On April 22, 2025 OS Therapies (NYSE-A: OSTX) ("OS Therapies" or "the Company"), a clinical-stage immunotherapy and Antibody Drug Conjugate (ADC) biopharmaceutical company, reported that the US Food & Drug Administration ("FDA") granted the Company’s meeting request to gain alignment on the surrogate endpoint to support Breakthrough Therapy Designation & Accelerated Approval of OST-HER2 in the Prevention of Recurrence of Fully Resected, Lung Metastatic Osteosarcoma (Press release, OS Therapies, APR 22, 2025, View Source [SID1234652033]). FDA granted a written response-only meeting and confirmed that its response would be received by mid-June 2025, in time for the Company to present the statistical analysis as part of the keynote presentation closing out major osteosarcoma conference MIB Factor on June 28, 2025 at 3:30pm MDT.

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"We are pleased that FDA agreed to the requested meeting forward and work within the timelines of the meeting type requested so that we would be able to share our analysis with the patient and physician communities that will be represented at MIB Factor in late June," said Robert Petit, Chief Medical & Scientific Officer of OS Therapies.

"We remain on track for an early third quarter submission and are hopeful to receive approval by year-end 2025 in order to bring this life saving treatment to patients in early 2026," said Paul Romness, CEO of OS Therapies.

OST-HER2 has received Rare Pediatric Disease Designation (RPDD) for osteosarcoma from the US FDA, and if it receives a conditional BLA via Accelerated Review prior to September 30, 2026, it will become eligible to receive a Priority Review Voucher (PRV) that it intends to immediately sell. The most recent PRV sale, valued at $150 million, occurred in February 2025.

The osteosarcoma treatment market was estimated at $1.2 billion in 2022 according to Data Bridge Market Research. Approximately 50% of patients are diagnosed with a lung metastasis at some point following surgical resection and chemotherapy. 3-year survival rates in patients who were not diagnosed with a metastasis are 59%. 3-year survival rates in patients who were diagnosed with pulmonary metastasis were 30%. The Company believes the market opportunity for OST-HER2 in the prevention of lung metastases is over $500 million.

OST-HER2, an immunotherapy for osteosarcoma using a HER2 bioengineered form of the bacteria Listeria monocytogenes to trigger a strong immune response against cancer cells expressing HER2, is being featured in the upcoming movie Shelter Me: The Cancer Pioneers. The movie offers a look into canine comparative oncology, a field that compares treatment of cancers in dogs to those in people and covers developing treatments for rare forms of cancer. A trailer for the movie is available here. The movie will air live nationally on PBS and be available via streaming on PBS’ website in early May 2025.

On April 22, 2025 Charles River Laboratories International, Inc. (NYSE: CRL), ahead of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago, IL, reported updates to its comprehensive portfolio of products and services supporting the discovery and development of novel oncology drugs (Press release, Charles River Laboratories, APR 22, 2025, View Source [SID1234652032]). Based solely on projected population growth, the number of cancer cases is predicted to increase to 35 million by 2050, and approximately 1 in 5 individuals will develop cancer in their lifetime.

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"Having worked on over 80 percent of the FDA-approved cancer therapies over the last five years, Charles River has a well-established track record of delivering innovative solutions for oncology researchers," said Julia Schueler, DVM, PhD, Therapeutic Area Lead, Oncology, Charles River. "We are consistently looking for ways to leverage new technologies and techniques to enhance our ability to deliver life-changing therapeutics to patients."

Developing a Fully Human Platform
Across several modalities, Charles River is developing and implementing humanized platforms to promote a more effective, translatable way of identifying human-specific drug targets and disease mechanisms, as well as treatments for individual patients. These methodologies include:

3D Tumoroids
Patient-derived xenograft (PDX) models are an increasingly common tool drug developers use when evaluating their new therapies, particularly immunotherapies. Charles River researchers have developed PDX-derived organoids, or tumoroids, which are self-organized 3D cell cultures that aim to mimic the structure, function, and cellular complexity of human organs, making them, in some ways, more translatable than animals.

Julia Schueler is presenting on PDX-derived breast cancer tumoroids on Sunday, April 27, 2025, at 2:00 p.m. and on establishing tumoroids from PDX tissue for in vitro applications on Tuesday, April 29, 2025, at 2:00 p.m.

2D Tumor Killing Assays
Charles River has a range of immune-mediated tumor-killing assays, including cytotoxic T-cell assays and natural killer (NK) cell assays. These assays are analyzed using flow cytometry and live cell imaging with IncuCyte. IncuCyte-based assays quantify the number of viable target cells and can be multiplexed with an apoptotic readout. The selection of target and effector cells can be tailored to the anticipated mechanism of action by selecting from a panel of validated target cell lines.

Ina Rohleff is presenting a comparison of NK-92MI cell line vs. primary NK cells derived from peripheral mononuclear blood cells on Wednesday, April 30, 2025, at 9:00 a.m.

Tumor Microenvironment
Leveraging knowledge of the tumor microenvironment (TME), Charles River is able to target different components of the TME that promote tumor growth and metastasis through anti-tumor immune suppression.

Louise Brackenbury is presenting on leveraging the tumor microenvironment for candidate vaccine screening on Tuesday, April 29, 2025, at 2:00 p.m.

Through a partnership with Cypre, Charles River clients can access Cypre’s 3D tumor models to predict therapeutic efficacy and mechanism of action in an accurate tumor microenvironment model. Utilizing Cypre’s patented 3D hydrogel technology and proprietary methods that synergize with Charles River’s PDX tumor model collection, the platform recreates the tumor microenvironment and enables predictive screening of innovative immune-oncology compounds.

Humanized Models
Charles River offers a comprehensive line of humanized mice models to support researchers in replicating human immune interactions and therapeutics responses in controlled, in vivo environments. Charles River has expanded its offering of humanized mouse models by introducing two new PBMC models that reduce the onset of graft versus host disease (GvHD). Along those lines, the Company offers a PBMC Select Humanization Kit, giving researchers the ability to leverage pre-qualified PMBCs and engraft specific NCG mice for improved study flexibility.

Eva Oswald is presenting on a PDX biobank in humanized mice and Steve Bronson is presenting on PMBC-humanized, MHC-deficient NCG mice that support human tumor xenographs without rapid onset GvHD, both beginning on Monday, April 28, 2025, at 9:00 a.m.

Leveraging AI in Oncology
The influence of artificial intelligence (AI) and machine learning (ML) tools is beginning to impact how researchers analyze cancer research data. Charles River recently collaborated with Revvity, Inc., to determine the feasibility of automatic organ volumetric analysis from 3D ultrasound images of mice using deep learning. Segmentation of 3D imaging data is labor intensive and measurements derived from human-annotated data are prone to inter-user variability and user error. To address these challenges, an AI model was generated to segment spleens from diverse 3D ultrasound images taken from several in vivo models.

David Harris is presenting on longitudinal ultrasound imaging as a novel pharmacodynamic marker of graft versus host disease progression in mice with Revvity on Monday, April 28, 2025, at 2:00 p.m.

Charles River is also partnering with Aitia to develop virtual control groups (VCGs) supported by Charles River’s extensive and well-categorized library of PDX data. VCGs, combined with insights from PDXs, allow researchers to predict tumor evolution over time in a specific model, reducing the need for control animals in future experiments and supporting the principles of the 3Rs (Replacement, Reduction, and Refinement).

Global Support for the RACE Act
As part of our broad oncology portfolio, Charles River, via the ITCC-P4 consortium, offers over 200 annotated, well-characterized, and Research to Accelerate Cures and Equity (RACE) for Children Act-compliant pediatric PDX models to accelerate time to clinic. These are critical models for research, as the U.S. Food and Drug Administration’s RACE Act requires nearly all oncology drugs to be tested for pediatric indications before approval.

"Globally, 400,000 children and adolescents develop cancer each year, and approximately one in four cannot be cured with currently available therapies," added Schueler. "Combined with our comprehensive portfolio of oncology drug discovery and development services, we are positioned to assess the safety and efficacy of new oncology treatments, specifically in this critically important patient population."

Charles River is proud to sponsor the Richi Childhood Cancer Foundation at AACR (Free AACR Whitepaper) 2025. Attendees can stop at Booth #1642 to learn how to actively support the mission to connect, support, and uplift children, youth with cancer, and survivors through the Richi House.

AACR Annual Meeting 2025: Transforming Oncology Drug Development
Charles River’s team has the capability to optimize workflows and maximize success across modalities including small and large molecules, cell and gene therapies, vaccines, and combination therapies. Charles River also recently launched an alliance with NJ Bio to optimize the development and manufacturing of antibody drug conjugates (ADCs.)

During the show, Charles River is presenting a Spotlight on HER2 therapy development on Monday, April 28, 2025 from 3:00-4:00 p.m. Visit Charles River during AACR (Free AACR Whitepaper) at Booth #1642 during the show, and visit criver.com to schedule a meeting, view a full schedule of activities, and explore oncology resources.

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.

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

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."