EFTA00292520.pdf

ANGIOCRINE BIOSCI ENCE Investor Update May 4th 2017 OO Nig9FRPA EFTA00292520  Fact: Endothelial Cells Instruct Stem Cells Endothelial Cells --- Angiocrine Factors Organ-Specific Stem Cells Signaling proteins Z\ C_N, This is how vital organs in humans regenerate MA19$:A!Ng 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 arum CALIFOR111111 /TEM CELL ',Gene* 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 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) 2. E-CEL Cellular Therapy • Goal: Accelerate recovery from radiation/chemotherapy • Clinical stage: H1 2018 (1" trial, various cancers) e199R1I'M EFTA00292524  Substantial Global Markets Gene Therapy Market Cancer Care Market • Genetic diseases (e.g. Sickle Cell • 40% of adults will be Anemia, Primary Immuno-Deficiency, diagnosed with cancer during etc.) are responsible for a heavy their lifetimes5 loss of life."' • 1,685,210 new US cancer • > 6,000 known single-gene cases diagnosed in 20165 defect disorders2 • Significant number of patients • "worth USD 11 billion by will receive high-dose 2025"3 chemotherapy and/or • "an area of important future radiation treatment growth" (GlaxoSmithKline)4 , 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. RINE 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 Phase lb Trial Phase 2 Trial Pre-Clinical & (Leukemia with high- IND (Sickle Cell Anemia) risk genetic profile) Done H2-2017 H 2-2019 C\\ Ml,g9FP,M 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-Human Clinical Trial E-CEL Ste 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\ • Clinical supply material manufactured in GMP facility for trial .99$71Plc EFTA00292528 Phase 2 Clinical Trial E-CEL Stem-Cell Gene Therapy NYSTEM Ne irogram to advance scientific discoveries in stem cell biology • Award Amount - $15.7M • Award Recipients: Weill Cornell Medical College Consortium led by Angiocrine Scientific founder — Shahin Raffi, MD (Weill Cornell Medical) and collaborator Michel Sadelain, MD (Memorial Sloan Kettering Cancer Center) Memorial Sloan Kettering Cancer Center,. • Disease Focus: Sickle Cell Anemia • Purpose: • Combine Angiocrine's E-CEL Technology with Sloan Kettering's gene correction technology to develop a curative therapy for sickle cell disease • Funds research, pre-clinical development and IND C\ [Angiocrine retains commercial rights to E-CEL ANR9c:firm EFTA00292529  E-CEL Cellular Th rapy Program RECOVER Phase lb Phase 2 Pre-Clinical & Clinical Trial Clinical Trial IND (High-Dose Chemo/ (High-Dose Chemo/ Radiation Recovery) Radiation Recovery On-going H1-2018 H1-2020 M1919c:111`lc EFTA00292530  E-CEL Infusion Accelerates Blood & Immune System Recovery White Blood Cells Red Blood Cells Platelets 3 12 1200 2.5 10 1000 2 8 800 1.5 6 600 1 4 400 0.5 2 200 0 0 0 Day 7 Day 14 Day 21 Day 28 Day 7 Day 14 Day 21 Day 28 Day 7 Day 14 Day 21 Day 28 Control Treated with E-CEL 12 Immunocompetent C57BL/6 mice — Treatment Day +1 post-LD50 irradiation EFTA00292531  And Also... Systemic Recovery Normal No Treatment E-CEL Treated -,isr‘svitiewk-motrtr Bone Marrow 13 MG19$=1"A No Radiation Day 28 Post-Irradiation EFTA00292532  Multiple Clinical Applications of E-CELTM Applies to virtually all organ regeneration • Brain & Spine _; ; • Heart ➢ 1St Clinical Focus: • Lungs Bone Marrow • Liver (Blood & Immune System) • Kidneys • Muscles & Tendons ANGOCRINE EFTA00292533  Angiocrine's Active Research Pipeline Partnering with Experts — Resource Efficient Program Aim Collaborator W-CELTM Stem Cells Produce one's own blood stem cells Weill ComeII Medical College from own fat Neural E-CEL Restore function in spinal cord 44. SPINAL CORD Drexel RESEARCH CENTER injury UMIVe AAAAA Lung E-CEL Restore function in chronic lung Weill ComeII Medical College disease Liver E-CEL Repair without fibrosis in chronic Weill ComeII Medical College liver disease Tendon/Muscle E-CEL Accelerate healing, strengthen tendon repair 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 potentialfor longevity and rejuvenation science Direct Conversion of Adult Endothelial Cells into Immune- Competent Long-Term Engraftable Clinically Scalable Hematopoietic Stem Cells: Pathway to Therapeutic e Abstract,. Translation Meals, ProgNor Lis R et. al., Blood 2016 128:372; Weill Cornell Medical College NATURE I VOL 511 I 17 JULY 2014 Reprogramming human endothelial nature cells to haematopoietic cells requires vascular induction 16 GI G I INE hneph M Scanclun. & Shahan kalif EFTA00292535  Series A-1 Equity Financing ANA9FP,IrN 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 + complete 2nd IND (cancer chemo/rad treatment recovery program) & apply for 2nd $5M CIRM grant + initiate pre-clinical gene therapy studies et99FITIc EFTA00292536  Anticipated Inflection Points Initiate Interim Initiate Interim 1° Data Trial Safety Trial Safety H1-2019 H2-2017 H1-2018 H2-2019 H2-2020 E-CEL Stem-Cell Phase lb Trial Phase 2b Trial Gene Therapy (High-Risk Leukemia) (Sickle Cell Anemia) Initiate Interim Initiate Trial Safety 1° Data Trial H1-2018 H2-2018 H2-2019 H2-2020 E-CEL Cellular Therapy Phase 2/3 Phase lb Trial (Cancer Care Recovery) Trial 18 April2017 EFTA00292537  Angiocrine Bioscience 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 C\ ANR9q1,111c 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