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Transkript BioNTech JPMorgan "40th annual health conference" 2022-01-11
By: detwin | Date: Jan 12 2022 15:43 | Format: None | Expires: never | Size: 27.42 KB | Hits: 12

  1. BioNTech SE Presents at JPMorgan 40th Annual Healthcare Conference, Jan-11-2022 09:45 AM
  2. 1/11/22
  3. Cory Kasimov
  5. Great. Good morning or good afternoon from the 40th Annual JPMorgan Healthcare Conference. My name is Cory Kasimov. I'm the senior large-cap biotech analyst. And it's my pleasure to introduce BioNTech and CEO, Ugur Sahin, who really needs no introduction. [Operator Instructions].
  7. Thanks so much for joining us today. Let me turn things over to you for an update.
  9. Ugur Sahin
  11. So thank you, Cory. Thank you for the introduction and invitation to present at the conference. Good morning, good afternoon, and thank you to everyone listening to our presentation today. In my presentation, I'm going to start with a recap of 2021 and the historic impact BioNTech had on [ June half ]. I will then highlight our multi-platform strategy and diversified pipeline. And I will close with a review of our outlook for the coming years. After my presentation, I will be joined by other members of our management team for the Q&A session.
  13. Slide 2 to 4. Before I get started, please note that where we will be making forward-looking statements during our presentation today, as noted on Slide 2. Slides 3 and 4 provide more detailed important safety information regarding our COVID-19 vaccine.
  15. Slide 5. BioNTech was founded in 2008 on a big vision to harness the power of the immune system in new ways to fight cancer. We focus on the human immune system because it is one of the most complex and powerful systems in the human body, which plays an important role in a broad set of diseases.
  17. In early 2020, we recognized the onset of the COVID-19 pandemic as a unique global threat. We felt the responsibility to leverage our technologies to address this global health challenge, which is unlike anything we have experienced in the last century.
  19. Whilst success in doing so, has opened up an entirely new area beyond cancer, where we believe we can make also a big difference. As we embark on a new phase of growth for our company, our core vision remains unchanged. It is to harness the power of the immune system to fight human diseases.
  21. Slide 6, please. Looking back into 2021 was a historic year with a historic impact that BioNTech has made on human health and the economy around the globe.
  23. Our COVID-19 vaccine, BNT162b2 was the first ever approved mRNA therapy, which is now first to Asia in new areas of classes of medicine. This was the fastest pharmaceutical product ever developed. And I'm proud to say, one of the most successful pharmaceutical launches in history, by almost any measure.
  25. Last year, we and our partner Pfizer produced 3 billion doses and shipped about 2.6 billion doses. About 1 billion of those doses was shipped to low and middle income countries. Our efforts resulted in greater than 1 billion people fully vaccinated across 162 countries and regions. And I'm incredibly grateful and proud of the hard work of so many people at our company and our partners to make this a reality. And I feel also blessed and humbled of the human impact we have made, with millions of cases of severe illness or death likely averted along with potentially even trillions of doses in positive global economic impact.
  27. Next slide. This was not the only accomplishment last year. In 2021, we made significant progress across the company that I believe sets the stage for another impactful year in 2022. We greatly expanded our clinical pipeline with 9 oncology trials, including 4 Phase II trial starts and 5 first-in-human studies.
  29. We have also expanded our R&D organization by more than 40% to more than 850 professionals to advance our growing pipeline. Our global footprint continued to grow with the acquisition and integration of new setup in manufacturing site in Gaithersburg, Maryland in the United States, and establishment of new offices in Singapore, Shanghai and in Turkey.
  31. We successfully implemented in-house commercial quality manufacturing and scaled up our manufacturing production capacity to a global commercial scale. With the Gaithersburg site addition, we have now established cell therapy manufacturing capabilities on both sides of the Atlantic. We also deployed our first commercial sales force in Germany, where we are responsible for distributing BNT162b2.
  33. On the next slide, looking back at 2021, we generated a strong financial performance. This positions us to accelerate our vision to transform medicine by reinvesting the COVID-19 vaccine costs into our company and advancing our broad pipeline immunotherapies toward patients around the world.
  35. Our ability to provide vaccine to so many people in 2021 was the direct result of our early efforts to scale up manufacturing to an annual production capacity together with our partner, Pfizer, of 3 billion vaccine doses. As a result, we estimate that as of December of 2021, BNT162b2 will help 70% -- 74% of market share in the United States and approximately an 80% market share in Europe.
  37. Turning to the outlook for COVID-19 vaccine revenues. We have guided to full year 2021, COVID-19 vaccine related revenues in the range of EUR 16 billion to EUR 17 billion. Additionally, we expect continued strong demand this year and estimate full year 2022 COVID-19 vaccine-related revenues in the range of EUR 13 billion to EUR 17 billion.
  39. On Slide 9, I would like to emphasize our structure. We are a 21st century immunotherapy powerhouse built on 3 pillars, which are centered around our core vision. The first is our fully integrated model. that combines world-class translational discovery, development, GMP manufacturing, commercial capabilities all under one roof.
  41. Second, we employ a multi-platform strategy, powered by a technology-agnostic innovation engine with strong leadership competencies in emerging technologies like mRNA and synthetic biology. That innovation engine has generated a diversified product pipeline of 1 approved vaccine, 16 clinical stage product candidates and over 30 programs.
  43. We take our approach to global social responsibility seriously. It is part of our DNA. It is core to who we are as a company. We believe that people all around the globe should benefit from advances in innovation, not just those in the wealthiest countries. As we enter into this new era of mRNA technology and synthetic biology, we remain focused on our approach of making impact on global health. This means addressing diseases with high medical need by innovation and democratizing access to novel medicines.
  45. On Slide 10, I would now like to take time to explore the aspects of BioNTech that enable our continued success, starting with our multi-platform strategy. On the next slide, we show a depiction of the broad toolbox we are building across our technology platform, which includes a diverse range of potentially first-in-class therapeutic approaches. This includes, on the left, various mRNA vaccine platforms, engineered T-cell receptor and CAR-T cell therapies, targeted antibodies, small molecule immune modulators, ribologicals that means mRNA encoded molecules such as antibodies and cytokines and next-generation checkpoint immunomodulators that combine immune checkpoint blockade and co-stimulatory activity.
  47. Turning to Slide 12. We are entering a new era of mRNA technology and synthetic biology, the door to which has been opened by the success of our vaccine. We believe that mRNA's impact on medicine could be similar to the introduction of recombinant protein expression technology in the pharmaceutical industry exactly 40 years ago. We are at the forefront and are helping to drive this transformation. mRNA is now a validated new drug class. We have pioneered and developed multiple approaches to use mRNA to deliver a variety of biologically active molecules with therapeutic potential, some of which can be seen here, right?
  49. Initially, we focused our efforts in oncology and infectious disease, we continue to see tremendous potential for mRNA to reach beyond those therapeutic areas as evidence by our publication in Science in January of last year, establishing mRNA for potential in autoimmune diseases.
  51. We also believe that mRNA therapeutics could help treat inflammatory diseases, cardiovascular, neurodegenerative diseases have the same applications in regenerative medicine. With all of these applications in mind, we believe that in 15 years, 1/3 of all newly approved drugs will be based on mRNA.
  53. On the next slide, at BioNTech, we are poised to drive that trend with significant pipeline progress in 2022. On Slide 14, you see our goals for 2022. We expect 2022 to be a year of tremendous expansion and maturation of our pipeline as we ramp up our investment. We aim to continue our global leadership in COVID-19 vaccines with multiple new product launches and label expansions, new formulation, pediatric dosage forms and potentially variant specific vaccines.
  55. In oncology, we are having 5 ongoing randomized Phase II trials across a range of solid tumor indications. We also expect to have 5 mRNA infectious disease vaccines in human priced by the year-end, including the influenza vaccine. Finally, we will also continue to invest in broadening our impact by expanding our platforms into new areas of unmet need, including cardiovascular, neurodegenerative and autoimmune diseases.
  57. On Slide 15, in 2022, we anticipate another year with strong demand for our vaccine. We have planned a 4 billion dose manufacturing capacity for the year. We expect demand to come from multiple drivers. Our aim to provide equitable access with 2 billion doses pledged to low and middle income countries through 2022. Doses for the unvaccinated, pediatric doses for nearly 25% of the global population, which is under 14 years old.
  59. Third booster for those who qualify. And if necessary, and we currently believe it is necessarily a variant specific vaccine.
  61. We are starting a clinical trial of our Omicron vaccine by end of January. In parallel, we have already started manufacturing for commercial scale the of Omicron vaccine. We anticipate to be ready for market supply by March 2022, subject to regulatory approval.
  63. Slide 16. The vast majority of scientists believe that COVID-19 will be in the long run become an endemic disease. We believe we will move to a long haul toward an influenza like vaccination paradigm with both periodic boosters against new variants and more routine boosting and vaccination in certain populations, but we are not yet there. There are clear differences between influenza and this corona virus. We can't make safe predictions today. And the reason is that we have to consider multiple factors. There's a huge global SARS-CoV-2 virus reservoir, which drives fast evolution of the virus and the virus is still in its infancy. There are new variants emerging every 3 months, some of them rapidly spreading at a global level.
  65. We can't get ignore mild and modulate disease. There appears to be a risk for long COVID and organ damage that is even associated with mild and moderate infections. This will have a significant impact on infrastructure and economy. And we need to consider other factors, the waning of protection over time in the broader population, for children vaccination addresses the risk of COVID-19-related multi-inflammatory syndrome, and vaccines ensuring the prevention of infection might be particularly relevant for frontline workers.
  67. And of course, we have to pay special attention to protect individuals with underlying or chronic health conditions who are at risk or increased risk of severe disease.
  69. On the next slide, the COVID-19 pandemic is highly dynamic. There are more than 10,000 novel variant sequences currently discovered every week. Human experts simply cannot cope with the complex data at this scale. To address this challenge, we have developed an AI-based early warning system in collaboration with our partner InstaDeep. Our approach combines structural modeling of the virus spike protein in the machine learning algorithms to analyze alteration of antibody binding epitopes.
  71. On the right side, you can see in red the changed epitopes for the better coronavirus variant and on the bottom to the Omicron variant. Our AI system identifies and scores potential high risk immune escape variants. The only information which is needed the sequence data from SARS-CoV-2 sequence repositories. The system is able to evaluate new variants in minutes and ensures risk monitoring of variant lineages in near real time.
  73. During the prior period, the system has identified 90% of the WHO designated variance on average 2 months in advance. We are happy to make this tool publicly available to support early detection and quick response to new variants and we will continue to improve it.
  75. In the next slide, we want to rapidly advance our programs, addressing diseases that continue to have a major impact on global population health and disproportionally impact developing countries such as malaria, which the WHO estimate has 229 million cases and 409,000 deaths annually.
  77. Tuberculosis, which the WHO estimates, 10 million people contracted in 2019 and HIV, which has more than 35 -- which affects more than 35 million people worldwide. It is not enough to successfully develop this innovation. We also have to take the appropriate steps to ensure access in the areas that need the most. To that end, we are now expanding our COVID-19 manufacturing network in Africa and South America, and we aim to establish state-of-the-art mRNA manufacturing sites in Africa and in Asia in the middle of this year, starting in the middle of this year.
  79. Finally, our infectious disease toolkit includes multiple technology platforms, including mRNA vaccines, ribologicals and a new class of precision anti-bacterials, synthetic lysins, which we added to our platform technology last year.
  81. Next slide. We have rapidly expanded our infectious disease pipeline on the back of our COVID-19 vaccine success. Our pipeline now includes 3 mRNA vaccines partnered with Pfizer and 10 other wholly-owned programs.
  83. In 2022, we will accelerate our infectious disease pipeline development to aim -- and aim to have 5 clinical stage programs. This includes our third collaboration with Pfizer, which we announced last week to develop the first mRNA shingles vaccines, which we expect to enter clinic in the second half of the year.
  85. Shingles is a debilitating and painful disease, which is the chronic recurrent form of the varicella zoster virus infection, which cause chickenpox infection. The virus can lay dormant in human nerve cells and can reactivate in life due to potentially leading to postherpetic neuralgia and in rare cases, facial paralysis, deafness and blindness. While they are currently approved vaccines for shingles, there's a need to develop an improved mRNA a vaccine that potentially combines high efficacy and better tolerability and is more efficient to produce globally.
  87. Alongside our vaccines developed with our partner, Pfizer, we are developing additional candidates in collaboration with the Bill & Melinda Gates Foundation from malaria and tuberculosis. The first of which is supposed to enter clinical stage in the second half of this year. We also plan to initiate clinical trials for our mRNA vaccine for HSV 2 in the second half of the year.
  89. Slide 20, please. Last year, we have advanced multiple programs into randomized Phase II studies and initiated 5 first-in-human clinical trials. In December of last year, our randomized Phase II trial for BNT311, our PD-L1x4-1BB bispecific antibody developed in collaboration with Genmab, evaluating it as a monotherapy and in combination with pembrolizumab in patients with checkpoint inhibitor relapsed or refractory metastatic non-small cell lung cancer.
  91. We have Phase II clinical trials ongoing with our off-the-shelf FixVac therapeutic vaccine candidates, BNT111 and BNT113 in advanced melanoma and in combination with Regeneron's PD-1 inhibitor Libtayo and in HPV16 head and neck cancer in combination with pembrolizumab respectively.
  93. For our personalized neoantigen targeted mRNA cancer vaccines, BNT122, developed in combination with Genentech, we have 2 ongoing Phase II trials in adjuvant setting for the treatment of advanced melanoma in combination with pembrolizumab and in postsurgical resection CTD [indiscernible] cancer as a monotherapy. Each of this program in Phase II have demonstrated single-agent activity.
  95. We continue to believe it is important to explore an established single agent activity as early as possible in clinical development. We'll continue to rapidly advance and expand our oncology pipeline this year. While we don't have time to highlight all of the amazing work behind each of these programs, I would like to discuss one program for which we have recently presented new clinical data, BNT211. Growth in 6 CAR-T cells are equipped with a second-generation chimeric antigen receptor of higher sensitivity and specificity for the tumor-specific antigen Claudin 6. Claudin 6 is absent in healthy adult tissue and frequently expressed in high medical need cancers, making this antigen as an ideal candidate for CAR-T setup. Our preclinical studies demonstrated that CARVac drives in vivo expansion of transferred CAR-T cells, increasing their persistence and efficacy. Based on our preclinical studies, we anticipate that BNT211 could overcome CAR-T cell therapy limitations in patients with solid tumors.
  97. On the bottom right of the slide, you can see that the sign of the first-in-human Phase I dose escalation trial, which evaluates the safety and efficacy of the Claudin 6 CAR-T cell monotherapy and in combination with the mRNA vaccine as CARVac. The clinical trial is performed in patients with Claudin 6 positive relapsed or refractory advanced solid tumors.
  99. The Part 1 comprise Claudin 6 CAR-T cell monotherapy dose escalation cohort. And Part 2 is a dose escalation of CAR-T cell combined with a fixed dose of CARVac. There are 3 dose levels for each part. The subsequent dose expansion will focus on ovarian, testicular and endometrial cancers as well as other Claudin 6 positive cancers.
  101. Slide 22. At the ESMO-IO Virtual Annual Meeting in December, we presented updated data seen on Slide 22, which is highlighted, which highlighted 15 extensively pretreated patients suffering from different tumors treated with BNT211. Let me quickly summarize the data for you. While we continue to improve the lymphodepletion regimen, Claudin 6, CAR-T cells as monotherapy or combined with CARVac were well tolerated.
  103. So far, we have observed a single dose-limiting toxicity DLT case, namely prolonged cytopenia in a patient with recent history of high-dose chemotherapy followed by a stem cell transplantation. And as a consequence, we have opened up the lymphodepletion 3 cohort.
  105. Mild forms of CRS were seen in 7 of 15 patients and were manageable. We have adapted the CARVac scheduled accordingly. Interestingly, 7 patients experienced CRS Grade 1 and 2, accompanied with high IL-6 levels which was manageable with IL-6 inhibitor without signs of neurotoxicity.
  107. With respect to efficacy, we would like to highlight a robust engraftment of CAR-T cells that was seen in all patients. In 9 of 10 patients evaluable for efficacy assessment, we have observed initial disease control including 4 partial responses, 3 of which occurred in patients with testicular cancer with recently relapsed after transplantation.
  109. Slide 23. You can see the progression of all 15 dose patients along with examples of 2 patients with CT scans, who have demonstrated significant too much shrinkage post infusion with BNT211. The first patient who received the dose level 1 boosted with CARVac showed an ongoing response on numerous metastasis. The second patient depicted here, has received those levels 2 as monotherapy and his high tumor burden in liver and multiple other tissues shrank tremendously, in line with the decrease of the tumor marker AFP of more than 99%. These are, of course, very early data and we await data updates from which we will focus on persistence, especially in combination with CARVac.
  111. Slide 24. To wrap up, in 2022, we plan to continue to realize this once in a generation opportunity to transform medicine. We will continue to address the challenges of COVID-19 around the world with additional formulations, pediatric dosage launches while continuing to develop our Omicron vaccine variant. We will continue to accelerate our late-stage oncology programs toward the market and expand our earlier stage pipeline. This pipeline growth will be enabled by continued R&D investment and further strategic investment in cutting edge digital technologies and competencies. And we will complement this by licensing acquisitions of synergistic technologies, infrastructure and product candidates.
  113. We will expand our global organization in Europe, in the United States, Asia and Africa, so that we can continue to bring long-term value to patients, shareholders and the society. 2021 set the stage for us and enabled by our success of last year, I feel more than optimistic than ever about our future.
  115. Thank you. And I'm opening the question-and-answer session. And I would like to ask my colleagues to join this part of the session.
  117. Cory Kasimov
  119. All right. Terrific. Thank you, Ugur, and welcome to the rest of the team. So we have just about 10 minutes for Q&A. [Operator Instructions]
  121. To start, though, I want to ask if you can talk more about the perceived value of an Omicron specific vaccine. If it's out by March, by the time a meaningful number of people are vaccinated against this variant, wouldn't that be too late to have a meaningful effect against Omicron?
  123. Ugur Sahin
  125. It is difficult to answer this question. We do not know how much immunity is associated with an Omicron infection. Because we see that most of the infections are mild. It is quite possible that even an infection less Omicron requires an additional boost with an Omicron to ensure a prolonged protection.
  127. Cory Kasimov
  129. Okay. So then a question from our portal is, should those eligible for a fourth booster in countries like Israel, wait for an Omicron specific vaccine in your opinion?
  131. Ugur Sahin
  133. This is a difficult-to-answer question. We have just to see what is the value of a fourth dose and we have, of course, to see -- to wait for the further dynamics of the pandemic. It is really difficult to see what -- to predict what is going to happen in the next 2 to 3 months.
  135. Cory Kasimov
  137. Okay. And then Ugur, what's your view and thoughts around the value of a potential multivalent vaccine? And where are you on that front?
  139. Ugur Sahin
  141. This is -- we see that, of course, as an opportunity. But at the moment, we have a situation. We have a situation where we do not know what is the next variant and Omicron infection and an Omicron vaccine will most likely with a high probability, also boost immune responses against all existing variants. So the question is, what is the value if we now combine an -- potential Omicron vaccine with another variant as the Omicron vaccine alone would most likely do the job.
  143. Cory Kasimov
  145. Okay. And then a question regarding the current guidance for 2022. Is that just based on existing advance purchase agreements?
  147. Ugur Sahin
  149. Jens, could you take the question or Sean?
  151. Jens Holstein
  153. Yes. Sean, do you want to take that question or I'll...
  155. Sean Marett
  157. Yes. You go ahead, Jens.
  159. Jens Holstein
  161. Yes. So I mean, of course, right now, we have around about 2 billion of contracts signed and options on this. We expect further contracts to be signed in the months to come and throughout the year, of course, also for '23 and then '24. So as we -- and Ugur pointed it out, as we expect that the pandemic might become an endemic over time.
  163. But at the end of the day, there is a high likelihood that to a certain extent, the area of additional vaccinations needed. So we have included additional volumes in that guidance. But we feel, I have to say also very confident of that we reached this guidance as we are confident about the 2021 figures.
  165. Cory Kasimov
  167. Okay. And I have a couple of questions in the portal about progress you're making in China. And what are the potential time lines to approval there?
  169. Ugur Sahin
  171. Sean, would you like to take the question or Ryan?
  173. Sean Marett
  175. Yes. So we're continuing to conduct a Phase II trial in China. And it is a continued dialogue with the Chinese authorities. It's very difficult to predict actually when we will get approval. And but China remains for us an extremely important market and we're very, very committed to it.
  177. Cory Kasimov
  179. Okay. And then another question we are keep getting is, what are your thoughts in terms of the time lines to the pandemic turning endemic? And what do you think an endemic COVID looks like?
  181. Ugur Sahin
  183. Ozlem, would you like to take the question?
  185. Özlem Türeci
  187. Yes, sure. Again, as Ugur pointed out earlier, very difficult to predict how the dynamics of COVID-19 will be. We do not know what variants will occur, how they will escape in a broader way immunity. So that the only thing we can say is, at some point, we will reach the paradigm of an endemic disease, but there will be quite a path towards reaching that.
  189. Cory Kasimov
  191. Okay. And then I wanted to ask for more on, if you could talk more about this early warning system to detect emerging variants. I found that to be very interesting. How do you see something like this implemented in the real world?
  193. Ugur Sahin
  195. Yes, it is important. We need this surveillance system and digital tools to enable rapid understanding what is going on. We have seen that different variants could emerge from different regions. And it is nearly impossible to continue to monitor that and timely respond. Therefore, we need to combine the information which are collected in the sequence repositories. This type of digital tools, implement all the know-how also with regard to example of understanding of the impact of certain mutations. And thereby, ensure that these tools can be used by researchers but also by health service providers and drug developers to make decisions earlier on where the new vaccines should be implemented and have more time for manufacturing of vaccine variants.
  197. Cory Kasimov
  199. Okay. We have another China-related question here in the portal. What is the level of interest for mRNA-based vaccines in China, particularly in the face of the fact that the Chinese vaccines do not result in much immunity against on the Omicron variant?
  201. Ugur Sahin
  203. Ryan, would you like to take the question?
  205. Ryan Richardson
  207. Sure. Yes. I mean we think there is significant interest in mRNA vaccines in China and indeed our market research supports that view. There are a number of locally developed mRNA vaccines that are in clinical trials currently. Of course, there's no approved vaccine in China based on mRNA technology yet. But we've had very positive signals, both from both from the population research we've done, but also from government officials and the regulatory bodies in the country.
  209. And I think the question is, as we mentioned, is how do we open up the regulatory pathway in Mainland. I would note, we do have approval and have had for most of 2021 authorization in Hong Kong and Macau, and we've seen very strong uptake of our vaccine in that region.
  211. Cory Kasimov
  213. Okay. Well, unfortunately, we are out of time. We have a ton of questions in the portal, I'll try to get to those offline. But thank you guys all very much for your time and for all that you're doing. So appreciate it and best of luck with the conference for the rest of the week.
  215. Ugur Sahin
  217. Thank you, Cory.
  219. Ryan Richardson
  221. Thank you.
  223. Özlem Türeci
  225. Thank you.
  227. Jens Holstein
  229. Thank you.