RARE Daily

Taking a Different Approach to Rare Epilepsies

June 20, 2024

Lennox-Gastaut syndrome and Dravet syndrome are two rare, developmental and epileptic encephalopathies. Drug developers have sought to address epilepsies by altering the electrical activity in the brain. Ovid therapeutics, though, has taken a novel approach with its experimental therapy soticlestat by seeking to restore homeostasis to the brain. We spoke to Meg Alexander, chief strategy officer of Ovid, about rare epilepsies, how the company’s experimental therapy soticlestat works, and the potential to apply the approach to other CNS conditions. Since recording this episode, there have been new results on soticlestat released. Ovid’s partner Takeda this week reported that soticlestat narrowly missed its primary endpoint in its phase 3 Dravet syndrome study while showing clinically meaningful and nominally significant effects in multiple key secondary efficacy endpoints. It also missed its primary endpoint of reduction in major motor drop seizures as compared to placebo in a separate phase 3 study in Lennox-Gastaut syndrome. Takeda said it will be engaging with regulators to determine the best path forward.

Daniel Levine: Meg, thanks for joining us.

Meg Alexander: Thanks for having me, Danny.

Daniel Levine: Well, we’re going to talk about rare epilepsies, Ovid, and its efforts to develop therapies to treat these conditions. Let’s start with the rare epilepsies, Lennox-Gastaut syndrome and Dravet syndrome. Can you explain what these are?

Meg Alexander: I can. So maybe let me just take a step back out, and I’ll zoom into Lennox-Gastaut and Dravet syndrome. Epilepsy is actually one of the oldest known conditions to humankind. We’ve literally known it for millennia, three to four millennia. Despite that, and despite many, many medicines that have come to the fore over the course of the last century, many, many epilepsies, and most particularly rare epilepsies called developmental epileptic encephalopathies, are not cured. More so, many of them don’t even have treatments that can abate the vast majority of the seizures, and that is very true of the two conditions that you just referenced, Lennox-Gastaut syndrome and Dravet syndrome. So both Lennox-Gastaut and Dravet are developmental epileptic encephalopathies. They’re rare and very, very, very severe epilepsy types, and I’ll walk you through each just from a high level and we can talk more about it together. But essentially, Lennox-Gastaut is a clinical diagnosis of a rare epilepsy. That means that patients that have a certain type of EEG pattern and experience a certain set of heterogeneous seizure types are diagnosed with Lennox-Gastaut syndrome, and it’s very severe. These are people who live with tens, even hundreds of seizures over the course of a month. Some even report thousands. Dravet syndrome is a little bit different. Dravet syndrome is actually and most often diagnosed as a genetic epilepsy, and it’s actually a mutation that occurs in a gene called SCN1A. And it’s a little bit more rare than Lennox-Gastaut, but it’s equally as devastating for the people and the family living with it. It’s most commonly associated with a different seizure type. People who are born with Dravet syndrome start with something called focal seizures that over time start to translate into convulsive seizures. Those are the types of seizures sometimes you see portrayed in movies where you see someone shaking or literally convulsing. Regardless, these are indications that just have complete very, very difficult lives for the people who have them. And many, many, many are refractory. We can talk a little bit more about that together.

Daniel Levine: I imagine people who don’t live with these conditions would be shocked by the frequency of seizures. How heterogeneous are these diseases?

Meg Alexander: Dravet is a little bit more consistent so we’ll start with that one and then we’ll talk about Lennox-Gastaut next. So Dravet, as I mentioned to you, is marked more by convulsive seizure types, and these can be quite extreme. People who live with Dravet can have 20 even 80 seizures in a month’s time, and some of these seizures when they’re prolonged, can actually convert into status epilepticus, which is terrifying. That’s essentially a medical emergency where a patient is having a seizure for more than five minutes or having seizures in a span of every five minutes. So really, really scary. And what is perhaps even more worrying than that for the parents and the families of these people with Dravet syndrome is they actually can have something called sudden death associated with epilepsy, where oftentimes you’ll hear accounts of someone who has passed in the night because of their epilepsy. So that gives you a sense of how traumatic living with Dravet is. Lennox-Gastaut, on the other hand, is a little bit more heterogeneous. Like I mentioned earlier, there’s more seizure types that someone living with LGS may experience. And I would say the characteristics seizure type that you hear the most often about is major motor drop seizures. And you can identify people who have these major motor drop seizures because oftentimes they are wearing a helmet to protect their head. So, if you see a child in an ER or a pediatrician’s office, they do that because the characteristic seizure type is actually an atonic seizure where your muscles lose tone and you essentially fall to the ground. So it’s very dramatic and not only is the patient at risk of potentially cracking their skull, but of also other broken bones. So that’s the primary seizure type that these people living with Lennox-Gastaut have, though there are many, and you can just imagine how terrifying this is to be a loved one or a family member with someone in your family who experiences these seizures. It’s like a crisis almost every day. And Lennox-Gastaut can have seizure rates sometimes in the hundreds per month. Some people, as I mentioned earlier, even have a thousand plus in a month. So very, very refractory conditions.

Daniel Levine: What is it like to be in a household where these episodes occur? Do people require any specialized training? Do they have to be prepared to make a decision about whether to go to a hospital? What happens in a house?

Meg Alexander: So, the short answer is yes. First of all, I have to say for caregivers and people living with Lennox-Gastaut and Dravet, I can only begin to say how much I admire what they are able to live with, cope with, and thrive with on a day-to-day basis. So, I’ve been very fortunate to work with these communities for now seven or eight years of my life, and I’ve been greatly privileged to launch one and what will hopefully be two medicines for these indications. But to give a sense of what it’s like to live in the households of these families, many of the people I know sleep next to their children all night long because they’re so worried about that sudden death, SUDEP. So their quality of life, their quality of rest is very, very diminished. These are parents, moms and dads, who are worried about trying to go to the grocery store with their child because what if their child has an attack and seizures that they can’t get under control? I mean, these are parents who’ve had to calculate in the past, I can’t go to the grocery store. I need to be thinking about giving my child a rectal suppository if he or she can’t stop seizing. So their lives are very, very challenging. And there’s fear in several of these indications about status epilepticus, right? If I can’t stop the seizure fast enough, my child or my loved one is going to be in a state of a medical emergency, in which case I’m calling the ambulance, I’m going into the ER, and I’m worried about trying to stop that seizure. So it is a crisis. It is terrifying, and it’s truly exhausting for both the people living with these conditions and their loved ones who care for them.

Daniel Levine: There are a number of anti-seizure medications available today. How effective are they on these rare epilepsies?

Meg Alexander: Sadly, they’re not effective enough. So I’ve been, as I said, privileged to work with this community and I’ve been extremely gratified to be part of the teams that have brought new medicines to these patients. But all of these families, these communities, need a true step change. And Danny, the reason why is that roughly 80 to 90 percent of the people living with Lennox-Gastaut and Dravet syndrome still continue to experience breakthrough seizures despite taking not just one, but oftentimes multiple medicines. These are not a simple situation of someone taking a statin every day for their health. These are children and adults who are oftentimes on four, even five anti-seizure medicines at once, and they still continue to experience breakthrough seizures. So the current medicines that we have today are better than what we had 20 years ago, but they’re not working. And even the ones that are working, unfortunately, are not what I would call friendly medicines. They have significant side effects like somnolence. Some of them decrease appetite, which is really hard, and almost all of them come with a boatload of different tests and monitoring. And that’s just not easy. It’s one thing for many of us to walk in and get a blood test, but for people living with these indications where they might also have developmental challenges, mobility challenges, it’s really rigorous. So the short answer is the medicines we have today are not effective enough and we need really a new generation of medicines and a step change in medicines for these communities.

Daniel Levine: One of the other concerns about these conditions is that seizures can cause progressive damage to the brain over time. What’s known about the progression of these conditions and the outcomes for people with them?

Meg Alexander: So, I’d like to say a lot is known, but we’re really still learning. The two indications are a bit different. So in Dravet syndrome, you’ll experience parents and caregivers telling you that they started to see symptoms oftentimes as early as toddlerhood. As I mentioned, it’s a genetic epilepsy. It’s a genetic developmental epileptic encephalopathy. And what you start to see in addition to the seizures is the children start to lag in their developmental markers relative to their peers and as they age over time, this can lead to, unfortunately, many challenges and many comorbidities, cognitive disabilities, behavioral challenges—those are really hard for the families, mobility challenges, and over time, even gastrointestinal issues. So that’s Dravet. Lennox-Gastaut is equally heartbreaking because in the case of Lennox-Gastaut, you may often see a child with LGS start to progress with typical development, but then as the seizures start to take hold, you start to see developmental setbacks that can lead to lifelong disability. And again, this can lead to just extremely heartbreaking situations where you hear the parents describe that they had a child who was starting to walk and talk and their personalities starting to come to the fore and then they start to lose these abilities over time. And you even hear that with parents as they talk about how their child responds to medicines and how responses to medicine wane over time. And you hear them talk about my child was speaking and we were even starting to do some school, and then she reversed. So this is very, very hard. And as I said, they’re lifelong indications.

Daniel Levine: Ovid is developing a pipeline of therapies that restore homeostasis to the neurons. Can you explain what happens in the brain during a seizure?

Meg Alexander: Yes. So essentially when you’re having a seizure, what’s happening inside the neurons in your brain is that you’re having too much excitation. And that can happen within the neuron itself and the broader neuronal network. And essentially what that’s leading to is involuntary changes in your body movement and your function and your overall awareness and your sensations and behaviors. So this can be very alarming to people who don’t know what they may be seeing. And some types of seizures, like the ones I mentioned to you, are very overt when they happen. For example, a major motor drop seizure like we discussed, it’s literally when your muscles lose tone and you fall to the ground, or in a convulsive seizure where you might see someone rock and convulse involuntarily. But other types of seizures are much more subtle and can be hard to diagnose. You might see someone’s eyes gloss over and they may be having an absence seizure where they don’t seem to be engaging or reacting with you as you might think. So it’s actually an interesting thing. Not only have we learned a lot about what causes seizures over time, but over the course of the last 10 years, the field has really helped us catch up in how we identify, define, and diagnose different seizure types and what that may mean relative to the underlying condition and how to treat it.

Daniel Levine: My sense is that anti-seizure medications generally seek to alter the electrical activity in the brain or inhibit it. How might Ovid’s therapeutic approach differ from what others are trying to do?

Meg Alexander: Thank you for asking that, Danny. So we have spent much of our careers and our lives trying to figure out how to conquer seizures, and we’re not alone. Many other dedicated scientists and physicians and parents and caregivers have tried to do this as well. When we looked out at the space, we had learned a lot from science and the underlying mechanistic rationale for different biological targets in seizures. So that exploratory was extremely important. But what we endeavored to do was to look at novel mechanisms of action and largely novel targets to be able to treat differently. And what we were endeavoring to do, and I can walk you through this, is we tried to look at what are the different causes of seizures? Was it extrinsic factors like neurotransmitters, like glutamate and GABA, which are the primary essential vehicles by which you get too much excitation or too little inhibition in the brain? We looked at intrinsic factors within a neuron. So was there an underlying chemical imbalance in a neuron that was making it excited? And then there’s other things that we went out to look at—are there anatomical reasons? For example, are there lesions or malformations occurring in a brain that are leading neurons to be hyper excited? So, we went out and we really scoured the universe, and what was important to us at Ovid is we want more and we want better for these families and for these people. So when we did our research, it was important for us as we started to build new mechanisms of action into our pipeline is that we weren’t going where the rest of the field was going to have a step change. We believe we need to be looking at new targets, new mechanisms, and it was important to us to not be the fifth or sixth next best in an existing category because we feel that it really is going to require new mechanisms and more precise underlying targeting of the biology that drives the pathophysiology in order for us to make an impact.

Daniel Levine: What’s the case for seeking to restore homeostasis and pursuing targets linked to the cause of hyperexcitability of neurons? And how well understood or unique are these targets?

Meg Alexander: So, the field has really advanced dramatically over the course of the last 20 to 30 years. Thankfully, we’ve got a lot more work to do, but before, if you think about the first couple generations of anti-seizure medicines that are many of the names that you know and hear about, for example, when you’re watching medical shows, a lot of the discoveries there, Danny, were pretty serendipitous. They didn’t understand how the mechanism of action was directly modulating the underlying biological target that impacted disease. So that’s been a huge step change. In fact, the way that we look at neurology, we’ve learned a lot, quite frankly, from oncology and more precision oncology. So, for us, when you ask how unique are our targets as we have built our pipeline over the course of the last several years, we’ve gone out and we’ve scoured the universe looking for mechanisms of action that we think will operate on novel targets or operate on a target in a novel way. So, you’re asking how unique are they? They’re very unique. So, we’ll tell you a little bit about our lead program in a few minutes, soticlestat, but that’s acting on basically a cholesterol enzyme in the brain that no one has done before. Our secondary pipeline program is operating on an anatomical cause of seizures that also has not been done before. We have a program that’s called GABA-aminotransferase that’s operating on a known cause of seizures, but in a different way than what’s been done before. And then we have a very exciting program that’s early in our pipeline, but I think it’s going to be hugely important in a number of different neurological conditions. We think it’s going to have many therapeutic applications, and that’s also a completely novel target, but people have known about it for a long time. No one’s been able to drug it effectively because it’s been such a hard target to crack. So this is key to our strategy and we think it’s going to be the right thing for patients, Danny, because we want to be able to deliver a significant change and benefit than what we’ve seen before. But it also makes sense from a business perspective because after a while being the fourth or fifth medicine in a certain class might not be as differentiating and might not deliver the change that these communities deserve.

Daniel Levine: If these are such novel targets, though, how well validated are they?

Meg Alexander: That’s a good question. So I think the short answer is the nice thing about seizures and epilepsies is that the type of models and experiments that we can run in a lab and with animals tend to be much more clinically translatable to humans than other neurological indications. And I hope other conditions will get there. But the nice thing is animal models actually tell us an awful lot about how effective an anti-seizure medicine will be in humans. They’re not perfect, but they’re pretty good. So what we’ve done across our pipeline is we have a very standardized development approach where before we ever come close to getting to a human, we have run a battery of essentially seizure disease models in our compounds so that we know what types of seizures they work on, whether they work on refractory versus more chronic recurrent seizures. So, we have all of that and we know that well before we ever consider putting our medicine, potential medicine in a human. But in terms of their validation—our lead program, which is soticlestat, which we co-developed for many years with Takeda, has been validated in humans. We actually have one of the most rigorous clinical development programs ever done in Lennox-Gastaut and Dravet syndrome. But our other programs have good validation as well. We know for our ROCK2 inhibition program, which is for a condition called cerebral cavernous malformations, of which about half the people have seizures, we’ve seen very well that the data shows that we can ameliorate the underlying cause of disease in animals and actually modify the course of the disease in animals. So we feel pretty confident there. And then some of our other compounds like our GABA-aminotransferase programs, OV329, that’s actually a known and validated mechanism. It works very well. We know it does a terrific job at crushing the seizures. The problem with older classes of medicines, prior medicines that came before, had side effect profiles that were just simply too risky. They were intolerable. They made one in three people who took them blind actually. So we believe we’ve been able to essentially dial that out with a different compound and a different approach with some subtleties in our mechanism of action. So we know it works to reduce seizures, we just needed to make a safer and more effective medicine.

Daniel Levine: You mentioned soticlestat, your lead experimental therapy. What is it and how does it work?

Meg Alexander: Soticlestat, we believe and hope, will be an entirely new class of medicines for people with Lennox-Gastaut and with Dravet. And it’s very unusual. It is the only medicine of its kind. It’s an inhibitor of cholesterol 24 hydroxylase. What many of us may not know, I actually didn’t know this for most of my career, is that we store a fair amount of cholesterol actually in our brains. And what Octa essentially works by doing is it inhibits a chemical modification that occurs in this cholesterol in the brain that can lead to basically an increase in the neurotransmitter called glutamate. So that’s the neurotransmitter that creates too much excitation and it can also create a high degree of inflammation. So what CCOs stat does is it inhibits cholesterol 24-hydroxylase, and by doing that, it helps reduce the amount of the excitatory neurotransmitter glutamate, and it also reduces inflammation, which sounds very technical and scientific, but what it really means is that it gets into people’s brains very well. And in a matter of a relatively short period of time period of time, a couple weeks, we see a dramatic reduction in seizures and we see a durable response in seizure reduction over time. So, we know it’s working, and that’s of course why it matters.

Daniel Levine: What’s known about it from studies that have been conducted to date.

Meg Alexander: So, we know a lot, as I mentioned, from going from a field where serendipity was how anti-seizure medicines used to be discovered to today. With our partner Takeda, we have run the most extensive clinical development program that was ever done in Dravet syndrome and Lennox-Gastaut syndrome. And we know a lot about how soticlestat behaves as a result of that. So now we have hundreds of people who have been on soticlestat for years, and there’s some important findings that have come out of that in terms of how it works. We know that it is very effective at reducing a whole range of seizure types, including the primary seizure types that are experienced most by people with Dravet syndrome and Lennox-Gastaut syndrome. In Dravet syndrome, our phase 2 program showed that it reduced 46 percent of seizures, placebo adjusted. Those are convulsive seizures. And in Lennox-Gastaut, we saw that it reduced motor seizures by about 15 percent. And we believe with some modifications that we made in the phase 3, it will be more. But I think the big takeaway—that’s the kind of raw data. The raw data is not going to be the thing that matters for these families, that what’s going to matter more for these families is the total profile of how a medicine may behave for their loved one. And what we know of soticlestat is that it’s very effective at reducing seizures, the primary seizure types for these indications. But many more than that. As you heard me say earlier, people with Lennox-Gastaut have many different types of seizures. It’s extremely safe, unlike most other anti-seizure medicines. So, in our phase 2 program, we saw that it almost barely separated from placebo. So it’s a very friendly potential medicine to take in that regard. And then finally, it can work well with other medicines, and it shouldn’t have to require the level of intensive monitoring that current anti-seizure medicines do today. That means that you don’t need to be worried about taking the other three or four medicines you’re on and taking soticlestat should be really easy to add to it, whether you add it first or you add it at the end, and you don’t need to be worried about it suppressing your appetite or putting your loved one to sleep or needing to go get very challenging types of monitoring tests like echocardiograms and blood draws. And again, that might seem minor for people like you and me, Danny, but I’ll never forget, probably it was now, I guess about four or five years ago, and I was getting ready to launch one of the first anti-seizure medicines for these indications in many years. And I was really excited about it, Danny, because I thought the seizure reduction rates were miraculous for these families. The first approval came in a condition—that was Dravet, and I was so excited for the families. And I remember talking to one of the world’s leading physicians in the space at the time. He told me he was having a hard time getting the parents to try the new medicine. And that was so mind boggling to me. I couldn’t understand why. And what he explained to me, the physician is a man called Joe Sullivan. And what he explained was that these families are living such a stressful, challenged lifestyle that any form of control, if you go from having 200 seizures in a month to a hundred seizures in a month is better balanced than what they previously had. And that makes them very averse to trying new things. And it makes it very hard when you think about how do I take my child in to get something like an echocardiogram or my child already is a tough eater and now you’re going to give me a medicine that suppresses her appetite. These are very, very, very real and hard struggles for these families, and they have to deal with so much chaos that any level of stability makes them very wary to rock the boat. So, when you bring that back to the medicine that I’m very excited and very hopeful that we might bring to patients sometime soon through Takeda, is that soticlestat doesn’t have those challenges. It’s easy for these families and it should afford a significant seizure reduction benefit to the patients. So, I’m really hopeful that this will meet the needs of a community who deserves better, friendlier medicines

Daniel Levine: And what’s the development path forward?

Meg Alexander: it’s looking good, and it’s soon. So, we out-icensed soticlestat to Takeda, and Takeda is now finishing two pivotal phase 3e trials, both in Lennox-Gastaut and Dravet. They’ve said that that’ll be done in the first half of their fiscal year, this year, 2024. So that means we’ve got top line readouts coming soon, which is going to be great news, I believe, and hope for these families. And from there, our partner will be preparing for global registrations. What’s really nice about having a talented partner, like what we’ve had with Takeda, is that they’re a global company and they’re really effective at launching medicines on a global scale, which these communities haven’t had before. In the past, it’s usually been one company launching per region. So that means it’s slow in terms of access of getting effective medicines to people all over the world. So I’m very encouraged by this and we hope to be hearing more about the data soon.

Daniel Levine: The deal with Takeda dates back to 2021. It brought in an upfront payment of $196 million and up to $660 million in potential milestones as well as royalties. What has that agreement meant to Ovid, particularly in an environment like we’ve had these past couple of years?

Meg Alexander: You mean the biotech winter, Danny, that’s what I refer to it as. So listen, I’ll tell you about the financial reasons why it matters, but that’s probably only a small part of it. Of course, I joke about the biotech winter. It hasn’t been funny. It hasn’t been funny for the hundreds of companies that have had a hard time in the capital markets. So yes, of course having capital in an upfront was helpful. And it was honestly the most helpful for us because we learned so much through our development of soticlestat that when we had the right capital in hand, we knew the types of mechanisms and other compounds that we wanted to go out and bring into our pipeline that we felt we could develop better as a result of all the investment and all the time and energy that we put into developing soticlestat. So yes, the capital was helpful from that perspective, but I think what soticlestat has meant more is it really was the playbook that we crafted that we’ve subsequently refined to develop anti-seizure medicines effectively. And that, as you’ll see, is now starting to come to bear with many more milestones that are coming out of our pipeline with new programs well beyond soticlestat. But then finally, this is really about the patient communities. They helped us develop this medicine. We believe this should be a very effective medicine for these communities, and we learned a lot from them, Danny. I mean, through our partnerships with them, we managed to recruit the phase 2 program six months faster than what anybody predicted and had scheduled under budget. And that was one of the instances where we really married ourselves to the patient communities and the caregiver communities, and we learned so much from them. And that has been typical of our playbook ever since. So yes, Ovid benefited financially from the development of the program, but it really has been the learnings from it that coined our playbook, and this deep fidelity and commitment that we have with the patient communities that has served us not just to tickly step, but frankly well beyond with the other medicines in our pipeline.

Daniel Levine: And is the expectation that you would seek additional partnerships like this for the other candidates in your pipeline, or would you start to build a commercial team?

Meg Alexander: So yes and yes is the short answer to that. So collaborating is always a good option when you can bring two different areas of expertise together, two potential synergies together. We had that with Takeda in the case of soticlestat. The compound was actually developed in their labs by a gentleman named Toshiya Nishi who came to work with us subsequently thereafter. We had a lot of expertise in how to develop in seizures and in epilepsies, and they brought a lot of the strength and resources and commitment that a large pharmaceutical company can bring. So as we look forward to our future, we’ve got three programs in our pipeline, all of which have novel mechanisms of action, and many of them have multiple areas where we could take them from a development perspective, so they have multiple therapeutic applications. That’s super exciting. So, the way that we look at this is where can we play with strength for ourselves and where would the science and the medicine be benefited or run faster with a partner? In our case, we have our core competencies in seizures and epilepsies. We have a strong U.S. domestic presence. Those would be areas that we would look to commercialize in. With that said, some of the earliest programs in our pipeline have really exciting applications in areas like psychosis, for example, and psychosis and schizophrenia and Parkinson’s. Also areas with just incredible unmet need and medicines that are less friendly, shall we say. So those are obvious areas where we would be looking to work with partners to complement some of the capabilities that we might not have as robustly, but a larger company whose played in those therapeutic areas may.

Daniel Levine: You mentioned that there’s been learnings from developing the drug that you’re now applying to earlier candidates in your pipeline, but I’m wondering if you can expand on that and talk a little about the specifics, some of the things you’ve learned that you’re actually applying.

Meg Alexander: One of the areas that makes epilepsy very appealing from a medical and clinical development perspective is we can take a lot of steps in early development to make sure that the compound and that the mechanism of action that we think we have is really working. And what was a fantastic learning experience for our team, as I said, we really built our playbook around soticlestat with Takeda. So starting very early on, we looked at the mechanism of action. There were some who said this should go into a much broader therapeutic area, we should try it on Alzheimer’s. That’s what the large pharmaceutical companies oftentimes are looking at because of the unmet need in the population size. But we did the work to look at the way the mechanism of action was working, and we looked carefully at the animal models. And actually, Danny, there was an insight in one of the Alzheimer’s animal models where the patients at the end of their life and journey start to seize. And we had this insight where we were looking closely at the findings and we saw the animals weren’t succumbing at the end of this disease model. They were in fact living, and it was because they weren’t seizing. So that insight and then many more thereafter, has helped us build our playbook. So, starting with a clear link of the mechanism of action to the underlying biology, pathophysiology of the disease, using the best clinically translatable animal models to develop. Looking at the underlying genetic factors and seizure types to make sure that we’re marrying the way the mechanism works with the types of seizures that different patients have. And then working hand in hand with the patient communities to design our trials and to help us enroll them. I mean, that’s been so fundamental. Companies make so many mistakes. Just even looking at a formulation. People have a hard time swallowing certain formulations—just not going to work. Understanding the protocols in a trial and how hard it is for a caregiver to get their child into a hospital-like setting for certain tests. These are all very real considerations that are so important. And we’ve built a whole playbook around this with soticlestat that we’ve subsequently started using for our GABA-aminotransferase program and our ROCK2 program. And it’s important. It’s important not just to de-risking the program, but it’s important to the kind of company that we want to be working in partnership, whether that’s with the community or a development partner. So soticlestat gave us all of this and so much more. And what’s exciting is this is not a static field. It’s incredibly dynamic. So we’re learning more and more every year working with very committed physicians and academic opinion leading experts about how to better measure seizure types. And that is only making trials more effective over time, and it’s helping us learn more over time. So I’m very, very, very encouraged and we are grateful for having that experience, and we hope it’s going to make us much more successful for these families as we look to take our novel mechanisms into the clinic.

Daniel Levine: Meg Alexander, chief strategy officer for Ovid Therapeutics. Meg, thanks so much for your time today.

Meg Alexander: Thank you, Danny. Nice to talk.

This transcript has been edited for clarity and readability.

 

The RARECast podcast is made possible through support from the Global Genes’ Corporate Alliance. The members of the Corporate Alliance support Global Genes’ mission and programs, work to meet the vital needs of people with rare diseases, and address inequities they face. To learn more about the Corporate Alliance or how your organization can become a member, click here.

 

 

 

Stay Connected

Sign up for updates straight to your inbox.

FacebookTwitterInstagramYoutube