Full data from the Helios-A trial support vutrisiran’s profile as a more convenient Onpattro, though that’s probably as good as it gets.
If drugs acting on amyloid and tau can defy the sector’s history of failure, the testing landscape will change dramatically.
Success in a study in essential tremor is marred by a high rate of adverse events and patient dropouts.
Key catalysts approach for Orphazyme, Gemini and Immutep.
Concerns about a rise in complete response letters in 2020 come to naught, but this might not reassure drug developers.
Prilenia’s pridopidine is the last big late-stage hope in the disorder, but it already failed phase 2, and phase 3 under the name Huntexil.