Discovery
Pre-clinical Phase I Phase II Phase III
Lupin Pharmaceuticals
Mexiletine (NaMuscla)
Repurposed antimyotonia small molecule
DM (≥ aged 6
years)
Not listed Clinical trials
Tor Vergata University of Rome
Metformin
Repurposed type 2 diabetes compound
DM1 Italy Clinical trials
AMO Pharma
Tideglusib
Small molecule
Congenital DM1 North America and UK Clinical trials
Osaka University Hospital
Erythromycin (MYD-0124)
Repurposed antibiotic compound
DM1 Japan Clinical trials
Expansion Therapeutics
ERX-963
Small molecule
DM1 USA Clinical trials
MyoGem
MYODM (caffeine and theobromine)
Food supplement
DM1 Spain No study phase listed on Clinicaltrials.gov Clinical trials
The Scripps Research Institute,
University of Florida and Iowa State
University
Cugamycin
Small molecule
DM1 USA Publication
Harmony Biosciences
Pitolisant
Repurposed anticataplectic small molecule
DM1 USA Company pipeline
University of Florida and Osaka
University
Erythromycin and pafuramidine
Repurposed oral antibiotic enhanced by small molecule
DM1 USA/Japan Publication
Pompeu Fabra University
Mirtazapine
Repurposed antidepressant small molecule
DM1 Italy Publication
University of Valencia
Chloroquine
Repurposed antimalerial small molecule
DM1 Spain Publication
Arthex Biotech
ARTHEx-01
MicroRNA small molecule
DM1 Spain Company pipeline
Audentes Therapeutics
AT466
AAV-antisense gene therapy
DM1 USA Company pipeline
NeuBase Therapeutics
NT0200
Modular antisense peptide nucleic acid
DM1 USA Company pipeline
Enzerna Biosciences
ENZ-003
Artificial site-specific RNA endonucleases gene therapy
DM USA Company pipeline
Osaka University
JM642
Small molecule
DM1 Japan Publication
Genethon
CRISPR-Cas9
Gene editing
DM1 France Publication
PepGen
PGN-EDODM1
Peptide conjugated oligonucleotide
DM1 UK Conference presentation
Avidity Biosciences
AOC 1001
Antibody-oligonucleotide conjugate
DM1 USA Company pipeline
Nexien BioPharma
Cannabidiol and tetrahydrocannabinol
DM USA Company filing
Locana Biosciences
RNA-targeted gene therapy
DM1 USA Conference presentation
Dyne Therapeutics
Antibody-oligonucleotide conjugate
DM1 USA Company pipeline
University of Washington
RNAi gene therapy
DM1 USA Conference presentation
Massachusetts General Hospital
and Harvard Medical School
Antisense oligonucleotide
DM1 USA Conference presentation
University of Alabama and
University of Florida
Ascochlorin
Antibiotic compound DM1 USA Publication
IRCCS Fondazione Don Carlo
Gnocchi and Università Cattolica
del Sacro Cuore
Resveratrol
Natural compound
DM1 Italy Publication
The First Affiliated Hospital of
Chongqing Medical University
Rapamycin
Repurposed antibiotic compound
DM1 China Publication
Vertex Pharmaceuticals and
CRISPR Therapeutics
CRISPR-Cas9
Gene editing
DM1 USA Press release
University of Cardiff
CRISPR-Cas9
Gene editing
DM1 UK Funding award
Houston Methodist Research
Institute
LshCas13a
Gene editing
DM1 USA Publication
Dewpoint Therapeutics and Pfizer
Biomolecular condensates
DM1 USA Press release
Ramon Llull University
Small molecule
DM1 USA Publication
LoQus23 Therapeutics
Small molecule
DM UK Other
The Scripps Research Institute and
Expansion Therapeutics
Small molecule
DM1 USA Publication
University of Illinois at Urbana-
Champaign
Small molecule
DM1 USA Funding award
Faze Medicines
Small molecule
DM1 USA Company pipeline
AskBio
Gene therapy
DM USA Press release
Amicus Therapeutics
Gene therapy
DM USA Company pipeline
Ionis Pharmaceuticals and Biogen
Antisense oligonucleotide
DM1 USA Company pipeline
Triplet Therapeutics
Antisense oligonucleotides and small interfering RNAs
DM USA Company pipeline
Syros Pharmaceuticals
Small molecule
DM1 USA Company pipeline
Design Therapeutics
Small molecule
DM USA Company pipeline
Expansion Therapeutics
Undisclosed
DM2 USA Company pipeline
Fulcrum Therapeutics
Undisclosed
DM USA Company pipeline
Vertex Pharmaceuticals and
Affinia Therapeutics
AAV gene therapy DM1
USA Press release
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 ben.porter@newcastle.ac.uk
This information is accurate as of 08/03/21
Drug Development Pipeline for Myotonic Dystrophy Type 1 (DM1) and
Myotonic Dystrophy Type 2 (DM2)
Stage of development
Company
Drug name and approach
Location of clinical
trial/developer
Source of information
Condition
Study complete
