Last Update: June 18, 2023
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 Pharmaceuticals | Public | Mexiletine | Repurposed Na+ channel blocker | DM1 and DM2 ages 6-18 | III | NCT04624750 | Sep 2021 | Recruiting |
| Lupin Pharmaceuticals | Public | Mexiletine | Repurposed Na+ channel blocker | DM1 and DM2 ages 18+ | III | NCT04700046 | - | Not yet recruiting |
| AMO Pharma | Private | Tideglusib (AMO-02) | GSK3ß inhibitor small molecule | Congenital DM1 | III | NCT03692312 | Mar 2021 | Recruiting |
| Hôpitaux de Paris | N/A | Metformin | Multiple (repurposed) | DM1 | III | NCT05532813 | Sep 2022 | Not yet recruiting |
| Harmony Biosciences | Public | Pitolisant | Repurposed H3 receptor antagonist | DM1 (adults, excessive daytime sleepiness) | II | NCT04886518 | Jun 2021 | Recruiting |
| Osaka University Hospital | N/A | Erythromycin | Repurposed antibiotic (MBNL1 activity correction) | DM1 (adults) | II | jRCT2051190069 | Nov 2019 | Completed |
| Avidity Biosciences | Public | AOC 1001 | TfR1-targeted antibody (mAb) conjugated to siRNA targeting DMPK | DM1 | I/II | NCT05027269 | Oct 2021 | Recruiting |
| Dyne Therapeutics | Public | DYNE-101 | TfR1-targeted antibody (Fab) linked to ASO targeting DMPK | DM1 | I/II | NCT05481879 | Aug 2022 | Recruiting |
| Expansion Therapeutics | Private | ERX-963 (Flumazenil) | GABA receptor antagonist | DM1 (adults, excessive daytime sleepiness) | I | NCT03959189 | Jun 2019 | Completed |
| NeuBase Therapeutics | Public | NT-0200 | Modular antisense peptide nucleic acid (ASO) | DM1 | IND | - | - | - |
| Entrada Therapeutics & Vertex | Private | ENTR-701 | Phosphorodiamidate morpholino oligomer conjugate | DM1 | IND-enabling | - | - | - |
| PepGen | Public | PGN-EDODM1 | Peptide-conjugated ASO | DM1 | IND-enabling | - | - | - |
| ARTHEx Biotech | Private | ATX-01 | Anti-microRNA (single stranded oligonucleotide), MBNL upregulation | DM1 | IND-enabling | - | - | - |
| Juvena Therapeutics | Private | JUV-161 | Recombinant hESC-derived fusion protein (via SC injection) | DM1 | Preclin | - | - | - |
| Astellas (Audentes Therapeutics) | Public | AT466 | AAV vector delivering ASO | DM1 | Preclin | - | - | - |
| Enzerna Biosciences | Private | ENZ-003 | AAV-delivered RNA endonucleases gene therapy | DM | Preclin | - | - | - |
| Ionis Pharmaceuticals | Public | IONIS-877864 | C16-HA conjugated ASO | DM1 | Preclin | - | - | - |
| Locana Biosciences | Private | - | AAV vector encoding PIN-dCas9 | DM1 | Preclin | - | - | - |
| Design Therapeutics | Public | - | CTG repeat binding heterobifunctional small molecule | DM | Preclin | - | - | - |
| Vertex & CRISPR Therapeutics | Public | - | CRISPR-CAS9 gene editing | DM1 | Disc | - | - | - |
| Vertex & Affinia Therapeutics | Public | - | AAV based gene therapy | DM1 | Disc | - | - | - |
| Prime Medicine | Public | - | Viral/non-viral delivered gene therapy | DM1 | Disc | - | - | - |
| Dewpoint Therapeutics & Pfizer | Private | - | Biomolecular condensates | DM1 | R&D | - | - | - |
| LoQus23 Therapeutics | Private | - | Small molecule | DM | R&D | - | - | - |
| Faze Medicines | Private | - | Small molecule | DM1 | R&D | - | - | - |
| Bayer (AskBio) | Public | - | AAV gene therapy | DM | R&D | - | - | - |
| Amicus Therapeutics | Public | - | AAV gene therapy | DM | R&D | - | - | - |
| Triplet Therapeutics | Private | - | Antisense oligonucleotides and small interfering RNAs | DM | R&D | - | - | - |
| Syros Pharmaceuticals | Public | - | Small molecule | DM1 | R&D | - | - | - |
| Expansion Therapeutics | Private | - | RNA inhibiting small molecule | DM1 | R&D | - | - | - |
| Ionis Pharmaceuticals & Biogen | Public | - | Ligand-conjugated antisense oligonucleotide (ASO) | DM1 | R&D | - | - | - |
| Nexion Biopharma | Private | - | Cannabinoids | DM1 | R&D | - | - | - |
| Scripps+Expansion Therapeutics | Private | - | Small molecule binding r(CUG)exp | DM1 | Lit | - | - | - |