Shares of Insmed Inc. (NASDAQ: INSM) shot ahead over 130% in premarket trading on Tuesday and are still holding triple-digit percentage gains in afternoon trading as the drugmaker focused on deadly rare diseases said it hit a milestone by meeting the primary endpoint in a phase 3 study.
Moreover, Insmed thinks the data is strong enough to convince the Food and Drug Administration to award its inhaled drug called ALIS, (an acronym for “Amikacin Liposome Inhalation Suspension) for a quick approval.
In Insmed’s 336-patient CONVERT study, ALIS, a once-daily inhaled drug, was evaluated in adult patients with nontuberculous mycobacterial (NTM) lung disease caused by mycobacterium avium complex (MAC) that have failed other therapies. The patients were randomized 2-to-1 to either a group receiving ALIS plus standard of care or standard of care alone.
ALIS met its primary endpoint of culture conversion by month six with statistical and clinical significance. The data showed that adding ALIS to standard of care eradicated signs of NTM caused by MAC in sputum in 29% of patients, versus only 9% in the group only receiving standard of care.
Insmed also took a look at some secondary endpoints, namely the six-minute walk test that is often viewed as a barometer of efficacy. Broadly, there was no statistical difference between the two groups of patients.
A more detailed look at pre-defined measures did show, however, an improvement in performance for patients that achieved culture conversion in either arm versus those that did not.
Time to culture conversion was also significantly shorter for those in the combo group than those that converted in the standard of care (SOC) arm. Lower percentages of death and cardiac disorders were reported in the ALIS+SOC arm compared to SOC alone.
On the downside, at 20.2%, those in the ALIS arm demonstrated a modestly higher incidence of adverse events (17.9% for SOC only group).
Insmed intends to seek an accelerated approval of ALIS based upon the FDA previously awarding the drug a vaunted “Breakthrough Therapy” designation, "Fast Track” status and another designation as a Qualified Infectious Disease Product under the Generating Antibiotic Incentives Now Act.
There is currently no FDA-approved drug for NTM lung infections, a hard-to-treat condition caused by bacteria found in soil and/or water often requiring a cocktail of drugs to combat.
