Paratek Pharmaceuticals Announces FDA Orphan Drug Designation for NUZYRA (omadacycline) for the Treatment of Infections Caused by Nontuberculous Mycobacteria

Paratek Pharmaceuticals, Inc. (Nasdaq: PRTK), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel life-saving therapies for life-threatening diseases or other public health threats for civilian, government and military use, today announced that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for NUZYRA® (omadacycline) for the treatment of infections caused by Nontuberculous Mycobacteria (NTM). This orphan drug designation includes NTM pulmonary disease caused by Mycobacterium abscessus complex (MABc), which is the focus of an ongoing Phase 2b study initiated by Paratek.

The FDA's Office of Orphan Drug Products grants orphan status to drugs and biologics that demonstrate promise for the treatment of diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation provides Paratek with certain development incentives, including tax credits for qualified clinical testing, exemptions from certain FDA application fees, and potential market exclusivity, if approved.

"The orphan drug designation is an important regulatory milestone that further validates our efforts to investigate the utility of NUZYRA, a once-daily, broad-spectrum antibiotic currently approved for acute bacterial skin and skin structure infections and community-acquired bacterial pneumonia, as a potential therapy option for patients afflicted with M.abscessus pulmonary disease, an orphan disease for which there are no approved therapies," said Randy Brenner, Chief Development and Regulatory Officer of Paratek. "Earlier this year, we initiated our Phase 2b clinical study that is designed to examine the efficacy and safety of NUZYRA in patients with M.abscessuspulmonary disease. These clinical data will continue to build upon the expanding data and publications about NUZYRA and its potential utility in NTM abscessus.

About NTM Abscessus

Pulmonary infections caused by M. abscessus, an orphan disease with no FDA-approved antibiotic therapies, affects approximately 11,500 patients in the United States. Patients with pulmonary disease caused by M. abscessus have a myriad of symptoms including severe fatigue, fever, cough, and shortness of breath. The standard of care typically involves a combination of multiple antibiotics, most of which are intravenous, which can often require life-long treatment that is complicated by long-term tolerability challenges and multiple adverse events.

About the Phase 2b NTM Study

The Phase 2b study is a placebo-controlled, randomized monotherapy study of NUZYRA in patients with M.abscessus pulmonary disease who are in the early treatment phase and are not receiving other antibiotic treatments. The U.S.-based study will enroll approximately 75 subjects, randomized in a 1.5 to 1 ratio. The primary study endpoints are improvement in symptoms and safety and tolerability following 12 weeks of treatment. Due to the small numbers of patients with this orphan disease, Paratek expects the study will take about two years to complete enrollment.

About Paratek Pharmaceuticals, Inc.

Paratek Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel life-saving therapies for life-threatening diseases or other public health threats for civilian, government and military use.

The company's lead commercial product, NUZYRA® (omadacycline), is a once-daily oral and intravenous antibiotic available in the U.S. for the treatment of adults with community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections. A modernized tetracycline, NUZYRA is specifically designed to overcome tetracycline resistance and exhibits activity across a spectrum of bacteria, including Gram-positive, Gram-negative, atypicals, and other drug-resistant strains.

Paratek has a collaboration agreement with Zai Lab for the development and commercialization of omadacycline in the greater China region and retains all remaining global rights.

Paratek exclusively licensed U.S. rights and rights to the greater China territory for SEYSARA® (sarecycline), a once-daily oral therapy for the treatment of moderate to severe acne vulgaris, to Almirall, LLC (Almirall). Paratek retains the development and commercialization rights for sarecycline in the rest of the world.

In 2019, Paratek was awarded a contract from BARDA, valued at ~$285 million, to support the development and U.S.-based manufacturing of NUZYRA for the treatment of pulmonary anthrax.

About NUZYRA

NUZYRA® (omadacycline) is a novel once-daily antibiotic with both oral and intravenous (IV) formulations for the treatment of community-acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections (ABSSSI). A modernized tetracycline, NUZYRA is specifically designed to overcome tetracycline resistance and exhibits activity across a spectrum of bacteria, including Gram-positive, Gram-negative, atypicals, and other drug-resistant strains.