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BSH 2022 | A look at the future treatment landscape of PNH

In this video, Shreyans Gandhi, MBBS, MD, DNB(Internal Medicine), MRCP, FRCPath, M.Phil, King’s College Hospital NHS Foundation Trust, London, UK, discusses novel agents and upcoming trials investigating the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Dr Gandhi first mentions the use of eculizumab and ravulizumab in PNH treatment, as well as the excitement surrounding clinical trials which are looking at other agents along the complement pathway. To conclude, Dr Gandhi explains some future agents that may be used, such as oral compounds and anti-c5 agents, which may change the future treatment landscape of PNH. This interview took place at the 62nd Annual Scientific Meeting of the British Society for Haematology (BSH) 2022, in Manchester, UK.

Transcript (edited for clarity)

About 10 years back, we started off on this journey where eculizumab, the anti-C5 compound, was the only treatment we had available for PNH, and subsequently, more recently, the last couple of years also had ravulizumab, which is a sister of eculizumab. It’s a long-acting version of eculizumab from two very pivotal studies done over a year and across the world.

Some of the most successful studies done in PNH, but since then we have had a plethora of trials looking at various compounds all along the complement pathways, very interesting studies, looking at various options, some trying to improvise and looking at oral options, some trying to improvise and trying to look at subcutaneous options of delivery of the drug...

About 10 years back, we started off on this journey where eculizumab, the anti-C5 compound, was the only treatment we had available for PNH, and subsequently, more recently, the last couple of years also had ravulizumab, which is a sister of eculizumab. It’s a long-acting version of eculizumab from two very pivotal studies done over a year and across the world.

Some of the most successful studies done in PNH, but since then we have had a plethora of trials looking at various compounds all along the complement pathways, very interesting studies, looking at various options, some trying to improvise and looking at oral options, some trying to improvise and trying to look at subcutaneous options of delivery of the drug. Some looking at less frequent episodes of administration of drug, some looking at compounds that target different pathways along the complement tree including the alternate pathways.

So, there’ve been some very very exciting things that have been happening in the last year or so. In particular, we are looking at oral compounds factor D factor B inhibitors. They are at the advanced stages, Phase II and Phase III studies. We’re looking at standard anti-C5 treatment strategies that are given subcutaneously. That again is a Phase III study. So, the lots of interesting things happening and it’s a question of watching this space.

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