IONS Ionis

sefaxersen · NCT05797610 registry update

countries: {'total_sites': 171, 'us_sites': 20} → {'total_sites': 162, 'us_sites': 20}

salanersen · NCT07221669 registry update

countries: {'total_sites': 13, 'us_sites': 3} → {'total_sites': 14, 'us_sites': 3}

Ionis Hibsago (bepirovirsen) receives FDA approval for chronic HBV

The FDA approved bepirovirsen (Hibsago) injection in combination with existing antiviral therapy for chronic hepatitis B in certain adults without cirrhosis. This marks the first approval of its kind for this indication.

salanersen · NCT07221669 registry update

countries: {'total_sites': 12, 'us_sites': 3} → {'total_sites': 13, 'us_sites': 3}

salanersen · NCT07221669 registry update

countries: {'total_sites': 11, 'us_sites': 3} → {'total_sites': 12, 'us_sites': 3}

Ionis: CHMP positive opinion for TRYNGOLZA (olezarsen) in sHTG

The CHMP adopted a positive opinion recommending approval of olezarsen as an adjunct to diet plus other lipid-lowering therapies for adults with severe hypertriglyceridemia (fasting TG ≥880 mg/dL). A final EC decision is expected in Q4 2026. This follows U.S. approval for sHTG and builds on positive Phase 3 CORE and CORE2 data.

pelacarsen · NCT07625306 registry update

status: NOT_YET_RECRUITING → WITHDRAWN; primary_completion: 2028-12-07 → 2029-01-22; completion: 2028-12-07 → 2029-01-22; enrollment: 240 → 0; why_stopped: → Study terminated by sponsor following the outcome of the Lp(a)HORIZON study (NCT04023552), which did not meet its primary endpoint

olezarsen · NCT07727538 registry update

countries: {'total_sites': 4, 'us_sites': 4} → {'total_sites': 5, 'us_sites': 5}

pelacarsen · NCT06813911 registry update

status: RECRUITING → ACTIVE_NOT_RECRUITING; enrollment: 340 → 246; countries: {'total_sites': 95, 'us_sites': 36} → {'total_sites': 94, 'us_sites': 35}

sefaxersen · NCT05797610 registry update

primary_completion: 2026-08-31 → 2028-05-25; completion: 2029-03-31 → 2029-08-31; countries: {'total_sites': 178, 'us_sites': 20} → {'total_sites': 171, 'us_sites': 20}

salanersen · NCT07444476 registry update

countries: {'total_sites': 1, 'us_sites': 1} → {'total_sites': 3, 'us_sites': 2}

obudanersen · NCT07782827 registry update

status: NOT_YET_RECRUITING → RECRUITING; countries: {'total_sites': 0, 'us_sites': 0} → {'total_sites': 1, 'us_sites': 1}

Ionis/Roche: Positive Phase 3 IMAgINATION interim topline for sefaxersen in IgAN

The Phase 3 IMAgINATION trial of sefaxersen met its primary endpoint at the prespecified 37-week interim analysis, demonstrating statistically significant and clinically meaningful reduction in 24-hour UPCR versus placebo in adults with primary IgAN. The safety profile was consistent with prior data and no new signals were identified. The blinded study continues to the week-105 eGFR endpoint; interim data will be presented at a medical congress and shared with regulators.

Ionis ulefnersen Phase 3 FUSION topline positive in FUS-ALS

The Phase 3 FUSION trial of ulefnersen met its primary endpoint with statistically significant improvement on a joint rank analysis of function, survival, time to death/permanent ventilation, time to rescue, and change in ALSFRS-R versus placebo (p=0.0005). Multiple secondary endpoints including serum NfL and time to key progression events were also met with a favorable safety profile. Ionis and Otsuka will discuss results with FDA and global regulators for potential expedited approval pathways.

Royalty Pharma: Novartis pelacarsen HORIZON Phase 3 misses primary endpoint

Novartis’ Phase 3 HORIZON outcomes trial for pelacarsen failed to meet its primary composite cardiovascular endpoint versus placebo. Royalty Pharma will not make any milestone payments to Ionis and its Spinraza royalty interest will revert to Ionis after $550 million in payments, yielding a 1.1x return on the $500 million investment.

Ionis: FDA approves Zanvastro (zilganersen) for Alexander disease

The FDA has granted full approval for zilganersen (Zanvastro) injection, making it the first approved therapy for Alexander disease in both pediatric and adult patients. This converts the asset from an investigational candidate to an approved product, completing its regulatory path.

Ionis Pharmaceuticals, Inc. · FDA approves Zanvastro (zilganersen)

FDA lists Zanvastro (zilganersen) as novel drug approval #37 of 2026 on 2026-09-03: To treat Alexander disease in pediatric and adult patients

Ionis: zilganersen NDA under Priority Review, PDUFA 22 Sep 2026

The 8-K states the NDA for zilganersen in Alexander disease is under FDA Priority Review with a PDUFA target action date of September 22, 2026. Ionis also entered a license agreement with Recordati for ex-U.S. development and commercialization. This sets the next regulatory decision point for the asset.

Ionis: CARDIO-TTRansform Phase 3 missed primary endpoint for eplontersen in ATTR-CM

The Phase 3 CARDIO-TTRansform trial of eplontersen in ATTR-CM did not meet its primary composite endpoint of CV mortality and recurrent CV events versus placebo. A nominally significant benefit (HR 0.71) was seen only in the pre-specified monotherapy subgroup, while patients on background stabilizers showed no treatment effect. Full data will be presented at ESC Congress in August 2026.

Ionis completes pivotal-cohort enrollment in Phase 3 REVEAL study of obudanersen

Ionis announced completion of enrollment in the pivotal pediatric cohort (Cohort 1, n=136) of the Phase 3 REVEAL study. The adult cohort (Cohort 2) is still enrolling and expected to finish in Q3 2026. Topline data from the full REVEAL study are now guided for the second half of 2027.

Ionis: FDA approves TRYNGOLZA (olezarsen) for sHTG

FDA approved TRYNGOLZA (olezarsen) as the first and only therapy indicated to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (TG ≥500 mg/dL). The approval was based on Phase 3 CORE and CORE2 data showing up to 72% TG reduction and up to 91% fewer acute pancreatitis events. The product will be available in the U.S. in July.

Biogen’s salanersen receives FDA Breakthrough Therapy Designation for SMA

FDA granted salanersen Breakthrough Therapy Designation for SMA based on Phase 1b exploratory data. Designation recognizes potential substantial improvement over available therapy and expedites development and review. Biogen is advancing three global Phase 3 studies (STELLAR-1, STELLAR-2, SOLAR) to support the asset’s regulatory path.

Biogen: Phase 2 CELIA topline shows tau reduction and cognitive benefit for diranersen

The Phase 2 CELIA study met key secondary and exploratory endpoints, demonstrating robust reductions in CSF tau and tau-PET pathology plus slowing of clinical decline across all doses, particularly the 60 mg q24w arm. Although the primary endpoint (dose-response on CDR-SB at Week 76) was not met, the data provide the first randomized Phase 2 evidence that a tau-directed ASO can impact both biomarkers and cognition in early Alzheimer’s disease, prompting Biogen to advance the program to registrational development.

Metagenomi: MGX-001 IND submission planned 4Q 2026

The company confirmed its lead hemophilia A program remains on track for an IND filing in the fourth quarter of 2026. This filing is intended to enable first-in-human studies in 2027, subject to regulatory clearance.

Ionis: olezarsen sNDA accepted for Priority Review, PDUFA June 30, 2026

FDA accepted the sNDA for olezarsen in sHTG under Priority Review with a PDUFA target action date of June 30, 2026. The company raised its peak net sales guidance for olezarsen in sHTG to >$3B. Launch preparations remain on track for a potential 2026 approval.

Ionis/GSK: bepirovirsen NDA accepted for Priority Review, PDUFA Oct 26 2026

FDA has accepted GSK’s NDA for bepirovirsen and granted Priority Review and Breakthrough Therapy Designation. The agency set a PDUFA target action date of October 26, 2026, shortening the review clock to six months.

Ionis: zilganersen PDUFA set for September 22, 2026

The FDA has set a PDUFA action date of September 22, 2026 for zilganersen under Priority Review. The press release also presents additional positive pivotal-study data across motor, patient-reported and clinician-reported endpoints that support the previously announced topline results.

Ionis: zilganersen NDA accepted for Priority Review; PDUFA 22 Sep 2026

FDA accepted the NDA for zilganersen and granted Priority Review, setting a PDUFA target action date of September 22, 2026. The application is supported by the pivotal Phase 1-3 study showing statistically significant gait-speed stabilization at week 61. This advances zilganersen toward a potential first approved therapy for Alexander disease.

Metagenomi completes pre-IND meeting for MGX-001; IND filing guided for 4Q 2026

Company completed pre-IND meeting for MGX-001 after NHP data showed curative FVIII activity. Remains on track for global regulatory submission including IND in 4Q 2026. Subject to clearance, clinical trials are planned to start in 2027.

Ionis submits sNDA for olezarsen in severe hypertriglyceridemia

Ionis submitted an sNDA for olezarsen in severe hypertriglyceridemia (sHTG) following positive Phase 3 CORE and CORE2 results. The submission positions the company for a potential second independent launch in 2026, pending FDA acceptance and review.