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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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.

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.

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.

On September 10, 2024 Avistone Biotechnology Co., Ltd. (also referred to as Avistone Biotechnology or Avistone), an innovative biotechnology company focused on precision oncology therapeutics, reported results from an oral presentation at the IASLC 2024 World Conference on Lung Cancer (#WCLC24) in San Diego, CA (Press release, Avistone Pharmaceuticals, SEP 10, 2024, View Source [SID1234646491]).

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"The development of drugs for indications related to both c-MET and EGFR targets has always been a difficult one. We are now entering an era where the clinical combination of these targets has shown meaningful potential benefit to patients with NSCLC. We are excited to share these preliminary clinical results from the combination of our two novel (cMET and EGFR) inhibitors in NSCLC patients with EGFR mutation-positive, MET amplified or MET overexpression," said Dr. Hepeng Shi, Chairman, CEO, and Founder of Avistone.

Details and highlights from the oral presentation are as follows:

Title: Vebreltinib plus PLB1004 in EGFR-mutated, NSCLC with MET amplification or MET overexpression following EGFR-TKI

Results: Forty-four patients received Vebreltinib 100mg plus PLB1004 160mg (n=15), Vebreltinib 150mg plus PLB1004 160mg (n=13), Vebreltinib 200mg plus PLB1004 80mg (n=3), or Vebreltinib 150mg plus PLB1004 80mg (n=13) and were included in the overall safety analysis. Among the 44 patients evaluated, 36.4% of patients had brain metastases and 86.4% of patients had previously received a third-generation EGFR-TKI, respectively. Objective responses occurred across all Vebreltinib and PLB1004 dose levels tested, with partial responses (PRs) observed in 19/32 (59.4%) response-evaluable patients. Among patients with brain metastases, the ORR was 75.0% (9/12). The ORR was 58.6% (17/29) in patients who received prior third-generation EGFR-TKI. The most common TRAEs were rash and paronychia. No patients discontinued treatment due to TRAEs.

Clinical Trial Identifier: NCT06343064