Navigate the Sarepta Therapeutics $SRPT and eteplirsen information pages
Formerly AVI BioPharma $AVII
Introduction
Webcasts and Events
Stock Analysis and Reporting
Eteplirsen Info
Media Reports and Patient Testimonials
Scientific and Medical Presentations
DMD and Competition
Infectious Disease Pipeline
Get first-hand perspective and education information from an inspiring family with two boys with DMD, plus learn how you can make a difference - visit www.DMDHero.com
Introduction
Webcasts and Events
Stock Analysis and Reporting
Eteplirsen Info
Media Reports and Patient Testimonials
Scientific and Medical Presentations
DMD and Competition
Infectious Disease Pipeline
Get first-hand perspective and education information from an inspiring family with two boys with DMD, plus learn how you can make a difference - visit www.DMDHero.com
Competitor: SMT C1100 from Summit (UK)
Phase 1 data will be presented at the World Muscle Society in October 2012 - click for top-line press release.
Competitor: drisapersen from Prosensa and GSK (aka PRO051 or GSK2402968)
Also an exon-skipping therapy, more advanced in development (phase 3) than eteplirsen, but with greater safety concerns to date. Click here for background information.
- Granted Orphan Drug designation for muscular dystrophy by US FDA (link)
- Holds European patent for treatment of DMD with exon 51 skipping - currently would block SRPT and eteplirsen in EU
- "Prosensa has rights to patent families corresponding to WO2002/024906 and WO2004/083432, including issued US 7,973,015, US 7,534,879" (h/t to @SteveJo22)
- 8/2012 GSK announced drisapersen phase 3 trial (no US sites) completed enrollment, data expected late 2013
- 11/2012 comments from JMP re delayed data release (h/t Mark Lehman @markflowchatter)
Competitor: Ataluren (PTC124) from PTC Therapeutics
Check back for more details. Was partnered with Genzyme but they returned rights.
- March 2010: Preliminary ataluren phase 2b results announced. 174 pts randomized to placebo, 40 mg daily, or 80 mg daily ataluren. Missed primary endpoint of six minute walk test (6MWT) at 48 weeks.
- December 2012: EMA agrees to review MAA for ataluren in DMD for patients with nonsense mutations in dystrophin. Quotes data showing the low dose above had a 31.3m improvement vs placebo.
- December 2012: PTC announces new global phase 3 trial to start 1q-2013 to support conditional approval, 220 patients.
Others
- Cialis shows promise in rare muscle disease (yes, the erectile dysfunction drug)
- DART and Biovista announce drug repositioning effort for DMD (March 2013)
- DART Therapeutics and their two in-licensed DMD candidates DT-200 and HT-100 (Xconomy)