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.
The $65 million series A money banked by Vicinitas Therapeutics Inc. will boost efforts with the company’s Deubiquitinase Targeting Chimera (DUBTAC) platform, developed by way of an academic-industry research collaboration between the Novartis Institutes for Biomedical Research and researchers at the University of California, Berkeley.
The U.S. FDA’s summer 2021 approval of Skytrofa (lonapegsomatropin) from Ascendis Pharma A/S served to sharpen appetites for an even better therapy to treat for pediatric growth hormone deficiency (GHD). Among players in the forefront is Lumos Pharma Inc., with an oral candidate that could disrupt the competitive space.
Wall Street apparently wants to see longer-term data from Bridgebio Pharma Inc. with oral infigratinib in children with achondroplasia (ACH) before deciding about the drug’s chances against the approved therapy Voxzogo (vosoritide) from Biomarin Pharmaceutical Inc.
Ipsen SA’s $247 million buyout of Epizyme Inc., and the recent decision by Nordic Nanovector SA to dump its phase IIb program with CD-37-targeted Betalutin (177Lu lilotomab satetraxetan), served to highlight the hot space of relapsed/refractory follicular lymphoma, where bispecifics have been showing particular promise.
With enrollment set to finish any day in Selecta Biosciences Inc.’s Dissolve II study testing SEL-212 in chronic refractory gout (CRG), investor appetite runs high in the space, as contenders line up to take on Horizon Therapeutics plc’s Krystexxa (pegloticase), the only product approved for CRG.
