Solve FSH invests $3M to advance ARM-201 as FSHD treatment
MicroRNA-based therapy designed to target genetic cause of MD type
Solve FSH, a venture philanthropic organization, has invested $3 million to support the development of Armatus Bio‘s microRNA-based therapy ARM-201 as a treatment for facioscapulohumeral muscular dystrophy (FSHD).
ARM-201 is designed to block the production of DUX4, the protein that is abnormally produced in people with FSHD, a type of muscular dystrophy that primarily affects the muscles of the face, shoulders, and upper arms.
Armatus Bio late last year completed a pre-investigational new drug meeting with the U.S. Food and Drug Administration (FDA) — a type of meeting that takes place before an application to conduct clinical trials is submitted.
The new funding will be used toward preclinical studies supporting such an application, according to a joint press release.
“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.”
Funding will suport preclinical studies of FSHD treatment
FSHD is a type of MD, a group of genetic conditions that cause progressive muscle weakness and wasting. This specific disease type occurs when a region of DNA called D4Z4 — which includes the DUX4 gene — is activated, resulting in the production of the DUX4 protein in cells where it is not normally produced. This is believed to be toxic, particularly to muscle cells.
ARM-201 is an experimental therapy that uses an adeno-associated virus (AAV) engineered to deliver a microRNA-based therapy to muscle cells. microRNA is a form of RNA that regulates the activity of genes.
The treatment uses a next-generation AAV capsid, a virus’ protein shell, called AAV-SLB101, that was licensed from Solid Biosciences. According to the developer, that capsid has shown an improved ability to deliver its payload to skeletal and heart muscles, while maintaining a lower exposure in the liver. Also, a clinical trial in Duchenne muscular dystrophy has shown positive tolerability results.
By reducing DUX4 levels, the treatment is expected to halt muscle weakness and wasting, reduce inflammation, and lower oxidative stress —a type of cell damage that occurs when the production of oxidant molecules outweighs the body’s antioxidant defenses.
Preclinical studies of ARM-201 showed improvements in FSHD-associated biomarkers and muscle function, per the company.
We’re excited to take this next step together and accelerate the path toward a much-needed therapy for the FSHD community.
According to Rachel Salzman, CEO of Armatus Bio, said the funding “provides critical resources to advance ARM-201 toward the clinic.”
“Solve FSHD’s focused commitment to advancing therapeutic development for FSHD, combined with [its] collaborative approach and scientific expertise, has made [it] an invaluable partner for Armatus,” Salzman said. “We’re excited to take this next step together and accelerate the path toward a much-needed therapy for the FSHD community.”
The experimental therapy has been granted orphan drug and rare pediatric disease designations by the FDA. Such designations are meant to accelerate the development of therapeutic candidates for rare diseases, defined in the U.S. as those affecting fewer than 200,000 people.
Orphan drug status confers seven years of market exclusivity if a treatment is ultimately approved, and provides tax credits to its developer. Having rare pediatric disease status means that if ARM-201 is approved, Armatus Bio may receive a voucher for priority review of another therapy, which it could redeem or sell.
Solve FSHD is a philanthropic organization that supports research to slow or stop muscle degeneration and increase muscle regeneration and strength. It also aims to improve quality of life for people with FSHD.
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