HC Wainwright & Co., Leerink Partners, and Goldman Sachs have revised their price targets for Ionis Pharmaceuticals, reflecting varied perspectives on the company's valuation and market performance. HC Wainwright analyst Mitchell S. Kapoor maintained a Buy rating and increased the target to $130 from $125. Separately, Leerink Partners analyst Mani Foroohar kept an Outperform rating and lifted the target to $129 from $122. Goldman Sachs analyst Salveen Richter maintained a Neutral rating and raised the price target to $75 from $74.

Rating and Target Details

The revised targets suggest potential upside from current trading levels, though ratings vary by firm. The following table summarizes the updates:

Ionis Pharmaceuticals, Inc. has received approval from the U.S. Food and Drug Administration (FDA) for TRYNGOLZA (olezarsen) as an adjunct to diet to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG). The approval marks the first and only treatment indicated to reduce the risk of acute pancreatitis in this patient population. TRYNGOLZA is available in 50 mg or 80 mg doses and is self-administered once monthly via an autoinjector. The company also entered a global licensing agreement with Italy-based Recordati for its investigational Alexander disease therapy, zilganersen, outside the U.S.

The FDA approval was based on positive results from the Phase 3 CORE and CORE2 studies, which were published in The New England Journal of Medicine. These global, multicenter, randomized, double-blind, placebo-controlled trials investigated the safety and efficacy of olezarsen in patients with triglyceride levels of 500 mg/dL or higher. The studies met their primary endpoints, demonstrating statistically significant reductions in fasting triglycerides and acute pancreatitis events compared to placebo.

Clinical Efficacy and Safety

In the CORE and CORE2 studies, TRYNGOLZA demonstrated rapid and consistent triglyceride control. The therapy lowered fasting triglyceride levels by up to 72% compared to placebo at six months and sustained those reductions at 12 months. Additionally, TRYNGOLZA significantly reduced acute pancreatitis events by up to 91%. Among patients treated with TRYNGOLZA with baseline and 12-month data, 86% achieved triglyceride levels below 500 mg/dL, a critical threshold for reducing acute pancreatitis risk.

The number needed to treat (NNT) over one year to prevent one episode of acute pancreatitis was 20 in the overall cohort and four in patients with triglycerides of 880 mg/dL or higher and a prior history of acute pancreatitis. Across the clinical program, TRYNGOLZA demonstrated a favorable safety and tolerability profile. The most common adverse reactions in patients with sHTG (incidence of 2% or higher than placebo) were injection site reactions and liver enzyme increases.

Market Availability and Strategic Partnerships

TRYNGOLZA will be available for sHTG in the U.S. in July. The drug is also approved in the United States, European Union, and other countries for adults with familial chylomicronemia syndrome (FCS), a rare form of sHTG. Ionis stated that this approval represents its first independent commercial launch in a prevalent condition, building on its previous success in FCS.

Separately, Ionis entered into a licensing agreement with Recordati, granting the company exclusive rights to develop and commercialize Zilganersen in all markets outside the U.S. Ionis will retain sole commercial rights in the U.S. and continue leading global development efforts, while Recordati will oversee regulatory filings and commercialization activities internationally. Zilganersen is currently under FDA review with a Prescription Drug User Fee Act action date of Sept. 22.

Zilganersen Clinical Data and Financial Terms

Ionis recently reported additional positive data from the pivotal Zilganersen study. The trial met its primary endpoint in patients aged five years and older. The 50 mg dose produced statistically significant stabilization in gait speed at week 61 compared with controls. Secondary and exploratory measures also favored treatment with Zilganersen. The investigational therapy demonstrated a favorable safety profile, with most adverse events classified as mild or moderate.

Under the agreement, Ionis will receive a $30 million upfront payment and remain eligible for milestone payments and tiered royalties reaching the mid-20% range on annual net sales. William Blair commented that TRYNGOLZA's availability in July for sHTG is likely to be a meaningful contributor to cash flow breakeven guidance in 2028. Analyst Myles Minter reiterated the Outperform rating on the stock.