Biogen

Seven genetic variants—LRRK2, GBA1, SNCA, PINK1, PARK7, PRKN, and VPS35—account for 13% of Parkinson's disease cases. Genetic testing should be offered to explain causes and encourage clinical trial participation.

Biogen Canada's omaveloxolone, a potential treatment for Friedreich's ataxia, has been accepted for priority review by Health Canada, offering hope for patients with this rare, progressive neuromuscular disease.

Higher dose nusinersen (Spinraza) met primary end point in phase 2/3 DEVOTE study, showing significant motor function improvement in treatment-naïve SMA infants compared to sham control from ENDEAR study. The higher dose regimen, 2 separate 50-mg doses followed by 28-mg every 4 months, outperformed the approved 12-mg regimen on key measures and was generally well-tolerated.

DelveInsight's 'Spinal Muscular Atrophy Pipeline Insight, 2024' report details 18+ companies developing 20+ therapies, including key players like Scholar Rock, Biogen, and Roche. The report covers clinical trials, MOA, ROA, recent developments, and emerging therapies like ACTX-401, Apitegromab, and GYM329, expected to impact the SMA market.

The FDA approved Roche's Ocrevus Zunovo, an under-the-skin injection for multiple sclerosis, expanding treatment options to centers unable to administer the IV version. The 10-minute injection, given twice a year, targets CD20-positive B cells and is based on a study showing no significant difference in Ocrevus levels when administered subcutaneously.

Roche's subcutaneous Tecentriq approved for cancer treatment; Gilead's twice-yearly HIV shot reduces infection risk by 96%; Biogen appoints Lloyd Minor and Menelas Pangalos as independent directors; Sanofi secures rights to a rare cancer radiopharmaceutical; Eli Lilly to expand Irish manufacturing for Alzheimer's drug Kisunla.

Large drug-maker stocks surged, driven by FDA approvals and pipeline innovations. Eli Lilly, Roche, Novartis, and Pfizer are top picks for long-term gains. Eli Lilly's tirzepatide drugs and Novo Nordisk's semaglutide drugs are top sellers. Despite M&A scrutiny, activities remained strong. Large drug-makers offer robust revenue streams and profitability.

Ractigen Therapeutics receives FDA orphan drug designation for RAG-18, a saRNA for DMD/Becker muscular dystrophy. Biogen's nusinersen shows higher dose efficacy in SMA. Cartesian Therapeutics initiates Descartes-15 CAR-T phase 1 trial for r/r multiple myeloma. Imugene's azer-cel CAR-T produces complete responses in r/r diffuse large B cell lymphoma. Novartis licenses Voyager Therapeutics' capsid for neurological target. BridgeBio Pharma's BBP-812 receives FDA RMAT designation for Canavan disease. Genprex plans to spin off its diabetes program into NewCo. NCI awards TransCode $2 million for TTX-MC138 RNA therapy in advanced solid tumors.

Regeneron Pharmaceuticals' PE, PB, and PS ratios are low, suggesting potential undervaluation. However, its low ROE indicates lower profitability. High EBITDA and gross profit levels indicate strong financial performance, but low revenue growth may be a concern.