Sometimes, even clinical trials that have reached their endpoints may not give the confidence to move forward.
That’s what Pfizer found with its drug vupanorsen, an antisense therapy being tested for potential indications for reducing cardiovascular (CV) risk and severe hypertriglyceridemia. After reviewing Phase 2B clinical data, the New York drugmaker and its partner Ionis Pharmaceuticals announced on monday It is abandoning development of the drug, returning the rights to Ionis.
As early as November, Pfizer had Announce The drug achieved a “statistically significant reduction in non-high-density lipoprotein cholesterol (non-HDL-C) and a statistically significant secondary endpoint of triglycerides (TG) at all dose levels compared to placebo. reduce”. According to the press release, the study enrolled 286 participants aged 40 and older with dyslipidemia — those with non-HDL-C and TG elevations who were receiving stable doses of statins or not taking ezetimibe cloth.
However, a careful reading of press releases citing positive clinical trial results seems to suggest doubts even then. James RusnackPfizer’s senior vice president and chief development officer for internal medicine and hospitals said Pfizer will “carefully consider these findings to determine appropriate next steps for future development.”
Just two months later, Pfizer made the decision that, while “statistically significant,” the magnitude of the reduction was immaterial. Vupanorsen was also found to cause a dose-dependent increase in liver fat. Those who received higher doses also showed elevations in the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST), indicating liver damage.
So the logical next step in development is basically a complete stop.
“Pfizer remains committed to research and development in the cardiovascular field and to help address the unmet medical needs of patients with cardiovascular disease,” said James RusnackPfizer’s senior vice president and chief development officer, internal medicine and hospitals, at a news conference on Monday. “We are grateful to the patients, researchers and support staff who participated in this important research program.”
Pfizer has paid $250 million upfront to acquire rights to develop the drug from Carlsbad, Calif.-based Ionis and its affiliates. Shares of Ionis fell briefly on Monday, closing at $31.80 on Monday, up from $31.68 on Friday.
With the exit of the New York company, Regeneron, which won FDA approval for Evkeeza nearly a year ago, will now no longer have a big competitor. The drug is an antibody designed to bind to and block angiopoietin-like 3 – ANGPTL3 – a key protein in lipid metabolism. The approval marks the first FDA-approved therapy that works in this way.
ANGPTL3 has emerged as an emerging therapeutic target for understanding cardiovascular risk.
Image: Ekkaluck, Getty Images
