March 5, 2025 • 19 mins
This week on BioTech Nation, Catalyst Pharmaceuticals CEO Rich Daly discusses the complexities of treating epilepsy and rare diseases. From the challenges of polypharmacy—where patients need multiple drugs to manage a single condition—to the hurdles of diagnosing and treating ultra-rare diseases.
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Episode Transcript
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Dr. Moira Gunn (00:11):
As hard as we try, it's not for each medical
condition, one drug. It'sfrequently many drugs. For such
common conditions as diabetesand epilepsy to even ultra rare
diseases, Getting thecombination right is no easy
matter. Today, we'll talk aboutseveral approaches to that
(00:33):
challenge with Rich Daley, thepresident and CEO of Catalyst
Pharmaceuticals. Rich, welcometo the program.
Rich Daly (00:41):
Thanks, Moira. It's great to be here. Appreciate it.
Dr. Moira Gunn (00:44):
While many of us are used to having one medical
condition or another, thatusually means we get a
prescription, we go to apharmacy, and the most we are
asked is, do you want the brandname or a generic? Well, today,
we're gonna talk about twosituations, each of which are
different, but we may findourselves in that's far more
(01:05):
than this. And I'm speaking toyou as Catalyst Pharmaceuticals.
And among your many efforts, Ihappen to be dealing with both
of these situations. Mhmm.
The first is any single medicalcondition, which a person may
need multiple drugs working incombination to address their
(01:26):
needs foreseeably, sometimes forthe rest of their lives. That's
called polypharmacy. And thereare many conditions which fall
under this, but the example Iwanna use today is epilepsy.
Fairly common, well knowncondition, although not all
epilepsy is the same. Now, Rich,tell us about epilepsy and how
it may fit under this umbrellaof polypharmacy.
Rich Daly (01:49):
Sure. In The US, there are about three million
people who have epilepsy, andabout five hundred thousand of
those are children.Interestingly enough, Moira,
every year, about a hundred andfifty thousand people are
diagnosed with epilepsy, newlydiagnosed. And the really
interesting thing, and I thinkyour listeners will find this
(02:09):
incredibly interesting, is aboutfifty percent of those newly
diagnosed patients are above 65years of age. I was shocked when
I heard that.
That that was really surprisingto me. And the burden here on
patients is really incredible.The burden on their family is
really incredible. So being ableto stop a seizure is very, very
important. Being able to controlseizures is really important.
(02:32):
Anywhere between twenty five andsixty nine percent of people
with epilepsy are unemployed.It's amazing. It's absolutely
amazing. So really getting thisunder control, getting a
seizures under control is reallyimportant. And so your point
about polypharmacy is spot on.
And nobody knows which drug, andthere are many, many drugs that
(02:54):
can be used, to control,seizures. But nobody really
knows. The doctor doesn't knowwhen they give that first drug
if that's gonna work or if it'sgonna be another drug. So it's
really kind of a anexperimentation to start with.
Dr. Moira Gunn (03:07):
Well, 95% of those drugs are generic, which
means they're very affordable.New drugs are not very
affordable, and the numbers aresimple. I say them all the time.
It takes twelve to fifteen yearsto go from the lab bench to a
real product. It takes 1 to$2,000,000,000, and only one out
of nine attempts are successful.
(03:29):
And in this market with so manygenerics, why has Catalyst
developed a new epilepsy drug?It's called Ficombe. And what is
its aim? And I know it's alreadyproved it's on the market.
Rich Daly (03:43):
Yeah. So it's been on the market for about ten years.
And as I said earlier, there'sjust so many different ways to
approach, epilepsy and try andcontrol it. And Ficompa is one
of a kind. Without going into alot of science, it basically is
one of the drugs that's used tocontrol overactive brain
activity just like all the otherones, but it just does it in a
(04:05):
unique way.
And so when you see this, thephysicians can look at it and
say, okay. I'm trying theseother types of drugs. Let me
supplement my activity or myaction with the patient with
this other drug, Ficampa. Sothey try and use it to see if
they can offset the overactivityof the brain because the
recognition is quite strong thatyou do need more than one
(04:27):
approach for many of yourpatients. More than a third of
patients really do need to havemore than one drug on board, and
some of them need three.
Dr. Moira Gunn (04:36):
Now let's turn to another pharmaceutical
situation. And that's when youor your loved one has a rare
disease, even an ultra raredisease and something Sure. Or
something called a difficult totreat disease. You can't just
get a prescribed dose, go downto your local pharmacy, call us
in a week or two, tell us howyou're doing. You have to get
(04:57):
the dose right, and there's anart to the science of treating
these patients even with anapproved drug.
So let's talk about another ofcatalyst approved drugs. This
one is Firdapse, and it treats acondition called Lambert Eaton
myasthenic syndrome. So tell usabout Lambert Eaton. Who has it?
(05:19):
What is it like to have it?
And how do you get the doseright?
Rich Daly (05:23):
Well, this is a really interesting, opportunity
to talk about this. So on youhave on the one side, you have
epilepsy with three millionpatients as we talked about
earlier. And on this side,Lambert Eaton myasthenic
syndrome, maybe there are fortyfive hundred patients in The US
with this, and many of them areundiagnosed. The Lambert Eaton
(05:45):
myasthenic syndrome looks a lotlike myasthenia gravis. It's
generally muscle weakness, thatpatients really don't recognize.
And so it's very hard for aphysician to recognize it. In
fact, we have been in this spacefor six years. The drug our
drug, Firdapse, has beenapproved for six years. And the
(06:07):
average physician who isdiagnosing a patient right now
over or I should say over thelast six years, the average
physician will only see oneLambert neet and myasthenic
patient in their entire career.That's how challenging it is to
see, to identify, to diagnose,and then to treat.
So it's an incrediblychallenging space to work in for
(06:29):
the physician and obviously fora company like Catalyst.
Dr. Moira Gunn (06:33):
When we were talking about this drug earlier,
you said something that caughtmy attention. You said, if dad
or mom loses their job and withthat, their health insurance
goes away, You continue toprovide the drug?
Rich Daly (06:47):
We do. We do. I've been working in the orphan or
rare space, for more than twentyyears, and it's a very patient
commitment oriented space. Someof these conditions like Lambert
Eaton, if the patient stopstaking the drug, specifically
Lambert Eaton, if the patientmisses a dose, some of these
(07:09):
patients will lose the abilityto walk. And then when they get
the drug again, they have theability to walk within fifteen
or twenty minutes.
So having the ability to get thedrug is incredibly important. So
before I became the CEO ofCatalyst, I was on the board for
nine years. And I remember,before the company became a
commercial company, we weretalking about what's our
(07:30):
philosophy, how do we thinkabout the how we interact with
patients. And one of the thingswe talked about at the board
level was we think that it'simportant that once a patient
starts on the drug, whether ornot they have the ability to
continue to pay for the drug,they should be given access to
the drug. And there are certainlimits to this, because of some
(07:51):
federal laws, which we won't getinto right now.
But we give that patient thataccess to the drug regardless of
their ability to pay. So if apatient loses their insurance,
we give them free drug. Or wegive them access to, certain
charities, and we help them geton free drug. And then we give
the drug to the charity, andthen, obviously, the charity
gives the drug to the patient.Because we believe our
(08:12):
commitment is to make sure thatthat patient continues to get
drug no matter what.
Because in many cases, it's thedifference between that patient
working or not working,providing for their family or
not, or living what's as closeto a normal life as possible. So
this is a very, very importantthing to us.
Dr. Moira Gunn (08:30):
Well, I'm gonna go out on a limb, not checking
my data, but I'm gonna bet thereare more pharmacies in The
United States than there arepeople with this condition.
We're talking there's not thatmany people, but they need this
drug.
Rich Daly (08:44):
Right.
Dr. Moira Gunn (08:44):
And that doesn't answer the part of the question
that I think is so important,which is how do you I mean, you
got you gotta go to a pharmacyto get your prescribed drug. How
do you fine tune and get theright dose? I mean, how does
that all work?
Rich Daly (09:03):
So that's a great point. So we actually bring the
pharmacy to the patient. We workwith a company called the
Specialty Pharmacy. It's not ourcompany. It's, a a different
company.
We contract with them, and theyget to know the patient. So we
have the appropriate separationfrom the patient. We're HIPAA
compliant in this way. And thepharmacy, the specialized
pharmacy gets to know thepatient. They specialize in
(09:26):
Lambert Eaton.
They focus on the medication,Firdapse. They have high touch
with the patient, so they knowthe patient personally.
Obviously, the patient givesthem permission to know them. So
and the goal of this is toincrease access to the
medication, make sure that theycan navigate the insurance,
which is incredibly difficultfor a patient who has a rare
(09:47):
disease. They understand thediagnosis information, and it
helps the patient to increasetheir adherence to the drug,
making sure they get the drug ontime.
Again, as I said, if they misstheir doses, it can be
devastating. And then theycontinually outreach to the
patient to make sure they'redoing well. Are they contacting
their doctor? Are they seeingtheir doctor on a regular basis?
(10:08):
Just to make sure that theircare is up to par.
And so that's the purpose of thespecialty pharmacy is to have
that relationship with thepatient. And so we bring the the
pharmacy to the patient.Obviously, it's done virtually,
but, you know, that's the way wework.
Dr. Moira Gunn (10:23):
Well, losing your job or something like that
isn't the only condition youwere talking about that would
cause you to go and and findanother way to get drugs to a
patient. You were talking abouta young man who had been
receiving the drug via Medicaid.
Rich Daly (10:41):
Mhmm.
Dr. Moira Gunn (10:42):
And for some reason, he outgrew or no longer
qualified for his Medicaidcoverage. How are you able to
help him?
Rich Daly (10:50):
Yeah. So this is a really, really interesting
story, Maura. So this young manwas getting, the drug for a
condition for which we are notapproved. So we couldn't help
him per se. Right?
So he was getting it throughMedicaid, and he aged out of his
parents' Medicaid coverage. Andso the parents contacted us. He
(11:11):
contacted us and said, hey. Canyou help us? And we said,
actually, we can't because thedrug is not approved for you for
use for your, for yourcondition.
So there's not a lot we can do.But we what we were able to do
was to point him in thedirection of, of his own
physician and say, if yourphysician opens a new IND or a,
(11:32):
an investigative new drugapplication for him
individually, then we can supplythat drug to the physician
because while we can't encouragenew INDs for for what's called
off label use, so this is when adrug is not approved. But since
he seems to be getting benefitfrom the drug itself right now,
(11:55):
we can continue to supply thedrug to that, patient through an
IND. But we couldn't do that ifhe was starting on it de novo or
or brand new. So, we continue towork with patients in that
manner.
Dr. Moira Gunn (12:08):
So an IND is what a drug company would file.
It's an investigational new drugwhen they're ready to go with
their well tested compound andall of that, and yet and that's
with the FDA. This young man hadbeen using the drug, did not
have Lamborghini
Rich Daly (12:24):
Correct.
Dr. Moira Gunn (12:25):
But it proved to be effective. So it was the same
IND, the same government formwas able to then have the doctor
make him an investigation of onegiven the experience he'd
already had.
Rich Daly (12:38):
As long as the doctor files the paperwork
appropriately with the FDA, wecan we can work with that
physician. Again, if the patienthad not been on the drug and not
received any benefit, then thethe risks might outweigh the
benefit. We wouldn't know that.But as long as he was on that
drug previously and had gainedsome benefit or his physician
had assumed he had gained somebenefit, we're willing to work
(12:58):
with them on that. Yeah.
Dr. Moira Gunn (13:00):
Now I noticed from your website that an
increased dosage of Firdapse hasjust been approved by the FDA.
It went from an eighty milligrama day maximum up to a hundred
milligram a day maximum. How didthat come about?
Rich Daly (13:15):
So physicians have been asking for this flexibility
for a while. We've been on themarket. Firdapse has been on the
market for six years. And, acouple of years ago, we heard
this, request. And so we fileddata with the FDA at the request
of the physicians who areworking with the patients.
And we filed what's called ansNDA or a supplemental new drug
(13:36):
application for an increaseddose because of the request for
this flexibility. And in June oflast year, the new dose was
approved. And what we're seeingis patients are in fact using a
higher dose because this is asomewhat sometimes this can be a
progressive, condition. It's notalways, but it can be a
(13:57):
progressive condition. So apatient may be on, let's say,
sixty milligrams a day, but mayprogress.
And then actually, they mayactually find that their
condition or symptoms get worse,so they may need a higher dose
of drug. Or a patient may have aa condition associated with
cancer. Sometimes that happens,and they may need actually a
(14:18):
higher dose as well. So wewanted to give those physicians
who were requesting thatflexibility the ability to do
that and get insurance coveragefor it. And that's very
important.
Dr. Moira Gunn (14:28):
How did Catalyst come to this? I'll call it going
the extra mile commitment. Imean, you don't generally teach
this in business school.
Rich Daly (14:38):
Yeah. So this is a really good point. Working in
the orphan or rare diseasespace, starting in the middle
nineties, I worked in a very,very small even smaller than
this opportunity. And, it was alesson I learned there that,
many of the patients we workedwith, their families were,
(14:59):
unable to afford the medication.But once they started, we made a
commitment at the company I wasat.
And this is very early in theorphan commercialization. You
know, the Orphan Drug Act waspassed in 1983. And so the first
drug started coming out in thelate eighties, early nineties.
And so, we were, on the cusp ofdoing some things. And we've
(15:20):
just decided that once a patientstarted, we just couldn't see a
reason why the patient shouldn'tfinish.
And this is a short coursetherapy. And, like I said, when
I came on the board here, itjust was I mentioned it in one
board meeting. I said, this iswhat we have to do. And I joined
the board here as the, as aperson with the deepest
(15:41):
commercial experience in Orphan.And to a person, everybody on
the board, it was a two minutediscussion.
Everybody said, yeah. That makessense. We should do that. That's
the right thing to do. And so II think you you do well by doing
good.
And so it's the right thing. Sowe're we're really proud of it.
And, it's a you can feel theenergy when things when this
(16:03):
comes into play. So, we've had acouple of situations where, like
the young man we just talkedabout or broader situations
where we've had to, really putthis into effect, and people are
just so proud so proud that thecompany does this for patients.
Dr. Moira Gunn (16:22):
You've had your own experience with dealing with
polypharmacy.
Rich Daly (16:27):
Yeah. Yeah. I've had a lot of inflection points in my
life, you know, whether it's,you know, going to school or and
marrying Susan, my wife, andhaving a family. Or, you know,
my mom, actually had, diabetesand so was on polypharmacy. And
so I I learned a lot about it atthat point in time.
(16:47):
But I also was diagnosed in the,early two thousands with a a
brain tumor. And, thankfully forme, it was benign. It was a
benign brain tumor. But as aresult of the tumor, I developed
epilepsy. And so, I'm intimatelyfamiliar with the epilepsy
world.
And, I myself, have to takemultiple drugs twice a day, to
(17:13):
prevent seizures. And, it tookthree years for me to con to get
my seizures under control. And,I'm one of the the the lucky
ones who, actually is able tocontrol my seizures. And, it was
a struggle. It was a real, realstruggle to get them under
(17:35):
control and, you know, tryingmultiple drugs.
And as I said, it's an art morethan a science, to figure out
which ones are gonna work foryou. So, I'm really thankful I
have a great epileptologist.And, yeah. And so being in this
field is not by design for me,but it's just happenstance. And,
I can really and when I go outand speak with patients, it's
(17:58):
it's it's from personalexperience.
And I think most of the patientsare actually shocked, you know,
to see somebody in this rolethat can actually speak to them
and say, oh, no. I I knowexactly where you're coming
from, and not as a businessperson, but as a person. So it's
really, rewarding.
Dr. Moira Gunn (18:13):
Well, Rich, thank you so much for coming on.
I hope you'll come back and seeus again.
Rich Daly (18:17):
Alright. Thank you. It's been a pleasure, and I
really have enjoyed it. Sothanks very much.
Dr. Moira Gunn (18:22):
Rich Daley is the president and CEO of
Catalyst Pharmaceuticals. Moreinformation is available on the
web at catalystpharma.com.
As hard as we try, it's not for each medical
condition, one drug. It'sfrequently many drugs. For such
common conditions as diabetesand epilepsy to even ultra rare
diseases, Getting thecombination right is no easy
matter. Today, we'll talk aboutseveral approaches to that
(00:33):
challenge with Rich Daley, thepresident and CEO of Catalyst
Pharmaceuticals. Rich, welcometo the program.
Rich Daly (00:41):
Thanks, Moira. It's great to be here. Appreciate it.
Dr. Moira Gunn (00:44):
While many of us are used to having one medical
condition or another, thatusually means we get a
prescription, we go to apharmacy, and the most we are
asked is, do you want the brandname or a generic? Well, today,
we're gonna talk about twosituations, each of which are
different, but we may findourselves in that's far more
(01:05):
than this. And I'm speaking toyou as Catalyst Pharmaceuticals.
And among your many efforts, Ihappen to be dealing with both
of these situations. Mhmm.
The first is any single medicalcondition, which a person may
need multiple drugs working incombination to address their
(01:26):
needs foreseeably, sometimes forthe rest of their lives. That's
called polypharmacy. And thereare many conditions which fall
under this, but the example Iwanna use today is epilepsy.
Fairly common, well knowncondition, although not all
epilepsy is the same. Now, Rich,tell us about epilepsy and how
it may fit under this umbrellaof polypharmacy.
Rich Daly (01:49):
Sure. In The US, there are about three million
people who have epilepsy, andabout five hundred thousand of
those are children.Interestingly enough, Moira,
every year, about a hundred andfifty thousand people are
diagnosed with epilepsy, newlydiagnosed. And the really
interesting thing, and I thinkyour listeners will find this
(02:09):
incredibly interesting, is aboutfifty percent of those newly
diagnosed patients are above 65years of age. I was shocked when
I heard that.
That that was really surprisingto me. And the burden here on
patients is really incredible.The burden on their family is
really incredible. So being ableto stop a seizure is very, very
important. Being able to controlseizures is really important.
(02:32):
Anywhere between twenty five andsixty nine percent of people
with epilepsy are unemployed.It's amazing. It's absolutely
amazing. So really getting thisunder control, getting a
seizures under control is reallyimportant. And so your point
about polypharmacy is spot on.
And nobody knows which drug, andthere are many, many drugs that
(02:54):
can be used, to control,seizures. But nobody really
knows. The doctor doesn't knowwhen they give that first drug
if that's gonna work or if it'sgonna be another drug. So it's
really kind of a anexperimentation to start with.
Dr. Moira Gunn (03:07):
Well, 95% of those drugs are generic, which
means they're very affordable.New drugs are not very
affordable, and the numbers aresimple. I say them all the time.
It takes twelve to fifteen yearsto go from the lab bench to a
real product. It takes 1 to$2,000,000,000, and only one out
of nine attempts are successful.
(03:29):
And in this market with so manygenerics, why has Catalyst
developed a new epilepsy drug?It's called Ficombe. And what is
its aim? And I know it's alreadyproved it's on the market.
Rich Daly (03:43):
Yeah. So it's been on the market for about ten years.
And as I said earlier, there'sjust so many different ways to
approach, epilepsy and try andcontrol it. And Ficompa is one
of a kind. Without going into alot of science, it basically is
one of the drugs that's used tocontrol overactive brain
activity just like all the otherones, but it just does it in a
(04:05):
unique way.
And so when you see this, thephysicians can look at it and
say, okay. I'm trying theseother types of drugs. Let me
supplement my activity or myaction with the patient with
this other drug, Ficampa. Sothey try and use it to see if
they can offset the overactivityof the brain because the
recognition is quite strong thatyou do need more than one
(04:27):
approach for many of yourpatients. More than a third of
patients really do need to havemore than one drug on board, and
some of them need three.
Dr. Moira Gunn (04:36):
Now let's turn to another pharmaceutical
situation. And that's when youor your loved one has a rare
disease, even an ultra raredisease and something Sure. Or
something called a difficult totreat disease. You can't just
get a prescribed dose, go downto your local pharmacy, call us
in a week or two, tell us howyou're doing. You have to get
(04:57):
the dose right, and there's anart to the science of treating
these patients even with anapproved drug.
So let's talk about another ofcatalyst approved drugs. This
one is Firdapse, and it treats acondition called Lambert Eaton
myasthenic syndrome. So tell usabout Lambert Eaton. Who has it?
(05:19):
What is it like to have it?
And how do you get the doseright?
Rich Daly (05:23):
Well, this is a really interesting, opportunity
to talk about this. So on youhave on the one side, you have
epilepsy with three millionpatients as we talked about
earlier. And on this side,Lambert Eaton myasthenic
syndrome, maybe there are fortyfive hundred patients in The US
with this, and many of them areundiagnosed. The Lambert Eaton
(05:45):
myasthenic syndrome looks a lotlike myasthenia gravis. It's
generally muscle weakness, thatpatients really don't recognize.
And so it's very hard for aphysician to recognize it. In
fact, we have been in this spacefor six years. The drug our
drug, Firdapse, has beenapproved for six years. And the
(06:07):
average physician who isdiagnosing a patient right now
over or I should say over thelast six years, the average
physician will only see oneLambert neet and myasthenic
patient in their entire career.That's how challenging it is to
see, to identify, to diagnose,and then to treat.
So it's an incrediblychallenging space to work in for
(06:29):
the physician and obviously fora company like Catalyst.
Dr. Moira Gunn (06:33):
When we were talking about this drug earlier,
you said something that caughtmy attention. You said, if dad
or mom loses their job and withthat, their health insurance
goes away, You continue toprovide the drug?
Rich Daly (06:47):
We do. We do. I've been working in the orphan or
rare space, for more than twentyyears, and it's a very patient
commitment oriented space. Someof these conditions like Lambert
Eaton, if the patient stopstaking the drug, specifically
Lambert Eaton, if the patientmisses a dose, some of these
(07:09):
patients will lose the abilityto walk. And then when they get
the drug again, they have theability to walk within fifteen
or twenty minutes.
So having the ability to get thedrug is incredibly important. So
before I became the CEO ofCatalyst, I was on the board for
nine years. And I remember,before the company became a
commercial company, we weretalking about what's our
(07:30):
philosophy, how do we thinkabout the how we interact with
patients. And one of the thingswe talked about at the board
level was we think that it'simportant that once a patient
starts on the drug, whether ornot they have the ability to
continue to pay for the drug,they should be given access to
the drug. And there are certainlimits to this, because of some
(07:51):
federal laws, which we won't getinto right now.
But we give that patient thataccess to the drug regardless of
their ability to pay. So if apatient loses their insurance,
we give them free drug. Or wegive them access to, certain
charities, and we help them geton free drug. And then we give
the drug to the charity, andthen, obviously, the charity
gives the drug to the patient.Because we believe our
(08:12):
commitment is to make sure thatthat patient continues to get
drug no matter what.
Because in many cases, it's thedifference between that patient
working or not working,providing for their family or
not, or living what's as closeto a normal life as possible. So
this is a very, very importantthing to us.
Dr. Moira Gunn (08:30):
Well, I'm gonna go out on a limb, not checking
my data, but I'm gonna bet thereare more pharmacies in The
United States than there arepeople with this condition.
We're talking there's not thatmany people, but they need this
drug.
Rich Daly (08:44):
Right.
Dr. Moira Gunn (08:44):
And that doesn't answer the part of the question
that I think is so important,which is how do you I mean, you
got you gotta go to a pharmacyto get your prescribed drug. How
do you fine tune and get theright dose? I mean, how does
that all work?
Rich Daly (09:03):
So that's a great point. So we actually bring the
pharmacy to the patient. We workwith a company called the
Specialty Pharmacy. It's not ourcompany. It's, a a different
company.
We contract with them, and theyget to know the patient. So we
have the appropriate separationfrom the patient. We're HIPAA
compliant in this way. And thepharmacy, the specialized
pharmacy gets to know thepatient. They specialize in
(09:26):
Lambert Eaton.
They focus on the medication,Firdapse. They have high touch
with the patient, so they knowthe patient personally.
Obviously, the patient givesthem permission to know them. So
and the goal of this is toincrease access to the
medication, make sure that theycan navigate the insurance,
which is incredibly difficultfor a patient who has a rare
(09:47):
disease. They understand thediagnosis information, and it
helps the patient to increasetheir adherence to the drug,
making sure they get the drug ontime.
Again, as I said, if they misstheir doses, it can be
devastating. And then theycontinually outreach to the
patient to make sure they'redoing well. Are they contacting
their doctor? Are they seeingtheir doctor on a regular basis?
(10:08):
Just to make sure that theircare is up to par.
And so that's the purpose of thespecialty pharmacy is to have
that relationship with thepatient. And so we bring the the
pharmacy to the patient.Obviously, it's done virtually,
but, you know, that's the way wework.
Dr. Moira Gunn (10:23):
Well, losing your job or something like that
isn't the only condition youwere talking about that would
cause you to go and and findanother way to get drugs to a
patient. You were talking abouta young man who had been
receiving the drug via Medicaid.
Rich Daly (10:41):
Mhmm.
Dr. Moira Gunn (10:42):
And for some reason, he outgrew or no longer
qualified for his Medicaidcoverage. How are you able to
help him?
Rich Daly (10:50):
Yeah. So this is a really, really interesting
story, Maura. So this young manwas getting, the drug for a
condition for which we are notapproved. So we couldn't help
him per se. Right?
So he was getting it throughMedicaid, and he aged out of his
parents' Medicaid coverage. Andso the parents contacted us. He
(11:11):
contacted us and said, hey. Canyou help us? And we said,
actually, we can't because thedrug is not approved for you for
use for your, for yourcondition.
So there's not a lot we can do.But we what we were able to do
was to point him in thedirection of, of his own
physician and say, if yourphysician opens a new IND or a,
(11:32):
an investigative new drugapplication for him
individually, then we can supplythat drug to the physician
because while we can't encouragenew INDs for for what's called
off label use, so this is when adrug is not approved. But since
he seems to be getting benefitfrom the drug itself right now,
(11:55):
we can continue to supply thedrug to that, patient through an
IND. But we couldn't do that ifhe was starting on it de novo or
or brand new. So, we continue towork with patients in that
manner.
Dr. Moira Gunn (12:08):
So an IND is what a drug company would file.
It's an investigational new drugwhen they're ready to go with
their well tested compound andall of that, and yet and that's
with the FDA. This young man hadbeen using the drug, did not
have Lamborghini
Rich Daly (12:24):
Correct.
Dr. Moira Gunn (12:25):
But it proved to be effective. So it was the same
IND, the same government formwas able to then have the doctor
make him an investigation of onegiven the experience he'd
already had.
Rich Daly (12:38):
As long as the doctor files the paperwork
appropriately with the FDA, wecan we can work with that
physician. Again, if the patienthad not been on the drug and not
received any benefit, then thethe risks might outweigh the
benefit. We wouldn't know that.But as long as he was on that
drug previously and had gainedsome benefit or his physician
had assumed he had gained somebenefit, we're willing to work
(12:58):
with them on that. Yeah.
Dr. Moira Gunn (13:00):
Now I noticed from your website that an
increased dosage of Firdapse hasjust been approved by the FDA.
It went from an eighty milligrama day maximum up to a hundred
milligram a day maximum. How didthat come about?
Rich Daly (13:15):
So physicians have been asking for this flexibility
for a while. We've been on themarket. Firdapse has been on the
market for six years. And, acouple of years ago, we heard
this, request. And so we fileddata with the FDA at the request
of the physicians who areworking with the patients.
And we filed what's called ansNDA or a supplemental new drug
(13:36):
application for an increaseddose because of the request for
this flexibility. And in June oflast year, the new dose was
approved. And what we're seeingis patients are in fact using a
higher dose because this is asomewhat sometimes this can be a
progressive, condition. It's notalways, but it can be a
(13:57):
progressive condition. So apatient may be on, let's say,
sixty milligrams a day, but mayprogress.
And then actually, they mayactually find that their
condition or symptoms get worse,so they may need a higher dose
of drug. Or a patient may have aa condition associated with
cancer. Sometimes that happens,and they may need actually a
(14:18):
higher dose as well. So wewanted to give those physicians
who were requesting thatflexibility the ability to do
that and get insurance coveragefor it. And that's very
important.
Dr. Moira Gunn (14:28):
How did Catalyst come to this? I'll call it going
the extra mile commitment. Imean, you don't generally teach
this in business school.
Rich Daly (14:38):
Yeah. So this is a really good point. Working in
the orphan or rare diseasespace, starting in the middle
nineties, I worked in a very,very small even smaller than
this opportunity. And, it was alesson I learned there that,
many of the patients we workedwith, their families were,
(14:59):
unable to afford the medication.But once they started, we made a
commitment at the company I wasat.
And this is very early in theorphan commercialization. You
know, the Orphan Drug Act waspassed in 1983. And so the first
drug started coming out in thelate eighties, early nineties.
And so, we were, on the cusp ofdoing some things. And we've
(15:20):
just decided that once a patientstarted, we just couldn't see a
reason why the patient shouldn'tfinish.
And this is a short coursetherapy. And, like I said, when
I came on the board here, itjust was I mentioned it in one
board meeting. I said, this iswhat we have to do. And I joined
the board here as the, as aperson with the deepest
(15:41):
commercial experience in Orphan.And to a person, everybody on
the board, it was a two minutediscussion.
Everybody said, yeah. That makessense. We should do that. That's
the right thing to do. And so II think you you do well by doing
good.
And so it's the right thing. Sowe're we're really proud of it.
And, it's a you can feel theenergy when things when this
(16:03):
comes into play. So, we've had acouple of situations where, like
the young man we just talkedabout or broader situations
where we've had to, really putthis into effect, and people are
just so proud so proud that thecompany does this for patients.
Dr. Moira Gunn (16:22):
You've had your own experience with dealing with
polypharmacy.
Rich Daly (16:27):
Yeah. Yeah. I've had a lot of inflection points in my
life, you know, whether it's,you know, going to school or and
marrying Susan, my wife, andhaving a family. Or, you know,
my mom, actually had, diabetesand so was on polypharmacy. And
so I I learned a lot about it atthat point in time.
(16:47):
But I also was diagnosed in the,early two thousands with a a
brain tumor. And, thankfully forme, it was benign. It was a
benign brain tumor. But as aresult of the tumor, I developed
epilepsy. And so, I'm intimatelyfamiliar with the epilepsy
world.
And, I myself, have to takemultiple drugs twice a day, to
(17:13):
prevent seizures. And, it tookthree years for me to con to get
my seizures under control. And,I'm one of the the the lucky
ones who, actually is able tocontrol my seizures. And, it was
a struggle. It was a real, realstruggle to get them under
(17:35):
control and, you know, tryingmultiple drugs.
And as I said, it's an art morethan a science, to figure out
which ones are gonna work foryou. So, I'm really thankful I
have a great epileptologist.And, yeah. And so being in this
field is not by design for me,but it's just happenstance. And,
I can really and when I go outand speak with patients, it's
(17:58):
it's it's from personalexperience.
And I think most of the patientsare actually shocked, you know,
to see somebody in this rolethat can actually speak to them
and say, oh, no. I I knowexactly where you're coming
from, and not as a businessperson, but as a person. So it's
really, rewarding.
Dr. Moira Gunn (18:13):
Well, Rich, thank you so much for coming on.
I hope you'll come back and seeus again.
Rich Daly (18:17):
Alright. Thank you. It's been a pleasure, and I
really have enjoyed it. Sothanks very much.
Dr. Moira Gunn (18:22):
Rich Daley is the president and CEO of
Catalyst Pharmaceuticals. Moreinformation is available on the
web at catalystpharma.com.
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