STEM CELL REGENERATIVE MEDICINE - COMPANIES AND IN AUSTRALASIA Snapshot of Stem Cell and Regenerative Medicine Companies in Australasia - AusBiotech
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STEM CELL
AND
REGENERATIVE
MEDICINE
COMPANIES
IN AUSTRALASIA
Snapshot of Stem Cell and 6th Edition
Regenerative Medicine 2020
Companies in Australasia
INDEX
Foreword .........................4
Transforming Research into
Real Solutions - AusBiotech .........................6
Companies
Australian-based Stem Cell
and Regenerative Medicine
Companies
Cynata .........................9
Mesoblast .........................10
Orthocell .........................11
Regeneus .........................12
Clinical Trials .........................14
Directory of Stem Cell .........................18
and Regenerative .
Medicine Companies
2
ABOUT THIS SNAPSHOT
This is the sixth version of the snapshot, a list of companies was formulated, largely
compiled from information that was publicly available. A questionnaire was sent out
to relevant company representatives, and the information was compiled into the
Snapshot. The information contained within this Snapshot is up to date and relevant
as of 20th October 2020. The publication was launched at AusBiotech + Invest 2020
(28th October— 30th October 2020). The sixth edition of the Snapshot highlights key
regenerative medicine companies and contains details of current clinical trials run by
cell therapy companies in Australiasia (page 14).
Disclaimer:
The information contained in the Snapshot of Stem Cell and Regenerative Medicine Companies in Australia: October 2020 is
intended solely for general information. This publication must not be relied upon as a substitute for medical, investment or
other professional advice. You are encouraged to speak with the relevant contacts provided in the Snapshot for further
information. This Snapshot should not be read as reflecting the policies of the NSW Stem Cell Network, its Executive, officers or
members. Nor does the Snapshot represent an endorsement of the processes, procedures and technologies described therein.
The NSW Stem Cell Network does not accept any liability arising in any way from information contained in this publication,
including by reason of negligence for errors or omissions in the information. Only companies that have agreed to be published
in the Snapshot were included and, any liability resides with them.
3
FOREWORD
2020 has been a most unusual year. COVID-19 has meant that there have
been significant physical restrictions as attempts are made to prevent the
spread of this contagious virus. On the other hand, it has placed regenerative
medicine (RM) in a unique position with the possibility of using stem and
amniotic epithelial cells to treat those who are infected and in intensive care
with acute respiratory distress. Three Australian entities, Mesoblast, Cynata
and the Hudson Institute of Medical Research, have ethics approval to trial
their cell products for this purpose. Other organisations around the world also
have approval to test their cell products to try and save the lives of those
afflicted with the virus.
Within Australia, there are over 200 clinical trials currently registered involving
stem cell therapies, and many innovative projects that are in the research
phase. Often in these therapies, stem cells are used to directly replace
damaged cells or tissue, or to recruit surrounding cells to repair tissue or
organs which have regenerative potential. Blood disorders are the lead
disease treated by stem cell transplantation with more than 300 unrelated
haemopoietic donor transplants in Australasia annually. Corneal transplants
using limbal stem cells are growing in popularity and graft versus host disease
resistant to conventional drugs is now being increasingly treated with
mesenchymal stem cells. Slightly peripheral to this, but growing at a rapid rate
are CAR-T cell therapies, especially for treatment of haemopoietic tumours.
Internationally, especially In the commercial space there are a growing
number of stem cell and regenerative medicine products being tested in
clinical trials, for example, for treatment of diabetes, osteoarthritis, multiple
sclerosis, stroke, macular degeneration, spinal cord lesions and certain types
of cancer. Details of current clinical trials of stem cell and regenerative
medicine therapies by Australian companies can be found on pages 14- 16.
.Japan, United Kingdom, United States, Canada and China have all invested
significantly into stem cell and RM research and development. The growing
number of clinical trials registered in Australia and worldwide reflect ongoing
efforts to expand the use of cell therapies to a broader range of conditions.
The Australian Government has added its weight to this effort, with a $150
million 10-year Australian Stem Cell Therapies Mission, funded by the Medical
Research Future Fund.
4
Some of these funds were distributed this year to Australian researchers for
treatment of Parkinson’s disease, age related macular degeneration, leukaemia,
epilepsy and fatty liver disease. And to nurture companies wishing to progress
medical technologies from Proof of Concept to the stage where venture capital
might provide developmental funds, there are the Biomedical Translation Bridge
Program and BioMed Tech Horizons Program funded by the Department of
Industry, Science, Energy and Resources. This year, also, for the first time, the
Centre for Commercialisation of Regenerative Medicine (CCRM) Australia
introduced the National Regenerative Medicine SuperPitch, to connect startups
and others with early stage projects to venture capitalists.
The NSW Stem Cell Network has nurtured the stem cell and regenerative
medicine communities for the past 18 years to grow and advance research and
development in these areas. The Network has over 600 members around
Australia including clinicians, researchers, policy developers, members of
Government, lawyers, ethicists, students, patients and companies, who are all
passionate advocates working together for the growth of the stem cell industry.
With this Report, our intention is to elevate commercialisation of stem cell and
regenerative medicine companies in Australia. In this Snapshot we provide details
current regenerative medicine and stem cell companies and manufacturers some
of whom are licensed by the TGA, and are carrying out controlled clinical trials
registered with an appropriate entity.
We highlight some of Australia’s leaders in stem cell and regenerative medicine
with company information and pipeline projects on pages 9 - 12.
Professor Bernie Tuch Tamara Treleaven
NSW Stem Cell Network NSW Stem Cell Network
Director Manager
Professor Tuch is an endocrinologist, non-executive Director of the biotechnology company
Living Cell Technologies Ltd, and a Director of Sydney Cell Therapy Foundation Pty Ltd. Tamara
Treleaven is a Clinical Embryologist, Ph.D candidate and the Manager of the NSW Stem Cell
Network.
5
TRANSFORMING RESEARCH INTO
REAL SOLUTIONS
Regenerative medicine has the potential to revolutionise the treatment of injuries
and disease, and Australia is primed to secure a more prominent share of the global
RM market, which is estimated to be worth AU$120 billion by 2035[1]. Australia is
investing in creating a world-class RM ecosystem with internationally-recognised
basic and translational research, a clinical trials framework and clinical centres. This
growing ecosystem has seen the number of cellular therapy or RM companies in
Australia double over the last two years.
Australia has been a front-runner due to its research output in the RM field, and has
seen commercial success in the area with companies such as PolyNovo and
Regeneus achieving international success this past year.
The RM industry is further bolstered by over 30 up-and-coming, small to medium
enterprises (SMEs) as profiled in this document. Together they represent a
burgeoning industry in the Australian biotechnology space with a market
capitalisation of over $3 billion that can further be bolstered by the
commercialisation of the newer technologies emerging from research.
Growing role of RM and need for regulatory pathways to expedite products
to market.
The RM landscape in Australia has changed since the first CAR-T therapy was
approved by the Australian regulator at the end of 2018. The number of
organisations developing new therapies in Australia has increased and with a
growing pipeline of therapies that multinationals may bring to Australia, the focus
on regulatory and reimbursement conditions is expected to increase in the next five
years.
RM therapies are fundamentally different from more common medicinal products
as they generally have longer than average development times, more stringent
manufacturing requirements, and a limited shelf-life for products. Treatments and
technologies within this area can also be disparately different from one another. For
example, a CAR-T therapy is vastly different from a cell scaffold. This impacts many
areas of the development pathway, including regulatory approvals and
classifications. AusBiotech is working actively with Therapeutic Goods
administration, the Australian regulator for therapeutic products, to enhance
knowledge of cell and gene therapies and expedite pathways for approval.
Research and Development (R&D) incentives in Australia.
A cornerstone for innovation in Australia is the R&D Tax Incentive; this Incentive
actively supports innovative local companies with their R&D and attracts companies
from around the world to bring their R&D to Australia. Start-up innovation
companies, especially biotechnology companies that trade at a loss, are the biggest
2020 beneficiaries from the Tax Incentive’s 43.5% refundable component.
The legislation also benefits large innovative companies by reducing the cost of
conducting eligible R&D activities in Australia, making Australia a more competitive
location for conducting biomedical and pharmaceutical R&D and clinical trials.
6
Other incentives and programes available in Australia include:
Medical Research Futures Fund (MRFF) focusses on ongoing health and medical research in Australia
which, in July 2020, was valued at $20 billion.From the year 2022, fund dividends (approx. $650 million
a year), will focus on translatable discoveries. Since establishment in 2015, the MRFF has funded over
450 projects worth more than $1.1 billion, including the following:
Biomedical Translation Fund (BTF) available to selected early-stage companies developing
promising biomedical products or services.
Valued at $500 million; through co-investment by the Australian government via the MRFF ($250
million) and private sector capital ($250 million).
Biomedical Translation Bridge (BTB) and BioMed Tech Horizons (BMTH) programmes are MRFF
initiatives supporting development of new technologies to the proof-of-concept stage and
through to commercialisation. They are administered by the industry growth centre for the
medtech and pharma sector, MTPConnect. Recent projects approved under these programmes
help to unlock key health challenges, and include research into breakthrough treatments,
diagnostics, and ultimately, a vaccine in the fight against COVID-19.
AusBiotech’s commitment to regenerative medicine.
AusBiotech, as the national peak body for Australian biotechnology, has a focus on providing funding and
partnership opportunities for Australian companies and recognises RM as an area of innovation and
opportunity.Part of its role is to listen carefully to Australia’s companies commercialising RM, so it can
champion advocacy on what’s needed to achieve success, and to foster a supportive community that is
dedicated to continuous learning.
Global growth is facilitated through the AusBiotech Regenerative Medicine Advisory Group (RMAG),
helping companies to navigate the sector, and collaborating with international bodies in Japan (FIRM) and
Korea (CARM), with the aim to facilitate cross-border partnerships. Several success stories from such
collaborations have led to new RM technologies entering the Asia-Pacific market. AusBiotech showcases
these RM successes as well as Australian and international best practice at its national conference held
each year in October.
To support its members to seek alternative financing sources and partnerships for their commercialisation
needs, AusBiotech provides opportunities for SMEs to raise funds through pitching to investors at its
flagship investment event, Australia Biotech Invest & Partnering. It also offers early-stage project
owners the opportunity to pitch to these same investors at the Early Stage
Investment Forum.
Australia’s goal is to translate more RM research into the clinic, and beyond to reach patients – a laudable
goal, for a thriving new industry, as Australia recovers from the COVID-19 pandemic.
Lorraine Chiroiu
Chief Executive Officer, AusBiotech
[1] Regenerative Medicine Opportunities for Australia, MTP Connect, October 2018
7AUSTRALIAN BASED COMPANIES
Cynata Therapeutics Ltd is a clinical stage company developing a proprietary therapeutic stem cell platform technology,
Cymerus™, which arose from the University of Wisconsin-Madison, a world leader in stem cell research. Cymerus
addresses a critical shortcoming in existing methods of production of allogeneic mesenchymal stem cells (MSCs) for
therapeutic use: the ability to achieve consistent economic manufacture at commercial scale without reliance upon
multiple donors. There is extensive interest in the development of MSCs as therapeutic agents, in light of their ability to
secrete bioactive molecules such as cytokines, chemokines, and growth factors, in addition to their immunosuppressive
and immunoregulatory properties. There are currently over 1000 clinical trials of MSCs, covering an extremely wide range
of therapeutic indications, including haematological, cardiovascular, orthopaedic, gastrointestinal and autoimmune
disorders, among others. However, there are very major limitations in conventional methods of MSC production including
the dependence upon multiple donors, the variability between donors, the relative scarcity of MSCs in adult tissue, and the
low proliferative capacity of adult stem cells compared to pluripotent stem cells. Cynata believes that the Cymerus
technology addresses these issues, uniquely placing Cynata to capitalise on the flourishing field of stem cell therapeutics.
The Company has been covered in highly favourable equity research analyses published by Baillieu Holst, BBY, SeeThru
Equity, H.C. Wainwright and Shaw & Partners. In 2019 the Company executed a global license agreement with FUJIFILM in
respect of CYP-001, Cynata’s first therapeutic product, which has shown highly promising efficacy in a Phase 1 clinical trial
in graft-versus-host disease (GvHD). FUJIFILM is a major participant in the regenerative medicine sector and the second
largest shareholder in Cynata, behind Fidelity, with around 7% of the shares.
PRODUCT PIPELINE
Cynata is the world leader in developing iPSC-derived cell therapy products. Following excellent results in a Phase 1
clinical study, the Company has commenced a clinical trial in patients with severe complications of COVID-19. A phase 3
clinical trial is expected to commence in 2H20 in osteoarthritis in association with the University of Sydney and similarly a
Phase 2 trial of CYP-001 in GvHD is expected to commence also in 2H20 through the partnership with FUJIFILM. A Phase 2
clinical trial in critical limb ischemia (CLI) has been approved by UK and Australian regulatory bodies. The Phase 1 clinical
trial results in GvHD provide a sound foundation for further development in numerous other indications, such as those in
which MSCs from other sources have previously been investigated. Cynata has also reported clear efficacy in pre-clinical
proof-of-concept studies with its Cymerus MSC products in models of cytokine release syndrome (CRS), CLI, diabetic
wounds, myocardial infarction, asthma, idiopathic pulmonary fibrosis, sepsis, acute respiratory distress syndrome (ARDS)
and in GvHD. Cynata is pursuing a vigorous partnering agenda in order to fully exploit its outstanding cell therapy
platform.
LATEST NEWS
Cynata has successfully completed a two-year follow up of patients in the Phase 1 clinical trial with its first Cymerus
therapeutic MSC product, CYP-001, in acute steroid resistant (GvHD). The study met all endpoints, demonstrating an
excellent safety profile and a very clear indication of efficacy in this challenging condition. Data from this study has been
published in Nature Medicine, providing important endorsement of Cynata’s Cymerus technology. A Phase 2 trial in GvHD
is expected to commence in 2H20. In addition, the Company has commenced the MEND clinical trial in COVID-19 patients
and expects the SCulPtor Phase 3 trial in osteoarthritis to commence during the latter half of 2020.
CEO: Dr Ross Macdonald
Established in Australia in 2011
ABN: 98 104 037 372
Status: PUBLIC (ASX:CYP)
www.cynata.com
CONTACT
Dr Ross Macdonald
P: +61 (0) 412 119343
9MESOBLAST
Mesoblast is using its proprietary technology platform to develop and commercialize innovative allogeneic cellular
medicines to treat complex diseases resistant to conventional standard of care and where inflammation plays a
central role.
The Company’s portfolio of Phase 3 product candidates comprises RYONCIL™ (remestemcel-L) for steroid-refractory
acute graft versus host disease (acute GVHD), remestemcel-L for the treatment of moderate to severe acute
respiratory distress syndrome (ARDS) due to COVID-19 infection, REVASCOR® for advanced chronic heart failure and
MPC-06-ID for chronic low back pain due to degenerative disc disease. Mesoblast also has a promising emerging
pipeline of product candidates and next generation technologies.
Innovative technology platform enables scalable manufacturing
Mesoblast’s novel allogeneic product candidates are based on rare (approximately 1:100,000 in bone marrow)
mesenchymal lineage cells that respond to tissue damage, secreting mediators that promote tissue repair and
modulate immune responses.
Mesenchymal lineage cells are collected from the bone marrow of healthy adult donors and proprietary processes
are utilized to expand them to a uniform, well characterized, and highly reproducible cell population. This enables
manufacturing at industrial scale for commercial purposes. Another key feature of Mesoblast’s cells is they can be
administered to patients without the need for donor–recipient matching or recipient immune suppression.
Mesoblast has proprietary technology that facilitates the increase in yields necessary for the long-term commercial
supply of its product candidates, and next generation manufacturing processes using xeno-free technologies and
three-dimensional bioreactors to reduce labour, drive down cost of goods and improve manufacturing efficiencies.
Robust Intellectual Property Estate
Mesoblast has an extensive patent portfolio with over 1,100 patents and patent applications across 82 patent
families, and patent terms extending through 2040. These patents cover composition of matter, manufacturing, and
therapeutic applications of mesenchymal lineage cells, and provide strong commercial protection for our products
in all major markets, including the United States, Europe, Japan and China. Licensing agreements with JCR,
Grünenthal, Tasly and Takeda highlight the strength of Mesoblast's extensive intellectual property portfolio covering
mesenchymal lineage cells.
Mesoblast will continue to use its patents to prosecute its commercial rights as they relate to its core strategic
product portfolio. When consistent with the Company’s strategic objectives, it may consider providing third parties
with commercial access to its patent portfolio.
Evidence-based Science and Translational Medicine
Mesoblast’s approach to product development is to ensure rigorous scientific investigations are performed with
well-characterized cell populations in order to understand mechanisms of action for each potential indication.
Extensive preclinical translational studies guide clinical trials that are structured to meet stringent safety and efficacy
criteria set by international regulatory agencies. All trials are conducted under the continuing review of independent
Data Safety Monitoring Boards comprised of independent medical experts and statisticians. These safeguards are
intended to ensure the integrity and reproducibility of results, and to ensure that outcomes observed are
scientifically reliable.
Global Operations
Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities
Exchange (MSB) and on the Nasdaq (MESO).
Chief Executive: Dr. Silviu Itescu
Established in Australia 2004
ABN: 68 109 431 870
Status: Public
www.mesoblast.com
info@mesoblast.com
10Orthocell Ltd (ASX:OCC) is a regenerative medicine company dedicated to the development of products for
the repair human tendon, bone, nerve and cartilage injuries. Orthocell’s two lead products are CelGro®, a
naturally derived collagen medical device for tissue repair, and Ortho-ATI™, a world-leading cellular therapy
for the treatment of chronic tendon injury.
Products
CelGro® is a platform technology that can be used in multiple clinical indications. CelGro® has distinct
advantages over existing tissue repair scaffolds, particularly with respect to its mechanical strength and ease of
use, and the quality of tissue repair. CelGro® is approved in Europe for various dental bone and soft tissue
applications, including dental implant procedures. CelGro®’s ability to support rapid, high-quality bone
regeneration has been demonstrated by trial data and clinical experience from our network of referring clinicians.
Orthocell is currently evaluating the performance and safety of CelGro® in clinical trials for nerve and tendon
repair. Orthocell’s first-in-class Ortho-ATI™ (Autologous Tenocyte Implantation) facilitates healing of damaged
tendons that have failed to respond to existing therapies, addressing a significant unmet clinical need. Ortho-ATI™
uses the patient’s own tendon-derived cells to stimulate tendon regeneration. Orthocell’s clinical program for
Ortho-ATI™ includes tendon repair in the elbow, shoulder and hip. Ortho-ATI™ is manufactured at Orthocell’s
GMP-licensed laboratory and has been used to treat over 500 patients in Australia, Singapore and Hong Kong.
Latest News
Orthocell recently announced some exciting preliminary results from the CelGro® nerve regeneration trial.
Participants experienced an 83% improvement in muscle power, allowing them to return to work, sport and to
perform everyday activities like dressing and picking up their children. These results further validate the quality of
tissue repair reported in our guided bone regeneration trial, and evidence of CelGro®’s flexibility of use in multiple
indications. Orthocell intends to expand its regulatory approval for CelGro® to Australia, the US and Europe in
bone, nerve and tendon repair.
Orthocell’s most recent annual quality survey indicated that 82% of patients were ‘satisfied’ to ‘extremely satisfied’
with how Ortho-ATI® relieved symptoms (i.e. chronic pain) and improved ability to perform everyday activities at
home and at work. Orthocell is well-advanced in preparing an application to the FDA for approval Ortho-ATI™ in
the US market, and close to completing recruitment in a clinical trial comparing Ortho-ATI™ to steroid injection for
the treatment of rotator cuff tendinopathy. This trial is a collaboration with DePuy Synthes Products Inc., part of the
Johnson & Johnson group of companies.
CEO: Paul Anderson
Established in Australia in 2006
ABN: 57 118 897 135
Status: Public
www.orthocell.com.au
CONTACT: Paul Anderson
P: +61 411 861 626
paulanderson@orthocell.com.au
11REGENEUS
Regeneus Ltd is a clinical stage regenerative medicine company developing a portfolio of novel cellular
therapies or the global pain market. The Company is listed on the ASX under ticker code RGS.
Regeneus’ product pipeline is underpinned by two proprietary novel allogeneic stem cell platforms:
·
Progenza - Derived from expanded allogeneic mesenchymal stem cells (MSCs) from human adipose tissue and
contains the bioactive secretions of the cells
Sygenus – Allogeneic MSC secretion used as a topical application for the treatment of inflammatory skin
conditions such as acne and wound healing
MSCs are the body’s natural healing and repair cells. They target diseases and secrete a diverse variety of bioactive
factors, including cytokines and growth factors that are responsible for reducing inflammation, promoting tissue
repair and reducing scarring. Rather than treating the symptom, MSCs look to regenerate and cure.
PRODUCT PIPELINE
The Company is focused on the commercialisation of its lead platform technology Progenza for osteoarthritis (OA),
ith a view to complete a Phase 2 clinical trial and license the technology in Japan. A successful Phase I trial showed
Progenza met primary safety endpoints as well as demonstrate efficacy in significantly reducing knee pain and
improving cartilage volume.
Recent pre-clinical data have shown Progenza demonstrated positive results for the treatment of allodynia, a
condition in which pain occurs from what is normally non-pain stimulation of the skin.
The Sygenus platform technology has shown to be safe and tolerable in human safety studies, and in high doses
shown to be more potent and longer lasting than morphine in terms of Its analgesic effects.
LATEST NEWS
Regeneus has undergone a recent strategic review focused on the commercialisation of Progenza to treat OA. The
Company’s partnership model
eans it works with leading university and research bodies to assist with the on-going pre-clinical and clinical
development of Progezna and Sygenus.
The Company has strong IP coverage in key markets including the US, China, Japan and Europe for both platform
technologies. Regeneus is supported by a management team with a strong track record of developing and
licensing novel regenerative medical technologies.
Regeneus CEO: Leo Lee
Established in Sydney, Australia
in 2007
ABN 13 127 035 358
www.regeneus.com.au
CONTACT
Investor Relations | WE Buchan
E: regeneus@we-buchan.com
P: +61 2 9237 2805
12CLINICAL
TRIALS
Clinical trial registries (clinicaltrials.gov, anzctr.org.au) were accessed BEFORE 01/10/2020.
This is not an exhaustive list of studies. HREC: Human Ethics Research Committee; MPC(s):
mesenchymal progenitor cell(s); MSC(s): mesenchymal stem cell(s); N/A: not applicable.14
15
16
COMPANY DIRECTORY
COMPANY CONTACT WEBSITE
Avita N/A www.avitamedical.com
w
ww
ww.avitamedical.com
ww
+61 3 8572 2500
Cartherics info@cartherics.com www.cartherics.com
Cell Therapies +61 3 8559 7310 www.celltherapies.com.au
Pty Ltd admin@celltherapies.com.au
Clinical Stem Cells N/A www.clinicalstemcells.com
Cryosite N/A www.cryosite.com
CSL N/A www.csl.com.au
COMPANY CONTACT PROFILE
Cynata +61 (0) 412 119343
www.cynata.com
Therapeutics ross.macdonald@cynata.com
Eppendorf N/A www.eppendorf.com
In Vitro 1300 552 003
www.Invitro.com.au
Technologies daina.elliott@invitro.com.au
Living Cell
N/A www.lctglobal.com
Technologies
Magellan
N/A www.magellanstemcells.com.au
Melbourne Stem
N/A www.melbournestemcellcentre.com
Cell Centre
18COMPANY CONTACT WEBSITE
+61 3 9639 6036
Mesoblast www.mesoblast.com
Miltenyi Biotec +61 (0) 405 399 959 www.miltenyibiotec.com
Orthocell +61 (0) 411 861 626 www.orthocell.com.au
paulanderson@orthocell.com.au
PeproTech ofir@peprotechasia.com www.clinicalstemcells.com
+61 2 9499 8010
Regeneus sandra.mcintosh@regeneus.com.au www.regeneus.com.au
Scientifix Pty Ltd nikki@scientifix.com.au www.scientifix.com.au
COMPANY CONTACT PROFILE
STEMCELL 1800 060 350
www.stemcell.com
Technologies cyndy.nauer@stemcell.com
Sydney Cell
Therapy sctf@optusnet.com.au N/A
+61 (0) 411 461 604
Foundation
Terumo BCT N/A www.terumobct.com
Thermofisher N/A www.thermofisher.com.au
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