Swedish Orphan Biovitrum AB

Ionis Pharmaceuticals’ Tryngolza has succeeded in two crucial late-stage tests that could considerably broaden the drug’s commercial reach, the company said Tuesday. Ionis is mulling how to price the drug in these patients, who have severely high levels of fat molecules called triglycerides. The drug is already in the rare disease market with a list price of almost $600,000. In two Phase 3 trials in patients with severe hypertriglyceridemia (sHTG), the drug cut triglyceride levels by up to 72% at six months. It also reduced cases of acute pancreatitis (AP), a potentially fatal condition that can result from high triglyceride levels, by 85%. Both figures are adjusted for placebo. “The results were unprecedented, groundbreaking, just really remarkable,” Ionis CEO Brett Monia told Endpoints News ahead of the data release. “An 85% reduction in AP has never been shown before in this patient population,” he added. Ionis said it would file this year to expand the drug’s label into sHTG. Monia said the drug could be launched in this new indication in the second half of 2026. Tryngolza is already approved for a genetic disease called familial chylomicronemia syndrome (FCS), in which the body struggles to break down triglycerides. Tuesday’s data come from studies known as CORE and CORE2, which enrolled patients who were already on standard-of-care lipid-lowering therapy. Analysts from TD Cowen wrote in an Aug. 25 note that cardiologists were seeking a 50% triglyceride reduction in these studies. A 50% drop in triglycerides could lead to a 30-50% reduction in AP events, the analysts said. Tryngolza steamed past these benchmarks. In the CORE trial, a 72% triglyceride reduction was seen with the higher dose of 80 mg, given subcutaneously monthly. The lower dose, 50 mg, achieved a 63% placebo-adjusted drop. In CORE2, the placebo-adjusted triglyceride reductions were less pronounced, at 55% and 49% with the same high and low doses, respectively, owing to a much higher placebo response. Placebo recipients in CORE2 had a 14% drop in their triglycerides, whereas in CORE, the placebo reduction was just 0.5%. Monia said that he doesn’t have a clear explanation for why the placebo response was so much stronger in CORE2, although it might come down to where the trials were conducted. “We’re still working through the data. We’ll be looking at things like, were the sites different or similar?” he said. “We had some sites that were overlapping in CORE and CORE2, and then we had some unique sites for CORE and CORE2 as well.” Both trials had sites in the US, Europe and Canada, but CORE had unique sites in Australia, New Zealand, Israel, South Africa and Turkey. CORE2, but not CORE, had sites in Mexico, Argentina, India and Malaysia, according to the US federal clinical trials database. CORE and CORE2 enrolled 617 and 446 patients, respectively, with those in CORE having slightly higher baseline triglycerides — 836 mg/dL versus 749 mg/dL in CORE2. The trials were identical in recruitment criteria and endpoints. Monia stressed that the placebo-adjusted triglyceride cut in CORE2 was “still remarkable.” All the triglyceride reductions in both trials were highly statistically significant versus placebo, with p-values below 0.0001. The 85% reduction in AP events, which comes from data pooled across both doses and both trials and was measured at one year, was also statistically significant at p=0.0002. Ionis’ pivotal data in sHTG raise the stakes for Arrowhead’s similar candidate, plozasiran. That oligonucleotide is in two pivotal Phase 3 trials, named SHASTA-3 and -4, also for severe hypertriglyceridemia, with data expected next year. In its Phase 2 SHASTA-2 trial in sHTG, plozasiran achieved a 58% reduction in triglyceride levels after nearly one year, versus 7% for placebo. That study did not assess AP rates. Side effects in CORE and CORE2 were generally balanced across treatment groups, and serious adverse events occurred less frequently with Tryngolza than with placebo, Ionis said. Injection site reactions, which were mostly mild, happened more often with the drug, however. Tryngolza’s list price for the rare condition FCS is $595,000 per year. And that price would not be competitive in sHTG, a disorder estimated to affect around three million people in the US alone. “We will step the price down. We haven’t settled on the price yet,” Monia said. He added that the new price would be consistent with “drugs of similar nature” in the cardiovascular space, which he said have an annual price in the $10,000 to $20,000 range, though that is “not definitive.” He added that the new data put the company in a strong position for negotiations with payers. But Tryngolza will lose its rare disease price even for FCS patients if it is approved for sHTG, because FCS patients will simply be folded into the sHTG population. “FCS patients will benefit from the price change. Once we get there for sHTG, it’ll be one price for FCS and sHTG,” Monia said. “We’ll announce price once we get approval.” Ionis will launch the drug in the US, and its partner Sobi will take charge everywhere else except Canada and China. Tryngolza brought Ionis $19 million in the second quarter of 2025, a threefold increase over its first quarter sales. The product is an antisense oligonucleotide that works to reduce production of apolipoprotein C-III, which regulates triglyceride metabolism in the blood. Data in moderate HTG were presented at the annual meeting of the European Society of Cardiology in Madrid on Saturday.

STOCKHOLM, Aug. 29, 2025 /PRNewswire/ -- As per 29 August 2025, the total number of shares in Swedish Orphan Biovitrum AB (publ) (Sobi®) amounts to 357,412,837 shares, of which 356,000,049 are common shares and 1,412,788 are C-shares. The total number of votes is 356,141,327,8. The increase in the number of shares and votes results from issues of 1,412,788 class C shares. The class C shares have been issued for the purpose of ensuring fulfilment of commitments under the long-term incentive programmes. As per 29 August 2025 the company holds 11,343,74 common shares. Sobi Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. Gerard Tobin Head of Investor Relations This information is information that Sobi is obliged to make public pursuant to the Swedish Financial Instruments Trading Act. The information was submitted for publication on 29 August 2025 at 08.00 CEST. This information was brought to you by Cision The following files are available for download: SOURCE Swedish Orphan Biovitrum AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The 80mg dose of Dawnzera is self-administered through a subcutaneous autoinjector. Credit: Ionis Pharmaceuticals, Inc./Business Wire. The US Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals’ RNA-targeted prophylactic treatment, Dawnzera (donidalorsen), for use in preventing hereditary angioedema (HAE) attacks in those aged 12 years and above. Dawnzera is designed to inhibit plasma prekallikrein, a significant activator of the inflammatory mediators that cause acute HAE attacks. This 80mg dose of the medicine is self-administered through a subcutaneous autoinjector, with patients having the option to choose between dosing every four or eight weeks. The agency’s approval was influenced by the positive outcomes from the Phase III OASIS-HAE trial. The placebo-controlled, randomised, global, multicentre, double-blind trial’s primary endpoint was achieved, with an 81% reduction in the monthly rate of HAE attacks for those treated with Dawnzera every four weeks, as compared to placebo, over 24 weeks. This reduction rose to 87% from the second dose, which was a secondary endpoint. The frequency of moderate-to-severe HAE attacks was also reduced by 90% from the second dose onwards during the same timeframe. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Further evidence of Dawnzera’s performance comes from the OASISplus open-label extension trial, where the eight-week dosing schedule demonstrated a similar impact to the four-week regimen over time. After one year, a 94% decrease in the total mean attack rate from baseline was observed across both dosing groups. HAE is an uncommon genetic condition, characterised by recurrent, severe swelling in various body parts. Ionis Pharmaceuticals CEO Brett Monia stated: “Dawnzera represents a significant advance for people living with HAE who need improved treatment options. “With strong and durable efficacy, convenient administration and the longest dosing option available, we believe Dawnzera will be the prophylactic treatment of choice for many people living with HAE.” In March 2025, Ionis entered a licensing agreement with Sobi. This agreement provided Sobi with the exclusive rights to commercialise olezarsen for the treatment of familial chylomicronaemia syndrome and severely elevated triglycerides. Pharmaceutical Technology Excellence Awards - The Benefits of Entering Gain the recognition you deserve! The Pharmaceutical Technology Excellence Awards celebrate innovation, leadership, and impact. By entering, you showcase your achievements, elevate your industry profile, and position yourself among top leaders driving pharmaceutical advancements. Don’t miss your chance to stand out—submit your entry today! Nominate Now