Insmed Drug for Rare Pulmonary Disorder Meets Phase 2 Goals

Xconomy New York — 

Rare disease drug developer Insmed on Monday revealed data from a mid-stage trial of its daily pill for adults with non-cystic fibrosis bronchiectasis, a severe chronic pulmonary disorder with no FDA-approved therapies.

Patients with chronic inflammatory lung diseases end up with an overabundance of white blood cells called neutrophils in their airways. The Insmed drug, INS1007, is designed to inhibit an enzyme, dipeptidyl peptidase 1 (DPP1), which activates enzymes that are released by neutrophils and cause lung damage and inflammation.

In a Phase 2 test of the drug over 24 weeks, groups of patients with this form of bronchiectasis who received a 10 mg dose and a 25 mg dose both achieved the trial’s primary goal of reducing the time to first pulmonary exacerbation, an episode of worsening of respiratory problems, compared to a placebo. The 256 patients who participated had at least two documented pulmonary exacerbations in the 12 months prior to screening.

Shares of Bridgewater, NJ-based Insmed (NASDAQ: INSM) rose 40 percent on the news to about $29.

About 340,000 to 520,000 people in US have bronchiectasis unrelated to cystic fibrosis. The condition is characterized by a cycle of infection, inflammation, and lung tissue damage that results in the bronchi—the pathways that move air to and from the lungs—becoming permanently dilated. That leads to frequent pulmonary exacerbations which may require antibiotics and hospitalization to manage.

INS1007, in addition to meeting the trial’s main goal, also reduced the frequency of pulmonary exacerbations that patients experienced, a secondary goal of the study, compared to those taking a placebo. Those who received the 10 mg dose experienced a 36 percent reduction; those who received 25 mg reported a 25 percent reduction.

SVB Leerink analyst Joseph Schwartz in a research note said the results of the trial surpassed expectations given the variety of causes of the condition. And although the higher dose group didn’t see a greater reduction in the frequency of pulmonary exacerbations than the lower dose group, Schwartz said this lack of dose response “isn’t uncommon for many drugs which offer a clinical benefit.”

The most common side effects reported by patients in the trial were cough, headache, an increase in mucus secreted in the respiratory tract, difficult breathing, fatigue, and upper respiratory tract infection. Those taking the 10 mg or 25 mg doses also reported higher rates of side effects believed to be associated with the drug, periodontal disease and hyperkeratosis, than those treated with a placebo.

Insmed plans to present more data from the study at an upcoming medical meeting.