Preclinical in vivo data demonstrate hyper-precise mechanism directly targets the disease-causing mRNA repeats leading to correction of splicing defects and reversal of myotonia
Oral administration of lead drug candidate results in broad biodistribution, including to the muscle and heart
Company plans to file an IND with U.S. FDA in 2026 for an oral drug candidate to treat DM1
WALTHAM, Mass., Dec. 03, 2025 (GLOBE NEWSWIRE) — Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1), a form of muscular dystrophy. The results showed that oral administration of the company’s lead series of rSMs achieved broad biodistribution and corrected mRNA splicing and myotonia in an in vivo animal model of the disease. The data are being presented today at the 8th Annual RNA-Targeted Drug Discovery and Development Summit, taking place on December 2-4, 2025 in Boston, MA.
The DM1-targeted rSM selectively binds to the pathogenic trinucleotide RNA sequence, called a CUG repeat, that is responsible for DM1. This RNA-targeted mechanism of action disrupts the formation of nuclear aggregates, releases sequestered splicing factors, specifically Muscleblind-Like Splicing Regulator 1 (MBNL1), and corrects splicing defects responsible for myotonia. The preclinical data from in vivo studies demonstrated splicing correction and reversal of myotonia following oral administration with the lead series of rSMs. In addition, data demonstrated broad biodistribution, including to the skeletal muscle and heart, and pharmacokinetics that support once-daily dosing.
“We are thrilled to share the progress of our DM1 program as we move toward IND-enabling studies with our orally available RNA-targeted compounds showing efficacy in animal models of DM1,” said Michael Gilman, PhD, Chief Executive Officer of Arrakis Therapeutics. “DM1 is a multisystemic RNA-mediated disease in which morbidity and mortality extend beyond neuromuscular debilitation to include severe cardiovascular complications, emphasizing the need for biodistribution beyond skeletal muscle. We look forward to advancing our lead rSM drug candidate to an IND filing in 2026.”
The Company will describe additional progress emerging from several preclinical studies supporting its lead rSM drug candidate targeting DM1. The presentation will include the following highlights for results for the lead series of rSMs in the DM1 program: