The phase III miss disclosed Aug. 11 by Kubota Pharmaceutical Holdings Co. Ltd. subsidiary Kubota Vision Inc. in Stargardt disease put more eyes on the rare, inherited, juvenile-onset form of macular degeneration, for which nothing is approved.
The U.S. FDA’s effort to push companies toward more and better randomized, controlled trials ahead of accelerated approvals – apparently driven by the lack of confirmatory studies done afterward – is “an important and meaningful move by the agency,” said Day One Pharmaceuticals Inc. CEO Jeremy Bender. “The industry’s history in that space has been a little mixed.” Bender’s remarks came Aug. 9 as part of a wide-ranging panel discussion hosted by analyst Robert Driscoll during the Wedbush Pacgrow Healthcare Conference.
Prometheus Biosciences Inc.’s stock-boosting news July 26 put more investor eyes on inflammatory bowel disease (IBD), where the company during its R&D Day revealed the target of PRA-052, the clinical candidate that emerged from the program known as PR-600. PRA-052 takes aim at the CD30 ligand, which acts as a co-stimulatory molecule to drive T-cell proliferation and differentiation.
Shares of Poseida Therapeutics Inc. (NASDAQ:PSTX) closed Aug. 3 at $4.51, up $2.08, or 85%, as a result of the collaboration and licensing deal with Roche Holding AG that brings $110 million up front as well as the same amount in near-term milestone payments described by CEO Mark Gergen as “highly achievable,” and the arrangement could be worth as much as $6 billion if goals farther down the road are met.
Doctors will “unequivocally view as a major advance” Cincor Pharma Inc.’s oral baxdrostat for patients with treatment-resistant hypertension, said Deepak Bhatt, director of interventional cardiovascular programs at Brigham and Women’s Hospital and professor at Harvard Medical School. “There are a lot of these folks out there, and physicians don’t know what to do with them right now,” he said, noting that many end up in emergency rooms.
With data due later this summer from the phase II Pioneer trial testing Blueprint Medicines Corp.’s approved Ayvakit (avapritinib), many investor eyes are on the potential label expansion into indolent systemic mastocytosis – but the company has another potential ace in the hole with BLU-451, which targets EGFR exon 20 insertion mutations in non-small-cell lung cancer (NSCLC).
Shares of Poseida Therapeutics Inc. (NASDAQ:PSTX) closed at $4.51, up $2.08, or 85%, as a result of the collaboration and licensing deal with Roche Holding AG that brings $110 million up front as well as the same amount in near-term milestone payments described by CEO Mark Gergen as “highly achievable,” and the arrangement could be worth as much as $6 billion if goals farther down the road are met.
Idrx Inc. launched with a $122 million oversubscribed series A round to boost precision drug combinations in cancer, with a first focus on non-PDGFR-driven gastrointestinal stromal tumors (GIST). The Plymouth, Mass.-based firm aims to develop a pairing, potentially with add-ons, powerful enough to handle existing mutations and those that turn up during treatment. Combo therapy attacks “not just the driver mutations, but also the key secondary mutations,” co-founder and CEO Ben Auspitz told BioWorld, and thereby “block every escape route the cancer has.”
As Marengo Therapeutics Inc. gears up to start human trials with its lead compound, the company nailed down a deal with Ipsen SA worth $45 million up front and as much as $1.59 billion-plus in milestone rewards to advance a pair of candidates from the Selective T Cell Activation Repertoire (STAR) platform into the clinic.
Wall Street must wait to learn more about factors that might distinguish the Claudin18.2-targeting asset at the center of Elevation Oncology Inc.’s $1.2 billion license deal with CSPC Megalith Biopharmaceutical Co. Ltd., a subsidiary of CSPC Pharmaceutical Group Ltd.
