Two days after the U.S. FDA announced approval of gene therapy Lenmeldy (atidarsagene autotemcel), making it the first treatment option for rare disease metachromatic leukodystrophy, Orchard Therapeutics and parent firm Kyowa Kirin Co. Ltd. disclosed the wholesale acquisition price of $4.25 million for the one-time treatment, which edges out hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) to become the world’s most expensive drug.
An MHRA investigation that began in the U.K. 16 years ago has concluded with Kamlesh Vaghjiani, a former director of Kappin Ltd., being sentenced to concurrent prison sentences of eight and seven months, both of which are suspended for a year and a half.
Clearing the way for a U.S. regulatory bid in the second half of this year are positive top-line results from Pathfindr-2, the second of two successful phase III studies testing the efficacy and safety of Crinetics Pharmaceuticals Inc.’s oral, once-daily paltusotine for acromegaly.
The U.S. FDA approved Orchard Therapeutics plc’s BLA for gene therapy atidarsagene autotemcel, making it the first treatment option for metachromatic leukodystrophy in the U.S. The one-time treatment, branded Lenmeldy, is indicated for children with presymptomatic late infantile, presymptomatic early juvenile or early symptomatic early juvenile disease.
Spruce Biosciences Inc.’s results from two studies with tildacerfont in adult and pediatric classic congenital adrenal hyperplasia (CAH) spurred Wall Street to speculate – further, again – about the prospect’s odds against a drug in the works from Neurocrine Biosciences Inc.
Astrazeneca plc is acquiring rare diseases specialist Amolyt Pharma SA in a $1.05 billion deal, taking ownership of eneboparatide (AZP-3601), which is in phase III development for the treatment of hypoparathyroidism. Of the total, $800 million will be paid up front, with the balance of $250 million contingent upon achievement of a regulatory milestone.
Atrogi AB is raising a €30 million to €35 million (US$32.9 million to $38.4 million) series B round after announcing positive clinical data for ATR-258, a novel beta-2 adrenergic receptor agonist that is being lined up as a potential first-in-class insulin-independent treatment for type 2 diabetes.
An Italian group of researchers has used zinc finger editing to silence the PCSK9 gene and improve blood cholesterol levels in mice by applying a single dose of their modifier. The epigenetic-based method could be an alternative to genome editing.
The enticing prospect – and proven worth – of dually agonizing the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors gained more evidence in a big way from Viking Therapeutics Inc. with VK-2735 in a phase II weight loss study. Shares of San Diego-based Viking Therapeutics Inc. (NASDAQ:VKTX) closed Feb. 27 at $85.05, up $46.57, or 121%, as investors learned that the drug achieved the primary and all secondary endpoints in the phase II study called Venture, with significant body-weight drops at all doses compared to placebo.
Synlogic Inc. jolted Wall Street with news that the firm is scrapping for futility Synpheny-3, its pivotal study with labafenogene marselecobac (SYNB-1934) for phenylketonuria, and will evaluate strategic options. Shares of the Cambridge, Mass.-based firm (NASDAQ:SYBX) fell 48.7%, or $1.68, to end Feb. 9 at $1.77. Synlogic will cease operations and reduce its workforce by more than 90%, retaining only certain employees to help with the wind-down.
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