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 IAu cours d’un essai clinique de phase I un médicament dont l’intérêt thérapeutique a été montré sur des modèles animaux et/ou cellulaires (essais précliniques) est administré pour la première fois à un petit groupe de volontaires sains, plus rarement à des malades, afin d’évaluer leur tolérance à la substance en fonction de la dose (Comment le futur traitement est-il absorbé et éliminé ? Comment se fait sa répartition dans les organes ? Est-il toxique et à quelles doses ? Existe-t-il des effets secondaires ?)./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 myhttps://pubmed.ncbi.nlm.nih.gov/36804094/otonic 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