Ionis Pharmaceuticals’ launch of Tryngolza (olezarsen) is off to a flying start as a treatment for familial chylomicronemia syndrome (FCS). With the California drugmaker preparing to roll Tryngolza out in a new indication, it has amped up its peak sales projection for the RNA-targeted therapy based on positive feedback.
Favoring nice round numbers, Ionis has increased its guidance for peak sales of Tryngolza as a treatment for severe hypertriglyceridemia (sHTG) from $1 billion-plus to $2 billion-plus.
The company revealed the adjustment in a press release Monday, and CEO Brett Monia, Ph.D. explained why Tuesday morning at the J.P. Morgan Healthcare Conference.
The updated guidance comes from research in the healthcare professional community, which indicates a significant increase in projected sales volumes compared to the company’s previous expectation, Monia said.
“The enthusiasm for the reduction in acute pancreatitis (AP) was so overwhelming,” Monia said.
Much of the optimism in the indication comes from results of two phase 3 trials that were so convincing analysts from Citi characterized them as a “home run.”
The trials achieved their primary endpoints, showing placebo-adjusted reductions of 55% and 72% in patients' triglyceride levels after six months of treatment. Pooled data from the trials also showed as a secondary prespecified endpoint that Tryngolza reduced the risk of AP events by 85% after patients were on Tryngolza for a year.
Ionis submitted its application for Tryngolza in sHTG last month, requesting that the FDA grant a priority review. If that is accepted, it would set up Ionis for a potential launch in the indication in July of this year. The company has already hired a field force of approximately 200 to handle launch of Tryngolza in sHTG.
Ionis' confidence is bolstered by the market performance of Tryngolza in FCS. The therapy launched a year ago and generated sales of $105 million in 2025, according to a preliminary tally from the drugmaker.
“It’s been overwhelmingly positive,” Monia said. “We’re hearing so many great stories about patients living with this devastating disease, their ability to live daily lives without body pain, without going to the hospital for recurrent acute pancreatitis events.”
When Tryngolza was approved in December 2024, Monia called it a “pivotal moment” for Ionis as it evolved from an R&D organization to a commercial company. Since then, the company has pulled off successful but vastly different launches of two products, including Dawnzera, the first RNA-targeted therapy for the rare disease hereditary angioedema.
“We started [in] FCS where we were the first [therapy] to the market and patient identification was so critical,” Monia said. “Our second launch, with Dawnzera, we came into a market where there are preexisting treatments and that’s a switch market. That’s a different strategy.”
With sHTG, Ionis faces a new challenge, as the indication includes millions of potential patients.
“We’re building off the success of FCS and the sentiment by the endocrinologists, cardiologists, and pancreatologists, that have experienced Tryngolza in FCS, that it is resonating incredibly well for the sHGT opportunity,” Monia said. “Physicians are asking, ‘When can I put my patients that have sHGT onto olezarsen?’ They, of course, have to wait until we get the indication.”