Musculoskeletal
Deal snapshot: Preparing for submission and potential approval and launch of its Phase III sleep apnea drug, Apnimed is divesting its half of Shionogi-Apnimed Sleep Science for $100m.
Underwhelming efficacy led to the myostatin inhibitor's discontinuation of in SMA and FSHD, but Roche will continue a separate study in obesity. Meanwhile, Scholar Rock’s rival apitegromab could soon be back on track for approval.
The company aims to restore near-normal muscle regrowth with its novel in vivo gene therapy PBGENE-MD, which it is now pursuing alone.
Since being taken private by B-FLEXION, Paratek has been building out its commercial portfolio and Radius’s osteoporosis drug Tymlos will advance that strategy.
After recent upheaval at the US FDA, the company’s CEO is unsure that an agreed fast-track pathway will still be in place for its Duchenne gene therapy, RGX-202.
The company presented updated data from the Phase III pivotal trial and plans to file for US FDA approval in the first half of the year.
The company still plans to report data from the 5mg/kg cohort of the oligonucleotide in myotonic dystrophy type 1 in Q1 and from the 10mg/kg cohort in the second half of the year.
The controversial Doug Ingram will depart for personal reasons, following the diagnosis of two family members with myotonic dystrophy.
The company’s positive topline Phase III results for the oral drug showed improvements in growth and body proportionality, including gains not seen with standard-of-care injections.
The FDA cited various issues with the CAMPSIITE trial in Hunter syndrome and offered multiple paths forward, but the biotech said that could be a challenge given the rarity of the disease.
Another Phase III hit for Lilly’s Zepbound, the TOGETHER-PsA trial is the first to add an incretin therapy to anti-inflammatory biologic, potentially expanding its reach into another major comorbidity market.
A deal for the Duchenne muscular dystrophy treatment with Nxera is valued at up to $205m.
Analysts warn of an uphill battle for setrusumab despite secondary endpoint wins after disappointing Phase III results dampens the drug's regulatory prospects.
The drugmaker announced that the pivotal trial of fidrisertib in FOP did not meet its primary endpoint, but it already markets the first ever drug to win FDA approval for the disease.
The California firm announced positive data from its early-stage randomized study of TLC-6740 combined with Lilly’s tirzepatide in patients with obesity.
Satellos is moving to start a double-blinded Phase II trial of its experimental Duchenne muscular dystrophy candidate that looks to mimic the effects of dystrophin on muscle stem cells.
Five-year Agamree data shows the novel steroid is a safer and equally effective treatment to standard of care deflazacort or prednisone, the Swiss biotech argues.
The company’s third quarter earnings fell by $67.8m year-over-year, reflecting the tumultuous situation around its DMD therapy Elevidys in June.
Despite a prior agreement with the FDA to seek accelerated approval, with Phase III data showing statistical significance on biomarkers and functional measures, BridgeBio is aiming higher.
The deal comes on the back of promising data from Avidity's next-generation RNA therapeutics over the past two years and a subsequent meteoric rise in share value.