ORIEN to Present Abstracts at 65th Annual ASH Meeting Utilizing Aster Insights’ Avatar Platform

On December 8, 2023 Aster Insights, the leading provider of scientific and clinical intelligence for oncology discovery, reported upcoming research presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s (ASH) (Free ASH Whitepaper) 65th Annual Meeting, December 9-12 in San Diego, CA (Press release, Aster Insights, DEC 8, 2023, View Source [SID1234638349]).

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Three abstracts will be shared at this year’s ASH (Free ASH Whitepaper) meeting by Moffitt Cancer Center, which co-founded the Oncology Research Information Exchange Network (ORIEN) in 2006. The investigations all utilize patient data from Aster Avatar, the best-in-class, deepest multimodal dataset for discovery research in oncology.

The schedule of presentations and highlights include:

Rafael Renatino-Canevarolo, PhD, Ex Vivo Mathematical Myeloma Advisor (EMMA) – a Clinical, Molecular, and Phenotypic Platform to Tailor Personalized Therapeutic Strategies for Multiple Myeloma (abstract 2280)
Presentation time: Saturday, December 9, 2023, 5:30 PM-7:30 PM

Examining the impact of a state-of-the-art platform for clinical-informed decisions, research advancement, and preclinical compound testing called the "Ex Vivo Mathematical Myeloma Advisor" (EMMA) on drug discovery and understanding of drug interaction for multiple myeloma patients.

Praneeth Reddy Sudalagunta, PhD, Selinexor Disrupts Epigenetic Programing and Modulates Immunogenicity in Multiple Myeloma (abstract 3301)
Presentation time: Sunday, December 10, 2023, 6:00 PM-8:00 PM

Investigating the cellular mechanisms of Selinexor (SELI) resistance leading to an immunogenic cell state.

Ciara L. Freeman, PhD, MSc, FRCPC, MRCP, Single Cell RNA Sequencing of Sequential Samples before and after BCMA-Directed CAR-T Reveal Features Associated with Non-Durable Response, Exhausted T-Cells and Decreased Expression of Genes Encoding Key Surface Targets in Particular in Patients with Extramedullary Disease (abstract 3304)
Presentation time: Sunday, December 10, 2023, 6:00 PM-8:00 PM

Longitudinal single cell analysis to identify factors associated with response, or therapeutic failure, to FDA approved anti-B-cell maturation antigen (BCMA) directed CAR-T cell therapy.

"ASH is a unique opportunity to demonstrate the impactful discoveries resulting from ORIEN and Aster Insights’ discovery solutions," said Anand Shah, MD, Aster Insights CEO. "This year, we are proud to showcase our collaborative work with Moffitt Cancer Center’s scientists that continues to push the envelope of multiple myeloma research and patient care."

SkylineDx Presents Pioneering Data at ASH 2023: SKY92 Risk Stratification in Multiple Myeloma Patients

On December 8, 2023 SkylineDx, an innovative diagnostics company focused on research & development of molecular diagnostics reported the release of new data during the American Society of Hematology (ASH) (Free ASH Whitepaper) 2023 Annual Meeting (Press release, SkylineDx, DEC 8, 2023, View Source [SID1234638348]). Dr. Noa Biran, Associate Professor at Hackensack Meridian School of Medicine, will present two posters from the pioneering US study on risk assessment in multiple myeloma. These studies were conducted within the PRospective Observational Multiple Myeloma Impact Study (PROMMIS; NCT02911571) trial, which aim to assess the impact of SKY92 on risk stratification and treatment decisions in clinical practice for newly diagnosed multiple myeloma patients. Multiple Myeloma (MM) is a complex hematologic malignancy marked by genetic instability, variable survival rates, and diverse treatment responses. The SKY92 gene expression profiling (GEP) assay is a valuable tool for stratifying MM patients into high-risk and standard-risk groups for disease progression and survival. Despite advances in MM treatments, a subset of high-risk patients still faces limited benefits from current therapies. This study aims to assess risk classification based on conventional risk markers and the SKY92 risk classifier, providing insights for optimizing risk-adapted treatment.

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A total of 251 MM patients were enrolled in this prospective US multicenter trial. The results based on prospective real-world clinical data show significant differences in progression-free survival (PFS) and overall survival (OS) between SKY92 standard-risk and high-risk patients, confirming SKY92’s reliability as a prognostic marker.

Integrating R-ISS staging with SKY92 classification, as previously reported [Kuiper et al. 2020], resulted in three risk groups. This combined classification method identified a larger number of high-risk patients with a significantly shorter OS and PFS when compared to the R-ISS alone.

The trial results reveal a significant discrepancy between risk stratification derived from conventional clinical practice assessments and that determined by SKY92. Integration of SKY92 into clinical practice enables the identification of a subset of high-risk patients characterized by substantially shorter PFS and OS. These findings support the added value of integrating SKY92 into clinical practice for precise risk assessment of patients. Furthermore, the integration of SKY92 resulted in increased physician confidence in assessing patient risks.

Jvalini Dwarkasing, Chief Scientific Officer of SkylineDx, expressed the company’s enthusiasm for these presentations, saying, "At SkylineDx, our mission is to advance the field of hematological diagnostics and ultimately improve patient care. We are proud that Dr. Biran will be presenting the insights from these groundbreaking studies that have the potential to transform the way we understand and treat hematologic disorders."

Dr. Noa Biran’s presentations at ASH (Free ASH Whitepaper) 2023 promise to challenge risk stratification in multiple myeloma, with implications for personalized treatment approaches and improved patient outcomes.

MEDSIR presents promising results at SABCS 2023: ATRACTIB and DEBBRAH trials hold potential to improve the lives of advanced breast cancer patients

On December 8, 2023 MEDSIR, a global leader in oncology research, reported significant breakthroughs in the fight against aggressive breast cancer at the 46th San Antonio Breast Cancer Symposium (SABCS) that have the potential to transform the lives of breast cancer patients (Press release, MedSIR, DEC 8, 2023, View Source [SID1234638347]). The results from the ATRACTIB trial, focusing on advanced triple-negative breast cancer (TNBC), and DEBBRAH Cohort 5, centered on HER2[+] or HER2-Low advanced breast cancer with leptomeningeal carcinomatosis, offer new hope for patients with limited treatment alternatives.

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ATRACTIB: Transforming the Landscape for TNBC Patients

This phase II clinical trial highlights the success of a combined therapy approach as a first-line treatment for advanced TNBC patients. Specifically, the combination of anti-PD-L1 atezolizumab and antiangiogenic bevacizumab with chemotherapy agent paclitaxel, demonstrated significant antitumor activity. This breakthrough holds particular significance since most of the patients included in this trial had PD-L1 negative tumors.

Dr. María Gion, Medical Oncologist at Ramón y Cajal University Hospital and first author of the study, presented the study’s outcomes, revealing a median progression-free survival of 11 months—a substantial delay in cancer progression compared to previous data on similar patient populations—. Impressively, 63% of patients responded positively to the treatment, achieving 13 complete responses and 50 partial responses, with a median duration of response reaching 10 months. Notably, the clinical benefit was observed in 79% of patients.

In the trial, 100 adult patients with untreated advanced TNBC received intravenous atezolizumab, bevacizumab, and paclitaxel until disease progression, intolerable toxicity, death, or patient withdrawal. The safety considerations revealed that peripheral neuropathy (68%) and fatigue (62%) were the most common side effects. Grade 3/4 adverse events, occurred in 47% of patients, primarily peripheral neuropathy (13%) and neutropenia (12%). Importantly, there were no drug-related deaths.

The combination of immunotherapy, antiangiogenic therapy, and chemotherapy demonstrated a manageable safety profile and merits further research for PD-L1-negative advanced TNBC patients.

DEBBRAH Cohort 5: Shaping the future for HER2[+] and HER2-Low Advanced Breast Cancer with Leptomeningeal Carcinomatosis

The DEBBRAH trial, focusing on HER2[+] or HER2-Low advanced breast cancer patients with brain metastases and/or leptomeningeal carcinomatosis, showcased promising results from Cohort 5, which specifically included patients with pathologically confirmed leptomeningeal carcinomatosis. This rare but serious complication occurs in approximately 10% of advanced breast cancer patients and is associated with poor outcomes and limited therapeutic options. Currently, there are no specific drugs approved for metastatic breast cancer patients with brain metastases and/or leptomeningeal carcinomatosis, and there is no consensus on how to manage these cases.

This trial was designed to address this gap in knowledge, aiming to investigate whether an antibody-drug conjugate called trastuzumab deruxtecan, which has shown promise in treating breast cancer that has spread to the brain and elsewhere in the body, can help improve the treatment for this specific group of patients. The antibody-drug conjugate, under evaluation demonstrated notable activity with no new safety concerns. The results of the Cohort 5, which included 7 patients, were presented by Dr. Marta Vaz, Medical Oncologist at Hospital Professor Doutor Fernando Fonseca, showing a remarkable median overall survival of 13.3 months, meeting the primary endpoint. Five patients (71.4%) experienced prolonged disease stabilization for at least 24 weeks, and the median progression-free survival was 8.9 months. The safety profile was consistent with previous studies.

The results of the DEBBRAH trial are extremely interesting and suggest that leptomeningeal carcinomatosis may become a more treatable condition with the introduction of new antibody-drug conjugates targeting HER2. These encouraging data warrant further investigation to address the unmet need in this difficult-to-treat disease.

ABOUT ATRACTIB

The ATRACTIB phase II study explored a combined treatment approach for advanced triple-negative breast cancer (a type of breast cancer characterized by the fact that the cancer cells don’t have estrogen or progesterone receptors, and do not have any or much of a protein called Human Epidermal Growth Factor Receptor-2). The trial evaluated the first-line therapy atezolizumab (an immunotherapy drug), bevacizumab (an antiangiogenic drug), and paclitaxel (a chemotherapy agent) in a cohort of 100 patients, regardless of their PD-L1 tumor expression. Primarily, the study looked at progression-free survival, or how long the disease remained stable. Secondly, it also assessed overall survival, response to treatment, and safety. The results were encouraging, showing that this combination treatment had a positive impact on advanced TNBC patients, even when tumors didn’t express PD-L1. Additionally, the safety profile of the treatment was consistent with what was known from previous studies.

ABOUT DEBBRAH

The DEBBRAH phase II trial was designed to evaluate the efficacy and safety of trastuzumab deruxtecan in patients with HER2[+] and HER2-low advanced breast cancer (meaning that tumors were characterized by the presence of either high or low levels of HER2 protein) with a history of brain metastases and/or leptomeningeal carcinomatosis. Patients were enrolled into one of five cohorts based on the HER2 protein level and type of central nervous system involvement. At SABCS 2023, results of Cohort 5 were presented, which specifically included 7 patients with leptomeningeal carcinomatosis. The main objective for this cohort was to measure the overall survival, or amount of time from the start of the treatment until death from any cause. With a median follow-up of 12 months (range, 2.5-18.6), results showed that treatment with trastuzumab deruxtecan led to a remarkable median overall survival of 13.3 months, meeting the primary endpoint. Five (71.4%) patients experienced a prolonged stabilization of their disease for at least 24 weeks, and the median time before their disease worsened again (known as progression free survival), was 8.9 months.

STORM Therapeutics’ First-in-Class tRNA methyltransferase inhibitor of METTL1 to be Presented at the 65th Annual ASH Conference

On December 8, 2023 STORM Therapeutics Ltd. (STORM), the clinical stage biotechnology company discovering and developing novel small molecule therapies targeting RNA modifying enzymes (RMEs) for oncology and other diseases, reported that data on its METTL1 tRNA methyltransferase inhibitor will be presented by collaborator Dr Konstantinos Tzelepis at the 65th Annual ASH (Free ASH Whitepaper) Meeting in San Diego, USA on Friday 8 December, 2023 (Press release, STORM Therapeutics, DEC 8, 2023, View Source [SID1234638345]).

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The presentation entitled "Overcoming Therapy Resistance in AML Using Novel Epitranscriptomic Modalities" details results showing that pharmacological inhibition of a tRNA methyltransferase affects tumour cell growth in in vitro and in vivo models.

Preclinical data demonstrated that:

STORM METTL1 inhibitors exhibit METTL1 inhibition in vitro at low nanomolar concentrations while displaying high selectivity over other RNA and protein methyltransferases.
Mechanistically, METTL1 inhibition leads to reduced tRNA methylation and reduced stability of a subgroup of tRNAs.
In several cancer models, METTL1 inhibition impairs cell proliferation and cell cycle progression accompanied by elevation of differentiation markers.
In vivo, METTL1i treatment induced strong tumour growth inhibition in cell-line derived xenografts and syngeneic in vivo cancer models (haematological and solid malignancies).
Jerry McMahon, Chief Executive Officer & President of STORM Therapeutics, said: "It is very exciting to be able share for the first time that inhibition of the tRNA methyltransferase METTL1 with a small molecule shows anti-cancer properties. We continue to expand our pipeline of novel and proprietary chemistries to modulate enzymes which modify RNA. We look forward to advancing our first-in-class METTL1 program towards the clinic."

Dr Konstantinos Tzelepis, Principal Investigator at University of Cambridge and Cambridge Stem Cell Institute, commented: "We are very excited to present novel data on the development and characterisation of the first-in-class bioavailable tRNA methyltransferase inhibitor against METTL1. This is a fantastic collaborative effort which expands significantly the therapeutic landscape of the epitranscriptomics field and capitalises on our previous published work which shown that METTL1 is a key player in oncogenesis."

Details of the conference and presentation are as follow:

Presented Talk Title: Overcoming Therapy Resistance in AML Using Novel Epitranscriptomic Modalities
Presenter: Dr Konstantinos Tzelepis, Principal Investigator, University of Cambridge and Wellcome-MRC Cambridge Stem Cell Institute
Date and Time: 8 December, 2023 at 2:45pm PST

Actinium to Highlight Broad Potential of Targeted Radiotherapies Iomab-B and Actimab-A for Relapsed or Refractory and Elderly Acute Myeloid Leukemia Patients at the 65th ASH Annual Meeting & Exposition

On December 8, 2023 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of targeted radiotherapies, reported that clinical data from its Iomab-B and Actimab-A programs will be featured in four presentations at the 65th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (ASH) (Free ASH Whitepaper) being held in San Diego on December 9-12, 2023 (Press release, Actinium Pharmaceuticals, DEC 8, 2023, View Source [SID1234638338]). These include an oral presentation and two poster presentations detailing results from the completed and positive Phase 3 SIERRA trial of Iomab-B in patients age 55 and above with active relapsed or refractory acute myeloid leukemia (r/r AML) as well as one poster presentation of Phase 1b results from the novel Actimab-A + Venetoclax combination trial for r/r AML.

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Sandesh Seth, Actinium’s Chairman & CEO, said, "We are particularly excited for this year’s ASH (Free ASH Whitepaper) as our four presentations will highlight the differentiated capabilities of Iomab-B and Actimab-A. Better treatment options and outcomes are needed in AML, particularly for elderly patients and those with relapsed or refractory disease who represent a majority of the population. Our strong presence at ASH (Free ASH Whitepaper) is indicative of the potential that our targeted radiotherapies have in the management of patients with difficult to treat AML. We are progressing toward BLA and MAA filings for Iomab-B as well as initiating a pivotal trial Actimab-A next year, which will be significant steps toward achieving our vision of transforming the treatment of AML with our highly differentiated therapies."

Iomab-B and Actimab-A target CD45 and CD33, respectively, both of which are validated targets with significant expression on AML cells. As targeted radiotherapeutics, Iomab-B and Actimab-A have both demonstrated the ability to overcome genetic mutations, including TP53, producing high response rates and improving survival outcomes in patients with difficult to treat, high-risk r/r AML.

Details of Actinium’s presentations at ASH (Free ASH Whitepaper) are as follows:

Iomab-B Oral Presentation

Title: 131I-Apamistamab-Led Allogeneic Hematopoietic Cell Transplant Significantly Improves Overall Survival in Patients with TP53 Mutated R/R AML

Session Name: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities: Novel Conditioning Regimens for Myeloid Malignancies
Session Date: Sunday, December 10, 2023
Session Time: 9:30 AM – 11:00 AM Pacific Time
Presentation Time: 9:30 AM
Room: Marriott Marquis San Diego Marina, Pacific Ballroom Salons 18-19

Iomab-B Poster Presentations

Title: 131I-Apamistamab Effectively Achieved Durable Responses in Patients with R/R AML Irrespective of the Presence of Multiple High-Risk Factors

Session Name: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities: Poster I
Session Date: Saturday, December 9, 2023
Presentation Time: 5:30 PM – 7:30 PM Pacific Time
Location: San Diego Convention Center, Halls G-H

Title: High-Dose Targeted Radiation with 131I-Apamistamab Prior to HCT Demonstrated a Dose-Response for Durable Complete Remission in Patients with R/R AML

Session Name: 721. Allogeneic Transplantation: Conditioning Regimens, Engraftment and Acute Toxicities: Poster II
Session Date: Sunday, December 10, 2023
Presentation Time: 6:00 PM – 8:00 PM Pacific Time
Location: San Diego Convention Center, Halls G-H

Actimab-A Poster Presentation

Title: Updated Results from Phase 1 Study of Targeted Radiotherapy with Lintuzumab-Ac225 in Combination with Venetoclax in Relapsed/Refractory AML

Session Name: 616. Acute Myeloid Leukemias: Investigational Therapies, Excluding Transplantation and Cellular Immunotherapies: Poster I
Session Date: Saturday, December 9, 2023:
Presentation Time: 5:30 PM-7:30 PM
Location: San Diego Convention Center, Halls G-H