Clinical Trial Spotlight: Boston Pharma, Intellia, Arrowhead
A look at some of today’s top clinical trial news
Boston Pharma Presents Positive Phase II MASH Data
Boston Pharmaceuticals (Cambridge, Mass.) will present positive Phase II data of once-monthly efimosfermin alfa, a long-acting FGF21 analogue, in patients with stage F2/F3 fibrosis from metabolic dysfunction-associated steatohepatitis (MASH). MASH, formerly called NASH, can lead to liver cirrhosis but is not associated with alcohol intake. Efimosfermin treatment led to significant improvements in fibrosis without making MASH worse, and MASH resolution without worsening of fibrosis over 24 weeks. The company is presenting the data on Nov. 19 in a late-breaking oral presentation at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting in San Diego.
Intellia Announces Early Phase I Data in Gene Editing Therapy for Transthyretin Amyloidosis
Intellia Therapeutics (Cambridge, Mass.) announced positive new data from the ongoing Phase I trial of nexiguran ziclumeran (nex-z, or NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. The drug is an in vivo CRISPR-based gene editing therapy. It is being developed with Regeneron. ATTR amyloidosis is a rare, progressive and fatal disease caused by mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a tendency to misfold. This caused an accumulation of amyloid in the body, causing serious complications in various tissues, including the heart, nerves and digestive system.
Arrowhead Presents New Data from Phase III Atherosclerotic Cardiovascular Trial
Arrowhead Pharmaceuticals (Pasadena, Calif.) presented new results from the Phase III PALISADE trial and open-label extension from the Phase II MUIR and SHASTA-2 studies of plozasiran. Plozasiran is a first-in-class RNA interference therapeutic designed to decrease the production of apolipoprotein C-1III, a component of triglycerides-rich lipoproteins and a key regulator of triglyceride metabolism. In the Phase III PALISADE trial in patients with and without a genetic confirmation of familial chylomicronemia syndrome (FCS), the drug demonstrated deep and sustained decreases in triglycerides and effects on a broader spectrum of lipoproteins associated with atherosclerotic cardiovascular disease. In the OLE Phase II MUIR and SHASTA-2 trials, extended treatment with 25 mg of plozasiran in patients with moderate to severely elevated triglycerides showed mean reductions through 15 months follow-up in the OLE in triglycerides of up to -73% in patients originally from the MUIR study and -86% in patients originally from the SHASTA-2 study.
The company also reported that based on the results from the Phase III PALISADE study, it had submitted a New Drug Application (NDA) to the U.S. FDA for the treatment of familial chylomicronemia syndrome (FCS), a severe and rare genetic disease that currently has no treatments. It plans to submit marketing applications to other regulators next year. FCS leads to extremely high triglyceride levels.