In a late-stage trial, Insmed’s small molecule DPP1 inhibitor cut yearly exacerbation rates by around 20% in bronchiectasis patients, according to topline data.
Insmed on Tuesday unveiled topline data from the Phase III ASPEN study demonstrating that its investigational DPP1 blocker brensocatib could significantly lessen pulmonary exacerbations in patients with non-cystic fibrosis bronchiectasis.
The New Jersey-based biotech’s shares soared more than 100% in reaction to the news, according to Seeking Alpha.
Topline data showed brensocatib’s 10-mg dose cut the annualized rate of pulmonary exacerbation by 21.1% compared with placebo, while a 25-mg dose resulted in a 19.4% decrease. Both effects met statistical significance, with p-values of 0.0019 and 0.0046, respectively, according to the company.
Brensocatib also aced its key secondary endpoints. Both doses significantly prolonged the time to the first exacerbation events and increased the patients’ odds of having no such episode over 52 weeks. The higher dose group also saw a significant improvement in lung function after 52 weeks.
ASPEN found brensocatib to be safe overall and well-tolerated. There was no excess of treatment-emergent adverse events (TEAE) in both active treatment arms. A total of seven brensocatib-treated patients died due to TEAEs, while 47 had to discontinue dosing.
Insmed CMO Martina Flammer in a statement said that the company is “incredibly excited” about these topline data “and what they may mean for patients.” The biotech will continue analyzing data from ASPEN and will build toward a regulatory submission for brensocatib, which it plans to submit in the fourth quarter of 2024. The U.S. product launch is slated for mid-2025, pending approval.
“These findings not only underscore our belief that brensocatib has the potential to transform the treatment landscape for bronchiectasis, but they also further validate DPP1 inhibition as a mechanism of action that may hold promise in other neutrophil-mediated diseases,” Flammer said.
Designed to be taken orally, brensocatib is a small molecule inhibitor of DPP1, an enzyme that activates neutrophil serine proteases triggering lung damage and inflammation in various chronic immune-driven lung diseases. In addition to bronchiectasis, Insmed is also developing brensocatib for chronic rhinosinusitis without nasal polyps.
Brensocatib, which previously received the FDA’s Breakthrough Therapy designation for bronchiectasis, is Insmed’s “most important product,” according to Mizuho analysts Graig Suvannavejh and Jerry Gong. In an investor note ahead of the ASPEN data drop, the analysts called the readout a “major binary event” for the biotech and one that “will largely shape Insmed’s future direction.”
Given Tuesday’s promising data and positive market reaction, brensocatib could provide Insmed access to what the Mizuho analysts see as a potential $1 billion-plus market opportunity, pending FDA approval.
Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.
