20:21 , Feb 2, 2018 |  BC Week In Review  |  Company News

Amicus to build U.S. biologics facility

Amicus Therapeutics Inc. (NASDAQ:FOLD) plans to spend up to $200 million to build a new biologics facility in the U.S. for its Pompe's disease program, CEO and Chairman John Crowley told BioCentury. The company said...
00:07 , Jan 27, 2018 |  BC Extra  |  Company News

AbbVie gains on updated 2018 EPS guidance

AbbVie Inc. (NYSE:ABBV) gained $14.91 (14%) to $123.21 on Friday after increasing its full-year 2018 non-GAAP EPS guidance to $7.33-$7.43 from $6.37-$6.57. The pharma said the adjusted range reflects "the impact of U.S. tax reform...
16:28 , Jan 12, 2018 |  BC Week In Review  |  Company News

Spark unveils gene therapy program for Pompe's

Spark Therapeutics Inc. (NASDAQ:ONCE) said it is developing a preclinical gene therapy for Pompe's disease using its liver-targeting adeno-associated virus (AAV) platform. The therapy, SPK-GAA, will use a novel acid alpha glucosidase (GAA) transgene in-licensed...
22:47 , Jan 9, 2018 |  BC Extra  |  Company News

Spark to develop gene therapy for Pompe's

Spark Therapeutics Inc. (NASDAQ:ONCE) said it is developing a preclinical gene therapy for Pompe's disease using its liver-targeting adeno-associated virus (AAV) platform. The therapy, SPK-GAA, will use a novel acid alpha glucosidase (GAA) transgene in-licensed...
01:09 , Dec 13, 2017 |  BC Innovations  |  Distillery Therapeutics

Endocrine / Metabolic

INDICATION: Pompe's disease Mouse studies suggest gene therapies delivering secretion-optimized GAA variants could help treat Pompe’s disease. The gene therapies consist of a liver-tropic adeno-associated viral serotype 8 (AAV8) vector encoding one of two GAA variants...
22:59 , Nov 14, 2017 |  BC Innovations  |  Distillery Therapeutics

Endocrine / Metabolic

INDICATION: Pompe's disease Mouse studies suggest GAA-loaded nanoparticles could help treat Pompe’s disease without generating anti-GAA antibodies associated with resistance to enzyme replacement therapy. The nanoparticles consist of low-immunogenic anionic phosphatidylinositol liposomes loaded with GAA. In...
17:56 , Oct 6, 2017 |  BC Week In Review  |  Clinical News

Amicus reports function outcomes data from Phase I/II of Pompe's disease candidate

Amicus Therapeutics Inc. (NASDAQ:FOLD) reported functional outcomes data from the Phase I/II ATB200-02 Study showing that IV ATB200/AT2221 improved muscle and pulmonary function in patients with Pompe's disease. Amicus CEO John Crowley told BioCentury the...
20:23 , Oct 4, 2017 |  BC Extra  |  Clinical News

Amicus eyeing 'fastest path' for Pompe's therapy

Amicus Therapeutics Inc. (NASDAQ:FOLD) rose $1.37 to $16.24 on Wednesday after reporting additional efficacy data from a Phase I/II trial showing that combo therapy ATB200/AT2221 improved muscle strength and motor function in patients with Pompe's...
21:31 , Jan 20, 2017 |  BC Week In Review  |  Clinical News

ATB200/AT2221: Preliminary Ph I/II ATB200-02 Study data

Preliminary data from 9 evaluable patients with Pompe’s disease who previously received enzyme replacement therapy (ERT) in the open-label, international Phase I/II ATB200-02 Study showed that ATB200/AT2221 led to no serious adverse events. The trial...
07:00 , Sep 7, 2015 |  BC Week In Review  |  Company News

Scioderm, Amicus Therapeutics deal

Amicus will acquire Scioderm for $125 million in cash and $104 million in stock up front. The shareholders are eligible for up to $618 million in milestones, including $361 million for clinical and regulatory milestones...