Prime Medicine (PRME) showcased its prime editing platform and clinical development timeline at the H.C. Wainwright biotech conference on Tuesday, as shares fell 4.4% to $3.04.
The company, founded out of David Liu’s laboratory at Harvard, uses a reverse transcriptase enzyme and RNA template to write changes directly into DNA. CEO Allan described the approach as "the most versatile, and the safest way to edit the genome" compared with traditional nuclease-based CRISPR, which causes double-strand breaks and chromosomal rearrangements, or base editing, which carries bystander-editing risks.
The platform handles transition and transversion mutations, missense mutations, frameshifts, hotspot editing, repeat expansions, and large gene inserts via a landing-pad approach called PASSIGE technology.
On the clinical front, Prime Medicine's lead program targets chronic granulomatous disease (CGD) through an ex vivo approach. Two patients have been treated so far, with one additional adolescent dose planned. The company aims to file a Biologics License Application in the first half of 2025 and said the FDA has signaled potential approval based on data from just two patients, with some chemistry, manufacturing, and controls work deferred post-approval.
Its first in vivo liver program, targeting Wilson's disease, has an IND in hand. First-patient dosing is expected within two to three months of IND acceptance, with initial data targeted for 2027. The program will use a radio-labeled copper PET scan as a functional readout at baseline and six to eight weeks post-dose. The primary mutation target is H1069Q; a second target, R778L—which is more common in Asian patients—is supported by a potent editor already in hand.
A second in vivo liver program for alpha-1 antitrypsin deficiency plans a regulatory filing in the third quarter of 2024, with clinical data also expected in 2027.
Prime Medicine reported a cash position of just under $110 million, with runway extending into 2027 excluding non-dilutive funding sources, priority review vouchers, and potential milestone payments. The stock has fallen nearly 11% over the past week and trades between a 52-week high of $6.94 and a low of $2.67. Its beta stands at 2.35.
Allan characterized the company's pipeline as a flywheel that should create value as it expands into additional indications.
