Umoja Biopharma Inc.’s gene delivery platform that combines a third-generation lentiviral vector gene approach with a novel T-cell targeting and activation surface complex brought Abbvie Inc. to the table for a pair of deals that could be worth as much as $1.44 billion.
With positive initial phase I/II data in hand from two trials, Dyne Therapeutics Inc. plans to report more findings and start enrolling registrational cohorts in both studies by the end of this year for DYNE-101 in myotonic dystrophy type 1 (DM1) and a study called Deliver with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Longboard Pharmaceuticals Inc.’s positive – and then some – phase Ib/IIa top-line data with 5-HT2C receptor superagonist bexicaserin (LP-352) in developmental and epileptic encephalopathies (DEEs) sparked Wall Street speculation about competitive odds as well as the shape of the firm’s upcoming phase III effort.
Pharma-biotech pairings continued apace in the antibody-drug conjugate (ADC) space, with 2023 capped by Legochem Biosciences Inc. signing a $1.7 billion licensure deal with Johnson & Johnson arm Janssen Biotech Inc. for the former’s Trop2-directed compound, the second-biggest Korean technology transfer agreement.
The long, strange and nowhere-near-concluded trip taken by psychedelic drugs as a therapeutic modality continued in 2023, with regulators in the U.S. and Europe – and, perhaps in greater numbers, investors – warming to prospects in a space that once drew only laughter.
What Piper Sandler analyst Christopher Raymond called the “shocking” decision by Allovir Inc. to scrap development of posoleucel – which had advanced to three phase III trials – for all indications sent shares of the firm (NASDAQ:ALVR) in a tailspin, closing Dec. 22 at 76 cents, down $1.56, or 67%.
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.
Calliditas Therapeutics AB’s full approval from the U.S. FDA for Tarpeyo (budesonide) delayed release capsules in immunoglobulin A nephropathy (IgAN) revived speculation about competitor Travere Therapeutics Inc. which, like Calliditas, has gained accelerated approval for its prospect.
Argenx SE CEO Tim Van Hauwermeiren said that, with subcutaneous efgartigimod (efgartigimod alfa and hyaluronidase-qvfc) in pemphigus, the company is “facing a situation where, even with a strong scientific hypothesis and well-executed trial, we encountered the unknown-unknown,” and the phase III experiment failed. “We are committed to doubling down” on the execution of the firm’s business plan, he added.
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.
