Subscribe
Share
Share
Embed
Welcome to BIOTECH NATION !!! With understandable interviews requiring no background in science, BTN attracts a wide global audience. From everyday people looking for hope in treatments in development, to bioentrepreneurs interested in the experience of their fellow travelers, to venture capitalists looking for possibilities in cutting-edge breakthroughs, to scientists simply interested in the work of others, BioTech Nation is the voice of human endeavor, driving science to new realities for everyone. These interviews are drawn directly from the public radio program, "Tech Nation", which also can be heard in numerous global radio and podcasting venues.
As happens with many diseases, there is little to be done in their most advanced stages or for their more rare unique forms. Priovant Therapeutics is solely focusing on severe autoimmune diseases with its single daily drug, and this one drug is now being tested in 2 different autoimmune conditions. They are each in final phase three trials. Ben Zimmer is Priovant Therapeutics CEO. Well, Ben, welcome to the program.
Ben Zimmer:Thank you so much. It's great to be here.
Dr. Moira Gunn:Now Priovant Therapeutics works with severe autoimmune disease. What is severe autoimmune disease, and what's the difference between a general autoimmune disease and a severe form of the autoimmune disease?
Ben Zimmer:Yep. So there's obviously a lot of autoimmune diseases, and all of them can be very severe. So if you think about maybe some of the ones that people are most familiar with, things like rheumatoid arthritis or psoriasis, These are diseases where for for many patients, they are very severe and burdensome, but they affect tons of people and the severity varies. For some other patients, it it's maybe not as as burdensome. There's a lot of other diseases out there that tend to be rarer, you know, maybe affect tens of thousands of patients rather than 100 of 1,000 or or even millions.
Ben Zimmer:But for nearly all of those patients, these diseases, come with very burdensome and debilitating symptoms. And because they're rarer, there's generally not as many treatment options available. There's not as much research that's been done. There's not as many new medicines that have been developed. And so that compounds even further in terms of how hard it is for the patients living with these diseases.
Ben Zimmer:And that's really, where Priovant comes in to to try to solve that and to try to help develop new medicines for those patients.
Dr. Moira Gunn:Priovant has a treatment designed to be a once daily pill, which is in phase 3 trials right now on 2 different severe autoimmune conditions. Phase 3, always good news. The last trials before drug approval if everything works well.
Ben Zimmer:The end is in sight for us.
Dr. Moira Gunn:The end is in sight, the horizon. You can see the event horizon here. So let's start with this single drug, and then let's go to these two severe conditions. Your drug is blocking 2 particular proteins of which there are many in our bodies, many, many, many, many. But these ones drive this overexcited immune response in autoimmune patients.
Dr. Moira Gunn:They're JAK 1 and TIK 2. That's their actual names. Many times these names are completely unpronounceable, but we got lucky here. Their actual names, JAK 1, j a k 1, and TIK 2, t y k 2. Now in simple terms, when you produce more JAK 1 and or t 2 in your body, what happens?
Ben Zimmer:So what happens in in autoimmune diseases, as many people know, is you have the immune cells are attacking instead of attacking pathogens like viruses, they're attacking the body's healthy cells, which they're not supposed to. And how does that happen? People may remember the, well, the cytokine storm from from COVID. So the cytokines are proteins that are how the immune cells, communicate with each other to do this, to to do this overactivity. And then JAK 1 and TIK 2 are proteins that are actually, part of the cell themselves, part of the immune cells themselves, and are how the cytokines signal to the immune cell what to do.
Ben Zimmer:And so when you have these, these inflammatory autoimmune responses, you have just a lot more immune cells being produced, and they're doing a lot of things that they're not supposed to be doing. And these JAK 1 and TIK 2 proteins, as part of the cells, are are kind of key to this cascade, key to this kind of vicious cycle. And by blocking those proteins, we're able to to stop that cycle and stop the cascade and and bring the immune system back under control.
Dr. Moira Gunn:So it's good that JAK 1 and tick 2, invite these cytokines. It's bad when there's way too many of them, and there's no reason for it. So
Ben Zimmer:Exactly. That's
Dr. Moira Gunn:where we're at. Okay. So now let's go to the 2 severe autoimmune conditions you're currently in phase three trials on. The first is dermatomyositis. Now what's that?
Ben Zimmer:So so that is a a disease that involves, inflammation, mostly in the skin and muscles. So it it's a very bad disease that affects around 40,000 adults in the United States as as well as, children, as well, actually. The the skin disease is is very bad. We're not just talking about small cosmetic rashes here. We're talking about rashes that can cover the entire body, lead to a lot of pain and irritation.
Ben Zimmer:Hair loss, is quite common because the rashes can be on the scalp. And then, that is, accompanied by muscle weakness from from muscle inflammation that can be extremely debilitating. Large numbers of patients struggle with daily activities like, walking upstairs, carrying groceries. Many require mobility aids. So it's really a a quite, a quite horrible disease.
Ben Zimmer:And and as you can imagine, when you have all of these things go grow hog in your skin and muscles, lots of other, different things can kinda stack up on top of that. So, you know, patients are generally immobile or or at least impaired mobility, so they're not getting as much exercise or even just walking around. So patients tend to develop often cardiometabolic problems. And with all of the pain and irritation and and, you know, physical changes to the body from the skin disease, depression, and anxiety are very common. And so you really have, yeah, huge numbers of of problems, very debilitating for people's lives.
Ben Zimmer:And the disease affects, mostly the working age population. 2 thirds of people are under 65. So this is, you know, affecting people who might otherwise be able to live, with normal healthy lives. And like many autoimmune diseases, it disproportionately affects women. Around 70% of the patients are women.
Ben Zimmer:So, really a very bad disease, very high burden on patients, and, little to nothing available in terms of modern medicines, for these patients. And so we're, we're excited to be hopefully, bringing something that can can help treat them.
Dr. Moira Gunn:Now you mentioned 40,000 people suffering in the United States, but your trial is is global. Describe the the phase three trial for us.
Ben Zimmer:Yeah. So we're running a phase three trial. It has 241 patients, which for a rarer disease like this is a very large trial. It's actually the largest dermatomyositis trial I've ever ever conducted in the, in the history of science. So we're we're excited about that.
Ben Zimmer:And we work with doctor's offices all over the world, you know, hospital systems who, you know, work who treat patients with with this condition and enroll them, in the trial and then, you know, measure a series of of of endpoints that are defined in our protocol. So so the trial, as you mentioned, is all over the world. It it's around 40% in the United States, but also a lot in Europe, and then some in South America and Asia as well, Taiwan and South Korea. So so in a very cool, exciting way, it's part of what I find fun about working in the space. You're really working with doctors all over the world to try to come together and and find, and find solutions.
Ben Zimmer:And then we're testing, 2 doses of of our drug in in the trial. We're testing the the dose that we, believe is is the right dose and the one that we expect to get, approved most likely. And then we're also testing a lower dose just to make sure that, that you can't get the same benefit with the with the main doses with the lower dose, that that we expect. Most likely, it's the it's the dose that we're we have most confidence in that will be the one getting approved. And then for the 1st year of the trial, 1 third of the patients, are on a are on a placebo, and that's how we tell if the drug works.
Ben Zimmer:But then there's a 2nd year where all of the patients get on on the on the active drugs, so get an opportunity to to experience that.
Dr. Moira Gunn:Now you mentioned endpoints. How do you know if it worked? Yeah.
Ben Zimmer:So you you really are doing different measurements of, of the symptoms. And so, you know, it's these these, diseases, it's funny when you really dig dig into it, how how in some ways simple that they are. So they're in for a muscle disease, for instance, there's literally tests where, you know, patients, like, push against the doctors with you know, the doctors will, do what's called manual muscle testing to, you know, measure a patient strength and a patient strength over time in different muscle groups. And then for skin disease, you know, there's different, you know, kind of measurements that are accepted in the medical community for the disease, or I'm looking at different parts of the body and how the rashes change. And then there's also, patient, what's called patient reported outcomes as well.
Ben Zimmer:So, basically, health care questionnaires that the patients answer about how they're feeling and functioning, which is, which is very important, as well, obviously, to get that patient perspective. So all we we look at at all of these together, and and, hopefully, they all, they all or at least the majority of them show, you know, show that the patients receiving the drug are doing better than the ones receiving the placebo. And I and I should also mention that, you know, when these patients enroll in the trial, because they're so sick, many are, you know, continuing on the the other drugs that they were before the trial, most notably, steroids. You know, as I mentioned earlier, there's really nothing, available for these, patients by way of modern medicines. And so, oral steroids remain really the staple of treatment and and disease control.
Ben Zimmer:And and as we know, across autoimmune disease, you know, steroids, do work at controlling inflammation, but they carry many, many, problems of their own when used chronically over a long period of time. And so one of the things we're making an effort to do, in the trial is taper patients off of their steroids, and so that will be another measurement that we can we can look at and and see.
Dr. Moira Gunn:Now remember, same drug, you've got another trial with a second condition, and it's most easily called NIU, officially nonanterior, noninfectious uveitis, NIU. What is NIU like in its severe form?
Ben Zimmer:So so this is, again, a very bad disease. This is a inflammation of the of the eye, and, you know, even just take a step back from the side, so and imagine yourself with your eye having inflammation there, and just picture what that is like. And it it is it is really bad. Everything you would imagine in terms of I don't want that, that that's what, NIU is like. You get, you know, blurry vision, floaters, pain in your eye, redness, itching.
Ben Zimmer:And in many cases, if if not treated, pay patients actually, can go blind. It's one of the leading causes of blindness among the working age population in the developed world. So this is a a really bad, bad disease. And again, patients, you know, for that reason need to be very aggressively treated. As with dermatomyositis, steroids remain the kind of staple of of treatment, including, oral steroids.
Ben Zimmer:And and in this case, sometimes even steroid injections into the eye, which which is you can imagine, you know, not not something that, you you wanna do if you can avoid it. There is one modern therapy there is one modern therapy approved for NIU, which is Humira, a drug that many people are are familiar with. And and that works for some patients, but we know from data that about half of the patients who get that, ultimately are are not effectively treated, and so that still leaves, you know, a very large number of of patients who, you know, need to get off steroids or not benefiting from that and are not able to be treated with with HUMIRA. And so I think there's, you know, a great opportunity for our our medicine, hopefully. Again, as you mentioned, is a once daily oral therapy to come in and and help these patients.
Dr. Moira Gunn:So the idea is that Priovance starts at the edge here, you know, the the most severe cases and attempts to address that in any number of autoimmune diseases. But I must say, lending confidence to to marching through more and more of these phase three trials is the fact that this drug has been tested in phase 2 in numerous larger populations of autoimmune conditions, the non severe forms by Pfizer. They ran 8 phase two trials across a number of conditions before you got this drug.
Ben Zimmer:Yeah. So the drug was, initially being developed by by Pfizer. And and as you know, they ran a number of successful phase two trials in your kind of standard set of, of larger autoimmune diseases, things like psoriatic arthritis and, and psoriasis. But, you know, we really felt that the the kind of opportunity for this medicine to make the biggest impact was in these, rarer but even more severe diseases. And we felt that both because that was really a a gap area in terms of other medicines, And also, it's where we think that, kind of combination of of of inhibiting JAK 1 and tick 2 can be particularly, beneficial as well compared to other medicines.
Ben Zimmer:And so we really had this idea of a different direction of how to take the medicine, and Pfizer's, r and d team was very excited about that as well. You know, but but, ultimately, as you can imagine, these large pharmaceutical companies, like, large companies of any kind, you know, they're they don't, always easily pivot or or change directions. They're kinda like, you know, freight trades, that speedboats. And so and so, you know, they kind of felt that really the best, opportunity was to spin out the drug into a standalone biotech company, but where Pfizer, retains ownership in it. So Pfizer, continues to own a a minority interest in in Privant, and, I think that was a really, you know, creative and exciting way to to take the drug and really unlock its benefit for patients in the best possible way.
Dr. Moira Gunn:Well, so many times, these very large global pharmaceutical companies, aren't just interested in large populations of people who can take their drug. That's what they're set up to do. You know? So, this is actually kind of the best of both worlds. We're not asking it to be something it's not.
Dr. Moira Gunn:And yet here we have a small new agile biotech that could be focused on this without the burdensome backstory of, okay. No. We have really large we've got really large everything. Everybody has to approve. It's like, no.
Dr. Moira Gunn:No. We we can't do that. We don't have time for that, and it's not it's not the right solution to to work on this problem. Okay. So you've got 2 severe immune conditions in phase 3 trials.
Dr. Moira Gunn:What are Priovance plans?
Ben Zimmer:So, you know, I think priority, you know, priority number 1, 2, and 3 is to execute on those successfully. As as you can imagine, these things are not are not easy. And, you know, like I was describing with the dermatomyositis trial, you know, these are very complicated, you know, global endeavors, and and it requires a lot of time and attention from us to try to make sure everything is run properly and set up for success. And, you know, we're we're a small company, around 60 to 70 people right now, which I think is great and that it allows us to be, you know, agile and quick and nimble, but Hey.
Dr. Moira Gunn:You can all go to the same Christmas party.
Ben Zimmer:Exactly. We can all go to the same Christmas party.
Dr. Moira Gunn:Try that with 60,000 employees.
Ben Zimmer:Exactly. We all know each other. It it's great, but it also means we have to prioritize and focus. And so that's really, our top priority, but, you know, it's great. I I'm an ambitious person and really want to, fulfill the, the long term, potential of this of this medicine, and our mission in general around these, severe autoimmune diseases.
Ben Zimmer:You know, and grow grow our company from a a small company. May maybe not big in in, sort of bureaucracy, but big in impact at least. And and so they're they're actually, you know, dermatomyositis and noninfectious uveitis are are 2 of, I think, many similar types of diseases where, where we could have, you know, have a similar potential impact. And so we're looking into starting some additional clinical trials, possibly as soon as next year, in 2025. And then, you know, in in the longer term as well to think about additional medicines that can help this category of of patients as well.
Ben Zimmer:Because I think it's something that, you know, we're very excited about, and I think we're just at the at the tip of the ice berg in terms of really being able to help patients with these diseases.
Dr. Moira Gunn:Well, Ben, thank you so much for joining me. I hope you'll come back, keep us updated.
Ben Zimmer:Thank you so much. I would love to, and I I really appreciate the the time and opportunity.
Dr. Moira Gunn:Ben Zimmer is the CEO of Priovant Therapeutics. More information is available at priovanttx.com.