Neuromuscular diseases
28 public biotech companies we cover are developing treatments for muscular dystrophies, myasthenia gravis, myositis and related conditions. 26 upcoming catalysts are listed below, with dates as the companies gave them.
Companies
- Annexon (ANNX)Thesis Intact
Guillain-Barr Syndrome
- Arrowhead Pharmaceuticals (ARWR)Thesis Intact
Myotonic dystrophy type 1, Facioscapulohumeral muscular dystrophy
- Artiva Biotherapeutics (ARTV)Thesis Intact
Myositis
- Biohaven (BHVN)Thesis Challenged
Duchenne muscular dystrophy
- BridgeBio Pharma (BBIO)Thesis Intact
LGMD, LGMD2I/R9, LGMD2M/R13 and LGMD2U/R20, Limb-girdle muscular dystrophy, and 1 more
- Cabaletta Bio (CABA)Thesis Intact
Myositis, Dermatomyositis, Myasthenia gravis
- Capricor Therapeutics (CAPR)Thesis Broken
Becker muscular dystrophy, Duchenne muscular dystrophy
- Cartesian Therapeutics (RNAC)Thesis Intact
Myositis, Myasthenia gravis, Juvenile dermatomyositis, Generalized myasthenia gravis, and 1 more
- CRISPR Therapeutics (CRSP)Thesis Intact
Inflammatory myositis
- Design Therapeutics (DSGN)Thesis Intact
Friedreich Ataxia, Myotonic Dystrophy Type 1
- Entrada Therapeutics (TRDA)Thesis Intact
Myotonic dystrophy type 1, Duchenne muscular dystrophy
- Immunovant (IMVT)Thesis Challenged
Myasthenia gravis, Chronic inflammatory demyelinating polyneuropathy
- Intellia Therapeutics (NTLA)Thesis Intact
ATTR with polyneuropathy, Hereditary ATTR amyloidosis with polyneuropathy
- Ionis Pharmaceuticals (IONS)Thesis Intact
Spinal muscular atrophy
- Jade Biosciences (JBIO)Thesis Intact
Dermatomyositis
- Keros Therapeutics (KROS)Thesis Intact
Duchenne muscular dystrophy
- Kyverna Therapeutics (KYTX)Thesis Intact
Generalized myasthenia gravis
- Larimar Therapeutics (LRMR)Thesis Intact
Friedreich's ataxia
- Lexeo Therapeutics (LXEO)Thesis Intact
Friedreich ataxia, Friedreich ataxia cardiomyopathy
- Mesoblast (MESO)Thesis Intact
Duchenne muscular dystrophy
- PepGen (PEPG)Thesis Challenged
Myotonic dystrophy type 1
- Precision BioSciences (DTIL)Thesis Intact
Duchenne muscular dystrophy
- Rocket Pharmaceuticals (RCKT)Thesis Intact
Danon disease
- Roivant Sciences (ROIV)Thesis Intact
Dermatomyositis, Myasthenia gravis, Chronic inflammatory demyelinating polyneuropathy
- Sarepta Therapeutics (SRPT)Thesis Challenged
LGMD2E/R4, Myotonic dystrophy type 1, Duchenne muscular dystrophy, Limb girdle muscular dystrophy, and 1 more
- TG Therapeutics (TGTX)Thesis Intact
Myasthenia Gravis
- Ultragenyx Pharmaceutical (RARE)Thesis Challenged
GNE myopathy
- Xeris Biopharma (XERS)Thesis Intact
Primary periodic paralysis
Drug pages
Upcoming catalysts
Firm dates · a specific day set by the company or regulator
Next 30 days
- Nov 22in 28 daysDeramiocel · FDA decision · Duchenne muscular dystrophy
Next 60 days
- Nov 27in 33 daysBBP-418 · NDA accepted for review · LGMD2I/R9
Estimated timing · a month, quarter, half-year or year-end the company gave
Expected in November 2026
- November 2026PGN-EDODM1 · Phase 2 results (FREEDOM2) · Myotonic dystrophy type 1
Expected in Q4 2026
- Q4 2026Tanruprubart · BLA submission · Guillain-Barr Syndrome
Expected in H2 2026
- H2 2026Nomlabofusp · BLA submission · Friedreich's ataxia
- H2 2026Nex-z · Phase 3 enrollment complete (MAGNITUDE-2) · ATTR with polyneuropathy
- H2 2026UX016 · Phase 1/2 start · GNE myopathy
- 2H 2026Descartes-08 · Phase 1 start · Generalized myasthenia gravis · in vivo delivery
- H2 2026ENTR-601-50 · Regulatory submission · Duchenne muscular dystrophy · exon 50 skipping amenable
- H2 2026ENTR-601-50 · Trial start (ELEVATE-50-201) · Duchenne muscular dystrophy · exon 50 skipping amenable
Expected in Q1 2027
- Q1 2027DT-216P2 · Phase 1/2 results (RESTORE-FA) · Friedreich Ataxia
- Q1 2027Descartes-08 · Phase 3 topline results (AURORA) · Generalized myasthenia gravis
- Q1 2027ELEVIDYS · Phase 1b results (ENDEAVOR) · Duchenne muscular dystrophy · non-ambulant
- Q1 2027ENTR-601-44 · Phase 1/2 results (ELEVATE-44-201) · Duchenne muscular dystrophy · exon 44 skipping amenable
Expected by end of March 2027
- Early 2027AMONDYS 45 · FDA decision: conversion to full approval · Duchenne muscular dystrophy · exon 45 skipping eligible
- Early 2027VYONDYS 53 · FDA decision: conversion to full approval · Duchenne muscular dystrophy · exon 53 skipping eligible
Expected in H1 2027
- H1 2027; near futureBBP-418 · Trial start · LGMD · pediatric and additional subtypes
- H1 2027Rinvatercept · Phase 2 interim results (NCT07704099) · Duchenne muscular dystrophy
- H1 2027Nomlabofusp · FDA decision · Friedreich's ataxia
- H1 2027PGN-EDODM1 · Phase 2 results (FREEDOM2) · Myotonic dystrophy type 1
- H1 2027Descartes-08 · Phase 2 topline results (TRITON) · Myositis · dermatomyositis and antisynthetase syndrome
- H1 2027ENTR-601-45 · Phase 1/2 results (ELEVATE-45-201) · Duchenne muscular dystrophy · exon 45 skipping amenable
Expected by end of June 2027
- mid-2027Rese-cel · Pivotal topline results (RESET-Myositis) · Dermatomyositis · antisynthetase syndrome
- mid-2027Miv-cel · Phase 3 enrollment complete (KYSA-6) · Generalized myasthenia gravis
- mid-2027RP-A501 · Phase 2 enrollment complete · Danon disease · male patients
- mid-2027Descartes-08 · BLA submission · Generalized myasthenia gravis