Advancing cell therapeutic products for clinical use v2.… · Advancing cell therapeutic products...

Proprietary data of Pluristem Therapeutics Inc.

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Advancing cell therapeutic products for clinical use


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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


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• Cell therapy company (NasdaqCM: PSTI, TASE: PSTI) • Using off-the-shelf, placenta-derived cells to achieve both local

and systemic therapeutic effects – no tissue matching needed • First-in-class 3D cell culturing technology allowing for efficient,

controlled production of multiple cell products in commercial quantities – “the process is the product”

• Active with regulators in the U.S., EU, S. Korea, Australia & Israel

• Demonstrated safety and efficacy in 3 clinical studies (two Phase I and one Phase I/II study)

Corporate Overview


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• Market Cap: ~ $200 million • Cash and marketable securities: $41 million (March, 2015)

• No debt

• Net burn: ~ $23 million

• 165 employees (16 PhD, 4 MD)

• IP Ownership: over 40 granted patents and ~150 pending

applications

Financial Overview


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PLX Technology


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Selected for EU Adaptive Pathways –

potential for marketing approval in

2018


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Partner Indication Deal structure

Pulmonary Arterial Hypertension (PAH)

Worldwide license

Licensing - (upfront payment of $7M, additional $48M in milestones, cells supply (cost +) and royalties in gross margin

IC, CLI South Korea only

JV following marketing authorization of the Korean authorities

Acute Radiation Syndrome U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18

Pluristem keeps IP and manufacturing rights in all collaborations

Collaborations


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Clinical Programs 8

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PLX-PAD


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• Two completed Phase I studies in critical limb ischemia – positive results for both the U.S. and German trials

• One ongoing Phase II study in intermittent claudication (N=150)

• Planned studies in CLI:

o Phase II/III in Europe via Adaptive Pathways pilot project Pluristem selected to Adaptive Pathways pilot project

o Phase I/II in Japan via Accelerated Pathway for Regenerative

Medicine (application submitted; cell quality and production methods already recognized by PMDA)

Completed, Ongoing and Planned Studies with PLX-PAD in Peripheral Artery Disease


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Conditional Approval vs. Traditional Pathway to Market in EU

Phase II RCT Conditional MA*

2016 2017 2018 2019 2020 2021

Collection of real-world Data from patients treated with PLX-PAD

Full MA*

Phase II RCT Two Phase III RCTs Full MA*

Conditional Aprroval

Conservative Pathway

MA = marketing authorization


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Event Rate USA data (AFS- 100%) Germany data (AFS- 73%) Average (85%) Published historical data

Summary Data from Both Phase I Studies, N=27

12 month - 85% Amputation-Free Survival (AFS)


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PLX-PAD CLI program status in Europe

• 3-4 years earlier in a multi-billion market

• Manufacturing facility approved and inspected by European

Qualified Person for Phase III and marketing

• Granted European patent for the use of PLX in ischemic

disease

• Increased probability of a large pharma deal

• EMA new approach to expand the use of PLX-PAD to

additional indications within PAD and other ischemic

conditions


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PLX PAD- Orthopedic Indication Phase II Completed


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Strong Clinical Data Muscle Injury following Total Hip Replacement N=20


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Improvement of 500%

P=0.0067

Strong Data on Efficacy in Muscle injury Following Total Hip Replacement


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Correlation Between Improvement in the Muscle Force of Injured and Contralateral Leg

Contralateral (non–operated)

-20

-15

-10

-5

0

5

10

15

20

25

30

chan

ge in

stre

nght

of c

ontr

alat

eral

leg

Placebo150M PLX-PAD300M PLX-PAD

6 12 26

150M vs. Placebo p=0.0114

Time (Week

-15-10-505

10152025303540

cha

nge

in s

tren

ght

of o

pera

ted

leg

Time (Weeks)

Placebo150M PLX-PAD300M PLX-PAD

6 12 26

150M vs. Placebo : p=0.0067

Injured (operated)


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Ongoing Clinical Trials – PLX-PAD

• Intermittent Claudication (IC): Phase II multinational, randomized, double blind, placebo controlled study. Primary End Point: Maximal Walking Distance (MWD) at 12 months compared to baseline, N=150 (as of December 31, 2014, 64 patients enrolled)

• Pulmonary Arterial hypertension (PAH): - Phase I, open

label, dose escalation study. Primary End Point: safety & efficacy at weeks 6 and 12, and at 12 months, N=9 (as of December 31, 2014, 3 patients enrolled)


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PLX R18 Hematologic Indications


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PLX-R18 Target Indications Bone marrow failure including: • Incomplete engraftment after bone marrow transplantation • Acute Radiation Syndrome (ARS) – supported by the U.S. National Institutes of

Health (NIH) • Two significant animal trials announced

o Animal Rule pathway • Aplastic Anemia (AA) – FDA Orphan Drug designation • Chemotherapy induced bone marrow failure - Pre-Clinical PoC

• Bone Marrow Transplant failure (compassionate use – human data)

Support for Hematopoietic cell Transplantation (HCT): • Umbilical cord blood transplantation – Pre-Clinical PoC • Bone Marrow Transplantation – Pre-Clinical PoC


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U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18

US Government Collaboration for ARS


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• “Exploratory” Phase I/II trial in hematological indication with

PLX-R18 under new expedited approval pathway for regenerative therapy in Japan o Positive results potentially sufficient for limited marketing

authorization

• Phase I trial in hematological indication with PLX-R18 in U.S.

• Later-phase animal trials by U.S. National Institutes of Health for Acute Radiation Syndrome via Animal Rule pathway o NO human efficacy trials needed for approval o If approved, government stockpiling for ARS to be

integrated with storage for civilian use in other indications

Planned Clinical Trials


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The Process is the Product


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Allogeneic MSC “the process is the product”

Paracrine/endocrine effects leads to emphasis on the secretion profile

Proprietary 3D platform to enable controlled 3D culturing to ensure “batch-to-batch” comparability and to modify the secretion profile as needed- PLX-PAD, PLX-R18 and others.

Monolayer Vs. Multilayer • 3D Cell-Cell interaction

• PLX cells harvested following extracellular collagen-cells interaction

• High yield

Pro-angiogenic

Undetermined

Anti-angiogenic


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3D manufacturing = 150,000 doses annually


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PLX-PAD CMC Approved by 5 Regulatory Agencies

FDA, PEI (Germany), EMA, South Korean & Israeli Regulatory Agencies: • Confirmed Batch-to-Batch Comparability, Using Multiple Donors • CMC Certification for 3D culturing for Phase II, III and Marketing


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IC

CLI (Adaptive EU)

CLI (Japan)

Orthopedic indication

Hematology

ARS

PAH

Preeclampsia

End of enrollmen

t

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Company milestones H2-2014

H1-2015

H2-2015

H1-2016

H2-2016

H1-2017

H2-2017

H1-2018

H2-2018

indication End of

enrollment

Data Readout

Licensing deal

Study initiatio

n

Adaptive

Pathway

approval

Initial approva

l

Data Reado

ut

End of enrollmen

t

Data Readou

t

Study initiatio

n

Conditional approval

We will seek to include the indication under the Adaptive licensing pathway- based on discussion with EMA

IND Study initiation

End of enrollment

Data Readout

Adaptive or phase

II/III

Pivotal study

Data Reado

ut

BLA application

UT will pursue IND upon completion of the phase I

We will seek to include preeclampsia under the Adaptive licensing pathway- based on discussion with EMA


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Zami Aberman Chairman & CEO

Efrat Livne-Hadass VP Human Resources

Racheli Ofir, Ph.D. VP Research & Intellectual Property

Sagi Moran VP Operations

Erez Egozi VP Finance Karine Kleinhaus, M.D., MPH Divisional VP, North America

Yaky Yanay President & COO

Hillit Mannor Shachar, M.D., M.B.A. VP Business Development

Ohad Karnieli, Ph.D., M.B.A. VP Technology & Manufacturing

Esther Lukasiewicz Hagai, M.D., Ph.D. VP Clinical & Medical Affairs

Orly Amiran VP Quality Assurance

Management Team


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Date post:	19-Jul-2020
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Advancing cell therapeutic products for clinical use v2.… · Advancing cell therapeutic products...

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