SKY-1214, an IND-ready first-in-class FANCL/FANCI RNA splicing modulator, demonstrates tumor growth inhibition and regression to undetectable levels in NHL and difficult-to-treat MM xenograft mouse models

BOSTON, Oct. 24, 2024 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, today announced 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) Molecular Targets and Cancer Therapeutics Symposium ("EORTC-NCI-AACR" or the "Triple Meeting").

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 .

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.

About Skyhawk Therapeutics
Skyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world's most intractable diseases. Skyhawk's discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states. Skyhawk has built collaborations with multiple pharma partners that leverage Skyhawk's novel platform across disease areas including neurodegenerative disease, autoimmune disease, and oncology. For more information visit .

Skyhawk Contact
Kyle Dow, VP Corporate Development
[email protected]

SOURCE Skyhawk Therapeutics