Forward Looking Statement - Corporate-ir

advertisement
Forward Looking Statement
This presentation contains forward-looking statements within the meaning of the “safe harbor” provisions of the
Private Securities Litigation Reform Act of 1995. These statements are based on management’s current
expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results
to differ materially from those described in the forward-looking statements. The forward-looking statements
contained in this presentation include statements about future financial and operating results, and risks and
uncertainties that could affect CTI’s product and products under development. These statements are not
guarantees of future performance, involve certain risks, uncertainties and assumptions that are difficult to predict,
and are based upon assumptions as to future events that may not prove accurate. Therefore, actual outcomes
and results may differ materially from what is expressed herein. In any forward-looking statement in which CTI
expresses an expectation or belief as to future results, such expectation or belief is expressed in good faith and
believed to have a reasonable basis, but there can be no assurance that the statement or expectation or belief will
result or be achieved or accomplished.
The following factors, among others, could cause actual results to differ materially from those described in the
forward-looking statements: risks associated with the closing of the acquisition and with the synergies expected
from the acquisition, as well as risks associated with preclinical, clinical and sales and marketing developments in
the biopharmaceutical industry in general and in particular including, without limitation, the potential failure of
XYOTAX™, pixantrone, and Brostallicin to prove safe and effective for treatment of non-small cell lung and
ovarian cancers, non-Hodgkin’s lymphoma, and sarcoma, respectively, potential determinations by regulatory,
patent and administrative governmental authorities, competitive factors, technological developments, costs of
developing, producing and selling CTI’s products under development; and other economic, business, competitive,
and/or regulatory factors affecting CTI’s business generally, including those set forth in CTI’s filings with the SEC,
including its Annual Report on Form 10-K for its most recent fiscal year and its most recent Quarterly Report on
Form 10-Q, especially in the “Factors Affecting Our Operating Results” and “Management’s Discussion and
Analysis of Financial Condition and Results of Operations” sections, and its Current Reports on Form 8-K. Except
as may be required by Italian law, CTI is under no obligation to (and expressly disclaims any such obligation to)
update or alter its forward-looking statements whether as a result of new information, future events, or otherwise.
2
Why Acquire SMI

CTI gets WW rights to Brostallicin, a phase II/III “first in class”
drug candidate which could reach market by 2010



Fills pipeline gap with late stage drug candidate with low risk
development program
Leverages CTI’s solid tumor sales force under NVS agreement following
potential Xyotax launch in NSCLC in 2009
Synergistic partnership with prestigious Nerviano Medical
Sciences (NMS), Milan


NMS/Farmitalia discovered Anthracyclines first class of DNA MAJOR
groove binding agents
NMS scientists discovered Brostallicin first synthetic class of DNA MINOR
groove binding agents
3
Why Acquire SMI

Establishes partnership with unprecedented team of genomic
scientists and translational clinical trial experts coupled with
pharmaceutical drug development expertise (Dan Von Hoff)



Ideal complement to our Bresso development capabilities




Guide current and future drug development direction
Multiple partnership opportunities with big pharma
Cost effective to SMI’s business plan with ~$10M cost savings/year over stand
alone model
Purchase price attractive; similar to Series A venture investors
Superior terms than potential co-development relationship
Adds biotech expertise to CTI Board

Richard Love ex CEO Ilex Oncology
4
Deal terms summary

$20,000,000 in unregistered CTIC stock


$5,000,000 in cash or stock at CTI’s option upon


5 day average price prior to execution
FDA special protocol agreement to a single pivotal trial for Brostallicin®
$10,000,000 in cash or stock at CTI’s option upon regulatory
approval of Brostallicin®
5
Deal terms summary

SMI to become wholly owned subsidiary of CTI


Unit to focus TGEN relationship on acquiring new drug candidates and
targeting current drug candidates using COV approach to increase
probability of success and shorten time to market
Current management to continue to run operations





Jeff Jacobs, CEO SMI
Tim Williamson, CBO SMI
Dick Love to join CTI’s Board of Directors
Dan Von Hoff to lead CTI’s Strategic Portfolio Development Board
Operations and Brostallicin® development will add <10%
increase in operating expenses to combined company P&L
over next 2 years
6
Overview
SMi’s Competitive Edge
Using their expertise
in clinical trials and functional genomics to:

Define clinical and/or genomic abnormalities that
makes a patient’s tumor susceptible to a specific
therapeutic agent

Implement unique clinical trial designs to obtain
rapid approval of new anticancer agents
(“context of vulnerability”)
8
The Context of Vulnerability
Reducing the risk of drug development…
…by picking the right patients for a given drug
-Increased efficacy
-Faster, less expensive development.
-High value targeted markets
Informing clinical studies with genomic and mechanistic information
9
Brostallicin®
a new class of anti-cancer agents:
minor groove binder
H
N
Br
NH2
NH
O
NH
NH
O
H
N
N
NH
H
N
O
N
O
N
O
N
10
DNA STRUCTURE
MAJOR GROOVE
Proteins that interact with
DNA often make contact
with the edges of the base
pairs that protrude into the
major groove. The chemical
groups on the edges of GC
and AT base pairs that are
available for interaction in
the major & minor grooves
MINOR GROOVE
Examples of effective drugs
which bind to Major Groove
-Anthracyclines
-Camptothecins
Examples of Minor Groove
binding agents
-Brostallicin
-Trabectedin (ET-743)
11
H
N
Br
NH2
NH
Brostallicin Highlights
O
NH
NH
O
H
N
N
O
N
1. Distamycin-analog delivered intravenously.
NH
H
N
O
N
O
N
2. Unique minor groove binder without cumulative bone marrow
toxicity
3. 200 + patients in phase I and early phase II clinical trials,
demonstrated clinical antitumor activity

Patients with soft tissue sarcoma (23 PR/SD in 42 pts)

Head and neck cancer (12 PR/SD in 27 chemo-naïve pts) tissue sarcoma,
non-small cell lung cancer, head and neck cancer

Non-small cell lung cancer (10 PR/SD in 24 platinum resistant pts)
4. Strong World-Wide Patent rights: comp-of-matter, uses,
biomarkers
12
Brostallicin Highlights
H
N
Br
NH2
NH
O
NH
NH
O
H
N
N
NH
H
N
O
N
O
N
O
N
5. Strong candidate to demonstrate proof-of-principle for SMi
context of vulnerability approach

Previous clinical responses and disease stabilization

Preclinical in vitro and in vivo data re:


mismatch repair deficiencies,

GSH/GST,

T12:16,
Unique mechanism of action exploitable with genomic and clinical
selection
13
Context of Vulnerability Planned Trials for
Brostallicin

Tumors screened for T[12:16]





Tumors screened for mismatch repair deficiency


Hereditary non-polyposis colorectal cancer
Tumors screened for high Glutathione levels


Mixoid/liposarcoma
ET-743 shown to have high (51%) ORR in this population
Brostallicin has superior convenience and toxicity profile
Single pivotal trial may be adequate
Platinum resistant Ovarian cancer
Less expensive development, higher probability of success,
faster approval potential
14
EORTC (1) Decision re Brostallicin
in Patients with Sarcoma
EORTC
NMS sponsored Phase II trial in 42 previously treated
patients with Sarcoma:



2 objective responses (rarely seen in patients with sarcoma)
~50% patients stable disease at 3 months; ~23%% stable at 6 months
(“success” is > 40% and 20% respectively for untreated patients)
EORTC investigators decided to pursue continued
development of Brostallicin




Randomized Phase II trial comparing Brostallicin to Doxorubicin in
previously untreated patients
108 patients; tissue will be tested for genomic markers
Trial is underway
(1)
European Organization for the Treatment and Care of Cancer Patients
15
Nerviano Medical Sciences is
the largest pharmaceutical R&D facility in Italy
and one of the largest oncology-focused,
integrated discovery and development companies
in Europe. NervianoMS was incorporated in May
2004 following company re-organisation by
Pfizer.
A total of almost 700 highly skilled and experienced scientists, technicians and managers
are involved in oncology R&D projects from target validation through to clinical phase IIa.
Our aim is to discover and develop innovative medicines for the treatment of cancer and
set up partnerships with the biopharmaceutical industry and the scientific community.
Preclinical and pharmaceutical development services are made available to drug companies
and biotechs worldwide.
NervianoMS is a private company owned by the Congregazione dei Figli dell'Immacolata
Concezione (CFIC). Financial resources and the establishing of long term strategies are
managed by CFIC.
16
A not-for-profit
research institute
focused on using
genomic
technologies to solve
serious medical
problems
Key Strategic Relationship
Translational Genomics Research Institute (TGen)


SMi’s founders are driving forces behind TGen’s world
class scientific and clinical community
Formal Relationship




Master Services Agreement
SMi rights to key Product IP and know-how
TGen-founders equity in SMi and subsequently in CTIC
SMi is strengthened by its access to TGen people and
resources
SMi office/labs located in TGen-Mayo Clinic complex
Scottsdale,AZ.
18
Key Founders and Leadership
Richard Love
Chairman & Founder
Former CEO and Founder ILEX Oncology
Former CEO, Triton Biosciences
Former COO Cancer Therapy Research Center
Former COO Translational Genomics Res Inst (TGen)
BOD Parexel
Daniel Von Hoff, M.D.
Founder and Chief Medical Advisor
Founder ILEX Oncology,
Founder TGen,
CSO US Oncology,
Key role in multiple approved NDA’s including
gemcitibine, irinotecan, campath, clofarabine,
and others
Jeffrey Jacob
CEO & Founder
Founder/Director Critical Path Institute,
Former Sr. V.P. of Research Corporation
Technologies, Inc. (RCT products included
Cis/CarboPlatin (BMS), PSA test (Abbott/others), GMCSF(Shering Plough/Immunex), Cardiolite
(Dupont/Merck)
Steve Weitman, M.D., Ph.D.
Consulting Oncologist & Founder
Former Chief Medical Officer, ILEX Oncology
Led development of clofarabine (ODAC and FDA
approval)
Director, Institute for Drug Development in San
Antonio (Adult and Pediatric Translational
Research)
Tim Williamson
CBO & Founder
30+yrs in pharma industry
Marketing and Business Development
Merck, Boeringer Mannheim,Genentech,
Ilex Oncology
Patrick Shannon, Ph.D.
Sr. Dir. Project Management
Former Sr. Dir OSI Pharmaceuticals
(led Tarceva NDA team)
Former Director Dev ILEX Oncology
Former Dir, Pharma Development Matrix
Pharmaceuticals
19
Founders and Leadership (continued)
Spyro Mousses, Ph.D.
Founder & Chief Scientific Advisor
Director, Pharmaceutical Genomics
Division
Translational Genomics Research
Institute (TGen)
Senior Scientist, National Institutes of
Health: National Human Genome
Research Institute
Robert MacArthur, Ph.D.
Founder, Reg.& Clinical Affairs
Founder, Clinical Horizons Research, Inc.
Director, Research Pharmacy, Columbia
University Medical Center
Director, Phase I Unit, LAB Inc.
Director, Drug Safety and Product Labeling
Sandoz Pharmaceuticals
Consultant, National Institutes of Health, NCI
Chemopreventive Branch
Jeffrey Trent, Ph.D.
Founder and Chairman, SAB
Pres/CSO, Translational Genomics Research Institute
(TGen)
Director Intramural Research, National Institutes of
Health: National Human Genome Research Institute
Multiple Academic/Oncology/Genetics leadership
positions (U Michigan, Johns Hopkins, U. Arizona
20
Overview of SMi Business PlanExpenses 2 year look ahead
Original Plan
Revised
Plan-CTI
GA
$7 MM
~$4 MM
Clinical Studies
$11.5 MM
~$10 MM
Preclinical
Programs
$1 MM
~$1 MM
Laboratory
Support
$4MM
~$3 MM
New Drug
Program
$5MM
CTI internal
Totals
$ 28.5 MM
~18 MM
21
2007 Product Pipeline Strategy

Add additional phase II/III product to pipeline that could
permit a focused selling effort in targeted solid tumor
applications
-
•
Initial indications <20,000 patients annually
Allows utilization of the 35 FTEs under NVS agreement
Allows CTI to back integrate into the larger solid tumor market once
resources and infrastructure permit
“Jump start” commercial effort in blood-related cancer
market
-
Acquire marketed product that has synergies with pixantrone
Re-establish commercial infrastructure in advance of pixantrone launch
Generates awareness of pixantrone trials and indications
Synergies in selling to same target audience
-
Sales training, MSL support, clinical trial support
22
Oncology Pipeline
Proposed additions and target dates
2007
2008
2009
2010
2011
Marketed
Relapsed
FollicularN
HL
Phase III
1st line
DBCL
1st line
Follicular
Phase
III’s
results
Expand
Label1st line
DBCL
Expand
Label
1st line
Follicular
Pixantrone
Launch
DBCL
Follicular
Launch
Year +1
Launch
Year +2
XYOTAX
Launch
Lung
Launch
Ovarian
Launch
Year +2
PRODUCT A
2012
2013
Brostallicin®
Phase IIIII Liposarcoma
Pivotal
Liposarcoma
File
NDA
Launch
Liposarcoma
Launch
Year +1
Bis-platinates
Select
lead
File IND
Start
Phase 1
Start
Phase II
Start
phase III
Phase III
continues
NDA
filing
Launch
Hif-1a
Lead
Optimize
Select
Lead
Start
preclin
Start
IND
studies
File IND
Start
phase I
Start
phase II
Start
Phase
III
Phase III
(cont)
= commercial
2007 Year end targets
•
Marketed product with annual sales ~$15M-$18M
-
•
•
•
•
•
Market expansion phase III studie(s) initiated
Q3 FDA meeting to discuss PIX301 (EXTEND)
analysis and requirements for NDA filing
Pending FDA guidance- report on pixantrone
EXTEND phase III trial results
Completing MAA filing for XYOTAX in EU
Advancing phase III trials of XYOTAX in NSCLC and
ovarian cancer toward results in 1H-09, 2H-09
respectively
Advance Brosatallicin® into phase II/III trial
24
Summary

Phase II cancer product with strong safety and
activity proof of principle: Brostallicin

Adds Wall Street sophistication to CTIC’s
board: Dick Love, of Ilex Fame

Adds top clinical/scientific expertise: Dan VonHoff,
Steve Weitman, Jeff Trent to key consulting and
advisory positions

Advanced technology to improve clinical trial
design, choice of compounds through TGen
Strategic Affiliation and new in-house capability
25
Download