ION582

Ionis Pharmaceuticals' shares rose 5% after FDA approved its RNA-targeted therapy, Tryngolza, for familial chylomicronemia syndrome (FCS), marking the first treatment for FCS in the U.S. The approval is supported by phase III BALANCE study results showing significant triglyceride-lowering and reduced acute pancreatitis attacks. Tryngolza is Ionis' first wholly-owned drug and independent commercial launch, expected by year-end. The company is also evaluating Tryngolza for severe hypertriglyceridemia and has collaborations with major drugmakers for various treatments.

Ionis Pharmaceuticals plans a phase 3 trial for ION582, targeting Angelman syndrome (AS), with enrollment starting in H1 2025. The placebo-controlled study will assess ION582's efficacy and safety in 200 AS patients with maternal UBE3A gene issues over 12 months, using Bayley-4 for expressive communication as the primary endpoint. ION582, an antisense oligonucleotide, aims to increase UBE3A protein production, addressing AS's underlying pathology. Positive phase 2 results from HALOS support ION582's potential.

Ionis Pharmaceuticals (IONS) reported a narrower-than-expected Q3 2024 loss of 95 cents per share, with total revenues of $134 million, beating estimates. The company's shares have declined 22.9% YTD. Ionis receives royalties from Biogen for Spinraza and co-markets Qalsody with Biogen. Wainua, co-marketed with AstraZeneca, was approved in the U.S. and the UK, generating $30 million in milestone payments. Commercial revenues were $76 million, missing estimates. Ionis maintained its 2024 financial guidance, expecting total revenues over $575 million and an adjusted operating loss of less than $475 million.

Ionis Pharmaceuticals plans Phase III REVEAL trial of ION582 for Angelman syndrome, enrolling 200 with UBE3A mutation/deletion. Primary endpoint is expressive communication improvement via Bayley-4. Trial includes long-term extension phase and assesses multiple secondary endpoints. CEO Brett Monia anticipates trial start in H1 2025.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting progress with WAINUA U.S. launch, olezarsen FDA action date in December 2024, and donidalorsen HAE PDUFA date in August 2025. The company is on track to achieve 2024 financial guidance and increased cash guidance to $2.2 billion. Key pipeline milestones include positive CHMP opinion for WAINUA in the UK, QALSODY approval in China, and ongoing pivotal development programs for olezarsen and donidalorsen.

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting successful U.S. launch of WAINUA, upcoming FDA action dates for olezarsen and donidalorsen, and reaffirmed 2024 P&L financial guidance with increased cash guidance to $2.2 billion.

Ionis Pharmaceuticals announces Phase 3 study design for ION582, following FDA alignment, with Bayley-4 expressive communication as the primary endpoint. The study is planned to begin in H1 2025 and will enroll 200 AS patients with maternal UBE3A gene deletion or mutation. Ionis will provide an update on the Phase 3 program at the FAST Global Science Summit in November.