CritiCally Caring about CritiCal Care

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  • 1. Critically caring about critical care

2. 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 lifes 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 its 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. 3 { I karia 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: 4. 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 thats 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 5. ResearchDevelopment 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. SalesMarketing 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 moments 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, Ikarias 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. Distribution We 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 DevelopmentLicensing 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 6. 6 O ur 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 thats 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 7. Critically ill patients cant wait for a traditional approach to RD R esearchdevelopment 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 cant 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 8. 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, con