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Ionis' Tryngolza Cuts Triglyceride Levels in Late-Stage Study
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Ionis Pharmaceuticals (IONS - Free Report) recently announced positive top-line results from the phase III ESSENCE study evaluating its wholly owned drug Tryngolza in people with moderate hypertriglyceridemia who have or are at risk for atherosclerotic cardiovascular disease (ASCVD).
The study met its primary endpoint, with Tryngolza-treated participants showing a statistically significant, placebo-adjusted reduction in triglyceride (TG) levels. Specifically, treatment with the drug led to TG reductions of 61% and 58% after six months with 80 mg and 50 mg monthly doses, respectively.
Ionis noted that the “vast majority” of participants in the ESSENCE study saw their TG levels fall within the normal range (<150 mg/dL) following treatment with Tryngolza. The drugalso met all key secondary endpoints in the study.
TG is a type of fat in the blood that, at elevated levels, increases the risk of heart disease and pancreatitis. Hypertriglyceridemia, marked by elevated TG levels (≥150 mg/dL), is a known risk factor for cardiovascular disease.
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 by the EMA, with a final decision expected later this year.
Ionis is also evaluating the drug as a potential treatment for severe hypertriglyceridemia (sHTG) across two late-stage studies — CORE and CORE2. Data from these studies is expected in third-quarter 2025. People with sHTG, marked by TG levels higher than 500 mg/dL, are at higher risk of potentially life-threatening acute pancreatitis (AP) and ASCVD.
Like FCS, Ionis also has a first-mover advantage in sHTG indication. If data from the CORE and CORE-2 studies are positive, Ionis plans to submit an FDA filing for Tryngolza’s label expansion before the end of 2025.
IONS Stock Performance
As seen in the chart below, shares of Ionis have been in line with the industry year to date.
Image Source: Zacks Investment Research
Ionis Boasts a Diverse Revenue Stream
Ionis has collaborations with leading drugmakers/biotech companies, such as AstraZeneca (AZN - Free Report) , Biogen (BIIB - Free Report) , GSKplc (GSK - Free Report) 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 worldwide. The company licensed this drug to Biogen, which is responsible for commercializing it. 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 being developed 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.
Some of its other wholly owned candidates include donidalorsen (for hereditary angioedema), zilganersen (for Alexander’s disease) and ulefnersen (for ALS), 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.
Image: Bigstock
Ionis' Tryngolza Cuts Triglyceride Levels in Late-Stage Study
Ionis Pharmaceuticals (IONS - Free Report) recently announced positive top-line results from the phase III ESSENCE study evaluating its wholly owned drug Tryngolza in people with moderate hypertriglyceridemia who have or are at risk for atherosclerotic cardiovascular disease (ASCVD).
The study met its primary endpoint, with Tryngolza-treated participants showing a statistically significant, placebo-adjusted reduction in triglyceride (TG) levels. Specifically, treatment with the drug led to TG reductions of 61% and 58% after six months with 80 mg and 50 mg monthly doses, respectively.
Ionis noted that the “vast majority” of participants in the ESSENCE study saw their TG levels fall within the normal range (<150 mg/dL) following treatment with Tryngolza. The drugalso met all key secondary endpoints in the study.
TG is a type of fat in the blood that, at elevated levels, increases the risk of heart disease and pancreatitis. Hypertriglyceridemia, marked by elevated TG levels (≥150 mg/dL), is a known risk factor for cardiovascular disease.
Tryngolza was recently approved by the FDA 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.
Recent Developments With Ionis’ Tryngolza
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 by the EMA, with a final decision expected later this year.
Ionis is also evaluating the drug as a potential treatment for severe hypertriglyceridemia (sHTG) across two late-stage studies — CORE and CORE2. Data from these studies is expected in third-quarter 2025. People with sHTG, marked by TG levels higher than 500 mg/dL, are at higher risk of potentially life-threatening acute pancreatitis (AP) and ASCVD.
Like FCS, Ionis also has a first-mover advantage in sHTG indication. If data from the CORE and CORE-2 studies are positive, Ionis plans to submit an FDA filing for Tryngolza’s label expansion before the end of 2025.
IONS Stock Performance
As seen in the chart below, shares of Ionis have been in line with the industry year to date.
Image Source: Zacks Investment Research
Ionis Boasts a Diverse Revenue Stream
Ionis has collaborations with leading drugmakers/biotech companies, such as AstraZeneca (AZN - Free Report) , Biogen (BIIB - Free Report) , GSK plc (GSK - Free Report) 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 worldwide. The company licensed this drug to Biogen, which is responsible for commercializing it. 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 being developed 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.
Some of its other wholly owned candidates include donidalorsen (for hereditary angioedema), zilganersen (for Alexander’s disease) and ulefnersen (for ALS), 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.