00:12 , Jul 19, 2019 |  BC Extra  |  Company News

July 18 Company Quick Takes: Mallinckrodt, Silence in RNAi deal; plus Pear, Blueprint and more

Mallinckrodt licenses RNAi candidate from Silence  Mallinckrodt plc (NYSE:MNK) obtained an exclusive, worldwide license to C3-targeted RNAi therapy SLN500 from Silence Therapeutics plc (LSE:SLN), plus options to license up to two additional complement-targeted preclinical candidates....
21:57 , Jun 27, 2019 |  BC Innovations  |  Product Development

Gene therapy’s next frontier lies beyond rare, monogenic diseases

As in vivo gene therapies continue notching approvals for rare monogenic diseases, the next frontier for the modality will be in treating complex indications without obvious genetic drivers. At least seven companies have clinical or...
21:46 , Apr 2, 2019 |  BC Innovations  |  Distillery Therapeutics

Ethoxyindole-based CFB inhibitor for complement-mediated diseases

DISEASE CATEGORY: Renal; hematology; autoimmune disease INDICATION: Renal; nephropathy; paroxysmal nocturnal hemoglobinuria (PNH); arthritis In vitro , cell culture, mouse and rat studies identified an ethoxyindole-based CFB inhibitor that could help treat the complement-mediated diseases...
21:51 , Mar 7, 2019 |  BC Extra  |  Financial News

Follow-on roundup: Horizon, Apellis, Epizyme

Horizon, Apellis and Epizyme each priced follow-ons raising a total of $552 million. Horizon Pharma plc (NASDAQ:HZNP) raised $300 million late Wednesday through the sale of 12 million shares at $24.50. The price is a...
21:51 , Feb 22, 2019 |  BioCentury  |  Emerging Company Profile

Gyroscope complements dry AMD

Gyroscope Therapeutics Ltd.’s complement-targeting gene therapy for dry age-related macular degeneration could have an edge over competing therapies that are dosed chronically or target complement downstream of its key immune mediators. On Feb. 19, Gyroscope...
20:04 , Feb 4, 2019 |  BC Extra  |  Company News

Management tracks: PhRMA, Amgen, Apellis

Sanofi (NASDAQ:SNY; Euronext:SAN) CEO Olivier Brandicourt became chairman of PhRMA. Brandicourt was chairman-elect and succeeds Robert Bradway, who is chairman and CEO of Amgen Inc. (NASDAQ:AMGN). PhRMA named Giovanni Caforio chairman-elect. Caforio is chairman and...
02:49 , Jan 5, 2019 |  BioCentury  |  Finance

Ready to launch

  The proposed $74 billion takeout of Celgene Corp. by Bristol-Myers Squibb Co. will not shift buysider attention away from upcoming and maturing drug launches heading into 2019, with plenty to track following a record...
23:31 , Dec 21, 2018 |  BC Extra  |  Company News

Alexion gains early approval for long-acting PNH therapy

Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) received FDA approval for Ultomiris ravulizumab-cwvz (ALXN1210), the biotech's long-acting formulation of Soliris eculizumab, to treat paroxysmal nocturnal hemoglobinuria. While the approval came nearly two months ahead of the Feb. 18...
19:26 , Dec 21, 2018 |  BC Week In Review  |  Clinical News

Achillion discontinuing PNH monotherapy

Achillion Pharmaceuticals Inc. (NASDAQ:ACHN) said it will discontinue development of ACH-4471 as oral monotherapy treatment of paroxysmal nocturnal hemoglobinuria in favor of an earlier stage compound. The decision is a delay for Achillion as it...
00:47 , Dec 19, 2018 |  BC Extra  |  Clinical News

Achillion down after discontinuing PNH monotherapy

Achillion Pharmaceuticals Inc. (NASDAQ:ACHN) lost $0.45 (19%) to $1.95 on Tuesday after it said it will discontinue development of ACH-4471 as oral monotherapy treatment of paroxysmal nocturnal hemoglobinuria in favor of an earlier stage compound....