COLUMBUS, October 2024 – Armatus Bio, a late-preclinical stage biotech innovator developing vectorized RNAi medicines in neuromuscular disorders, has received Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA) for ARM-201, a vectorized engineered microRNA therapy under development to treat Facioscapulohumeral Muscular Dystrophy (FSHD), a devastating neuromuscular disorder with no approved disease-modifying drugs.
“We recognize the significant burden of FSHD on patients and their families, and are working with urgency to advance what we believe is a promising therapeutic innovation to address the underlying genetic cause of the disease,” said Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. “These designations from the FDA further emphasize the need for novel options and support our ability to move efficiently toward clinical evaluation.”
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.
ODD is granted to therapeutic candidates for conditions affecting fewer than 200,000 people in the U.S. This designation provides incentives to advance clinical development with potential for up to seven years of U.S. market exclusivity if the product is approved for its designated indication. RPD is available for therapeutics targeting diseases that primarily affect children ages 18 or younger and fewer than 200,000 people in the U.S. Once awarded, these vouchers may be transferred or sold to other companies.
About Facioscapulohumeral Muscular Dystrophy (FSHD)
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 the 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.