In this episode of the Capital Compass, we take a closer look at Marvel Biosciences (TSXV:MRVL,OTC:MBCOF), a company developing a novel neuroscience platform with potential applications across autism spectrum disorder, Rett syndrome, and Alzheimer’s disease.
Marvel is advancing its lead asset, MB-204, toward first-in-human studies, while strengthening its intellectual property position and laying the groundwork for strategic partnerships.
With recent financing activity, accelerating warrant exercises, and growing preclinical validation, the company is approaching an important inflection point.
Joining Stockhouse’s own Ricki Lee is Rod Matheson, CEO of Marvel Biosciences, and Dr. Mark Williams, President and Chief Science Officer. Watch the video above, or read the full transcript below.
Developing a neuroscience platform with potential applications across several indications
Ricki: So, you’ve moved to accelerate the expiry of your warrants following strong exercise activity. So, what does that tell you about investor confidence and how does it impact Marvel’s capital structure as you move closer to human trials?
Rod: Well, we’ve always had strong support from our various investors, and in actual fact, we had a warrant that had a bit of an extra timeframe on it, but we started getting those exercised without forcing the exercise which spoke to our shareholders wanting to keep funding alive and well, and overall, we’ve got some really positive shareholders and very loyal that really believe in what we’re doing.
Ricki: And so, biotech IP really underpins long-term value, right. So, Marvel recently secured a granted patent for MB-204 in China, the first allowance worldwide for the compound. So, can you walk us through your global IP strategy and why it matters for valuation and potential exits?
Rod: Good question. The composition of matter is a major milestone for us. And having the first approval come through in China is an important validation of the strength and the novelty of MB-204. We’re also applying in other jurisdictions, of course, the US, Japan, Europe to get our worldwide composition of matter patents together.
Ricki: And so, MB-204 is being positioned across several indications from rare neurodevelopmental disorders to Alzheimer’s disease. How do you prioritize across those programs and what’s the commercial logic behind taking a single molecule into multiple disease areas?
Rod: As a result of our studies in Alzheimer’s that led us into our models with social awareness, social confidence, social communication, taking us into another area which is pretty topical these days, autism.
And so that became the priority because it’s an unmet area. We’ve got what we believe to be something of significance for that particular condition. And we are in Rett, Fragile X and of course just general overall autism.
Ricki: And so, for investors watching from the outside, what do you see as Marvel’s near term catalysts, and just as importantly, what differentiates your risk profile from a typical early stage biotech?
Rod: We have several near term preclinical readouts coming that we believe will be quite unique, including data from our liquid pediatric formulation. We’re also working with FRAXA Research Foundation with MB-204 in their development pipeline.
We expect to be able to announce further progress on our IP funding and data releases, which together represent clear near term catalysts. From a risk perspective, Marvel is differentiated from typical early stage biotechs in that we are building value through multiple data sets, indications and IP milestones instead of human trials, rather than relying on a single binary outcome.
Overall, these catalysts, we believe, reflect our focus on steady execution and delivering on the milestones we’ve committed to.
Ricki: Great. Thank you, Rod. So, Mark, we’re going to shift themes now slightly for viewers without a neuroscience background. Can you explain some of this in plain terms, how MB-204 works, what the adenosine A2A receptor does and why that target is relevant across neurological and neurodevelopmental disorders?
Mark: That’s right. Thank you for the question. So, the adenosine A2A receptor is involved in actually a diverse set of states including neuroinflammation, Tal phosphorylation. And as it turns out, more recently, as Rod alluded to, social interaction.
And so, the A2A receptor is found specifically all over the body, but within the brain, it actually turns out it is highly involved in social reward pathways. So, this touches on autism where we believe that there’s a deficit in social reward. Children and adults who suffer from that particular sort of state don’t get a charge out of social interaction like you or I would normally do.
And the adenosine A2A receptor is pivotal in that problem. And if you block it, it’s actually able to restore this social interactivity pathway and social reward pathway essentially almost immediately within an hour.
And so, what we have now found is that in multiple models including depression and multiple models of autism, a single dose within an hour is able to restore this pathway.
And at least preclinical animals are more socially interactive. So, it’s a very interesting molecule and target. And the target itself, there is a drug that’s been developed for it called Istradefylline, and it was developed to treat Parkinson’s disease.
So, we have all this background on the target, and this original drug and MB-204 is now a unique derivative of Istradefylline that we’ve made to have longer activity, and it’s engineered specifically more for these other non-Parkinson’s disease states.
Ricki: That’s incredible. So, one of the most interesting data points you’ve released comes from your Rett syndrome work. Can you talk us through that study and why those findings are so important?
Mark: So, we’ve been working with a collaborative group out of France who were experts on autism and social behavior, and they’d already tested one model of autism and shown that, again, as I mentioned, essentially after one hour after oral dosing of our compound, we essentially reversed the social deficits.
Animals were acting as normal as other animals, but that was after a single dose in a lesser known model of autism. But Rett syndrome is a disease that affects a large number of children, although it’s an orphan disease, it’s quite extreme.
And we were able to show chronic dosing with MB-204, was able to also restore essentially all the endpoints that we looked at, which is touching, following, anything that involves social interactivity. And in that we also had a control drug called Trofinetide. Trofinetide is actually approved to treat Rett syndrome.
And essentially on every single measurement we beat Trofinetide hands down. Not only that, we actually stopped treatment and said, well, what’s the effect after we stopped treatment?
And it turns out, this benefit with social interactivity was still seen two, three weeks later, even though the animals were no longer on drug. And that is simply not true of Trofinetide with the endpoints that we tested.
So we have a drug that’s immediately active. It can stay active for a long time and actually even has a benefit once you are off the drug for a considerable amount of time. So that’s a very exciting discovery for us, and we think ultimately for the whole autism field and for clinicians who deal with that particular type of disease.
Ricki: Wow. And Marvel also presented Alzheimer’s data at AAIC in collaboration with Professor Emmanuel Planel, a globally recognized expert in tau pathology. So what have you demonstrated so far, and why was that presentation a meaningful milestone for the program?
Mark: That’s, that’s a good question too. So, in Alzheimer’s research right now, there’s two really big targets. One is beta-amyloid, which most people have probably heard at some point in their life.
We now have two drugs that can treat beta-amyloid. There is another target for which there are no drugs called tau, and you can kind of think of these diseases are related in that they’re caused by clumping of proteins. And that’s a problem because that causes all sorts of issues.
So, beta-amyloid clumps outside of cells and tau typically clumps inside cells. And that process is driven by a pretty fundamental biochemical process where the tau protein is modified with what’s called a phosphate group. And what we were able to show is after a very short period of time, our MB-204 was able to stop this phosphorylation of tau, which in turn should prevent the long-term clumping problem of tau, which is called tau tangles.
So, the fact that we were able to see this effect again after oral dosing, after this other very large target for which there are currently no drugs to treat for Alzheimer’s was exciting. And we were very pleased to be able to present that data in collaboration with Dr. Planel at the AAIC meeting.
Ricki: And as Rod alluded to earlier, you’re also working with the FRAXA Research Foundation on Fragile X syndrome. So where does that program stand today and how does it fit into Marvel’s broader platform strategy?
Mark: Thank you for that question too. So Fragile X is probably the most commonly inherited neurodegenerative disease and was certainly related to autism that way. The FRAXA group has been actively searching for compounds to test independently in their own labs to try and advance therapies for that particular class of autism and children thereof.
And so we have a collaboration with them. We are anticipating a readout from that study with the oral dosing of our MB-204 imminently, certainly this quarter or early next quarter as Rod alluded to. And again, that would be further validation that our drug is active in another third now form of autism where we know that genetic basis of the cause.
I think coupled with that, with the depression data that we’ve already previously reported on I think we have a very compelling compound to treat autism. And also, by the way, depression is a very common issue with children with autism.
They suffer; I think four times the rate of depression compared to their neurotypical peers that way. So, this drug could have a secondary benefit to that particular patient population as well.
Ricki: Wow, that’s incredible. So finally, then, on clinical readiness, where does MB-204 stand today in terms of toxicology and GMP manufacturing? And what can you tell us about the timeline and design for a first in human study?
Mark: Again, thank you for the question. So, we’re very fortunate, and this is another element of Marvel that needs to be appreciated. The major risks have sort of been dealt with now in terms of the toxicology.
We have completed that. We’ve completed kilogram batch manufacturing of our drug, that’s ready to go. We are essentially ready to go into clinic at any time. We would like to develop this as Rod had mentioned, a liquid based formulation for the drug, which would, I think, improve patient compliance, particularly in the autism group, people who often have difficulty swallowing, especially children with Rett syndrome and Fragile X, that might be an even larger issue for them.
And with the rest of our data that we’ve generated so far, we think we are ready to go into the clinic. We think we’re a de-risked asset, again composition of matter, manufacturing, toxicology, excellent preclinical data, known drug with good safety history that we based ours off of. In summary, I think it’s a very compelling reason to go into clinic and good time to be involved with Marvel Biosciences.
Ricki: Well, Rod, Mark — thank you both for walking us through the strategy and the science behind Marvel Biosciences.
That was Rod Matheson, CEO of Marvel Biosciences, and Dr. Mark Williams, President & Chief Science Officer. That’s all for this edition of The Capital Compass. I’m Ricki Lee — thanks for watching, and we’ll see you next time.
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