Phase III CONVERT study showed that the addition of ALIS (amikacin liposome inhalation suspension) to guideline-based therapy eliminated evidence of nontuberculous mycobacterial lung disease caused by mycobacterium avium complex.- Ismed.

The global CONVERT study met its primary endpoint of culture conversion by Month 6 with statistical and clinical significance. The study demonstrated that the addition of ALIS (Amikacin Liposome Inhalation Suspension) ( formerly Arikayce) from Ismed to guideline-based therapy (GBT) eliminated evidence of NTM (Nontuberculous Mycobacterial (NTM) Lung Disease) lung disease caused by MAC in sputum by Month 6 in 29% of patients, compared to 9% of patients on GBT alone (p < 0.0001). The trial was powered to detect a treatment effect of 15% between the two treatment groups.

The CONVERT study enrolled 336 adult patients with NTM lung disease caused by MAC (Mycobacterium Avium Complex) who were refractory to at least six months of GBT. Patients were randomized 2:1 to receive ALIS plus GBT versus GBT alone. The primary endpoint was the proportion of patients achieving culture conversion by Month 6.

Insmed plans to pursue accelerated approval of ALIS under subpart H based on the data from the CONVERT study, which will be reviewed by the Division of Anti-Infective Products. FDA previously granted this product breakthrough therapy designation and fast track status and designated ALIS as a qualified infectious disease product (QIDP) under the Generating Antibiotic Incentives Now (GAIN) Act.

The Company also reported top-line data for several secondary endpoints as of the 6-month timepoint. Top-line data for the 6-minute walk test indicates no statistically significant difference between patients in the two arms. However, an analysis of these data (per a pre-specified endpoint) shows that patients who achieved culture conversion in either arm demonstrated an improvement in 6-minute walk distance when compared to patients who did not culture convert (p=0.0108). Top-line data for the secondary endpoint of time to conversion demonstrated that patients on GBT took approximately 30% longer to convert when compared to patients on ALIS plus GBT (p < 0.0001). The Company is continuing its analysis of the impact of conversion on a variety of other clinical measures.

Comment: MAC has a population of about 26 400- 34 400 patients in US/Japan and five major EU countries.

Comment: The company plans to commercialize ALIS on its own, if the product wins accelerated approval from the FDA.