19:44 , May 9, 2019 |  BC Extra  |  Clinical News

May 9 Clinical Quick Takes: Polyphor, BMS, Cel-Sci, University of Pittsburgh, Merck, Scholar Rock, Promethera

Polyphor halts Phase III enrollment of pneumonia therapy  Polyphor Ltd. (SIX:POLN) said late Thursday it will suspend enrollment in the Phase III PRISM-MDR and PRISM-UDR trials of pneumonia antibiotic murepavadin (POL7080) after observing a 56%...
19:11 , Apr 12, 2019 |  BC Week In Review  |  Clinical News

Acceleron discontinues ACE-2494 due to antidrug antibodies

Acceleron discontinued development of ACE-2494 due to the frequency of antidrug antibodies observed among healthy volunteers in a Phase I trial. Acceleron Pharma Inc. (NASDAQ:XLRN) was developing ACE-2494 as a potential treatment of neuromuscular disorders....
23:20 , Mar 15, 2019 |  BioCentury  |  Emerging Company Profile

Neurogene: Genes over ERT for monogenic CNS diseases

Neurogene is developing disease-modifying gene therapies for two monogenic, pediatric disorders in which enzyme replacement is not feasible or hasn't been explored. Aspartylglucosaminuria (AGU) is a lysosomal storage disorder caused by autosomal recessive AGA mutations...
00:06 , Sep 28, 2018 |  BC Innovations  |  Emerging Company Profile

GDF11 against aging

Harvard University spinout Elevian Inc. is developing a regenerative platform around GDF11 to treat age-related diseases, including Alzheimer's disease. The company, which emerged from stealth on Sept. 6, aims to modulate the protein’s pathway with...
22:58 , Sep 14, 2018 |  BioCentury  |  Regulation

Closing NICE’s Orphan gap

As the list of Orphan drugs rejected by NICE grows, industry and patient groups are pressuring the agency to make broader use of tools that currently apply only to selected ultra-Orphan therapies. These tools allow...
19:43 , Aug 31, 2018 |  BC Week In Review  |  Clinical News

Pfizer ends Phase II trials of DMD candidate

Pfizer Inc. (NYSE:PFE) terminated two Phase II trials of domagrozumab (PF-06252616) to treat Duchenne muscular dystrophy after one of the studies missed its primary endpoint. The company will evaluate the data to determine next steps...
15:47 , Aug 30, 2018 |  BC Extra  |  Clinical News

Pfizer ends Phase II trials of DMD candidate

Pfizer Inc. (NYSE:PFE) terminated two Phase II trials of domagrozumab (PF-06252616) to treat Duchenne muscular dystrophy after one of the studies missed its primary endpoint. The company will evaluate the data to determine next steps...
03:00 , Aug 10, 2018 |  BC Innovations  |  Targets & Mechanisms

Getting to function

A new target for sarcopenia that increases muscle function directly, in addition to adding muscle mass, could help address calls from patients and regulators to make functional recovery central to drug development in the indication....
16:35 , Aug 3, 2018 |  BC Week In Review  |  Clinical News

Acceleron's ACE-083 increases muscle volume in Phase II for Charcot-Marie-Tooth disease

Acceleron Pharma Inc. (NASDAQ:XLRN) reported preliminary data from 18 patients with Charcot-Marie-Tooth disease in Part 1 of a Phase II trial showing that ACE-083 increased mean total muscle volume by 12.6-14.2%, increased mean contractile muscle...
20:06 , Aug 1, 2018 |  BC Innovations  |  Distillery Therapeutics

Cancer

INDICATION: Lung cancer Patient sample, cell culture and mouse studies suggest inhibiting activin signaling could enhance the efficacy of platinum-based chemotherapy in KRAS-mutant lung cancer. In 59 patients with stage IV non-small cell lung cancer...