J.P. Morgan Healthcare Conference January 2020 Ugur Sahin, MD CEO and Co-Founder Exhibit 99.1
This slide presentation includes forward-looking statements Forward-Looking Statements Various statements in this slide presentation concerning the future expectations of BioNTech, its plans and prospects, including the Company’s views with respect to the potential for mRNA therapeutics, its expectations with respect to the timing and results of clinical trials and release of clinical data (both in respect of its proprietary product candidates and of product candidates of its collaborators), the development of commercial capabilities and the transition of BioNTech to a fully integrated biopharmaceutical company, its expectations with respect to interactions with regulatory authorities such as FDA and EMA, including the potential approval of BioNTech’s or its collaborators’ current or future drug candidates, and expected royalty and milestone payments in connection with BioNTech’s collaborations, constitute forward-looking statements. Words such as "expects," "plans," "potential," "target," "continue" and variations of these words or similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are based on the current beliefs and assumptions of the management team of BioNTech and on the information currently available to the management team of BioNTech, and are subject to change. The Company will not necessarily inform you of such changes. These forward looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that could cause the Company’s actual results, performance or achievements to be materially different than any future results, performance or achievements expressed or implied by the forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the initiation, timing, progress, results and cost of the Company's research and development programs and its current and future preclinical studies and clinical trials; the timing of and the Company's ability to obtain and maintain regulatory approval for its product candidates; the Company's ability to identify research opportunities and discover and develop investigational medicines; the Company's expectations regarding the size of the patient populations for its product candidates, if approved for commercial use; the Company's estimates of its expenses, ongoing losses, future revenue and capital requirements and its needs for or ability to obtain additional financing; the Company's ability to identify, recruit and retain key personnel; the Company's and its collaborators' ability to protect and enforce its intellectual property protection for its proprietary and collaborative product candidates, and the scope of such protection; the development of and projections relating to the Company's competitors or its industry; the Company's ability to commercialize its product candidates, if approved; the rate and degree of market acceptance of the Company's investigational medicines; the Company's ability to manage its development and expansion; regulatory developments in the United States and foreign countries; the Company's ability to manufacture its product candidates with advantages in turnaround times or manufacturing cost; and the Company's ability to implement, maintain and improve effective internal controls. The preceding list is not intended to be an exhaustive list of all of the Company's forward-looking statements. Any forward-looking statements represent the Company's views only as of today and should not be relied upon as representing its views as of any subsequent date. The Company explicitly disclaims any obligation to update any forward-looking statements. The mRNA vaccines and other product candidates discussed in this slide presentation are investigational products being developed by BioNTech and its collaborators and are not currently approved by the FDA, EMA or any other regulatory authority.
Agenda Who we are and what we do Our platforms and programs Outlook for 2020 and beyond +
Building a 21st century individualized immunotherapy company Executive summary Large addressable market opportunity in solid tumors More than USD 90bn solid tumor market3 addressed Commercialization or co-commercialization rights retained in key geographies World-leading collaborators 7 pharmaceutical collaborators and multiple leading academic institutions 50:50 cost and profit share agreements with leaders in oncology1 Broad & diversified pipeline 10 product candidates in the clinic First registrational trial expected to start in 20202 Next generation immunotherapies for cancer and other diseases Technology agnostic approach Exploiting novel targets and mechanisms Vertical Integration with in house manufacturing Up to 7 clinical data updates expected in the next 18 months 1with Genentech and Genmab; 2BNT111; 3Source: Global Data Total WW Market, top 10 available products 2018-2024 + other
Our Vision: We aspire to individualize cancer medicine Overview mRNA Therapeutics Engineered Cell Therapies Antibodies Small Molecule Immunomodulators Off-the-shelf drugs Interindividual variability Tailored-on-demand immunotherapies Clinical samples Sample profiling bioinformatics big data, deep data AI platforms Drug classes In-house diagnostics & bio-informatics Multi-drug platform approach Off-the-shelf drugs and individualized therapies In-house manufacturing with on-demand production capabilities A patient centric approach
We collaborate with global leaders in our industry Strategic collaborations Oncology Collaborations with at least one program in the clinic 50:50 Cost and Profit share (2016) 50:50 Cost and Profit share (2015) Cost and Profit share (2015) Other Oncology, Infectious Diseases and Rare Diseases Collaborations Co-development Co-commercialization (2018) Licensing Agreement (2018) Strategic R&D Alliance (2018) R&D Agreement (2019) Licensing Agreement (2015) UPenn
Targeting Cancer Immunomodulation mRNA Cancer Vaccines Antibody Targeting Small Molecule Immunomodulators Next Generation Immunomodulators FixVac, iNeST Targeted Antibodies RiboMabs Engineered Cell Therapies CAR-T, TCRs Bispecific Antibodies (CPI + co-stimulation) TLR agonist Engineered Cytokines Intra-tumoral cytokines RiboCytokines Our IO strategy exploits complementary therapeutic platforms Our multi-platform approach + We expect to have all core platforms in the clinic by the end of 2020 1 2020 In the clinic 1 1 1 2020
A technology agnostic approach increases our addressable market Solid tumor market segmentation Cancer segment Patient Population Challenge Our Therapeutic Strategy High mutational burden/ adjuvant stage cancers Significant portion of cancer patients Poor risk-benefit profile of checkpoint inhibitors mRNA Neoantigen Immunotherapy (iNeST) Low mutational burden cancers >60% of cancers Poor response to checkpoint inhibitors Shared Antigens (FixVac, CAR-T cells, Antibodies) “Immune desert” cancers >40% of high-mutational cancers Poor infiltration and activation of T-cells in TME1 mRNA Immunotherapy Immunostimulatory Compounds (intratumoral, RiboCytokines) Cancers with MHC / B2M loss 20-30% of CPI-experienced advanced cancers Failure of immune system to recognize tumor cells Antibodies CAR-Ts Refractory tumors Patients with large tumors and multiple resistance mechanisms Few treatment options Engineered Cell Therapies Combination Therapies Portfolio approach based on molecular classification and segmentation of cancer types 1Tumor microenvironment
One of the broadest mRNA toolkits in the industry Local Intratumoral Systemic Tissue specific Adjustable activity in vivo from minutes up to weeks Various delivery routes Liposomes / LPX LNPs Polyplexes Broad formulation spectrum Multiple mRNA formats uRNA modRNA saRNA taRNA 5’ Cap 5’ UTR ORF 3’ UTR Poly(A) tail mRNA platform
Additional late stage trial starts planned for FixVac and iNeST in 2020 Our pipeline Drug Class Platform Product Candidate Indication (Targets) Preclinical Phase 1 Phase 2 Rights Collaborator Milestones Oncology mRNA FixVac (fixed combination of shared non-mutated cancer antigens) BNT111 advanced melanoma (adjuvant & metastatic) fully-owned report phase 1 data and phase 2 start 1H 2020; phase 3 start 2H 2020 BNT112 prostate cancer fully-owned first patient enrolled in phase 1/2 in Dec 2019 (plan: 2H 2019) BNT113 HPV16+ head and neck cancer1 fully-owned phase 2 start 2H 2020 BNT114 triple negative breast cancer fully-owned data update 1H 2020 BNT115 ovarian cancer1 fully-owned first patient dosed in Dec 2019 BNT116 NSCLC fully-owned - iNeST (patient specific cancer mutated antigen therapy) RO7198457 (BNT122) 1L melanoma with CPI2 Genentech (global 50:50 profit/loss) top line data 2H 20203 multiple solid tumors data update 2020 Intratumoral Immunotherapy SAR441000 (BNT131) solid tumors (IL-12sc, IL-15sushi, GM-CSF, IFNα) Sanofi (global profit/ loss share) data update 2H 20204 1BNT113 and BNT115 are currently being studied in an investigator-initiated phase 1 trials; 2Checkpoint Inhibitor; 3We expect this topline data update to include an update on the ongoing study, including patient enrollment numbers, with full efficacy and safety data for an interim update expected in the second half of 2021; 4As the trial is sponsored and conducted by Sanofi, the timing of data updates is not under our control, and is subject to change by Sanofi. new new new
We plan to start first-in-human trials for RiboMabs & RiboCytokines in 2020 Our oncology pipeline Drug Class Platform Product Candidate Indication (Targets) Preclinical Phase 1 Phase 2 Rights Collaborator Milestones Oncology mRNA RiboMabs (mRNA-encoded antibodies) BNT141 multiple solid tumors fully-owned phase 1 start 2H 2020 BNT142 multiple solid tumors (CD3+CLDN6) fully-owned phase 1 start 2H 2020 or 1H 2021 RiboCytokines (mRNA-encoded Cytokines) BNT151 multiple solid tumors (optimized IL-2) fully-owned phase 1 start 1H 2020 BNT152+ BNT153 multiple solid tumors (IL-7, IL-2) fully-owned phase 1 start 2H 2020 or 1H 2021
We now have 3 antibodies in clinical testing Our oncology pipeline Drug Class Platform Product Candidate Indication (Targets) Preclinical Phase 1 Phase 2 Rights Collaborator Milestones Oncology Engineered Cell Therapies CAR-T Cells BNT211 multiple solid tumors (CLDN6) fully-owned phase 1/2 start 1H 2020 BNT212 pancreatic, other cancers (CLDN18.2) fully-owned - TCRs Undisclosed undisclosed Eli Lilly (exclusive license) - To be selected all tumors fully-owned - Antibodies Next-Gen CP5 Immunomodulators GEN1046 (BNT311) multiple solid tumors (PD-L1×4-1BB) Genmab (global 50:50 profit/loss) data update 1H 2021 GEN1042 (BNT312) multiple solid tumors (CD40×4-1BB) - Targeted Cancer Antibodies BNT321 (MVT-5873) pancreatic cancer (sLea) fully-owned patient enrolled to resume phase 1 in Dec 2019 SMIM6 Toll-Like Receptor Binding BNT411 solid tumors (TLR7) fully-owned phase 1 start 1H 2020 5Checkpoint; 6Small Molecule Immunomodulators new
2019 Highlights Year in review Pipeline Initiated clinical trials for 6 Investigational Medicinal Products (IMPs) across various cancer indications Started first randomized Phase 2 trial for iNeST Dosed more than 440 patients across all BNTX programs1 as of end 2019 Corporate Raised $225m in Series B financing and $149m in Nasdaq IPO Signed two additional agreements with Bill & Melinda Gates Foundation and Regeneron Purchased site for building new iNeST manufacturing facility and initiated planning and design work Management Team Agreed on new Management Board Member and Chief Strategy Officer (appointment of Ryan Richardson on Jan 12, 2020) 1 BNTX programs: all BioNTech trials including trials sponsored by collaborators
Agenda Who we are and what we do Our key platforms and programs mRNA vaccines – FixVac and iNeST Antibodies CARVac platform – CLDN6 CAR-T RiboCytokines Outlook for 2020 and beyond
Our mRNA vaccine platforms: FixVac and iNeST mRNA drug class | vaccine platforms Off-the-shelf mRNA immunotherapy Targeting a fixed combination of shared antigens Non-mutated antigens shared among patients with a specific cancer type Applicable for almost all types of tumor antigens FixVac iNeST FixVac Fully individualized mRNA immunotherapy Targeting 20 neo-antigens unique to each patient Vast majority of neo-antigens are unique to individual patients Applicable across solid tumor types Proprietary RNA-LPX formulation for systemic dendritic cell targeting Strong immunogenicity observed in vivo via TLR7-driven adjuvant effect Potent induction of strong ex vivo CD4+ and CD8+ T cell responses Kranz et al., Nature 2016
. 2.1% 10.1% 10.3% Our RNA-LPX vaccine approach mRNA drug class | vaccine platforms Strong vaccine-induced ex vivo CD8+ T cell responses1 across different cancer types FixVac iNeST HPV16-E7 Head Neck Cancer BNT113, HARE40 trial Mutant Neoantigen TNBC BNT114, TNBC MERIT trial MAGE-A3 Melanoma BNT111, Lipo-MERIT trial NY-ESO-1 Melanoma BNT111, Lipo-MERIT trial 5.0% 1T cell responses analyzed by ex vivo multimer staining analysis in blood
FixVac: BNT111 interim clinical activity data (dose range 14µg -100µg) 1CPI = Checkpoint Inhibitor, 2based on 18F-FDG-PET/CT analysis mRNA drug class | FixVac platform | BNT111 (FixVac advanced melanoma) Summary Advanced melanoma patients (Stage IIIB,C, Stage IV) Out of 74 patients with follow-up imaging 42 patients were eligible for exploratory analysis of objective responses as of July 29, 2019 25 patients with pretreated and CPI1-experienced metastatic melanoma who received BNT111 monotherapy 3 patients with partial response (PR) 1 with metabolic complete remission2 7 patients with stable disease (SD) 14 progressive disease (PD) 17 patients with CPI-experienced metastatic melanoma who received BNT111 in combination with CPI 6 patients with partial response (PR) 2 patients with stable disease (SD) 9 patients with progressive disease (PD) Adjuvant cohort of 32 patients still in study Shared Antigens Targeted NY-ESO-1 / MAGE-A3 / Tyrosinase / TPTE
BNT112: FixVac Prostate Cancer mRNA drug class | FixVac platform | BNT112 (FixVac prostate cancer) Ph1/2: first patient enrolled in December 2019 Multipronged vaccine: Targeted antigens of BNT112 are 5 prostate cancer specific antigens (PAP, PSA and 3 undisclosed antigens) RNA-LPX vaccine format validated by our FixVac Melanoma program Antigen 1 PSA PAP Antigen 4 Antigen 5 Antigen 1 PSA PAP Antigen 4 Antigen 5
FixVac: an expanding pipeline of clinical stage programs Product candidate mRNA drug class | FixVac platform FixVac Pipeline Advanced melanoma HPV positive head & neck cancer (IIT) Triple negative breast cancer Prostate cancer BNT111 BNT113 BNT114 BNT112 BNT115 Preclinical Phase 1 Phase 2 Ph. 3 start H2 2020 Ph. 2 start 2H 2020 5 programs in human trials NSCLC BNT116 Ovarian cancer (IIT)
Conclusions from iNeST clinical trials mRNA drug class | iNeST platform | clinical trials Clinical efficacy evaluation in randomized phase 2 trials initiated Long-term follow-up of completed trial with BNT121 (Intra-nodal administration, RNA): Long-term relapse free disease activity with BNT121 iNeST in adjuvant melanoma Preliminary observations in ongoing trials with BNT122 (RO7198457) (IV administration, RNA-LPX): iNeST can be manufactured for individual patients with clinically relevant turn-around times across a range of tumor types iNeST +/- atezolizumab (Tecentriq) has a manageable safety profile Strong iNeST immunogenicity across a range of tumor types
Update for BNT121 (as of October 2019) mRNA drug class | iNeST platform | clinical trials Metastatic relapse analyses 9 of 13 patients without documented PFS Events Melanoma Stage IIIb, IIIc, and IV, 13 patients, intranodal delivery against 10 neoantigens Stable progression free survival in adjuvant melanoma Sahin et al., Nature 2017
A technology agnostic approach increases our addressable market Solid tumor market Cancer segment Patient Population Challenge Our Therapeutic Strategy High mutational burden/ adjuvant stage cancers Significant portion of cancer patients Poor risk-benefit profile of checkpoint inhibitors mRNA Neoantigen Immunotherapy (iNeST) Low mutational burden cancers >60% of cancers Poor response to checkpoint inhibitors Shared Antigens (FixVac, CAR-T cells, Antibodies) “Immune desert” cancers >40% of high-mutational cancers Poor infiltration and activation of T-cells in TME mRNA Immunotherapy Immunostimulatory Compounds (intratumoral, RiboCytokines) Cancers with MHC / B2M loss 20-30% of CPI-experienced advanced cancers Failure of immune system to recognize tumor cells Antibodies CAR-Ts Refractory tumors Patients with large tumors and multiple resistance mechanisms Few treatment options Engineered Cell Therapies Combination Therapies Portfolio approach based on molecular classification and segmentation of cancer types
Individualized Neoantigen Specific Immunotherapy (iNeST) mRNA drug class | iNeST platform | clinical trials Overview Targeting multiple neoantigens Intended to be a universal approach applicable for the majority of cancers 50:50 profit/loss share with Genentech Turnaround time reduced from three months to six weeks BNT121 (i.n.) BNT122 (IV) Preclinical Phase 1 Phase 2 Up to 41 mon follow-up data Data update 2020 Top line data 2H 2020 New planned trial expected to start in 2020 Multiple solid tumors Metastatic melanoma (N=13) First-line advanced melanoma in combination with pembrolizumab (Keytruda) First-line adjuvant solid cancer in combination with atezolizumab (Tecentriq) First-line adjuvant solid cancer Currently being evaluated in ≥ 8 solid tumor indications
Digitization and automation for neo-antigen vaccine manufacturing Paperless documentation Semiautomatic manufacturing 2 mRNA GMP production facilities: Idar-Oberstein (GMP since 2011) and Mainz (GMP since 2018) Construction and GMP licensure of new Mainz facility for iNeST expected in 2022/2023 Partnered with Siemens to develop automated production processes mRNA drug class | iNeST platform | clinical trials
Agenda Who we are and what we do Our platforms and programs mRNA vaccines – FixVac and iNeST Antibodies CARVac platform – CLDN6 CAR-T RiboCytokines Outlook for 2020 and beyond
BNT311 (GEN1046) BNT312 (GEN1042) Next-Gen checkpoint immunomodulators PD-L1 4-1BB K409R F405L Fc-silencing mutations matched CH3 mutations L234F L235E D265A CD-40 4-1BB K409R F405L Fc-silencing mutations matched CH3 mutations L234F L235E D265A Two bispecific antibodies partnered with Genmab Potential “first-in-class” bispecific antibodies Conditional activation of immuno-stimulatory checkpoint activity 50:50 profit/loss share Both programs are now in the clinic Preclinical Phase 1 Phase 2 PD-L1x4-1BB Product Candidate CD-40x4-1BB Ph1/2a Ph1/2a Data update 1H 2021 Antibodies drug class | bispecific antibodies
Characteristics Bi-specific antibody combining constitutive CPI blockade and conditional co-stimulatory activity Enhanced proliferation of antigen specific activated T cells in the presence of PD-L1-positive cells Constitutive PD-L1 blockade & Conditional 4-1BB agonism 1 2 Increased tumor infiltrating lymphocyte (TIL) expansion in human tumor tissue cultures ex vivo 3 Induced tumor regression of murine tumors superior to pure PD-L1 blockage associated with an increase in tumor-specific CD8 T-cells Mode of Action Antibodies drug class | bispecific antibodies | anti-PDL1, anti-4-1BB Preclinical antitumor activity beyond PDL1 blockade PDL1 Blockade 41BB Agonism *SITC 2018, Altintas et al Next-Gen checkpoint immunomodulators
A technology agnostic approach increases our addressable market Solid tumor market Cancer segment Patient Population Challenge Our Therapeutic Strategy High mutational burden/ adjuvant stage cancers Significant portion of cancer patients Poor risk-benefit profile of checkpoint inhibitors mRNA Neoantigen Immunotherapy (iNeST) Low mutational burden cancers >60% of cancers Poor response to checkpoint inhibitors Shared Antigens (FixVac, CAR-T cells, Antibodies) “Immune desert” cancers >40% of high-mutational cancers Poor infiltration and activation of T-cells in TME mRNA Immunotherapy Immunostimulatory Compounds (intratumoral, RiboCytokines) Cancers with MHC / B2M loss 20-30% of CPI-experienced advanced cancers Failure of immune system to recognize tumor cells Antibodies CAR-Ts Refractory tumors Patients with large tumors and multiple resistance mechanisms Few treatment options Engineered Cell Therapies Combination Therapies Portfolio approach based on molecular classification and segmentation of cancer types
BNT321 (MVT-5873) BNT321: Cancer antibody targeting Cancer Associated Carbohydrate sLea sLea Phase 1 resumed in 2H 2019 Pancreatic ductal adenocarcinoma Colon carcinoma Lung adenocarcinoma Urinary bladder, mucinous adenocarcinoma Colon metastatic to ovary Breast carcinoma, lymph node sLea expression in human cancers Characteristics Fully human IgG1 mAb with subnanomolar affinity, potent cell killing by ADCC &CDC activity. Targets sialyl Lewis A epitope (sLea) epitope present in a range of glyco-proteins collectively known as CA19-9 CA19-9 is specifically expressed in pancreatic and various other cancers. Shedded CA19-9 is a prognostic marker in these cancers CA19-9 is functionally associated with carcinogenesis1 Preliminary data Six patients evaluated in combination with chemotherapy; four of them met the criteria for partial response and two patients met the criteria for stable disease. BNT321 was generally well tolerated by all six patients First patient enrolled to resume the BNT321 trial against pancreatic cancer in December 2019 Antibodies drug class | targeted antibodies | anti-CA19.9 AACR 2016, Abstract CT026, Ragupathi_Maffuid 1Engle et al, Science 2019: The glycan CA19-9 promotes pancreatitis and pancreatic cancer in mice
First-in-human Ph1 trial also supports theranostic potential Lohrmann et al., Clin Cancer Res, 2019 in press PET/CT imaging study with MVT-2163 (PET conjugated Ab version; 89Zr-DFO-HuMab-5B1) Robust accumulation in tumors lesions; tumor uptake increasing over time. Validates the target and the antibody and indicates utility of BNT321 also for detection by radio-imaging and for radiotherapy. Antibodies drug class | targeted antibodies | anti-CA19.9
Agenda Who we are and what we do Our platforms and programs mRNA vaccines – FixVac and iNeST Antibodies CARVac platform – CLDN6 CAR-T RiboCytokines Outlook for 2020 and beyond
Complete eradication of advanced tumors demonstrated in an ovarian carcinoma xenograft model BNT211: Next generation CAR-T targeting CLDN6 with CARVac “primer” Engineered Cell Therapies | CAR-T therapies | BNT211 CAR-T cell therapy + RNA Vaccine to amplify CAR-T cell in vivo CLDN6 is not present in healthy tissues CLDN6 is expressed in multiple cancers Ovarian cancer Testicular tumor Lung cancer
BNT211: Next generation CAR-T targeting CLDN6 with CARVac “primer” Engineered Cell Therapies | CAR-T therapies | BNT211 RNA-lipoplex vaccine enhances expansion & persistence of CAR T Applicability shown for CLDN6, CLD18.2, CD19 CAR-T cells 1Reinhard et al, Science 2020: An RNA vaccine drives expansion and efficacy of claudin-CAR-T cells against solid tumors
A technology agnostic approach increases our addressable market Solid tumor market Cancer segment Patient Population Challenge Our Therapeutic Strategy High mutational burden/ adjuvant stage cancers Significant portion of cancer patients Poor risk-benefit profile of checkpoint inhibitors mRNA Neoantigen Immunotherapy (iNeST) Low mutational burden cancers >60% of cancers Poor response to checkpoint inhibitors Shared Antigens (FixVac, CAR-T cells, Antibodies) “Immune desert” cancers >40% of high-mutational cancers Poor infiltration and activation of T-cells in TME mRNA Immunotherapy Immunostimulatory Compounds (intratumoral, RiboCytokines) Cancers with MHC / B2M loss 20-30% of CPI-experienced advanced cancers Failure of immune system to recognize tumor cells Antibodies CAR-Ts Refractory tumors Patients with large tumors and multiple resistance mechanisms Few treatment options Engineered Cell Therapies Combination Therapies Portfolio approach based on molecular classification and segmentation of cancer types
Further development of engineered T cell therapies Engineered Cell Therapy Manufacturing Key Plans Start first-in-human trial for CLDN6 CAR-T in solid tumors Second CAR-T in pipeline for solid tumors: CLDN18.2 CAR-T Develop CARVac with other CAR-T therapies Plan to announce first TCRs for TCR engineered therapies Expansion of certified GMP T cell manufacturing facilities planned to be completed in 2020 Idar-Oberstein: GMP certified Cell Therapy Manufacturing Front view model of final layout with the existing buildings A/B and the new buildings C and D (D behind B). C D B A
Agenda Who we are and what we do Our platforms and programs mRNA vaccines – FixVac and iNeST Antibodies CARVac platform – CLDN6 CAR-T RiboCytokines Outlook for 2020 and beyond
A technology agnostic approach increases our addressable market Solid tumor market Cancer segment Patient Population Challenge Our Therapeutic Strategy High mutational burden/ adjuvant stage cancers Significant portion of cancer patients Poor risk-benefit profile of checkpoint inhibitors mRNA Neoantigen Immunotherapy (iNeST) Low mutational burden cancers >60% of cancers Poor response to checkpoint inhibitors Shared Antigens (FixVac, CAR-T cells, Antibodies) “Immune desert” cancers >40% of high-mutational cancers Poor infiltration and activation of T-cells in TME mRNA Immunotherapy Immunostimulatory Compounds (intratumoral, RiboCytokines) Cancers with MHC / B2M loss 20-30% of CPI-experienced advanced cancers Failure of immune system to recognize tumor cells Antibodies CAR-Ts Refractory tumors Patients with large tumors and multiple resistance mechanisms Few treatment options Engineered Cell Therapies Combination Therapies Portfolio approach based on molecular classification and segmentation of cancer types
RiboCytokines: a novel therapeutic platform k Preclinical Product Candidate Optimized IL-2 Phase 1 Phase 2 IL-7, IL-2 BNT151 BNT152 Pharmacokinetic Profile RiboCytokine Recombinant cytokine Expected to enter the clinic in 2H 2020 k Preclinical Product Candidate Optimized IL-2 Phase 1 Phase 2 IL-7, IL-2 BNT151 BNT152 / BNT153 combo Expected to enter the clinic in 1H 2020 Expected to enter the clinic in 2H 2020 The Concept Cytokines encoded by mRNA and produced in the patient Improved PK properties to improve tolerability and activity Cytokine design to improve immunological properties and tolerability Therapeutic Goals Overcome resistance mechanisms by therapeutic synergy Improve activity of mRNA Vaccines Worldwide rights; wholly owned RiboCytokines
RiboCytokines boost clinical activity of vaccination and PD-L1 blockade RiboCytokines CT26 tumor model, vaccine antigen: gp70 CT26 tumor model, tumor size: 60mm2 CR: complete response, vaccine antigen:gp70 Vaccine + aPD-L1 + Effect of tumor size on treatment success of vaccination + aPD-L1 RiboCytokines boost the clinical activity of vaccination + aPD-L1 in large tumors
Multiple angles for therapeutic synergy across platforms Our multi-platform approach BNT211: First-of-kind CLDN-6 CAR-T approach utilizing CAR-T Amplifying RNA Vaccine (CARVac). Significant amplification of CAR-T cells in preclinical studies (published in SCIENCE, 2020) mRNA Cancer Vaccines Engineered Cell Therapies + Engineered Cytokines mRNA Cancer Vaccines Ribocytokine IL-2 (BNT151): Amplification of vaccine induced T cell response in pre-clinical studies + FixVac Melanoma (BNT111): Induces objective responses in CPI-experienced patients iNeST (BNT122): Currently in Phase 2 in combination with CPI in 1L Melanoma. 2 adjuvant trials planned in 2020 mRNA Cancer Vaccines Approved PD1/PL1 Inhibitors + Broad therapeutic potential across a range of solid tumors
Agenda Who we are and what we do Our platforms and programs Outlook for 2020 and beyond +
We expect a significant news flow in the upcoming next 18 months Expected news flow Platform Candidate Indication (Target) 1H-2020 2H-2020 20213 20223 FixVac BNT111 Advanced Melanoma BNT112 Prostate Cancer BNT113 HPV16+ H&N Cancer BNT114 Triple Negative Breast Cancer iNeST RO7198457 (BNT122) 1L Melanoma with CPI Multiple ST (basket trial) Intratumoral Immunotherapy SAR441000 (BNT131) Solid tumors (IL-12sc, IL-15sushi, GM-CSF, IFNα) RiboMabs BNT141 Multiple ST BNT142 Multiple ST (CD3+CLDN6) RiboCytokines BNT151 Multiple ST (Optimized IL-2) BNT152/153 Multiple Solid Tumors (IL-7, IL-2) CAR-T Cells BNT211 Multiple ST (CLDN6) Next-Gen CP Immunomodulators BNT311 Multiple ST (PD-L1x4-1BB) BNT312 Multiple ST (CD40x4-1BB) Antibodies BNT321 Pancreatic Cancer (CA19-9) TLR7 Ligand BNT411 Multiple ST (TLR7) Infectious and Rare Diseases Influenza Up to 10 Infectious Disease Indications 5 Rare Disease Indications mRNA Others Phase 2/3 Phase 1/2 Phase 2 Phase 1 Start Phase 1 Start Phase 1 Start Phase 1 Report Phase 1 Phase 1/2 Report Phase 1/2 1We expect this topline data update to include an update on the ongoing study, including patient enrollment numbers, with full efficacy and safety data for an interim update expected in the second half of 2021; 2As the trial is sponsored and conducted by Sanofi, the timing of data updates is not under our control, and is subject to change by Sanofi. 3Our expectations for timing of milestones beyond 2020 are premised on and subject to the achievement of earlier milestones on their expected timelines. Press releases will be issued once first patient has been dosed. Start Phase 2 Start Phase 3 Start Phase 2 Start Phase 1 Report Phase 1/22 Trial progress update1 Data update Phase 1/2 Start Phase 1/2 Expected begin of trial Expected data readout / update Legend Data update Phase 1 Start first study Start first Phase 1 Start first Phase 1 Start Phase 1 Report Phase 1/2 ST: solid tumors
Building a 21st Century Immunotherapy Company Our goals 2020 Outlook 5 trial updates (incl. publishing BNT111 FixVac Melanoma Phase 1/2 data in peer reviewed journal) Initiate Phase 3 registrational trial for BNT111 FixVac Melanoma Initiate 2 additional iNeST trials in adjuvant stage cancers Initiate Phase 1/2 trial using CARVac (BNT211) in CLDN6+ solid tumors (e.g., ovarian, testicular) Initiate Phase 2 trial in HPV16+ H&N cancer Continue to build global clinical development organization (US development team on East Coast) 1 2 3 4 5 6
© Copyright BioNTech SE 2019. All Rights Reserved. Jan 15, 2020 An der Goldgrube 12 55131 Mainz Germany T: +49 6131 9084-0 F: +49 6131 9084-390 M: info@biontech.de