Lou Arcudi, Chief Financial Officer
Noble Financial Capital Market’s Seventh Annual Equity Conference
Clinical
Pipeline
Discovery Translational
Platform Research
+
Nasdaq: IDRA
May 2011
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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Safe Harbor Statement
Any statements that we may make in this presentation about future expectations,
plans and prospects for the Company constitute forward-looking statements for
purposes of the safe harbor provisions under The Private Securities Litigation Reform
Act of 1995. Actual results may differ materially from those indicated by these
forward-looking statements as a result of various important factors, including risks as
to whether results obtained in preclinical studies or early clinical trials will be
indicative of results obtained in future studies or trials; whether products based on
Idera’s technology will advance through the clinical trial process and receive
regulatory approval on a timely basis or at all; whether, if such products receive
approval, they will be successfully distributed and marketed; whether the Company’s
collaborations will result in successful product development and in the Company
receiving payments thereunder; whether the patent and patent applications owned or
licensed by Idera will protect the Company's technology and prevent others from
infringing it; whether Idera's cash resources will be sufficient to fund product
development and other operations; and such other important factors as are set forth
under the caption "Risk Factors" in Idera’s Quarterly Report on Form 10Q for the
three months ended March 31, 2011. Idera disclaims any intention or obligation to
update any forward-looking statements.
© 2011 Idera Pharmaceuticals
www.iderapharma.com
2
Idera: Clinical Stage Development Company
• Diverse clinical-stage pipeline of proprietary and partnered drug
candidates targeting Toll-like receptors (TLRs)
• Clinical data validating scientific rationale in Oncology,
Chronic Hepatitis C Virus Infection, and Autoimmune Diseases
• In 2011, multiple clinical programs in Phase 2
• Entire development portfolio generated by Idera’s chemistry-based TLR
drug discovery capability
• Strong patent position, over 500 patents in portfolio
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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Idera’s Pipeline of Candidates
PRECLINICAL
PHASE 1
PHASE 2
PHASE 3
Oncology
IMO-2055
(EMD 1201081)
• Head and Neck, Second-line
• Other Cancer Indications
COLLABORATION WITH
Hepatitis C
IMO-2125
• Null-responder patients
• Treatment-naïve patients
Autoimmune Diseases
IMO-3100
• Healthy Subjects
• Lupus, Rheumatoid Arthritis, Psoriasis
• Hyperlipidemia
Respiratory Disease
IMO-2134
• Healthy Subjects
Hematological Malignancies
IMO-4200
Vaccine Adjuvants
COLLABORATION WITH
TLR7, 8 and 9 Agonists
• Cancer, Infectious Diseases, Alzheimer’s Disease
TLR3 Agonists
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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Idera’s TLR-Targeted Candidates
TLR Signaling
Chemistry
Drug Candidates
Endosome
Cytoplasm
IMO-2055
IMO-2125
IMO-3100
IMO-2134
IMO-4200
IMO-5001
IMO-2311
IMO-2314
IMO-2295
Nucleus
NK Cells
pDCs
B cells
Macrophages/
Monocytes
mDCs
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-2055: for the Treatment of Cancer
• IMO-2055, a TLR9 agonist, induces
innate and adaptive immune responses
IMO-2055
• IMO-2055 shows anticancer activity in
multiple preclinical tumor models
• IMO-2055 potentiates anticancer
activity of Erbitux® in preclinical tumor
models
• Mechanism of action of IMO-2055
provides a rationale for targeted
immunotherapy of cancer
B cells
pDCs
Th1-type immune responses
NK Cell Activation
Antitumor Activity
Erbitux®
Enhanced antitumor activity
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-2055: Collaboration with Merck KGaA
COLLABORATION WITH
• Entered into collaboration with Merck KGaA in December 2007 for
discovery and development of TLR9 agonists for cancer treatment
• Business terms
•
•
•
•
$40M upfront payments received
Total potential milestones remaining €255M
Commercialization royalties: mid-single digit escalating to low-double digit
R&D costs covered by Merck KGaA
• Milestone Payments Received
• $12M associated with initiation of three trials
• Merck KGaA is conducting clinical trials of IMO-2055 (EMD 1201081) in
combination with other cancer therapy agents including a Phase 2 clinical
trial in head and neck cancer
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-2125: A Novel Immune Modulator
for Treatment of Hepatitis C Virus Infection
• IMO-2125 induces TLR9-mediated
immune responses including:
IMO-2125
• Endogenous antiviral cytokines
• IFN-α, IP-10, 2’,5’-OAS, etc.
• Cellular activation:
• NK cells, monocytes, neutrophils
• Enhances Th1-type immune response
B cells
pDCs
• IMO-2125 provides rationale for novel
immune modulator as an alternative to
recombinant interferon
Th1-type immune responses
Type I Interferons
Cellular Activation
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-2125: Phase 1 Clinical Trials in NullResponder and Treatment-Naïve HCV Patients
• Two Phase 1 trials completed in 118 patients, four-week treatment periods
• Null-responder patients, IMO-2125 monotherapy
• 58 patients, 6 dose regimens
• Treatment-naïve patients, IMO-2125 plus ribavirin
• 60 patients, 4 dose regimens
• Comparative arm: Pegasys® plus ribavirin
• Study endpoints
• Safety and tolerability
• Antiviral activity
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-2125: Safety Profile
• IMO-2125 was well-tolerated in approximately 96 patients
• No discontinuations, no treatment-related SAEs
• Most common AEs in patients receiving IMO-2125 were
• Mild-moderate injection site reactions (e.g., erythema, induration, tenderness)
• Mild-moderate flu-like symptoms (e.g., fever, chills, myalgias)
• Treatment-naïve patients receiving IMO-2125, compared to Pegasys®,
experienced:
•
Shorter duration of flu-like symptoms
•
Limited neutropenia and thrombocytopenia
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-2125: Antiviral Activity
• IMO-2125 induced a broad array of antiviral cytokines and chemokines,
including interferon-alpha in both patient populations
• At all dose levels IMO-2125 induced declines in viral levels at 48 hours
after the first dose in treatment-naïve patients
• IMO-2125 at all dose levels produced substantial viral load reductions
after four weeks of treatment in both patient populations
• IMO-2125 treatment led to AST and ALT levels within normal limits by the
end of the fourth week of treatment in treatment-naïve patients
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-2125: Planned Phase 2 Clinical Trial
• Patient Population and Treatment
• Treatment-naïve HCV-infected patients
• 12-week treatment period
• Treatment with IMO-2125 + ribavirin
• Control arm treated with Pegasys®+ribavirin
• Study endpoints:
• Detailed monitoring of safety and tolerability
• Antiviral activity
• Path forward to be determined after evaluation of data from nonclinical
toxicology studies, which we expect will be available in 2H 2011
Planned Phase 2 trial designed to guide clinical development of
IMO-2125 for use in combination with Direct-Acting Antiviral agents
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-3100: Novel Approach for Treating
Autoimmune Diseases
• TLR7 and TLR9 are implicated in
autoimmune and inflammatory diseases
• IMO-3100 inhibited immune responses
mediated through TLR7 or TLR9
Immune complexes act as
Endogenous Ligands
• IMO-3100 activity confirmed in several
preclinical models of autoimmune
diseases including: lupus, psoriasis,
rheumatoid arthritis and hyperlipidemia
• IMO-3100 inhibits induction of multiple
cytokines and provides a novel
approach for the treatment of
autoimmune diseases
IMO-3100
B cells
pDCs
X X
IFN-α, IP-10, IL-12, IL-6, TNF-α,
IL-1β, Anti-RNP, Anti-ds DNA
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-3100: Safety and Mechanism of Action in
Clinical Studies
• Two Phase 1 clinical trials completed in 60 healthy subjects
• Single-dose study
• 36 healthy subjects, 5 dose levels, placebo controlled
• Multiple-dose study for four weeks
• 24 healthy subjects, two dose cohorts, placebo controlled
• Study endpoints:
• Safety
• IMO-3100 well tolerated at all dose levels
• Mechanism of action
• Engagement of TLR7 & TLR9 confirmed in MO-3100-treated subjects through
suppression of TNF-α, IL1β, IL-6, IFN-α and other cytokines
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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IMO-3100: Planning for Phase 2 Trial
• Anticipated completion of ongoing nonclinical safety studies 1H 2011
• Submission of Phase 2 protocol expected 3Q 2011
• Initiation of Phase 2 anticipated by year-end 2011
• Study design anticipated to establish safety and activity of IMO-3100 over
12 weeks of treatment in a selected autoimmune disease indication
IMO-3100 inhibits induction of multiple cytokines
including TNF-α, IL-6, IL-1β, IP-10 and INF-α and provides
a novel approach for the treatment of autoimmune diseases
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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Pipeline of Clinical and Preclinical
Candidates
• Clinical Candidate: IMO-2134
• TLR9 agonist for the treatment of respiratory diseases
• Phase 1 trial conducted in healthy subjects by Novartis
• Idera regained rights to this program from Novartis in 2010
• Lead Candidate: IMO-4200
• Dual TLR7 and 8 agonist for the treatment of hematological malignancies
• In preclinical lymphoma models, antitumor activity and potentiation of activity of
Rituxan® and Velcade®
• Novel Discoveries: TLR3 agonist compounds
• Use as vaccine adjuvants being studied in infectious diseases and other
indications
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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TLR Agonists: For Use as Vaccine Adjuvants
• Entered into collaboration with Merck & Co. in December 2006 for use of
agonist of TLR7, 8 and 9 as vaccine adjuvants in the field of cancer,
infectious diseases, and Alzheimer’s disease.
• Business terms
• $30M upfront payment
• Up to $425M potential milestone payments depending on vaccines and
agonists employed
• Commercialization royalties: mid to upper single digit
• R&D costs covered by Merck
• Ongoing research
• Multiple scientific presentations by Merck on immunogenicity and adjuvant
activity of TLR agonist in preclinical models
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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Novel Gene-silencing Oligonucleotide
Technology
• Gene-silencing oligonucleotides (GSOs) are novel single-stranded DNA
and RNA structures
• In vitro and in vivo data demonstrate that GSOs:
• Inhibit gene expression with 19-mer and 21-mer having most potent activity
• Engage a similar cellular mechanism as siRNAs
• Achieve systemic delivery without need for enhancement technology
• Provides new platform for target validation and discovery of novel drug
candidates
• Next steps: conduct studies of GSOs targeted to mRNA and miRNA
GSOs may overcome significant obstacles of antisense and
siRNA technologies including stability, delivery and specificity
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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Financial Highlights
1Q 2011
2010 FY
Net Loss
$(6,845)
$(17,963)
Cash, Cash
Equivalents &
Investments
$28,346
$34,643
Net Cash Used in
Operating Activities
$6,300
$19,556
$0
$0
27,615,933
27,595,920
Total Debt
Total Common
Shares Outstanding
Dollars in 000’s
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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2011 Anticipated Milestones
• Advancement of multiple clinical programs
COLLABORATION WITH
• Completion of Phase 2 of IMO-2055 in oncology
• Determine path forward for IMO-2125 in HCV
• Initiation of Phase 2 clinical trial of IMO-3100 in an autoimmune indication
• Advancing the pipeline of candidates
• Outline development strategy of IMO-2134 in respiratory diseases and
IMO-4200 in hematological malignancies
• Vaccine adjuvant application
• Advancement of ongoing preclinical studies
COLLABORATION WITH
• Building alliances
• Assets include IMO-2125, IMO-3100, IMO-4200, and agonists of TLR3, TLR7,
TLR8 and TLR9, and gene-silencing oligonucleotide technology
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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Clinical
Pipeline
Discovery Translational
Platform Research
+
Nasdaq: IDRA
© 2011 Idera Pharmaceuticals
www.iderapharma.com
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