In 2016, Insmed bet $30 million upfront—and millions more in milestones—on a pulmonary drug from AstraZeneca. Now, that bet looks to be paying off as the New Jersey biotech reports positive topline data in a phase 2 study of non-cystic fibrosis bronchiectasis (NCFBE), a chronic condition in which the bronchi are damaged from a cycle of infection and inflammation.
After 24 weeks of treatment, two doses of the drug, INS1007, beat placebo at staving off patients’ first pulmonary exacerbation—an episode during which their symptoms, such as chronic cough and repeated lung infections, worsen. The phase 2 study enrolled 256 patients who suffered at least two pulmonary exacerbations in the year before they were screened for the trial. The study also found that both dose levels, 10 mg and 25 mg, reduced the number of exacerbations. The lower dose did slightly better, reducing 36% of these episodes, compared to 25% for the higher dose.
Evercore ISI analysts dubbed the data a "surprise," in a note, saying "Few investors expected a positive outcome of this trial, but many say a positive risk-reward with downside protection provided by Arikayce," its inhaled formulation of an old antibiotic. It was approved in 2018 to treat mycobacterium avium complex (MAC) lung disease as part of combo regimen for patients who don't respond to conventional treatment.
The company's stock leaped 46% on the news when the market opened Monday morning. It will present detailed results at an upcoming medical meeting.
Given as a daily pill, INS1007 is a selective, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), an enzyme that plays a role in the development of neutrophils in the bone marrow. These white blood cells collect in the airways of people who have chronic inflammatory lung diseases. By blocking DPP1 and its activation of enzymes called neutrophil serine proteases, Insmed believes that INS1007 may tamp down on the damage caused by diseases like NCFBE.
"This molecule represents a novel, potentially first-in-class mechanism that utilizes an anti-inflammatory approach to treat the debilitating cycle of inflammation, infection, and lung damage associated with NCFBE," said Insmed Chief Medical Officer Martina Flammer, M.D., in a statement.
"Importantly, in addition to achieving the primary and a key secondary endpoint, we saw significant reductions in sputum neutrophil elastase, an important biomarker that reflects the mechanism of action of INS1007,” Flammer added. “These data provide a strong rationale for continued development in this disease and potentially other neutrophil-driven inflammatory conditions. We look forward to further analyzing the data and discussing next steps with regulatory authorities."
Insmed licensed INS1007 from AstraZeneca in October 2016 for $30 million upfront and $120 million in milestones down the line. Under that deal, AstraZeneca also holds the cards to negotiate a future agreement with Insmed to sell the drug in chronic obstructive pulmonary disease or asthma.