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Company PresentationSeptember 2019
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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• 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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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
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
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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/
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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
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
*
PLX-PAD
Peripheral Arterial Disease
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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
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
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
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
PLX-PAD
Muscle Regeneration
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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
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
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
PLX-PAD
Hematological programs
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• 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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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
Acute Radiation Syndrome (via the FDA Animal Rule)
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Pluristem keeps IP and manufacturing rights in all collaborations
Partnerships and Collaborations
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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
