Why One Drug Isn't Always Enough... Rich Daly, President & CEO, Catalyst Pharmaceuticals
As hard as we try, it's not for each medical condition, one drug. It's frequently many drugs. For such common conditions as diabetes and epilepsy to even ultra rare diseases, Getting the combination right is no easy matter. Today, we'll talk about several approaches to that challenge with Rich Daley, the president and CEO of Catalyst Pharmaceuticals. Rich, welcome to the program.
Rich Daly:Thanks, Moira. It's great to be here. Appreciate it.
Dr. Moira Gunn:While many of us are used to having one medical condition or another, that usually means we get a prescription, we go to a pharmacy, and the most we are asked is, do you want the brand name or a generic? Well, today, we're gonna talk about two situations, each of which are different, but we may find ourselves in that's far more than this. And I'm speaking to you as Catalyst Pharmaceuticals. And among your many efforts, I happen to be dealing with both of these situations. Mhmm.
Dr. Moira Gunn:The first is any single medical condition, which a person may need multiple drugs working in combination to address their needs foreseeably, sometimes for the rest of their lives. That's called polypharmacy. And there are many conditions which fall under this, but the example I wanna use today is epilepsy. Fairly common, well known condition, although not all epilepsy is the same. Now, Rich, tell us about epilepsy and how it may fit under this umbrella of polypharmacy.
Rich Daly:Sure. In The US, there are about three million people who have epilepsy, and about five hundred thousand of those are children. Interestingly enough, Moira, every year, about a hundred and fifty thousand people are diagnosed with epilepsy, newly diagnosed. And the really interesting thing, and I think your listeners will find this incredibly interesting, is about fifty percent of those newly diagnosed patients are above 65 years of age. I was shocked when I heard that.
Rich Daly:That that was really surprising to me. And the burden here on patients is really incredible. The burden on their family is really incredible. So being able to stop a seizure is very, very important. Being able to control seizures is really important.
Rich Daly:Anywhere between twenty five and sixty nine percent of people with epilepsy are unemployed. It's amazing. It's absolutely amazing. So really getting this under control, getting a seizures under control is really important. And so your point about polypharmacy is spot on.
Rich Daly:And nobody knows which drug, and there are many, many drugs that can be used, to control, seizures. But nobody really knows. The doctor doesn't know when they give that first drug if that's gonna work or if it's gonna be another drug. So it's really kind of a an experimentation to start with.
Dr. Moira Gunn:Well, 95% of those drugs are generic, which means they're very affordable. New drugs are not very affordable, and the numbers are simple. I say them all the time. It takes twelve to fifteen years to 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.
Dr. Moira Gunn:And in this market with so many generics, why has Catalyst developed a new epilepsy drug? It's called Ficombe. And what is its aim? And I know it's already proved it's on the market.
Rich Daly:Yeah. So it's been on the market for about ten years. And as I said earlier, there's just so many different ways to approach, epilepsy and try and control it. And Ficompa is one of a kind. Without going into a lot of science, it basically is one of the drugs that's used to control overactive brain activity just like all the other ones, but it just does it in a unique way.
Rich Daly:And so when you see this, the physicians can look at it and say, okay. I'm trying these other types of drugs. Let me supplement my activity or my action with the patient with this other drug, Ficampa. So they try and use it to see if they can offset the overactivity of the brain because the recognition is quite strong that you do need more than one approach for many of your patients. More than a third of patients really do need to have more than one drug on board, and some of them need three.
Dr. Moira Gunn:Now let's turn to another pharmaceutical situation. And that's when you or your loved one has a rare disease, even an ultra rare disease and something Sure. Or something called a difficult to treat disease. You can't just get a prescribed dose, go down to your local pharmacy, call us in a week or two, tell us how you're doing. You have to get the dose right, and there's an art to the science of treating these patients even with an approved drug.
Dr. Moira Gunn:So let's talk about another of catalyst approved drugs. This one is Firdapse, and it treats a condition called Lambert Eaton myasthenic syndrome. So tell us about Lambert Eaton. Who has it? What is it like to have it?
Dr. Moira Gunn:And how do you get the dose right?
Rich Daly:Well, this is a really interesting, opportunity to talk about this. So on you have on the one side, you have epilepsy with three million patients as we talked about earlier. And on this side, Lambert Eaton myasthenic syndrome, maybe there are forty five hundred patients in The US with this, and many of them are undiagnosed. The Lambert Eaton myasthenic syndrome looks a lot like myasthenia gravis. It's generally muscle weakness, that patients really don't recognize.
Rich Daly:And so it's very hard for a physician to recognize it. In fact, we have been in this space for six years. The drug our drug, Firdapse, has been approved for six years. And the average physician who is diagnosing a patient right now over or I should say over the last six years, the average physician will only see one Lambert neet and myasthenic patient in their entire career. That's how challenging it is to see, to identify, to diagnose, and then to treat.
Rich Daly:So it's an incredibly challenging space to work in for the physician and obviously for a company like Catalyst.
Dr. Moira Gunn:When we were talking about this drug earlier, you said something that caught my attention. You said, if dad or mom loses their job and with that, their health insurance goes away, You continue to provide the drug?
Rich Daly:We do. We do. I've been working in the orphan or rare space, for more than twenty years, and it's a very patient commitment oriented space. Some of these conditions like Lambert Eaton, if the patient stops taking the drug, specifically Lambert Eaton, if the patient misses a dose, some of these patients will lose the ability to walk. And then when they get the drug again, they have the ability to walk within fifteen or twenty minutes.
Rich Daly:So having the ability to get the drug is incredibly important. So before I became the CEO of Catalyst, I was on the board for nine years. And I remember, before the company became a commercial company, we were talking about what's our philosophy, how do we think about the how we interact with patients. And one of the things we talked about at the board level was we think that it's important that once a patient starts on the drug, whether or not they have the ability to continue to pay for the drug, they should be given access to the drug. And there are certain limits to this, because of some federal laws, which we won't get into right now.
Rich Daly:But we give that patient that access to the drug regardless of their ability to pay. So if a patient loses their insurance, we give them free drug. Or we give them access to, certain charities, and we help them get on free drug. And then we give the drug to the charity, and then, obviously, the charity gives the drug to the patient. Because we believe our commitment is to make sure that that patient continues to get drug no matter what.
Rich Daly:Because in many cases, it's the difference between that patient working or not working, providing for their family or not, or living what's as close to a normal life as possible. So this is a very, very important thing to us.
Dr. Moira Gunn:Well, I'm gonna go out on a limb, not checking my data, but I'm gonna bet there are more pharmacies in The United States than there are people with this condition. We're talking there's not that many people, but they need this drug.
Rich Daly:Right.
Dr. Moira Gunn: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 pharmacy to get your prescribed drug. How do you fine tune and get the right dose? I mean, how does that all work?
Rich Daly:So that's a great point. So we actually bring the pharmacy to the patient. We work with a company called the Specialty Pharmacy. It's not our company. It's, a a different company.
Rich Daly:We contract with them, and they get to know the patient. So we have the appropriate separation from the patient. We're HIPAA compliant in this way. And the pharmacy, the specialized pharmacy gets to know the patient. They specialize in Lambert Eaton.
Rich Daly:They focus on the medication, Firdapse. They have high touch with the patient, so they know the patient personally. Obviously, the patient gives them permission to know them. So and the goal of this is to increase access to the medication, make sure that they can navigate the insurance, which is incredibly difficult for a patient who has a rare disease. They understand the diagnosis information, and it helps the patient to increase their adherence to the drug, making sure they get the drug on time.
Rich Daly:Again, as I said, if they miss their doses, it can be devastating. And then they continually outreach to the patient to make sure they're doing well. Are they contacting their doctor? Are they seeing their doctor on a regular basis? Just to make sure that their care is up to par.
Rich Daly:And so that's the purpose of the specialty pharmacy is to have that relationship with the patient. And so we bring the the pharmacy to the patient. Obviously, it's done virtually, but, you know, that's the way we work.
Dr. Moira Gunn:Well, losing your job or something like that isn't the only condition you were talking about that would cause you to go and and find another way to get drugs to a patient. You were talking about a young man who had been receiving the drug via Medicaid.
Rich Daly:Mhmm.
Dr. Moira Gunn:And for some reason, he outgrew or no longer qualified for his Medicaid coverage. How are you able to help him?
Rich Daly:Yeah. So this is a really, really interesting story, Maura. So this young man was getting, the drug for a condition for which we are not approved. So we couldn't help him per se. Right?
Rich Daly:So he was getting it through Medicaid, and he aged out of his parents' Medicaid coverage. And so the parents contacted us. He contacted us and said, hey. Can you help us? And we said, actually, we can't because the drug is not approved for you for use for your, for your condition.
Rich Daly:So there's not a lot we can do. But we what we were able to do was to point him in the direction of, of his own physician and say, if your physician opens a new IND or a, an investigative new drug application for him individually, then we can supply that drug to the physician because while we can't encourage new INDs for for what's called off label use, so this is when a drug is not approved. But since he seems to be getting benefit from the drug itself right now, we can continue to supply the drug to that, patient through an IND. But we couldn't do that if he was starting on it de novo or or brand new. So, we continue to work with patients in that manner.
Dr. Moira Gunn:So an IND is what a drug company would file. It's an investigational new drug when they're ready to go with their well tested compound and all of that, and yet and that's with the FDA. This young man had been using the drug, did not have Lamborghini
Rich Daly:Correct.
Dr. Moira Gunn:But it proved to be effective. So it was the same IND, the same government form was able to then have the doctor make him an investigation of one given the experience he'd already had.
Rich Daly:As long as the doctor files the paperwork appropriately with the FDA, we can we can work with that physician. Again, if the patient had not been on the drug and not received any benefit, then the the risks might outweigh the benefit. We wouldn't know that. But as long as he was on that drug previously and had gained some benefit or his physician had assumed he had gained some benefit, we're willing to work with them on that. Yeah.
Dr. Moira Gunn:Now I noticed from your website that an increased dosage of Firdapse has just been approved by the FDA. It went from an eighty milligram a day maximum up to a hundred milligram a day maximum. How did that come about?
Rich Daly:So physicians have been asking for this flexibility for a while. We've been on the market. Firdapse has been on the market for six years. And, a couple of years ago, we heard this, request. And so we filed data with the FDA at the request of the physicians who are working with the patients.
Rich Daly:And we filed what's called an sNDA or a supplemental new drug application for an increased dose because of the request for this flexibility. And in June of last year, the new dose was approved. And what we're seeing is patients are in fact using a higher dose because this is a somewhat sometimes this can be a progressive, condition. It's not always, but it can be a progressive condition. So a patient may be on, let's say, sixty milligrams a day, but may progress.
Rich Daly:And then actually, they may actually find that their condition or symptoms get worse, so they may need a higher dose of drug. Or a patient may have a a condition associated with cancer. Sometimes that happens, and they may need actually a higher dose as well. So we wanted to give those physicians who were requesting that flexibility the ability to do that and get insurance coverage for it. And that's very important.
Dr. Moira Gunn:How did Catalyst come to this? I'll call it going the extra mile commitment. I mean, you don't generally teach this in business school.
Rich Daly:Yeah. So this is a really good point. Working in the orphan or rare disease space, starting in the middle nineties, I worked in a very, very small even smaller than this opportunity. And, it was a lesson I learned there that, many of the patients we worked with, their families were, unable to afford the medication. But once they started, we made a commitment at the company I was at.
Rich Daly:And this is very early in the orphan commercialization. You know, the Orphan Drug Act was passed in 1983. And so the first drug started coming out in the late eighties, early nineties. And so, we were, on the cusp of doing some things. And we've just decided that once a patient started, we just couldn't see a reason why the patient shouldn't finish.
Rich Daly:And this is a short course therapy. And, like I said, when I came on the board here, it just was I mentioned it in one board meeting. I said, this is what we have to do. And I joined the board here as the, as a person with the deepest commercial experience in Orphan. And to a person, everybody on the board, it was a two minute discussion.
Rich Daly:Everybody said, yeah. That makes sense. We should do that. That's the right thing to do. And so I I think you you do well by doing good.
Rich Daly:And so it's the right thing. So we're we're really proud of it. And, it's a you can feel the energy when things when this comes into play. So, we've had a couple of situations where, like the young man we just talked about or broader situations where we've had to, really put this into effect, and people are just so proud so proud that the company does this for patients.
Dr. Moira Gunn:You've had your own experience with dealing with polypharmacy.
Rich Daly: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, and having a family. Or, you know, my mom, actually had, diabetes and so was on polypharmacy. And so I I learned a lot about it at that point in time.
Rich Daly:But I also was diagnosed in the, early two thousands with a a brain tumor. And, thankfully for me, it was benign. It was a benign brain tumor. But as a result of the tumor, I developed epilepsy. And so, I'm intimately familiar with the epilepsy world.
Rich Daly:And, I myself, have to take multiple drugs twice a day, to prevent seizures. And, it took three 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 to control my seizures. And, it was a struggle. It was a real, real struggle to get them under control and, you know, trying multiple drugs.
Rich Daly:And as I said, it's an art more than a science, to figure out which ones are gonna work for you. 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 out and speak with patients, it's it's it's from personal experience.
Rich Daly:And I think most of the patients are actually shocked, you know, to see somebody in this role that can actually speak to them and say, oh, no. I I know exactly where you're coming from, and not as a business person, but as a person. So it's really, rewarding.
Dr. Moira Gunn:Well, Rich, thank you so much for coming on. I hope you'll come back and see us again.
Rich Daly:Alright. Thank you. It's been a pleasure, and I really have enjoyed it. So thanks very much.
Dr. Moira Gunn:Rich Daley is the president and CEO of Catalyst Pharmaceuticals. More information is available on the web at catalystpharma.com.
