Positive olezarsen Phase 3 data in familial chylomicronemia syndrome to be presented at 2024 American College of Cardiology (ACC) annual meeting

Rhea-AI Summary

Ionis Pharmaceuticals, Inc. presents positive Phase 3 Balance results for olezarsen at ACC Annual Meeting, showcasing significant reductions in triglycerides and acute pancreatitis events in patients with FCS, a rare disease with no approved treatments in the U.S.

Positive

• Ionis Pharmaceuticals, Inc. presents positive Phase 3 Balance results for olezarsen at ACC Annual Meeting
• Significant reduction in triglyceride levels and acute pancreatitis events in patients with familial chylomicronemia syndrome (FCS)
• Olezarsen shows robust reductions in apolipoprotein C-III and favorable safety profile
• Late-breaking abstract on efficacy and safety of olezarsen in patients with hypertriglyceridemia and high cardiovascular risk to be presented at ACC Annual Meeting

Negative

• None.

Medical Research Analyst

The recent announcement by Ionis Pharmaceuticals regarding the positive Phase 3 Balance results for olezarsen is a significant development within the biopharmaceutical sector. Familial chylomicronemia syndrome (FCS) is a rare and life-threatening disease characterized by extremely high triglyceride levels, leading to severe pancreatitis. The absence of approved treatments in the U.S. underscores the unmet medical need and potential market opportunity for Ionis.

Olezarsen's efficacy in reducing triglyceride levels and acute pancreatitis events could translate into a substantial clinical benefit for patients. The reduction in apolipoprotein C-III (apoC-III) is particularly noteworthy, as it is a key regulator of triglyceride metabolism. A favorable safety profile enhances the drug's commercial viability, potentially leading to a smoother regulatory path and swift adoption upon approval.

For stakeholders, the short-term focus will be on the FDA's response, which could lead to significant stock volatility. In the long-term, the success of olezarsen could bolster Ionis's revenue streams and solidify its position in the rare disease market.

Healthcare Industry Analyst

Ionis Pharmaceuticals' positive trial results are poised to have a ripple effect on the healthcare industry. The rarity of FCS means that olezarsen would likely qualify for orphan drug designation, offering benefits like tax credits and market exclusivity upon approval. This exclusivity could protect Ionis's market share and pricing power, contributing to a potentially lucrative niche market.

From an industry perspective, Ionis's progress with olezarsen may encourage further investment and research into treatments for rare diseases, a sector that has been gaining traction due to the high impact on patient populations and favorable regulatory incentives. The presentation at the ACC meeting also serves as a strategic platform for Ionis to engage with cardiologists and key opinion leaders, which is important for building advocacy and awareness ahead of a potential launch.

Analyzing the broader market implications, competitors in the hypertriglyceridemia space might experience pressure, especially if olezarsen's safety and efficacy data surpass existing therapies. This could lead to market share shifts and influence future R&D directions.

Financial Analyst

The financial implications of the positive Phase 3 results for olezarsen cannot be overstated. Ionis Pharmaceuticals has the potential to tap into a market void with this drug. Given the rarity of FCS and the lack of approved treatments, pricing strategies will likely aim high, supported by the drug's orphan status. The cost-effectiveness of reducing acute pancreatitis events, a significant driver of healthcare expenses in FCS patients, will further justify the drug's value proposition to payers.

Investors will be closely monitoring the upcoming presentations at the ACC Annual Meeting. The detailed data could sway investor sentiment and influence Ionis's market capitalization. It's essential to consider the company's pipeline robustness and the scalability of its platform technology when evaluating long-term prospects. Should olezarsen receive FDA approval, it may serve as a proof of concept for Ionis's antisense technology, potentially leading to premium valuations.

However, investors should also be aware of the risks associated with drug development, such as regulatory hurdles and potential commercialization challenges. The market's reaction to the upcoming presentations will provide insight into investor confidence in Ionis's ability to navigate these challenges.

–  First presentation of pivotal results showing significant reduction in triglycerides and substantial reduction in acute pancreatitis events in patients with rare, life-threatening disease, for which there are no approved treatment options in U.S.  –

CARLSBAD, Calif., March 25, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will present positive Phase 3 Balance results in patients with familial chylomicronemia syndrome (FCS) for the company's lead independent, investigational medicine, olezarsen, at the 2024 American College of Cardiology (ACC) Annual Meeting in Atlanta, Georgia.

The oral presentation will take place on April 7, 2024 at 10:08am ET during the session on Prevention and Health Promotion and the Year in Review. Ionis previously announced positive topline results. As previously shared, monthly dosing of olezarsen 80 mg met the primary endpoint with a statistically significant reduction in triglyceride levels at six months, demonstrated robust reductions in apolipoprotein C-III (apoC-III) and marked reductions in acute pancreatitis (AP) events versus placebo. Olezarsen also demonstrated a favorable safety and tolerability profile.

In addition, a late-breaking abstract entitled "Efficacy and Safety of Olezarsen in Patients with Hypertriglyceridemia and High Cardiovascular Risk: Primary Results of the Bridge-TIMI 73a Trial" will be presented on April 7, 2024 at 8:30am ET during the session on Late-Breaking Clinical Trials II.

About the Balance Study

The global, multicenter, randomized, double-blind, placebo-controlled Phase 3 Balance study (NCT04568434) enrolled 66 patients aged 18 and older with confirmed FCS. Patients in the study received background therapies including statins, fibrates and omega-3 fatty acids. Patients were randomized in a 1:1:1 ratio to receive olezarsen 80 mg or 50 mg or placebo via subcutaneous injection once every four weeks for 53 weeks. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months compared to placebo. Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters, and adjudicated acute pancreatitis event rates over the treatment period.

About Olezarsen

Olezarsen is an RNA-targeted investigational LIgand Conjugated Antisense (LICA) medicine being evaluated for people at risk of disease due to elevated triglyceride levels, including those with familial chylomicronemia syndrome (FCS). Olezarsen is designed to inhibit the body's production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood.^1,2 The U.S. FDA granted olezarsen Fast Track designation for the treatment of FCS in January 2023, as well as Orphan Drug designation and Breakthrough Therapy designation in February 2024. In addition to FCS, Ionis is evaluating olezarsen for the treatment of severe hypertriglyceridemia (sHTG) in Phase 3 clinical trials.

About FCS

FCS is a rare, genetic disease characterized by extremely elevated triglyceride levels. It is caused by impaired function of the enzyme lipoprotein lipase (LPL).³ Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are 90% triglycerides.^3,4 FCS is estimated to impact one to 13 people per million in the U.S.^5,6,7 People living with FCS are at high risk of acute pancreatitis (AP) in addition to other chronic health issues such as fatigue and severe, recurrent abdominal pain.^3,8,9 People living with FCS are sometimes unable to work, adding to the burden of disease.⁹

Currently, there are no U.S. FDA-approved therapies for the treatment of FCS and standard triglyceride lowering therapies are generally ineffective in patients with FCS.^10,11 People living with this condition currently rely solely on nutrition management through extremely restrictive and difficult to manage diets to navigate the health risks associated with FCS.^11,12

About Ionis Pharmaceuticals, Inc.

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionispharma.com and follow us on X (Twitter) and LinkedIn.

Forward-looking Statements

This press release includes forward-looking statements regarding olezarsen, Ionis' business, and the therapeutic and commercial potential of Ionis' commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.ionispharma.com.

Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc.

¹Alexander VJ, et al. Eur Heart J 2019;40(33):2785-2796.
²Tardif JC, et al. Eur Heart J 2022;43(14):1401-1412.
³Gaudet D, et al. N Engl J Med. 2014;371:2200-2206.
⁴Ginsberg HN, et al. Eur Heart J. 2021;42:4791-4806.
⁵Pallazola VA, et al. Eur J Prev Cardiol 2020;27(19):2276-8.
⁶Warden BA, et al. J Clin Lipidol 2020;14(2):201-6.
⁷Tripathi M, et al. Endocr Pract 2021;27(1):71-6.
⁸Bashir B, et al. Metabolites. 2023;(5):621.
⁹Davidson M, et al. J Clin Lipidol. 2018;12(4):898-907.e2.
¹⁰Gouni-Berthold I. J Endocr Soc. 2019;4(2):bvz035.
¹¹Paquette M, et al. Atherosclerosis. 2019;283:137-142.
¹²Williams L, et al. J Clin Lipidol. 2018;4:908-919.

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SOURCE Ionis Pharmaceuticals, Inc.

What are the key findings of Ionis Pharmaceuticals, Inc.'s presentation at the ACC Annual Meeting?

Ionis Pharmaceuticals, Inc. presented positive Phase 3 Balance results for olezarsen, showing significant reductions in triglyceride levels and acute pancreatitis events in patients with FCS.

When will the oral presentation of olezarsen take place at the ACC Annual Meeting?

The oral presentation of olezarsen will take place on April 7, 2024, at 10:08am ET during the session on Prevention and Health Promotion and the Year in Review.

What was the primary endpoint met by monthly dosing of olezarsen 80 mg?

The primary endpoint met by monthly dosing of olezarsen 80 mg was a statistically significant reduction in triglyceride levels at six months.

What additional abstract will be presented at the ACC Annual Meeting regarding olezarsen?

A late-breaking abstract on the efficacy and safety of olezarsen in patients with hypertriglyceridemia and high cardiovascular risk will be presented on April 7, 2024, at 8:30am ET.