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 (LAD-I), a rare, inherited immune disorder. The CRL requests “limited” chemistry manufacturing and controls (CMC) information – additional CMC data were also cited as the three-month review extension earlier this year – and Rocket said it has already met with the FDA’s Center for Biologicals Evaluation and Review to address the outstanding issues.

At low end of range, Alumis raises $250M in IPO, private placement

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. However, the IPO is a step forward as there were none from biopharma in May. The shares were going for $13.10 each, an 18% drop, at midday June 28. Alumis is developing two tyrosine kinase 2 inhibitors, ESK-001, a small molecule for moderate to severe plaque psoriasis, systemic lupus erythematosus and non-infectious uveitis, and A-005, a central nervous system penetrant molecule for neuroinflammatory and neurodegenerative diseases. The South San Francisco-based company, formerly Esker Therapeutics Inc., was founded in 2021.

Novo’s ocedurenone study flop ricochets, hits Mineralys stock

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. News of ocedurenone’s curtailed development dropped Bagsværd, Denmark-headquartered Novo shares (NASDAQ:NVO) down 2.2% to $143.67 June 26, although it inched up to $144.49 by June 27. Shares of hypertension drug-competitor, Radnor, Pa.-based Mineralys Therapeutics Inc. (NASDAQ:MLYS), fell nearly 9%. The setback is expected to cost Novo around DKK 5.7 billion (US$817.3 million) in impairment loss in the second quarter of 2024.

Immutep's eftilagimod phase IIb positive in head and neck cancer

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). A lymphocyte activation gene-3 fusion protein and major histocompatibility complex class II agonist, eftilagimod and Keytruda led to overall response rates that exceed Keytruda monotherapy across all levels of PD-L1 expression.

UK agrees to payment scheme for costly gene therapy Hemgenix

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.

May’s 298 clinical trial updates led by Astrazeneca, Hutchmed and Novartis

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. The average monthly count of phase I-III updates for the first five months of 2024 is 274, compared to a monthly average of 305 throughout 2023.

Also in the news

2seventy, Abbvie, Abdera, Bicara, Bluesphere, Brainstorm Cell, Calcimedica, CASI, Celsius, Coherus, Genmab, GRI, Harbour, Holoclara, Inmagene, Kala, Medivir, Meitheal, Merck, Neural, NMDP Biotherapies, Novo Nordisk, Oncopeptides, Purple, Recode, Repare, Silence, Tenaya, TME, Tonix, Tubulis, Vigil, Vogenx, X4