Welcome to Herantis Pharma 2024 results webinar. In this webinar, we will also cover a business update, and we will discuss the very exciting year that we have ahead of us in 2025. During the webinar, you may ask questions, and the webinar ends with a Q&A session. Please be very welcome to listen to us. My name is Antti Vuolanto. I'm the CEO of Herantis, and together with me, I have CFO Tone Kvåle. Let's start with the necessary forward-looking statements. Not going to read them through, but then a short summary of Herantis. As a reminder, we are a clinical stage public company listed at Helsinki First North. We develop a novel type of therapeutics to address the unmet clinical need in Parkinson's disease and other CNS disorders.
Our lead compound that we called HER-096 is a disease-modifying peptide that mimics the active side of a protein called CDNF, which is a neurotrophic factor, which has been shown to reverse the cycle of Parkinson's disease pathogenesis. We already have clinical data from phase 1a and ongoing phase 1b studies where HER-096 is administered subcutaneously. We have shown already a very good safety profile in healthy volunteers, and also what is really important for us is the efficient brain penetration. We are continuing the phase 1b trial with treatment of Parkinson's patients. I will come into those details a bit later. What makes Herantis unique is the efficacy data that we already have.
Of course, we have great preclinical efficacy data with HER-096, but as we already have experience with the mother molecule, the neurotrophic factor CDNF, which has the same mechanism of action as HER-096. With CDNF, we conducted a phase I clinical study where we showed biological responses and clinical responses in advanced Parkinson's patients. That places us very uniquely among the competition in disease modification in Parkinson's disease. As an ambition as a company, we want to engage with a partner based on the phase IB results that we expect to report later this year and to engage with the partner before the phase II so that the partner will participate in the phase II program. We already have very strong interest from potential partners. A bit of background in HER-096. We believe that HER-096 is actually an ideal candidate for Parkinson's disease modification.
You will see in the middle of the slide that HER-096 mimics the active side of the CDNF protein. HER-096 is a synthetic molecule, easy to manufacture, and what is the main benefit compared to the CDNF protein is that HER-096 penetrates the blood-brain barrier, so we can use patient-friendly administration, subcutaneous administration to deliver the activity into the brain tissue. What we expect from HER-096 in Parkinson's disease treatment, what we have seen in preclinical settings, we actually have a robust symptomatic improvement, but of course, eventually we want to have a long-term effect, so disease modification, slowing down or even stopping the progression of the neuron degeneration and the disease. With this very differentiated mechanism that we have, we have also many opportunities in combination therapies and also in other indications. Why is Parkinson's disease such an important target?
There is an unmet clinical need currently in Parkinson's disease. There are therapies that can treat the symptoms, but nothing currently can modify the disease progression. There is nothing that can slow down or stop the progression of the disease. Even with the current symptomatic treatments, definitely not all of the patients benefit from those treatments. There is a large number of patients that are untreated even in the early stage of the disease. As the disease progresses, eventually the current symptomatic treatments are not enough, and the quality of life of the patients gets worse. How we do this, how we do the slowing or stopping the Parkinson's disease. Basically no one knows what triggers the degeneration of the dopaminergic neurons in the target area in the brain.
However, we know that the neurons suffer from many different characteristics that make the neurons suffer. There is endoplasmic reticulum stress, meaning an imbalance of proteostasis. There is accumulation of toxic alpha-synuclein aggregates, and there is general neuroinflammation in the surrounding glial cell tissue. Actually, these form a vicious cycle. Basically, each of these components strengthen each other, and eventually this results in decreased dopamine secretion and movement disorder. Once this cycle continues, eventually these neurons die. With HER-096, we can rescue the function of these neurons. The target pathway is called unfolded protein response pathway, and HER-096 actually addresses that pathway both at the target neurons but also in the surrounding glial cell tissue. Normalizing, stabilizing the function of both of these cell types, which means results in increased dopamine secretion and normalized motor function.
This we have shown very robustly in preclinical settings. Today, I'm not going to go into those details at all. Let's go into last year's reporting. First, the business highlights. On the financial side, we were able to get the second tranche of the European Innovation Council Accelerator grant financing, altogether EUR 750,000. Maybe the biggest news during the year was that the Michael J. Fox Foundation and Parkinson's UK decided to finance the phase 1b clinical study or trial of HER-096. We got EUR 3.6 million that covers the cost of phase 1b and some ongoing biomarker projects. This is, of course, extremely important external validation for Herantis as we were selected among a large number of applicants for this program. We also had a new option rights program that was decided by the board.
What is really important for the scientific progress is that the structural rationale of HER-096 was published in one of the best journals called Nature Communications. Extremely happy about that. Last year we set up the phase 1b clinical trial, got the approval, and we actually were able to successfully complete the first part of that study, and the second part is currently ongoing. We continued this rapid progress in the first two months of 2025. The second part of the phase 1b study started in January. The first time HER-096 was used for treating Parkinson's patients is now in the Part 2 of the phase 1b study. We also reported encouraging pharmacokinetic data from the Part 1 of the phase 1b. This provides us insights to how to design a phase two clinical study.
Of course, very important that we were able to raise EUR 5.2 million in a direct share issue with strong support from the existing investors and also a selected number of new investors. Now Tone will provide us insight into last year's figures.
Yes. As you can see here is three boxes. On the left side, you had the other operating income. For us, this means grant from European Innovation Council. That is booked here both in 2024 and also part of it, part of 2023 consists of that. On top of that, we had a one-off loan payment from Business Finland last year of EUR 4.5 million. That is the reason why the other operating income was so high in 2023 compared to what we have this year. For the operating expenses, this reflects the progress we have had during the year of phase 1b, the clinical trial we're running there, and also the ongoing biomarker project. On the right side of the slide, you have the profit and loss. Last year, we had the positive results there.
That's basically what I mentioned regarding the wave of the Business Finland loan. Next slide. We move into the cash flow and the balance sheet. For the cash securities during the year, we ended up including securities EUR 2.1 million in cash position compared to last year. It was a higher amount. You can also see the cash flow, which reflects the operation cost of that year. The equity for 2024 ended just below zero, so negatively compared to EUR 4.7 million positively last year. As you know, we raised money, new fresh cash in the beginning of February 2025 of EUR 5.2 million. That improved the equity significantly.
Okay. Thank you, Tone. Let's go then to the business update. Basically, we are currently running the phase 1b clinical study or trial. We expect to have the top line data by or during Q3 of 2025. As mentioned, we already completed a Part A in which we dosed HER-096 in elderly healthy individuals, in eight individuals, 300 milligram dose. The purpose of this study was to complete the pharmacokinetic profile of HER-096 in CSF that we started in phase 1a, and now we complete it in phase 1b. Of course, confirming the safety profile that we already established at the phase 1a clinical trial. Now we are running the Part B of the phase 1b. There we dose HER-096 in Parkinson's patients. Each of the patients will get eight injections during four weeks, either 200 milligrams, 300 milligrams HER-096 or a placebo.
We first run the 200 milligram cohort and then the 300 milligram cohort. As mentioned, this is a placebo-controlled study. The outcome measures, definitely safety and tolerability of the repeated dosing, are very important towards the phase two planning. Of course, pharmacokinetics, again, important outcome for the phase two planning. We have a rather comprehensive biomarker program in phase 1b. I will provide you with the details of that. We also assess symptoms both with using clinical scale and a wearable device that monitors the movements of the patients continuously. Related to the biomarker plan, we have different purposes why we collect biomarkers and what we want to establish in this phase 1b trial. First of all, we want to study the patients, their type of disease. We study the alpha-synuclein presence in CSF.
We do genetic testing so we can identify some genes potentially that are associated with Parkinson's disease. Actually, the fluid biomarkers is in a way a key part here. We study these biomarkers both in plasma and CSF, cerebrospinal fluid, some of them also from urine. There are different analytes and purposes why we take these samples. On a high level, we want to see changes which are known to be related to Parkinson's disease, for example, mitochondrial damage or lysosomal dysfunction. We want to find target pathway-related markers, for example, from extracellular vesicles that provides us a tool to actually get biomarkers from plasma that are originated from the brain tissue. We do novel biomarker discovery. Also very important is the digital biomarker, the wearable device.
In this measurement, if we have a potential symptomatic effect in the very short study, there is a possibility to observe even that in phase 1b trial setting. What is the clinical data about the CSF or the brain penetration of HER-096? This slide contains a combined dataset from the phase 1a and phase 1b clinical studies, the phase 1b Part 1. In the figure, you see the red dots represent one individual in the phase 1a clinical study, and the blue ones are the individuals in phase 1b study. As you can see, we have a very nice and clear CSF exposure profile that demonstrates that we are actually within the pharmaceutically active concentrations. The predicted elimination of HER-096 from the CNS is somewhere around 48 hours that supports our previous hypothesis that HER-096 should be administered every two or three days.
Extremely happy about the results, which provides us a tool to start designing the phase two clinical study. The key advantages of HER-096, we have a neurorestorative compound that really can slow down or even reverse the damage that we have in Parkinson's patients' brain. Easy route of administration, and we already have a nice safety profile. I already mentioned about the research funding that we received last year from the Michael J. Fox Foundation, Parkinson's UK I want to highlight that together with the support from the EIC Fund, we have raised over EUR 6 million non-dilutive research funding during the past two years. Of course, very important for a company like Herantis Pharma. We also have secured EUR 15 million investment commitment from the European Investment Bank, sorry, from which we have now raised EUR 3.2 million in two previous financing rounds.
Again, an important support for Herantis, which also allows us to exploit that vehicle going forward if we choose to raise additional financing, for example, for phase two. What kind of business opportunity do we have in Parkinson's disease? Based on several market researches that are available, the market size for Parkinson's disease drugs will be more than EUR 10 billion, $10 billion once the first disease-modifying therapies will enter the market. Maybe by 2030 or around that time. Based on our own market research with very conservative estimates, we believe that at least 20% of that market could be very well covered by HER-096. We have a multi-billion market opportunity in only Parkinson's disease.
Why I say only in Parkinson's disease is that the same mechanism of action that HER-096 has been already shown to be relevant with the CDNF molecule in ALS, in Alzheimer's disease, and some ischemic diseases. Basically, we have much, much larger market opportunity. Of course, the ambition of the company is to first demonstrate that in Parkinson's disease and then continue with other indications. Currently, we are running the phase 1b clinical trial. We are preparing for the phase two trial. Of course, once we have the phase 1b readout, top line data Q3, and the full dataset with all the biomarkers by the end of the year, that allows us then to design the phase two study. For the phase two, the primary objective is to show clinical benefit of HER-096.
At the same time, we are really actively seeking out licensing or collaboration with Big Pharma. The partnering status, basically during the past two years, we have met around more than 50 pharma companies. We have signed confidentiality agreements with 10 companies so far. We have got really clear feedback from the pharma what can trigger a partnering agreement. There, the positive phase 1b clinical trial data is, of course, the key. We have the biomarker data both from the clinical study and preclinical studies that are accumulating on a continuous basis, the pharmacokinetic data that is also coming from the phase 1b. What is also important is that we will have a readiness to launch a phase two program. We need to plan the phase two and plan it in such a way that Herantis can run it.
All this together will most likely put us in a very favorable position during the second half of 2025. This is really an exciting year for Herantis. We are really looking forward to get the phase 1b readout and to start the discussions with pharma based on these results. It is just like really great to be in this position only half a year until we have this great value inflection point coming. As a summary, we believe that we are in the forefront of the development of disease-modifying therapies for Parkinson's disease. We already have great clinical data with both HER-096 and the previous CDNF compound sharing the same mechanism of action. We have the exciting phase 1b trial ongoing. We have the readout in September. We are currently preparing for the phase two.
We are in a great position to go forward. I can't wait until we see what are the results and how the year will end with Herantis. I think we are now ready for the Q&A.
Yes. Just continue sending in questions from the system, and we will take it here. First question, Herantis reported encouraging pharmacokinetic data from Part 1 of the phase 1b clinical trial in January. What was so important with this data? You have mentioned it several times today, but if you can kind of concrete say what was so important for this data.
Basically, as mentioned, it's key for us to design a phase two study that really addresses the question of the clinical efficacy. There, the pharmacokinetics and pharmacodynamics is the key to design how we administer HER-096 in terms of what is the dose level in terms of milligrams and how often we dose the patients, two or three times per week. It really seems that all the assumptions that we have made with the preclinical data is very nicely translated into clinical data. We have a very good insight now how to design the phase two study in terms of these practical administration-related items. Of course, we need to design what are the endpoints, and that requires quite a bit of additional intelligence to be able to do a design that can show our primary purpose of the study.
Yeah. Good. Can you explain what is the main objective with the Part 2 of the phase 1b clinical trial? Are you on track to report the top line data in Q3 2025?
The Part 2 of the phase 1b trial, now we have for the first time we administer HER-096 into Parkinson's patients, two dose cohorts, and placebo control. Of course, the main purpose is safety. For us, very important to establish the biomarker studies and get good outcome from there, again, something that we can exploit in phase two. Those are the main objectives. Of course, we will observe also the symptoms of the patients, but we need to keep in mind that one month treatment is very short considering the target indication. Let's see how the data then pans out.
Yeah. Next question. You raised EUR 5.2 million in a very difficult funding market in February this year. How long is your cash runway?
Maybe you will answer that.
Maybe I will answer it. We are really happy that we were able to raise this amount of cash in a very difficult funding market, as you all know. We are so grateful to our investors who share our vision for HER-096. With this funding secure, our cash runway takes us to or into Q2 2026, and that's far beyond the data readout for the phase 1b clinical trial. Next question is, let me see here. There are so many new questions. When did the first Parkinson's patient receive a dose of HER-096 in the phase 1b study?
That's a question that I cannot actually answer very accurately. We know that it was, was it the 24th or 23rd of January when the second part of the phase 1b study started. As it's placebo controlled, we don't know whether the first patient got placebo or HER-096. First patients have already received HER-096 because we have several patients currently under administration.
Next one. Are your phase I results translatable for other indications in addition to Parkinson's? Can you or are you planning to partner for phase II trials also for other neurodegeneration diseases, for example, Alzheimer's, based on your current phase IA data?
That's a very good question. Basically, the safety data that we have collected from the phase 1a and now in phase 1b, that will be applicable also for other indications. Potentially, also some of the biomarker work can be exploited, especially if you're talking about the target mechanism or maybe changes in mitochondrial function or lysosomal functions. Yes, it's really much, in a way, taking forward also the opportunity with other indications. About the potential licensing or partnering, it's a bit premature to say. Of course, we need to wait until we have the phase 1b data, then we start negotiations with pharma.
It really depends on what kind of terms we might have on the table, depending on the price tag and additional value of these other indications, which is, of course, something that it's beyond what I can comment right now because it's not yet concrete.
Next one is kind of the same topic. Which is more likely to be next disease, ALS or Alzheimer's?
Again, speculation for which I don't have the answer right now. We are looking at these different indications from several angles, the scientific angle, but also commercial angle. We need to also consider what is the appetite from potential partners and how the situation looks like in the autumn and when we have resources to actually invest heavily in other indications, which we haven't done yet.
Yeah. Next one. It is nice to see the support from Michael J. Fox and Parkinson's UK. Can you provide a bit more color on the involvement from these external groups' collaboration? Do they provide any support beyond funding, such as insight into navigating the regulatory side of things in Parkinson's disease?
Again, very good question. First of all, the consortium of Michael J. Fox and Parkinson's UK, they have provided only support for a couple of companies so far. We have been selected among very few ones that have received that kind of support. That has included also very thorough due diligence on the science. Now, during this project or phase 1b study, we actually have follow-up calls with both of these parties on a regular basis. We discuss about the progress. We discuss about the data. We get their insight as they are maybe the best experts for Parkinson's disease during this program. We also hear what else they are doing in the field, for example, towards the regulatory processes. They have said that their ambition is that they can support Herantis and HER-096 to become a true treatment.
They have a true incentive to help us as much as they can. I'm really happy about the collaboration and look forward to continuing that during this year and beyond.
Good. Next. Given the historical biomarkers or the history, biomarkers have been challenging in Parkinson's disease. How do you see the sentiment of regulators towards biomarkers and their use in latest state clinical trial? Would the consideration of phase two and beyond come down to Herantis' plans for a phase two, or would this be the responsibility of a potential partner?
Yeah. If you look at the if you think about the biomarkers, there are multiple different objectives for the biomarker work. We are currently primarily concentrating on demonstrating that, yes, HER-096 affects the target pathway so that there is a biological response. We also want to learn other changes that HER-096, how it affects different processes. Primarily, currently, it is like collection of that kind of evidence. We are not currently looking at biomarkers that we could use in later stage clinical development as outcome measures. That is a big challenge, definitely. As I mentioned, our objective is to have a fairly quick symptomatic improvement with HER-096. Currently, at least, based on the preclinical data and the data that we have with the CDNF program, is that we might have a very nice opportunity to really address the symptomatic side.
Actually, potentially, we would not need to have a biomarker endpoint, regulatory endpoint for approval. Of course, this is something that we need to address together with the partner in phase two and phase three to see how that path goes and, of course, depending on the findings. Of course, a key milestone is during the second half of this year when we get the results from phase 1b.
Yeah. I think the last question coming up. Why should investors invest in Herantis?
Yeah. That's a good question. If you look at our position currently, we are six months from a major value inflection time point. If the data pans out to be good, there is huge market and there is actually huge unmet clinical need. If we look at the Parkinson's disease modification space currently, there are not too many companies out there who are as advanced as we are. Basically, we have actually a true window of opportunity here in the coming 6-12 months. It's a great possibility, I would say. Of course, it comes with the risk.
Okay. I think that ends the Q&A session.
All right. Thank you for listening to us today. It was really a pleasure to be here. Great questions. I hope that we were able to successfully address those ones. We hope that you will continue following us and share the enthusiasm that we have for this year. Thank you.
Thank you.