All articles tagged with #phase iii
Despite the FDA's reluctance to pull fully-approved drugs from the market, the significant failure of Amylyx's Phase III data in ALS, with a failed primary endpoint and missed secondary endpoints, may leave the company with few options, especially considering past promises to consider withdrawing the drug if the final-stage study didn't succeed.
Roche has announced the removal of RG6358, or SPK-8016, from its gene therapy pipeline for hemophilia A, while initiating a Phase III trial for RG6357, or SPK-8011. The company plans to open enrollment for the study, named Keystone1, later this year.
FibroGen's pamrevlumab failed in a Phase III study for idiopathic pulmonary fibrosis, marking the company's third late-stage miss in two months. The drug was not better than placebo at a lung function test and did not delay disease progression. However, it was generally safe and well-tolerated.
AstraZeneca and Ionis have announced positive Phase III results for eplontersen, a drug for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), a fatal neurodegenerative disease. Patients taking eplontersen recorded a 0.28-point average increase from baseline compared to a 25.06-point average increase in the placebo group. The drug is expected to generate over $3 billion in sales.