Otsuka and Ionis Report P-III (FUSION) Trial Results for Ulefnersen in FUS-ALS

Shots: The P-III (FUSION) trial met its 1EP with ulefnersen, showing improvements in functional impairment & survival in a joint rank analysis of time to death or permanent ventilation, time to rescue, & change in ALSFRS-R from baseline to Day 505 vs PBO in pts with ALS caused by mutations in the fused in sarcoma…

ByRidhi RastogiSeptember 23, 2026

NEWS

Ionis and Roche Report Interim P-III (IMAgINATION) Trial Data on Sefaxersen in IgA Nephropathy (IgAN)

Shots: Ionis & Roche have reported interim P-III (IMAgINATION) trial results assessing sefaxersen (QM, SC) in 459 adults with IgAN for 105wks., after which eligible pts may switch to open-label treatment IMAgINATION met its 1EP, showing improvements in proteinuria reduction at 37wks., as measured by 24hr. UPCR; data to be presented in future & shared with…

ByRidhi RastogiSeptember 23, 2026

NEWS

Ionis’ Zanvastro (Zilganersen) Receives US FDA Approval for Alexander Disease (AxD)

Shots: FDA has approved Zanvastro (50mg, Q3M, intrathecal injection) for the treatment of AxD in pediatric & adult pts, with launch expected in the coming wks. FDA also granted Ionis a Rare Pediatric Disease PRV Approval was backed by the Phase I-III trial assessing Zanvastro (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.) for…

ByRidhi RastogiSeptember 4, 2026

NEWS

Ionis Completes Patient Enrollment in Cohort 1 of P-III (REVEAL) Study of Obudanersen in Angelman syndrome (AS)

Shots: Ionis has completed enrollment in Cohort 1 (n=136; 2 to <18yrs.) of the global P-III (REVEAL) study assessing obudanersen (ION582) in pts with AS with genetic confirmation of either a UBE3A deletion or UBE3A mutation The Cohort 2 comprises adult pts (18 to ≤50yrs.) living with AS, with enrollment expected to be completed in…

ByRidhi RastogiJuly 7, 2026

NEWS

Ionis’ Tryngolza Receives the US FDA Approval for Patients with Severe Hypertriglyceridemia

Shots: The US FDA has approved Tryngolza (olezarsen; 50 or 80mg, QM) as an adjunct to diet to reduce triglycerides (TG) & the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG); commercially available in Jul 2026 Approval was based on the P-III (CORE: n=617 & CORE2: n=446) trials assessing Tryngolza (SC) vs PBO…

ByRidhi RastogiJune 26, 2026

NEWS

Ionis Inks ex-US Deal with Recordati for Zilganersen in Alexander Disease

Shots: Ionis has granted Recordati exclusive rights to develop & commercialize zilganersen for Alexander disease outside the US, while Ionis retains US commercialization rights & will continue to lead global development Ionis will receive $30M upfront & additional milestone payments, with tiered royalties of up to the mid-20% range on annual net sales. Recordati will…

ByRidhi RastogiJune 25, 2026

NEWS

Biogen Reports P-II (CELIA) Trial Results on Diranersen in Early Alzheimer’s Disease

Shots: Biogen has reported the global P-II (CELIA) trial data assessing diranersen [60mg (Q24W), 115mg (Q24W), or 115mg (Q12W); intrathecal) vs PBO over 76wks. in 416 pts with mild cognitive impairment due to Alzheimer’s disease (AD) or mild AD dementia Although CELIA did not meet its 1EP of dose response on CDR-SB change from baseline…

ByRidhi RastogiMay 15, 2026

NEWS

Ionis’ Zilganersen Receives US FDA Priority Review for Alexander Disease

Shots: The US FDA has accepted NDA & granted priority review to zilganersen for the treatment of Alexander disease (AxD), with PDUFA target action date of Sep 22, 2026 NDA was backed by the P-III trial assessing zilganersen (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.) for 60wks., followed by an open label…

ByRidhi RastogiMarch 25, 2026

NEWS

Ionis’ Olezarsen sNDA Secures the US FDA Priority Review for Severe Hypertriglyceridemia

Shots: The US FDA has accepted sNDA & granted priority review to olezarsen for the treatment of severe hypertriglyceridemia (sHTG), with a PDUFA action date of Jun 30, 2026 sNDA was backed by the P-III (CORE: n=617 & CORE2: n=446) trials assessing olezarsen (50 or 80mg, SC, Q4W) vs PBO for 12mos. in sHTG adults…

ByRidhi RastogiFebruary 27, 2026

NEWS

The EC Approves Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Shots: The EC has approved Dawnzera (SC autoinjector; Q4W/Q8W) for the routine prevention of HAE attacks in pts (≥12yrs.) Approval was based on P-III (OASIS-HAE) trial & OASISplus study, showing improvements across multiple domains, incl. sustained reduction in mean monthly HAE attack rate, with 94% overall mean monthly reduction at 1yr. in the OASISplus OLE study Approval…

ByRidhi RastogiJanuary 22, 2026

Otsuka and Ionis Report P-III (FUSION) Trial Results for Ulefnersen in FUS-ALS

Ionis and Roche Report Interim P-III (IMAgINATION) Trial Data on Sefaxersen in IgA Nephropathy (IgAN)

Ionis’ Zanvastro (Zilganersen) Receives US FDA Approval for Alexander Disease (AxD)

Ionis Completes Patient Enrollment in Cohort 1 of P-III (REVEAL) Study of Obudanersen in Angelman syndrome (AS)

Ionis’ Tryngolza Receives the US FDA Approval for Patients with Severe Hypertriglyceridemia

Ionis Inks ex-US Deal with Recordati for Zilganersen in Alexander Disease

Biogen Reports P-II (CELIA) Trial Results on Diranersen in Early Alzheimer’s Disease

Ionis’ Zilganersen Receives US FDA Priority Review for Alexander Disease

Ionis’ Olezarsen sNDA Secures the US FDA Priority Review for Severe Hypertriglyceridemia

The EC Approves Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

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