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.

Ultragenyx Pharmaceutical Inc. CEO Emil Kakkis said his company seized "an opportune moment" to enter gene therapy via the cash counter-offer for Dimension Therapeutics Inc. at $5.50 per share, or about $138 million, stacking the proposal against last month's all-stock bid valued at around $110 million by Regenxbio Inc.

Halozyme Corp. CEO Helen Torley told BioWorld that she expects the latest news about the firm's subcutaneous (subcu) hyaluronidase technology "on the heels of the Rituxan Hycela approval in June of this year by the FDA [will] spark even more rapid growth of companies wanting to sign an Enhanze deal."

Nanology LLC's strategy is "almost like having a new molecular entity, in terms of the broadness of the intellectual property [IP] around it, but on a more streamlined path to potential approval" if safety and efficacy pan out, Marc Iacobucci, managing director of DFB Pharmaceuticals Inc. told BioWorld, with a nod to the 505(b)(2) regulatory pathway that the Fort Worth, Texas-based firm intends to trod.

Shingrix (varicella zoster vaccine [recombinant], adjuvanted) from Glaxosmithkline plc (GSK) sailed through a hearing by the FDA's Vaccines and Related Biological Products Committee, with panelists voting 11-0 in favor of the product's efficacy and safety in people 50 years of age and beyond.

Achillion Pharmaceuticals Inc.'s chief commercial officer, Joseph Truitt, said the firm "will certainly assess all the options for the hepatitis C virus [HCV] portfolio" after the ending of the deal by Johnson & Johnson (J&J) unit Janssen Pharmaceuticals Inc., but had no further guidance.

Adaptimmune Therapeutics plc CEO James Noble said Glaxosmithkline plc (GSK) exercised its option "much earlier than they needed to," exclusively licensing rights to research, develop and commercialize Adaptimmune's NY-ESO SPEAR T-cell therapy program.

"I'd hate for people to just assume, 'Oh, this is another cancer vaccine, therefore it's going to fail like so many others have failed in big clinical trials,'" Gritstone Oncology Inc. CEO Andrew Allen told BioWorld. Investors in the company's individualized, tumor-specific neoantigen strategy – able to sort the plethora of mutations in cancer for key ones – apparently made no such assumption, as they put a handsome $92.7 million into the series B round.