Good news from San Diego-based Acadia Pharmaceuticals Inc.'s phase III trial with Nuplazid (pimavanserin) in dementia-related psychosis (DRP) had analysts trotting out music metaphors in reports about the study called Harmony, while talking already about would-be sales of the drug, a selective serotonin inverse agonist and antagonist that preferentially targets the 5-HT2A receptor.
Findings disclosed from Synlogic Inc.'s phase Ib/IIa trial with SYNB-1020 in hyperammonemia surprised the company and Wall Street, dealing a blow to the compound but leaving in place the Synthetic Biotic platform, which genetically engineers probiotic microbes.
Vertex Pharmaceuticals Inc.'s deal to take over Cambridge, Mass.-based Semma Therapeutics Inc. for $950 million in cash – thereby bringing aboard a potential cure for type 1 diabetes – may have brought to mind another Cambridge player in the space: Sigilon Therapeutics Inc., which inked a whopper deal with Eli Lilly and Co. in April of last year.
CEO Travis Mickle said prodrug-focused Kempharm Inc.'s potential $493 million deal with Gurnet Point Capital (GPC) marks "the end of a long journey to reach a very valuable point" in the story of the company, which has "ended with the same goal it started with" – gaining the best possible partner for attention deficit and hyperactivity disorder (ADHD) prospects KP-415 and KP-484.
Shares of San Diego-based Cidara Therapeutics Inc. (NASDAQ:CDTX) enjoyed a skyward ride, closing Tuesday at $2.35, up 67 cents, or 40%, on word of a deal with Mundipharma International Ltd., of Cambridge, U.K., to develop and commercialize rezafungin for invasive fungal infections. "Historically, [antifungal] pricing has been much stronger in the EU, so that is an important opportunity for us and now for Mundipharma as well," Cidara CEO Jeffrey Stein said.
Merus N.V.'s second-quarter earnings report included an important update on phase I MCLA-117, which Jefferies analyst Maury Raycroft said could be a best-in-class "game-changer" for the treatment of acute myeloid leukemia (AML) – a candidate that "alone is a reason to own the stock," in his view.
Newark, Calif.-based Protagonist Therapeutics Inc.'s third-quarter earnings call included an update on hepcidin mimetic PTG-300 in beta-thalassemia, but other prospects in the pipeline are starting to excite investors as well.
Kidney-disease player Chinook Therapeutics Inc. CEO Eric Dobmeier told BioWorld that the company's $65 million series A round provides "a few years of money at the current pace. I'm hopeful we're going to make progress, have really good use of proceeds, and be out there raising money again to advance the company, because there's just such a huge unmet need right now."
The FDA cleared Rozlytrek (entrectinib) for the treatment of adults with ROS1-positive, metastatic non-small-cell lung cancer, also granting accelerated approval to the tyrosine kinase inhibitor for adult and pediatric patients (12 and older) with solid tumors that have a NTRK gene fusion.
