Key Takeaways Ionis' phase III study showed zilganersen stabilized gait speed in Alexander disease patients.The drug demonstrated favorable safety and consistent benefit across secondary measures.Ionis plans an FDA filing in Q1 2026 as it expands its wholly owned medicine portfolio.Ionis Pharmaceuticals (IONS) announced positive topline results from a pivotal phase III study evaluating its investigational RNA-targeted therapy zilganersen for treating a rare neurological condition called Alexander disease (A ...

Ionis Pharmaceuticals (NASDAQ:IONS) has reported promising results from a pivotal study of its experimental medicine, zilganersen, for the treatment of Alexander disease, a rare and progressive neurological disorder. The study found that a 50 mg dose of zilganersen significantly improved ...

Core Insights - Ionis Pharmaceuticals reported second-quarter 2025 adjusted earnings per share (EPS) of 86 cents, significantly exceeding the Zacks Consensus Estimate of 27 cents, compared to an adjusted loss of 24 cents in the same period last year [1] - Total revenues reached $452 million, doubling year-over-year and surpassing the Zacks Consensus Estimate of $271 million [2] Revenue Streams - Ionis has licensed Spinraza to Biogen, which is responsible for its commercialization, and receives royalties from its sales. Spinraza is approved for treating spinal muscular atrophy globally [3] - Ionis also earns royalties from Biogen's Qalsody, approved for treating amyotrophic lateral sclerosis with SOD1 mutations, launched in the U.S. in 2023 and in the EU in May 2024 [3] - The FDA approved Wainua (eplontersen) for treating hereditary transthyretin-mediated amyloid polyneuropathy in December 2023, co-marketed with AstraZeneca in the U.S. [4] - Following Wainua's U.S. launch, Ionis began receiving royalties from AstraZeneca, which are included in commercial revenues [5] Commercial Revenue Performance - Commercial revenues rose 43% year-over-year to $103 million, driven by Tryngolza product sales and Wainua royalties, exceeding the Zacks Consensus Estimate of $88 million [7] - Tryngolza contributed $19 million in sales, up from $6 million in the previous quarter, indicating strong launch momentum [8] - Spinraza royalties totaled $54 million, down 5% year-over-year, with sales of $393 million, reflecting an 8% decline compared to the previous year [8] - Wainua royalty revenues amounted to $10 million, with sales of $44 million recorded by AstraZeneca [9] R&D Revenue Growth - R&D revenues surged 128% year-over-year to $349 million, driven by a $280 million upfront payment for out-licensing rights for a rare blood cancer drug to Ono Pharmaceutical [12] - Collaborative agreement revenues totaled $337 million, compared to $141 million in the year-ago quarter [13] Cost and Guidance Updates - Adjusted operating costs rose 8% year-over-year to $282 million, with SG&A costs increasing 42% to support commercialization efforts [14] - Ionis raised its 2025 revenue outlook to $825-$850 million, up from the previous guidance of $725-$750 million, reflecting strong uptake for Tryngolza [15] - The adjusted operating loss is now expected to be between $300-$325 million, down from previous guidance of less than $375 million [18] Updates on Wholly-Owned Candidates - Tryngolza is being evaluated in three late-stage studies for severe hypertriglyceridemia, with positive data from the ESSENCE study [20] - Donidalorsen is under FDA review for hereditary angioedema, with a decision expected next month [21] - Zilganersen is in a late-stage study for Alexander disease, with data expected in 2025 [22] Partnered Candidates Developments - AstraZeneca and Ionis are developing Wainua for ATTR-CM, with data from the phase III CARDIO-TTRANSform study expected in the second half of 2026 [23] - Novartis is developing pelacarsen for elevated Lp(a)-driven cardiovascular disease, with data expected this year [24] - AstraZeneca initiated a phase IIb study on opemalirsen for APOL1-mediated kidney disease, triggering a $30 million milestone payment to Ionis [25]

Core Insights - Ionis Pharmaceuticals announced positive top-line results from the phase III ESSENCE study for its drug Tryngolza, targeting moderate hypertriglyceridemia in patients at risk for atherosclerotic cardiovascular disease (ASCVD) [1][2] - The study achieved its primary endpoint, showing a statistically significant reduction in triglyceride (TG) levels, with reductions of 61% and 58% for 80 mg and 50 mg monthly doses, respectively [2] - A majority of participants in the study had their TG levels fall within the normal range (<150 mg/dL) post-treatment, and the drug met all key secondary endpoints [3] Recent Developments - Tryngolza received FDA approval for treating familial chylomicronemia syndrome (FCS), marking it as the first approved treatment for this rare genetic condition in the U.S. and Ionis' first independent commercial launch [4] - Ionis out-licensed ex-U.S. rights for Tryngolza to Sweden-based Sobi, which will handle future regulatory filings and commercialization outside the U.S. [5] - The drug is also being evaluated for severe hypertriglyceridemia (sHTG) in two late-stage studies, with data expected in Q3 2025 [6][7] Financial and Revenue Streams - Ionis has a diverse revenue stream through collaborations with major pharmaceutical companies like AstraZeneca, Biogen, GSK, and Novartis, providing funds for its development pipeline [10] - The company earns commercial revenues from royalties on Spinraza, which treats spinal muscular atrophy, and is also involved in marketing Qalsody for amyotrophic lateral sclerosis [11] - Recent approvals for drugs like Wainua and ongoing studies for other candidates could further enhance Ionis' revenue and reduce reliance on collaboration partners [12][14]

Core Viewpoint - Ionis Pharmaceuticals reported a narrower adjusted loss in Q1 2025 compared to the previous year and exceeded revenue expectations, driven by strong sales from its licensed products and new drug approvals [1][2][6]. Financial Performance - The adjusted loss per share for Q1 2025 was 75 cents, better than the Zacks Consensus Estimate of a loss of $1.11 and an adjusted loss of 77 cents in the same quarter last year [1]. - Total revenues reached $132 million, surpassing the Zacks Consensus Estimate of $120 million, marking an 11% increase year over year [2]. - Commercial revenues amounted to $76 million, a 29% increase year over year, exceeding the Zacks Consensus Estimate of $67 million [6]. - R&D revenues declined 7% year over year to $56 million, but still beat the Zacks Consensus Estimate of $50 million [10]. Revenue Streams - Ionis earns royalties from Biogen on the sales of Spinraza and Qalsody, with Spinraza royalties totaling $48 million, up 26% year over year [7]. - Wainua generated $9 million in royalty revenues, with sales recorded at $39 million by AstraZeneca [7]. - Tryngolza contributed $6 million in product sales during its first quarter of recognition [6]. Cost Structure - Adjusted operating costs rose 5% year over year to $249 million, with SG&A costs increasing by 52% to support commercialization efforts [11]. - R&D costs decreased by 6% as several late-stage studies concluded [11]. Guidance and Future Outlook - Ionis raised its 2025 revenue guidance to between $725 million and $750 million, up from over $600 million, reflecting new licensing deals [12]. - The adjusted operating loss is now expected to be less than $375 million, improved from the previous guidance of less than $495 million [15]. - The company anticipates ending 2025 with approximately $1.9 billion in cash, up from a prior projection of $1.7 billion [16]. Drug Development Updates - Tryngolza is under evaluation in three late-stage studies for severe hypertriglyceridemia, with data expected in 2025 [18]. - Donidalorsen is awaiting FDA approval for hereditary angioedema, with a decision expected by August 21, 2025 [19]. - Zilganersen is in a phase III study for Alexander disease, with data expected in 2025 [20]. Partnerships and Collaborations - AstraZeneca and Ionis are co-marketing Wainua for hereditary transthyretin-mediated amyloid polyneuropathy in the U.S. and have plans for further development in other forms of amyloidosis [21]. - Ionis has out-licensed rights for a rare blood cancer drug to Ono Pharmaceutical, receiving an upfront payment of $280 million and potential milestone payments [23].