Skyhawk Therapeutics Shares Promising Preclinical Data on SKY-1214 at Cancer Symposium

Skyhawk Therapeutics, Inc., a biotechnology company at the clinical stage of development, has announced promising preclinical findings for their innovative 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. SKY-1214, an oral modulator of RNA splicing, is being developed for the treatment of difficult-to-treat multiple myeloma (MM) and non-Hodgkin’s lymphoma (NHL).

SKY-1214 targets FANCL/FANCI, crucial components of the Fanconi anemia DNA damage repair pathway, which is exploited by MM and NHL cells to sustain their genomic integrity. Preclinical studies indicate that SKY-1214 exhibits potent anti-cancer activity in both MM and NHL models, including those with high-risk genetic alterations, such as t(14;16), t(4;14), 1q+, and double-hit lymphoma. Furthermore, in vivo studies with SKY-1214 as a monotherapy have shown significant tumor growth inhibition and regression to undetectable levels in NHL and difficult-to-treat MM xenograft mouse models, such as KMS-28BM.

Dr. Sergey Paushkin, Co-founder and Head of R&D at Skyhawk, highlighted the significant anti-cancer activity of SKY-1214 and its potential as a new therapeutic option for patients with MM and NHL. Despite existing treatments, patients with MM inevitably experience disease progression, and only half of those with certain NHL subtypes achieve a cure. These facts underscore the urgent need for novel therapies with unique mechanisms of action. SKY-1214’s robust anti-cancer capabilities, particularly in high-risk tumor lines, support its further development both as a standalone treatment and in combination therapies for challenging cancer types.

Skyhawk has discovered SKY-1214 through its proprietary RNA-splicing platform and is advancing it towards clinical development. SKY-1214 has shown anti-tumor activity in various preclinical cell models, including those with prevalent high-risk genetic alterations. It has also demonstrated efficacy in human tumor cell-derived xenograft mouse models by correlating with reductions in FANCL/FANCI mRNA levels. SKY-1214 has completed studies required for IND (Investigational New Drug) submission, paving the way for future clinical trials.

Skyhawk Therapeutics, based in Boston, MA, focuses on the discovery and development of small molecule therapies that target RNA splicing mechanisms. Their innovative drug discovery platform enables the identification and testing of RNA splicing targets across various therapeutic areas and diseases. This platform has facilitated partnerships with multiple pharmaceutical companies to leverage Skyhawk’s unique approach in areas such as neurodegenerative diseases, autoimmune disorders, and oncology.

The company’s recent presentation at the EORTC-NCI-AACR symposium highlights their commitment to developing groundbreaking therapies for some of the most challenging diseases. The advancement of SKY-1214 represents a significant milestone in Skyhawk’s mission to revolutionize patient treatment through the modulation of RNA targets.