US-based biotech company Alnylam Pharmaceuticals has reported positive results from a Phase 2 study of an investigational RNAi therapeutic, cemdisiran.

Developed in collaboration with Regeneron Pharmaceuticals, cemdisiran targets the C5 component of the complement pathway for the treatment of adult patients with immunoglobulin A nephropathy.

Alnylam presented the results at the 18th European Meeting on Complement in Human Disease (EMCHD) being held in Bern, Switzerland on 26-28 August.

The Phase 2 study is a randomised, double-blind, placebo-controlled multicentre study to evaluate the efficacy and safety of cemdisiran.

The trial was done in three periods: 14 weeks, 32 weeks and 52 weeks with 31 adult patients (≥18 years and ≤ 65 years of age) with a clinical diagnosis of primary IgAN in a 2:1 cemdisiran to placebo ratio.

According to the data, cemdisiran showed a reduction in 24-hour urine protein to creatinine ratio at Week 32, the study’s primary endpoint, with a reduction of 37% in 24-hour UPCR seen in comparison to placebo.

New results also showed a higher percentage of patients treated with cemdisiran as compared to those on placebo, 32 versus 13%, respectively, achieved greater than or equal to 50% reduction in 24-hour UPCR.

Spot urine data were in agreement with 24-hour urine data, and the treatment impact started to become noticeable as early as Week 8 and persisted over time.

Patients using cemdisiran specifically experienced a 46% reduction in spot UPCR from baseline at 32 weeks.

In addition, the outcomes demonstrated that cemdisiran was generally well tolerated in IgAN patients, with no adverse events (AEs) necessitating therapy or study discontinuation during the double-blind treatment phase.

Injection site responses (41%) and peripheral edoema were the AEs in the cemdisiran arm that were reported by more than or equal to 10% of participants (14%).

The firm reported no drug-related serious or severe AEs.

IgAN is a common inflammatory condition affecting the kidney’s glomerulus that frequently leads to kidney failure.