VIDEO: Monoclonal antibodies, oral medications, gene therapy showing promise in HAE

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There's a lot going on right now, a whole sort of wave of new therapies that are either have just completed studies or undergoing clinical trials. The ones that have made their way now through phase three studies, there's a couple of preventative medicines. There's a monoclonal antibody called garadacimab, which targets factor XII in the bradykinin system. That's a once-a-month Sub-Q medication that's been shown to be very effective, and so far, very safe in preventing HAE attacks. That's in front of the regulatory bodies currently.

There's another preventative medicine called donidalorsen, which locks the translation or expression of plasma kallikrein. This is a RNA-targeted therapy. Not a new technology, but new to hereditary angioedema, and this is also given once a month as a small Sub-Q injection, and has been through phase three studies, again, showing it to be very effective, and so far, very safe and tolerable. So those are medicines we might have in the next couple of years to prevent HAE attacks.

There's also been action in the on-demand therapy, so treating attacks when they occur. And the big advance, there is an oral medicine, a pill that could stop an an HAE attack when it occurs. It's called sebetralstat. This is an oral kallikrein inhibitor in phase three study. It was shown to stop attacks very quickly, and can easily be taken rapidly at the onset of the symptoms. So this, if it's approved by regulatory bodies, would be a big advance over having to give injections or infusions, which is what people with HAE currently have to do to stop a swelling attack.

So those are the things that are kind of the closest to being available in the clinic. There are other things a little earlier in developments that include other oral medications, like nucriptabant. This blocks the B2 receptor, the bradykinin receptor, and is being studied now in phase three trials for both treating attacks, but also preventing attacks. There's another monoclonal antibody called novenobart, which might be given every three months as an injection to prevent attacks. And then the next big leap is gene therapy, and there is a phase two CRISPR-Cas9 technology looking to prevent HAE attacks as potentially one treatment that may last for a very long time, and those patients in phase two studies have been followed out now close to well over a year, close to two years, some of them.

And it looks to be effective so far in preventing attacks. So there's a lot of work to be done to determine the safety of some of these things, particularly I think gene therapy. But we've got a lot on the horizon. It's pretty likely we're gonna see, you know, more evolution of how we're managing patients over the next few years as we have these additional tools.