Malaria is a disease that even today it's the single largest cause of death in children.
And it's mostly a disease of sub-Saharan Africa in terms of death. Those patient populations
are both economically and logistically-underserved. So that's a big motivator for us.
And then on the science side, my focus has always been developing new approaches to therapeutics.
So we're most interested in finding a new way to attack a disease that didn't exist
before and really proving that works, which means moving it into the clinic and working
with it in humans. You need different drugs to treat different
patient populations, and we have a very small number of available drugs for malaria.
The majority of the ones we have been around for 20, 40, 50 years.
So the way we think about safety, the way we think about usability, those rules were
very different 40, 50 years ago. We're treating children, we're treating pregnant women is
another target population, travelers who don't know what malaria is. We need really safe drugs.
Drugs last a year to 10 years in the clinic
before you have clinical resistance. So, having that continual introduction of new drugs that
work by different mechanisms ensures you can deal with resistance.
Right now every first line therapeutic depends on one class of antimalarial drugs. We're
starting to see resistance to that class emerge in Central Asia, Thai-Cambodian border.
We're seeing 40% to 60% in presenting patients who have resistance. When that happens, it's not
that long, likely, before those drugs are failing.
This was the second compound that acted on a novel mechanism. Novartis has developed
an inhibitor that works on the same target. It's a protein in the parasite called Pf-ATP4.
The mechanism is really unique. So it effects, directly, the parasite. But we believe what
actually happens is it primes the infected red blood cells to be cleared by the host.
So the mechanism by which the drug works takes advantage of the way your body gets rid of
old red cells, and that means the way resistance would develop is different. So we're utilizing
host in order to get rid of infected tissue, and that's potentially a real advantage.
We really won't know if that's important until we get deeper into clinical trials.
But that's one of the things we're really excited about. We've done two phases of testing.
So the classic phase I is safety.
That we did in Memphis in the University of Tennessee Health Sciences Center.
So this has, to date, been a really, really
safe drug. We really had no serious adverse events. We had nobody stop taking it while
they were on it. That's been really encouraging. When we finished that, we partnered with an
Australian institution that's developed a challenge model. They enroll a healthy subject,
they actually give them malaria in the clinic, and then we test the drug in that individual
in a really controlled setting. We look at the level of parasites in their blood. When
they're detectable but they're not causing symptoms, we treat.
And that lets us see, how does the drug work. Is it fast or slow? Is it still tolerated
in a patient? The answers to which is, this is a really rapidly acting drug.
That means if you're really sick and we give it to you, a day or two later you're starting
to feel better. We're reducing the likelihood that you would die. So that rapid action is
a characteristic we really look for. And the safety seems to be the same in patients who
have malaria. So now, we're expanding that into a country
where it's patients who walk into the clinic actually ill, Peru. And we're working in Peru
because they get both types of major malarias that are seen worldwide. So, in one clinical
trial we can test our compound against both types of malaria.
Most people who do what I do for a living, they're doing really well to get one compound
in the clinic. So it feels really good.