Sanbio Co Aktie

Hello, everyone. This is Keita Mori, Founder and CEO of SanBio. I'm really excited today to bring the news as we have met the largest milestone of our company, which is the launch of our first product, new drug, AKUUGO. So in this slide, you will see cell therapy product aiming for brain regeneration, launch of AKUUGO injection for intracerebral transplantation. So this is what I mentioned at the beginning of my talk. This year, we were able to finally launch this product, AKUUGO in Japan.

The indication is for chronic traumatic brain injury patients and the indication is to improve the motor impairment for these patients. There are many, many traumatic brain injury patients who are suffering with motor disability, unable to conduct daily living of life. And we are -- I think our mission is to really bring this new drug to the patients in a solid manner so that we can help our patients. We have come this far, and thanks again to everyone who's been involved, especially obviously, the patients and families who have decided to participate in our clinical trials that we have done to date.

So along the line of the launch of our product, I'd like to also share with you another highlight of this 6 months. Last month in August, on the 22nd, we had a Japan National Launch Commemorative Seminar for AKUUGO. We were able to -- we are grateful to have had about 100 physicians coming from all over Japan from Kyushu to Hokkaido to everyone everywhere, and we are grateful for the time that they have taken to travel to Tokyo, be in person and get involved in this commemorative seminar.

We had wonderful talks by the leading physicians from Japan. Also, we had a special lecture invited doctor from the United States, Dr. David O. Okonkwo, Professor of Neurosurgery from the University of Pittsburgh. He brought an inspirational talk at this seminar covering 2 things. I mean, for one, he was -- he congratulated the launch of AKUUGO, himself being the leading physician in the TBI clinical trial for AKUUGO. Also, in addition, he shared with the physicians in Japan, the latest news or development from the United States.

That is Dr. Okonkwo's group has worked for many years to try to come up with new biomarkers for acute TBI, and his team has been successful in getting FDA's acceptance to use certain biomarkers for acute TBI. So he's very happy that to present the 2 areas of the big success and especially the launch of AKUUGO, where historically speaking, it has been a very difficult disease to develop a new product against. Dr. Okonkwo is very much looking forward to conduct a Phase III clinical trial in the United States and launch AKUUGO in the United States as soon as possible.

So these are 2 highlights I'd like to sort of bring to your attention. We have done many more accomplishments this past 6 months, and I would like to go over this by myself as well as Naoki Tsukahara in later part of this presentation. I'd like to spend a couple of minutes going over the financials. So this is the consolidated statement of the income. And as you can see, in this past 6 months, we had R&D expense of about JPY 1.2 billion. This was recorded and mainly for manufacturing-related expenses for AKUUGO.

Next, I'd like to go over the balance sheet of our company. We are maintaining a very good level of cash and cash equivalents, which is JPY 12 billion approximately, and this is going to meet our foreseeable short to midterm needs. We believe we are very stable finance-wise. Lastly, I'd like to go over the revision of the consolidated earnings forecast. So in this 6 months, I'd like everyone to look at the left part of this slide, comparing the gray forecast versus the green results. We have some substantial difference in this 6 months.

We had a difference of about JPY 996 million in the last 6 months. And this was due to the timing shift of certain manufacturing-related R&D expenses to the second half of this year. Also, I would like everybody to look at the right-hand side of this slide. We now have for our first time in our forecast, revenue forecast. We have JPY 396 million forecast in this fiscal year. So this is the financial statement update. Now I would like to pass on to Naoki to go over the number of the progresses we had in the first half year. So Tsukahara-san, please.

Okay. Thank you, Keita. Okay. Let me introduce the progress in the first half year at my session. As Keita mentioned already, AKUUGO was listed on the NHI drug price list on May 20 and launched on May 21. This is a really great achievement in the past 25 years since SanBio was founded in 2001. This slide provides basic information on AKUUGO, including its dosage, administration and efficacy. As you may be aware, the NHI price was set at more than JPY 72 million, and this is probably the third highest drug price in Japan as far as I know.

And this is our initiative this fiscal year, and we promote the adoption of AKUUGO focused on ensuring patient access to treatment and establishing a system for stable product supply. Regarding patient access, we are working to introduce AKUUGO at a total of 8 institutions consisting of the 5 original clinical trial sites plus 3 additional institutions. Preparations have now been completed at 4 institutions. Treatment of the first patient is scheduled for the first -- for the next month -- and the first patient was scheduled to treat at next month and we expect 6 patients to be treated during this fiscal year.

For stable supply, we will utilize R-SAT to secure the stability of the product and establish an operational framework that enables product delivery in line with each patient's scheduled treatment. This is the AKUUGO Smart Regional Health Care Collaboration model, which we have introduced to you several times in the past biannual earnings call. It illustrates the pathway of the patients who may not generally be under the care of a neurosurgeon and who are -- then how they are referred to an administration institutions.

Centered around each administrating institutions, we are working to establish these regional networks by holding regional health care collaboration seminars and informational sessions by our sales reps called Area Medical Partners. These are information materials designed to support the establishment of an AKUUGO treatment framework. The materials on the left are intended to raise disease awareness among patients and provide consultation contact and facilitate consultation and referrals. The materials in the center provide physicians and other health care professionals with information about AKUUGO itself, including information on the post-marketing clinical trial.

The materials on the right explains the cell preparation procedures and surgical techniques required for AKUUGO administration. In terms of promotional activities for physicians and other health care professionals, Keita introduced already at the beginning of this presentation that we held a nationwide commemorative launch seminar. In addition to that, we have held regional seminars, cosponsored seminars at major scientific meetings such as the rehab -- Annual Meeting of rehab hospital physicians. And also, we have exhibited at conference booths and we did a survey to the doctors who visited our booth to check their awareness of AKUUGO and also provide the product information to them.

As part of our interest to ensure stable supply, we have entered into a trial manufacturing agreement with JCR Pharmaceuticals in addition to Minaris Advanced Therapies to build a framework for commercial manufacturing. By establishing 2 manufacturing sites, we aim to create a supply system capable of providing the increasing demand of AKUUGO, not only in Japan, but also globally. This is the progress in SB623 development and commercialization for this fiscal year.

The main highlight is its inclusion in the Japanese drug price list and market launch. And also, as we previously reported, we have reached an agreement on the trial design in the U.S. for TBI. Additionally, regarding ischemic strokes, we are currently preparing to begin discussion with the PMDA in Japan during this fiscal year. That concludes my presentation. I'll hand it back to Keita for the next session.

Thank you, Naoki. As Naoki covered, we have achieved a lot of things in the last 6 months. Now I would like to communicate our strategy to become a global leader in regenerative medicine. Our strategy is quite simple. We have a near-term focus of 3 things, 3 pillars, as we say. One of them is Japan. We now already launched our product in Japan, and we will be learning a lot of things. We will be having in-depth experiences through the activities in Japan. This is going to be our home base and provide strength to our global expansion.

So number 1 strategy is Japan. Number 2 is the United States. And as everyone agrees, the United States is by far the most important end market in the biotech innovative products of the biopharmaceuticals. We have already come to an agreement with the FDA on the clinical design of the Phase III clinical trial, and we'll conduct this with much speed. This is the second strategy, U.S. Lastly, the third strategy is ischemic stroke. TBI is a very important indication for us and for the patients.

When it comes to sort of the number of patients, ischemic stroke is lot of patients. There are millions of patients in Japan, United States, China, Europe, everywhere. And we will take AKUUGO and expand the indication to ischemic stroke. So this is our third strategy. So from the top to the bottom, Japan, U.S. and ischemic stroke. Let me talk about the first strategy of Japan. This one, Naoki has already gone into quite details of what we have done and what we are planning to do.

As we have lots of activities in Japan, we will be really increasing our capabilities and experiences, especially we have 2 areas that we think are important. One of them is the secure patient access of AKUUGO through the regional medical collaboration. This is something that we will be doing better and better over the months, and this is going to become one of the strengths. The second area is the whole manufacturing and logistical supply chain systems. So we have already built and we will be getting more and more experiences through this system, and this is going to serve another strength when it comes to our future global expansion.

So now I'd like to sort of map out our pipeline plan in terms of the indications and the territories. And as I mentioned in our strategy being Japan, U.S. and stroke, this is again the same focus. I'd like everyone to look at the left upper corner, which is the Japan/TBI. So in the short term, we will be focusing on initial dosing of the patient, adoption of the patients for AKUUGO in Japan and also post-marketing clinical study. So this is the Japan activities.

The second, the U.S., I would like everyone to look at the yellow box at the top -- so what we are going to do is the next fiscal year, we plan on starting our Phase II -- Phase III clinical trial. And as I mentioned, we have already obtained the agreement from the FDA on the clinical trial design. So this is our second focus. The third area is the yellow box on the right hand. So ischemic stroke/Japan, our team has been working diligently in this area. Clinical trial design agreement with the FDA is planned in the next fiscal year, and we are also preparing for clinical trial initiation. So that's our focus in the near term.

And beyond that, as we go through this, and we will become a global leader. And beyond that, we also plan on expanding indications to dementia or Alzheimer's and also territories to Europe and China, et cetera. In order to achieve our strategy, U.S. continues to be a very important area. And this is something that I have shared with everyone in the past, but I wanted to highlight again our rich and deep experiences and track record in the United States. This spans from running 3 clinical trials about 200 subjects in the United States with about 80 medical centers, including TBI and stroke.

Also, we have done a huge grant obtained $20 million in the past, and we have done very good publications, et cetera, et cetera, in the United States. So with the very strong product AKUUGO and our strengths in Japan as well as in the United States, we foresee a dramatic growth of our business in the near term as well as in the midterm. We see ourselves in the middle at the present. Right now, our value drivers are threefold: Japan AKUUGO adoption for one, Japan. The second is the U.S. TBI. The third one is the ischemic stroke.

And as we pursue these 3 strategies, we will be growing our business. And in the 4 years and beyond, we see ourselves getting AKUUGO adopted for TBI as well as ischemic stroke in Japan and also in the United States. And as Naoki mentioned, we have a planned first dosing of the patient next month, and this is going to be a beginning. SanBio will move and move forward. Thank you.

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.