Arrakis Therapeutics Presents Data Highlighting Progress of Oral RNA-targeted Small Molecule to Treat Myotonic Dystrophy Type 1 (DM1)
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:
- DM1-targeted rSMs demonstrated reversal of myotonia in the HSALR DM1 animal model.
- DM1-targeted rSMs demonstrated favorable overall drug properties and dose-dependent splicing recovery after oral dosing.
- DM1-targeted rSMs showed good exposure in heart tissue and demonstrated splicing correction in cultured cardiomyocytes.
“Our structure-based optimization of rSMs has accelerated over the past year, culminating in a lead compound that is orally active and has successfully completed preliminary safety and ADME characterization,” said Domi Stickens, PhD, Chief Scientific Officer of Arrakis Therapeutics. “This shows the success of our approach to rationally design a small molecule purpose-built to address an RNA disease target, setting the stage for advancing our DM1 candidate as well as building our pipeline of rSMs for other diseases.”
About the rSM Program to Treat Myotonic Dystrophy Type 1 (DM1)
Myotonic dystrophy type 1 (DM1) is a form of muscular dystrophy and a genetic neuromuscular disease affecting at least 1 in 8,000 people worldwide or approximately 45,000 people in the United States. It is a multisystem disease, affecting the skeletal muscle, heart, diaphragm, central nervous system, and gastro-intestinal tract. DM1 is caused by a trinucleotide (CUG) repeat expansion of the RNA encoding myotonic dystrophy protein kinase (DMPK), resulting in the formation of nuclear aggregates that bind and sequester splicing factors important for cellular function. Arrakis’s rSM selectively binds to CUG repeats, disrupting aggregate formation in cells, liberating splicing factors, and correcting splicing defects in genes that drive DM1 pathology, including myotonia.
About Arrakis Therapeutics
Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on genetically validated targets and numerous disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. For more information, please visit www.arrakistx.com and engage with us on LinkedIn.
Media Contact Kathryn Morris, The Yates Network LLC 914-204-6412 kathryn@theyatesnetwork.com
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