As many of the young people and families we support are affected by biliary atresia, we’d like to update you on two pieces of research in this area:
Ipsen evaluation of Bylvay
Ipsen have announced the results of their Phase III BOLD trial evaluating Bylvay (odevixibat) as a potential treatment for biliary atresia. The trial was investigating the potential of using Bylvay to improve native liver survival post Kasai.
Unfortunately, the trial did not meet the primary endpoint of improvement in native liver survival vs placebo. Lead investigator Dr Saul J. Karpen commented: “As the first global Phase III trial in biliary atresia, BOLD has generated the most comprehensive dataset ever assembled in this disease. Although the study did not meet its primary endpoint, the commitment of participating children and families has produced valuable insights that will deepen our understanding of biliary atresia and provide a crucial basis for future research and patient care.”
More details on the trial are available here Ipsen provides update on Phase III BOLD trial in biliary atresia
ProQR announces positive Phase 1 target engagement data for AX-810
On a more positive note, data from ProQR’S Phase 1 trial of AX-0810 in healthy volunteers has demonstrated dose-dependent target engagement on all key biomarkers.
AX-0810 has demonstrated up to 8-fold change in total bile acids in serum, exceeding the 2-fold threshold identified by the Company as a meaningful indicator of NTCP modulation. The NTCP biomarker findings support advancement of next-generation candidate AX-0811 and future clinical studies in biliary atresia.
More information on the AS-0810 trial is available here
Whilst both trials demonstrate that there is a long way to go, we’re pleased to see that there is commitment from major pharma companies to advancing understanding and treatment of biliary atresia. We shall continue to keep our families update on future developments.
More information on biliary atresia is here .
