Arrowhead Pharmaceuticals has launched legal action against Ionis Pharmaceuticals, aiming to neutralize Ionis' claim that Arrowhead's potential rival to Tryngolza breaches a patent.
Arrowhead claims that Ionis’ patent is “invalid and not infringed by Arrowhead’s planned commercialization of Arrowhead's investigational rare disease drug plozasiran,” according to a Sept. 10 complaint filed in the U.S. District Court for the District of Delaware.
Plozasiran is an RNAi therapeutic designed to reduce production of apolipoprotein C-III (APOC3) in order to lower triglycerides and restore lipid levels. The candidate holds first-in-class potential and is currently under FDA review as a treatment for a rare genetic disorder called familial chylomicronemia syndrome (FCS), with a decision expected on or before Nov. 18.
The asset, previously dubbed ARO-APOC3, has secured breakthrough therapy, orphan drug and fast track tags from the FDA.
If Arrowhead’s candidate gains the FDA green light, it will face Ionis’ Tryngolza (olezarsen), which secured approval for FCS in December 2024. The antisense oligonucleotide was the first treatment to gain FDA clearance in the rare metabolic condition and is the first drug Ionis has commercialized on its own.
Like plozasiran, Tryngolza is also made to lower the body's production of APOC-3.
According to Arrowhead’s lawsuit, Ionis has claimed that its patent No. 9,593,333—titled “Modulation of apolipoprotein C-III (APOC3) expression in lipoprotein lipase deficient populations”—would be infringed if plozasiran were to be marketed.
“Arrowhead has multiple issued U.S. patents that cover plozasiran for the treatment of patients with FCS based entirely on work developed internally at Arrowhead, which Ionis was not involved with and provided no contribution to whatsoever,” Arrowhead President and CEO Christopher Anzalone, Ph.D., said in a Sept. 11 release.
Arrowhead’s newly filed lawsuit is seeking a declaratory judgment to establish the patent as invalid when related to the manufacturing or sale of plozasiran.
“It is unfortunate and troubling that Ionis Pharmaceuticals is attempting to take action that clearly puts their corporate goals ahead of the needs of patients with familial chylomicronemia, a severe and rare disease characterized by extremely high triglyceride levels which can lead to acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis and cognitive issues,” Anzalone said in the release.
“Arrowhead will not tolerate efforts by Ionis to limit the availability of a potentially important new medicine to members of the FCS community,” the CEO added.
A spokesperson for Ionis told Fierce Biotech that the company is “deeply committed to the FCS community, and we welcome new advances that can help raise awareness of FCS and improve the lives of people who are in need.”
“Importantly, we are not seeking to block the launch of this new therapy to patients,” the spokesperson added. “We are confident in our IP around FCS and will vigorously defend our patent position.”