Spero Receives Orphan Drug Designation for Nontuberculous Mycobacterial Treatment

Spero Therapeutics announced 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.

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.

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