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Rznomics Inc. Secures Fast Track Designation from the U.S. FDA for RZ-001

On November 10, 2023 Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, reported that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Glioblastoma (GBM) (Press release, Rznomics, NOV 10, 2023, View Source [SID1234637473]).

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Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner. If relevant criteria are met, the drug with Fast Track Designation may also be eligible for Accelerated Approval and Priority Review.

GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies. RZ-001, the RNA replacement enzyme-based cancer gene therapy for the treatment of GBM, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. This induces anti-cancer activity and cytotoxic effect by trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. RZ-001 has demonstrated very promising responses in preclinical studies in which an enhanced anti-cancer efficacy and a higher survival rate was observed.

"We are proud to receive Fast Track Designation from the FDA," said Dr. Seong-Wook Lee, Chief Executive Officer of Rznomics. "This designation signifies an important milestone in developing a safe and effective treatment for patients with GBM who are in need of new therapeutic options."

Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in Glioblastoma and the clinical trial will investigate the safety, tolerability, and efficacy of RZ-001 in patients with GBM. Subjects will be treated with RZ-001 according to the planned dose escalation design.

SeekIn Presents Retrospective and Prospective Validation Studies of SeekInCare Multi-omics Multi-cancer Early Detection test at the 20th ICGC Workshop and 7th ARGO Meeting in New York

On November 10, 2023 SeekIn Inc., a leader in blood-based pan-cancer early detection and monitoring technology, reported the validation studies consisting of one retrospective cohort and one prospective cohort that evaluate SeekInCare’s ability to detect cancer patients and predict the tissue of origin with one tube of blood (Press release, SeekIn, NOV 10, 2023, View Source [SID1234637472]). The results demonstrate the performance of SeekInCare with the consistent results among the two cohorts in ~2,400 cancer patients and non-cancer individuals. SeekInCare achieved overall 65.5% sensitivity at 97.9% specificity in the retrospective studies and 60.0% sensitivity at 96.1% specificity in the prospective cohort study, respectively, which is best-in-class in blood-based multi-cancer early detection (MCED). The findings were presented at the 20th ICGC Workshop and 7th ARGO Meeting in New York.

"Recent studies have demonstrated that blood-based multi-cancer early detection approaches may hold promise for identifying asymptomatic cancer patients from general population. However, most studies only exploit a single aspect of cancer hallmarks, which is challenging to achieve highly sensitive results because cancer is a heterogenous disease," said Mao Mao, MD, PhD, Founder and CEO of SeekIn. "For example, our OncoSeek test for multi-cancer early detection was designed with seven protein tumor markers. With the assistance of artificial intelligence (AI), the sensitivity of OncoSeek was 51.7% at 92.9% specificity. While OncoSeek significantly reduced false positive rates comparing with traditional methods, we believe sensitivity can be further improved when more omics data are included. And this is how SeekInCare was conceived."

SeekInCare was developed using proprietary AI- and big data-driven algorithm to distinguish cancer patients from non-cancer individuals by calculating cancer risk score (CRS) incorporating copy number aberrations (CNAs), fragment size (FS), end motifs and oncogenic viruses via shallow whole genome sequencing (sWGS) from cell-free DNA (cfDNA), and seven plasma tumor markers (PTMs), and to predict the possible affected tissue of origin (TOO) for those who have been detected with cancer signals in blood. SeekInCare’s performance was well-demonstrated by the overall 65.5% sensitivity at 97.9% specificity in the retrospective study and 60.0% sensitivity at 96.1% specificity in the prospective study. A critical attribute of a blood-based multi-cancer detection test is the ability to localize the TOO to direct the diagnostic workup. The overall accuracy of two most possible organs SeekInCare achieved was 85.4%, improved by 20.0% from OncoSeek.

"In addition to the performance improvement of SeekInCare in TOO prediction comparing with OncoSeek, significant improvements were also observed in major metrics such as sensitivity, specificity and accuracy. Unlike OncoSeek designed for low- and middle-income countries, SeekInCare enables us to provide a more sensitive and specific multi-cancer detection solution to the regions with purchasing power and high demands of advanced cancer screening," said Dr. Mao. "More importantly, comparing with the results of both retrospective and prospective studies from GRAIL and Exact Sciences, SeekInCare showed higher sensitivities with similar specificities."

The excellent consistency of validation results from both retrospective and prospective studies established SeekInCare’s clinical feasibility. With these achievements, SeekIn is planning for future investigation of SeekInCare’s clinical utility as a large-scale cancer screening test in average-risk populations.

RZNOMICS Inc. Announces Clinical Trial Collaboration and Supply Agreement to Evaluate RZ-001 in Combination with anti-PDL1 in liver cancer patients

On November 10, 2023 Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, reported that it has entered into a clinical collaboration agreement with F. Hoffmann-La Roche Ltd (Roche) to study RZ-001, a gene therapy approach utilizing the company’s proprietary trans-splicing ribozyme-based RNA reprogramming and editing technology, in combination with Roche’s atezolizumab, in patients with hepatocellular carcinoma (HCC) (Press release, Rznomics, NOV 10, 2023, View Source [SID1234637471]). Under the terms of the agreement, Roche will provide atezolizumab to be used in combination with RZ-001 for the planned Phase 1b/2a study.

RZ-001, the RNA replacement enzyme based cancer gene therapy, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. Specifically, RZ-001 induces anti-HCC activity by suppressing hTERT expression selectively in cancer cells, which express hTERT, and simultaneously inducing a cytotoxic effect by trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. It also induces effective immune cell infiltration into treated tumors and reduces VEGF expression in the tumors, which leads to the anti-cancer efficacy and transformation of tumor microenvironment to be more responsive to immunotherapy.

RZ-001 has demonstrated therapeutic activity in preclinical studies and significant improvement in anti-cancer efficacy in a dose dependent manner in combination with atezolizumab. The clinical study will evaluate the safety and efficacy in liver cancer patients and it is anticipated that the combination of RZ-001 with atezolizumab may enhance response rate and anti-cancer efficacy that could benefit patients with HCC.

"We are excited to initiate collaboration with longstanding oncology leader, Roche, to further explore the potential benefits of combining checkpoint blockade with RZ-001," said Dr. Seong-Wook Lee, Chief Executive Officer of RZNOMICS. "We look forward to having this collaboration, evaluating combination therapy and continuing our mission to offer innovative therapies for patients in need."

About RZ-001

RZ-001 is the first Group I intron based trans-splicing ribozyme-based RNA reprogramming approach approved by the FDA for evaluation in patients. The treatment was developed utilizing Rznomics’ proprietary RNA reprogramming and editing technology and takes the form of an adenoviral vector that expresses an hTERT targeting trans-splicing ribozyme to treat hepatocellular carcinoma (HCC) patients. HCC is the most common type of primary liver cancer, accounting for 80 percent of cases worldwide.

Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in HCC and Glioblastoma as well.

XNK Therapeutics to present at Investival Showcase Meeting

On November 10, 2023 XNK Therapeutics AB ("XNK") reported that Johan Liwing, CEO, will present the company at the at Investival Showcase Meeting in London, on November 13th (Press release, XNK Therapeutics, NOV 10, 2023, View Source [SID1234637470]).

The LSX Investival Showcase meeting is one of Europe’s biggest pitch events for early and growth stage companies. The meeting brings together over three hundred qualified life science investors. In addition, it serves as the kickoff to the Jefferies week, leading into the Jefferies London Healthcare Conference, where XNK will also participate.

The presentation is followed by a short Q&A session.

Mirati Therapeutics Receives Positive Opinion from CHMP for KRAZATI (adagrasib) as a Targeted Treatment Option for Patients with Advanced Non-Small Cell Lung Cancer (NSCLC) with a KRASG12C Mutation Following a Re-Examination Procedure

On November 10, 2023 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a commercial stage biotechnology company, reported that following a re-examination procedure, the Company has received a positive opinion from the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) on KRAZATI (adagrasib) as a targeted treatment option for adult patients with KRASG12C -mutated advanced non-small cell lung cancer (NSCLC) and disease progression after at least one prior systemic therapy (Press release, Mirati, NOV 10, 2023, View Source [SID1234637469]).

"Today’s positive opinion from the CHMP for KRAZATI marks an important step on the path to providing access to a potentially best-in-class therapeutic option to patients living with this difficult-to-treat disease," Alan Sandler, M.D., chief medical officer, Mirati Therapeutics, Inc. "We look forward to approval from the European Commission and the opportunity to positively impact the lives of eligible patients living in the European Union."

"This is an important day for the oncology community as we step closer to a new therapeutic option being available to patients living with KRASG12C mutated NSCLC in the European Union," said Martin Reck, MD, PhD, Lung Clinic Grosshansdorf, Germany. "Every patient has a slightly different case so as more options become available physicians will better be able to tailor their treatment for each patient."

About KRAZATI (adagrasib)

Mirati has risen to meet one of the most challenging mutations in cancer research by developing KRAZATI, a highly selective and potent oral small-molecule inhibitor of KRASG12C.

Intentionally designed to meet the challenge of KRASG12C, adagrasib is optimized to sustain target inhibition, an attribute that could be important to treat KRASG12C-mutated cancers, as the KRASG12C protein regenerates every 24−48 hours.1Adagrasib has shown clinically to be a CNS penetrant, which may be important given that CNS metastases frequently occur in NSCLC and lead to poor prognosis.2,3,4

The FDA provided KRAZATI Accelerated Approval (Subpart H), allowing for the approval of drugs that treat serious conditions, and that fill an unmet medical need based on surrogate endpoints. KRAZATI was also granted a conditional marketing authorization by the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) on November 3, 2023.

Adagrasib continues to be evaluated as monotherapy and in combination with other anti-cancer therapies in patients with advanced KRASG12C-mutated solid tumors, including NSCLC, colorectal cancer, and pancreatic cancer. For more information, visit Mirati.com/science.

About the KRYSTAL-1 Study

KRYSTAL-1 is an open-label Phase 1/2 multiple-expansion cohort trial evaluating adagrasib as monotherapy and in combination with other anti-cancer therapies in patients with advanced solid tumors harboring the KRASG12C mutation.

About KRASG12C in NSCLC

Lung cancer is one of the most common cancers worldwide, accounting for 2.21 million new cases and 1.8 million deaths worldwide in 2020.5 Lung cancer consists of NSCLC in approximately 85% of cases and small cell lung cancer (SCLC) in approximately 15% of cases.6 KRASG12C is the most common KRAS mutation in NSCLC, present in approximately 14% of patients with lung adenocarcinoma, and is a biomarker mutation of poor prognosis.