Nearly two years after Novartis AG signed a $1.7 billion deal for options to Voyager Therapeutics Inc.’s adeno-associated virus capsids for central nervous system disorders, the Basel, Switzerland-based company secured rights to develop gene therapies for Huntington’s disease and spinal muscular atrophy in a licensing agreement potentially worth $1.3 billion.
In July, Leqembi (lecanemab, Biogen Inc./Eisai Co. Ltd.) became the first amyloid-targeting drug to win traditional approval from the U.S. FDA, after getting accelerated approval in January based on the surrogate endpoint of plaque removal.
A new self-injectable therapy for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) will be available in January 2024 now that the U.S. FDA has approved Ionis Pharmaceuticals Inc.’s Wainua (eplontersen), a ligand-conjugated antisense oligonucleotide.
Adam Lenkowsky, chief commercial officer for Bristol Myers Squibb Co. (BMS), said his firm plans to launch Karxt (xanomeline-trospium) in the U.S. as soon as it’s approved by the U.S. FDA, and “expect[s] to accumulate sales in early 2025.” BMS tied a bow on the year by disclosing its plan to pay $330 per share to take over Karuna Therapeutics Inc. in a deal valued at $14 billion to bring aboard Karxt, which acts as a dual M1/M4 muscarinic acetylcholine receptor agonist. The FDA has assigned Sept. 26, 2024, as the PDUFA date for Karxt as a new treatment for schizophrenia in adults.
On-again, off-again investor enthusiasm for Uniqure NV’s Huntington’s disease (HD) gene therapy AMT-130 got another boost as the company followed this summer’s news from phase I/II trials with additional interim data. Shares of Uniqure (NASDAQ:QURE) closed Dec. 19 at $6.64, down $1.34, or 17%, as the company offered results on up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European experiment.
Neuren Pharmaceuticals Ltd. plans to progress NNZ-2591 to phase III trials following positive top-line phase II results in children with Phelan-McDermid syndrome, a genetic neurodevelopmental disorder for which there are no approved treatments.
Just over a year since the last anti-nerve growth factor (NGF) antibody program was axed, the target is poised for a revival, with Levicept Ltd. announcing it has completed recruitment to a phase II trial of LEVI-04 in the control of chronic pain caused by osteoarthritis.
All treatment groups in Vertex Pharmaceuticals Inc.’s phase II study of the non-opioid VX-548 showed meaningful reductions in pain from baseline in treating diabetic peripheral neuropathy (DPN), pointing the company toward pivotal phase III studies and prompting analysts to applaud enthusiastically.
Abbvie Inc.’s encore to its $10.1 billion buyout of antibody-drug conjugate specialist Immunogen Inc. took the form of another takeover – this time of Cerevel Therapeutics Inc. for $45 per share in cash, which puts the total equity value at about $8.7 billion and provides Abbvie with “one of the most attractive growth portfolios in the industry,” CEO Richard Gonzalez said.
China’s National Medical Products Administration (NMPA) approved Zhejiang Jingxin Pharmaceutical Co. Ltd.’s dimdazenil (EVT-201) for short-term treatment of insomnia. Jingxin filed the NDA in China in April 2022.
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