Annexon Touts Final Huntington's Trial Data Confirming No New Safety Issues

Annexon Inc ANNX shared final data from the Phase 2 trial for ANX005 in patients with or at risk for Huntington's disease, which causes a progressive breakdown of nerve cells in the brain. 

• The 28 patients were dosed for six months and followed for three more. The goal of the study was safety and tolerability.
• ANX005 demonstrated full and durable C1q target inhibition as measured by two biomarkers.
• Results showed stabilization of disease progression in the overall population for the entire nine months of the study.
• Related: Annexon's Interim Mid-Stage Results On Huntington's Disease Candidate Fails To Cheer.
• Three dropouts were potentially linked to the treatment. The adverse events experienced by the three patients included a case of systemic lupus and idiopathic pneumonitis, a type of noninfectious pneumonia. But no serious cases of infection or death were reported.
• According to the final data release, the case of lupus resolved after treatment stopped, while the idiopathic pneumonitis event "improved" once dosing ceased. 
• The third patient experienced hemolytic anemia, which was asymptomatic and also resolved with the end of treatment. 
• Annexon plans to talk with regulators in the U.S. and the EU to discuss the design of a well-controlled confirmatory trial.
• ANX005 is also tested for amyotrophic lateral sclerosis, Guillain Barré syndrome, and warm autoimmune hemolytic anemia.
• Price Action: ANNX shares are up 16.3% at $3.78 during the market session on the last check Tuesday.