Biocryst joins the push against Soliris

Full data on Biocryst’s oral factor D inhibitor BCX9930 are good enough to warrant a push into phase III. The longer-term question here is how the company will compete in a crowded space against much larger rivals. Mid-stage data in the rare severe anaemia paroxysmal nocturnal haemoglobinuria are encouraging, showing BCX9930 to improve patients’ blood biomarkers and prevent the transfusions on which many sufferers rely. The small study recruited treatment-naive subjects and those non-responsive to Alexion’s Soliris. In the former group haemoglobin levels were returned to close to normal and transfusions reduced to zero once patients were on higher doses of ‘9930. Haemoglobin also reached almost normal levels in the latter, harder-to-treat group, and although one patient required a transfusion the results are not hugely different from phase II data generated with experimental projects from Novartis and Alexion/Astrazeneca. Roche and Apellis are also taking aim at PNH, and pivotal data from all of these projects are needed to make more rigorous comparisons. All are trying to improve on the hugely successful Soliris with more convenient agents, but it is worth remembering that Alexion managed to build a blockbuster franchise by being the only option available.

Cross trial comparison of mid-stage trials in PNH patients inadequately controlled on Soliris or Ultomiris
  Iptacopan Danicopan BCX9930
Company Novartis Alexion/Astrazeneca Biocryst
Mechanism factor B inhibitor factor D inhibitor factor D inhibitor
Study* NCT03439839 NCT03472885 NCT04330534
Number of patients 10 11 6
Baseline haemoglobin not given mean 7.9g/dl mean 8.9g/dl
Increase from baseline mean 2.9g/dl mean 2.4g/dl mean 3.2g/dl
End of study haemoglobin >12g/dl in 8 of 10** mean 10.3g/dl*** mean 12.2g/dl****
Transfusion avoidance? At mean 241 days on treatment, no transfusions 95.8% reduction (one transfusion during 24 week trial)  5/6 remained transfusion free (83%) 
Note: *all given in combination with Soliris; **at week 13; ***at week 24, ****median 13 weeks on therapeutic dose. Source: company presentations.


Chasing Soliris: the crowded late-stage PNH pipeline
Project Description Company  Note
APL-2 (pegcetacoplan) Subcutaneous C3 inhibitor Apellis  Pegasus trial in treatment-experienced patients succeeded, Pdufa May 14; Prince trial in naïve due to read out H1 2021
Iptacopan Oral factor B inhibitor Novartis  Two pivotal trials under way in treatment-experienced and naive; results due from 2022
Danicopan (ALXN2040) Oral factor D inhibitor Alexion/Astrazeneca Phase III under way as add-on to Soliris; results due late 2022
Crovalimab Subcutaneous C5 inhibitor Roche  Three pivotal trials listed in treatment-experienced, naïve and H2H vs Soliris; results due late 2022/23
BCX9930 Oral factor D inhibitor Biocryst Phase III trial due to start H2 2021
And don't forget the Soliris biosimilars… 
ABP 959 Intravenous C5 inhibitor Amgen Phase III under way
BCD-148 Intravenous C5 inhibitor Biocad Phase III under way
SB12 Intravenous C5 inhibitor Samsung Bioepis Phase III under way
Source: EvaluatePharma &

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