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CritiCally Caring about CritiCal Care
A s a fully integrated biotherapeutics
company, we have a deep
understanding of critical care.
This understanding drives us to focus on
the needs of critically ill patients both inside
and outside the ICU.
In life’s most fragile moments, we know
what it takes to make a difference. We have
a keen insight into the complete clinical
picture, and a strong understanding of the
biology of the conditions affecting critically
ill patients. This combination fuels our
scientific and research endeavors. And
it’s this work that we hope will have an
impact on critically ill patients and the
healthcare professionals who care for them.
Above all, we recognize the urgent needs
of this environment. Which is why helping
clinicians succeed and giving patients a
fighting chance is what fuels us to move ahead.
Over the last several decades there have
been very few breakthroughs in critical
care therapies. We are committed to
changing that. When the next significant
breakthrough occurs in critical care,
we plan to be on the front lines leading
the way.
Superb clinical and biologic insight drives our exploration into critical conditions
Disclaimer:This document includes or may include certain statements, estimates, and forward-looking projections with respect to our anticipated performance. Such statements, estimates, and forward-looking projections reflect various assumptions of our management that may or may not prove to be correct and involve various risks and uncertainties.
2{
More than 56,000 patients are in the iCu on any given day.1
3 {
Ikaria was formed in 2007 when research
and discovery company Ikaria, Inc. joined
forces with INO Therapeutics, a company
with expertise in developing and commercializing
novel treatments for critically ill patients.
Today, Ikaria boasts a robust and growing
product pipeline that spans commercial,
preclinical, and clinical development. Rapid
global expansion and strategic licensing and
acquisitions are helping propel Ikaria forward.
The basis of our growth is a unique business
model and a range of product and proprietary
drug-delivery systems supported by an
all-inclusive offering seen nowhere else
in the industry.
At Ikaria, we are rapidly establishing a
leadership role in providing innovative solutions
to advance critical care for the future.
Fueled by passion, a mission that leads to results
our ideas drive us forward
• Our innovative therapies that directly influence patient outcomes and provide hope to families
• Our dedication to healthcare professionals who provide care when it is most critical
• Our value of the diverse talents, energy, and pride of all our employees
• The integration of our scientific,clinical, and caregiver expertise
• Our foundation, which is built on integrity and unwavering quality standards
Our mission is to address
the significant unmet needs
of critically ill patients every
single day through:
A t Ikaria, our competencies run deep, enabling
us to advance innovative solutions and provide
critical support. Our approach of critically caring
underpins everything that we do.
Fully integrated and focused on the needs of patients and customers
a strong team for a complex world
Throughout Ikaria, we work hard, smart, and with urgency,
because that’s the environment of critical care. We are a
customer-centric organization, and never forget that patients,
families, and clinicians are counting on us.
4{
More than five million patients are admitted annually to iCus in the united States.2
research & Development Along with investigating additional potential benefits
of our flagship product INOMAX® (nitric oxide)
for inhalation, we currently are investigating the
therapeutic potential of other promising critical care
compounds, and are aggressively pursuing the
development of new therapies across the spectrum
of drug and drug-delivery systems.
Clinical research With significant capabilities in pharmacology,
preclinical development, and clinical development
across all phases, we focus on applications in
ischemia/reperfusion injury, cardiovascular and
pulmonary medicine and surgery, transplantation,
and neonatology.
Sales & Marketing Our relationships with clinicians and hospital
staff are based not only on technical and clinical
expertise but also on our commitment to go the
extra mile at a moment’s notice. Our elite sales team
is supported by a growing integrated marketing and
customer service staff. The combined efforts of this
group embody the highest level of customer care.
Manufacturing We are the only company to develop and
commercialize an endogenous signalling
molecule in gas form for human use. In fact,
Ikaria’s drug manufacturing facility is the only
FDA-regulated nitric oxide manufacturing site
in the US. Separately, we manufacture the
complementary device that delivers the therapy
safely and reliably through an integrated
drug-delivery system.
Delivery System Development Beyond providing novel biotherapeutics, we have
special competencies in developing drug-delivery
systems for proper administration of our therapy.
Our marketed product INOMAX (nitric oxide)
for inhalation and some of our development-stage
compounds require drug-delivery systems for
proper administration.
DistributionWe meet the needs of our North American customers
with seven regional service centers that are on call
24/7/365. Overseas, we work with local partners to
assure the same high level of competence, access,
and service.
global relationships To share our critical care expertise and impact upon
patients worldwide, we have established our presence
in Canada, Japan, and Australia, and have teamed
with like-minded organizations in Europe, Mexico,
and South America.
business Development & licensing With a goal of bringing additional much-needed
solutions to the critical care market, we actively pursue
development and commercial opportunities that will
benefit from our unique expertise in this arena.
IkarIa CapabIlITIeS
{5
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Our flagship commercialized
product, INOMAX® (nitric oxide)
for inhalation, demonstrates
our ability to develop unique therapies
that meet the significant unmet needs
of the most fragile patients. But that’s
just the start.
Harnessing powerful science to produce promising therapies
We discovered that certain naturally
occurring or endogenous signalling
molecules, when properly delivered in low
doses, may demonstrate several essential
critical care benefits. Our investigation of
these powerful therapeutic molecules is
an ongoing journey.
Our drug development program is broad
and far reaching, with roots in nitric oxide,
carbon monoxide and hydrogen sulfide
molecules. Through groundbreaking
research, these molecules have been
established as powerful influencers
of many cellular functions. They are just
the beginning of our further exploration
into developing promising products for
critical care.
Leveraging our deep clinical insight and
understanding of pathophysiology, we also
are exploring other promising compounds
that will help address significant unmet
needs of critically ill patients.
Proven execution of complex clinical studies in iCu settings
Ikaria brings substantial clinical development
expertise to all aspects of critical care trial
design and management, executing clinical
trials in more than 4,000 critically ill patients
across multiple ICU settings in the US,
Canada, the EU, and Australia.
Vast clinical and preclinical critical care experience
{
the five primary iCu admitting diagnoses are, in order, respiratory insufficiency/failure, postoperative management, ischemic heart
disorder, sepsis, and heart failure.2
Critically ill patients can’t wait for a traditional approach to r&D
Research & development is vital
for clinicians and their patients
in the critical care community.
Meeting their unmet needs is a powerful
driver for Ikaria.
Where breakthroughs are born
Our preclinical discovery and exploratory
research facility is equipped to conduct
state-of-the-art synthetic chemistry, analytical
and bioanalytical chemistry, formulation,
pharmacology, and toxicology studies.
reducing risk and shortening development time
Translational medicine and adaptive trial
design have emerged as two powerful
alternatives to traditional research and
drug development. At Ikaria, we also utilize
biomarkers to help guide and de-risk clinical
development. These strategic approaches have
the potential to reduce costs by facilitating
more efficient clinical trials. Additionally, they
can determine at a significantly earlier date
which formulations are likely to fail in later
trials, potentially saving money and time.
What this means for patients
By identifying earlier which compounds may
have a therapeutic benefit and which patients
may benefit most, translational medicine has
the potential to offer breakthroughs sooner for
patients who need them.3,4 This can make all
the difference for patients who can’t wait.
transforming the future
At Ikaria we continue to define our
leadership role. Fully integrated and
critically positioned, we intend to
advance critical care well into the future
and plan to be on the forefront of the
next major breakthrough.
7 {
nearly 80% of all americans will experience a critical care illness or injury, as a patient, a family member, or as a friend of a patient.5
inoMaX® DS Delivering and maintaining precise levels of a pharmacological gas is complex. but our state-of-the-art transportable INOMaX DS is engineered to simplify the complex administration of INOMaX® (nitric oxide) for inhalation and is fully compatible with more than 45 makes of ventilation devices and anesthesia machines.1,6-8
Our proprietary INOMaX DS delivery system features multiple back-up systems to ensure safe, consistent, and reliable delivery and monitoring at any ventilation setting for all patients.
Warning: The INOMaX DS delivery system must only be used in accordance with the indications, usage, contraindications, warnings, and precautions described in the nitric oxide drug package inserts and labeling.
inoMaX®—a therapy of great possibilities
From the moment it was discovered, nitric oxide has inspired
countless researchers and clinicians to realize the benefits of
this potent pulmonary vasodilator. A compound that is naturally
produced in the human body, nitric oxide is the active substance in
INOMAX, the first and only FDA-approved endogenous signalling
molecule in gas form for use in humans.
At Ikaria, we have been equally inspired by this Nobel Prize-winning
discovery and are dedicated to realizing its full potential. Nitric oxide is
able to penetrate cell walls to deliver a signal to nearby muscles to relax.9
As these muscles relax, blood flow increases, helping the heart and lungs
process more oxygen and deliver more oxygenated blood to the body.
therapy that calls for state-of-the-art medication management
Delivering the full benefit of INOMAX requires advanced medication
management technology for accurate dosing, patient monitoring,
and individualized record keeping. To facilitate optimal delivery,
Ikaria designs, develops, manufactures, and globally distributes
these drug-delivery systems through its FDA-regulated facility.
Please visit www.inoMaX.com.
8{
offering sophisticated multifaceted support
Whether we’re scheduling an emergency
delivery of medication, flying in equipment
from one of our regional centers, or providing
middle-of-the-night technical assistance,
we do what it takes to ensure that clinicians
and their patients are able to benefit from
this vital therapy.
Clinical & technical Support
• Emergency technical, medical,
and respiratory therapist support;
medical affairs assistance; in-service
training; technical support; and
reimbursement services
Distribution Services & Support
• Emergency deliveries in 4 hours in most
cases; seven wholly owned and operated
regional service/distribution centers;
service and maintenance of all devices;
technical training
ongoing training
Clinical professionals provide on-site training,
education, and scientific and technical
expertise to both new and existing customers.
On average, we interact with and train
approximately 1,800 customers each month.
inotherapy®—an exclusive offering
The value of INOMAX includes a comprehensive, all-inclusive offering of service and
technologies called INOtherapy. This includes the drug, device, distribution, emergency
supply, technical assistance, clinical assistance, and quality maintenance. INOtherapy
is delivered by an exclusive interdisciplinary team of clinical and logistical professionals who are
available to customers 24/7/365.
9 {
Since its commercialization in 2000, we believe that approximately 360,000 patients have been treated with inoMaX® worldwide.1
Committed to advancing critical care worldwide
We have established wholly owned operations in Canada,
Japan, and Australia and have created relationships with
organizations in those regions, as well as in Europe, Mexico,
and South America. This global reach is part of our commitment to
provide advanced critical care therapies around the world.
Our US regional service centers are located
in or around New York, Chicago, Dallas, atlanta,
los angeles, San Francisco, and baton rouge.
10{
european union Austria Belgium Czech Republic Denmark Finland France Germany Hungary Iceland Ireland Italy Netherlands Norway Poland Portugal Spain Sweden Switzerland United Kingdom
Southeast asia/ South Pacific Australia Japan (as INOflo) Malaysia Singapore
north america Canada United States
latin america Argentina Chile Colombia (as INOflo®) Mexico Uruguay
inoMaX® (nitric oxide) for inhalation is available in the following countries:
11 {
building alliances for the future
Partners
Born of collaboration, Ikaria pursues
paths that lead to the development
and commercialization of acute care
therapies for the significant unmet needs of
critically ill patients. At Ikaria, we are looking
to create future relationships that build upon
our three unique areas of expertise:
• Our insight into the clinical needs
of critically ill patients
• Our deep understanding of the
biology of critical conditions
• Our intimate understanding of the
needs of healthcare providers who
practice critical care medicine
Expertise in preclinical and clinical development
through product commercialization makes us
an ideal partner for any company with assets
focused on critical care conditions. We seek
to acquire additional products and technologies,
including drugs, devices, and drug-delivery
systems at any stage of their development.
therapies on the horizon
In addition to our organic work on NO, CO,
and H2S, strategic licensing and acquisition
enhances the Ikaria pipeline. Our acquisition of
the North American rights to terlipressin puts us
on the forefront of delivering the first treatment
for hepatorenal syndrome Type 1, a rare
and often fatal condition of advanced kidney
failure in patients with cirrhosis.
The acquisition of several exclusive worldwide
licenses further expands our offering of
novel therapies:
• IK-5001* is a potential breakthrough
treatment for preventing pathological
cardiac remodeling following acute
myocardial infarction†10
• Investigational fibrin-derived compounds,
known as IK-6001, IK-6002, and IK-6003,‡
preserve endothelial barrier function,
prevent tissue injury,† and hold great
promise for exploration in a multitude
of critical care conditions11,12
For a full list of our areas of focus, visit www.ikaria.com.
*IK-5001 – Licensed from BiolineRx (BL-1040).† In animal disease models.‡ IK-6001, IK-6002, IK-6003 – Licensed from Fibrex Medical (FX06, FX201, FX107).
11 {
there are approximately 6,000 iCus in the united States.2
References
1. Data on file. Clinton, NJ: Ikaria Holdings Inc.; 2009.
2. Society of Critical Care Medicine. Critical care statistics in the United States. http://www.sccm.org/AboutSCCM/Public%20Relations/Pages/ Statistics.aspx. Accessed July 24, 2009.
3. Littman BH, Di Mario L, Plebani M, Marincola FM. What’s next in translational medicine? Clin Sci. 2007;112(4):217-227.
4. Chow SC, Chang M. Adaptive design methods in clinical trials: a review. Orphanet J Rare Dis. 2008;3:11.
5. Society of Critical Care Medicine. Critical care questions. http://www.myicucare.org/Critical_Care_Questions/Pages/default.aspx. Accessed July 24, 2009.
6. Datex-Ohmeda, Inc. INOvent Delivery System Operational and Maintenance Manual. Madison, WI: Datex-Ohmeda, Inc; 2000. Publication 1605-0014-000.
7. INO Therapeutics. INOmax DS Operation Manual. Clinton, NJ: INO Therapeutics LLC; 2002.
8. INO Therapeutics. INOblender Operation and Maintenance Manual. Clinton, NJ: INO Therapeutics LLC; 2001.
9. Bryan NS, Bian K, Murad F. Discovery of the nitric oxide signaling pathway and targets for drug development. Front Biosci. 2009;14:1-18.
10. Landa N, Miller L, Feinberg MS, et al. Effect of injectable alginate implant on cardiac remodeling and function after recent and old infarcts in rat. Circulation. 2008;117:1388-1396. http://circ.ahajournals.org/cgi/content/abstract/117/11/1388. Accessed August 28, 2009.
11. Gröger M, Pasteiner W, Ignatyev G, et al. Peptide Bß15-42 preserves endothelial barrier function in shock. PLoS One. 2009;4(4):e5391. http://www.plosone.org/article/info:doi/10.1371/journal.pone.0005391. Accessed August 28, 2009.
12. Roesner JP, Petzelbauer P, Koch A, et al. Bß15-42 (FX06) reduces pulmonary, myocardial, liver, and small intestine damage in a pig model of hemorrhagic shock and reperfusion. Crit Care Med. 2009;37(2):598-605.
www.ikaria.com
In the United States:6 Route 173 Clinton, NJ 08809Voice +1-908-238-6600Fax +1-908-238-6633
Customer Service 24/7/365: +1-877-KNOW-INO (+1-877-566-9466)
© 2009 Ikaria, Inc. October 2009.The product information here is presented for use in the United States. IMK111-01164R 10/09