Pexidartinib Hydrochloride

CAMBRIDGE, England, Nov. 1, 2023 /PRNewswire/ -- STORM Therapeutics Ltd. (STORM), a clinical stage biotechnology company discovering and developing novel small molecule therapies targeting RNA modifying enzymes (RMEs) for oncology and other diseases, is pleased to announce the appointment of Marguerite Hutchinson, J.D., as Chief Business Officer. Marguerite has over ten years' extensive experience in leading licensing agreements and collaborations with large, high profile healthcare companies as well as experience in legal and strategic operations. She was previously the Chief Operating Officer and General Counsel of Plexxikon Inc., a member of the Daiichi Sankyo Group, establishing and managing collaborations with Roche, Merck, AstraZeneca, Array and Cancer Research UK. She played a pivotal role in the out-licensing of bezuclastinib to Cogent Biosciences and plixorafenib to Fore Biotherapeutics, securing upfront payments and additional developmental milestones, and royalty payments. She was instrumental in the Phase 3 development and approval of TURALIO® (pexidartinib), a prescription medicine which is the first and only oral therapy for tenosynovial giant cell tumor. In addition, Marguerite successfully pursued the patent infringement case Plexxikon v. Novartis, resulting in a significant jury verdict award and ongoing royalties from dabrafenib sales. Jerry McMahon, Chief Executive Officer of STORM Therapeutics, said: "We are excited by the addition of Marguerite to the STORM leadership team as we continue to maximise collaborations for our developing pipeline of products targeting RNA modifying enzymes. Marguerite brings strategic vision to align our business with existing and new shareholders as we advance our current clinical program into additional advanced clinical studies." Marguerite Hutchinson, new Chief Business Officer of STORM Therapeutics, commented: "STORM is advancing the first RNA methyltransferase inhibitor to enter clinical development and I am delighted to be joining the team at a pivotal stage of clinical development with its lead candidate STC-15, which has the potential to significantly improve patient outcomes. I look forward to working closely with the stellar management team at STORM." Marguerite joins STORM from Tatara Therapeutics, where she was Chief Executive Officer, a UCSF spin-out which she set up and raised seed financing. Prior to this, Marguerite was Chief Business Officer at Frontier Medicines Corporation where she oversaw and led all business development activities including a collaboration with AbbVie. Marguerite received a B.A. from Harvard University and a J.D. from the University of San Diego School of Law and is based in US. About STORM Therapeutics STORM Therapeutics (STORM) is a clinical stage biotechnology company creating novel therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. There are more than 150 RNA modifications reported and approximately 300 RNA modifying enzymes which represent novel therapeutic targets. STORM has leveraged its first-mover advantage to establish a novel drug discovery and RNA analytics platform leading to the identification of novel targets and a proprietary pipeline of first-in-class small-molecule drug candidates for potential use in oncology, inflammation, viral infection and CNS diseases. The pipeline is exemplified by STORM's METTL3 inhibitor, STC-15, which has received IND approval and commenced its Phase 1 clinical study in cancer patients in November 2022. STC-15 represents the first ever RNA modifying enzyme inhibitor to enter clinical evaluation in humans. Additional programs are planned for advancement into IND-track activities in 2023. STORM investors include M Ventures, Pfizer Ventures, Taiho Ventures LLC, Seroba Life Sciences, Cambridge Innovation Capital I Limited, IP Group plc, UTokyo Innovation Platform Co., Ltd. (UTokyo IPC) and the Fast Track Initiative (FTI). For more information, please visit About STC-15 STORM's lead clinical program STC-15 is a first-in-class inhibitor of RNA modification and is the first ever RNA methyltransferase inhibitor to enter clinical development. STC-15 is an oral small molecule that inhibits METTL3, an RNA methyltransferase implicated in oncology and other diseases. Certain RNA methyltransferases are important regulators of RNA sensing and innate immune activation and represent novel immune-regulatory targets. STC-15 has also been shown preclinically to inhibit tumour growth through mechanisms involving anti-cancer immune responses, such as changes in interferon signalling and synergy with T cell checkpoint blockade. STORM commenced the dosing of the first patient in a Phase 1 clinical study of STC-15 in patients living with solid tumours in November 2022 and anticipates presenting first results from its study in 2024. Details of the study can be found on clinicaltrials.gov under the identifier NCT05584111. SOURCE STORM Therapeutics

XI'AN, China, Oct. 17, 2023 /PRNewswire/ -- Single-cell and spatially resolved omics have helped scientists take a closer look at tissue composition, structure, and function. This comprehensive understanding paves the way for them to uncover some intricate and previously unknown disease mechanisms. Three articles in the most recent special issue of the Journal of Pharmaceutical Analysis (JPA) discuss how single-cell and spatially resolved omics help unravel intercellular dynamics, aiding the development of novel therapeutic modalities. The first study focuses on microglia-astrocyte communication which may improve the therapeutic landscape of intracerebral hemorrhage (ICH). It is based on the notion that after a spinal cord injury or stroke, reactive astrocytes in the brain form a 'glial scar', that is lesser known in the context of ICH. To this end, the authors used spatial transcriptomics to investigate how microglial depletion (induced using PLX3397) affects astrocytic scar formation in the early and late stages of ICH. The article was available online in February 2023 and published in Volume 13, Issue 8 of JPA in August 2023. The researchers discovered that during early ICH, microglia-derived insulin-like growth factor 1 (IGF1) regulated protective astrocytic scar formation by activating the mechanistic target of rapamycin (mTOR) signaling pathway, which was further activated by the repopulating microglia. In chronic ICH, however, the scar had less protective and more neurotoxic effects. Interestingly, this effect could be partially reversed by delayed microglial depletion. The corresponding author Jianmin Zhang says, "Delayed microglial depletion may help with chronic ICH. Combined early-stage IGF1/osteopontin and late-stage PLX3397 treatment could be a promising therapeutic strategy for ICH." The second study attempted to investigate the testicular toxicity of triptolide, an active component of the traditional Chinese medicine Tripterygium wilfordii Hook. F., at the single cell level. Although triptolide is effective in the treatment of inflammatory diseases, its side effects, particularly testicular toxicity, limit its clinical use. The researchers used triptolide to treat mice, then isolated their testes and prepared single-cell suspensions. These were used to create a transcriptome map, which revealed that in treated mice, inflammatory response and reactive oxygen species signaling were upregulated, while germ cell and spermatid development were downregulated, indicative of triptolide-induced toxicity. "Our findings provide an invaluable resource for identifying the therapeutic targets of triptolide-induced male reproductive toxicity", explains corresponding author Dr. Chuanbin Yang. The third study elaborated on the single-cell and spatial analysis of epicardial cells in mouse heart tissue from embryonic day 9.5 to postnatal day 9. The team identified that the gene markers Msln, C3, Efemp1, and Upk3b were specific to postnatal (mature) epicardial tissue and were associated with increased extracellular matrix (ECM) signaling. Furthermore, while Wt1, Tbx18, and Upk3b were expressed at all stages, Msln, C3, and Efemp1 were expressed only in mature stages. "The findings of our research can enhance the current understanding of epicardial cells, contributing to the development of more effective cardiac regenerative therapy strategies. This, in turn, improves the prognosis of patients with myocardial infarction and propels advancements in the field of cardiac repair," comments author Dr. Ling Zhang. Together, these articles enlighten us with a deeper understanding of cellular mechanisms at the single-cell and spatial levels, offering hope for improved treatment strategies in the future. Reference Title of original paper: Temporal dynamics of microglia-astrocyte interaction in neuroprotective glial scar formation after intracerebral hemorrhage Journal: Journal of Pharmaceutical Analysis DOI: Media contact Mengjie Wang 367011@email4pr.com Xi'an, China +86-(0)29-88960092 View original content to download multimedia: SOURCE Journal of Pharmaceutical Analysis