Insmed Announces Disappointing Results from Phase 2b CEDAR Study; Discontinues Hidradenitis Suppurativa Program
Key Highlights from the Report
- CEDAR Study Misses Efficacy Endpoints: Insmed’s Phase 2b CEDAR study evaluating brensocatib in adults with moderate to severe hidradenitis suppurativa (HS) failed to meet its primary and secondary efficacy endpoints at both the 10 mg and 40 mg dosing arms.
- Program Discontinued: Insmed will discontinue the development of brensocatib for HS following these results.
- Safety Profile Remains Positive: No new safety signals were identified, even at the highest studied dose (40 mg), and the drug was generally well tolerated.
- Placebo Outperformed Drug: At 16 weeks, the placebo arm showed a greater reduction in total abscess and inflammatory nodule count (57.1%) compared to the brensocatib 10 mg (45.5%) and 40 mg (40.3%) arms.
- Adverse Events: The incidence of any treatment-emergent adverse events (TEAEs) was comparable across the groups, with no increase in severe or serious adverse events in the brensocatib arms.
Details Investors Need to Know
The Phase 2b CEDAR study was a randomized, double-blind, placebo-controlled trial conducted at 72 sites globally, enrolling 214 patients. Participants were randomized equally to receive either 10 mg or 40 mg of brensocatib or placebo, once daily for 16 weeks. The primary endpoint was the percent change from baseline in total abscess and inflammatory nodule count at week 16.
The results are disappointing for Insmed and its investors, as the trial not only failed to demonstrate efficacy for brensocatib in HS, but the placebo arm outperformed both dosing arms of brensocatib. Specifically, at week 16, the reduction in abscess and nodule count was 45.5% for the 10 mg group, 40.3% for the 40 mg group, and 57.1% for placebo. These results led Insmed to the decision to halt further development of brensocatib in HS.
Safety signals were reassuring across the board. Any TEAEs were reported in 55.4% (10 mg), 42.9% (40 mg), and 45.7% (placebo) of participants. Severe TEAEs were rare (1.4% in 10 mg, none in 40 mg or placebo), and serious TEAEs occurred in 4.1% (10 mg), 1.4% (40 mg), and 1.4% (placebo).
Potential Price-Sensitive Information
- Pipeline Setback: The discontinuation of the HS program is a significant pipeline setback for Insmed, potentially impacting investor sentiment and share value. The CEDAR study was a proof-of-concept trial in a challenging indication due to the lack of established animal models, but its failure reduces near-term pipeline catalysts and may prompt a reassessment of the company’s valuation.
- No Immediate Safety Concerns: The lack of new safety signals may reassure stakeholders regarding brensocatib’s safety profile in other indications, but the efficacy miss is likely to overshadow this.
- Company’s Focus: Insmed will continue to focus on its other advanced programs, particularly in pulmonary and inflammatory conditions, which include two approved therapies for chronic lung diseases. The company’s early-stage portfolio remains broad, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue platforms.
Management Commentary
Dr. Martina Flammer, Chief Medical Officer of Insmed, stated, “While we are disappointed in the results, we hope that insights gained from this study will contribute to the broader scientific understanding of HS. We are grateful to the patients and investigators who participated in this study.”
Summary Table of Adverse Events (16-Week Placebo-Controlled Period)
|
Brensocatib 10 mg (N=74) |
Brensocatib 40 mg (N=70) |
Placebo (N=70) |
| Any TEAE, n (%) |
41 (55.4%) |
30 (42.9%) |
32 (45.7%) |
| Severe TEAE, n (%) |
1 (1.4%) |
0 |
0 |
| Serious TEAE, n (%) |
3 (4.1%) |
1 (1.4%) |
1 (1.4%) |
Forward-Looking Statements and Risks
Insmed cautioned investors that the forward-looking statements made in this press release involve substantial risks and uncertainties. These include potential changes in trial data interpretation, challenges in enrolling or retaining clinical trial participants, unexpected safety or efficacy concerns in other programs, and possible litigation. Investors should refer to Insmed’s Annual Report and SEC filings for a comprehensive list of risk factors.
Contact Information
- Investor Relations: Bryan Dunn, Vice President, Investor Relations – (646) 812-4030, [email protected]
- Media: Claire Mulhearn, Vice President, Corporate Communications – (862) 842-6819, [email protected]
Disclaimer: This article is for informational purposes only and does not constitute investment advice. Investors should conduct their own research or consult with their financial advisors before making investment decisions. The author and publisher are not responsible for any losses resulting from investment decisions based on this article.
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