22:18 , May 20, 2019 |  BioCentury  |  Product Development

Making Orphan drug prices work for society

The Orphan Drug Act worked too well. It didn’t just stimulate development of therapies for rare diseases, it reoriented huge swaths of the industry to the pursuit, all attracted by the high prices and product...
18:22 , May 7, 2019 |  BC Extra  |  Clinical News

May 7 Clinical Quick Takes: AZ, Merck, Osmotica, AlzeCure

AZ's Calquence meets in Phase III for CLL  AstraZeneca plc (LSE:AZN; NYSE:AZN) said an interim analysis of the Phase III ASCEND trial to treat relapsed or refractory chronic lymphocytic leukemia showed that Calquence acalabrutinib as...
19:37 , May 6, 2019 |  BC Extra  |  Company News

May 6 Company Quick Takes: Esperion, BioMarin, WuXi AppTec, Axsome, Harbour BioMed/CTTQ, GW, Adynxx

FDA accepts Esperion NDAs for LDL-C-lowering indications  Esperion Therapeutics Inc. (NASDAQ:ESPR) gained $5 (12%) to $48.24 on Monday after FDA accepted for review a pair of NDAs for bempedoic acid (ETC-1002) and a bempedoic acid/ezetimibe...
19:03 , Jan 11, 2019 |  BC Week In Review  |  Clinical News

Aevi's AEVI-001 misses in Phase II for children with ADHD

Aevi Genomic Medicine Inc. (NASDAQ:GNMX) said AEVI-001 missed the primary endpoint in both Parts A and B of the Phase II ASCEND trial to treat ADHD in patients ages 6-17. The company plans to conduct...
17:47 , Jan 11, 2019 |  BC Week In Review  |  Clinical News

Axsome's AXS-05 meets in Phase II for MDD

Axsome Therapeutics Inc. (NASDAQ:AXSM) added $4.24 (161%) to $6.87 on Jan. 7 after reporting that AXS-05 met the primary endpoint in the Phase II ASCEND trial to treat moderate-to-severe major depressive disorder (MDD). On the...
22:10 , Jan 7, 2019 |  BC Extra  |  Clinical News

Sage rebounds after next-gen postpartum depression readout

Sage Therapeutics Inc. (NASDAQ:SAGE) gained $41.62 (43%) to $139.13 on Monday after it said SAGE-217 met the primary endpoint in the Phase III ROBIN trial to treat women with severe postpartum depression. The move translates...
20:46 , Oct 5, 2018 |  BC Week In Review  |  Clinical News

Sarepta reports microdystrophin expression data for fourth patient in DMD gene therapy trial

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said AAVrh74.MHCK7.micro-Dystrophin led to mean sarcolemma-localized transduced microdystrophin expression, as measured by immunohistochemistry, of 81.2% and a mean fiber intensity of 96% compared with normal controls in four Duchenne muscular dystrophy...
18:19 , Jul 27, 2018 |  BC Week In Review  |  Clinical News

FDA places clinical hold on Sarepta's DMD gene therapy

Sarepta Therapeutics Inc. (NASDAQ:SRPT) and partner Nationwide Children’s Hospital said FDA placed a clinical hold on a Phase I/IIa trial of their Duchenne muscular dystrophy gene therapy, AAVrh74.MHCK7.micro-Dystrophin. The partners said the hold is due...
18:27 , Jun 22, 2018 |  BC Week In Review  |  Clinical News

Sarepta's DMD candidate leads to microdystrophin expression of 76.2% in Phase I/IIa

Sarepta Therapeutics Inc. (NASDAQ:SRPT) reported on June 19 preliminary data from a Phase I/IIa trial evaluating AAVrh74.MHCK7.micro-Dystrophin to treat Duchenne muscular dystrophy (DMD). In three DMD patients in the open-label, U.S. trial, the microdystrophin gene...
17:12 , Dec 2, 2016 |  BC Week In Review  |  Clinical News

ASN-002: Interim Ph I/IIa data

Interim data from 9 patients with nodular BCC in an open-label, Australian Phase I/IIa trial showed that once-weekly intratumoral ASN-002 for 3 weeks led to an overall response rate (ORR) of 100%, comprising 67% complete...