Musculoskeletal
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.
The gene therapy for Duchenne muscular dystrophy passed the blockbuster sales threshold in 2024. Analysts said the drug’s overall risk/benefit profile still appears solid.
Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.
Scrip surveys some of the more interesting Phase III clinical trial readouts expected this year.
On the back of a stellar showing in Phase III, apitegromab is going to be filed in the US and Europe in the coming weeks for spinal muscular atrophy and CEO Jay Backstrom believes it will be a $2bn blockbuster.
The US biotech’s Duchenne muscular dystrophy drug is “undoubtedly the most successful gene therapy launch in all of history,” according to CEO Doug Ingram. “We have barely scratched the surface of the opportunity in front of us.”
Deal Snapshot: December is just over a week old but the UK giant has packed a lot in, signing collaborations with likes of Duality Biologics (cancer) and Muna Therapeutics (neurodegeneration). Now it is targeting fibrotic diseases and osteoarthritis after inking a deal with Relation Therapeutics.
The company has reached agreement with the US FDA on a pivotal trial for RGX-202 through an accelerated pathway.
Avidity hopes to complete enrollment in mid-2025 for an ongoing Phase III trial of its antibody-oligonucleotide conjugate in myotonic dystrophy type 1, which has no approved drug therapy.
A new muscle-building front is opening in the fight against obesity, with Lilly among the leaders. But it plans to remain tight-lipped on upcoming Phase II data for its myostatin inhibitor in combination with Novo’s Wegovy for fear of playing into the hands of its rival.
The Mereo-partnered drug is in pivotal development for osteogenesis imperfecta. Ultragenyx also recently reported positive Phase III data for a rare disease gene therapy.
The company sees a $5bn opportunity to treat children with achondroplasia and the less severe growth disorder variant, hypochondroplasia.
Phase I/II data for its ADC candidates in myotonic dystrophy type 1 and Duchenne muscular dystrophy indicate potential for biomarker-based accelerated approval in both diseases, Dyne asserts.
Just a month after an EU rejection, the French drugmaker’s oral retinoic acid receptor gamma agonist Sohonos has won a US thumbs up for the treatment of fibrodysplasia ossificans progressiva, an ultra-rare disorder that turns soft tissue into bone.
The UK major’s older C5 inhibitor product could soon receive EU approval for children and adolescents with the rare disease, but emerging competition from biosimilar developers and rival firms are starting to stack up.
The US approval of a more convenient formulation of its myasthenia gravis therapy, Vyvgart, should cement its market position as well as change the definition of what being "well-controlled means for patients", Argenx hopes.
Emerging Company Profile: Fresh from a $73m follow-on offering last year, the US biotech is readying its lead asset for a pivotal trial in ultra-rare but debilitating mineralization disorders that affect infants and children.
The US firm’s lead antibody oligonucleotide conjugate candidate has improved muscle strength and mobility in an early muscular dystrophy trial but some experts are disappointed by dose-specific data and a serious adverse event.
Emerging Company Profile: US firm Peptilogics is advancing through the clinic with its lead orthopedic anti-infective drug candidate based on an AI platform that utilizes algorithms to improve peptide design.