Ionis Pharmaceuticals Inc. (NASDAQ: IONS) has announced that Pfizer Inc. (NYSE: PFE) has given an update on the Phase 2b vupanorsen study. Vupanorsen is an experimental antisense treatment under development for severe hypertriglyceridemia and cardiovascular risk reduction indications.
The study’s primary goal of no-HDL cholesterol reduction achieved
The primary goal of the dose-ranging trial in participants with increased non-HDL-C and triglycerides (TG) was reached, with a statistically significant non-HDL-C reduction at all doses evaluated after 24 weeks, relative to placebo. Furthermore, when compared to placebo, patients treated with vupanorsen experienced statistically significant TG and ANGPTL3 reduction at all dosage levels after 24 weeks.
Ionis’ global cardiovascular development VP and cardiovascular franchise head Sotitios “Sam” Tsimikas, said, “We were pleased to see statistically significant reductions in the primary endpoint, non-HDL-cholesterol, and in the secondary endpoint of triglycerides at all doses tested. The topline results of the Phase 2b study also showed that vupanorsen dose-dependently lowered its target, angiopoietin-like 3. Pfizer is continuing to review the findings to determine the next steps regarding future development. We look forward to the full data set being presented at a medical meeting next year.”
Study showed no adverse events with no Hy’s Law cases reported
Injection site responses were the most common adverse effects, occurring most frequently in the high vupanorsen dose group. Increases in liver enzymes, aspartate aminotransferase (AST), and alanine aminotransferase (ALT) were the most common laboratory abnormalities, and they were mainly detected at the higher doses. In vupanorsen-treated patients, there were no Hy’s Law cases and no significant changes in bilirubin levels.
However, when compared to placebo, certain vupanorsen doses were related to increases in a hepatic fat fraction from baseline, as measured by magnetic resonance imaging (MRI) proton density fat fraction after24 weeks. There were no confirmed platelet count abnormalities or estimated glomerular filtration rate decline in any of the subjects. Equally, there were no major side effects associated with the treatment. Interestingly serious adverse events (SAEs) were equally common in both the study and placebo groups.