Santhera Eyes US Market Potential After DMD Candidate Tests Positive
Top-Line Phase IIb Results For Vamorolone
Executive Summary
Just under a year since Santhera restructured after disappointing late-stage clinical results with another DMD candidate, the Switzerland-headquartered company has reported positive top-line results in the condition with licensed-in vamorolone, and is planning for commercialization in late 2022/early 2023.
You may also be interested in...
Santhera Restructuring: Discontinues Lead DMD Candidate
Switzerland’s Santhera is to restructure its business and seek extra funding after the failure of Puldysa (idebenone) in a Phase III Duchenne muscular dystrophy study at interim analysis.
Pipeline Watch: Phase III Readouts in Wilson Disease, Parkinson's Disease And COVID-19
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
AZ's Rare Disease Unit Reports Positive Phase III Results For Copper-Binding ALXN1840
Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.