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Company Presentation September 2019

Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

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Page 1: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Company PresentationSeptember 2019

Page 2: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

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

Forward looking Statement

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Page 3: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

• Cell therapy company focused on Regenerative Medicine

• Off-the-shelf placenta-derived cell product candidates

• Two ongoing Phase III studies

• Publicly listed in Nasdaq and TASE

• Favorable safety profile and efficacy data from hundreds

of patients treated worldwide

• Cash- $25 million (as of June 30, 2019)

• Best-in-class cell manufacturing technology producing

highest quality cell products at a commercial scale

• Strong IP portfolio (over 120 granted patents)

• Full time employees: 160

Pluristem Therapeutics

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Page 4: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

PLURISTEM in one slide

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Placenta

Technology

Allogeneic off-the-shelf

Simple IM administration

Adaptive slow release secretion of cytokines

Long term regenerative effect

Page 5: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Complexity of the diseaseInnovative treatments are needed to treat complex diseases

The Economic ImpactSome of the world’s largest economies are nowfacing subsequent increases in health-care costs.

The Human ImpactAging is often associated with debilitating medicalconditions, many of which are still unmet needs.

Longer lifespansLifespan has increased significantly, Nearly 2 billion people across the world are expected to be over 60 years old by 2050 (World Health Organization)

The Need for Cell Therapy

Page 6: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

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Placenta-Derived Cells

• Ethically accepted

• Rich & diverse

• Pro-angiogenic & Immunoregulatory

• Young donors

• Unlimited source & easy to collect

• Ability to manufacture treatments for over 20,000 patients per placenta

The Placenta Project was

Launched by the US National

Institutes of Health (NIH) in 2013

to further explore the role of the

placenta in health and disease

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

Page 7: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

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Best In Class GMP FacilityCommercial Scale Highest Quality Cell Products

Manufacturing Process Approved by:7

Most advanced cell manufacturing technology. Tightlycontrolled, completely automated, efficient andscalable

State-of-the-art, proprietary bioreactor system whichprovides a 3D micro-environment for cells that mimicsthe human body condition

Ability to manufacture treatments for over 20,000patients per placenta

Controlling the conditions within our bioreactors allowsus to produce several unique patented products

Page 8: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

IndicationProduct

CandidateLocation Pre-Clinical Phase I Phase II Phase III Funding

Clinical Pipeline

FDA Animal Rule

Critical Limb Ischemia

Intermittent Claudication

Muscle Regeneration following Hip Fracture

Acute Radiation Syndrome*

PLX-PAD

PLX-PAD

PLX-PAD

PLX-R18

Graft Versus Host Disease PLX-PAD

incomplete recovery following bone marrow transplantation PLX-R18

U.S., Europe Israel

U.S., Europe South Korea,

Israel

U.S., Europe Israel

U.S.

Israel

U.S., Israel

* FDA Orphan Drug Designation

*

Page 9: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

PLX-PAD

Peripheral Arterial Disease

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Page 10: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Critical limb ischemia (CLI)Severe pain at rest, skin wounds, tissue necrosis and poor quality of life

High risk of leg amputation and deathApproximately 40% of patients are unsuitable for revascularization

Peripheral Arterial DiseasePeripheral Arterial Disease (PAD) is caused by fatty deposits in leg arteries that obstruct blood flow.

Risk factors include smoking, diabetes, obesity, cardiovascular problems and hypertension.

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5 Year Mortality Rate

Page 11: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Clinical Development in Peripheral Arterial Disease

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Completed two Phase I studies in CLI- U.S. & Germany (N=27)

Good safety profile

Increase in tissue perfusion, 60% reduction in the risk for death or amputation

Dose identification- two treatments of 300 million cells, two months apart

Completed Phase II study in intermittent claudication (IC)- U.S., Germany, S. Korea & Israel (N=172)

Good safety profile

Significant increase in walking distance , reduction in surgical events and HbA1c and CRP levels

Confirmation of Phase III design including- dose (300m cells), dose regimen (2 administrations)

Ongoing Phase III study in CLI- U.S., Europe & Israel (N=246)

Completed enrollment of half of the study’s population (n=123) in April 2019. An interim analysis following 12 months follow-up period under the EU Adaptive Pathways Program, targeting conditional marketing approval in Europe

Page 12: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Ongoing CLI Phase III Study - Overview

Design Phase III, randomized, Double-Blind, Placebo-controlled (2:1)

Study population CLI subjects with minor tissue loss, unsuitable for revascularization

Countries Germany, UK, U.S., Poland, Hungary, Czech republic, Bulgaria, Macedonia, Israel

Sample size 246 patients

Doses tested 300M cells vs. Placebo (randomization ratio 2:1)

Administration IM injections in the affected leg, 2 treatments at 8-week interval

Primary efficacy endpoint Time to occurrence of major amputation of leg or death (AFS)

Main Secondary & exploratory efficacy endpoints

Composite efficacy endpoint; Pain; Complete wound healing; Quality-of-life; Adjudicated amputations; TcPO2; cytokine levels

Follow Up length 52 Weeks

Expected Data H1 2020 (Europe), H1 2021 (U.S.)

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€7.6 million grant from the EU Horizon 2020 program

Page 13: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Disclaimer: The results presented above are a small sample of the ongoing trial, chosen by the principal investigator, and are not representative of the full trial population. These results may not be typical and could be materially different from the results reported at the completion of the trial. Investors are cautioned to consider this sample data at their own risk.

Before 8 Weeks After

Area:

4.3 cm²

Area: 0.2

cm²

Reduction

of 96%

Area:

9.8 cm²

Area: 0.5

cm²

Reduction

of 95%

Area:

6.8 cm²

Area: 3.1

cm²

Reduction

of 54%

• CLI Expanded Access Program cleared

by FDA to enroll patients unsuitable for

inclusion in the ongoing Phase 3

clinical trial

• Program to enroll an initial 100 CLI

Rutherford Category 5 patients

• FDA approved cost recovery for the

treatment

CLI Expanded Access Program (EAP)

Ongoing CLI Phase III Study

Page 14: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

PLX-PAD

Muscle Regeneration

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Page 15: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Hip Fracture- Overview

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• Hip fracture often results in serious long-term complications,including pain, functional decline and disability

• Up to 30% of hip fracture patients die within 1 year due toimmobility associated diseases

• Annual treatment costs in the U.S. are between $10 to $15billion, and are expected to rise due to aging population

• Attributable costs for hip fracture are $80,000, demonstratingthe cost burden of long-term care after fracture

There are currently no approved treatments for the post-operative regeneration of injured or weak skeletal muscle

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

Page 16: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Contralateral(non–operated)

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Muscle Regeneration

Phase I/II Study of PLX-PAD for Muscle Injury Following Total Hip Replacement (N=20)

Change in Volume Improvement of 300%

P=0.004

Change in Strength Improvement of 500%

P=0.0067

Change in Strength Improvement of 4000%

P=0.012

• PLX-PAD demonstrated a significant increase in muscle strength & volume compared to placebo

• First study to show efficacy of cell therapy in skeletal muscle injury

Page 17: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Ongoing Muscle Regeneration Phase III Study

Design Phase III, randomized, Double-Blind, Placebo-controlled

Study population Patients suffering from muscle injury following arthroplasty for hip fracture

Countries U.S., Germany, UK, Denmark, Israel

Sample size 240 patients

Doses tested 150M cells vs. Placebo (randomization ratio 1:1)

Administration IM injections in the operated leg on the day of surgery

Primary efficacy endpoint Short Physical Performance Battery (SPPB) score at week 26

Main Secondary & exploratory efficacy endpoints

Muscle strength, muscle mass & volume, hospitalization time, lower extremity measure

Follow Up length 26 (efficacy), 52 weeks (safety)

Expected Data H2 2020

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€ 7.4 million grant from the EU Horizon 2020 program

Page 18: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

PLX-PAD

Hematological programs

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Page 19: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

• Acute Radiation Syndrome (ARS)

- Studies conducted and funded by U.S. government (NIH, DOD)

- FDA has cleared Pluristem’s Investigational New Drug (IND) application for PLX-R18 in the treatment of ARS in case of nuclear events

- FDA Orphan Drug Designation

• Phase I - Incomplete Hematopoietic Recovery Following Hematopoietic Cell Transplantation (HCT)

− N= 24

− Open-label trial allows for interim data analysis

− Clinical sites in U.S and Israel

− FDA Orphan Drug Designation

Hematological Programs

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Page 20: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Acute Radiation Syndrome (ARS) – the need

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

• Screening- PLX-R18 release a combination of therapeutic proteins in response to the patient’sneeds and doesn’t require pre-screening

• Treatment in mass casualty event- PLX-R18 can be quickly administered by simpleintramuscular injections and does not require genetic or tissue matching, suitable to treat largenumber of victims exposed

• Comprehensive therapy- currently approved MCMs are all myeloid colony stimulating factors(increase white blood cells only), and may lead to severe side effects. PLX-R18 stimulates theregeneration of all three blood lineages

Page 21: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Acute Radiation Syndrome (via the FDA Animal Rule)

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Page 22: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Pluristem keeps IP and manufacturing rights in all collaborations

Partnerships and Collaborations

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Page 23: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

Zami AbermanExecutive Chairman

Efrat Livne-Hadass VP Human Resources

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

Chen Franco-YehudaCFO

Yaky YanayPresident & CEO

Management Team

Efrat KaduriDirector of Business, Investor & Public Relations

Lior RavivVP Operations & Development

Orly AmiranVP Quality Assurance

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Dan PeresVP Medical & Clinical Affairs

Page 24: Company Presentation - Pluristem Therapeutics · Good safety profile Increase in tissue perfusion, 60% reduction in the risk for death or amputation Dose identification- two treatments

[email protected] +972-74-710-8600

U.S. +1-347-973-2098www.Pluristem.com

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