Good morning, and thank you so much for joining us at the H.C. Wainwright Third Annual Cell Therapy Virtual Conference. I'm Douglas MacPherson. I'm an Equity Research Associate on the healthcare team here at H.C. Wainwright. Now, I would like to introduce you to our speaker for this session. Please welcome William Ho, who is the CEO and Co-Founder of IN8bio, a company focused on developing cell therapies for cancer treatment. Will.
Thanks, Doug. Hi, everybody. Great to be here. Thanks for the H.C. Wainwright team for having us again. Really excited to be here and providing an update today on our company, IN8bio. As Doug had mentioned, I'm the co-founder and CEO of the company. My background: this year will mark 24 years I've been in the biotech industry.
A little bit of a unique background: I've been on all sides of the business, from investment banking as a sell-side research analyst and on the buy-side running a portfolio. I met my CSO and Chief Scientific Officer, Dr. Larry Lamb, almost a decade ago, where Dr. Lamb is an expert in the area of cell therapy and gamma delta T- cells. He has been working with these cells for over 30 years.
Today, our team is one of the best in the world at developing novel cell therapies targeting the gamma delta T- cells. We believe these are powerful cells with unique properties that make them suitable to target cancer. Today, across two trials, we have patients across multiple indications, across multiple centers being treated with different therapies who remain with durable remissions for the long term. Multiple patients who actually remain in remission beyond three years.
Our team has strong capabilities with over 30 years of experience with gamma delta T- cells. Our platform, what we call DeltEx, enables us to expand, activate, and genetically modify gamma delta T- cells at scale. With this knowledge and know-how, we're advancing rapidly towards trying to achieve our mission of what we call Cancer Zero, the safe elimination of all cancer cells in these patients.
Ultimately, the goal for us is, how do we keep patients alive and in remission for the long term? To accomplish this, we believe that we need continued immune surveillance to prevent or eliminate cancer relapses. To achieve this, our tool is the gamma delta T- cell. We know a lot of people have used other cells, such as NK cells or alpha beta T- cells, but we believe the gamma delta T- cell is a unique tool.
Many cancer relapses are due to the heterogeneity of a tumor. Ultimately, when we think of a tumor, we think of a single tumor. The reality is, cancer is more like 2 million different tumors. It is that heterogeneity that causes the problem, and we believe is a challenge that prevents some of our competitors from demonstrating long-term durable remissions.
It shouldn't be too surprising to learn that the gamma delta T- cell receptor is highly complex. It has polyclonal binding abilities. The gamma delta T- cell has the ability to distinguish between healthy and tumor tissue. Ultimately, it's the complexity of the T- cell receptor that allows it to determine the heterogeneity of the tumor and kill what's dangerous while leaving the healthy tissue alone.
In many cases, people have called the gamma delta T- cell nature's CAR T- cell. Multiple studies have also demonstrated that higher levels of gamma delta T- cells are associated with better survival outcomes. Just last year, two papers came out. They're at the bottom of the slide in front of you. One in melanoma patients being treated with pembrolizumab, a checkpoint inhibitor, showing that higher levels of gamma delta T- cells were predicted to have better outcomes.
A second study, a paper last year, looking at CAR T therapy, in particular CD19 CAR T- therapy, again showing that higher levels of gamma delta T- cells are associated with better survival outcomes. Today, we believe that IN8bio possesses a comprehensive gamma delta T- cell and a unique base of know-how and knowledge that makes us one of the best in developing gamma delta T- cells out there.
These are our pipeline programs to date. INB-100 is the first allogeneic gamma delta T- cell program. We very recently presented new data at the TCT conference about two weeks ago. This is what we call DeltEx Allo in leukemia patients undergoing transplantation. Preclinically, we have three programs. INB-300 is our unique gamma delta T- cell-based CAR T- program that can distinguish between healthy and tumor tissue, widening the therapeutic index and the safety window.
We can create iPSC-derived gamma delta T- cells with what we call INB-500. We're actively looking for potential partners for both of those programs. Very recently, we announced we have a new gamma delta T cell engager platform. We will be providing additional update or preliminary data at a medical meeting this spring. Behind that, our solid tumor program, what we call INB-200 and 400. INB-200 was the investigator-initiated phase one trial. I'll provide a slight update.
We'll provide some data on that program today. INB-400 was the phase two program that we suspended further enrollment last year. We will provide some data from this program later this summer. Ultimately, higher levels of gamma delta T- cells are associated with greater survival in cancer patients. It was actually our own Dr. Lamb who first made that observation in the 1990s.
This was his publicaton and his data. In leukemia patients undergoing transplantation, those patients who had higher levels of gamma delta T- cells had a greater than three and one-half times greater survival than those who had low to normal levels of gamma delta T- cells. This has been demonstrated multiple times since. Multiple parties have demonstrated additional data showing that higher levels of gamma delta T- cells result in better outcomes.
Another study in 2019 coming from the University of Copenhagen again demonstrates that higher levels of gamma delta T- cells result in better survival outcomes. Just read the title to this publication yourself. Today, we are advancing forward our INB-100 program. This is the allogeneic delivery of gamma delta T- cells into leukemia patients undergoing transplantation. I can say we are demonstrating that we are increasing the levels of gamma delta T- cells.
These are the data we have previously presented. Here in black are patients undergoing transplantation without receiving gamma delta T cells. You can see the trend is generally downwards. In cohort one, where we infuse 1x 10 to the six cells per kg, in blue, you can see the increased levels of gamma delta T cells. In green, the second cohort, our recommended phase two dose, we infuse three times the dose at 3x 10 to the six cells per kilo.
At 60 days, we are generating eight times the number of gamma delta T cells, showing that we are actually getting in vivo or in the body expansion of gamma delta T cells. Surprisingly, these gamma delta T cells are persisting beyond 365 days, the first time anybody has infused any allogeneic cell therapy and demonstrated long-term persistence. What is important is the dotted gray line.
We're actually showing that we are surpassing the numbers of gamma delta T cells that have been shown in prior studies that have been associated with better survival outcomes. We are going forward in leukemia patients undergoing transplantation because ultimately, relapse is the biggest challenge. Very recently, we had a conference call. We invited Dr. Michael Bishop from the University of Chicago, who spoke on our behalf, who outlined the challenge of relapse.
Up to 25% of patients will relapse within the first 100 days, and up to 50% will relapse within the first year. Even if you are transplanting patients and the patients are in complete remission or CR and are MRD negative or minimal residual disease negative, it doesn't mean that they don't have any residual leukemia. It simply means you are below the level of detection, which is at about 1 in a million cells.
Ultimately, if patients are relapsing, it means there was residual leukemia that we couldn't detect. All it takes is a single leukemic cell that doubles and doubles. Over time, we get these high rates of relapses. We are advancing forward in acute myeloid leukemia. There is a significant unmet need. There are about 21,000 patients newly diagnosed every single year.
We think we can help with these AML patients because the goal, despite whatever treatment's available, whether it is venetoclax, azacitidine, 7+3, FLT3 inhibitors, IDH inhibitors, or even the more recent menin inhibitors, the goal is always to bridge to transplant because transplantation is the only path towards a potential cure. We now realize to cure these patients, you need to transplant because the stem cells themselves are faulty and spitting out leukemic blasts. We need to reset the bone marrow.
Ultimately, to prevent relapse, we need continued immune surveillance to look for residual disease. Our hypothesis was that if we can eliminate any residual leukemic cells or keep pressure on the tumor, the patients will not relapse. This was the design of the trial. It was a 3+3 design, looking to dose escalate from 1 times 10 to the 6 cells upwards now to 3x 10 to the six cells, which we called our recommended phase two dose.
We initially enrolled all patients, including AML, CML, MDS, and ALL patients. We have made the decision to move forward specifically in AML based on specific guidance from the FDA. We initially ran this study at the University of Kansas Cancer Center. We are currently actively looking or seeking to add additional centers to this trial. At the University of Kansas to date, we have treated 16 patients.
We have enrolled 17 with one awaiting dosing. These were generally older patients receiving reduced intensity conditioning because they were not eligible for full myeloablative conditioning. That means there is a higher rate of risk of relapse. We had very complex disease with patients with chromosomal abnormalities such as a trisomy of chromosome 8, deletion of chromosome 7, current FLT3 or IDH inhibitor or IDH mutations.
These patients received no maintenance therapy, so no FLT3 inhibitor, no IDH inhibitor, only a single dose of gamma delta T cells. We believe we are doing quite well. We did ultimately have three patients who relapsed. None of them AML patients to date. We had Patient 9 who had ALL with a TP53 mutation, who had seven prior lines of therapy, patient 11, an MDS-MPN overlap, who also had a TP53 mutation. Ultimately, also patient 15, who also had MDS.
Currently, we are demonstrating durable remissions, especially in our complex AML patients. These are the patients that we reported to date. Our last update to the data cutoff is as of January 17. Across all of our AML patients, the median follow-up is currently at 20.1 months. In the first two cohorts, our initial 10 patients, the follow-up is currently at 23.3 months. We have yet to see a single relapse across any of our AML patients, which is where we're moving forward to.
Now, people often look at this data and say, "Wow, that's amazing data. Is the University of Kansas just very good? That's why you're keeping patients in remission?" We do realize this is in combination with transplantation. Very recently, we presented additional data looking at retrospective real-world control data.
We pulled data from the CIBMTR, which is the National Database of Transplantation and Outcomes. That includes all centers across the United States. That database pulled 684 patients. Here, it includes community centers and patients who have less complex disease. As you can see from the CIBMTR, about 33% of their patients with AML relapsed at one year, and about a quarter of them died within the one year.
We also pulled the data from the University of Kansas, both all across all leukemic patients and the AML patients. Again, we are outpacing what is expected of KU. The University of Kansas is a very robust leading transplant center. Their patient outcomes are worse than CIBMTR because they are a tertiary referral center. They take the more complex disease patients.
When we look at our data, in general, they are seeing about 40% of patients relapse by one year and about 33% ultimately passing by one year. Across all of our patients, we have 91% of patients remaining in remission at one year and 100% alive. In the AML patients, we have 100% alive and 100% in remission. We're quite excited about this data. We'll continue to enroll patients. We've given guidance. We are looking to complete enrollment up to 25 total patients in 2025.
We're looking to add additional centers and potentially add a prospective parallel observational arm as a potential control. We will provide additional updates later this year, but we're excited about the progress we've made so far. Ultimately, the holy grail of cell therapy has been to be able to target solid tumors. This is a glioma. You can see the dark purple tumor.
There are billions and billions of tumor cells. Our goal is ultimately, how do we prevent relapse? The reality is the bulk can be eliminated through the surgeon's knife. Relapse is often due to residual tumor cells that are left behind. If you look on the left margin within the healthy pink tissue, you can see individual purple dots.
Those are the tumors that are often left behind. Our INB-200 was designed to target those residual tumors and to prevent relapse. 80%-90% of newly diagnosed glioblastoma patients relapse locally within a centimeter or two of the original resection cavity. This trial was designed in the upfront setting, patients who are going the Stupp protocol. In 2025, marks the 20th anniversary for the last drug approval for glioblastoma, temozolomide. Patients are undergoing the Stupp protocol.
The median progression-free survival remains at about 6.9 months, with overall survival being between 14 and 16 months. In this trial, we take a patient, undergoes surgical resection. We insert a catheter that remains in place that allows direct delivery directly into the tumor bed. After a few weeks, we take blood from the patients and manufacture a product.
It is genetically altered so that our gamma delta T- cells are resistant to the lymphodepleting or the killing effects of the chemotherapy. The product is frozen, and the patient undergoes treatment such that in the maintenance phase, we combine dosing. Our chemotherapy-resistant gamma delta T- cells are dosed in combination with the chemotherapy.
We use the chemotherapy to lymphodeplete the patient, to debulk the tumor, to reduce immunosuppression, and to increase the immune signal on the surface of the residual tumor cells, even if they are resistant to chemotherapy or a part of the stem cell population that is often left behind. This trial was run at the O'Neal Comprehensive Cancer Center at the University of Alabama at Birmingham.
We've treated 13 patients to date, a mix of MGMT unmethylated patients who are not responsive to chemo, a mix of total and subtotal resections. We believe we are doing quite well to date. Our patients are living longer than expected, and many patients today are remaining in remission longer than we expected them to be alive. Here, the patients receive one, three, or up to six doses. In blue, the first cohort, the patients received a single dose.
This is marked by the orange circle. In purple, they received three doses. In green, those are the patients who received up to six doses. Of the patients who received a single dose, ultimately, everybody ultimately relapsed and died. The median progression-free survival was 8.3 months. Across all of our patients, we have a median progression-free survival of about 9.9 months. When we look at the patients who receive multiple doses, as of October 18 of last year, our median progression-free survival is sitting at 12.4 months.
The last approval for a medical device in glioblastoma was Novocure with the tumor treating fields, the Optune hat. Last year, they presented the Phase II top study of tumor treating fields in combination with pembrolizumab. The active arm showed a median progression-free survival of 12 months. As of October of last year, we are surpassing that median progression-free survival.
We believe we are demonstrating activity and keeping patients alive longer. We have multiple patients now who have remained in remission longer than they should have been alive. In fact, we have one patient, Patient 9. It is the one IDH mutant, but that particular patient is now out beyond 40 months in remission. Last year, Servier presented the Phase III data of the IDH inhibitor from Agios. In that data, the median progression-free survival of the control arm was 11.1 months, and all patients progressed by 31 months.
Small N, but we have a patient out beyond 40 months remaining in remission, and we have multiple patients with severe GBM with unmethylated disease who remain in remission long-term. We expect to provide an update in this program later this summer. Moving forward, we have a unique T- cell engager.
We will be providing first data in this at a medical meeting this spring. We're excited about this particular program. We have a unique T- cell engager platform that we created in-house. This one has a cassette-like target antigen binding domain that we can switch the targets. We believe we have a unique gamma delta T- cell engager in that it can drive significant expansion of the gamma delta T- cell compartment that nobody else has demonstrated that they can do.
Sit tight. We'll provide additional data later this spring. We continue to be excited about our progress. We raised an additional $11.6 million last fall. We have given guidance that we currently have cash available through 2025, and we have a lot of significant milestones, both clinical and preclinical, throughout 2025. We're excited about all the progress we've made. We've built a very strong team.
We believe that gamma delta T- cells are powerful tools against cancer cells. To date, the gamma delta T- cells appear across multiple studies to be linked with better survival outcomes. We are achieving the higher levels of gamma delta T- cells that are associated with those higher outcomes, and we are demonstrating long-term persistence of our gamma delta T- cells. Ultimately, our goal is to achieve long-term durable remissions with an enhanced quality of life.
Many therapies have been plagued by toxicities, cytokine release syndromes, neurotoxicities such as ICANS, and we have not seen any of it in our patients to date. We're excited about the data we have demonstrated. We are excited about the fact that we have treated patients across multiple centers with different therapies with complex disease, both in acute myeloid leukemia and glioblastoma, two indications that are thought to be complex, difficult, and deadly.
We have multiple patients beyond three years who have remained in remission. I want to thank you for your time. I want to thank you for listening to our story, and I welcome you to join us in our journey as we try to accomplish our mission of achieving Cancer Zero. Thank you.
That's outstanding, Will. Thank you so much for your presentation. We really appreciate you being here with us. Thank you again to everybody from H.C. Wainwright.