Nanobiotix S.A. Q1 FY2024 Earnings Call

Good day, and welcome to the Nanobiotix Corporate Strategy Update and Q1 2024 Conference Call. 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.

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 €58.9 million and reaffirmed our cash runway into Q3 2025, which is inclusive of the $20 million milestone we received. With that said, I'd like to turn the call over to Laurent. Please go ahead.

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 an 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. 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 in our vision and our plans to get there. Last summer, we entered into a collaboration with Janssen worth potentially more than $2.5 billion plus royalties ranging from low teens to low twenties to expand the worldwide potential of our lead therapeutic candidate, NBTXR3, a potential first-in-class radioenhancer with universal application across various tumor types. As part of the agreement, NBTXR3 is being developed and potentially commercialized globally by Janssen Pharmaceutica, a Johnson & Johnson company. Our global 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 radioenhancer's 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 a 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 a 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's many stakeholders. Alongside this, Nanobiotix is working to explore and push forward the next generation of nanoparticle-based therapy in healthcare. 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 three nanoparticle-based therapy platforms. 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 nanoparticle-based therapy, and we will discuss more on this shortly. But first, let's start with our efforts to deliver potentially curative outcomes for patients with locally advanced and metastatic cancer with NBTXR3, as it remains our primary focus. As a reminder, for those new to the story, NBTXR3 is a biologically electron-dense nanoparticle designed to be a strong X-ray absorber and radiation amplifier. It is a one-time treatment designed to minimize impact on the patient journey as it is injected directly into the tumor before the first course 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 lends itself to NBTXR3's ability to be combined with many other modes of therapy, including chemotherapy regimens, targeted therapy, and importantly, immuno-oncology therapy. NBTXR3 is being touted as a therapeutic candidate with potential to easily integrate into the cancer treatment paradigm for 60% of solid tumors 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 3 evaluating the radioenhancer for elderly patients with locally advanced head and neck cancer, as well as the launch of a randomized Phase 2 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 positive head and neck trial results, Nanobiotix and Janssen aligned at the collaboration's Joint Strategy Committee to transfer the global sponsorship of the Phase 3 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 product's value and its efficient path to market. In conjunction with the sponsorship transfer, following discussions that began in late 2023, the Joint Strategy Committee aligned modifications 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 Study 102, a Phase 1 study evaluating NBTXR3 in a similar population, which provided satisfactory support for the NANORAY-312 trial design, which made the futility analysis unnecessary. This amendment is subject to review and approval by health authorities in all countries where NANORAY-312 is operational, as well as by the sites participating in the study. To date, the amendment has been accepted by health authorities in several regions, including the United States, Europe, and Japan. Additionally, Nanobiotix and Janssen agreed to a change in the approach to the planned interim efficacy analysis, such that Janssen will realize and report on the interim data after both the requisite number of events are observed and at the end of recruitment, which is expected in the first half of 2026, rather than immediately after the requisite number of events, as originally planned. This revised approach helps 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 expects 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 operations since executing the Global Licensing Agreement this July. We started with the formation of the Joint Strategy Committee comprised of executives from both companies to drive strategic decision-making for the global development program. We also activated joint working teams staffed by both companies, with an initial focus on manufacturing and preparing NANORAY-312 for global regulatory submission in the event of positive trial results. In addition to establishing governance methods, 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 to increase product availability. So we have started to leverage increased discretionary income contributions that were allocated from Janssen to Nanobiotix. That includes staff support for NANORAY-312 along with payments for manufacturing, technical assistance, 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 2 study for patients with Stage 3 non-small cell lung cancer. Fifth, we began elaboration of the overall clinical development plan for NBTXR3, including new indications beyond head and neck cancer and non-small cell lung cancer. And lastly, Janssen consolidated global development and commercialization rights by assigning 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 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 indications in the near, medium, and long term. The Joint Strategic Committee will recommend the next indication beyond the immediate operational 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 expansive potential of NBTXR3 and continues to progress. In our immuno-oncology 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 two cohorts in Study 1100, an ongoing Nanobiotix-led Phase 1 clinical trial that has completed dose escalation and is 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 from multiple primary cancers, will be presented later in 2025. Expansion opportunities in additional cancer settings 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 1 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 1 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 1 studies have been completed with primary endpoints met in patients with hepatocellular carcinoma with and without liver metastasis and in rectal cancer. Our main focus and priority for the 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 expects to lead a new Phase 2 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 moves toward the ultimate goal of reaching millions of patients with cancer around the world through the execution of our ongoing and planned studies, we are working to expand the impact of nanoparticle-based therapy in healthcare through continued early stage development of our nanoparticle expertise, which includes two additional platforms, Curadigm and Oocuity. Like NBTXR3, these platforms have been years in the making, and we are excited to share some of our progress with you today. Curadigm has produced a nanoprimer as its first product candidate that is designed with specific physicochemical properties to allow its nanoparticles to transiently occupy liver cells responsible for therapeutic clearance. This allows subsequently delivered therapeutics to bypass the liver's clearance mechanism. This is intended to increase bioavailability and subsequent accumulation of therapeutics in targeted tissues, thus allowing the medicine to do its work. This has the potential to see increases in efficacy or decreases in toxicity, or reductions in the needed dose of subsequently administered medicine. The Oocuity platform is based on the principle that nanoparticle materials can interact with and influence neuronal networks via their electrical properties. The goal is to potentially enable modulation of malfunction in neuronal networks towards a normal state. In particular, the reduction of neuronal hyperexcitability associated with neuropathic pain has been observed in both in vitro and mouse models with several nanoparticle candidates. We expect to provide the next update on our plans 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, modifications to 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 positions 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 for 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.

We will now take the first question from Jonathan Chang at Leerink Partners. Please go ahead.

Hi guys. Thanks for taking the questions. 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? Thank you.

Thank you, Jonathan. Let's address the second question first before moving to the first. To provide some context regarding the shift in sponsorship, if we reflect on a year ago, the company was primarily focused on advancing the 312 trial to the interim readout as swiftly as possible. Last July, we entered a partnership aimed at maximizing patient reach and expanding our presence in the market. Consequently, our focus has transitioned from solely prioritizing the interim readout to broadening the range of indications, such as lung cancer, which represents a larger market compared to head and neck cancer, while also pursuing a more secure approach. This led us to discuss the timing of the sponsorship transfer, ultimately resulting in the decision we reached. Internal discussions took place at the partner level, followed by agreement at the Joint Strategy Committee and endorsement from our Board. Regarding the timing of the transfer, J&J must be the sponsor to register the product, initiate pre-market activities, and effectively launch it. We had the option to transfer now or wait until after the interim readout. Considering the complexity and time required to execute such a transfer for a global trial involving 500 patients, we determined it was best to handle the transfer now. This decision alleviates that burden later, allowing us to concentrate fully on product registration and market activities following a positive interim readout. This is the context of our operations and the press release we issued. As for your point on the cash runway and financing, I'll let Bart address that.

Thank you, Laurent. Thank you, Jonathan, for the question. Just to recap, we believe that it's an important step forward in de-risking the assets, positioning it for success, and really optimizing its potential for the benefit of patients and healthcare professionals. 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-2025 in line with the milestones that we shared in the PR. Based on prior study results, we look forward with conviction to share those updates. As with 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.

Understood. Thanks for taking my questions.

Thank you.

Thank you. We will now take the next question from the line of Lucy Codrington from Jefferies. Please go ahead.

Thank you for taking my questions. Can you provide clarity on the timing of the sponsorship transfer process? It appears you anticipate it will be completed before the interim readout scheduled for the first half of 2026. Once the sponsorship is transferred, will J&J assume the financing of that trial, which could potentially extend your cash runway if it occurs sooner? Additionally, how confident are you in completing recruitment by the first half of 2026, and what is the minimum period required after recruitment completion for the interim to take place? Lastly, regarding funding for the new indications you plan to advance to Phase 2, would that be in addition to your commitments for funding 312, or could J&J’s takeover of 312 free up some resources? Thank you.

Thank you, Lucy. First, regarding the timing of the transfer of the 312, we are still in the early stages as the intent was only agreed upon in the last few days. It will indeed take some time. However, the goal is to complete this transfer well before the interim readout, which is why we are initiating this process now. This will require some investment from our partner, and we are currently working on a joint plan to both transfer the trial and continue running it without interruption during the transition. We believe this is critical. As for the financing, we cannot discuss our partner's costs, but we view our expenses as neutral for the time being. In the future, it may appear a bit positive, but we cannot confirm that yet since we are early in the process. Regarding your second question about completing recruitment by the first half of '26, I think that's a reasonable conservative estimate for a biotech. Considering our recruitment experience so far and the enhancements we anticipate with our partner's involvement, achieving this target is quite feasible. You also asked about the timing of the interim readout relative to the last patient enrolled. We expect to have the 283 events completed before recruitment ends, and significant preparatory work can occur before the interim analysis is triggered. Ideally, we aim to synchronize the last patient enrollment and the interim readout closely. Your third question was about new indications and our approach to their selection along with financing. Overall, with the transfer of the 312, we still have a considerable amount of work ahead. We do not expect to have additional capacity during the transfer period since our team will be focused on that. However, we have a substantial pipeline that indicates various potential opportunities for NBTXR3. Based on this and other factors, we will identify any new indications for Nanobiotix. Once the transfer is complete and we have some bandwidth, we can consider initiating a new trial. This will also depend on acquiring additional funding and milestone payments from our contract with J&J. We will keep you informed about these developments. Over time, we intend to explore all future opportunities we have for NBTXR3, as well as beyond what we mentioned in the press release.

Thank you.

Thank you. We will now take the next question coming from the line of Suzanne van Voorthuizen from Van Lanschot Kempen. Please go ahead.

This is Chiara Montironi. I'm on behalf of Suzanne. Thanks a lot for taking my question. I have two questions, if possible. The first one will be, can you provide some color on how imminent will be the Phase 2 in lung cancer? And if it's not imminent, what are the gatekeeping items here? And the second question is more about the new programs, the new platform, Curadigm and Oocuity—what kind of updates should we expect in H2 '24? How concrete is that? Are you already working on some product candidates? Thank you.

Thank you. So about lung cancer, which is the randomized Phase 2 trial that J&J is going to run, we've been publishing recently a good update explaining that the FDA got the green light about the protocol design. 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 J&J is working heavily to get that done and started as soon as possible. I think we'll be able to give some updates 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 provide 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 opportunities. Some of the key things and questions we would like to answer and discuss with you in H2 is how we're going to build a sustainable business model with the various possibilities 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 leads in this technology? Yes, we have multiple products that could be used to further develop this platform. But obviously, we will provide much more on this in H2.

Okay, thank you very much.

Thank you.

Thank you. We will now take the next question from the line of Swayampakula Ramakanth from HCW. Please go ahead.

Thank you. This is RK from H.C. Wainwright. Good afternoon, Laurent. 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 two 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 do you need to generate data; and only with that sort of data would a partner actually sit at the table?

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. There is a lot of untapped value for this first product within the collaboration we have, and the primary focus of this collaboration is to make that work and 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. 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 healthcare. 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 co-develop 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 are 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.

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 trying to do this parallel track. I'm just trying to understand how you would be developing all three programs, albeit at different levels of development, of course.

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

Perfect. Thank you. Thank you very much.

Thank you.

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

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. We believe this data and this specific application of NBTXR3 are very important regarding opening new doors and also potential new indications 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. 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.

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