Sangamo Therapeutics, Inc. · NASDAQ:SGMO
Sangamo Therapeutics — J.P. Morgan 36th Annual Healthcare Conference (January 2018)
35 slides from Sangamo Therapeutics's conference presentation, Jan 8, 2018
Slide 1: J.P. Morgan 36th Annual Healthcare Conference
J.P. Morgan 36th Annual Healthcare Conference Dr. Sandy Macrae CEO January 10, 2018 Exhibit 99.1
Slide 2: Forward Looking Statements
This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include, but are not limited to, the duration for which existing capital resources can provid
Slide 3: Committed to translating science into genomic therapies
We are committed to translating ground-breaking science into genomic therapies that transform patients’ lives
Slide 4: Four technological platforms for genomic medicines
Sangamo is investing across four technological platforms for genomic medicines Genome Editing Gene Therapy Cell Therapy Gene Regulation
Slide 5: 2017 Accomplishments: Technology
2017 Accomplishments Technology Optimized Zinc Finger technology platform Improved cell therapy editing: >90% T-cell multiplexing efficiency Demonstrated AAV.SGMO crosses blood-brain barrier via IV delivery in mice Advanced LNP platform after demonstrating highly efficient editing and protein knock
Slide 6: 2017 Accomplishments: Clinical
2017 Accomplishments Clinical Initiated 3 Phase 1/2 clinical studies for hemophilia A, MPS I and MPS II Dosed patients in SB-525 hemophilia A gene therapy study First ever patient treated with in vivo genome editing FDA acceptance of IND for ST-400 beta-thalassemia
Slide 7: 2017 Accomplishments: Corporate
2017 Accomplishments Corporate Global collaboration agreement with Pfizer for SB-525 (hemophilia A) Strengthened balance sheet; cash runway of more than 2 years Secured new corporate HQ in South San Francisco biotech hub Expanded manufacturing capacities and secured cGMP vector supply chain ZFP-TF g
Slide 8: ZFNs: The platform of choice for therapeutic genome editing
ZFNs: The platform of choice for therapeutic genome editing ZFN ZFN
Slide 9: ZFNs: The platform of choice for therapeutic genome editing
ZFNs: The platform of choice for therapeutic genome editing Efficiency Precision Specificity ZFNs
Slide 10: Recent innovations drive exceptional performance
Recent innovations drive exceptional performance Innovation Result New dimer architectures yield higher modification activity Increase DNA editing efficiency to as high as 99.5% Phosphate contact tuning via replacement of key residues Off-target cleavage undetectable (>1000 fold reduction) Specifici
Slide 11: Sangamo Therapeutic Development
SB-318: MPS I SB-913: MPS II SB-FIX: Hemophilia B SB-525: Hemophilia A ST-920: Fabry Disease ST-400: Beta-thalassemia BIVV-003: Sickle Cell Disease Sangamo Therapeutic Development Therapeutic programs Research T-cell Immuno-Oncology CNS: ALS/FTLD (C9ORF72) CNS: Tauopathies Technology: ZFNs, AAV, LNP
Slide 12: Product portfolio diversified across tissue, disease and technology
Tissue Research Preclinical Phase 1/2 Phase 3 Liver (Rare Disease) MPS I (SB-318) MPS II (SB-913) Hemophilia B (SB-FIX) Hemophilia A (SB-525) Fabry Disease (ST-920) Brain (CNS Diseases) Tauopathies ALS/FTLD - C9ORF72 Huntington’s Disease T Cells (Immunology, Oncology) Oncology - allogeneic Immunolog
Slide 13: Genome Editing and Gene Therapy: Liver (Rare Diseases)
Genome Editing SB-318: MPS I SB-913: MPS II SB-FIX: Hemophilia B Gene Therapy Liver (Rare Diseases) SB-525: Hemophilia A ST-920: Fabry disease
Slide 14: In vivo genome editing of albumin
In vivo genome editing of albumin: harnessing the liver’s most highly expressed locus albumin gene H TG H transgene transgene + zinc finger nucleases Packaging into AAV vectors In the liver Delivery Strong albumin promoter AAV vectors
Slide 15: Genome editing program summaries for SB-318, SB-913 and SB-FIX
Phase 1/2 Clinical Trial Status INDs open Studies initiated 14 sites active by YE 2017 First patient treated (MPS II) Preliminary data expected 1H 2018 Genome editing program summaries for SB-318, SB-913 and SB-FIX SB-318: MPS I SB-913: MPS II SB-FIX: Hemophilia B Regulatory Designations US Orphan D
Slide 16: Sangamo's AAV cDNA gene therapy platform
Sangamo’s AAV cDNA gene therapy platform: potential for potent therapeutic solutions for adults with rare monogenic diseases transgene AAV vectors liver cell DNA therapeutic gene nucleus liver cell Delivery Packaging into AAV vectors In the liver
Slide 17: Gene therapy program summary for SB-525: Hemophilia A
Amended study protocol with FDA – allows flexibility to dose-escalate after 2 patients / cohort Strong collaborative relationship with Pfizer focused on long-term success Gene therapy program summary for SB-525 Regulatory Designations US Orphan Drug Fast Track EMA Orphan Medicinal Product Phase 1/2
Slide 18: Gene therapy program summaries for SB-525 and ST-920
Gene therapy program summaries for SB-525 and ST-920 Regulatory Designations US Orphan Drug Fast Track EMA Orphan Medicinal Product Phase 1/2 Clinical Trial Status IND open Study initiated 8 sites active at YE 2017 3 patients treated Protocol amended with FDA – 2 pts / cohort Preliminary data expect
Slide 19: Cell Therapy: Stem Cells and T Cells
Stem Cells ST-400: Beta-thalassemia BIVV-003: Sickle Cell Disease T Cells Allogeneic CAR-T / TCR Immunotherapy Cell Therapy
Slide 20: Autologous, gene-edited cell therapies for beta-thalassemia and sickle cell disease
Autologous, gene-edited cell therapies for beta-thalassemia and sickle cell disease Gene editing Manufacture ST-400 stem cells via transient, non-viral delivery of ZFN mRNA Harvest ST-400 stem cells are cryo-preserved and shipped to treatment center BCLA11 Enhancer Sequence BCLA11 Enhancer Knocked O
Slide 21: Cell therapy program summaries for ST-400 and BIVV-003
Cell therapy program summaries for ST-400 and BIVV-003 ST-400: Beta-thalassemia BIVV-003: Sickle Cell Disease Phase 1/2 Clinical Trial Status IND filing expected in 2018 Phase 1/2 Clinical Trial Status IND open Study initiation early 2018 First enrolled subject expected mid-2018 Highly efficient, pr
Slide 22: Manufacturing allogeneic T-cell therapies with ZFNs
Manufacturing allogeneic T-cell therapies with ZFNs HLA knock-out TCR knock-out Targeted insertion of CAR gene cGMP Manufacturing Facility Healthy Donor Apheresis Isolate T-cells from a healthy donor’s blood T-cell Manufacturing Transient, non-viral delivery of ZFN mRNA to manufacture universal CAR-
Slide 23: High efficiency is critical for single-step multiplexed genome editing
Allogeneic cell therapy production: high efficiency is critical for single-step multiplexed genome editing 1 KO+1 KO +1 TICompounded Efficiency Very High 99% x 99% x 99% = 97% High 90% x 90% x 90% = 73% Medium 70% x 70% x 70% = 34% Low 50% x 50% x 50% = 13%
Slide 24: Reliably hit >90% gene editing efficiency in T-cells
We can reliably hit >90% gene editing efficiency in T-cells Transgene Expression 91% Targeted Insertion (TI) HLA Expression T-cell Receptor Expression 93% Double KO Precise KO at TRAC and B2M loci with no detectable off-target activity Precise TI with no detectable off-target activity
Slide 25: Simultaneous multiplex editing efficiencies
Simultaneous multiplex editing efficiencies: 3x ZFN KO + 1x TI 76% of cells have all 4 edits KO: TCR, β2M and CISH; TI: GFP (TRAC) % EDITING POTENTIAL APPLICATION: Universal T cells with checkpoint gene knock-out SINGLE STEP EDITING ZFN Knock-out TCR (TRAC) HLA-class I (β2M) CISH (checkpoint gene) T
Slide 26: Gene Regulation: Brain (CNS Diseases)
Gene Regulation Huntington’s disease Tauopathies C9ORF72 for ALS and FTLD Brain (CNS Diseases)
Slide 27: Sangamo's gene regulation platform
Sangamo’s gene regulation platform: precise and specific regulation of a mutated gene allele to treat various CNS diseases Packaging into AAV vectors Potential Routes of Administration In Neurons Intravenous Intrathecal Intracranial ZFP-TF represses tau DNA ZFP-TF does not integrate into the genome
Slide 28: New R&D collaboration with Pfizer for ALS and FTLD
New R&D collaboration with Pfizer to develop gene therapy for ALS and FTLD using Sangamo’s ZFP-TF gene regulation platform Gene Regulation C9ORF72 gene target Transcription Start Site ATG Wildtype allele Disease allele G4C2 < 30 G4C2 > 100
Slide 29: Pfizer collaboration financial terms
New R&D collaboration with Pfizer to develop gene therapy for ALS and FTLD using Sangamo’s ZFP-TF gene regulation platform $12M Upfront payment $60M Development, regulatory and first commercial sale milestones $90M Sales milestones Tiered royalties on net sales Gene Regulation C9ORF72 gene target
Slide 30: AAV.SGMO delivery results in greater neuronal transduction than AAV9
AAV.SGMO delivery via IV or ICV administration results in greater neuronal transduction efficiency compared to AAV9 Motor Cortex Cerebellum Hippocampus AAV.SGMO IV ICV AAV9 IV ICV Route of Admin.
Slide 31: AAV.SGMO ZFP-TF: potent tau reduction
AAV.SGMO ZFP-TF: potent tau reduction via IV / ICV administration Up to ~50-70% tau reduction in targeted brain regions for tauopathies N=3-6. Mean +/- SD. * P <0.05; ** P <0.01; *** P <0.001; **** P < 0.0001.
Slide 32: 2018 Priorities
2018 Priorities yy
Slide 33: Owned cGMP facility and Brammer Bio agreement enable flexible supply
Sangamo owned cGMP facility along with expanded agreement with Brammer Bio enables flexible supply to support expanding pipeline Sangamo cGMP facility initially focused on product development and Phase 1/2 clinical trial supply for new pipeline programs Brammer agreement provides dedicated capacity
Slide 34: 2018 steps toward long-term success of Sangamo
1 Clinical 2018 steps toward long-term success of Sangamo 2 3 4 5 Technology Partnerships Corporate Pipeline Demonstrate clinical progress on core assets with preliminary clinical data beginning 1H 2018 Advance ST-400 BT program with FPI in mid-2018 and support Bioverativ IND filing for SCD. File IN
Slide 35: Thank you.
Thank you.
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