Sanbio Co Aktie

Hello, everyone. This is Keita Mori from SanBio. Thank you, everyone, for your interest in SanBio and coming to this update meeting. Today, before I present anything, I'd like to just spend a few moments to talk about. Actually, we are extremely delighted that at the end of last year, December of 2025, we have gotten the our company first approval on our cell therapy product. This name used to be a development code SB623. Now the product name is AKUUGO has been approved in Japan for the patient who has disability -- motor disability from TBI.

So this year, we will be able to start commercializing our product, AKUUGO. And this is after 25 years of rigorous efforts by everyone involved. And I'd like to also thank all the stakeholders who has been helping out and also have interest to our company. So thank you very much.

I'd like to also introduce two new executive members to our company, which is also extremely great news for us. This time, the two new executive officers will be building up or continuing to build up the one area is the production. This is Tetsuya Isono. And then another area is regulatory affairs and quality assurance, quality compliance. This is Soyoku Nobeyama. Let me introduce briefly exceptionally incredible two officers. Mr. Isono comes to SanBio with over 30 years of pharma experiences, biopharma experiences at the Chugai Pharmaceuticals. As you know, Chugai Pharmaceutical is one of the leading global biopharma companies, and they have a lot of successful biologics product such as Actemra and other products. And Mr. Isono has been instrumental in developing product development and production development and also the actual manufacturing of these biopharmaceuticals over the years and has contributed tremendously to the success of Chugai Pharmaceuticals. We are welcoming Mr. Isono, and he's going to be bringing all his expertise to the new challenge of a cell therapy.

A little bit about the Mr. Nobeyama. He comes from a few -- several global pharma companies such as Johnson & Johnson, Biogen and most recently, CSL. And through his experiences, he has been successfully launching -- launched approximately 10 new products and has a track record of getting approvals and getting products launched in many new areas. Mr. Nobeyama is also bringing very valuable experiences to SanBio, and he's also looking forward to creating a new field of regenerative medicine.

So going to the next slide, I already mentioned about the -- our AKUUGO approval. Let me just add a little more details to clarify the current status. Actually, we got the approval initially in 2024, July of 2024. However, this approval came with some conditions, and there was some restrictions for the shipment of our product. And after 2024, we have worked and conducted additional manufacturing campaigns and build up more data and satisfied the regulatory agencies' requirements.

And thereby, in December of 2025, we got approval on the partial change. And this now gives us the ability to actually ship products to the hospitals to be used for patients. And so based on this December 2025 approval, this has now become a real approval.

So now I'd like to start the sort of the usual part of the update. The first section is the financials. First is our income statement. And as you can see, we used R&D expense of about JPY 2.6 billion, and this was primarily consisting of costs related to our activities aimed at obtaining marketing approval of AKUUGO. And this level has been about the same as the previous year.

So next, I'd like to go over our balance sheet. And I'm very pleased to update that we have much more cash on hand than the previous several years. In fact, at the end of January 31, 2026, we had the cash and equivalent of about JPY 15 billion. And this was enabled by the finance that we conducted in the last 6 months. We raised a total of JPY 16 billion, and we are very pleased because we are now going to be able to invest in our company's next level growth.

Now going into the financial forecast. This current fiscal year, we plan on using more money than the previous years. We will be spending money of about JPY 4.1 billion for the R&D expense. And as an overall total operating expense, we plan on using about JPY 5.6 billion. And what we will be doing is the manufacturing, distribution and the sales activities to promote the AKUUGO. Also, in addition, our activities will include obtaining more data so that we will be positioned very well for the upcoming full approval.

So going from the financials, I'd like to just touch base on our sort of midterm strategy. As we indicated in the past, these are the 3 things that we are focusing in the next several years. One is Japan, that's our home base. And the second is the U.S. And the third is the new indication of the stroke. And we believe that by tackling these 3 pillars in the next several years, we believe that we will be becoming a global leader in regenerative medicine.

Now I'd like to turn over to Naoki, who is going to talk about our very active activities in Japan as a home base. So please, Naoki.

Thank you, Keita. So in my turn, let me first talk about the importance of Japan market as homebase and also how it relates to the expansion of SanBio business in the future. As Keita explained, SanBio obtained the partial change approval to ship AKUUGO to the market in December 2025. We anticipate the NHI price listing in May this year and the first shipment and administration to the patient will be in the second half of this year.

Japan will serve as a key hub for SanBio's future growth. First and foremost, it will function as a research and development hub. We haven't had our own lab since we closed our lab in the U.S. in 2023. However, we will open new lab for future R&D at Mitsui Link Lab Shinkiba 3 in this April. With this lab, we will promote R&D through the high-quality data to go into the future business.

The next priority is to establish a system to cover the entire process from AKUUGO manufacturing to patient administration. It is essential to establish a workflow for transporting allogenic cells to medical institutions, preparing the cells at institutions and admin administrating to patients via stereotactic brain surgery. We believe this will also help in facilitating rapid market launch in the U.S. in the future. We need to learn how to do the cell business. That's the important thing.

Naturally, we will -- while we are providing information on proper usage, we will collect real-world data to the Japan market and which we can apply to the future clinical trials.

Now let me introduce the market activities we have carried out so far. This year, in order to raise awareness of AKUUGO among health care professionals, we have hosted sponsor seminars at major medical conferences. Additionally, following the enactment of the Act on Support for Persons with Higher Brain Dysfunction, we held a media roundtable aimed at raising awareness of TBI.

We also plan to hold national launch event in AKUUGO for neurosurgeons and similars for Media as a PR event.

This chart illustrates the regional health care collaboration involved in AKUUGO treatment. AKUUGO patients are in chronic phase and typically do not receive regular care even at nearby medical facilities. However, since AKUUGO must be administered at the major university hospitals or large institutions, it is essential to establish a system for referring patients from clinics to these hospitals. Additionally, rehab is required after administration, so we think it is very important to establish this entire workflow to ensure administration of AKUUGO to proper patients who have motor paralysis resulting from chronic TBI. Now let's give the slide back to Mr. Keita. Keita?

Yes. Thank you very much. As Naoki mentioned, we, SanBio as a company who's gotten the world's first approval on the brain regeneration drug. We are in the process of getting a lot of knowledge and know-how and expertise in this new field. And we will be applying our strengths and go beyond.

And our approach, one is the United States and the other area is the stroke. First, I'd like to talk about the -- our initiative in the United States. And the United States is actually the place where we started this company. This was back in 2001 in California. This was where we started where at the time, we thought the center of our innovation.

During the over 25 years of activities, we have built a lot of a track record in the United States. This includes conducting 3 clinical studies in cell therapy, having over 80, 80 clinical centers indeed throughout the United States. We have also been working very rigorously with the FDA. In the -- in our journey, we have been successful in getting our RMAT, which is a Regenerative Medicine Advanced Therapy designation. This gives us a priority review and provides us a chance to get approval earlier than -- by this designation.

And also, we have been very successful in the academic peer-reviewed journal. In 2024, our TBI clinical results, we were able to publish in the top Journal of Neurology. And all of this, we have a lot of the network of strong network, and we believe that we will be accelerating our development in the United States and get the approval and deliver the therapy to our patients. So based on this, most recently, we have gotten from the FDA the agreement on the clinical trial design for our Phase III study for traumatic brain injury. We are very pleased with this progress.

So I'd like to again sort of talk about sort of where our product pipeline is using this matrix. So Japan TBI, we already have gotten approval. In the United States, we have agreement with the FDA on the Phase III clinical trial design. Going down the matrix, let me talk about the ischemic stroke. So this one, we, for us, this is a very important indication, big indication, very important for many patients. And we plan on discussing about this ischemic stroke clinical trial with the FDA within this fiscal year. So please stay tuned.

For other areas, we are right now trying to determine the good timing to really activate our activities. But right now, our focus is TBI, stroke and Japan and the U.S. So I already talked about the stroke in the last slide. But here, I'd like to also just mention that we have conducted already two clinical trials in ischemic stroke. And we have learned a lot from these two studies and a post-hoc analysis of these studies have provided great insights for the next clinical trial.

So our next is ischemic stroke, but that's not the only programs that we will be going after. We will be going beyond. And these include hemorrhagic stroke, spinal cord injury or diseases of the retina, Parkinson's disease and Alzheimer's is also our big interest because this still presents severe conditions for our patients and for our society in all.

So with our strong base as a company who has gotten the world first approval in the brain regeneration drug in Japan, we'll be expanding to the United States, we will be expanding to stroke. And in the 4 years' time frame also, with the launching in the United States and with a substantial progress in stroke, we believe we will be able to sort of grow the company to the next level and really help a lot of the patients. Our vision is to become a global leader in regenerative medicine and really provide a cure to the patients who are in need. So thank you for your attention, and we'll continue and go beyond what we have accomplished to date. Thank you.

Good afternoon. This is Keita Mori from SanBio, and thank you very much for coming to this quarterly update meeting. Today, I have 3 agendas. First agenda is the financial results. The second agenda is the progress in the first half year 6 months. And then thirdly, I will go over future outlook.

So first, financial results, and I would like to explain the consolidated statements of income. So as we are working to get obtaining approval of AKUUGO, this partial change approval, our spending pattern has been quite similar to the last quarters or last year or so. And this 6 months, we spent about JPY 1.8 billion, and this was primarily a component was the expenses related to activities for obtaining a partial change approval AKUUGO.

Now I'd like to move on to the balance sheet. As always, I would like everyone to look at the cash and cash equivalents to ensure the health of -- economic health of our company. We maintain a prudent level of cash and cash equivalents. Specifically, we have JPY 2.7 billion, and this meets our foreseeable short- to medium-term needs.

This time, we revised the consolidated earnings forecast. So I'd like to highlight this to our audience. The primary change is due to an increase in manufacturing-related expenses and the intent here is to secure inventory of AKUUGO at the early stage. So if you could look at this, the operating expense, the original plan or forecast was JPY 3.5 billion and now the revised forecast is at JPY 3.9 billion.

Now I'd like to move on to the sort of the meat of this presentation, the progress of the -- our progress in the last 6 months. Before I dive into the actual specifics of what we accomplished in the last 6 months, I'd like to again briefly introduce what our company is about and what our product AKUUGO is.

SanBio, our company is a regenerative medicine company, and we have over 20 years of history of trying to develop the new field of regenerative medicine. And primarily, we have been focusing on brain diseases. There are 2 reasons. One reason is brain diseases is one of the last frontiers of drug development, and we believe this is an important area for the patients worldwide.

The second reason is brain diseases are set to have no immunorejection, and this allows us and other companies also to implement the regenerative medicine in an allogeneic cell therapy way. And allogeneic cell therapy is a -- we use a standardized product approach, large-scale manufacturing, and this allows us to make our products available to many patients around the world. So we focus on brain diseases.

And after continuous efforts of pushing this regenerative medicine for brain diseases for over 20 years, last year, we finally got the world's first approval for this product AKUUGO. I'd like to also highlight that this product is the first ever -- the world's first ever therapeutic agent for regeneration or regenerating brain. Our team is very proud that we have been able to do this in this very important field and before any competitors around the world.

So last July in 2024, we got an approval for this new product AKUUGO. I'd like to spend maybe just a little bit more time what this product does. And the patient population, target population is a chronic TBI, traumatic brain injury patients. Typically, these patients have more automobile or motorcycle or car accidents and resulting in a severe injury into their head and brain.

And this results in patients' inability to move their arms or hands or legs, resulting in disability in like walking or daily living activities. And our product indication is the improvement of motor paralysis associated with the TBI.

So we'd like to really help many patients improve their motor paralysis and get back their living once again. So I'd like to move on to the next page. AKUUGO, as I mentioned, is approved for TBI at this point, and it's very important. But for the patients who are waiting, TBI is the beginning.

As a company, we intend to expand the disease areas to diseases like stroke, spinal cord injury or retinal disease or even to Parkinson's disease or Alzheimer's disease. We believe this AKUUGO has a big potential, and we like to make sure that we expand the use of this product in the future as near future as possible.

So going to the next page, I just want to set the background of our company in AKUUGO. And last July, it was a very, I think, important approval. However, we were left with certain homework from the regulatory agency. And this homework was for us to conduct some runs of the manufacturing runs and submit the additional manufacturing data to the regulatory agency. This was the homework before we are allowed to ship our products to hospitals for doctors and patients to use.

So in the last year, we -- our team has been working rigorously and conducting the manufacturing runs. We conducted 3 commercial manufacturing runs and of the 3, we succeeded in 2 commercial manufacturing runs. And based on the homework, we submitted this data in June of this year.

So the regulatory agency is reviewing right now, and our anticipation is that the approval for this partial change will be coming in the second half fiscal year of this year, so between August of this year and January of the next year. So this is one of the accomplishments that we had in the last 6 months. We completed filing to the Japanese agency of the partial change for AKUUGO. The second, I would say, quite substantial accomplishment we got in the 6 months is the United States initiative.

So as I mentioned in the last quarterly meeting, we started interacting with the FDA, the regulatory agency in the United States to discuss the Phase III clinical trial for TBI in the United States. And very recently, if you could look at this red box, we were successfully, we reached an agreement with the FDA on the Phase III clinical trial design.

We are very excited about this as we -- as this is a very important progress towards the Phase III, and we're very, very happy about this. We will be continuing to discuss the details with the FDA to conduct this Phase III clinical trial. So far, I've mentioned about 2 quite substantial progresses we had in this 6 months.

I'd like to move on to the -- yet the third progress we had in the last 6 months, and this is related with the financial situation. We always want to keep a financial stability or financial health of the company in order to innovate and bring innovative products to the patients. And in the last 6 months or so, we secured 3 commitment lines from major, major banks in Japan.

We secured JPY 1 billion commitment line from Resona Bank in March and then another JPY 1 billion from Mizuho Bank in June and yet another JPY 1 billion from Mitsubishi, MUFG Bank in July of this year. And this gives us additional stability and the health of the finance to our company. So this marks the 3 major progresses of this 6 months, and our team is very proud of what we accomplished in this 6 months.

Now based on this progress, I'd like to now move on to the future outlook. We have the mission to become the global leader in regenerative medicine, and I'd like to set the path how we get there. So in our growth strategy in the next 4 years or so, we are focusing on 3 pillars. One is Japan, and we consider Japan as a home base and Japan is an important starting point for expansion because Japan has the most innovative and proactive regulatory framework of regenerative medicine.

The second pillar is obviously the biggest health care market in the United States. We always had the activities in the U.S. and operations and the network. We are restarting or we restarted the U.S. clinical initiatives.

And the third pillar is ischemic stroke. TBI is an important indication, but ischemic stroke is consist with many more patients and ischemic stroke is where we started the company's initiative with, and we have lots of inquiries from patients all over the world, and we owe to the patients to make sure that we engage with ischemic stroke and get this program going eventually to the approval and commercialization.

So in the next few minutes, I'd like to dive into somewhat more details of how and what the path we have in the future. But before this, I'd like to sort of step back and share with -- share with you audience our strengths in the United States. So in the United States, actually, United States is the place where we started SanBio. This was back in 2001.

And since then, we have had a lot of activities in the United States. And the original idea back then was we found many innovative technologies in Japan, and we were looking for place -- best place to develop and commercialize. And at the time, United States was the best place and that's how we started in the United States. I'd like to just highlight since this is a busy slide, I'd like to highlight a few things.

One is in this slide, you can see that we conducted 3 clinical trials in the United States, 2 clinical trials in ischemic stroke from 2011 to 2015 and another one 2016 to 2018. And the number of patients we treated is 18 in the first study and 163 patients in the second study. And this made us work with 65 clinical centers throughout the United States, California, New York, East Coast, West Coast, Midwest, pretty much all the major areas in the United States, we have done clinical trials.

Also, we did, of course, TBI study in the United States. This was between 2016 and 2019. And the number of patients was 63 patients, and we operated 21 hospitals in the United States. So we have a lot of track record of conducting clinical trials. And we are by far the company who have done the regenerative medicine for brain regeneration. Over 200 patients is by far the most number.

I'd like to also mention 3 more things just quickly. We had the recognition from the funding agency. You can see 2017, we got $20 million from California Institute for Regenerative Medicine as a research grant. This is a really, really big brand.

The second recognition came from the regulatory agency. In 2019, we got the RMAT, which stands for Regenerative Medicine Advanced Therapy designation. This is a priority review, et cetera. This only goes to promising and solid programs in regenerative medicine.

And the third recognition is came from the academia. In 2022, we were selected to present our clinical study results at the American Academy of Neurology at the Plenary Session. So this is a special session which our PI stood up at the podium in front of thousands of audiences wanting to hear the latest clinical outcome.

So we have a lot of recognitions in the United States and a lot of the clinical experiences, including our network with key opinion leaders and all the practitioners throughout the United States. So I guess that's probably enough for today to go our strength in the United States.

So next, I'd like to go over the road map for us in this near future. So here, we present the Japan initiative at the top and U.S. initiative. I'd like to go from the Japan TBI first. So from left to right, we already applied for the partial change. And so this is already done. And then this half -- this 6 months, we estimate that we will be obtaining approval for this partial exchange.

And then in the first half of the next year, we anticipate getting a drug price listing and also the sales -- product sales will begin something we anticipate. And ultimately, our goal is to obtain the official approval and accelerate the commercial activities. Next, ischemic stroke in Japan. So what we estimate as the road map is the first half of next year, we estimate we will be beginning discussions with the PMDA. And ultimately, our goal is to expand the indications into this ischemic stroke.

Now moving down to U.S. TBI. So in the last -- I mean, in the first half of this fiscal year, we already resumed or restarted the discussions with the FDA regarding the Phase III clinical trial. And in this 6 months, our goal was to reach an agreement with the FDA on the Phase III clinical trial. Even though this is still early part of this 6 months, we actually already obtained this agreement from the FDA, and we are very happy about this, as I mentioned.

And ultimately, our goal is to commercialize in the United States, TBI as well as stroke in the United States. Now I would like to just highlight the near-term sort of the events or projected events in the next 12 months. So the left side is this 6 months and then the right-hand side is the following 6 months, which is the first 6 months of the next year.

So Japan, this 6 months, we project obtaining the approval for the partial exchange. And then in the next 6 months, we anticipate the NIH drug price listing and product sales. For stroke, we estimate beginning of the discussions with the PMDA. And then going to the lower part of the slide in the United States. As I mentioned, this we already agreed. We already got an agreement from the FDA on the clinical trial study design.

And then in the next 6 months of next year, we will be conducting a preparation for TBI clinical trial. I hope this gives sort of what to look for the people who are cautiously or curiously watching and looking at our company SanBio. And finally, I would like to sort of present more of the midterm view of our company to our audience, we have a big vision to become the global leader in regenerative medicine.

We have done quite a substantial work getting the approval of the world's first ever brain regeneration drug, but we still think this is the beginning. We consider us as on the left-hand side, the green sphere. This is our current status, we believe. And we are about to launch our first TBI Japan product, and this is where we are today.

And moving to the next stage, which we consider this current fiscal year to the next fiscal year we anticipate getting to the next phase. And this includes launching our product in Japan, seeing the patients happy, restart the TBI in the United States and the preparation for stroke. And this would give us, I think, a quite growth for our company, and we would like to get there very quickly.

And then if you look at the right-hand side in the pink sphere, this is the image that we have in the 4-year time frame or so. And in this time frame, we anticipate launching our product in the U.S. We also anticipate a substantial progress in the stroke initiative. And at this point, we will be looking at many patients that total of over 10 million patients spanning from TBI to stroke and spanning from Japan to United States.

I think by that time, we should be able to ready to start so many patients, and we really look forward to getting there very quickly and healthy patients. And lastly, our mission is -- we say this again and again, we would like to really create a new field of medicine, and that is the regenerative medicine. We have many patients waiting for innovative medicine products.

And to do so, we put ourselves with the vision of becoming a global leader in regenerative medicine. Thank you, as always, for your attention, and thank you for your continued support. Thank you.