Pharmacyte Biotech, Inc. · NASDAQ:PMCB
Pharmacyte Biotech — Investor Presentation (March 2022)
44 slides from Pharmacyte Biotech's investor presentation, Mar 9, 2022
Slide 1: Introduction of PharmaCyte Biotech
PHARMACYTE BIOTECH Introduction of PharmaCyte Biotech March 7, 2022
Slide 2: Safe Harbor Statement and Disclaimer
• This document may include statements by PharmaCyte Biotech that constitute “forward - looking statements.” Such statements are often characterized by the terms “may,” “believes,” expects” or “anticipates” and do not reflect facts. • Forward - looking statements involve risks, uncertainties and oth
Slide 3: Safe Harbor Statement and Disclaimer (cont'd)
• This presentation shall not constitute an offer to sell or a solicitation of an offer to buy any securities, nor shall there be any sale of such securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the secu
Slide 4: Platform Technology for Cancer and Diabetes
Encapsulate Genetically Modified Live Cells to Treat Diseases • Cancer : o Encapsulate genetically engineered live cells that convert a prodrug from its inactive form to its cancer - killing form o Encapsulated live cells are implanted near the site of the tumor; low dose chemotherapy prodrug ifosfa
Slide 5: Pipeline
Pipeline Preclinical Phase 1 Phase 2b Phase 3 Encapsulated live cells converting Ifosfamide – antitumor effectiveness and much improved quality of life* Pancreatic Cancer: Preclinical Phase 1 Phase 2 Phase 3 Preclinical Phase 1 Phase 2 Phase 3 Encapsulated live cells producing insulin on demand for
Slide 6: Platform Technology Targeted Chemotherapy for Solid Tumors
Platform Technology Targeted Chemotherapy for Solid Tumors • Cell - in - a - Box ® encapsulated live cells + cancer prodrug ifosfamide are used • Ifosfamide at its “normal dose” has shown success in treating some cancers, but cannot be used at the normal dose due to severe toxicity • Cell - in - a -
Slide 7: Cell-in-a-Box Capsules Unique Encapsulation Material
Cell - in - a - Box ® Capsules Unique Encapsulation Material • Capsules are made of bio - inert material (cellulose/cotton) • Capsules have pores for nutrient and waste transfer • The pores are too small for immune system cells to enter or encapsulated live cells to leave the capsules • The encapsul
Slide 8: Targeted Deployment and Activation
Targeted Deployment and Activation 8 • 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 Arr
Slide 9: Mechanism of Action
Mechanism of Action Immune system cells too large to enter capsule Single capsule containing ifosfamide - activating live cells Inactive ifosphamide “prodrug” “Activated” ifosfamide molecules cancer killing in - situ Pancreas tumor 9
Slide 10: Placement of Encapsulated Cells
Placement of Encapsulated Cells 10 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
Slide 11: Pancreatic Cancer Program: Aggressive Cancer with Poor Prognosis
Pancreatic Cancer Program Aggressive Cancer with Poor Prognosis • Increasing in most industrialized countries • Third leading cause of cancer - related deaths in the western world • Expected pancreatic cancer patients in 2021: U.S. >60,430; 48,480 deaths • Approximately 80% die within the first year
Slide 12: Current First Line Therapy
Current First Line Therapy 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 to 38% with Abraxane ® + gemcitabine • Severe si
Slide 13: Clinical Trials in Pancreatic Cancer
13 Clinical Trials in Pancreatic Cancer Phase 1/2 Clinical Trial with Two Courses of Low Dose Ifosfamide (1998 - 1999) • Fourteen patients were treated with only two courses of ifosfamide at 1/3 (1 g/m) of the dose normally used to treat other forms of cancer • Median survival : gemcitabine = 5.7 mo
Slide 14: Phase 1/2 Clinical Trial: Trial Design and Endpoints
Phase 1/2 Clinical Trial . 14 Trial Design and Endpoints • Patients with advanced, inoperable pancreatic cancer were treated in a single - arm (no comparator arm) trial at a single study site in Rostock, Germany • Feasibility, safety, tolerability and clinical benefit were endpoints • Tumor responsi
Slide 15: Planned Clinical Trial in Pancreatic Cancer
Planned Clinical Trial in Pancreatic Cancer Addressing Critical Unmet Medical Need • A critical unmet medical need exists for patients with pancreatic cancer whose tumors are locally advanced and inoperable but no longer respond to Abraxane ® + gemcitabine or FOLFIRINOX • These patients have no effe
Slide 16: Clinical Trial Design
Clinical Trial Design Elements of Trial Design • Design: Trial will be two - armed • Location: Trial will be conducted in the U.S. • Objective: Trial is designed to show Cell - in - a - Box ® + low - dose ifosfamide can serve as an effective and safe consolidation chemotherapy for patients whose tum
Slide 17: Clinical Trial Design (cont'd)
Clinical Trial Design (cont’d) • Start Date: Trial is expected to start in Q3 of 2022, subject to the FDA’s timely lifting of the clinical hold • Eligibility: Only patients whose tumors are locally advanced, inoperable and non - metastatic will be eligible to be enrolled . Patients must have been tr
Slide 18: Clinical Trial Design: Secondary Endpoints
• Secondary Endpoints: o Overall - survival is evaluated as the time from randomization to death by any cause o Objective response rate is evaluated as the combined incidence of complete response and partial response o Conversion from inoperable at randomization to operable post - treatment is evalu
Slide 19: Trial Design Oncologists
Trial Design Oncologists Leading Experts in Development of Therapies to Treat Pancreatic Cancer 19 Dr. Daniel D. Von Hoff Dr. Manuel Hidalgo Dr. Matthias Löhr
Slide 20: Trial Design Oncologists (cont'd)
Trial Design Oncologists (cont’d) 20 Dr. Daniel Von Hoff • A world leading oncologist in the development of drugs to treat pancreas cancer • Involved in clinical trials of more than 200 anticancer and biologic drugs • Conducted early clinical trials for most of the cancer agents approved in the U.S.
Slide 21: Manufacturing Capability and Trial Preparations
Manufacturing Capability and Trial Preparations 21 Manufacturing Capability • GMP facility successfully audited and deemed ready for production of clinical trial product candidate known as “CypCaps Ρ ” • Two staggered and back - to - back manufacturing runs of clinical trial product candidate succes
Slide 22: Trial Preparations (cont'd)
Trial Preparations (cont’d) 22 Trial Preparations • Protocol and Investigator Brochure prepared, subject to input from the FDA • Practical Clinical retained to function as the Director of Clinical Operations • Medpace selected as CRO to conduct the trial. Medpace is among the top 10 CROs in the worl
Slide 23: IND Submission to FDA
IND Submission to FDA • IND filed with FDA on September 1, 2020, to commence Phase 2b clinical trial in locally advanced and inoperable pancreatic cancer (LAPC) • On October 1, 2020, received notice that the FDA had placed the IND on clinical hold • On October 30, 2020, FDA sent a letter to PharmaCy
Slide 24: FDA Granted Orphan Drug Designation for Pancreas Cancer Therapy
FDA Granted Orphan Drug Designation for Pancreas Cancer Therapy • Provides 7 years of market exclusivity in the U.S. upon FDA approval EMA Granted Orphan Drug Designation for Pancreas Cancer Therapy • Provides 10 years of market exclusivity in the E.U. upon EMA approval Eligible for Biologics Price
Slide 25: Malignant Ascites Program
25 Malignant Ascites Program Targeted Chemotherapy to Treat Malignant Ascites • Malignant ascites 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 causing swelling of the abdomen, s
Slide 26: Bio-Artificial Pancreas for Diabetes
Bio - Artificial Pancreas for Diabetes • PharmaCyte’s Diabetes Program consists of encapsulating genetically engineered live cells (Melligen cells and stem cells) that produce insulin in proportion to a patient’s blood glucose level and then implanting the capsules in the body to treat Type 1 diabet
Slide 27: Diabetes Program (cont'd)
o In the past, PharmaCyte’s International Diabetes Consortium encountered difficulties related to the stability of the Melligen cells o PharmaCyte then spent two years recreating a stable version of the Melligen cells • UTS entered into an agreement with PharmaCyte to create a new, stable and advanc
Slide 28: Development Timeline Accelerated
Development Timeline Accelerated • PharmaCyte 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; Heidelberg University, Heidelberg, Germany; U
Slide 29: Cannabis Program: Anti-Cancer Effects of Cannabinoids
29 Cannabis Program* Anti - Cancer Effects of Cannabinoids • Properties of cannabinoids (THC and CBD): o Anti - proliferative: slow tumor growth o Anti - metastatic: slow tumor spread o Anti - angiogenic: slow blood vessel penetration o Pro - apoptotic: initiate programmed cell death • In vitro and
Slide 30: Cannabis Program (cont'd)
30 Cannabis Program (cont’d)* Cannabis - derived cannabinoid “prodrug” Bio - engineered cell line encapsulated with Cell - in - a - Box® produces activating enzyme Targeted therapy “active” cannabinoid cancer cell death Targeted Cannabinoid Chemotherapy • PharmaCyte has an exclusive, worldwide lic
Slide 31: Research Program with University of Northern Colorado
Research Program with University of Northern Colorado • Initial goal was to develop methods for identification, separation and quantification of constituents of Cannabis which are prodrugs requiring activation. Methods have now been developed • The focus of this aspect of the Cannabis Program is: o
Slide 32: Development of Pain Therapy
Development of Pain Therapy • Chronic pain is linked to restricted mobility, opioid dependency, anxiety, depression and reduced quality of life • Estimated $560 billion annually in direct costs, lost productivity and disability programs in U.S. • Additional goal of Cannabis Program is to utilize Cel
Slide 33: Leadership Team
Kenneth L. Waggoner, JD – 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 clients such as C
Slide 34: Leadership Team (cont'd)
Carlos A. Trujillo, CPA – Chief Financial Officer • Has 36 years of experience in finance, accounting and management • For the last thirteen years, he has been the Chief Financial Officer for privately held, publicly traded and multinational companies 34 Leadership Team (cont’d)
Slide 35: Medical and Scientific Advisory Board
Medical and Scientific Advisory Board Dr. Matthias Löhr (Chairman ) • A world - renowned European oncologist/gastroenterologist at the Karolinska Institute in Stockholm, Sweden. Expert in the treatment of pancreas cancer and diabetes • Served as Principal Investigator for the Phase 1/2 and Phase 2 c
Slide 36: Medical and Scientific Advisory Board (cont'd)
Dr. Mark L. Rabe • Leader in use of cannabis to treat diseases and their symptoms • Served as Chief Medical Officer of California’s largest network of physician - owned cannabis evaluation centers David A. Judd • Cellular biologist with the Grand Island Biological Company for over 30 years with a pa
Slide 37: FDA Guidance to Have Clinical Hold Lifted
FDA Guidance to Have Clinical Hold Lifted • Provide additional sequencing data and genetic stability studies • Conduct a stability study on the final formulated product candidate as well as the cells from PharmaCyte’s Master Cell Bank (MCB) • Evaluate the compatibility of the delivery devices (prefi
Slide 38: FDA Requested Issues to Address as an Amendment to the IND
The FDA Requested PharmaCyte to Address the Following Issues as an Amendment to the IND • Provide a Certificate of Analysis for pc3/2B1 plasmid that includes tests for assessing purity, safety and potency • Perform qualification studies for CypCaps Ρ filling process to ensure that the product candid
Slide 39: Scientific and Regulatory Team Working on FDA Requests
Scientific and Regulatory Team of Experts Working on FDA Requests PharmaCyte assembled a scientific and regulatory team of experts to address the FDA’s requests. PharmaCyte is working to complete the items requested by FDA and making the following progress: • Additional Regulatory Expertise Added to
Slide 40: Cytochrome P450 2B1 Gene Sequence Determination
• Determination of the Exact Sequence of the Cytochrome P450 2B1 Gene . PharmaCyte has completed the determination of the exact sequence of the cytochrome P450 2B1 gene inserted at the site previously identified on chromosome 9 using state - of - the - art nanopore sequencing, a cutting edge, unique
Slide 41: Biocompatibility Studies
• Biocompatibility Studies . PharmaCyte has designed and commenced 8 biocompatibility studies, 6 of which have been completed successfully. The remaining 2 studies are underway. Those studies are the Acute Systemic Toxicity Study of Empty Cellulose Sulphate Capsules in Mice and the Skin Sensitizatio
Slide 42: Capsule Compatibility with Microcatheter Delivery System
• CypCaps Capsules Compatibility with the Syringe and Other Components of the Microcatheter Delivery System . PharmaCyte has commenced studies designed to show that CypCaps Ρ are not in any way adversely affected by the catheters used by interventional radiologists to deliver them into a patient. Co
Slide 43: Appendix A (cont'd): Additional Documentation Requested by the FDA
• Additional Documentation Requested by the FDA . The Company is in the process of updating its IND submission documentation, including extending its discussion on immunological aspects of its treatment for LAPC • Pig Study . Finally, the Company has designed a study in pigs to address biocompatibil
Slide 44: Contact Investor Relations for Further Information
Contact Investor Relations for Further Information InvestorRelations@PharmaCyte.com Thank you Investor Relations 44
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