iBio, Inc. (IBIO) November 10, 2026 Conference Transcript

Thanks, everyone, again, for joining us for this year's Guggenheim Healthcare Innovation Conference on day two here today. Vamo Devon, one of the biopharm analysts here from Guggenheim, joined on the Guggenheim side up here on stage by Daniel Krizay from the team. And next in this room, very pleased to have the iBio team. Next to myself, I have Martin Brenner, the CEO and CSO, and Felipe Duran, who's the CFO. So thanks so much for joining us again. You were here last year, 12 months ago. It's been a pretty productive time for the company. In the prior 12 months, maybe you can just give an overview for those less familiar with the company on the iBio story and the progress you've made over these last 12 months, including becoming a clinical stage company now.

Perfect. Felipe, do you want to take that?

Thank you, Martin. So you hit it on the nose, right? We became a clinical stage company this year, and I think that's the major milestone. We took myostatin into the clinic. We've completed the SAD study, and we're planning to read that out in the first quarter of next calendar year. The other things that we've done over the last 12 months is we've advanced our portfolio, right? Active in E is now basically getting ready to get approved in Australia. And we hope to be dosing patients in the first quarter of next year. Our 800 molecule is way into CMC and toxicology. And we're planning to file that in Australia sometime in the summer of next year. And also, we've made strides on our Amlin molecule, right? We've mentioned in the past that we're the only company that can interrogate a Dacra, Sarah, R1 receptor, and R3 receptor. And the only reason we've been able to do that is we've also advanced our platform, right? Now we've got GPCR technology, we've got cryo-EM technology, and we're able to then interrogate these molecules further and actually not go blind, right, Right, when we're developing and discovering these assets. And then lastly, we've strengthened our balance sheet, right? In January of last year, we bought Frazier in a pipe. And that allows us to now extend our cash runway, which gets us into the second quarter 2028 calendar year. We have multiple inflection points, and I know you'll ask that in a little bit, but we've got multiple inflection points that that cash can drive.

Okay, all right, great. So thanks for the overview, and obviously a lot of programs to kind of go through this morning. So maybe let's start on the 610, the active in E. You do present some additional non-human primate data, EASD, or these are key takeaways from that data presentation, and how do they inform what you're looking to do on the clinical side?

So our iVirus 610 program is an active in E antibody, so we're still, to this day, the only company with an antibody against the target. There's obviously liver knockdown pursued by multiple companies right now, but we feel like with an antibody we have a few points of differentiation against those. We have started the non-human primate study because we were not convinced that the mouse data would translate into humans, and at that time there was no human data. And so what we found in the monkey study was actually that the monkey model is quite translational. So that was the first finding. The body composition data was a little anticlimactic because it's exactly reproducing what we see in humans now, which is good. What we've shown two weeks, three weeks ago at EASD, the European Diabetes Meeting, is that if you combine GLP-1... And aggressively lose weight in these monkeys and you add an active knee on top of that, you basically shift your body weight loss from 10-20% muscle to almost exclusively fat. So this weight loss almost exclusively came out of fat and obviously we want to retain muscle, we want to retain bone density. The one tissue that makes us all sick is visceral fat and fat tissue in general and that's what this is actually reducing. Now, that data set is not complete. This Saturday, we're gonna show body weight data, which is, we ran this monkey study all the way out to 28 weeks, and that's gonna be. It's enlightening because of the mechanism, and it explains a little bit better what this mechanism does. I think a lot of people get confused with Active N-E, it is not a GLP-1, it is not reducing your food intake, it is actually directly working on fat tissue, which takes a lot more time to actually have an effect. But there's also this massive benefit that not a lot of people need to lose 40% of their body weight, and they probably should not take a GLP-1 or, if you will, in the hardest In this case, a reddit rutide, they might actually be much, much better off with something that slowly reduces fat mass and actually retains muscle completely. And that monkey study should be eye-opening, and hopefully it will go very nicely with the clinical phase two data we're expecting from Arrowhead and WAVE over the next few quarters. So that's going to be really interesting how these studies in species actually compare.

I think you mentioned you're on track now to file in Australia by year-end, first in human data, first half of next year. Maybe you can just share with us what we should expect from the study, what should it look like, and then when we get to that interim, so what is going to be the key things to look at?

Yeah, so first of all, I know there's always this pressure in obesity to show nearly phase three level endpoints on obesity. I would just want to highlight that phase 1a is a safety study and a PK study. This is a long-acting antibody. We intend to dose this very infrequently. So the most important thing is to correlate the dose we give, the inhibition of the pathway we see, because that's going to be an important differentiator for an antibody. We have shown we can block this pathway completely, not just about 85%. And the third thing obviously will be we're going to take body composition images. And we have enrolled this time in our first study in my stat, and we took patients that were just overweight. This time we're really taking obese patients in, so we will do body composition scans. But the study is not long enough to really show body weight loss. We already know that, but we want to see are we going directionally in the right direction with this antibody. But most importantly, it's really a safety and a PK study with the added benefit that we see early on signs of efficacy.

Okay, and you mentioned Arrowhead and Wave. They showed some data this year. What did that... What did you learn from what they shared, especially in terms of this relationship, as you talked about, between visceral fat, total weight loss, and how is it impacting how you're thinking about developing your workout?

Yeah, so I think it is very important if you have a new mechanism. If you're the first one going in the clinic, you're kind of blind. You don't know what you don't know yet, and so we're eternally grateful to wave an arrowhead to kind of pave a little bit of a path. We've learned specifically from Arrowhead that type 2 diabetic patients that are obese and are typically resistant to GLP-1 treatment probably have a much better response to GLP-1 if you add an active and E. Say you can double the weight loss in a type 2 diabetic. That is really, really meaningful. The mechanism also might play a role in can we influence how the progression of type 2 diabetes happens. Most type 2 diabetics, if they actually get to a certain age, will have complications like diabetic foot ulcers, diabetic nephropathy, retinopathy, neuropathy. Can we actually turn the clock back and prevent that they ever actually get these diseases? So this would be a disease-modifying treatment for type 2 diabetes, which would be really important. But we've also learned that liver fat reduction is a really, really massive improvement that we're seeing when you block active N-E in humans. So that's another area where we're looking at very carefully, is this going to play out as something as a mesh or in other, you know, larger indications, dyslipidemia drugs that can be used for that. The one thing we're still missing is on the cardiovascular side, because if you look at the genetics of active N-E, you see that there's not only protection against metabolic disease, there's also protection against cardiovascular disease. So our plans for the monkey study and for the human trials are to really look also into exploratory biomarkers. Can we actually see if a population is specifically benefiting from active N-E that is obese and, for example, has HEF-PEF? As you might know, half-pef patients are, to a very, very large majority, all obese. And it doesn't seem that obesity drives the disease. It seems that adiposity drives the disease. Adiposity is really kind of how much fat do you have around your heart that is highly inflamed is adding cytokines to the heart that actually acutely disturbs heart function and then chronically likely leads to half-pef. So there's a strong correlation. But we have to establish first kind of a biomarker relationship before we actually can then plan to enroll or enrich the cohorts with patients that have comorbidities.

Okay, maybe last one on the 610 program, just, you know, I think a lot of people think about how do you see this ultimately from a commercial perspective, positioning-wise? Is this a monotherapy? Is it a combination with incretins? Is it more of a maintenance approach? How do you see this fitting in?

So from a regulatory perspective, we still believe that this can be approved as a monotherapy purely on weight loss because we believe after a year, 5% weight loss is possible. Of course, you would not take a 5% weight loss drug if you have one that lowers your body weight by 30%. We see monotherapy in a very limited population where we feel like they would not benefit from a GLP-1. Say you're frail, you have low muscle mass, you're highly obese, your doctor is really worried about losing more muscle mass because you might fall, break a bone, that might be a monotherapy indication. But we see the biggest benefit in combining this with a GLP-1 because you can either lower the GLP-1 dose to kind of get away from kind of the most severe adverse effects but still have the same floor of weight loss that you would achieve with a high dose. And second, what we truly believe is that the biggest unmet medical need we have in obesity right now is weight maintenance, because we all know people come off of GLP-1, and they cycle on GLP-1, and at least most studies show that if you cycle too often, you lose the effect of GLP-1 to a large degree. So we want to avoid this weight cycling and want to keep patients permanently off that. It's a chronic disease. We will likely have to treat this for the rest of people's lives. And GLP-1s are very, very likely not the right tool for that. So we believe that weight maintenance component, basically keeping that body weight down for good, is something that activity might actually be able to address.

Okay. Let me turn to Daniel.

Yeah, great. So maybe even some of the other programs, starting with 600, the myostatin. So maybe if you can just kind of give a brief overview of that program, where it stands, how the phase one trial is going, and then what data we should be expecting. Yeah.

So iBio600 is a long-acting myostatin and GDF11 inhibitor. We have designed this molecule from the ground up, having the knowledge that others did not have at the time when they designed their myostatin molecules. So we have a few advantages in this molecule. We added GDF11 because GDF11 not only works also on muscle, but it also reduces fat. And we believe this can be a large differentiator. As you know, Regeneron had a combination with myostatin and active NA that might or might not have been, the adverse effects we're seeing there might not have been because of the molecule itself or because of the pathway, but that was leading to a lot of muscle spasms, and so that was not something patients would tolerate. And we have finalized dosing the single ascending dose study. And we went all the way to 10 milligrams per kilogram, so that's one gram of antibody per person, and the adverse effects have been really benign. So we have not unblinded the data, but there was nothing that would concern us in any way or shape. We've also seen that the exposure is very linear to the dose, which is a really good indicator that this antibody behaves really well. And although we cannot yet say what the half-life is, it looks like the half-life is quite significantly extended. That you need this linear elimination phase that happens way back at the end to determine your half-life, and we're not there yet, even after 12 weeks. That's a pretty good indicator that this is actually a half-life extended molecule. We believe with this molecule, we've threaded kind of the needle, we'll find a good common ground, right? We know that myostatin molecules are usually very safe. There's nothing that would kind of tell you that you cannot use this chronically for the rest of people's lives. But the efficacy might be somewhat limited because there's three negative regulators of muscle, myostatin active in A and GDF-11. And myostatin is not very potent on fat tissue, so it doesn't reduce fat that much. So we believe that GDF-11 component helps us. The second component was the long half-life, and that is important because that antibody has to get from the blood compartment into the muscle. And the muscle, as we know, is multiple sheaves of material that the antibody has to get through, and that's hard. So you need to constantly have high levels in blood that antibody will diffuse into muscle. In a regular antibody with a shorter half-life, if your plasma concentration goes down, the gradient collapses. And although you still can measure drug in blood, nothing is arriving in muscle. So having a long half-life where your exposure levels in blood stay up and keep pushing antibody in the muscle, that should actually give us a nice benefit. So first quarter next year, we're going to read out the half-life, the safety data. And although those patients were not obese, we did some DEXA scans for body composition. If that is in any way or shape showing directionality of effect, we're going to present that data Okay, great.

So as you mentioned, there's some other competitors looking at the, you know, anti-myostatin approaches, some looking for lean mass preservation, while others are going more into neuromuscular disorders, like SMA. So I guess maybe if you can discuss your strategy of going after this more lean mass preservation, as opposed to neuromuscular disorders. And you mentioned, obviously, the long half-life. Is that the main differentiator, you believe, for this program? Or is there other things that this molecule can provide?

I think the long half-lap and the dual inhibition are the key differentiators, right? And I think we're grateful to companies like Regeneron and Eli Lilly who move molecules like that forward and also help us pave that path, right? Regeneron is, at least their comment was at EASD, that they're moving Travogromab back into a phase two, but in aged patients. So for the first time, we actually have evidence that myostatin treatment actually improves the quality of muscle. There was always the question, is it increasing muscle strength? That is still debatable, but the quality of muscle, reducing fat inside muscle, that clearly happened now. So kudos to Regeneron to really do a very, very careful imaging approach to that. And what they found, and this was really counterintuitive, actually they had the best effect in aged people with low muscle mass. You would imagine if you have more muscle, you have more myostatin, and you have a better effect. So in younger people, it works better. It actually works better in aged people. So I think that shows you where this is going, right, where you need to lose actually weight, but you cannot afford losing muscle, right, and if you shift this weight loss, specifically in sarcopenic obesity, towards purely coming from fat, and these patients really need to lose fat tissue, but cannot afford to lose muscle, I think that's the right indication for now, and luckily Regeneron will likely go in that trial long before we're in a phase two, so we'll know what endpoints to look for. There will likely be negotiated endpoints with the FDA how to actually approve a drug like this. And so this is going to help dramatically not to be the only ones being in that space.

Okay. Maybe in just a time, we can move to 800, your myostatin, active NA, bispecific. So I guess what drew you to PHF as an indication up front, and why is this approach the right approach for this disease?

I think, you know, almost every indication has a breakthrough drug, if you will. And for PHF, I think Sotardacet was that breakthrough drug. If you look at the development of this molecule from Merck, it literally is a game-changer, a life-changing medication for patients with HEPF. People who couldn't even barely stand up out of a chair can walk for 60 yards now. This is truly life-changing. The problem with Sotardacet was that it causes bleeding, which limits its dose, and it also limits how much or how broad of a population you can enroll. So the risk-benefit factor for PHF is clearly there, but if you broaden the population, there's likely going to be better molecules that are safer. What we did, we actually dissected the pathways that actually we believe contribute to efficacy versus those that contribute to the safety signals. And instead of building a ligand trap where you have to engineer certain things out, and if you follow the space with carers stopping their trial with their ligand trap, It is very, very complicated to engineer these ligand traps. We took the opposite approach. We said, which are the pathways that contribute to efficacy, and just build a multi-specific antibody. Our platform allows us to do these very efficiently and also in a very drug-like manner. So our molecule expresses at 8 to 10 grams per liter, which is honestly at that stage early on beyond what monoclonals actually do, and it's still a bispecific. So you have to keep in mind you need to produce this for a lot of people, which we can. But given that we're not even binding to BMP9 and 10, which are the components that are believed to cause the bleeding risk, we feel like we can build a safer molecule that can be dosed higher, that can be dosed to a much broader population. We're very early. We're in CMC. So CMC looks very good. We're in the middle of doing these safety studies for bleeding and also for erythropoiesis. So adult humans should not make blood cells in the spleen. They should make them in the bone marrow. And one of the adverse effects we're seeing is actually more blood cells being built in the spleen, which, as you can imagine, in patients with disturbed hemodynamics, could lead to a detrimental effect. So that's what we're figuring out right now. And then the last two pieces of evidence is there's animal models for half-pef. None of them is good, but we've chosen probably two that most of the industry is using to show that it's actually efficacious. That data should read out maybe the second quarter and second quarter next year, and that coincides very closely with us submitting that molecule to HREC in Australia, so for approval to start phase one trials. We're very excited about that molecule. It keeps positively surprising us on safety on CMC, and if this keeps going, I think we have a very strong contender.

Okay. Yeah, looking forward to seeing that data. So it also has potential in obesity, I believe, as I mentioned. So I guess why choose PHF-PEF as the lead indication? And are you still definitely thinking of obesity as a later indication? How does that kind of fit in with your other approaches?

So we have early on taken the approach that we didn't want to consider just a single obese population. There is no obese population. There's obese people with half-peft. There's obese people with kidney disease. There's obese people with metabolic disease. So I think this fits in the spectrum. These patients will still be obese, and it will still work on their obesity. But active in A, specifically that component, renders people, men and women, infertile. And so it's not as benign as a myostatin and a GDF11 alone. So, for a broader obese population, it is still at a stage where they think about reproduction. Active in A might not be the right molecule to inhibit, so it would be more limited to an aged population as well. And, obviously, you're highly unlikely to develop half-PEF when you're younger, and so we felt this is a first really good indication. Second is also that we feel the regulatory path for half-PEF is clearly defined by Sotardacet, So we know exactly what to look for. And it's a little bit, you know, no path is easy, but it's a more described path that we can tread on when it comes to regulatory. And it's also going to be a little bit better path when it comes to how do you commercialize a drug like this versus obesity. The question in obesity is still who's going to pay for all these drugs. I think that's one of the biggest things people try to wrap their heads around. If you're being diagnosed with half-pef, there will be somebody who will prescribe a drug and there will be somebody who pays for it.

Okay, so great. So let's maybe move to the AMLIN, which you touched on at the beginning also. So here again, we have some companies ahead of you, and you're entering phase three now, or moved into phase three already. How do you see your approach around AMLIN differentiating from these competitors, and are you deciding sort of what kind of profile to move forward?

Yeah, interesting question. So there's a lot of knockout studies happening, but this is very complex biology, and it's likely not translating very well from rodents to humans. So what we're building is, like Felipe mentioned, we're building four different profiles to basically understand how MLM is actually working, and we are in a prime position to actually find this. These are two molecules. Among those, we will find the one that has the best. Effect versus adverse effect profile, that's the one we're likely going to move forward. So these are antibody-based, and if it comes to amylin development, there's three important components. Component one is the half-life. The longer the half-life, at least from what we've learned up to this day, from a three-times-a-day dosing to now once-a-week dosing, the longer the half-life, the better the effect. Second is what is the receptor profile. There's a total of three, four receptors, if you take it seriously, the calcitonin receptor alone, And then calcitonin receptor with a RAM protein that makes amylin receptors 1, 2, and 3. At the moment, the thinking is you only need amylin receptor 1 and 3, potentially only 3. And that obviously means all of the peptides that also hit calcitonin receptor find a sink. So you need to inject a lot more to get to the same effect size, which also drives adverse effects. So we can create profiles against each of these receptor subclasses and combinations thereof. They will likely go into non-human primates to see in the head-to-head comparison which is the right profile that gives efficacy but stays away from the nausea and vomiting, and that profile is likely the one we're going to move forward. And the last component is the signaling bias, that peptide can signal inside the cell through different pathways. And there's a belief that if you're stimulating one versus the other, you see fewer adverse effects. That's hypothetical, but we have the means to kind of create these molecules to test that hypothesis. So it'll take us until probably mid-next year to find the right profile. And then hopefully mid-next year, we're going to declare a development candidate. And then it's your typical 9 to 12 months in CMC and TOX before we're in the clinic.

Okay, great. So a couple more minutes here. It's just maybe from a big picture, corporate-wise, a lot of programs going on that you're working on. You've talked before about trying to get programs maybe through phase two and then partnering. How are you thinking about that at this point in terms of your overall portfolio partnering strategy and then timing across all these different programs?

I'll hand this over to Filipe because I think it's highly dependent on what we have as cash runway right now.

Yeah, thank you, Martin. So that's a good question, Venmo. The way that we see it is, when we built out this portfolio back, what, two, two and a half years ago, we wanted to look at the obesity white space, right? So we're very well positioned. We feel that we are one of the only obesity companies that have a portfolio that will meet all the strategic objectives for big pharma. We are going to have dual pathways. Once we have phase one data, we'll see if we raise capital for phase two or partner that asset out. But we also want to be good stewards to our shareholders and make sure that we're maximizing shareholder value. How do you maximize shareholder value? You know, most of the time it's post-phase two. So we want to basically look at dual pathways every time, show that these assets are very critical to strategic partners, and either raise capital or partner those assets out. And that non-dilutive would then feed the platform to then drive other assets forward.

Okay. And you mentioned the cash runaway earlier, but just maybe, so what are the key events we should be looking at over the next 12 months across the program? Sure.

So, as I mentioned, we have cash into second quarter 2028 calendar year, that gets us phase one readout for myostatin, gets us phase one readout, interim readout for active and E, it gets us basically interim readout for 800, and allows us to drive ambulance and through CMC and TOCS. So, you know, obviously, we are always looking when we would need to raise, and with that time horizon, we should be raising sometime in the next year.

Okay. All right, great. Thanks so much again for joining us this year. Congrats on all the progress. We'll look forward to keeping track on how things move from here.

Great.

Thank you for having us.