The delay in reporting data from the phase II trial by Ultragenyx Pharmaceutical Inc. with UX007 (triheptanoin, or trihep) in glucose transporter type-1 deficiency syndrome (Glut1 DS) patients with seizures may have tipped some investors that mixed results lay ahead with the compound, but Raymond James analyst Christopher Raymond said that, "with the Glut1 DS filing strategy still dependent on the pivotal phase III movement-disorder study, we do not see [the latest] data meaningfully changing trihep's opportunity in Glut1 DS."
With Pfizer Inc.'s oxycodone Troxyca ER still unlaunched after approval last summer and Collegium Pharmaceutical Inc. hammering away for market share with April-cleared Xtampza ER, the quest for a better abuse-resistant, extended-release oxycodone goes on, as does Purdue Pharma LP, selling Oxycontin in the $2.5 billion pain market.
Asked whether U.S. regulators might change their minds about guidance on the regulatory path for bempedoic acid to lower low-density lipoprotein C (LDL-C), Esperion Therapeutics Inc. CEO Tim Mayleben said “the answer is that we don’t know. We know what we know today. That’s probably a question better addressed to the FDA and what they’re planning.”
In what CEO Mark Murray told BioWorld Today may signal "the first of a number of deals," hepatitis B virus (HBV)-focused Arbutus Biopharma Corp. is bringing aboard $7.5 million up front and as much as $75 million in development, regulatory and commercial milestone payments from Alexion Pharmaceuticals Inc. for the lipid nanoparticle (LNP) technology.
As Biocryst Pharmaceuticals Inc. prepared to close its $45 million financing round last week, work continued with BCX7353 and second-generation compounds for hereditary angioedema (HAE). So did speculation about the oral, second-generation kallikrein inhibitor's chances in the crowded HAE space, as company watchers mulled interim outcomes unveiled in late February from APeX-1, a phase II dose ranging trial.
Aerpio Pharmaceuticals Inc. has "a very different way" of activating Tie2, CEO Joseph Gardner told BioWorld Today. "What I don't know is whether an anti-angiopoietin-2 [Ang2] antibody activates Tie2, and how much activation you would get," he said. "We feel very confident with our approach. Our program is on solid ground scientifically, in terms of the data we have generated and published."
"There are many forms of graft vs. host disease [GVHD], many types, so there will not be one solution for all of them," Gil Efron, deputy CEO and chief financial officer of Kamada Ltd., told BioWorld Today, but the Ness Ziona, Israel-based firm may have a fix for major unmet need.
Details were scant about the FDA holdup of Radius Health Inc.'s abaloparatide-SC for postmenopausal osteoporosis – regulators pushed the PDUFA date from March 30 to June 30, saying they need more time – but at least the hitch isn't related to manufacturing, apparently.
Beyondspring Inc. priced its IPO of 174,286 shares at $20 each and sold about 2.5 million shares at the same price in a private placement, hauling down about $54.3 million, which "probably for some companies is not that much," CEO Lan Huang told BioWorld Today. "But it can really help us to run our current defined clinical programs," since the New York-headquartered company bases its clinical operations in China. Shares (NASDAQ:BYSI) closed Thursday at $16.80, down 16 percent.
