Word that big pharma firms are sniffing around Horizon Therapeutics plc as a takeover candidate caused not only that firm’s shares to jump but also provided a boost for Viridian Therapeutics Inc., which has a thyroid eye disease (TED) candidate set to challenge Horizon’s Tepezza (teprotumumab-trbw), cleared by the U.S. FDA in January 2020.
Wall Street’s reaction to phase I news in chondrosarcoma from Inhibrx Inc. puzzled some onlookers and brought renewed attention to the rare disease, which is also the most common form of primary bone cancer in adults. Big pharma and smaller concerns have been throwing drug prospects at the condition for years without much luck.
Rgenta Therapeutics Inc.’s $52 million in a series A money will let the RNA-focused firm pursue its small-molecule drug efforts “for the next two or three years,” as candidates in cancer and neurology make their ways toward the clinic, said co-founder and CEO Simon Xi. “We’ll go where the science leads us,” he told BioWorld, adding that the cash on hand is sufficient to complete a phase I study.
Shares of Cincor Pharma Inc. (NASDAQ:CINC) took a serious hit on top-line findings from the completed phase II Halo study with once-daily oral baxdrostat, but the clinical program with the aldosterone synthase inhibitor remains on track, and an NDA submission is targeted for 2025. The stock closed at $14.11, down $12.42, or 46.8% after Waltham, Mass.-based Cincor informed Wall Street that results from Halo, which enrolled patients taking as many as two blood pressure medications at the maximally tolerated doses without satisfactory results, missed statistical significance on its primary endpoint evaluating change from baseline in mean seated systolic blood pressure in the intent-to-treat population, which included 249 patients.
Avenue Therapeutics Inc.’s takeover of Baergic Bio Inc. pursuant to the previously disclosed share contribution agreement with its parent company, Fortress Biotech Inc., highlighted the potential of targeting GABAA, an approach under investigation in various quarters.
The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
Merck & Co. Inc. is adding to its hematology assets in the takeover of Imago Biosciences Inc. for $36 per share, an arrangement with an equity value of about $1.35 billion. Shares of Imago (NASDAQ:IMGO) took flight as Wall Street learned of the deal, ending the day at $35.59, up $18.19, or 104%. Imago is working on new drugs for myeloproliferative neoplasms and other bone marrow diseases. Lead candidate bomedemstat (IMG-7289), an orally available inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1), is undergoing phase II trials for the treatment of essential thrombocythemia, myelofibrosis and polycythemia vera, along with other indications.
The stock rally by Tango Therapeutics Inc. over the past month or so has further revved the long-percolating interest in protein arginine methyl transferase 5 (PRMT5) and in synthetic lethality, where a number of parties have programs ongoing.
Provention Bio Inc. scored approval from the U.S. FDA of the BLA for Tzield (teplizumab-mzwv), an intravenously given, anti-CD3-directed antibody, as the first and only immunomodulatory treatment to delay the onset of stage 3 type 1 diabetes (T1D) in adult and pediatric patients ages 8 and older with stage 2 T1D.
As Wall Street waits to find out later this year whether Stoke Therapeutics Inc.’s positive results with a low, single dose of STK-001 for Dravet syndrome pans out in more extensive research, a number of players large and small are investigating candidates for the rare but dismal form of epilepsy.
