Amorfix Life Sciences Leaders in Misfolded Protein Diseases Q4 2014 1

Amorfix Life Sciences
Leaders in Misfolded Protein Diseases
Q4 2014
1
Amorfix Life Sciences
Founded 2004 by Dr. Neil Cashman
2005 Public listing on TSX (AMF)
Corporate Headquarters Mississauga, Ontario CANADA
Relocating to South San Francisco Q1 2015
Focus on Misfolded Protein Diseases
Neurodegenerative diseases and cancer
Proprietary platform technologies for development of targeted
therapeutics and companion diagnostics
Epitope Protection
ProMIS Discovery Technology
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Management and Board of Directors
Management Team
Board of Directors
Robert Gundel, PhD, MBA
• President and CEO
William Wyman, MBA
• Chairman of the Board
• 30 years of Pharmaceutical industry experience
• Booz Allen, Oliver Wyman
Neil Cashman, MD
• Founder and CSO
William Copeland, MBA
• Director
• World renowned neurologist
• Leader in misfolded protein diseases
• Vinik Asset Management
Warren Whitehead, MBA
• CFO
• 30 years of Pharmaceutical industry experience
Johannes Roth, MA
• Director
• Five T Capital
Stuart Hwang, PhD
Elliot Goldstein, MD
• Director
• VP of Business Development
• 25 years of Pharmaceutical industry experience
• Immune Pharmaceuticals, Virtua, British
Biotech, SmithKline
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Amorfix Platform Technologies
Amorfix Technology
Disruptive Idea 1: Protein Misfolding and Disease
Cancer
Cancer
Neurodegeneration
Neurodegeneration
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Disruptive Idea 2: Antibodies Can Selectively Target
Misfolded Proteins while Sparing Native Isoforms
Efficacy: Specific targeting of a
pathogenic species
Safety: Selective sparing of
normal proteins
–
–
–
–
– Preservation of normal function
– Minimization of autoimmunity
– Minimal regimens in
therapeutic vaccines
Neutralization of toxicity
Blockade of propagation
Acceleration of degradation
Minimal “target distraction”
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Amorfix: The Leader in Misfolded Protein Diseases
Leveraging Platform Technology and Intellectual Property
Licensing Deals with Upfront Payments, Milestones and Royalties
CJD
Chronic Wasting
Disease
Misfolded Protein Diseases
Prion
Disease
Cancer
ProMIS Discovery
Epitope Protection
Neurodegenerative
Ovarian
Lung
Prostate
Melanoma
Lymphoma
Leukemia
Core technology can be used
across multiple disease areas:
- therapeutic antibodies
- companion diagnostics
- vaccines
ALS
Alzheimer’s
disease
Parkinson’s
disease
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ProMIS™ Algorithm: Rational Selection of DSEs
ProMis™ Discovery – An Enabling Technology
• A computational algorithm that predicts where proteins will
misfold or partially unfold
• Used to identify specific areas of misfolded proteins used to
generate novel targeted therapeutics
INPUT
Calculate free
energy of
unfolding ΔGij for
region starting at
residue i with
length j
Loop over i = 125 to 228 - j
OUTPUT
Loop over j = 1 to 25
DSE
Candidates
Targeted
Therapeutics
High resolution
structure of the
natively folded
protein
OUTPUT: Unfolding Energy Landscape = Disease Specific
Epitopes CANDIDATES
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Amorfix Technology: Beauty in Simplicity
Therapeutic Antibodies
1. ProMIS™ Discovery Technology
Identifies unique areas on misfolded
protein targets
2. These misfolded proteins are only
expressed on the surface of tumor
cells, not normal cells
3. Antibodies generated against these
unique areas don’t ‘recognize’ the
normal cell surface receptor and
only bind to tumors.
Normal Cell Surface
Receptor
Misfolded Cell Surface
Receptor
Antibodies
Multiple ways to kill tumors with antibodies
- Antibody alone
- Antibody recruits immune system
- Antibody drug conjugate
Normal Cell
Cancer Cell
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Proof of Concept of Disruptive Ideas:
Therapeutic Benefit of Targeting Misfolded Epitopes
ALS
Treatment with antibodies directed
against misfolded SOD1 increases
survival time. Vaccine treatment
produces the same therapeutic
results.
Inhibition
of
misfolding
propagation
Ovarian Cancer
Treatment with antibodies directed against
misfolded PrP specifically targets tumors,
not normal tissue, and significantly reduces
tumor growth
% increase in tumor volume
(compared to day 0)
SKOV-3
450
400
350
300
250
200
150
100
50
0
Vehicle
Taxotere
AMF-1c-120
0
5
10
15
20
Day After Treatment Initiation
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Options for therapeutic antibodies:
- Antibody alone; recruiting the body’s
own immune defense systems
- Antibody drug conjugates; precise
delivery of toxins directly to the tumor
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ProMIS™ Discovery Partnering Activity
• Currently in discussions with eight big pharma
organizations about platform technology deals and
Research Agreements.
• Amorfix provides partner with patentable disease specific
epitopes (targets) for the generation of therapeutic
antibodies
• Deals include upfront cash payments, development
milestone payments and royalties on sales
• We expect to complete the first deal end of 2014/early 2015
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Amorfix Epitope Protection™ Technology
Finding the “Needle in the Haystack”
Misfolded proteins are present in a million fold excess of normally folded proteins
Technical hurdle: how to identify and measure these small amounts of misfolded proteins
Amorfix Epitope Protection and AMFIA technologies solve this problem.
Epitope Protection™ Technology
NO2
Normal
Monomers
NO2
Chemical Modifier
Disease
Oligomers
Aggregates
NO2
NO2
NO2
NO2
NO2
NO2
NO2
NO2
NO2
NO2
Disaggregation
NO2NO2 NO2
= epitope available for chemical modification
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Alzheimer’s Disease Diagnostic
Identification of Early-Stage Disease
Alzheimer’s disease diagnostic technology:
- Measures levels of aggregated A oligomers in CSF
- A oligomers are a “hallmark” of Alzheimer’s disease
Diagnostic sensitivity for early-stage disease = 90%
Highest sensitivity
in the industry
Diagnostic sensitivity for AD individuals = 97%
Separate Detection of A 40 and 42
No other company in the world has this capability.
Enables research into Abeta oligomer’s role in early
Alzheimer’s disease.

Can be used as a biomarker to ‘monitor’ target
engagement, drug mechanism of action and efficacy
over time.

Additional secondary endpoint in clinical trials to
support observations of cognitive function.
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Signal / Unspiked CSF


Original A40/42
New A40
New A42
64
32
16
8
4
2
unspiked CSF
1
0.5
10
2
10
3
10
4
10
5
10
6
Dilution of 10% AD Brain Homogenate
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AD Diagnostic Partnering
Option 1
Exclusive worldwide license to develop and
commercialize
•
•
•
Upfront payment
Milestone payments
Royalties on sales
Non-exclusive license for use of technology
Option 2
•
•
•
Option 3
Amorfix commercializes the test
•
•
•
Upfront technology access fee
Tech Transfer based milestone payments
Yearly maintenance payments
Go with CLIA approved lab or CRO
Clinical validation of the assay
Service-based pricing
In Discussions with Four Big Pharma Companies
for Licensing the AD Diagnostic Technology
Plan to complete the first deal in Q4 2014
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One, Three, Ten Year Plan
Evolving The Business Over Time
Fully Integrated Platform Technology Business
Year 10
- Cutting edge technology across a wide variety of businesses
- Large number of collaborations using our technology
platforms to develop products
- Continue to improve and understand misfolded proteins as a
means to expand the therapeutic arena
Partially Integrated Drug Development Business
Year 3
-
Develop strength in Alzheimer’s, ALS and certain cancers
Increase the number of partners using our technology platforms
Expand capabilities and technology toolbox (e.g. protein crystallography)
Begin to expand beyond neurodegenerative disease and cancer
Revenue generated from partnered programs
Obtain US Listing
Building The Platform Technology Based Business
Year 1
-
ProMIS Discovery technology
Epitope Protection
Move operations to San Francisco Bay Area
Improve visiblilty to investors and partners
Obtain funding to achieve three year plan
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Current Partnerships
May 2010
High Affinity Rabbit Antibodies. Amorfix pays technology access upfront,
milestone payments and royalties.
November 2011
Research Agreement (50/50 cost/profit split). Urease-antibody drug conjugate for
Ovarian cancer
July 2012
Exclusive license for Preclinical AD diagnostic. They do all sales, marketing and
perform the assay. Amorfix gets double-digit % of sales.
November 2012
Multiple cancer antibody targets. Modest technology access fee paid.
April 2013
Research agreement. Lung cancer targets. No money exchanged.
Kyowa Hakko Kirin
January 2014
Non-exclusive license to AD CSF assay. Funded R&D for assay optimization and
validation. .
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Short-Term Priority List
Bring in non-dilutive revenue
• Close at least one ProMIS discovery deal
• License AD diagnostic
• Complete other partnering deals
Get the message out/Rebranding the
company
• Publish data in top tier scientific journal
• Hire IR/PR firm
• Present at high visibility conferences
Stops the need for continued PP’s
Validates our technology
Sets stage for more deals
Gets the company on radar screen
Increases share price
Sets stage to bring in more cash
Secure additional financing
• Angel investors
• VC firms
• Public offering
Stabilizes the company
Enables further growth of the business
Generates further value
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Financing Strategy/Options
• Generate non-dilutive revenue through partnering deals
for ProMIS Discovery technology and the AD assay.
• Venture Capital funding, Angel Investors
• Financing Goal: $10M
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