Hello, and welcome to Abliva Audiocast Teleconference 2022. Throughout the call, all participants will be in a listen-only mode, and afterwards, there will be a question and answer session. Today, I'm pleased to present CEO Ellen Donnelly. Please go ahead with your meeting. Hi. Thanks all for joining us today. I'm Ellen Donnelly. I'm the CEO of Abliva. I'm joined today by our Chief Financial Officer, Catharina Johansson, and our Chief Medical Officer, Magnus Hansson. We're here today to talk about the financing we completed last night, the use of the proceeds in that financing, and our exciting path forward as we work to develop new innovative therapies for patients with PMD. I'm gonna start today with a short presentation, and then we'll open up the floor to questions. If you have questions while I'm speaking, please email them to us at ir@abliva.com, and we'll make sure we address your questions at the end of the presentation. If we can pull the slides, please. I'm just gonna give you a bit of background on Abliva and the financing we just completed. As you can see on the first slide, our goal at Abliva is to target the powerhouse of cells to improve the lives of primary mitochondrial disease patients. The second slide. Abliva is focused on becoming a global leader in mitochondrial medicine. We can do that because we have an experienced team with over 20 years of experience working in mitochondrial research. This is very unique. The mitochondria kind of are thought of as a new therapeutic target. They exist in every cell in your body except your red blood cells, and thus it's been a hard thing to understand how to target until recently. Our team, both our CMO and our CSO, have been working in this area for over 20 years. We started in Lund, Sweden, but have recently expanded and now have a subsidiary located in Boston as well. In our company, we may be small, but we're mighty. We have both research and development capabilities, and we plan to build out a commercial organization to commercialize our lead asset, KL1333. We're also differentiated from other companies in that we are focused solely on primary mitochondrial diseases. These are rare diseases that are devastating. Patients often have reduced life expectancy, and there are no options for them. It's a genetic disease. They get diagnosed, and they're told by their physician, "I'm sorry, you have primary mitochondrial disease, and there's very little I can do for you." It's a hard place to be. We're gonna try to solve that gap. Because we're in the rare disease space, we have a lot of opportunities here. Working in the rare disease provides you opportunities with regulatory authorities, for a more seamless development plan, and we obviously have the opportunity to take commercial advantages as well with the opportunity of premium price. This means that we have blockbuster sales potential for our compounds. As many of you know, we are publicly traded on Nasdaq Sweden, and our stock ticker is ABLI. If we can go to slide three. Here you can see our portfolio of assets. KL1333 is our lead asset. Most of the proceeds raised today will go towards the initiation of the phase II/III study for KL1333. We'll talk more about that in a minute. Proceeds will also go to be used on NV354, our second asset in the portfolio. We also, as indicated here, have some earlier stage programs that are currently in development. Important to note is that KL1333 asset does have orphan drug designation in both the U.S. and Europe, which is good news for us as we develop that compound. On slide four, you can see that today we announced an unprecedented raise. We raised SEK 200 million through a directed share issue and a fully underwritten rights issue. We're excited about this. This really shows a position of strength and support for both our strategy, our portfolio and our team. Right now, the market conditions are suboptimal. It's a very challenging environment, and most companies are only able to raise a small rights issue at a significant discount in order to stay alive. What we have managed to do here is raise a directed share issue and a rights issue over our market cap. We raised SEK 200 million on a market cap of SEK 150 million. We did it at a discount of only 10%, and we brought in a great group of new high-quality life science and institutional investors that are able to commit to Abliva for the long term. This is really important as we think about our portfolio and all the needs we're gonna have. We've also importantly added a rights issue to this so that our current shareholders have an opportunity to participate in this deal as well. We recognize that we have a lot of shareholders that are interested in this company, and we encourage you to come into this rights issue that will be open from June 10th through June 27th. This is an exciting raise for us. The company is now fully financed to a key milestone for our lead asset, KL1333, and we now have cash runway for 24 months, which is a very unique position in this current market environment. On slide five, we can talk a bit more about our lead asset, and we're happy to answer any questions after the presentation on this as well. KL1333 is being developed for adults with primary mitochondrial diseases who suffer from both fatigue and muscle weakness. These patients have a devastating disorder. Many of them can't leave the house. They have trouble getting up. They have trouble finding the energy to go to the grocery store. Many have proximal muscle weakness, so their legs don't work as well as they'd like. They may have problems standing up and sitting down. We are trying to address both of those factors. This asset is a modulator of two coenzymes in your body called NAD+ and NADH. The reason those are important is in patients with mitochondrial disease, the imbalance exists between these two. We are fixing that balance so that these patients' cells have more energy, and thus the patients have more energy. The program has a lot of safety information. We have dosed over 100 patients and healthy volunteers, so we know what this molecule looks like when it's put into patients. That's really important. We also have signals of efficacy from a Phase I-B study that we read out last year, where you can see that KL1333, when dosed for 10 days, does appear to affect both fatigue and muscle weakness. That's after only 10 days of dosing. What we're about to commence on, and the financing round was done for, is to start our Phase II/III registrational study. We'll start that with our first 40 patients. As I mentioned, we did a cohort earlier, that was eight patients. Now we'll do our first 40. They'll be dosed for six to 12 months, and we'll go to a meaningful interim analysis. Well, why should we care about this crazy PMD that I've never heard of? Well, this is very devastating, high unmet need, no therapies, but there's also a big commercial opportunity here for our investors. When we look at the sales potential of KL1333, it exceeds $1 billion in peak annual sales. There is a big opportunity here to address a very high unmet medical need. On slide six, you can see what we're about to do. We're excited to start our global registrational Phase II/III study. The details are all on this slide, and I won't read through them, but we will be dosing our patients for 12 months. They'll get a pill in the morning and a pill at night, and we'll see what KL1333 does to those patients, how it helps their fatigue and their myopathy over that 12-month period. On slide seven, you can see some more details about the interim analysis. I bring this up because this financing has supported these 40 patients to be dosed for six to 12 months, and to read out an interim analysis. This interim analysis is really important because it'll give us our first signals of efficacy and safety information. We'll get that efficacy through a conditional power analysis that will allow us to move seamlessly into our platform design, Part B of the study. Once we do that IA, we'll have a chance to understand the direction that we will move in three ways. We will either find out that we'll continue the study to the planned size of 120 patients total. We'll expand the study to a predefined cap, or the DSMB will come back and say, "I'm sorry, the study doesn't look like it's gonna work. We'll stop the study now." This is extremely important because this milestone comes quite soon. By late 2023 or early 2024, we will have the interim analysis. We will know what this drug looks like in patients, and we will know the safety profile of patients dosed for six to 12 months. That's extremely important for both investors, potential partners, and for our work because if that looks good at that interim analysis, we'll trigger a lot of activities to get us ready for a commercial launch after an NDA approval. That's a little bit about KL1333. We've also announced that our proceeds will be used towards our second program, NV354. This is a complementary program. It will still work for patients with PMD, but with NV354, we're targeting children. This drug is different than KL1333 also because it gets into the brain. This program addresses Leigh syndrome. It's a severe disease with multi-organ deterioration. Many of these patients don't live beyond the age of five. We have an energy replacement therapy here that has the opportunity to start in Leigh syndrome and then move forward. This program finished preclinical development earlier this year. We went to a scientific advice meeting with the MHRA, had a good discussion with them. Now we need some additional activities. We need to make more drug in order to start the Phase I study. We need to continue our regulatory documentation process. [Inaudible]. We'll be translating documents into the foreign languages we need to run the study, and we'll be working to submit our country documentation and have the final regulatory discussions to start this study in late 2022. The team at Abliva is very accomplished and manages to do a lot in a very short time. I think this is the most dedicated and driven team I've ever worked with. We are passionately interested in helping these patients with PMD. On slide 10, you can see a list of the accomplishments last year. It was a great year for us, and we're looking forward to another great year this year with this financing. In 2021, we accomplished a lot. We showed efficacy of our lead compound, KL1333. We completed a drug-drug interaction study. We did two long-term toxicology studies. We validated our primary endpoint, and we engaged our CRO. At the end of the year, we also announced our IND approval. We are busy, and we are excited. We are motivated. We are ready to dose our patients with KL1333. In summary, we're focused on becoming the global leader in mitochondrial medicine. We have an exciting portfolio of compounds with KL1333 and NV354. We have an extremely motivated and dedicated team, found in Sweden and the U.S. We really think we have the opportunity here to do something quite amazing. We just wanted to take the time today to thank our current shareholders, our new shareholders, our new investors, our board of directors, and everyone that helped with this financing. It was not a great time to be raising money, and we couldn't be more happy with what we managed to accomplish. A huge thank you goes out to our entire network of Abliva friends, family, and support network because this was a job well done. Thank you. We'll open it up to questions now. Thank you. If you do wish to ask a question, please press zero one on your telephone keypad. If you wish to withdraw your question, you may do so by pressing zero two to cancel. There'll just be a brief pause while any questions are being registered. Just as a reminder, that was zero one on your telephone keypad if you wish to ask a question. Maybe we can start with one question that I'm seeing come through. You've mentioned that the use of proceeds is gonna be directed at this phase II/III study of KL1333. You spoke about the interim analysis, but not much about the rest of the study. Can you tell us what you'll be doing in that phase II/III study? That's a question from the floor. I'd like to turn this over to Magnus Hansson. Magnus is our Chief Medical Officer at Abliva, and he's focused on our phase II/III study. Magnus, tell us a little bit about that study. Yes, I'm happy to. Thank you, and hi, everybody. The phase II/III study, when we designed this, we really tried to focus on what is most important for the patients. We spoke to patient advocate groups, and we've also done in-depth interviews as part of validating the primary endpoint, one of the primary endpoints in this study. We really have designed this study with the mitochondrial disease patients in mind. We selected what the patients themselves told us limited their daily lives the most. The two most common things that bothers them and really hinders them to do everyday life activities is, one, muscle weakness or like a lack of endurance in the muscles. They very easily get restrained from doing physical activity. Two, a really pervasive sense of fatigue, this lack of energy. That makes sense. I mean, we are focused on mitochondrial medicine, mitochondria being the energy-producing part of the cell. It really makes sense that what the patients feel is this lack of energy and symptoms from the muscles, which requires a lot of energy when they are required to work. We really designed the study, how we include specifically the patients and how we measure efficacy of the compounds based on those two important aspects of disease, so muscle function and general fatigue. Importantly, we have validated for the first time for mitochondrial disease patients a fatigue questionnaire. A scale that the patients will fill in every week on their own device, so an iPhone or a smartphone or a device that we will supply to them. We will be able to follow really how effective our compound is throughout the study and at the same time, at regular intervals, measure their muscle function with a test called sit-to-stand. We ask the patients to, within half a minute, to sit-to-stand as many times as they can and then measure that. That's an endpoint that has been used in several trials before. That's really the key aspect. We believe that this will give us two complementary sets of information. We have discussed with the regulators, so that we can have both of those measurements as primary endpoints of the study. Really that gives us two shots on goal to show the benefit of KL1333. Fantastic. Thanks, Magnus. We have another question here about the terms of the financing. I'm gonna turn this over to Catharina Johansson, our Chief Financial Officer. Catharina, can you give us more details about the terms of the directed share issue and the rights issue? Yes, of course. Happy to meet you all today. The rights issue of SEK 150 million, that was an opportunity to broaden and diversify our shareholder base with new specialist biotech and institutional investors. We were also very happy that our largest shareholder, Hadean Ventures, were able to participate in the directed share issue. The directed share issue was closed yesterday, with a discount of 10%, which is a remarkable discount compared to other companies during these times. We're very happy about that as well. Hadean also decided to convert their loans into shares yesterday. On top of this rights issue, we would like to give our current shareholders an opportunity to also participate. We will launch preferential rights issue of SEK 50 million. If you are a shareholder on June 8, one share would entitle you to one subscription right, and 11 subscription rights will entitle to subscription of four shares. The subscription price in the preferential rights issue will be SEK 0.35, the same price as in the directed issue. The subscription period will be June 10 to June 27. The preferential rights issue is also 100% guaranteed by underwriters. Hopefully, a lot of our current shareholders will take the opportunity to participate in this preferential rights issue. Thank you. Hi. Tell us about that data that we read out? As you know, it was read out last spring, and we were really happy to see the results of that trial. The primary goal of any phase I trial and our trial as well was to see how our compound KL1333, how well it's tolerated in humans, if there is any unexpected safety issues, and how the compound is taken up in the body, and how long it stays around and then excreted. These aspects look really good. We first explore that in normal, healthy volunteers. We wanted, as we want to progress this compound quickly, to get it to patients as soon as possible and in a lean, cost-effective manner, we included a cohort of genetically confirmed primary mitochondrial disease patients. Our target group for KL1333. There, the primary goal was as well to see safety, tolerability, and pharmacokinetics. Those aspects look very similar to healthy volunteers. We also had the opportunity to test some efficacy endpoints to get some initial data. We knew already back then that the most important aspects to study in mitochondrial disease is the muscle function and this pervasive fatigue that these patients suffer from. We had two fatigue scale and the same functional measure that we have in the phase II/III study. Essentially, we show that the actively treated patients, they responded with an improvement, so a less degree of fatigue and an improved ability to perform their functional muscle test compared to the patients that received placebo. An identical pill, but without the active substance. It was a small study with really short duration, but there were really promising signals of efficacy that really has made us confident in going forward with phase II/III study and also with our choice of these important efficacy endpoints, the fatigue and muscle weakness, that we can really be able to show a significant benefit for the patients from this trial design. The phase 1b study was really supportive of the strategy. We have been able to kind of finish the final design of the phase II/III study further. We are really well-positioned with that data in the background and now with the financing of the initial part of this study to really get this going towards approval of the compound. Great. Thanks, Magnus. We have a question about NV354. The question acknowledges that we have communicated that we're gonna use about 5% of the net proceeds to complete the preparation of NV354 for the clinic. The question continues, when that work is finished, how are you gonna take it to the clinic? The plan for NV354, we're super excited about this program. The plan there is to look for additional ways to finance that program in the phase one study. That could include partnering. A partnership obviously has many different ways. It could include a grant, or other sources of revenue. I think there's been a lot of external interest in that program because it is a brain penetrable manipulator of mitochondrial function in the brain. That program has gotten a lot of interest. Still working on the final plans there. Maybe I can turn it over to Magnus to talk a little bit about why he's excited about NV354. Sure. Yeah, I mean, both our compounds, KL1333 and NV354, are really exciting because they have been developed specifically with mitochondrial disease in mind. We have looked at what is causing this disease and found two really important aspects. One being a bit more important in children, and that's where we focus NV354, and the other being more important in the adults with this disease, KL1333. Even though we think that both of these strategies will be complementary. NV354, we focus on children, as I said, with mitochondrial disease, and a typical expression of mitochondrial disease in childhood is called Leigh syndrome. This compound was actually... The whole idea was based on earlier research from our research group here within Abliva, where I was also part of that invention. We really got the idea from looking at cells from patients, from a small child with Leigh syndrome, seeing how the mitochondria didn't work and got an idea, okay, so how can we fix this? We tested that and then developed that into a promising pharmaceutical. As Ellen mentioned, it's brain penetrant, and these children usually suffers from brain damage. We are really excited to also take this compound into clinical trials and really explore how it can benefit mitochondrial disease patients. Okay, we've been talking a lot about the patients and these patients with primary mitochondrial diseases. We just had a question come in about the landscape in this area. Who else is out there? What other competitors do we have? Maybe I can start that. It's interesting, we are kind of in the wave of companies that are at the same stage of development. There are a few other companies in the phase II/III space who are all looking to test their compounds in patients right now. Those compounds tend to split between two different groups. I would say they're the brain penetrant compounds and there are the compounds that aren't brain penetrant. The nice thing about the population of candidates out there is that we're all relatively complementary to each other. If one drug got approved before ours, we could still be used in conjunction with that therapy. There aren't many of us out there developing it, so there's still an opportunity that Abliva could be first to market, which would be very exciting. Also I think one of the opportunities we have here is that in the rare disease space, we are all good friends. We have an upcoming meeting, the annual mitochondrial meeting that's gonna be held next week in Phoenix, Arizona. That will be an opportunity for us all to get together and talk about how we're gonna address this massive medical unmet need. I know Magnus and I are looking forward to attending that meeting some more patients, talking to investigators, and working with the other companies in this space to really make sure we get our therapies to these patients who need them so desperately. It's a neat space to work in. We've spent a lot of time talking to patients, understanding what the patients need, and trying to help them address those needs. I think we all find this to be a very dynamic and passionate place to work, because every day we're reminded by the fact that the patients are waiting for our medicine, and that's personally very, very satisfying to know that we can help them. We're all looking forward to starting this phase II/III study. I think that's our next step, and we're all excited to get this study started and get this drug tested in patients for a year of treatment. Are there any more questions that anyone can see from my team that have come in? No more questions from the IR website. What about the phone? Should we open it up? Are there any questions on the phone line? Yes. We have a question from the line of Jacob McCall from Kempen. Please go ahead. Hi there, and thanks for taking my question. Also congrats on the raise. I was just curious if you could maybe tell us a bit about PMD and how the patients are currently managed, and what is the size of the opportunity maybe here? Great question, Jacob. Thanks for joining us. Magnus, I'll hand that one over to you. The question was how these patients are managed currently, and then I didn't catch the second part. The second part was the size of the opportunity here. You know, how many patients are roughly. Oh, yeah. Yeah, like with this condition. Yeah. These patients are usually taken care of by specialist centers. Most countries have a few or up to a handful of specialist centers where there are geneticists and neurologists, and they're really like a multidisciplinary team, really making sure to optimize a kind of symptomatic treatment for these patients. Because there is really nothing specific that can slow down the disease or really target the underlying pathology of the disease. What can be done is trying to avoid things that we know will worsen the disease, such as I mean, these patients, since they have a lack of energy, they should definitely not fast. They can suffer quite badly if they have a severe infection, so they were really trying to avoid COVID, and of course still are. It's more of a supportive treatment. As we've mentioned in some of our press releases, even though it's a rare disease, when you look at the total numbers. There are about 40,000 adult patients in Europe and U.S. It is a significant number of patients out there that currently have nothing really to be offered to them except maybe some vitamin supplements, et cetera, where there's no proof whatsoever that it has any effect. There is really great opportunity and a huge unmet medical need that we're really looking forward to get rid of that unmet need and really offer these patients hope. Okay, thank you very much. We have just one more question from the line of Dominic Rose from Intron Health. Please go ahead. Hi, this is Dominic. Congratulations on the raise, and thanks for taking my question. I've just got the one. Considering that KL1333 is for an indication with no treatment, I just thought it would be eligible for accelerated approval. That being the case, for this interim readout, is there any possibility that this data could be fileable in the U.S.? Thanks. Great question, Magnus. Yeah, we have discussed at length and repeatedly with the FDA. What they have agreed to is that it's sufficient with one pivotal trial. I mean, usually there is a requirement for first a proof of concept and then two confirmatory trials. They know how challenging it is. They have agreed that we can have a combined phase Ii/III trial, and that could be a registrational, of course, depending on the data. We don't think that we can file on interim data. I mean, we are making sure that we will get good information from that, but at the same time preserving the data integrity of the overall study. There are several other opportunities to get a quick approval, but we think that we will need this trial with robust evidence of efficacy. Okay, thanks. Very helpful. As there are no further questions, I'll hand it back to the speakers. Great. We'll give you one more minute here. Any further questions? There are no further questions on the phone. Okay. Well, thank you everyone for joining us. We really appreciate your support and your interest in Abliva. I hope you can hear our excitement and our passion for what we're doing. We're really, really excited at this point to jump into the Phase II/III study and test KL1333 to see if it has the efficacy, if it makes improvements in fatigue and myopathy to help these patients suffering from primary mitochondrial diseases. We appreciate everyone's support and hope that if you're a current shareholder, you'll participate in the rights issue that's gonna open up in June. Thank you all for your time today. Thanks, Catharina and Magnus, and best wishes for your day. Thank you. This concludes our conference call. Thank you all for attending. You may now disconnect your lines.