IONS Hits 52-Week High on Tryngolza's Success in Lowering Triglyceride

Shares of Ionis Pharmaceuticals IONS surged 35% on Tuesday after it announced positive top-line results from two late-stage studies — CORE and CORE2 — evaluating its wholly owned drug Tryngolza (olezarsen) in people with severe hypertriglyceridemia (sHTG).

Both studies met their primary endpoint, with Tryngolza-treated participants showing a statistically significant, placebo-adjusted reduction in triglyceride (TG) levels across both 80 mg and 50 mg monthly doses. TG is a type of fat in the blood that, at elevated levels, increases the risk of heart disease and pancreatitis.

At the 80 mg dose, treatment with the drug led to TG reductions of 72% and 55% after six months in the CORE and CORE2 studies, respectively. At the 50 mg dose, Tryngolza-treated patients in the CORE study achieved TG reductions of 63%, while those in the CORE2 study achieved reductions of 49%.

People with sHTG, marked by TG levels higher than 500 mg/dL, are at higher risk of potentially life-threatening acute pancreatitis (AP) and atherosclerotic cardiovascular disease (ASCVD). Both studies enrolled nearly 1,100 patients, all of whom remained on standard-of-care lipid-lowering therapy throughout the treatment period.

Both studies also showed a significant reduction in AP events — a key secondary endpoint. Pooled data from the studies showed that treatment with Tryngolza for 12 months reduced the risk of AP events by 85% compared with placebo.

Tryngolza was approved by the FDA last year for treating familial chylomicronemia syndrome (FCS), a rare genetic disease marked by extremely elevated TG levels (>880 mg/dL). This nod makes the drug the first approved treatment for FCS in the country. It also marks Ionis’ first independent commercial launch.

Ionis to Seek Label Expansion for Tryngolza Later This Year

Earlier in May, Ionis reported positive top-line results from the phase III ESSENCE study evaluating Tryngolza in people with moderate hypertriglyceridemia (marked by TG levels ≥150 mg/dL) who have or are at risk for ASCVD. The study met its primary endpoint of a statistically significant, placebo-adjusted reduction in TG levels following treatment with the drug.

Based on data from the ESSENCE, CORE and CORE2 studies, Ionis plans to submit an FDA filing for Tryngolza’s label expansion before the end of 2025. Like FCS, Ionis also has a first-mover advantage in the sHTG indication.

In March, Ionis out-licensed ex-U.S. rights for Tryngolza to Sweden-based Sobi. Per the deal terms, Sobi will be responsible for all future regulatory filings and commercialization activities for the drug outside the United States (except Canada and China). A regulatory filing for Tryngolza in the FCS indication is currently under review in the EU, with a final decision expected later this year.

IONS Stock Performance

Following the news, shares of Ionis hit a 52-week high. Several Wall Street analysts praised the results, with some describing the outcome as a “home run” scenario. While Tryngolza is already approved for FCS, the market for sHTG is significantly larger. Per Ionis, nearly 3,000 people in the United States have FCS, compared with around 3 million affected by sHTG — presenting access to a substantial, untapped market with blockbuster potential.

Year to date, shares of Ionis have soared over 64% compared with the industry’s 8% growth.

Ionis Boasts a Diverse Revenue Stream

Ionis has collaborations with leading drugmakers/biotech companies, such as AstraZeneca AZN, Biogen BIIB, GSK plc GSK and Novartis. These deals provide the company with funds in the form of license fees, and upfront and milestone payments to invest in the development of its wholly owned pipeline.

Ionis earns commercial revenues in the form of royalty payments on net sales of Spinraza, approved in the United States to treat spinal muscular atrophy (SMA) worldwide. The company licensed this drug to Biogen, which is responsible for its commercialization. Ionis and Biogen also market Qalsody, which was approved by the FDA in April 2023 for amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 mutations.

AstraZeneca, Novartis and GSK are its partners for Wainua, pelacarsen and bepirovirsen, respectively. The FDA approved Wainua in December 2023 to treat adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (ATTRv-PN) in the United States. The drug recently obtained approval in the EU for a similar indication and will be marketed under the name Wainzua.

While the GSK-partnered drug is being developed in two late-stage studies for chronic hepatitis B, the Novartis-partnered drug is in a late-stage study for patients with cardiovascular disease due to elevated Lp(a) levels. Data from all these studies are expected in 2026.

Based on funds received from the above partners, Ionis is expanding the portfolio of its wholly-owned medications. The company recently secured FDA approval for its second wholly-owned drug, Dawnzera, to prevent hereditary angioedema (HAE) attacks in individuals aged 12 years and older.

Some of its wholly owned candidates include zilganersen (for Alexander’s disease), ulefnersen (for ALS) and ION582 (for Angelman syndrome), which are being evaluated in late-stage studies. Ionis expects commercial launches for these drugs over the next three years, which could further reduce its reliance on collaboration partners.

Ionis Pharmaceuticals, Inc. Price

 

Ionis Pharmaceuticals, Inc. price | Ionis Pharmaceuticals, Inc. Quote

IONS’ Zacks Rank

Ionis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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