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PART IItem 1.Financial Statements2Item 2.Management’s Discussion and Analysis of Financial Condition and Results of Operations18 Any forward-looking statements in this Quarterly Report reflect our current views with respect to future events or to our future financial be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. Factors thatmay cause actual results to differ materially from current expectations include, among other things, those listed under Part I, Item 1A. Risk Factors ofour most recent Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 26, 2025 (the "2024 Annual no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future. certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions.Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actualevents or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, weobtained this industry, business, market and other data from reports, research surveys, studies and similar data prepared by third parties, industry,medical and general publications, government data and similar sources. (In thousands, except share and per share amounts)(Unaudited) Interest income659Interest expense(180) (In thousands, except share amounts)(Unaudited) 1. Natureof business development of innovative therapeutics for rare diseases. The Company has developed a portfolio of late-stage clinical product candidates, and its tworare disease product candidates are setrusumab for the treatment of osteogenesis imperfecta (“OI”) and alvelestat primarily for the treatment of severealpha-1 antitrypsin deficiency-associated lung disease (“AATD-LD”). 2. Basis of presentation and summary of significant accounting policiesBasis of presentation its subsidiaries have been eliminated on consolidation.The unaudited condensed consolidated financial statements presented in this Quarterly Report should be read in conjunction with theconsolidated financial statements and accompanying notes included in the Company’s Annual Report on Form 10-K filed with the SEC on March 26,2025 (the “2024 Annual Report”). The condensed consolidated balance sheet as of December 31, 2024 was derived from audited consolidated Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have beencondensed or omitted from these interim financial statements. However, these interim financial statements include all adjustments which are, in the ended March 31, 2025 and 2024 and cash flows for the three months ended March 31, 2025 and 2024. The interim results are not necessarily indicativeof results to be expected for the full year.Going concernThe Company has prepared its condensed consolidated financial statements on the basis that it will continue as a going concern. TheCompany is subject to risks common to companies in the biotechnology industry, including but not limited to, risks of delays in initiating or continuing The Company has historically been loss making, anticipates that it will continue to incur losses for the foreseeable future, and had anaccumulated deficit of $472.0million as of March 31, 2025. The Company has funded these losses through a combination of public equity financings,private equity and debt financings and various license and collaboration agreements, and it expects it will continue to do so until such time as it cangenerate significant revenue from product sales, or other commercial revenues, if ever, or through licensing and/or collaboration agreements for its raredisease or oncology product candidates. Although management continues to pursue these plans, there is no assurance that the Company will be The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affectthe reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reportedamounts of income and expenses during the reporting periods. There are no estimates and assumptions made in the consolidated financial statementsthat are considered to be critical. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Changes in estimates 3. Recent accounting pronouncements There have been no recent accounting pronouncements, changes in accounting pronouncements or recently adopted accounting guidanceother than those previously included in the 2024 Annual Report that are of significance or potential significance to the Company. The Co