On March 22, 2022 KSQ Therapeutics (KSQ), a clinical-stage biotechnology company developing therapies to treat cancer and autoimmune diseases using its proprietary, integrated discovery CRISPRomics platform, reported that will give an oral presentation as part of the "New Drugs on the Horizon: Part 1" session at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting in New Orleans (Press release, KSQ Therapeutics, MAR 22, 2022, View Source [SID1234610615]). The company will provide an overview of preclinical data on its first-in-class USP1 inhibitor, KSQ-4279, which supports the ongoing Phase 1 clinical trial of KSQ-4279 being conducted in patients with advanced solid tumors.
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Presentation Title: ND01 – KSQ-4279: A first-in-class USP1 inhibitor for the treatment of cancers with homologous recombination deficiencies
Session Title: DDT001 – New Drugs on the Horizon: Part 1
Session Date and Time: Sunday, April 10, 2022, 1:05-1:20 pm CT
Location: La Nouvelle Orleans A-B, Convention Center
Data Highlights:
First disclosure of KSQ-4279 structure, a potent and selective USP1 inhibitor
KSQ-4279 has a unique mode of allosteric binding to USP1 and a high degree of selectivity over other family members
Efficacy was observed both as a single agent and in combination with PARP inhibitors across multiple BRCA/HRD xenograft models
Functional genomic resistance screens indicate that the major genetic drivers of resistance to USP1 and PARP inhibitors are distinct, indicating that combination treatment may be able to delay or prevent the emergence of resistance
Favorable safety profile without overlapping toxicities suggests broad combination potential with PARP inhibitors
"Based on the preclinical data we have observed from our studies of KSQ-4279, we believe it has great potential in a number of solid tumors, and we are looking forward to sharing the results from our ongoing Phase 1 clinical study in the future," said Frank Stegmeier, Ph.D., Chief Scientific Officer of KSQ. "USP1 is the first of multiple exciting targets that our CRISPRomics platform identified. In addition to KSQ-4279, we’re advancing multiple engineered TIL (eTILTM) adoptive cell therapy programs towards the clinic."
KSQ-4279
KSQ-4279 is a first-in-class small molecule targeting USP1, a protein regulating DNA damage response (DDR). USP1 was identified by KSQ’s CRISPRomics platform as a novel synthetic lethal target in cancers with certain types of genomic instability. KSQ-4279 is currently in a Phase 1 study in patients with advanced solid tumors.