EHA23: With Novartis, Roche circling, AstraZeneca hopes danicopan can bolster PNH franchise

09 Jun 2023
firstwordpharma.com
Clinical ResultPhase 3Phase 2
AstraZeneca on Friday detailed results from a Phase III study showing that the addition of the oral factor D inhibitor danicopan to standard-of-care therapy in patients with paroxysmal nocturnal haemoglobinuria (PNH) led to statistically significant and clinically meaningful increases in haemoglobin (Hb) levels versus placebo. The findings from the ALPHA trial were presented at the European Hematology Association (EHA) annual meeting.
In September last year, AstraZeneca reported that the study had met its primary endpoint of change in Hb from baseline to 12 weeks. The trial was designed to enrol 84 patients with PNH who experience clinically significant extravascular haemolysis (EVH). Participants were randomised to receive danicopan or placebo, both in addition to their ongoing C5 inhibitor therapy of Ultomiris (ravulizumab) or Soliris (eculizumab).
Results presented at the EHA congress showed that in an interim analysis of 63 patients, those treated with danicopan experienced an increase in Hb levels of 2.94 g/dL on average at 12 weeks, compared to 0.50 g/dL for placebo. Study investigator Jong Wook Lee said the "results suggest danicopan has the potential to be an important option for the small subset of patients with PNH who experience clinically significant EVH while being treated with [Soliris or Ultomiris]."
AstraZeneca noted that all key secondary endpoints also met statistical superiority in favour of danicopan, with 59.5% of those given the drug experiencing an improvement in Hb of ≥2 g/dL at week 12 in the absence of transfusion, compared to 0% for placebo. Further, 83.3% of patients on danicopan avoided transfusion through week 12, versus 38.1% for the placebo group. Meanwhile, improvements in fatigue, as measured by the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue score, and absolute reticulocyte count were also shown.
Danicopan was generally well tolerated in the study, with the most common treatment-emergent adverse events being headache at 10.2%, while nausea, arthralgia and diarrhoea all occurred at rates of 8.2%. AstraZeneca indicated that regulatory submissions in this indication are currently under review with a number of health authorities. In addition to PNH, AstraZeneca, via its Alexion division, is evaluating danicopan in a Phase II study as a monotherapy for geographic atrophy.
The positive data for AstraZeneca's drug come shortly after Novartis said that it would submit regulatory filings for its targeted factor B inhibitor iptacopan after success in a second Phase III study for PNH. Meanwhile, Roche is also preparing to seek approval of crovalimab in PNH, having detailed late-stage results for the anti-C5 recycling monoclonal antibody at the EHA conference.
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