Sana Biotechnology, Inc. · NASDAQ:SANA
Sana Biotechnology — Investor Presentation (January 2024)
36 slides from Sana Biotechnology's investor presentation, Jan 9, 2024
Slide 1: Corporate Presentation January 2024
Corporate Presentation January 2024 Exhibit 99.1
Slide 2: Cautionary Note Regarding Forward-Looking Statements
This presentation contains forward-looking statements about Sana Biotechnology, Inc. (the “Company,” “we,” “us,” or “our”) within the meaning of the federal securities laws. All statements other than statements of historical facts contained in this presentation, including, among others, statements r
Slide 3: Sana's hypoimmune technology goal is to overcome allogeneic rejection
Sana’s hypoimmune technology goal is to overcome allogeneic rejection HIP technology provides foundation for potential multiple drugs across many therapeutic areas Begin 2024 with four clinical programs treating seven diseases SC291 oncology – NHL and CLL SC291 B-cell mediated autoimmune – lupus nep
Slide 4: Sana pipeline positioned to deliver meaningful clinical data
Sana pipeline positioned to deliver meaningful clinical data 1Investigator sponsored trial. Abbreviations: AAV, ANCA-associated vasculitis; CLL, chronic lymphocytic leukemia; ERL, extrarenal systemic lupus erythematosus; HD, Huntington’s disease; LN, lupus nephritis; MM, multiple myeloma; NHL, non-H
Slide 5: Allogeneic cell rejection remains the largest problem in transplants
Allogeneic cell rejection ~75 years of transplants – immune rejection remains the largest problem Lifelong immunosuppression is current standard Genome modification efforts to date have generally been incomplete Autologous therapies have limited scalability and are only available for a small number
Slide 6: HIP-modified cells successfully transplanted in allogeneic models across species and cell types
HIP-modified cells successfully transplanted in allogeneic models across various species and cells types Mouse iPSCs Mouse iPSC-derived endothelial cells Mouse iPSC-derived smooth muscle cells Mouse iPSC-derived cardiomyocytes NHP iPSCs (16 weeks follow-up) NHP donor-derived islets (40 weeks follow-
Slide 7: HIP-modified allogeneic islet cells to control glucose in a type 1 diabetic NHP model
HIP-modified allogeneic islet cells to control glucose in a type 1 diabetic NHP model Type 1 diabetes is a disease of missing pancreatic beta cells Abbreviations: NHP, non-human primate; STZ, Streptozotocin. Study Design (N=1) NHP treated with STZ Glucose stabilized with exogenous insulin Allogeneic
Slide 8: NHP islet study design and results
Study Design (N=1) NHP primary islet cells isolated and HIP-modified Cells injected intramuscularly into a diabetic, allogeneic NHP without immunosuppression Fasting glucose Survival and function of allogeneic hypoimmune pancreatic islet cells in diabetic NHP 6 months without immunosuppression STZ a
Slide 9: Near-term opportunities to apply HIP modifications to validated mechanisms
1Avezbakiyev et al. Blood. 2022 2Durie et al. The Oncologist. 2020 3NIH Autoimmune Diseases Coordinating Committee: Autoimmune Diseases Research Plan, October 2017, U.S. 4t1dindex.org Near-term opportunities to apply HIP modifications to validated mechanisms with unmet need Blood cancers: >100,000 p
Slide 10: Hematologic cancers continue to have a high unmet need
Challenges Autologous CAR T cell scalability Many patients fail CAR T treatment Allogeneic CAR T cell immune rejection limits persistence and efficacy Opportunity Known targets Known efficacy and safety bar Sana’s HIP CAR T platform can address challenges and exploit opportunities Hematologic cancer
Slide 11: Defining success for SC291 in oncology
Understanding levels of evidence as data mature Defining success for SC291 in oncology Does SC291 evade allogeneic immune rejection? Do we make great T cells? How do early responses translate into durable CRs? Can we make the drug from multiple donors with a consistent and predictable safety and eff
Slide 12: ARDENT trial: patient responses and safety
3. LBCL* 4. LBCL** 5. MZL*** 6. CLL 1. CLL 2. MCL ARDENT: 3 of 4 evaluable patients had at least a partial response, with 2 ongoing complete responses Clinical data as of: January 5, 2023 “evaluable” defined as patients treated with SC291 and had at least one disease assessment *Transformed DLBCL fr
Slide 13: Translating preclinical data to people
Translating preclinical data to people Immune response data provide important early insights T Cell Population Genetic Modifications WT CAR T CD47-CD19 CAR dKO T HLA I/II deficient HIP CAR T CD47-CD19 CAR; HLA I/II deficient SC291 is a mixture of HIP and non-HIP CAR T cells Test the patient’s immune
Slide 14: Patient T cells do not kill dKO T cells or HIP CAR T cells
Patient T cells kill WT CAR T cells but do not kill dKO T cells or HIP CAR T cells Patient T cells kill WT CAR T cells Patient T cells do not kill dKO T cells Patient T cells do not kill HIP CAR T cells No Killing Killing No Killing Day 28 Baseline Day 28 Baseline Day 28 Baseline No Killing No Killi
Slide 15: HIP CAR T cells do not induce an antibody response
WT CAR T cells induce an antibody response HIP CAR T cells do not induce an antibody response dKO T cells do not induce an antibody response Patient generates antibodies against WT CAR T cells but not dKO T cells or HIP CAR T cells From Patient #1 in the ongoing ARDENT trial.
Slide 16: NK cells kill dKO T cells with HLA-E overexpression
NK cells kill dKO T cells with HLA-E overexpression NK cells kill dKO T cells NK cells taken from patient’s blood Only HIP CAR T cells avoid NK cell killing Patient’s NK cells from day 13 after SC291 dosing T cell with editing profile in column title NK cells NK cells do NOT kill HIP CAR T cells Act
Slide 17: Only HIP CAR T cells avoid NK cell killing
still image after movie Only HIP CAR T cells avoid NK cell killing NK cells taken from patient’s blood Patient’s NK cells from day 13 after SC291 dosing NK cells kill dKO T cells NK cells kill dKO T cells with NK cells do NOT kill HIP CAR T cells HLA-E overexpression ‘Actual assay time = 4 hours. @
Slide 18: No detectable immune response toward HIP CAR T cells
D28 blood sample No detectable immune response in the patient toward HIP CAR T cells From patient #1 in the ongoing ARDENT trial. Target: WT CAR T cells Target: dKO T cells Target: HIP CAR T cells Killing No Killing Killing
Slide 19: Early data suggest safe dosing, immune evasion, and clinical efficacy
Early data suggest ability to dose safely, the desired immune evasion profile, and clinical efficacy More data to come Immune evasion Safety profile Response rate Cell persistence Durability of responses SC291: ARDENT trial continues enrollment with more data expected in 2024 An effective allogeneic
Slide 20: Autoimmune diseases have emerged as promising opportunity
Autoimmune diseases have emerged as promising opportunity 1Adapted from Zhang et al. Frontiers in Immunology. 2023; Oh et al. Immune Network. 2023; Lee et al. Nature Reviews Drug Discovery. 2021. 2Anolik et al. Arthritis and Rheumatism 2007 3 Mendez et al. Clinical Journal of the American Society of
Slide 21: Autologous CD19 CAR T therapy results in durable drug-free remission in refractory SLE
Autologous CD19 CAR T therapy results in durable drug-free remission in refractory SLE patients Mackensen et al. Nature Medicine. 2022 Abbreviations: BL, baseline; CRS, cytokine release syndrome; ICANS, immune effector cell-associated neurotoxicity syndrome; M, months; SLE, systemic lupus erythemato
Slide 22: SC291 treatment leads to deep B cell depletion in oncology patient
ARDENT trial: SC291 treatment leads to deep B cell depletion in oncology patient From Patient #4 in the ongoing ARDENT trial. CD19 CD19+ cells in blood in %
Slide 23: No anti-HLA antibody production suggests complete B cell depletion
No anti-HLA antibody production against WT CAR T cells suggests complete B cell depletion HIP CAR T cells do not induce an antibody response dKO T cells do not induce an antibody response Complete B cell depletion may be even more important in autoimmune than oncology patients From Patient #4 in the
Slide 24: SC291 potential treatment for B-cell mediated autoimmune diseases
SC291 offers potential for transformative treatment for B-cell mediated autoimmune diseases Targeting multiple indications Lupus nephritis >230K1,2 patients3 Extrarenal SLE >200K1 patients3 ANCA-associated vasculitis >60K4 in US 1 2 3 1 2 3 4 No patient apheresis Product availability Scaled manufact
Slide 25: SC291 GLEAM Phase 1 trial goals
Key features of Phase 1 trial (GLEAM) Patients with refractory lupus nephritis, extrarenal SLE, and AAV Starting dose of 90 million CAR T cells Potential to expand beyond these indications over time Data expected in 2024 from multiple indications Safety and tolerability Early response rates SC291: G
Slide 26: SC262: Targeting patients with inadequate response to CD19 therapy
SC262: Targeting growing population of patients with inadequate response to CD19 therapy 1US, EU5, and Japan.2Clarivate DRG NHL Market Forecast Nov 2021; 2027 Forecast is 2L+ LBCL patients; internal analysis of secondary EPI data.3Di Blasi et al. Blood.2022; DESCAR-T registry. Estimated ~12,000 B ce
Slide 27: SC262: Licensed CD22 CAR produced strong clinical data in CD19 failures
2023 ASH Yi-Jiun Su N=38 SC262: Licensed CD22 CAR produced strong clinical data in CD19 failures when part of autologous CAR T N=16 N=21 >50% 6-month CR rate in CD19 CAR failure DLBCL patients High rate of CRs in CD19 failure ALL patients ~80% patients with prior CD19 therapy 2022 ASH Miklos/Stanfor
Slide 28: Type 1 diabetes is a large unmet need
Disease caused by autoimmune destruction of insulin-producing pancreatic beta cells, resulting in no insulin production Type 1 diabetes is a large unmet need with >8M WW2 Short-term complications result from hypo- and hyperglycemia Long-term complications result from micro- and macrovascular disease
Slide 29: Sana combining stem cell, gene editing, and immunology expertise
Sana – combining stem cell, gene editing, and immunology expertise Emerging data suggest a cure is possible Eliminate need for immunosuppression Primary islet cell transplants offer long-term glucose control with immunosuppression Stem-cell derived islets with immunosuppression can create an efficac
Slide 30: Survival and function of hypoimmune islet cells in diabetic NHP
Survival and function of allogeneic hypoimmune pancreatic islet cells in diabetic NHP 6 months without immunosuppression Study Design (N=1) NHP primary islet cells isolated and HIP-modified Cells injected intramuscularly into a diabetic, allogeneic NHP without immunosuppression Fasting glucose STZ a
Slide 31: Type 1 diabetes model highlights potential to overcome autoimmune rejection
D0 D3 D7 D5 D9 No glucose control Patient T cells eliminate islet cells due to autoimmunity Type 1 diabetes model highlights potential to overcome autoimmune rejection of pancreatic beta cells Abbreviations: T1DM, type 1 diabetes mellitus Hu et al. Sci Transl Med. 2023 Patient with T1DM PBMCs Autolo
Slide 32: HIP iPSC-derived islet cells evade autoimmune killing and control glucose
D7 D0 D23 D29 D13 HIP iPSC-derived pancreatic islet cells from patient with T1DM evade autoimmune killing and control glucose Abbreviations: BLI, bioluminescence imaging Hu et al. Sci Transl Med. 2023. BLI: Islet Survival C-peptide Glucose Control
Slide 33: Trial authorized at Uppsala University Hospital
Trial authorized at Uppsala University Hospital Primary human HIP islet cells transplantation in type 1 diabetes patients Intramuscular administration in forearm No immunosuppression Insights for SC451 Potential clinical validation of hypoimmune islet cells in T1DM patients Cell survival & immune ev
Slide 34: Sana's approach to treat type 1 diabetes
Sana’s approach to treat type 1 diabetes Make hypoimmune islet cells from stem cells 1 + CD47 MHC I MHC II Manufacture at scale 2 Deliver as a single therapy 3 SC451 program – HIP stem cell-derived islet cell therapy – delivered with no immunosuppression
Slide 35: Meaningful clinical data in multiple diseases in 2024
Meaningful clinical data in multiple diseases in 2024 Unlocking the potential of our hypoimmune platform across multiple patient populations Value B-cell mediated Autoimmune Unlocked by HIP validation Oncology Type 1 Diabetes SC291 SC262 SC291 LN SC291 ERL SC291 AAV UP421
Slide 36: Thank You
Thank You Biotechnology"
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