IONIS PHARMACEUTICALS, INC.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting progress with WAINUA U.S. launch, olezarsen FDA Priority Review (PDUFA Dec 19, 2024), and donidalorsen HAE NDA (PDUFA Aug 21, 2025). The company reaffirms 2024 P&L financial guidance and increases cash guidance to $2.2 billion. Key milestones include WAINUA sales generating $23 million and $44 million, positive CHMP opinion, UK approval, and Canada launch. Olezarsen and donidalorsen are positioned for potential launches in 2025. Ionis continues to advance its pipeline, including Phase 3 studies for olezarsen, donidalorsen, and ION582 for Angelman syndrome.

FDA accepted Ionis' NDA for donidalorsen, an RNA-targeted medicine for HAE prophylaxis, with an action date of August 21, 2025. Donidalorsen aims to reduce PKK production, preventing HAE attacks, supported by positive Phase 3 and Phase 2 OLE study results.

Ionis Pharmaceuticals announced FDA acceptance of donidalorsen NDA for hereditary angioedema, with PDUFA date set for August 21, 2025. Donidalorsen, targeting prekallikrein, aims to prevent HAE attacks and could be Ionis' second independent commercial launch.

Ionis Pharmaceuticals announced FDA acceptance of donidalorsen NDA for HAE prophylaxis, with a PDUFA date of August 21, 2025. Donidalorsen, targeting prekallikrein, showed significant HAE attack reduction in Phase 3 and 2 OLE studies.

Immunic's IDMC advises continuation of phase 3 ENSURE program for vidofludimus calcium in RMS; CHMP recommends approval for Ionis and AstraZeneca's eplontersen for ATTRv-PN; first patient dosed in ARTHEx's phase 1/2 trial for ATX-01 in DM1.

Roche reported strong Q3 and nine-month results, with sales up 9% at constant exchange rates. Sanofi is in exclusive talks with CD&R to sell a controlling stake in its consumer healthcare business, Opella. The FDA approved Pfizer’s RSV vaccine, Abrysvo, for adults under 60. Novo Nordisk’s cardiovascular outcomes study on oral semaglutide met its primary endpoint, and AstraZeneca’s Wainzua received a positive CHMP recommendation for EU approval.

CHMP recommends approval for Ionis’ and AstraZeneca’s eplontersen (Wainua) for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), based on phase 3 NEURO-TTRansform trial results. Eplontersen, a once-monthly RNA-targeted medicine, demonstrated benefits across ATTRv-PN spectrum and improved quality of life. The therapy is currently under study for amyloid transthyretin cardiomyopathy (ATTR-CM) in the CARDIO-TTRansform trial.

AstraZeneca and Ionis' Wainzua (eplontersen) recommended for approval by EU's CHMP for treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or 2 polyneuropathy. Wainzua demonstrated consistent and sustained benefit on serum transthyretin concentration, neuropathy impairment, and quality of life in NEURO-TTRansform Phase III trial. If approved, Wainzua would be the only self-administered monthly treatment for ATTRv-PN in the EU.