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Proprietary data of Pluristem Therapeutics Inc.3 Cell therapy (Biotechnology) company. Using off the shelf placental expanded cells to achieve local and systemic therapeutic effect. First in class 3D cell culturing technology allowing for efficient, controlled production of different cell products in commercial quantities. Active with regulators in USA, EU, South Korea, Australia and Israel. Pluristem Corporate Overview
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Proprietary data of Pluristem Therapeutics Inc.1
Advancing cell therapeutic products for clinical use
Proprietary data of Pluristem Therapeutics Inc.2
This presentation concerning Pluristem Therapeutics may include forward-looking statements which represent Pluristem Therapeutics' expectations or beliefs regarding future events. I caution that such statements involve risks and uncertainties that may cause actual results to differ materially from those in the forward-looking statements. Consequently, all such forward-looking statements are qualified by the cautionary language and risk factors set forth in Pluristem Therapeutics' periodic and other reports filed with the SEC.
There can be no assurance that the actual results, events or developments referenced in such forward-looking statements will occur or be realized. Pluristem Therapeutics assumes no obligation to update these forward-looking statements to reflect actual results, changes in assumptions or changes in factors affecting such forward-looking statements.
www.pluristem.com
Forward Looking Statement
Proprietary data of Pluristem Therapeutics Inc.3
• Cell therapy (Biotechnology) company.
• Using off the shelf placental expanded cells to achieve local and
systemic therapeutic effect.
• First in class 3D cell culturing technology allowing for efficient,
controlled production of different cell products in commercial
quantities.
• Active with regulators in USA, EU, South Korea, Australia and Israel.
Pluristem Corporate Overview
Proprietary data of Pluristem Therapeutics Inc.4
• Public company, Traded in:
• Strong balance sheet• Market Cap: ~ $100 million• Cash and marketable securities: $47 million (September 30, 2015)• No debt• Net burn: ~ $22 million• No royalty liability (other than Israeli government) • 165 employees (16 PhD, 4 MD)• IP Ownership: over 50 granted patents and ~150 pending
applications
Financial Glance
PSTI PSTI/ PLTR
Proprietary data of Pluristem Therapeutics Inc.5
Partner Indication Deal structure
IC, CLISouth Korea only
Joint Venture following marketing authorization by the South Korean authorities
Pluristem keeps IP and manufacturing rights in all collaborations
Collaborations
Government Partnership Indication Deal structure
Acute Radiation Syndrome U.S. National Institutes of Health (NIH) to Support Development of PLX-R18
Acute Radiation Syndrome
Pluristem will contribute cells and scientific knowledge, FMU will conduct the studies and provide the required resources.
Proprietary data of Pluristem Therapeutics Inc.6
The PLX Technology
Proprietary data of Pluristem Therapeutics Inc.7
Placenta derived cells• Rich & Diverse• Highly potent - pro-angiogenic - immunoregulatory
• Young donors • Unlimited source• Easy to collect • Ethically accepted• Over 20,000 Doses of 300
million cells per placenta
The Placenta Project- Launched by the US National Institutes of Health (NIH) to Further explore the role of the placenta in health and disease.
Proprietary data of Pluristem Therapeutics Inc.8
3D manufacturing 150,000 doses annually
Proprietary data of Pluristem Therapeutics Inc.9
Culture conditionsPLX-R18Hematological
PLX-PADAngiogenesis
Culture conditions
VEGFIL-10
IL-6bFGF
BDNF
TGFβM
MP-9
NGF
PLX-CNSNeuronal
PLX-IMMUNEImmunological
Human Placenta- A platform for cell products
Culture conditionsCulture conditions
Proprietary data of Pluristem Therapeutics Inc.10
• Broad platform with tailored products per indication• PLX cells can be customized to secrete a specific cytokine
secretion profile to target different indications• Real off the shelf product requiring no additional manipulation
at bedside
• Unique Immunological Properties• No HLA- matching required• Low immunogenicity confirmed :• PLX does not induce in vivo priming of Th1 responses • Repeated IM injections of PLX cells from the same placenta
does not induce patients memory T-cells activation.
• Distinctive MOA• Products demonstrate both local and systemic efficacy via
intramuscular injection
How is PLX Different from the Competition
Proprietary data of Pluristem Therapeutics Inc.11
• Superior Manufacturing• In-house manufacturing which guarantees batch to
batch consistency and high margins
• Near Term Product Approval• First product approval expected 2018 for CLI
How is PLX Different from the Competition
Proprietary data of Pluristem Therapeutics Inc.12
Product 1: PLX-PAD• Reduces inflammation• Stimulates growth of collateral blood vessels• Stimulates repair f damaged muscle
Proprietary data of Pluristem Therapeutics Inc.13
Demonstrated clinical safety and significant efficacy in 3 clinical studies (Two Phase I and one Phase I/II study)
• Muscle Injury following Total Hip Replacement (N=20)
PLX-PAD- Strong Clinical Data
Improvement of 500%
P=0.0067
-10-505
1015202530
Placebo
Time (Weeks)
Change in Volume
from Day 0
6 12 26
150M vs. Placebo : p=0.0040
-20-15-10-505
1015202530
Placebo150M PLX-PAD
6 12 26
150M vs. Placebo p=0.0114
Time (Weeks)
-15-10-505
10152025303540
Placebo150M PLX-PAD
Time (Weeks)6 12 26
150M vs. Placebo : p=0.0067
Injured (operated) Contralateral (non–operated)
Proprietary data of Pluristem Therapeutics Inc.14
Demonstrated clinical safety and significant efficacy in 3 clinical studies (Two Phase I and one Phase I/II study)
• Two Phase I/II Critical Limb Ischemia (N=27)
Pre-Treatment 8 Weeks After TreatmentPre-Treatment
8 Weeks After Treatment
PLX-PAD- Strong Clinical Data
6-8 month follow-up
Proprietary data of Pluristem Therapeutics Inc.15
PLX PAD in CLI via new regulatory pathways
European initiative intended to grant earlier access to drugs meant to treat debilitating and/or life-threatening diseases with unmet medical need.
May 2015 - PLX-PAD cells have been selected for the project. Manufacturing facility approved and inspected by European Qualified Person
for Phase III and marketing. Earlier entry in a multi-billion market (2018)- Conditional marketing approval
in selected population of CLI patient following Initial Phase II study, then post-marketing extension to more CLI patients.
Pluristem intends to expand development of PLX-PAD to additional indicationsvia the Adaptive Pathway project
Pluristem is committed to provide early access of PLX-PAD to Critical limb ischemia (CLI) patients all over the world
Europe - Adaptive Pathways pilot project
1.7 M patients in the major pharmaceutical markets*. Expected to reach 2M in 2020. Two Phase I data with PLX-PAD in CLI patients showing good safety profile and efficacy. Safety data from 150 patients enrolled into IC study to support initial approval in CLI.
*U.S., France, Germany, U.K., Italy, Spain and Japan.
Phase II Trial* Conditional MA*
2016 2017 2018 2019 2020 2021
Collection of real-world Data from treated patients** Full MA
Phase II Trial Two Phase III Trials Full MA
Adaptive Pathways
Traditional Pathway
MA = marketing authorization*Can start marketing product in restricted, niche population**Additional studies in broader population of CLI patients
Target 2018 Product Launch
2021 Traditional Product Launch
Proprietary data of Pluristem Therapeutics Inc.16
New Japanese regulations to accelerate development of drugs in the field of regenerative therapy.
Conditional, time-limited approval for marketing and reimbursement upon proof of safety and a signal of effectiveness. No formal Phase III trial is expected to be required.
April 2015- PLX-PAD quality standards and large-scale manufacturing methods accepted by PMDA.
August 2015- PLX-PAD cells cleared by PMDA for use in clinical trials in Japan.
Pending approval of clinical study design to initiate a Phase II study during H1 2016.
Japan - Accelerated Pathway for Regenerative Medicine
PLX PAD in CLI via new regulatory pathwaysPluristem is committed to provide early access of PLX-PAD to
Critical limb ischemia (CLI) patients all over the world 1.7 M patients in the major pharmaceutical markets*. Expected to reach 2M in 2020. Two Phase I data with PLX-PAD in CLI patients showing good safety profile and efficacy. Safety data from 150 patients enrolled into IC study to support initial approval in CLI.
Conditional MA**
Collection of data from treated patients*** Full MA
Additional Trials to Confirm Safety & Efficacy Full MA
Pathway for Regenerative Medicine
Traditional Pathway
Initial Clinical Study
Exploratory Study*
MA = marketing authorization* If results likely to predict efficacy and confirm safety then conditional, time-limited marketing authorization granted; no confirmation of efficacy needed.** Time limit of 7 years till full MA or withdrawal***Post-marketing safety-monitoring, surveillance and studies
Initial Clinical Study
Target Early Product Launch
Traditional Product Launch
Proprietary data of Pluristem Therapeutics Inc.17
Product 2: PLX-R18• Stimulates regeneration of damaged bone
marrow to produce blood cells (white, red and platelets)
PLX-R18 Pipeline:
Proprietary data of Pluristem Therapeutics Inc.18
PLX-R18 - Targeting Bone Marrow Failure
Main clinical manifestations of low blood counts: Neutropenia: recurrent or severe infections Anemia: fatigue, shortness of breath, pallor, tachycardia Thrombocytopenia: bruising, purpura, petechiae, bleeding
• Bone Marrow Transplant Failure / Incomplete Engraftment FDA IND application approved for Phase I study in the US
• Acute Radiation Syndrome (ARS) – Supported by the NIH (NIAID) Animal Rule pathway Two significant animal trials announced Preparing to submit protocol for large animal trial
• Potential indications include- Chemotherapy, Aplastic Anemia (AA), Acute myeloid leukemia (AML), Myelodysplastic syndrome (MDS)
Proprietary data of Pluristem Therapeutics Inc.19
U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18
Collaboration for ARS with US Government
Proprietary data of Pluristem Therapeutics Inc.20
CMC & Manufacturing Facility for PLX-PAD approved by FDA,
German, EU, South Korean, Japanese & Israeli Regulatory Agencies for 3D culturing for
Phase II, III trials and marketing
Proprietary data of Pluristem Therapeutics Inc.21
IC
CLI-EuropeCLI-JapanOrthopedic Indication
PAH
Preeclampsia
Hematology
ARS
End of enrollment
Proprietary data of Pluristem Therapeutics Inc.21
Company milestonesH1-2016 H2-2016 H1-2017 H2-2017 H1-2018 H2-2018indication
End of enrollment
Data Readout
Licensing deal
Study initiation
Initial approval
Data Readout
End of enrollment
Data Readout
Study initiation
Conditional approval
We will seek to include the indication under the Adaptive licensing pathway- based on discussion with EMA
Pivotal study
Data Readout
BLA application
Conducting additional pre-clinical studies based on discussions with FDA
Product
PLX-
PAD
IND Study initiation
End of enrollment
Data Readout
Adaptive or phase
II/III
PLX-
R18
End of follow up Cohort 2
Proprietary data of Pluristem Therapeutics Inc.22
Zami AbermanChairman & CEO
Efrat Livne-Hadass VP Human Resources
Racheli Ofir, Ph.D.VP Research & Intellectual Property
Sagi MoranVP Operations
Erez Egozi VP Finance
Karine Kleinhaus, M.D., MPHDivisional VP, North America
Yaky YanayPresident & COO
Hillit Mannor Shachar, M.D., M.B.A.VP Business Development
Lior RavivDirector of Development
Esther Lukasiewicz Hagai, M.D., Ph.D. VP Clinical & Medical Affairs
Orly AmiranVP Quality Assurance
Management Team
Proprietary data of Pluristem Therapeutics Inc.23
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