Clinical research
Gene therapy company Sarepta Therapeutics provided additional data for SRP-5051 in treating Duchenne muscular dystrophy patients who are amenable to exon 51 skipping.
After VX-548 demonstrated significant pain relief in surgical and non-surgical settings, Vertex Pharmaceuticals is preparing to file a New Drug Application for the non-opioid candidate by mid-2024.
Bristol Myers Squibb’s PD-1 inhibitor was unable to outperform the placebo arm in improving disease-free survival in renal cell carcinoma patients at high risk of relapse after surgery.
The blockbuster immunotherapy secured separate late-stage victories as an adjuvant-setting treatment in renal cell carcinoma and muscle-invasive urothelial carcinoma.
While the FDA continues to investigate reported cases of T cell malignancies in patients who received CAR-T therapies, the cause of the secondary cancers remains unclear.
Despite myriad stumbling blocks over the past two years, experts still see the potential of Bruton’s tyrosine kinase inhibitors to quiet progressive multiple sclerosis.
In the CONTACT-02 pivotal trial, the combination of Exelixis’ Cabometyx and Roche’s Tecentriq reduced the risk of disease progression or death by 35% in patients with metastatic castration-resistant prostate cancer.
After 26 weeks of follow-up, five of six children treated with an experimental gene therapy developed in China showed strong improvements in speech perception and could hold normal conversations.
While decentralized trials are expediting innovation and promoting diversity, challenges remain, including complex stakeholder ecosystems, skills gaps and regulation.
The company said it’s preparing to file a New Drug Application with the FDA on the strength of late-stage study data for its investigational RNA-targeted donidalorsen in patients with hereditary angioedema.
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