January 15, 2025 • 21 mins
This week on Biotech Nation, Priovant Therapeutics CEO Ben Zimmer shares how their once-a-day pill could transform the treatment of severe autoimmune diseases like dermatomyositis and non-infectious uveitis. With phase 3 trials in progress, Priovant is offering hope to patients with few modern treatment options.
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Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Dr. Moira Gunn (00:11):
As happens with many diseases, there is little
to be done in their mostadvanced stages or for their
more rare unique forms. PriovantTherapeutics is solely focusing
on severe autoimmune diseaseswith its single daily drug, and
this one drug is now beingtested in 2 different autoimmune
(00:33):
conditions. They are each infinal phase three trials. Ben
Zimmer is Priovant TherapeuticsCEO. Well, Ben, welcome to the
program.
Ben Zimmer (00:44):
Thank you so much. It's great to be here.
Dr. Moira Gunn (00:46):
Now Priovant Therapeutics works with severe
autoimmune disease. What issevere autoimmune disease, and
what's the difference between ageneral autoimmune disease and a
severe form of the autoimmunedisease?
Ben Zimmer (01:03):
Yep. So there's obviously a lot of autoimmune
diseases, and all of them can bevery severe. So if you think
about maybe some of the onesthat people are most familiar
with, things like rheumatoidarthritis or psoriasis, These
are diseases where for for manypatients, they are very severe
and burdensome, but they affecttons of people and the severity
(01:27):
varies. For some other patients,it it's maybe not as as
burdensome. There's a lot ofother diseases out there that
tend to be rarer, you know,maybe affect tens of thousands
of patients rather than 100 of1,000 or or even millions.
But for nearly all of thosepatients, these diseases, come
(01:50):
with very burdensome anddebilitating symptoms. And
because they're rarer, there'sgenerally not as many treatment
options available. There's notas much research that's been
done. There's not as many newmedicines that have been
developed. And so that compoundseven further in terms of how
hard it is for the patientsliving with these diseases.
(02:11):
And that's really, wherePriovant comes in to to try to
solve that and to try to helpdevelop new medicines for those
patients.
Dr. Moira Gunn (02:20):
Priovant has a treatment designed to be a once
daily pill, which is in phase 3trials right now on 2 different
severe autoimmune conditions.Phase 3, always good news. The
last trials before drug approvalif everything works well.
Ben Zimmer (02:39):
The end is in sight for us.
Dr. Moira Gunn (02:41):
The end is in sight, the horizon. You can see
the event horizon here. So let'sstart with this single drug, and
then let's go to these twosevere conditions. Your drug is
blocking 2 particular proteinsof which there are many in our
bodies, many, many, many, many.But these ones drive this
(03:03):
overexcited immune response inautoimmune patients.
They're JAK 1 and TIK 2. That'stheir actual names. Many times
these names are completelyunpronounceable, 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 youproduce more JAK 1 and or t 2 in
(03:28):
your body, what happens?
Ben Zimmer (03:30):
So what happens in in autoimmune diseases, as many
people know, is you have theimmune cells are attacking
instead of attacking pathogenslike viruses, they're attacking
the body's healthy cells, whichthey're not supposed to. And how
does that happen? People mayremember the, well, the cytokine
(03:51):
storm from from COVID. So thecytokines are proteins that are
how the immune cells,communicate with each other to
do this, to to do thisoveractivity. And then JAK 1 and
TIK 2 are proteins that areactually, part of the cell
(04:12):
themselves, part of the immunecells themselves, and are how
the cytokines signal to theimmune cell what to do.
And so when you have these,these inflammatory autoimmune
responses, you have just a lotmore immune cells being
produced, and they're doing alot of things that they're not
(04:33):
supposed to be doing. And theseJAK 1 and TIK 2 proteins, as
part of the cells, are are kindof key to this cascade, key to
this kind of vicious cycle. Andby blocking those proteins,
we're able to to stop that cycleand stop the cascade and and
bring the immune system backunder control.
Dr. Moira Gunn (04:54):
So it's good that JAK 1 and tick 2, invite
these cytokines. It's bad whenthere's way too many of them,
and there's no reason for it. So
Ben Zimmer (05:04):
Exactly. That's
Dr. Moira Gunn (05:06):
where we're at. Okay. So now let's go to the 2
severe autoimmune conditionsyou're currently in phase three
trials on. The first isdermatomyositis. Now what's
that?
Ben Zimmer (05:19):
So so that is a a disease that involves,
inflammation, mostly in the skinand muscles. So it it's a very
bad disease that affects around40,000 adults in the United
States as as well as, children,as well, actually. The the skin
(05:40):
disease is is very bad. We'renot just talking about small
cosmetic rashes here. We'retalking about rashes that can
cover the entire body, lead to alot of pain and irritation.
Hair loss, is quite commonbecause the rashes can be on the
scalp. And then, that is,accompanied by muscle weakness
(06:03):
from from muscle inflammationthat can be extremely
debilitating. Large numbers ofpatients struggle with daily
activities like, walkingupstairs, carrying groceries.
Many require mobility aids. Soit's really a a quite, a quite
(06:24):
horrible disease.
And and as you can imagine, whenyou have all of these things go
grow hog in your skin andmuscles, lots of other,
different things can kinda stackup on top of that. So, you know,
patients are generally immobileor or at least impaired
mobility, so they're not gettingas much exercise or even just
(06:46):
walking around. So patients tendto develop often cardiometabolic
problems. And with all of thepain and irritation and and, you
know, physical changes to thebody from the skin disease,
depression, and anxiety are verycommon. And so you really have,
yeah, huge numbers of ofproblems, very debilitating for
(07:09):
people's lives.
And the disease affects, mostlythe working age population. 2
thirds of people are under 65.So this is, you know, affecting
people who might otherwise beable to live, with normal
healthy lives. And like manyautoimmune diseases, it
(07:32):
disproportionately affectswomen. Around 70% of the
patients are women.
So, really a very bad disease,very high burden on patients,
and, little to nothing availablein terms of modern medicines,
for these patients. And sowe're, we're excited to be
(07:52):
hopefully, bringing somethingthat can can help treat them.
Dr. Moira Gunn (07:56):
Now you mentioned 40,000 people
suffering in the United States,but your trial is is global.
Describe the the phase threetrial for us.
Ben Zimmer (08:06):
Yeah. So we're running a phase three trial. It
has 241 patients, which for ararer disease like this is a
very large trial. It's actuallythe largest dermatomyositis
trial I've ever ever conductedin the, in the history of
science. So we're we're excitedabout that.
And we work with doctor'soffices all over the world, you
(08:29):
know, hospital systems who, youknow, work who treat patients
with with this condition andenroll them, in the trial and
then, you know, measure a seriesof of of endpoints that are
defined in our protocol. So sothe trial, as you mentioned, is
all over the world. It it'saround 40% in the United States,
(08:52):
but also a lot in Europe, andthen some in South America and
Asia as well, Taiwan and SouthKorea. So so in a very cool,
exciting way, it's part of whatI find fun about working in the
space. You're really workingwith doctors all over the world
to try to come together and andfind, and find solutions.
(09:13):
And then we're testing, 2 dosesof of our drug in in the trial.
We're testing the the dose thatwe, believe is is the right dose
and the one that we expect toget, approved most likely. And
then we're also testing a lowerdose just to make sure that,
that you can't get the samebenefit with the with the main
(09:36):
doses with the lower dose, thatthat we expect. Most likely,
it's the it's the dose thatwe're we have most confidence in
that will be the one gettingapproved. And then for the 1st
year of the trial, 1 third ofthe patients, are on a are on a
placebo, and that's how we tellif the drug works.
(09:58):
But then there's a 2nd yearwhere all of the patients get on
on the on the active drugs, soget an opportunity to to
experience that.
Dr. Moira Gunn (10:07):
Now you mentioned endpoints. How do you
know if it worked? Yeah.
Ben Zimmer (10:12):
So you you really are doing different measurements
of, of the symptoms. And so, youknow, it's these these,
diseases, it's funny when youreally dig dig into it, how how
in some ways simple that theyare. So they're in for a muscle
(10:32):
disease, for instance, there'sliterally tests where, you know,
patients, like, push against thedoctors with you know, the
doctors will, do what's calledmanual muscle testing to, you
know, measure a patient strengthand a patient strength over time
in different muscle groups. Andthen for skin disease, you know,
(10:53):
there's different, you know,kind of measurements that are
accepted in the medicalcommunity for the disease, or
I'm looking at different partsof the body and how the rashes
change. And then there's also,patient, what's called patient
reported outcomes as well.
So, basically, health carequestionnaires that the patients
(11:15):
answer about how they're feelingand functioning, which is, which
is very important, as well,obviously, to get that patient
perspective. So all we we lookat at all of these together, and
and, hopefully, they all, theyall or at least the majority of
them show, you know, show thatthe patients receiving the drug
(11:36):
are doing better than the onesreceiving the placebo. And I and
I should also mention that, youknow, when these patients enroll
in the trial, because they're sosick, many are, you know,
continuing on the the otherdrugs that they were before the
trial, most notably, steroids.You know, as I mentioned
(11:57):
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 anddisease control.
And and as we know, acrossautoimmune disease, you know,
steroids, do work at controllinginflammation, but they carry
(12:20):
many, many, problems of theirown when used chronically over a
long period of time. And so oneof the things we're making an
effort to do, in the trial istaper patients off of their
steroids, and so that will beanother measurement that we can
we can look at and and see.
Dr. Moira Gunn (12:41):
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 (12:58):
So so this is, again, a very bad disease. This
is a inflammation of the of theeye, and, you know, even just
take a step back from the side,so and imagine yourself with
your eye having inflammationthere, and just picture what
that is like. And it it is it isreally bad. Everything you would
imagine in terms of I don't wantthat, that that's what, NIU is
(13:23):
like. You get, you know, blurryvision, floaters, pain in your
eye, redness, itching.
And in many cases, if if nottreated, pay patients actually,
can go blind. It's one of theleading causes of blindness
among the working age populationin the developed world. So this
(13:48):
is a a really bad, bad disease.And again, patients, you know,
for that reason need to be veryaggressively treated. As with
dermatomyositis, steroids remainthe kind of staple of of
treatment, including, oralsteroids.
And and in this case, sometimeseven steroid injections into the
(14:12):
eye, which which is you canimagine, you know, not not
something that, you you wanna doif you can avoid it. There is
one modern therapy there is onemodern therapy approved for NIU,
which is Humira, a drug thatmany people are are familiar
with. And and that works forsome patients, but we know from
(14:35):
data that about half of thepatients who get that,
ultimately are are noteffectively treated, and so that
still leaves, you know, a verylarge number of of patients who,
you know, need to get offsteroids or not benefiting from
that and are not able to betreated with with HUMIRA. And so
(14:55):
I think there's, you know, agreat opportunity for our our
medicine, hopefully. Again, asyou mentioned, is a once daily
oral therapy to come in and andhelp these patients.
Dr. Moira Gunn (15:06):
So the idea is that Priovance starts at the
edge here, you know, the themost severe cases and attempts
to address that in any number ofautoimmune diseases. But I must
say, lending confidence to tomarching through more and more
of these phase three trials isthe fact that this drug has been
(15:27):
tested in phase 2 in numerouslarger populations of autoimmune
conditions, the non severe formsby Pfizer. They ran 8 phase two
trials across a number ofconditions before you got this
drug.
Ben Zimmer (15:45):
Yeah. So the drug was, initially being developed
by by Pfizer. And and as youknow, they ran a number of
successful phase two trials inyour kind of standard set of, of
larger autoimmune diseases,things like psoriatic arthritis
(16:06):
and, and psoriasis. But, youknow, we really felt that the
the kind of opportunity for thismedicine to make the biggest
impact was in these, rarer buteven more severe diseases. And
we felt that both because thatwas really a a gap area in terms
(16:29):
of other medicines, And also,it's where we think that, kind
of combination of of ofinhibiting JAK 1 and tick 2 can
be particularly, beneficial aswell compared to other
medicines.
And so we really had this ideaof a different direction of how
to take the medicine, andPfizer's, r and d team was very
(16:53):
excited about that as well. Youknow, but but, ultimately, as
you can imagine, these largepharmaceutical companies, like,
large companies of any kind, youknow, they're they don't, always
easily pivot or or changedirections. They're kinda like,
you know, freight trades, thatspeedboats. And so and so, you
(17:14):
know, they kind of felt thatreally the best, opportunity was
to spin out the drug into astandalone biotech company, but
where Pfizer, retains ownershipin it. So Pfizer, continues to
own a a minority interest in inPrivant, and, I think that was a
(17:37):
really, you know, creative andexciting way to to take the drug
and really unlock its benefitfor patients in the best
possible way.
Dr. Moira Gunn (17:48):
Well, so many times, these very large global
pharmaceutical companies, aren'tjust interested in large
populations of people who cantake 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 notasking it to be something it's
not.
(18:08):
And yet here we have a small newagile biotech that could be
focused on this without theburdensome backstory of, okay.
No. We have really large we'vegot really large everything.
Everybody has to approve. It'slike, no.
No. We we can't do that. Wedon't have time for that, and
it's not it's not the rightsolution to to work on this
(18:30):
problem. Okay. So you've got 2severe immune conditions in
phase 3 trials.
What are Priovance plans?
Ben Zimmer (18:38):
So, you know, I think priority, you know,
priority number 1, 2, and 3 isto execute on those
successfully. As as you canimagine, 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
(19:03):
know, global endeavors, and andit requires a lot of time and
attention from us to try to makesure everything is run properly
and set up for success. And, youknow, we're we're a small
company, around 60 to 70 peopleright now, which I think is
great and that it allows us tobe, you know, agile and quick
(19:26):
and nimble, but Hey.
Dr. Moira Gunn (19:28):
You can all go to the same Christmas party.
Ben Zimmer (19:30):
Exactly. We can all go to the same Christmas party.
Dr. Moira Gunn (19:33):
Try that with 60,000 employees.
Ben Zimmer (19:35):
Exactly. We all know each other. It it's great, but
it also means we have toprioritize and focus. And so
that's really, our top priority,but, you know, it's great. I I'm
an ambitious person and reallywant to, fulfill the, the long
term, potential of this of thismedicine, and our mission in
(19:59):
general around these, severeautoimmune diseases.
You know, and grow grow ourcompany from a a small company.
May maybe not big in in, sort ofbureaucracy, but big in impact
at least. And and so they'rethey're actually, you know,
dermatomyositis andnoninfectious uveitis are are 2
(20:20):
of, I think, many similar typesof diseases where, where we
could have, you know, have asimilar potential impact. And so
we're looking into starting someadditional clinical trials,
possibly as soon as next year,in 2025. And then, you know, in
(20:45):
in the longer term as well tothink about additional medicines
that can help this category ofof patients as well.
Because I think it's somethingthat, you know, we're very
excited about, and I think we'rejust at the at the tip of the
ice berg in terms of reallybeing able to help patients with
these diseases.
Dr. Moira Gunn (21:05):
Well, Ben, thank you so much for joining me. I
hope you'll come back, keep usupdated.
Ben Zimmer (21:10):
Thank you so much. I would love to, and I I really
appreciate the the time andopportunity.
Dr. Moira Gunn (21:15):
Ben Zimmer is the CEO of Priovant
Therapeutics. More informationis available at priovanttx.com.
As happens with many diseases, there is little
to be done in their mostadvanced stages or for their
more rare unique forms. PriovantTherapeutics is solely focusing
on severe autoimmune diseaseswith its single daily drug, and
this one drug is now beingtested in 2 different autoimmune
(00:33):
conditions. They are each infinal phase three trials. Ben
Zimmer is Priovant TherapeuticsCEO. Well, Ben, welcome to the
program.
Ben Zimmer (00:44):
Thank you so much. It's great to be here.
Dr. Moira Gunn (00:46):
Now Priovant Therapeutics works with severe
autoimmune disease. What issevere autoimmune disease, and
what's the difference between ageneral autoimmune disease and a
severe form of the autoimmunedisease?
Ben Zimmer (01:03):
Yep. So there's obviously a lot of autoimmune
diseases, and all of them can bevery severe. So if you think
about maybe some of the onesthat people are most familiar
with, things like rheumatoidarthritis or psoriasis, These
are diseases where for for manypatients, they are very severe
and burdensome, but they affecttons of people and the severity
(01:27):
varies. For some other patients,it it's maybe not as as
burdensome. There's a lot ofother diseases out there that
tend to be rarer, you know,maybe affect tens of thousands
of patients rather than 100 of1,000 or or even millions.
But for nearly all of thosepatients, these diseases, come
(01:50):
with very burdensome anddebilitating symptoms. And
because they're rarer, there'sgenerally not as many treatment
options available. There's notas much research that's been
done. There's not as many newmedicines that have been
developed. And so that compoundseven further in terms of how
hard it is for the patientsliving with these diseases.
(02:11):
And that's really, wherePriovant comes in to to try to
solve that and to try to helpdevelop new medicines for those
patients.
Dr. Moira Gunn (02:20):
Priovant has a treatment designed to be a once
daily pill, which is in phase 3trials right now on 2 different
severe autoimmune conditions.Phase 3, always good news. The
last trials before drug approvalif everything works well.
Ben Zimmer (02:39):
The end is in sight for us.
Dr. Moira Gunn (02:41):
The end is in sight, the horizon. You can see
the event horizon here. So let'sstart with this single drug, and
then let's go to these twosevere conditions. Your drug is
blocking 2 particular proteinsof which there are many in our
bodies, many, many, many, many.But these ones drive this
(03:03):
overexcited immune response inautoimmune patients.
They're JAK 1 and TIK 2. That'stheir actual names. Many times
these names are completelyunpronounceable, 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 youproduce more JAK 1 and or t 2 in
(03:28):
your body, what happens?
Ben Zimmer (03:30):
So what happens in in autoimmune diseases, as many
people know, is you have theimmune cells are attacking
instead of attacking pathogenslike viruses, they're attacking
the body's healthy cells, whichthey're not supposed to. And how
does that happen? People mayremember the, well, the cytokine
(03:51):
storm from from COVID. So thecytokines are proteins that are
how the immune cells,communicate with each other to
do this, to to do thisoveractivity. And then JAK 1 and
TIK 2 are proteins that areactually, part of the cell
(04:12):
themselves, part of the immunecells themselves, and are how
the cytokines signal to theimmune cell what to do.
And so when you have these,these inflammatory autoimmune
responses, you have just a lotmore immune cells being
produced, and they're doing alot of things that they're not
(04:33):
supposed to be doing. And theseJAK 1 and TIK 2 proteins, as
part of the cells, are are kindof key to this cascade, key to
this kind of vicious cycle. Andby blocking those proteins,
we're able to to stop that cycleand stop the cascade and and
bring the immune system backunder control.
Dr. Moira Gunn (04:54):
So it's good that JAK 1 and tick 2, invite
these cytokines. It's bad whenthere's way too many of them,
and there's no reason for it. So
Ben Zimmer (05:04):
Exactly. That's
Dr. Moira Gunn (05:06):
where we're at. Okay. So now let's go to the 2
severe autoimmune conditionsyou're currently in phase three
trials on. The first isdermatomyositis. Now what's
that?
Ben Zimmer (05:19):
So so that is a a disease that involves,
inflammation, mostly in the skinand muscles. So it it's a very
bad disease that affects around40,000 adults in the United
States as as well as, children,as well, actually. The the skin
(05:40):
disease is is very bad. We'renot just talking about small
cosmetic rashes here. We'retalking about rashes that can
cover the entire body, lead to alot of pain and irritation.
Hair loss, is quite commonbecause the rashes can be on the
scalp. And then, that is,accompanied by muscle weakness
(06:03):
from from muscle inflammationthat can be extremely
debilitating. Large numbers ofpatients struggle with daily
activities like, walkingupstairs, carrying groceries.
Many require mobility aids. Soit's really a a quite, a quite
(06:24):
horrible disease.
And and as you can imagine, whenyou have all of these things go
grow hog in your skin andmuscles, lots of other,
different things can kinda stackup on top of that. So, you know,
patients are generally immobileor or at least impaired
mobility, so they're not gettingas much exercise or even just
(06:46):
walking around. So patients tendto develop often cardiometabolic
problems. And with all of thepain and irritation and and, you
know, physical changes to thebody from the skin disease,
depression, and anxiety are verycommon. And so you really have,
yeah, huge numbers of ofproblems, very debilitating for
(07:09):
people's lives.
And the disease affects, mostlythe working age population. 2
thirds of people are under 65.So this is, you know, affecting
people who might otherwise beable to live, with normal
healthy lives. And like manyautoimmune diseases, it
(07:32):
disproportionately affectswomen. Around 70% of the
patients are women.
So, really a very bad disease,very high burden on patients,
and, little to nothing availablein terms of modern medicines,
for these patients. And sowe're, we're excited to be
(07:52):
hopefully, bringing somethingthat can can help treat them.
Dr. Moira Gunn (07:56):
Now you mentioned 40,000 people
suffering in the United States,but your trial is is global.
Describe the the phase threetrial for us.
Ben Zimmer (08:06):
Yeah. So we're running a phase three trial. It
has 241 patients, which for ararer disease like this is a
very large trial. It's actuallythe largest dermatomyositis
trial I've ever ever conductedin the, in the history of
science. So we're we're excitedabout that.
And we work with doctor'soffices all over the world, you
(08:29):
know, hospital systems who, youknow, work who treat patients
with with this condition andenroll them, in the trial and
then, you know, measure a seriesof of of endpoints that are
defined in our protocol. So sothe trial, as you mentioned, is
all over the world. It it'saround 40% in the United States,
(08:52):
but also a lot in Europe, andthen some in South America and
Asia as well, Taiwan and SouthKorea. So so in a very cool,
exciting way, it's part of whatI find fun about working in the
space. You're really workingwith doctors all over the world
to try to come together and andfind, and find solutions.
(09:13):
And then we're testing, 2 dosesof of our drug in in the trial.
We're testing the the dose thatwe, believe is is the right dose
and the one that we expect toget, approved most likely. And
then we're also testing a lowerdose just to make sure that,
that you can't get the samebenefit with the with the main
(09:36):
doses with the lower dose, thatthat we expect. Most likely,
it's the it's the dose thatwe're we have most confidence in
that will be the one gettingapproved. And then for the 1st
year of the trial, 1 third ofthe patients, are on a are on a
placebo, and that's how we tellif the drug works.
(09:58):
But then there's a 2nd yearwhere all of the patients get on
on the on the active drugs, soget an opportunity to to
experience that.
Dr. Moira Gunn (10:07):
Now you mentioned endpoints. How do you
know if it worked? Yeah.
Ben Zimmer (10:12):
So you you really are doing different measurements
of, of the symptoms. And so, youknow, it's these these,
diseases, it's funny when youreally dig dig into it, how how
in some ways simple that theyare. So they're in for a muscle
(10:32):
disease, for instance, there'sliterally tests where, you know,
patients, like, push against thedoctors with you know, the
doctors will, do what's calledmanual muscle testing to, you
know, measure a patient strengthand a patient strength over time
in different muscle groups. Andthen for skin disease, you know,
(10:53):
there's different, you know,kind of measurements that are
accepted in the medicalcommunity for the disease, or
I'm looking at different partsof the body and how the rashes
change. And then there's also,patient, what's called patient
reported outcomes as well.
So, basically, health carequestionnaires that the patients
(11:15):
answer about how they're feelingand functioning, which is, which
is very important, as well,obviously, to get that patient
perspective. So all we we lookat at all of these together, and
and, hopefully, they all, theyall or at least the majority of
them show, you know, show thatthe patients receiving the drug
(11:36):
are doing better than the onesreceiving the placebo. And I and
I should also mention that, youknow, when these patients enroll
in the trial, because they're sosick, many are, you know,
continuing on the the otherdrugs that they were before the
trial, most notably, steroids.You know, as I mentioned
(11:57):
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 anddisease control.
And and as we know, acrossautoimmune disease, you know,
steroids, do work at controllinginflammation, but they carry
(12:20):
many, many, problems of theirown when used chronically over a
long period of time. And so oneof the things we're making an
effort to do, in the trial istaper patients off of their
steroids, and so that will beanother measurement that we can
we can look at and and see.
Dr. Moira Gunn (12:41):
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 (12:58):
So so this is, again, a very bad disease. This
is a inflammation of the of theeye, and, you know, even just
take a step back from the side,so and imagine yourself with
your eye having inflammationthere, and just picture what
that is like. And it it is it isreally bad. Everything you would
imagine in terms of I don't wantthat, that that's what, NIU is
(13:23):
like. You get, you know, blurryvision, floaters, pain in your
eye, redness, itching.
And in many cases, if if nottreated, pay patients actually,
can go blind. It's one of theleading causes of blindness
among the working age populationin the developed world. So this
(13:48):
is a a really bad, bad disease.And again, patients, you know,
for that reason need to be veryaggressively treated. As with
dermatomyositis, steroids remainthe kind of staple of of
treatment, including, oralsteroids.
And and in this case, sometimeseven steroid injections into the
(14:12):
eye, which which is you canimagine, you know, not not
something that, you you wanna doif you can avoid it. There is
one modern therapy there is onemodern therapy approved for NIU,
which is Humira, a drug thatmany people are are familiar
with. And and that works forsome patients, but we know from
(14:35):
data that about half of thepatients who get that,
ultimately are are noteffectively treated, and so that
still leaves, you know, a verylarge number of of patients who,
you know, need to get offsteroids or not benefiting from
that and are not able to betreated with with HUMIRA. And so
(14:55):
I think there's, you know, agreat opportunity for our our
medicine, hopefully. Again, asyou mentioned, is a once daily
oral therapy to come in and andhelp these patients.
Dr. Moira Gunn (15:06):
So the idea is that Priovance starts at the
edge here, you know, the themost severe cases and attempts
to address that in any number ofautoimmune diseases. But I must
say, lending confidence to tomarching through more and more
of these phase three trials isthe fact that this drug has been
(15:27):
tested in phase 2 in numerouslarger populations of autoimmune
conditions, the non severe formsby Pfizer. They ran 8 phase two
trials across a number ofconditions before you got this
drug.
Ben Zimmer (15:45):
Yeah. So the drug was, initially being developed
by by Pfizer. And and as youknow, they ran a number of
successful phase two trials inyour kind of standard set of, of
larger autoimmune diseases,things like psoriatic arthritis
(16:06):
and, and psoriasis. But, youknow, we really felt that the
the kind of opportunity for thismedicine to make the biggest
impact was in these, rarer buteven more severe diseases. And
we felt that both because thatwas really a a gap area in terms
(16:29):
of other medicines, And also,it's where we think that, kind
of combination of of ofinhibiting JAK 1 and tick 2 can
be particularly, beneficial aswell compared to other
medicines.
And so we really had this ideaof a different direction of how
to take the medicine, andPfizer's, r and d team was very
(16:53):
excited about that as well. Youknow, but but, ultimately, as
you can imagine, these largepharmaceutical companies, like,
large companies of any kind, youknow, they're they don't, always
easily pivot or or changedirections. They're kinda like,
you know, freight trades, thatspeedboats. And so and so, you
(17:14):
know, they kind of felt thatreally the best, opportunity was
to spin out the drug into astandalone biotech company, but
where Pfizer, retains ownershipin it. So Pfizer, continues to
own a a minority interest in inPrivant, and, I think that was a
(17:37):
really, you know, creative andexciting way to to take the drug
and really unlock its benefitfor patients in the best
possible way.
Dr. Moira Gunn (17:48):
Well, so many times, these very large global
pharmaceutical companies, aren'tjust interested in large
populations of people who cantake 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 notasking it to be something it's
not.
(18:08):
And yet here we have a small newagile biotech that could be
focused on this without theburdensome backstory of, okay.
No. We have really large we'vegot really large everything.
Everybody has to approve. It'slike, no.
No. We we can't do that. Wedon't have time for that, and
it's not it's not the rightsolution to to work on this
(18:30):
problem. Okay. So you've got 2severe immune conditions in
phase 3 trials.
What are Priovance plans?
Ben Zimmer (18:38):
So, you know, I think priority, you know,
priority number 1, 2, and 3 isto execute on those
successfully. As as you canimagine, 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
(19:03):
know, global endeavors, and andit requires a lot of time and
attention from us to try to makesure everything is run properly
and set up for success. And, youknow, we're we're a small
company, around 60 to 70 peopleright now, which I think is
great and that it allows us tobe, you know, agile and quick
(19:26):
and nimble, but Hey.
Dr. Moira Gunn (19:28):
You can all go to the same Christmas party.
Ben Zimmer (19:30):
Exactly. We can all go to the same Christmas party.
Dr. Moira Gunn (19:33):
Try that with 60,000 employees.
Ben Zimmer (19:35):
Exactly. We all know each other. It it's great, but
it also means we have toprioritize and focus. And so
that's really, our top priority,but, you know, it's great. I I'm
an ambitious person and reallywant to, fulfill the, the long
term, potential of this of thismedicine, and our mission in
(19:59):
general around these, severeautoimmune diseases.
You know, and grow grow ourcompany from a a small company.
May maybe not big in in, sort ofbureaucracy, but big in impact
at least. And and so they'rethey're actually, you know,
dermatomyositis andnoninfectious uveitis are are 2
(20:20):
of, I think, many similar typesof diseases where, where we
could have, you know, have asimilar potential impact. And so
we're looking into starting someadditional clinical trials,
possibly as soon as next year,in 2025. And then, you know, in
(20:45):
in the longer term as well tothink about additional medicines
that can help this category ofof patients as well.
Because I think it's somethingthat, you know, we're very
excited about, and I think we'rejust at the at the tip of the
ice berg in terms of reallybeing able to help patients with
these diseases.
Dr. Moira Gunn (21:05):
Well, Ben, thank you so much for joining me. I
hope you'll come back, keep usupdated.
Ben Zimmer (21:10):
Thank you so much. I would love to, and I I really
appreciate the the time andopportunity.
Dr. Moira Gunn (21:15):
Ben Zimmer is the CEO of Priovant
Therapeutics. More informationis available at priovanttx.com.
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