Publication of results from the IONIS-DMPKRx trial in DM1

IONIS-DMPKRx, also known as baliforsen is an antisense oligonucleotide developed by IONIS Pharmaceuticals in DM1. It was tested in a Phase I/II clinical trial in 48 patients between 2014 and 2016. The results were published in the Lancet Neurology journal in March 2023, confirming the initial results announced by press release in 2017:

  • The product was well tolerated.
  • However, its efficacy was too low to consider further development.

Other antisense oligonucleotides are being developed for Steinert’s disease, based in particular on the lessons learned from this trial.


Antisense oligonucleotide targeting DMPK in patients with my dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial. Thornton CA, Moxley RT 3rd, Eichinger K et al. Lancet Neurol. 2023 Mar