Jess Ferko

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.

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.

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.

COLUMBUS, October 2023 – 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 TVR110, an advanced engineered miRNA under development for Charcot-Marie-Tooth disease type 1A (CMT1A), a progressively debilitating disorder with no approved treatments.

“Our receipt of ODD and RPD from the FDA highlights the overwhelming need for therapeutic innovation to support people living with CMT1A in the U.S.,” said Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. “We continue to build momentum with TVR110 based on robust preclinical signals, and are now actively advancing this asset toward the clinic.”

TVR110 is an AAV-delivered microRNA that safely reduces PMP22 overexpression, which is the underlying cause of CMT1A. Preclinical evaluations have demonstrated encouraging findings that the vectorized RNAi strategy can bring PMP22 down to healthy levels, with signs of improvement in nerve histology, repaired myelin and restored functional behavior.

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 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 leads to progressive muscle weakness and sensory loss in the extremities that often results in severe disability, pain, and loss of independence. There are currently no approved disease-modifying therapies or curative interventions 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.