‘Highly Accurate’: Telix’s Phase III ZIRCON Trial for Kidney Cancer Imaging Published in The Lancet Oncology

On September 10, 2024 Telix Pharmaceuticals Limited reported that primary results from its Phase III ZIRCON[1] trial have been published in The Lancet Oncology, reporting that Telix’s first-in-class investigational PET[2] agent, TLX250-CDx (Zircaix[3], 89Zr-girentuximab), is highly accurate in detecting and characterising clear cell renal cell carcinoma (ccRCC) in patients with indeterminate renal masses (IRMs) (Press release, Telix Pharmaceuticals, SEP 10, 2024, View Source [SID1234646489]).

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In this peer-reviewed manuscript, Professor Brian Shuch (University of California, Los Angeles, UCLA) and colleagues report results from this prospective, open-label, multicentre, Phase III trial in which 300 patients with an IRM ≤7cm (cT1) received TLX250-CDx. Authors conclude that TLX250-CDx "has a favourable safety profile and is a highly accurate, non-invasive imaging modality for the detection and characterisation of ccRCC, which has the potential to be practice changing."

Authors explain that small masses in the kidney are increasingly being detected incidentally when patients undergo abdominal imaging, often for other health conditions, contributing to "an era of gross overtreatment". Diagnosis and treatment are limited by current imaging techniques, and renal mass biopsy is invasive, which can often lead to complications. Up to 30% of patients undergo unnecessary surgery, removing masses that are later determined to be benign[4]. If confirmed, however, ccRCC is the most common and aggressive form of kidney cancer, and delays in diagnosis can significantly reduce survival rates.

Professor Brian Shuch, MD, Director of the Kidney Cancer Program and the Alvin & Carrie Meinhardt Endowed Chair in Kidney Cancer Research at UCLA Institute of Urologic Oncology, and a ZIRCON principal investigator, said, "Until now, assessing whether renal masses are cancerous has been difficult and often involves invasive surgery or percutaneous biopsy. This is because standard imaging technology – either a CT or MRI scan – cannot reliably differentiate between benign or malignant renal lesions or provide information about disease biology.

"The challenges and uncertainty in diagnosing ccRCC underscore a critical unmet need for a new, non-invasive technique that accurately detects and differentiates ccRCC from other renal masses in patients, to inform clinical decision making. The ZIRCON trial has shown that TLX250-CDx is a breakthrough technology that can address this need."

TLX250-CDx is a zirconium-89 (89Zr) radiolabelled monoclonal antibody that targets carbonic anhydrase IX (CAIX), a tumour-associated antigen highly expressed in ccRCC. Following successful Phase I and II trials to establish safety and preliminary efficacy, the ZIRCON trial was designed to assess sensitivity and specificity of TLX250-CDx PET/CT imaging to non-invasively detect ccRCC in patients with cT1 IRMs (≤7 cm in diameter) who underwent nephrectomy, using central histological confirmation as standard of truth.

Key findings of the ZIRCON trial, outlined in the paper, include:

89Zr-girentuximab PET/CT imaging accurately detected ccRCC in patients with cT1 IRM (≤7cm), demonstrating a mean sensitivity of 86%, specificity of 87% and positive predictive value of 93%
89Zr-girentuximab PET/CT imaging has high diagnostic performance for detection and characterisation of small and very small renal masses
The primary and secondary endpoints were met by all three radiology readers and exceeded pre-specified thresholds. Inter-reader variability indicated robust agreement among the readers and intra-reader variability was 100%, indicating perfect agreement
No safety concerns associated with the administration of 89Zr-girentuximab were revealed
Imaging performed 5±2 days after administration is sufficient to visualise and assess ccRCC lesions, with the flexible imaging window offering several advantages for patient management
The non-invasive nature of this technique may be especially beneficial to those at risk of complications from a surgical renal mass biopsy.
Dr David N. Cade, Chief Medical Officer at Telix said, "The results of the ZIRCON trial make a compelling case for TLX250-CDx as a breakthrough product for kidney cancer imaging, and validate CAIX as a novel target to accurately identify renal cell carcinoma.

"Professor Shuch and his co-investigators, at 36 sites worldwide, found that its high diagnostic performance, including for very small lesions, may support early and accurate diagnosis, inform patient risk stratification and clinical decision making, and reduce over- and under-treatment. We believe this result will lead to improved patient outcomes".

If approved by the United States (U.S.) Food and Drug Administration (FDA), TLX250-CDx will be the first and only targeted PET agent specifically for kidney cancer to be commercially available in the U.S.

The full paper can be found at: https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(24)00402-9/fulltext

About TLX250-CDx (Zircaix3)

TLX250-CDx (Zircaix3) is an investigational radiodiagnostic PET agent that is under development to characterise IRMs as ccRCC or non-ccRCC in a non-invasive manner. Telix’s pivotal Phase III ZIRCON trial evaluating TLX250-CDx in 300 patients, of which 284 were evaluable, was completed in 2022 and met all primary and secondary endpoints[5].

PDX Pharma’s PETTRA drug candidate has been awarded to Phase II

On September 10, 2024 PDX Pharma reported that it has successfully met all the milestones of our Phase I SBIR and has received approval from the National Cancer Institute (NCI) to advance to the Phase II project, valued at $2 million (Press release, PDX Pharmaceuticals, SEP 10, 2024, View Source [SID1234646513]). This project (R44CA285233) focuses on the development of our nanotherapeutic, PETTRA (PLK1 and EGFR Targeted Therapy and Radiation Sensitizer). PETTRA is built upon our patented nanoparticle delivery platform, Pdx-NP, which enables the co-delivery of an anti-EGFR antibody (serving as both a cancer-homing agent and an EGFR inhibitor) and PLK1 siRNA (killing cancer cells and sensitizing them to radiation). We have overcome the limitations of traditional nanoparticle delivery systems, achieving a long circulation half-life (e.g., 25 hours in monkeys), a tenfold increase in siRNA accumulation in tumors, and specific delivery to target cells (by 5 to 8-fold over normal cells). Additionally, we have demonstrated excellent PLK1 gene knockdown (e.g., 84%) and significant tumor inhibition (e.g., 91%) in mouse models. We would like to extend our gratitude to the NCI for their continued support and congratulate our team on this achievement!

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Mural Oncology to Host First Virtual Investor Day on September 26, 2024

On September 10, 2024 Mural Oncology plc (Nasdaq: MURA), a clinical-stage immuno-oncology company developing novel, investigational engineered cytokine therapies designed to address areas of unmet need for patients with a variety of cancers, reported that it will host a virtual Investor Day on Thursday, September 26, 2024, beginning at 10 a.m. ET (Press release, Mural Oncology, SEP 10, 2024, View Source [SID1234646474]).

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Mural leadership, including Caroline Loew, Ph.D., CEO, and Vicki Goodman, MD, Chief Medical Officer, will provide new clinical insight into the trial design and assumptions of the company’s late-stage and potentially registrational trials of nemvaleukin, an engineered IL-2. Those trials are on track to deliver topline results in 1H 2025. Mural will also provide an overview and data presentation of its IL-18 program.

The Investor Day will also feature clinician discussion as follows:

Clinical proof of concept of Mural’s lead program, nemvaleukin: Ulka Viashampayan, MD, Professor, Internal Medicine, Division of Hematology/Oncology, University of Michigan

Unmet need in platinum-resistant ovarian cancer: John Hays, MD, PhD, an associate professor in the Divisions of Medical Oncology and Gynecologic Oncology, and member of the Translational Therapeutics Program at The Ohio State University Comprehensive Cancer Center

Mucosal melanoma and need for dedicated treatments: Rich Carvajal, MD, Deputy Physician-in-Chief and Director of Medical Oncology at the Northwell Health Cancer Institute
The event will conclude with a live Question & Answer section.

Register to attend the Investor Day webcast at ir.muraloncology.com/events-and-presentations. A replay of the webcast will be archived and available following the event.

About Nemvaleukin
Nemvaleukin alfa (nemvaleukin) is a novel, engineered cytokine designed to leverage antitumor effects of the IL-2 pathway while mitigating the hallmark toxicities that limit its use. Nemvaleukin selectively binds to the intermediate-affinity IL-2 receptor (IL-2R) and is sterically occluded from binding to the high-affinity IL-2R. Because of this molecular design, nemvaleukin treatment leads to preferential expansion of antitumor CD8+ T cells and natural killer cells, with minimal expansion of immunosuppressive regulatory T cells. Nemvaleukin is currently being evaluated in two potentially registrational late-stage trials.

About Mural Oncology’s IL-18 Program
IL-18 is a potent immune-stimulating cytokine, but its efficacy is blunted by IL-18 binding protein (IL-18BP), a high affinity decoy receptor that binds to, and neutralizes, IL-18, thereby rendering it ineffective. Native IL-18’s potency is also limited by its short half-life. Mural Oncology’s novel approach to protein engineering is designed to mitigate these issues. First, Mural introduced mutations to IL-18 that eliminate binding to IL-18BP while minimally impacting the native IL-18 structure. Second, it fused IL-18 to protein scaffolds which extend the half-life and increase IL-18’s exposure. Together, these have demonstrated more durable immunological effect in preclinical studies. Mural intends to nominate a development candidate for its IL-18 program by the end of this year.

MAIA Biotechnology Announces Positive Survival Updates in Phase 2 Study of THIO in Non-Small Cell Lung Cancer

On September 10, 2024 MAIA Biotechnology, Inc., (NYSE American: MAIA) ("MAIA", the "Company"), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, reported favorable interim survival benefit from its lead clinical candidate THIO, a telomere-targeting treatment for patients with advanced non-small cell lung cancer (NSCLC) (Press release, MAIA Biotechnology, SEP 10, 2024, View Source [SID1234646490]). A Phase 2 clinical trial, THIO-101, is evaluating THIO sequenced with Regeneron’s immune checkpoint inhibitor (CPI) cemiplimab (Libtayo) in patients with advanced NSCLC who failed two or more standard-of-care therapy regimens.

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Published available results suggest that overall survival (OS) in third-line patients is 5.8 months.1

As of August 01, 2024, 16 patients had survival follow-up surpassing 12 months, including 9 in third line treatment (3L). Interim median survival follow-up in 3L was 10.6 months.

"THIO is showing a survival benefit for patients with advanced NSCLC. As our follow-up continues, we have noted that three of the earliest patients enrolled are approaching 17-month survival. We’re on track to achieve our survival goals in third-line therapy," said Vlad Vitoc, M.D., Chairman and Chief Executive Officer of MAIA. "THIO’s outperformance to date supports our thesis that our telomere targeting agent could become a treatment option for people suffering from advanced NSCLC."

The 12-month survival data corresponds to the Company’s most recent data from THIO-101 demonstrating favorable disease control and overall response rates. As announced in April 2024, THIO 180mg + CPI in third-line treatment showed, in part, overall response rate (ORR) of 38%, disease control rate (DCR) of 88% and median progression-free survival (PFS) of 5.5 months.

MAIA expects to release full efficacy results of THIO-101 this year.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About THIO-101, a Phase 2 Clinical Trial

THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate THIO’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of THIO administered prior to cemiplimab (Libtayo) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of THIO administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of THIO using Overall Response Rate (ORR) as the primary clinical endpoint. Treatment with THIO followed by Regeneron’s cemiplimab (Libtayo) has been generally well-tolerated to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.

Neurocrine Biosciences to Participate at the 2024 Cantor Global Healthcare Conference

On September 10, 2024 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will participate at the 2024 Cantor Global Healthcare Conference in New York at 3:05 p.m. Eastern Time on Tuesday, September 17, 2024 (Press release, Neurocrine Biosciences, SEP 10, 2024, View Source [SID1234646475]). Chief Business Development and Strategy Officer and CEO-elect Kyle Gano, Chief Financial Officer Matt Abernethy, and Vice-President of Investor Relations Todd Tushla will present at the conference.

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The live presentation will be webcast and may be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.