EX-99.1 2 d851245dex991.htm EX-99.1 EX-99.1
CORPORATE PRESENTATION
San Francisco –
January 13, 2015
©
Copyright 2015 CTI BioPharma Corp. All rights reserved.
Exhibit 99.1
Conquering
Cancer.
It’s
in
our
blood.


Forward Looking Statement
2
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 BioPharma's securities. Such statements
include, but are not limited to, statements regarding CTI BioPharma’s expectations with respect to the development of CTI
BioPharma and its product and product candidate portfolio, and expectations concerning the benefits of our recently expanded
rights to tosedostat, the anticipated reporting of top-line results from a Phase 2 trial of tosedostat, the development of a
registrational strategy and development program for tosedostat, the anticipated completion of enrollment in PERSIST-2 in mid-
2015, potential release of top-line data from PERSIST-1 late in the first quarter of 2015, the potential submission of a rolling NDA
to the FDA for pacritinib in late 2015, the advancement of a pacritinib development program in other hematologic malignancies in
the second half of 2015, the initiation of a first-line study in elderly patients with AML in the second half of 2015, enrollment
projections and expected outcome of the PIX306 post-marketing commitment study, liquidity, revenue and expense projections,
the anticipated achievement of partner-related milestones and product and geographic sales, initiation of investigator-sponsored
trials, the potential commencement of a pivotal program for tosedostat in early 2016, and the execution of CTI BioPharma’s
strategy to commercialize globally and its strategy to continue the growth in aNHL in 2015. 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 BioPharma and its product and product candidate portfolio in particular including, among others, risks associated
with the following: that CTI BioPharma cannot predict or guarantee the pace or geography of enrollment of its clinical trials, that
CTI BioPharma cannot predict or guarantee the outcome of preclinical and clinical studies, that CTI BioPharma may not obtain
favorable determinations by other regulatory, patent and administrative governmental authorities, that CTI BioPharma may
experience delays in the commencement of preclinical and clinical studies, risks related to the costs of developing pacritinib and
CTI BioPharma’s other product candidates, and other risks, including, without limitation, competitive factors, technological
developments, that CTI BioPharma may not be able to sustain its current cost controls, further reduce its operating expenses or
achieve its anticipated liquidity, revenue and expense projections, that CTI BioPharma may not achieve previously announced
goals and objectives as or when projected, that CTI BioPharma’s average net operating burn rate may increase, that CTI
BioPharma 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.


Accomplishments in 2014
3
Entered into a $133 million* collaboration for PIXUVRI to
expand commercial potential of product
Reported preclinical data highlighting pacritinib’s potential in
other indications beyond myelofibrosis; ISTs in additional
indications underway (AML, CMML, MDS)
Secured worldwide rights to tosedostat and retired
$209mm in potential payments and royalties to Chroma
Grew revenue by 71% to approximately $60mm** reducing
development and capital requirements risk
Advanced pacritinib Phase 3 program in myelofibrosis;
completed 1
st
Phase 3 trial and received $20mm from Baxter
Building Shareholder Value
* Milestone and upfront payments excluding royalties.
** Unaudited 2014 financial results.


2015 Key Objectives
Pacritinib
4
Report top-line results from an investigator-sponsored Phase 2 AML trial
Based
on
clinical
data,
define
registration
directed
strategy
in
AML
Tosedostat
Generate sufficient E.U. sales to achieve a net positive contribution
margin in 2015
Complete enrollment of PIXUVRI post-marketing commitment study
Together with Servier, support the initiation of additional investigator-
sponsored combination studies
Report top-line results from PERSIST-1 Phase 3 MF trial late 1Q-2015
Complete enrollment of PERSIST-2 mid-2015
Begin rolling submission of NDA late 2015
Advance development program in other hematologic malignancies
PIXUVRI


5
Preliminary 2015 Revenue Projections 
Projected range of
$55mm -
$72mm
Preliminary Year-Over-Year Revenue Comparison*
Actual
$-
$10
$20
$30
$40
$50
$60
$70
$80
2013
2014
E*
2015E
*
*This estimate is unaudited, preliminary and may change. CTI BioPharma and its auditors have not completed our normal annual closing and
review procedures for the year ended December 31, 2014, and there can be no assurance that final results for the year ended December 31, 2014
will not differ materially from our estimates, including as a result of annual closing procedures or review adjustments and other developments that
may arise between now and the time the financial results for this period are finalized and other factors described in “Forward Looking Statements”
at the beginning of this presentation and in CTI BioPharma’s most recent filings with the SEC. Investors are cautioned not to rely on preliminary 
financial information.  
   Revenue projections are subject to a number of risks and uncertainties and may change. Actual revenue for 2015 may vary based upon a
number of factors, including the ability to complete and the timing of completion of events triggering milestone payments, the degree of market
acceptance and other factors described in “Forward Looking Statements” at the beginning of this presentation and in CTI BioPharma's most recent
filings with the SEC. Investors are cautioned not to rely on financial projections.  


Strategic Approach to Commercialize Globally
U.S.
Europe
ROW
3 core products focused on overlapping heme/onc
patients, prescribers and payors
*In collaboration with BAXTER in U.S.
6
Develop Full
Commercial
Capabilities
For pacritinib*; PIXUVRI
and tosedostat
Core Markets 
[North Western Europe]
Commercializing PIX
and future products
SERVIER for PIX in
other markets
BAXTER for pacritinib
Collaboration
For PIXUVRI, pacritinib
and tosedostat
Build comprehensive
field force, MSLs
Reimbursement
specialists
Cost-effective
commercial structure
CTILS Medical Affairs
& Reimbursement
specialists
SERVIER for PIXUVRI
BAXTER for pacritinib
Partner for tosedostat
ROW


Building a Successful Oncology Business
Therapeutic Focus:  Novel Targeted Therapies
7
Lymphoma
Leukemia
MPNs
NHL and CLL
AML and MDS
MF and PV
Blood-Related Cancers


CTI BioPharma Opportunity
8
*Under the license and collaboration agreement with Servier, monetary amounts are generally denoted in euros, while
the corresponding U.S. dollar amounts are based upon conversion on September 12, 2014.
pacritinib
tosedostat
High (>50%) CR rates in elderly AML, MDS in combination with HMA
or LDAC
Acquired worldwide rights, extinguished potential $209 million obligations
Targeting registration directed Phase 3 trial in early 2016
Fast Track designated product candidate
Phase 3 top-line data expected late in 1Q’15; Second trial enrolling, FDA-SPA
Data beyond myelofibrosis expected in 2015
Collaboration with BAXTER International of up to ~$360 million
1st approved therapy in the E.U. for relapsed aggressive B-cell NHL beyond 2
nd
line; ongoing confirmatory Phase 3 trial
Targeting cash flow positive operations in 2015
Collaboration with SERVIER of up to ~$133* million


Pacritinib


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 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%
10


Pacritinib: Market Opportunity in MF
Approved drug Jakafi
®
/Jakavi
®
(ruxolitinib)
Total 2014 U.S. net sales estimated at $350-360mm
1;
$600mm -
$1bn 2015
run-rate worldwide
Treatment emergent thrombocytopenia and anemia are limitations of most
JAK1/JAK2 inhibitors, including ruxolitinib
Market research estimates 1/3 of patients do not respond adequately to
ruxolitinib; dose adjustments used to “manage the drug”
Low dose (
10mg bid) provides minimal spleen reduction/symptom control
2
If ruxolitinib therapy is interrupted, symptoms return within 7 days
2
>50% of U.S. patients on low dose (5-10mg)
Average time on drug 6-8 months at doses
20 mg/day
3
Minimal activity JAK/STAT pathway at lower doses
Survival correlates with spleen response
Ideal product profile for new MF market entrant would include the following:
Efficacy not dose-limited by tolerability concerns
Ability to treat entire spectrum of patients with MF
Ability to maintain dose over a long period of time
Demonstrated long-term symptom control and splenomegaly control
No overlapping myelosuppression with other MF directed targeted agents
11
1 Incyte 3Q’14 financial results conference call
2 Verstovsek
S,
European
Focus
on
MPNs
&
MDS
2014,
Tips
on
using
rux
in
everyday
practice.
3 IMS data.


Evaluating Pacritinib in All MF Patients
12
Fast Track Designation; Global Registration Program
PERSIST-1
compares pacritinib to BAT, excludes ruxolitinib
Fully enrolled; Top-line results expected late in 1Q-2015
Most inclusive randomized Phase 3 study
All MF
Patients
PERSIST-2
designed to demonstrate full dose pacritinib
potential superior symptom and disease control over titrated
dose ruxolitinib or other BAT in patients with disease or drug
related thrombocytopenia (
100,000/µL)
Conducted under Special Protocol Assessment (SPA)
Enrollment completion expected in mid-2015
MF Patients with
Low Platelets
Pacritinib Phase 3 trials incorporate FDA and EMA accepted endpoint
We believe the design is comparable to Incyte’s COMFORT II with the exception of inclusion
INT-1, no exclusion on low platelet counts and primary endpoints at 24 weeks
INT-1, 2 High risk, no restrictions on platelet counts


PERSIST-1 Representative of MF Population
13
1 Visani G
et al. Br J Haematol 1990;75:4.
2 Caramazza D et al. Leukemia 2011;25:82.
3 Tam C et al. J Clin Oncol 2009;27:5587.
4 Based on blinded data.
The duration of therapy is comparable across patient subsets with thrombocytopenia at
baseline or patients with normal platelet counts at baseline.
Distribution (%) Platelets
in MF Patients
Projected Distribution (%)
Platelets in MF Patient in
PERSIST-1
4
63
11
10
16
>150,000/µL
100
-
150,000/µL
50-100,000/µL
<50,000/µL
>150,000/µL
100
-150,000/µL
50-100,000/µL
<50,000/µL
54
12
15
19
1-3


14
March 2014:
Open
enrollment for
PERSIST-2
March 2015:
Expect to report
PERSIST-1 top-line
results
PERSIST Program Phase 3 Trial -
Timeline
June 2012:
Acquired
pacritinib
from
S*BIO for
$30mm
January
2013:
PERSIST-1
trial initiated
October 2013:
Reached
agreement with
FDA on SPA for
PERSIST-2
November 2013:
BAXTER
collaboration
460mm upfront
July 2014:
PERSIST-1
enrollment
complete;
$20mm
milestone


Pacritinib: Unique Kinome Profile
1
15
Pacritinib is an oral multikinase
inhibitor with specificity for kinases
in several unrelated families
The inhibition of IRAK1 and c-fms
(CSF1R) may contribute to its
anti-inflammatory activity and
early onset of symptomatic benefit
in MF
Suggests its potential therapeutic
utility in AML, MDS and,
particularly CMML, CLL due to its
potent inhibition of c-fms, IRAK1,
JAK2 and FLT3
1 Singer J, et al., Abstract #1874 American Society of Hematology Annual Meeting 2014.


Pacritinib: AML Target Indication
Able to target multiple pathways, including
suppression of microenvironmental tumor
interactions that are key to overcoming
longer-term drug resistance in this disease
FLT3 strong prognostic factor for AML;
presents in 33% of patients
1
Pacritinib also inhibits JAK2, which is
independently associated with resistance to
FLT3 inhibition and a poor prognosis in AML
2
Phase 2 trial in patients with relapsed AML
and FLT3 mutations is currently underway; a
first-line study in elderly patients with AML is
expected to be initiated in 2H-2015
16
1 Brunet S (2012) Impact of FLT3 Internal Tandem Duplication on the Outcome of Related and Unrelated Hematopoietic
Transplantation for Adult Acute Myeloid Leukemia in First Remission: A Retrospective Analysis. J Clin Oncol 30(7):735-41.
2 Hart S et al., Pacritinib (SB1518), a JAK2/FLT3 inhibitor for the treatment of acute myeloid leukemia. Blood Cancer Journal
(2011) 1, e44; doi:10.1038/bcj.2011.43.
Signaling pathways initiated by FLT3
ligand activation of the FLT3 receptor


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 with potential application in a spectrum of blood-
related cancers
Pacritinib: Key Takeaways
17
Patent protection through 2026 / 2029 (+ possible patent term extension)
Orphan drug designation in the U.S. and E.U.


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
Potential 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
Announced November 2013
18


PIXUVRI®


PIXUVRI: Fulfilling An Unmet Clinical Need
20
1
st
LINE
2
nd
LINE
3
rd
LINE+
Typically
anthracycline-
based treatment
R-CHOP
Curative in 50% -
55% of patients
1
Cardiac toxicity
prevents re-use of
anthracyclines in
relapsed patients
Intensive (toxic)
non-anthracycline
based salvage
therapy (R-DHAP),
+/-
ASCT
Also, ESHAP, ICE
are commonly
used
2
95% of patients
will relapse after
2
nd
-line therapy
3
Palliative care
(symptom control)
or clinical trials
1 Adult Non-Hodgkin Lymphoma Treatment (PDQ®), Aggressive DLBCL, National Cancer Institute. Available at
www.cancer.gov. Last accessed January 2015.
2 Papadatos-Pastos D, et al. Expert Rev. Hematol. 2013; 6(1), 25-33.
3 Hagemeister FB. Cancer Chemother Pharmacol 2002;49(suppl 1):S13-20.


Novel MOA: DNA alkylation/mitotic instability
Works in anthracycline-resistant aggressive B-cell NHL
No label warning for cardiotoxicity or cumulative dose restrictions
PIXUVRI: Overview
One of the most active agents in R/R aggressive B-cell
NHL where there was no standard therapy
21
1 Beeharry N, et al., AACR 2013.
PIXUVRI appears to be impairing chromosomal segregation during mitosis, thereby
generating loss of genetic material in daughter cells, an abnormality, which is ultimately lethal,
and
that
PIXUVRI
would
likely
be
effective
in
cells
resistant
to
other
cytotoxic
agents
such
as
doxorubicin.
1


PIXUVRI: Demonstrated Benefit in Phase 3
Patient Population
Heavily pre-treated patients; median of 3 prior treatments
Median age was 60 years
Safety profile
Predictable and manageable side effects; uncomplicated neutropenia
-
No severe nausea, vomiting or hair loss
Administration
Pixantrone (50 mg/m
2
iv.) weekly infusion on days 1, 8 and 15 of a 28-day cycle, for
maximum of 6 cycles on an outpatient basis
22
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
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)
OS
13.9 mos.
7.8 mos.
0.275
(HR=0.76)
3
rd
/4
th
Line B-cell Results
PIX301* -
Only randomized controlled Phase 3 trial in
3
rd
-line aggressive
B-cell NHL (n=140 patients)
*
PIX301
(EXTEND
Study)
published
in  Lancet
Oncology,
May
2012.


PIXUVRI: Approved as Monotherapy by EMA
1
st
and only approved monotherapy in the
E.U. for aggressive B-cell NHL 3
rd
/4
th
line
2
nd
salvage (3
rd
line) = 2
nd
chance at
potentially curative ASCT*
Durable remissions and disease control
(3
rd
/4
th
line) in non-SCT eligible
Label expansion study underway
with potential as basis for filing U.S. NDA
Prospective investigator-sponsored studies
underway evaluating potential in
combination-salvage regimens for various
hematological malignancies
23
*Autologous Stem Cell Transplant


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, could 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
24
*Based on Kantar forecast model.


PIXUVRI EU Sales Since Launch
25
Net PIXUVRI Sales by Quarter ($ in millions)
2014
2013
Germany
UK
Netherlands
Focus on Reimbursement
0
0.5
1
1.5
2
2.5
Q2
Q3
Q4
Q1
Q2
Q3


PIXUVRI: Maximizing the European
Commercial Opportunity
Exclusive license and
collaboration agreement with
SERVIER to develop and expand
commercial reach
Reimbursed in Austria,
England/Wales, Germany,
Netherlands
Standard of care per treatment
guidelines in certain E.U.
countries
Weighted average pricing:
€16,500 or $20,000/patient/year
26
10+
Countries
~5,000
Current eligible
patients


PIXUVRI Collaboration with SERVIER
Announced September 2014
Exclusive license and collaboration agreement to develop and expand
commercial reach of PIXUVRI
CTI retains 100% U.S. rights, continues its commercial presence in UK,
Germany/Austria, Nordics, and via distribution partners in Turkey, Israel
SERVIER gains rights in ROW
27
Potential deal value: ~$132mm
~$18mm upfront
~$63mm in potential clinical/regulatory milestone payments
-
~$12mm tied to PIX306 Phase 3 trial enrollment
~$52mm in potential sales milestone payments
CTI receives tiered royalty payments: low double-digit to mid-twenties
on net sales in SERVIER’s territories
Summary of Financial Terms


Continued Growth Expected in aNHL in 2015
Continue to build awareness and educate HCPs
Expand field force; medical science liaisons key
Growing body of evidence on potential for combination
studies
Limited near-term competition expected
28


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 cytarabine in 1
st
line
elderly high risk AML/MDS
Several Phase 2 ISTs in AML or MDS
underway
30


Financial Overview
Capital Structure and Financial Statistics
Exchanges
NASDAQ and MTA: CTIC
Market Capitalization*
~$417mm
Shares Outstanding**
~177mm
Cash as of November 30, 2014
$82mm
Debt
$20mm
*As of January 9, 2015 stock price of $2.36 per share.
** As of December 15, 2014.
31


Why Invest in CTI BioPharma?
32
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 high potential near-term catalysts


NASDAQ & MTA: CTIC
THANK YOU!