Results from a Phase 3 study (NCT05305664) investigating Pelacarsen in patients with Hyperlipoproteinemia(a) were posted on 2025-01-28. The trial demonstrated that Pelacarsen significantly reduced the rate of weekly lipoprotein apheresis sessions, with a mean of 0.16 apheresis sessions per week compared to 0.93 sessions per week for placebo.
Background
Pelacarsen was investigated for its potential to lower lipoprotein(a) levels and reduce the need for lipoprotein apheresis in 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 Phase 3 multicenter trial that enrolled 51 participants with Hyperlipoproteinemia(a) and established cardiovascular disease, who were undergoing weekly lipoprotein apheresis. The trial aimed to assess if treatment with Pelacarsen (TQJ230) 80 mg Q4W significantly reduced the rate of lipoprotein apheresis sessions compared to placebo over 52 weeks.
Key results
The study reported significant reductions in the rate of lipoprotein apheresis sessions for patients treated with Pelacarsen compared to placebo. Over 52 weeks, the mean rate of lipoprotein apheresis sessions normalized to the weekly schedule was 0.16 apheresis sessions per week for the Pelacarsen group, versus 0.93 apheresis sessions per week for the placebo group. This difference was statistically significant, with an Odds Ratio of 0.006 (95% Confidence Interval: 0.003 to 0.013; p-value 0.0001).
Regarding total lipoprotein apheresis avoidance from Week 12 to Week 52, 18 participants in the Pelacarsen group achieved avoidance, while 0 participants in the placebo group did. The median time to lipoprotein apheresis avoidance for the Pelacarsen group was 6.14 weeks. A Cox Regression analysis showed a Hazard Ratio of 88.26 (95% Confidence Interval: 4.71 to 1653.15; p-value 0.0014) for time to avoidance, and a Logistic Regression analysis for total avoidance yielded an Odds Ratio of 163.2 (95% Confidence Interval: 7.66 to 3477.25; p-value 0.0005).
Furthermore, Pelacarsen demonstrated a substantial reduction in lipoprotein(a) levels. The geometric mean ratio for the change from baseline to Week 52 in log-transformed Lp(a) (reported as mg/dL) was 0.32 for Pelacarsen versus 1.11 for placebo. Similarly, when reported as nmol/L, the geometric mean ratio was 0.27 for Pelacarsen versus 1.14 for placebo. Both reductions were highly significant (ANCOVA geometric mean ratio of 0.28 (95% Confidence Interval: 0.21 to 0.39; p-value 0.0001) for mg/dL and 0.23 (95% Confidence Interval: 0.16 to 0.33; p-value 0.0001) for nmol/L).
For the period from Week 12 to Week 52, the mean rate of apheresis sessions was 0.11 apheresis session per week for Pelacarsen compared to 0.93 apheresis session per week for placebo, with an Odds Ratio of 0.003 (95% Confidence Interval: 0.001 to 0.011; p-value 0.0001).
What this means
The results from this Phase 3 study indicate that Pelacarsen has a significant impact on reducing the burden of lipoprotein apheresis for patients with Hyperlipoproteinemia(a) and established cardiovascular disease. The substantial reduction in both the rate of apheresis sessions and lipoprotein(a) levels suggests that Pelacarsen could offer a therapeutic alternative to frequent apheresis treatments, potentially improving patient quality of life and clinical outcomes by lowering Lp(a) levels.
Source
This information was sourced from ClinicalTrials.gov, documenting the primary completion results of trial NCT05305664, which were posted on 2025-01-28. The study details are available on clinicaltrials.gov.