SanBio Company Limited (4592) Earnings Call Transcript & Summary

Good afternoon. This is Keita Mori from SanBio. Thank you very much for spending time with us today and eager to listen to our latest update. With SanBio, as most of you probably know, we have been working since the beginning of the company history 2001, brain regeneration. This is something that has been believed to be impossible for over 100 years. But with our persistent work, last year, July of 2024, we were able to obtain the marketing approval for our brain regeneration drug called “AKUUGO. This is the world's first ever brain regeneration new drug. We are very proud that we have come this far, but this is still the beginning. And since the last quarter update meeting, as I mentioned, we have changed the gear to further a much more dramatic expansion of our company, U.S., United States and stroke. So now I'd like to go over the updates in detail. Financial results. Consolidated income statement. So for the last year, we spent JPY 3.5 billion in operating expense, and this is primarily for the activities of the SB623 TBI, and now this is “AKUUGO. Next slide, consolidated balance sheet. Here, at the end of the fiscal year, end of January this year, we had JPY 2.9 billion. And after this fiscal year-end, in fact, on March 3 of this year, we raised additional JPY 2 billion to strengthen our financial situation. Altogether, we do have and maintain a very good level of cash and deposits necessary for our current year activities. Now moving on to the earnings forecast. This year, we expect to spend about JPY 3.5 billion, and this is mainly for obtaining the partial change of the manufacturing and marketing approval for “AKUUGO and all the activities that are associated with the launch of the product. Now I'd like to move on to the second section, the birth of the brain regeneration therapeutics. As I touched base a little bit at the beginning of my talk, it has been really believed that the conventional wisdom is that the brain, an adult human brains do not regenerate. This has been the conventional wisdom for over 100 years. But with our “AKUUGO approval of last year, we have been able to overcome or overturn this conventional wisdom. Now we can say that we can regenerate human brain. So about the “AKUUGO, this is the world's first brain regeneration drug on the market on this planet. And the indication is for TBI patients. And the great thing about “AKUUGO is that “AKUUGO can be applied to patients who are like 6 months after TBI but not only that, it can also be administered -- applied to patients who had TBI, for example, 5 years ago or 10 years ago or even like 20 years ago. So this really gives hope to the patients for the improvement of their motor paralysis or motor function. As I mentioned, our AKUUGO is for TBI, and we do have many, many indications above and beyond TBI. The next in line is ischemic stroke, which we know that a lot of the patients are waiting for, also hemorrhagic stroke. These three indications, we regard as clinical stage indications. In addition, we have in our plan spinal cord injury, macular degeneration, this is an eye disease, retinitis pigmentosa, this is another eye disease, retina disease, Parkinson's disease and Alzheimer's disease and even beyond. We have a lot of the very severe brain diseases or central nervous diseases to tackle. And we believe our first regeneration -- brain regeneration drug, AKUUGO, has a huge potential to help patients suffering with these disease and conditions. As I mentioned that we do now have the AKUUGO approved and now we have shifted a gear upwards, and we are working on three areas. One is Japan as a mother base and the other two areas are driving growth drivers, United States initiatives and ischemic stroke initiatives. By accomplishing these three initiatives, we believe we have a clear path to becoming a global leader in regenerative medicine and most importantly, helping many, many patients in many severe diseases. Now I'd like to turn over to Mr. Tsukahara to talk about the Japan as a mother base.

Okay. Thank you, Keita. Like Keita just talked about SanBio's aim to become a global leader in regenerative medicine, let me explain here our progress establishing Japan as a mother base for SanBio in this part. The first milestone we must highlight was the success of second manufacturing run. As we already announced in the press release on February 6, the second manufacturing run cleared all specification requirements and was deemed compliant. The company already started one more manufacturing run. And according to the schedule, the yield of this run will be available by end of April. After confirming all the specs are cleared, again, we will file a partial change application immediately and will obtain approval to lift the condition of shipping AKUUGO to the market by end of July. Another approach SanBio took in last quarter was the contract manufacturing agreement with JCR Pharma. JCR is a Japanese pharmaceutical company. It has about 10 years history of manufacturing and marketing the first regenerative medicine in Japan called TEMCELL. TEMCELL is the same as AKUUGO in that it is allogeneic made from MSC. We signed a contract with JCR for trial production of AKUUGO, which finally aim to commercial production. With this agreement, SanBio expects to stabilize the supply of AKUUGO when we obtain indication for ischemic stroke and also to enter the U.S. market. AKUUGO needs to be distributed separately from other regular pharmaceutical products because it must be kept under minus 150 degrees Celsius. AKUUGO is consisted by three components: vials containing cells, dedicated preparation solution and dedicated delivery device set. Since these are manufactured separately, a wholesaler will put them together and deliver to a hospital as one product. We developed RSAT system that enables to manage the information from patient registration to product delivery, dosing and follow-up. This chart illustrates the patient flow of AKUUGO. AKUUGO is administrated by neurosurgeons at university hospitals or large key hospitals. However, chronic phase TBI patients with motor paralysis don't usually see a doctor at these hospitals. Some of them go to recovery or chronic care facilities, but others just stay at home without seeing a doctor regularly. So we have to establish a referral system to connect patients with neurosurgeons via local consultation facilities. The first step is to increase disease awareness to see consultation facilities. And after implanting AKUUGO, the patient will receive follow-up on rehab at local rehab facilities. This is TBI Navi, a website that's created as one of the initiatives to increase the disease awareness. Professor of Keio University and President of Japanese Society for Regenerative Medicine, Dr. Okano, gave us a short video clip to explain what is regenerative medicine. Patients can register to receive further information or notice, and once AKUUGO is launched, patients can search a hospital to get implantation of AKUUGO. This is my last slide. SanBio has also made significant progress in R&D. The first one is sub-analysis of Phase II trial of SB623 by Dr. Kawabori from Hokkaido University. This analysis suggested the way to identify the optimal place to inject AKUUGO. The second one is by Dr. Nagase from Okayama University about the effects of SB623 transplantation combined with exercise in a rat cerebral infarction model. And the last one is the presentation at the Japanese Biochemical Society by Dr. Maeda from Keio University about the possibility of SB623 cell for Alzheimer's disease. Now let's turn back to Keita.

Yes. Thank you. So Naoki went over the Japan as a mother base initiative. Now I would like to go over the outlook of our growth drivers. So just as we saw the world market and I'm sure that the audience today is very aware that United States is the #1 important market for the biotech and biopharma industry. About 40% of the sales or revenues come from the United States and more than 50% of the profit comes from U.S. is the general understanding. For us, it's the same or even more important. As you can see, for TBI in the United States, there are over 5 million patients. This is about 100 more patients -- 100x more patients than that of Japan. In addition, in the United States, there are over 6 million ischemic stroke patients, and there are over 1 million hemorrhagic stroke patients. So United States is without question, very important country or the market that we need to serve. In the United States, our activities have been very rigorous. Since our company's founding in 2001 in California, our activity has been most rigorous in the United States. For example, as you can see on the upper right-hand corner, we -- this is from our first clinical trial, and this was done in the United States, Stanford University and the University of Pittsburgh. This is one of the patients who benefited from our SB623 product. She had a stroke, and she was not able to move her arms for 2 years. But after she received SB623, she started being able to raise her arm, use her hands and improve her motor activities. Through this, this patient got so much confidence to get married, to have a baby and now is leading the day-to-day lives that I think she wanted -- always wanted to enjoy. The map, which is on the right-hand corner, describes the clinical sites that we worked with for our TBI study. There were over 20 clinical sites. This included university hospitals such as UCLA, Stanford University and University of Pittsburgh and so forth. For the stroke studies, we had a similar engagement or even more engagement. We had over 60 clinical sites for stroke. So altogether, we have more than 80 clinical sites that we worked with throughout the United States. So as you can see this, we have a rigorous and very rich experiences. I would like to go through very briefly on the table on the left-hand side corner. You can just go through this on understanding the magnitude of our clinical studies. Beyond that, I'd like to highlight that in 2016, we received the Innovation Award 2016 based on our first clinical study. This award is a very prestigious award, which in the past went to a blockbuster type of product like a tPA, the only acute stroke drug on the market. Others, like 2017, we received the largest grant, $20 million, from the California Institute for Regenerative Medicine, which is the largest funding agency for regenerative medicine. And in 2019, we received the RMAT designation. This is called Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration, FDA. This gives us accelerated review, et cetera, so that our product can get early approval through the system. This only is extended to promising regenerative medicine products, needless to say. And finally, in 2022, we -- our presentation of the TBI clinical study results was selected as the plenary session, the special session by the American Academy of Neurology. This attracted a lot of attention for the cutting-edge cell therapy work for TBI patients and chronic TBI patients, where conventionally speaking, no scientists or no doctors had ever imagined about. So we have a very strong base in the United States and our Phase III initiative that we have just launched is based on these strong experiences and expertise and network. Now I'd like to sort of summarize our U.S. activities to date. In the past, we already had consulted with the Food and Drug Administration in 2019 and 2022. And more recently, this is between the last quarterly update meeting 6 months ago and between now, we resumed or we restarted the discussions with the Food and Drug Administration to talk about and discuss the Phase III clinical trials in TBI. In addition, our preparation for the ischemic stroke is also underway. I'd like to also talk about some on the stroke rechallenge. One clear thing is that we learned very important lesson from our Phase IIb study. And after conducting extensive ad hoc or post-hoc analysis, I should say, we have gained lots of insights. In this slide, we are demonstrating two pieces of great insights that we have. By now, we know the optimal patient segment, which we'd like to take forward, patient segment. Secondly, what we learned is the optimal endpoint to measure. So with these two things, we conducted a simulation what if scenario, what if we use the patient segment that we now best know and what if we use the endpoint that we know the best now, and we ran the statistics based on the Phase IIb data, we got the outcome that the therapeutic group met the endpoint by 49%, whereas the test group was 19%, the difference was 30%, and we obtained a p-value of 0.02, indicating that we would have met the primary endpoint if we had conducted the way that we now envision to do. So based on this, and we have lots of insights by this very precious Phase IIb study, we now have very solid confidence that we can do the next study very well and lead this next study to a success. More recently, we -- our publication was successful related to stroke. This publication went out earlier this year in January. And this is coming from many years of collaboration research between our company, SanBio, and Gladstone Institutes in California, which everybody knows as a leading research institute in the United States. We are very excited about this publication because we have done a lot of analysis using SB623, using animal models, using electrophysiology, using a lot of tools to understand what SB623 is actually doing in the brain tissues. And now we have many new findings what's happening and why SB623 is so efficacious. And I'd like to just share the quote by the joint team with the Gladstone Institutes and SanBio. This is on the right-hand corner. There are currently no treatments that can be given weeks or months after stroke to prevent long-term symptoms, so this is incredibly exciting. So the team is referring to the potential of the SB623 and everything that we found out. So with all these activities that we have been doing, our confidence level has dramatically increased, and we are ready for expansion. I'd like to summarize where we are and where we are heading. This is a summary of the area and indication expansion outlook. In the past, in the last couple of years or so, we have really sort of pin focused on Japan TBI and trying to get the world first ever approval on the brain regeneration drug. We did it, and we were able to get that approval. And now we have now embarked on growth opportunities. And by now, we restarted the discussions with the FDA. So we are expanding into the U.S. opportunities. Also, our preparation for the ischemic stroke is underway. I'd like to also mention not only our big picture of where we are going, I would like to also share what to anticipate the remaining of this year. So in the first half of this year, in Japan, an anticipated event is the approval of the ‘AKUUGO, which is going to result in the release of the shipment of ‘AKUUGO. This is going to require three batches of the production run, which two of them are already complete and the third one underway. So this is one event we anticipate. Another one down at the bottom is the United States. And this one, we already achieved this milestone of resuming the discussions with the FDA regarding the Phase III clinical trial. So this is already done and the discussions are underway. In the second half of this year, in Japan, we anticipate listing of the AKUUGO on the drug pricing list and the launching of our AKUUGO product finally. And we also anticipate ischemic stroke progress. More specifically speaking, we anticipate start of the negotiation with the agency PMDA. And lastly, in the second half of the year, for the United States initiative, we anticipate obtaining agreement with the Food and Drug Administration for our Phase III clinical trial for TBI. Finally, I would like to summarize our vision to become a global leader in regenerative medicine. This is sort of the image that we have for our company growth. We have now the vision of a back to original vision and that we aspire to become a global leader in regenerative medicine. How are we going to do that? The answer is simple. We will obtain a dramatic growth through two things: one is restarting stroke; and another one is restarting the United States initiative. So in the 4 years' time frame or beyond, we envision launching products in the United States. And this is going to give us opportunity to help many patients in Japan, in the United States, and we believe that this is going to create a huge growth for our company. Right now, we already got the approval for the world's first brain regeneration drug, but this is a beginning. We, ourselves -- we see ourselves currently as a green sphere in the left lower corner, and we envision to really grow the company dramatically. What's happening this year? If you could look at the middle of this slide, within this fiscal year, fiscal year 2026, which is the calendar year of 2025, we anticipate launching in Japan, we anticipate restart TBI in the United States. We also have preparations underway for stroke, both in Japan as well as the United States. So we have a clear vision and -- to become a global leader in regenerative medicine. And I hope that our path to the global leader and how we are going to help many patients through this journey is now more clear to the -- to you, who has kindly come to this presentation today. Thank you very much.