COLUMBUS, November 2023 – 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 TVR110, an engineered miRNA therapeutic in development for Charcot-Marie-Tooth disease type 1A (CMT1A), a progressively debilitating disorder with no approved treatments.
“We appreciated a productive dialogue with the FDA that provided clarity on our path forward to first-in-human trials of TVR110,” said Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. “The robust body of evidence we have generated to date indicates that TVR110 could serve as a powerful new option for people living with CMT1A. Accordingly, we are working expediently towards an IND submission.”
TVR110 is an AAV9-delivered engineered microRNA that reduces the PMP22 overexpression that is the underlying cause of CMT1A. Armatus is evaluating the safety and efficacy of a one-time intrathecal delivery for TVR110, which is designed to provide durable benefit. Preclinical data have demonstrated encouraging findings that vectorized RNAi can reduce PMP22 to healthy levels, with signs of improvement in nerve histology, repaired myelin and restored functional behavior.
About CMT1A
Charcot-Marie-Tooth disease type 1A is a peripheral nerve demyelination and axon degeneration disease caused by a duplication of the PMP22 gene, which leads to the production of an abnormal amount of peripheral myelin protein 22 (PMP22). The disease affects over 150,000 people in the U.S. and EU. It typically presents in the teenage or young adult years, and results in progressive muscle weakness and sensory loss in the extremities that often leads to severe disability, pain, and loss of independence over time. There are currently no disease-modifying therapies and no cure for CMT1A.
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.