1. A
New
Drug
to
Restore
Func1on
A2er
Spinal
Cord
Injury
Team
15
A.
Taylor
Bright,
C-‐Level
Officer
Travis
L.
SDles,
PI
Total
in-‐person
interviews
to-‐date
(total):
89
(112)
Weekly
Interview
Average:
11.2
Shawn
Gahr,
Industry
Expert
December
10,
2014
2. Oh,
how
naive
we
were....
2
• Quickly
validated
the
underlying
paDent
need
– 15,000
new
cases
of
SCI
in
US
each
year
– NO
drug
approved
to
treat
SCI
– Pa1ents
and
Physicians
clamoring
for
any
drug
to
help
those
afflicted
• IdenDfied
very
large
unmet
market
with
great
revenue
potenDal
$50B
$3B
The
customers
of
our
customers
are
demanding
our
solu1on!
This
will
be
easy!!!
3. BMC
–
Where
We
Started
3
Key
Partners
Key
AcDviDes
Value
Props.
Customer
Rel.
Customer
Seg.
Key
Resources
Channels
Cost
Structure
Revenue
Streams
University
FoundaDons
NIH
CRO
Venture
Phil.
Sci.
Comm.
IP
for
unmet
need
Fast
Track
candidate
Block
RhoA
Admin.
via
IV
Sanofi
J&J
Genentech
Amgen
NovarDs
Merck
Raptor
BioAxone
Drug
ProducDon
Data
ProducDon
IP
Acquire
KOL
IP
assets
Data
ScienDfic
Team
Direct
Sales
ScienDsts
Lab
Space
Animal
Studies
IP
DivesDture
License
Fees
4. BMC
–
Right
Side
4
Value
Props.
Customer
Rel.
Customer
Seg.
Channels
Revenue
Streams
IP
for
unmet
medical
need
FDA
Fast
Track
candidate
AdministraDon
via
IV
Target
validated
human
biomarker
(RhoA)
Sanofi
J&J
Genentech
Amgen
NovarDs
Merck
Raptor
BioAxone
Direct
Sales
DivesDture
License
Fees
5. Customer
Delivery
Diagram
5
~18
MONTHS
FROM
CUSTOMER
AWARENESS
TO
PROVE
PRE-‐CLINICAL
RELEVANCE
Pharma/Biotech
Addresses
unmet
market
need
“signal
of
efficacy”
Strong
FDA
Fast
Track
candidate
PharmaceuDcal
Company
IP
&
Data
Novoron
Bioscience’s
Value
ProposiDons
Channel
Customer
Segment
Intravenous
administraDon
6. What
We
Did:
Interviewed
Customers
6
Development
Scouts:
8
BD
Execs:
4
Senior
ScienDst:
14
Biologic
Dev
Execs:
7
7. What
We
Learned
7
“We
do
not
believe
there
is
a
valid
pre-‐clinical
model
for
SCI
or
stroke.
No
pre-‐clinical
data
will
convince
Genentech
to
buy
early-‐stage
SCI
drug.
Would
look
at
data
a2er
a
successful
Phase
2
Clinical
Trial.”
-‐
Senior
ScienDst,
Genentech
“We
are
not
interested
in
SCI
but
are
interested
in
{AD,
MS,
etc.}.”
-‐
Senior
ScienDst,
Amgen;
BD
Regional
Director,
Sanofi
“No
one
[Big
Pharma/Biotech]
will
partner
early
stage
in
SCI.”
-‐
CEO,
BioAxone
Bioscience
“We
are
not
interested
in
early-‐stage
SCI
drugs.”
-‐
Senior
Director
of
New
Ventures,
J&J
8. BMC
–
Right
Side
8
Value
Props.
Customer
Rel.
Customer
Seg.
Channels
Revenue
Streams
IP
for
unmet
medical
need
FDA
Fast
Track
candidate
AdministraDon
via
IV
Block
RhoA
acDvaDon
in
damaged
neurons
Next
SCI
clinical
candidate
SCI
PoC
candidate
Sanofi
J&J
Genentech
Amgen
NovarDs
Merck
Raptor
–
Poten1al
Partner
BioAxone
Direct
Sales
DivesDture
License
Fees
9. BMC
–
LeK
Side
9
Key
Partners
Key
AcDviDes
Value
Props.
Drug
ProducDon
Data
ProducDon
IP
Acquire
KOL
Key
Resources
Cost
Structures
University
FoundaDons
NIH
Contract
Research
Org.
Venture
Philanthropist
ScienDfic
CommunicaDon
IP
for
unmet
medical
need
FDA
Fast
Track
candidate
AdministraDon
via
IV
Block
RhoA
acDvaDon
in
damaged
neurons
Next
SCI
clinical
candidate
SCI
PoC
candidate
IP
assets
ScienDfic
Team
PoC
in
Animal
Model
ScienDsts
Lab
Space
Animal
Studies
IP
10. Key
Resources
Learning
10
Customer
Interviews:
“Not
only
more
data,
but
more
rigorous,
replicated
data
is
required
for
an
SCI
drug.”
Academic
KOLs:
20
SCI
FoundaDons:
8
“Field
liJered
with
failures
and
no
successes”
-‐
BioAxone
BioSciences,
CEO
11. BePer
Data
Required
11
• Randomized,
double-‐blind
animal
trials
at
mulDple
independent
research
centers.
• Each
trial
must
assess
mulDple
behavioral,
histological,
and
biochemical
outcome
measures.
Outcome
Measures
for
Proof
of
Concept
Study
Gait
and
ambulaDon
(BBB,
gridwalk)
Sensory
(Von
Frey)
Signal
of
efficacy/biochemistry
(AcDvaDon
of
RhoA)
Immune/scar
effects
(histology)
Neuronal
growth
proximal
and
distal
of
lesion
site
(tracing)
Cervical
injury
(forepaw
grip
strength,
pellet
retrieval)
12. BMC
–
LeK
Side
12
Key
Partners
Key
AcDviDes
Value
Props.
Drug
ProducDon
Data
ProducDon
IP
Acquire
KOL
Key
Resources
Cost
Structures
University
FoundaDons
NIH
Contract
Research
Org.
Venture
Philanthropist
ScienDfic
CommunicaDon
IP
for
unmet
medical
need
FDA
Fast
Track
candidate
AdministraDon
via
IV
Block
RhoA
acDvaDon
in
damaged
neurons
Next
SCI
clinical
candidate
SCI
PoC
candidate
Data
PoC
in
Animal
Model
Replicated,
MulD-‐outcome
PoC
in
Man
ScienDsts
Lab
Space
Animal
Studies
IP
13. AcSviSes,
Resources
&
Partners
13
Grant
funding
ScienDfic
team
Lab
faciliDes
Pharma/
Biotech
Drug
producDon
KOL
support
IP
related
acDviDes
acquisiDon
Proof-‐of-‐Principle
data
acquisiDon
Research
InsDtutes
FORE-‐SCI
Specialty
IP
law
firm
–
Biologic
Drugs
SAB/KOL
advisors
NIH/FoundaDons/
Venture
philanthropists
Linle
Dog
CommunicaDons
Brand
ScienDfic
communicaDons
The
Miami
Project
InsDtute
for
Neural
RegeneraDon
$
$
$
Non
Accrued
Expenses
(under
consideraSon)
$
$
$
ACTIVITIES
PARTNERS
$
14. What’s
Next?
14
• 36-‐month
plan
to
develop
our
putaDve
SCI
drug
to
a
stage
where
Big
Pharma/Biotech
would
find
value.
• IdenDfied
financing
mechanisms
to
enable
longer
development
path.
– NIH
SBIR
Phase
2
– NINDS
CREATE
Bio
Discovery/Development
– Private
foundaDons:
Rick
Hansen,
Spinal
Research
• Secured
partners
needed
to
assist
in
more
complex
development
plan.
15. What
We
Thought:
Finance
&
OperaSons
Timeline
15
Refine/Scale
up
producTon
In
vivo
proof
of
concept
0
12
Time
(months)
$1.0
MM
Opportunity
for
license
or
acquisiDon
18
16. We
What
We
Know:
Finance
&
OperaSons
Timeline
16
Refine/Scale
up
producTon
In
vivo
proof
of
concept
0
12
ConfirmaTon
and
non-‐primate
24
36
large
mammal
studies
Time
(months)
IND
Enabling:
ADME/tox
and
CMC
$1.0
MM
$1.6
MM
$5.8
MM
17. What’s
Next?
17
• 36-‐month
plan
to
develop
our
putaDve
SCI
drug
to
a
stage
where
Big
Pharma/Biotech
would
find
value.
• IdenDfied
financing
mechanisms
to
enable
longer
development
path.
– NIH
SBIR
Phase
2
– NINDS
CREATE
Bio
Discovery/Development
– Private
foundaDons:
Rick
Hansen,
Spinal
Research
• Secured
partners
needed
to
assist
in
more
complex
development
plan.
18. What’s
Next?
18
• 36-‐month
plan
to
develop
our
putaDve
SCI
drug
to
a
stage
where
Big
Pharma/Biotech
would
find
value.
• IdenDfied
financing
mechanisms
to
enable
longer
development
path.
– NIH
SBIR
Phase
2
– NINDS
CREATE
Bio
Discovery/Development
– Private
foundaDons:
Rick
Hansen,
Spinal
Reseach
• Secured
partners
needed
to
assist
in
more
complex
development
plan.
19. AddiSonal
Opportunity
19
2014
2014
We
can
cure
SCI!!!
What
about
MS?
Tell
us
about
MS?
I
wonder
if
anyone
around
here
is
talking
about
MS…
Novoron
and
an
MS
therapeuTc?
• SubmiJed
STTR
for
MS
on
December
5th.
• Strong
early
indicaTon
for
MS
from
technology.
• Customer
interviews
indicate
much
stronger
interest
in
MS
therapeuTc.
20. We
are
a
Go
20
Go
with
a
Nominal
Pivot
The
feasibility
data
generated
in
the
Phase
1
grant
will
provide
the
appropriate
technical
founda1on
for
a
Phase
2
applica1on,
AND
we
are
largely
targe1ng
the
customer
segments
that
we
had
originally
an1cipated.
Investment
Readiness
Level:
Valida1on
of
product/market
fit
is
needed
to
get
us
to
level
5.