SPR720:  An Oral Antibiotic Designed to Treat Nontuberculous Mycobacterial Pulmonary Disease, a Rare Orphan Disease

Nontuberculous Mycobacterium (NTM) pulmonary disease is a growing global health concern and major unmet medical need due to a lack of new medications being developed to combat these bacteria.

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. It is estimated that approximately 130,000 patients suffer from NTM in the U.S. and Europe, a figure that is growing at a rate of 8% annually. The elderly and people with compromised immune or lung function are at greatest risk, as are patients with bronchiectasis for whom it is estimated that up to 50% may also have active NTM pulmonary disease.

Treatment of NTM pulmonary disease requires prolonged therapy (continuing for approximately 12 to 24 months) with a combination of drugs approved for other infections and is frequently complicated by tolerability and/or toxicity issues. There are currently no oral antibiotics specifically approved for use to treat NTM pulmonary disease. NTM is also associated with high healthcare costs and high mortality. In 2014, the annual cost in the U.S. alone was estimated at $1.7 billion.

Spero Therapeutics is developing SPR720, a novel, investigational oral agent to treat NTM pulmonary disease.