Shares of Marinus Pharmaceuticals Inc. climbed 51.7% to close at $3.20 on Sept. 15 following news that the company's sole clinical asset, ganaxolone, met the primary endpoint of its phase III Marigold study in CDKL5 deficiency disorder, a rare genetic condition that can leave children unable to walk, talk or feed themselves. Read More

The FDA has granted Plus Therapeutics Inc. fast track designation for its lead candidate, Rhenium Nanoliposomes (RNL), for treating recurrent glioblastoma, propelling it into the sixth cohort of a phase I dose-finding trial. Read More

Antibody-drug conjugates (ADCs) are making a comeback. After a relatively slow start with Adcetris (brentuximab vedotin, Seattle Genetics Inc.) and Kadcyla (ado-trastuzumab emtansine, Roche Holding AG) approved by the FDA in 2011 and 2013, respectively, the regulatory activity has swelled with four FDA approvals over the last nine months. Read More

HONG KONG – Osaka-based Takeda Pharmaceuticals Co. Ltd. has divested a portfolio of non-core prescription pharmaceuticals products to Greifswald, Germany-based Cheplapharm Arzneimittel GmbH as part of an ongoing program intended to optimize and simplify its portfolio. Read More

Researchers at the University of California at San Diego have used RNA-targeted CRISPR to reverse symptoms in an animal model of myotonic dystrophy type 1 (DM1). They reported their findings in the Sept. 14, 2020, issue of Nature Biomedical Engineering. Read More

What’s the plan? The U.S. CDC is being asked that question a lot these days – not just about COVID-19, but also about preparing for what could be a tough influenza season as flu bugs circulate with the coronavirus. Read More

The latest global regulatory news, changes and updates affecting biopharma, including: NIH adds funding to Latino Alzheimer’s research; Sprout warned for Addyi promotion; Scripps to pay $10M over use of NIH grants. Read More

New hires and promotions in the biopharma industry, including: Adverum, Aravive, Atea, Aviceda, Haima, Immatics, Kronos, Matinas, Miragen, Nextcure, Partner, Rhythm, Rockwell, Veracyte, Voyager, Vtv, Tizona, Ziopharm. Read More

Biopharmas raising money in public or private financings, including: 89bio, Ansa, Aziyo, Hexagon, Humanigen, Imcheck, Novaremed, Proteodesign, Sunshine. Read More

Clinical updates, including trial initiations, enrollment status and data readouts and publications: Adocia, Aerpio, Arbutus, Atossa, Atyr, Axsome, Clinuvel, Codiak, Curasen, Curevo, Galecto, Kyorin, Marinus, Opko, Teva, TFF, Tyme. Read More

Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Adicet, Alimera, Anelixis, Applied Stemcell, Axovant, Bayer, Biontech, BMS, Bostongene, C3.ai, Cell and Gene Therapy Catapult, Cellinta, Combigene, Corino, Cullinan, Cue, ERS Genomics, Exelixis, Fosun, Genexine, Humanigen, Iconic, Innovation Pharma, Kiadis, Mabion, Medis, Melior, Mindset, Miragen, Momenta, Mylan, Novavax, Novus, Obsidian, Oligomerix, Pfizer, Protokinetix, Restorbio, Serum Institute of India, Soligenix, Systems Oncology, Teva, Tonix, Vaxine. Read More

Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Aerie, Amryt, Apellis, Ardelyx, Astrazeneca, BMS, Boehringer, Epimab, Equillium, Gensight, Gilead, Kiniksa, Lilly, Mateon, Matinas, Novartis, Plus, Takeda, Zynerba. Read More