In the fast-advancing hepatitis C virus (HCV) space, to make a splash isn't easy, as Regulus Therapeutics Inc. proved by unveiling phase I data that hold promise but were met by Wall Street with a lukewarm, wait-and-see attitude.
Going it alone with the oral, once-daily factor Xa inhibitor betrixaban, for which Merck & Co. Inc. handed back rights in 2011, Portola Pharmaceuticals Inc. marches ahead, following a pre-specified futility analysis by the APEX phase III trial's monitoring board.
G1 Therapeutics Inc.'s $33 million in series B money gives the firm "flexibility to hang onto [the lead program] and continue to move it further into development on our own," said CEO Mark Velleca.
What was described last week by Roche Holding AG CEO Severin Schwan as a "chance for readouts" this year with anti-CD20 therapy Gazyva (obinutuzumab) came to pass in a big way, as Roche unit Genentech scored a phase III win in the indolent form of non-Hodgkin's lymphoma (NHL), a slow-growing type of the disease in which patients are prone to multiple relapses.
Chemically separating the antifungal efficacy of the amphotericin B scaffold from the mechanism that can lead to kidney damage – thus creating more potency without harm – is among the first projects of Revolution Medicines Inc., expected to disclose its $45 million round of series A financing Wednesday.
Tenapanor, the NHE3 sodium transporter inhibitor that yielded positive phase IIb data last fall in constipation-predominant irritable bowel syndrome (IBS-C) for Ardelyx Inc. and partner Astrazeneca plc, hit its efficacy endpoint in a second indication, but disappointed investors with "distinctly" higher-than-expected instances of diarrhea.
The FDA's granting of breakthrough designation for Intercept Pharmaceuticals Inc.'s obeticholic acid (OCA) in nonalcoholic steatohepatitis (NASH) put the U.S. regulators on record about the disease – its dangers and the need for new therapies, "the first time that this has been acknowledged formally by the agency" outside of workshops, noted Deutsche Bank analyst Alethia Young in an email alert to investors Friday.
With "our crown jewel back in our hands and the opportunity to bring it all the way to the market," Symphogen A/S found cause to celebrate, said CEO Kirsten Drejer, as the firm makes plans to finish the phase IIb trial begun by Merck KGaA with Sym004 and move the cancer antibody product forward.
Mustering hope in the obesity space – marked by drugs' generally disappointing sales despite a large and growing market – is tough, but Boston-based Zafgen Inc. recently drew investor enthusiasm and pulled off a $138 million public offering to advance what RBC Capital Markets analyst Simos Simeonidis called "a really impressive drug."
Basilea Pharmaceutica Ltd.'s licensing partner Astellas Pharma Inc. won the favor of the FDA's Anti-Infective Drugs Advisory Committee for the antifungal isavuconazole, achieving a slam-dunk vote of 8-0 to recommend approval in the invasive aspergillosis indication, though the panel's balloting proved less certain about the rarer invasive mucormycosis: 8-2 in favor, with one abstention.
