Prime Medicine, Inc. (PRME) announced that New Zealand Medicines and Medical Devices Safety Authority (Medsafe) has cleared its Clinical Trial Application for PM577a, an investigational Prime Editing therapy targeting the H1069Q mutation in Wilson disease. This marks the company's first clinical authorization for an in vivo Prime Editing therapy, enabling initiation of a global Phase 1/2 study in the second half of 2026.
Wilson disease is a serious genetic disorder caused by mutations in the ATP7B gene, leading to copper buildup in the body. Current treatments require lifelong adherence and carry significant side effects. PM577a is designed as a one-time therapy that corrects the root cause at the genomic level, offering the potential for durable benefit.
The Phase 1/2 trial will evaluate safety, tolerability, and biological activity in adults and adolescents, with endpoints including copper efflux, serum ceruloplasmin, and hepatic copper levels. Initial clinical data are expected in 2027.
CEO Allan Reine called the clearance "a defining moment for Prime Medicine and the global Wilson Disease community," while CMO Mohammed Asmal highlighted the use of 64Cu PET imaging as a non-invasive functional readout of ATP7B activity.
Prime Medicine noted that its modular lipid nanoparticle (LNP) delivery platform may enable expansion into additional Wilson disease mutations, including R778L, which is more common in East Asian populations.
PRME has traded between $1.70 and $6.94 over the past year. The stock closed Wednesday's trading at $3.16, up 10.10%. In pre-market trading the stock is at $3.10, down 1.90%.
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