Insmed Inc. (NASDAQ: INSM) saw its shares more than double early on Tuesday after the firm released late-stage results from its clinical study in the treatment of nontuberculous mycobacteria (NTM) lung disease. Specifically, the Phase 3 Convert 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.
For some quick background: NTM lung disease is an infection that can slowly scar and damage lungs. Although this disease is somewhat rare, (about two out of every 100,000) it can take as long as two years of ongoing treatment to clear it up.
As a result, Insmed plans to pursue accelerated approval of ALIS. The U.S. Food and Drug Administration (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 firm also reported top-line data for several secondary endpoints as of the six-month timepoint. Top-line data for the six-minute walk test indicates no statistically significant difference between patients in the two arms. However, an analysis of these data shows that patients who achieved culture conversion in either arm of the study demonstrated an improvement in six-minute walk distance when compared to patients who did not culture convert. 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.
Will Lewis, president and CEO of Insmed, commented:
We consider these compelling top-line data to be a remarkable accomplishment in a rare disease state with no currently approved therapies. We are particularly encouraged by the consistency of these data when compared with our Phase 2 study results, and look forward to additional data as the Convert study continues over the next two years. We want to thank all the patients who participated in this trial around the world as well as the physicians who supported them. Treatment of this serious and potentially debilitating disease is an unmet medical need, and we expect these important data will enable us to submit for accelerated approval.
Shares of Insmed traded up about 124% Tuesday morning at $27.50, with a consensus analyst price target of $25.43 and a 52-week range of $10.21 to $29.52.