Mission Bio Launches Innovative Single-cell Genome Integrity Solution to Measure Whole Genome Copy Number Variation in Advanced Therapies and Tumor Samples

On September 26, 2024 Mission Bio, a leader in single-cell multiomics solutions for precision medicine, reported the launch of its Tapestri Genome Integrity CNV Solution (Press release, Mission Bio, SEP 26, 2024, View Source [SID1234646890]). This new product is the only single-cell high-throughput solution for measuring genome-wide copy number variants (CNVs) on the market and is designed to fulfill important needs in the critical areas of therapeutic development oncology research.

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While genome-editing and stem cell therapies continue to harbor tremendous promise for treating complex diseases but have encountered safety concerns associated with genomic instability. The latest FDA guidance suggests that assessment of genomic integrity should include chromosomal abnormalities like CNVs (i.e., duplications and deletions) as indicators of clinical safety. Conventional methods of assessing genomic instability such as g-banding fail to provide adequate answers due to their low throughput, as they may not analyze enough cells to detect aberrations. Oncology researchers have faced similar issues unlocking the potential of CNVs to serve as prognostic or therapeutic markers associated with tumor evolution, immune evasion, and therapeutic resistance. Existing bulk techniques provide only a limited view of CNV-based clonal architecture.

Mission Bio’s Tapestri Genome Integrity CNV Solution enables both CGT developers and oncology researchers with high-throughput, single-cell multiomic analysis of genome-wide CNVs, combined with automated reporting and multiplexing capabilities to ensure even more accessibility. Compared to current methods, the solution equips CGT developers with improved assay throughput to uncover potentially adverse chromosomal events, while simultaneously measuring genome editing outcomes. For oncology researchers, the solution enables the assessment of cell-to-cell aneuploidy and CNV events across the genome with the potential to co-measure SNVs and focal CNVs. Tapestri enables these researchers the rapid characterization of clonal heterogeneity behind tumorigenesis and therapy resistance.

"While working on a new way of investigating chromosomal instability and aneuploidy within tumors, we found that Mission Bio’s Tapestri Platform was able to provide the combination of high throughput and high sensitivity we needed," said Dr. Teresa Davoli of the NYU School of Medicine’s Institute for Systems Genetics and Department of Biochemistry and Molecular Pharmacology. "Mission Bio’s Tapestri platform gave our team the flexibility and customizability necessary to push our understanding of aneuploidy across thousands of single cells simultaneously." For details of her study see "KaryoTap Enables Aneuploidy Detection in Thousands of Single Human Cells."

"The launch of the Tapestri Genome Integrity CNV Solution represents a significant advancement in therapeutic development and cancer research," said Vanee Pho-Conners PhD, Senior Director of Product Management at Mission Bio. "By offering genome-wide CNV analysis at the single-cell level, we’re bridging a critical safety gap for CGT developers, who require precise genomic stability measurements to ensure the safety and efficacy of their therapies. This innovation highlights our commitment to equipping researchers with the most comprehensive tools to drive transformative breakthroughs in treatment."

The assay will be a key focus of Mission Bio at the 31st annual European Society of Gene & Cell Therapy Meeting, taking place Oct. 22-25 in Rome. For more information, please visit booth B24 or View Source

Tempus Announces Expansion of Collaboration with Takeda to Leverage Multimodal Real-World Datasets and Biological Model Systems in Oncology Research and Development

On September 26, 2024 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine and patient care, reported an expansion to its collaboration with Takeda (TSE:4502/NYSE:TAK) that takes a data-first approach to research and development, with the aim of enhancing Takeda’s oncology research and development efforts (Press release, Tempus, SEP 26, 2024, View Source [SID1234646889]).

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After an initial collaboration that leveraged Tempus’ de-identified multimodal datasets, this new agreement will expand upon that work. Takeda will combine Tempus’ multimodal real-world datasets and Tempus’ biological modeling capabilities to advance Takeda’s pipeline of cancer therapeutics, which includes antibody-drug conjugates (ADCs), small molecules, bispecifics and gamma delta T-cell therapies.

Takeda researchers will use Tempus’ analytics platform, Lens, to gain real-time access to de-identified patient records as well as a suite of AI-enabled tools to accelerate critical insights for Takeda’s drug development efforts. Initial work has resulted in research that will be presented at an upcoming medical congress.

In parallel, the two companies have embarked on a multi-phase biological modeling project leveraging one of the largest repositories of patient-derived tumor organoids in the world. Each organoid model can be used to reflect the biology of a distinct patient tumor, and then be genetically linked through Tempus’ next-generation sequencing assays to Tempus’ real-world multimodal dataset, potentially enhancing the predictability of a drug’s effectiveness. This approach enables efficient hypothesis generation and rapid validation that could guide early drug candidate prioritization. For this unique scope of work, Tempus is working with Takeda on a panel of 60 organoids spanning 10 cancer indications that closely reflect real-world patients to functionally evaluate several preclinical candidates.

"We are excited to expand our relationship with Takeda, combining real-world multimodal data and biological modeling capabilities to better understand targets of interest," said Ryan Fukushima, Chief Operating Officer at Tempus. "The results we’ve seen thus far demonstrate how the collaboration between the Takeda and Tempus teams can assist in efforts to accelerate Takeda’s growing oncology therapeutic pipeline, which may lead to the next generation of cancer treatments."

Bayer Submits Supplemental New Drug Application to U.S. FDA Seeking Expanded Indication for NUBEQA® (darolutamide) in Metastatic Hormone-Sensitive Prostate Cancer

On September 26, 2024 Bayer reported the submission of a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for the oral androgen receptor inhibitor (ARi) NUBEQA (darolutamide) in combination with androgen deprivation therapy (ADT) for the treatment of patients with metastatic hormone-sensitive prostate cancer (mHSPC) (Press release, Bayer, SEP 26, 2024, View Source [SID1234646888]).

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"Simply put, our ambition is to help more patients with prostate cancer," said Christine Roth, Executive Vice President, Global Product Strategy and Commercialization and Member of the Pharmaceuticals Leadership Team at Bayer. "We are proud of the role NUBEQA currently plays in the treatment of mHSPC and with this FDA submission, hope to expand the use of NUBEQA to more patients with the disease, regardless of chemotherapy use."

The submission is based on positive results from the investigational pivotal Phase III ARANOTE trial. Data from the trial were presented at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress and published in The Journal of Clinical Oncology.

NUBEQA is developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company.

About the ARANOTE Trial1
The ARANOTE trial (NCT04736199) was a randomized, double-blind, placebo-controlled Phase III study designed to assess the efficacy and safety of NUBEQA plus androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer (mHSPC). 669 patients were randomized to receive 600mg of NUBEQA twice daily or matching placebo in addition to ADT.

The primary endpoint of this study was radiological progression-free survival (rPFS), measured as time from the date of randomization to the date of first documentation of radiological disease progression or death due to any cause, whichever occurs first. Secondary endpoints included overall survival (time from randomization to the date of death from any cause), time from randomization to the date of first castration-resistant event, time to initiation of subsequent anti-cancer therapy, time to prostate-specific antigen (PSA) progression, PSA undetectable rates, time to pain progression, and safety assessments.

About NUBEQA (darolutamide)2
NUBEQA (darolutamide) is an androgen receptor inhibitor (ARi) with a distinct chemical structure that competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription.

In addition to the ARANOTE trial, NUBEQA is being evaluated in a robust clinical development program, which includes studies across various stages of prostate cancer, including in the ARASTEP Phase III trial evaluating NUBEQA plus ADT versus ADT alone in HSPC patients with high-risk biochemical recurrence (BCR) and no evidence of metastatic disease by conventional imaging and a positive PSMA PET/CT at baseline, as well as in the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP) led international Phase III co-operative group DASL-HiCaP (ANZUP1801) trial evaluating NUBEQA as an adjuvant treatment for localized prostate cancer with very high risk of recurrence. Information about these trials can be found at www.clinicaltrials.gov.

INDICATIONS
NUBEQA (darolutamide) is an androgen receptor inhibitor indicated for the treatment of adult patients with:

Non-metastatic castration-resistant prostate cancer (nmCRPC)
Metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel
IMPORTANT SAFETY INFORMATION

Warnings & Precautions
Ischemic Heart Disease – In a study of patients with nmCRPC (ARAMIS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA versus 2.5% receiving placebo, including Grade 3-4 events in 1.7% vs. 0.4%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA vs. 0.2% receiving placebo. In a study of patients with mHSPC (ARASENS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA with docetaxel vs. 2% receiving placebo with docetaxel, including Grade 3-4 events in 1.3% vs. 1.1%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA with docetaxel vs. 0% receiving placebo with docetaxel. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Discontinue NUBEQA for Grade 3-4 ischemic heart disease.

Seizure – In ARAMIS, Grade 1-2 seizure occurred in 0.2% of patients receiving NUBEQA vs. 0.2% receiving placebo. Seizure occurred 261 and 456 days after initiation of NUBEQA. In ARASENS, seizure occurred in 0.6% of patients receiving NUBEQA with docetaxel, including one Grade 3 event, vs. 0.2% receiving placebo with docetaxel. Seizure occurred 38 to 340 days after initiation of NUBEQA. It is unknown whether antiepileptic medications will prevent seizures with NUBEQA. Advise patients of the risk of developing a seizure while receiving NUBEQA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others. Consider discontinuation of NUBEQA in patients who develop a seizure during treatment.

Embryo-Fetal Toxicity – Safety and efficacy of NUBEQA have not been established in females. NUBEQA can cause fetal harm and loss of pregnancy. Advise males with female partners of reproductive potential to use effective contraception during treatment with NUBEQA and for 1 week after the last dose.

Adverse Reactions
In ARAMIS, serious adverse reactions occurred in 25% of patients receiving NUBEQA vs. 20% of patients receiving placebo. Serious adverse reactions in ≥1% of patients who received NUBEQA included urinary retention, pneumonia, and hematuria. Fatal adverse reactions occurred in 3.9% of patients receiving NUBEQA vs. 3.2% of patients receiving placebo. Fatal adverse reactions in patients who received NUBEQA included death (0.4%), cardiac failure (0.3%), cardiac arrest (0.2%), general physical health deterioration (0.2%), and pulmonary embolism (0.2%). The most common adverse reactions (>2% with a ≥2% increase over placebo), including laboratory test abnormalities, were increased AST, decreased neutrophil count, fatigue, increased bilirubin, pain in extremity and rash. Clinically relevant adverse reactions occurring in ≥2% of patients treated with NUBEQA included ischemic heart disease and heart failure.

In ARASENS, serious adverse reactions occurred in 45% of patients receiving NUBEQA with docetaxel vs. 42% of patients receiving placebo with docetaxel. Serious adverse reactions in ≥2% of patients who received NUBEQA with docetaxel included febrile neutropenia (6%), decreased neutrophil count (2.8%), musculoskeletal pain (2.6%), and pneumonia (2.6%). Fatal adverse reactions occurred in 4% of patients receiving NUBEQA with docetaxel vs. 4% of patients receiving placebo with docetaxel. Fatal adverse reactions in patients who received NUBEQA included COVID-19/COVID-19 pneumonia (0.8%), myocardial infarction (0.3%), and sudden death (0.3%). The most common adverse reactions (≥10% with a ≥2% increase over placebo with docetaxel) were constipation, rash, decreased appetite, hemorrhage, increased weight, and hypertension. The most common laboratory test abnormalities (≥30%) were anemia, hyperglycemia, decreased lymphocyte count, decreased neutrophil count, increased AST, increased ALT, and hypocalcemia. Clinically relevant adverse reactions in <10% of patients who received NUBEQA with docetaxel included fractures, ischemic heart disease, seizures, and drug-induced liver injury.

Drug Interactions
Effect of Other Drugs on NUBEQA – Combined P-gp and strong or moderate CYP3A4 inducers decrease NUBEQA exposure, which may decrease NUBEQA activity. Avoid concomitant use.

Combined P-gp and strong CYP3A4 inhibitors increase NUBEQA exposure, which may increase the risk of NUBEQA adverse reactions. Monitor more frequently and modify NUBEQA dose as needed.

Effects of NUBEQA on Other Drugs – NUBEQA inhibits breast cancer resistance protein (BCRP) transporter. Concomitant use increases exposure (AUC) and maximal concentration of BCRP substrates, which may increase the risk of BCRP substrate-related toxicities. Avoid concomitant use where possible. If used together, monitor more frequently for adverse reactions, and consider dose reduction of the BCRP substrate.

NUBEQA inhibits OATP1B1 and OATP1B3 transporters. Concomitant use may increase plasma concentrations of OATP1B1 or OATP1B3 substrates. Monitor more frequently for adverse reactions and consider dose reduction of these substrates.

Review the Prescribing Information of drugs that are BCRP, OATP1B1, and OATP1B3 substrates when used concomitantly with NUBEQA.

For important risk and use information about NUBEQA, please see the accompanying full Prescribing Information.

About Metastatic Hormone-Sensitive Prostate Cancer
Prostate cancer is the second most common cancer in men and the fifth most common cause of cancer death in men worldwide.3 In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, including almost 300,000 cases in the U.S., and about 375,000 died from the disease worldwide.4,5

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Approximately 10% of men will already present with mHSPC when first diagnosed.6,7,8 Men with metastatic hormone-sensitive prostate cancer (mHSPC) will start their treatment with hormone therapy, such as ADT, androgen receptor inhibitor (ARi) plus ADT or a combination of the chemotherapy docetaxel and ADT. Despite this treatment, most men with mHSPC will eventually progress to castration-resistant prostate cancer (CRPC), a condition with limited survival.

858 Therapeutics Announces $50 Million Series B Financing

On September 26, 2024 858 Therapeutics reported that it has raised $50 million in a Series B financing led by Avidity Partners with participation from Insight Partners, Mirae Asset Capital, and Alexandria Venture Investments, as well as existing investors Versant Ventures, NEA, and Logos Capital (Press release, 858 Therapeutics, SEP 26, 2024, View Source [SID1234646887]). As part of the financing, Monal Mehta, Ph.D., Managing Director at Avidity Partners, will join the company’s Board of Directors.

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"We are pleased to have strong backing from an elite group of life science investors," said Jeffrey Stafford, Ph.D., CEO of 858 Therapeutics. "The Series B financing is a testament to the 858 team and the progress we’ve made across our portfolio, including the advancement of our PARG inhibitor ETX-19477 into the clinic."

858 Therapeutics plans to use the proceeds to advance its pipeline of small molecule therapeutics. The company’s lead asset, ETX-19477, is a potent and selective inhibitor of the DNA repair protein PARG and is currently being evaluated in patients with advanced solid tumors. Data from the ongoing clinical trial will provide insights for advancing ETX-19477 through clinical development and for understanding the patients who may benefit most from PARG inhibition.

The Phase 1 trial for ETX-19477 is a multi-center, open-label, dose escalation and expansion study designed to evaluate safety, tolerability, dose, pharmacokinetics, pharmacodynamics, and preliminary efficacy. The dose escalation portion of the study is preferentially enrolling patients with specific tumor types and genetic alterations that are likely to confer sensitivity to PARG inhibition. Once a recommended dose for expansion is identified, the company will initiate tumor- and biomarker-focused Phase 2 cohorts. For more information, visit www.clinicaltrials.gov (NCT06395519).

"858 Therapeutics has a seasoned management team with a track record of drugging challenging targets," said Dr. Mehta. "We are excited to partner with the company to help address unmet needs across a variety of disease types in an effort to bring transformative medicines to patients."

About ETX-19477

The company’s lead asset, ETX-19477, is a novel and potent small molecule inhibitor of PARG, a glycohydrolase that plays a pivotal role in the regulation of DNA repair mechanisms. Pharmacological inhibition of PARG results in hyperPARylation, which leads to the death of cancer cells undergoing replication stress. In multiple animal models, ETX-19477 shows potent tumor growth inhibition, which is associated with specific genetic biomarkers. 858 Therapeutics is evaluating ETX-19477 in a Phase 1 study in patients with advanced solid tumors at multiple sites in the U.S. For more information on the Phase 1 study, please visit: View Source

RefleXion Showcases Pioneering Multi-Target Treatment (MTT) and Early Clinical Highlights at ASTRO

On September 26, 2024 RefleXion Medical, an external-beam theranostic oncology company, reported it will showcase Multi-target Treatment (MTT), the groundbreaking upgrade for its RefleXion X1 platform (Press release, RefleXion, SEP 26, 2024, View Source [SID1234646886]). This innovation allows physicians to combine SCINTIX biology-guided radiotherapy with conventional stereotactic body radiotherapy (SBRT) in a single plan to treat patients with metastatic disease. The company will also highlight learnings from its early patient treatments.

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RefleXion will showcase MTT at the American Society for Radiation Oncology (ASTRO) Annual Meeting, Sept. 29 – Oct. 3, in Washington D.C. (booth #1313), where researchers will present new scientific evidence in 22 presentations, including four oral presentations, showcasing the X1 machine with SCINTIX therapy.

"The results from our first-year patient treatments are highly encouraging," said Sean Shirvani, M.D., M.P.H., chief medical officer at RefleXion. "We are consistently observing that SCINTIX therapy detects and adapts to tumor motion in an autonomous fashion. In the lung, this means that less normal lung tissue is damaged by the ablative zone of radiotherapy. And in the bone, our system provides insurance against unanticipated shifts by the patient when the radiotherapy beam is on.

"Moreover, our clinical partners recognize the immense value of having positron emission tomography or PET data underpinning both treatment planning and delivery. This information has, in several cases, influenced the course of patient therapy," continued Shirvani.

The RefleXion X1 with SCINTIX therapy uses information from an injected PET radiopharmaceutical produced by the cancer itself in real time to determine where to deliver radiation. Because PET data is used to direct treatment, it is also an integral part of the treatment planning process.

In early SCINTIX therapy cases, PET data during treatment planning or delivery demonstrated tumor progression, uncovered additional tumors, or confirmed significant tumor movement in bone tumors, which are generally considered relatively immobile. In the bone tumor cases, SCINTIX technology accurately followed the live PET signal and delivered the dose as planned, a feat not achievable with conventional image-guided radiotherapy.

The following oral presentations highlight some of the new research being presented at ASTRO 2024. Detailed information on all 22 presentations may be found here.

Sunday, Sept. 29, 3:37 pm, room 147 – Scientific Development and Clinical Deployment of BgRT
Monday, Sept. 30, 3:00 pm, room 145 – Early Clinical Insights into FDG-Guided Radiotherapy Planning and Delivery on a Novel PET-Linac Platform in Patients with Lung and Bone Lesions: Pioneering Real-Time Biology-guided Radiotherapy
Monday, Sept. 30, 3:50 pm, room 145 – Feasibility of Biology-guided Radiotherapy with Gallium-68 PSMA-11 Radiotracer for Bony Metastases in Prostate Cancer Patients
Tuesday, Oct. 1, 8:40 am, room 152 – Impact of Synthetic PET Evaluation Prior to PET-Guided Functional Modeling in Optimizing Patient Selection for Biology-guided Radiotherapy
RefleXion’s booth will feature a multi-target SCINTIX technology interactive demonstration and SCINTIX treatment planning demonstrations.