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ASX RELEASE
17 May 2016
Shareholder Update Presentation
Brisbane, 17th May 2016. Factor Therapeutics Limited’s (ASX:FTT) management team will be giving a series of investor updates and presentations in Sydney, Melbourne and Brisbane during the week of the 16th of May.
This presentation material incorporates various progress updates that will be communicated to shareholders and investors in the course of those presentations.
- ENDS -
Corporate Contact Dr. Christian P. Behrenbruch Executive Director Factor Therapeutics Limited Tel: +61 7 3334 3900 Email: c.behrenbruch@factor-‐therapeutics.com
About Factor Therapeutics
Factor Therapeutics Limited is a biomedical technology company that is developing significantly more effective treatments for acute and chronic wound healing applications, including chronic skin ulcers and burns. Factor Therapeutics is commercialising VF-‐001 (previously denoted VitroGro® ECM), a technology created by cell biology, tissue engineering and protein engineering experts at the Institute of Health and Biomedical Innovation at the Queensland University of Technology. The business owns various patent families related to wound healing and other therapeutic uses. The Company’s shares are traded on the Australian, Berlin and Frankfurt stock exchanges. For more information, please visit www.factor-‐therapeutics.com
Important information
This announcement does not constitute an offer to sell, or a solicitation of an offer to buy, securities in the United States, or in any other jurisdiction in which such an offer would be illegal. The securities referred to herein have not been and will not be registered under the United States Securities Act of 1933 (the ‘US Securities Act’), or under the securities laws of any state or other jurisdiction of the United States and may not be offered or sold within the United States, unless the securities have been registered under the US Securities Act or an exemption from the registration requirements of the US Securities Act is available. This document may not be distributed or released in the United States.
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Shareholder
Update
May, 2016
Factor Therapeutics Limited
ASX : FTT
FACTORT H E R A P E U T I C S
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Notices
2
The information contained in the presentation is not intended to be an offer for subscription, invitation or
recommendation with respect to shares in any jurisdiction.
No representation or warranty, express or implied, is made in relation to the accuracy or completeness of the
information contained in this document or opinions expressed in the course of this presentation. The information
contained in this presentation is subject to change without notification.
This presentation contains forward-looking statements which can be identified by the use of words such as “may”,
“should”, “will”, “expect”, “anticipate”, “believe”, “estimate”, “intend”, “scheduled” or “continue” or similar expressions.
Any forward-looking statements contained in this presentation are subject to significant risks, uncertainties,
assumptions, contingencies and other factors (many of which are outside the control of, and unknown to Factor
Therapeutics Limited, and its officers, employees, agents or associates), which may cause the actual results or
performance to be materially different from any future result so performed, expressed or implied by such forward-
looking statements.
There can be no assurance or guarantee that actual outcomes will not differ materially from these statements.
The photographs of clinical subjects used in this presentation are illustrative of medical conditions associated with
potential applications of VF-001 (formerly marketed as VitroGro®). Actual clinical results may vary from those shown.
Relevant images accessed under Creative Commons.
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Corporate Dashboard
3
ASX ticker FTT
Product focus area Advanced Wound Care
Headquartered Brisbane, Australia
Clinical stage Phase II
Total issued capital 724,328,499 shares
Options 2,500,000 options
Share price* $0.036
Market capitalisation* $26m
Cash position ~$15m
Top 20 ownership 52% of cap table
*As at 13/05/2016
AUD $15m financing completed 3rd of May (1st placement tranche : 18th of March)
Corporate adviser : Taylor Collison
$10m over-subscribed placement / $5m rights issue
Shareholder base has been significantly institutionalised
Primary use of proceeds : US multi-centre Phase II trial (to be reported Q3, 2017)
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Key activities since first closing
of financing (18th of March)
Process transfer for manufacture
of clinical lots for US Ph II study
Establishment of medical
advisory board (MAB)
Contracting of CRO for Phase II
study
Finalisation of clinical protocol
for Phase II
Submission of IND amendments
to the FDA
Off clinical hold
Approximate timeline for further
shareholder information
Engineering run ok, cGMP lots by
end-May
Excellent progress in recruiting,
contracted by end-May
Critical path – complete ASAP
(final qualification in progress)
Under review, finalisation contingent
on selecting CRO
Target : end-June (lot certificates,
amended protocol)
End-July is the target*
4*Subject to no further questions/issues raised by the FDA
In parallel : site selection/qualification, validation of enrolment estimates,
contracting, ethics
Activity SummaryF
or p
erso
nal u
se o
nly
Focus : Manufacturing of Clinical Lots
Initiated on the 18th of March
Validation and transfer of materials
from Europe to the US (Boston)
Establishment of Phase II appropriate
(small scale) fill-and-finish process that
is “comparable” to prior large-scale EU
manufacturing.
Site qualification / audit
Engineering run – successfully completed during the last week of April
GMP run to be finished by end-May (standard dose, high dose,
placebo) but release assays still to be completed in Europe (our
validated assays) post-manufacturing
Lot release assays : end-May / early June
5
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CRO
QA/RA
Manufacturing & Supply Chain
Medical Advisory
Board (MAB)
Advisory / Services Overhaul
6
FTT’s new FDA
authorised
representative,
Vast experience
with all stages
of biologics
development
Four proposals received. Two
firms undergoing proposal
refinement / qualification
1 x AU
2 x US
1 x EU
KOLs*
Fill and finish
manufacturing
moved to the
US (scale-
appropriate)Labelling, storage, cold chain
and logistics
FTT has completely
overhauled its advisory
and service provider
relationships
*KOL = Key Opinion Leaders. TBA
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Ok – Financing done,
manufacturing in progress.
What about the trial?
Precision Wound Care
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Value Inflection : Recap
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Phase II Efficacy
Study (US)
EMA
Resubmission
VF-001 Partnering
Indication
Expansion
Ocular
Collaboration
~18 months
• Build a partnering-
quality data set
• Move the company
toward Phase III as a
biologic drug in the US
• Meet the additional
safety database
requirements for EMA
to enable CE Mark
resubmission
• Our Phase II study will
meet the additional
clinical data
requirements for EMA
• Some limited non-
clinical work should
pave the way for re-
submission end-2017
• Focus : DFUs*
• Will be supported
by clinical data
• New applications
• IP “ever-greening”
*Diabetic foot ulcers
• New market
opportunities
Our planned Phase II study in
the US underpins most of the
value creation in the next 18
months.
Designing this study for
maximum impact, both
clinically and commercially,
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VLUs : Market Characteristics
Venous Leg Ulcers (VLUs) : a volume market
Oft-cited “3% of the population, millions of potential patients”
Chronic wounds constitute 5-7% of healthcare costs
The reality?
For the majority of patients, nothing has yet beaten high
quality compression bandaging / moisture dressings
The general trend is toward treating VLUs in the
community setting. Anything that involves a “procedure”
therefore does not address the clinical unmet need:
• Tissue-derived products must be used by a physician, really
only applicable to the most serious (5-6%) of ulcer patients
• Anything that sutures or involves grafting, tissue harvesting,
etc. is not applicable to the “community setting”
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Venous Leg
Ulcer
Topical
Delivery
Accelerated
Healing
Treatments for VLU are highly price sensitive and need to be able to be used outside of the
specialty wound care setting. Expensive and complex procedures don’t fit this market.
This is Factor Therapeutics’ unique opportunity because our product is simple to administer
and our COGS is low
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VLUs : Margolis Wound “Severity” Scores
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Severity
Proportion in
the real world
population
Probability of Healing within 24
Weeks with Limb Compression
(%)*
Baseline
Ulcer Area
(cm2)
Ulcer
Duration
(months)
0 (least
severe)~ 69% 93.0 % ≤ 5 ≤ 6
1 (middle)~ 25%
65.0 %
≤ 5 > 6
> 5 ≤ 6
2 (most
severe)~ 6% 13.0 % > 5 > 6
* Margolis DJ, Berlin JA, Strom BL. Which venous leg ulcers will heal with limb compression bandages? Am J Med. 2000 Jul;109(1):15-9.
The vast majority of these patients
will be treated in the community
setting, not the specialty care setting
Seminal work by epidemiologist and wound care expert, Dr. David Margolis
(U. Penn), provides a simple classification for VLUs that is very meaningful to
understanding the clinical strategy for VF-001
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Potential Impact of Margolis on VF-001
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Marg
olis 1
Ma
rgo
lis
0
• Severe disease – treated in the specialty wound care setting.
• High cost-benefit supports expensive treatments
• Grafts, cell therapies, scaffolds, advanced materials.
• Highly fragmented / congested market
6% 31% 100%Cumulative Patient Population
Severity
of
venous d
isease
High
Low
• Mild disease – community care
• Compression bandaging, dressings
• Highly fragmented / congested
market (low “value”/differentiation)
Clinical unmet need, low competition.
A cost-effective treatment that can be used
in the community setting would be a game-
changer
Marg
olis 2
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z
Marg
olis 2
Ma
rgo
lis
0
6% 31% 100%Cumulative Patient Population
Severity
of
venous d
isease
High
Low
Our VF-001 Clinical Data Supports This View
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No benefit
observed
3x faster healing
in moderately
severe patients*
*Based on a retrospective cross-trial analysis of 48 patients in an open-label study
3xP=0.103
P=0.002M
arg
olis 1
Time (Days)
0 20 40 60 80 100
Cum
ulat
ive
Prob
abili
ty o
f Non
-Hea
ling
0.0
0.2
0.4
0.6
0.8
1.0
Community (N=33)VitroGro ECM (N=12)Clinic (N=36)
~ 10%
improv.
P=0.029
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Impact on Clinical Trial Design
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Margolis 0 Patients
VF-001 demonstrates limited benefit. These patients respond
well to “standard care” (bandaging / moisture dressings)
Contributes to cost-benefit but is not our biggest value-add
Wish to exclude the “easy” healers because they increase
the number of patients (and $) required to show efficacy
Margolis 2 Patients
Severe venous disease, effectively “multi-pathology” (co-morbidities)
Based on our limited data, VF-001 has no detectable efficacy in this patient population
We want to exclude these patients because they don’t contribute to efficacy
Margolis 1 Patients : Target Patient Population for Trial Efficacy
Moderately severe ulcers – treated in the community setting
Impacting the health of these patients is of enormous benefit. These are the “hard to
heal” patients that cost the most in the community care setting, and where our product
has the strongest signal
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Impact on Product Label?
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For Margolis 0/1 patients, Standard Care will
always involve compression bandaging and
moisture dressings in the community setting. It is
cheap, effective, not yet beaten
Negative pressure wound care systems may
have future applicability but are not yet clinically
proven for VLU – more of a novelty at this stage
Margolis “classification” is a high-level strategy
for patient delineation. It is an “effect”, not a
“score”. Qualitative, not quantitative
In the “real world”, we will exclude patients with
severe venous disease (Margolis 2s) by
size/duration of ulcer
In the “real world”, we will not exclude Margolis 0
patients from treatment eligibility, rather we will
require that those patients have not responded
to a duration of Standard Care (say, 4-8 weeks)
Post-Treatment
(Week8)
Baseline
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Precision Wound Care
Based on expert consultation we have refined a clinical
design that targets our optimal patient population
The use of a “precision” medicine approach is relatively
unexplored in wound care and likely why many products
have failed
We will pre-screen and eliminate “easy” Margolis 0 patients
(by giving a duration of Standard Care and excluding
patients that stabilise/improve). We will also exclude severe
venous disease (Margolis 2s)
We learned a great deal from the last clinical trial, which
was “enriched” for Margolis 1 and Margolis 2 patients
We now need to further validate the efficacy signal we have
previously seen in a blinded, placebo-controlled study
Primary end-point : % of patients fully healed after 12 weeks
of treatment (+ standard care) per FDA guidance
Important secondary end-points : time to healing, pain
15
Cell attachment to
the wound bed via
VF-001
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Concluding Remarks
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Immediate launch of clinical activities post-financing
Manufacturing of clinical lots on track
Refinement of clinical trial protocol based on expert review of our prior
clinical data, optimisation of inclusion/exclusion criteria for our target patient
population
As previously disclosed, revised protocol will be submitted to FDA along with
certifications of analysis (target : end-June)
The next general shareholder update will focus on medical advisory board,
CRO selection, site selection progress, etc.
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