MyotonicPipeline.com
Expansion Therapeutics
Drug #1: ERX-963
Approach: Unknown
Status: In June 2019 Expansion commenced a Phase 1 trial in 12 adult-onset myotonic dystrophy patients. Trial failed to show efficacy. See AAN 2021 presentation.
Drug #2: Small molecule RNA inhibitor
Status: Preclinical
Company Website with description of approach
Video from 2018 MDF Conference
January 3, 2018 Press Release on Expansion's launch
Article on preclinical tests of Expansion's DM1 drug
Publication of preclinical tests
September 14, 2019 Presentation at Myotonic Conferenct (1:00)
Patent Application: MODULARLY ASSEMBLED SMALL MOLECULES FOR THE TREATMENT OF MYOTONIC DYSTROPHY TYPE 1
April 2020 presentation by Dr. Disney: Advancing Precision Medicines. Discussion of myotonic dystrophy at 41:50 and 1:05:00
April 2021 presentation for Myotonic Dystrophy Foundation
Sept. 2021 Press Release announcing $80 million capital raise