Myotonic Dystrophy (DM1 & DM2) | Global Pipeline

Last Update: Nov 25, 2022

Purchase the Myotonic Dystrophy database for this data in an Excel spreadsheet, plus added notes.


Company Company Candidate MOA Indications Stage Trial Trial Initiation Status
Lupin PharmaceuticalsPublicMexiletineRepurposed Na+ channel blockerDM1 and DM2 ages 6-18IIINCT04624750Sep 2021Recruiting
Lupin PharmaceuticalsPublicMexiletineRepurposed Na+ channel blockerDM1 and DM2 ages 18+IIINCT04700046-Not yet recruiting
AMO PharmaPrivateTideglusib (AMO-02)GSK3ß inhibitor small moleculeCongenital DM1IIINCT03692312Mar 2021Recruiting
Hôpitaux de ParisN/AMetforminMultiple (repurposed)DM1IIINCT05532813Sep 2022Not yet recruiting
Harmony BiosciencesPublicPitolisantRepurposed H3 receptor antagonistDM1 (adults, excessive daytime sleepiness)IINCT04886518Jun 2021Recruiting
Osaka University HospitalN/AErythromycinRepurposed antibiotic (MBNL1 activity correction)DM1 (adults)IIjRCT2051190069Nov 2019Completed
Avidity BiosciencesPublicAOC 1001TfR1-targeted antibody (mAb) conjugated to siRNA targeting DMPKDM1I/IINCT05027269Oct 2021Partial clinical hold
Dyne TherapeuticsPublicDYNE-101TfR1-targeted antibody (Fab) linked to ASO targeting DMPKDM1I/IINCT05481879Aug 2022Recruiting
Expansion TherapeuticsPrivateERX-963 (Flumazenil)GABA receptor antagonistDM1 (adults, excessive daytime sleepiness)INCT03959189Jun 2019Completed
NeuBase TherapeuticsPublicNT-0200Modular antisense peptide nucleic acid (ASO)DM1IND---
Entrada TherapeuticsPrivateENTR-701Phosphorodiamidate morpholino oligomer conjugateDM1IND-enabling---
PepGenPublicPGN-EDODM1Peptide-conjugated ASODM1IND-enabling---
ARTHEx BiotechPrivateATX-01Anti-microRNA (single stranded oligonucleotide), MBNL upregulationDM1Preclin---
Juvena TherapeuticsPrivateJUV-161Recombinant hESC-derived fusion protein (via SC injection)DM1Preclin---
Astellas (Audentes Therapeutics)PublicAT466AAV vector delivering ASODM1Preclin---
Enzerna BiosciencesPrivateENZ-003AAV-delivered RNA endonucleases gene therapyDMPreclin---
Ionis PharmaceuticalsPublicIONIS-877864C16-HA conjugated ASODM1Preclin---
Locana BiosciencesPrivate-AAV vector encoding PIN-dCas9DM1Preclin---
Design TherapeuticsPublic-CTG repeat binding heterobifunctional small moleculeDMPreclin---
Vertex & CRISPR TherapeuticsPublic-CRISPR-CAS9 gene editingDM1Disc---
Vertex & Affinia TherapeuticsPublic-AAV based gene therapyDM1Disc---
Prime MedicinePublic-Viral/non-viral delivered gene therapyDM1Disc---
Dewpoint Therapeutics & PfizerPrivate-Biomolecular condensatesDM1R&D---
LoQus23 TherapeuticsPrivate-Small moleculeDMR&D---
Faze MedicinesPrivate-Small moleculeDM1R&D---
Bayer (AskBio)Public-AAV gene therapyDMR&D---
Amicus TherapeuticsPublic-AAV gene therapyDMR&D---
Triplet TherapeuticsPrivate-Antisense oligonucleotides and small interfering RNAsDMR&D---
Syros PharmaceuticalsPublic-Small moleculeDM1R&D---
Expansion TherapeuticsPrivate-RNA inhibiting small moleculeDM1R&D---
Ionis Pharmaceuticals & BiogenPublic-Ligand-conjugated antisense oligonucleotide (ASO)DM1R&D---
Nexion BiopharmaPrivate-CannabinoidsDM1R&D---
Scripps+Expansion TherapeuticsPrivate-Small molecule binding r(CUG)expDM1Lit---