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TPIP Phase 1 Topline Results Forward Looking Statements

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TPIP Phase 1 Topline Results Forward Looking Statements February 19, 2021 TPIP Phase 1 Topline Results Forward Looking Statements This presentation contains forward-looking statements that involve requirements related to ARIKAYCE or the Lamira® Nebulizer System; the agreements with PARI and AstraZeneca AB, and failure of the Company to substantial risks and uncertainties. "Forward-looking statements," as that Company's inability to obtain adequate reimbursement from government or comply with its obligations under such agreements; the cost and potential term is defined in the Private Securities Litigation Reform Act of 1995, are third-party payors for ARIKAYCE or acceptable prices for ARIKAYCE; reputational damage resulting from litigation to which the Company is or statements that are not historical facts and involve a number of risks and development of unexpected safety or efficacy concerns related to may become a party, including product liability claims; the Company's uncertainties. Words herein such as "may," "will," "should," "could," ARIKAYCE or the Company’s product candidates; inaccuracies in the limited experience operating internationally; changes in laws and "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," Company's estimates of the size of the potential markets for ARIKAYCE or regulations applicable to the Company's business, including any pricing "predicts," "intends," "potential," "continues," and similar expressions (as its product candidates or in data the Company has used to identify reform, and failure to comply with such laws and regulations; inability to well as other words or expressions referencing future events, conditions or physicians, expected rates of patient uptake, duration of expected repay the Company's existing indebtedness and uncertainties with respect circumstances) may identify forward-looking statements. treatment, or expected patient adherence or discontinuation rates; the to the Company's ability to access future capital; and delays in the Company's inability to create an effective direct sales and marketing execution of plans to build out an additional third-party manufacturing infrastructure or to partner with third parties that offer such an infrastructure facility approved by the FDA and unexpected expenses associated with The forward-looking statements in this presentation are based upon the for distribution of ARIKAYCE or any of the company’s product candidates those plans. Company's current expectations and beliefs, and involve known and that are approved in the future; failure to obtain regulatory approval to unknown risks, uncertainties and other factors, which may cause the expand ARIKAYCE's indication to a broader patient population; failure to Company's actual results, performance and achievements and the timing successfully conduct future clinical trials for ARIKAYCE, brensocatib, The Company may not actually achieve the results, plans, intentions or of certain events to differ materially from the results, performance, trepostinil palmitil inhalation powder (TPIP) and the Company's other expectations indicated by the Company's forward-looking statements achievements or timing discussed, projected, anticipated or indicated in product candidates, due to the Company's limited experience in conducting because, by their nature, forward-looking statements involve risks and any forward-looking statements. Such risks, uncertainties and other factors preclinical development activities and clinical trials necessary for regulatory uncertainties because they relate to events and depend on circumstances include, among others, the following: the risk that brensocatib does not approval and the Company's potential inability to enroll or retain sufficient that may or may not occur in the future. For additional information about prove to be effective or safe for patients in ongoing and future clinical patients to conduct and complete the trials or generate data necessary for the risks and uncertainties that may affect the Company's business, please studies, including the ASPEN study; the risk that TPIP does not prove to be regulatory approval, among other things; risks that the Company's clinical see the factors discussed in Item 1A, "Risk Factors," in the Company's effective or safe for patients in ongoing and future clinical studies; business studies will be delayed or that serious side effects will be identified during Annual Report on Form 10-K for the year ended December 31, 2019, or economic disruptions due to catastrophes or other events, including drug development; failure to obtain, or delays in obtaining, regulatory Quarterly Reports on Form 10-Q for the quarters ended March 31, 2020, natural disasters or public health crises; impact of the novel coronavirus approvals for ARIKAYCE outside the U.S. or European Union, including June 30, 2020 and September 30, 2020, and any subsequent Company (COVID-19) pandemic and efforts to reduce its spread on our business, the United Kingdom as a result of its exit from the EU, or for the Company's filings with the SEC. employees, including key personnel, patients, partners and suppliers; product candidates in the U.S., Europe, Japan or other markets; failure of failure to successfully commercialize ARIKAYCE®, our only approved third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE or the Company's product candidates for product, in the United States or European Union (amikacin liposome The Company cautions readers not to place undue reliance on any such commercial or clinical needs, to conduct the Company's clinical trials, or to inhalation suspension and Liposomal 590 mg Nebuliser Dispersion, forward-looking statements, which speak only as of the date of this comply with the Company’s agreements or laws and regulations that respectively), or to maintain U.S. or EU approval for ARIKAYCE; presentation. The Company disclaims any obligation, except as specifically impact the Company's business or agreements with the Company; the uncertainties in the degree of market acceptance of ARIKAYCE by required by law and the rules of the SEC, to publicly update or revise any Company's inability to attract and retain key personnel or to effectively physicians, patients, third-party payors and others in the healthcare such statements to reflect any change in expectations or in events, manage the Company's growth; the Company's inability to adapt to its community; the Company's inability to obtain full approval of ARIKAYCE conditions or circumstances on which any such statements may be based, highly competitive and changing environment; the Company's inability to from the FDA, including the risk that the Company will not timely and or that may affect the likelihood that actual results will differ from those set adequately protect its intellectual property rights or prevent disclosure of its successfully complete the study to validate a PRO tool and the forth in the forward-looking statements. confirmatory post-marketing clinical trial required for full approval of trade secrets and other proprietary information and costs associated with ARIKAYCE; inability of the Company, PARI Pharma GmbH (PARI) or the litigation or other proceedings related to such matters; restrictions or other obligations imposed on the Company by its agreements related to Company's.other third-party manufacturers to comply with regulatory 2 ARIKAYCE or the Company's product candidates, including its license Powered By Purpose Our commitment to transforming the lives of people with rare diseases drives what we do. 3 Treprostinil Palmitil Inhalation Powder (TPIP) Potentially Developing a Superior Treatment Paradigm Current TPIP Encouraging Further Therapies Phase 1 Results Development Prostanoids have long A Phase 1 study of Topline results Results support moving forward been considered the “gold TPIP in healthy demonstrate that TPIP with development of TPIP with standard” therapy for PAH; volunteers has recently was generally well once daily dosing been completed tolerated however, this class of drugs has also been TPIP may also have utility in plagued by challenges The highest dose treatment of Group 3 pulmonary tested as a single dose hypertension (PH-ILD) and was 675 µg, and the idiopathic pulmonary fibrosis (IPF) Treprostinil palmitil has highest dose tested in demonstrated substantial repeated dosing was promise for the treatment 225 µg We believe evidence of efficacy ® of pulmonary arterial with Tyvaso in these patient hypertension populations may translate to TPIP as well 4 Treprostinil Palmitil Harnessing the Full Potential of the Prostanoid Pathway Prostanoids have long been a cornerstone therapy for PAH, but clinical use has faced challenges related to rapid metabolism and dose-limiting tolerability issues TPIP could address the shortcomings of existing prostanoid therapies by potentially: • Providing prolonged, localized pulmonary vasodilation • Offering improved tolerability, with fewer prostanoid-related side effects • Enabling higher dosing for superior efficacy with the potential for disease-modifying effect • Simplifying dosing with once daily administration PAH = pulmonary arterial hypertension; RA = right atrium; RV = right ventricle; PA = pulmonary artery; mPAP = mean pulmonary arterial pressure; PAOP = pulmonary artery occluded pressure; PVH = pulmonary venous hypertension 5 Image reproduced from https://www.mayoclinic.org/, © Mayo Foundation for Medical Education and Research TPIP: A Potentially Differentiated Prostanoid • TPIP* is a dry powder formulation
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