AI Terminal

MODULE: AI_ANALYST
Interactive Q&A, Risk Assessment, Summarization
MODULE: DATA_EXTRACT
Excel Export, XBRL Parsing, Table Digitization
MODULE: PEER_COMP
Sector Benchmarking, Sentiment Analysis
SYSTEM ACCESS LOCKED
Authenticate / Register Log In

Pluri Inc.

Regulatory Filings Oct 29, 2017

6990_rns_2017-10-29_8f1b5ce7-97ff-4ca3-955d-81e6bb546c20.pdf

Regulatory Filings

Open in Viewer

Opens in native device viewer

COMPANY PRESENTATION October 2017

Forward looking Statement

This presentation contains express or implied forward-looking statements within the Private Securities Litigation Reform Act of 1995 and other U.S. Federal securities laws. For example, we are using forward-looking statements when we discuss the expected timing of obtaining regulatory approval for our various patient trials and clinical data readout, proposed trials that may occur in the future, the timing and implementation of our collaborations with various partners and the execution of definitive agreements relating to such collaborations and the potential benefits and impact our products could have on improving patient health care. These forward-looking statements and their implications are based on the current expectations of our management only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching and/or successfully completing our clinical trials; our products may not be approved by regulatory agencies, our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real clinical settings; results of preclinical studies may not correlate with the results of human clinical trials; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications; loss of market share and pressure on pricing resulting from competition, which could cause our actual results or performance to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, we undertake no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. For a more detailed description of the risks and uncertainties affecting us, reference is made to our reports filed from time to time with the Securities and Exchange Commission

CORPORATE OVERVIEW

  • Cell therapy company using off the shelf placentaderived cell products
  • Entering late-stage trials in 3 indications
  • Multifactorial therapy releasing a range of therapeutic proteins in response to signals from patient's body
  • First in class 3D cell culturing technology allowing for efficient, controlled production of different cell productsin commercial quantities

FINANCIAL GLANCE

Pluristem
Therapeutics Inc.
NASDAQ: PSTI
TASE:
PSTI
Stock Price
(As of 10/27/2017)
\$1.97
Market Capitalization ~\$192
million
Cash and Marketable
Securities (As of 6/30/2017)
\$26.7 million
Debt \$0
Employees 180
Intellectual Property
Ownership
115+ granted
~100 pending

5

The PLX Platform Technology

Best In Class GMP Facility

3D Manufacturing, In-house Cell Production Potential capability to manufacture up to 150,000 doses annually

* Chemistry, Manufacturing, and Controls

Placenta Derived Cells

  • Ethically accepted
  • Rich & Diverse
  • Highly potent Pro-angiogenic Immunoregulatory
  • Young donors
  • Unlimited source & Easy to collect
  • Over 25,000 Doses of 300 million cells per placenta The Placenta Project was

http://www.the-scientist.com/?articles.view/articleNo/43618/title/The-Prescient-Placenta/

Launched by the US National Institutes of Health (NIH) to further explore the role of the placenta in health and disease

From The Miracle of Birth to Therapeutics for All

Company Pipeline

Late-stage trials

* One Multinational trial- U.S- phase 3, Europe- via adaptive pathway potentially allowing early marketing approval

** Via PMDA's accelerated regulatory pathway for regenerative therapies

*** Pending FDA/EMA approval

12

A CHANGE IN REGULATORY ENVIRONMENT

Regulatory Status

FDA EMA PMDA
CLI (PLX-PAD)
Fast track approval
Single pivotal study
(n=246)


Adaptive regulatory
pathway
Single pivotal study (n=246)
Potential conditional
approval on interim report
(n=123)

Accelerated regulatory
pathway
Single pivotal study (n=75)
Hip fracture
(PLX-PAD)

Pivotal study
Subject to FDA
approval

Adaptive regulatory
pathway
Single pivotal study

  • ARS (PLX-R18) Animal rule pathway
    • Open communication, unlimited pre- IND

  • Reduces inflammation
  • Stimulates growth of collateral blood vessels
  • Stimulates repair of damaged muscle

Peripheral Arterial Diseases Orthopedic Injuries

PLX-PAD Mechanism of Action

Completed and Ongoing Clinical Studies with PLX-PAD

  • Two completed Phase I studies in Critical limb ischemia (CLI) in U.S. and Germany, N=27
    • Good safety profile
    • Trends of efficacy (pain reduction and increase in tissue perfusion)
  • Ongoing multinational Phase II study in Intermittent claudication (IC) in U.S., Germany, South Korea and Israel, N=172
    • Enrollment completed
    • Data readouts expected in H1 2018
  • Completed Phase II study in muscle injury following total hip replacement in Germany, N=20
    • Good safety profile
    • Strong efficacy (increase in muscle volume and muscle force)

Completed and Ongoing Clinical Studies with PLX-PAD

  • Ongoing multinational Phase III study In Critical Limb Ischemia (CLI) in U.S., Europe, N=246
    • Fast track designation from FDA
    • Adaptive regulatory pathway from EMA
    • Support from EU Horizon 2020 program
  • Planned multinational Phase III study In Hip Fracture in U.S., Europe
    • Adaptive regulatory pathway from EMA
    • Support from EU Horizon 2020 program
  • Planned Pivotal study in CLI in Japan, N=75
    • PMDA's accelerated regulatory pathway for regenerative therapies
    • Form joint venture

Pre-Treatment

8 Weeks post treatment

Company Pipeline

Late-stage trials

* One Multinational trial- U.S- phase 3, Europe- via adaptive pathway potentially allowing early marketing approval

** Via PMDA's accelerated regulatory pathway for regenerative therapies

*** Pending FDA/EMA approval

Peripheral Arterial Disease (PAD)

  • PAD is caused by fatty deposits in leg arteries that obstruct blood flow
  • Intermittent claudication is the early stage while critical limb ischemia (CLI) is the more advanced stage of PAD
  • CLI Patients suffer from severe pain at rest, skin wounds, tissue necrosis and poor quality of life with a high risk of leg amputation and death
  • 5-6 million people in U.S. and Europe suffer from CLI*
  • Estimated cost for treatment in the U.S. is over \$25 billion per year*
  • Up to 40% of patients are unsuitable for revascularization and experience up to a 40% amputation rate at 1 year**

*Source: Sage Group- (link, link, link)

**Source: European Society for Vascular Surgery (link)

CLI Phase III Study -U.S./ Europe (N=246)

  • Accelerated regulatory pathways in U.S. (Fast Track), Europe (Adaptive regulatory pathway) & Japan (accelerated regulatory pathway for regenerative therapies)
  • An interim analysis (N=123) of efficacy will be performed in support of an application to the EMA for Conditional Marketing Authorization (CMA)
  • Interim analysis could lead to CMA based on the success of either the primary or one of the key secondary endpoints, or a composite endpoint that includes death, major amputation, and certain measures of severity of wounds and gangrene
  • Primary endpoint is time to event (amputation or death); other measures of efficacy include AFS, quality of life, TcPO2, and pain score
  • Dosing regimen: two doses of 300 million cells, two months apart (n=144), placebo (n=72)
  • No HLA matching or immunosuppression required
  • Follow-up of 12-36 months increases the study's power allowing for a smaller trial

\$8 million grant from the EU Horizon 2020 program to support Phase III trial

Clinical Development of CLI in Japan

  • Accepted to the PMDA's accelerated regulatory pathway for regenerative therapies
  • A single 75 patient study may lead to early conditional marketing approval and reimbursement
  • Binding term sheet with Sosei CVC to establish joint venture for the clinical development and commercialization of PLX-PAD for CLI in Japan

Company Pipeline

Late-stage trials

* One Multinational trial- U.S- phase 3, Europe- via adaptive pathway potentially allowing early marketing approval

** Via PMDA's accelerated regulatory pathway for regenerative therapies

*** Pending FDA/EMA approval

23

Muscle Regeneration- clinical data

Muscle injury following total hip replacement (N=20)

Improvement of 500% P=0.0067

Improvement of 4000% P=0.012

Muscle Regeneration- clinical data

Muscle Injury following Total Hip Replacement (N=20)

Change in Volume from Day 0

Phase III Hip Fracture Study

  • Femoral neck fracture is the most common form of hip fracture
  • Annual treatment costs in the U.S. are estimated to be between \$10 to \$15 billion, and are expected to rise due to the aging population, with mortality rates of up to 36%*
  • Positive feedback from FDA and EMA on the proposed study design and endpoints of Phase III trial in treatment for muscle recovery following arthroplasty for hip fracture
  • PLX-PAD program in hip fracture might be eligible for Breakthrough Therapy designation and benefit from the 21st Century Cures Act as well as the EMA's Adaptive Pathways pilot project

\$8.7 million grant from the EU Horizon 2020 program to support this Phase III trial

* Source: Simran Mundi, Bharadwaj Pindiprolu, Nicole Simunovic, Mohit Bhandari

PLX-R18

Stimulates regeneration of damaged bone marrow to produce blood cells (white, red and platelets)

Acute Radiation Syndrome (ARS) Hematologic Indications

PLX-R18 Mechanism of Action

PLX-R18 Programs

Acute Radiation Syndrome (ARS)

In Preparations for pivotal study

Bone Marrow Failure

Following or in support of a transplant of hematopoietic stem cells (HCT) Ongoing Phase I study in U.S and Israel

Hematological Disorders

Autoimmune diseases, Genetic disorders, Chemotherapy, Radiation therapy, Side effects from treatments Covered by patent

Company Pipeline

Late-stage trials

* One Multinational trial- U.S- phase 3, Europe- via adaptive pathway potentially allowing early marketing approval

** Via PMDA's accelerated regulatory pathway for regenerative therapies

*** Pending FDA/EMA approval

Acute Radiation Syndrome ARS

ARS occurs following acute exposure to very high levels of radiation, and involves severe, potentially lethal injury to the bone marrow as well as to other organs and systems within the body

High doses of radiation can destroy the bone marrow's ability to produce white cells, red cells and platelets; without these cells patients are at high risk of death

Collaboration on ARS with U.S. Government

Collaboration on ARS with U.S. Government

Studies are conducted and funded by the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH) and U.S the department of defense

PLX-R18 Data- Phase I equivalent study (FDA animal rule)

PLX-R18 Data- Phase II equivalent study (N=62) (FDA animal rule)

PLX-R18- Treatment of ARS

  • Allogeneic, ready to use as an off the shelf product
  • Easy IM administration
  • Beneficial when administered even 48 hrs. following exposure to radiation
  • No need for prescreening no effect if injected to those who were not exposed to radiation
  • Supports recovery of all three blood lineages (red and white cells and platelets)
  • Long shelf life
  • Showed increased survival rates In irradiated non-human primates (NHPs)

PLX-R18 Hematological Program

  • Ongoing U.S. and Israeli Phase I clinical trial of R18 for the treatment of insufficient hematopoietic recovery following hematopoietic cell transplantation
    • − N= up to 30
    • − Open-label trial allows for interim data analysis
  • Collaboration with the to evaluate PLX-R18 as an adjuvant therapy to umbilical cord blood transplantation in animal studies
    • − Grant of \$900,000 from Israel-U.S. Binational Industrial Research and Development Foundation (BIRD)
  • Granted European patent to cover indications related to the bone marrow's inability to produce blood cells, such as autoimmune diseases, genetic disorders, chemotherapy, radiation therapy, and side effects from other treatments

Commercialization Strategy

  • 1. Out-licensing commercialization deals with partners
  • 2. Direct sales of indications with small patients population & high market price
  • 3. Direct sales of our PLX-R18 product for Acute Radiation Syndrome (governments)

Partner Indication Deal structure
Collaborations IC, CLI
South Korea only
Joint
Venture
following
marketing
authorization
by
the
South
Korean
authorities
Pluristem keeps IP
and manufacturing
rights in all
collaborations
Acute Radiation
Syndrome
U
S
National
Institutes
of
Health
(NIH)
to
examine
the
effectiveness
of
PLX
-
R18
as
a
treatment
for
ARS
following
24
hoursfrom
exposure
Acute Radiation
Syndrome
U
S
Department
of
Defense
to
examine
the
effectiveness
of
PLX
-
R18
prior
to,
and
within
the
first
24
hours
of
exposure
to
radiation
Acute Radiation
Syndrome
Pluristem
will
contribute
cells
and
scientific
knowledge,
FMU
will
conduct
the
studies
and
provide
the
required
resources
Acute Radiation
Syndrome
Conducting
trials
to
test
PLX
-
R18
cells
in
the
treatment
of
ARS
and
understanding
of
MOA
CLI,
Immunology,
Cardiovascular,
Orthopedic
Research
to
test
the
unique
immunology
of
the
placenta
and
cells
MOA
39 Umbilical Cord
Blood
Transplantation
Evaluating
PLX
-
R18
as
an
Adjuvant
Therapy
to
Umbilical
Cord
Blood
Transplantation

Investment

  • Late-stage pipeline with products advancing towards commercialization and 3rd parties funding
  • Advanced regulatory pathways that could shorten time to commercialization
  • Expected near-term data readouts
  • "Off the shelf" product, no HLA-matching required
  • Unique multifactorial MoA with a vast scientific background
  • Major technological competitive advantages
  • Strong collaborations and partnerships

Upcoming Milestones – 12 Months

Initiate pivotal trials

  • Critical limb ischemia (CLI) U.S., Europe (Japan yet to start)
  • Hip fracture U.S., Europe
  • ARS

Clinical data readout

  • Phase II Intermittent Claudication (IC)
  • Phase I incomplete engraftment of hematopoietic cell transplantation open label
  • Pivotal study in ARS

Business development

  • U.S. Advance discussions with U.S. government regarding stockpiling of PLX-R18 for ARS
  • Japan- Finalize joint venture
  • Asia Licensing/ joint venture with partner for Asian market

Management team

Zami Aberman Chairman & Co-CEO

Erez Egozi CFO

Sagi Moran VP Operations

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

Yaky Yanay President & Co-CEO

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

Orly Amiran VP Quality Assurance

Lior Raviv VP Development

Karine Kleinhaus, M.D., MPH Divisional VP, North America

[email protected] Israel +972-74-710-8600

U.S.- +1-914-512-4109

www.Pluristem.com

Talk to a Data Expert

Have a question? We'll get back to you promptly.