Musculoskeletal

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.  

Faced with a scarily short cash runway and an ongoing expensive mid-stage study of an allogeneic cell therapy for the treatment of delayed-union bone fractures, Bone Therapeutics has found a saviour in France's Medsenic.

Major Korean firm Yuhan is seeing progress across multiple novel drug candidates licensed out to global partners, with two now entering pivotal trials. An innovative therapy for degenerative disc disease licensed to Spine BioPharma has received an FDA nod for a Phase III program and may change the standard of care for chronic low back pain patients.

The company said the study of JTA-004 failed to meet its primary and key secondary endpoints, though a post-hoc analysis indicated activity in a patient subset.

Ipsen has withdrawn the US NDA for palovarotene in an ultra-rare bone condition in order to conduct additional data analyses, but intends to resubmit once they are complete.  

The knee osteoarthritis injection was hit hard by COVID-19 restrictions, at first. But CEO Mike Clayman reflected in an interview about how the pandemic experience is helping fuel further growth for Zilretta.   

South Korean stem cell therapy company Medipost is speeding up the entry of flagship product Cartistem into global markets, with an initial focus on ASEAN countries and Japan and helped by regulators' allowance of early local trials to be waived on the back of strong Korean data.

Trial of high-need gene therapy set to restart following the deaths of several participants and the lifting of a clinical hold imposed by the US FDA last year.

Ultragenyx hopes to expand its bone disease franchise in a deal that frees up Mereo to focus on its anti-TIGIT immuno-oncology pipeline asset.    

Buoyed by an $81m Series A financing round, Faze Medicines joins Dewpoint Therapeutics and Nereid Therapeutics in the race to develop small-molecule drugs targeting biomolecular condensates, an emerging field of cell biology.