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ENTROPY NEURODYNAMICS LIMITED — Investor Presentation 2021
Apr 15, 2021
64855_rns_2021-04-15_ebee75cb-9cd6-4666-8e05-27b36daabd11.pdf
Investor Presentation
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ASX ANNOUNCEMENT
Investor Presentation - Delivering Transformative Medicines
16 April 2021, Melbourne, Australia: Exopharm Limited (ASX:EX1), a clinical-stage company at the forefront of developing transformative exosome therapeutics, is pleased to provide the attached Investor Presentation titled “Delivering Transformative Medicines.”
By the Board - this announcement has been authorised for release by the Board.
Company and Media Enquiries:
Dr Ian Dixon, MBA Founder and Managing Director P: +61 (0)3 9111 0026 [email protected]
Rudi Michelson Monsoon Communications Tel: +61 (0)411 402 737 [email protected]
Join our mailing list to receive updates: http://exo.ph/ExoMails www.exopharm.com P: +61 (0)3 9111 0026
EXOPHARM LIMITED ACN: 163 765 991 ASX:EX1 Address: Level 17, 31 Queen Street Melbourne 3000 Telephone: 03 9111 0026 Email: [email protected] Web: www.exopharm.com
ABOUT EXOPHARM
Exopharm (ASX:EX1) is a clinical-stage biopharmaceutical company using exosomes to deliver a new class of transformative medicines and generate revenue from multiple partnership deals.
Exosomes are seen by the Biopharma industry as a highly differentiated platform with the potential to enhance tissue delivery for a variety of payloads like mRNA and proteins – part of the global market for drug delivery systems which is growing at a compound annual growth rate (CAGR) of 5% and valued at around US$170 billion in 2021.
For some medicines, exosomes are an alternative and superior means for delivery inside the body, alongside technologies such as lipid nanoparticles (LNP), cell penetrating peptides, viral vectors and liposomes.
Exopharm’s LEAP technology solves the challenge of purifying clinical-grade exosomes at large scale and low cost.
Exopharm also has two exclusive proprietary technologies that allow advanced customisation of exosomes – the LOAD technology improves loading of nucleic medicines into exosomes and the EVPS technology allows exosomes to be directed towards selected cell types.
Exopharm uses variations and combinations of LOAD and EVPS to enable its Biopharma partners to improve delivery of their drug candidates and help them design and test new exosome medicines aimed at treating a wide scope of medical problems including neurological disease, infectious disease, cancer, and fibrosis.
FORWARD LOOKING STATEMENTS
This announcement contains forward-looking statements which incorporate an element of uncertainty or risk, such as ‘intends’, ‘may’, ‘could’, ‘believes’, ‘estimates’, ‘targets’, ‘aims’, ‘plans’ or ‘expects’. These statements are based on an evaluation of current corporate estimates, economic and operating conditions, as well as assumptions regarding future events. These events are, as at the date of this announcement, expected to take place, but there cannot be any guarantee that such events will occur as anticipated or at all given that many of the events are outside of Exopharm’s control or subject to the success of the Development Program. Furthermore, the Company is subject to several risks as disclosed in the Prospectus dated 6 November 2018.
INHERENT RISKS OF INVESTMENT IN BIOTECHNOLOGY COMPANIES
There are a number of inherent risks associated with the development of biopharmaceutical products to a marketable stage. The lengthy clinical trial process is designed to assess the safety and efficacy of a drug prior to commercialisation and a significant proportion of drugs fail one or both of these criteria. Other risks include uncertainty of patent protection and proprietary rights, whether patent applications and issued patents will offer adequate protection to enable product development, the obtaining of necessary drug regulatory authority approvals and difficulties caused by the rapid advancements in technology. Companies such as Exopharm are dependent on the success of their research and development projects and on the ability to attract funding to support these activities. Investment in research and development projects cannot be assessed on the same fundamentals as trading and manufacturing enterprises. Therefore, investment in companies specialising in drug development must be regarded as highly speculative. Exopharm strongly recommends that professional investment advice be sought prior to such investments.
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Exopharm Ltd Delivering Transformative Medicines
April 2021
Updated Investor Presentation
2.5
IMPORTANT INFORMATION
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Purpose of presentation: This presentation (including this document, any related video or oral presentation, any question and answer session and any written or oral material discussed or distributed in relation to this presentation) has been prepared by Exopharm Limited (ACN 163 765 991) (Exopharm or Company). This presentation is intended for sophisticated or professional investors (as those terms are defined in the Corporations Act 2001 (Cth)), and their professional investment advisors and has been prepared for the sole purpose of providing general high-level information on Exopharm and its operations.
Not an offer or solicitation: This presentation is not investment advice nor an offer to subscribe for securities or otherwise invest in Exopharm, and it should not be relied upon to make any investment decision.
Nature of presentation: This presentation is not a prospectus, product disclosure statement or other investment disclosure document, and the level of disclosure in this presentation is less than such disclosure documents. This presentation does not purport to contain all of the information that a prospective investor may require to make an evaluation of Exopharm or its business activities and nothing in this presentation is, or is intended to be, a recommendation to invest in Exopharm. Exopharm does not purport to give financial or investment advice. No account has been taken of the objectives, financial situation or needs of any recipient of this presentation.
Forward-looking statements: This presentation may contain forward-looking statements which may be predictive in nature and incorporate an element of uncertainty or risk, such as ‘intends’, ‘may’, ‘could’, ‘believes’, ‘estimates’, ‘targets’ or ‘expects’. These statements are based on an evaluation of current economic and operating conditions, as well as assumptions regarding future events. These events are, as at the date of this presentation, expected to take place, but there cannot be any guarantee that such will occur as anticipated, or at all, given that many of the events are outside Exopharm’s control. The stated events may differ materially from results ultimately achieved. Accordingly, neither Exopharm nor any of its directors, employees, contractors or advisors make any warranty or assurance that the results, performance or achievements expressed or implied by the forward-looking statements contained in this presentation will actually occur. Further, other than as required by law, Exopharm may not update or revise any forward-looking statement if events subsequently occur or information subsequently becomes available that affects the original forward-looking statement.
Disclaimer: Neither Exopharm nor its officers, employees, contractors or advisors give any warranty or make any representation (express or implied) as to the accuracy, reliability, relevance or completeness of the material contained in this presentation. Nothing contained in this presentation is, or may be relied upon as a promise, representation or warranty, whether as to the past or the future. Except for statutory liability which cannot be excluded, Exopharm, its officers, employees, contractors and advisors expressly disclaim any responsibility for the accuracy or completeness of the material contained in this presentation and exclude all liability whatsoever (including in negligence) for any loss or damage which may be suffered by any person as a consequence of any information in this presentation or any error or omission therefrom.
Professional advice: Recipients of this presentation should consider seeking appropriate professional financial, taxation and legal advice in reviewing the presentation and all other information with respect to Exopharm and evaluating its business, financial performance and operations.
Confidentiality and copyright: While this is a non-confidential presentation, it still provides confidential and commercially sensitive information which is provided for the intended recipient only. Organisations or Persons viewing this presentation must not disclose the presentation or its contents to any third parties other than external consultants of the recipient for the purposes of obtaining a professional review, financial, taxation or legal advice, or as required by law or court order. Exopharm holds the copyright in this paper. Except as permitted under the Copyright Act 1968 (Cth), this paper or any part thereof may not be reproduced without Exopharm’s written permission.
© Exopharm Ltd 2021
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Exopharm Ltd
Overview
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Australian clinical-stage company at the forefront of developing transformative medicines based upon exosomes (extracellular vesicles, EVs)
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40 staff based in Melbourne, Australia; 1 based in Europe
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Publicly traded on the ASX (ASX:EX1) (listed Dec 2018) 139.4 million shares on issue, current market cap. ~AU$100 million
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A platform technology company with application to many exosome medicines – using our exclusive LEAP, LOAD and EVPS technologies
Priorities
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Making exosome medicines available to established biopharmaceutical companies to enable successful delivery of existing drug candidates
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Generating revenue in the near-term through multiple deals
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Building a valuable leadership position in the emerging exosome medicines field through our exosome technologies and exosome medicines
Exosomes: Nature’s Solution to Delivery in the Body
Exosomes (also referred to as extracellular vesicles or EVs) are natural, multifunctional and stable nanoparticles that transfer cargo and messages between cells.
1. PACKAGE LIPID BILAYER
Natural exosomes can be produced from cells in a bioprocessing facility.
2. ADDRESS PROTEINS
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Outer membrane that forms the
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1. PACKAGE exosome (same membrane as human cells)
3. CARGO
- RNA, PROTEINS, & LIPIDS
External proteins that improve 2. ADDRESS targeting of exosomes to specific cell types
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The materials delivered by exosomes, 3. CARGO including instructions (RNA) and building materials (lipids, enzymes, proteins)
© Exopharm Ltd 2021
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Uncovered by Scientists in 2007, in 2021 Exosomes are Emerging as a New Frontier in Medicines Delivery
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Exosomes represent a dynamically growing segment in life sciences with opportunities in research, diagnostics and therapeutic applications.
Dr Uwe Gottschalk, Chief Technology Officer for Lonza (2017)
Targeted and nontargeted exosomes offer a highly differentiated platform with the potential to enhance tissue delivery for a variety of payloads like mRNA and proteins.
Dr Madhu Natarajan, Head of the Rare Diseases Drug Discovery Unit at Takeda (2020)
Now is the time for researchers to usher in a new era of therapeutic possibilities using RNA-delivering, natural exosome vesicles.
Professor Phillip Askenase MD, Yale University School of Medicine (2020)
Exopharm is at the forefront of exosome medicines
© Exopharm Ltd 2021
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Many Modern Drugs Need Packaging and Addressing to Solve the ‘ Delivery Problem’
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| Drug type | Common delivery problems |
|---|---|
| Small molecules | • Inability to cross the blood-brain barrier • Metabolism (degradation) in the blood • Poor delivery to inside the cell • Not targeted |
| Nucleic acids (e.g. mRNA, miRNAs) |
• Degradation in the blood • Stimulation of immune response • Poor delivery efficiency |
| Gene therapy | • Inability to reach nucleus of the cell • Degradation in the blood |
The global market for drug delivery systems is projected to reach USD 175 billion by 2021
https://formulations.pharmaceuti calconferences.com
Pharma companies have medicines that need better delivery to become transformative
There is a USD175 billion p.a. industry around drug delivery and formulation
© Exopharm Ltd 2021
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SARS-CoV-2 RNA Vaccines Use “Artificial Exosomes”
Pfizer and Moderna SARS-2 vaccines package RNA into artificial synthetic lipid nanoparticles (LNPs) However, LNPs have major limitations for drug delivery: • Toxicity
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Anti-LNP immune response
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• Inefficient cellular delivery
Naturally occurring exosomes from human cells have evolved an optimal composition over billions of years and have none of these limitations .
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Solid lipid nanoparticle
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LNP
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DOI: 10.3389/fmolb.2020.587997
Exosomes Overcome Significant Issues Associated with Other Drug Delivery Technologies
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| Delivery technology |
Efficient delivery |
Non toxic | Tropism | High stability |
Multiple dosings |
|---|---|---|---|---|---|
| Lipid nanoparticles (LNPs) |
|||||
| Liposomes | |||||
| Cell-penetrating peptides |
|||||
| Viral vectors | |||||
| Engineered exosomes with EVPS and LOAD |
© Exopharm Ltd 2021
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Exosome Medicines Can Have Broad Application to Solve Many Medical Problems
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Orphan indications/ genetic conditions (e.g. cystic fibrosis)
Infectious disease (viral, bacterial, parasite) Cancers/oncology
Psychiatric & pain
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CNS/neurological
Disability (hearing, taste, mobility)
Cardiovascular
Immune conditions (e.g. GvHD, TID, transplant rejection, IBD)
© Exopharm Ltd 2021
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Exopharm Has the Only Known Technology for Commercial-scale Exosome Medicine Purification
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State of the Art, Exosome Purification as of June 2020*
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Unlike all alternatives, LEAP
AF4 technology:
Research Scale
Nano-FCM
UC
Immunoaffinity 1. Uses industry-standard
Diagnostic Scale
Commercial reagents equipment/processes
Microfluidics Phase I Scale 2. Uses low-cost, reusable
consumables
Density gradient
Phase II/III Scale 3. Scales economically beyond
thousands of doses
SEC
4. Is proprietary (i.e. patent
Commercial
Filtration applied for)
Manufacturing
Scale
LEAP unlocks the potential
Low of Exosome Medicines
SCALABILITY High
High
COST
Low
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** LEAP assessment from Exopharm, based on industrial use to date; LEAP Patents processing through National phases at present.
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Adapted from https://doi.org/10.1016/j.tibtech.2020.05.012
© Exopharm Ltd 2021
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Three Unique & Powerful Technologies Underpin our Partnering and Exosome Medicines
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Exclusivity
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Scalable, economical GMP process for purifying exosomes
Activity enhancing method for delivering RNA by exosomes
Tropism-conferring approach for engineering exosome surface proteins
100% Exopharm
100% Exopharm
100% Exopharm
EVPS US patent is granted, LOAD patent is progressing and LEAP patent is progressing through national phase in USA under fast-track process.
© Exopharm Ltd 2021
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Exopharm is Proud to Be One of Few Exosome Medicine (EM) Companies Worldwide
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Leading EM
EM IP Status Pharma Partner Date Area Upfront Milestones
Company
Jun
CNS > $50 million $1.7 billion
2020
Mar Rare
$63 million $1.3 billion
2020 diseases
Jan
Cancer $77 million $1.8 billion
2019
Jun
CNS > $100 million Not disclosed
2020
Discussions are underway with
multiple potential partners
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Other emerging exosome medicine companies include PureTech, ARUNA, Carmine, Mantra, Ciloa, Anjarium, Ilias
© Exopharm Ltd 2021
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Exopharm is Building Financial Value via Two – Main Streams concurrent business opportunities
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Outlicensing LEAP technology to others for upfront fees, milestone fees and royalties*
Developing exosome medicines utilising Exopharm’s three technologies (LEAP, LOAD & EVPS)
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Early revenue from tech transfer & licensing for research & clinical trial exosome medicines
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License to blood plasma, cell product and bioprocessing companies, CMOs, blood centres, etc
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Early revenue from partnership deals incl. upfront & milestone payments – _Pharma company nominates the drug to deliver and invests in the development themselves*_
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Development of our own Exosome Medicines – we own and invest in the development of the exosome medicine
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- Example possible potential partnerships – inclusion of company name and brand does not indicate an actual commercial or legal relationship with Exopharm at this time. The brand remains the property of the company.
© Exopharm Ltd 2021
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Right Place, Right Time, Right People
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© Exopharm Ltd 2021
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Thank you
Exopharm Ltd (ASX:EX1)
Chris Baldwin Deputy CEO/Chief Commercial Officer +61 450 290 280 [email protected] www.exopharm.com