On May 30, 2024 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that enrollment has been completed and dosing initiated for the fifth dose cohort of its Phase 1/2 study of ONCT-534 for the treatment of patients with metastatic castration-resistant prostate cancer who are relapsed or refractory to approved androgen receptor pathway inhibitors (ARPI) (Press release, Oncternal Therapeutics, MAY 30, 2024, https://investor.oncternal.com/news-releases/news-release-details/oncternal-announces-enrollment-completed-and-dosing-initiated [SID1234643864]). Patients in the fifth cohort are receiving ONCT-534, the company’s dual-action androgen receptor inhibitor (DAARI), at a dose of 600 mg taken orally once each day. The decision to proceed to this higher dose level was made by the study’s Safety Review Committee (SRC) after reviewing data from the fourth dose level of 300 mg ONCT-534 daily. An initial update on ONCT-534 safety and efficacy based on prostate-specific antigen (PSA) levels from this study is expected in the third quarter of 2024 and will include data from this 600 mg dose cohort.

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"We are encouraged by the rapid enrollment in the dose escalation portion of our Phase 1/2 study with ONCT-534. The drug has been well tolerated, with no dose limiting toxicities observed to date. We continue to open new sites and patient demand continues to be strong," said Salim Yazji M.D., Chief Medical Officer at Oncternal Therapeutics. "We are looking forward to sharing initial safety and efficacy data soon, which will include a larger, more robust set of clinical and biomarker results, as well as longer follow-up from the initial dosing cohorts."

About Study ONCT-534-101
Study ONCT-534-101 is a Phase 1/2, single-arm, open-label, multi-center study to evaluate the safety and tolerability, pharmacokinetics, and preliminary anti-tumor activity of ONCT-534 in patients with mCRPC who have relapsed or are refractory to approved ARPIs including enzalutamide, abiraterone, apalutamide, and darolutamide. After the safety and tolerability and preliminary antitumor activity of ONCT-534 have been assessed in Phase 1, Phase 2 will commence to further evaluate the safety and antitumor activity of ONCT-534 to support selecting an optimal dose.

On May 30, 2024 NuCana plc (Nasdaq: NCNA) reported that Hugh Griffith, Chief Executive Officer, and Don Munoz, Chief Financial Officer, will present and host one-on-one meetings at the Jefferies Global Healthcare Conference (Press release, Nucana BioPharmaceuticals, MAY 30, 2024, View Source [SID1234643863]).

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Event: Jefferies Global Healthcare Conference
Date: Thursday, June 6, 2024
Time: 12:30 PM EDT
Location: New York, NY

The presentation will be webcast live and available for replay under "Events & Presentations" in the Investors section of the Company’s website at www.nucana.com.

On May 30, 2024 – NH TherAguix (NHT), a phase II clinical stage biotechnology company specializing in the development of novel nanomedicine solutions applicable to precision radiotherapy in oncology, reported that its lead drug candidate, AGuIX, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) as a next-generation radio-enhancer for the treatment of malignant gliomas, and in particular glioblastoma (GBM), the most common and deadliest brain cancer globally (Press release, NH TherAguix, MAY 30, 2024, View Source [SID1234643862]).

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A nanodrug capable of improving the precision and effectiveness of radiotherapy without damaging surrounding tissues

The result of over 10 years of research, AGuIX is a nanodrug designed to meet the growing medical need in brain cancer, by significantly improving the efficacy and precision of radiotherapy directly within tumors.

Its structure mostly made of gadolinium provides it with strong contrast imaging properties, coupled with the potential to indirectly increase the delivered X ray dose. AGuIX thus enables precise tumor delineation via MRI and can significantly improve radiotherapy efficacy. AGuIX also boasts an excellent safety profile, as demonstrated by the results of the first In Human Phase Ib clinical trial NANORAD-1.

This new designation marks an important regulatory milestone for NH TherAguix, paving the way for accelerated development of its lead drug candidate, and is in line with the deployment of the company’s new development strategy, following the recent appointment of Vincent Carrère as CEO.

Vincent Carrère, a pharmaceutical industry expert, appointed as CEO to steer the late-stage clinical development of AGuIX

Last September, Vincent joined NH TherAguix to lead the final steps of AGuIX clinical developments through registration trials, until expected market approval. Formerly Vice President-Head of the Northern and Central Europe Region at Ipsen, he brings over 15 years of experience in thepharmaceutical and biotech industry. His deep knowledge of the processes and final stages of drug development and commercial launch will be instrumental in finalizing the upcoming developments of AGuIX.

"We are delighted to have received this FDA Fast Track designation for AGuIX in the treatment of malignant glioma. It demonstrates the strong interest of the US regulatory authorities in our nextgeneration radio-enhancer. We are convinced that AGuIX holds great potential to offer an effective clinical and therapeutic response to patients suffering from these deadly cancers, for whom existing treatments remain largely insufficient. 2024 should be a major turning point in the development of our promising nano-drug, and we look forward to sharing the first results from our clinical trials later this year," said Vincent Carrère, CEO of NH TherAguix.

Major clinical inflection points expected in H2 2024

AGuIX is currently being evaluated in four Phase II clinical trials, out of which three should provide major inflection points before the end of 2024:

• The Phase II NANORAD 2 study, conducted by Grenoble University Hospital (p.i. Pr. C. Verry) on 100 patients with multiple brain metastases, is evaluating AGuIX in combination with whole brain radiotherapy1. Recruitment has been finalized and results of the interim efficacy analysis are expected by the end of 2024 at the latest.

• The Phase II NANOBRAINMETS trial, conducted in collaboration with the Dana Farber Cancer Institute (p.i. Dr. A. Aizer), the world’s leading institute for adult and pediatric cancer research and treatment, is evaluating AGuIX in 134 patients with brain metastases in combination with stereotactic radiotherapy2. A futility analysis (50% of patients enrolled) is scheduled for August 2024 to assess the first potential effects of the treatment on patients. Validation of the continuation of the study would underline the strong therapeutic potential of AGuIX in the treatment of these patients. Initial results from an interim efficacy analysis are then expected in November 2024.

• The Phase I/II NANOGBM trial, conducted by Clermont Ferrand Centre Jean Perrin (p.i. Dr. J. Biau), is evaluating AGuIX in the treatment of glioblastoma in 62 patients. Results from the interim efficacy analysis are expected by the end of 2024.

"Glioblastoma are the most common type of malignant primary brain tumor and account for most deaths among patient with primary tumors. Although there has been progresses in understanding the biology of these tumors, the unmeet therapeutic need remains very important. This Fast Track designation will facilitate NH TherAguix more frequent interactions with the FDA as well as accelerated approval and priority review in glioblastoma indications. This program will be led in parallel to our ongoing clinical development program in brain metastases, where preliminary signal of efficacy of AGuIX has already been detected," concluded Olivier de Beaumont, CMO of NH TherAguix.

On May 30, 2024 MimiVax Inc, a clinical-stage biotechnology company developing immunotherapeutics for cancer and autoimmune diseases, reported that the United States Food and Drug Administration (FDA) has awarded a supplemental orphan drug designation to MimiVax’s SurVaxM vaccine to now include malignant glioma (Press release, MimiVax, MAY 30, 2024, View Source;utm_medium=rss&utm_campaign=us-fda-awards-supplemental-orphan-drug-designation-to-survaxm-to-now-include-treatment-of-malignant-glioma [SID1234643861]). Currently, SurVaxM vaccine is being studied as a treatment for newly diagnosed glioblastoma (nGBM) in a phase 2b randomized clinical study (the SURVIVE trial) which will assess the efficacy of SurVaxM in a large patient population with nGBM. This supplemental orphan drug designation greatly expands the potential for SurVaxM’s use in other forms of pediatric and adult malignant gliomas. An orphan drug designation plays a critical role in encouraging the development of treatments for rare diseases, ultimately improving the lives of patients who might otherwise have few or no treatment options.

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Gliomas are brain tumors that are initiated from glial cells that and support nerve cells in the brain and spinal cord. Gliomas comprise about 80% of all malignant primary brain tumors and include such tumors as astrocytomas, oligodendrogliomas and ependymomas.

"Receiving orphan drug designations for SurVaxM emphasizes the critical demand for novel and enhanced therapeutic options for people living with malignant gliomas," said Michael Ciesielski, Ph.D., CEO of MimiVax. "We hope that this designation may help to advance SurVaxM’s application for important indications beyond adult GBM including for pediatric brain cancers."

Orphan designation is granted by the FDA to advance the evaluation and development of new therapies intended to treat a rare disease or condition that generally affects fewer than 200,000 individuals each year in the United States. Under the Orphan Drug Act, regulatory agencies may offer certain benefits to expedite the approval process for orphan drugs, recognizing the urgent need for treatments for rare diseases. This can include accelerated review timelines and flexibility in clinical trial design, tax credits for qualified clinical trials, FDA user-fee benefits, and seven years of market exclusivity in the United States after new drug approval.

On May 30, 2024 Labcorp (NYSE: LH), a global leader of innovative and comprehensive laboratory services, reported that it will present several abstracts at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago (May 31 – June 4, 2024) (Press release, LabCorp, MAY 30, 2024, View Source [SID1234643860]).

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Labcorp’s oncology research highlights the company’s dedication to advancing meaningful and actionable insights that enhance the understanding of tumor biology and immune system mechanisms. These insights will contribute to facilitating patient access to novel targeted therapies with the end goal of improving patient outcomes.

"Labcorp is committed to deepening the understanding of cancer biology and enhancing therapeutic strategies. Through our presentations at the ASCO (Free ASCO Whitepaper) Annual Meeting investigating critical aspects of triple-negative breast cancer, liquid biopsy, and employing advanced machine learning algorithms for tumor profiling, we are paving the way for more accurate diagnostics and effective treatments," said Shakti Ramkissoon, M.D., Ph.D., MBA, vice president, medical lead for oncology at Labcorp. "These studies exemplify Labcorp’s deep scientific and medical expertise enabling delivery of compelling evidence through internal and external collaborations to drive advancements in precision oncology."

Of the eight abstracts to be presented at the ASCO (Free ASCO Whitepaper) Meeting, four were internal studies by Labcorp researchers and four were conducted in collaboration with research partners from premier academic institutions and medical centers.

Among those being presented by Labcorp researchers, the findings of two studies examine various aspects of triple-negative breast cancer (TNBC) to provide a deeper understanding of the factors causing tumor progression and therapy resistance in the disease. Triple-negative breast cancer accounts for approximately 10-15% of all breast cancers but disproportionately impacts Black women in the U.S., who have almost twice the rate of the disease compared to white women.

Session: Breast Cancer-Metastatic
Abstract: 1096 – Interaction between VEGF-A and immune checkpoint targets in triple-negative breast cancer suggests a mechanism of immune evasion and tumor progression.
Poster Board: 74
Date: Sunday, June 2, 9:00 a.m. – 12:00 p.m. CDT
Vascular endothelial growth factor (VEGF) promotes angiogenesis and potentially modulates tumor immune evasion in breast cancer. Labcorp researchers performed comprehensive genomic and immune profiling on 143 formalin-fixed paraffin-embedded breast cancer samples to investigate the interaction between VEGF and immune gene expression. In triple-negative breast cancer (TNBC) samples, VEGF was co-expressed with immune checkpoint genes such as PD-1 and PD-L1. Labcorp’s findings support that angiogenesis mediators may enhance immunosuppression, leading to immune evasion and tumor progression in TNBC.

Session: Breast Cancer—Metastatic
Abstract: 1095 – Novel HLA-Ilo/HLA-IIhi phenotype and immune evasion in triple-negative breast cancer.
Poster Board: 73
Date: Sunday, June 2, 9:00 a.m. – 12:00 p.m. CDT
Resistance and non-response to immunotherapy remain an unmet clinical need for patients with triple-negative breast cancer (TNBC). Aberrant expressions of human leukocyte antigens (HLAs) are one mechanism by which cancer cells evade immune response. Labcorp researchers performed a targeted RNA-sequence-based assay on 143 breast cancer patient samples, demonstrating that concurrent loss of HLA class I with increased HLA class II expression was associated with co-expression of biomarkers indicative of immune escape but not survival outcomes. These data offer an opportunity for developing novel approaches to overcome immunotherapy resistance in TNBC.

Session: Care Delivery/Models of Care
Abstract: 1554 – A machine learning algorithm based on multi-omics biomarkers for the detection of tumor microsatellite instability
Poster Board: 425
Date: Saturday, June 1, 9:00 a.m. – 12:00 p.m. CDT
Labcorp researchers will present data on an innovative machine learning model developed to predict microsatellite instability (MSI) status in patients with solid tumors using comprehensive genomic and immune profiling, independent of direct sequencing data from microsatellite sites. Researchers analyzed genomic and gene expression data from over 2,000 colorectal cancer samples to generate and test a model for predicting MSI status, which was confirmed using MSI status from The Cancer Genome Atlas (TCGA) studies of colorectal and endometrial carcinoma. This study highlights an algorithmic method to identify patients with potential MSI-high status for orthogonal screening when current methodologies fall short.

Session: Developmental Therapeutics-Molecularly Targeted Agents and Biology
Abstract: 3063 – Analytical Validation of the Labcorp Plasma Complete Test to Enable Precision Oncology Through Solid Tumor Liquid Biopsy Comprehensive Genomic Profiling
Poster Board: 208
Date: Saturday, June 1, 9:00 a.m. – 12:00 p.m. CDT
The Labcorp Plasma Complete test is a next-generation-sequencing, cell-free DNA comprehensive genomic profiling test that identifies actionable and clinically relevant variants in advanced and metastatic solid cancers across 521 genes. In this validation study, test performance demonstrated highly specific (>99.99%), accurate (97.3% positive percent agreement and >99.99% negative percent agreement) and sensitive (down to 0.35% or 1.63-fold) variant detection. Plasma samples from a broad range of solid tumors demonstrated that this assay offers a highly precise, accurate, and robust comprehensive genomic and immune profiling assay to complement tissue-based testing and inform clinical decision-making.

To connect with Labcorp at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting in Chicago, visit View Source