Spero Therapeutics Receives FDA Orphan Drug Designation for SPR720 for the Treatment of Nontuberculous Mycobacterial (NTM) Infection

CAMBRIDGE, Mass., March 11, 2020 (GLOBE NEWSWIRE) -- Spero Therapeutics, Inc. SPRO, a multi-asset clinical-stage biopharmaceutical company focused on identifying, developing and commercializing treatments in high unmet need areas involving multi-drug resistant bacterial infections and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for oral SPR720 for the treatment of nontuberculous mycobacterial (NTM) infection.

"We are happy to receive this important designation from the FDA, which highlights the critical unmet need that SPR720 is being developed to address," said Ankit Mahadevia, M.D., CEO of Spero Therapeutics.  "We look forward to our continued work with the FDA to advance SPR720 for a population of patients suffering from NTM pulmonary disease in need of novel treatments."

In December 2019, Spero announced positive preliminary data from its Phase 1 clinical trial of SPR720, which was designed as a double-blind, placebo-controlled clinical trial to assess the safety, tolerability and pharmacokinetics (PK) of SPR720 at single and multiple ascending doses in healthy volunteers.  These results indicated that SPR720 in healthy volunteers was generally well-tolerated at doses up to 1000 mg over the maximum studied duration of 14 days, with a PK profile that Spero believes supports SPR720's further development as an oral agent for the treatment of NTM pulmonary disease. Spero expects to present final data from the SPR720 Phase 1 clinical trial at a medical conference during 2020.  Spero plans to meet with the FDA in the first half of 2020, submit an investigational new drug (IND) application for SPR720 to the FDA in the second half of 2020 and, subject to FDA acceptance of the IND, initiate a dose-ranging Phase 2a clinical trial evaluating SPR720 in patients with NTM pulmonary disease due to Mycobacterium avium complex (MAC) in the second half of 2020. 

The FDA's Office of Orphan Products Development (OOPD) grants orphan designation status to a drug that is intended to treat a rare disease or condition that affects fewer than 200,000 persons in the United States.  Orphan drug designation provides certain benefits and incentives that may include tax credits towards the cost of clinical trials and prescription drug user fee waivers and the potential for seven years of market exclusivity in the United States upon regulatory approval.

About SPR720
SPR720 represents a novel class of antibacterial agents that target enzymes essential for bacterial DNA replication. SPR720 was acquired from Vertex and is currently under development by Spero as an oral therapy for the treatment of non-tuberculous mycobacterial (NTM) pulmonary disease, a rare orphan disease.  NTM are ubiquitous environmental pathogens that can cause progressive lung damage and respiratory failure, particularly in patients with compromised immune systems or underlying pulmonary disorders. Although rare, the incidence of NTM pulmonary disease is increasing worldwide. Treatment of NTM pulmonary disease requires prolonged therapy (continuing for approximately 12 to 24 months) with a combination of mostly unapproved drugs and is frequently complicated by tolerability and/or toxicity issues. Additionally, there are currently no oral antibiotics specifically approved for use to treat NTM pulmonary disease. Thus, if successfully developed, SPR720 has the potential to address an important unmet need as the first oral antibiotic approved for the treatment of this debilitating disease.  Under Spero's collaboration with Gates MRI, SPR720 will also be developed for the treatment of Mycobacterium tuberculosis (Mtb) infections in select economically disadvantaged countries. Tuberculosis is a priority pathogen as defined by the World Health Organization with it being one of the top ten causes of death worldwide, and a situation where resistance is increasing and current treatment approaches are not optimal.  Spero believes that its intellectual property portfolio for SPR720 will provide protection globally, including in the United States and Europe, through 2033. SPR720 has been granted Qualified Infectious Disease Product (QIDP) designation by the U.S. Food and Drug Administration (FDA) for the treatment of lung infections caused by non-tuberculous mycobacteria and lung infections caused by Mycobacterium tuberculosis (Mtb) and orphan drug designation by the FDA for the treatment of nontuberculous mycobacterial (NTM) infection.

SPR720 Research Support
Research reported in this publication was partially supported by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, under Award Number R44AI131749. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

About Spero
Spero Therapeutics, Inc. is a multi-asset, clinical-stage biopharmaceutical company focused on identifying, developing and commercializing novel treatments for multidrug-resistant (MDR) bacterial infections and rare diseases. 

Spero's lead product candidate, tebipenem HBr (tebipenem pivoxil hydrobromide; formerly SPR994), is designed to be the first oral carbapenem-class antibiotic for use in adults to treat MDR Gram-negative infections. 

Spero is also advancing SPR720, its novel oral therapy product candidate designed for the treatment of rare, orphan disease caused by non-tuberculous mycobacterial (NTM) pulmonary disease. 

Spero also has a platform technology known as its Potentiator Platform that includes an IV-administered product candidate, SPR206, being developed to treat MDR Gram-negative infections in the hospital setting. 

For more information, visit https://sperotherapeutics.com. 

Forward Looking Statements 
This press release may contain forward-looking statements. These statements include, but are not limited to, statements about Spero's expectation that positive results from a single pivotal Phase 3 clinical trial of tebipenem HBr and ancillary supportive studies to be conducted in parallel with the Phase 3 trial will support the approval of tebipenem HBr; the design, initiation, timing, progress and results of Spero's preclinical studies and clinical trials and its research and development programs, including the timing of Spero's regulatory meeting with the FDA regarding SPR720, the timing of Spero's IND submission with the FDA regarding SPR720, the commencement of Spero's planned Phase 2a clinical trial of SPR720 and the commencement of Spero's planned Phase 1 bronchoalveolar lavage (BAL) clinical trial assessing the penetration of SPR206 into the pulmonary compartment; management's assessment of the results of such preclinical studies and clinical trials; the timing of clinical data, including the availability of pharmacokinetic data from the lead-in cohort in the Phase 3 clinical trial of tebipenem HBr, final data from the Phase 1 clinical trial of SPR720 and final data from the Phase 1 clinical trial of SPR206; and Spero's cash forecast and anticipated expenses, anticipated payments under Spero's agreement with Everest Medicines, potential payments under Spero's agreement with BARDA, the sufficiency of its cash resources and the availability of additional non-dilutive funding from governmental agencies beyond any initially funded awards. In some cases, forward-looking statements can be identified by terms such as "may," "will," "should," "expect," "plan," "aim," "anticipate," "could," "intent," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including whether the FDA will accept a single pivotal study for approval of tebipenem HBr; whether results obtained in preclinical studies and clinical trials will be indicative of results obtained in future clinical trials; whether Spero's product candidates will advance through the preclinical development and clinical trial process on a timely basis, or at all, taking into account the effects of possible regulatory delays, slower than anticipated patient enrollment, manufacturing challenges, clinical trial design and clinical outcomes; whether the results of such trials will warrant submission for approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies; whether Spero will satisfy all of the pre-conditions to receipt of the development milestone payment under its agreement with Everest Medicines; whether BARDA elects to exercise its second option under Spero's agreement with BARDA; whether Spero's cash resources will be sufficient to fund its continuing operations for the periods and/or trials anticipated; and other factors discussed in the "Risk Factors" set forth in filings that Spero periodically makes with the U.S. Securities and Exchange Commission. The forward-looking statements included in this press release represent Spero's views as of the date of this press release. Spero anticipates that subsequent events and developments will cause its views to change. However, while Spero 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 Spero's views as of any date subsequent to the date of this press release.

Spero Investor and Media Contact: 
Sharon Klahre 
Senior Director, Investor Relations
857-242-1547 
IR@sperotherapeutics.com