INTRINSIC

Arrakis Therapeutics is developing an oral RNA-targeted small molecule (rSM) for the treatment of myotonic dystrophy type 1 (DM1). This lead wholly-owned program, known as the INTRINSIC program, utilizes a first-in-class small molecule that selectively binds to pathogenic CUG repeat expansions within the DMPK (myotonic dystrophy protein kinase) mRNA. By binding these repeats, the drug disrupts the formation of toxic nuclear RNA-protein aggregates and releases sequestered Muscleblind-Like Splicing Regulator 1 (MBNL1) protein. The restoration of functional MBNL1 levels corrects the mRNA splicing defects responsible for myotonia and other clinical symptoms of the disease. Preclinical studies have demonstrated broad biodistribution to skeletal muscle and heart tissue, as well as the correction of splicing and myotonia in animal models. Arrakis is currently conducting IND-enabling studies and expects to file an Investigational New Drug (IND) application with the FDA in late 2026.

Other names

INTRINSIC ProgramArrakis DM1 programCUG repeat RNA-targeted small molecule