Latest news with #Tryngolza
Yahoo
04-08-2025
- Business
- Yahoo
Why Morgan Stanley Upgraded This Biotech Stock to a Buy
Sitting at a market cap of $6.8 billion, Ionis Pharmaceuticals (IONS) is a U.S.-based biotech company that develops RNA-targeted therapeutics to treat diseases caused by genetic or biochemical dysregulation. Ionis has many approved products in the market, most in collaboration with large pharmaceutical companies. However, it independently launched its sole product, Tryngolza, in December of last year. The drug had a blockbuster performance following its commercial launch. IONS stock has surged 21.5% year-to-date, outperforming the market. Based on the biotech's strong second-quarter results, Morgan Stanley analyst Michael Ulz upgraded the stock to a "Buy" with about 45% upside potential, based on the target price of $62. More News from Barchart Find Winning Momentum Trades With This Moving Average Stock Screener Tariffs, Earnings and Other Can't Miss Items this Week Dear Nvidia Stock Fans, Mark Your Calendars for August 27 Get exclusive insights with the FREE Barchart Brief newsletter. Subscribe now for quick, incisive midday market analysis you won't find anywhere else. Ulz cited a combination of strong operational performance and promising future catalysts as reasons for his bullish stance on Ionis. Let's take a look at what those drivers are, and whether Ionis stock is truly a 'Buy' now. Why Did Morgan Stanley Upgrade IONS Stock? According to Michael Ulz, the launch of Tryngolza was the standout factor in Ionis' second quarter earnings, exceeding not only Wall Street's expectations but also initial internal sales forecasts. Its robust commercial debut shows Ionis' management's strong execution capabilities and solidifies investor confidence in the company's strategic direction. Tryngolza (olezarsen) is used to treat familial chylomicronemia syndrome (FCS), a rare and life-threatening genetic lipid disorder in adults, in combination with diet. It was the drug's second full quarter on the market, with net product sales of $19 million in Q2. Management emphasized in the Q2 earnings call that physicians are prescribing it to a large number of patients, indicating increased confidence in the drug. Furthermore, approximately 60% of patients are covered by commercial insurance and 40% by government programs, with more than 90% paying no out-of-pocket costs, contributing to the drug's success. Crucially, with a positive opinion from the European CHMP (Committee for Medicinal Products for Human Use), Ionis is planning to expand Tryngolza's reach throughout Europe. This will lay the groundwork for significant international expansion. The company also earned a royalty revenue of $70 million from the following products, among others: Spinraza (nusinersen) — for spinal muscular atrophy in partnership with Biogen (BIIB) Wainua (eplontersen) — for ATTRv PN, co-commercialized with AstraZeneca (AZN) Total revenue in the second quarter increased by an impressive 100.8% year-on-year to $452 million. The company also reported a profit of $0.70 per share, up from a loss of $0.45 per share in the year-ago quarter. Morgan Stanley's Ulz also mentioned many upcoming events that could boost the stock, including the anticipated Phase 3 readouts for severe hypertriglyceridemia (SHTG) and other pipeline updates scheduled for 2026. Furthermore, early feedback from key opinion leaders on the upcoming Phase 3 CORE/CORE2 data for Olezarsen to treat patients with sHTG has been overwhelmingly positive, adding to the sense of optimism. The company expects topline data from the trial in September of this year. Notably, Ionis has an extensive late-stage pipeline, with many in Phase 2-3 clinical development. Donidalorsen is expected to be approved in August for hereditary angioedema (HAE), a rare but well-known genetic condition that causes painful swelling episodes. Management believes Donidalorsen has the potential to significantly broaden the company's footprint, as up to 20% of HAE patients switch prophylactic therapies each year, indicating dissatisfaction with current options. Furthermore, ION582 is beginning a Phase 3 trial to determine its efficacy for Angelman syndrome (AS), a serious and rare neurodevelopmental disorder. With a successful drug launch under its belt and multiple potential catalysts on the horizon, Morgan Stanley believes Ionis is well-positioned for continued growth. Management is also confident that the company's current momentum will lead to long-term revenue growth and positive cash flow. For the full year, Ionis expects revenue to increase by 17% to 20%, to $825 million to $850 million, while analysts predict $869.6 million in revenue. What Are Other Analysts Saying About IONS Stock? Aside from Morgan Stanley, Oppenheimer, Needham, Raymond James, and H.C. Wainwright analysts also maintain a 'Buy' rating for IONS stock. Furthermore, H.C. Wainwright has raised the target price to $65 from $50. Separately, Wells Fargo analyst Yanan Zhu maintained a 'Buy' rating and a target price of $77 for IONS after the company's strong second-quarter performance, which the firm says reflected both top-line momentum and operational discipline. Zhu also highlighted the company's strategic execution, specifically the successful launch of Tryngolza. Looking ahead, Zhu expects additional upside from several key catalysts, including upcoming pivotal trial readouts and potential regulatory milestones. These developments could further strengthen the company's pipeline and unlock long-term value for shareholders. Overall, on Wall Street, IONS remains a 'Moderate Buy.' Of the 23 analysts covering the stock, 15 rate it a 'Strong Buy,' two say it is a 'Moderate Buy,' five rate it a 'Hold,' and one suggests a 'Strong Sell.' The average target price of $57.77 suggests an upside potential of 35% from current levels. Additionally, the Street-high estimate of $83 suggests the stock has an upside potential of 93% over the next 12 months. With successful products and a robust pipeline approaching key milestones, Ionis appears to be on track to become a multi-product, multibillion-dollar biotech leader, which explains analysts' optimism for the stock. Ionis is developing a long-term, growth-oriented business model capable of delivering transformational therapies to patients while providing strong returns to investors, making it a compelling biotech stock for the long haul. On the date of publication, Sushree Mohanty did not have (either directly or indirectly) positions in any of the securities mentioned in this article. All information and data in this article is solely for informational purposes. This article was originally published on
Medscape
28-07-2025
- Health
- Medscape
EMA Committee Backs Tryngolza for Rare Lipid Disorder
The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization in the European Union to Tryngolza (olezarsen; Ionis Ireland Limited) for treating adults with familial chylomicronemia syndrome (FCS). The drug, to be available as an 80-mg solution for injection in prefilled pens, will be used as an adjunct to dietary modifications in adult patients with genetically confirmed FCS. FCS is a rare inherited disorder caused by loss-of-function variants in LPL or related genes, leading to absent or severely reduced lipoprotein lipase activity. This results in extreme hypertriglyceridemia (often > 880 mg/dL or 10 mmol/L) due to accumulation of chylomicrons and very low-density lipoprotein (VLDL). FCS affects about 13 people per million in Europe and causes recurrent acute pancreatitis (in about 85% of patients), severe abdominal pain, and chronic complications. Standard lipid-lowering therapies are typically ineffective, and strict fat-restricted diets are difficult to maintain, making disease management challenging. Tryngolza, a lipid-modifying agent, is an antisense oligonucleotide-GalNAc₃ carbohydrate ligand conjugate that targets apolipoprotein C-III (apoC-III) mRNA in hepatocytes. The GalNAc₃ moiety facilitates selective uptake by liver cells and, once internalized, the antisense oligonucleotide blocks apoC-III protein synthesis. This enhances lipolysis and hepatic clearance of triglyceride-rich lipoproteins, including VLDL and chylomicrons, ultimately lowering plasma triglyceride levels. Significant Benefits in Clinical Trials The CHMP decision relied on positive findings from the phase 3 Balance trial involving 66 patients with genetically confirmed FCS and severe hypertriglyceridemia, including 71% with a history of acute pancreatitis. Participants were randomly assigned to receive Tryngolza 80 mg, 50 mg, or placebo subcutaneous injections every 4 weeks for 49 weeks. Tryngolza 80 mg significantly reduced triglyceride levels by 43.5 percentage points at 6 months compared with placebo, while the 50-mg dose showed a nonsignificant reduction. Both doses markedly decreased apoC-III concentrations, with the 80-mg group achieving a 73.7-percentage point drop and the 50-mg group a 65.5-percentage point decline vs placebo. Acute pancreatitis events decreased significantly, with 11 episodes occurring among placebo recipients compared with just one in each Tryngolza group over 53 weeks, indicating an 88% risk reduction. The most common side effects of Tryngolza include injection-site erythema, headache, arthralgia, and vomiting.
Business Insider
01-07-2025
- Business
- Business Insider
Ionis Pharmaceuticals upgraded to Overweight from Equal Weight at Barclays
Barclays upgraded Ionis Pharmaceuticals (IONS) to Overweight from Equal Weight with a price target of $57, up from $51, ahead of Tryngolza Phase 3 CORE/CORE2 data in severe hypertriglyceridemia due likely in September. After having spoken with three key opinion leaders and doing a deep dive on historical clinical trials, natural history data, and statistical analysis, the firm sees a greater than 90% probability that the trial will hit the primary endpoint and sees a high probability it will hit all key second biomarker endpoints, the analyst tells investors. Don't Miss TipRanks' Half-Year Sale Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with TipRanks' Smart Investor Picks, delivered to your inbox every week.
Yahoo
20-05-2025
- Health
- Yahoo
Ionis reports positive data from study of olezarsen for hypertriglyceridemia
Ionis Pharmaceuticals has reported positive topline outcomes from the randomised, global Phase III Essence study of Tryngolza (olezarsen) in individuals with moderate hypertriglyceridemia and, or at risk for, atherosclerotic cardiovascular disease (ASCVD). This condition is characterised by fasting triglyceride (TG) levels between ≥150mg/dL and <500mg/dL. The multicentre, double-blind, placebo-controlled Essence study was carried out with the TIMI Study Group, the company's research partner. It enrolled 1,478 subjects aged 18 years and above diagnosed with or at risk for ASCVD, and 9% of them had fasting triglycerides of ≥500mg/dL at baseline. Subjects received either 50mg or 80mg of olezarsen or a placebo every four weeks via subcutaneous injection for 12 months. The primary endpoint focused on the percentage change from baseline in fasting TG levels at the six-month mark, compared to the placebo. Key secondary endpoints were changes in TG levels at 12 months, the proportion of subjects achieving fasting TG levels of <150mg/dL, and the percentage variations in other lipid parameters against the placebo over the treatment period. The trial, which included subjects already on standard of care, lipid-lowering medications, achieved its primary endpoint. A significant decrease in TG levels was observed, with a placebo-adjusted 61% decrease at the 80mg monthly dose and a 58% decrease at the 50mg dose over a six-month period, compared to the placebo. In addition, the therapy met all key secondary endpoints, with the majority of subjects achieving normal TG levels of <150mg/dL. Olezarsen also demonstrated a favourable tolerability and safety profile in the trial. Ionis Pharmaceuticals global cardiovascular development senior vice-president Sam Tsimikas said: 'Following the Food and Drug Administration (FDA) approval and encouraging launch of Tryngolza (olezarsen) for people living with familial chylomicronaemia syndrome (FCS), a rare, genetic form of severely elevated TGs, these data support olezarsen's potential tobenefit the much broader population of people living with severe hypertriglyceridemia (sHTG).' The investigational RNA-targeted medicine is designed to decrease the body's apolipoprotein C III (apoC-III) production, a protein for regulating the metabolism of triglyceride in the blood. Last November, Ionis announced the design for a pivotal Phase III trial of ION582, an investigational treatment for Angelman syndrome (AS), following discussions with the US FDA. "Ionis reports positive data from study of olezarsen for hypertriglyceridemia" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
11-02-2025
- Business
- Yahoo
Why Insiders Are Selling Ionis Pharmaceuticals, Inc. (IONS) Recently
We recently compiled a list of the . In this article, we are going to take a look at where Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) stands against the other feminist stocks. We previously covered 10 Mid-Cap Stocks Insiders Are Buying Recently. Why should investors analyze insider trading activity? Insiders, or people in high positions within a company—such as executives and directors—can have crucial insights into the company's strategic moves, plans, and initiatives. When, for example, a CEO invests their own capital in company stocks, it can indicate strong confidence in the company's future results. What does it mean when insiders are selling their shares? It doesn't necessarily mean bad news for the stock. Sometimes, large shareholders just want to trim their holdings to more appropriate position sizes based on the risk/reward potential. It is important to note that both insider selling and buying can be driven by various motives. Therefore, any insider trading activity should be analyzed within the broader context of the company's fundamentals, industry trends, and overall market conditions. Many insider purchases don't necessarily mean the company will be successful. That's why due diligence before any investment is of the utmost importance. Investors should also take into account and carefully analyze underlying reasons for insider transactions. However, insider trading activity in combination with other relevant factors can offer valuable insights into a company's capabilities, helping investors make more informed investment decisions. To identify the 10 mid-cap stocks insiders are selling recently, we considered only stocks with a market capitalization of between $2 billion and $10 billion. We first used Insider Monkey's insider trading stock screener and looked for stocks with at least two insiders buying over the last two months. With each stock we note the number of recent insider sales and the company's current market capitalization. But why is it important to follow insider activity? Our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds, focusing on insider trading and stock picks from hedge fund investor newsletters and conferences. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 275% since May 2014, beating its benchmark by 150 percentage points (see more details here) A scientist in a laboratory making a breakthrough discovery in biotechnology. Number of Insiders Selling: 10 Market Capitalization: $5.152B Ionis Pharamceuticals is a biotechnology company focused on discovering and developing RNA-targeted therapeutics. Currently, it has six marketed medicines and a pipeline in innovative therapies across areas of neurology, cardiology and rare diseases. In December 2024, the U.S. Food and Drug Administration (FDA) greenlighted Tryngolza, an RNA-targeted therapy developed by Ionis for the treatment of familial chylomicronemia syndrome (FCS). Tryngolza is the first-ever FDA-approved treatment that significantly and substantially reduces triglyceride levels in adults with FCS and offers clinically meaningful reduction in AP events when used with an appropriate diet (≤20 grams of fat per day). In January and February, 10 insiders, including CEO, CCO, and CBO sold approximately $5.12 million worth of Ionis Pharmaceuticals shares at an average price of around $32.54 per share. The stocks is now trading at $31.48, having lost 9.95% year-to-date. As many as 18 analysts have 'Buy' as the average rating for Ionis stock, while the stock price target is $62.0. David Lebowitz, an analyst from Citi, recently gave a 'Buy' rating on the company's shares, while the associated price target remains the same at $67.00. Ionis is also one of the 12 Best Healthcare Stocks to Buy According to Analysts. Over the last 12 months, the company's shares lost 37.51%. Overall IONS ranks 3rd on our list of the mid-cap stocks insiders are selling recently. While we acknowledge the potential of IONS as an investment, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than IONS but that trades at less than 5 times its earnings, check out our report about the . READ NEXT: 20 Best AI Stocks To Buy Now and Complete List of 59 AI Companies Under $2 Billion in Market Cap Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
