Arrowhead Pharmaceuticals has dosed the first participants in a Phase I/IIa clinical trial of ARO-DM1, an investigational RNA interference (RNAi) therapeutic, aimed at treating type 1 myotonic dystrophy (DM1).
The study is a double-blinded, placebo-controlled, dose-escalating trial set to enrol up to 48 patients.
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It will assess the tolerability, safety, pharmacokinetics, and pharmacodynamics of ARO-DM1 in comparison to a placebo.
Both male and female subjects with DM1 will be part of the study, receiving either single (Part I) or multiple (Part II) doses of the therapeutic or a placebo.
ARO-DM1 is engineered to reduce the dystrophia myotonica protein kinase (DMPK) gene expression in muscle tissue.
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The pathogenesis of DM1 involves an expanded CUG trinucleotide repeat in the DMPK gene transcripts. The abnormal transcripts lead to spliceopathy, which is directly associated with DM1 symptoms.
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By GlobalDataPreclinical studies have shown promising results, with ARO-DM1 achieving over 80% silencing of DMPK in skeletal muscle of nonhuman primates, a result that persisted for more than 85 days.
Furthermore, in a DM1 mouse model, a species-specific version of ARO-DM1 rectified spliceopathies by reducing pathogenic DMPK-CUG expression.
In November, Arrowhead Pharmaceuticals sought regulatory approval to begin the Phase I/IIa trial of ARO-DM1, with submissions to a local Ethics Committee and the New Zealand Medicines and Medical Devices Safety Authority.
DM1 patients typically experience muscle weakness and wasting, cataracts, myotonia, and cardiac conduction issues. These symptoms often lead to physical disability and a reduced lifespan.
Currently, treatments for DM1 are limited to managing symptoms, as no disease-modifying therapy has been approved.
Last October, Arrowhead and GSK entered an agreement with Janssen Pharmaceuticals (Janssen), a Johnson & Johnson company, to acquire the sole global rights for the subsequent development and marketing of JNJ-3989 to GSK.
Janssen in-licensed JNJ-3989 from Arrowhead in 2018.
