The recent trading session observed a remarkable surge in the stock value of Prime Medicine, Inc. (NASDAQ: PRME), marking a substantial rise of 9.83% to $4.86. This noteworthy uptick in PRME stock price followed the regulatory clearance for a groundbreaking treatment.
On Monday, Prime Medicine (PRME) announced that the U.S. Food and Drug Administration (FDA) has approved the company’s application for an investigational new drug (IND) , PM359. The objective of this IND filing, which was made on March 29, is to treat chronic granulomatous disease (CGD). This approval empowers PRME to commence its global Phase 1/2 clinical trial in the United States.
The attainment of this pivotal milestone for its inaugural product candidate signifies the first-ever IND clearance for a Prime Editor product candidate, representing a significant stride in the domain of next-generation gene editing. Drawing upon data from Prime Medicine’s preclinical investigations, PM359 exhibits the potential to effectively rectify a prevalent disease-causing mutation of CGD, thereby potentially mitigating the disease burden for afflicted patients.
Prime Medicine is poised to initiate Phase 1/2 trials to further elucidate the potential therapeutic efficacy of PM359 in patients grappling with this debilitating ailment. The Phase 1/2 clinical trial constitutes a multinational, pioneering endeavor designed to evaluate the safety, biological activity, and preliminary efficacy of PM359 in both adult and pediatric participants.
Initial study cohorts will comprise adults exhibiting stable disease. Should safety and biological activity be demonstrated within this cohort, the trial protocol encompasses the enrollment of participants presenting with active infection or severe inflammation, alongside adolescent and pediatric participants.
Throughout the trial duration, participants will be subjected to rigorous safety monitoring, encompassing assessments of hematopoietic system engraftment and reconstitution, early indicators of restored immune function, as well as the long-term resolution and prevention of infectious and inflammatory complications associated with CGD. Prime Medicine anticipates disclosing preliminary clinical findings from the trial in the year 2025.
