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spk06: And welcome all to the audio webcast of Galapagos' H1 2024 results. I'm Sophie van Heersel, Investor Relations, representing the reporting team at Galapagos. This recorded webcast is accessible via the Galapagos website homepage and will be available for download and replay later on today. I would like to remind everyone that we will be making forward-looking statements during today's webcast. These forward-looking statements include remarks concerning future developments of the pipeline and our company and possible changes in the industry and competitive environments. Because these forward-looking statements involve risks and uncertainties, Galapagos' actual results may differ materially from the results expressed or implied in these statements. Today's speakers will be Paul Stoffels, CEO, and Thad Houston, CFO and COO. Paul will reflect on the first half of 2024 and discuss our pipeline and programs. Ted will provide a financial and operational update and discuss the outlook for 2024 and present concluding remarks. You will see a presentation on screen. We estimate that the prepared remarks will take about 20 minutes. Then we'll open it up to Q&A with Paul and Ted, joined by Jeevan Shetty, Head of Development Oncology. And with that, I'll now turn it over to Paul.
spk01: Thank you, Sophie, and good afternoon, everyone. I would like to start with bringing back this slide that we showed at the first quarter results, summarizing our vision and our strategy to drive value creation for all stakeholders. We transferred Jaiselica and successfully transformed Calabros into a pure-play biotech with a revitalized pipeline, and we are investing in the elements that we believe we need to make our strategy successful. We focus on our key therapeutic areas, oncology and immunology, where significant unmet medical needs remain for patients. Our strategy is to spearhead our efforts with indications that have breakthrough designation potential in oncology and immunology, and we are building a broad pipeline of potential best-in-class cell therapies and small molecule drugs. We put in place strong leadership with a track record of delivering transformative drugs to patients around the globe, taking a collaborative approach, combining internal and external innovation. And finally, our strategy is supported by a strong cash position of 3.4 billion euros at the end of June 2024. We are building a pipeline and a manufacturing network to support our cell therapy vision, and we are optimistic that it will enable us to accelerate future growth. In the first half of this year, we delivered on a number of important milestones. Let's start with the regulatory achievements. We are pleased to share that we have submitted the IND for the FDA for Atalanta Phase 1-2 study of 5-101 in refractory relapsed non-Hodgkin lymphoma. Obtaining regulatory approval is a crucial step to conduct clinical trials with our cell therapies in the U.S., which is a key element of our growth strategy. We submitted a CTA with the European authorities for the eplagia phase 1-2 study of 5201 in refractory relapsed chronic lymphocytic leukemia and Richter's transformation. We made important clinical progress with our Phase 1-2 studies of 5-101 and 5-201 respectively, and we presented additional encouraging safety, efficacy, durability, and translational data at scientific conferences. We are making significant progress with our proprietary pipeline now comprising of 20 small molecules and cell therapy programs. I will give more color on this in the following slide. We're also significantly expanding the footprint of our cell therapy manufacturing network, not in the least through the agreement signed with Blood Centers of America for the U.S. territory. And finally, with the collaboration announced with Adaptimmune, we took an important first step into the cell therapy solid tumor space with TCRT cell therapy. Now over to our pipeline. In our two therapeutic areas of oncology and immunology, we aim to deliver best-in-class therapeutics. In oncology, we are progressing our Phase I-II CAR-T programs, 5-101 in NHL and 5-201 in CLL and Richter Transformation. As mentioned, I'll come back to the clinical progress presented at EHA for 5-101. We presented additional encouraging safety data, safety, efficacy, and translational data with 5201 at EBMT in April this year in 14 patients with relapsed refractory CLL and Richter's transformation, showcasing deep and durable responses in this critically ill patient population. In the Papilio Phase 1-2 BCMA-directed multiple myeloma program with 5301, we observed one case of Parkinsonism. Patient safety is a key priority, and therefore, we temporarily post enrollment protocol guidelines to evaluate this event. Parkinsonism has been reported previously in BCMA and CAR-T cell therapy studies, and we believe that with the implementation of a comprehensive and specific measure, this can be safely managed. The study protocol was amended and submitted to the EMA in June, and we anticipate resuming recruitment in the coming months and aim to report data from the study at the medical conference in 2025. While we advance therapies that we have developed internally, we believe that a robust pipeline should also include promising therapies developed externally. Thanks to the collaboration and licensing agreement with Adaptimmune, signed and announced at the end of May, we also added TCRT cell therapy to our pipeline. To option to exclusively license Adaptimmune's next generation TCRT cell therapy user cell, targeting May's age four, we took a first step in head and neck cancer at potentially additional solid tumor indications with our decentralized manufacturing platform. In their phase 1 CIRPAS trial with centrally manufactured Uza cell, Adaptimmune has already shown encouraging results in head and neck cancer with an overall response in 4 out of 5 patients. Initial in vitro results suggest that UsaCell produced on Galapagos decentralized manufacturing platform yields fresh, fit, early phenotype T cells in seven days that could potentially improve efficacy and durability compared to the UsaCell centrally manufactured on Adapt Immune's platform. In addition, the vein-to-vein time of seven days is important for patients in whom rapid access to treatment is vital. In immunology, we are progressing our two phase two studies with 3667 in lupus and dermatomyositis. As we work to advance programs in development, we are also investing in our discovery portfolio in small molecules and cell therapies to identify the programs of tomorrow. We are making important progress through our therapeutic areas, and we have over 15 internal programs in discovery across oncology and immunology with small molecules and cell therapies. We also continue to scout for external innovation to further build our early-stage pipeline. We recently expanded our strategic collaboration and licensing agreement with BRIDGENE Biosciences to include the discovery and development of a highly selective oral SMARCA2 small molecule, or PROTAC. In 2025, we expect to initiate at least four IND or CTA-enabling studies and at least one first-in-human study. From 26 onwards, our aim is to fuel the clinical pipeline with at least two new clinical assets annually across cell therapy and small molecules. As mentioned, we were happy to present encouraging new data from our Atlanta study with 5101 in NHLN at the EHAC conference in June. A quick reminder about the design of the study, which consists of a Phase I dose escalation part and a Phase II expansion part. The study is a basket trial in critically ill NHL patients. The CAR-T cells are manufactured using a decentralized platform with a vein-to-vein time of seven days. The Phase I primary objectives are to establish the safety profile and recommended Phase II dose. The Phase II primary objective is efficacy as measured by objective response rate. Here you see the pooled Phase I-II efficacy results for 31 patients. We observed high objective response and complete response rates over the different indications. In patients with diffuse large B-cell lymphoma, 7 out of 9 patients responded, and complete responses were seen in 5 out of 9 patients. In patients with follicular lymphoma or marginal zone lymphoma, complete responses were observed in 16 out of 17 patients. In patients with mantle cell lymphoma, complete responses were observed in all five patients. Looking at the responses over time, we saw that the results were durable in the majority of responding patients. 10 of 14 patients responding in Phase 1 had an ongoing response at data cutoff with a median follow-up of 13.1 months. Four patients in Phase 1 progressed after initial response. Two had the CD19 positive relapse. One had the CD19 negative relapse, and one patient was unconformed. In Phase 2, which started more recently, All 14 patients responding had an ongoing response at data cutoff with a median follow-up of 4.2 months, and one patient in Phase 2 progressed after an initial response. Turning over to the safety results now, it is a busy slide and I will not get into detail, but summarizing, we see the vast majority of CRS and ICANN events were low-grade. This is very encouraging and confirms the data that were previously shared at ASH in December of last year. As we previously disclosed, two deaths occurred during the study. One intra-abdominal hemorrhage caused by grade four disseminated intravascular correlation in dose two level, dose level two, and one grade five urosepsis event was reported in follow-up more than six months after infusion and while the patient was in complete response. one additional patient died due to disease progression. This updated patient set of 33 patients in total confirms the data presented earlier on the 23 patients. The observed safety, efficacy, and durability are very encouraging, providing additional data to show potential beneficial role of a short vein-to-vein time, robust in vivo expansion, and preservation of early T-cell phenotype on clinical outcomes. The data demonstrate that with our innovative cell therapy manufacturing platform, we can infuse fresh fit cells with a median vein-to-vein time of just seven days with heightened complete response rates across indications in heavily pretreated patients. I would now like to hand it over to Tad for the financial update and the outlook for the remainder of 2024. Tad?
spk05: Thank you, Paul, and thanks, everyone, for joining today. Let's take a look at the financial results for the first half of 2024. During the first half of 2024, we have worked on strengthening the foundation for future growth. We divested Giseleca, which enabled us to focus on our priority programs and invest in our pipeline and building our global cell therapy network. You will see our strategy reflected in our financial results. In January of 2024, we transferred the Giseleca business to Alpha Sigma, As a result, the Giseleca results moved to discontinued operations. For our continued operations, revenue remained fairly stable year over year, and mainly consists of the linear recognition of the platform for the Gilead collaboration. A small part is coming from the remaining Giseleca royalty income and inventory sale of the product to Alpha Sigma. As explained in our Q1 earnings call, we see an increase in R&D costs as compared to last year, which is mainly driven by our investments in oncology, both in cell therapy and small molecules. We reported a net profit driven by fair value adjustments in Forex, as well as €49 million in interest income. Also, we reported a net profit from discontinued operations of €71 million, mainly driven by the one-time gain of €52 million for the Giseleca transaction with Alpha Sigma. Now over to our 2024 guidance. Excluding business development activities to date, we reconfirm our full-year cash burn guidance of €280 to €320 million. The updated cash burn guidance accounting for the deals that we executed in the first half of the year is €370 million to €410 million for 2024. This is mainly driven by the Adaptimmune collaboration, which accounts for €79 million. We reported an operational cash burn of €250 million in the first half of 2024. Note that this comes on the higher end due to the announced collaborations with Adaptimmune and other smaller business development transactions. the phase transition services for Jselek at Alpha Sigma, which mainly happened in Q1, as well as the timing of interest, income, and tax credits. Our cash balance at the end of H1 amounts to €3.4 billion, supporting us to execute on research and development and collaboration opportunities. In the last couple of years, we closed strong partnerships to build our cell therapy capabilities entered into R&D collaborations and licensing agreements, and executed on acquisitions and equity investments. We continue to focus on strategic business development to bolster our pipeline and optimize our operations. I would like to highlight the collaboration that we closed a few months ago with Blood Centers of America as a partner for our cell therapy platform in the United States. Following the earlier announced collaborations with Landmark Biome Boston, and Thermo Fisher in the Bay Area. The collaboration significantly advances the expansion strategy of our platform in the US as BCA's national network will allow us to manufacture our cell therapy products close to the patient across the US. The network will support our planned pivotal trials and help us gear up for commercial readiness. Now turning to our outlook for 2024. We anticipate important regulatory progress with our CAR T trials in the U.S. To recap what Paul explained, we submitted the IND for our NHL trial. We also submitted a CTA to start the phase two dose expansion with 5201, and we are on track to submit the IND for 5201 in CLL and Richter transformation later this year. In H2, we will also share further data on the safety, efficacy, and durability of our ongoing CAR T programs 5101 and 5201. Operationally, we are working on opening additional sites for our decentralized cell therapy manufacturing, both in the U.S. and in Europe. We will leverage the BCA collaboration to open sites across the U.S. and are exploring additional partnerships for our cell therapy network across the globe. We also aim to execute on additional licensing agreements and acquisitions as well as research collaborations. Our business development efforts serve our overarching purpose of accelerating breakthrough solutions to patients in need. Let me conclude by coming back to the strong fundamentals that we have put in place to build an innovative biotech company and the clear path that we have towards value creation. We are progressing our early stage pipeline, building on our renewed discovery portfolio based on best-in-class targets towards best-in-class medicines. Thanks to the progress made, our goal now is to initiate at least four IND CTA-enabling studies and at least one first-in-human study in 2025. While we push forward internal programs, we remain active in business development. The ADAPT Immune Collaboration announces a good example of how our BD efforts broaden our portfolio as it marks our first expansion into TCRT cell therapy in solid tumor. We continue to execute on our scientific progress in our key therapeutic areas of oncology and immunology, and most notably our CAR T programs with 5101 and 5201. We have invested and continue to invest in strengthening our team in key positions globally. Finally, we benefit from a very strong balance sheet. We commit to staying disciplined in our use of cash to focus our investments to maximize value. And we want to thank our investors for their continued support as we deliver on our strategy to generate sustainable long-term value for shareholders.
spk06: Thank you, Paul and Ted. That concludes the presentation portion of today's audio conference call. I would now like to ask the operator to open up the line for Q&A.
spk07: Thank you, dear participants. As a reminder, if you wish to ask a question, please press star 1-1 on your telephone keypad and wait for your name to be announced. To withdraw a question, please press star 1-1 again. To ensure everyone has the opportunity to ask a question today, please limit yourself just to one question. Mr. Bauer will compile the Q&A role study. This will take a few moments. And now we're going to take our first question. And the first question comes to the line of Brian Abrahams from RBC Capital Markets. Your line is open. Please ask your question.
spk02: Hi there. Thanks so much for taking my question. Congrats on the progress. I'm wondering if you could maybe talk about some of the key aspects that you were able to kind of get through with regards to manufacturing, site readiness, QC to get the FDA, I guess, over the line and facilitate IND submission for 5101. And then kind of along those lines, curious what's left to be done for 5202 in order to facilitate that IND filing. Thanks.
spk05: Yeah, so we've been working actively, obviously, to prepare for some time with the tech transfer to Landmark Bio to help set up the the IND filing. We have completed that process and have just recently submitted the IND.
spk01: Let me add, Brian, we have multiple interactions with the FDA pre-IND submission in order to be able to understand what the expectation was. And so we could optimize our proposal for the FDA. And we just now submitted and is being evaluated. And we'll follow up as we get more questions through the process and hopefully finish this within the timeframe.
spk04: And then we anticipate we're going to file the 52-1 later this year.
spk01: Yeah, we first had to get, like we said before, we first had to have the transfer of technology to Landmark Bio. And so the second one, as we complete the first one, we'll start to continue with the second one, hopefully before the end of the year. That's the goal. Great.
spk02: Thanks so much.
spk07: Thank you. Just give us a moment. Now we're going to take our next question. And the next question comes to the line of Philip Nadeau from TD Cohen. Your line is open. Please ask your question.
spk00: Philip Nadeau Good morning. Thanks for taking our question. We just had one on the Parkinsonism that you mentioned for 5301. Is that similar to what's been seen for the other BCMA CAR Ts? Any difference in the severity or quality of the side effect? And can you go into a bit more detail about the protocol amendments that were submitted? Were those changes to monitoring or were there dosing changes recommended? Thanks.
spk09: Thank you. Thank you very much for the question. With regard to the papillary study in the case that you're describing, it is a feature of patients increasingly more so with the increasing use of BCMA-targeted CAR-T therapies. It's becoming more recognized. We haven't seen anything in this particular patient, which was an atypical patient presentation that is different from what is out there in the limited literature that there actually is In an abundance of caution, we ourselves undertook to pause the study and really interrogate this particular patient history. We did this by internally reviewing all the components of the patient's characteristics as well as seeking external guidance and advice, and it's consistent with what has previously been recognized. We have undertaken a number of steps, additional specific safety measures, so moving forward we feel very comfortable with the continuation of the study and in fact have submitted the protocol to the EMA in June, and we anticipate resuming the recruitment imminently. I concur with you that the changes that we've made are two components of monitoring, and I hope that answers your question.
spk00: That's very helpful. Thank you.
spk07: Thank you. Now we're going to take our next question. And the next question comes in line of Judah from Morgan Stanley. Your line is open. Please ask your question.
spk03: Yes. Hi. Thanks for taking the questions. Just a couple on adapting. I guess first, just any thoughts you can share on how the first gen of Famicel asset may have influenced your decision to partner on the next gen asset. And then secondarily, I guess from a size perspective, And from an indication perspective, I think you said this is fairly indicative of what business development efforts could look like going forward. Just could you be a little more specific on whether that's modality, indication, or just size of the deal? Thank you.
spk01: Yeah, let me say we were very impressed with the Adaptimmune team as well as with what they have been doing in the clinic and the result on Afamisel. As we saw that initially 12 months ago, even 15 months ago, when we started the discussion on the DCRT, which piqued their interest since a long time. And so that led to evaluating whether we could produce these DCRTs on our platform. It took about 12 months to do that, and came out, in my prepared remarks, Well, there's some feedback here. In my prepared remarks, that the phenotype which could create in a seven-day production process was very encouraging for potential additional benefit with the TCRT product. That led us to a further discussion and conclude a collaboration on Usacell, starting with head and neck, where they had their initial data. They remarked on four out of five patients are responders. And there we are starting now, collaborating on expanding those studies and working together on Uzacel. But yes, Afamicel was a very important driver for us to start a discussion and lead to the conclusion.
spk05: Yeah, I would just add, I think, I mean, to me, it's clear that, you know, business development is going to be key to broadening our portfolio and, of course, doing great licensing deals with great partners like Adapt Immune is one way to do that. We also continue to look at doing acquisitions as well and to try to find potential partners. We see that we have and are building a nice pipeline in cell therapy as well as small molecules. You also see our early pipeline in discovery can really be complemented through business development activities as well. Thanks.
spk07: Thank you. Now we're going to take our next question. And the next question comes from the line of Brian Abrahams from RBC, or maybe his colleague. Please ask a question. Okay, just give us a moment. There are no questions from this line. Now we're going to take our next question. And the next question comes from the line of Jason Gerbery from Bank of America. Your line is open. Please ask your question.
spk10: Hey, guys. Thank you for taking my questions. Just one quick follow-up on the protocol amendments. It sounds like I just want to confirm this is more to do on the monitoring side rather than the mitigation side, as I guess the understanding out there is that there's limited strategies you can incorporate to mitigate the severity of the Parkinsonianism. And how many patients did you dose with 5303 before you saw it, and was the onset of action consistent with the what we've seen with other CAR-Ts, which is typically kind of 90 to 120 days after the infusion. Thanks.
spk09: Yeah, thank you for the question. Yeah, just to reiterate, with regard to the changes made to the protocol, they are indeed to do with monitoring and to more closely follow the history of patients. As I said, this was an atypical patient, and the neurological component of monitoring has been further fortified. We will share imminently. Clearly, the study is about to be reinitiated. We will share all of the data regarding safety, efficacy, patient numbers in 2025. Thanks.
spk07: Thank you. Dear participants, as a reminder, if you wish to ask a question, please press star 11 on your telephone keypad. And now we're going to take our next question. And the next question comes from Sean McCutcheon from Raymond James. Your line is open. Please ask your question.
spk11: Hi, guys. Thanks for the question. Can you speak to the BRIDgene SMART A2 project and the BRIDgene approach as it compares to the other assets in the space, so maybe more broadly the landscape in SMART A2? And then beyond that, you know, what's your view on the opportunity within SMARC A4 low-frequency mutation, which drove you to select SMARC A2 as the first target molecule for the collaboration. Thanks.
spk09: Thank you for your question, Sean. As you know, SMARC A2, it is a dependency in the SMARC A4 deficient non-small cell lung cancer population. So we think 4% to 8% of the population, in fact. So the big challenge really is determining the selectivity against the SMARC A2. The problem of toxicity is quite well known, particularly cardiovascular in essence. And so looking at our report, which is really about how best to innovate and using combinatorial skill sets, the strategies that we are familiar with are really ProTech and other companies that are in this space. We have a certain expertise in small molecule oral ATPase. So the strategy, what we propose is really using the best of our own internal capabilities and knowledge and the best partner, which we believe is Bridgene with the ProTac. So really leveraging Bridgene's ProTac expertise with our own internal innovation machine, and specifically the selective small molecule ETPA's expertise. And so our intention is to build the best-in-class oral selective ProTac and to accelerate time to patients, we believe. While there are a number of companies working on different parts of this approach, we are combining both to what we believe will be a best in class product for patients. More widely, we have a significant pipeline of small molecules, which we will share more information with you in the coming months and years.
spk01: Yes, as Jeevan is saying, we have set up a team which is focused on best-in-class targets, combining a lot of expertise brought together by different experts on platforms and especially small molecules. In addition, we have the CAR-T platform, but we'll start to disclose this as soon as we go into the clinic with the first ones. At the moment, we consider this as a competitive edge for from our side to not disclose yet what type of approaches we take to the new targets we have chosen.
spk07: Thank you. Dear participants, as a reminder, if you wish to ask a question, please press star 11 on your telephone keypad. And now we're going to take our next question. And the next question comes to the line of Jacob McHale from KBC Security. If your line is open, please ask a question.
spk08: Hi there, and thanks for taking my question. I just want to come back to the adverse event that was seen in the multiple myeloma trial. Just curious what happened to the patient and if that adverse event was reversed. And perhaps a follow-up on that, given the competitive nature of the multiple myeloma space, how much of a priority is 5301 compared to your other programs and does this adverse events scene change how you think about this program at all? Thank you.
spk01: Yes, we have a principle and I think it's a clinical principle that we can't share private data on individual patients so that we can't share the status of the patient at this moment. This is the first part of this study with the dose finding, so we evaluate further as the study restarts what the doses and the safety efficacy profile will be with the fresh cell therapy production approach. And based on that, we evaluate whether there is a future for the product in the competitive landscape of the BCMA. Of course, what you have seen with CLL and NHL data as I presented, we see very encouraging data in very advanced patients who really can benefit from our approach close to the patient, fast access, as well as a high-quality cell therapy which can be given. And that's resulting in good safety and efficacy. And so the 5-101, 5-201 are key priorities. With 5-301, we are evaluating the benefit risk, benefit safety, and we'll come back to that with the first when we have our dose finding done. So all focus on making sure we start in the U.S. We start our next studies in Europe and trying to get our critical studies all started in 25 and still focusing on submission and approval in 28. Yeah, so that is the goal, what is top priority for us. Okay, thank you.
spk07: Thank you. Dear participants, just a last reminder, if you wish to ask a question, please press star 11 on your telephone keypad. Dear speakers, for further questions, I would now like to hand the conference over to Sophie Van Giesel for the closing remarks. Please go ahead.
spk06: Thank you, operator. Please feel free to reach out to the IR team if you still have questions. Our next financial results call will be our Q3 2024 results on October 31st. Thank you all for participating and have a great rest of your day.
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