08:00 , Jan 12, 2015 |  BioCentury  |  Finance

Buyside view XXIII: Milestones galore

Stephen Hansen, Associate Editor and Jennifer Rhodes, Staff Writer   Buyside view XXIII The maturation of the biotech sector over the past few years finds fund managers focusing on a slew of clinical milestones and commercial launches...
07:00 , Sep 8, 2014 |  BC Week In Review  |  Clinical News

Vatiquinone alpha-tocotrienol quinone: Phase II data

A double-blind, Italian Phase II trial in 24 female Rett syndrome patients ages 2.5-8 showed that thrice-daily 15 mg/kg EPI-743 missed the primary endpoint of improving the Rett Syndrome Clinical Severity Scale score from baseline...
00:49 , Sep 4, 2014 |  BC Extra  |  Clinical News

Edison's EPI-743 misses Rett syndrome endpoint

Edison Pharmaceuticals Inc. (Mountain View, Calif.) said EPI-743 missed the primary endpoint of improving the Rett Syndrome Clinical Severity Scale score vs. placebo in a Phase II trial. The compound did meet a secondary endpoint...
07:00 , Aug 11, 2014 |  BioCentury  |  Emerging Company Profile

AAVLife: Fixing Friedreich's heart

AAVLife S.A.S. is developing an adeno-associated viral vector-based gene therapy that could become the first disease-modifying treatment for Friedreich's ataxia-associated cardiomyopathy. The therapy has the potential to decrease the risk of cardiac events and improve...
07:00 , Jun 16, 2014 |  BC Week In Review  |  Clinical News

Vatiquinone regulatory update

Edison said FDA granted EPI-743 Orphan Drug designation to treat Leigh syndrome. The compound is in a U.S. Phase IIb trial and a Japanese Phase IIb/III trial for the indication, for which it already has...
07:00 , May 26, 2014 |  BC Week In Review  |  Clinical News

Vincerinone vatiquinone regulatory update

Edison said Japan's Ministry of Health, Labor and Welfare (MHLW) granted EPI-743 Orphan Drug designation to treat Leigh syndrome. The compound is in a U.S. Phase IIb trial and a Japanese Phase IIb/III trial for...
07:00 , Apr 24, 2014 |  BC Innovations  |  Targets & Mechanisms

Reversing (heart) failure in Friedreich's ataxia

Heart failure accounts for over half the deaths in patients with Friedreich's ataxia, but no therapies exist to treat this neurodegenerative disease or its associated cardiomyopathy. Now, a team of French researchers has shown that...
07:00 , Mar 24, 2014 |  BC Week In Review  |  Clinical News

Vatiquinone regulatory update

Edison said FDA granted Fast Track designation to EPI-743 to treat Friedreich's ataxia. The compound is in Phase II testing for the indication, for which it has Orphan Drug designation in the U.S. EPI-743 also...
07:00 , Mar 24, 2014 |  BC Week In Review  |  Clinical News

Vatiquinone: Phase II started

Edison began a 12-month, international Phase II trial to evaluate oral EPI-743 in children with Pearson syndrome, an ultra-rare, fatal mitochondrial disease characterized by sideroblastic anemia, pancytopenia and pancreatic dysfunction. EPI-743 is in Phase IIb...
07:00 , Mar 17, 2014 |  BC Week In Review  |  Clinical News

EPI-743: Phase IIb/III started

Edison and Dainippon began a 6-month, open-label, Japanese Phase IIb/III trial to evaluate EPI-743 in >=5 children with clinical and radiographic evidence of Leigh syndrome with or without genetic confirmation. EPI-743 has Orphan Drug designation...