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...
18:11 , Jul 27, 2018 |  BC Week In Review  |  Company News

Biogen acquires neuromuscular assets from AliveGen

Biogen Inc. (NASDAQ:BIIB) said it acquired two early stage neuromuscular assets from AliveGen USA Inc. (Thousand Oaks, Calif.) and reported rising U.S. sales of spinal muscular atrophy (SMA) therapy Spinraza nusinersen in its 2Q18 earnings...
17:58 , Jul 24, 2018 |  BC Extra  |  Company News

Biogen acquires neuromuscular assets, reports 2Q earnings

Biogen Inc. (NASDAQ:BIIB) said it acquired two early stage neuromuscular assets from AliveGen USA Inc. (Thousand Oaks, Calif.) and reported rising U.S. sales of spinal muscular atrophy (SMA) therapy Spinraza nusinersen in its 2Q18 earnings...
19:26 , Jun 1, 2018 |  BC Week In Review  |  Financial News

Scholar Rock raises $75M IPO

Scholar Rock Holding Corp. (NASDAQ:SRRK) raised $75 million on May 23 through the sale of 5.4 million shares at $14 in an IPO. Jefferies, Cowen, BMO Capital Markets and Wedbush PacGrow underwrote the deal. Scholar...