AKAO 2017 Annual Report

85 Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations. You should read the following discussion and analysis of our financial condition and results of operations together with the section of this report entitled “Selected Financial Data” and our consolidated financial statements and related notes included elsewhere in this Annual Report on Form 10-K. This discussion and other parts of this report contain forward-looking statements that involve risk and uncertainties, such as statements of our plans, objectives, expectations, and intentions. As a result of many factors, including those factors set forth in the “Risk Factors” section of this Annual Report on Form 10-K, our actual results could differ materially from the results described in or implied by these forward-looking statements. Overview We are a late-stage biopharmaceutical company passionately committed to the discovery, development, and commercialization of innovative antibacterial treatments against multi-drug resistant (“MDR”) gram-negative infections. We are developing plazomicin, our lead product candidate, for the treatment of serious bacterial infections, including complicated urinary tract infection (“cUTI”), blood stream infections (“BSI”) and other infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae (“CRE”). In 2013, the Centers for Disease Control and Prevention (“CDC”) identified CRE as a “nightmare bacteria” and an immediate public health threat that requires “urgent and aggressive action” and in 2017 the World Health Organization (“WHO”) identified CRE as a Global Priority 1 Pathogen: Critical Need for Research and Development of New Antibiotics. Our second antibacterial candidate is C-Scape, an orally-administered combination of clavulanate and ceftibuten, which targets serious bacterial infections due to expanded spectrum beta-lactamases (“ESBL”) producing Enterobacteriaceae. In 2017, the WHO identified ESBL as a Global Priority 1 Pathogen: Critical Need for Research and Development of New Antibiotics. We also have other programs in early and late preclinical stages focused on other MDR gram-negative infections and additional disease areas. New Drug Application (“NDA”) for substantive On January 2, 2018, we announced the acceptance of a review to the U.S. Food and Drug Administration (“FDA”) for plazomicin, seeking approval to treat complicated urinary tract infections (“cUTI”), including acute pyelonephritis (“AP”) and bloodstream infections (“BSI”) due to certain Enterobacteriaceae in patients who have limited or no alternative treatment options. The NDA is supported by data from two Phase 3 clinical trials, EPIC (Evaluating Plazomicin In cUTI) and CARE (Combating Antibiotic Resistant Enterobacteriaceae). The FDA has accepted review of the NDA and has granted the NDA Priority Review as well as set a Prescription Drug User Fee Act (“PDUFA”) target action date of June 25, 2018. We expect a commercial launch of plazomicin in the United States in 2018, if our NDA is approved. We also intend to submit an application for marketing authorization (“MAA”) in the European Union (“EU”) in 2018. On May 23, 2017, we announced that the FDA granted Breakthrough Therapy designation (“BTD”) for plazomicin for the treatment of BSI caused by certain Enterobacteriaceae in patients who have limited or no alternative treatment options. BTD was created by the FDA to expedite the development and review of drugs that target serious or life-threatening conditions. Plazomicin has also received Qualified Infectious Disease Product (“QIDP”) designation from the FDA, which provides incentives for the development of new antibiotics, including priority review and an extension by an additional five years of any existing non-patent market exclusivity the product may be awarded upon approval. We have global commercialization rights to plazomicin, which has patent protection in the United States estimated from 2030 to 2032. Plazomicin has been evaluated in two Phase 3 clinical trials, entitled EPIC and CARE. The EPIC study was a Phase 3 trial of plazomicin for the treatment of patients with cUTI and AP and enrolled 609 patients. In the EPIC trial, plazomicin successfully met the objective of non-inferiority compared to meropenem for the FDA-specified primary efficacy endpoints, and achieved superiority for the primary efficacy endpoints specified by the European Medicines Agency (“EMA”). Results for the FDA pre-specified composite endpoint of clinical cure and microbiological eradication in the microbiological modified intent-to-treat (“mMITT”) population at Day 5 achieved statistical non-inferiority, and outcomes at the Test-of-Cure (Day ~17) statistically favored plazomicin. Results for EMA-specified endpoints of microbiological eradication at the test-of-cure visit achieved statistical superiority in both the mMITT and microbiologically evaluable (“ME”) populations. Plazomicin was also generally well tolerated with no new safety concerns identified in the EPIC trial.