Nanobiotix SA

PAR:NANO

Earnings Call Analysis

Earnings Call Transcript 2024-Q1

[Operator]

Good day, and welcome to the Nanobiotix Corporate Strategy Update and Q1 2024 Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded.

At this point, I will turn the call over to Craig West, Senior Vice President of Investor Relations of Nanobiotix.

[Craig West, executive]

Thank you. Good afternoon and good morning, and welcome to the Nanobiotix conference call to discuss updates to our NBTXR3 development plan and our first quarter.

Joining me on the call today are Laurent Levy, Co-Founder and Chief Executive Officer; and Bart Van Rhijn, Chief Financial Officer, who will be available during the Q&A section. As a reminder, today's call is being webcast and will be available on our website for replay. I would like to remind you that this call will include forward-looking statements, which may include statements regarding the progress, success and timing of our ongoing and planned clinical trials, collaborations, regulatory filings, dates of presentation and future research and development efforts, among other things.

These forward-looking statements are based on current information, assumptions and expectations that are subject to change. They are subject to significant risks and uncertainties that could cause the company's actual results to differ materially from our current expectations. Accordingly, you are cautioned not to place undue reliance on forward-looking statements. Please review the full description of risk factors that can be found in the documents we filed with the AMF in France and SEC in the United States, which are available in the Investor Relations section of our website, along with the press releases issued yesterday and today.

In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, Nanobiotix undertakes no obligation to update them to reflect subsequent events or future circumstances.

Before turning the call over to Laurent, allow me to highlight that we announced in a separate press release this morning, our first quarter 2024 unaudited cash balance of EUR 58.9 million and reaffirmed our cash runway into Q3 2025, which is inclusive of the $20 million milestone we meanwhile received.

With that said, I'd like to turn the call over to Laurent. Please go ahead.

[Laurent Levy, executive]

Thank you, Craig, and thank you, everyone, for joining us today. As Craig mentioned, we issued a press release yesterday, highlighting the company's progress in the robust development of NBTXR3 and overview of our robust pipeline and expansion strategy. For today's call, I would like to begin with updates to our NBTXR3 collaboration and discuss our pathway to long-term growth. Then I will provide closing remarks before opening up the call for questions.

Nanobiotix has been long known for the disruptive potential of NBTXR3, a nanoparticle treatment we've been developing for patients with cancer around the world. The accomplishments we have achieved through our 20-year history stand as a testament to the hard work of our team, the potential value the industry at large sees in our technology as well as the tremendous faith and confidence of our investors and other stakeholders all in our vision and our plans to get there.

Last summer, we entered into a collaboration with Janssen was potentially more than $2.5 billion plus royalties ranging from low teens to low 20s to expand the worldwide potential of our lead therapeutic candidate, NBTXR3, a potential first-in-class radioenhancer with universal application across solid tumor. As part of the agreement, NBTXR3 is being developed and potentially commercialized globally by Janssen Pharmaceutica, a Johnson & Johnson company. Overall license agreement with Janssen has brought us the possibility to deliver outcome changing innovation for millions of patients. Today's focus with our partner is to secure the path to market for NBTXR3 and our commitment to realize our [indiscernible] medical and economic value remains our most important goal.

We believe this agreement underscores the therapeutic and market opportunity of NBTXR3 and importantly, further validates our platform and scientific approach. We believe that this collaboration with our partner has the potential to impact the lives of many patients. We believe this because NBTXR3 can treat patients at stage where the disease is local with radiation therapy, which is a treatment utilized by millions of patients. Better local control of disease at this stage, we believe could have the fundamental impact on the overall outcome for patients. The alliance with Janssen for the co-development and global commercialization of NBTXR3 has achieved significant progress, which we will overview today.

We'll remain focused on the execution to secure our partnership outcomes and drive as much value in the alliance as possible for patients and Nanobiotix' many stakeholders. Alongside this, Nanobiotix is working to explore and push forward the next generation of nanoparticle-based therapy in health care. I'm excited to discuss with you today our corporate strategy, which is designed to enable long-term growth for Nanobiotix. Our company is developing 3 nanoparticle-based therapy platform. We plan to leverage the sustainable revenue expected to come from the development and commercialization of NBTXR3 to further advance development of our second platform, Curadigm and the third platform. Building on the success of NBTXR3, we are energized to expand our leadership in naroparticle-based therapy and we will discuss more on this shortly.

But first, let's start with our efforts to deliver potentially curative outcome for patients with locally advanced and metastatic cancer with NBTXR3 as it remains our primary focus. As a reminder, all those new to the story, NBTXR3 is biologically electron-dense nanoparticle designed to be a strong X-ray absorber and radiation amplifier. It is a onetime treatment designed to minimize impact on the patient journey as it is injected directly in the tumor before the first cause of radiation treatment where it has been shown to amplify the anti-tumor activity of the radiation without increasing exposure to soft tissue. We call this mode of action universal.

We do so because it's based on physics and radiation interacts with the nanoparticle and not on the specificity of the tumor type or the biology of the patient. This mode of action also lands itself to NBTXR3's ability to be combined with many other mode of therapy, including chemotherapy regimen, targeted therapy and importantly, immuno-oncology therapy. NBTXR3 is being iterated as a therapeutic candidate with potential to easily integrate into the cancer treatment paradigm for 60% of solid tumor of patients who receive radiation as part of their therapeutic regimen each year.

To date, NBTXR3 has been injected in hundreds of patients by leading physicians at several pre-eminent and clinical research institutions in the United States, Europe and Asia. To help enable the practice-changing potential of NBTXR3 in collaboration with the Interventional Oncology Group at Johnson & Johnson, the current NBTXR3 development program is initially focused on the completion of NANORAY-312, an ongoing pivotal Phase III evaluating the radioenhancer for elderly patients with locally advanced head and neck cancer, as well as the launch of a randomized Phase II study evaluating NBTXR3 for patients with Stage 3 non-small cell lung cancer for which Janssen is fully responsible.

In preparation for potential regulatory submission in the event of a positive head and neck trial results, Nanobiotix and Janssen aligned at the collaboration's Joint Strategy Committee or JSC to transfer the global sponsorship of the Phase III pivotal head and neck cancer trial. Nanobiotix will continue to support Janssen in the execution of the NANORAY-312 during and after the sponsorship transfer is complete. This was a deliberate decision to pursue the transfer sooner rather than later because we believe it's the best decision for the products, value and its efficient paths to market. In conjunction and with the sponsorship transfer following discussion that began in late 2023, the Joint Strategy Committee aligned to modification to NANORAY-312 that include a protocol amendment removing the planned futility analysis in light of the robust positive top line data from the expansion part of the Study 102, a Phase I study evaluating NBTXR3 in a similar population which provided satisfactory support for the NANORAY-312 trial design, which in the futility analysis not useful.

This amendment is subject to review and approval by health authorities in all countries where NANORAY-312 is operational as well as by the [indiscernible] industry. To date, the amendment has been accepted by health authority in several regions, including United States, countries in Europe and Japan. Additionally, Nanobiotix and Janssen agreed to a change in the approach to the planned interim efficacy analysis, such as Janssen will realize and report on the interim data after both the requisite number of events are observed and at the end of the recruitment has occurred, which is expected in the first half of 2026, rather than immediately after the requisite number of events as originally planned.

This revised approach helped to ensure that potentially positive interim analysis results do not introduce a potential bias in the remaining recruitment prior to completion of the study. As such, Nanobiotix now expect the interim analysis to be reported after the last patient is recruited in H1 2026.

Now let's review other significant progress on our NBTXR3 collaboration with Janssen in establishing development operation since executing the global licensing agreement this July. We started with the formation of the Joint Strategy Committee compared equally of executive from both companies to drive strategic decision-making for the global development program. We also activated joint working team that are staffed by both companies with an initial focus on manufacturing and preparing NANORAY-312 from global regulatory submission in the event of positive trial results.

In addition to establishing good [indiscernible] key operational progress has been achieved. Highlights include: first, Janssen has begun replicating the Nanobiotix manufacturing process to allow Johnson & Johnson to produce NBTXR3 in parallel with our own facility in order to increase product availability. So we have started to leverage increased discretionary income contribution that were allocated from Janssen to Nanobiotix. That includes staff's report for NANORAY-312 along with payments for manufacturing, technical assistant and other product development support services.

Third, we achieved the first operational milestone in NANORAY-312 triggering a $20 million payment in May from Janssen to Nanobiotix. Then the U.S. FDA has issued a Study May Proceed letter regarding the protocol for the Janssen-led Phase II study for patients with Phase III non-small cell lung cancer. Fifth, we began elaboration of the overall clinical development plan for NBTXR3, including new indication beyond head and neck cancer and non-small cell lung cancer. And lastly, Janssen consolidated global development and commercialization rights by the assignment to Janssen of the licensing agreement relating to China and other Asian markets and territories from our former Nanobiotix partner, LianBio.

As you have heard, we've made significant progress with Janssen advancing NBTXR3. It is key to understand that bringing a therapeutic candidate from concept to global registration is an exciting, yet daunting proposition for any development stage biotechnology company, even those working within well-known asset classes with well-established development and regulatory pathways. That is why we believe that our global licensing agreement with Janssen was and continues to be the right move for the patients, for healthcare professionals and for all the supporters of NBTXR3's potential.

We are confident that the leadership of the Joint Strategy Committee will optimize the probability of success on NANORAY-312. Importantly, our global licensing agreement with Janssen also includes plans to further evolve the NBTXR3 development pipeline. These plans include an established framework for potential complementary co-development of relevant indication in the near, medium and long term. The Joint Strategic Committee will recommend the next indication beyond the immediate operation priorities in head and neck and non-small cell lung cancer. Our current pipeline beyond Stage III lung and locally advanced head and neck cancer has already shown the expensive potential of NBTXR3 and continues to progress.

In our I-O combination program, NBTXR3 in combination with anti-PD-1 therapies for patients with recurrent or metastatic head and neck cancer that is naive or resistant to anti-PD-1 treatment with and without metastasis is being explored in 2 cohorts in Study 1100 and ongoing Nanobiotix-led Phase I clinical trial that has completed dose escalation and ongoing dose expansion.

We expect to present initial data from the head and neck cancer trial expansion cohort at ASCO this June. The third part of this study, including patients with metastasis for multiple primary cancer will be presented later in 2025. Expansion opportunity in additional cancer setting in the medium to long term are being investigated in several trials that are part of our ongoing collaboration with MD Anderson Cancer Center in Texas.

This includes Phase I studies that have completed dose escalation and are in ongoing dose expansion in locally advanced pancreatic cancer and inoperable recurrent lung cancer and an ongoing Phase I study in dose escalation evaluating NBTXR3 in combination with chemotherapy in patients with locally advanced esophageal cancer. We expect multiple readouts from this study in the next 12 months. Additionally, several Nanobiotix-led Phase I study have been completed with primary end points met in patients with hepatocellular carcinoma with and without liver metastasis and in rectal cancer.

Our main focus and priority for NBTXR3 program is validating the safety and efficacy of radiotherapy activated NBTXR3 and moving to global registration in locally advanced head and neck cancer. These efforts account for the majority of operational and financial resource allocation. Nanobiotix expect to lead a new Phase II randomized study in a new indication in due time once NANORAY-312 sponsorship transfer has been completed, additional funding has been secured and approval has been confirmed by the Joint Strategic Committee.

Now let's turn to the promising future of Nanobiotix beyond NBTXR3. As the NBTXR3 program moved toward the ultimate goal of reaching millions of patients with cancer around the world through the execution of our study both ongoing and planned, we are working to expand the impact of nanoparticle-based therapy in health care through continued early stage development of our nanopartical expertise, which include 2 additional platforms, Curadigm and Oocuity. Like NBTXR3, this platform has been here in the making, and we are excited to share some of our progress with you today.

Curadigm has produced a nanoprimer, has its first product candidate which is designed with specific physicochemical property to allow its nanoparticles to transiently occupy liver cells responsible for therapeutic clearance. This allows a subsequently delivered therapeutics to bypass the liver's clearance mechanism. This is intended to increase bioavailability and subsequent accumulation of therapeutic in targeted tissue thus allowing the medicine to do its work. This has the potential to see increases in the efficacy or decreases in the toxicity or reduction in the needed dose of subsequently administered medicine.

The Oocuity platform is based on the principle that nanoparticles materials can interact withwith and influence neuronal network via the electrical properties. The goal is to potentially enable modulation of malfunction in neuronal network towards a normal state. In particular, the reduction of neuronal hyperexcitability associated with neuropathic pain, has been observed in both in vitro and [tumor] model with several nanoparticle candidate. We expect to provide the next update on plan and operational progress for our Curadigm platform in the second half of 2024.

As you heard today, we've made significant strategic and operational progress in our NBTXR3 global alliance with Janssen. This includes the intended sponsorship transfer of the pivotal NANORAY-312 trial, modification of the NANORAY-312 to optimize the regulatory pathway and continued ongoing exploration of expansion opportunities for NBTXR3 in other indications. This achievement validates the potential of our nanoparticle platform and strongly position us to expand our Nanobiotix' corporate strategy. We plan to allocate some of the expected proceeds from NBTXR3 to further develop our Curadigm nanoparticle platform to foster and enhance long-term growth potential and value of Nanobiotix. We look forward to providing you updates along the way.

With that, I will now ask the operator to begin the Q&A session. Operator?

[Operator]

[Operator Instructions] We will now take the first question coming from the line of Jonathan Chang from Leerink Partners.

[Unknown Analyst]

First question, can you discuss how you're thinking about strategies to extend the company's cash runway? And then second question, can you provide any color on the alignment process with J&J on a global development plan for NBTXR3? And when we might hear more on the potential medium- to long-term opportunities?

[Laurent Levy, executive]

Thanks, Jonathan. So maybe let's answer the question #2 before going to question #1. So -- and maybe before, let's give some context in this shift in sponsorship. I think when we -- if we put ourselves one year ago, the focus of the company was about getting the 312 to the interim readout as fast as possible and it was the context in which we've been operating. Then last July, we've been signing this deal and the whole idea of this deal being to -- with our new partner, making sure that we can reach the largest number of patients possible and to grow the [indiscernible] and to grow the market, if I may say.

So we've been shifting from having all focus on getting this interim readout as fast as possible to focus, which is enlarging the number of indications, like having the lung cancer which is again a bigger market than head and neck, but also to go in a more secure or derisked way. So that's why along and around all this discussion we have had on what's the best timing to transfer the sponsorship, we ended up in this decision. So there have been some internal discussion, of course, at our partner level, then there have been an agreement at the JSE and then an endorsement by our Board. So that has been the process to get to this decision.

And I think there's a big question about why now versus later. So first of all, in order to -- for J&J to be able to register the product, to start premarket activity and to launch the product effectively, they need to be sponsored of this trial. So there was a choice to do it either now or after the interim readout. Given the work and the heavy lifting that represents such a transfer for global trial with 500 patients, that will take months and months to get there effectively.

So we thought the best thing to do is to do it now, take the burden now and don't have this burden post interim readout. Then after the interim readout assuming the data are positive, we can be fully focused and J&J could be fully focused on registering the products and starting up all market activities. So that's the context in which we operate, and we've been, of course, doing this press release. Now as you mentioned, this is opening a question about the current cash runway and financing, but maybe I'll let Bart answer that question.

[Bart Van Rhijn, executive]

Thank you, Laurent. Thank you, Jonathan, for the question. Just to recap, we believe that it's an important step forward in derisking of the assets, positioning it for success and really optimizing its potential for the benefit of patients and HCPs. As we indicated in the press release, the cash runway is unchanged. And yes, it implies that we need to finance through the updated timeline that we communicated. That said, we have much data to deliver between now and mid of 2025 to the milestones that we shared in the PR. And based on prior study results, we look forward with conviction to share those updates. And as any other biotech, we have multiple options, and we're very confident that we will work through this. And as you may appreciate, we cannot message on either the timing or the composition of sizing of the milestones.

[Operator]

We will now take the next question from the line of Lucy Codrington from Jefferies.

[Lucy-Emma Codrington-Bartlett, analyst]

So just again, sorry, the transfer of the sponsorship just in terms of what does the timing look like for that whole process? It sounds like you do expect it to be done before then now at the interim readout in -- from first half '26. But I guess once that sponsorship is transferred, does J&J then take over financing of that trial, and therefore, that alone could help extend your cash runway where it happens sooner rather than later?

And secondly, just your level of confidence in completing recruitment by first half '26 and therefore, the potential timing of that interim. And is there a minimum time after the completion of recruitment at which point that interim would need to take place? And then just in terms of the funding for the new indications that you might take into Phase II, I guess, that's on top of your commitments to running -- funding 312 or if J&J would take over 312, that might free up some funds?

[Laurent Levy, executive]

Thank you, Lucy. So first of all, the expected timing for the transfer of the 312. So we're early in the process as the intent has just been agreed in the past few days. So it will take time, that's for sure. But the intention, of course, is to complete that way before the interim readout. That's the whole intention of making this transfer now.

Now in terms of -- obviously, this will add some investment on the partner side. Now we were just establishing what's the joint plan not only to transfer the trial, but to continue to run the trial up to the end, knowing that there would be no interruption of the trial during the transfer, that's the whole game we are trying to achieve here. So for financing part and how much this will cost Nano, how much will cost to our partner, we can't talk for our partner, but on our side, we think for now it's neutral, may look at it in the future to be a bit positive, but we can't say more right now because we are, as I said, early in the process.

So now to your second question about H1 '26 to get the end of recruitment, I think that's a good estimate that we could qualify as a biotech as conservative estimate. But we think given the experience of recruitment we have had so far, plus what we expect to improve the equipment with the involvement of our partner, we should be able to reach this target. That's within the realm of high possibility.

So now you asked a question about how the interim will play versus the last patient in and timing for that. So we expect, of course, to get the 283 event before, of course, at the end of recruitment. And there's plenty of work that could be done prior to trigger the interim per se. So talking theoretically here because we, again, can't talk for our partner, the idea will be to be ready to just push the button and to have almost concomitant last patient in and the interim readout for the trial.

So your third question, if I recall correctly, was about new indication and how we select and move forward in that direction and what about the finance. First of all, I think that overall, in our plan, including the transfer of the 312, there's still a lot of work to be done here. So we don't expect to have opening bandwidth from the team within the period of the transfer because we'll be heavily working on that versus what we're doing today already. But a new indication, I think we have a large pipeline today showing all the possibility or at least a big part of the possibilities of NBTXR3. And based on that and some other considerations, we will define what could be a new indication for Nanobiotix.

And the goal here being when we start freeing up some bandwidth of the team when the transfer is complete, then we could start thinking about having a new trial. And this will, of course, depend on potential additional funding and other later stage when the milestone payment coming from also the contract we have with J&J. So we'll keep you update on that, obviously. And over time, we can start building all the next opportunity we have for NBTXR3, but also, as we mentioned, in the PR, beyond NBTXR3.

[Operator]

We will now take the next question coming from the line of [ Suzanne ] [indiscernible].

[Unknown Analyst]

This is [indiscernible]. I'm on behalf of [Suzanne]. I have 2 questions, if possible. The first one will be, can you provide some color on how imminent will be the Phase II in lung cancer? And if it's not imminent, which are the gatekeeping items here? And the second question is more about the new programs, the new platform, Curadigm and Oocuity, which kind of updates should we expect in H2 '24? How concrete is that? Are you already working on some product candidates?

[Laurent Levy, executive]

Thank you. So about lung cancer, which is the randomized Phase II trial that J&J is going to run, we've been publishing recently a good update explaining that FDA got the green line or get the green light about the protocol design. And obviously, that's going to trigger a number of activities and work around that by our partner. So we can't say more at that stage, but time always comes in what we do, then you can imagine that is the one is working heavily to get that done and started as soon as possible. I think we'll be able to give some update over time in agreement with our partner about the development of this clinical trial.

Now for the question on Curadigm, the expected update we want to give in H2 is about how we're going to build the strategy and the business model around this new platform which could give not only one product but multiple products and in any case, multiple applications in different therapeutic areas. So as I think I have mentioned in the past few calls, that's a technology that offers many, many opportunities. And some of the key things and key questions we would like to answer and discuss with you in H2 is how we're going to build a sustainable business model with all different possibility we have with this type of technology, both on internal development and of course, external collaboration and business development activities. Now to your question, do we have already lead in this technology? Yes. We have multiple products that could be used to continue to develop this platform. But obviously, we will give you much more on this in H2.

[Operator]

We will now take the next question from the line of Swayampakula Ramakanth from HCW.

[Swayampakula Ramakanth, analyst]

This is RK from H.C. Wainwright. Just on the long term, thinking about Curadigm and Oocuity. So you have certainly generated a lot of validation on your NBTXR3 program. And how much of that validation can get transferred into these 2 new nanoparticle platforms? The reason for that question is, can you point to NBTXR3 if a partner comes and wants to talk about either Curadigm or Oocuity and is that enough? Or you need to generate data and only with that sort of data, would a partner actually sit at the table?

[Laurent Levy, executive]

Thank you, RK. That's a good question. Maybe just before I answer this question, I just want to reiterate that on NBTXR3, we still have a lot of work to do. And there is a lot of untapped value for this first product within the collaboration we have and the primary focus of this collaboration being -- and the company being to make that work and also to get the value of this product and collaboration recognized in the value of the company.

Now we're starting to build the future as we've been discussing that. And in terms of Curadigm or Oocuity, there is a broader validation that our product is bringing in the introduction of nano physics-based mode of action into the health care. Now all products are different and have different therapeutic applications. Of course, we need to generate a number of data to get to a potential partnership and so on. The main difference being that for both platforms, Oocuity and Curadigm, we are talking here about a wide technology platform where we can not only work with product and codevelop our licensed product, but we could also start licensing part of the technology to partners for them to develop their own product. So again, there's multiple opportunities here from very short term to medium and long term, and that's what we intend to be able to explain to you in the second part of this year.

[Swayampakula Ramakanth, analyst]

So should we expect some sort of preclinical data from both Curadigm and Oocuity if not in the second half, but over the next, say, 12 months? I'm you trying to do this parallel track. I'm just trying to understand how you would be developing all 3 programs, albeit at different levels of development, of course.

[Laurent Levy, executive]

Yes, without giving you any precise date for second half, yes, we'll be able to show to [shareholders.]

[Operator]

There are no further questions. I will now turn the call over to Dr. Levy for his closing remarks.

[Laurent Levy, executive]

Thank you. And maybe before I make some closing remarks, I just want to remind the audience that we have been publishing that we will present at ASCO our data on the immuno-oncology program with our lead product, NBTXR3. And we think this data and this specific application of NBTXR3 is very important in regards to opening new doors and also potential new indication for Nanobiotix. So I hope to see you at that call when we will disclose and explain and discuss the data.

And on that note, I'm going to thank you and wish you a very good day and rest of the week and let's stay tuned for the next piece of news that will come in the coming few days. Thank you very much. Have a great day.

[Operator]

Ladies and gentlemen, this concludes today's presentation. Thank you once again for your participation. You may now disconnect.