COLUMBUS, December 2024 – Armatus Bio, a late-preclinical stage biotech innovator developing vectorized RNAi medicines in neuromuscular disorders, completed a successful pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration to advance ARM-201. This engineered microRNA therapeutic is in development to treat Facioscapulohumeral Muscular Dystrophy (FSHD), a devastating neuromuscular disorder with no approved disease-modifying drugs.
“This encouraging feedback from the FDA allows us to advance our development program, which is intended to represent an important new therapeutic strategy for people living with the significant physical, mental and social burden of FSHD,” said Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. “With FDA input, we are working to rapidly advance through IND-enabling activities to pursue first-in-human studies of this novel therapy.”
ARM-201 is an AAV-delivered microRNA engineered with a second-generation myotropic capsid that has been designed to safely, effectively and durably silence toxic DUX4 expression. Preclinical evaluations have generated multiple datasets that strongly support continued pursuit of the vectorized RNAi strategy, including improvements in FSHD-linked biomarkers and neuromotor function.
About Facioscapulohumeral Muscular Dystrophy
FSHD is a devastating neuromuscular disorder caused by mutations that lead to abnormal activation of the DUX4 gene, which is highly toxic to muscle cells. This progressive disease affects up to 70,000 people in the U.S. and EU and can be diagnosed at any age. FSHD often manifests as a profound physical disability affecting the upper and lower extremities with as many as 20% of individuals becoming confined to a wheelchair. Severe chronic fatigue, pain, and emotional distress are also commonly reported. There are currently no FDA-approved disease-modifying therapies or curative interventions for FSHD.
About Armatus
Armatus Bio is a late-preclinical stage, privately held biotech innovator developing advanced medicines that leverage vectorized RNAi (RNA interference). Armatus’ uniquely specific, engineered microRNAs are noncoding RNAs responsible for regulating gene expression by mirroring innate cellular biogenesis processes without altering the underlying genetic make-up. The company’s two lead assets are designed to target neuromuscular disorders: TVR110 for Charcot-Marie-Tooth disease type 1A (CMT1A), and ARM-201 for Facioscapulohumeral Muscular Dystrophy (FSHD), which together affect more than 225,000 people in the U.S. and European Union. In preclinical studies, these investigational drugs demonstrated robust signals of target engagement and biomarker improvement, and both are advancing toward the clinic. For more information, visit www.ArmatusBio.com.