On September 10, 2024 enGene Holdings Inc. (Nasdaq: ENGN, or "enGene" or the "Company"), a clinical-stage genetic medicines company whose non-viral lead investigational product detalimogene voraplasmid, (also known as detalimogene, and previously EG-70), is in an ongoing pivotal study in patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis), reported its financial results for the third quarter ended July 31, 2024 and provided a business update (Press release, enGene, SEP 10, 2024, View Source [SID1234646493]).

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"Detalimogene was designed to be the most practical therapy for patients living with NMIBC and the urologists caring for them," said Ron Cooper, Chief Executive Officer of enGene. "We believe the unmet need for bladder cancer patients is significant and that detalimogene has the potential to offer a highly differentiated profile with a unique combination of clinical activity, tolerability, and ease of use. We look forward to sharing preliminary results from our pivotal LEGEND study later this month."

Anticipated Milestones and Strategic Corporate Updates

Release of preliminary data from LEGEND Cohort 1: The Company expects to release preliminary data from the LEGEND study’s pivotal BCG-unresponsive cohort by the end of September.

Key leadership hires and board additions: In July 2024, enGene announced that Ron Cooper joined the Company as Chief Executive Officer and member of the Board of Directors. The Company also announced the promotion of Dr. Raj Pruthi to Chief Medical Officer.

Third Quarter 2024 Financial Results

Cash and cash equivalents, as of July 31, 2024, were $257.7 million. The Company expects that its existing cash and cash equivalents will fund operating expenses, debt obligations and capital expenditures into 2027.

Three Months ended July 31, 2024

Total operating expenses were $16.8 million for the three months ended July 31, 2024, compared to $6.2 million for the three months ended July 31, 2023. Research and development expenses increased by $7.6 million, mainly due to increasing manufacturing and clinical costs related to our pivotal LEGEND study and headcount costs. General and administrative expenses increased by $2.9 million, primarily driven by headcount costs and other expenses driven by director and officer insurance expense as the Company scales its general and administrative function to support the operation of a public company.

For the three months ended July 31, 2024, net loss attributable to common shareholders was approximately $14.1 million, or $0.32 per share, compared to approximately $6.0 million, or $8.55 per share, for the same period for the three months ended July 31, 2023. The increase in net loss is mainly attributed to the increase in operating expenses partially offset by net interest income earned during the period.

On September 10, 2024 Delta-fly pharma reported that a phase I/II clinical trial of DFP-10917 in combination with venetoclax (VEN) in patients with acute myeloid leukemia (AML) who have received prior VEN-containing therapy was approved by the U.S. Food and Drug Administration (FDA) on April 8, 2024 ( Delta-Fly Pharma Inc: Notice of Authorization to Conduct Phase I/II Study of DFP-10917 in Combination with Venetoclax | Business Wire ) (Press release, Delta-Fly Pharma, SEP 10, 2024, View Source [SID1234646492]). Today, we are pleased to announce that the first patient has been enrolled at the University of Virginia Hospital based on Investigational Review Board (IRB) approval. The University of Virginia Hospital is the clinical site that has enrolled the most patients in the Phase III study of DFP-10917 as a single agent in patients with relapsed/refractory AML.

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Pharmaceutical companies have already expressed interest in the Phase III study of DFP-10917 as a monotherapy. In addition, many companies, including pharmaceutical giants, have expressed interest in the Phase I/II combination study of DFP-10917 with VEN, due to the large potential market size for the treatment of AML.

VEN alone is not effective against AML, but it becomes effective in combination with azacitidine (DNA methylation inhibitor), which is currently recognized as a standard treatment for AML despite safety concerns. We will therefore provide significantly more combination therapy of VEN and DFP-10917 (G2/M arrest) compared to the current standard treatment for AML. Up to 39 patients will be enrolled in this study. The endpoints are complete remission rate and progression-free survival. After the completion of the Phase I/II study, Delta-Fly Pharma, Inc. will cooperate with a pharmaceutical giant to file a New Drug Application (NDA).

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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

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 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].