Jess Ferko

COLUMBUS, May 12, 2025 – Armatus Bio, a late-preclinical stage biotech innovator developing vectorized RNAi medicines in neuromuscular disorders, today announced its recognition as one of eight finalists in the XPRIZE Healthspan FSHD Bonus Prize, led by XPRIZE, the world’s leader in designing and operating large-scale incentive competitions to solve humanity’s grand challenges.

SOLVE FSHD, a venture philanthropy organization dedicated to catalyzing innovation and overcoming barriers to accelerate new therapies for facioscapulohumeral muscular dystrophy (FSHD), is a co-sponsor of XPRIZE Healthspan and funder of the FSHD Bonus Prize, which is being run in parallel with the XPRIZE Healthspan.

“The XPRIZE Healthspan FSHD Bonus Prize is designed to solicit bold solutions intended to deliver measurable improvements in muscle function and biomarkers of FSHD disease progression in individuals affected by FSHD,” said Eva Chin, PhD, Executive Director of SOLVE FSHD. “Armatus’ drug candidate, ARM-201, represents a highly promising strategy that addresses the underlying genetic defect that causes FSHD. Furthermore, Armatus has devised a clear roadmap for bringing this experimental therapeutic into human clinical trials to demonstrate its ability to transform outcomes for this population.”

ARM-201 is a vectorized microRNA engineered with a second-generation myotropic capsid that has been designed to effectively, safely, 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 motor function.

“Recognition as a finalist in this highly competitive forum is a reflection of the promise, hard work, and rigor embedded within our scientific strategy for ARM-201, which originated in the Harper Lab at Nationwide Children’s Hospital,” said Rachel Salzman, DVM, Chief Executive Officer of Armatus Bio. “We applaud XPRIZE and SOLVE FSHD for their visionary leadership to champion innovative new ideas that carry the potential to dramatically impact care.”

The $101 million XPRIZE Healthspan is a 7-year global competition to catalyze the development of proactive, accessible therapeutic solutions that restore muscle, cognition, and immune function by a minimum of 10 years, with an ambitious goal of 20 years, in persons aged 50-80 years, in one year or less. Read more at https://www.xprize.org/healthspan.

About Armatus

Armatus Bio is a late-preclinical stage, privately held biotech innovator developing advanced medicines that leverage vectorized RNAi.  RNAi is a well-validated therapeutic approach that modifies protein expression via innate cellular biogenesis pathways without altering the cell’s genetic make-up. The company is advancing two assets addressing urgent unmet needs in neuromuscular disorders: TVR110 for Charcot-Marie-Tooth disease type 1A (CMT1A), and ARM-201 for Facioscapulohumeral Muscular Dystrophy (FSHD). In preclinical studies, these investigational drugs demonstrated robust early signals of precision target engagement and biomarker improvement, and both are now advancing toward preparations for clinical trials. For more information, visit www.ArmatusBio.com.

Vancouver, British Columbia and Columbus, Ohio [May 6, 2025] — SOLVE FSHD, a venture philanthropy organization committed to accelerating therapies for facioscapulohumeral muscular dystrophy (FSHD), today announced a $3 million investment in Armatus Bio, a biotechnology company developing next-generation vectorized RNAi therapeutics for neuromuscular diseases. The investment will support ARM-201, Armatus Bio’s AAV-delivered microRNA therapy designed to silence the expression of DUX4, the toxic protein responsible for muscle degeneration in individuals with FSHD.

The investment is part of SOLVE FSHD’s growing portfolio of therapeutic programs aimed at addressing FSHD through a range of innovative approaches. ARM-201 is a potential best-in-class, single-dose therapeutic for the treatment of FSHD. Its proprietary miRNA payload is designed to reduce DUX4 expression and thereby arrest muscle weakening and atrophy, prevent further degeneration, and reduce inflammation and oxidative stress associated with the disease.

ARM-201 incorporates AAV-SLB101, a next-generation AAV capsid licensed from Solid Biosciences, which was rationally designed to target integrin receptors and has shown enhanced skeletal and cardiac muscle transduction, with decreased liver exposure in preclinical studies. It has also been shown to be well tolerated with robust transduction and expression levels seen in a clinical trial for Duchenne muscular dystrophy. With a pre-IND meeting successfully completed with the FDA, Armatus Bio will deploy SOLVE FSHD’s investment towards critical IND-enabling activities, positioning ARM-201 for regulatory submission and entry into FSHD clinical trials.

“We’re thrilled to support Armatus Bio in advancing a differentiated therapeutic approach that directly targets the genetic cause of FSHD,” said Eva Chin, PhD, Executive Director of SOLVE FSHD. “This program represents an important step toward delivering meaningful treatments to the FSHD community. We believe Armatus has a strong scientific foundation, experienced leadership, and a focused development strategy that position them well for clinical and commercial success.”

“SOLVE FSHD’s focused commitment to advancing therapeutic development for FSHD, combined with their collaborative approach and scientific expertise, has made them an invaluable partner for Armatus,” said Rachel Salzman, DVM, CEO of Armatus Bio. “This investment provides critical resources to advance ARM-201 toward the clinic and positions us for continued growth as a company. We’re excited to take this next step together and accelerate the path toward a much-needed therapy for the FSHD community.”

FSHD is one of the most common forms of muscular dystrophy, affecting approximately 1 in 8,000 individuals globally. With no approved treatments available, advancing promising therapies is critical to meeting the urgent needs of the FSHD community.

About SOLVE FSHD

SOLVE FSHD is a venture philanthropic organization established to catalyze innovation and accelerate key research in finding a cure for FSHD. Established by renowned Canadian entrepreneur and philanthropist, Chip Wilson, widely known as the founder and part owner of various technical apparel companies including lululemon and Amer Sports. The Wilson family has committed $100 million to kick-start funding into projects that support the organization’s mission to solve FSHD by 2027. The goal of SOLVE FSHD is to find a solution that can slow down or stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for those living with FSHD.

https://www.solvefshd.com

About Armatus Bio

Armatus Bio is a privately held late preclinical stage biotechnology company leveraging vectorized RNAi to address urgent unmet medical needs in genetically-driven neurological diseases. Based in Columbus, Ohio, the company is led by a seasoned team with expertise in drug development and delivery, and partnered with world renowned experts in vector biology, genomics, and neurology. In addition to ARM-201, Armatus is building a pipeline of precision medicines that can transform care for people with other genetic neuromuscular disorders.

https://www.armatusbio.com

Media Contacts:

SOLVE FSHD:
Andrea Mestrovic
Very Polite Agency
andrea@weareverypolite.com

Armatus Bio:
Kellie Hotz
khotz@armatusbio.com

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.