Novartis takes another swipe at AstraZeneca's rare disease gems Soliris and Ultomiris

Novartis has its eye on the market held by a pair of AstraZeneca's rare disease gems. The latest data showing that the Swiss drugmakers' iptacopan increases hemoglobin levels in a disease called paroxysmal nocturnal hemoglobinuria could be the keys to the safe.

Iptacopan is being studied in a clutch of trials across a group of rare disorders, and Novartis is now detailing the phase 3 PNH study called APPOINT today. The disease is characterized by the destruction of red blood cells, blood clots and impaired bone marrow function, which leaves patients dependent on blood transfusions. 

Novartis found that patients taking iptacopan met the study's main goal, with increases in hemoglobin without the need for blood transfusions at week 24.

This latest readout is a nice companion to results issued in October from the phase 3 APPLY trial, which showed that the drug beat the incumbent therapies on the market, Alexion’s Soliris and Ultomiris. AstraZeneca bought Alexion for $39 billion in 2021, meaning a head-to-head Big Pharma rivalry is on the horizon should Novartis’ med get approved.

Novartis said the APPOINT data is consistent with that seen in APPLY. The former trial featured patients who had not previously received Soliris or Ultomiris, while APPLY compared iptacopan directly with the AstraZeneca meds.

It seems Novartis is trying to create a new standard of care. The pharma said that many PNH patients are not served by the existing anti-C5 therapies, with many continuing to experience anemia, fatigue and dependency on blood transfusions. Iptacopan is also an oral medication, which would provide patients with an easier option than the Soliris and Ultomiris infusions.

Ultomiris brought in $688 million in 2021, while Soliris clocked a whopping $1.87 billion, according to AstraZeneca's year-end 2021 financial report issued this February. 

The APPLY data will be presented at the American Society of Hematology conference Tuesday, followed by an investor call. Novartis promised that more data from APPOINT beyond the initial top-line peak will be presented at a future medical conference. The data will also be used to form a regulatory submission in 2023.

Novartis is also studying iptacopan in phase 3 trials for the complement-mediated kidney diseases C3 glomerulopathy, IgA nephropathy and atypical hemolytic uremic syndrome. In addition, a number of phase 2s are underway.