EX-99.1 2 d789826dex991.htm EX-99.1 EX-99.1
CORPORATE PRESENTATION
September 2014
Exhibit 99.1


Forward Looking Statement
This presentation includes forward-looking statements within the meaning of the Safe Harbor provisions of the Private
Securities Litigation Reform Act of 1995.  Such statements are subject to a number of risks and uncertainties, the outcome
of which could materially and/or adversely affect actual future results and the trading price of CTI's securities.  Such
statements
include,
but
are
not
limited
to,
statements
regarding
expectations
with
respect
to
the
following:
the
development
of CTI and its product and product candidate portfolio, including expectations with respect to existing or potential
partnerships; expected receipt and timing of milestone progress payments; market size/potential for our compounds; timing
of PIXUVRI reimbursement and pricing decisions; potential advantages of pacritinib and expansion of pacritinib market
opportunity; timing of our Phase 3 trial for pacritinib and projected availability of trial data; timing of opening of E.U. sites for
PIXUVRI post-marketing commitment study; potential growth of PIXUVRI sales; benefits and results from tosedostat and
our ability to manage expenses and generate non-equity based operating capital; liquidity projections; and our 2014 target
milestones.  Risks that contribute to the uncertain nature of the forward-looking statements include, among others, risks
associated with the biopharmaceutical industry in general and with CTI and its product and product candidate portfolio in
particular including, among others, risks associated with the following: that CTI cannot predict or guarantee the pace or
geography of enrollment of its clinical trials, that CTI cannot predict or guarantee the outcome of preclinical and clinical
studies, that CTI may not obtain reimbursement for PIXUVRI in certain markets in the E.U. as planned, that the conditional
marketing
authorization
for
PIXUVRI
may
not
be
renewed,
that
the
second
Phase
3
clinical
trial
of
pacritinib
will
not
occur
as planned, that CTI may not obtain favorable determinations by other regulatory, patent and administrative governmental
authorities, that CTI may experience delays in the commencement of preclinical and clinical studies, risks related to the
costs of developing, producing and selling PIXUVRI, pacritinib, tosedostat and CTI's other product candidates, and other
risks, including, without limitation, competitive factors, technological developments, costs of developing, producing and
selling PIXUVRI, that CTI's operating expenses continue to exceed its net revenues, that CTI may not be able to sustain its
current
cost
controls
or
further
reduce
its
operating
expenses,
that
CTI's
average
net
operating
burn
rate
may
increase,
that
CTI
will
continue
to
need
to
raise
capital
to
fund
its
operating
expenses,
but
may
not
be
able
to
raise
sufficient
amounts
to
fund its continued operation, as well as other risks listed or described from time to time in CTI's most recent filings with the
Securities and Exchange Commission on Forms 10-K, 10-Q and 8-K.  Except as required by law, CTI does not intend to
update any of the statements in this presentation upon further developments.
2


CTI BioPharma Opportunity
1
st
approved
therapy
in
the
E.U.
for
relapsed
aggressive
B-cell
NHL
beyond
2
nd
line
Partnership
goal:
expand
market
potential,
upfront
&
cost
share;
retain
U.S./E.U.
rights
PIXUVRI
®
: Marketed in 10 E.U. Countries
Pacritinib: JAK2/FLT3 Inhibitor Completing Phase 3
Fast
Track
designated
drug;
Registration
trial
conducted
under
FDA
-
SPA
1
st
Phase
3
trial
enrollment
completed;
Top-line
data
1Q-2015
2
nd
Phase
3
trial
enrollment
completion
estimate:
1H-2015
$360mm collaboration with BAXTER International
Cash-flow Focus
Targeting net positive product margin contribution from PIXUVRI sales 4Q-2014
Generating non-equity based revenue/operating capital through product partnerships
Current cash balance, plus BAXTER upfront/progress payments, potential PIXUVRI
collaboration
upfront
and
projected PIXUVRI
European
sales
expected
to
provide
adequate
capital
into
3Q-2015
Marketed Oncology Product & Late Stage Pipeline 
3


Therapeutic Focus:  Novel Targeted Therapies
Blood-Related Cancers
Lymphoma
Leukemia
MPNs
Aggressive NHL
AML and MDS
Myelofibrosis
4



1
st
and only approved mono-therapy in the E.U.
for
aggressive
B-cell
NHL
3
rd
/4
th
line
Novel MOA: DNA alkylation/mitotic instability
Works in anthracycline-resistant aggressive B-cell NHL
No label warning for cardiotoxicity or cumulative dose
restrictions
Predictable and manageable toxicity (neutropenia)
-
No severe nausea, vomiting or hair loss
PIXUVRI
®
: Overview
One of the most active agents in R/R aggressive
B-cell NHL where there is no standard therapy
6


Relapsed Aggressive NHL: High Unmet Need
~37,000 new cases in the E.U. each year
1,2
1 Harris NL, et al. Ann Oncol.
1999;10(12):1419-32.
2 European Cancer Observatory, Cancer Fact Sheets, 2008.
3 Swain S, Whaley FS, Ewer MS. Cancer
2003;97:2869-79.
4 Mordente A, Meucci E, Silvestrini A et al. Curr Med Chem 2009; 16:1656-72.
5 Hagemeister FB. Cancer Chemother Pharmacol 2002;49(suppl 1):S13-20.
7
Typically Anthracycline-based treatment
Anthracyclines
limited
mostly
to
1
st
line
use
due
to
association
with
irreversible,
cumulative
dose
related
heart
damage
3,4
Intensive non-anthracycline based toxic salvage therapy (R-
DHAP), +/-autologous stem cell transplantation
95%
of
patients
will
relapse
after
2
nd
-line
therapy
5
Prior options for patients were palliative care or clinical trials
1
st
Line
2
nd
Line
3
rd
Line


PIXUVRI Highlights
3
rd
/4
th
line B-cell Results
Only
randomized
controlled
Phase
3
trial
in
3
rd
-line
aggressive NHL (n=140 patients)
PIXUVRI
(n=50)
Comparator
(n=49)
p value
CR/CRu
28%
4.0%
0.002
ORR
48%
12.2%
<0.001
PFS
5.8 mos.
2.8 mos.
0.002
(HR=0.50)
Median
OS
13.9 mos.
7.8 mos.
0.275
(HR=0.76)
PIXUVRI
(n=70)
Comparator
(n=70)
p value
CR/CRu
24%
7.1%
0.009
ORR
40%
14.3%
0.001
PFS
5.3 mos.
2.6 mos.
0.005
(HR=0.60)
OS
10.2 mos.
7.6 mos.
0.251
(HR=0.79)
ITT Population
EXTEND (PIX301) study published in Lancet Oncology, May 2012
8


PIXUVRI: E.U. Commercial Update
Attractive Market Opportunity
Potential >8,000 patients on product at peak in E.U.**
Label expansion study underway (PIX306)
Reimbursement in Key Major Market Countries
Reimbursement/market access in U.K., Germany, France, Italy
NICE: cost effective for NHS in England and Wales
Standard of care per treatment guidelines in key E.U. countries
Weighted average pricing: €16,500 or $20,000/patient/year
*Autologous Stem Cell Transplant
**Market penetration projections provided by Kantar Research.
9
2
nd
salvage
(3
rd
line)
=
2
nd
chance
at
potentially
curative
ASCT*
13,500** addressable market


PIXUVRI: PIX306 Study
Eligibility Criteria
Relapse after CHOP-R
therapy or an equivalent
regimen and are
ineligible for stem cell
transplant
1:1
Randomization
n = ~220
Primary Endpoint
PFS
Secondary
Endpoints
CR   ORR   OS
Gemcitabine*
Rituximab
Pixantrone*
Rituximab
Post-marketing commitment Phase 3 study
Sites currently open in U.S. and E.U.
Target enrollment completion 4Q-2015; data in 2016
If
positive,
should
extend
label
to
2
nd
line
combination
therapy
-
Expected to increase E.U. addressable market by 12,000* patients
-
Potential to provide basis for U.S. NDA
10
*Friedberg J 2011 American Society of Hematology.


Pacritinib


12
Myelofibrosis: A Chronic Disease
Malignant bone marrow disorder triggers an inflammatory response,
scarring the bone marrow and limiting its ability to produce blood cells
prompting the spleen and liver to compensate
Symptoms include: enlargement of the spleen, anemia,
thrombocytopenia, extreme fatigue, pain, severe itching and GI side
effects
Estimated
prevalence
~18,000
30,000
patients
in
the
U.S.,
1
~37%
of
whom
are
thrombocytopenic
with
platelet
counts
below
150,000/µL
2
1
Based on Mesa R, ASH 2012 poster
2
Visani et al. 1990 Br J Haematol; Caramazza et al. 2011 Leukemia; Tam et al. 2009 JCO
<50,000/µL
>50,000/µL but <100,000/µL
>100,000/µL but <150,000/µL
>150,000/µL
10%
11%
16%
63%


Clinical trials of JAK inhibitors before PERSIST excluded
patients with platelet counts <100,000/µL or <50,000/µL
Treatment emergent thrombocytopenia is a limitation of
most JAK1/JAK2 inhibitors (ruxolitinib)
Spleen Volume
Total Symptom Score
1
Harrison C, et al. JAK Inhibition with Ruxolitinib vs Best Available Therapy for Myelofibrosis. N Eng J Med 2012;366;787-98.
2
Verstovsek S, European Focus on MPNs & MDS 2014, Tips on using rux in everyday practice.
3
Fonseca
E,
ASH
2013
Abstract
2833
Ruxolitinib
Discontinuation
in
Patients with Myelofibrosis: An Analysis from Clinical Practice.
Thrombocytopenia in Myelofibrosis
13
92%
patients
in
COMFORT
II
(ruxolitinib)
had
normal
platelet
counts
1
n=101
n=103
Low
dose
(
10mg
bid)
provides
suboptimal
disease/symptom
control
2
Discontinuation
rates
for
ruxolitinib
(60%)
within
the
first
3
months
3


Baseline
Platelets
100,000/µL
(n=28)
Baseline
Platelets
100,000/µL
(n=37)
All
(n=65)
Platelet Count (x10
3
/µl)
Median (IQR)
59
(34-70)
227
(163-315)
121
(60-238)
Range
15-97
104-859
15-859
Duration on Treatment
Median Weeks
(range)*
46.9 (5.7-86.4)
40.9 (1.3-87.4)
44.1 (1.3-87.4)
Komrokji, R; Versovsek, S, et al. ASH 2013 Oral Presentation of Phase 2 Trial
*In the 003 study drug was not provided beyond 12 months
Pacritinib Phase 2: Treatment Duration
Independent of Platelet Counts
14


Endpoint
Time
Period
Baseline
Platelets
100,000/µL
(n=28)
Baseline
Platelets
100,000/µL
(n=37)
All
(n=65)
35%
Reduction
in spleen
volume by
MRI
Up to 24
weeks
7 / 23 (30%)
6 / 26 (23%)
13 / 49
(27%)
Up to last
visit on
treatment
10 / 23 (44%)
8 / 26 (31%)
18 / 49
(37%)
Pacritinib Phase 2: Spleen Response
Independent of Platelet Counts
15
Komrokji, R; Versovsek, S, et al. ASH 2013 Oral Presentation of Phase 2 Trial


50% Reduction In Patient-Reported Symptom
Score Independent of Platelet Counts*
16
Baseline
Platelets
100,000/µL
(n=28)**
Baseline
Platelets
100,000/µL
(n=37)**
All
(n=65)
Up to 24 weeks
11 / 28 (39%)
16 / 34 (47%)
27 / 62 (44%)
Up to last visit on
treatment
13 / 28 (46%)
17 / 34 (50%)
30 / 62 (48%)
* The symptom score is the sum of the individual scores for worst fatigue,
early satiety, abdominal pain or discomfort, night sweats, itching and bone
pain reported on the MFSAF (Mesa et al, Leuk Res 2009, 33(9)1199).
** No significant differences were observed between the two groups
Komrokji, R; Versovsek, S, et al. ASH 2013 Oral Presentation of Phase 2 Trial


Pacritinib: Addressing Unmet Need in MF
Fast Track Designation; Phase 3 Program Underway
PERSIST-1
Topline results expected early 2015
INT-1, 2 High risk, no restrictions on platelet counts
PERSIST-2
Conducted under Special Protocol Assessment (SPA)
Enrollment completion expected 1H-2015
17
Most inclusive randomized trial of MF patient population representative
of actual clinical practice
-
Designed to demonstrate impact of full dose pacritinib over titrated dose
ruxolitinib or BAT in patients with disease or drug related
thrombocytopenia (
100,000/µL)


Sites:
Europe, Australia, New Zealand, Russia and U.S.
Patient Accrual:
Completed
Anticipated Topline Results:
1Q-2015
Principal Investigators:
*Cross-over from BAT allowed after progression or assessment of the primary endpoint.
Eligibility Criteria
No exclusion for
platelet levels,
stratified for platelet
counts of 100,000/µL
and 50,000/µL
No prior treatment
with JAK2 inhibitors
2:1
Randomization*
n = ~320
Primary Endpoint
% of patients achieving
35% reduction in
spleen size from
baseline to Week 24
Pacritinib
Pacritinib: PERSIST-1 Phase 3
18
Best Available
Therapy (BAT)
excluding ruxolitinib
Ruben Mesa, M.D., Mayo Clinic Cancer Center, Arizona
Claire Harrison, M.D., Guy’s Hospital, London


Sites:
Predominantly North America, Europe, Australia, and New Zealand
Anticipated Patient Accrual:
Principal Investigator:
* Cross-over from BAT allowed after progression or assessment of the primary endpoint.
**  BAT may include ruxolitinib at the approved dose per its label.
Pacritinib: PERSIST-2 Phase 3 
19
Co-Primary
Endpoints
% of patients achieving
35% reduction in
spleen volume from
baseline to Week 24
Srdan (Serge) Verstovsek, M.D.
MD Anderson Cancer Center, Texas
1H-2015
Eligibility Criteria
Patients with platelet
counts   100,000/µL,
prior/current JAK2
therapy allowed
1:1:1
Randomization
n = ~300
Best Available
Therapy
(BAT)
**
Pacritinib
400 mg QD   
Pacritinib
200 mg BID  
Patients achieving       
50% reduction in total
symptom score (TSS)
from baseline to   
Week 24
*


Announced November 2013
CTI and BAXTER: Joint commercialization, profit split in U.S.
BAXTER: Exclusive rights to commercialize in all indications outside U.S.,
BAXTER generally responsible for 75% of global development program
Total value: $362mm; potential milestones of up to $127mm through
regulatory submission
-
$60mm upfront payment, includes $30mm equity investment in CTI
-
$67mm potential milestone progress payments expected through 2015
($20mm received in conjunction with completion of PERSIST-1 enrollment)
CTI to receive tiered high single-digit to mid-teen percentage royalties  
based on net sales for myelofibrosis; higher double-digit royalties for all 
other indications
Summary of Financial Terms
Pacritinib: Collaboration with Baxter
20


Oral JAK2/FLT3 inhibitor that lacks treatment emergent
myelosuppression associated with marketed JAK1/JAK2
agent
Potential to demonstrate superior disease and symptom control over
titrated doses of ruxolitinib
May be used in combination regimens w/o overlapping
myelosuppression
Potential for longer duration of therapy than observed with JAK1/JAK2
inhibitors
Durable improvement in splenomegaly (24+ months) and
MF-related symptoms
Unique kinome profile -
potential application in spectrum
of blood-related cancers (AML -
MDS)
Pacritinib: Key Takeaways
21
-
-
-


Tosedostat


Tosedostat
Novel Treatment for Blood-Related Cancers: MDS & AML
Oral, once-daily, aminopeptidase
inhibitor interferes with protein
recycling, prevents breakdown of
peptides into amino acids necessary
for tumor cell survival
Synergy with targeted agents (HMA,
proteosome inhibitors)  or
chemotherapy
Encouraging CR rates (54%) and
median OS (12 months) in combination
with HMA or LDAC in 1
st
line elderly
high risk AML/MDS
Several ISTs in AML or MDS underway
23


Financial Overview
Capital Structure and Financial Statistics
Exchanges
NASDAQ and MTA: CTIC
Market Capitalization*
~$390mm
Shares Outstanding
~149.9mm
Pro Forma Cash as of June 30, 2014**
$53.2mm
Debt (unsecured)
$15mm
*As of September 12, 2014 stock price of $2.61 per share
24
**In August 2014, CTI received a $20mm development milestone payment related to the PERSIST-
1 trial under the agreement with Baxter


2014 Key Business Priorities
Initiate PERSIST-2 (2
nd
Phase 3 trial of pacritinib in MF)
Earn $20mm milestone from BAXTER after completion of enrollment
in PERSIST-1
Be in position to report PERSIST-1 top-line results in 1Q-2015
Secure ROW partner (ex-U.S.) to expand commercial potential for
PIXUVRI in countries where CTI doesn’t have a commercial
presence
Advance PERSIST-2 to complete enrollment in 1H-2015
Generate PIXUVRI E.U. sales to achieve a net positive contribution
margin by year-end 2014
25


Why Invest in CTI BioPharma
Biopharmaceutical company with a marketed oncology drug in the E.U.
Strategic partner provides validation, financial resources and commercial
support for high-potential JAK2/FLT3 inhibitor Phase 3 program
Building value through development of late-stage pipeline, with emphasis in
areas that address unmet medical needs
of patients with blood-related cancers
Commitment to manage expenses within guidance
Stock with good liquidity and potential near-term catalysts
26


NASDAQ & MTA: CTIC
THANK YOU!