20150108- biOasis Q1 - IR Presentation.pptx

Transcription

20150108- biOasis Q1 - IR Presentation.pptx
OTCQX: BIOAF
TSX.V: BTI
A natural solution
to deliver medicine
to the brain !
biOasis.ca!
QUARTER 1 !
2015!
Forward Looking Statements
Certain statements in this presentation contain forward-looking statements within the meaning of the Private Securities Litigation
Reform Act of 1995 or forward-looking information under applicable Canadian securities legislation that may not be based on
historical fact, including without limitation statements containing the words “believe”, “may”, “plan”, “will”, “estimate”, “continue”,
“anticipate”, “intend”, “expect” and similar expressions. Such forward-looking statements or information involve known and unknown
risks, uncertainties and other factors that may cause our actual results, events or developments, or industry results, to be materially
different from any future results, events or developments express or implied by such forward-looking statements or information. Such factors include, among others, our stage of development, lack of any product revenues, additional capital requirements, risk
associated with the completion of clinical trials and obtaining regulatory approval to market our products, the ability to protect our
intellectual property, dependence on collaborative partners and the prospects for negotiating additional corporate collaborations or
licensing arrangements and their timing. Specifically, certain risks and uncertainties that could cause such actual events or results
expressed or implied by such forward-looking statements and information to differ materially from any future events or results
expressed or implied by such statements and information include, but are not limited to, the risks and uncertainties that: products
that we develop may not succeed in preclinical or clinical trials, or future products in our targeted corporate objectives; our future
operating results are uncertain and likely to fluctuate; we may not be able to raise additional capital; we may not be successful in
establishing additional corporate collaborations or licensing arrangements; we may not be able to establish marketing and the costs
of launching our products may be greater than anticipated; we have no experience in commercial manufacturing; we may face
unknown risks related to intellectual property matters; we face increased competition from pharmaceutical and biotechnology
companies; and other factors as described in detail in our filings with the Canadian securities regulatory authorities at
www.sedar.com.
Given these risks and uncertainties, you are cautioned not to place undue reliance on such forward-looking
statements and information, which are qualified in their entirety by this cautionary statement. All forwardlooking statements and information made herein are based on our current expectations and we undertake no
obligation to revise or update such forward- looking statements and information to reflect subsequent events
or circumstances, except as required by law.
2"
Central Nervous System Diseases
AFFECT 1 IN 6 PEOPLE !
CNS diseases account
for 12% of global
deaths1
Incidence of CNS
diseases will increase by
12% by 20301
Over 1000 different CNS
diseases with drugs
hindered by BBB2
Total cost of CNS
diseases is over $500
billion in USA alone2
CNS disease market is
over $81.2 billion US
and rising3
ALL TREATMENTS HINDERED BY THE BLOOD BRAIN BARRIER !
1)  (2012). Brain Facts; A Primer on the Brain and Nervous System . Publication, Society for Neuroscience.
2)  (2007). Neurological disorders: Public health challenges . Study, UN World Health Organization .
3)  bcc Research . (2010, September ). Therapeutic Drugs for Central Nervous System (CNS) Disorders: Technologies and Global Markets, PHM068A.
3"
"
Challenge "
The Blood Brain Barrier (“BBB”) A protective barrier that separates the brain from the
circulatory system, which blocks harmful chemicals from
entering the brain tissue
Problem
The BBB prevents medicines (anti-cancer drugs, enzymes,
antibodies, gene silencing siRNA, etc.) from entering the
brain at levels effective for treatment, blocking about:
•  98% of small molecule drugs •  100% of large molecule drugs1
Solution
biOasis has discovered a naturally occurring biological
process that can transport drugs across the BBB and into
the brain tissue for the treatment of thousands of CNS
diseases and brain disorders Capillary network of tightly woven endothelial
cells lining the blood vessels in the brain,
regulating transport of essential molecules and
maintaining a stable environment
1)  Royal Society of Chemistry
4"
biOasis Presents: The Transcend Program The Transcend Drug Delivery Platform
Patented carriers that deliver medicines across
the BBB for the treatment of CNS diseases Transcend offers potential treatments for:
"
Brain Cancers &
Tumours
Neurodegenerative
Diseases like
Alzheimer's Disease
Stroke and
Traumatic Brain
Injury
Metabolic
Diseases like
Lysosomal Storage
Diseases
Gene
Silencing
Therapy
for ALS
5"
Breakthrough Solution
The Transcend Program Exploits a natural, biological process to deliver medicines into the brain tissue by linking preexisting drugs to a human protein that freely crosses the BBB through receptors located on the
brain capillary wall in a process called Receptor Mediated Transcytosis. MTf (Melanotransferrin)
•  Iron-binding human protein found at low concentrations in
the blood
•  When linked to existing drugs, MTf can deliver effective doses
of medicine into the brain, distributing the medicine with a
wide distribution throughout the brain tissue
MTfp (MTf peptide) •  biOasis has discovered the peptide found within the full-
length protein (MTf) that is responsible for transport of MTf
across the BBB
•  MTfp has shown improved brain penetration and greater
commercial potential over the full-length MTf protein 6"
Advantage of MTfp
! 
Higher commercial value over MTf
! 
More efficient delivery across the BBB
! 
Lower cost of production ! 
Greater consistency in manufacturing ! 
Improved quality control
! 
Simpler to link to medicines ! 
! 
! 
MTfp in Action
Brain Tissue RMT!
Blood Vessels Transports a variety of compounds
with no apparent size limitation
Extends biOasis patent portfolio for
additional ~20 years
Pharmaceutical collaborators currently
licensing MTfp MTfp
Therapeutic
RMT -Receptor
Mediated
Transcytosis 7"
Market Opportunities for Transcend Lysosomal
!
!
2014 Drug Market Estimates Storage
Disease !
Billion
USD
~$2
Neurodegenerative
Disease"
~$20
Billion
USD
Disorders
Stroke &
Traumatic
Brain Injury
Billion
USD
~$81.2
Billion
USD
~$32.3USD
Central Nervous System Psychiatry
Billion
Clinical
need met but
improved
BBB
penetration
could
increase
efficacy
Pain &
Migraine
"
~$33.7USD
Billion
~$7.5
Infection
~$138Billion
USD
A carrier to cross the BBB would establish a
foothold within these major markets
8"
Brain Cancer Drugs 2013 drug sales for cancers that frequently metastasize in the brain1 Herceptin® (trastuzumab)
$6.6Billion
USD
HER2+ Breast Cancer
~40% Metastasize
Rituxan® (Rituximab)
Blood cancers: lymphoma, leukemia
~24% Metastasize Erbitux® (Cetuximab) Lung.
colon cancer, etc.
~35% Metastasize Taxol® (Paclitaxel)
Lung, ovarian, etc.
~35% Metastasize 1)  Company Reported Data $1.9Billion
USD
$92Million
USD
$7.5Billion
USD
Improved cancer
treatments have
led to an increase
in brain tumours,
creating a need
for anti-cancer
drugs that target
the brain The Transcend
Program has been
shown to reduce
brain tumour
volume by 84%
when anti-cancer
drugs are linked to
MTf Anti-cancer
drugs linked to
MTf penetrate
the blood
tumour barrier
10 times more
efficiently 9"
Business Opportunities Patent Cliff Estimated ~$250 billion in lost
revenue from patent
expirations from 2012-20151
"
Offers potential patent extensions
to the pharmaceutical industry
through New Chemical Entity
designation for many preexisting,
patent-expiring drugs
1) 
2) 
3) 
Market Growth Unmet Medical Need
CNS Drug Market is the
second largest pharmaceutical
segment behind oncology3 Over 1000 CNS diseases and brain
disorders with treatments hindered
by drug delivery across the BBB2
"
Aid in the development of new
therapies and revitalize dormant
candidates that are proven
efficacious but do not cross the
BBB
drug market by
Expand the CNS
" opening up an industry that has
been heavily stagnated by
ineffective drug delivery to the
brain
DeRuiter, J., & Holston, P. L. (2012, June 20). Drug Patent Expirations and the “Patent Cliff”. U.S. Pharm. 2012;37(6)(Generic suppl):12-20.
(2007). Neurological disorders: Public health challenges . Study, UN World Health Organization . http://www.un.org/apps/news/story.asp?newsid=21689&cr=neurological#.U7iNnBa4klI
(2012). Brain Facts; A Primer on the Brain and Nervous System . Publication, Society for Neuroscience.
10"
Management
Board of Directors Rob Hutchison, Chair & CEO
Former: CEO & Chief Scientist at eCharge Corporation (One of the world’s first internet banking system) Chris Fibiger, PhD Former: Head of Neuroscience, Amgen; VP
Neuroscience, Eli Lily & CSO, Biovail Laboratories
Greg Gubitz, LLB
Former: VP, Biovail Corporation & COO, MDS Capital
Terry Pearson, PhD
Professor: Biochemistry & Microbiology, UVIC
Ron Erickson Current: Chair & CEO, Visulant Technologies
Michael Hutchison, QC, LLB
Current: Partner, Smith Hutchison Law Corporation Team
Judi Dalling, LLB, CFO Seasoned public company CFO and corporate lawyer Wilfred Jefferies, PhD, Founding Scientist Discovered MTf’s ability to cross the BBB
Professor: Microbiology & Immunology, UBC
Reinhard Gabathuler, PhD, Chief Scientist
Former: VP Brain Research, Biomarin & CSO,
Angiochem Inc.
Mei Mei Tian, PhD, Scientist UBC, Microbiology and Immunology (MTf expert) Laura Ho, MSc, Scientist UBC, Microbiology and Immunology
Tiffany Tolmie, Corporate Communications Honours, Wilfrid Laurier University Corporate Strategy
Obtain
independent,
3rd party
validation of
MTf & MTfp
•  National Research
Council •  BC Cancer Research
Centre •  Texas Tech University
Health Sciences Center
•  Southern Research
Institute
•  University of Alabama Expand &
protect
intellectual
property
portfolio •  Over 30 patents granted for
blood-brain barrier drug
delivery and
neurodegenerative
diseases •  Strong pending patent
portfolio •  Over 20 year patent
protection on MTfp Advance
internal
development
programs at
UBC •  Successful delivery of over
12 therapeutic compounds
and over 6 imaging agents
to the brain (enzymes,
antibodies, small molecule
drugs & biological agents)
•  Potential to achieve major,
near-term milestones
Collaborate
with reputable
pharmaceutical
companies &
institutions •  Many “shots on goal”
leading to commercial
transactions with
numerous licensees
•  Collaborations with large
pharmaceutical partners "
12"
Strategic Collaborations The Transcend Program
MTfp
MedImmune
Brains for Brain Foundation
(AstraZeneca: AZN)
Licensing Agreement
Research Agreement
AbbVie
UCB Pharmaceuticals
MTf (Abbott Labs: ABT )
Research Agreement
(UCB)
Research Agreement
Ongoing discussions with large
pharmaceutical companies on
licensing and research
agreements. 13"
MTf: Rate of Uptake in Brain
Compound
Kin (x10-6;
mL-1/s-1/g-1)
Glucose
9500
Reference
Smith (2003)
Melanotransferrin (MTf) 640
biOasis’ Transcend Program is the only drug delivery platform
that utilizes a natural process to deliver drugs into the brain1
Morphine
200
Cisternino et al. (2001)
Aprotinin
160
Angiochem in Stage 2 Clinical Trials2
Insulin Rec Antibody 100
ArmaGen licensing to Shire (2014)3
Leu-Enkephalin
60
Zlokovic (1987)
Morphine-6-Glucuronide
24
Temsamani et al. (2005)
RAP 10
Raptor Therapeutics – Licensing Issues4
Beta Amyloid 6.5 Banks (1991)
DADLE
6.5 Chen (2002)
TNF-α 4.3 Pan (2002)
1) 
2) 
3) 
4) 
More
Transport Efficiency !
Competitors
Compounds that freely cross the BBB compared to MTf
Less
Demeule et al. (2002)
Demeule et al. (2008)
Pardridge (1997)
Pan (2004)
14"
Development Program: "
biOasis’ MTf + Anti-Aβ antibody Alzheimer’s Disease Results
biOasis’ MTf + anti-Aβ antibody conjugate increases
transport into brain tissue by ~5-fold
Anti-Aβ antibodies have
undergone clinical trials for
Quantitative Image Analysis by Laser
treatment of Alzheimer’s disease Opportunity Scanning Confocal Microscopy
Using antibodies directed at Aβ
are potential therapeutics for
reducing amyloid plaques in the
brain, which are indicative of
Alzheimer's disease
MTf + Anti-Aβ
Anti-Aβ
National Research Council of Canada (NRC)
15"
Development Program:"
biOasis’ MTf +
α-L-iduronidase (“IDU”)
Lysosomal Storage Diseases
IDU Brain Enzymatic Activity !
Rare inherited metabolic diseases caused by an
enzyme deficiency primarily affecting children 1.2"
1"
Problem
•  MPS I is caused by an IDU enzyme deficiency •  Currently CNS effects untreatable Results •  biOasis’ MTf + IDU (lysosomal enzyme)
conjugate increased IDU brain enzyme activity ~ 4-fold, showing delivery to the brain tissue Opportunity •  Offers the promise of an efficient enzyme
replacement therapy
Total IDU activity
Hurler Syndrome (MPS I)
Parenchyma
Capillaries
0.8"
0.6"
0.4"
0.2"
0"
IDU
MTf - IDU
16"
Development Program:"
biOasis’ MTf + iduronate-2-sulfatase (“IDS”) Hunters Syndrome (MPS II)
•  Lysosomal Storage disease, MPS II is caused
by an IDS enzyme deficiency •  Currently CNS effects untreatable Results •  biOasis’ MTf + IDS (lysosomal enzyme)
conjugate increased IDS brain enzyme activity Native Enzyme
~20-fold, showing delivery to the brain tissue does not enter the brain tissue efficaciously Opportunity •  Brains for Brain Foundation are in studies with
MTfp + IDS, which offers the opportunity to
fast-track to human trials •  Offers promise of an efficient enzyme
replacement therapy MTf + IDS Enzyme enters the brain tissue 17"
Development Program: "
biOasis’ MTf + Doxorubicin •  Doxorubicin is currently used as a cancer
treatment for breast, lung, ovarian, thyroid and
stomach cancers; multiple myeloma, leukemia,
Hodgkin's lymphoma, etc.
Problem •  These cancers metastasize in the brain but
MTf Significantly Enhances
Doxorubicin Transport into the Brain
% INJECTED DOSE (G TISSUE/G BODY MASS)*100%
3.00"
Doxorubicin does not efficiently penetrate the
BBB
Results 2.00"
Conjugate retains
full activity once
released in the
brain
•  Significant INCREASE in brain uptake with the
MTf + Doxorubicin conjugate vs. Doxorubicin
alone 1.00"
Opportunity •  MTf + Doxorubicin conjugate could be used to
treat brain cancers that metastasize in the brain
0.00"
MTf + DOXO
DOXO 18"
MTf Reduces Doxorubicin Uptake in the Heart
•  Doxorubicin’s most serious adverse
effect is life-threatening heart
damage Results
•  biOasis’ MTf + Doxorubicin vs.
Doxorubicin on its own showed a
significant DECREASE in uptake in
the heart
Opportunity
•  Could be used to treat brain
cancers and decrease heart-uptake
side effect, offering increased
usage of Doxorubicin for brain
cancer treatment
% INJECTED DOSE
(gram brain tissue/gram body mass)*100 Problem 20.0"
15.0"
10.0"
5.0"
0.0"
MTf + DOXO
DOXO 19"
Development Program: "
biOasis’ MTf + Trastuzumab (Herceptin®) •  Trastuzumab (Herceptin®)1 is a humanized
monoclonal antibody used to treat HER2+
breast cancer with annual global sales ~$6.6
billion2
MTf + Trastuzumab (Herceptin®) Confocal Images Two Hours Post IV Administration
Brain Capillaries
•  Herceptin® increases survival rate but ~40%
of patients eventually develop breast cancer
metastasis in the brain3
Cell Nuclei
•  Trastuzumab (Herceptin®) does not cross the
BBB on its own
•  biOasis aims to, not only prevent and treat
HER2+ breast cancer that metastasizes in the
brain, but also for Transcend to become the
‘Standard of Care’ for HER2+ breast cancer MTf + Trastuzumab (Herceptin®)
Confocal Image Performed by iCapture at
St. Paul’s Hospital Vancouver Canada
1)  Herceptin® is a registered trademark of Roche/Genentech
2)  Company Reported Data – 2013 3)  Britta Weigelt, Johannes L. Peterse & Laura J. van't Veer. (2005, August). Breast cancer metastasis: markers and models. Nature Reviews
Cancer 5 , 591-602.
20"
MTf + Trastuzumab (Herceptin®) "
Texas Tech University - Breast Cancer Model
Highlights • 
• 
• 
• 
• 
Reduced the number of HER2+ breast
cancer tumours in the brain by 68%
Total tumor volume reduced by 84% in
only 14 days, after 4 treatments
Penetrated the Blood-Tumour Barrier
10 times better than Herceptin® alone Increased cancer killing effect of
Herceptin® in tumours throughout the
body when linked with MTf Offers the potential to be used in
conjunction with HER2+ cancer
therapy as a treatment and
preventative measure BEFORE the
cancer metastasizes to the brain Number of Tumours
90"
80"
70"
60"
50"
40"
30"
20"
10"
0"
68%
Reduction MTf
TZM
MTf-TZM
Saline
Control !
TZM = Trastuzumab (Herceptin®) 21"
Development Program: "
MTfp + siRNA Small Interfering RNA (siRNA)
•  Silences target gene expression by knocking down disease
causing genes associated with human diseases (i.e. cancers, neurodegenerative and metabolic diseases, etc.)
Problem
•  Gene targeting within the brain is currently unachievable because
siRNA does not cross the BBB NO siRNA detected in Brain
Results •  MTfp + siRNA conjugate delivered into the brain tissue •  Demonstrated 40% to 50% decrease in target gene vs. siRNA
alone
Opportunity •  A potential cornerstone technology for the development of new
therapeutics suppressing disease-linked genes in the brain •  siRNA is a rapidly emerging field of medicine National Research Council MTf + siRNA detected in Brain
22"
Market Profile "
TSX.V:BTI; OTCQX:BIOAF
Shares Issued & Outstanding
44,024,257
Fully Diluted 51,158,704
Options 5,190,000
Warrants 1,944,477 Market Cap
Directors, officers & employees
IP Warrants ~$44m @ $1.00 Share Price (Nov. 3, 2014) Cash Position ~$3m Ownership
Insiders & Affiliates ~35% Institutional ~10%
(May 2014 Financials + August 2014 Financing)
Data source: S&P Capital IQ
23"
Pipeline Brain
Transport
Quantity in
the Brain
Localization in
Brain (Cellular) Efficacy
Models
Brain Cancer Herceptin: Breast Cancer
Lysosomal Storage Disease Hunters Syndrome: MPS II Model Sandhoff Disease: Hex B Model siRNA Stroke Model A
ALS Model B
24"
Summary Market Profile
Large Market
Opportunities
Good cash position
Clean share structure
Exit strategy in place
High internal ownership
Transcend
Program
Enhanced technology “The first natural
Greater commercial
carrier to effectively
potential transport therapeutic "
drugs into the brain.”
Ability to extend patent life
Commercialize new therapeutics
and/or revitalize dormant
candidates Contribute to the growth
in CNS drug market
"
Intellectual !
Property & Collaborations Strong patent protection
Patent portfolio of >30 patents Large pharmaceutical
collaborations Positive results; successful demonstration Management
Major success in: Drug commercialization
Biotechnology & venture
capital startups
Academia Treatment Potential
"
Awarded TSX.V Top
50® in 2013 Leader in providing shareholder value
Discovery of MTfp
"
Metabolic diseases
Neurodegenerative
diseases
Brain Cancer 25"