRAS-mutated cancers

KRAS mutations drive approximately 25 percent of all human cancers, leading to an estimated 3.9 million new cases each year. Despite the recent approvals of two KRAS inhibitors (KRASi), these drugs show limited efficacy and durability. Response rates hover around 40 percent, and the median patient benefit lasted just 8 to 10 months, in part because the available biomarkers for patient selection rely on genotype alone. The Genialis Supermodel addresses this challenge by mapping over twenty biological modules relevant to KRASi-therapy in every tumor sample rather than focusing solely on the allele.

The first poster, led by Genialis, incorporates analysis of therapeutic mechanisms related to EGFR, immune checkpoint inhibitors (ICIs), and standard-of-care chemotherapy to stratify patients with KRAS-mutated cancers using the company’s first-in-class RNA biomarker algorithm, Genialis krasID. By broadening the biological space considered by the algorithm, Genialis is better able to predict results from both well-established and investigational therapies, to model various combinations, and to suggest the most effective sequencing/indication of various compounds. This new research expands on Genialis’ novel work presented at AACR (Free AACR Whitepaper) 2024. Genialis krasID serves as a sterling example of how the Genialis Supermodel scales to align treatment selection with the underlying tumor biology and therapeutic context.

"Genotype determines eligibility, but it often fails to predict actual clinical benefit. The Supermodel addresses this gap by capturing the functional biology that drives response," said Josh Wheeler, MD PhD, Director of Research and Innovation at Genialis. "The biology-first approach engendered by the Genialis Supermodel provides the mechanistic understanding and the necessary precision for informing and stratifying both emerging and standard of care therapeutic selection in patients."

Predicting response to WEE1 inhibitor Debio 0123

The second poster, presented in collaboration with Swiss-based global biopharmaceutical company Debiopharm, describes early results from the development of a predictive model for Debio 0123, a WEE1 inhibitor currently in phase 1 clinical trials. While the collaboration has since expanded to clinical studies, this poster describes the use of the Supermodel to identify biological modules in the DNA damage response space that contribute to Debio 0123 WEE1 response, training of a first-generation machine learning algorithm, and exploration of that biomarker across various translational models.

Using the DNA damage response (DDR)-related modules as input features, the ML algorithm was trained on patient-derived organoid datasets and tested on xenografts. The resulting biomarker algorithm achieved promising performance in leave-one-out cross-validation. Notably, it accurately predicted an in vivo responder that had been missed by organoid IC50 testing, showing the model’s ability to capture biologically relevant signals that may not be observed through traditional in vitro assays. These early findings set the foundation for further validation in independent clinical datasets and point to broader use of this biomarker framework for other DDR-targeting agents.

"The findings from this study show the Genialis Supermodel is able to learn components of complex biology that are translationally relevant and likely to prove important in human datasets. We see strong potential in using this kind of RNA-based, ML-driven approach to guide patient selection and boost development success," said Luke Piggott, Director, Global Business Development & Principal Scientist at Debiopharm. "The ability to predict, and soon validate, response is now being born out in clinical data and gives us an important strategic advantage."

These two AACR (Free AACR Whitepaper) posters demonstrate how Genialis supports drug development through its modular, interpretable Supermodel. Whether targeting KRAS-driven tumors or DDR mechanisms or entirely novel target biology, Genialis’ predictive biomarker algorithms reflect tumor biology, making it possible to match patients to successful treatment options and improve clinical outcomes.

Q1 2025 Highlights

On April 23, 2023 Boston Scientific reported first quarter for 2025 highlights (Presentation, Boston Scientific, APR 23, 2025, View Source [SID1234654129]).

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Verastem Oncology Announces U.S. IND Clearance of VS-7375, Oral KRAS G12D (ON/OFF) Inhibitor, Enabling Phase 1/2a Trial in Advanced Solid Tumors

On April 23, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application of VS-7375, an oral KRAS G12D (ON/OFF) inhibitor for clinical evaluation (Press release, Verastem, APR 23, 2025, View Source [SID1234652065]). The Company expects to initiate a Phase 1/2a study in mid-2025 with plans for multiple expansion cohorts, including combinations, in advanced solid tumors, such as pancreatic cancer, colorectal cancer, and non-small cell lung cancer.

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The Company also announced the acceptance of an abstract, by our partner GenFleet Therapeutics, for rapid oral presentation of the preliminary dose escalation phase of the Phase 1/2 study of VS-7375 (also known as GFH375) conducted by GenFleet at the upcoming 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held on May 30-June 3, 2025, in Chicago, IL. The abstract is under embargo until May 22, 2025, at 5:00 pm EDT, when additional data will be released.

"We’re excited to advance the clinical program for VS-7375 in the U.S. and build on the initial dose escalation work conducted by GenFleet in China that demonstrated oral bioavailability and no dose-limiting toxicities across six dose levels, with partial responses achieved among multiple patients with both pancreatic and advanced lung cancers," said Dan Paterson, president and chief executive officer of Verastem Oncology. "We believe there remains a significant opportunity to improve on the efficacy seen to date with other KRAS G12D-selective agents. VS-7375’s dual inhibition of both the ON/OFF states has the potential to drive deep and durable cancer responses and allow for better combinability with other agents. We look forward to our partner GenFleet’s oral presentation that will include updated safety and efficacy data from the Phase 1 study at the ASCO (Free ASCO Whitepaper) annual meeting."

The Phase 1/2a study will be conducted in the U.S., with the potential to expand globally, and will evaluate the safety and efficacy of VS-7375 in patients with advanced KRAS G12D mutant solid tumors. The starting dose for the Phase 1 study is based on an efficacious dose identified in the initial data from the GenFleet study to accelerate the trial’s progress. Verastem plans to dose escalate across levels where cancer responses were observed in GenFleet’s study and will assess in the Phase 2a portion the efficacy and safety of VS-7375, both as monotherapy and in combination, in patients with advanced solid tumors, such as pancreatic, colorectal, and non-small cell lung cancers.

ASCO 2025 Presentation Details:

The abstract is under embargo until May 22, 2025, at 5:00 pm EDT.

Title: A First-in-Human Phase I/II Study of GFH375, a Highly Selective and Potent Oral KRAS G12D Inhibitor in Patients with KRAS G12D Mutant Advanced Solid Tumors
Abstract Number: 3013
Session: Rapid Oral Abstract Sessions: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology
Session Date/Time: Monday, June 2, 2025, from 8:00 am to 9:30 am CDT
About KRAS G12D

KRAS G12D represents 26% of all KRAS mutations, making it the most prevalent KRAS mutation in human cancers. The KRAS G12D mutation occurs most commonly in pancreatic (37%), colorectal (12.5%), endometrial (8%), and non-small cell lung (5%) cancers. Currently, no therapies are approved by the U.S. Food and Drug Administration specifically targeting KRAS G12D mutations in cancer.

About VS-7375, an Oral KRAS G12D (ON/OFF) Inhibitor

VS-7375 is a potential best-in-class, potent, and selective oral KRAS G12D dual ON/OFF inhibitor. VS-7375 is the lead program from the Verastem Oncology discovery and development collaboration with GenFleet Therapeutics. Verastem announced in April 2025 that the U.S. Investigational New Drug (IND) application for VS-7375 was cleared and plans to initiate a Phase 1/2a clinical trial in mid-2025. GenFleet’s IND for VS-7375 (known as GFH375 in China) was approved in China in June 2024, and the first patient was dosed in a Phase 1/2 study in July 2024.

Tempus Signs Expanded Strategic Agreements with AstraZeneca and Pathos to Develop the Largest Multimodal Foundation Model in Oncology

On April 23, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported multi-year, strategic collaborations with AstraZeneca (LSE/STO/Nasdaq: AZN) and Pathos AI, Inc., in which the companies will work together to build a multimodal foundation model in oncology which can be used to gather biological and clinical insights, discover novel drug targets, and develop therapeutics for the broader oncology community (Press release, Tempus, APR 23, 2025, View Source [SID1234652081]).

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Tempus’ de-identified oncology data will be used to build the foundation model. Upon completion, the model will be shared among all three parties to advance their individual efforts to improve patient care. The agreements include $200 million in data licensing and model development fees to Tempus.

The agreement with AstraZeneca expands on the strategic partnership between the two companies announced in 2021 and aims to leverage Tempus’ AI-enabled platform and vast repository of multimodal data to advance novel therapeutic programs in oncology on a global scale.

"Generative AI and the emergence of large multimodal models is the final catalyst needed to usher in precision medicine in oncology at scale," said Eric Lefkofsky, Founder and CEO of Tempus. "Tempus has spent the last decade investing billions of dollars into collecting the necessary data needed for a foundation model of this kind to take shape. We look forward to working with AstraZeneca and Pathos to apply AI-enabled solutions to advance therapies in an effort to help patients live longer and healthier lives."

"Cancer drug discovery and clinical development are being transformed by the ability to analyze vast amounts of rich data using artificial intelligence," said Jorge Reis-Filho, Chief AI and Data Scientist, Oncology R&D, AstraZeneca. "We are excited to collaborate with Tempus and Pathos to advance our data and AI-driven R&D strategy through the development of a multimodal oncology foundation model that we believe will accelerate and increase the probability of clinical success across our diverse pipeline."

"As artificial intelligence becomes more prominent in drug discovery and development, the opportunity for companies like Pathos to build foundation models that seemed almost unimaginable a few years ago is now taking shape," said Mohamad Makhzoumi, Co-CEO of NEA and Pathos Board Member. "We couldn’t be more excited to collaborate with Tempus and AstraZeneca given the potential of these models to improve patient outcomes."

Interim Report 1-3/2025

On April 23, 2025 Orion reported interim financial report (Presentation, Orion, APR 23, 2025, View Source [SID1234654297]).

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