On September 10, 2024 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global oncology company, reported that it will present new data on TEVIMBRA (tislelizumab) at the European Society for Medical Oncology 2024 Congress (ESMO 2024) in Barcelona, ​​Spain, September 13-17, 2024 (Press release, BeiGene, SEP 10, 2024, View Source [SID1234646494]). Seven BeiGene abstracts have been selected for the ESMO (Free ESMO Whitepaper) 2024 Congress, including one that has been selected for the special session revisiting the virtual ESMO (Free ESMO Whitepaper) plenary held in February 2024.

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New data strengthen evidence for TEVIMBRA’s efficacy in multiple conditions

As a reminder of the ESMO (Free ESMO Whitepaper) plenary session, interim results from the RATIONALE-315 study show a statistically significant benefit in terms of event-free survival (EFS) and overall survival (OS) trend supporting neoadjuvant tislelizumab plus chemotherapy with adjuvant tislelizumab compared with placebo plus chemotherapy with adjuvant placebo for patients with resectable non-small cell lung cancer (NSCLC) (session #VP1-2024, 13 September from 16:17 to 16:29 CEST). These results confirm data presented at ESMO (Free ESMO Whitepaper) 2023, showing that the major pathological response (MPR) and pathological complete response (pCR) rates were significantly improved: 56.2% vs. 15.0% (P<0.0001) and 40.7% vs. 5.7% (P<0.0001), respectively. The safety profile of the tislelizumab arm was consistent with that of the individual therapies, with 72.1% (vs. 66.4% in the placebo arm) of patients in the tislelizumab arm experiencing grade ≥3 treatment-related adverse events (TRAEs) and 15.5% (vs. 8.0% in the placebo arm) experiencing serious TRAEs. The most common treatment-related adverse events were neutrophil count decreased, white blood cell count decreased, and alopecia. Symptom improvement achieved with RATIONALE-315 will also be presented as patient-reported outcomes (Poster #1213P, September 14).
Three-year overall survival data from the RATIONALE-305 study continue to demonstrate the long-term efficacy and safety of tislelizumab in combination with chemotherapy in patients with first-line advanced or metastatic gastric cancer/gastroesophageal junction cancer (GC/GEJC) (Poster #1437P, September 16), as well as improved patient-reported outcomes (Poster #1449P, September 16).
Long-term results from the RATIONALE-307 study in the ITT population as well as in the population with long-term exposure to tislelizumab plus chemotherapy for the first-line treatment of squamous cell NSCLC show a continued OS benefit, with clinically promising four-year OS rates (Poster No. 1323P, September 14).
Relative efficacy of tislelizumab compared with other anti-PD-1 therapies approved in the European Union and the United Kingdom for the second-line treatment of esophageal squamous cell carcinoma (ESC) using a grounded theory simulated treatment comparison of data from the RATIONALE-302 study and comparative clinical studies (poster #1417P, September 16).
"TEVIMBRA has demonstrated potential in multiple disease states, and the data presented at ESMO (Free ESMO Whitepaper) 2024 reinforce its position as a cornerstone asset in our solid tumor pipeline," said Dr. Jan-Henrik Terwey, Vice President, Medical Affairs, Europe, BeiGene. "As part of our commitment to bring innovative cancer medicines to more patients, we recently launched TEVIMBRA in EMA-approved indications in Germany, Austria and Norway, and are working to make TEVIMBRA available across Europe."

TEVIMBRA in Europe

BeiGene recently launched TEVIMBRA in the first European countries following EU marketing authorizations for the treatment of eligible patients with EOC and NSCLC. TEVIMBRA is also approved in the United Kingdom and Switzerland for eligible patients with advanced or metastatic EOC.

"Advanced or metastatic EOC and NSCLC are aggressive cancers with limited treatment options," said Markus Moehler, MD, PhD, of Johannes Gutenberg University Medical Center Mainz in Germany. "The availability of tislelizumab for these patients represents an important next step in changing the treatment landscape."

The European Commission’s authorisations are based on the results of four randomised phase 3 studies in the RATIONALE programme: RATIONALE-302 ( NCT03430843 ) in EOC and RATIONALE-307 ( NCT03594747 ), RATIONALE-304 ( NCT03663205 ) and RATIONALE-303 ( NCT03358875 ) in NSCLC. The approved indications for TEVIMBRA in the EU are:

In combination with carboplatin and paclitaxel or nab-paclitaxel for the first-line treatment of adult patients with locally advanced squamous cell NSCLC who are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
In combination with pemetrexed and platinum-containing chemotherapy for the first-line treatment of adult patients with non-squamous NSCLC whose tumors have PD-L1 expression on ≥50% of tumor cells, without positive EGFR or ALK mutations, and who have locally advanced NSCLC and are not candidates for surgical resection or platinum-based chemoradiation, or metastatic NSCLC.
As monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC after prior platinum-based therapy. Patients with EGFR mutation-positive or ALK-positive NSCLC should also have received targeted therapies prior to receiving tislelizumab.
As monotherapy for the treatment of adult patients with unresectable, locally advanced or metastatic EOC after prior platinum-based chemotherapy.
About TEVIMBRA (tislelizumab)

Tislelizumab is a uniquely designed humanized anti-programmed death protein 1 (PD-1) immunoglobulin G4 (IgG4) monoclonal antibody with high affinity and specificity of binding against PD-1. It is designed to reduce binding to Fc-gamma (Fcγ) receptors on macrophages, which helps the body’s immune cells detect and fight tumors.

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.