PIPELINE
First-in-class, oral genetic medicine to treat Myotonic Dystrophy Type 1 (DM1)
A novel oral approach to
treat the root cause of DM1
Arrakis’s RNA-targeted small molecule (rSM) selectively binds to the pathogenic trinucleotide RNA sequence, called a CUG repeat, that is responsible for DM1. Our rSM has broad biodistribution to address the multisystemic manifestations of DM1 that extend beyond neuromuscular debilitation to also cause severe cardiovascular complications.
DM1 is a debilitating disease with no approved treatments
DM1 is a genetic neuromuscular disease affecting at least 1 in 8,000 people worldwide or ~45,000 people in the United States. It is a multi-systemic disease, affecting the heart, skeletal muscle, the central nervous system, and the gastro-intestinal tract. Cardiovascular complications are the leading cause of mortality in patients with DM1.
There are currently no approved drugs to treat DM1, and while there are promising oligonucleotide therapies in clinical development, these may not address the full systemic manifestations of DM1 because of their limited biodistribution.
Myotonic dystrophy type 1 (DM1) is caused by an abnormal expansion of CTG repeats in the DMPK (myotonic dystrophy protein kinase) gene. This change produces an mRNA with an unusually long stretch of CUG repeats that folds into unstable shapes. These RNA structures form toxic aggregates that bind and sequester important regulatory proteins like MBNL1 (muscleblind-like protein), disrupting normal RNA processing and leading to the symptoms of the disease.
Our hyper-precise mechanisn to target DM1’s underlying pathology
Our therapeutic approach is a small molecule that selectively and potently binds to the toxic CUG-repeat RNA, breaking down the aggregates and releasing MBNL1 to restore normal splicing and cellular function.
Promising initial data for our first-in-class rSM
Our lead drug candidate, is a first-in-class, orally administered, RNA-targeted small molecule in development for myotonic dystrophy type 1 (DM1). It blocks the formation of toxic RNA aggregates and restores normal splicing in patient-derived cell models, while correcting splicing and reversing myotonia in a validated animal disease model.
With encouraging preclinical data, we expect to enter the clinic in late 2026 to address the broad, multisystem manifestations of DM1—including cardiovascular complications—with an oral therapy designed for wide biodistribution.
