Prime Medicine stock surges on FDA IND clearance

Investing.com -- Prime Medicine Inc (NASDAQ: PRME) shares gained over 12% in after-hours trading Thursday following the FDA's clearance of the company's Investigational New Drug application for PM647, an in vivo gene editing therapy for Alpha-1 Antitrypsin Deficiency.

PM647 is designed to correct the E342K mutation in the SERPINA1 gene, the most common cause of AATD. The therapy aims to restore production of functional M-AAT and address both liver and lung manifestations of the disease with a one-time intravenous infusion.

The planned Phase 1/2 clinical trial will be a global, single-arm, open-label study evaluating the safety, tolerability and preliminary efficacy of ascending doses of PM647 in adults with AATD. The study will initially enroll adult participants with lung-only manifestations of the disease. Upon demonstration of tolerability, the study will expand to include a separate cohort enrolling adults with significant liver disease, with or without concurrent lung manifestations.

The clearance marks the company's second regulatory milestone in recent months, following clearances for PM577a, its investigational program for Wilson disease. PM647 uses the same liver-directed lipid nanoparticle as PM577a.

AATD is an inherited genetic disorder caused by variants in the SERPINA1 gene. Approximately 200,000 people are estimated to carry the PiZZ genotype across the United States and Europe. Patients currently have no approved curative treatment that addresses the underlying genetic cause of both liver and lung manifestations of the disease.

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