EFTA00292520.pdf

ANGIOCRINE BIOSCI ENCE Investor Update May 4th 2017 OO Nig 9FRPA EFTA00292520 Fact: Endothelial Cells Instruct Stem Cells Organ-Specific Stem Cells Z\ C_N, MA19$:A!Ng Endothelial Cells --- Angiocrine Factors Signaling proteins This is how vital organs in humans regenerate 2 EFTA00292521 Uniquely Angiocrine Bioscience E-CELTM Injectable engineered human endothelial cells that can orchestrate regeneration of vital organs 4.J-CELTM Create your own stem cells from your own endothelial cells obtained via skin or fat samples No other company has technologies like ours ANGOGUNE EFTA00292522 Rapid Progress Over the Last Year 1) Awarded non-dilutive $3.8M grant for pre-clinical development 2) Created two clinical therapeutic candidates 3) Filed with FDA 1" Investigational New Drug (IND) application to enter clinic 4) New IP in cellular biopharmaceutical and stem cell gene therapy 5) Established full R&D capabilities in San Diego AN OC arum CALIFOR111111 /TEM CELL ',Gene* 4 EFTA00292523 E-CELTM Clinical Development Focus on Transforming Olt. Hematology & Oncology 1. E-CEL Stem-Cell Gene Therapy • Goal: Cure genetic blood diseases(e.g., Sickle Cell Anemia) • Clinical stage: H2 2017 (1" trial, Leukemia with high-risk genetic characteristics) e199R1I'M 2. E-CEL Cellular Therapy • Goal: Accelerate recovery from radiation/chemotherapy • Clinical stage: H1 2018 (1" trial, various cancers) EFTA00292524 Substantial Global Markets Gene Therapy Market • Genetic diseases (e.g. Sickle Cell Anemia, Primary Immuno-Deficiency, etc.) are responsible for a heavy loss of life."' • > 6,000 known single-gene defect disorders2 • "worth USD 11 billion by 2025"3 • "an area of important future growth" (GlaxoSmithKline)4 RINE Cancer Care Market • 40% of adults will be diagnosed with cancer during their lifetimes5 • 1,685,210 new US cancer cases diagnosed in 20165 • Significant number of patients will receive high-dose chemotherapy and/or radiation treatment , www.whoint/genomics/public/geneticdiseasesien/index2.html MIT Technology Review, May 16° 2016, Gene Therapy's First Out-and-Out Cure , www.medicinenet.com/genetic disease/article.htm is Here. www.pmewswire.cominews-releases/gene-therapy-market-2015- 'Cancer Statistics - National Cancer Institute 2016 --2025-30008282Zhml EFTA00292525 E-CEL Stem-Cell Gene Therapy Program CIRM Funded Pre-Clinical & IND Done C\\ Ml,g9FP,M Phase lb Trial (Leukemia with high- risk genetic profile) H2-2017 Phase 2 Trial (Sickle Cell Anemia) H 2-2019 EFTA00292526 Clear US FDA Regulatory Path E-CEL Stem-Cell Gene Therapy First-In-Human Clinical Trial (Phase lb) • Safety of stem cells expanded in culture with E-CEL cells • Stem cells are not gene corrected and obtained from donor umbilical cord blood (allogeneic) • Patients with blood/marrow cancer with high-risk genetic profile Second Clinical Trial (Phase 2) • Safety and efficacy of stem cells expanded in culture with E-CEL cells • Stem cells are gene corrected and obtained from patient's bone marrow (autologous) • Patients with genetic blood disease (e.g., sickle cell anemia) c...x.-., AN.99$T,FIc EFTA00292527 First-in E-CEL Ste -Human Clinical Trial rn-Cell Gene Therapy Financial Support from: arum CALIFORIIIA7 /TEM CELL RGEnCY State of California Stem Cell Research and Cures Initiative publicly funded program to advance scientific discoveries in stem cell biology • Pre-clinical grant award (April, 2016) - $3.8M / Covered 80% of all pre-clinical development costs • Clinical grant application — (Q2, 2017) — up to $5M / Covers 70% of Phaselb Clinical trial costs Therapeutic focus: • Rebuild new blood and immune system by transplanting E-CEL cord blood stem cells (transplantation) in high-risk leukemia patients who cannot be cured by chemotherapy or other donors Key milestones: • Submitted IND to enter first clinical trial in 2H17 ANO\ c • Clinical supply material manufactured in GMP facility for trial .99$71Pl EFTA00292528 Phase 2 Clinical Tr E-CEL Stem-Cell Gene Th ial erapy NYSTEM Ne irogram to advance scientific discoveries in stem cell biology • Award Amount - $15.7M • Award Recipients: Consortium led by Angiocrine Scientific founder — Shahin Raffi, MD (Weill Cornell Medical) and collaborator Michel Sadelain, MD (Memorial Sloan Kettering Cancer Center) Weill Cornell Medical College Memorial Sloan Kettering Cancer Center,. • Disease Focus: Sickle Cell Anemia • Purpose: • Combine Angiocrine's E-CEL Technology with Sloan Kettering's gene correction technology to develop a curative therapy for sickle cell disease • Funds research, pre-clinical development and IND C\ [Angiocrine retains commercial rights to E-CEL ANR9c:firm EFTA00292529 E-CEL Cellular Th Pre-Clinical & IND On-going M1919c:111`lc rapy Program Phase lb Clinical Trial (High-Dose Chemo/ Radiation Recovery) RECOVER Phase 2 Clinical Trial (High-Dose Chemo/ Radiation Recovery H1-2018 H1-2020 EFTA00292530 E-CEL Infusion Accelerates Blood & Immune System Recovery White Blood Cells 3 12 2.5 10 2 8 1.5 6 1 4 0.5 2 0 0 Day 7 Day 14 Day 21 Day 28 Day 7 Red Blood Cells 1200 1000 Day 14 Day 21 Day 28 800 600 400 200 0 Platelets Day 14 Day 21 Day 7 Control Treated with E-CEL Day 28 Immunocompetent C57BL/6 mice — Treatment Day +1 post-LD50 irradiation 12 EFTA00292531 And Also... Systemic Recovery Bone Marrow MG19$=1"A Normal No Treatment E-CEL Treated -,isr‘svitiewk-motrtr No Radiation Day 28 Post-Irradiation 13 EFTA00292532 Multiple Clinical Applications of E-CELTM Applies to virtually all organ regeneration • Brain & Spine • Heart • Lungs • Liver • Kidneys • Muscles & Tendons ANGOCRINE _; ; ­ 1St Clinical Focus: Bone Marrow (Blood & Immune System) EFTA00292533 Angiocrine's Active Research Pipeline Partnering with Experts — Resource Efficient Program Aim Collaborator W-CELTM Stem Cells Neural E-CEL Lung E-CEL Produce one's own blood stem cells from own fat Restore function in spinal cord injury Restore function in chronic lung disease Liver E-CEL Repair without fibrosis in chronic liver disease Tendon/Muscle E-CEL Accelerate healing, strengthen tendon repair Weill ComeII Medical College Drexel UMIVe AAAAA 44. SPINAL CORD RESEARCH CENTER Weill ComeII Medical College Weill ComeII Medical College 0 HOSPITAL FOR SPECIAL SURGERY E-CEL Gene Therapy Treat and cure HIV patients with Cltyof Hope Lymphoma RESEARCH • TREATMENT CURES ANGIOCRINE EFTA00292534 Angiocrine Research: 1P-CEL Technology Create your own stem cells from your skin or fat sample Transformative potential for longevity and rejuvenation science nature e Abstract,. Meals, ProgNor Direct Conversion of Adult Endothelial Cells into Immune- Competent Long-Term Engraftable Clinically Scalable Hematopoietic Stem Cells: Pathway to Therapeutic Translation Lis R et. al., Blood 2016 128:372; Weill Cornell Medical College NATURE I VOL 511 I 17 JULY 2014 Reprogramming human endothelial cells to haematopoietic cells requires vascular induction GI G I INE hneph M Scanclun. & Shahan kalif 16 EFTA00292535 Series ANA9FP,IrN A-1 Equity Financing Pre-Money Valuation: $27M Minimum raise: $4M Maximum: $8M Series A-1 Proceeds towards objectives: $4M 'matching' funds to obtain additional $5M CIRM clinical grant for 1st E-CEL study & complete trial -) $6M + advance E-CEL chemo/rad recovery to 2nd IND + in-license gene therapy vector (e.g., sickle cell) $8nn et99FITIc + complete 2nd IND (cancer chemo/rad treatment recovery program) & apply for 2nd $5M CIRM grant + initiate pre-clinical gene therapy studies EFTA00292536 Anticipated Inflection Points Initiate Interim Trial Safety H2-2017 H1-2018 E-CEL Stem-Cell Gene Therapy 1° Data H1-2019 Phase lb Trial (High-Risk Leukemia) Initiate Interim Trial Safety H1-2018 H2-2018 E-CEL Cellular Therapy (Cancer Care Recovery) Initiate Trial H2-2019 Interim Safety H2-2020 Phase 2b Trial (Sickle Cell Anemia) 1° Data H2-2019 Phase lb Trial Initiate Trial H2-2020 Phase 2/3 Trial April2017 18 EFTA00292537 Angiocrine Bioscie C\ ANR9q1,111c nce Investment Thesis 1. Prior to value inflection points • Transition to clinical stage • Two clinical assets • Expanding pipeline & partnerships 2. Risk-mitigated: • Multiple shots to succeed • Pre-clinical toxicology done on E-CEL • Thoroughly vetted by experts (grants, publications) • Value in robust IP 3. Leveraged: opportunity for more non-dilutive grants 4. Experienced Leadership: solid records, prior exits EFTA00292538 THANK YOU! Contacts: Paul Finnegan, MD, MBA - CEO John Jaskowiak - VP, Corporate Development E-CELTM Therapies tli-CELTM Technology Transforming Hematology and Cancer Care April 2017 via the powers of C.... Engineered Endothelial Cells ANRWIPM EFTA00292539