Trouble surfaced early in the joint meeting of the FDA's Arthritis Advisory Committee (AAC) and the Drug Safety and Risk Management Advisory Committee for Takeda Pharmaceuticals Co. Ltd. with the supplemental NDA for xanthine oxidase inhibitor (XOI) Uloric (febuxostat).
What Oppenheimer analyst Leland Gershell a few months ago called "the shift toward non-opioid/locally delivered treatment strategies [in pain], driven by increasing regulatory/payer/institutional hurdles" has brought a handful of players to the fore.
With a new CEO imported from Novartis AG, Urogen Pharma Ltd. popped the cork on eagerly awaited top-line data from the ongoing pivotal phase III trial called Olympus with UGN-101 (mitomycin gel) for instillation, a nonsurgical treatment of low-grade upper tract urothelial cancer. "It's a very creative solution to a nagging problem, which is how to take care of these patients, most of whom end up losing their kidneys because we don't have a lot of alternatives," New York-based Urogen's chief medical officer, Mark Schoenberg, told BioWorld.
Eli Lilly and Co. CEO Dave Ricks called the $8 billion buyout of Loxo Oncology Inc. "completely in line with what we've been talking about all along," although it's a deal "bigger than we've done before. In this case, you have four either clinical or recently approved medicines in one package, which is a bit unique for us." The bundle comes with a platform that may prove more broadly useful, he pointed out during a conference call with investors.
The first question for Esperion Therapeutics Inc. CEO Timothy Mayleben during a conference call with investors Friday reflected skepticism, albeit mild, about the potential $1 billion-plus European licensing deal with Daiichi Sankyo Europe GmbH (DSE).
Codiak Biosciences Inc. scored its first partnership, putting ink to a contract with Dublin-based Jazz Pharmaceuticals plc to develop exosome-based therapeutics for cancer, "an area of biology that has really exploded in the last few years, probably starting with the realization that exosomes represent a fundamental communication system that cells use all the time, particularly in places like the tumor microenvironment," Codiak CEO Douglas Williams said. "There's a great understanding now that there's a lot of cross talk taking place between the tumor cells, the immune cells and the microenvironment, using exosomes as the vehicle."
Myokardia Inc. CEO Tassos Gianakakos said officials at his company "anticipated and were prepared for" Sanofi SA's decision not to extend the partnership begun in 2014, instead returning rights to pivotal phase III-stage candidate mavacamten (previously known as MYK-461), an oral, allosteric modulator of cardiac myosin to treat obstructive hypertrophic cardiomyopathy (HCM), as well as MYK-491, which works similarly and is expected to yield phase IIa proof-of-concept data in dilated cardiomyopathy (DCM) before the end of this year.
South San Francisco-based Denali Therapeutics Inc.'s dosing of the first patient in a phase Ib study with DNL-201 for Parkinson's disease (PD) brought renewed attention to the affliction and to the race with Glaxosmithkline plc (GSK), of London, in the development of leucine-rich repeat kinase 2 (LRRK2) inhibitors.
Capricor Therapeutics Inc. CEO Linda Marbán told BioWorld that the anaphylaxis case that caused her firm to suspend dosing voluntarily in the blinded, pivotal Hope-2 trial with stem cell therapy CAP-1002 was "likely caused by something in the infusion solution. Because the timing of the reaction was so immediate – literally within one minute of the beginning of the infusion – the immunologists that we've consulted do not believe it was related to cells, even if the patient was on cells."
