Although trading nipped shares of United Therapeutics Corp. when the news broke, few onlookers likely felt shock when the data monitoring committee (DMC) decreed earlier this month that the company keep going with its Freedom-Ev trial testing vasodilator Orenitram (treprostinil extended-release tablets).
BOSTON – At last week's Biopharm America meeting, Astellas Pharma Inc.'s Salim Mujais sat down with BioWorld for a high-level look at the Tokyo-based firm's efforts in therapeutic areas outside of cancer.
BOSTON – How to get around venture capital (VC) firms when raising money in a company's early days yielded a lively panel discussion among alternative funders who explored various routes.
BOSTON – The "softer side of negotiation" was among topics covered during Biopharm America's second day, where Anjan Aralihalli, chief business officer of Glypharma Therapeutic Inc., of Montreal, asked attendees: "Are humans rational? Are we like Spock, where we think very rationally, or are we more like Homer Simpson?" Economic policy, he said, is "all premised on the underpinning that we think and make decisions rationally, but we don't."
BOSTON – Although a panel of venture capital (VC) investors set up to mull "the next big thing" on their wish lists appeared shy about answering the question, the talk turned up some important insights for attendees of BioPharm America on the event's first day.
Regenera Pharma Ltd. CEO Jordan Rubinson told BioWorld that “there’s over $1 billion dollars of market value, of revenues, in the first five years” if the company can succeed in a planned phase III trial and win approval for RPH-201 in nonarteritic anterior ischemic optic neuropathy (NAION), “essentially a stroke of the optic nerve.”
"In our minds, you really need [growth hormone] to be small and unmodified," Jonathan Leff, chief medical officer (CMO) for Ascendis Pharma A/S, told BioWorld, since such therapy must "get out of the bloodstream, penetrate into small nooks and crannies such as bone growth plates, and adipose tissue that's poorly vascularized. You don't mess with Mother Nature. It sounds simple and trite, but it's absolutely true in this case."
Josep Bassaganya-Riera, president and CEO of Landos Biopharma Inc., told BioWorld that the firm's lead compound, BT-11, in mouse models is "acting as a double-barrel shotgun" against inflammatory bowel disease (IBD), in both Crohn's disease and ulcerative colitis.
Solid efficacy and a distinguishing safety profile – so far, anyway – should give Alnylam Pharmaceuticals Inc. and Sanofi SA an edge over competitor Ionis Pharmaceuticals Inc., analysts said, as the partnered pair scored a top-line phase III win testing RNAi therapy patisiran in hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy.
For scientists aiming to combat the long-known problem of axonal degeneration, "the issue was that there wasn't a lever to pull, there wasn't a drug target," Disarm Therapeutics Inc.'s acting CEO, Jason Rhodes, told BioWorld.
