The pharmaceutical company Arrowhead Therapeutics has begun recruitment for their phase 1 randomised placebo-controlled trial of ARO-ATXN2 in adults with SCA2. The programme is a partnership with the pharmaceutical company Sarepta Therapeutics.
The trial has begun recruitment in Australia, New Zealand, Canada and Taiwan. The company also plans to open sites in a number of other countries including in Newcastle and London in the UK. We will provide updates on when the UK sites will open, which is expected to be in late 2025.
ARO-ATXN2 is designed to silence the expression of the toxic Ataxin 2 protein produced in SCA2. The company is exploring the potential of trialling a version of this drug in SCA1 and SCA3, with plans to initiate trials in 2026.
The trial will investigate the safety and tolerability of the drug, as well as identify any side effects. It will also study whether the drug decreases levels of Ataxin 2, and whether it has any effect on other markers of nerve cell degeneration.
Read more about the trial and its eligibility criteria here and here.
