Phase 2/3 Pivotal Trial Will Test Intravenous, Oral Capsule and Oral Suspension Formulations of Solithromycin in Pediatric Patients From Age 2 Months to 17 Years
Cempra to Receive $25.5 Million in Funding for the Next Phase of Its Existing Agreement With BARDA
CHAPEL HILL, N.C. , March 07, 2016 (GLOBE NEWSWIRE) -- Cempra, Inc. (Nasdaq:CEMP), a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases, today announced it has received authorization under its existing contract with the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services, to receive funding of $25.5 million through mid-2018 for a Phase 2/3 clinical study of solithromycin. Cempra is responsible for an additional designated portion of the cost of the planned Phase 2/3 study. Pediatric patients from age 2 months to 17 years with community-acquired bacterial pneumonia (CABP) who are enrolled in the study may receive intravenous, oral suspension or oral capsule formulations of solithromycin, an investigational, fourth generation macrolide antibiotic, or a comparator treatment. The planned enrollment of 400 patients for the Phase 2/3 study is expected to begin during the first half of 2016, with full enrollment expected during the first half of 2018.
"We are excited to receive funding approval for the next planned phase of our investigational studies with solithromycin in pediatric patients, and with the progress we are making under our ongoing collaborative relationship with BARDA,” stated Prabhavathi Fernandes, Ph.D., president and chief executive officer of Cempra. “Solithromycin has shown promising potential to fill a medical need across all patient age groups and we appreciate BARDA’s continuing support. Currently, Cempra’s regulatory submissions in the U.S. and EU, for the approval of oral and intravenous solithromycin for use in adult patients with CABP, are on track for completion in the first half of 2016.”
About the Solithromycin Pediatric Development Program
The Phase 2/3 pivotal open label trial is planned for enrollment of 400 pediatric patients aged 2 months to 17 years with CABP and will be conducted at 60 centers worldwide. Patients will be randomized 3:1 to receive solithromycin (n~300) as an oral, IV or suspension formulation, or a comparator drug (n~100). The primary endpoint will be safety and the secondary endpoints will be efficacy and pharmacokinetics in the pediatric population. Patients will receive solithromycin for 5 to 7 days and the dose will be based on the age and weight of the patient. The duration of treatment will be up to the treating physician. Comparator treatments include amoxicillin, amoxicillin-clavulanic acid, ampicillin, azithromycin, ceftriaxone and erythromycin.
Primary safety endpoints for the Phase 2/3 study will be the proportion of subjects experiencing an adverse event (AE) and the proportion of subjects discontinuing study drug due to a related AE. The secondary efficacy endpoint is defined as clinical improvement on the last day of treatment (end of treatment response), and additional efficacy endpoints are defined as early clinical response at days 2-4 and clinical success (cure) at the short-term follow-up visit which is 10 days (+/– 4 days) after the last dose of treatment. For additional information about the Phase 2/3 pediatric study please visit clinicaltrials.gov.
In a completed Phase 1a study conducted in pediatric patients aged 12 to 17 years, solithromycin oral capsules were well tolerated and demonstrated a pharmacokinetic profile similar to that seen in adults. The Phase 1b portion of the study continues to enroll patients aged newborn to 17 years with suspected or confirmed bacterial infections. Patients will receive oral capsules, oral suspension or intravenous solithromycin dosed by weight once per day as add on therapy for up to 5 days. The study is open label and the primary endpoint will be to determine pharmacokinetics in the pediatric population. Safety data is also being collected. For additional information about the Phase 1b study please visit clinicaltrials.gov.
About the Cempra BARDA Agreement
In May 2013, Cempra entered into a five year agreement with the Biomedical Advanced Research and Development Authority of the U.S. Department of Health and Human Services (Contract No. HHS0100201300009C) for the evaluation and development of solithromycin for the treatment of bacterial infections in pediatric populations and infections caused by bioterror threat pathogens, specifically anthrax and tularemia. The BARDA agreement is a cost plus fixed fee development contract with a base performance segment plus four option segments. Under the agreement, $34.7 million has been previously awarded to fund the Phase 1a and 1b pediatric clinical trials as well as a portion of the Phase 2/3 startup costs.
About Cempra, Inc.
Cempra, Inc. is a clinical-stage pharmaceutical company focused on developing antibiotics to meet critical medical needs in the treatment of bacterial infectious diseases. Cempra's two lead product candidates are currently in advanced clinical development. Solithromycin (CEM-101) has successfully completed two Phase 3 clinical trials for community-acquired bacterial pneumonia (CABP) and is licensed to strategic commercial partner Toyama Chemical Co., Ltd., a subsidiary of FUJIFILM Holdings Corporation, for certain exclusive rights in Japan. Solithromycin is also in a Phase 3 clinical trial for uncomplicated urogenital urethritis caused by Neisseria gonorrhoeae or chlamydia. Cempra is contracted with BARDA for the development of solithromycin for pediatric use. Three formulations, intravenous, oral capsules and a suspension formulation are in a Phase 1b trial in children from birth to 17 years of age. Taksta™ is Cempra's second product candidate, which is being developed for acute bacterial skin and skin structure Infections (ABSSSI) and is also expected to be tested in an exploratory study for chronic oral treatment of refractory infections in bones and joints. Both products seek to address the need for new treatments targeting drug-resistant bacterial infections in the hospital and in the community. Cempra has also synthesized novel macrolides for non-antibiotic uses such as the treatment of chronic inflammatory diseases, endocrine diseases and gastric motility disorders. Cempra was founded in 2006 and is headquartered in Chapel Hill, N.C. For additional information about Cempra please visit www.cempra.com.
Please Note: This press release contains forward-looking statements regarding future events. These statements are just predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially. These risks and uncertainties include, among others: the costs, sources of funds, enrollment, timing, regulatory review and results of our studies and clinical trials and those of our strategic commercial partners; our and our strategic commercial partners' ability to obtain FDA and foreign regulatory approval of our product candidates; our ability to commercialize and launch, whether on our own or with a strategic partner, any product candidate that receives regulatory approval; our need to obtain additional funding and our ability to obtain future funding on acceptable terms; our anticipated capital expenditures and our estimates regarding our capital requirements; the unpredictability of the size of the markets for, and market acceptance of, any of our products, including solithromycin and Taksta; our ability to produce and sell any approved products and the price we are able to realize for those products; our ability to retain and hire necessary employees and to staff our operations appropriately; the possible impairment of, or inability to obtain, intellectual property rights and the costs of obtaining such rights from third parties; our ability to compete in our industry; our dependence on the success of solithromycin and Taksta; innovation by our competitors; and our ability to stay abreast of and comply with new or modified laws and regulations that currently apply or become applicable to our business. The reader is referred to the documents that we file from time to time with the Securities and Exchange Commission.