CRISPR Therapeutics AG CRSP reported new topline study results on Wednesday to establish risk factors for atherosclerotic heart disease.
The company disclosed data regarding the first 10 patients across its Phase 1 first-in-human dose escalation trial, targeting ANGPTL3 in four patient groups with elevated low-density lipoprotein (LDL), triglyceride (TG), or both.
The data comes from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg], DL2[0.3 mg/kg], DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg]) with at least 30 days of follow-up for each participant as of a data cutoff date of April 16.
A single dose of CTX310 demonstrated dose-dependent decreases in ANGPTL3, TGs, and LDL.
- Based on ANGPTL3 knockdown, DL1 and DL2 were minimally active doses, whereas treatment at DL3 and DL4 reduced up to 75% of baseline levels in ANGPTL3.
- A DL4 patient with sHTG had an 82% reduction in triglycerides from a baseline of 1073 mg/dL at day 30, and a DL3 patient with HeFH had an 81% reduction in LDL-C from a baseline of 256 mg/dL at day 90 – supporting the potential for targeted efficacy in high-risk populations.
CTX310 has been well-tolerated, with no treatment-related severe adverse events (SAEs) and no grade ≥3 adverse events (AEs) reported.
- No clinically significant changes in alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, or platelets were observed at any dose level. There were no dose-dependent trends in any of these laboratory measurements.
The company plans to present the CTX310 Phase 1 data at a medical meeting in the second half of 2025.
Chardan Research lowered the price target from $84 to $82 on Wednesday, with a Buy rating.
Chardan analyst Geulah Livshits writes, “Topline PI results for CTX310 indicate safety and activity, though more detailed data in 2H25 will help understand competitiveness.”
On Wednesday, CRISPR reported that cash, cash equivalents, and marketable securities were $1.85 billion as of March 31.
The topline readout did not include patient-level data. They also comprise of results from patients with a range of disease pathologies, which could contribute to variability. However, the biomarker numbers generally align with “benchmarks set by RNAi assets for ANGPTL3,” Livshits adds.
The CRISPR’s update, plus Verve Therapeutics Inc’s VERV data on its second-generation PCSK9 gene editor, points to a promising balance of benefits and risks for gene-editing treatments.
The analyst also combed through CBER Director Vinay Prasad‘s social media. Commentary reveals criticism of Verve’s 1st-gen PCSK9 approach/data. This may signal negative sentiment for advanced therapies that CRSP, Verve and other players might need to navigate, Livshits says.
Health Secretary Robert F. Kennedy picked Prasad to be the Food and Drug Administration’s vaccine regulator. Prasad replaces former CBER Director Peter Marks, who Kennedy forced to resign.
Price Action: CRSP stock is up 2.99% at $34.15 at the last check Wednesday.
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