BIOTRON LIMITED — Investor Presentation 2014

May 26, 2014

==> picture [227 x 36] intentionally omitted <==

Level 2, 66 Hunter Street Sydney NSW 2000 Tel: (61-2) 9300 3344 Fax: (61-2) 9221 6333 E-mail: pnightingale@biotron.com.au Website: www.biotron.com.au

27 May 2014

The Manager Companies ASX Limited 20 Bridge Street Sydney NSW 2000

(26 pages by email)

Dear Madam

PRESENTATION TO INVESTORS

I attach a PowerPoint presentation as presented by Biotron Limited's CEO, Dr Michelle Miller, to investors.

For further information please contact Dr Michelle Miller on (61-2) 9805 0488.

Yours sincerely

==> picture [106 x 67] intentionally omitted <==

Peter J. Nightingale Company Secretary

pjn7742

BIOTRON
LIMITED (ASX:BIT)

Investor
Update May
2014

==> picture [720 x 50] intentionally omitted <==

This
presenta,on
may
contain
forward-­‐looking
statements
with
respect
to
the
financial
condi,on, results
and
business
achievements/performance
of
Biotron
Limited
(ACN
086
399
144)
and
certain of
the
plans
and
objec,ves
of
its
management.
These
statements
are
statements
that
are
not historical
facts.
Words
such
as
“should”,
“expects”,
“an,cipates”,
“es,mates”,
“believes”
or
similar expressions,
as
they
relate
to
Biotron
Limited,
are
intended
to
iden,fy
forward-­‐looking
statements. By
their
nature,
forward-­‐looking
statements
involve
risk
and
uncertainty
because
they
reflect Biotron’s
current
expecta,ons
and
assump,ons
as
to
future
events
and
circumstances
that
may
not prove
accurate.
There
is
no
guarantee
that
the
expected
events,
trends
or
results
will
actually occur.
Any
changes
in
such
assump,ons
or
expecta,ons
could
cause
actual
results
to
differ materially
from
current
expecta,ons.

==> picture [720 x 50] intentionally omitted <==

**Key

Financial
Metrics**

**Shareholder

Register**

	Ticker Code	ASX: BIT		No. shareholders	2100
	Share Price (26 May 2014)	A $0.09		Top 20 shareholder ownership	78.6 million (34.44%)
	Market cap	A $20.4 million		Director/Management ownership	14.6 million (6.40%)
	12 Month Trading Range	A $0.075 – 0.315
	Shares Outstanding	228 million
	Op,ons	5 million (incen,ve op,on plan)
	Cash Posi,on (03/14)	A $2.65mn
	Monthly Burn rate (2013 FY)	A $0.33mn

Slide
1

• HCV
  market
  is
  high
  growth,
  mul,-­‐billion
  dollar
  market

• Treatment
  gaps
  remain

• Phase
  2a/2
  clinical
  trials
  with
  BIT225
  have
  demonstrated
  efficacy

• BIT225
  is
  well
  posi,oned
  to
  fill
  significant
  HCV
  treatment
  gaps

• HCV
  Genotype
  3

• HIV/HCV
  co-­‐infected
  pa,ents

• Cirrho,c
  pa,ents

• Flexibility
  to
  combine
  with
  any
  DAA
  or
  IFN/RBV
  op,ons

• Significant
  value
  already
  created

• Short-­‐term
  key
  value
  inflec,on
  events

• HIV
  provides
  addi,onal
  value
  opportunity

• Significantly
  undervalued
  in
  comparison
  with
  other
  HCV
  companies

Slide
2

• Worldwide
  market
  for
  Hepa,,s
  C
  forecast
  to
  grow
  from
  $4.7bn
  in
  2012
  to
  over
  $18bn
  by
  2018

• 180
  million
  people
  infected
  worldwide
  (3%
  world
  popula,on)

• 5
  million
  pa,ents
  in
  US

• 30
  million
  pa,ents
  in
  China

• New
  drugs
  have
  demonstrated
  significant
  pricing
  power

• Gilead’s
  Solvaldi
  (Sofosbuvir)
  at
  US$84,000
  for
  a
  12
  week
  course

• JNJ’s
  Olysio
  (Simeprevir)
  at
  US$65,000
  for
  a
  12
  week
  course

• Gilead’s
  Solvaldi
  –
  Q1
  2014
  sales
  US$2.3
  bn

• Addi,onal
  new
  drugs
  are
  soon
  to
  enter
  the
  market

• Not
  pan-­‐genotypic

• 12
  weeks
  treatment
  minimum

• S,ll
  room
  for
  new
  players
  such
  as
  BIT225

Slide
3

• Focused
  on
  crea,ng
  shareholder
  value
  with
  clinical
  programs
  for
  lead
  drug,
  BIT225

• Novel
  approach
  to
  trea,ng
  HCV
  and
  HIV
  infec,ons

• Unique
  mechanism
  of
  ac,on

• Significant
  value
  has
  been
  already
  created

  • Completed
    Phase
    2
    clinical
    trials
    have
    demonstrated
    efficacy
    against
    HCV
    and
    HIV

• Ready
  to
  progress
  into
  Phase
  2b
  trials

• Patents
  issued
  and
  pending
  in
  US
  and
  other
  key
  markets

• A
  solid
  package
  of
  data
  will
  support
  a
  licensing/M&A
  transac,on
  for
  late-­‐stage
  clinical
  development

Slide
4

• Leader
  in
  developing
  viroporin
  inhibitors
  for
  the
  treatment
  of
  viral
  infec,ons

• Target
proteins
are
present
in
influenza
(M2),
HIV
(Vpu),
Hep
C
(p7),
Dengue and
West
Nile
(M
protein),
SARS
(E
protein)
and
others

• Rapid
  proprietary
  primary
  screening
  assays
  for
  target
  proteins

• Pipeline
  of
  first-­‐in-­‐class
  small
  molecule
  viroporin
  inhibitors
  for
  key
  markets

Slide
5

INDICATION	COMPOUND	DISCOVERY	PRECLINICAL	PHASE 1	PHASE 2	PHASE 3
Hep C	BIT225
HIV/Hep C	BIT225
HIV	BIT225
Next generaHon - HCV	BIT314
Dengue	Leads

Slide
6

**BIT225

–
First
of
a
New
Class
of
HCV
DAA
Drugs**

ü Novel,
oral,
small
molecule
compound

POLYMERASE/PROTEASE INHIBITORS
e.g. Sofosbuvir/Simeprevir BIT225
-­‐
ASSEMBLY/ BUDDING
INHIBITOR

ü Only
one
of
its
class
(p7
inhibitor)
in
clinical
trials

ü Inhibits
viral
assembly
and
infec,vity

ü Pan-­‐genotype
ac,vity:

ü Ac,ve in
vitro against
all
main
genotypes

• ü Clinically
  ac,ve
  against
  hard-­‐to-­‐treat
  HCV Gen
  1
  (1a
  and
  1b)
  and
  Gen
  3

• ü Also
  ac,ve
  against
  HIV
  hiding
  in
  reservoir
  cells

Slide
7

• Novel
  target
  and
  mechanism
  of
  ac,on

• Target
  difficult
  to
  treat
  popula,ons

• Current
  DAAs
  are
  poor
  in
  certain
  pa,ent
  groups

• Gen
  3
  and
  Cirrho,cs

• Issues
  with
  some
  DAAs
  (e.g.
  protease
  inhibitors)
  in
  HIV/HCV
  co-­‐infecteds

• Poten,ally
  safe
  to
  use
  in
  combina,on
  with
  IFN/RBV,
  new
  DAAs
  and
  HIV
  ART
  regimens

• New
  class
  –
  may
  prevent
  resistance
  to
  other
  DAAs
  in
  combina,on

• High
  barrier
  to
  resistance
  in
  trials
  to
  date

• Strong
  IP

• Available
  for
  licensing

Slide
8

**BIT225

–
DifferenHated
Product
Profile**

Drug Class	HCV Genotype 1	HCV Genotype 3	HIV/HCV co-infected	CirrhoHcs
Interferon & Ribavirin		ü		
NS5A inhibitors	ü	ü	ü
NS5B polymerase inhibitors	ü		ü
NS3/NS4A protease inhibitors	ü		û
BIT225 (p7 inhibitor)	ü	ü	ü	?

• ~~Mul,ple pathways to demonstrate clinically relevant improvements in efcacy~~

• Improved
  SVR
  rates
  in
  difficult-­‐to-­‐treat
  popula,ons

• Shorter
  treatment
  dura,on

• Interferon
  and
  Ribavirin
  (IFN/RBV)
  free
  regimens

• Demonstrate
  3
  months
  safety
  and
  efficacy
  with
  new
  and
  old
  SOC
  in
  HCV
  Gen
  3

• BIT225-­‐008
  Trial
  –
  Gen
  1
  &
  3
  pa,ents
  with
  IFN/RBV
  (Thailand
  –
  in
  progress)

• • BIT225-­‐009
  Trial
  –
  Gen
  3
  pa,ents
  in
  combina,on
  with
  DAA

• RBV
  (USA/EU
  -­‐
  proposed)

Slide
10

• :

• BIT225-­‐001

Phase
1a,
single
dose,
dose
escala,ng
study
in
healthy
volunteers
(48
subjects;
Aust)

• BIT225-­‐003:

Phase
1b,
7-­‐day,
repeat
dose
study
in
HCV+
pa,ents
(35
and
200
mg
BID;
18
subjects;
Aust)

• B ~~IT225-004:~~

• B ~~IT225-005 :~~

• B ~~IT225-006:~~

• B ~~IT225-007:~~

• BIT225-­‐008:

• ~~Phase 2a, 10-day, repeat dose study in HIV+ pa,ents (400 mg BID; 21 subjects; Thailand) Phase 2a, 28-day, repeat dose study in HCV G1 pa,ents in combina,on with PEG/RBV (20~~ 0
  and ~~400 mg BID; 24 pa,ents; Thailand)~~

~~Phase 2, 28-day, repeat dose, open label study in HIV/HCV G1 and 3 co-infected pa,ents~~ in ~~combina,on with PEG/RBV (300 mg BID; 12 pa,ents; Thailand)~~

~~Phase 1, BE/PK study in healthy volunteers, cross-over, single dose comparing capsule formula,on with exis,ng powder (400 mg BID; 12 subjects; Aust)~~

Phase
2,
3
month,
repeat
dose
study
in
HCV+
pa,ents
(G1
&
3)
in
combina,on
with
PEG/RBV ~~(200 mg BID; 60 subjects; Thailand) IN PROGRESS~~

NB
BIT225-­‐002
was
an ex
vivo study
of
BIT225
on
HIV-­‐infected
cells
isolated
from
HIV-­‐posiFve
paFents

• BIT225-­‐001

&
BIT225-­‐003
Trials

• Phase
  1
  studies
  to
  demonstrate
  safety
  and
  iden,fy
  ini,al
  dose
  response

• 35mg
  to
  600mg
  BIT225
  single
  dose
  escala,on
  in
  48
  healthy
  volunteers

• 35mg
  and
  200mg
  BIT225
  BID
  for
  7
  days
  in
  18
  HCV-­‐infected
  pa,ents

• Results
  and
  Conclusions

• BIT225
  was
  well
  tolerated

  • The
    most
    common
    AEs
    were
    nausea,
    vomi,ng
    and
    headaches

  • No
    SAEs

• First
  demonstra,on
  of
  BIT225
  efficacy
  in
  HCV
  pa,ents

Slide
12

• Phase
  2a
  trial

• ~~Double blind, placebo controlled trial in treatment naïve HCV Genotype 1 pa,ents (n=24)~~

• 200mg
  and
  400mg
  BID
  doses
  of
  BIT225
  for
  28
  days
  with
  SOC
  IFN/RBV
  for
  48weeks;
  randomized
  1:1:1

• Results

• BIT225
  demonstrated
  good
  an,viral
  ac,vity
  against
  HCV
  Genotype
  1a
  and
  1b

  • Numerical
    superiority
    at
    all
    ,me
    points
    in
    both
    doses

  • BIT225
    accelerated
    the
    decline
    in
    HCV
    viral
    load
    vs
    SOC
    in
    both
    doses

  • 100%
    receiving
    400mg
    BIT225
    were
    virus-­‐free
    at
    48
    weeks

• BIT225
  was
  well
  tolerated
  with
  IFN/RBV
  where
  most
  common
  AEs
  were
  fever,
  vomi,ng
  and
  headache

• 5
  SAEs
  in
  the
  trial

  • Ø 1
    SAE
    occurred
    2
    days
    awer
    comple,on
    of
    treatment
    and
    symptoms
    resolved
    without
    sequelae

  • Ø 1
    case
    of
    horizontal
    diplopia
    leading
    to
    blurred
    vision;
    pa,ent
    discon,nued;
    symptoms
    resolved
    without
    sequelae

  • Ø 3
    SAEs
    were
    related
    to
    vomi,ng
    and
    ver,go
    during
    week
    1;
    pa,ents
    ceased
    treatment
    for
    1
    to
    5
    days
    un,l
    symptoms resolved
    and
    all
    returned
    and
    completed
    the
    full
    28
    day
    treatment
    with
    no
    further
    issues

Slide
13

**BIT225

-­‐
Clinical
AcHvity
in
BIT225-­‐005**

~~n=8~~		~~Placebo~~ ~~+~~ ~~IFN/RBV~~			~~IFN/RBV~~		~~IFN/RBV~~
n=8		BIT225 200 mg BID + IFN/RBV				EVR		ETVR
					IFN/RBV		IFN/RBV
n=8		BIT225 400 mg BID + IFN/RBV
	Week			4		12		48
	BIT225-005: HCV RNA Response (Baseline Adjusted Median Change)					Median log reducHon	% Complete EVR	% ETVR
		BIT225 treatment window			Treatment	at 35 days	(<50 IU/ml at 12 weeks)	(<50 IU/ml at 48 weeks)
			Placebo
			200 mg 400 mg		400 mg BIT225 + SOC	-4.957	86	100
					200 mg BIT225 + SOC	-4.351	88	88
					Placebo + SOC	-3.649	63	75

Slide
14

• Phase
  2
  trial

• ~~On-going, open label study in HIV/HCV Gen 1 & 3 co-infected pa,ents on stable ART regimens (n=12)~~

• 300mg
  BID
  BIT225
  for
  28
  days
  in
  combina,on
  with
  IFN/RBV,
  with
  a
  7
  day
  lead
  in
  with
  IFN/RBV
  prior
  to
  commencing
  BIT225

• • Results
  (Interim)

• 7
  pa,ents
  achieved
  clearance
  of
  the
  HCV
  virus
  by
  week
  12,
  with
  2
  non-­‐responders
  and
  3
  drop
  outs

  • 3
    drop
    outs
    due
    to
    nausea,
    vomi,ng
    and
    headache
    within
    14
    days
    of
    star,ng
    treatment

  • 2
    non-­‐responders
    were
    both
    Gen
    1a
    pa,ents
    and
    IL28b
    CT/GT
    heterozygous

• 300mg
  BIT225
  BID
  with
  IFN/RBV
  was
  well
  tolerated
  in
  pa,ents
  on
  stable
  ART
  regimens

  • Most
    common
    AEs
    were
    headache,
    hypokalemia
    and
    vomi,ng

  • 1
    SAE
    of
    nausea
    and
    vomi,ng
    in
    a
    pa,ent
    with
    neutropenia;
    withdrew
    from
    study

•

Conclusions

(Interim)

• All
  Gen
  3
  paHents
  on
  remaining
  on
  trial
  have
  HCV
  viral
  loads
  below
  detectable
  levels
  at
  24
  weeks

• HCV
  viral
  load
  reduc,ons
  were
  par,cularly
  rapid
  in
  pa,ents
  with
  HCV
  Gen
  3
  (n=8)

• 300mg
  BID
  of
  BIT225
  generally
  well
  tolerated
  in
  combina,on
  with
  IFN/RBV
  and
  ART
  in
  pa,ents
  co-­‐infected
  with
  HCV
  Gen
  1
  or 3
  and
  HIV-­‐1

Slide
15

**BIT225

-­‐
Clinical
AcHvity
in
BIT225-­‐006
(interim
data)**

==> picture [720 x 334] intentionally omitted <==

----- Start of picture text -----

NB
All
paFents
were
stable
on
ART
before
and
throughout
the
study;
BIT225
300
mg
BID
+
IFN/RBV
–
HCV Genotype 1 n=4
HIV remained undetectable during the treatment period
n=12
IFN/RBV
IFN/RBV
BIT225
300
mg
BID
+
IFN/RBV
–
n=8
ETVR
HCV
Genotype
3
Week
1
5
48
----- End of picture text -----

Slide
16

**HIV

–
Towards
a
Cure**

• Currently,
  pa,ents
  need
  to
  stay
  on
  an,retroviral
  drugs
  (ART)
  to keep
  virus
  levels
  under
  control

• BIT225
  targets
  HIV
  Vpu;
  interferes
  with
  assembly
  and
  budding
  of new
  virions

• Industry
  is
  now
  focused
  on
  developing
  drugs
  to
  eradicate
  or
  cure HIV
  infec,on

• Phase
  2a
  trial
  demonstrated
  BIT225
  can
  reduce
  HIV
  levels
  in macrophage
  cells in
  vivo ,
  paralleling in
  vitro studies

==> picture [273 x 226] intentionally omitted <==

• BIT225
  may
  provide
  addi,onal
  benefit
  in
  HIV/HCV
  co-­‐infected pa,ents
  due
  to
  an,-­‐HIV
  ac,vity

==> picture [302 x 182] intentionally omitted <==

----- Start of picture text -----

In
vitro
Effect
on
HIV
Replica3on
d14
infec,on
&
d21
BIT225
addi,on
200
150
100 +BIT225
50
16 17 19 21 22 23 24 25 26 27 28
Time
(days)
+HIV-­‐1
----- End of picture text -----

Slide
17

• Efficacy

• Pan-­‐genotypic
  ac,vity in
  vitro

• Clinical
  efficacy
  demonstrated
  in
  HCV
  Gen
  1
  and
  Gen
  3
  and
  HCV
  in
  HCV/HIV
  co-­‐infected
  pa,ents

• Accelerated
  decline
  in
  HCV
  viral
  load
  over
  IFN/RBV

•

Safety

• Safety
  profile
  generally
  consistent
  with
  IFN/RBV

• Well
  tolerated
  in
  doses
  up
  to
  400mg
  BID
  for
  28
  days

• Most
  common
  adverse
  events
  are
  nausea,
  vomi,ng
  and
  headache
  within
  the
  first
  7
  days
  of
  treatment

• Next

Steps

• 12
  week
  treatment
  with
  BIT225
  in
  combina,on
  with
  IFN/RBV
  (in
  progress),

• Ini,ate
  12
  week
  study
  of
  BIT225
  with
  newly
  approved
  DAA

• Demonstrate
  benefit
  for
  difficult
  to
  treat
  popula,ons
  such
  as
  the
  HCV
  Gen
  3,
  HCV/HIV
  co-­‐infected
  and
  cirrho,c
  pa,ents

• Partner
  BIT225
  for
  combina,on
  with
  other
  DAAs
  in
  development

Slide
18

**BIT225-­‐008:

Phase
2
HCV
Three-­‐Month
Dosing
Trial**

==> picture [653 x 134] intentionally omitted <==

----- Start of picture text -----

Placebo
+
IFN/RBV
n=20
(HCV
Genotype
1
or
3)
IFN/RBV
ETVR
n=20
BIT225 200 mg BID + IFN/RBV HCV Genotype 1 IFN/RBV
ETVR
BIT225
200
mg
BID
+
IFN/RBV
HCV
Genotype
3
IFN/RBV
n=20
Week
12
24
48
----- End of picture text -----

Design:

• -­‐ Randomized,
  placebo-­‐controlled,
  double-­‐blind
  trial
  (n=60) -­‐ Treatment
  naïve,
  HCV
  gen
  1
  and
  3

Aims:

  - -­‐ Demonstrate

safety
of
BIT225
with
3
months
dosing

  - -­‐ Extend

HCV
gen
3
efficacy
data

• -­‐ 3
  months
  dosing
  with
  BIT225
  in
  combina,on
  with
  IFN/RBV

• -­‐ Using
  new
  capsule
  formula,on

• -­‐ 1.6
  fold
  higher
  blood
  levels
  than
  previous
  formula,on

• -­‐ IN
  PROGRESS
  (Thailand);
  expect
  to
  complete
  recruitment
  mid-­‐2014

Slide
19

**BIT225-­‐009:

HCV
Phase
2
CombinaHon
Trial
with
DAA
(proposed)**

==> picture [651 x 130] intentionally omitted <==

----- Start of picture text -----

Placebo
+
DAA+
RBV
n=25
HCV
Genotype
3
BIT225+DAA+
RBV
n=25
HCV
Genotype
3
Week
4
8
12
----- End of picture text -----

NB
–
Trial
design
and
dosing
is
yet
to
be
finalized
and
is
subject
to
regulatory
approval

Design:

• -­‐ Randomized,
  placebo-­‐controlled,
  double-­‐blind
  trial -­‐ Treatment
  naïve
  Gen
  3

• -­‐ 3
  months
  dosing
  with
  BIT225
  in
  combina,on
  with
  approved direct-­‐ac,ng
  an,viral
  DAA)
  drug

Aims:

• -­‐ Demonstrate
  safety
  of
  BIT225
  with
  3
  months
  dosing

• -­‐ Demonstrate
  efficacy
  of
  BIT225
  in
  combina,on
  with
  DAA

• -­‐ Show
  improvement
  in
  treatment
  of
  difficult
  to
  treat
  popula,on

• -­‐ Using
  new
  capsule
  formula,on

• -­‐ Expected
  to
  commence
  2H14

(Subject
to
funding
and
regulatory
approval)

Slide
20

**PotenHal

to
Create
Further
Value
of
BIT225
Program**

• HCV
  cirrho,c
  pa,ents

• Safety profile
  suggests
  suitability
  for
  this
  hard
  to
  treat
  popula,on
  with
  major
  unmet
  need

• HIV/HCV
  co-­‐infected
  study

• In
  combina,on
  with
  DAA(s)

• Supported
  by
  DDI
  study

• Poten,al
  for
  part-­‐funding
  from
  bodies
  such
  as
  ACTG

• Progress
  next
  genera,on
  BIT314
  through
  IND-­‐enabling
  studies
  into
  first-­‐in-­‐man
  phase
  1
  trial

• Promising
  clean
  preclinical
  profile

• Potent
  an,-­‐HCV
  ac,vity in
  vitro

• Pipeline
  development
  of
  earlier
  stage
  an,viral
  programs,
  including
  Dengue

Slide
21

CONFIDENTIAL

• Novel
  approach
  to
  trea,ng
  Hepa,,s
  C
  and
  HIV
  infec,ons

• Phase
  2
  clinical
  trials
  have
  demonstrated
  efficacy

• Strategically
  posi,oned
  for
  use
  in
  hard-­‐to-­‐treat
  subpopula,ons
  of
  Hepa,,s
  C,
  including

  • Genotype
    3

  • HCV/HIV
    co-­‐infected

  • Cirrho,cs

• Next
  trials
  will
  demonstrate
  3
  months
  safety
  and
  efficacy

  • Will
    assist
    with
    partnering
    within
    the
    DAA
    landscape

• Patents
  issued
  and
  pending
  in
  US
  and
  other
  key
  markets
  covering
  composi,on
  of
  maxer
  and
  use

• Promising
  next
  genera,on
  BIT314
  ready
  to
  progress
  to
  IND-­‐enabling
  studies

• Company
  is
  well
  posi,oned
  to
  axract
  a
  pharma
  partner
  at
  conclusion
  of
  outlined
  clinical
  program
  in
  a
  rela,vely short
  ,meframe

• Significant
  valua,on
  upside
  compared
  to
  other
  HCV
  companies

Slide
24

Dr
Michelle
Miller Managing
Director +61
412
313329 mmiller@biotron.com.au www.biotron.com.au

==> picture [420 x 60] intentionally omitted <==

==> picture [720 x 50] intentionally omitted <==