Results from a PHASE3 study (NCT05305664) investigating Pelacarsen in patients with Hyperlipoproteinemia(a) were posted on 2026-07-17. The trial demonstrated that Pelacarsen significantly reduced the mean rate of lipoprotein apheresis sessions by 83% compared to placebo over 52 weeks.

Background

Pelacarsen was investigated for its ability to lower lipoprotein(a) levels and reduce the need for lipoprotein apheresis. The study focused on patients with Hyperlipoproteinemia(a) and established cardiovascular disease who were already undergoing weekly lipoprotein apheresis in Germany.

Trial design

The study, identified as NCT05305664, was a PHASE3 trial that enrolled 51 participants with Hyperlipoproteinemia(a) and established cardiovascular disease. The trial aimed to test the hypothesis that treatment with Pelacarsen (TQJ230) 80 mg Q4W would significantly reduce the rate of lipoprotein apheresis compared to placebo in patients undergoing weekly apheresis.

Key results

The trial reported several key outcomes related to lipoprotein apheresis sessions and Lp(a) levels:

  • Rate of Lipoprotein Apheresis Sessions Performed Over 52 Weeks: The mean rate of apheresis sessions was 0.16 sessions per week for the Pelacarsen (TQJ230) group, compared to 0.93 sessions per week for the Placebo group. A fractional regression model analysis showed an Odds Ratio (OR) of 0.006 (95% CI: 0.003, 0.013) with a p-value of 0.0001.
  • Time to Lipoprotein Apheresis Avoidance: The median time to lipoprotein apheresis avoidance was 6.14 weeks for the Pelacarsen (TQJ230) group, while it was NA for the Placebo group. A Cox regression analysis yielded a Hazard Ratio (HR) of 88.26 (95% CI: 4.71, 1653.15) with a p-value of 0.0014.
  • Number of Participants With Total Lipoprotein Apheresis Avoidance From Week 12 to Week 52: 18 participants in the Pelacarsen (TQJ230) group achieved total apheresis avoidance, compared to 0 participants in the Placebo group. A logistic regression analysis showed an Odds Ratio (OR) of 163.2 (95% CI: 7.66, 3477.25) with a p-value of 0.0005.
  • Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as mg/dL: The geometric mean ratio from baseline was 0.32 for Pelacarsen (TQJ230) and 1.11 for Placebo. An ANCOVA analysis reported a geometric mean ratio of 0.28 (95% CI: 0.21, 0.39) with a p-value of 0.0001.
  • Change From Baseline to Week 52 in the Log-transformed Lp(a) Reported as Nmol/L: The geometric mean ratio from baseline was 0.27 for Pelacarsen (TQJ230) and 1.14 for Placebo. An ANCOVA analysis reported a geometric mean ratio of 0.23 (95% CI: 0.16, 0.33) with a p-value of 0.0001.
  • Rate of Lipoprotein Apheresis Sessions From Week 12 to Week 52: The mean rate of apheresis sessions was 0.11 sessions per week for the Pelacarsen (TQJ230) group, compared to 0.93 sessions per week for the Placebo group. A fractional regression model analysis for this period showed an Odds Ratio (OR) of 0.003 (95% CI: 0.001, 0.011) with a p-value of 0.0001.

What this means

The results indicate that Pelacarsen substantially reduced the frequency of lipoprotein apheresis sessions and enabled complete avoidance for a significant proportion of patients with Hyperlipoproteinemia(a). The observed reduction in Lp(a) levels correlates with the clinical benefit, suggesting that Pelacarsen could offer a meaningful therapeutic alternative or adjunct for patients reliant on apheresis.

Source

This information was sourced from ClinicalTrials.gov, documenting the results of trial NCT05305664, which were posted on 2026-07-17. The study details are available on clinicaltrials.gov.