Astrazeneca pulls ahead in haemoglobinuria

Astra is claiming a hit in the pivotal Alpha trial of danicopan in a late-stage paroxysmal nocturnal haemoglobinuria (PNH) trial, though it has not disclosed any actual data. The oral factor D inhibitor, given on top of Astra’s injected PNH blockbusters Ultomiris or Soliris in PNH patients who have clinically significant extravascular haemolysis, met its primary endpoint of change in haemoglobin from baseline – presumably an increase – at 12 weeks. Secondary goals including transfusion avoidance and change on a score measuring fatigue were also hit, but the company did not say whether another secondary, the proportion of patients with a haemoglobin increase of at least 2g/dL in the absence of transfusion, was met. If the extent of the hit is good enough for approval, Astra will be able to reach a subset of PNH patients that are not well treated now. Filings are planned for “the coming months”, but other groups are nipping at Astra’s heels. The phase 3 trial of Novartis’s Iptacopan should report by the end of the year, and data from Roche’s exhaustive phase 3 programme for crovalimab could come soon after. Roche’s project arguably poses less of a threat to Astra, though, since it is injected.