Replay
Transcript of Call:
Participants
- Mike Kelly, NervGen CEO
- Dan Mikol, NervGen CMO
- Rich Macary, KOL
- Deniel Mero, PropThink Editor
PropThink
Good morning everyone, welcome to PropThink’s Digital Conference. My name is Deniel Mero, Editor of PropThink. I will be hosting today’s event. PropThink Digital Conferences is a platform designed to give you access to subject-matter experts, including prominent investors, executives, KOLs, and others with unique insights into the healthcare space.
Before we jump into the discussion, just as a reminder for the audience that there will be forward looking statements made, which are based on assumptions. We are not registered investment advisors so nothing in this presentation should be construed as investment advice. Always consult a professional before making any decisions.
Alright, with that said, let’s get into the agenda and speakers today. We’ve invited 3 renowned experts in neurology and spinal cord injury to discuss the current landscape and ongoing trends in the space.
The experts include Mike Kelly, who is President & CEO of NervGen. He previously served as President of US Operations for Adapt Pharma, which commercialized NARCAN Nasal Spray used for opioid overdoses and was eventually sold to Emergent BioSolutions for $735M. Mike has also held executive roles with Covis Pharma, which had a $1.2B sale and Azur Pharma that merged with Jazz Pharmaceuticals.
Also on the call today is Dan Mikol, the Chief Medical officer of NervGen. Dan previously served as Amgen’s Head of clinical development in neuroscience and nephrology. He’s also held senior development positions at big pharmas like Biogen, Novartis and EMD Serono.
And the third expert on the call today is Rich Macary, a biotech investor, executive and consultant that has worked with numerous companies in neurology, most notably Sarepta.
We will open the call with a roundtable discussion and close with Q&A, where the audience can participate. So, with that, let’s get to it.
Gentlemen, welcome. I’m looking forward to this discussion. Mike, let’s start with you. So, you have a lot of commercial experience most recently at Adapt Pharma launching the Narcan nasal spray. You recently jumped into a clinical stage biotech when joining NervGen, which comes with career risk, and reputational risk. What about the data, story and people drove you to make such a jump?
Mike Kelly, NervGen CEO
Thank you. I was approached about five months ago by leading recruiting firm and encouraged to take alook at NervGen. At the time, I saw a lot of similarities with what you talked about for ADAPT and Narcan. There was a great product, a strong team and there was a significant unmet need. That unmet need was very unappreciated by the world.
I think that the world thought the unmet need was, “this is going to be roughly a $100 million product”, that ultimately went on to be a $400 million product. So that was kind of the backdrop when I first started to look at NervGen.
The first thing I did was I dug into the product candidate, which is NVG-291. We'll talk about that a lot today. I spoke to a lot of KOLs in the area, key opinion leaders.
I think probably one of the biggest turning points for me is when I spoke to Jerry Silver, who was the discoverer of the technology. He walked me through and explained the difference between this product and why he felt that this was something that was going to be transformational in the world of treating CNS disorders. I didn't just take his word for it. I did my own research and dug and talked to several other key opinion leaders who collaborated. Dug into the studies myself. And there was some commonalities here.
So the product has the ability to treat many different CNS disorders. We're starting with spinal cord injury. Because of the mechanism, which we'll talk about today, it has the ability to allow the body to repair itself, which is the differentiator. It's not growing cells. It's not trying to grow nerves. It's not working on the immune response. It's actually working on allowing the body to actually regenerate or remodel. I will talk a little bit about that today.
The product itself is transformational. The unmet need of spinal cord injury is significant. A lot of market research on that area. We have both acute and chronic that we're going after. Both of those markets are billion dollar plus markets. It’s a great start and it's a small commercial footprint.
I felt that with my background and my ability to see the pathway for this product to the ultimate commercialization starting with spine, that I could really add some value here. That's what led me to meeting the team. My last step was meeting Dan and the rest of the management team. I realized there's a strong group, management here that we can build off of. Within 12 months, we could be really changing the way that the world looks at spinal cord injury.
So, I think it's the product, the people and the opportunity. think that the linchpin for me was how quickly that this is all going to happen. I think within the next 12 months, we're going to be in a totally different place as a company. That's the reason I jumped ship and took this opportunity.
PropThink
Well, I can tell you, I think a lot of the nerve gen stakeholders are excited to have you on board.Rich a question for you. So many people are familiar with Sarepta today, but not many know of its predecessor AVI Biopharma, in which you were an early investor, activist shareholder, and helped bring in new management to what transitioned to become Sarepta. Then you also spent several years as the VP of business development for Sarepta. Can you speak on the parallels you see between NervGen and the early stages of Sorepta?
Rich Macary
Oh, absolutely. I think the first area to look at is it was a very similar market cap. Small company in the pacific northwest, sub-100 million, of course, eventually became a multi-billion dollar company. But the parallels are many. I'm just going to pick out a few that I think are important for investors, that are important to me. I came here based on, like Mike and Dan, the science and the data.
The first one was when I got involved with AVI, RNA therapeutics was a new thing. This idea that you could program a drug and it would go through the body and find a sequence and you could ameliorate a rare disease or you could stop a virus from replicating. I mean, it seemed like science fiction when I was talking to investors. In reality, if you were looking at it and understanding it, you saw the pathway forward to work. And of course, you could have taken all the companies and added them up to a couple billion back then. Today, it's hundreds of billions. So, I see the same thing here.
This is a new class of therapeutics. It's the first thing that's gotten me and the pharmaceutical world as excited as I was when I first saw and understood what RNA-based therapeutics could be. Up until the 90s, it wasn't even believed that you could repair nervous system damage. So the idea that NervGen has this, what could be considered a drug class, a platform starting with SCI, the idea of nervous system repair is exciting.
I don't think it's something that people are looking at and expecting to happen. I think when it does, the prospect of it, it's going to get investors very, very excited. It's transformative. It's the only company really doing it. At least back then, AVI had other companies like Alnylam and Ionis sort of taking this along because of where this research came from.
Second area that I would say is a great parallel is the indication itself. I mean, what made AVI/Sarepta was a pivot away from chasing viruses and bio tear agents to going after rare disease, focusing on Duchenne muscular dystrophy. A lot of the investors said, “What the hell are you doing? What are you going after the small little indication for? Go after millions of patients.”
What they didn't understand, which is what the incoming CEO understood, was rare disease had its own model. This wasn't going to be a $25,000 or $50,000 drug. It was going to be a quarter of a million or a half a million dollar drug. So 20,000 patients meant a multi-billion dollar market. So DMD had no therapies. Nobody was clamoring. There was a handful of companies looking to develop in that space. And so DMD became the reason that Sarepta exists.
SCI is that, I believe, obviously for NervGen. There's unmet need. Almost everybody that's tried to do it, it's in the rear-view mirror, whether that was a Acorda, Vertex, Pfizer. There's very few that are moving forward with promising candidates. And somebody crosses the line here. This is incredibly valuable. And I don't think that's understood. Wall streets are not breaking out their calculator or investors trying to understand this because why should they? Nobody's come close to having a drug that's crossed the line. But when they do, they're going to see, this is 10, 15 times larger than what Sarepta had in DMD.
The last parallel that I think is super important, I'll end there, is the change from AVI to Sarepta was because of a new CEO. We brought in Chris Garabedian, who came out of Gilead and came out of Celgene with a lot of experience. He came in, saw the opportunity in DMD, focused the limited resources of the company on that, and drove it forward. And that was really the big, big change and the important thing. For me, Mike Kelly coming in here with his experience, doing exactly the same thing, driving the focus, prioritizing the pipeline, the R&D, grabbing onto a great team that's already here. It feels like Sarepta all over again, except in my opinion, better, bigger in terms of market opportunity, and I think it's going to happen a lot faster.
PropThink
That is exciting. Mike, the spinal cord injury, like Rich was mentioning, is the first indication for NervGen, where surgery and rehab are the current standard of care. Can you talk about this field and how big this is as a market commercially?
Mike Kelly, NervGen CEO
Sure. So we're looking at this market, there's really two populations, what we're calling the sub-acute population and the chronic population. The sub-acute population is folks, and Dan will talk about this when we talk about the clinical trial that we're doing. But those are folks that have had a spinal cord injury in the past year. Then there's the chronic population of people who've been living with a spinal cord injury for many years. So there's two groups.
There's 18,000 new cases a year. There's a pool of 300,000 people living with spinal cord injury in the United States annually. When we did market research and we talked to patients, then we also talked to payers and we also talked to physicians, we found that their modest improvements in function would be a huge step forward for patients.
If patients could get more functional recovery, if they can get bladder improvement, if they can move their arms or move their legs a little bit more than they currently are, it would be significant improvements. And the goal for them is to live independently. So that's really our goal is to provide a product that could give functional recovery or functional repair to allow folks to get some independence again.
So this is an affliction that happens immediately. The average age is roughly in the young 40s, 70% are men and they happen to either in a car accident or a fall boom and their life has changed forever. The size of this market, if you look at the cost, so what we try to do is you look at the incidence numbers, then you look at the cost of the system.
The spinal cord injury costs over $50 billion in the U.S. market health care system. Whenever you have a small group, like 300,000 driving $50 billion worth of cost, it's a massive opportunity if you even show a small improvement in that cost. When we spoke to payers, they would put this in the category of an orphan type of drug. If you take a small fraction of this patient population, multiply it by an orphan type of drug reimbursement that we would expect to get, that's why I said early on that the acute market is well over $1 billion and the chronic market is much greater than $1 billion. So that's why we focused commercially in spinal cord injury first. That combined with the data. The clinical data models, we have robust preclinical data package in spinal cord injury.
So those several things together really drove us to say, okay, this is the first indication. That's really going to unlock the value for us to go after multiple indications.
PropThink
Let's touch on the landscape for a bit in spinal cord injury. There have been several innovations that have received hype and praise, but they've ultimately fallen short of expectations. Can you share insights on why these innovations might have had difficulty with adoption or just showing clinical efficacy?
Mike Kelly, NervGen CEO
There's many different things trying to attack this market. Many different ways, right? So, you have the drugs, you have the devices. Let's separate the two. So, the device world is looking at stimulation. Stimulation to actually help the body with plasticity and regeneration and potentially growing new connections. It's definitely shown some modest improvement. The challenge is that it's modest improvement and it's a device. So, devices have their own luggage when it comes to getting paid for, and when you have a modest improvement and tough reimbursement, your uptake is not going to be there.
As far as the drugs, many of the earlier drugs focused on nerve growth, you know, growth factor types or modulating the recovery for the CNS. It's a tough path to go down when you have such a wide range of patients and various different functional recoveries that you're measuring. When you take all of the different types of endpoints you can pick in this patient population that's not ubiquitous at all, and then you apply those types of mechanisms, it's going to be a tough road to hoe.
I think the mechanism that we're working on, which allows the body to enable and repair, and the unique design that Dan put together in the clinical trial, which he'll walk through, allows us to get a homogeneous population and focus on allowing the body to repair itself. So those things I think are going to separate and differentiate our pathway as compared to some of the others that have tried this before.
PropThink
Dan, let me get your insights on NervGen’s NVG-291 and how it's different from the disease modifying therapies that are approved or that are being developed for neurodegenerative conditions.
Dan Mikol, NervGen CMO
Sure. Well, most drugs in the space targeting, for example, Alzheimer's disease, ALS, sclerosis, these drugs aim to slow progression of the disease. In other words, to slow further damage and further loss of functions, but patients are still expected to deteriorate just at a slower rate. They were called disease modifying therapies.
NervGen’s approach is entirely different. It's about neuro repair. The objective is to repair nervous system damage and improve function. I'll talk about this a little bit more as we move forward, but NVG-291 acts through general repair mechanisms. It doesn't act by modifying the biology of any particular disease.
PropThink
Understood. Rich, you're part of a few different neurology projects from Imeka's AI based neural imaging platform to Synaptica’s neurostimulation approach to treating Alzheimer's. What have you learned about the brain and the nervous system that gets you excited for NervGen’s approach?
Rich Macary
I've learned a ton and a lot of that was focused on neurodegenerative and being involved with NervGen is educating me on the trauma side when we're talking about spinal cord, traumatic brain injury, or stroke.
I think the commonality, especially what I'm doing with Synaptica in stimulation, is understanding the importance of plasticity in both sides of this, neurodegenerative or in traumatic injury. That loss of plasticity or the ability to generate plasticity, strength and connections, making new connections, it's of vital importance. There are a lot of other ways to do that right now, pharmaceuticals.
For me, the thing that gets me most excited is that core aspect of what this technology does, which Dan will speak to, is really where I believe is a game change because it does so turn to all these other repair mechanisms that are really exciting about what is happening in SCI and other clinical problems.
PropThink
Dan, can you talk a bit about the repair mechanisms and what have been shown with NVG-291 in animal studies?
Dan Mikol – NervGen CMO
Yeah, before I talk about the actual repair mechanisms, let me walk through what happens as a result of injury to the nervous system. So, after injury, let's say to the spinal cord or the brain, there are three key biological effects and summarized on the screen.
First, there's a glial scar that forms around the side of an injury and the scar and the components around it inhibit regeneration of neurons, nerve cells. The neurons are shown in sort of a pinkish purple color.
Secondly, the connections between neurons are lost as a result of injury. And then third, the myelin coating of neurons can be damaged partially or completely. The myelin is shown in sort of a greenish color covering the long extensions of the neurons. It's the myelin that insulates the neurons, the long processes of the neurons called axons. And this insulation allows the neurons to communicate, to transmit electrical impulses very swiftly.
Now, NVG-291 has been shown in preclinical studies, to enhance repair. Tt's been shown to enhance repair by each of these three mechanisms I just mentioned that are adversely affected after injury. It's been shown to promote the regeneration of neurons through this growth inhibitory environment of the glial scar. It's been shown to rewire neurons that had lost connections through the process called plasticity, where neurons essentially establish new connections with other neurons to regain function.
And then thirdly, it's been shown to promote remyelination. That is the re-insulation of neurons in order to transmit the signals more efficiently.
PropThink
Mike, let me ask you, we've heard some of the benefits and advantages of NervGen’s approach. But what do you see as challenges in the next few years?
Mike Kelly, NervGen CEO
Well, I think in the next few years, I think our biggest challenge is a company is going to be to remain focused and execute. I am bullish on the company and think that once we have a data readout of this chronic cohort, there could be a little bit of chaos. We as a company need to remain focused. I'll draw back to my analogous situation with Narcan.
When we launched Narcan, we didn't know what to expect. The product took off and it was chaos. We were trying to grow the company as fast as the product was growing. I think the thing that remained, why we were so successful is we had a strong group of people who could execute on a plan and remain focused. I think that's going to be our biggest challenge next year as a company. Once we have the chronic data readout, there's many different ways we can go with this product. We're going to be tempted to do a lot of different things and be masters to many, but we have to remain focused and get this product on the market for patients or people who are suffering from a spinal cord injury. That's going to be the biggest challenge is to just remain focused and execute on a plan.
PropThink
I want to play this quick video just showing the impact of NVG-291 on motor function and nerve regeneration. I think it demonstrates the potential here on this mouse model, as you see, regains mobility and is able to walk again.
Dan, obviously the preclinical data here has been undeniably positive. What gives you confidence about translating this into humans with spinal cord injury?
Dan Mikol, NervGen CMO
Well, what gives me confidence, first of all, is that the preclinical data are undeniably positive, as you said. I mean, that's really important. If you don't have very strong preclinical data, you're on very shaky ground. In our case, we have undeniably positive data in animal models of acute spinal cord injury, which is shown on this slide, where treatment with the peptide begins very early after the injury within the first 24 hours. You can see this is measuring mobility, and you can see for the NVG-291 group of animals, they had significant improvement in mobility compared to the placebo group.
We also see significant improvements in models of chronic spinal cord injury. In this case, the treatment was begun 12 weeks, three months after the injury, when the animals had already achieved the extent to recovery they were going to achieve. This is a different outcome measure we're looking at here. It measures forearm function and fine digit function. But you can see there's separation from placebo. There's significant improvement in the animals who received peptide. And again, this is with chronic intervention.
We see similar results with NVG-291 and some other conditions associated with nervous system damage such as stroke. We're not going to have time to go into that. Most of our data comes from animal models of traumatic spinal cord injury.
So, another thing that gives me some confidence going into the clinical trial is that animal models of traumatic spinal cord injury should be very good models of traumatic injury in humans. Nothing has successfully crossed the line yet, but it should be a very good model. We have very strong data from multiple different investigators. It's not like this came out of one investigator who showed some positive results. It's been reproduced over and over or many times over.
That's why spinal cord injury is our lead indication. And the last thing I'll say is we designed the proof of concept trial, a lot of thought went into it. We designed it in such a way to optimize the probability of success in clinical translation. That is, taking these positive results from animal studies into humans. Hopefully we'll have positive results in humans as a result.
PropThink
Rich, let me get you to chime in here because you got involved with Sarepta when the company was at a similar stage to where NervGen is presently. How do you compare the quality of the preclinical data that NervGen has to what Sarepta had back then?
Rich Macary
Yeah, sure. Always being driven by the science and the data. And with AVI/Sarepta, that preclinical work was done in what they call the MDX model, which emulates Duchenne muscular dystrophy. But as many of these models, they're genetically bred models. They're developed to emulate the disease, they're not the exact disease. So, you always get this risk when you're dealing with not the exact disease in a rodent model, trying to translate that up to humans. Whether it's an EAE model and an MS or there's just a lot of these. And it brings risk into that translation.
I got excited about this because the magnitude of the result. What I got investors excited about in the MDX model, you know, this difference in the progression of the disease over time, you look at that acute model and you see this incredible recovery. I've never seen that in preclinical models of any disease that I've looked at, at that magnitude. That's why it's always talked about as being unprecedented.
The work that they have in stroke is unprecedented against other preclinical models. As Dan said, the trauma model, again, not a genetically modified rodent, it's a healthy rodent, sustains an injury. And then you're seeing this effect. An important note that I'll make in all research, especially in SCI, a lot of things that didn't move forward or fail, you'll see that they weren't replicated by other researchers. Somebody got a result, wow, it looks great. But either the work never moved forward or didn't work when they got into the clinic.
Those rat videos that you see, which really bring to life that change in the BBB scale, that's a replication study of Dr. Jerry Silver's work, where not only they replicated, they got a much better result because they used a much bigger dose. So my confidence level, because a lot of work with NervGen’s technology has been done with third party independent researchers in multiple different models, neurodegenerative and trauma, really creates a lot of confidence that this may translate and this has a great potential to translate to humans. It’s one of the reasons that I'm here and excited about it.
PropThink
Let's talk a little bit about this proof of concept study in humans. Dan, you touched on it a little bit before, but can you explain how this study is designed to optimize the probability of success?
Dan Mikol, NervGen CMO
Yeah, it has to do with careful clinical translation. I've used that term a little bit earlier. We have to apply what we've learned from animal studies to the design of our clinical trial to give us the best chance of successful translation.
We designed the trial to mirror what was successfully done in the animal models of spinal cord injury. For example, in the proof of concept trial, we will be enrolling participants who have some residual motor function. It's called motor incomplete. We're doing this because the animal models of spinal cord injury are motor incomplete models. The animals have some residual motor function, whether you're studying them shortly after the injury or after they've recovered, they have some residual motor function. We will enroll motor incomplete subjects.
We're also going to focus our assessments on motor function. This was done in the animal studies. A lot of, again, independent studies with NVG-291 have had positive results in animal models of spinal cord injury. All of these studies have focused on motor and locomotor function, and that's going to be the focus of our clinical trial.
Moreover, to provide even more detailed information about motor recovery in this trial, we're going to be incorporating electrophysiological measurements throughout the study, because this is the sensitive, objective, and quantitative way to monitor motor improvements, more sensitive than clinical outcome measures, and it's going to give us much more information.
PropThink
Can you talk a bit about what this electrophysiology that you're using in the trial? I don't know if it's commonly used in spinal cord injury trials, is it?
Dan Mikol, NervGen CMO
No, the incorporation of electrophysiological testing is quite innovative in this trial, and that's being recognized. It's just not being done in other trials of spinal cord injury. Let me first explain why we're doing it, and then I can answer the how part of it.
In someone without spinal cord injury, the brain and body communicate back and forth, and the spinal cord acts as sort of an electrical autobahn, if you will. The brain sends electrical signals through this spinal cord to specific muscle groups when you're trying to move an arm or leg, and when the body senses, let's say when your skin is touched, this sends signal the opposite way up to the brain. But after spinal cord injury, the electrical connections between the brain and the rest of the body are disrupted. The wiring has been cut, and it's this loss of electrical connectivity that results in loss of function.
Based on the animal results, our main hypothesis going into our proof-of-concept trial is that NVG291 will improve electrical connectivity. We've seen that in animals, and we hypothesize that this is going to result in improved function.
Electrophysiological testing is quite sensitive in monitoring motor recovery, and I should point out that a number of different studies have demonstrated that if you can show improvements in electrophysiological measures, this predicts improvements in clinical outcome measures of motor function.
Without going into all the details, in the clinical trial, we'll be measuring the strength of electrical signals and the speed of electrical signals that are passing through this spinal cord to 10 different muscle groups on both sides of the body, and we'll be doing this in every participant at regular intervals during the trial using a highly standardized approach.
You asked about other trials in spinal cord injury, and really in 99% of trials, electrophysiology is just simply not used. I think it's a missed opportunity, and maybe that's going to be changing in the future. Electrophysiology has been explored in some recent studies, but it's never been a primary objective in a proof-of-concept trial, as it is in our trial.
I should also mention that we have an outstanding group of clinical advisors. You can find them on our listed on our website, and during discussions about study design, they agreed that incorporating electrophysiology in a proof-of-concept trial is a very smart approach that will tell us a lot about how our drug works, and will also increase the probability of observing efficacy in this trial.
PropThink
Given the design of the trial and what Dan spoke about, Mike, do you think you can get expedited approval for 291?
Mike Kelly, NervGen CEO
I do. I think the FDA has laid out many different pathways for products like this, and there's two main criteria. The number one criteria is an unmet need in the marketplace. If this is a debilitating disease, and I think we check that box pretty simply with spinal cord injury. Then the other is what other products are out there to treat these patients? Is there anything that they're currently using, or are we layering on top of another product? And the answer is no. So, there is nothing for these folks.
Those are the two common themes for fast-track, for breakthrough therapy, for accelerated review, for prior review. The common two themes amongst the four different pathways that the FDA is giving companies like ours the ability to go in and have discussions with them about it. So, we will be applying for fast-track. Once we get clinical data, we'll be applying for breakthrough. We're going to be applying for everything that we possibly can to see if we can get this product to market as quickly as possible.
The goal here is, even though there's small numbers in this study, if we can align what Dan laid out here in the primary endpoints and the secondary endpoints, and we have statistical significance in both categories, and we can predict that electrophysiological measures were positive and functional improvements were positive, I think that gives us a pretty strong approach to go in and have a discussion with the FDA about how much more do we need to do.
Once we have a good idea and sit down with data, I think that's when we'll be able to give the investment community a clearer path to the speed of which we're going to get approved. But I'm pretty confident here that if we get positive in the primary and the secondaries here, it's going to give us a pretty good approach to be able to get this product to prove faster than typical traditional means.
PropThink
Staying on this on the speed topic. Dan, this is a single center study. Two questions, why is that? And wouldn't it be faster to conduct the trial if you went to more than one site?
Dan Mikol, NervGen CMO
Yeah, for every clinical trial, quality of data and speed of the trial are very important considerations. We'd always like to have the best quality data fast as we can get it. So, for speed of recruitment, yeah, trial could be faster with more sites, but the sacrifice in this case would be data quality.
A lot of thought went into this. This is the first single center study I've done, but we have a very strong rationale for why we're doing it. The main objective of the trial is to demonstrate improved connectivity with NVG-291. By using a single site that's highly experienced in electrophysiology, this allows us to ensure that the same exact equipment, electrophysiology equipment, same placement of equipment, electrodes, coils, same technique, same analysis of all the electrical signals being measured is consistent for all subjects across all study visits.
This helps reduce the variability of results in such a way that you really would be very difficult to do this if you included multiple centers. So, we're better off in this case using a single center trial and one that's highly experienced in electrophysiology.
The center we're using is Shirley Ryan ability lab in Chicago, formally known as Rehab Institute of Chicago. They have been ranked the number one rehabilitation hospital in the U.S. for more than 30 years. Our lead investigator is Dr. Monica Perez. She has extensive expertise in applying electrophysiology in exactly the way we're going to be doing in this trial to monitor motor recovery. She uses it both in clinical practice but mostly in clinical trials. She does things in a very standardized way to reduce the noise, to ensure that if there's an efficacy signal that she will see it.
I should also point out that in terms of this being a single center trial, it's feasible to conduct this study as a single center trial because the cohort size isn't large and it doesn't have to be large to demonstrate improved connectivity.
PropThink
The focus of the trial seems to be on the electrophysiological measurements. Will you also be looking at clinical outcomes?
Dan Mikol, NervGen CMO
Yeah, the focus is on assessing motor function using both clinical measures and electrophysiological testing. Electrophysiology is just a biomarker of clinical motor performance. It's telling you something but in a more objective quantitative way. More sensitive.
The primary objective of this trial and a number of secondary objectives will look at different types of electrophysiological assessments. But there's six secondary clinical outcome measures and several exploratory clinical outcome measures included in this trial.
This trial is loaded with a lot of different assessments. The clinical measures will assess, for example, grasping, finger dexterity, mobility, muscle strength, ability to do self-care, bowel and bladder function, quality of life. It's a very extensive list and it will complement the electrophysiological testing.
I think at the end of the day, the greatest value will come from looking at changes in both. Both the electrophysiological measures and the clinical measures throughout the study from start to finish at the end of the 16-week trial.
PropThink
So the treatment period here is 12 weeks, correct me if I'm wrong. So why is the study continuing for 16 weeks?
Dan Mikol, NervGen CMO
It comes back to what we saw in preclinical studies, animal studies, as spinal cord injury. Again, we're trying to translate what we've learned and try to learn more about what NVG-291 does in humans. In some of the animal studies, not all, functional assessments were followed after the treatment period for about a month. And in these studies, there was continued improvement seen even after stopping treatment.
I think if you can go to slide 10, the chronic study, there's a nice example actually on that slide. As you can see, the treatment period is shown in the box from week 12. Treatment began 12 weeks after injury, continued for eight weeks up until week 20. And you can see there's a continued improvement in NVG-291 treated animals for the month after treatment was stopped. That was the last assessment. We don't know what happens after week 24. This is very intriguing and this has been observed in some other studies.
We'd like to understand in our first proof of concept trial whether this kind of continued improvement might also occur in humans. So, we're going to be assessing, conducting our assessments throughout the 12-week treatment period of our trial as well as at a later time point. Four weeks later, week 16, which is the end of the study.
PropThink
Understood, okay. The study plans to enroll two cohorts. Why is that? And do you expect that it is going to be easier to see improvements in one cohort over the other?
Dan Mikol, NervGen CMO
Yeah, we saw a glimpse earlier and just now with the chronic data. We saw a glimpse of NVG-291 being effective when treatment was started either very soon after the injury in acute intervention models, or after animals had recovered after their recovery had plateaued - in chronic injury.
In our trial, we thought it was important to test both of these scenarios in humans because we really don't know whether in humans, NVG-291, will work if you give it soon after injury or after individuals have achieved the full extent of their recovery. Maybe it works in both. So, our trial is really two trials in one. Each cohort is essentially a different trial.
We'll be so we'll be enrolling the two cohorts of people with chronic injury defined as one to 10 years after injury and subacute, which is defined as 10 to 49 days after injury. Each of the two cohorts will enroll about 20 subjects.
Now to answer the second question about where we might expect to see more easily improvements in which cohort. The simple answer is that improvement should be easier to see in the chronic injury participants because there should be extremely little, if any, placebo effect in this population.
These individuals have already achieved whatever recovery they were able to achieve after spinal cord injury because recovery usually stops after about a year after injury. For the subacute cohort of participants, because we're intervening before full spontaneous recovery will have been achieved, one would expect some degree of improvement both clinical and electrophysiological in the placebo group. NVG-291 has to overcome that placebo effect.
This is true for any spinal cord injury trial that intervenes before full spontaneous recovery has occurred. I should say by the way that most spinal cord injury trials are acute intervention trials that begin very very soon after injury. Like 12 to 24 hours after injury. So we're able to extend that because of our results in the animal models.
Bottom line is going into this trial, we cannot predict whether NVG-291 might work better in one cohort or the other. It may work in both. So we feel like we essentially have two shots and goal here.
PropThink
Why is the trial not enrolling subject to have no motor function below the level of the injury?
Dan Mikol, NervGen CMO
Well, interestingly, most trials in spinal cord injury, both prior and ongoing, have frequently enrolled subjects with no motor function below the level of the injury. It's what we call motor complete, zero motor function below the level of the injury. This is the most severe type of injury. But the disconnect, again, is that the animal models are motor incomplete models. The animals have some residual motor function. It really, to me, makes absolutely no sense to go after the most severe population people who have no motor function when we have no results from animals to support that.
The approach we're taking in our proof of concept trial is to enroll humans with motor incomplete injury. They have some residual motor function. We define exactly what that is in the protocol. This stays consistent with the animal models that demonstrated efficacy of NVG-291.
If we can demonstrate efficacy in this trial, then we can consider testing NVG-291 more severe populations further down the road.
PropThink
Mike, I want to touch on what's going on in the field. There was news a few weeks ago that Elon Musk's chip company, Neuralink, raised $280 million at a reported $5 billion valuation. From a health care investor point of view, having an early stage company raised out of $5 billion valuation seems quite absurd, especially because it's only presented preclinical data and just entering into humans now.
Can you explain the excitement around brain computer interfaces and why there is such a discrepancy with NervGen’s valuation?
Mike Kelly, NervGen CEO
Yeah, I think the first two words under the Bloomberg heading explain the hype slash excitement around that evaluation. If my name were Elon Musk, I think we would probably have a higher valuation than we currently have right now.
Media drives a lot of attention to a lot of different things. One of the reasons why we're here today to talk to you is we got to get our story out. I think our story is not out as broadly as Elon Musk's Neuralink story. And that's okay for now. Our story will get out. I think we’re getting on a lot of people's radar. We've been doing a lot of talking to investors, a lot of talking to different banks in the area. And folks are starting to understand who we are, what we have, and how quickly what we have is going to materialize into something.
Our expectation is that we're going to enroll a chronic cohort this year. We're going to have data readout of a drug in the spinal cord area by midyear next year. I don't think there's any other company out there that could say that right now. With the preclinical package that we have and the study that's underway to be within 12 months of a chronic data readout for us is, I think we're undervalued, but so does every other CEO of every other public company in the world.
We're a smaller company based on the TSX and the OTC. And I think it's just going to be a matter of time where the discrepancies and value start to merge. I think we're undervalued. I think others may be overvalued in this space. Media plays a lot in that. I think we're taking a measured approach. We're focusing on execution and allowing our executing of our plans to be the value that we create for our shareholders.
I think the shareholders that do their homework and dig into the data that I dug into when I joined this company, they're going to be rewarded when we start to have uplifts and value. Those uplifts are starting.
PropThink
Marketing is great and obviously, Musk is phenomenal with that. Especially in biotech investments, at the end of the day, it's all about data and you have a chance to really just go above and beyond everyone else.
Rich, I want to get your take because you've been involved in the biotech investment community and the industry ecosystem for a long time now. Why do you think that NervGen has mostly been off the radar of the investment community? What do you think is going to take for the company to capture the attention of investors and the biotech community?
Rich Macary
Yeah, well, certainly, when you look at what I do with AVI and other situations, I tend to look where others aren't looking, either because they don't understand or they haven't done the homework or there's a misperception.
In this case, in all fairness, I was asked three times over a two month period almost two years ago to look at this company and I didn't do it. And I didn't do it because I've been on Wall Street a long time. You tell me about a public company in Vancouver on the TSXV with a dollar-something share price and that I'm going to end up finding neuroscience innovation that I'd be interested in, of the hundreds of companies and opportunities out there. It just didn't seem plausible. So, I didn't look. It was only because of the credibility of the people asking me to look that I finally did.
I'll tell you, when I did, and again, I'm five years into digging in deep and doing diligence on companies and everything I heard from this company was like new to me. You know, I heard a little bit about glial scars, about CSPGs, PTP Sigma, you know, extracellular matrix and paraneurons. What is this? This isn't tau and amyloid. This isn't, you know, some of the things that I've been looking at.
So, I feel that that's part of what this is. This is a new area. You literally have a single company pioneering this area because a single researcher, Dr. Jerry Silver, went on a 30-year journey to do this. He overturned nearly a hundred years of neuroscience precedent that said, you can't regenerate the nervous system. Then went on to discover receptor and then went on in his lab to create a drug which we're about to put into spinal cord injury, those suffering spinal cord injuries.
So, I believe it's really a matter of, first of all, education around what this technology is, how exciting it is. Again, to me, it's as big as that beginning of the RNA therapeutic revolution, being able to repair nervous system damage.
I think another part of it is spinal cord injury. The people that knew about DMD when Sarepta made that pivot, is people watching Jerry Lewis telethons. They didn't know about DMD. Spinal cord injury people know about it. They just don't view it as something that a pharmaceutical can become a game-change in. If they did, and they busted out the calculator, look at what this market could be. That this is about to happen. This is a clinical study starting. I think that creates an inflection.
That's exactly, by the way, what happened in Sarepta. When Garabedian came in, they started to run this trial. People started to look at their preclinical data and said, “Oh, wow, if this works, you're going to revalue this company almost overnight.”
So, for me, it's really what's happening right now with the company. New CEO, great team, taking this preclinical asset, and it's becoming a clinical asset right now. With a relatively fast readout, I think we're at the very doorstep of that valuation inflection starting. I think the data itself is going to be the key. But I think a lot of people are going to start to be excited right now.
As an individual investor, I'm not thinking about multi-billion-dollar hedge funds, some of them came in early, like Perceptive, on AVI/Sarepta. Others waited till the coast was clear - it was a billion plus valuation. They helped drive at the 10 billion.
I feel that as an investor, you know, you want to be in something exciting. You want to be in something to change the game. Innovation, nervous system repair. This is the holy grail of neuroscience. If it happens here, you're not talking about stock doubling or tripling. You're talking about a long-term building of incredible value. Like it happened with Sarepta. I can guarantee you, those folks that were there in the beginning didn't ride it all the way to 10 billion.
I believe that this works, and because of what it is and where it could be applied, the upside is so much larger than what was there at Sarepta. It could happen so much faster, so, again, I just think people haven't paid attention for all those reasons.
One last thing, this goes to Elon Musk. This didn't walk out of Robert Langer's lab at MIT. It walked out of Dr. Jerry Silver's lab at Case Western. There wasn't a line of RA Capita, Orbimed and Flagship waiting to grab it. Instead, there was a father who wanted to help his daughter-in-law, who saw this as being potential, got money together, took it public to be able to turn into the company it is today. That's not how this normally happens. That's one of the reasons why it's not on everybody's radar screen. That's about to change the model.
PropThink
You know, it could be the needle in the haystack. Just a point on Sarepta, if I'm not mistaken, they're also from the Northwest. So maybe there's something in the water up there that is driving some of this innovation.
Rich Macary
Yeah, they were in Bothell, Washington, and Portland, so you could throw a rock and hit NervGen's headquarters. I'll say that as part of that game, Chris eventually moved the company to Cambridge to sort of be amongst the biotech behemoths, and that's exactly what Sarepta has become. I'm feeling that same way here in NervGen. I think this is the time and the inflection point is now.
PropThink
Let's start with the Q&A portion of the event. The audience may submit a question using the text box at the bottom right of your screen. I think we already have a few questions submitted.
First question here. What concentration of NVG-291 will be used in this Phase 1-2 study?
Dan Mikol, NervGen CMO
I can take that. So first I'll say we haven't gone into all the details, which is just not enough time, but in across all the different preclinical studies of spinal cord injury, a very broad range of doses was tested. Doses as low as 0.007mg/kg was shown to be effective, and the highest dose that was tested was 0.32 mg/kg. That's a dose based on body weight.
In humans, we have selected a fixed dose that will give us exposures that are at least comparable to, if not exceeding, the very highest doses that were tested in preclinical studies. So again, a very broad range was tested and found to be effective. We're aiming at the very high end of the efficacy bar.
It could be that lower doses are effective, but you know, in this first trial, we want to give ourselves the best chance of success.
PropThink
Perfect. And another question here, just talking about the enrollment of this ongoing trial, are patients excited? What are physicians telling them to convince patients to go on your trial?
Dan Mikol, NervGen CMO
Well, first of all, as far as excitement, it's interesting when I joined NervGen, I kind of felt like a rock star in the spinal cord injury community because people in the spinal cord injury community are very familiar with Jerry Silver's work. We get contacted all the time by people who are just learning about the some of the data. And there's a lot of data that's been amassed over the last few decades coming out of Jerry Silver's lab in particular.
There's a lot of excitement. We get contacted. Shirley Riley Ability Lab gets contacted about potential participation in the study. So, there's a tremendous amount of excitement. We just had IRB approval. I think we announced that in a press release about a week or two ago. And screening will be happening very, very soon. Enrollment will be happening very, very soon after that. So we're on the cusp of enrollment. We haven't enrolled anyone just yet, but it's because we're in the final stages of making sure that the site is ready to go. And that's going to happen imminently. And we'll have a press release when we do actually dose the first subject.
So there's something about competitors. We don't really see any competition for participation in our study. I'm not sure if that's implied in the question. That has to do with the fact that our first cohort we're going to enroll is the chronic cohort. We're also going to enroll subjects with subacute injury. Most of the trials enrolling subjects, again, within 12 or 24 hours after injury. We're not competing with that at all. We're also targeting a single center. They have a vast amount of people with spinal cord injury that they can recruit from.
Question
So how much time will the trial require in terms of weeks or months?
Dan Mikol, NervGen CMO
The first cohort we're recruiting is the chronic cohort. This will be very easy to recruit in the sense that these people already exist, people who've had their injury one to 10 years ago. They have to be eligible and satisfy eligibility criteria. The site has been doing a lot of prescreening. We have, we have a lot of subjects who are ready to go and to begin the formal screening process very, very soon. The site has resources to enroll subjects rather quickly.
What we're saying publicly in terms of a readout for the chronic cohort, we expect to read out sometime in the middle of next year, which has a little bit of variability in terms of what that actually means. But it's less than a year away, for sure, for the chronic cohort. Then the sub-acute cohort would be it'll take a little longer to recruit because the window is 10 to 49 days after injury. We anticipate having data for the sub-acute cohort at the end of next year, possibly beginning of 2025.
Question
The motor incomplete versus motor complete. What are the prospects for motor complete, which is sometimes referred to as Asian impairment scale A and B?
Dan Mikol, NervGen CMO
Motor complete is no motor function below the level of the injury. As I alluded to, if we can demonstrate efficacy in this trial, then we would be very interested in pursuing other populations. We're trying to first successfully translate from animals. We don't know how this might work.
First, let's see what we do in our targeted population. If we can show efficacy, we would consider exploring other populations. The thing I will say is that even people with motor complete injury will often have evidence of electrophysiological connectivity.
So even though they don't have enough electrical connectivity to get a muscle to move below the level of the injury, the signal is still there. I think there is hope that if that can be amplified, that there could be a benefit in the more severe people as well.
Question
Kringle is using HGF protein for SCI. They showed some efficacy but not stat sig. Currently in Phase 3, they’re enrolling the least mobile patients with an AIS score A. Is this patient population the hardest to treat? How are you different?
Dan Mikol, NervGen CMO
So I've talked to colleagues from other companies, and there aren't a lot of them who are developing therapies in spinal cord injury, like Kringle or Mitsubishi. They're all targeting similar populations, which is leaning towards the more severe spectrum.
To me, I have difficulty understanding this. I've shared that with some of my colleagues at other companies. So I think, again, to successfully translate, it makes most sense to try to reproduce what we've done in animal studies in humans. That is going after people who have some residual motor function.
Question
Are you planning to list on the NASDAQ exchange?
Mike Kelly, NervGen CEO
Short answer is yes, but timing is going to be key. I think we're going to likely attempt to uplist when the timing is right. Right now, we're kind of happy that we're not on NASDAQ. The way that the XBI had been hammered over the past year and a half to two years. But I think we do have it in our sites. We think that this company is the type of company that will get a lot of attraction from larger institutional investors whose core business is on NASDAQ. So it's just a matter of time.
Question
Is this a treatment an SCI patient would take for a short time period, or ongoing throughout their life?
Mike Kelly, NervGen CEO
Well, we're studying it in the 12-week period. We feel that obviously this is a lifecycle management question. We expect if people will want to continue to treat if they do get a beneficial effect. If you think about the mechanism that Dan walked through, if we could help the body regenerate connections, and we could show it in a 12-week study, we feel that those connections can be built upon. Patients could continue to improve through exercise and rehabilitation once connections are there. So, will they have to go through other treatments? We're not sure yet. We're studying it for the 12 week period of treatment. We expect that we're going to see some results. We'll approach that question after we see the results from this study.
Question
There seems to be a number of miracle recoveries in rodent models over the years. Why has none of this translated to primates and why not test NVG in dogs or chimps as well?
Mike Kelly, NervGen CEO
I think Dan alluded to the fact that why a lot of other companies are going after a different patient set. They're going after the acute patient set. Most of these clinical trials are complete and acute. We are going after incomplete and subacute and chronic. It's just a different target that we're going after.
We're following what the preclinical results that we've seen and NVG-291 show that we share with you on this presentation. Are there plans to study this in dogs and chimps as well? Not from an efficacy perspective. We're in the middle of starting a human clinical trial. So there's no reason to go back and, and try it in other larger animal models.
We will be doing other studies in dogs and chimps as part of the development plan. Probably dogs, not chimps in the development plan as we move forward.
Question
If you see positive results from the Phase 1b-2a trial at chronic cohort, will you continue to just focus on SEI or will you initiate a trial, another indication like stroke?
Mike Kelly, NervGen CEO
So this is when I answered the question, focus and execution in chaotic times. I think we'll have the ability to go in many different areas, post this chronic cohort readout. If we have positive results in this chronic cohort readout, we need to remain focused. Obviously we will be growing the company as fast as we possibly can to go into these other areas.
Stroke is on the top of our radar, underneath the spinal cord injury because there's some fantastic preclinical data in stroke that's up on our website. You can go and take a look at it as well. We have other different disease states.
We have NVG-300, which is another product candidate in our pipeline. And the reason we're setting that up is to start to diversify into other different disease states with other different potential products and grow this company.
We can see a path where we can take spinal cord injury ourselves and we can potentially partner some of the larger indications with other companies. That planning process is underway. We recently had a strategic planning process with the management team. And we decided to focus heavily on spinal cord injury. And then after we start to get some traction in that spinal cord injury space, broaden out indications under NVG-291 and then advance NVG-300 as well.
PropThink
Next question asks if someone's going to let Elon Musk know that Rich called him out. So Rich, I don't know if you want to do that or I will, but we can both call him and let him know what's going on here. *laughter*
Rich Macary
I don't recall calling him out. And I don't recall telling Mike that I think he should change the name to NervGenX either. *laughter*
Mike Kelly, NervGen CEO
Yeah, if he wants to invest, he can call us anytime. You know, we'd be happy to talk to him. If somebody wants to reach out to him, let him know. *laughter*
Question
Do you plan to raise additional funds through a share offering before the chronic cohort readout or after?
Mike Kelly, NervGen CEO
Yeah, we just announced our financials I think about a week ago. We have enough cash in the bank to conduct this clinic trial. Obviously, there's a lot of other things that I just mentioned that we'd like to do. We'd like to get proof of concept in other areas. We're going to be opportunistic with financing. We do have enough cash in the bank to to get us through chronic cohort readout.
I think as Rich mentioned earlier, we believe that there's we're gaining some traction getting the story out there. If investors want to come in for a big chunk of money, we're a small public company. We're always, as a development company, looking for more cash, but it really comes down to timing and valuation.
Question
Will you look for a partner or can you handle the development?
Mike Kelly, NervGen CEO
We can handle spinal cord injury. And I think we can also handle several other different disease states. It's a matter of how quickly can we grow the company. It's our goal to hold on to this product. Obviously, being a part of several transactions that have happened in the past, I don't think I was ever with a company where the executive team wanted to sell the company.
Our goal is to build a company. But sometimes that that is out of your control, especially if you're a public company, if somebody comes and makes an offer you have to bring it to the board and we have a fiduciary responsibility to look at it. I truly think that we can navigate spinal cord injury by ourselves. And that value uplift will allow us to build out an organization and start to get into some other indications ourselves.
When the resources of four or five of these indications and the size of the organization becomes onerous, we will see if a partner can help us expedite an indication and get it to market faster. We'll talk to partners, but then it really comes down to how much are they going to want for that? And how much are we going to keep for that?
Dan has built organizations much bigger than the organization he's residing in now. I've built organizations much bigger than this organization. So, we have the team here that could build out this company as big as we need to build it without relying on anybody else. But we also are business people and we're going to look at this from a getting the drug to market for each indication on an individual basis. So, that's kind of the thought process.
I've avoided answering the question because there's no there's no answer here. But I think I'd love to get across to you that we feel that we can build out this organization and take this product to the promised land for the first indication, which is going to unlock the value for all of the other indications.
PropThink
There's a question here discussing the partial clinical hold that was announced time ago. And if there's been any progress made there, if that's even necessary to move forward?
Mike Kelly, NervGen CEO
Well, the short answer is it's not necessary to move forward right now. We've gotten the green light from the FDA to conduct the trial that we're conducting. We will address it as we continue to progress the product through the development stage. We were collecting the information that the FDA wants us to provide. But we don't see it as a roadblock today. We've gotten the green light from the FDA to conduct this clinical trial and we don't need to resolve the partial clinical hold prior to the chronic data readout.
Quite honestly, you don't necessarily need to resolve a partial clinical hold to get a product approved either. So, we're going to be addressing it, but it's not a real limit for the company today.
PropThink
Have there been any other drugs that have shown what you have shown in terms of efficacy in animal models that you can think of?
Dan Mikol, NervGen CMO
You can look back over the last few decades. There have been a number of phase two and phase three clinical trials and spinal cord injury based to different extents and some animal data. The vast majority of cases, to my recollection, is based on the small number of animal studies. Again, motor incomplete models and the trials have enrolled largely Asian Impairment Scale A and B individuals, sometimes also C, sometimes also D. But by including the motor complete population, it could be diluting effect.
I think one of the problems may just be lack of adequate replication of the data. So, positive results in one animal experiment alone would be very risky going into a clinical trial. I feel we're on much steadier ground because there's so much data in spinal cord injury by independent investigators.
PropThink
The next question here talks about the expectations and how you would rank them in success for chronic and acute groups. And as far as the endpoints would go, I think the question is getting at what defines a positive proof of concept trial?
Dan Mikol, NervGen CMO
Typically a proof of concept trial, what you'd like to see is you'd like to see efficacy on a surrogate outcome measure, which in our cases, like physiology. In other clinical trials, it could be changes on brain MRI or beta amyloid levels in Alzheimer's disease. You'd like to have a surrogate tool because clinical measures are just noisy. To show an effect on a clinical measure, you need much larger sample sizes and more subjects in the trial.
So what we're looking for, and that's why we included electrophysiology in this trial, is it's much easier for us to do this kind of a trial than to do a several hundred subject trial with clinical outcome measures only.
We'll be looking for changes in both the clinical measures and electrophysiological measures and both together. Our expectations, I don't think we can define upfront what success looks like, but I think we'll know it when we see it. We're going to be looking for changes in electrophysiology and clinical measures.
In the chronic cohort, this will be much easier. These individuals are stable. They should not improve with placebo because their recovery had already been achieved. So, I think it'll be easier even with the sample size we have to see effects on clinical measures. We have a lot of them included. I'll just try to adjust this briefly. We like to include performance measures because these can be quantitative. It gives you more information than a clinical outcome measure like a motor score, which is very widely used, where you're measuring muscle strength in people.
I think our six secondary clinical outcome measures, sort of four of them are performance tests. The other two are basically upper and lower extremity motor scores, which is just an improvement in strength in specific muscle groups.
Frankly, I would expect to see directional improvements in all of these, if this drug works. If we can show an effect on electrophysiology, we're going to see directional improvements. Whether we're going to see P values that are statistically significant is kind of irrelevant. That's not something that you have to have in a proof of concept trial. It's great if you can see it. But if we can see directional movements safe for the chronic cohort that has just been static for years, that's going to give us the confidence to move forward.
The extent of improvement that we see on different endpoints will help us design the next, trial multi-center trial.
PropThink
Mike, this one is for you. Can you talk about any catalyst if there are any before the SCI data readout in mid-2024?
Mike Kelly, NervGen CEO
Yeah, I think the biggest catalyst is going to be the anticipation of enrolling in this trial and us getting the story out. We're going to be on the road, talk to a lot of folks once Labor Day comes. Once everyone goes back to work after Labor Day, we're going to be traveling quite a bit and talk to a lot of people. So I think that the story getting out plus the enrollment kicking in are going to be two major catalysts for the organization.
In addition to things that we've talked about previously, we'll be applying for fast-track designation and several other different catalysts that we have in our communication plan. But I think the biggest thing for us is the execution of this clinical trial and getting enrollment going as quickly as possible so that we can arm Dan with the data to go out and start to talk about analyses of the study at medical meetings. That's really going to be the catalyst for the company moving forward.
PropThink
Assuming data is positive, what would the timeline look like to get 291 commercial? It's probably early, but what the cost would look like as well.
Mike Kelly, NervGen CEO
It's really hard to answer that question because it comes down to the magnitude of the fact that we see in this trial. If we hit a homerun, it's very different than if we hit a single or double. But a single is a magnitude difference here. We talked about seeing primary endpoints with aligned secondary endpoints.
If we have statistical significance in several different primary and secondary endpoints, I think that's a different ball game. It gives us a lot more to talk to when we sit down with the FDA and map out what that plan looks like. We feel we'll have to do one more, at least one more clinical trial. If we file for a priority review, folks could do the math and try to figure out how quickly this thing can get to market.
As far as costs, we haven't talked about what it's going to totally cost. We've given the cost of this trial. We haven't given the cost for future trials because we don't know how big those future trials have to be. Our hope is that they're going to be a modest size, a multi-center and fast. As fast as this chronic cohort is going to enroll.
Not a lot of folks can do a study starting today and get a readout mid-year next year. So, you can see how fast we can do this proof of concept. It's likely going to be as fast, maybe a little bit longer because it's going to be a bigger study, the next study, and then a short review period.
That's kind of the high-level timeline that we're talking about to get approval. We'll be a lot more specific on what the cost of that trial is after we sit down with the FDA and map out the path to regulatory approval.
Rich Macary
I would just add to that, when you look at the accelerated approvals that have happened, where there's significant unmet need, there is no other therapy in the market. Whether you look at, you know, Amylyx in ALS, you look at Aduhelm in Alzheimer's, you just look at the most recent one from Sorepta. They were open-label extensions or ad-hoc, post-hoc analysis. They're not statistically significant. They didn't hit their endpoints and yet they were approved because there were signals that they have to now prove in a confirmatory study.
You just got to also be thinking about, when you look at indications like this, where the bar might be, and that's something the company can't predict, but there is a source by virtue of what's happened in a lot of these other rare or unmet conditions.
PropThink
Are there any upcoming preclinical data for Alzheimer's?
Mike Kelly, NervGen CEO
There will be, but not at this time. We haven't announced when we're going to be doing those studies, but we will put up press releases when we actually kick them off and we'll give you an expected timeframe for readout.
PropThink
One of the questions talks about complete versus motor incomplete, and if the FDA would require another study for motor complete, or if that would impact your, label, just to include Asian A-B, and not just the C-D population.
Dan Mikol, NervGen CMO
We'll see. I don't know how they would respond. I mean, this is an area of high unmet need. Sometimes the FDA is a little bit broad in terms of their label. Payers can be a little bit more strict, so there may be a difference there and what might get paid for.
But as I said earlier, if we can show efficacy in motor incomplete individuals, I don't think we want to stop there. We want to try to help as many people as we can with spinal cord injuries, so we need to begin to investigate that. Once we can show efficacy and have discussions with the FDA, we'll be able to have a little bit more clarity about what might be necessary in terms of other trials or not. It's just too premature to say much more than that.
Mike Kelly, NervGen CEO
I can add to that a little bit. I think from a market perspective, the majority of cases are motor incomplete. I think if we show that we can have improvement in motor incomplete patients, it would be hard for anyone to say, oh, you got to prove this in motor complete.
There's several other disease states that folks pick a patient population and they prove that they can have an effect in that population, then it's up to the FDA to decide, well, do they want to restrict the label to to just that population or give them the open label to the entire population?
I could tell you there's going to be a lot of pressure to look at the broader population. But even if they give us the narrow population of motor incomplete, let's take that. That's the billion dollar opportunity I was talking about. It's not a small marketplace. It's the majority of cases and payers that we did market research with have given us the answer that we had expected, which is if you can help these patients become independent, they're going to pay for it.
We will do studies in motor complete eventually, but our goal if we show motor and chronic complete, we’re going after chronic motor incomplete.
It's the same discussion we have about subacute. If the chronic readout is positive, do you continue on with the subacute? Well, ethically, I think we have to, but practically, who's not going to treat somebody who's injured from days 10 to 49 when we've shown that we can affect folks a year to 10 years have. It's just common sense that they're going to treat the entire population. But we will continue on with that segment, that cohort, we're not going to stop the trial.
As Dan mentioned, this is two clunk of trials in one. We have to remember, this is a proof of concept trial. Based on the results in this trial, we may go after one group, the other group, or both groups. The market will dictate coverage for this product. Regardless of if it’s subacute or chronic, it's going to be very hard for payers to say, well, hold on, you got to wait. Your injuries day 10, you got to wait a year for me to cover this product. It's not going to happen. The political pressure put on that payer is going to be so high.
PropThink
The last question that I see here, how is NVG-300 chemically different than NVG-291?
Mike Kelly, NervGen CEO
We're not disclosing the chemical structure. As of yet, we're right now in the mode of building the patent estate around NVG-300. What we could tell you, it's the next generation molecule that was internally discovered. And we're extremely excited about it. I can tell you more once we firm up our patent position, but we still have a little work to do to make sure that we protect this asset as best we possibly can, because it does give us much longer life if we do it the right way.
PropThink
If anyone would like to add any final remarks just to close.
Mike Kelly, NervGen
Yeah, first off, I want to thank you and PropThink for helping us get our story out. I think the more of these we do, the more we can inform the world on what we have and what we're embarking on and how quickly we're going to see something. I appreciate Dan's time, Rich's time, and your time to make sure that we as a company continue to do what we need to do, which is get our story out to as many people as possible. So thank you.
PropThink
Thanks for participating. Everyone, thanks for attending. I think the event obviously proved to be very insightful. It was much longer than I anticipated, but this concludes the presentation and the Q&A portion. The event was sponsored by NerveGen. If you're interested in getting in touch with the company, you may do so at their website nervgen.com. If you're interested in learning about PropThink, you can visit our website at propthink.com or follow us on Twitter with a handle @propthinker. Thank you for attending and I hope to see everyone again.
Disclaimer:
PropThink Digital Conferences are organized and marketed by PropThink LLC ("PropThink" or "we") with the aim of connecting subject matter experts with investors. This Digital Conference is sponsored by NervGen Pharma Corp. (the "Company") who has paid fifteen thousand dollars for PropThink to conceptualize, market and host a Digital Conference featuring the Company. PropThink is not a registered investment advisor in any jurisdiction. We do not publish investment advice and remind readers that investing involves considerable risk. PropThink urges all readers to carefully review the Company's regulatory filings and consult with an investment professional before making any investment decisions. Please read our full disclaimer at https://propthink.com/disclaimer
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