Biogen

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.

ASHG welcomed 2024–2026 Human Genetics Scholars, including Amber M. Abram, Kynon J. Benjamin, Razaq Durodoye, Tania Fabo, Gabrielle Ferra, JP Flores, Renée Fonseca, and Esteban Vazquez-Hidalgo, recognized for research and commitment to diversity, equity, and inclusion in human genetics and genomics.

Cognition Therapeutics presented data from its Phase II SHINE trial showing CT1812 significantly slowed cognitive decline in mild-to-moderate Alzheimer’s patients with low baseline plasma p-tau217 levels, with 95% slowing measured by ADAS-Cog 11 and 108% by MMSE. The company plans to discuss Phase III with the FDA in 2025, considering subgroup recruitment strategies and dosing, while facing funding challenges.

The Korean Dementia Association reassures on Leqembi's safety, noting rare side effects like brain hemorrhage. Phase 3 trial results show Leqembi delayed Alzheimer’s progression by 27%, leading to MFDS approval in Korea. Despite EU and Australia's refusal to approve due to ARIA concerns, Korean data reveals fewer side effects in Asians, with only 6.5% incidence of cerebral edema among Korean participants.

Despite approvals for Leqembi and Kisunla, attrition in Alzheimer’s and Parkinson’s pipelines persists, with Roche, Johnson & Johnson, Sage Therapeutics, and Otsuka discontinuing programs. Analysts attribute these decisions to clinical trial data and evolving commercial opportunities, noting the Alzheimer’s market is projected to be worth $15.5 billion by 2031, while Parkinson’s drugs are expected to reach $6.63 billion by 2029.

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Eisai and Biogen complete rolling submission to FDA for a subcutaneous maintenance dosing option of Leqembi, an Alzheimer's disease treatment. The BLA, granted Fast Track designation, builds on Clarity AD study data, aiming to enable weekly home or facility-administered SC doses. Results show Leqembi slows cognitive and functional decline by 27% over 18 months. Common adverse events include infusion reactions and amyloid-related imaging abnormalities. The SC formulation is designed to sustain effective drug levels post-plaque clearance, potentially improving convenience and reducing site visits.

Eisai and Biogen announce completion of BLA submission to FDA for lecanemab-irmb (LEQEMBI) subcutaneous autoinjector for weekly maintenance dosing in early Alzheimer's disease, granted Fast Track designation. LEQEMBI targets highly toxic protofibrils to sustain clearance of amyloid-beta plaque, potentially more convenient than IV administration.

Eisai and Biogen complete US BLA for subcutaneous Leqembi, aiming to replace IV version with once-weekly autoinjector after initial IV course. This could differentiate Leqembi from Eli Lilly's Kisunla and boost uptake. Lilly is developing IV and subcutaneous forms of its follow-up drug remternetug. FDA has fast-track status for subcutaneous Leqembi but hasn't accepted the filing yet. Eisai and Biogen also filed for a monthly IV maintenance dose. Leqembi is approved in several countries but faces re-examination in the EU. Biogen reports $67 million in Q3 sales, with plans to expand subcutaneous use into induction phase by Q1 2026.

Roche may seek accelerated U.S. approval for trontinemab, its Alzheimer’s drug, if it continues to show significant amyloid reduction. Trontinemab, designed to easily pass the blood-brain barrier, demonstrated quicker amyloid clearance than Kisunla in Phase 2 testing. Roche plans to expand the trial to 120 volunteers, with preliminary safety data showing lower ARIA rates than other drugs.