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IPIG 2025 | A Phase II trial of zaltenibart in patients with PNH with a suboptimal response to ravulizumab
Jens Panse, MD, Aachen University Hospital, Aachen, Germany, provides insight into the Phase II proof-of-concept clinical trial (NCT05972967) investigating zaltenibart, an alternative pathway MASP-3 inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH) with a suboptimal response to ravulizumab. Dr Panse highlights that the trial showed promising results. This interview took place at the 2nd International PNH Interest Group (IPIG) Conference in Paris, France.
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Transcript
You might not imagine it, but there are even newer drugs within the field of PNH, despite the fact that we just discussed we have six different ones. So zaltenibart is a so-called MAS3 inhibitor. And MASP-3 is basically an enzyme that allows Profactor D to become Factor D. And Factor D is basically produced during 24 hours because of MASP-3. Now if you inhibit MASP-3, then you would tackle the alternative pathway very early in a very specific way...
You might not imagine it, but there are even newer drugs within the field of PNH, despite the fact that we just discussed we have six different ones. So zaltenibart is a so-called MAS3 inhibitor. And MASP-3 is basically an enzyme that allows Profactor D to become Factor D. And Factor D is basically produced during 24 hours because of MASP-3. Now if you inhibit MASP-3, then you would tackle the alternative pathway very early in a very specific way. And the beauty of the MASP-3 inhibitor is that it can be given intravenously every eight weeks, so like ravulizumab. So the trial basically looks like all the other switch trials at patients suboptimally responding to ravulizumab and then you have an overlap where you give zaltenibart, the MASP-3 inhibitor, together with ravulizumab and then in those patients responding well with an increase of the hemoglobin level and a decrease in the reticulocytes, those patients are put on zaltenibart only. And what the trial showed that it is feasible that the dose, it was three milligrams and five milligrams per kilogram body weight, might not be the ideal dose because that leads to the Phase III trial which is done with eight milligram per kg, but that the proof of concept in this patient group, the same proof of concept that has been done in naive patients, basically works. And so 12 out of those patients went into the extension study and they all had stable levels.
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Disclosures
Consulting fees: Alexion, Sobi, Omeros, Sandoz; Payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events: Alexion, Amgen, Apellis, AstraZeneca, Blueprint Medicines, BMS, Boehringer Ingelheim, Gilead, MSD, Novartis, Pfizer, Roche, Sanofi, Sobi; Support for attending meetings and/or travel: Alexion, Blueprint Medicines, Boehringer Ingelheim, Novartis, Omeros, Pfizer, Roche, Sobi; Participation on a Data Safety Monitoring Board or Advisory Board: Alexion, Amgen, Apellis, AstraZeneca, Blueprint Medicines, BMS, Boehringer Ingelheim, Gilead, MSD, Novartis, Omeros, Pfizer, Roche, Sanofi, Sobi; Leadership or fiduciary role in other board, society, committee or advocacy group, paid or unpaid: www.lichterzellen.de; Receipt of equipment, materials, drugs, medical writing, gifts or other services: Apellis, Blueprint Medicines, Novartis, Roche, Sobi.
