BioCentury | Oct 15, 2020
Product Development

Lingering questions as Vertex sheds $15B after Phase II setback

With a CF franchise that is generating operating income with triple digit growth, it is difficult to square why investors slashed nearly $15 billion from Vertex’s valuation over the failure of a single...
BioCentury | Oct 15, 2020
Product Development

Oct. 14 Quick Takes: FDA approves first Ebola therapy; plus Vertex tumbles after hours on AAT discontinuation

Regeneron’s Ebola mAb cocktail approvedFDA approved Inmazeb atoltivimab/maftivimab/odesivimab-ebgn from Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) to treat Zaire ebolavirus infection. The mAb cocktail had raised the chance of survival vs. ZMapp, a mAb combination used as a control, in...
BioCentury | Oct 9, 2020

Arrowhead gains Takeda firepower as it seeks edge over Dicerna in AAT-associated liver disease

Arrowhead has found a partner in Takeda that could help it compete with Dicerna as the two biotechs advance their RNAi therapies to treat alpha-1 antitrypsin (AAT)-associated liver disease. The deal...
BioCentury | Jul 29, 2020
Distillery Therapeutics

Inhibiting proteolytic activation of the receptor PAR2 for eosinophilic esophagitis

DISEASE CATEGORY: Inflammation INDICATION: Eosinophilic esophagitis Inhibiting the extracellular serine protease KLK5 or its substrate PAR2 could treat eosinophilic esophagitis, a food antigen-driven inflammatory disease. Single-cell sequencing revealed aberrant KLK5 expression in multiple cell populations...
BioCentury | May 2, 2020
Translation in Brief

ORIC’s CD73 inhibitor for cancer; plus Beam, CRISPR-Cas13 for respiratory infections, COVID-19 complex structure, and more

Small molecule CD73 inhibitor for cancer A small molecule CD73 inhibitor from ORIC Pharmaceuticals Inc. (NASDAQ:ORIC) has shown significant antitumor activity in vivo . ORIC-533 is slated for an IND submission in 1H21. CD73, which is...
BioCentury | Apr 7, 2020
Product Development

April 6 Quick Takes: Second anemia approval for BMS, Acceleron therapy; plus EU approves trio of metabolic disease drugs, Dicerna-Alnylam and Axsome

Reblozyl gets second approval FDA approved Reblozyl luspatercept-aamt from Bristol Myers Squibb Co. (NYSE:BMY) and Acceleron Pharma Inc. (NASDAQ:XLRN) to treat anemia in certain patients with myelodysplastic syndromes. It is the second indication for the...
BioCentury | Mar 21, 2020
Product Development

Under duress, biotechs will have to make hard decisions for patients and themselves about which trials to run

While COVID-19’s risk to clinical trials was initially a function of geography, the fallout for companies is now less a matter of where the studies are being run, and more about which patient population is...
BioCentury | Mar 18, 2020

FDA’s clinical trial guidance stresses patient safety, studies’ integrity amid outbreak

As disruptions begin to mount among biopharmas’ planned or ongoing clinical trials during the COVID-19 outbreak, newly issued FDA guidance clarified the agency’s thinking on how sponsors can ensure participants’ safety and implement modifications to...
BioCentury | Nov 27, 2019
Company News

With shares rising on M&A chatter, Arrowhead points to pipeline moves in 2020

Buoyed by news of this week's takeout of Medicines Co., Arrowhead is riding a wave of investor enthusiasm for RNAi therapeutics that has quintupled the company’s share price this year, and has boosted its valuation...
BioCentury | Nov 22, 2019
Company News

Alnylam outlines ambitions, pipeline progress at R&D day

In its R&D day presentation Friday, Alnylam outlined its goals of becoming a self-sustaining company with at least eight approved drugs and 10 late-stage clinical candidates by 2025, with safer GalNAc conjugates, a late-stage amyloidosis...
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