Ionis Pharmaceuticals

Ionis Pharmaceuticals reports Q3 2024 financial results, highlighting successful U.S. launch of WAINUA, UK approval, and positive CHMP opinion. Key milestones include olezarsen FCS PDUFA on December 19, 2024, and donidalorsen HAE PDUFA on August 21, 2025. The company is on track to achieve 2024 financial guidance and increased cash guidance. Financial highlights show revenue from diverse sources, including new royalty revenue from WAINUA. Operating expenses increased as planned, reflecting investments in late-stage development and commercialization efforts. Ionis' cash, cash equivalents, and short-term investments increased to $2.5 billion, with plans to deploy capital toward growth opportunities.

Ionis Pharmaceuticals announces successful Phase 2 discussion with FDA, aligning on Phase 3 design for ION582, targeting Angelman syndrome. Bayley-4 expressive communication selected as primary endpoint, with Phase 3 study initiation planned for H1 2025. Ionis to update ION582 program at FAST Global Science Summit in November.

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

Viatris (VTRS) to report Q3 results on Nov. 7, 2024; Zacks Consensus Estimate for revenues at $3.68B and earnings at $0.68/share. Developed Markets expected to contribute $2.2B, Emerging Markets $567M, JANZ $396M, and Greater China $562M. Brand business growth in Emerging Markets, JANZ, and Greater China offset by unfavorable dynamics in North America and price regulations in Japan/Australia. Generics business improved due to new product launches and strong performance in Europe. Operating expenses likely increased due to SG&A investments and R&D progress.

FDA accepts Ionis Pharmaceuticals' donidalorsen NDA for hereditary angioedema prophylaxis, with a PDUFA date of August 21, 2025. Donidalorsen aims to reduce prekallikrein production, preventing HAE attacks. Positive 3-year data from phase 2 and phase 3 OASIS-HAE studies show significant improvements in quality of life and disease control, with no serious adverse events.

Goldman Sachs maintains Buy rating on Biogen with $290.00 price target, citing Q3 revenue meeting expectations due to Leqembi's performance. The firm observes Biogen's pipeline, including felzartamab and dapirolizumab, and notes potential growth from business development. Biogen's market cap is $26.39B, with a P/E ratio of 22.76, and is trading near a 52-week low.

A single dose of CRISPR-based NTLA-2002 reduced angioedema attacks by 75-77% in patients with hereditary angioedema, with 80% of patients in the 50 mg group experiencing complete response.

The EU's CHMP recommended AstraZeneca and Ionis Pharmaceuticals' Wainzua for ATTRv-PN, a progressive disease causing nerve damage. If approved, Wainzua would be the only self-administered monthly treatment in the EU, offering consistent TTR suppression and improved quality of life. The recommendation is based on the NEURO-TTRansform Phase III trial results.