The second orphan drug indication discussed by Mr. Lewis was Arikace for non-TB mycobateria lung infections. Currently there are no approved treatments for it and according to Mr. Lewis, most treatments are off label with significant toxicities and limitations (including a systemic formulation of amicacin). Non-TB mycobateria lung infections is a chronic, deliberating progressive infection that can lead to lengthy and repeated hospitalizations. Mr. Lewis also noted that with average hospital stays of 10 days, Insmed will examine the efficacy of Arikace in Non-TB mycobacteria lung infections with the premise that improved efficacy will also reduce hospitalizations and thereby reduce overall costs of care. A concept Insmed refers to as Orphan 2.0 – value for your money.
The company is currently conducting a phase 2 study comparing Arikace to placebo in patients also receiving background therapy. The primary efficacy endpoint will be reduction bacterial density at the end of the 12 week study. Mr. Lewis hopes to have results of the study by the end of the year.
Mr. Lewis also noted that the study was designed in communication with both regulators and key opinion leaders to best create a study design that they hope will lead to regulatory approval. For example, the study also has numerous secondary endpoints (eg, time to sputum conversion, time to pulmonary exacerbation, change in PRO/QoL, 6 minute walk test, change in clinical signs/symptoms, safety) to hopefully encourage the drug to be approved if the drug proves to be effective. We look forward to see that data.