ConcertAI Announces Strategic Agreement with Bayer to Accelerate Clinical Development in Precision Oncology

On April 22, 2025 ConcertAI reported a multi-year agreement with Bayer to leverage ConcertAI’s Translational360 — powered by Guardant Health liquid biopsy genomic data — and AI SaaS solutions, which use artificial intelligence and machine learning (AI/ML)-derived insights to accelerate clinical development in precision oncology (Press release, Bayer, APR 22, 2025, View Source [SID1234652029]).

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The agreement will fully leverage ConcertAI’s newly launched Translational360, an integrated research-grade longitudinal clinical molecular database that taps into the CancerLinQ network of de-identified cancer patient data from over 9 million records coming from all 50 states in the U.S.

Translational360 combines clinical, genomic, transcriptomic, and whole-slide imaging (WSI) from comprehensive molecular testing for deep phenotypical and genomic insights. Transcriptomics is increasingly a foundation of biopharma translational sciences, allowing researchers to understand disease molecular mechanisms, basis of patient response, and inter-patient variability that’s essential for developing new therapeutics. Too often, clinical trial results in early phases are ambiguous with idiosyncratic positive and negative responses. The integrated data solutions and advanced AI enable selection of programs with the highest likelihood of success and design trials informed by multi-modal, multi-genomic, and transcriptomic data and AI.

"This partnership furthers causal biological inferences, where multi-modal and multi-molecular data can be integrated with AI/ML-based approaches across discovery, translation, and development, accelerating oncology pipelines, allowing our biopharma partners to deliver better medicines faster," said Jeff Elton, Ph.D., CEO of ConcertAI. "This partnership builds on a multi-year history of working together and is unique in offering both tissue and liquid biopsy molecular data, allowing insights into patterns of treatment response, acquisition of resistance, AI modeling of likely success and benefit, informing program priority and clinical study design."

"Data play a critical role in providing a multi-dimensional understanding of a tumor, the clinical environment, and the patient’s response to therapy," said Helmy Eltoukhy, Chairman and co-CEO of Guardant Health. "The real-world data can provide access to unprecedented insights into a patient’s cancer journey, including a tumor’s complex systemic interactions, to help users navigate critical decision points as they develop novel targeted therapies."

"As cancer rates continue to rise, we’re committed to advancing next-generation solutions that can speed up drug discovery and clinical development, enabling us to bring precision oncology treatments to patients faster," said Sai Jasti, Head of Data Science and AI for Pharma R&D at Bayer. "By combining ConcertAI’s powerful data solutions with Bayer’s scientific and AI expertise, we aim to enhance the use of real-world data and cutting-edge AI to boost R&D productivity and ultimately deliver transformative precision therapies to those who need them."

"More AI-designed new molecular entities are progressing faster in first-in-human trials, which is a great milestone," said Claudio D’Ambrosio, Ph.D., Chief Revenue Officer of ConcertAI. "Our entire R&D process has been historically built on studying controlled cell lines and animal models that don’t reflect the interspecies differences, the physiology, and the different biological barriers we have in humans. We are flipping this paradigm on its head. We need to start with human cancer genomes and phenotypes and ‘reverse translate.’ Unlike only a few years ago, we now have powerful human data."

Circle Pharma to Present Late-Breaking Data at AACR 2025 Demonstrating Mechanism of Action for Novel Cyclin A/B RxL Inhibitors

On April 22, 2025 Circle Pharma, a clinical-stage biopharmaceutical company focused on developing cell-permeable macrocycle therapeutics, reported a late-breaking poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 in Chicago, Illinois (Press release, Circle Pharma, APR 22, 2025, View Source;utm_medium=rss&utm_campaign=circle-pharma-to-present-late-breaking-data-at-aacr-2025-demonstrating-mechanism-of-action-for-novel-cyclin-a-b-rxl-inhibitors [SID1234652013]).

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The poster will present new mechanistic insights into the anti-cancer activity of cyclin A/B RxL inhibitors, including their impact on DNA repair pathways and mitotic progression in E2F-high cancers. Circle Pharma’s CID-078, an oral macrocycle Cyclin A/B RxL inhibitor, is being evaluated in a Phase 1 clinical study (NCT06577987).

Presentation Details
Title: Anti-cancer effect of Cyclin A/B RxL inhibitors is mediated in part by disruption
of the ATR/Chk1 DNA repair pathway

Session: Late-Breaking Research: Experimental and Molecular Therapeutics 3

Session Start: 4/29/2025 9:00:00 AM CT

Session End: 4/29/2025 12:00:00 PM CT
Location: Poster Section 52
Poster Board Number: 19
Abstract Number: LB296

The study, led by researchers from Circle Pharma in collaboration with the University of Oxford and Dana-Farber Cancer Institute, highlights the dual mechanism by which Cyclin A/B RxL inhibitors induce tumor regression.

Contributing Authors:
Catherine E. Gleason, Ranya Odeh, Frances Hamkins-Indik, Iolanda Vendrell, Roman Fischer, Benedikt Kessler, Shilpa Singh, Matthew Oser, Marie Evangelista, and Pablo D. Garcia, Circle Pharma Inc., South San Francisco, CA, University of Oxford, UK,
Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA

Guardant Health to Present Data Highlighting Advances in Multi-Cancer Detection and Liquid and Tissue Molecular Profiling at AACR

On April 22, 2025 Guardant Health, Inc. (Nasdaq: GH), a leading precision oncology company, reported it will present 18 abstracts, including four oral sessions, showcasing advances in multi-cancer detection and multiomic precision oncology testing enabled by its Guardant Infinity smart liquid biopsy platform at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, April 25-30 in Chicago (Press release, Guardant Health, APR 22, 2025, View Source [SID1234652030]).

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Key data that will be highlighted include:

An oral presentation of performance data for the Shield MCD test, selected for the National Cancer Institute’s Vanguard Study evaluating emerging multi-cancer detection (MCD) technology
Additional oral presentations on methylation-based cancer signal of origin identification, methylation profiling for lung cancer subtyping, and a real-world data analysis of BRAF mutations in non-small cell lung cancer
Analytical validation of Guardant’s new tissue profiling assay with integrated multiomics, showing a high success rate with minimal tissue input and supporting the acceleration of novel biomarker discovery
New methylation-based applications for liquid biopsy, including molecular subtyping of lung and breast cancer, rule-out of actionable genomic mutations, and identification of promoter methylation and MTAP deletions
"We look forward to sharing new data demonstrating the tremendous potential of the Guardant Infinity smart liquid biopsy platform to provide researchers and healthcare providers with a more complete picture of cancer, including genomics, epigenomics and more," said Helmy Eltoukhy, Guardant Health chairman and co-CEO. "This multidimensional view of cancer biology is enabling advances in precision oncology ranging from blood-based multi-cancer detection to identification of novel biomarkers that can help bring the next generation of cancer therapeutics to patients sooner."

The full abstracts for Guardant Health and a list of all abstracts being presented at AACR (Free AACR Whitepaper) 2025 can be found on the AACR (Free AACR Whitepaper) website.

For information and updates from the conference, follow Guardant Health on LinkedIn, X (Twitter) and Facebook or visit AACR (Free AACR Whitepaper) booth #1523.

Full List of Guardant Health Presentations

Date and Time (CDT)

Title

Abstract and Poster Section/Board

Guardant Product

Multi-Cancer

Sunday, April 27, 2:00-5:00pm

Analytical validation of a tissue-free epigenomic assay for circulating tumor DNA (ctDNA)-based molecular residual disease (MRD) detection in early-stage cancer

Abstract #692

Poster Section 29 Poster Board #25

Guardant Reveal

Sunday, April 27, 2:00-5:00pm

Development and characterization of a negative prediction algorithm for actionable mutations utilizing genomic and epigenomic profiling in cfDNA

Abstract #733

Poster Section 31 Poster Board #18

Guardant Infinity

Sunday, April 27, 3:00-5:00pm

A novel methylation-based classifier to identify cancer signal of origin using blood-based testing

Oral Minisymposium Session

Abstract #6365

Guardant Infinity Screening

Monday, April 28, 9:00am-12:00pm

Inferring immune and tissue cell type contributions to cell-free DNA (cfDNA) with a DNA methylation assay

Abstract #1951

Poster Section 28

Poster Board #19

Guardant Infinity

Monday, April 28, 9:00am-12:00pm

Detection of clinically actionable somatic variants in post-operative samples from patients with molecular residual disease

Abstract #1953

Poster Section 28

Poster Board #21

Guardant Reveal

Monday, April 28, 9:00am-12:00pm

Blood-based mapping of the personalized tumor epigenomic landscape

Abstract #1955

Poster Section 28 Poster Board #23

Guardant Infinity

Monday, April 28, 2:00-5:00pm

Analytical validation of cancer gene promoter methylation detection in cfDNA liquid biopsy assay

Abstract #3255

Poster Section 29 Poster Board #20

Guardant Infinity

Tuesday, April 29, 2:30-4:30pm

Evaluation of a plasma cell-free DNA methylation-based multi-cancer detection test

Oral Minisymposium

Session

Abstract #6425

Shield MCD Test

Tuesday, April 29, 2:00-5:00pm

A tumor-specific, methylation-based algorithm to identify MTAP gene deletions via tissue-free circulating tumor DNA

Abstract #5924

Poster Section 30 Poster Board #16

Guardant Infinity

Tuesday, April 29, 2:00-5:00pm

Analytical validation of a robust, integrated multiomics tissue assay powered by the Guardant Infinity platform

Abstract #5935

Poster Section 30 Poster Board #27

Guardant Infinity

Tuesday, April 29, 2:00-5:00pm

Landscape of epigenomic tumor fraction in large pan-cancer cfDNA cohort

Abstract #5936

Poster Section 30 Poster Board #28

Guardant Infinity

Wednesday, April 30, 9:00am-12:00pm

Analytical validation of a new plasma-only bioinformatics classifier to identify variants from clonal hematopoiesis (CH) in cfDNA liquid biopsy assays

Abstract #6603

Poster Section 9 Poster Board #10

Guardant Infinity

Lung

Sunday, April 27, 3:00-5:00pm

Non-invasive cell free DNA (cfDNA) methylation profiling for accurate proportional quantification of lung cancer subtypes

Oral Minisymposium

Session

Abstract #1142

Guardant Infinity

Tuesday, April 29, 9:00am-12:00pm

EGFR PACC mutations occur more frequently as compound mutations with better responses to EGFR TKIs

Abstract #4594

Poster Section 30 Poster Board #14

Guardant360

Tuesday, April 29, 2:30-4:30pm

Unveiling the potent anti-tumor activity and underlying mechanism of action of the novel pan-RAF inhibitor exarafenib in BRAF-mutated NSCLC

Oral Minisymposium Session

Abstract #6389

Guardant Inform

Breast

Monday, April 28, 2:00-5:00pm

Pre- and post-operative analysis of circulating tumor DNA in patients with breast cancer treated with neoadjuvant therapy using a tissue-free, methylation-based approach

Abstract #3360

Poster Section 32 Poster Board #30

Guardant Reveal

Monday, April 28, 2:00-5:00pm

Liquid based methylation profiling for quantification of breast cancer subtypes

Abstract #3247

Poster Section 29 Poster Board #12

Guardant Infinity

Colorectal

Monday, April 28, 9:00am-12:00pm

Employing joint modeling to support clinical interpretation of ctDNA dynamics during the treatment of advanced colorectal cancer

Abstract #2481

Poster Section 41 Poster Board #2

Guardant Inform

Genprex Announces Research Collaborators’ Abstract Published in Cancer Research Supplement Proceedings

On April 22, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its research collaborators’ abstract was published in the online Proceedings supplement of Cancer Research, a scientific journal published by the American Association for Cancer Research (AACR) (Free AACR Whitepaper) (Press release, Genprex, APR 22, 2025, View Source [SID1234652014]).

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Genprex collaborators will present the published abstract in a poster presentation at the 2025 AACR (Free AACR Whitepaper) Annual Meeting being held April 25-30, 2025 in Chicago, Illinois. The collaborators will present positive preclinical data from a study of Genprex’s lead drug candidate, Reqorsa Gene Therapy (quaratusugene ozeplasmid), for the treatment of KRASG12Cmutant non-small cell lung cancer (NSCLC).

"We are pleased to have our collaborators’ abstract selected for both publication and presentation before the world’s top cancer researchers, investigators and healthcare professionals," said Ryan Confer, President and Chief Executive Officer at Genprex. "This publication highlights research-driven advances against cancer, and we are gratified to be featured among those advances. We look forward to this week’s presentation of positive preclinical data on our lead drug candidate, REQORSA, as a potential therapeutic treatment for Ras inhibitor resistant lung cancer."

The featured Genprex-supported poster to be presented at AACR (Free AACR Whitepaper) 2025:

Title: Overcoming sotorasib acquired resistance in KRASG12C mutant NSCLC by TUSC2 gene therapy

Session Category: Experimental and Molecular Therapeutics

Session Title: Drug Resistance in Molecular Targeted Therapies 3

Session Date and Time: Tuesday, April 29 from 2-5 p.m. CT

Location: Poster Section 17

Poster Board Number: 12

Abstract Presentation Number: 5517

The featured Genprex-supported abstract to be presented at AACR (Free AACR Whitepaper) 2025:

Acquired resistance (AR) to Lumakras (sotorasib), the first FDA-approved KRASi, poses a significant challenge in the treatment of KRASG12C mutant NSCLC. Despite initial responses, patients invariably develop resistance, necessitating alternative therapeutic strategies. The mechanisms underlying AR include the emergence of additional mutations in the KRAS gene, reactivation of the KRAS pathway, or activation of alternative signaling pathways. TUSC2, a potent tumor suppressor gene with immunogenic properties, exhibits multifunctional activity by inhibiting downstream signaling pathways, including MAPK and mTOR; arresting the growth and proliferation of cancer cells; inducing tumor cell death; and activating innate and adaptive immune responses. In this study, researchers demonstrated that TUSC2 gene therapy (REQORSA) effectively overcomes sotorasib AR in KRASG12C mutant NSCLC mouse xenografts.

The data indicate that TUSC2 transfection significantly reduced colony formation in two AR cell lines. Transfection of TUSC2 also markedly increased apoptosis in AR cells. Re-expression of TUSC2 into AR PDXOs significantly decreased the viability of organoids compared with the empty vector. The H23AR tumors exhibited significantly lower sensitivity to sotorasib than their parental counterparts. However, treatment with REQORSA was highly effective in controlling tumor growth compared to treatment with sotorasib alone or the control groups. REQORSA alone also exhibited a strong antitumor effect on TC314AR PDXs. Sotorasib alone showed no significant antitumor activity in these models. However, a synergistic antitumor effect was observed when TC314AR PDX tumors were treated with the combination of REQORSA and sotorasib.

In conclusion, researchers demonstrated that REQORSA, alone or in combination with sotorasib,

induced apoptosis, inhibited colony formation, and showed significant antitumor efficacy in KRASG12C mutant sotorasib-acquired resistant xenograft and PDX tumors.

About Reqorsa Gene Therapy

REQORSA (quaratusugene ozeplasmid) consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles in a lipoplex form (the Company’s Oncoprex Delivery System), which has a positive charge. REQORSA is injected intravenously and specifically targets cancer cells. REQORSA is designed to deliver the functioning TUSC2 gene to negatively charged cancer cells while minimizing uptake by normal tissue. Laboratory studies conducted at MD Anderson show that the uptake of TUSC2 in tumor cells in vitro after REQORSA treatment was 10 to 33 times the uptake in normal cells.

Ensem Therapeutics Announces U.S. FDA IND Clearance to Initiate Clinical Development of ETX-636

On April 22, 2025 Ensem Therapeutics, Inc. (ENSEM) reported the clearance of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for ETX-636, a potential best-in-class novel allosteric pan-mutant-selective PI3Kα inhibitor and degrader, with its first-in-human study anticipated in the second quarter of 2025 (Press release, ENSEM Therapeutics, APR 22, 2025, View Source [SID1234652031]). In addition, ENSEM will present preclinical data on April 28th at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting in Chicago, Illinois, supporting a differentiated profile of ETX-636 and its potential to deliver clinical benefit to patients with tumors harboring activating PI3Kα mutations.

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Mutant PI3Kα is a key and frequent oncogenic driver across a broad spectrum of cancers, including up to 40 percent of hormone receptor-positive (HR+) /HER2-negative advanced breast cancers. However, wildtype PI3Kα is central to glucose homeostasis, and ATP-binding-site inhibitors target both mutant and wildtype PI3Kα, resulting in hyperglycemia which limits the clinical utility of these therapeutics.

"Targeting mutant PI3Kα within tumors while sparing wildtype PI3Kα in normal tissues represents a significant clinical challenge, which ETX-636 overcomes. The IND clearance is an important milestone for the company, and we are laser-focused on driving ETX-636 through clinical development," said Shengfang Jin, PhD, CEO and Co-Founder of ENSEM.

Dr. Jin noted that ETX-636 is the second novel ENSEM compound entering clinical development. ETX-197, an oral selective CDK2 inhibitor licensed to BeOne and being developed as BG-68501, is currently undergoing Phase 1 clinical development in advanced or metastatic solid tumors associated with CDK2 dependency.

Jeffery Kutok, MD, PhD, ENSEM’s Chief Scientific Officer, added, "ETX-636 is a potent pan mutant-specific allosteric PI3Kα inhibitor and degrader, which differentiates it from other compounds in its class. We are excited to evaluate ETX-636’s therapeutic potential in patients in our upcoming clinical trial. ENSEM also eagerly anticipates INDs for additional pipeline programs in 2026."

The initial first-in-human, Phase 1/2 study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of ETX-636 in participants with advanced solid tumors harboring a PI3Kα mutation. ETX-636 will be administered alone and in combination with fulvestrant, a selective estrogen receptor degrader approved for the treatment of advanced hormone receptor HR+/HER2- breast cancer.

Poster Details

Title: ETX-636, a novel allosteric pan-mutant-selective PI3Kα dual inhibitor and degrader with best-in-class potential
Poster ID: 1659
Sessions: Experimental and Molecular Therapeutics – Degraders and Glues 2
Date/Time: Monday, April 28, 2025, from 9am-12pm CT
Location: Poster session 18, Board 19
About ETX-636
ETX-636 was designed to optimally fit into a specific allosteric binding site in p110α, the catalytic subunit of PI3Kα. This allosteric binding site has been shown to selectively inhibit multiple activating mutant forms of PI3Kα, including hotspot kinase and helical domain PI3Kα mutants, while sparing wildtype PI3Kα. The selectivity of ETX-636 for mutant PI3Kα greatly reduces the risk for hyperglycemia and other wildtype PI3Kα-related adverse events compared to non-mutant selective PI3Kα inhibitors. In addition to its potent inhibitory effect on mutant PI3Kα catalytic activity, ETX-636 also leads to proteasome-dependent degradation of mutant PI3Kα, while sparing wildtype protein (a feature not seen with other pan-mutant allosteric inhibitors). This dual mechanism of action results in deep and durable pathway inhibition and induces concordant tumor regression in kinase and helical domain PI3Kα-mutant breast cancer xenograft models as monotherapy and in combination with fulvestrant. In preclinical toxicology studies, blood glucose levels after dosing in multiple species indicate that ETX-636 did not disrupt glucose homeostasis at predicted human efficacious doses.