On September 10, 2024 Incendia Therapeutics, a precision oncology company discovering and developing a novel class of therapies that reprogram the tumor microenvironment (TME), reported that the first patient has been enrolled in the Phase 1c study of PRTH-101 for the treatment of patients with advanced or metastatic solid tumors (Press release, Incendia Therapeutics, SEP 10, 2024, View Source [SID1234646496]).

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"In our Phase 1a and 1b studies, we were able to select an optimal dose and identify potential biomarkers to carry forward" said Irena Webster, SVP Development and Operations of Incendia. "The data from this study will inform the design of the Phase 2/3 program, for which planning and global feasibility are currently underway."

Dr. Joseph Paul Eder, CMO, added "Incendia, with its outstanding team of investigators and investigative sites, has determined a recommended Phase 2 dose of PRTH 101. PRTH 101 has shown consistent safety as a single agent and in combination with pembrolizumab. We will now focus on tumor types that have demonstrated signs of clinical activity and patient benefits."

The Phase 1c study will evaluate the safety, tolerability and anti-tumor activity of PRTH-101 as both a monotherapy and in combination with KEYTRUDA (pembrolizumab).

About PRTH-101
PRTH-101 is a therapeutic antibody that targets DDR1, a collagen binding protein and kinase present on epithelial cells. Some tumor cells are believed to co-opt DDR1 – collagen binding to build an impenetrable barrier around the tumor. By allosterically disabling DDR1, PRTH-101 disrupts tumor associated collagen alignment to permit immune cell access into the tumor core. In preclinical experiments, PRTH-101 mediated DDR1 blockade has demonstrated both single agent anti-tumor activity as well as marked augmentation of checkpoint inhibitor function. Tumor types that express high levels of DDR1-associated collagen barriers include thymic, colorectal, pancreatic, ovarian, glioma, and non-small cell lung cancer. Currently, there are no approved drugs that target DDR1.

About the Phase 1 Trial
The Phase 1 trial (NCT05753722) is a multi-center, open-label, dose escalation and dose expansion study that is expected to enroll up to 270 patients in the US with advanced or metastatic solid tumors. The goals of the study are to assess safety and tolerability of PRTH-101, evaluate anti-tumor activity in select indications alone and in combination with anti-PD-1 inhibitors, and determine dosing regimens for the Phase 2 clinical program. In addition to examining the clinical profile of PRTH-101, the trial will evaluate DDR1 and pathway-related proteins as predictive biomarkers for patients whose tumors respond to treatment.

On September 10, 2024 CEL-SCI Corporation (NYSE American: CVM) reported that it will report new data from its Phase 3 study of Multikine (Leukocyte Interleukin, Injection)* at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress which takes place from September 13 – 17, 2024 in Barcelona, Spain (Press release, Cel-Sci, SEP 10, 2024, View Source [SID1234646495]). A poster titled "Prognostic significance of diagnostic staging in treatment naïve, resectable locally advanced primary oral cavity squamous cell carcinoma for neoadjuvant Leukocyte Interleukin Injection immunotherapy" will be presented by the study’s co-author József Tímár MD, PhD, DSc, a prominent and highly respected pathologist.

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"Prognostic significance of diagnostic staging in treatment naïve, resectable locally advanced primary oral cavity squamous cell carcinoma for neoadjuvant Leukocyte Interleukin Injection immunotherapy"

Post this
CEL-SCI has received the go-ahead from the U.S. Food and Drug Administration (FDA) to commence a confirmatory Registration Study of Multikine in the treatment of head and neck cancer based on strong safety and efficacy data from its IT-MATTERS completed Phase 3 study.

Dr. Timar is Professor Department of Pathology, Forensic and Insurance Medicine at Semmelweis University in Budapest, Hungary, and served as the Director of the Central Pathology Laboratory for the IT-MATTERS study. With 174 peer reviewed studies published, Dr. Timar is a founding editor, editor in chief, or a member of the editorial board of four oncology journals. He is the recipient of a dozen honors and awards for excellence in cancer research and teaching.

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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