Ionis Pharmaceuticals Inc（IONS）资讯与事件IONSIonis Pharmaceuticals Inc

Ionis Pharmaceuticals Receives Positive EU Opinion for Tryngolza in Severe Hypertriglyceridemia

Ionis Pharmaceuticals announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion recommending the indication extension for tryngolza as an adjunct to diet, in combination with other lipid-lowering therapies, for the treatment of adult patients with severe hypertriglyceridemia. If confirmed by the European Commission, tryngolza would be the first and only RNA-targeted therapy authorized in the EU for adults with sHTG and triglyceride levels greater than or equal to880 mg/dL. The positive CHMP opinion comes ahead of the previously anticipated timeline, with a final EC decision expected in the fourth quarter of 2026.

Genentech Reports Positive Interim Results in IgA Nephropathy Study

Genentech, a member of the Roche Group, announced positive prespecified interim results from the ongoing Phase III Imagination study evaluating investigational sefaxersen in adults with primary IgA nephropathy. The study met its primary endpoint with sefaxersen achieving statistically significant and clinically meaningful improvements in proteinuria reduction, compared to placebo at 37 weeks, as measured by 24-hour urine protein-to-creatinine ratio. Proteinuria is a key indicator of kidney damage and a reduction in UPCR is strongly associated with the preservation of long-term kidney function. Sefaxersen is a once-monthly subcutaneous injection designed to enable self-administration for people with IgAN. The safety and tolerability profile of sefaxersen was consistent with previously reported data, with no new safety signals identified. The Imagination study will continue as a blinded study to evaluate the change in kidney function over two years, as measured by estimated glomerular filtration rate at week 105. Interim analysis data will be presented at an upcoming medical congress and shared with health authorities, with the goal of bringing this treatment to patients as soon as possible.

Ionis and Otsuka Announce FUSION Trial Results

Ionis Pharmaceuticals and Otsuka Pharmaceutical Development & Commercialization announced topline results from the Phase 3 FUSION trial evaluating ulefnersen, an investigational RNA-targeted medicine, in people living with amyotrophic lateral sclerosis caused by mutations in the fused in sarcoma gene, also known as FUS-ALS. The study met its primary endpoint, with ulefnersen demonstrating a statistically significant improvement on the primary endpoint assessing functional impairment and survival using a joint rank analysis of time to death or permanent ventilation, time to rescue and change in ALS Functional Rating Scale Revised score from baseline to Day 505 compared to placebo, providing evidence from the first-ever placebo-controlled clinical study targeting the underlying genetic cause of FUS-ALS. Ulefnersen, discovered and developed by Ionis, is the first treatment to target the underlying genetic cause of FUS-ALS and demonstrate the potential to modify disease progression. The FUSION study also demonstrated statistically significant improvements across important secondary endpoints, including change from baseline in serum neurofilament light chain and time to death, permanent ventilation, rescue, or withdrawal due to disease progression, further supporting the potential of ulefnersen to improve the course of disease in people living with FUS-ALS. Ulefnersen demonstrated a favorable safety and tolerability profile, with most adverse events being mild or moderate in severity. Further prespecified and exploratory analyses of the data will be conducted to determine the full potential of ulefnersen for the treatment of FUS-ALS. Otsuka plans to move with urgency to review results of the FUSION trial with the U.S. Food and Drug Administration and continue discussions with other health authorities globally regarding potential expedited submission pathways for ulefnersen approval. Ionis and Otsuka plan to present detailed results from the FUSION trial at a future medical congress and submit for publication in a peer-reviewed journal in the coming months. Otsuka licensed ulefnersen from Ionis in 2024 under a collaborative development and licensing agreement. Under the terms of the agreement, Ionis received an upfront payment and is eligible to receive additional regulatory and sales milestone payments as well as tiered royalties on net sales of ulefnersen.