On October 24, 2024 Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, reported that the Company presented compelling preclinical data highlighting the therapeutic potential of its first-in-class compound, SKY-1214, at the European Organization for Research and Treatment of Cancer (EORTC)-National Cancer Institute (NCI)-American Association for Cancer Research (AACR) (Free AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium ("EORTC-NCI-AACR" or the "Triple Meeting") (Press release, Skyhawk Therapeutics, OCT 24, 2024, View Source [SID1234647373]).
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SKY-1214 is a first-in-class, oral RNA splicing modulator discovered through the company’s novel RNA-splicing platform and being developed for difficult-to-treat multiple myeloma (MM) and non-Hodgkin’s lymphoma (NHL). SKY-1214 targets FANCL/FANCI, critical components of the Fanconi anemia DNA damage repair pathway, which MM and NHL cells use to maintain their genome integrity.
Study authors concluded that SKY-1214 shows potent and efficacious anti-cancer activity in MM and NHL models in vitro, including in models with high-risk cytogenetic and/or genetic alterations (e.g., t(14;16), t(4;14), 1q+, and double-hit lymphoma). Additionally, in vivo treatment with SKY-1214 as a monotherapy resulted in tumor growth inhibition and regression to undetectable levels at tolerated doses in NHL and difficult-to-treat MM xenograft mouse models, including KMS-28BM.
"We’re excited to present these data and to introduce SKY-1214, our most-advanced oncology program, ready for IND submission," said Sergey Paushkin, M.D., Ph.D., Co-founder, Head of R&D at Skyhawk. "There are a number of approved treatments for both MM and NHL, but all patients with MM eventually progress and only 50% of patients with certain NHL subtypes can be cured, underscoring the urgent need for new therapies with novel mechanisms of action. SKY-1214’s profound anti-cancer activity, including in these high-risk tumor cell lines, supports further development and exploration as a single agent and in combination treatment in these difficult-to-treat cancers."
The poster is available on the Skyhawk website at www.skyhawktx.com/resources.
About SKY-1214
SKY-1214 is a first-in-class, oral FANCL/FANCI targeting RNA splicing modulator being developed for difficult to treat multiple myeloma (MM) and non-Hodgkin’s lymphoma (NHL). FANCL/FANCI are critical components of the Fanconi anemia DNA damage repair pathway, which MM and NHL tumor cells use to maintain their genome integrity. Skyhawk has observed anti-tumor activity for SKY-1214 in a number of preclinical cell models, including those representing prevalent high-risk cytogenetic alterations. SKY-1214 also demonstrated anti-tumor activity in human tumor cell-derived xenograft mouse models correlating with FANCL/FANCI mRNA reduction. SKY-1214 has completed IND-enabling studies.