Conference Transcript - TNYA 2026-08-11

Whitney Ejim, Analyst — Center Court

Thanks, everyone, for joining. My name is Whitney Ejim. I'm one of the Biotech Analyst here at the Center Court. And it's my pleasure to be joined by Tanaya.

Whitney Ejim, Analyst — Center Court

I think I said the good afternoon. So Tanaya this afternoon, pleased to be joined by. And Eric Heiligren, CFO. So thank you for making the trip. Thank you for coming. We have a lot to cover. So diving right in. And for anybody who's not familiar with the story, can you provide just a high-level overview? You know, what attracted you to the company? Because you're relatively new. And what is the team trying to build over the next five, ten years?

Speaker 1

Yeah, great one. Thanks for having me, Whitney. I've been in the chair almost a month, so still a new guy. And, yeah, I mean, when I thought about, you know, Tanai and joining the company, really it's about the mission, right? So we are looking for precision medicines for heart disease. We're modality agnostic, so we have gene therapy, we have small molecule. but really we're trying to address the underlying cause of the disease and so the mission of the company really resonated with me also the science when i looked at the data presented to date strong data very encouraging and the team you know the team that i met with during the interview process very excited and energized to succeed in this space and then we have a big big year here Q4, there'll be, and we'll get into this, but regulatory updates coming, our plans for advancing our small molecule 301. Also, we'll talk about those in Q4. So a lot of near-term events for the company as well.

Whitney Ejim, Analyst — Center Court

Okay, excellent. So maybe starting with TN201, the lead gene therapy, I think. And so can you just briefly review what that is? What is MYBPC3? It's a mouthful associated HCM and the kind of key unmet need as patient population.

Speaker 1

Right. So as you said, with MYBPC3 in HCM, we're talking about about 120,000 patients in the U.S. here. So that's about 20% of all HCM. And really what we see with these patients is a thickening of the left ventricle and so the heart's not able to pump blood where it needs to go in the body effectively and there's really a lack of treatment effective treatments out there and so especially with folks with more severe disease and so you know we feel like targeting this area really you know our gene therapy can improve outcomes and and you know results for these folks okay and which endpoints are most important to us as you guys are monitoring the data and as you're talking to the physicians as well? Sure. So as I mentioned, you know, these left ventricles thicken or they get large. So reducing the thickness of the left ventricle LVMI is very important to us. We feel like that then would contribute to better outcomes. You know, we're having discussions with regulators on appropriate endpoints but the data we've shown to date has been very strong in that area and so we would think that would be appropriate.

Whitney Ejim, Analyst — Center Court

Okay, got it. And the expansion cohort in the study is ongoing. Can you remind us which dose is being used, what the target enrollment is, and I guess, yeah, if there's been any changes there as the study has evolved over time?

Speaker 1

Yeah, so in this cohort two it's the higher dose, so 6E13, cohort one was half of that. and really well-tolerated from a safety perspective. And again, the LVMI reductions we saw were very meaningful in the majority of patients. And I think also what's important is from a feel and function kind of symptoms, those have improved as well for most patients. So it's not just LVMI, but it's how these folks are feeling.

Whitney Ejim, Analyst — Center Court

Okay, perfect. And you just touched on it as well, but safety, all important in general, but particularly against the bad opposite things that have been happening in the broader cardiac gene therapy space. So can you talk about what you've seen there?

Speaker 1

Yeah, so, right. Very well tolerated thus far, favorable safety profile at both doses. And we really haven't seen, you know, I think maybe what you're hinting at is some other folks in the space have seen some issues. We really haven't seen that. So we're very confident that what we're moving forward is safe.

Whitney Ejim, Analyst — Center Court

Okay. And the study, the data so far in the ongoing expansion cohort in adults, you're also running a natural history study in pediatric patients. So I guess what was the rationale from that study and what are you learning so far?

Speaker 1

Yeah. So when we started, there really wasn't a whole lot of data out there in pediatrics. And so we thought that there was a need there to look at that population. and really, you know, the younger the age of the symptom onset, the higher the complications later on. So it's really, can you catch these patients early? So I thought that that was, you know, a good thing to look into and we are. And yes, we think that there could be a possibility that ultimately we study this in pediatric down the road.

Whitney Ejim, Analyst — Center Court

Okay. And you're planning to engage with regulators to figure out the path forward. Is it both in adults and peds or should we be thinking about the ongoing conversations that don't focus?

Speaker 1

Yeah, I think both are on the table, and those conversations are iterative. So, you know, and we're undergoing those right now as we speak. And so, you know, we're going to have an update in Q4, you know, but I think the expectation is we'll have some pieces of the puzzle put together. Will it be this, you know, all-encompassing one shot? Maybe, maybe not. But, you know, these are ongoing conversations that we're having with regulators.

Whitney Ejim, Analyst — Center Court

And which regulators? Is that mostly a U.S. discussion?

Speaker 1

Both the U.S. and Europe.

Whitney Ejim, Analyst — Center Court

Okay, perfect. And I guess probably you might say TBD, but I'll ask it anyway. How might the pivotal path of pediatric patients look different than adults? Well, we'll start there.

Speaker 1

Yeah. Yes, so answer is TBD for sure. But, again, we think the unmet needs high in the pediatric space, and so we think that we could go maybe faster there. That's part of the discussion we're having with regulators, just given that there really are no effective treatment options for pediatrics in this space. And so, yeah, we'd like to look at that for sure. Okay.

Whitney Ejim, Analyst — Center Court

And would the endpoints be different there, or is that kind of what the natural history studies inform it?

Speaker 1

Right, right. And that also is part of these discussions. So we'll have to see. Yeah, we'll have to see how that plays out.

Whitney Ejim, Analyst — Center Court

Okay, perfect. And as I recall, the plan has always been to move forward in peds, hence this natural history study. So first of all, correct me if I'm wrong. But second of all, what did regulators want to see or why haven't you been dosing pediatric patients as yet?

Speaker 1

Well, I think it's just, you know, from a safety perspective, right, you want to make sure it's good in adults before you're going to move. into pediatrics. I really just think that's it. And we feel like, you know, what we've shown so far has been favorable. And so would think that would make sense to move in to PEDS as a next step.

Whitney Ejim, Analyst — Center Court

Okay, got it. And maybe talking about the competitive landscape, you mentioned nothing for the pediatric patients. What does it look like in adults? And how should people, investors, be thinking about 201 competing in that landscape?

Speaker 1

Yeah, we think there's space. I mean, if you if you think of the opportunity of approximately 120,000 patients in the U.S., you know, as I mentioned, there's space there. And so, you know, we feel like there's some unmet need and we can play with existing therapies out there and especially in the pediatric area where there really is nothing. So it's small, but it's not ultra rare disease at 120,000 patients in the U.S.

Whitney Ejim, Analyst — Center Court

Okay, got it. All right, I think we'll switch over. I could ask more, but I'll switch over to TN-401, which is the secondary therapy program targeting PTP2-associated ARVC. Same line of questioning here. So can you briefly talk about the disease, the patient population, and the key MND?

Speaker 1

Sure, sure. So start out with patient population, about 70,000, 75,000 patients in the U.S., so slightly smaller. And this is really just around the electrical signaling of the heart, right? So, you know, 90% of these patients have over 500 of these PVCs, these premature ventricular contractions per day. And if, you know, if you've ever had a couple, you're like, what's going on with my heart? So these are, you know, serious, serious complication. And so we feel like if we can regulate and reduce those PVCs, it's going to then lead to better outcomes and prevent, you know, events down the road that you don't want, including, you know, sudden cardiac death. So that's really been our target so far.

Whitney Ejim, Analyst — Center Court

Okay, got it. And can you briefly review the clinical data there as well? Kind of what have you seen? What are the key endpoints that we all should be focused on from an efficacy perspective?

Speaker 1

Sure, sure. So first, starting out safety clean, still, you know, clean safety profile there. And really, we are looking to see what is the impact on electrical instability, and so measuring PVC reductions. And what we've seen in all patients is about a 60% to 67% reduction in PVCs, and with that's out to 52 weeks in some patients. So again, you know, seeing those reductions and also comparing to, you know, what some others have done, we feel that that our data is very favorable it's you know small number of patients but but very impressive so far uh perfect and on that pvc production i think it's an average of 64 reduction um uh for six patients that you've talked about so is that what are you hearing from patients is that clinically meaningful or how should we all be putting that into context yeah so what we hear and you know what kales have said is about a 50 reduction is meaningful Um, and by doing that, you're also cutting your odds of having a future VA event in half as well. So, um, we, we like how our data stacks up against that and, um, and think that, you know, hopefully future data can repeat, but, um, it's a, it's a good start.

Whitney Ejim, Analyst — Center Court

Okay. And you mentioned the competitive landscape. There are, um, two other GDP competitors out there doing similar things, I guess.

Speaker 1

So how do you think the data compares so far between what you, uh, against what you seen from others and how might 401 differentiate yeah so i think we've seen greater reductions in pavcs versus others and again clean safety profile so um you know it'll all kind of play out here in terms of you know who the winner is i don't necessarily think there's only going to be one winner space for for one winner but you look at our data even out to 52 weeks and you know in in In Q4, we'll have a little bit more durability data that we'll bring across for 401, as well as a regulatory update there. We feel like we're in a good spot.

Whitney Ejim, Analyst — Center Court

Okay. And is there a similar pediatric angle here that could be faster or shorter as 201?

Speaker 1

Could be, although I think we would probably focus more on adults, but it's still in play, and those discussions are still ongoing. Okay, perfect.

Whitney Ejim, Analyst — Center Court

Excellent. All right. I think I'm going to switch over to TN301. And honestly, it's been a while since we've thought about this program. We've talked about it. So can you remind us of the mechanism? This is the small molecule, as you mentioned earlier. Why are we hearing about it again? Why is it not really interesting?

Speaker 1

Yeah, right. So this is the HDAC6 inhibitor that we have. And we've run phase one. We've had exciting preclinical data. I think, you know, there was a prioritization choice early on to focus on the gene therapies, you know, just given resources that we had at the time. There's been some activity kind of in the broader space around not only HDAC6 or HDACs, but just heart failure and kind of these indications that we're looking at as well. And so, So, you know, we're kind of reintroducing it, I guess you could say, and we really are encouraged by the preclinical and the phase one data that we've seen thus far and, you know, are looking forward to thinking about indications and what types of studies we might want to run with this. And again, that's another Q4 event for us.

Whitney Ejim, Analyst — Center Court

Okay, got it. And I guess, yeah, what did you learn from the preclinical and the phase one study? I think the phase one was in 72 patients. Yeah, key learnings.

Speaker 1

Yeah, I think three things. One, you know, safe, well-tolerated. Two, we're hitting the target. And then three, the PKPD is implying probably a daily dosing. So I think those three things were very important for us and, you know, would make us feel really good about moving into phase two.

Whitney Ejim, Analyst — Center Court

And you mentioned this, but there was a time when it was that data came out and you said, okay, great, we're going to be, you know, We'll be focusing on partnerships. We're going to focus on gene therapy internally. I guess you talked a little bit about what has changed. Can you get more specific there? Is it data that's come out of the space? Is it progress with other similar molecules or just maybe more internal data that's generated? Help people get confident in the choice to bring it back.

Speaker 1

Yeah, I think it's internal data conviction. Also, there's been some external validation with Jovinistat and DMD. you know and I think just in general more excitement even from the investment community around okay for a very minimal investment or moderate investment there could be a very large opportunity here in heart failure for example so I think just more of a general you know swell of enthusiasm behind it okay got it and so what work is ongoing now internally that will help set Yeah, so we're doing phase two enabling work, including talks, and including phase two design. So, again, figuring out indications, studies, size, cost, and the good thing is that's more in our control versus waiting for a regulatory alignment with the FDA, for example. So our plan is to come out in Q4 and unveil those plans and then start phase two in the second half of next year. Okay, got it.

Whitney Ejim, Analyst — Center Court

And on the indication selection point, you've talked about HEFPEF and D&D as potential indications. I guess two different, very different indications. So I guess why did you throw out those two and are those the two that we should be thinking about? Like, well, could both move forward? Were those just examples and it could be something else?

Speaker 1

Yeah, those two definitely are the ones that internally we have studied the most and pursued the most and have the most knowledge about and conviction. But there could be others, as you mentioned. The HDAC6 inhibition, I think, can apply to others. And so we'll take a look at that. And, you know, we're listening to the community of where maybe it might make sense to go. So, but definitely HFPAF and DMD are the, at least the first two that are on our minds right now.

Whitney Ejim, Analyst — Center Court

Okay. And could, could you move, would you start two phase two studies or is it really going to be choosing?

Speaker 1

That's my, it depends answer. And as you know, CFO, I would love to have, you know, and give my head of R&D all the money he wants to do what he wants with these exciting molecules. So we're looking at that. So what I would say is we're looking at potentially multiple indications, maybe multiple different types of studies, quick, maybe your more traditional development path. So all that to say, we'll have some ideas in Q4 about what we could run.

Whitney Ejim, Analyst — Center Court

I'll wait for Q4.

Speaker 1

It's going to be a big quarter.

Whitney Ejim, Analyst — Center Court

Just last quick one. There was an HDAC6 deal done by Novartis maybe a couple years ago. Have there been any updates, or is there any evolution of that program that's kind of informing you or reinvigorating?

Speaker 1

We're interested to see how that goes is what I would say. Obviously, very smart people over there. But, yes, we're watching closely, and based on their card flip, sure, it could inform which direction we go.

Whitney Ejim, Analyst — Center Court

Excellent. So on the all-important topic of cash then, which will inform a lot of this, you ended the second quarter with about $78 million about a year, I believe. So how should investors be thinking about the prioritization, particularly now as 301 is kind of coming back to the next?

Speaker 1

So that's right. $78 million cash through Q3 of next year. But these pivotal studies, phase two studies I'm talking about, are not contemplated in that runway. So we're looking to get this regulatory clarity, number one, to determine, all right, what do the pivotal studies look like for 2141? What are the phase two plans for 301? And then based on that capital need, you know, we'll likely come back to investors and ask them to help us finance that. We're looking at non-dilutive capital options as well. I'm taking a close look, obviously, internally at our spend. But really, it's going to play out here, again, in Q4 to get that clarity on shape and size of pivotal studies and how much does that cost.

Whitney Ejim, Analyst — Center Court

Okay. And I guess, is there a scenario where, thinking specifically, I guess, of any of the programs, thinking specifically about 301, but you do the phase two enabling work, you figure out the path forward, and then, I guess the question is, are you still evaluating partnerships in there? Is that still a potential option there, or are you now very focused on doing phase two in turn?

Speaker 1

Yeah, I think we'd like to execute phase two internally on 301, but then I think given the potential large indications that it could go into, partnerships probably make sense, both from a financial and then just a capability perspective of running phase threes with thousands of patients in them, we would be open to that.

Whitney Ejim, Analyst — Center Court

Okay, got it. So it's more about generating phase two to maybe get to a different and next level kind of valuation inflection point, but still be interested in the partnership.

Speaker 1

Yes, I'd agree with that.

Whitney Ejim, Analyst — Center Court

And then I guess assuming both positive data, we've seen both gene therapy programs, assuming those both move forward as well, if you were to be in a position to have to kind of prioritize one or the other, how would you do that? What would you be thinking about?

Speaker 1

So now you're going to make me pick my favorite child, right? Which, no, I think we all have one and they know who it is, right? But I really do think, you know, I hate the it depends answer, but we're going to look at, from a capital deployment perspective, what makes the most sense? What are the, you know, I'd say highest probability bets, but, you know, this is a tough business we're in. But we need to look at that. And then also, you know, when are the next milestones coming for these programs? You know, quite honestly, it's hard to ask investors to wait three years before you turn a card over on a phase two, for example, right? So we would look at both timing of data, quantum of spend, and then where we think we have the biggest bang for the buck. We'd love to fund it all, right? And maybe we can. But it's also going to depend on what those pivotal study designs do ultimately look like.

Whitney Ejim, Analyst — Center Court

Okay, perfect. Speaking of non-dilutive capital, you recently, fairly recently, announced a collaboration with Onylam that brought in a $10 million upfront payment. Can you discuss, how did that partnership come together? Honestly, it caught, I think, myself and some other people off guard a little bit. So, yeah, how did it come together? What was the strategic rationale? And I guess, what are you guys doing? What are they doing?

Speaker 1

Yeah, so this is all about identifying, you know, novel genetic targets for cardiology indications. They are, it's been going great. I mean, in terms of a collaboration perspective, they are reimbursing us for our research capabilities. As you mentioned, we have the $10 million upfront, over a billion dollars of potential milestones down the road. And really, they get access to our cardio expertise, and then they get a license to develop and commercialize these down the road. But it's really been a great enabler, I think, for us and our research organization. And yeah, my head of research is thrilled with the partnership. this far.

Whitney Ejim, Analyst — Center Court

Totally. I mean, it's great validation of the platform. I mean, that everybody's heard of Lnylam in the cardio space, no less, looking at you guys. So can you talk a little bit more about the internal capabilities or what is the secret sauce that you all have that Lnylam was interested in?

Speaker 1

Yeah, I think it's our identification of the targets and, you know, the deep knowledge and expertise in this space that our research folks have. And so I think they identified that as something that was attractive to them. And, you know, obviously the idea is together we move faster down the road.

Whitney Ejim, Analyst — Center Court

Okay. And I guess, are there, is, should we be thinking about potential for additional collaborations kind of with that same thought in mind? Or was that kind of, you know, the one that you were pursuing?

Speaker 1

Yeah. I mean, this one certainly made sense. I think we're always looking at things, you know, for whatever could make sense um you know non-dilutive capital sure it's good but you're giving up something in value um at some point and you know so uh i don't go on blindly um there but um you know we have a a very a strong bd organization and team and we're always looking at things okay got it all right uh we covered a lot of ground anything else that i didn't ask you or that investors aren't asking you that you wish I think, and we've talked about this a little bit today, but I think there are really two categories where one is this regulatory clarity on 201-401 that we'll be making progress on here in Q4. We'll give an update. I think that hopefully will take a lot of uncertainty out of the ultimate pivotal path for the programs. And then number two, we owe the clarity on the phase two and our plans there for 301. And we're already kind of sensing renewed excitement around that program. And so, as I mentioned, that's in our control, and we'll come out in Q4 with that as well. So kind of two sides of the coin there. But the good news is we don't have to wait that long, and we'll have some hopefully good and exciting news to bring across.

Whitney Ejim, Analyst — Center Court

Okay, awesome. And, yeah, I should ask, early for Q?

Speaker 1

Stick with that.

Whitney Ejim, Analyst — Center Court

Well, thank you so much for taking the time today. This has been super helpful.

Speaker 1

Thank you, Whitney.

Whitney Ejim, Analyst — Center Court

Thanks, everyone, for listening.