Herantis Pharma Oyj (HRTIS.HE) Earnings Call Transcript & Summary

Good morning, and welcome to Herantis Pharma's First Half '25 Webinar. I am the CEO, Antti Vuolanto. And with me, I have here CFO, Tone Kvale. Before going into the webinar presentation, just a disclaimer about the forward-looking statements. I won't read them through, but let's continue with the presentation. So Herantis Pharma, we are a clinical stage public company listed at Helsinki First North, and we develop neuro regenerative therapies to stop Parkinson's disease progression. And our lead asset can actually become a game-changing therapy as the first disease-modifying treatment for Parkinson's disease. HER-096 is a small peptide that mimics the CDNF protein that the company has studied earlier. And with the mechanism of action coming from CDNF protein, HER-096 can protect dopamine neurons from further degeneration and also support their functional restoration. And in addition to that, with the peptide, we have efficient brain penetration that enables patient-friendly administration under the skin. We have very convincing set of preclinical data showing that, yes, we actually capture the full activity of CDNF protein into HER-096 in terms of the protection of dopaminergic neurons. We also have advanced nicely in clinical development with HER-096. So we earlier or previously have demonstrated strong data with brain penetration and safety in Phase I settings. We are currently finalizing the Phase Ib clinical trial, expecting the top line data by mid-October this year and the full data set by the end of the year. And of course, we are currently also preparing for Phase II proof-of-concept clinical trial. And as already many times earlier mentioned, the ambition of the company is to engage with a development partner even before Phase II. And of course, the Phase Ib data readout will be a strong catalyst towards that goal. If we go into Parkinson's disease market. So Parkinson's news today has recently estimated that the economic impact or burden of Parkinson's disease globally exceeds USD 250 billion, huge number. And if we look at the therapeutic market, which is currently approximately between USD 5 billion and USD 6 billion, it's projected to grow to around USD 13 billion by 2033. And this growth will be catalyzed by the development of new therapies like HER-096 that can modify the disease, but we also have estimates that the number of Parkinson's patients globally will increase tremendously during the next 25 years. So currently, we have approximately 10 million to 12 million Parkinson's patients globally and by 2050, it's estimated to grow to 25 million patients due to improved diagnostics tools and aging population globally and the awareness of the disease. So we have a disease, which has a huge global burden, but we also have an unmet clinical need. And when Parkinson's disease is diagnosed, it's always based on the appearance of the first symptoms. And actually, at that stage, the disease is already rather advanced, meaning that maybe half of the dopaminergic activity in the affected brain area has been lost. The challenge is that current therapies can only treat symptoms, not the disease itself. So the current therapies are based on a increasing the amount of dopamine in the brain area, but they cannot effect on the disease progression at all. Many patients benefit from the current treatment symptomatically, but there are a big number of patients who actually don't get the right benefit or they have significant side effects. And the effectiveness of the current treatments decline over time. So basically, all Parkinson's patients currently know that there will be the time when the current treatments cannot manage their disease properly. So there is a huge, huge unmet clinical need. And we, of course, want to address that with HER-096. And HER-096 mechanism of action is rather unique. So we are currently the only company in clinical settings, exploiting a pathway called unfolded protein response pathway, which we can modulate with HER-096. And modulating that pathway at the same time in the neurons, the affected neurons and also the surrounding glial cells, so the immune cells, we can reduce cell stress. We can reduce protein misfolding, so the accumulation of toxic alpha-synuclein aggregates, and we also can reduce neuroinflammation. So we can break down the cycle of the pathology in the target tissue. And so we are able to protect the dopamine neurons from further degeneration. And based on preclinical data, we are also able to have some degree of functional restoration. And here, it's also good to remember that this mechanism of action is not specific for Parkinson's disease, and there is compelling evidence with CDNF related to other neurodegenerative diseases and also other indications as well. But in Parkinson's disease treatment, what we are looking at is rather quick symptomatic improvement due to the functional restoration of the affected dopamine neurons and also on the longer-term, disease modification, so stopping -- slowing down or stopping the disease progression, which would be, of course, game-changing for Parkinson's disease treatment. And currently, it seems or we believe that HER-096 will be subcutaneously administered most likely 2 times or from 1 to 3x per week. We do have a very strong and compelling set of evidence for the biology and for HER-096. So with the previous molecule CDNF, we have 15 years of experience studying its effect in Parkinson's disease. In preclinical settings, we have shown regenerative effects. We have shown effects on both motor and nonmotor functions. We have completed a Phase I clinical study with intracranial CDNF demonstrating clinical and biomarker responses in advanced Parkinson's patients. With HER-096 in preclinical settings, we have shown that, yes, we capture the full activity of the CDNF protein. So we are hitting the target pathway. We see the expected biological changes, and that's transformed into protection of the dopaminergic neurons and improved motor functions. We have also demonstrated very efficient brain penetration and clean and nice safety and tolerability data. With earlier Phase Ia, that showed efficient brain penetration in healthy volunteers and good safety profile as well. And now the brain penetration and safety profile is further strengthened in the Phase Ib data that we announced earlier this year related to pharmacodynamics with healthy volunteers, and of course, currently, we are just finalizing the study with Parkinson's patients where we had 2 dosing cohorts, 200 and 300-milligram doses for 4 weeks in early to mid-stage Parkinson's patients. And these 2 cohorts were also placebo-controlled. And as announced last week, the patient treatments are completed, and now we are actively working towards having the top line data by mid-October. So the key advantages of HER-096, which are the right ingredients to bring HER-096 into commercial settings, we have neurorestorative mechanism of action. We have compelling evidence that it works in preclinical settings, and now we are studying this in clinical settings as well. We have patient friendly subcutaneous administration, and so far, the safety profile has been very nice at promoting or enabling us going forward with the clinical development. So let's now jump into business update and looking at what we achieved during the first half of this year. So the objectives for this year were the completion of Phase Ib. And during the first half, we have progressed the program according to the plan, of course, very nice progress there. Then a key objective has been advancing the partnering discussions. And of course, the Phase Ib readout will be a key catalyst to continue with those and have hopefully a conclusion there. And then, of course, the next step will be a Phase II, and the company has been very actively preparing for the Phase II study. Of course, we need to remember that we need to have the Phase Ib results in order to finalize the trial design. So if we look at the business highlights from the first half in January, we reported encouraging pharmacokinetic data from the Part 1 of the ongoing Phase Ib clinical study, and this data help us to design the Phase II trial. We also, in January, started the treatment of the Parkinson's patient cohorts in the Phase Ib. In February, we completed a directed share issue. We raised EUR 5.2 million gross, of course, important milestone and supporting future company development. In May, we announced that we completed the first Parkinson's disease cohort with -- who received 200-milligram doses or placebo in the Phase Ib. And we also further in May, announced that we started the second cohort with 300-milligram doses or placebo in the Phase Ib. And right -- and after the reporting period, last week on August 14, we announced that the last patient visit has been completed in the Phase Ib and now we are actively in the process of analyzing the results during the next 5 to 8 weeks to have the data announced by mid-October. And Tone, if you tell us about -- a little bit about the financial figures.

Yes, thank you. So the majority of our resources are today spent on the Phase Ib clinical development and also the ongoing biomarker project and preparing for the Phase II, as Antti mentioned. You can see the loss has increased compared to the same period last year. We are now minus EUR 3.2 million compared to EUR 2.6 million. Reason for that, you can see on the other side of the slide, the -- we have been running the European Innovation Council Accelerated grant project for more than close to 2.5 years now. And that project ended in April this year. So that's the reason why both the operating expenses related to that and also the income decreased. We see some increase in the payroll and related expenses due to we are hiring more staff and also we had bonus payment in Q1 2025. As you remember, we have the fundraising in February. And in connection with that, we had some finance expenses when we raised that amount of money. So that's the reason why we have increased in the loss. Next slide. The cash position is strengthened compared to the last year period. We raised EUR 5.2 million in February, and we also have received the second installment from the Michael J. Fox Foundation of EUR 0.5 million during this period. So we have now EUR 4.5 million in cash position and that compared to EUR 3.4 million same period last year. Equity is at EUR 1.7 million positively and compared to last year, EUR 2 million. So we have a cash runway into Q2 2026, but we need funding to be able to start the Phase II clinical trial. So as you have been reading, if you read press releases and also the previous presentation, we have seen really strong external validation of our science, especially from leading patient organizations in Europe and also Michael J. Fox in U.S. So EUR 3.6 million was secured last year from Michael J. Fox in Parkinson's U.K. And that is -- they are paying basically for the Phase Ib clinical trial, which is now coming to an end of this year. We also had this EUR 2.5 million grant funding from European Innovation Council has ended now in April. And as you also remember, we had the commitment from the European Innovation Council of direct investment in equity of up to EUR 15 million. It's been utilized. They have participated in the last 2 funding rounds with EUR 3.2 million. So rest of the amount is still left. And they will then participate in every direct equity fundraising up to 1/3 of what we are raising. So that's really, really good for us.

All right. Thank you, Tone, for that financial update. So in this webinar, we have covered pretty much about what is the status of the company and the first half activities and the financial status. So we really are looking forward to see the Phase Ib results and advance HER-096 as a potentially game-changing therapy for Parkinson's disease. But this concludes the webinar or the presentation part, and let's continue with the Q&A session. So my colleague, Jaako, who has been our summer trainee will ask the questions from the audience.

All right. You reported the last patient visit a week ago. Why will it take 6 to 9 weeks until you report the top line data?

That's a very good question. So -- at the time of the last patient visit, also -- there are some samples, biological samples taken from the patient. And of course, those needs to be also analyzed. And then we have a database that -- where we collect all the information, all the data points, all the measurements from each of the patients. And that database can only be closed after we have all the data collected from all the patients, all the visits and then we also need to -- the database has to be quality controlled. And only after that, it can be closed. And the randomization code can be in a way, broken and the data analysis itself can start. And after that, it will take quite some time to analyze the actual data to have in a way, the raw data for Herantis. And after that, we also need to have the right interpretation and conclusion. And the estimation from the last patient visit was that we will be able to release the top line data within 6 to 9 weeks. And compared to industry average, I would say that this is quite short time frame. So of course, we are working towards that time line. And of course, as already mentioned, we are really enthusiastically looking at seeing the data and being able to update the market also what we see in that data.

How are you preparing for Phase II? And what would it look like?

Again, very important question. First of all, we need to remember that we can't finalize Phase II design until we have seen the data, analyze the data with Phase Ib. So the final design is definitely not yet there, and we need to wait until we see the data. But of course, Phase II will be addressing the question what is the clinical benefit of HER-096 in treating Parkinson's patients. So we are looking at a study that can detect the symptomatic improvement caused by HER-096 and also potentially disease modification and also biomarkers caused by -- or the changes caused by HER-096 treatment.

What might be the possible design or structure of the Phase II study?

Right now, I won't start speculating about what is the actual setup. However, most likely, it will be a placebo-controlled study with HER-096.

How long is the patient recruitment expected to take for Phase II? And has it already started?

Of course, as mentioned, we are still in the process of finalizing the Phase II study design, and it will take quite some time to actually then start the clinical operations. And as Tone mentioned, starting a clinical study or collaboration with pharma. So the study hasn't started yet, and there is no patient recruitment started yet. And of course, the company will update the market about the progress in the design phase. And of course, once we are able to start the study, that will be announced separately.

How much is required? And how will you fund the Phase II study? What are your plans to raise additional funding?

Tone, maybe you will address this.

Yes. So we can't comment on the Phase II trial and the cost because the design is not ready yet, and we need to wait for the Phase Ib data until that is ready. But of course, we are exploring different options to secure additional capital to support the next development phase. That could be, for instance, partnering agreement, scientific collaborations, soft funding and equity. But of course, as I mentioned, remember on the previous slide that we have very strong support from European Innovation Council. So they are committed to invest 1/3 of what we are raising externally.

Do the current funders have any plans or interest in continuing to support Phase II if the results are promising?

Yes. At this stage, the company won't start speculating about this.

Could you share anything about potential partners? Are all of them focused on Parkinson's disease? Or are their funders interested in expanding the research into other conditions as well?

Yes. So as mentioned, the Phase Ib data will be a key catalyst for continuation of the partnering discussions and hopefully having a conclusion there as well. Currently, the vast majority of the evidence of the biology is within Parkinson's disease. So that will be the primary focus also in these partnering discussions. However, of course, there have been already discussions about other indications, but I won't start speculating about those here right now.

Can you tell us a bit more about the outcome of the biomarker project? How might shape -- how that might shape the design of the Phase?

That's a very good question. Biomarkers that we have or the biomarker program that we have run for the past years is -- the focus there is to find evidence of HER-096 affecting the target pathways. And of course, Phase Ib readout again will be key in understanding what kind of biomarker changes we see in patients. If we see robust changes, this will help us to design the Phase II, but it also can function as an important data set for partnering discussions.

Is there a potential to expand HER-096 to other neurodegenerative indications? Is this something Herantis may explore? Or will it more likely be picked up once an appropriate partnership is secured?

So Herantis has so far concentrated on Parkinson's disease to have the focus there and create enough evidence so that we can have a meaningful partnering deal covering that. But of course, the company is really looking forward to start development also in other neurodegenerative diseases and maybe also in some other indications, utilizing the same biology, so addressing the unfolded protein response pathway. Of course, that would require resources, and we believe that once HER-096 in Parkinson's disease has a collaboration -- pharma collaboration, we can -- we might be able to start advancing the other indications. Okay. I guess this concludes also the Q&A session. So thank you for the very good questions. And as maybe final words, we are looking at a very interesting autumn. We are really looking forward to see the data and be able to communicate that to the market and to the potential partners. So this will be a very, very nice autumn for us in Herantis, and I wish the same for the audience. So thank you for watching this webinar, and I hope that you will follow the progress of Herantis through the autumn. So thank you.