Qualified Infectious Disease Product (QIDP) status granted for oral lefamulin formulation
Top-line data from LEAP 2 study expected in 2H17
VIENNA, Austria, and KING OF PRUSSIA, Pa., April 11, 2016 (GLOBE NEWSWIRE) -- Nabriva Therapeutics AG (NASDAQ:NBRV), a clinical stage biopharmaceutical company engaged in the research and development of novel anti-infective agents, with a focus on the pleuromutilin class of antibiotics to treat serious infections, today announced the initiation of the second of two Phase 3 multinational pivotal trials evaluating the safety and efficacy of Nabriva’s lead product candidate, lefamulin, for the treatment of adults with community-acquired bacterial pneumonia (CABP). The trial, Lefamulin Evaluation Against Pneumonia (LEAP) 2, will assess the efficacy and safety of oral lefamulin compared with oral moxifloxacin in subjects with moderate CABP.
Additionally, the U.S. Food and Drug Administration (FDA) recently designated oral lefamulin as a qualified infectious disease product, or QIDP, which provides for the extension of statutory exclusivity periods in the United States for an additional five years upon FDA approval of the product for the treatment of CABP. The FDA also granted fast track designation to the oral formulation of lefamulin. Fast track designation is granted by the FDA to facilitate the development of drugs that treat serious conditions and fill an unmet medical need. The fast track designation for the oral formulation of lefamulin will allow for more frequent interactions with the FDA, eligibility for priority review and a shortening of the FDA’s goal for taking action on a marketing application from ten months to six months. The intravenous (IV) formulation of lefamulin has previously been designated QIDP with fast track status.
Lefamulin is being developed to be the first pleuromutilin for systemic use in humans, and is the first new class of antibiotic to reach late-stage clinical development for CABP in over a decade. In microbiological studies, lefamulin showed potent in vitro activity against the most common pathogens associated with CABP, specifically S. pneumoniae, H. influenzae, M. pneumoniae, L. pneumophila, and C. pneumoniae, as well as S. aureus, including multi-drug resistant (MDR) strains of S. pneumonia and methicillin resistant S. aureus (MRSA).
“Given its novel mechanism of action, targeted spectrum of activity, lack of cross resistance with other commonly used respiratory antibiotics, and favorable tolerability profile, we believe lefamulin is well-positioned to become a first-line empiric monotherapy for the treatment of moderate to severe CABP,” said Elyse Seltzer, MD, Chief Medical Officer of Nabriva. “We look forward to the progress of our clinical program and to potentially bringing a first-in-class systemically available antibiotic to patients suffering from CABP.”
The Phase 3, multinational, randomized, double-blind, placebo-controlled, non-inferiority trial will assess safety and efficacy of oral lefamulin therapy compared to oral moxifloxacin for the treatment of adults with moderate CABP (PORT Risk Class II, III, or IV). The study is designed to enroll approximately 740 patients at approximately 150 centers in the United States, Europe, Asia and selected countries in the southern hemisphere. Patients will be randomized 1:1 to receive either 5 days of active lefamulin (600 mg BID) or 7 days of active moxifloxacin (400 mg QD). At least 50% of patients will have PORT scores III or IV.
The study is designed to address both the FDA and European Medicines Agency (EMA) regulatory guidelines for the development of antibacterial agents to treat CABP. The FDA primary efficacy endpoint is the early clinical response (ECR). ECR is a programmatically derived endpoint, based on improvement of the patient’s baseline symptoms of CABP at 72 to 120 hours after the first dose of study drug. The EMA primary endpoint is the investigator assessment of clinical success rate (IACR) at test of cure (5-10 days after the last dose of study drug). IACR is based on the investigator’s assessment of a patient’s clinical course. Secondary objectives will evaluate these endpoints in other relevant study populations, microbiologic endpoints to assess pathogen eradication, 28-day mortality, and safety and tolerability.
“The initiation of our second of two Phase 3 studies in patients with CABP is an important milestone for Nabriva. Our first study, LEAP 1, is enrolling patients who require initiation of therapy with IV antibiotics, but allows patients to switch to subsequent oral therapy. We plan to conduct a blinded interim analysis of LEAP 1 when 60% of patients have been enrolled to check the assumptions used to determine the sample size of the study. While we are focused on execution of these Phase 3 studies, we look forward to having top-line data from each of these trials in the second half of next year,” stated Dr. Colin Broom, Chief Executive Officer of Nabriva. “If the results of these studies are favorable, we expect to submit applications for marketing approval to both the FDA and EMA in 2018.”
About Nabriva Therapeutics AG
Nabriva Therapeutics is a clinical stage biopharmaceutical company engaged in the research and development of novel anti-infective agents to treat serious bacterial infections, with a focus on the pleuromutilin class of antibiotics. Nabriva's medicinal chemistry expertise has enabled targeted discovery of novel pleuromutilins, including both intravenous and oral formulations of its lead product candidate, lefamulin, which is being developed as the first systemically available pleuromutilin antibiotic for human use. Nabriva's lead pleuromutilin product candidate, lefamulin, is being developed to be the first systemically available pleuromutilin for human use and is the first new class of antibiotic to reach late stage clinical development for CABP in over a decade. Nabriva believes lefamulin is well positioned for use as a first-line empiric monotherapy for the treatment of moderate to severe CABP due to its novel mechanism of action, targeted spectrum of activity, encouraging resistance profile, achievement of substantial drug concentration in lung tissue and fluid, oral and IV formulations and favorable tolerability profile. Nabriva also intends to further pursue the development of lefamulin for additional indications, including the treatment of acute bacterial skin and skin structure infections, and is developing a formulation of lefamulin appropriate for pediatric use.
Nabriva owns exclusive, worldwide rights to lefamulin, which is protected by composition of matter patents issued in the United States, Europe and Japan.
Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for Nabriva, including but not limited to statements about the development of Nabriva's product candidates, such as plans related to the conduct and timelines of Phase 3 clinical trials of lefamulin for CABP, the clinical utility of lefamulin for CABP, and Nabriva's plans for filing for regulatory approvals and efforts to bring lefamulin to market, the development of lefamulin for additional indications, the development of additional formulations of lefamulin, plans to pursue research and development of other product candidates and other statements containing the words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "likely," "will," "would," "could," "should," "continue," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials in different disease indications will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including for use as a first-line empiric monotherapy for the treatment of moderate to severe CABP, the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption "Risk Factors" in Nabriva's final prospectus dated September 17, 2015 for its U.S. initial public offering and in other filings it makes with the United States Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Nabriva's views as of the date of this release. Nabriva anticipates that subsequent events and developments will cause its views to change. However, while Nabriva may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Nabriva's views as of any date subsequent to the date of this release.