The oral drug reduced DUX4 in FSHD patient muscle cells and increased lean mass and muscle power in a human study
· Targets the cause: reduced DUX4 and its target genes in FSHD patient-derived muscle cells, pointing to disease-modifying potential
· Builds muscle: +0.8 kg lean mass and about 13% more lower-extremity muscle power in an eight-week human study
· €3 million Eurostars programme led by Atrogi in collaboration with LUMC, Karolinska Institutet, Stockholm University, Optics11 Life and NIRI
STOCKHOLM, Sweden — 22/10/26 — Atrogi AB has been awarded Eurostars funding for STAMINA, a €3 million programme to develop its first-in-class oral drug ATR-258 for facioscapulohumeral muscular dystrophy (FSHD) and, in time, other muscle-wasting diseases such as Duchenne muscular dystrophy, spinal muscular atrophy and age-related muscle loss. FSHD is one of the most common muscular dystrophies, affecting roughly one in 8,500 people, and has no approved treatment.
“This Eurostars award is a strong endorsement of our platform,” said Dr Paul Little, Chief Executive Officer of Atrogi. “With preclinical data pointing to disease modification and human data showing gains in muscle power, FSHD is a natural first rare disease for ATR-258.”
FSHD is driven by DUX4, a gene that is normally silent in adult muscle but switched on in people with the disease, progressively weakening the muscles of the face, shoulders and limbs. ATR-258 is designed to act on both the cause and its consequence. In preclinical studies in FSHD patient-derived muscle cells, it reduced DUX4 and the genes it switches on, pointing to disease-modifying potential. In an eight-week human study in 13 adults with overweight or obesity, it increased lean mass by 0.8 kg and lower-extremity muscle power by approximately 13%, and was well tolerated.
“This program represents an exciting opportunity for both the FSHD field and the patients who urgently need new treatment options,” said Professor Silvère van der Maarel, whose group at Leiden University Medical Center (LUMC) led the discovery of the genetic causes of FSHD. “The unmet medical need in FSHD remains substantial, and ATR-258 has the potential to offer a unique dual benefit by promoting muscle growth while simultaneously suppressing DUX4, the underlying driver of the disease. I am excited to work with Atrogi to advance this innovative approach.”
ATR-258 comes from Atrogi's GRK2-biased β2-agonist platform, described in Cell in 2025 together with a 69-subject Phase 1 study. Conventional β2-agonists have increased muscle mass in FSHD trials, but lose their effect as the receptor desensitises and carry cardiovascular side effects. ATR-258 is designed to avoid both, as a once-daily oral treatment.
“What began as basic research in my lab at Stockholm University has now gone from bench to clinic,” said Professor Tore Bengtsson, founder and Chief Scientific Officer of Atrogi. “Our first-in-human trial and an investigator-initiated study have shown that ATR-258 is well tolerated and acts on human muscle, opening clear opportunities in muscle-sparing weight loss and sarcopenia. We now see great potential in rare muscle disease, and FSHD is where we start.”
Within STAMINA, ATR-258 will be tested in 3D muscle models grown from FSHD patients' own cells at LUMC. The results will inform a first clinical trial of ATR-258 in people with FSHD, which Atrogi is preparing for 2027.
“Helping patients has always been the reason behind this work,” said Hamza Bokhari, Chairperson of the STAMINA Steering Committee. “STAMINA is an opportunity to bring that ambition closer to reality by taking a promising scientific approach forward in FSHD
“The lack of approved treatments makes every promising drug development program important to the FSHD community,” said the Dutch FSHD Community. “We are thrilled that Atrogi has selected FSHD as the first rare disease indication for ATR-258 and look forward to seeing the potential of this novel muscle-targeting therapy explored in FSHD.”
About STAMINA
STAMINA (Stimulation of Adrenergic Receptors for the Inhibition of Muscular Atrophy) is a 36-month programme co-funded through Eurostars [and Vinnova]. It is led by Atrogi with Karolinska Institutet, Stockholm University, Leiden University Medical Center, Optics11 Life and the National Research and Innovation Institute. Its focus is FSHD, and it will also assess other muscle-wasting conditions. [Project reference: Eurostars 11249.]
About Atrogi
Atrogi AB is a Stockholm-based, clinical-stage biotech pioneering novel GPCR pathway-signaling modulators to transform metabolic and muscle health. Its lead candidate, ATR-258, is a first-in-class oral GRK2-biased β2-agonist designed to safely stimulate muscle metabolism and preserve lean mass, offering a unique profile in muscle sparing weight loss, sarcopenia and diseases characterised by muscle loss. Atrogi's proprietary compound library, developed under the scientific leadership of Professor Tore Bengtsson, serves as the foundation for a broader platform aimed at discovering selective GPCR modulators across multiple disease areas. Learn more at www.atrogi.com.
Media contact
Paul Little, Chief Executive Officer, Atrogi AB · info@atrogi.com
