JP Morgan upgraded Ionis Pharmaceuticals (NASDAQ:IONS) to overweight from neutral citing a potential label expansion for Tryngolza (olezarsen) and promising candidates in its pipeline. The bank raised its price target to $80 from $49 (~16% upside based on Oct. 7 close). ...

Core Insights - Ionis Pharmaceuticals' shares surged 35% following positive results from late-stage studies CORE and CORE2 for its drug Tryngolza (olezarsen) targeting severe hypertriglyceridemia (sHTG) [1][12] - Both studies achieved their primary endpoint, demonstrating significant placebo-adjusted reductions in triglyceride (TG) levels [2][3] Study Results - At the 80 mg dose, Tryngolza resulted in TG reductions of 72% in CORE and 55% in CORE2 after six months; at the 50 mg dose, reductions were 63% in CORE and 49% in CORE2 [3] - The treatment also significantly reduced acute pancreatitis (AP) events by 85% compared to placebo over 12 months [5][12] Regulatory and Market Potential - Tryngolza was previously approved by the FDA for familial chylomicronemia syndrome (FCS), marking it as the first approved treatment for this condition [6] - Ionis plans to seek FDA label expansion for Tryngolza to include sHTG, targeting a much larger market of approximately 3 million affected individuals compared to 3,000 with FCS [10][8] Collaborations and Revenue Streams - Ionis has partnerships with major pharmaceutical companies like AstraZeneca, Biogen, GSK, and Novartis, providing funds through license fees and milestone payments [14] - 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 [15] Future Developments - Ionis is expanding its portfolio with several wholly-owned candidates in late-stage studies, including drugs for Alexander's disease, ALS, and Angelman syndrome, with expected commercial launches in the next three years [19]

Core Insights - Ionis Pharmaceuticals reported strong second-quarter results with adjusted earnings of 85 cents, a significant improvement from an adjusted loss of $35 million a year ago, and beating analysts' expectations of a 52-cent loss [1] - Revenue for the first quarter of 2025 increased by 10% to $132 million, surpassing the consensus estimate of $125.32 million, driven by higher commercial revenue [1] Revenue Performance - Ionis' revenues more than doubled to $452 million, exceeding the consensus of $282.95 million, primarily due to the successful launch of Tryngolza and increased royalty and R&D revenues [2] - Tryngolza generated net product sales of $19 million in the second quarter of 2025 and $26 million in the first half of 2025 [2] Product Developments - Tryngolza received a positive opinion from the Committee for Medicinal Products for Human Use, with a European Commission decision expected by Q4 2025 [3] - Wainua generated sales of $44 million in the second quarter of 2025, resulting in royalty revenue of $10 million [3] - Spinraza generated global sales of $393 million, leading to royalty revenue of $54 million [4] Financial Guidance - Ionis raised its fiscal 2025 sales guidance from $725 million-$750 million to $825 million-$850 million, compared to the consensus estimate of $754.67 million [6] - The company expects a 2025 adjusted operating loss of $300 million-$325 million, an improvement from previous guidance of less than $375 million [7] Market Reaction - Following the announcement, Ionis stock rose by 5.23% to $43.65 [7]

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].

Core Insights - Ionis Pharmaceuticals reported a first-quarter EPS loss of 93 cents, an improvement from a loss of 98 cents, and better than the consensus estimate of a $1.12 loss [1] - Revenue for the first quarter of 2025 increased by 10% to $132 million, surpassing the consensus of $125.32 million, driven by higher commercial revenue [1] Financial Guidance - The company raised its 2025 revenue guidance by over 20% to a range of $725-$750 million, compared to previous guidance of over $600 million and the consensus of $659.37 million [4] - Ionis expects a 2025 adjusted operating loss of less than $375 million, improved from prior guidance of less than $495 million, with cash and equivalents projected at approximately $1.9 billion [4] Product Performance - The drug Tryngolza (olezarsen) generated over $6 million in net product sales in its first full quarter post-approval in the U.S. [6] - Wainua (eplontersen) for polyneuropathy generated $39 million in sales, resulting in $9 million in royalty revenue for the first quarter of 2025 [6] - Spinraza (nusinersen) for spinal muscular atrophy achieved global sales of $424 million, leading to $48 million in royalty revenue in the first quarter of 2025 [6]