PharmaCyte Biotech, Inc. · NASDAQ:PMCB
PharmaCyte Biotech — Investor Presentation (May 2016)
34 slides from PharmaCyte Biotech's investor presentation, May 29, 2016
Slide 1: PharmaCyte Biotech Investor Presentation
PHARMACYTE BIOTECH Investor Presentation
Slide 2: Safe Harbor Statement
Safe Harbor Statement • This document may include statements that constitute “forward looking statements,” which are often characterized by the terms “may,” “believes,” expects” or “anticipates” and do not reflect facts. • Forward-looking statements involve risks, uncertainties and other factors tha
Slide 3: Pancreatic Cancer Targeted Chemotherapy
Pancreatic Cancer Targeted Chemotherapy Pancreatic Cancer is the 3rd Leading Cause of Cancer Deaths in the U.S. • Overall survival is only 8% • Typically not diagnosed until cancer is advanced and inoperable • No cure unless the cancer is surgically removed in its earliest stages • No targeted chemo
Slide 4: PharmaCyte Platform Technology
PharmaCyte Platform Technology Targeted Chemotherapy for Solid Tumors • Using the Cell-in-a-Box® technology, encapsulate genetically engineered live cells to convert prodrug into its active form • Targeted and activated therapy at site of tumor • Allows for use of lower and safer doses of cytotoxic
Slide 5: Properties of Cell-in-a-Box Capsules
Properties of Cell-in-a-Box® Capsules Unique Encapsulation Material and Design • Capsules are made of bio-inert material (cellulose/cotton) • Capsules have pores for nutrient and waste transfer • Pores are too small for immune system cells to enter or encapsulated live cells to leave • Long-term (5+
Slide 6: Cell-in-a-Box Capsules vs. Alginate Capsules
Cell-in-a-Box ® Capsules vs. Alginate Capsules Property Cell-in-a-Box® Alginate No purification required Yes No Can exist intact for >2 years in the body Yes No Protect encapsulated live cells from immune Yes No system attack for >2 years Do not cause damage to surrounding tissues Yes No while in th
Slide 7: Pancreatic Cancer
Pancreatic Cancer Aggressive Cancer with Poor Prognosis • Third leading cause of cancer-related deaths in the western world • Overall survival rate is 8% • Expected pancreatic cancer patients in 2016: U.S. >53,000; Europe >80,000 • Approximately 72% die within the first year of diagnosis • More than
Slide 8: Pancreatic Cancer (cont'd)
Pancreatic Cancer (cont’d) Current “gold standard” is Combination of Abraxane® + Gemcitabine • Combination approved by FDA in September 2013 • Increased median survival by 1.8 months as compared to gemcitabine alone • Increased the percentage of one-year survivors from 22% with gemcitabine alone to
Slide 9: PharmaCyte Pancreatic Cancer Therapy
PharmaCyte Pancreatic Cancer Therapy Targeted Chemotherapy • Cell-in-a-Box® encapsulated live cells + cancer prodrug ifosfamide • Ifosfamide at “normal dose” has shown success in treating testicular cancer • Cannot be used for pancreatic cancer at normal dose due to severe toxicity • Cell-in-a-Box®
Slide 10: Targeted Deployment and Activation
Targeted Deployment and Activation • Capsules containing live ifosfamide-activating cells are implanted in the blood vessels leading to the pancreatic tumor • Low dose ifosfamide is given intravenously • Ifosfamide is converted to its cancer-killing form by the encapsulated live cells Blue Arrow: If
Slide 11: Mechanism of Action
Mechanism of Action Inactive prodrug Activated ifosfamide molecules ifosfamide cancer killing in-situ Pancreas tumor Immune system cells too large to enter capsule Nutrients Single capsule containing Waste products ifosfamide-activating live cells 11
Slide 12: Placement of Encapsulated Live Cells
Placement of Encapsulated Live Cells A. Angiography of blood vessels to the pancreas B. Insertion of catheter into the pancreas blood vessel (arrow) C. Injection of microcapsules D. Angiography shortly after capsule implantation (arrow) Blood supply to the pancreas is NOT impeded by the capsules 12
Slide 13: Phase 1/2 Clinical Trial
Phase 1/2 Clinical Trial Trial Design and Endpoints • Fourteen patients were treated with only two courses of ifosfamide at 1/3 (1 g/m2) of the dose normally used to treat other forms of cancer • Patients with advanced, inoperable pancreatic cancer were treated in a single- arm (no comparator arm) t
Slide 14: Previous Clinical Trials
Previous Clinical Trials Phase 1/2 Clinical Trial with Two Courses of Low Dose Ifosfamide • Median survival: gemcitabine – 5.7 months vs. Cell-in-a-Box® + ifosfamide – 10 months • Percentage of 1-year survivors: gemcitabine – 18% vs. Cell-in-a-Box® + ifosfamide – 36% • Treatment-related side effects
Slide 15: Survival Summary of Clinical Trials
Survival Summary of Clinical Trials 14 Patients 13 Patients 36 Patients Kaplan–Meier curves describing the survival of patients from the Phase 1/2 clinical trial (green boxes), the Phase 2 clinical trial (blue triangles) and an age and disease stage matched historic control group receiving the best
Slide 16: Phase 2b Clinical Trial
Phase 2b Clinical Trial Therapy to Address Critical Unmet Medical Need • A critical unmet medical need exists for patients with advanced pancreatic cancer whose tumors are locally advanced, non-metastatic and inoperable but no longer respond to Abraxane® + gemcitabine or to FOLFIRINOX • These patien
Slide 17: Phase 2b Clinical Trial (cont'd)
Phase 2b Clinical Trial (cont’d) Elements of Trial Design • Trial will be two-armed. Patients will be randomized to receive PharmaCyte’s therapy or the standard of care consisting of capecitabine + radiation • Trial will be conducted in the United States and Europe • Trial is designed to show Cell-i
Slide 18: Phase 2b Clinical Trial (cont'd)
Phase 2b Clinical Trial (cont’d) • Only patients whose tumors are locally advanced, inoperable and non- metastatic will be eligible to be enrolled • Patients must have been treated with either Abraxane ® + gemcitabine or FOLFIRINOX for 4-6 months and their tumors must no longer respond to this thera
Slide 19: Phase 2b Clinical Trial Design Oncologists
Phase 2b Clinical Trial Design Oncologists Leading Experts in Development of Therapies to Treat Pancreatic Cancer Dr. Daniel D. Von Hoff Dr. Manuel Hidalgo Dr. Matthias Löhr 19
Slide 20: Phase 2b Clinical Trial Design Oncologists (cont'd)
Phase 2b Clinical Trial Design Oncologists (cont’d) Dr. Daniel D. Von Hoff • The world’s leading oncologist in the development of drugs to treat pancreatic cancer • Involved in clinical trials of more than 200 anticancer and biologic drugs • Conducted early clinical trials for most of the cancer age
Slide 21: Clinical Trial Preparations
Clinical Trial Preparations Manufacturing Capability • cGMP facility successfully audited and deemed ready for production • Production of clinical trial material being validated • Clinical trial material expected to be available in August 2016 Phase 2b Trial in United States and Europe • Protocol sy
Slide 22: Pancreatic Cancer Addressable Market
Pancreatic Cancer Addressable Market Approximately $3.0 Billion Globally within Next Five Years • Current pancreatic cancer addressable market in United States o New cases in U.S. in 2016* 53,070 o Average price range per cycle: $50,000 $75,000 o Average price per cycle: $62,500 o Potential patient
Slide 23: Malignant Ascites Fluid Accumulation
Malignant Ascites Fluid Accumulation Targeted Chemotherapy to Treat Malignant Ascites Fluid Accumulation • Malignant ascites fluid is secreted by abdominal tumors into the abdomen • Contains cancer cells that can seed and form new tumors throughout the abdomen • Accumulates in the abdominal cavity c
Slide 24: PharmaCyte Diabetes Program
PharmaCyte Diabetes Program Develop Artificial Pancreas for Diabetes • PharmaCyte has the exclusive world-wide license to use Melligen cells to treat diabetes o Melligen cells are human liver cells that have been genetically modified to produce, store and release insulin in response to concentration
Slide 25: Cell-in-a-Box + Pancreatic β-Islet Cells
Cell-in-a-Box® + Pancreatic β-Islet Cells Successful Proof of Principle • Preclinical proof of principle for an “artificial pancreas” has been established using Cell-in-a-Box® capsules containing pig pancreatic β-islet (insulin-producing) cells in a rat model of Type 1 diabetes • Soon after Cell-in-
Slide 26: International Diabetes Consortium
International Diabetes Consortium Clinical Development Accelerated • PharmaCyte has established an international Diabetes Consortium of world renowned physicians and scientists specializing in the treatment of diabetes and diseases of the pancreas o Karolinska Institute, Stockholm, Sweden; Heidelber
Slide 27: Diabetes Market
Diabetes Market Type1 and Insulin-Dependent Type 2 Diabetes and Related Healthcare • Diabetes has reached epidemic proportion globally • 422 million people have diabetes – 314 million more than in 1980 • 8.5% of adults worldwide have diabetes • ~$920 billion spent annually in treatment of diabetes a
Slide 28: Orphan Drug Status and IP Portfolio
Orphan Drug Status and IP Portfolio FDA Granted Orphan Drug Designation for Pancreatic Cancer Therapy • Provides 7 years of market exclusivity in the United States EMA Granted Orphan Drug Designation for Pancreatic Cancer Therapy • Provides 10 years of market exclusivity in the European Union Eligib
Slide 29: Leadership Team
Leadership Team Kenneth L. Waggoner, J.D. - Chief Executive Officer, President and General Counsel • Over four decades of experience in law, management, operations and business • Senior partner with Brobeck, Phleger and Harrison, one of the top law firms worldwide providing services to biotechnology
Slide 30: Medical and Scientific Advisory Board
Medical and Scientific Advisory Board Dr. Matthias Löhr (Chairman) • One of Europe’s leading authorities in diseases of the pancreas (pancreatic cancer, diabetes) • Principal Investigator for previous clinical trials of PharmaCyte’s pancreatic cancer therapy Dr. Manuel Hidalgo • Internationally reno
Slide 31: Key Company Facts
Key Company Facts SELECTED FINANCIAL INFORMATION Stock Price (05/27/2016) ~$0.07 52-Week Range ~$0.04-$0.16 Trading Symbols: OTCQB: PMCB Shares Outstanding ~ 780 million 90-Day Average Trading Headquarters: Silver Spring, MD ~ 1.2 million shares Volume Fiscal Year End: April 30 Market Capitalization
Slide 32: 2016 Milestones
2016 Milestones Achieved and Remaining Thai FDA inspected and approved encapsulation facility for product production Encapsulation facility deemed ready for cGMP production of encapsulated live cells • Pre-IND meeting is held with the U.S. FDA regarding Phase 2b clinical trial • IND for the Phase
Slide 33: 2016 Milestones (cont'd)
2016 Milestones (cont’d) • Additional preclinical studies are carried out by TD2 to determine if PharmaCyte’s cancer therapy is effective in slowing the production and accumulation of malignant ascites fluid in the abdomen • Preparations made to conduct clinical trial in ascites in early 2017 • Nume
Slide 34: Questions and Answers
Questions and Answers Discussion and Next Steps Thank you 34
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