Right. I'm going to start. Good afternoon, everyone. Thanks for joining us to have a conversation with Mr. Bart Van Rhijn CFO of Nanobiotix. Nanobiotix is developing a radio enhancer, NBTXR3, which is a hafnium oxide nanoparticle that can be induced by radiotherapy to cause significant tumor cell death in injected tumor. In 2023, the company forged a global co-development and commercialization agreement with Johnson & Johnson pharmaceutical. I would welcome Bart to this fireside.
Thank you, Arthur. A pleasure being here, and thanks for having us.
Sure. Maybe the—so Bart, the radiotherapy seems to be coming back as a potential therapy since the science has started seeing it as a new inducing therapy. With that backdrop, how do you see Nanobiotix as a company that can become a major player in the radiation therapy?
Excellent questions. Allow me to say a few words about the company, and then I'll get into the radiation piece. Nanobiotix is headquartered in Paris with a group of folks in Boston. It's a phase III oncology company, and we have two other platforms. Maybe a few words on those platforms. The first one is QR9. It's a bioavailability and biodistribution platform that transiently occupies the liver clearance pathway. If you were to use a nanoprimer, as we call it, first, you basically condition the body to then receive a therapeutic agent that is IV administered. It can do its work because it can go through the bloodstream and reach its intended target with the right dose and toxicity. That's our second platform. The third platform we have is what we call Ocuity CNS.
It's basically leveraging the neuronal pathways and electric properties for which we designed nanoparticles to up or downregulate the electrical conduction in the brain. That can have big implications and applications. The main space where we've been playing and what we're known for is in the radiation space because we have these functionalized hafnium oxide nanoparticles that get into tumor, re-injected, exposed to radiation. That physical mode of action does a lot of the cancer cell killing you reference. Where we see the market going is an excellent question because if you think about radiotherapy, it's not designed to cure. Radiotherapy, you can only provide as much to a human as the healthy tissue will endure, right? There's a massive opportunity to basically move that standard of care.
That is what we have been focused on with our lead product, NBTXR3, or now also known as JNJ-1900, further to the partnership with Johnson & Johnson. When we look at radiotherapy as a field, it is a massive field. There are 60% of all cancer patients that get treated who have radiotherapy in their treatment regimen. That is about 12 million people annually. As population grows and there is more early detection, we see the radiotherapy field growing. Recently, maybe a small note, we have done a study with MD Anderson with regards to lung reirradiation, whereby we gave patients a subtherapeutic dose, and we saw very compelling effects. I think the standard of care is a standard of care moving to have a greater impact. With innovation, we believe that impact can become a lot greater in the years to come.
I see. From the previous data you guys generate, the R3 molecule seems like it's tumor agonistic and also combination therapy agonistic. Could you help highlight data that can support that for the, as it be a true, it can be used for a different kind of cancer treatment?
Yeah, happy to. If we look back at what we've done and what we're currently doing, our clinical development pipeline has 13 trials spanning nine different tumor types. We have feasible, we have good safety data, and we've got very compelling efficacy responses throughout all of these different tumor types. Indeed, to your point, right, we believe this is a product that can address many tumors. It's patient agnostic. It's target agnostic. It's combination agnostic. It has great versatility in the field. We're very fortunate to work with MD Anderson that does a lot of the signal finding and going to the edges of innovation, if you will, in order to help demonstrate what this asset can do.
Let's dig a little bit deeper into the Johnson & Johnson partnership. Could you give us a—definitely, that's a big validation for the technology and platform. Could you give us a little bit of overview about what the contract looks like for the partnership?
Yeah, happy to. It is a global co-development and commercialization deal. With this pipeline and a product, they can play in all the different tumor types. We're very happy because it is a partner that obviously has demonstrated many times over what they can do on a global basis. They have the rights not only in the rest of the world, but they acquired the rights for Greater China from LianBio at the end of 2023. They have the global rights. They have launched one of their lung studies from CONVERGE. We can talk about it later. The contract is one where we have a lot of milestones in excess of $2.6 billion and royalties in the low teens to low 20s%.
In the recording mid-last year, you guys did a common environment of the partnership. Could you tell us more about what's the—
Yeah. Earlier this year, we basically, on the back of the transfer of sponsorship of our phase III trial, the 312 study, as we call it, in a locally advanced head and neck, we transferred the sponsorship to Johnson & Johnson. It gives us, it gives Johnson & Johnson specifically an ability to take it through their regulatory process, but also to get ready for potential launch should the data come in positive. It is shortening next steps in the development of this product to go to market. Earlier this year, we were able to conclude this license amendment with them, which allowed us to transfer the cost of this phase III pivotal study to Janssen for the vast majority. It avoids that we have to bear these clinical trial costs. It extended the runway to mid-2026.
Also, beyond mid-2026, we'll have a lower run rate because also cost beyond that point will no longer be in our P&L. It was a very deliberate move in exchange for some of the milestones to get direct support at this time.
Speak of the 312 study. Could you give us a little bit of a walk us through how the design? I also record the—you did some amendment to the study together with the transfer. Could you tell us more about that?
Yeah, happy to. It is a locally advanced head and neck for patients that are cisplatin ineligible. It is randomized 1- 1, 250 in each panel. It has stratification factors for Johnson Comorbidity Index for GEO, HPV status. It is a big study that should deliver some results with an ability to go deeper with primary endpoint being PFS and key secondary being overall survival.
I see. What's the next biggest update from the study?
That will be the interim analysis. Maybe going back to your prior question, I think it's also good to explain what the bar for success looks like in this 312 study. That would be nine months+ four, so 13 months for PFS, and then 12 + four or 16 for OS. You may recall our Study 102, the phase I study that we did in a small population, which showed us median overall survival of 23 months in the evaluable population and 16.9 months for the PFS in the evaluable population.
I see. So besides that, you mentioned before Johnson & Johnson also initiated a lung study coverage, right?
Converge.
Converge. Could you tell us a little bit more about that study design and what's the—
Happy to.
Okay. Yeah.
That's a randomized study, 130 patients. It is locally advanced, non-resectable, non-metastatic, non-small cell lung cancer, stage 3, just the specific population. It uses standard chemoradiation and then with a follow-on of durvalumab. That's an open-label study. I think that's very interesting. We're very much looking forward to seeing what comes out of that study. The primary endpoint in this study is ORR, so it's response rate driven. We have a track record of delivering response in many, many tumor types. We look forward to that with confidence. I think for us, although it's not the same population, but depending on where you sit, you may want to take a look at that lung reirradiation study that MD Anderson did.
The fact that with a subtherapeutic dose, you can create the responses we did, I think, personally speaking, I think that bodes well for our conversion.
I see. Back to the head and neck cancer. You also have another, your own study, Study 1100 ongoing. What's the clinical update in this year for that study?
Quick backdrop on this 1100 study. It has three arms. One is naive patients, naive to PD-1, resistant to PD-1, and then a cohort of patients with primary tumors that metastasize to soft tissue, lung, and liver. We have shared data on naive and resistant previously. For naive, we had PFS of 73 and mean overall survival of 26.2. On the resistant, we had 4.3 of PFS or 4.2 and 7.8 for OS. When you look at that resistant population, you see results that are significantly more than if you were providing these patients with PD-1 alone. We believe there is a strong signal. It looks good. The data that we will be sharing in the remainder of this year will be rent patient in and data for both the naive and resistant population. That is nearing its end.
For that third cohort, it will be data and that will have a focus on melanoma. We haven't reported on melanoma previously. We believe that that is something that will generate some interest.
That's very encouraging. What's the regulatory plan for that?
We've had discussions with the FDA in the past, leading us to believe that that might be a design that could be response-driven with patient numbers between 100 and 150. It moves us to state that ultimately, this will be dependent on the final design of the study. This is also with the Joint Steering Committee, right? It's something that J&J would need to take forward. Ultimately, the FDA will have its say what that ought to be, but it could be response-driven, which is interesting.
I see. Through your collaboration with MD Anderson, I know you report some interesting data in the pancreatic cancer. Could you highlight those for us? What's your plan for that cancer indication?
Yeah. So indeed, I think that was a week and a half ago that we shared an update. We had some compelling overall survival. Median overall survival was 23 months. LPFS was 13.3 months. Pretty spectacular for this type of patient. We have very engaged investigators at MD Anderson. We look forward to bringing this to a close and continue to recruit patients in this study. I think maybe the last point to highlight is the MD Anderson investigators also saw some interesting biomarker points of attention with circular tumor mutational burden linked to LPFS and OS. There was a pretty high CA19-9 normalization in these patients of 59%. That was also encouraging.
That might be an opportunity for us to do some more work on the biomarker front in order to be well-informed about patients and how that may play out in terms of these evolutions.
Do you guys plan to give you more update this year from that?
Most likely not this year. We haven't announced it. To come.
Okay. I see. I guess for the close, could you remind us what's the other major milestone we should have been on the lookout for over the next two months- three months?
Yeah. We have, other than the 1100 update, so that's the naive and the resistant in head and neck. Then this third cohort, melanoma, we'll have an update on esophageal. Also an MD Anderson-run study that will take place later this year. It is going to be the interim analysis for Study 312, which is guided for H1 2026. We believe that this pivotal study and the data there and/or the data that will come from the CONVERGE trial, the phase II non-small cell lung cancer stage III trial that J&J is running, will really inform what the next steps in the development pathway will look like.
Got you. Obviously, the Johnson&Johnson transaction has alleviated most of your financial issues. What other additional levers do you have to regulate that you can have a catch-y2 to make sure you guys go through all the milestones you want to get?
Yeah. Thank you, Arthur. Yeah. It's important for us that with the asset where it's at, we bring it into what's called a safe harbor because we have those other two platforms where we believe we can make an impact. So Curadigm and Ocuity CNS platform. There's a lot of value that's tied to R3 or JNJ-1900. We don't feel that the equity markets are in a place that they can value the potential of these platforms at this time. That may correct itself. One of the ways to think through solving for that is to think about non-dilutive funding because you create temporary earnings dilution, but not permanent capital dilution.
I think we can be smart about these things so that it is in the best interest for both shareholders as well as the company and these platforms because there's a lot for us to prove that we need to keep working on.
Awesome. Thank you, Bart. Thank you for coming down.
Thank you, Arthur. Pleasure.