On December 7, 2023 Medidata, a Dassault Systèmes company and leading provider of clinical trial solutions to the life sciences industry, reported the launch of the Medidata Research Alliance (Press release, Medidata, DEC 7, 2023, View Source [SID1234638274]). This unique consortium brings together leading clinician-researchers and key opinion leaders from academia, non-profit organizations, and the life sciences industry to leverage Medidata’s expertise in Artificial Intelligence (AI) capabilities and clinical trial data to drive cutting-edge research into innovative treatments.

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The Medidata Research Alliance bridges insights from data into clinical practice, fostering scientific exploration that can be applied across multiple therapeutic areas.

"This initiative combines the deep expertise of physician-scientists with Medidata AI and data-driven research to help advance medical breakthroughs – ultimately supporting patients and the broader healthcare and life sciences communities," said Fareed Melhem, SVP, Medidata AI, Medidata. "Our collective ambition is to drive medical progress by drawing insights from the volume, depth, and richness of our historical clinical trial data."

One significant area of focus for the Research Alliance is providing a greater understanding of Chimeric antigen receptor T (CAR-T) cell therapy, a promising new therapeutic approach to treat cancer. CAR-T clinical developers face a number of challenges, including life-threatening side effects and a lack of relevant data. Medidata and academic researchers can now partner and leverage the insights from an exclusive CAR-T dataset, generated through the Medidata Data Collaboration Program. And through this effort, collaborators explore clinically impactful hypotheses and jointly conduct research that provides tangible improvements in patient care.

"It’s been a dream come true to partner with such a great group of people at Medidata and to utilize such a robust dataset to explore these hypotheses in a more granular and highly statistically powered way than a single institution may otherwise be able to," said Theodore Scott Nowicki, MD, PhD, Assistant Professor of Pediatric Hematology/Oncology at the David Geffen School of Medicine, UCLA.

Results from the Research Alliance collaborations will be presented in poster sessions at the 65th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, being held from December 9-12, 2023:

Poster 3626: The association of phosphorus disruption and neurotoxicity in patients undergoing CAR-T therapy
Poster 3629: Significant CRS risk model with T-cell engaging therapies
Poster 5018: Machine learning-based decision tree for clinical management of neurotoxicity in patients undergoing CAR-T therapy

On December 7, 2023 NextPoint Therapeutics, a clinical-stage biotechnology company developing precision immuno-oncology therapeutics targeting the novel HHLA2 pathway, reported the acceptance of its investigational new drug (IND) application by the U.S. Food & Drug Administration (FDA) for NPX887 (Press release, NextPoint Therapeutics, DEC 7, 2023, View Source [SID1234638273]). The company plans to initiate a first-in-human Phase I clinical trial in early 2024 evaluating NPX887 in patients with solid tumors known to express HHLA2, a tumor antigen strongly upregulated in many human cancers.

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"NPX887 represents our second clinical program targeting the HHLA2 checkpoint axis. NextPoint’s approach is to advance therapeutics targeting this axis from different angles to prevent tumors from cloaking themselves from the immune system," said Leena Gandhi, MD, PhD, Chief Medical Officer of NextPoint Therapeutics. "This important milestone allows us to advance our clinical evaluation of NPX887 and brings us one step closer to our goal of expanding treatment options for patients with cancer."

About NPX887

NPX887 is a fully human monoclonal antibody targeting HHLA2 (B7-H7), a novel immune checkpoint and tumor target antigen highly expressed in many cancers independently of PD-L1. NPX887 is designed to prevent immune escape in solid tumors by blocking KIR3DL3-mediated immunosuppression upon binding to HHLA2. Treatment with NPX887 is believed to promote T and NK cell antitumor activity within the tumor microenvironment.

On December 7, 2023 Mission Bio, a leader in single-cell multiomic solutions for precision medicine, reported new data generated by clinical researchers from leading biopharma companies and top academic institutions will be showcased at the 65th ASH (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Mission Bio, DEC 7, 2023, View Source [SID1234638272]). This announcement follows on the heels of the successful launch of the company’s scMRD Assay, to which the latest data on 48 samples from the Clínica Universidad de Navarra will be featured in a poster presentation at the conference. The widespread adoption of Tapestri for hematological research and therapeutic development signifies the importance of single-cell multi-omic data and indicates a potential new gold standard in precision medicine.

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At the American Society of Hematology (ASH) (Free ASH Whitepaper) conference, being held in San Diego on December 9-12, world-leading scientists will present results demonstrating Tapestri’s capabilities to uncover critical insights into clonal dynamics, therapeutic response mechanisms, and clones driving disease persistence and relapse, reflecting the platform’s unique capabilities and position in the market.

Included among the presentations is the first data demonstrating the potential actionability of Mission Bio’s Tapestri Single-Cell MRD (scMRD) AML Multiomics Assay, which will be presented from the lab of Felipe Prosper, MD, PhD, Co-director of the Hematology and Cell Therapy Unit at the Clínica Universidad de Navarra, highlighting its feasibility for assessing MRD in AML patients.

"Understanding the genetic heterogeneity at the single-cell level is crucial to the discovery and development of safer and more effective treatments for blood cancers," said Todd Druley, MD, PhD, Chief Medical Officer of Mission Bio. "We’re excited to see so many of our customers leverage Tapestri, validating the scientific merit of our technology, demonstrating its practical application in a clinical setting, and substantiating the impact of single-cell data to potentially transform care by guiding more personalized treatments in AML, multiple myeloma (MM), and other blood cancers."

Among the ASH (Free ASH Whitepaper) presentations, researchers from Dr. Prosper’s lab will present results demonstrating the concordance between Mission Bio’s scMRD assay and the gold-standard techniques for MRD, such as PCR and multiparameter flow cytometry (MFC), in AML patient samples. The data suggest the scMRD Multiomics Assay’s potential role in advancing targeted therapeutic approaches and its influence on treatment paradigms in the future.

In the presentation, "Minimal Residual Disease Detection By Single Cell DNA Sequencing Technology: A Feasible Approach for Clinical Application and Identification of the Landscape of MRD Clones," the researchers showed that Mission Bio’s scMRD AML Multiomics Assay:

Identified rare leukemic clones that were missed by conventional assays.
Distinguished clonality of clinically actionable mutations, compared to MFC and PCR data, which only designated MRD status.
Detected MRD-positive patients, which were deemed negative by MFC and PCR,
Differentiated between clonal hematopoiesis (CH) clones and leukemic clones.
"Patients suffering from relapsed AML have poor overall survival, necessitating more effective methods for MRD monitoring and detection to better understand the genomic landscape of clonal evolution," said Dr. Prosper. "This impressive data is a significant step towards demonstrating the feasibility of Mission Bio’s assay to detect and monitor MRD in AML patients, to ultimately enable more effective therapeutic strategies, including combination and targeted therapies, based on insights from single-cell DNA and protein multiomics for improved patient outcomes in the future."

These results follow Mission Bio’s successful commercial launch of the scMRD AML Multiomics Assay, designed to offer deep genotypic and immunophenotypic insights into MRD signatures within individual cells.

"Multiple Myeloma therapy, similarly to other blood cancers like AML, remains a challenge and huge unmet medical need because of the poor prognosis and high relapse rate. Tapestri’s ability to combine SNVs, CNVs, and surface protein expression at a single-cell level will be crucial to myeloma surveillance and understanding clonal evolution, and our ability to fully realize precision medicine," said Cedric Dos Santos, PhD, Director AML/MDS/MM, Translational Medicine, at Genentech.

Data from other blood cancer studies will be presented by leading researchers in over 24 presentations leveraging Mission Bio’s Tapestri Platform, showcasing its broad application in hematology. These studies from institutions including Genentech, Merck, MD Anderson Cancer Center, Broad Institute of MIT and Harvard, and St. Jude Children’s Research Hospital embody Mission Bio’s commitment to advancing hematological research. For a complete list of ASH (Free ASH Whitepaper) presentations using Tapestri, please visit our website.

On December 7, 2023 BostonGene, a leading provider of AI-driven molecular and immune profiling solutions, reported the selection of four abstracts for oral presentation, four abstracts as poster presentations, and two abstracts for online publication at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 9-12, 2023, in San Diego, California and virtually (Press release, BostonGene, DEC 7, 2023, View Source [SID1234638271]). BostonGene will exhibit in booth #2350.

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"We look forward to showcasing compelling data at ASH (Free ASH Whitepaper), which will underscore the pivotal role of molecular and immune profiling alongside advanced analytics in propelling precision medicine for individuals battling cancer," said Nathan Fowler, MD, Chief Medical Officer at BostonGene.

Details of the presentations are below:

Oral presentations:

Abstract: 601
Title: Clinical Outcomes of Patients with Relapsed/Refractory Follicular Lymphoma Treated with Tisagenlecleucel: Phase 2 Elara 3-Year Follow-up
Date & time: Sunday, December 10, 2023 | 4:30 PM
Location: Room 6CF – San Diego Convention Center
Presenter: Stephen J Schuster, MD, Abramson Cancer Center, University of Pennsylvania

This presentation highlights results from the Phase 2 ELARA trial that show efficacy, safety, pharmacokinetic, and exploratory biomarker analyses in relapsed/refractory follicular lymphoma patients treated with tisagenlecleucel after a median follow-up of more than 3 years. Higher baseline levels of CD8+ naive T cells are associated with improved long-term clinical outcomes.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center, Abramson Cancer Center, University of Pennsylvania, The University of Melbourne, Hospital Universitario, Oslo University Hospital, Royal Brisbane Hospital, University of Michigan, City of Hope National Medical Center, Moffitt Cancer Center, Hospices Civils de Lyon and Claude Bernard Lyon 1 University, Kyushu University Hospital, Tohoku University Graduate School of Medicine, University of Amsterdam, University of California San Francisco, University of Chicago, Royal Prince Alfred Hospital, Universidad de Sevilla, Oregon Health and Science University, University of Cologne, West German Cancer Center, University of Duisburg-Essen, IRCCS San Raffaele Scientific Institute, Hokkaido University, King’s College London, University of Kansas Medical Center, Ordensklinikum Linz Barmherzige Schwestern-Elisabethinen, Universitair Ziekenhuis Gent, University Hospital of Ulm, University of Bologna, Cambridge University, Novartis Pharmaceuticals Corporation, Novartis Pharma AG, Hôpital Saint-Louis, Ludwig-Maximilians-University Hospital

Abstract: 851
Title: A CD5 Gene Signature Identifies Diffuse Large B-Cell Lymphomas Sensitive to Bruton’s Tyrosine Kinase Inhibition
Date & time: Monday, December 11, 2023: 3:45 PM
Location: Room 6DE – San Diego Convention Center
Presenter: Alan Cooper, University of Chicago

This study highlights the discovery of a novel transcriptomic-based biomarker of BTK inhibitors (BTK-i) response in DLBCL patients. The biomarker, a CD5 gene signature, expands upon existing genetic DLBCL classifications by identifying DLBCL patients that can benefit from BTK-i, warranting further prospective validation in BTK-i clinical trials.

Research conducted in collaboration with the University of Chicago, City of Hope National Medical Center, The University of British Colombia, Janssen Research & Development, Centre for Lymphoid Cancer

Abstract: 905
Title: Machine Learning (ML)-Enabled Automation for High-Throughput Data Processing in Flow Cytometry
Date & time: Monday, December 11, 2023 | 3:45 PM
Location: Room 6CF – San Diego Convention Center
Presenter: Anna Kamysheva, MSc, BostonGene

This presentation describes BostonGene’s development of an ML-based algorithm for automated cell-type labeling that enables the detection of rare and/or new cell populations with high speed and accuracy. This algorithm is applicable in clinical settings, particularly for detecting hematological abnormalities and cancers.

Abstract: 983
Title: Addition of Acalabrutinib to Lenalidomide and Rituximab Induces High Complete Response Rates in Patients with Previously Untreated Follicular Lymphoma: Results of a Phase II Study
Date & time: Monday, December 11, 2023 | 5:30 PM
Location: Grand Hall C – Manchester Grand Hyatt San Diego
Presenter: Paolo Strati, MD, MD Anderson

The study demonstrates the safety and efficacy of lenalidomide and rituximab treatment in combination with acalabrutinib in previously untreated FL patients and highlights the use of bulk RNA sequencing with deconvolution to analyze the biological effects of this treatment combination on peripheral blood immune cells.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center

Poster presentations:

Abstract: 1676
Title: Preliminary Analysis of an Ongoing Phase II Study of Response-Adapted Therapy with Copanlisib and Rituximab for Untreated Follicular Lymphoma
Date & time: Saturday, December 9, 2023 | 5:30 PM – 7:30 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Rahul Lakhotia, MBBS, National Cancer Institute, National Institutes of Health

This study describes the preliminary analysis (including safety and efficacy) of an ongoing "window of opportunity" study of copanlisib, followed by response-adapted copanlisib and rituximab in treatment-naïve patients with follicular lymphoma (FL). Gene expression profiling was used to identify high-risk patients and find a predictive signature of response to PI3K inhibitor.

Research conducted in collaboration with the National Cancer Institute, National Institutes of Health and Mayo Clinic

Abstract: 3644
Title: A Novel Comprehensive Tumor-Informed Plasma cfDNA Assay to Monitor Minimal Residual Disease for Hematological and Solid Malignancies
Date & time: Sunday, December 10, 2023 |6:00 PM – 8:00 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Anastasiya Yudina, MSc, BostonGene

This presentation describes our cell-free DNA (cfDNA) testing platform for minimal residual disease (MRD) monitoring. It encompasses all major clinically reliable genetic hotspots events and can detect clinically relevant mutations in plasma samples before clinical manifestation.

Abstract: 3001
Title: Genomic and Transcriptomic Profiles of Blastoid and Pleomorphic Mantle Cell Lymphoma Are Distinct from Classic Histology Mantle Cell Lymphoma
Date & time: Sunday, December 10, 2023 |6:00 PM – 8:00 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Preetesh Jain, MD, MBBS, PhD, DM, MD Anderson

This presentation highlights the use of BostonGene’s integrated genomic and transcriptomic analysis platform to provide a comprehensive portrait of the molecular differences between blastoid and pleomorphic mantle cell lymphoma (MCL), revealing mechanistic insights that can be used for the development of more effective therapeutic strategies.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center

Abstract: 4872
Title: Long-Term Durable Responses in Relapsed/Refractory (r/r) ALL, DLBCL, and FL Patients Treated with Tisagenlecleucel and Its Association with Persistence of CAR T-Cells
Date & time: Monday, December 11, 2023: 6:00 PM – 8:00 PM
Location: Halls G-H – San Diego Convention Center
Presenter: Rakesh Awasthi, PhD, Novartis Institutes for BioMedical Research

This study highlights a positive association between chimeric antigen receptor (CAR) persistence in peripheral blood post tisagenlecleucel (tisa-cel) infusion and durable clinical responses across three indications.

Research conducted in collaboration with Novartis Institutes for BioMedical Research, Novartis Pharmaceuticals Corporation, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Intermountain Primary Children’s Hospital, Huntsman Cancer Institute, Spencer Fox Eccles School of Medicine at the University of Utah, Goethe University Frankfurt, University Hospital, Knight Cancer Institute, Oregon Health & Science University, Emory University School of Medicine, Medical University of Vienna, Université de Paris, Klinikum der Universität, LMU München, MD Anderson Cancer Center

Online publications:

Title: Test-the-Test: Clinical Utility of Comprehensive Whole Exome Sequencing and RNA-Seq for Patients with Lymphoma

This study shows an acceptable turn-around-time (TAT) of 8 days for the delivery of clinical reports that include clinically relevant findings and matched clinical trials, demonstrating the feasibility of using comprehensive WES and RNA-seq molecular profiling for lymphoma patients.

Research conducted in collaboration with The University of Texas MD Anderson Cancer Center

Title: Molecular Findings on Plasma Cell-Free DNA Analysis Among Adults with Histiocytic Neoplasms

This study describes the use of cell-free DNA (cfDNA) analysis to evaluate the molecular findings in adults with histiocytic neoplasms, highlighting cfDNA as a potential viable tool for discovering driver mutations in multiple cancers.

Research conducted in collaboration with The University of Alabama, Mayo Clinic

In addition to the poster presentations, the abstracts have been published online in the November supplemental issue of "Blood."

For more information, please visit the 65th ASH (Free ASH Whitepaper) Annual Meeting and Exposition website at View Source

ON December 7, 2023 Remix Therapeutics (Remix), a biotechnology company developing small molecule therapies designed to modulate RNA processing and address underlying drivers of disease, reported an upcoming presentation introducing REM-422, the Company’s potent, selective, oral small molecule messenger RNA (mRNA) degrader, as a potential treatment for MYB-driven cancers (Press release, Remix Therapeutics, DEC 7, 2023, View Source [SID1234638270]). Data describing the activity of REM-422 in AML xenograft models will be highlighted at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper), taking place from December 9-12, 2023 in San Diego. Results demonstrate oral dosing of REM-422 degrades MYB mRNA, inducing anti-leukemic activity in multiple xenograft models.

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"These compelling data support the therapeutic rationale of targeting MYB with REM-422 as a potential treatment for acute myeloid leukemia (AML) and myelodysplastic syndromes," said Peter Smith, Ph.D., President and Chief Executive Officer of Remix Therapeutics. "REM-422 is the first compound entering the clinic from the REMaster discovery platform and we look forward to the start of clinical development in patients with MYB-dysregulated malignancies."

Lead author Charles Kung, PhD, Vice President of Biology, concludes "MYB is an important oncogenic transcription factor that is dysregulated in the majority of AML and MDS patients and we look forward to bringing REM-422 to patients in need of new treatment options."

Details for the poster presentation are as follows:

Title: REM-422, a potent, selective, oral small molecule mRNA degrader of the MYB oncogene, demonstrates anti-tumor activity in mouse xenograft models of AML
Abstract Number: 1425
Session Name: Poster Session I – Presentations
Session Date and Time: Saturday, December 9, 2023, 5:30 PM-7:30 PM
Session Location: Poster Hall