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.
相关临床试验
323
171 进行中
药物批准
2
批准总数
监管机构
1
监管机构数
成立时间
1989
进行中（未招募）
169
52.3%
Approved For Marketing
4
1.2%
已完成
96
29.7%
Enrolling By Invitation
1
0.3%
尚未招募
1
0.3%
招募中
43
13.3%
终止
5
1.6%
撤回
4
1.2%
- ADARx Pharmaceuticals priced an upsized IPO of 26.25 million shares at $17 each, raising about $446.3 million in gross proceeds. - AbbVie agreed to a concurrent private placement that would give it roughly 4.9% of ADARx, capped at $100 million. - Combined gross proceeds are expected to reach approximately $535.2 million before any underwriters' option exercise. - The capital funds onvuzosiran, an siRNA for hereditary angioedema now enrolling in the Phase III STOP-HAE trial.
- Roche's investigational antisense oligonucleotide sefaxersen met the primary endpoint of the Phase 3 IMAgINATION study in adults with primary IgA nephropathy at 37 weeks. - The prespecified interim analysis showed statistically significant and clinically meaningful reductions in 24-hour urine protein-to-creatinine ratio versus placebo, with no new safety signals. - Roche did not disclose the magnitude of the proteinuria reduction, limiting cross-trial comparison with approved and investigational IgAN therapies. - The 459-patient trial remains blinded and will continue to week 105 to assess kidney function change measured by eGFR.
- Otsuka Pharmaceutical Development & Commercialization has initiated a global early access program for ulefnersen, an investigational antisense oligonucleotide for FUS-ALS, announced September 22, 2026. - The program targets patients with a confirmed genetic diagnosis of FUS-ALS who cannot enroll in an ongoing clinical trial and meet predefined eligibility criteria. - Ulefnersen binds FUS pre-mRNA to reduce FUS protein production and is given by intrathecal injection; it remains unapproved worldwide and is being studied in the Phase 1-3 FUSION trial. - FUS-ALS accounts for an estimated 0.6% of all ALS cases but 43 to 52% of juvenile and pediatric ALS, with no approved therapies targeting its genetic cause.
- The Phase 3 FUSION trial met its primary endpoint, with ulefnersen showing a statistically significant improvement in functional impairment and survival versus placebo in FUS-ALS (p=0.0005). - Ulefnersen is the first treatment to target the underlying genetic cause of FUS-ALS and the first to show potential to modify disease progression in a placebo-controlled study. - Secondary endpoints, including serum neurofilament light chain and time to death, permanent ventilation, rescue or withdrawal, also favored ulefnersen with a favorable safety profile. - Ionis and Otsuka plan to discuss the results with the FDA and global health authorities to pursue potential expedited regulatory submission pathways.
- Novartis' pelacarsen significantly lowered lipoprotein(a) but failed to produce a statistically significant reduction in major cardiovascular events in the Phase 3 Lp(a)HORIZON trial. - The trial enrolled 8,323 patients with elevated Lp(a) and established heart disease, testing whether biomarker lowering translates into fewer heart attacks and strokes. - Citi analysts expect the result to push CRISPR Therapeutics toward its more potent next-generation candidate CTX321 over CTX320, which achieved up to 73% Lp(a) reductions. - Pelacarsen was discovered by Ionis Pharmaceuticals and licensed to Novartis in 2019, and the miss weakens validation of antisense technology in Lp(a)-driven cardiovascular disease.
- 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.
