Pharmacyte Biotech, Inc. · NASDAQ:PMCB
Pharmacyte Biotech — Investor Presentation (September 2021)
40 slides from Pharmacyte Biotech's investor presentation, Sep 10, 2021
Slide 1: Presentation of PharmaCyte Biotech
PHARMACYTE BIOTECH Presentation of PharmaCyte Biotech (September 9, 2021)
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 Encapsulated cells convert a prodrug from its inactive form to its cancer - killing form o Encapsulated cells are implanted near the site of the tumor; low dose chemotherapy prodrug is given intravenously. Encapsulated cells
Slide 5: Pipeline
Pipeline Pre cli ni c a l Phase 1 Phase 2b Phase 3 Encapsulated live cells converting Ifosfamide – antitumor effectiveness and good quality of life* Pancreatic Cancer: Preclinical Phase 1 Phase 2 Phase 3 Preclinical Phase 1 Phase 2 Phase 3 Diabetes : Encapsulated live cells producing insulin on dema
Slide 6: Platform Technology Targeted Chemotherapy for Solid Tumors
Platform Technology 6 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 it cannot be used at the normal dose due to severe toxicity • Cell - in
Slide 7: Cell-in-a-Box Capsules Unique Encapsulation Material
Cell - in - a - Box ® Capsules 7 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 encaps
Slide 8: 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 8 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 P anc r e as tumor 9
Slide 10: Placement of Encapsulated Cells
Placement of Encapsulated 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 10
Slide 11: Pancreatic Cancer: Aggressive Cancer with Poor Prognosis
Pancreatic Cancer 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 of diag
Slide 12: Pancreatic Cancer: Current First Line Therapy
Pancreatic Cancer (cont’d) 12 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 Abraxa
Slide 13: Trials in Pancreatic Cancer Using Our Product Candidate
Trials in Pancreatic Cancer Using Our Product 13 Candidate 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 : g
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 Trial in Pancreatic Cancer Addressing Critical Unmet Medical Need
Planned Trial in Pancreatic Cancer 15 Addressing Critical Unmet Medical Need • A critical unmet medical need exists for patients with pancreatic cancer whose tumors are locally advanced, non - metastatic and inoperable but no longer respond to Abraxane ® + gemcitabine or FOLFIRINOX • These patients
Slide 16: Trial Design
Trial Design 16 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 tumors no
Slide 17: Trial Design (cont'd)
Trial Design (cont’d) 17 • Eligibility: Patients must have been treated with Abraxane ® + gemcitabine or FOLFORINOX for 4 - 6 months until their tumors no longer respond to this therapy or the therapy is refused • Randomization: 100 patients will be randomized into two groups. 50 patients will recei
Slide 18: Trial Design: Secondary Endpoints
Secondary Endpoints: • Overall - survival is evaluated as the time from randomization to death by any cause • Objective response rate is evaluated as the combined incidence of complete response (CR) and partial response (PR) • Conversion from inoperable at randomization to operable post - treatment
Slide 19: Trial Design Oncologists
Trial Design Oncologists Leading Experts in Development of Therapies to Treat Pancreatic Cancer Dr. Daniel D. Von Hoff 19 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’s 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.
Slide 21: Trial Preparations: Manufacturing Capability
Trial Preparations 21 Manufacturing Capability • GMP facility successfully audited and deemed ready for production of GMP clinical trial product, known as “CypCaps Ρ ” • Two staggered and back - to - back manufacturing runs of clinical trial successfully completed • FDA required “release testing” co
Slide 22: Trial Preparations (cont'd)
• Dr. Manuel Hidalgo selected as Principal Investigator • Protocol and Investigator Brochure finalized, 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 t
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, 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 PharmaCyte
Slide 24: Orphan Drug Designations and IP Portfolio
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 Fluid Accumulation
Malignant Ascites Fluid Accumulation 25 Targeted Chemotherapy to Treat Malignant Ascites • 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 causing swelling
Slide 26: Bio-Artificial Pancreas for Diabetes
Bio - Artificial Pancreas for Diabetes • PharmaCyte’s Diabetes Program consists of encapsulating genetically modified 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 diabetes and
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: Cannabis Program: Anti-Cancer Effects of Cannabinoids
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 in
Slide 29: Cannabis Program (cont'd)
Cannabis Program (cont’d)* Cannabi s - d er i v ed 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 l
Slide 30: 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 31: Development of Pain Therapy
Development of Pain Therapy • Chronic pain 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 Cell -
Slide 32: Kenneth L. Waggoner, J.D. – Chief Executive Officer, President and General Counsel • Over
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 clients such as
Slide 33: Medical and Scientific Advisory Board
Medical and Scientific Advisory Board 33 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
Slide 34: 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 part
Slide 35: FDA Guidance to Have Clinical Hold Lifted
FDA Guidance to Have Clinical Hold Lifted • Provide additional sequencing data and genetic stability studies • Conduct stability study on CypCaps Ρ and cells from MCB • Evaluate the compatibility of the delivery devices (prefilled syringes and microcatheter used to implant CypCaps Ρ ) with our drug
Slide 36: Appendix A (cont'd)
• Revise Investigators Brochure to include the preclinical studies conducted in response to the clinical hold and remove any statements not supported by the data • Provide data from new pig toxicology study The FDA requested we address the following issues as an amendment to the IND • Provide a Cert
Slide 37: Scientific and Regulatory Team of Experts
Scientific and Regulatory Team of Experts Assembled a scientific and regulatory team of experts to address FDA’s requests. Working to complete the items requested by FDA. At varying stages of addressing the studies and information requested by the FDA • Successfully completed a 3, 6, 9 and 12 - mont
Slide 38: Appendix A (cont'd)
• Austrianova manufactured and delivered 400 syringes of empty capsules to laboratory conducting biocompatibility studies • Designed and commenced studies designed to show CypCaps Ρ were not adversely affected by catheters used by interventional radiologists to deliver CypCaps Ρ nor by contrast medi
Slide 39: Appendix A (cont'd)
• In the process of updating our IND submission documents to include (i) more pre - clinical data; (ii) additional parameters for release of CypCaps Ρ ; (iii) recommendation of the catheters and contrast medium to be used to deliver CypCaps Ρ ; and discussion of the potential for cellular and humora
Slide 40: Contact Investor Relations for Further Information
Contact Investor Relations for Further Information ( InvestorRelations@PharmaCyte.com ) Thank you Investor Relations 40
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