Good morning, everyone, and thank you for joining us. I am Amir Hishmat for Executive Chairman, President, CEO of Neon Technologies Holdings Incorporated.
I'm joined today by our leadership team and scientific team members.
Dr. Thomas Chan, our Chief Medical Officer. Dr. Josh Neiman, our Chief Clinical Officer. Heathly Garnett, our Chief Financial Officer, Dr. Henry Friedman, Chair of our Scientific Advisory Board, and Dr. Alex Miller, a key member of our Scientific Advisory Board. Together, this team reflects the depth and experience of innovation driving NEON forward. Today marks an important milestone for NEON. Through the dedication of our investigators, patient, and scientific collaborators, we have achieved full enrollment in our LEAD Phase II clinical study, NEO-100, our internasal therapeutic design for recurrent IDH1 mutated high-grade gliomas. This morning, we will be discussing significant radiographic response and long-term survival in recurrent World Health Organization Grade 3, Grade 4, IDH1 mutant high-grade gliomas, treated with intranasal NEO-100. findings that underscore the promise of our platform and its potential transform outcome in some of the most challenging brain cancers. This program embodies our mission, translating innovation into hope for patients who have long been without effective options. This data we'll review today not only will demonstrate meaningful radiographic response, but also durable survival benefits that are beginning to redefine what is possible in aggressive CNN malignosis. With that, I will turn it over to Dr. Thomas Chen. Thank you, Mayor.
So today, what we will be doing is presenting our data from the Phase 2A, as Mr. Hatchman-Borres stated. And what we will be looking at is initial introduction to the trials that NEONC has currently in progress. And what we have here is basically our NEON100-01. These are the results that we will be talking about today in patients with recurrent grade 3 and grade 4 astrocytomas. All these patients have IDH1 mutations. These patients have been treated with standard of care therapy and now have recurrence of their disease. This is a phase 2A trial, and as mentioned, we have now completed enrollment of 25 to 25 patients. Our NEO100-02 is also given intranasally. It is still in progress. It is given for treatment of meningiomas. Meningiomas are the most common type of benign brain tumors. However, they can also become atypical organolignant. And these are the patients that we will be treating in our phase 2. It's currently at 7 out of 30 for enrollment. Our next trial, NEO212, is given orally. It's a chemical conjugate of temozomide with NEO-100. We're currently in phase one. We're treating all brain tumors. We are now in our last cohort. We've enrolled 13 out of 15 patients. And this is the maximum tolerated dosage has not been reached. In our last trial, it's NEO-100-03. It is our pediatric application. We are going to be giving it intranasally as well. We will be treating pediatric brain cancer, usually in the midline. It is currently in Phase 1, and it will be starting enrollment.
Thank you, Tom. Now I'll go over the NEO 100-1 Phase 2 objectives, which we are fully enrolled in. Quickly, I'll go over the eligibility requirements, which are for patients with who-recurrent IDH1 mutant, grade 3, grade 4 astrocytomas who have gone through standard of care therapy. This includes radiation and temozolomide. Our primary objectives for this phase 2A trial has been progression-free survival at six months. Our secondary objectives have been to assess objective response rates, overall survival, evaluate safety and tolerability, and confirm every exposure to perillic acid and collect quality of life data. As you can see from our swimmer's chart, these are all patients enrolled within the Phase 2a trial. Amongst patients in our Phase 2a cohort who have been on study for at least six months, we have achieved a 44% progression-free survival. These are 8 out of 18 evaluable patients. This rate, as you can see, significantly exceeds historical benchmarks of 21% to 31%, indicating a meaningful improvement in disease control compared to prior studies. Now I'd like to go over our significant radiographic response and long-term survivor cohort summary. These were patients with, again, recurrent IDH1 mutant who grade 3-4 astrocytes illness. In this cohort summary, we looked at 24 patients receiving intranasal NEO-100 only across our phase 1, which was 5 patients, and phase 2A, which were 18 patients, as well as one compassionate use. Again, all patients were enrolled in study for a minimum of six months prior to data cutoff. MRI confirmed radiographic response via MRI contrast and perfusion. We saw no major toxicity reported in any chronic intranasal dosing. These are a summary of the five patients that we will be going over with respect to our radiographic response. Again, these patients are alive, all are alive, and are on NEO-100. I will be going over the patient history, and Dr. Chen will be going over the radiographic response imaging. For patient one, we have a female who's 63 years old. She was diagnosed with WHO grade 4 IDH1 meet and astrocytoma with unmethylated MGMT status. she presented with on on on february 2022 showing a partially necrotic left partial mass she then went on rose total resection then adjuvant therapy which included radiation and temozolomide followed by six maintenance of temozolomide on follow-up on december 22nd there was confirmed metabolic activity consistent with recurrence she had no other active therapies after October 22nd. On her MRI in January of 2023, she showed an irregular left periventricular enhancing mass and started NEO100. She has been on NEO100 for 35 cycles, which corresponds to 35 months. Dr. Chen.
This is the radiographic imaging of this patient. What you can see is in her left bridal periventricular area is a post-contrast imaging showing a contrast-enhancing mass. This is at baseline. After cycle 8, without any other treatment, you can see that this mass has significantly decreased in size. And then after cycle 34, almost all the mass is gone from the sample of contrast enhancement. And on the perfusion scan, which demonstrates that there's increased blood flow to the tumor, it is negative.
Our second patient is a female, 46 years of age. She was diagnosed again with a blue grade 4 IDH1 mutant astrocytoma with an unknown MGMT status. She went through initial management of surgical resection, followed by adjuvant therapy, which included radiation therapy and four cycles of temozolomide and an optume device. Prior to study, there was clinical progression of evidence of relapse for this patient. She went on 58 cycles of NEO-100 and is currently alive. Dr. Chen.
This shows the patient's MRI scan at the time of enrollment, and you can see the area of contrast enhancement. After two cycles, you can still see the mass, but on her perfusion scan, it is cold. And after cycle 57, that mass is pretty much gone, and her perfusion scan remains cold.
Our third patient, again, is a 32-year-old female. She was diagnosed with an IDH4 mutant astrocytoma, again, with an unknown MDMT methylation status. Her initial management was biopsy, and adjuvant therapy was started with radiation and one cycle of temozomite. On pre-study MRI, she showed that she had recurrence, and her first dose was on October of 2024, and she is currently on 12 cycles of Neo100 and is alive.
Dr. Chan. This shows the patient has a small recurrence. This is in her left frontal lobe. and with treatment you can still see at cycle two you can still see that patient has a lesion at that same area. At cycle 12 the contrast enhancing portion is now gone and with the perfusion scan you do not see the lesion anymore as well.
Our fourth patient is a female of 30 years old. She was also diagnosed with a grade 4 IDH1 mutant astrocytoma. Her initial management included surgical resection with pathological confirmed diagnosis. She went on adjuvant therapy, which was radiation therapy and temozolomide. Again, she went on screening in March of 2025 and began Neo100 alone. She is currently on seven cycles of Neo100 and alive.
Dr. Chang. This shows that she has a right frontal lesion at the time of recurrence. After two cycles, that area enhancement is not seen, and her perfusion scan is full. After cycle seven, you can see that there is still some enhancement. The question was whether this was just pseudoprogression, and you can see here that her perfusion scan remains cold.
Our last patient is a male, 34 years of age, diagnosed again with a grade 4 IDH1 mutant astrocytoma with MGMT unmethylated status. He went on, underwent surgical resection, followed by adjuvant therapy of radiation and temozolomide. He was enrolled in NEO-100 and has been on cycle 7 or 7 months of NEO-100 and is alive with no other current therapies.
Dr. Chen. This shows the patient has a small right frontal lesion. And you can see an area of hypermedicolic activity on this MRI perfusion scan. After two cycles, you still see the lesion, but now that area of hypermetabolic activity on the perfusion scan is no longer seen. And after six cycles, the lesion has stayed about the same size, but the perfusion scan shows that the lesion is not seen with no activity.
So now we're going to move on to our data for our long-term survivors. These include our World Health Organization Grades 3 and 4 IDH mutant astrotypomas. These subjects are from our Phase 1, Phase 2, and Compassionate Care Program. Out of patients who have been on NEO 100, as you can see, 8 out of 24 of these patients are termed long-term survivors, meaning that they have been on NEO-100 and surviving following initiation of the therapy for a minimum of 18 months. The median survival for these patients is 88 months in total. We do not see any major toxicity reported across the cohorts, and there are no adverse events as well. In summary, our NEO100 clinical outcomes for recurrent who-grade 3-4 IDH1 mutant apocytomas are that for progression-free survival at six months, we have 44% of patients who've achieved this progression-free survival. This outperforms historical benchmarks of 21% to 31% for IDH1 mutant recurrent high-grade gliomas. With respect to our radiographic response, 21% of patients achieved significant radiographic remission. This response rate exceeds the 8% typically observed with salvage therapies for recurrent gliomas. Finally, with our long-term survivors, 33% of patients demonstrated a durable survival of greater than 18 months post-initiation of Neo100 with immediate overall survival of 88 months.
Dr. Chai. Thank you, Josh. First of all, I want to say that this is the first of its kind signal for durable response. And with our NEO 100 trial, we are seeing MRI confirm significant response in a setting that's rarely seen. This provides a potential paradigm shift with multi-year survival, and demonstrates that NEO100 is a CNS-penetrant metabolic therapy that is durable. Our differentiation of our drug from current therapy is that it's minimal toxicity, it's a nasal brain delivery system, and it's much more durable and can be performed at home compared to standard chemotherapy. This provides us with translational momentum to support future phase 2b slash 3 and global trials.
Now I'd like to open it up to discussions and remarks from Drs. Henry Friedman and Alex Miller. Dr. Henry Friedman is our chair of our scientific advisory board and our distinguished professor of neuro-oncology and leader of the Preston-Robert Tisch Brain Tumor Center at Duke University. Dr. Alex Miller is chief of neuro-oncology and co-director of the Brain and Spine Tumor at the NYU Langone Health. Thank you.
Alex, you want to go first?
Sure. Thank you. Thank you so much for the exciting presentation and the data and for the kind introduction, Dr. Neiman, you know, honored to be part of this team and very excited about the work that was just presented. You know, as everyone here alluded to, existing treatments for glioma are really inadequate, and often our patients are sadly left with few options, most of which are palliative. And our effective treatments currently, you know, for glioma really represent an urgent unmet need for innovative new strategies, especially ones with minimal side effects and easy administration. So NEOWONC really represents a unique approach to the treatment of phleomas. And as I mentioned, I think particularly appealing is the ease of administration, the lack of significant side effects associated with the drug, and the fact that because there are so few side effects, it really has the potential to enable it to be layered on top of many of our other treatment strategies as well. So the encouraging preliminary results that were just presented, I believe really provide clear justification for continued research on how neo-onc may be best deployed to become part of our armamentarium as neuro-oncologists that we can use in order to counter the devastating effects of gliomas on our patients and their families. And I'm really looking forward to the next phase and a bigger clinical trial to follow the signal and see how this work unfolds.
Well, how am I supposed to follow that. That was quite eloquent and quite spot on. I've been doing this a long time and only rarely does an agent come out that gives one the hope that we're going to see something really meaningful. I remember I was there with the FDA for temozolomide. Our team at Duke led the push for Avastin. This reminds me of Avastin in the sense that we saw a small cohort of patients who had some extremely good responses, and that led reasonably quickly to approval for recurrent disease and an allowance to use it in newly diagnosed disease with bulk disease present. Everything I see tells me there's a strong signal here. Everything tells me, for all the reasons that Alex elucidated, that this is something we really should be pursuing. Duke will participate in these trials, of course. This is something that needs to be pursued, but I am heartened that we've got something that's going to prove to be very meaningful. And I don't say that very often in this field. Thank you, Dr. Friedman.
Thank you, Dr. Friedman, Dr. Miller. We're going to go to the web for questions, and I see that we have several already. Dr. Chen, maybe I'm going to point this question towards you. The first question is, is there any toxicity that is considered on target? Does NEO100, does your drugs metabolism suggest any drugs that NEO100 can't be combined with?
The answer is that there are no visible toxicity with NEO100. In terms of combination therapy, we have done some compassionate use patients with combination therapy, but we have not had a formal trial.
And a second follow-up question, Dr. Chen, is the resistance mechanism to NEO-100 understood? And any ideas of what might predict the long responses? this?
That's a great question. It still illustrates the fact that we do not understand fully all the mechanisms of NEO-100. NEO-100, to me, the response that we're seeing is really the tip of the iceberg in terms of what we can potentially achieve with this stroke. We know for sure that it's not toxic. We also know for sure that it has activity. How it combines with other therapies, how it combines with either recurrent disease or newly diagnosed disease are topics that we will be exploring. Great.
I'm going to, Dr. Friedman, there's another question in the chat that I'm going to ask you. Given the durable responses and favorable safety profile, could New 100 qualify for breakthrough therapy, fast track, or even orphan drug designation expansion?
Yes. I think that those are things that we need to pursue. They're all different pathways, but all of them need to be explored. The problem, of course, is the FDA a year ago is not the FDA of today. So I'm not sure where we are. But one very heartening step was that Rick Pazder was just made head of CEDAR, and that's usually important. He was head of the Oncology Center. Now he's head of CEDAR, and he brings to bear a predictable way of responding to the kinds of issues that we're talking about now. So I think that orphan disease, well, that's easy. It's an orphan disease. That's a no-brainer. Breakthrough or other ways that one can expedite the review, again, depends on their perception of the data. But I think that the thing that's most striking to me, frankly, is less the responses than the duration of time that patients who were progressing go on the drug and stay on the That is the single most important thing as far as I'm concerned. You know, responses are nice to show people, and I'm not poo-pooing them in any way. But when I see a curve or a slide that shows me you've got patients on there for years on the drug, and they were actively progressing at the time they entered that therapy, that's a signal that needs to be pursued. I think the FDA will probably feel the same way. Thank you, Dr. Friedman.
Dr. Miller, there's one question that I'm going to, in the chat, that may be pointed to you. Again, based on its safety profile, could NEO100 be explored in combination with temozomide, CCNU, slash lomustine, or other inhibitors, including immunotherapy?
Absolutely. And I was just, you know, thinking as I was going through trial designs in my head during the presentation that, you know, I think that's a clear place to go is to layer it on top of standard of care, especially for the, you know, IDH mutant population where we often see better responses to chemotherapy. But, you know, even though we see better responses, we know that those responses are inadequate and not durable. And so anything we can do to lengthen that amount of time for our patients, I think we should pursue.
Okay, great. Thank you. We have several other questions, but I know we're running out of time. So I'm going to now turn it over to Mr. Heshmapur for closing remarks.
Thank you. Thank you, Josh.
And it looks like Dr. David Ashley has joined us, as well as Axel. So with the closing remarks, as of today, NEO 100 is fully enrolled, a pivotal achievement that positions us to deliver the full FDA six-month readout by May 12th of 2026. We are deeply encouraged by the safety, durability, and clinically meaningful benefits we've seen to date, and even more inspired by what lies ahead. On behalf of our entire NEONC team, I want to extend my heartfelt thanks to our investigators, collaborators, and above all, our patients. your commitment and courage continues to to push our mission forward together we are not just advancing our therapy we are advancing hope for those fighting brain cancer and thank you and god
