On May 22, 2024 Veracyte, Inc. (Nasdaq: VCYT), a leading cancer diagnostics company, reported that data from three studies will demonstrate the power of the Afirma GRID (Genomic Resource for Intelligent Discovery) tool to help unlock new molecular insights into thyroid tumors, which may ultimately help to further personalize treatment of the disease (Press release, Veracyte, MAY 22, 2024, View Source [SID1234643539]). The findings will be presented at ENDO 2024, the annual meeting of The Endocrine Society, which will take place June 1-4 in Boston.

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"Molecular testing in thyroid cancer currently focuses primarily on whether a patient’s thyroid nodule is benign or cancerous, and whether specific gene mutations are present," said Joshua Klopper, M.D., Veracyte’s medical director for Endocrinology. "The data to be presented at ENDO 2024 looks beyond individual gene mutations and explores the potential of novel molecular signatures and alterations to provide more-granular, prognostic information. This may ultimately help clinicians guide more individualized care based on the molecular characteristics of each patient’s tumor. These findings are from studies using Afirma GRID, a research-use-only tool that leverages Veracyte’s whole-transcriptome approach, which enables the scientific community to advance understanding of thyroid cancer biology."

Following are details for the Afirma GRID-focused research studies that will be presented at ENDO 2024 in the Boston Convention & Exhibition Center:

Title:

Retrospective Analysis of mRNA Expression Based Signatures of Thyroid Tumor Invasion and Metastases

Presenter:

Sara Ahmadi, M.D., Brigham and Women’s Hospital

Format:

Poster Presentation (MON-640)

Time:

Monday, June 3, 2024, 12:00 p.m. – 1:30 p.m. ET

Location:

ENDO Expo Poster Area

Title:

Prostate-specific Membrane Antigen (PSMA) Expression in Cytologically Indeterminate and Malignant Thyroid Nodules

Presenter:

Rabail Sadiq, M.B.B.S., Johns Hopkins University School of Medicine

Format:

Rapid-Fire Oral Presentation (RF28-01) AND Poster Presentation (MON-649)

Poster Presentation (MON-649):

Time:

Monday, June 3, 2024, 12:00 p.m. – 1:30 p.m. ET

Location:

ENDO Expo Poster Area

Rapid-Fire Oral Presentation (RF28-01):

Time:

Monday, June 3, 2024, 1:45 p.m. – 1:50 p.m. ET

Location:

Room 257AB

Title:

Cancer-associated Fibroblasts Correlate with Aggressive Thyroid Cancer Behavior: Insights from Four Large Patient Cohorts

Presenter:

Matthew A. Loberg, B.A., Vanderbilt University Medical Center

Format:

Oral Presentation (OR28-04)

Time:

Monday, June 3, 2024, 2:45 p.m. – 3:00 p.m. ET

Location:

Room 257AB

Additional information about these presentations and Veracyte’s participation at ENDO 2024 can be found at the company’s booth (#709).

About Afirma GRID

The Afirma GRID database is inclusive of the sequencing of over 21,000 expressed genes for nearly 200,000 patients with thyroid nodules (benign and malignant) and is used by Veracyte and its partners to contribute to continued research that helps advance understanding of thyroid tumors. Afirma GRID information is available on a Research-Use-Only basis. More information about Afirma GRID can be found here.

About the Afirma GSC

Veracyte’s flagship Afirma Genomic Sequencing Classifier (GSC) was developed with RNA whole-transcriptome-derived sequencing and machine learning technology and helps physicians identify patients with benign thyroid nodules among those whose fine needle aspiration (FNA) biopsy results are indeterminate by cytopathology so that they can potentially avoid unnecessary thyroid surgery. The Afirma GSC also includes Xpression Atlas, the largest thyroid gene and fusion variant panel available, to help inform treatment decisions for patients whose genomic test or cytopathology results are suspicious for cancer. Veracyte also enables physicians to order DNA testing of the TERT promoter gene, which is performed on the same FNA sample, to help further guide treatment decision-making. More information about the Afirma GSC can be found here.

On May 22, 2024 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that new interim tumor response data from the ongoing randomized, controlled Phase 2b study of VBI-1901, the Company’s cancer vaccine immunotherapeutic candidate in recurrent glioblastoma (GBM), were accepted for poster presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, VBI Vaccines, MAY 22, 2024, View Source [SID1234643538]).

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The presentation at ASCO (Free ASCO Whitepaper) will provide an update to the encouraging data previously shared at the World Vaccine Congress Washington in April, including additional data from those initially evaluable patients, as well as data from new patients who have since been randomized into the Phase 2b study.

Presentation Details

Title: Randomized Phase 2b trial of a CMV vaccine immunotherapeutic candidate (VBI-1901) in recurrent glioblastomas
Date: Saturday, June 1, 2024
Poster Session: Central Nervous System Tumors
Poster Session Time: 9:00 AM – 12:00 PM CDT
Phase 2b Study Design

Multi-center, randomized, controlled, open-label study in up to 60 patients with first recurrent GBM

Patients will be randomized in a 1:1 ratio across two study arms:
Intradermal VBI-1901 + GM-CSF: 10 µg dose every 4 weeks until clinical disease progression
Monotherapy standard-of-care: either intravenous carmustine or oral lomustine, every 6 weeks until disease progression or intolerable toxicity
Endpoints include:
Safety and tolerability
Overall survival (OS) – median and overall
Tumor response rate (TRR)
Progression-free survival (PFS)
Immunologic responses
Reduction in corticosteroid use relative to baseline
Change in quality of life compared to baseline
The U.S. Food and Drug Administration (FDA) has considered demonstration of a statistically significant improvement in overall survival relative to a randomized control arm to be clinically significant and has recognized this as criteria to support the approval of new oncology drugs.1

For more information about the Phase 2b study, visit clinicaltrials.gov and reference trial identifier: NCT03382977.

About GBM and VBI-1901

Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, more than 12,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. The FDA has granted VBI-1901 Fast Track Designation and Orphan Drug Designation for the treatment of recurrent glioblastoma. These designations are intended to provide certain benefits to drug developers, including more frequent meetings with the FDA, and Accelerated Approval and Priority Review, if relevant criteria are met, among other benefits.

References:

1. Oncology Center of Excellence, Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics; Guidance for Industry. FDA.gov. December, 2018

On May 22, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that management will participate in a virtual fireside chat at the TD Cowen 5TH Annual Oncology Innovation Summit on Tuesday, May 28, 2024 at 3:20 PM ET (Press release, UroGen Pharma, MAY 22, 2024, View Source/news-releases/news-release-details/urogen-pharma-present-td-cowen-5th-annual-oncology-innovation" target="_blank" title="View Source/news-releases/news-release-details/urogen-pharma-present-td-cowen-5th-annual-oncology-innovation" rel="nofollow">View Source [SID1234643537]).

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A live webcast of the fireside chat can be accessed by visiting the Events and Presentations Section of the Company’s website: View Source

On May 22, 2024 The University of Birmingham and Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of orphan paediatric medicines, reported the extension of their collaboration to leverage patient-level data of the BEACON refractory or relapsed neuroblastoma trial to support the marketing authorisation of KIZFIZO (Press release, ORPHELIA Pharma, MAY 22, 2024, View Source;utm_medium=rss&utm_campaign=orphelia-pharma-and-university-of-birmingham-strengthen-their-collaboration-to-support-the-registration-of-kizfizo [SID1234643536]).

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Neuroblastoma is a rare type of cancer usually diagnosed in children under 2 years of age, and for which age-specific therapeutic options are needed.

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is the first ready-to-use oral liquid paediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carries a very poor prognosis.

The BEACON trial (NCT02308527) was a hypothesis-generating phase II trial that served to identify active regimens in relapsed or refractory neuroblastoma, some of which are now being further investigated. The trial recruited paediatric patients between 2013 and 2021 and involved the University of Birmingham as the sponsor, the European expert groups SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative therapies for children with cancer).

KIZFIZO was not available in time for the BEACON trial but Orphelia Pharma and the University of Birmingham are collaborating to assess the clinical benefit and the safety of temozolomide-based regimens in relapsed or refractory high-risk neuroblastoma patients. Overall, anonymised data from the BEACON trial are being used to support the registration dossier of KIZFIZO, according to a data sharing agreement drawn up by University of Birmingham Enterprise.

BEACON-2 is the follow-on trial that will test bevacizumab and dinutuximab beta in combination with the backbone chemotherapy temozolomide and irinotecan. As part of its ongoing commitment to child cancer, Orphelia Pharma has also agreed to support the new BEACON-2 trial, also sponsored by University of Birmingham, by making KIZFIZO available as a child-friendly temozolomide option, in addition to the standard form of temozolomide, where participating investigators can have access to this medicine upon regulatory approval of its use as part of the BEACON-2 trial.

Jeremy Bastid, Chief Development Officer at Orphelia Pharma, commented: "The support of the University of Birmingham has been invaluable to us. The BEACON data has already demonstrated a clear clinical benefit and an improved overall survival over historical controls."

Dr Lucas Moreno, Head of Paediatric Haematology and Oncology at Vall d’Hebron University Hospital, Barcelona, Spain and Chief Investigator for BEACON said: "We are delighted that the data generated could not only delineate the best treatment options for those patients, but also support the registration of a new paediatric formulation of temozolomide, which is much sought-after for our younger patients."

Professor Amos Burke, Director of the Cancer Research Clinical Trials Unit concluded: "A liquid formulation of temozolomide is welcomed for younger patients and the use of data from the standard capsule formulation of temozolomide used in BEACON to support registration for a new liquid formulation demonstrates the importance of the collaborative use of results from this practice-changing trial."

On May 22, 2024 NUCLIDIUM and Guerbet (GBT) reported a strategic partnership to advance NUCLIDIUM’s diagnostic and therapeutic copper-based radiopharmaceuticals (Press release, NUCLIDIUM, MAY 22, 2024, View Source [SID1234643534]). This partnership will provide NUCLIDIUM access to Guerbet’s knowledge and extensive network in diagnostic imaging, manufacturing, and clinical trials, aiding the progression of NUCLIDIUM’s theranostic candidates towards commercialization. Guerbet has made a strategic, non-exclusive investment in NUCLIDIUM as part of the agreement. As part of the agreement, François Nicolas, Senior Vice President of R&D at Guerbet, will join NUCLIDIUM’s Board of Directors as an observer. No further details of the agreement have been disclosed.

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"Guerbet is a global leading expert in diagnostic imaging with a longstanding record in developing, manufacturing, and globally distributing medical imaging solutions. This collaboration emphasizes the value of our innovative copper-based theranostic pipeline, which aims to provide cancer patients with comprehensive solutions from the precise diagnosis to effective treatments. Our goal is to accelerate the clinical development of our unique product candidates and we look forward to gaining valuable strategic insights from François and the Guerbet team," said Leila Jaafar, PhD, CEO and Co-Founder of NUCLIDIUM.

François Nicolas, Senior Vice President of R&D Guerbet added: "NUCLIDIUM leads in pioneering a new generation of targeted radiotheranostics that could vastly extend patient benefits. Targeted radiopharmaceuticals represent a significant advancement in cancer treatment, yet face supply limitations and production challenges. NUCLIDIUM’s copper-based strategy presents a promising approach to address these issues, enhancing patient access and improving disease staging and treatment. This aligns with Guerbet’s growth strategy to broaden our core business areas. I am excited to support Leila and her team in demonstrating the clinical value of its platform."

NUCLIDIUM is advancing a unique pipeline of targeted copper-based radiopharmaceuticals leveraging isotopes 61Cu and 67Cu. Its innovative platform facilitates a seamless transition from diagnostic to therapeutic applications by merely exchanging the isotopes on the same tumor-targeting molecule. The company will apply its easy-to-produce manufacturing process to enhance product availability and address major challenges in radiotheranostic medicine to develop precise and effective theranostics for multiple solid tumor types including prostate, colorectal, gastric, breast and pancreatic cancers as well as neuroendocrine tumors. The partnership with Guerbet is a critical pillar in NUCLIDIUM’s strategy to establish valuable collaborations to advance its proprietary pipeline and ensure reliable supply chains for its copper-based radiopharmaceuticals.