Discovery
Pre-clinical Phase I Phase II Phase III
Tor Vergata University of Rome
Metformin
Repurposed type 2 diabetes medication that modifies RNA
splicing, autophagy, insulin sensitivity or glycogen synthesis
DM1 Italy Clinical trials
AMO Pharma
Tideglusib
Glycogen synthase kinase 3 beta inhibiting small molecule
Congenital DM1 North America and UK Clinical trials
Osaka University Hospital
Erythromycin (MYD-0124)
Repurposed oral antibiotic to reduce RNA toxicity
DM1 Japan Clinical trials
Expansion Therapeutics
ERX-963
RNA inhibiting small molecule
DM1 USA Clinical trials
The Scripps Research Institute,
University of Florida and Iowa
State University
Cugamycin
Small molecule
DM1 Publication
Lupin Pharmaceuticals
Mexiletine
Repurposed antimyotonia small molecule
DM Company pipeline
Harmony Biosciences
Pitolisant
Repurposed anticataplectic small molecule
DM1 Company pipeline
University of Florida and Osaka
University
Erythromycin and pafuramidine
Repurposed oral antibiotic enhanced by small molecule
DM1 Publication
Pompeu Fabra University
Mirtazapine
Repurposed antidepressant small molecule
DM1 Publication
University of Valencia
Chloroquine
Repurposed antimalerial small molecule
DM1 Publication
Nexien BioPharma
NX04S1
Sublingual cannabinoid formulation
DM1 Company pipeline
Nexien BioPharma
NX04S2
Sublingual cannabinoid formulation
DM2 Company pipeline
Arthex Biotech
ARTHEx-01
MicroRNA small molecule
DM1 Company pipeline
Audentes Therapeutics
AT466
AAV-antisense gene therapy
DM1 Company pipeline
NeuBase Therapeutics
NT0200
Modular antisense peptide nucleic acid
DM1 Company pipeline
Enzerna Biosciences
ENZ-003
Artificial site-specific RNA endonucleases gene therapy
DM Company pipeline
Locana Biosciences
RNA-targeted gene therapy
DM1 Funding award
Avidity Biosciences
Antibody-oligonucleotide conjugate
DM1 Company pipeline
Dyne Therapeutics
Antibody-oligonucleotide conjugate
DM1 Company pipeline
MDUK Oxford Neuromuscular
Centre
Peptide-conjugated oligonucleotide
DM1 Funding award
University of Washington
RNAi gene therapy
DM1 Conference presentation
Massachusetts General Hospital
and Harvard Medical School
Antisense oligonucleotide
DM1 Conference presentation
University of Illinois at Urbana-
Champaign
Small molecule
DM1 Funding award
Genethon
CRISPR-Cas9
Gene editing
DM1 Publication
Vertex Pharmaceuticals and
CRISPR Therapeutics
CRISPR-Cas9
Gene editing
DM1 Company press release
University of Cardiff
CRISPR-Cas9
Gene editing
DM1 Funding award
AskBio
Gene therapy
DM Company press release
Amicus Therapeutics
Gene therapy
DM Company press release
Ionis Pharmaceuticals and
Biogen
Antisense oligonucleotide
DM1 Company pipeline
Triplet Therapeutics
Antisense oligonucleotides and small interfering RNAs
DM Company pipeline
Syros Pharmaceuticals
Small molecule
DM1 Company press release
Design Therapeutics
Small molecule
DM Company press release
Expansion Therapeutics
Undisclosed
DM2 Company pipeline
Fulcrum Therapeutics
Undisclosed
DM Company pipeline
Vertex Pharmaceuticals and
Affinia Therapeutics
AAV gene therapy
DM1 Press release
NeuBase Therapeutics
Undisclosed
DM2 Company pipeline
Locations of clinical trials are taken from publically available clinical trial registry entries
International non-proprietary names or developmental codes are provided in bold
We appreciate all those developing new treatment options for DM patients and would be very thankful for any researchers who may have been
missed, to update us on your research. Please contact Ben Porter at [email protected]
This information is accurate as of 19/05/20
Drug Development Pipeline
Myotonic Dystrophy Type 1 (DM1) and Myotonic Dystrophy Type 2 (DM2)
Stage of development
Company
Drug name and approach
Clinical trial location
Source of information