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. 

The PNH pipeline - selected projects
Project Company 2028e PNH sales ($m) Mechanism Trial(s)
Danicopan (ALXN2040/
Alexion (Astrazeneca) 213 Oral factor D inhibitor Ph3 Alpha, as add-on to Ultomiris or Soliris, toplined as hit
Iptacopan (LNP023) Novartis - Oral factor B inhibitor Ph3 Apply-PNH in treatment-experienced pts, data due 2022
Crovalimab (RG6107) Roche  329 Subcutaneous C5 inhibitor Ph3 in treatment-experienced (Commodore-1); -naive (Commodore-3); and H2H vs Soliris (Commodore-2), results due late 2022/early 23
BCX9930 Biocryst 100 Oral factor D inhibitor Pivotal Ph2 trials in treatment-experienced (Redeem-1) and -naïve (Redeem-2) resumed Aug 2022 after clinical hold. 
ALXN2050 (ACH-5228) Alexion (Astrazeneca) - Oral factor D inhibitor Ph2 (NCT04170023), data due 2023
PNH=paroxysmal nocturnal haemoglobinuria. Source: EvaluatePharma &

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