Cue Biopharma Inc., which in September filed for an IPO to raise up to $40 million – having only emerged from stealth mode in January – inked a deal with Merck & Co. Inc. that brings an undisclosed up-front payment along with as much as $374 million in research, development, regulatory and commercial milestone payments, plus tiered royalties on sales if all goes well.
Sangamo Therapeutics Inc.'s chief medical officer Edward Conner said the first patient to be treated with SB-913 "was clear that he wasn't just looking for help for himself, but he wanted to help future generations of MPS patients as well," and the in vivo genome-editing therapy could do just that for people with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Exithera Pharmaceuticals Inc. CEO Neil Hayward told BioWorld he's hoping to build a "continuum of care" around factor XIa inhibitor EP-7041, after phase I data showed the intravenous (I.V.) version of the antithrombotic safe and well-tolerated in healthy volunteers after single or multiple ascending intravenous doses – and even turned up some clues to efficacy.
Nektar Therapeutics Inc.'s vice president of clinical development, Mary Tagliaferri, told BioWorld that the compelling objective response rate and impressive disease control rate (DCR) with NKTR-214 when paired with Opdivo (nivolumab) from Bristol-Myers Squibb Co. (BMS) in cancer – along with a clean safety profile and an "antibody-like dosing regimen" once every three weeks – likely put the program in line for accelerated approval.
After the approval last week of Dynavax Technologies Corp.'s Heplisav-B hepatitis B virus (HBV) vaccine, backers are looking forward to the launch in the first quarter of next year and the meeting of the Advisory Committee on Immunization Practices (ACIP), which can vote to add the vaccine to its "preferred" list for easier coverage by payers.
Otonomy Inc. CEO David Weber told BioWorld that researchers at his firm "have what we need to continue moving forward" in Ménière's disease with Otividex, the sustained-exposure formulation of dexamethasone that cheered investors by ringing the phase III bell after a similar late-stage experiment failed.
An apparent screw-up by organizers of the Society for Immunotherapy of Cancer (SITC) meeting led to trouble on Wall Street for Five Prime Therapeutics Inc., which spent time explaining not only the import of its phase Ia/Ib data in pancreatic cancer (PC) but also how the abstract related thereto appeared online unexpectedly.
Oyster Point Pharmaceuticals Inc.’s $22 million series A financing will push two lead therapies in corneal surface disorders through phase II trials, but CEO Jeffrey Nau told BioWorld he was “not willing at the moment to comment on the mechanism of action. We want to make sure that we keep a competitive advantage for a little bit. Once we have our first clinical trial under our belt – and we’re very optimistic at the moment – we’ll share more information,” he said.
In developing aducanumab, one of the more promising therapies for Alzheimer's disease (AD), Neurimmune Therapeutics AG took a creative approach – one that caught the eye of Biogen Inc. "Aducanumab actually comes from a human individual, someone who was in his or her late 90s or early 100s," Ajay Verma, previously with Cambridge, Mass.-based Biogen, told BioWorld.
United Neuroscience Inc.'s chief medical officer, Ajay Verma, told BioWorld that research into Alzheimer's disease (AD) has "positioned us now to finally test the hypothesis" that amyloid beta (a-beta) is the culprit, or at least a main one, in the scourge.
