Despite the approval of Biogen Inc.’s Qalsody (tofersen) for treating amyotrophic lateral sclerosis (ALS), there are still hurdles for the drug to clear, including a confirmatory study and setting a price. Because the U.S. FDA granted Qalsody accelerated, not full approval, there are plenty more data to collect in the ongoing confirmatory phase III Atlas study of those who develop ALS symptoms during the trial compared to placebo. The randomized, double-blind, placebo-controlled study is being conducted with those carrying the superoxide dismutase 1 gene genetic mutation but are symptom free. Read More
Orbital Therapeutics Inc. raised $270 million in a series A round to fund a big push into the next generation of mRNA-based therapies. The Cambridge, Mass.-based firm is building out a comprehensive RNA platform from which it will launch programs in oncology, autoimmune disease and indications involving protein replacement approaches. Read More
The industry has hit out at the European Commission’s proposals for new pharmaceuticals regulations, saying they risk “sabotaging” life sciences in Europe. “Today’s proposals manage to undermine research and development in Europe while failing to address access to medicines for patients,” said Nathalie Moll, director general of the European Federation of Pharmaceutical Industries and Associations. Read More
U.S. Centers for Medicare & Medicaid Services (CMS) Administrator Chiquita Brooks-LaSure made her first appearance April 26 before the House Energy and Commerce’s Subcommittee on Health, ostensibly to discuss legislative solutions to increase transparency and competition in health care. But member after member, regardless of political party, demanded answers about why CMS continues to severely restrict access to Eisai Co. Ltd.’s Alzheimer’s drug, Leqembi (lecanemab), especially since another government agency is covering it for all veterans that meet the labeling requirements. Read More
Albatroz Therapeutics Pte Ltd. has secured $3 million in seed funding to develop therapeutic antibodies against a new target that degrades the extracellular matrix, a key contributor to cancer and arthritis. Read More
The U.S. Supreme Court has declined to hear a case that tests the notion that artificial intelligence (AI) can be an inventor, a development that may be nothing more than the beginning of the AI-as-inventor story under U.S. law. The Patent and Trademark Office’s (PTO) April 25 webinar on the subject included some remarks that AI could be used to produce a tsunami of potentially duplicative patent applications, but the event demonstrated that there is almost no at-large support for AI-as-inventor, suggesting that the status quo will stand for the time being. Read More
It used to be a lot easier to find partners. After two years of strong dealmaking, the volume and value of partnerships slid in the first quarter of 2023. M&As also are down compared to prior years. But there are some reasons to be cheerful. Looking at first quarter numbers and surveying the big picture on this BioWorld Insider podcast are Karen Carey, BioWorld’s managing editor and senior data analyst, along with Tim Shannon, a general partner at Canaan. Carey just wrote about biopharma deals and completed M&As in the first quarter. Shannon, who’s been with Canaan since 2009, has a new fund sporting $850 million in new capital, including an oversubscribed $650 million fund for seed and series A financings. They both offer their insights into the first quarter’s numbers and what may lie ahead. Read More
New hires and promotions in the biopharma industry, including: Alloy, Entera, Epsilogen, Finch, Immutep, Linkinvax, NRG, Oculis, Oxford Biomedica, Purple, Systimmune, Xencor. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Amo, Aphaia, Arrowhead, Artelo, Atsena, Baudax, Erasca, First Wave, Hillevax, Lyra, Morphic, Okyo, Omeros, Parthenon, Samsung, Trefoil, Ultimovacs, Vaccinex. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Aldena, Algorithm, Alkermes, Endo, Evelo, Hoth, J&J, Liuqiang, Nanoviricides, Provention, Sable, Sanofi, Slayback, TC. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Arbutus, Atea, GSK, Hipra, In8bio, Invivyd, Myrtelle, Santhera, Stoke, Tarsier, Virios. Read More
