Investigational agent OMS906 performs well in PNH

The MASP-3 inhibitor OMS906 appeared safe and displayed encouraging signs of efficacy in the interim analysis of a phase 1b trial involving patients with treatment-naïve paroxysmal nocturnal haemoglobinuria (PNH). Several trials are underway to further evaluate this agent in the PNH population.

“Established therapies for PNH come with the risk of extravascular haemolysis,” said Dr Jens Panse (University of Aachen, Germany). The investigational agent OMS906 selectively targets MASP-3, which is a key activator of the alternative complement pathway. “Since this agent inhibits the alternative complement pathway proximally, it may block intravascular haemolysis and prevent extravascular haemolysis,” added Dr Panse. He presented the findings of the phase 1b trial (NCT05889299) which evaluated OMS906 in treatment-naïve participants with PNH who received 4-weekly subcutaneous injections of the agent up to 13 times. Safety was the primary endpoint [1].

OMS906 was well-tolerated in participants with PNH. “There were no cases of clinical breakthrough haemolysis, no major adverse vascular events, no serious adverse events or discontinuations,” stated Dr Panse. Low-grade headache or itching was observed in 20% and 30% of the participants, respectively, and 3 participants with underlying bone marrow failure showed evidence of cytopenia.

All participants had an increase in haemoglobin of at least 2g/dL after 1 injection and participants without myelodysplastic syndrome (n=8) achieved sex-normal haemoglobin levels. LDH normalisation was achieved in 7 out of 10 participants and reticulocyte normalisation was reached in 9 out of 10 participants.

The interim results of the current phase 1b trial demonstrate that OMS906 is a promising candidate to target PNH.

1. Karnabeda O, et al. OMS906, a mannan-binding lectin-associated serine protease-3 (MASP-3) inhibitor, normalizes hemoglobin levels in treatment-naïve PNH patients: interim data from a proof-of-concept clinical trial. Late-breaking oral session, EHA 2023 Annual Congress, 8─11 June, Frankfurt, Germany.

Copyright ©2023 Medicom Medical Publishers



Posted on 2 August, 202329 February, 2024