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Hematology

Danicopan shows promising results in PNH clinical trial

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The ongoing international Phase 3 clinical trial, ALPHA, has reported positive interim results for danicopan (ALXN2040) as an add-on therapy to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PNH) and clinically significant extravascular haemolysis. The interim analysis indicates that danicopan effectively increased haemoglobin levels compared to a placebo, with a favorable safety profile.

In the ongoing international randomized, double-blind, placebo-controlled trial,

73 individuals were randomly assigned and received treatment, with 49 individuals receiving danicopan and 24 receiving a placebo. The interim analysis set, comprising the first 63 participants, revealed promising results at the 12-week mark.

The primary endpoint, change in haemoglobin concentration from baseline to week 12, demonstrated a substantial improvement in the danicopan group compared to the placebo group. The least squares mean change from baseline in haemoglobin was 2.94 g/dL (95% CI 2.52 to 3.36) in the danicopan group, while the placebo group showed a change of 0.50 g/dL (-0.13 to 1.12). The difference between the groups was statistically significant (P < 0.0001), with a least squares mean difference of 2.44 g/dL (1.69 to 3.20).

Grade 3 adverse events in the danicopan group included increased alanine aminotransferase, leukopenia, neutropenia, cholecystitis, COVID-19, increased aspartate aminotransferase, and increased blood pressure. In the placebo group, adverse events included anaemia, thrombocytopenia, and asthenia. Serious adverse events were reported in both groups but were not related to the study drug, and there were no deaths reported.

Reference
Lee JW, Griffin M, Kim JS, et al; ALXN2040-PNH-301 Investigators. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. Lancet Haematol. 2023;10(12):e955-e965. doi: 10.1016/S2352-3026(23)00315-0. PMID: 38030318.

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