Two days before the PDUFA date, the U.S. FDA handed down a complete response letter (CRL) for Rocket Pharmaceuticals Inc.’s Kresladi (marnetegragene autotemcel), delaying potential approval of the lentiviral-based gene therapy as the first therapeutic option for leukocyte adhesion deficiency type I, a rare, inherited immune disorder. But the Cranbury, N.J.-based company has suggested that delay won’t be long, as the CRL requests only “limited” chemistry manufacturing and controls (CMC) information – additional CMC data were also cited as the reason for the three-month review extension earlier this year. Read More
Alumis Inc. began trading (NASDAQ:ALMS) earlier this morning, with the company’s IPO debuting at $16 per share to raise $210 million, with another $40 million coming through a concurrent private placement at the same price. That was the lower end of its previously announced price range, which had stretched as high as $18 per share. Read More
Just seven months after in-licensing ocedurenone (KBP-5074) from KBP Biosciences Pte. Ltd. in a $1.3 billion deal, Novo Nordisk A/S halted its late-stage study after ocedurenone failed to significantly control hypertension in chronic kidney disease patients. Read More
On the heels of an AU$100 million (US$66.5 million) capital raise, Immutep Ltd. announced positive top-line results in first-line head and neck cancer with its lead candidate, eftilagimod, in combination with Merck & Co. Inc.’s Keytruda (pembrolizumab). Read More
CSL Behring’s expensive hemophilia B gene therapy is to be reimbursed by the U.K. National Health Service, after the company agreed to an outcomes-based payment scheme. The therapy, Hemgenix (etranacogene dezaparvovec), which has a U.K. list price of £2.6 million (US$3.3 million), was approved under a managed access scheme, in which data will be collected over five years to enable both the long-term effectiveness, and any adverse liver toxicity caused by the transgene, to be monitored. Read More
In May 2024, BioWorld covered 298 updates across phase I-III clinical trials, surpassing March (261), February (236) and January (252), though falling short of April’s high of 323. Additionally, at the end of May BioWorld revised its reporting criteria for clinical trial updates, focusing mainly on data readouts and excluding trial initiations, enrollment changes and initial patient dosing, thereby impacting the update count relative to prior months. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Bicara, Calcimedica, Inmagene, Medivir, Novo Nordisk, Repare, Silence, Vogenx, X4. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: 2seventy, Abbvie, Bluesphere, BMS, CASI, Celsius, Coherus, Lykos, Meitheal, Novo, Oncopeptides. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Abdera, Daiichi Sankyo, Immunovant, Merck, Recode, Tubulis. Read More
