The approval of Ionis Pharmaceuticals’ Tryngolza for severe hypertriglyceridemia could spur “substantial growth” for the product, according to William Blair.
The FDA has greenlit the use of Ionis Pharmaceuticals’ antisense oligonucleotide Tryngolza to help control blood lipid levels and pancreatitis risk in patients with severe hypertriglyceridemia. The biotech expects to make the product available for the indication in July.
For Ionis, the approval, announced Wednesday, marks a “major market expansion for [a] wholly owned commercial opportunity,” analysts at William Blair told investors in a note the same day. Severe hypertriglyceridemia (sHTG) “should provide substantial growth” to Tryngolza, the firm added, noting that the drug will be “a likely meaningful contributor to [Ionis’] cash flow breakeven guidance in 2028.”
The biotech set that goal for itself during the 44th J.P. Morgan Healthcare Conference in January, with CEO Brett Monia saying at the time that the company is “well-positioned to deliver accelerating revenue growth.”
Tryngolza is an RNA-targeting medicine that targets the serum apolipoprotein C-III, in turn promoting the clearance of triglycerides and LDL cholesterol from the bloodstream. The product was first approved in 2024 for familial chylomicronemia syndrome, a rare and genetic disease that raises the risk of acute pancreatitis and causes severe hypertriglyceridemia.
Tryngolza has been off to a roaring start. The product made around $102 million last year, “a sign of the company’s impressive salesforce in cardiometabolic disease,” William Blair wrote in a Jan. 12 note. The firm forecasted at the time that Tryngolza sales could soar to $2 billion in 2034.
Thursday’s approval made William Blair analysts more bullish about the product’s market potential, prompting an upward adjustment of peak sales forecast to $3 billion.
Data from the Phase 3 CORE and CORE 2 studies showed that Tryngolza could suppress fasting triglyceride concentrations by up to 72% at six months versus placebo. The antisense agent achieved this benefit rapidly and consistently, sustaining those reductions at 12 months, according to Ionis.
Importantly, Tryngolza also lowered acute pancreatitis events by up to 91%, making it the only sHTG therapy that has been shown to reduce the risk of this complication, according to the biotech. William Blair pointed to this acute pancreatitis benefit as a driver of the drug’s future growth.
As for safety, Tryngolza’s updated label references potential “increases in liver enzyme and hepatic fat,” and encourages prescribers to consider liver enzyme testing before initiating treatment or upping dosage. “We doubt this hinders uptake,” Leerink Partners wrote on Thursday, citing findings from a survey it conducted among healthcare providers. The firm nevertheless expects doctors to start patients on the lower 50-mg dose before uptitrating as needed.
Tryngolza currently comes in 50-mg and 80-mg autoinjectors, designed for monthly self-administration.
