Targeting cadherin-11 (cad-11) in rheumatoid arthritis (RA) could provide a complement to existing therapies, Adheron Therapeutics Inc. CEO Hari Kumar told BioWorld Today, and "that's exactly how we pitched it" to Roche AG, which is shelling out $105 million up front and potentially $475 million in milestone payments to take over the company, as toxicity studies continue ahead of phase II trials with SPD051.

Quartet Medicine Inc. CEO Kevin Pojasek told BioWorld Today that the finished series A round brought aboard "a financial investor with a long-term view, making sure we can carry this [program] as far as we want, really, into the future" research on tetrahydrobiopterin (BH4) as it relates to chronic pain and inflammation.Cambridge, Mass.-based Quartet snagged a final $6.25 million for a total of $23.25 million, drawing three new backers, including Remeditex Ventures LLC and a pair of undisclosed Shanghai-based funders.

CEO Vivek Ramaswamy told BioWorld Today that the special protocol assessment (SPA) from the FDA for Axovant Sciences Ltd.'s new phase III trial with RVT-101, a 5-hydroxytryptamine6 (5-HT6) serotonin receptor antagonist to treat Alzheimer's disease (AD), should bolster confidence that his firm is pursuing "the best-validated target" for the stubborn indication.

Chromocell Corp. CEO Christian Kopfli told BioWorld Asia that payments in his firm's potential $500 million deal with Astellas Pharma Inc. are "evenly spaced" along the development path for the oral Nav1.7 inhibitor CC8464 to treat neuropathic pain, for which trials are expected to start next year.

Spark Therapeutics Inc.'s pleasing phase III top-line data with its compound for blindness-causing, RPE65-mediated, inherited retinal dystrophies (IRDs) whetted investor appetite for more-detailed results and sent hope surging through the sector, as SPK-RPE65 could become the first gene therapy cleared for marketing in the U.S.

After what Amicus Therapeutics Inc. characterized last month as an upbeat pre-new drug application (NDA) meeting, U.S. regulators have sent word that the package for migalastat, its oral small-molecule pharmacological chaperone to treat the alpha-galactosidase A shortage in Fabry disease, needs to include more, and the company said the NDA won’t be submitted by the end of this year as planned.

Biomarin Pharmaceutical Inc. followed up its win over Sarepta Therapeutics Inc. in the patent dispute regarding Duchenne muscular dystrophy (DMD) therapies with news that the firm will regain global rights to the phenylketonuria (PKU) drugs Kuvan (sapropterin dihydrochloride) and Peg-Pal (pegvaliase) from Merck Serono.

Interim CEO Cary Pfeffer told BioWorld Today that Neon Therapeutics Inc.'s $55 million series A round will fuel the firm for "at least a couple of years," as researchers work to further exploit findings made possible by checkpoint inhibitors and target neoantigens in cancer.

Chromocell Corp. CEO Christian Kopfli told BioWorld Today that payments in his firm's potential $500 million deal with Astellas Pharma Inc. are "evenly spaced" along the development path for the oral Nav1.7 inhibitor CC8464 to treat neuropathic pain, for which trials are expected to start next year.

As the market awaits first data from a phase II trial in non-Hodgkin's lymphoma (NHL) around the middle of next year, Epizyme Inc. disclosed results showing that none of three patients with synovial sarcoma in the phase I trial with first-in-class EZH2 inhibitor tazemetostat showed a clinical response, but the firm is continuing with a phase II experiment in such patients as well.