Prime Medicine Announces First Patient Dosed in Global Phase 1/2 Clinical Trial of PM577a for H1069Q-Mutated Wilson Disease
Key Highlights
- ➤First patient dosed in global Phase 1/2 trial of PM577a for Wilson disease
- ➤FDA grants Rare Pediatric Disease designation to PM577 for Wilson disease
- ➤PM577a targets H1069Q, the most prevalent Wilson disease-causing mutation in North America and Europe
- ➤Initial clinical data expected in 2027
- ➤Follow-on R778L candidate targets the most common mutation in East Asian populations
Expert Statements
Allan Reine, Chief Executive Officer of Prime Medicine
“Dosing the first patient with PM577a is a meaningful step toward our goal of delivering a transformative, one-time treatment for people living with Wilson disease”
Allan Reine, Chief Executive Officer of Prime Medicine
“We’ve seen strong interest from patients and physicians that, together with our patient identification and prescreening efforts, positions us well for efficient enrollment.”
Allan Reine, Chief Executive Officer of Prime Medicine
“More broadly, PM577a reflects the modularity of our platform.”
Allan Reine, Chief Executive Officer of Prime Medicine
“While this initial candidate is designed to correct H1069Q, the most prevalent disease-causing mutation in the United States and Europe, the same shared LNP delivery approach provides a path to expand into additional Wilson disease patient populations globally, beginning with our follow-on candidate designed to correct the R778L mutation, the most common mutation in East Asian populations.”
Mohammed Asmal, Chief Medical Officer of Prime Medicine
“Wilson disease is a serious, progressive genetic disorder with no approved curative therapy.”
Mohammed Asmal, Chief Medical Officer of Prime Medicine
“Current pharmacologic treatments require lifelong management and can be limited by tolerability and adherence challenges”
Mohammed Asmal, Chief Medical Officer of Prime Medicine
“PM577a is designed to correct the H1069Q mutation at its genetic root through a single administration, with the potential to offer patients a one-time curative treatment, ideally before irreversible liver and neurological damage occur.”
Mohammed Asmal, Chief Medical Officer of Prime Medicine
“Because Wilson disease frequently presents during childhood and adolescence, we are particularly encouraged by the FDA’s Rare Pediatric Disease designation for PM577, which underscores the significant unmet need for therapies capable of altering the course of disease early in life.”
-- Global, first-in-human study underway designed to evaluate safety, tolerability, and initial clinical efficacy of PM577a --
-- PM577a is designed as a one-time therapy to correct the H1069Q mutation in ATP7B, the most prevalent Wilson disease-causing mutation in North America and Europe --
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