Ionis Pharmaceuticals Inc

Ionis Pharmaceuticals reported a narrower-than-expected adjusted loss and higher revenues for the second quarter of 2026, while reaffirming its full-year guidance. The company posted an adjusted loss of 43 cents per share, beating the Zacks Consensus Estimate of a loss of 89 cents, and revenues of $268 million, surpassing the $190.7 million estimate. Total revenues fell nearly 41% year over year due to a $280 million upfront payment from Ono Pharmaceutical in the prior-year period, but excluding that payment, revenues rose 56%. Ionis reiterated its 2026 revenue guidance of $875 million to $900 million, with Tryngolza net product sales expected between $100 million and $110 million and Dawnzera between $110 million and $120 million, and maintained its adjusted operating loss forecast of $425 million to $475 million.

Cathie Wood's ARK Genomic Revolution ETF has purchased $15.3 million worth of Ionis Pharmaceuticals shares since the beginning of July, following a sharp decline in the stock after a late-stage trial failure for eplontersen in ATTR-CM. The purchases came after Ionis and partner AstraZeneca announced the surprise setback, suggesting Wood is capitalizing on the drop while maintaining a long-term view. Ionis recently won FDA approval for Tryngolza to treat severe hypertriglyceridemia, expanding its target market to more than 3 million U.S. patients, and is pivoting to commercialize its own assets with peak sales guidance raised to $3 billion. The company reported first-quarter revenue of $246 million, up 86% year over year, and expects to reach cash flow break-even by 2028. Ionis also has late-stage pipeline catalysts including a priority review for zilganersen in Alexander disease and an upcoming phase 3 readout for pelacarsen partnered with Novartis.

Ionis Pharmaceuticals announced the completion of enrollment in Cohort 1 of the global Phase 3 REVEAL study evaluating obudanersen, an investigational RNA-targeted therapy for Angelman syndrome. The pivotal cohort enrolled 136 participants between the ages of 2 and under 18 with genetically confirmed UBE3A deletion or mutation. Enrollment in the adult cohort, which includes participants aged 18 to 50, is expected to conclude in the third quarter of 2026, with topline results from the REVEAL study anticipated in the second half of 2027. The trial was designed with input from the Angelman syndrome community to evaluate the therapy across a broad patient population. Separately, H.C. Wainwright raised its price target on Ionis to $130 from $125 and maintained a Buy rating following the FDA approval of Tryngolza.

TimesSquare Capital Management's U.S. Mid Cap Growth Strategy fell 7.72% net in the first quarter of 2026, underperforming the Russell Midcap Growth Index's decline of 6.35%. The strategy navigated geopolitical tensions, temporary global tariffs, and supply chain disruptions following U.S. and Israeli involvement in Iran, which prompted a shift to safer assets. Central banks maintained steady policies despite energy-driven inflation. The strategy added Ionis Pharmaceuticals, a commercial-stage biotechnology company focused on neurology and cardiovascular indications, citing several commercially approved drugs and a deep pipeline with important clinical trial readouts this year. Ionis reported total revenues of $246 million in the first quarter, an 87% year-over-year increase, and its shares gained 84.16% over the past 52 weeks.

Ionis Pharmaceuticals has signed an ex-U.S. licensing agreement with Recordati for zilganersen, an investigational RNA therapy for Alexander disease. The deal grants Recordati rights to commercialize zilganersen outside the United States, while Ionis retains U.S. rights and global development leadership. Ionis is set to receive upfront and milestone payments, plus potential royalties on ex-U.S. sales, as FDA review of zilganersen for Alexander disease continues.

Ionis Pharmaceuticals shares rose 9.5% after the FDA expanded the label for Tryngolza to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia, moving beyond its initial approval for familial chylomicronemia syndrome. The approval makes Tryngolza the first and only therapy in the US with a label specifically addressing both triglyceride reduction and acute pancreatitis risk in this high-need population. The expanded indication opens a much broader market, though it also introduces pricing pressure and payer negotiation risks as Ionis shifts from a rare disease to a larger patient group. Ionis' investment narrative projects $2.3 billion in revenue and $300.8 million in earnings by 2029, with a fair value estimate of $100.92 per share, representing a 24% upside. The most bullish analysts see revenue reaching about $2.8 billion and earnings $456 million by 2029, but the broader label and pricing dynamics could either reinforce or challenge those targets.

This week brought a mix of FDA and EU approvals, layoffs, acquisitions, and clinical trial updates across the biotech sector. Ionis Pharmaceuticals secured FDA approval for TRYNGOLZA as an adjunct therapy for severe hypertriglyceridemia, while Merck received European Commission approval for Keytruda in combination with Padcev as a neoadjuvant treatment for muscle-invasive bladder cancer. Gilead's Trodelvy was cleared by the FDA for first-line metastatic triple-negative breast cancer, and Pfizer's IBRANCE won expanded approval for HR+/HER2+ metastatic breast cancer. Natera gained Japanese approval for its Signatera MRD test in colorectal cancer, and AbbVie's MAVIRET was approved in the EU for both acute and chronic Hepatitis C. Achieve Life Sciences received an FDA Complete Response Letter for its Cytisinicline NDA due to manufacturing deficiencies, though no efficacy or safety issues were raised. ADC Therapeutics announced a 17% workforce reduction as part of a strategic reorganization to focus on its ZYNLONTA lymphoma franchise. In dealmaking, Merck KGaA agreed to acquire Bio-Techne for $73 per share in cash, representing an enterprise value of $11.3 billion, while Ionis inked a licensing deal with Recordati for Zilganersen, an investigational therapy for Alexander disease. Passage Bio and Remix Therapeutics entered an all-stock merger agreement, and Boundless Bio and Serapha Bio also announced an all-stock merger. On the clinical front, IMUNON's Phase 3 OVATION 3 trial passed a safety review, Takeda's Zasocitinib showed superiority over Deucravacitinib in a Phase 3 psoriasis study, and Eledon reported positive long-term kidney transplant data for Tegoprubart. However, Pfizer's Sigvotatug vedotin missed its primary overall survival endpoint in a Phase 3 non-small cell lung cancer study, and MapLight's ML-004 failed to meet its primary endpoint in autism spectrum disorder, though it showed an irritability signal in adolescents. Definium Therapeutics reported positive Phase 3 results for DT120 ODT in major depressive disorder.

The IgA nephropathy clinical trial space is intensifying, with more than 25 companies actively developing over 30 pipeline drugs, according to a new report from DelveInsight. Key players include Haisco Pharmaceutical Group, Novartis, Vertex Pharmaceuticals, Biogen, Vera Therapeutics, AstraZeneca, Roche, Ionis Pharmaceuticals, Takeda, Arrowhead Pharmaceuticals, and others. Promising therapies in various trial phases include HSK39297, Zigakibart, Povetacicept, Felzartamab, Atacicept, ULTOMIRIS, Sefaxersen, TAK-079, ARO-C3, NM8074, WAL0921, KP104, PS-002, BHV-1400, CM313, NTQ5082, RNK288, and IFX 301. Approximately 12 or more drugs are in late-stage development, targeting mechanisms such as Complement Factor B inhibition, BAFF and APRIL antagonism, antibody-dependent cell cytotoxicity, Gd-IgA1 degradation, RNA interference, and CD38 antagonism. Recent milestones include positive Phase III data for Povetacicept and Atacicept, and publication of telitacicept results in the New England Journal of Medicine.

The U.S. Food and Drug Administration has approved Ionis Pharmaceuticals' TRYNGOLZA as the first and only treatment indicated to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia. TRYNGOLZA, available in 50 mg or 80 mg doses self-administered once monthly via an autoinjector, was approved based on Phase 3 CORE and CORE2 studies showing it lowered fasting triglyceride levels by up to 72% compared to placebo at six months and reduced acute pancreatitis events by up to 91%. Among patients 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 over one year to prevent one episode of acute pancreatitis was 20 in the overall cohort and four in patients with triglycerides of at least 880 mg/dL and a prior history of acute pancreatitis. Ionis plans to launch TRYNGOLZA in the U.S. in July, marking its first independent commercial launch in a prevalent condition.

Deciphera Pharmaceuticals announced the first patient has been dosed in the global pivotal Phase 3 INTREPID study evaluating sapablursen for the treatment of polycythemia vera, a rare and potentially life-threatening hematologic disease. The study will compare the efficacy and safety of sapablursen to placebo over a 32-week double-blind treatment period followed by up to 124 weeks of open-label treatment, with the primary endpoint being response defined by the absence of phlebotomy eligibility. Sapablursen has received Fast Track, Orphan Drug, and Breakthrough Therapy designations from the U.S. Food and Drug Administration. The trial has initiated in the United States and is planned in additional regions including North America, Latin America, Asia Pacific, and Europe. Deciphera is a member of Ono Pharmaceutical, which obtained exclusive global rights to sapablursen through a license agreement with Ionis Pharmaceuticals in March 2025.