On Monday, CRISPR Therapeutics AG (NASDAQ:CRSP) presented clinical data demonstrating that a single-course treatment of CTX310 deeply reduced lipid levels in patients with severe hypertriglyceridemia.
These results reinforce the therapy’s potential to serve as a one-time gene editing treatment for this high-risk patient population.
The investigational therapy utilizes in vivo CRISPR/Cas9 editing to target angiopoietin-like protein 3 (ANGPTL3). At Day 30, a single course of CTX310 produced a 65% mean reduction in circulating ANGPTL3 and a 63% mean drop in triglycerides among participants with median baseline triglyceride levels exceeding 1,000 mg/dL.
Significant Reductions In Atherogenic Lipoproteins
The Phase 1b clinical trial evaluated 10 patients with severe hypertriglyceridemia who each received a single-course treatment of CTX310. All 10 participants reached at least 30 days of follow-up as of the data cutoff.
Along with triglyceride reductions, CTX310 generated substantial decreases across atherogenic lipoproteins. Participants achieved a 51% mean reduction in non-HDL-C and a 23% average drop in apolipoprotein B (ApoB).
Patients also achieved a 16% mean decrease in LDL-C from a low mean baseline of 60 mg/dL.
In participants monitored for at least 90 days, the reductions in ANGPTL3 and triglycerides persisted over time.
Notably, the study reported zero cases of acute pancreatitis, even though 30% of enrolled participants entered the trial with a history of the condition.
Favorable Safety Profile And Pipeline Expansion
CTX310 proved well tolerated among study participants. Seven patients completed at least 90 days of safety monitoring as of the data cutoff date.
The trial recorded no serious adverse events related to CTX310 and no Grade 3 or higher treatment-related adverse complications.
Furthermore, patients showed no clinically relevant elevations in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) liver enzymes.
One patient experienced a Grade 2 infusion-related reaction, which resolved and permitted the individual to complete the infusion.
CRISPR Therapeutics continues to advance CTX310 across multiple lipid disorders, with recruitment in additional Phase 1b cohorts actively ongoing.
Analyst Take
William Blair on Monday wrote, ” The Phase Ib CTX310 data at AHA came in ahead of our expectations, with the 63% triglyceride (TG) reduction at Day 30 comparing favorably with our roughly 50% expectation heading into the presentation.”
Analyst Myles Minter said, “With RMAT designation granted for CTX310 in sHTG and for elevated LDL-C, we believe the dataset has preliminary regulatory buy-in and look forward to additional Phase Ib trial updates.”
William Blair reiterates the Outperform rating on CRISPR shares.
CRSP Price Action: CRISPR Therapeutics shares were down 8.17% at $45.95 at the time of publication on Tuesday, according to Benzinga Pro data.
Photo by T. Schneider via Shutterstock
