Ionis Pharmaceuticals Touts $3B TRYNGOLZA Opportunity, Pipeline Readouts at Conference

Key Points

• Ionis Pharmaceuticals said TRYNGOLZA could become a more than $3 billion U.S. product in severe hypertriglyceridemia, citing strong phase 3 efficacy, no expected monitoring burden, and a planned June 30 FDA decision date. The company is targeting high-risk patients first and has set a $40,000 wholesale acquisition cost, which it says supports a higher peak-sales outlook.
• Ionis also reported solid momentum for DAWNZERA in hereditary angioedema and outlined upcoming pipeline catalysts across several programs, including WAINUA in ATTR cardiomyopathy, APOC3 dosing expansion, pelacarsen, Tau, and Angelman syndrome data. The company emphasized that multiple readouts over the next year could broaden its commercial and clinical growth story.
• Neurocrine Biosciences highlighted strong commercial performance from INGREZZA and CRENESSITY, while discussing the strategic addition of VYKAT XR through its Soleno deal. The company also pointed to a deep pipeline in psychiatry and obesity, with key data expected from osavampator, dereclidine and 2118 over the next few years.

Executives at a healthcare investor event outlined commercial launch plans, pipeline readouts and market-development priorities across several biotechnology franchises, with discussion spanning Ionis Pharmaceuticals NASDAQ: IONS products including TRYNGOLZA, DAWNZERA and WAINUA, as well as Neurocrine Biosciences’ commercial and pipeline strategy.

Ionis Highlights TRYNGOLZA Opportunity in Severe Hypertriglyceridemia

Todd Tushla, vice president of investor relations, said Ionis is preparing for a potential June 30 FDA action date for olezarsen in severe hypertriglyceridemia, or sHTG. The therapy, marketed as TRYNGOLZA in familial chylomicronemia syndrome, has already launched in that ultra-orphan indication.

Tushla said the FCS launch has been “very, very strong,” citing demand from physicians and patients, triglyceride reductions and prevention of acute pancreatitis as factors supporting adoption. He said the company expects those early physician relationships to help with the broader sHTG opportunity because many of the same specialists treat both groups of patients.

According to Tushla, phase 3 data in sHTG showed an 85% reduction in acute pancreatitis and triglyceride reductions exceeding 70% on top of standard of care. He described the indication as a “multi-billion dollar product opportunity” and said Ionis has hired and trained its field team ahead of a potential launch.

Tushla said the company plans to focus initially on high-risk patients, including those with a prior acute pancreatitis event and patients with triglycerides of 880 milligrams per deciliter or higher. He said patients who have experienced acute pancreatitis are highly motivated to avoid another event, while physicians are also motivated to prevent first-time attacks in high-risk patients.

On competition, Tushla said there are more than 3 million people in the U.S. with severe hypertriglyceridemia, including about 400,000 currently treated “ineffectively” with generic fibrates and fish oils. He said the market has room for multiple players but added that being first to market would help Ionis identify high-volume prescribers, establish payer relationships and set pricing.

Pricing, Access and Safety Discussed for sHTG Launch

Tushla said Ionis set a $40,000 wholesale acquisition cost for TRYNGOLZA in sHTG, effective April 1, after extensive research with payers and physicians. He said the expected label is for patients with sHTG, defined as triglycerides of 500 and above, as an adjunct to diet. He also said the company expects acute pancreatitis data to appear in the clinical trial section of the label.

He said the $40,000 WAC is expected to produce a net price above the company’s earlier assumed $10,000 to $20,000 range. Tushla said that was the main driver behind Ionis raising its U.S. peak sales expectation for TRYNGOLZA in sHTG to more than $3 billion from more than $2 billion.

On safety, Tushla said the company does not expect monitoring requirements in sHTG, similar to the FCS label. He addressed liver fat observations from the phase 3 study, saying the increase was small, dose-dependent and not associated with clinical consequences. He said Ionis continues to follow patients in an open-label extension and expects to present additional liver fat data at a medical congress later this year.

DAWNZERA, WAINUA and Ionis Pipeline Updates

Tushla also discussed DAWNZERA, a prophylactic treatment for hereditary angioedema, or HAE. He said demand from patients and healthcare providers has been high and that the launch has gone “exceptionally well.” He described HAE as a switch market, with patients moving from other prophylactic treatments, on-demand treatments and newly diagnosed patients starting therapy.

For WAINUA in ATTR cardiomyopathy, Tushla said the company’s study is the largest conducted in the indication and is well-powered for its primary endpoint. He said baseline demographics showed similarities to the HELIOS-B study, with about 56% of patients on tafamidis at baseline. Tushla said combination-use data could be a key differentiator if the study supports benefit from adding a silencer to tafamidis.

Tushla said Ionis is also advancing a less-frequent dosing APOC3 program, with phase 2 work in FCS expected to start soon. He also highlighted anticipated data for pelacarsen in the second half of the year, Tau data around midyear and Angelman syndrome phase 3 data expected next year after enrollment completion in the second half of this year.

Neurocrine Reviews Commercial Base and Soleno Deal

In a separate discussion, Matt Abernethy, chief financial officer of Neurocrine Biosciences, said the company is in a stronger position than it was when INGREZZA was first approaching launch. He cited three commercial products: INGREZZA, CRENESSITY and VYKAT XR, the latter through Neurocrine’s announced acquisition of Soleno Therapeutics.

Abernethy said INGREZZA, approved in 2017 for tardive dyskinesia, has 2025 guidance of $2.7 billion to $2.8 billion and loss of exclusivity in 2038. He said the company continued to add record numbers of new patients in the first quarter despite typical seasonality, adding that about half of the tardive dyskinesia market has been diagnosed and only one in 10 patients is currently on a VMAT2 inhibitor.

Abernethy said CRENESSITY, approved in December 2024 for classic congenital adrenal hyperplasia, has had a strong launch, with more than 10% of patients on therapy within the first five quarters and more than $150 million in first-quarter sales. He said reimbursement has been a positive factor, with more than 80% of prescription requests reimbursed.

On Soleno, Abernethy said VYKAT XR is a rare endocrine product that fits strategically with CRENESSITY. He said he could not discuss the acquisition in detail during the offer period, but described VYKAT XR as a durable asset with intellectual property extending into the 2040s.

Neurocrine Pipeline Focuses on Psychiatry and Obesity

Abernethy said Neurocrine has close to 20 clinical-stage pipeline programs, including osavampator in major depressive disorder and dereclidine in schizophrenia, both expected to produce data in 2027 or 2028. He described osavampator, an AMPA potentiator, as one of his preferred programs because phase 2 efficacy was significant and the market opportunity is large.

He also discussed dereclidine, a selective M4 agonist in phase 3 schizophrenia studies, with the first trial expected to read out in 2027 and a second in 2028. Abernethy said the company is also studying the drug in bipolar mania.

Neurocrine is also entering obesity research with 2118, a CRF2 agonist. Abernethy said the company expects data in healthy obese people in late 2027 and sees the phase 1 program as a relatively modest investment that can inform later development strategy.

On business development, Abernethy said Neurocrine’s priority after the Soleno deal will be integration and rebuilding its cash position, though the company would still evaluate opportunities if the right acquisition emerged.

About Ionis Pharmaceuticals NASDAQ: IONS

Ionis Pharmaceuticals, Inc is a biotechnology company focused on the discovery and development of RNA-targeted therapies designed to modulate gene expression. The company's proprietary antisense oligonucleotide (ASO) technology enables the selective binding of short synthetic strands of nucleic acids to messenger RNA (mRNA), thereby inhibiting or altering the production of disease-causing proteins. Ionis' pipeline spans a range of therapeutic areas, including neurological disorders, cardiovascular conditions, metabolic diseases and rare genetic disorders.

Since its founding in 1989 by Dr.

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