Date: 2017-09-05
Type of
information: Results
phase: 3
Announcement: results
Company: Insmed Limited (USA - NJ)
Product: Arikayce® (amikacin sulfate - liposomal amikacin for inhalation)
Action
mechanism:
- antibiotic. Arikayce® is a form of the antibiotic amikacin, which is enclosed in liposomes. This advanced pulmonary liposome technology prolongs the release of amikacin in the lungs while minimizing systemic exposure. The treatment uses biocompatible lipids endogenous to the lung that are formulated into small (0.3 micron), charge-neutral liposomes. Arikayce® is administered once-daily using an optimized, investigational eFlow® Nebulizer System manufactured by PARI Pharma GmbH, a novel, highly efficient and portable aerosol delivery system. Arikayce® has received Orphan Drug Designation from the EMA for both NTM lung infections and for Pseudomonas aeruginosa lung infections. In addition, Arikayce® has received Breakthrough Therapy, Orphan Drug, Qualified Infectious Disease Product (QIDP) and Fast Track designations from the FDA for the treatment of NTM lung infections.
Disease: treatment-refractory Nontuberculous Mycobacterial (NTM) lung disease caused by Mycobacterium Avium Complex (M
Therapeutic
area: Infectious diseases
Country:
Trial
details:
Latest
news:
- • On September 5, 2017, Insmed announced top-line data from its Phase 3 CONVERT study. 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 to guideline-based therapy (GBT) eliminated evidence of NTM 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 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 prespecified 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.
- Safety and Tolerability In the study, serious treatment emergent adverse events were similar between treatment arms. There were no distinctions between treatment arms due to hearing loss or renal impairment, side effects commonly associated with intravenous use of amikacin. The overall dropout rate was 16.1%, with an 8.9% dropout rate in the GBT arm and a 19.6% rate in the ALIS plus GBT arm. Overall the rate of reported adverse events in the ALIS plus GBT arm was higher and these events were predominately mild or moderate in nature and generally declined after the second month of treatment. These findings are consistent with the Phase 2 study results and demonstrate adverse events similar to those seen in other clinical studies of inhaled antibiotics.
Is
general: Yes
