CTI BIOPHARMA CORP - FORM 8-K - EX-99.1

Attached files

file	filename
8-K - FORM 8-K - CTI BIOPHARMA CORP	d789826d8k.htm

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

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

renewed,

that

the

second

Phase

clinical

trial

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

further

reduce

its

operating

expenses,

that

CTI's

average

net

operating

burn

rate

may

increase,

that

CTI

will

continue

need

raise

capital

fund

its

operating

expenses,

but

may

not

able

raise

sufficient

amounts

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.

CTI BioPharma Opportunity

•

approved

therapy

the

E.U.

for

relapsed

aggressive

B-cell

NHL

beyond

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

•

Phase

trial

enrollment

completed;

Top-line

data

1Q-2015

•

Phase

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

provide

adequate

capital

into

3Q-2015

Marketed Oncology Product & Late Stage Pipeline

Therapeutic Focus: Novel Targeted Therapies

Blood-Related Cancers

Lymphoma

Leukemia

MPNs

Aggressive NHL

AML and MDS

Myelofibrosis

•

and only approved mono-therapy in the E.U.

for

aggressive

B-cell

NHL

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

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.

•

Typically Anthracycline-based treatment

•

Anthracyclines

limited

mostly

line

use

due

association

with

irreversible,

cumulative

dose

heart

damage

3,4

•

Intensive non-anthracycline based toxic salvage therapy (R-

DHAP), +/-autologous stem cell transplantation

•

95%

patients

will

relapse

after

-line

therapy

•

Prior options for patients were palliative care or clinical trials

Line

PIXUVRI Highlights

line B-cell Results

Only

randomized

controlled

Phase

trial

-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

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)

10.2 mos.

7.6 mos.

0.251

(HR=0.79)

ITT Population

EXTEND (PIX301) study published in Lancet Oncology, May 2012

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.

salvage

line)

chance

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

•

positive,

should

extend

label

line

combination

therapy

Expected to increase E.U. addressable market by 12,000* patients

Potential to provide basis for U.S. NDA

*Friedberg J 2011 American Society of Hematology.

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

–

30,000

patients

the

U.S.,

~37%

whom

are

thrombocytopenic

with

platelet

counts

below

150,000/µL

Based on Mesa R, ASH 2012 poster

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

Harrison C, et al. JAK Inhibition with Ruxolitinib vs Best Available Therapy for Myelofibrosis. N Eng J Med 2012;366;787-98.

Verstovsek S, European Focus on MPNs & MDS 2014, Tips on using rux in everyday practice.

Fonseca

ASH

2013

Abstract

2833

Ruxolitinib

Discontinuation

Patients with Myelofibrosis: An Analysis from Clinical Practice.

Thrombocytopenia in Myelofibrosis

•

92%

patients

COMFORT

(ruxolitinib)

had

normal

platelet

counts

n=101

n=103

•

Low

dose

(

10mg

bid)

provides

suboptimal

disease/symptom

control

•

Discontinuation

rates

for

ruxolitinib

(60%)

within

the

first

months

Baseline

Platelets

100,000/µL

(n=28)

Baseline

Platelets

100,000/µL

(n=37)

All

(n=65)

Platelet Count (x10

/µl)

Median (IQR)

(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

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

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*

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

•

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

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

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

•

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

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

line elderly

high risk AML/MDS

•

Several ISTs in AML or MDS underway

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

**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

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

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

NASDAQ & MTA: CTIC

THANK YOU!

Attached files

Stock widget

Investing forum

CTI BIOPHARMA CORP Reports