Ionis Pharmaceuticals, Inc. (formerly Isis Pharmaceuticals)
Biotechnology company specializing in antisense therapy, RNA interference and CRISPR therapeutics, developing medications targeting neurological and cardiovascular diseases. Renamed from Isis Pharmaceuticals in December 2015.
Clinical Trials
323
171 active
Approvals
2
Total approvals
Agencies
1
Regulatory bodies
Founded
1989
Active, not recruiting
168
52.0%
Approved For Marketing
3
0.9%
Available
1
0.3%
Completed
96
29.7%
Enrolling By Invitation
1
0.3%
Not yet recruiting
2
0.6%
Recruiting
43
13.3%
Terminated
5
1.6%
Withdrawn
4
1.2%
- Novartis's pelacarsen and Novo Nordisk's ziltivekimab both failed to separate from placebo in late-stage cardiovascular outcome trials despite strong genetic support for their targets. - Cardiologist Ethan Weiss said the field can no longer claim that genetics is undefeated in predicting which drugs will work in outcome studies. - Novartis shares fell 14% in a single session and Amgen dropped 9.8% as investors repriced roughly 35,000 patients still enrolled in Lp(a) outcome trials. - Weiss attributed the Lp(a) failure largely to background therapy, arguing the risk signal diminishes in patients already on statins and other powerful medicines.
- The FDA has approved Ionis Pharmaceuticals' Zanvastro (zilganersen), the first disease-modifying treatment for Alexander disease, an ultra-rare and often fatal neurological disorder. - Approval was based on a global randomized, double-blind, placebo-controlled Phase 1-3 study in which the 50 mg dose stabilized gait speed in patients aged five and older. - Zanvastro is given as a 50 mg intrathecal injection every three months and will launch at $285,000 per dose, an implied annual list cost of roughly $1.14 million. - Ionis expects peak annual sales above $100 million and will roll out the drug through 12 U.S. leukodystrophy centers of excellence to reach eligible patients.
- Novartis' Phase 3 HARBOR study of del-desiran failed to show statistically significant improvement over placebo on video hand opening time in myotonic dystrophy type 1. - The antibody oligonucleotide conjugate targets DMPK messenger RNA, and Novartis reported clinical activity on secondary endpoints and exploratory analyses including muscle strength. - Novartis shares fell about 9-10% while Dyne Therapeutics and Sarepta Therapeutics dropped sharply on read-through risk to their related DMPK programs. - Novartis is evaluating the full HARBOR dataset and will engage health authorities on a development path; no approved treatment exists for the disease itself.
- Once-daily deucrictibant extended-release tablets reduced hereditary angioedema attack rates by 83% versus placebo in the Phase 3 CHAPTER-3 trial, with a p-value below 0.0001. - The global, double-blind study randomized 85 adolescents and adults across 21 countries to 40 mg deucrictibant XR or placebo for 24 weeks, with all secondary endpoints met. - Pharvaris plans to submit a prophylaxis New Drug Application to the FDA in the first half of 2027, following an accepted NDA for the immediate-release formulation. - Deucrictibant blocks the bradykinin B2 receptor, potentially making it the first oral HAE therapy spanning both on-demand treatment and long-term prophylaxis.
- The ANGPTL3 inhibitors market was valued at USD 0.21 billion in 2025 and is projected to reach USD 3.48 billion by 2035, at a 32.4% CAGR. - Monoclonal antibodies led the market with 71% share in 2025, driven by Regeneron's EVKEEZA, the sole FDA-approved ANGPTL3 inhibitor for homozygous familial hypercholesterolemia. - Arrowhead's zodasiran is advancing into Phase III development following positive Phase IIb ARCHES-2 results, positioning RNAi therapeutics as the fastest-growing drug class. - Verve Therapeutics' VERV-201, an in vivo base-editing therapy, is advancing toward potentially one-time, permanent ANGPTL3 reduction for refractory hypercholesterolemia and HoFH.
- Roche announced the discontinuation of the Phase II GENERATION HD2 trial of tominersen after the antisense oligonucleotide failed to meet clinical efficacy endpoints despite achieving biomarker reductions. - The company also halted the Phase I POINT-HD study of RG6496, a mutant-selective huntingtin-lowering ASO, after concurrent animal studies revealed it cannot be given chronically with repeated dosing. - In both cases, no new safety concerns were identified in trial participants; Roche emphasized the decisions were independent and data-driven. - Roche's gene therapy program RG6662 continues unchanged, and the company remains committed to exploring multiple therapeutic approaches for Huntington's disease.
- The Phase III CARDIO-TTRansform trial of Wainua (eplontersen) in ATTR-CM failed to meet its primary composite endpoint of cardiovascular mortality and recurrent CV events. - A predefined subgroup analysis showed nominally significant benefit in patients receiving Wainua as standalone therapy without concomitant stabiliser treatment. - Wainua was generally well tolerated with a safety profile consistent with prior clinical findings; full data will be presented at the European Society of Cardiology Congress. - The result represents a setback for expanding Wainua beyond its current approval in hereditary transthyretin-mediated amyloid polyneuropathy.
- Deciphera Pharmaceuticals, a member of Ono Pharmaceutical, announced the first patient dosed in the global pivotal Phase 3 INTREPID study of sapablursen in polycythemia vera. - The trial compares sapablursen to placebo over a 32-week double-blind period, followed by up to 124 weeks of open-label treatment, with phlebotomy-free response as the primary endpoint. - Sapablursen, designed to reduce TMPRSS6 and raise hepcidin, holds FDA Fast Track and Orphan Drug designations from 2024 and Breakthrough Therapy Designation from 2025. - Ono obtained exclusive global development and commercialization rights to sapablursen from Ionis Pharmaceuticals under a license agreement signed in March 2025.
- The average biopharmaceutical employee earned $116,648 in 2025, roughly 30% more than the average S&P 500 worker at $89,744, according to an Endpoints News study. - Gilead topped Big Pharma with a median salary of $239,000, while biotech firms like Alnylam ($312,000) and Ionis ($305,000) far exceeded even the most generous large drugmakers. - Johnson & Johnson, the industry's perennial revenue leader, ranked only 10th in median salary at $91,000, demonstrating that company size and profitability do not directly correlate with employee pay. - The 32 large biotech companies studied collectively employ over one million people with a combined market capitalization of $3.88 trillion.
- GSK announced positive results from two pivotal phase III trials (B-Well 1 and B-Well 2) evaluating bepirovirsen, an investigational antisense oligonucleotide for chronic hepatitis B treatment in over 1,800 patients across 29 countries. - Bepirovirsen demonstrated statistically significant and clinically meaningful functional cure rates when combined with standard care, representing a potential breakthrough for a disease affecting more than 250 million people worldwide. - The drug could become the first finite six-month therapeutic option for chronic hepatitis B, compared to current treatments that typically require lifelong therapy with functional cure rates of only 1%. - GSK plans to begin global regulatory filings from Q1 2026, with the potential to transform treatment goals for chronic hepatitis B patients and reduce long-term liver complications including cancer.
