SOCS1

Phase 1/2 clinical study will evaluate the safety, pharmacokinetics, and preliminary efficacy of KSQ-001EX in melanoma, head and neck squamous cell carcinoma, and non-small cell lung cancerLEXINGTON, Mass., Nov. 29, 2023 (GLOBE NEWSWIRE) -- KSQ Therapeutics, Inc. (KSQ), a clinical-stage biotechnology company developing novel treatments for solid tumors, in collaboration with The University of Texas MD Anderson Cancer Center and the Cell Therapy Manufacturing Center (CTMC), today announced the Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for a Phase 1/2 clinical study of KSQ-001EX, KSQ’s lead engineered tumor-infiltrating lymphocyte (eTIL®) program. KSQ-001EX consists of TILs in which the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing. KSQ's CRISPRomics® platform identified SOCS1 as a key gene regulating the anti-tumor potency and persistence of TIL and provides KSQ-001EX with best-in-class potential in the treatment of solid tumors. "Our team has made great progress advancing our KSQ-001EX and KSQ-004EX eTIL® programs. Through our platform insights, we've identified how to enhance the effectiveness of T cells in the treatment of solid tumors. We believe our eTIL® cell therapies have tremendous potential to benefit patients with cancer who have limited treatment options. We look forward to working closely with the clinical experts at MD Anderson, where the study will be initiated,” said Qasim Rizvi, Chief Executive Officer of KSQ. “Our partnership with CTMC has yielded a robust manufacturing process for our eTIL® cell therapy that is derived from patient tumor samples, including core biopsies. The collaborative effort allowed the IND to be submitted in an accelerated timeframe.” “New treatment options are urgently needed in melanoma and other solid tumors, and the potential to improve outcomes and patient experience is very exciting. KSQ-001EX exhibited considerable efficacy in preclinical studies,” said Rodabe Amaria, M.D., professor of Melanoma Medical Oncology at MD Anderson and principal investigator of the KSQ-001EX study. “We look forward to working closely with KSQ as we advance this program.” Phase 1/2 Trial Design The Phase 1/2 clinical trial is an open-label, safety lead-in study for patients with melanoma, head and neck squamous cell carcinoma (HNSCC), and non-small cell lung cancer (NSCLC). The primary objective of the Phase 1 portion of the trial is to evaluate safety and tolerability of KSQ-001EX. In the safety lead-in portion, a cohort of patients will be initially dosed without IL-2. The primary objective of Phase 2 is to evaluate antitumor activity in indication-specific cohorts. About KSQ-001EX KSQ’s lead eTIL® cell therapy program, KSQ-001EX, in which TILs are edited to inactivate the SOCS1 gene, has the potential to revolutionize the treatment of solid tumors. In preclinical studies, KSQ-001EX demonstrated enhanced anti-tumor function in solid tumor models refractory to PD-1 inhibition, as well as enhanced persistence, and memory formation. About KSQ Therapeutics KSQ Therapeutics is advancing a pipeline of novel drug candidates to treat cancer across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies. KSQ's proprietary CRISPRomics® discovery engine enables genome-scale insights driving novel therapeutic discovery. For more information, please visit the company's website at www.ksqtx.com. KSQ Media Contact: Cory Trombleecory@scientpr.com

Moderna’s combination vaccine candidate for Covid-19 and influenza, mRNA-1083, has shown early potential in a mid-stage trial, sparking plans for registrational development later this year. The ongoing Phase I/II trial is comparing mRNA-1083 to a standard dose of Fluarix or high-dose Fluzone, depending on patient age, and Moderna’s own Spikevax booster for Covid-19. The combination vaccine is one of 15 products the US biotech aims to bring to market over the next five years. Moderna’s ambitious plans also include advancing up to 50 mRNA programs into the clinic within the same timeframe. But its jab candidate for influenza only, mRNA-1010, has yielded mixed data in the past, although it has since succeeded at Phase III.

- Research study demonstrates regulation of chromatin structure and cancer gene expression – - Findings to augment clinical trial in liver cancer - Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a clinical stage biopharmaceutical company focused on Artificial Intelligence (“AI”)-driven therapeutic drug development for the treatment of non-alcoholic steatohepatitis (“NASH”), fibrotic diseases, hepatocellular carcinoma (“HCC”), and other chronic diseases, today announced new research findings uncovering a previously unknown mechanism by which Hepion’s lead drug candidate, rencofilstat, may exert anti-cancer activity. The research involved a technique called ATAC-Seq (“assay for transposase-accessible chromatin with sequencing”) that determines what specific DNA sequences are in open or closed states. Gene expression occurs from DNA sites that are in an open state, whereas genes within closed regions are silent. Hepion’s ATAC-Seq experiments on two liver cancer cell lines revealed that rencofilstat significantly changed the open-closed state of DNA. Approximately 4,500 sites were altered within one hour of treatment in both cell lines, which was slightly more than a chemotherapeutic compound, azacytidine, known to alter DNA structure. The predominant effect of rencofilstat – opening or closing – depended on the cell line. Rencofilstat predominantly closed DNA in the cell line in which it also suppressed cell growth, suggesting that the drug candidate’s anti-cancer effect on the cells resulted in part from DNA closure and gene silencing. Another interesting finding was the types of genes that became accessible/inaccessible due to rencofilstat treatment. DNA sites that were opened by rencofilstat were enriched for several well-known tumor suppressor genes such as CDKN1A (p21), PTEN, HINT1, KLF6 and SOCS1; whereas a tumor-promoting oncogene, NOTCH1, was predicted to be silenced by rencofilstat. Accessibility also changed for many genes related to apoptosis, consistent with increasing cancer cell death. Thus, changes in gene accessibility produced by rencofilstat favored an anti-cancer mode of action. Follow-up experiments are planned to further understand the impact on cancer cell growth. “Anti-tumor effects of rencofilstat and other cyclophilin inhibitors observed in many preclinical studies have been attributed to several mechanisms, but changes in DNA accessibility and especially those that potentially limit oncogenesis have never before been reported for this class of drug,” explained Dr. Daren Ure, Hepion’s Chief Scientific Officer. “Knowing now that rencofilstat can modify DNA chromatin structure and regulate what genes are turned on and off gives us new appreciation for the scope of rencofilstat’s actions.” “Rencofilstat previously received Orphan Drug designation for the treatment of NASH. Hepion has also received FDA clearance to begin a Phase 2 clinical trial of rencofilstat in HCC, which remains the most common type of liver cancer, accounting for up to 90% of all primary liver cancers. We believe rencofilstat may be well-positioned to offer a unique approach to treating both NASH and HCC, as these diseases are often intertwined,” commented Hepion’s CEO, Robert Foster. “These newest findings reported today strengthen our resolve to initiate Phase 2 HCC trials pending adequate financial resources. More broadly, rencofilstat’s cumulative properties – liver-targeting, antifibrotic, anti-cancer, antiviral – reinforce Hepion’s focus on two of the most serious liver diseases of the day, NASH and HCC.” About Hepion Pharmaceuticals The Company's lead drug candidate, rencofilstat, is a potent inhibitor of cyclophilins, which are involved in many disease processes. Rencofilstat has been shown to reduce liver fibrosis and hepatocellular carcinoma tumor burden in experimental disease models and is currently in Phase 2 clinical development for the treatment of NASH. In November 2021, the U.S. Food and Drug Administration (“FDA”) granted Fast Track designation for rencofilstat for the treatment of NASH. That was followed in June 2022 by the FDA’s granting of Orphan Drug designation to rencofilstat for the treatment of HCC. Hepion has created a proprietary Artificial Intelligence deep machine learning (“AI/ML”) platform designed to better understand disease processes and identify patients that are rencofilstat responders. This AI/ML has the potential to shorten development timelines and increase the observable differences between placebo and treatment groups. In addition, Hepion’s AI/ML can be used to drive its ongoing NASH and HCC clinical development programs and identify other potential therapeutic indications for cyclophilin inhibition with rencofilstat. The content above comes from the network. if any infringement, please contact us to modify.