What happened

Shares of Insmed (NASDAQ:INSM), a commercial-stage biotech focused on rare diseases, had risen 41% as of 11:50 a.m. EST on Monday. The bullish move is linked to the release of positive top-line results from its phase 2 WILLOW trial today.

So what

Insmed's WILLOW trial was designed to test the efficacy, safety, and pharmacokinetics of an experimental compound known as INS1007 as a potential treatment for non-cystic fibrosis bronchiectasis (NCFBE).

Here are the key takeaways from today's news update:

  • The primary endpoint of the 24-week trial was achieved. Specifically, INS1007 showed a statistically significant improvement in time to first exacerbation for both its 10 mg dosage and its 25 mg dosage when compared to placebo. The p-values of the dosages were 0.027 and 0.044, respectively.
  • A key secondary endpoint was also achieved: Patients who were treated with INS1007 demonstrated a statistically significant reduction in the frequency of pulmonary exacerbations when compared to those treated with placebo. The reduction was 36% in the 10 mg arm and 25% in the 25 mg arm. 
  • INS1007 was well tolerated by patients at both dosage levels.
  • Management plans to move INS1007 into a phase 3 trial.
Ben Franklin on hundred dollar bill surrounded by pills

Image source: Getty Images.

Dr. James Chalmers, the study's lead investigator, commented:

These results are incredibly encouraging and highlight the potentially important role INS1007 may play in the management of bronchiectasis. Today, many bronchiectasis patients suffer from persistent symptoms and frequent exacerbations, with no pharmaceutical therapies available that are approved to help them manage this disease. There is an urgent need for approved, effective therapies that can break the vicious cycle of inflammation, lung damage, and infection for these patients.

Given the news, it's easy to understand why shares are flying high today.

Now what

The WILLOW study results should give investors confidence that INS1007 could be the real deal. With $1 billion in estimated peak sales potential, if INS1007 can live up to its potential, it would certainly be needle moving for Insmed.

This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.