It was a battle of the companies with drugs for transthyretin-mediated amyloidosis on the first day of the J.P. Morgan 2026 Healthcare Conference with Alnylam Inc., Bridgebio Pharma Inc. and Pfizer Inc. all presenting at the annual kickoff conference. One company disclosed 2025 sales as well as 2026 revenue guidance, the second only looked back, while the third was too big to do either for a specific drug.
Peptidream Inc. has announced a preclinical development milestone in its collaboration with Alnylam Pharmaceuticals Inc. under their siRNA conjugate discovery collaboration.
Amid the increasingly competitive myasthenia gravis drug development space, siRNA candidate cemdisiran met phase III endpoints, with the monotherapy showing numerically higher results than a combination product. Regeneron Pharmaceuticals Inc., which has a worldwide license to cemdisiran from Alnylam Pharmaceuticals Inc., plans to file for U.S. approval in generalized myasthenia gravis, a rare and chronic autoimmune disease leading to life-threatening muscle weakness, in the first quarter of 2026.
The BioWorld Biopharmaceutical Index extended its downward trend over April and May, closing the latter month down 6.47% after reaching a peak gain of 9.64% at the end of February. The Nasdaq Biotechnology Index followed a similar trajectory, finishing May with a 5.37% decline. Meanwhile, the Dow Jones Industrial Average dipped through April but rebounded slightly in May, ending the month down 0.64%.
Moderna Inc. once again emerged the winner in a court skirmish over claims that its COVID-19 vaccine infringed two Alnylam Pharmaceuticals Inc. patents. The U.S. Court of Appeals for the Federal Circuit issued a precedential opinion May 4, agreeing with a federal district court in Delaware that Moderna didn’t infringe the patents. For both courts, the decision was based on a single issue of claim construction.
A development deal with Biogen Inc. could eventually bring City Therapeutics Inc. about $1 billion in milestone payments. It’s a step in the direction the company had been going: looking for the right partners, including those from big pharma. Privately held City Therapeutics is getting $46 million in the deal. That includes $16 million as an up-front payment and an investment of $30 million in exchange for a City Therapeutics convertible note.
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors.
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.
The BioWorld Biopharmaceutical Index (BBI) ended 2024 down 2.24%, despite hitting a peak of 25.19% in late August. While it remained in positive territory through November, up 3.58%, the steady decline through the later months of the year pushed the index into the red by December’s close.
