THERAPEUTIC AREAS AND TECHNOLOGIES

COMBINING OUR STRENGTHS
SHARING OUR SUCCESSES
THERAPEUTIC AREAS
AND TECHNOLOGIES
Areas of Interest
June 2011
Merck is known as MSD outside the United States and Canada.
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C O M B I N I N G
S H A R I N G
2011
O U R
O U R
Dear Colleagues,
Deal Making—A Creative and Flexible Process
I am delighted to invite you to discover more about Merck and our partnership interests.
Our four-step partnership is clear and straightforward. From initial discussions
to signing the deal, through to execution and global commercialization, you’ll
experience flexibility, creativity, and the utmost professionalism.
Whether you are working for a biotechnology company, an academic institution, or a financial
organization, we welcome the opportunity to discuss our shared interests and opportunities
to partner. We are actively seeking new alliances that accelerate the discovery and
development process, improve R&D productivity, and increase the probability of successfully
commercializing novel therapeutics and vaccines.
We understand that you are using your unique strengths to identify innovative new
compounds and technologies. With Merck as your partner, you will find an organization
devoted to you and the rigorous, expeditious development of your discovery.
Inside this book you will find a list of our currently defined therapeutic areas of interest,
including research technologies and biologics. We welcome:
• Novel patented chemical or biological entities and vaccines in discovery and development
• Targets with proof of concept
• Molecules with a defined mechanism of action or testable hypothesis
• Technologies with patent protection that provide a competitive advantage
Step 1
Connecting
With You
Step 2
Understanding
Your Science
Step 3
Doing the
Deal
Step 4
Working
Together
We are interested in compounds that have a large market potential for unmet medical needs.
Late-stage clinical compounds with proven therapeutic value (Phase III-ready or later) are of
particular interest in any therapeutic area.
To begin a discussion about partnering with Merck, contact our scientific licensing expert in
your region. Contact information for our licensing experts can be found in the back of this
book. Or, if you prefer, please visit our Web site at merck.com/licensing.
I’m confident that, together, we can combine our strengths and translate cutting-edge science
into breakthrough medicines.
Sincerely,
David Nicholson, PhD
Senior Vice President and Head
Worldwide Licensing and Knowledge Management
S T R E N G T H S
S U C C E S S E S
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Therapeutic Areas and Technologies
Table of Contents
Atherosclerosis and Cardiovascular Diseases
Biologics . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 23
Lipids / Metabolic Syndrome . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 4
Specialty Hypertension / Cardiovascular . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 5
Thrombosis and Other Areas . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 6
Vaccines . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 24
Diabetes and Endocrinology
Diabetes . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Obesity . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Osteoporosis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Contraception and Fertility . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Other Areas of Women’s Health . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Technologies and Capabilities . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
7
8
8
8
9
9
Infectious Diseases
Antibacterials . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 10
Antifungals . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 11
HIV . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 12
Hepatitis C . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 13
Antivirals – Other Interests . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14
Antiviral and Anti-infective Technologies . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 14
Research & Enabling Technologies
RNA Therapeutics . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Drug Delivery and Formulation . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Translational Models / In Vivo Pharmacology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Biomarkers . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Lead Identification and Screening Assays . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Chemical Synthesis and Purification . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Modeling Tools . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Automated Workflows . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Information Technology / Software . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Analytical Technology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Technology Areas of Interest by Franchise . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
25
26
27
27
27
28
29
30
30
31
32
Neurosciences and Ophthalmology
Migraine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Pain . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Alzheimer’s Disease . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Parkinson’s Disease . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Ophthalmology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
Psychiatric Diseases . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . .
15
15
16
17
17
18
Oncology
Oncology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 19
Technologies and Capabilities . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 20
Respiratory and Immunology
Arthritis and Immune-Based Diseases . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 21
Asthma / COPD / Rhinitis . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22
Urology . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 22
2
•We have aligned our areas of interest
with our franchises, plus new technologies
and biologics.
•Additionally, we will continue to pursue
external licensing opportunities in other
disease areas where clinical proof of
concept exists.
•We will also pursue niche acquisitions and
partnerships in diagnostics and devices
where it complements our pipeline, and
not as a stand-alone business.
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EMBRACING PARTNERSHIPS
Lipids / Metabolic Syndrome
Areas of Interest:
Not Interested in:
• Lipid-lowering therapies
–Agents with effect on LDL / Apo B
–Agents with additional effect on glucose,
triglycerides, blood pressure, body weight,
and / or HDL
–Basic research collaborations on novel targets
• HDL-raising therapies
–Acute HDL therapy for high-risk patients
(eg, HDL infusion)
–Oral compounds, with known mechanism of
action, that increase HDL-C or Apo-A1 levels
and have additional evidence to increase
confidence that such mechanism will reduce
cardiovascular risk
–Basic research collaborations on novel targets
• Fibrates or other PPAR-alpha agonists
•Discovery
–Collaborations for identifying, validating, and
developing novel lipid / metabolic and / or other
emerging targets with pre-clinical or human
genetic validation and high confidence for
reducing CV risk
•Biomarkers
–Methods, biomarkers, or platforms to assess
antiatherogenic properties of HDL or other
lipoproteins
–Biomarkers of patient subtypes
–Acute methods to assess cholesterol transport in
humans (especially reverse cholesterol transport),
including imaging and kinetic models
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Disease­s
specialty Hypertension / Cardiovascular
Specialty hypertension includes hypertensive segments
with high unmet medical need such as systolic HTN,
resistant HTN and HTN associated with obesity,
and diabetes HTN with associated heart failure; and
pulmonary artery HTN
Atherosclerosis and
Cardiovascular Disease­s
Technology / Methods:
Disease­s
Areas of Interest:
Not Interested in:
•Diuretics
• Long-acting vasorelaxants
• Agents that inhibit aldosterone synthesis
and / or action
• Agents that lower blood pressure and have pleiotropic
benefits on end-organ protection (especially cardiac,
renal, and vascular)
• Antihypertensive agents with additional benefits on
other components of metabolic syndrome and other
cardiovascular risk factors
• Agents for pulmonary hypertension
• Heart failure agents that affect structural cardiac
remodeling and have antihypertensive effects
• Basic research collaborations on novel targets in
above areas of interest
• ACE inhibitors, ARBs, CCBs, beta-blockers,
adrenergic agents
•Nutraceuticals
Technology / Methods:
•Discovery
–Animal models of hypertension and end-organ
damage (cardiac, pulmonary vascular, renal) with
strong evidence of translational value
• Biomarkers (clinical and preclinical)
–Technologies for measuring vascular dynamics
(eg, flow, shear stress, vascular compliance) and
intravascular pressure beyond tonometry
–Markers for renal sequelae of HTN (eg, renal
perfusion and renal injury)
–Platform technologies that enable personalized
medicine for HTN
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
In addition to those areas cited at left, we
are particularly interested in agents that treat
heart failure.
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Disease­s
Thrombosis and Other Areas (Outside Lipids and Hypertension)
Areas of Interest:
Not Interested in:
Thrombosis (discovery onward):
• Oral direct thrombin inhibitor for the treatment of
stroke in patients with atrial fibrillation and venous
thromboembolic disorders
• Novel anti-coagulants that offer potential for
improved efficacy and / or reduced bleeding risk as
compared to warfarin, DTIs, Factor Xa inhibitors
• Basic research collaborations on novel targets
• Agents without clinical POC for:
–Acute treatments for MI
–Post-MI therapeutics for myocardial preservation
or perfusion-reperfusion injury
–Vascular inflammation targets
• Treatment of restenosis
Technology / Methods:
• Preclinical models, clinical models, and / or
biomarkers that can be used to differentiate anticoagulant mechanisms based on efficacy and/or
bleeding risk
• Preclinical models, clinical models, and / or
biomarkers that can be used to differentiate antiplatelet mechanisms based on efficacy and / or
bleeding risk
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EMBRACING PARTNERSHIPS
Diabetes and Endocrinology
Diabetes
Areas of Interest:
Not Interested in:
• Any treatments for glucose with risk reduction of
co-morbidities (eg, dyslipidemia, hypertension)
•Novel or best-in-class protein / peptide therapeutics
–Insulin
•Novel “breakthrough” insulins (eg, glucosedependent, liver-targeted) to complement
existing efforts
•Insulin delivered by alternate routes (eg, oral,
buccal, transdermal, nasal) that also offers an
improved clinical profile
–Best-in-class GLP-1 analogs
•Oral therapies: Best-in-class and novel mechanisms
–Non-PPAR insulin sensitizers
–Glucose-dependent insulin secretagogues (GDIS)
–Beta-cell protection or regeneration if also
affects GDIS
–Ability to combine with metformin is a must-have
–Additive / synergistic in combination with
sitagliptin
–Best-in-class SGLT inhibitor
•Microvascular complication treatments with human
clinical efficacy data
–Agents that halt / reverse complications in
preferably >1 target (diabetic nephropathy,
neuropathy, retinopathy)
• Nutraceuticals or natural product mixtures
• Non–glucose-dependent insulin secretagogues
for diabetes
• Cell-based insulin replacement for type 1 diabetes
unless long-term clinical POC data exists
• Compounds with unknown molecular target unless
clinical POC exists
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage development (Phase III-ready and later). In addition
to those areas cited at left, we are particularly
interested in antiarrhythmic agents for atrial
fibrillation, parenteral anti-coagulants, novel
anti-platelet agents.
6
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
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Other Areas of Women’s Health
Areas of Interest:
Not Interested in:
Not Interested in (unless in Phase III or later):
• Completed Ph II data are required for obesity
licensing opportunities
–Must meet registration criteria for obesity
indication with better safety and tolerability vs
current agents
• Best-in-class compounds and novel mechanisms
for weight loss or both weight loss and prevention
of weight gain
–Any effective mechanism, eg, those acting
centrally or peripherally on appetite, satiety, or
metabolic rate, and nutrient absorption inhibitors
–Peptide, protein, or oral small molecule
–Independent effects on comorbidities (blood
pressure, glucose, lipids) desirable
–Mechanisms that are additive or synergistic as
combination therapy
• Nutraceuticals or natural product mixtures
• Compounds with unknown molecular target unless
clinical POC exists
• Compounds, biologics, medications, and / or
treatments in the following areas are outside
strategy unless in Phase III with a differentiated
product profile
–Endometriosis
–Menopausal complaints
–Uterine fibroids
–Female sexual dysfunction
–Dysmenorrhea
Technologies and Capabilities
Areas of Interest:
Osteoporosis
Areas of Interest:
Not Interested in:
• Osteoanabolic agents
–Novel mechanisms with known molecular target
(prefer clinical POC data)
–Novel delivery for well-validated targets
(eg, PTH) with clinical data to demonstrate a
differentiated profile
• Nutraceuticals or natural product mixtures
• Growth hormone or secretagogues for osteoporosis
• ER alpha modulators for osteoporosis
• Classic antiresorptive agents, including
bisphosphonates with improved formulations that
allow less frequent dosing or better tolerability
for osteoporosis
• Compounds with unknown molecular target unless
clinical POC exists
Contraception and Fertility
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Areas of Interest:
Not Interested in:
• Compounds with a differentiated product profile
and with completed Phase IIB data for female
contraception
• Compounds or Biologics with a differentiated
product profile and with completed Phase IIB data
for treatment of female infertility
• Basic research collaborations towards validation
of novel targets for contraception or infertility
treatment
• Male contraception
• Emergency contraception
• Progestagens for luteal support
•Obstetrics
• Alternate delivery of peptides
• Platform technologies for the identification
and validation of novel targets for diabetes to
complement existing efforts
• Imaging agents for monitoring pancreatic
beta-cell mass
• Pharmacogenetics /-genomics, proteomics, or other
for prediction of diabetes, obesity, drug response,
and sub-phenotyping of disease. Clinical validation
data are needed
• Clinical methods for early prediction of long-term
weight loss
• Novel delivery or formulation to support monthly
dosing for odanacatib
• Novel delivery or formulation technologies to
support Merck’s late-stage product candidates for
contraception or treatment of infertility
• Biomarkers for Osteoporosis
–Biomarkers of fracture risk and treatment response
for patient stratification (eg, genetics / genomics)
–Non-invasive biomarkers of bone strength, quality,
and microarchitecture with clinical translation data
(eg, imaging)
–Novel translatable biomarkers of bone changes
• Responders to Cat K inhibitor therapy
• Early read-out of gain in bone mass
•Selective for cortical vs trabecular
bone anabolism
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
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Diseases
EMBRACING PARTNERSHIPS
Infectious Diseases
Antibacterials
Areas of Interest:
Not Interested in:
• Antibacterial agents that can be meaningfully
clinically differentiated from current agents in terms
of safety, efficacy, route of administration, or activity
against resistant bacterial strains. Products /
programs should only be considered if compelling
data exists, including: resistance frequency, an
understanding of the mechanism of action, efficacy
in animal models of infection, and preliminary
toxicology data*
• Topical antibiotics
• Antibodies without clinical POC
• Quinolones (mechanism of action for quinolones is
of interest, but not the structural class)
Areas of highest interest include:
•Broad-spectrum agents that cover problematic
pathogens included on the IDSA ESKAPE list
• Broad-spectrum agents that can cover either intrinsic
or acquired resistance to existing agents. Though
candidates with broad G+ and G- coverage are
preferred, agents that cover a side spectrum of G+
or G- pathogens will be considered
• Narrow spectrum agents are lower priority due
to: (1) the need for rapid, accurate, and affordable
diagnostics to succeed clinically and commercially;
and (2) difficulty in designing, conducting, and
registering clinical trials
• Agents co-dosed with antibacterials to overcome
resistance (eg, beta-lactamase inhibitors) or
enhance spectrum of coverage (eg, synergists).
Note: beta-lactamase inhibitors should have at least
Class A and C coverage, but will be more highly
valued if they can also provide Class B or Class D
coverage as well
• IV-only dosing is acceptable, but IV / PO is preferred
• Novel approaches to C. difficile that address the
recurrence rate
*Criteria that determine meaningful clinical differentiation may vary by region of the world.
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Antifungals
Areas of Interest:
Not Interested in:
• Antifungal agents with compelling data including: an
understanding of the mechanism of action, efficacy
in animal models of infection, and preliminary
toxicology data
–IV (preferably with oral formulation) broadspectrum agents with activity over azoles and
echinocandins against Candida, Aspergillus, and
rare moulds for hospital use
–Aspergillus- or mold-specific small molecule or
mAb (therapeutic and / or prophylactic, clinical
POC required)
• Incremental improvements upon inhibitors of
ergosterol biosynthesis
• Known antifungal structural types unless they
are differentiated from current agents by safety,
efficacy, route of administration, or activity on
resistant strains
• Antibodies without clinical POC
Late-stage opportunities (Phase III-ready
and later):
• Candida, Aspergillus, or rare mould antifungals
(IV, preferably with oral formulation) that can be
meaningfully clinically differentiated from current
treatment options*
• Oral agents for community use active against
Candida and dermatophytes that can be
meaningfully clinically differentiated from current
treatment options*
*Criteria that determine meaningful clinical differentiation may vary by region of the world
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
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Diseases
HIV
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Diseases
Hepatitis C
Areas of Interest:
Not Interested in:
Areas of Interest:
Not Interested in:
•Preclinical:
–Novel agents with compelling data including an
understanding of the mechanism of action, activity
in cell culture, resistance profile, and PK with
particular interest in:
• Inhibitors of viral budding and / or maturation
• Latency targets
• HIV gene expression inhibitors
• Non-nucleoside inhibitors
•Nucleoside reverse transcriptase inhibitors
with differentiated resistance profile to existing
agents, QD (or less frequent, eg, once weekly)
dosing, >1 month tox
• PK enhancers
• Phase I or later in development:
–Integrase inhibitors with new MOA or novel
structural scaffold with QD dosing
–QD protease inhibitors that do not require
ritonavir-boosting
–Non-nucleoside reverse transcriptase inhibitors
dosed QD with a high barrier to resistance /
efficacy against NNRTI (including 2nd-generation
NNRTI)-resistant virus
• Other new mechanisms, eg, host targets
• Late-stage opportunities (Phase III-ready and later):
–Novel or differentiated agents, ideally combinable
in fixed-dose combination with existing in-line
or pipeline assets that are complementary to
in-house MOAs
–Global / regional promotional partnerships
• Agents administered parenterally, including
antibodies
•Preclinical:
–Novel agents with compelling data including an
understanding of the mechanism of action, activity
in HCV replicon, and PK in one species with
particular interest in:
•Highest interest in nucleoside inhibitors with
acceptable safety / toxicology profile
• NS5A inhibitors
• Novel mechanism agents
• PK enhancers
• Phase I or later in development:
–Non-nucleoside inhibitors of NS5B
–Second-generation protease inhibitor with activity
against genotypes 1, 2, and 3, and key mutants
with preclinical safety data and PK suggestive of
QD dosing suitable for development as fixed-dose
combinations
• Late-stage opportunities (Phase III-ready and later):
–Novel or differentiated agents, ideally suitable
for fixed-dose combination with existing in-line
or pipeline assets that are complementary to
in-house MOAs
–Global / regional promotional partnerships
–Cyclophillin inhibitors and other host cell factors
• Parenterally administered interferons
• TLR agonists or activators
• HCV viral entry inhibitors
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
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Areas of Interest:
Not Interested in:
• Preclinical or later in development:
–Novel approaches to HBV with the potential to
cure / eradicate
• Phase IIb or later in development:
–HBV, RSV, CMV, EBV, flu agents with pristine
safety profiles
• Late-stage opportunities (Phase III-ready and later):
–We will continue to consider external licensing
and partnership opportunities for differentiated
products directed at other viral infections in
late-stage development
–Global / regional promotional partnerships
•Agents administered parenterally
Antiviral and Anti-infective Technologies
•Animal or cellular models of HIV latency
•In vitro tissue models for HCV
•Robust cell culture systems for HCV replication
•Biomarkers / noninvasive assay technology for HIV
and HCV with enhanced sensitivity and / or lower
cost; particularly interested in prognostics
•Genotyping and phenotyping technologies
for HCV polymerase and HCV protease
•HCV and specific gene chimera replicons
•PK enhancers
Antifungal and Antibacterial:
•Rapid (<2 hours), point-of-care, pathogen-specific
(ESKAPE organisms), and host-response
diagnostic tests
•Molecular biomarkers of susceptibility
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Neurosciences and Ophthalmology
Migraine
Areas of Interest*:
Not Interested in:
• Novel therapeutic agents which, alone or in
combination, address unmet needs in migraine or
migraine prophylaxis
• Biologics for prophylaxis
• Mechanisms that mimic or complement CGRP
antagonists for acute migraine
•Triptans
• COX-2 inhibitors and NSAIDs
• Synergistic combination approaches
• Serotonin agonists
• New formulations of existing migraine drugs
Tools and Technologies:
• Novel and patented delivery systems (oral, buccal,
injectable, intranasal) for novel low dose molecules
Pain
Areas of Interest*:
Tools and Technologies:
• Novel therapeutic agents for neuropathic or
inflammatory pain in mechanisms or pathways with
human genetic or clinical validation
–Subtype selective sodium channel blockers
–Selective Trk inhibitors
• Novel proprietary targets for neuropathic and
osteoarthritis pain
–With selective compounds showing efficacy and
safety in Humans
• Agents for treatment of postoperative pain showing
efficacy and safety in Humans
• Synergistic combination approaches showing
efficacy and safety in Humans
–Therapies providing improved efficacy or safety as
add-on or part of novel combination therapy
• Anticonvulsant mechanisms of action showing
efficacy and safety in Humans
• Abuse deterrent opioids at or near registration
• Biomarkers and genetic markers for chronic pain
• Glutamate sensors
• Characterized neuropathic pain patient populations
• Alternate formulation (eg, topical) technologies
showing efficacy and safety in Humans
*Biomarkers: An important aspect for all successful neuroscience licensing
submissions is a focus on target engagement, PK / PD, and efficacy and
safety biomarkers to be used in conjunction with the program.
14
N eurosciences
EMBRACING PARTNERSHIPS
Antivirals – Other Interests
Areas of Interest:
Diseases
Not Interested in:
• Undifferentiated reformulations of marketed
products
• NSAIDs and COX-2 inhibitors
• iNOS inhibitors
•Serotonergics
• Opioids delivered by device
• FAAH inhibitors or cannabinoid agonists
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
15
J U N E
2011
N eurosciences
an d
O p htha l mo l ogy
JU NE
N eurosciences
2011
an d
O p htha l mo l ogy
Parkinson’s Disease
Alzheimer’s Disease
Areas of Interest*:
Not Interested in:
Areas of Interest*:
Tools and Technologies:
• Broad interest in agents and novel mechanisms with
potential disease modifying activity
–Agents must have demonstrated preclinical in vivo
activity
–Targets of interest include:
•Tau pathophsyisology targets, e.g. targets
that modifying Tau acetylation, gylcosylation,
ubiquination, etc.
• Broad interest in agents and novel mechanisms with
potential for symptomatic improvement
–Characteristics at least comparable to existing
approved medications
–With clinical POC, or within pathways with
clinical POC
–May be used as mono-therapy or in combination
with current therapies
• Acetylcholinesterase inhibitors
• NMDA antagonists
• AD vaccines
•Antioxidants
• Metal chelators
• Other general neuroprotectants
• Agents and mechanisms for disease modification
in genetically or clinically validated pathways
• Nondopaminergic agents with preclinical or
clinical proof of concept in palliative therapy for
Parkinson’s disease
• Nondopaminergic agents that preclinically or
clinically reduce / eliminate L-dopa-induced
dyskinesias
• Different formulations of approved products that
have demonstrated clinically significant benefits in
efficacy, safety / tolerability, and / or dosing
vs standard of care
• Approaches and biomarkers that may identify
prodromal stages of disease with pre-clinical data
• Animal models of Parkinson’s disease progression
• Pathway biomarkers of neurodegeneration
Tools and Technologies:
• Collections of CSF from healthy, MCI, AD, and
non-AD dementia subjects
• Prognostic, diagnostic, and progression biomarkers
of disease and disease state with clinical validation
–Tau and amyloid imaging agents
–Fluid-based assays for Aβ and tau species (eg,
oligomers) with clinical validity data
• Novel animal models for target evaluation
–Models that develop both plaques and tangles,
neuronal loss, cognitive decline correlated with
age and pathology
• Novel genetic / RNA-based approaches to target
validation or therapeutics
Not Interested in:
• Metal chelators
•Antioxidants
• Other general neuroprotectants
Ophthalmology
Areas of Interest**:
Retinal diseases
•AMD
–Wet AMD therapies showing less-invasive dosing
than Lucentis™ / VEGF-Trap (topical, periocular)
and / or additional efficacy
–Dry AMD therapies to reduce geographic
atrophy progression
• Diabetic retinopathy / diabetic macular edema
–Mechanisms to reduce progression of NPDR in
moderately-to-severely affected patients
–Mechanisms to treat patients with existing vision
loss with superior efficacy and / or favorable
AE profile vs laser and steroids
•Glaucoma
–Nonprostanoid MOAs with efficacy and / or
tolerability ≥ Xalatan™
–Additional properties of interest: trabecular
outflow enhancers, nontopical delivery
formulations, neuroprotective activity
• Anterior segment disease
–Allergic conjunctivitis
–Bacterial conjunctivitis
–Dry eye
–Uveitis
Tools and Technologies:
• Ophthalmic delivery technologies that improve
patient compliance, efficacy and/or tolerability,
especially in a preservative free fashion
The trademarks contained herein are the property of their respective owners.
**We would consider compounds/agents that have established POC in clinical phase 2 or later
Late-Stage Opportunities:
*Biomarkers: An important aspect for all successful neuroscience licensing
submissions is a focus on target engagement, PK / PD, and efficacy and
safety biomarkers to be used in conjunction with the program.
16
*Biomarkers: An important aspect for all successful neuroscience licensing
submissions is a focus on target engagement, PK / PD, and efficacy and
safety biomarkers to be used in conjunction with the program.
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
17
J U N E
2011
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O p htha l mo l ogy
Areas of Interest*:
Not Interested in:
Novel compounds and chemical leads for:
Schizophrenia
• Agents for monotherapy or add-on therapy for
positive, negative, and / or cognitive symptoms
–In novel mechanisms that modulate clinically
validated pathways, or
–In mechanisms with clinical POC that lack the
adverse event profile of atypical antipsychotics
Bipolar, Depression, and Anxiety
• Agents with clinical POC for monotherapy and / or
add-on that are at or near registration
• Monoamine-based atypical antipsychotics
• SSRI / SNRI
• Animal models for psychiatric diseases
• Imaging target engagement tools for novel
mechanisms
ON C OL OGY
2011
EMBRACING PARTNERSHIPS
Psychiatric Diseases
Tools and Technologies:
JU NE
Oncology
Oncology
Areas of Interest:
Late-stage clinical
•Must demonstrate clinical benefits (improvements
in clinical end points) based on results from a
Phase IIB program
•Clinical data in medium to large market solid
tumor indications or hematological malignancies –
synergies with current portfolio is a plus
•Global or regional deals (US, EU, and / or
Asia Pacific)
Early-stage (up to and including clinical POC)
•Agents that have synergy with Merck Oncology
pipeline therapies in clinical development; agents
that have a well-defined responder ID strategy
–Signaling pathways
–DNA damage and repair pathways
–Select others
•Agents that have efficacy in tumors that are resistant
to SOC therapy (preclinical POC data or Ph I data
with a clear registration strategy)
Not Interested in (applies to Early and
Late Stage):
• Preventive care
• Personalized immunotherapy / autologous therapies
• Gene therapy
• Intra-tumoral delivery (except for RNAi delivery)
•Radio-pharmaceuticals
• Autologous vaccines
Late-Stage Opportunities:
*Biomarkers: An important aspect for all successful neuroscience licensing
submissions is a focus on target engagement, PK / PD, and efficacy and
safety biomarkers to be used in conjunction with the program.
18
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
19
J U N E
2011
Technologies and Capabilities
O NC O L O G Y
JU NE
R ES P I R AT OR Y
2011
AN D
I M M UN OL OGY
EMBRACING PARTNERSHIPS
Areas of Interest:
•Biomarkers and diagnostics
–Diagnostic platforms that are ready for clinical
application, including imaging tracers, cell-based,
blood, nucleic acid, and IVD capabilities with
WW distribution to support Merck Oncology
pipeline programs
–Pharmacodynamic and response biomarkers for
PI3K and DNA damage / checkpoint pathways
–Minimally invasive platforms for measuring
tumor biology: circulating tumor cells, circulating
nucleic acids, novel imaging tracers (particularly
those in the PI3K pathway or those used to
assess apoptosis)
•RNAi
–Tumor-targeted delivery systems for systemic
administration of RNAi
•Other
–Robust technology that could predict in vitro
therapeutic index of combination therapies
–Platforms that have high predictive value for
clinical efficacy (eg, primary tumors
passaged in mice)
–Tumor models for assessment of antitumor
activity of tumor immunotherapy programs
–Technology that enables multiplexing of in vivo
biomarker/efficacy studies
–Genome-wide scanning with the ability to
measure copy # variation in tumor samples
in a CLIA-certified lab
–Gene expression from FFPET in a CLIA-certified lab
–Sequencing in a CLIA-certified lab
–Software tools to correlate genomic data with
clinical outcomes for oncology
Respiratory and Immunology
Arthritis and Immune-Based Diseases
Areas of Interest:
•Disease-modifying antirheumatic drugs (DMARDs)
with a strong preference for:
–Mechanisms targeting clinically proven pathways
–Mechanisms effective in patients with an
inadequate response to anti-TNFa therapies
•Th17 pathway modulators
•Cartilage / joint imaging technologies
•Biomarkers that can be used for patient
segmentation
•Disease-modifying drugs for psoriasis, SLE, or
IBD that have achieved POC in Phase II trials
•Disease-modifying osteoarthritis drugs
(DMOADs) with clinical POC and a clearly
defined path to registration
Not Interested in:
• Drugs whose primary clinical indication is the
prevention of graft rejection
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
20
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J U N E
2011
RESPIRATO RY
A N D
IMMU NO L O G Y
B I OL OGI C S
2011
EMBRACING PARTNERSHIPS
Asthma / COPD / Rhinitis
Areas of Interest:
Not Interested in:
• Compounds / biologics for asthma
–Anti-inflammatory agents with novel mechanisms
–Anti-inflammatory agents, additive or
differentiated from a glucocorticoid
–Anti-inflammatory agents that are effective in
glucocorticoid-resistant asthma
–Selective glucocorticoid receptor modulators
–Novel and / or differentiated bronchodilator
mechanisms
–Inhaled compounds including combination
therapies
• Compounds / biologics for COPD with clinical
POC or compelling biological rationale
–Oral or inhaled compounds including
combination therapies
• Disease-modifying compounds for rhinitis,
atopic dermatitis, and allergy with clinical POC
•Technologies
–Biomarkers
–Predictive animal models of asthma
pathophysiology
–Human lung cell / tissue-based systems for
investigating new therapeutic mechanisms
–Translational medicine platforms
–Inhaled technologies / formulations that
provide improved drug delivery / compliance /
patient acceptance
• Acute lung injury
• Antioxidants (ie, direct scavengers)
• Adenosine antagonists (A1, A2, and A3)
• Antihistamine antagonists (H1, H2)
• IL5 antagonists
• Leukotriene receptor antagonists
• VLA4 antagonists
Biologics
BIOLOGICS
Areas of Interest:
Not Interested in:
•Biosimilars
• Novel biologics and biobetters that fit
franchise strategies
• Novel devices for SQ and IV delivery
• Transgenic animal-based or plant-based production
systems for therapeutics
Technologies of Interest:
Urology
22
JU NE
Areas of Interest:
Not Interested in:
• Novel therapies for OAB (Human POC or greater)
• Novel therapies for stress urinary incontinence
(Phase I or greater)
• Novel therapies for BPH (Human POC or greater)
•Biomarkers
–Surrogate biomarkers of bladder dysfunction
–Imaging approaches for indices of bladder
function, including control of bladder function
• Diagnostics to improve specificity of diagnosing
OAB from stress incontinence
• Diagnostics to predict response or nonresponse
to therapy including anticholinergics or other
mechanisms of action
• Anticholinergics for OAB (unless Phase III or later)
•5a reductase inhibitors for BPH
• Alpha-1 adrenergic blockers for BPH
• Fc engineering to enhance / improve effector
functions, half-life
• Targeted delivery technologies that:
–Address / overcome the blood brain barrier
–Glycan mediated targeting
• Bi-specific platforms with promising stability
and manufacturing yield
• Validated antibody-drug conjugate targets
and technology
• Technologies that enhance expression, production,
formulation, stability, and bioavailability
(via SQ admin) of proteins
• Platforms for the identification, generation, and
modification of monoclonal antibodies (mAbs,
Fabs, scFvs) and engineered proteins displaying
pharmaceutical properties
–High throughput screening
• Including function-based screens
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
23
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VA C C I NES
2011
EMBRACING PARTNERSHIPS
Vaccines
JU NE
2011
R ES EA R C H
&
E N AB L I N G
T E C HN OL OGI E S
EMBRACING PARTNERSHIPS
Research & Enabling Technologies
Vaccines
RNA Therapeutics
Areas of Interest:
Tools and Technologies:
Areas of Interest:
• Viral, bacterial, fungal, and parasitic vaccine
candidates in areas of high medical need /
high incidence
–Must have robust pre-clinical immunogenicity
and safety data
–Efficacy in a relevant pre-clinical model where
one exists
–Clinical POC an advantage but not required
–Must be amenable to production in a
platform acceptable for human use and to
scalable production
•HIV
–Novel immunogens that elicit broadly crossneutralizing immunity antibodies
•Influenza
–Universal flu vaccines: Preclinical data
demonstrating broadly cross-reactive HAI and /
or neutralizing antibodies (ie, not just cellular
immunity) and protection from challenge (survival
and weight loss) comparable to seasonally
matched control vaccines
–Seasonal influenza vaccines that have clinical
data demonstrating differentiation from marketed
products, eg, higher HAI and / or neutralizing
antibodies in elderly subjects
• Improvements on existing in-house vaccines,
which would allow for reduced dosing or increased
cross-strain protection
• Novel adjuvants and immunomodulators
–Highly desirable if preclinical efficacy or clinical
POC achieved, but not necessarily required
–Robust preclinical toxicology data demonstrating
acceptable safety profile
• Novel technologies for:
–Antigen selection, discovery, and identification
–Viral vector approaches
–Vaccine administration, eg, dermal or mucosal
delivery of vaccines
–Multiplexed clinical assay platforms
–Production of virus-like particles
–Studying human cellular and B cell responses to
vaccination, including novel human tissue culture
systems and humanized mouse systems
–Enhance thermostability of vaccines during
transport and storage
• Novel cell lines for vaccine or recombinant protein
production, preferably with Phase I safety data
• siRNA delivery platforms for systemic and / or
local administration
–Ability to demonstrate dose-dependent, RNAimediated gene silencing in vivo with at least a
10-fold margin for severe toxicities
–Chemical and / or biological components suitable
for enhancing cellular uptake, intracellular
trafficking, endosomal escape, and cytosolic
release of oligonucleotides
–Chemical and / or biological components capable
of conferring improved biocompatibility of RNA
nanoparticles / formulations
–Targeting ligands (antibodies, peptides, aptamers,
or small molecules) suitable for direct siRNA
conjugation or for nanoparticle, polymer, or
liposome delivery
•Assays
–Novel particle size characterization methods
–Novel colloid surface characterization methods
–Biochemical assays for Ago / RISC binding and
catalytic activity
• siRNA sequence, structure, and modification
–Novel chemistries for:
•Improving resistance to enzymatic and
chemical degradation
•Reducing immunostimulation
•Enhancing Ago2 / RISC incorporation
and potency
•Improving target specificity
–Predictive bioinformatic and molecular models
Not Interested in:
• Biodefense targets
• Products containing thimerosal or unmodified
animal / human components
• Seasonal vaccines (eg, influenza) that are not
clinically differentiated from marketed products
• DNA-based vaccines for infectious diseases without
clinical proof of concept
• Viral vectors based on pox viruses, retroviruses,
adenovirus or adeno-associated viruses
•miRNA
–Novel chemistries to create miRNA mimics and /
or antagonists
–Assays for pharmacodynamic evaluation of
miRNA activity
–Potential therapeutic agents that:
•Reduce miRNA levels in animals and generate
the expected phenotypic effects
•Mimic natural miRNA, reduce mRNA levels,
and have the expected phenotypic effects
• RNA manufacturing
–Advancements in large-scale production of
modified siRNA
–Improved processes for increased quality,
efficiency, and reduced COG
–Novel chemistries
–Universal linker-based solid support for
production of siRNAs
–Robust LC-MS method for determination of
impurity profile of phosphoramidite raw
materials and oligonucleotides (siRNAs)
–Alternative methods to produce siRNA
clinical supplies
• AAV production
–High-titer, high-volume AAV production for
non-clinical applications and POC
Not Interested in:
• Plasmid DNA-based methods for RNA therapies
• Viral delivery methods for RNA therapies
• Aptamers as therapeutics
Late-Stage Opportunities:
We will continue to pursue external licensing
and partnership opportunities for differentiated
products in all disease areas in late-stage
development (Phase III-ready and later).
24
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2011
RESEA RCH
&
EN A BLIN G
TEC H NO L O G I ES
JU NE
2011
R ES EA R C H
Drug Delivery and Formulation
Translational Models / In vivo Pharmacology
Areas of Interest:
Areas of Interest:
• Oral delivery technologies:
–Technologies for delivery of water-insoluble
compounds (especially mitigation of food effect)
–Oral-controlled release technologies to modify
pharmacokinetics (eg, increase Cmax, AUC, and / or
T1/2, reduce peak-to-trough ratio):
• For poorly soluble compounds (<0.1 mg / mL)
•For compounds with narrow absorption windows
(eg, those with poor colonic absorption)
–Ingredients or formulations that can enhance
permeability
–Orally disintegrating tablet (ODT) technology or
film technology with robust in vivo performance
and simple packaging
–Sublingual delivery for fast onset
–Technologies that protect actives from the GI
environment (eg, gastric acid or GI metabolism)
–Novel oral protein / peptide delivery systems
(eg, insulin)
• Inhalation / nasal delivery:
–Delivery systems for small and large molecules
–Novel in situ modeling (eg, lung solubility or IVIVC)
• Injectable delivery, alternative routes of
administration:
–Injection devices (eg, novel and easy to use
self-injection systems, improved liquid / dry
reconstitution technologies)
–Infusion systems (IV and subcutaneous
delivery that facilitate hospital and home
infusion therapies)
–Novel skin-based delivery technologies for high
concentration antibody formulations and large
volumes of liquid (>2 mL)
–Technologies for delivery of water-insoluble
compounds (eg, IV-administered nanosuspensions
or dispersed systems for small molecules
and peptides)
–Systems (including implants) for sustained release
of small and large molecules from one week to
several months, up to years (long-acting implants)
–Vaginal ring and intrauterine delivery
• Back-of-the-eye delivery systems (eg, invasive
and noninvasive, clinically proven technologies
for retinal delivery of small molecules, siRNA* and
trophic factors)
• Novel passive and active transdermal and topical
technologies for small molecule and peptide delivery
• Intelligent delivery systems capable of modulated
delivery (eg, signal or drug concentration);
personalized / feedback control
&
E N AB L I N G
T E C HN OL OGI E S
• In vivo platforms that replicate human metabolism and disease state
• Technologies to assess gene function in tissue-specific manner for MOA and target tissue specificity
• In vivo models with improved assessment of human specific ADME and safety / toxicity
• In vitro platforms that robustly reproduce clinical indications, ADME, and clinical safety / toxicity
• Label free assay / non-invasive in vivo technologies capable of translating from basic to clinical for MOA, drug
distribution, and safety / toxicity
Biomarkers
Areas of Interest:
•Quick-turnaround mid-density expression, genotyping, protein expression platforms
•Technologies that can perform multiplexed biomarker analysis with wide dynamic range
–RNA, proteins, peptides, and / or small molecules
–Animals and / or clinic
–More streamlined with fewer repeats due to dilutions for individual analytes qualified biomarkers
•Identify vendors / academics that have identified and / or discover / validate / qualify biomarkers
–Translation to humans (cell lines → human cells → animals → humans)
–Target engagement, pharmacologic activity, efficacy, and toxicity
–Sources of samples or model systems for biomarker discovery and development
Lead Identification and Screening Assays
Areas of Interest:
• Improved miniaturized high throughput assays for MoA and toxicity assays
–Toxicity Assessment with limited amounts of compounds
–Continuous kinetic cell-based assays of second messenger kinetics (Ca++, cAMP, phosphorylation, Ion
Channels, etc) for determination of On rate, Off rate, desensitization in cellular contest
–Measurements of the GPCR-mediated physiological responses, including heterodimer formation
–Cellular protein-protein interactions
–Technologies for automated siRNA and cDNA screening
• Physiological cellular models and translational ex vivo cellular models
–In vitro / ex vivo / in vivo predictive models
–Improved technologies for primary cell immortalization of disease tissues
–Metabolically competent hepatocyte-like cellular systems
–Improvement on BACMAM vectors for cell transfection
–Disease predictive physiological cellular models
• High throughput imaging and flow cytometry platforms
–Advanced cellular image analysis software
–Imaging-based tools to measure morphological / biomechanical changes in cells
–High throughput flow-based imaging for screening primary stem cells
–3D-imaging tools and analysis software
–Novel fluorophores for measurements of the cellular changes, including structural proteins
See RNA Therapeutics section for complete listing of interests related to
delivery of nucleic acids.
*
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2011
RESEA RCH
&
EN A BLIN G
Chemical Synthesis / Purification
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TEC H NO L O G I ES
JU NE
2011
R ES EA R C H
&
E N AB L I N G
T E C HN OL OGI E S
Modeling Tools
Areas of Interest (Chemical Synthesis):
Areas of Interest (Purification):
Areas of Interest:
• Improved reactions
–Asymmetric cyclopropanation
–Selective functionalization of
heterocyclic compounds
–Nitroreductases as a platform for accessing
chiral aliphatic amines, and anilines
–Greener oxidations
• Method to determine the cost of separations
for scale up reactions
• Enzyme immobilization
• t-Bu cross-coupling
• Automation tools for DOE experiments
• Analysis and purification of lipids
• 14C labeled carbonylation
• N=O chemistry
• Preparation of alkyl fluorides
• C-H oxidations
• Reactions to introduce fluorine and CF3
• Scalable membrane separations
• “One shot” separations system
•ADME models and databases
–Large, diverse datasets for less common metabolic pathways, such as aldehyde oxidase,
sulfotransferases, BCRP, and other transporters, etc
–Models for observable phenomena that are composites of multiple processes, eg, bioavailability,
volume of distribution, clearance
•Ligand design
–Fragment-based de novo design software
–Assessment of relative synthetic tractability with performance appropriate for prioritizing thousands to
millions of novel structures
•Protein and protein-ligand modeling
–Reliable screening of multiple mutations including multiple templates
–Antibody structure prediction and protein loop prediction
–Flexible backbone design and simultaneous de novo modeling of multiple loops
–Ab-initio prediction of protein structures, including cases where predicted domain is part of a
multi-domain protein or complex
–3D docking software for pose prediction that incorporates binding site flexibility and alternate
protonation states
•Molecular mechanics and dynamics
–Setup and analysis software for MD calculations
–Molecular mechanics force-fields that can reliably model geometries and energies of small molecules and
proteins including solvent effects
•Virtual screening for lead finding and lead optimization
–Ligand or protein-based methods to find actives more efficiently with demonstrated performance on
experimental datasets for multiple targets
–Ligand or protein-based methods to find actives representing greater diversity of chemical classes with
demonstrated performance on experimental datasets for multiple targets
–Improved scoring for compound ranking that include explicit waters or configurational entropy)
with demonstrated ranking performance on experimental datasets for multiple targets in 3D docking
experiments (eg, methods)
29
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&
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TEC H NO L O G I ES
JU NE
2011
R ES EA R C H
&
E N AB L I N G
T E C HN OL OGI E S
Automated Workflows
Analytical Technology
Areas of Interest:
Areas of Interest:
•Microfluidics
–Coupling microfluidics to conventional automation and analytical tools to maximize productivity, eg,
Empyrean microfluidic UV / Vis plate reader
–Miniaturization of screening tools compatible with organic solvents capabilities for analytical measurements,
reactions, formulations
• Nanoscale sample transfer automation
–1–100 nL range with improved precision / accuracy and speed, solvent compatibility
–Acoustic, inkjet, piezo-based technologies
• MicroArray type methodologies applied to chemical / analytical questions
• Consolidation of sample prep unit operations with analytical autosamplers
–Sample filtration, dilution, quenching, pretreatment, etc, for compounds, tablets, etc
• Coupling conventional HTS liquid handling approaches with techniques beyond plate readers
• Automation of routine sample / buffer preparation
• Quick-turnaround mid-density expression, genotyping, protein expression platforms
•Improved efficiency of analytical workflows
–Fast and high throughput analysis tools
–More efficient analytical method development
–Novel PAT tools
–Automation of analysis
• Methods to analyze large molecules (siRNAs, peptides, polymers), biologics, and vaccines
• Improved platform analytical tools (HPLC, MS, etc) that require smaller samples, including microfluidic
devices to run complex analytical assays
Information Technology / Software
Areas of Interest:
•Knowledge management and data-mining tools
–Finding and accessing existing data
–Ability to integrate external and internal data sets
–Technology to improve information connectivity and sharing within MRL as well as with external partners
–Information and document tagging technologies for smart retrieval of information and documents for
export and data / text-mining
–Technology to support the reusability of information, eg, through collaborative / structured authoring
–Knowledge capture technology to capture tacit knowledge as well as when knowledge is generated
through collaborative research
•Collaboration tools
–Collaborative contribution / decision-making tools, eg, such as predictive market technology
•Workflow management
–Ability to track work requests between groups and perform resource management / prioritization
•In vivo information technology platforms
–Global in vivo database and analytical platform for preclinical data and ability to compare associated
clinical data
–Animal facility management tools
•Modeling, and predictive analysis capabilities that:
–Allow better prediction of in vivo (human) drug performance based on in vitro testing
–Sharing of macromolecular structure models
•Efficient, easy-to-use software for visualizing, manipulating, and processing large amount of data
–Multidimensional data analysis and reporting tools
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2011
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TEC H NO L O G I ES
JU NE
R ES EA R C H
2011
Mapping of Technology Areas of Interest by Franchise
Target Identification
/ Validation
Athero and Cardio
Respiratory and
Immunology
Diabetes and
Endocrinology
Novel lipid / metabolic
and / or other
emerging targets
with high confidence
for reducing CV risk
Human lung cell /
tissue-based systems
for investigating
new therapeutic
mechanisms
Platform technologies
for the identification
and validation of novel
diabetes targets
Infectious
Diseases
Neuro and
Ophthalmology
Novel genetic / RNAbased approaches to
target validation or
therapeutics
Oncology
Animal models of
hypertension
Preclinical or
clinical models
and/or biomarkers
that can be used
to differentiate
anti-coagulant
mechanisms
Predictive animal
models of asthma
pathophysiology
Animal or cellular viral
latency models for
diabetes
Animal or cellular viral
latency models
Robust cell culture
systems for HCV
replication
Biologics
Vaccines
Athero and Cardio
Respiratory and
Immunology
Diagnostics /
Imaging Techniques
and Genotyping /
Phenotyping
Platform technologies
for the identification
and validation of novel
targets
Diabetes and
Endocrinology
Imaging agents for
monitoring pancreatic
beta-cell mass
Biomarkers of patient
subtypes
Technologies for
measuring vascular
dynamics and
intravascular pressure
beyond tonometry
Markers that
link HTN to other
metabolic syndrome
phenotypes and / or
atherosclerosis
Markers for renal
sequelae of HTN
Platform technologies
that enable
personalized medicine
for HTN
Acute methods to
assess cholesterol
transport in humans
(especially reverse
cholesterol transport),
including imaging and
kinetic models
CSF collections from
healthy, MCI, and AD
subjects
Tau and amyloid
imaging agents
Fluid-based assays
for Aβ, ADDLs, and tau
SN-MRI volumetry
Oncology
Biomarkers that can
be used for patient
segmentation
Translational medicine
platforms
Pharmacogenetics
/-genomics,
proteomics, or other
for prediction of
diabetes, obesity,
drug response, and
sub-phenotyping of
disease
Biomarkers for
Osteoporosis
(eg, biomarkers
for fracture risk
and treatment
response for patient
stratification;
non-invasive
biomarkers of bone
strength, quality, and
micro-architecture
with clinical
translation data;
novel translatable
biomarkers of bone
changes) of disease
Markers for chronic
pain
Molecular biomarkers
of susceptibility
Parkinson’s disease
markers that may
identify prodromal
stages
Neurodegeneration
pathway markers
Biologics
Vaccines
Genome-wide
scanning with the
ability to measure
copy number variation
in tumor samples
Gene expression from
FFPET
Fc engineering to
enhance / improve
effector functions,
half-life
Bispecific platforms
with promising
stability and
manufacturing yield
Drug Delivery and
Formulation
In vitro therapeutic
index of combination
therapies
Prognostic HIV and
HCV markers
T E C HN OL OGI E S
Imaging tracers,
cell-based, blood,
nucleic acid, and IVD
diagnostics
Production,
Purification, and
Analysis
Biological tools
to address
nicotinic receptor
pharmacology
In vitro tissue models
for HCV
Methods, biomarkers,
or platforms to assess
antiatherogenic
properties of HDL or
other lipoproteins
Genotyping and
phenotyping
technologies for HCV
polymerase and HCV
protease
Neuro and
Ophthalmology
Models for psychiatric
diseases
Robust In Vitro
Platforms That
Translate to Animal
and Human Studies
Near Real-time
Assays to Screen
Patients Faster for
Clinical Trials
New models for
chronic pain
Infectious
Diseases
Rapid point-of-care,
pathogen-specific,
and host-response
diagnostic tests
Antigen selection,
discovery, and
identification
Tumor models
that can be used
for assessment of
antitumor activity of
tumor immunotherapy
programs
Parkinson’s disease
progression models
Preclinical or clinical
models and/or
biomarkers that
can be used to
differentiate antiplatelet mechanisms
Biomarkers
Models that develop
both plaques and
tangles and that show
neuronal loss
E N AB L I N G
Mapping of Technology Areas of Interest by Franchise
Novel targets for
specialty hypertension
Translational
Models / In Vivo
Pharmacology
&
Identification,
generation, and
modification of
monoclonal antibodies
and engineered
proteins displaying
pharmaceutical
properties
Inhaled technologies /
formulations
Alternate delivery of
peptides
PK enhancers
Novel delivery
systems for novel
or existing migraine
treatments
Tumor-targeted
delivery systems
for systemic
administration of RNAi
Methods to address
blood brain barrier
Dermal or mucosal
delivery
Glycan mediated
targeting
New formulations of
existing anti-migraine
drugs
Markers for PI3K
and DNA damage /
checkpoint pathways
Minimally invasive
platforms for
measuring circulating
tumor cells,
circulating nucleic
acids, novel imaging
tracers
Clinical methods for
early prediction of
long-term weight loss
The table represents a summary of technologies which are further detailed in the Franchise sections.
32
33
J U N E
2011
NOTES
34
A REA S
of
interest
JU NE
2011
AR E AS
o f
interest
NOTES
35
J U N E
2011
areas
of
interest
NOTES
Merck is passionate about our commitment to partnering.
Let’s explore the possibilities of combining our strengths to deliver
novel medical breakthroughs that save and improve lives.
—David Nicholson, PhD, Senior Vice President and Head
Worldwide Licensing and Knowledge Management
36
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