Sana Biotechnology, Inc. · NASDAQ:SANA
Sana Biotechnology — Investor Presentation (June 2022)
30 slides from Sana Biotechnology's investor presentation, Jun 14, 2022
Slide 1: Corporate Presentation June 2022
Corporate Presentation June 2022 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 Biotechnology: Engineered Cells as Medicines
Ability to modify the genome and use engineered cells as medicines has the potential to be one of the most important advances in healthcare over the next several decades: Nearly every disease is caused by damage to or dysfunction of a cell Sana’s ambition is to repair or replace any cell in the body
Slide 4: Sana goal: Repair cells in the body when possible or replace them when needed
Sana goal: Repair cells in the body when possible or replace them when needed Deliver any payload… (DNA, RNA, protein, organelle, integrating vs non-integrating) To any cell… (unlimited volume of distribution) In a specific… (e.g., just T cell) And repeatable way (limit immunogenicity) Manufacture a
Slide 5: Sana's platforms, technology, and programs
Sana’s platforms, technology, and programs 1Ornithine transcarbamylase deficiency
Slide 6: Hypoimmune technology: Protecting cells from immune rejection
Hypoimmune technology: Protecting cells from immune rejection
Slide 7: Hypoimmune cells evade rejection from the adaptive and innate immune system in mice
Hypoimmune cells evade rejection from the adaptive and innate immune system in mice Evade the adaptive immune system Deuse T, …, Schrepfer S. Nat Biotechnology. 2019; 37:252-258 T cell Activation (ELISPOT) No systemic T cell activation with HIP cell transplantation IgM Binding (FACS) No binding of d
Slide 8: Hypoimmune cells survive in vivo in NHP while unmodified iPSCs get rejected
Hypoimmune cells survive in vivo in NHP while unmodified iPSCs get rejected A A CO, cross over; Txp, transplant NHP unmodified iPSCs (wt) & NHP hypoimmune iPSCs (HIP) were transplanted into 8 allogeneic recipients Representative of results across 4 NHPs per arm. B Representative of results across 4
Slide 9: Hypoimmune cells evade rejection in NHPs
T cell Activation (ELISPOT) No systemic T cell activation by hypoimmune cells IgM Production (ELISA) Implantation of hypoimmune cells does not cause activation of antibody production Hypoimmune cells do not activate the “missing self” response from the NK cells Anti-CD47 safety switch Killing by NK
Slide 10: Sana is pursuing a broad ex vivo cell engineering strategy
Sana is pursuing a broad ex vivo cell engineering strategy T cells Pancreatic islets Glial progenitor cells Cardiomyocytes Differentiate pluripotent stem cells with hypoimmune edits Programs that benefit from, but do not require hypoimmune edits
Slide 11: Significant unmet need in B cell malignancies and multiple myeloma
Significant unmet need in B cell malignancies and multiple myeloma in the US and EU alone: ~250,000 new cases annually1 Est. 100,000 deaths annually1 <10,000 patients have been treated with CAR T therapy to date2 Of those treated, many do not respond and many relapse Manufacturing and access challen
Slide 12: Sana's hypoimmune allo T is potentially best-in-class
Sana’s hypoimmune allo T is potentially best-in-class Cell engineering CD19 targeted HIP allogeneic T cell GvHD, graft versus host disease; HvGD, host versus graft disease. 2 3
Slide 13: CD19 HIP CAR T cells clear tumor in vivo
Day 0 Day 3 start of CAR dosing Day 7 Day 15 Untreated Day 23 Day 27 HIP CAR T cells CD19 HIP CAR T cells clear tumor in vivo
Slide 14: Clinical study shows CD22 CAR T efficacy in relapsed lymphoma after prior CD19 CAR T
Clinical study shows CD22 CAR T efficacy in treating relapsed lymphoma after prior CD19 CAR T therapy Miklos et al, ASH 2021 Blood 2021 Apr 29;137(17):2321-2325. doi: 10.1182/blood.2020009432. Minimal ICANS / CRS observed across dose levels Miklos et al, ASH 2021 Total is a combination of DL1 and DL
Slide 15: Expect to file our first allo T IND targeting CD19 as early as this year
Expect to file our first allo T IND targeting CD19 as early as this year CD19/CD22 dual targeting offers potential of higher and more durable complete response rates Fully-human, clinically validated CD22 CAR with potential to address CD19 CAR T failures Fully-human, clinically validated BCMA CAR to
Slide 16: Large unmet need remains in type 1 diabetes
Large unmet need remains Disease where destruction of insulin-producing beta cells in the pancreas results in inability to control blood glucose 1.6M patients with type 1 diabetes in the US and 2.4M in Europe2; 51k new patients/year combined3 Long-term complications: end-organ damage, including hear
Slide 17: Progress toward turning beta cells into medicines
Progress toward turning beta cells into medicines Make functional beta cells from iPSCs cells Hide beta cells from allogeneic rejection Hide beta cells from autoimmune reaction Create GMP supply chain
Slide 18: Stem cell-derived pancreatic islet cells lead to robust function
Stem cell-derived pancreatic islet cells lead to robust function Superior insulin secretion and faster kinetics in vitro Robust rescue of type 1 diabetes mouse model
Slide 19: Hypoimmune pancreatic islet cells evade immune detection and regulate glucose levels
Hypoimmune pancreatic islet cells evade immune detection and regulate glucose levels Glucose levels stay elevated Allogeneic human hypoimmune islet cells Glucose levels normalized Allogeneic human unmodified islet cells
Slide 20: Hypoimmune islet cells evade autoimmune killing in type 1 diabetes patient blood
T cells from PBMCs of type 1 diabetes patients kill unmodified islets, but not HIP islet cells Hypoimmune pancreatic islet cells evade autoimmune killing in testing of blood from type 1 diabetes patients Serum from healthy volunteers or type 1 diabetes patients Antibodies from sera of type 1 diabete
Slide 21: Robust GMP supply chain required to use iPSC-based therapies as medicines
Robust GMP supply chain required to use iPSC-based therapies as medicines 1 2 3
Slide 22: Fusosome technology: cell-specific in vivo delivery platform
Fusosome technology: Development of cell-specific in vivo delivery platform Source: CDC website 3 2 1 Sana approach: Leverage insights from nature to deliver various payloads to specific cells Fusosome
Slide 23: In vivo cell engineering: targeted medicines across diverse cell types
In vivo cell engineering: Creating targeted medicines across a diverse set of cell types T cells Hepatocytes Hematopoietic stem cells
Slide 24: High unmet need remains for blood cancers
High unmet need remains for blood cancers Current ex vivo approaches have limitations Fusogen platform offers potential to overcome these limitations
Slide 25: T cell fusosome carrying CAR construct infused into patient
T cell fusosome carrying CAR construct infused into patient CAR activity Expression/recognition Amplification Target cell killing Target cell killing Specificity CD4/CD8 T cell transduction Expression Transgene integration and CAR expression Function Targeted cell killing T cell Fusosome Cancer cell
Slide 26: IV fusosome-delivered CD19 CAR clears B cell tumors in humanized mice
IV administration of CD19 CAR delivered by fusosome can clear B cell tumors in humanized mice comparably to ex vivo CD19 CAR T CD19 CAR delivered by fusosome: in vivo CD19 CAR T: ex vivo Saline CD19 CAR T
Slide 27: Sana aspiration: Engineered cells as medicines
Sana aspiration: Engineered cells as medicines Address obstacles to using engineered cells as medicines Validate platforms and create important medicines Hypoimmune allo CD19 CAR T Fusosome for CD19 CAR T in vivo Unlock the potential of engineered cells as medicines in multiple diseases Hypoimmune c
Slide 28: Thank You
Thank You
Slide 29: Appendix
Appendix Sana@
Slide 30: BCMA CAR T (CT103A) initial clinical data promising in relapsed/refractory multiple myeloma
Safety profile (All patients n=79): Grade 3+ CRS: 2.5% i.e., 2 patients Grade 3+ ICANS: 0% Efficacy profile (All patients n=79): ORR: 94.9% CR/sCR: 58.2% MRD negativity at least once after infusion: 93.7% In prior BCMA CAR T treated patients (n=13): ORR (76.9%); CR/sCR (46.2%) Persistence of CT103A
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