13:05 , Sep 14, 2018 |  BC Week In Review  |  Clinical News

Eagle to meet with FDA to discuss Ryanodex exertional heat stroke data

Eagle Pharmaceuticals Inc. (NASDAQ:EGRX) said it plans to meet with FDA to discuss next steps for Ryanodex dantrolene to treat exertional heat stroke after the candidate improved neurologic function in three of four patients in...
03:42 , Mar 9, 2018 |  BioCentury  |  Emerging Company Profile

Reviving lixivaptan

Single-asset play Palladio Biosciences Inc. is repurposing lixivaptan as a safer alternative to Jinarc tolvaptan, a competing vasopressin 2 (V2) receptor antagonist associated with hepatotoxicity in patients with autosomal dominant polycystic kidney disease. ADPKD is...
19:22 , Jul 28, 2017 |  BioCentury  |  Regulation

Ganging up in rare disease

A joint proposal from EMA and FDA that encourages companies to join multidrug platform trials for pediatric Gaucher’s disease came too late to serve its original purpose. But the proposal could provide a template for...
18:46 , Jul 21, 2017 |  BC Week In Review  |  Company News

NICE final guidance recommends Sanofi's Cerdelga for Type I Gaucher's

Last month, the U.K.'s NICE issued final guidance recommending the use of Cerdelga eliglustat from the Sanofi Genzyme unit of Sanofi (Euronext:SAN; NYSE:SNY) to treat Type I Gaucher’s disease in adults who are cytochrome P450...
20:25 , Feb 16, 2017 |  BC Week In Review  |  Clinical News

GZ/SAR402671: Ph II MOVES-PD started

Sanofi’s Genzyme Corp. unit began the double-blind, placebo-controlled, dose-escalation, international Phase II MOVES-PD trial to evaluate once-daily oral GZ/SAR402671 in about 231 patients heterozygous for the glucocerebrosidase (GBA; GCase) gene mutation associated with PD. Sanofi...
20:42 , Feb 10, 2017 |  BC Week In Review  |  Clinical News

CEVA101: Ph I data

Data from the Phase I portion of an open-label, dose-escalation, U.S. Phase I/II trial in 25 adults with severe TBI and without signs of irreversible brain injury showed that 6, 9 and 12×10 6 cells/kg...
22:38 , Jan 20, 2017 |  BioCentury  |  Emerging Company Profile

Aiming for durability

Avrobio Inc. is developing cell therapies that are transduced with lentivirus ex vivo to express therapeutic proteins. The approach could provide long-lasting treatments for patients with monogenic rare diseases or cancer. Data from Phase I...
08:00 , Jan 4, 2016 |  BC Week In Review  |  Clinical News

Dantrolene: Additional Phase II data

Additional data from the open-label, Saudi Arabian Phase II Study EGL-4104-C-1502 in 34 EHS patients showed that IV Ryanodex plus standard of care (SOC) consisting of body cooling by physical methods and supportive measures led...
07:00 , Oct 12, 2015 |  BC Week In Review  |  Clinical News

Dantrolene: Phase II data

The open-label, Saudi Arabian Phase II Study EGL-4104-C-1502 in 34 EHS patients showed that IV Ryanodex plus standard of care (SOC) consisting of body cooling by physical methods and supportive measures led to no significant...
07:00 , May 25, 2015 |  BC Week In Review  |  Clinical News

GZ/SAR402671: Phase IIa started

Sanofi began an open-label, international Phase IIa trial to evaluate once-daily oral GZ/SAR402671 for 6 months in 9 treatment-naive adult male patients. The trial includes a 2-year extension study. Sanofi (Euronext:SAN; NYSE:SNY), Paris, France  ...