Pivotal Phase 3 Data from CONVERT Study of ALIS in Patients with Treatment-Refractory NTM Lung Disease Caused by MAC Published in American Journal of Respiratory and Critical Care Medicine
--As previously reported, study met primary endpoint of culture conversion (p<0.0001)--
--ALIS under FDA Priority Review with PDUFA action date of
NTM lung disease is a chronic, debilitating condition that can cause severe, permanent damage to the lungs. If approved by the
“The high rate of culture conversion achieved by patients in this study who received ALIS plus guideline-based therapy is remarkable, particularly given that these patients had previously failed treatment with the guideline-based therapy,” said
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. Culture conversion was achieved if patients had three consecutive monthly negative sputum cultures, with all sputum samples collected at each visit required to be culture-negative.
“The CONVERT study is a landmark clinical trial that is evaluating, for the first time, a treatment for NTM lung disease in a controlled, global, Phase 3 setting. We are very pleased that the study met its primary endpoint and we look forward to evaluating further data, including the durability of culture conversion for patients treated with ALIS,” said
In the study, rates of serious treatment-emergent adverse events were similar between treatment arms (20.2% for ALIS+GBT vs. 17.9% for GBT alone). Overall the rate of reported adverse events in the ALIS plus GBT arm was higher (98.2% for ALIS+GBT vs. 91.1% for GBT alone). TEAEs led to discontinuation of ALIS in 17.4% of patients. The most common TEAEs (occurring in ≥10% of patients) were primarily respiratory events and were predominately mild or moderate in nature. Most of these common events were initially reported in the first month of ALIS treatment and infrequently led to discontinuation of ALIS (dyspnea, 3.1%; dysphonia, 2.2%; all others <1%) or withdrawal from the study. Side effects commonly associated with intravenous use of amikacin, including hearing loss or renal impairment, were infrequent and generally similar between treatment groups with the exception of tinnitus (7.6% for ALIS+GBT vs. 0.9% for GBT alone).
Insmed’s new drug application for ALIS is currently under Priority Review by the
About CONVERT (INS-212) and INS-312
CONVERT is a randomized, open-label, global Phase 3 trial designed to confirm the culture conversion results seen in
About NTM Lung Disease
NTM lung disease is a rare and serious disorder associated with increased rates of morbidity and mortality. There is an increasing prevalence of lung disease caused by NTM and
The prevalence of human disease attributable to NTM has increased over the past two decades. In a decade long study (1997 to 2007), researchers found that the prevalence of NTM lung disease in the U.S. was increasing at approximately 8% per year and that NTM patients on
ALIS is a novel, inhaled, once-daily formulation of amikacin that is in late-stage clinical development and under regulatory review by the
About PARI Pharma and the eFlow® Electronic Nebulizer
ALIS is delivered by a novel inhalation device, the eFlow® Electronic Nebulizer, developed by PARI Pharma GmbH. eFlow is a quiet, portable nebulizer that enables efficient aerosolization of liquid medications, including liposomal formulations such as ALIS, via a vibrating, perforated membrane. Based on PARI's 100-year history working with aerosols, PARI Pharma is dedicated to advancing inhalation therapies by developing innovative delivery platforms and new pharmaceutical formulations that work together to improve patient care.
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Source: Insmed, Inc.