INSMED Inc Q1 FY2025 earnings call

• Brensocatib: The full results of the Phase 3 ASPEN trial were published in the New England Journal of Medicine. The FDA review of the NDA filing for brensocatib in bronchiectasis is ongoing with the PDUFA date of August 12. Launch readiness progress includes over a million unique visits to the disease state awareness website and engagement with payers. International regulatory reviews for brensocatib in Europe, U.K., and Japan are ongoing.
• TPIP: The Phase 2 trial of TPIP in PAH is progressing toward a topline readout, with the expected timing narrowed to June. Focus is on PVR reduction (20% placebo-adjusted reduction as a clear win, 25% as a homerun) and a directional benefit of 15-20 meters in 6-minute walk distance.
• ARIKAYCE: Strong commercial performance with double-digit growth in all regions for the sixth consecutive quarter. Cash balance as of the end of the quarter was approximately $1.2 billion in cash, cash equivalents, and marketable securities.
• Next Gen DPP-1 Inhibitors: Research ongoing for indications like COPD and rheumatoid arthritis, with next gen molecules in development, anticipating entry into the clinic as soon as next year.

ARIKAYCE delivered double-digit year-over-year revenue growth in Q1 across all geographic regions, with 50% growth in Japan and Europe, and 14% growth in the U.S. for the quarter. ARIKAYCE's revenue contribution to the company's overall performance was significant, with the 2025 full-year net revenue guidance for ARIKAYCE set at $405 million to $425 million.

• ARIKAYCE 2025 full-year net revenue guidance: $405 million to $425 million.
• TPIP topline readout expected in June.
• Brensocatib FDA decision expected by the August 12 PDUFA date.

• Geopolitical uncertainties and tariffs could impact the business, but Insmed is well-positioned with U.S. intellectual property domiciled in the U.S. and ongoing efforts to expand U.S. manufacturing. Impact on business is estimated to be in the single-digit millions annually over the next few years.
• Variability in clinical trial results and regulatory outcomes for brensocatib, TPIP, and other programs.

Q: How does potential MFN legislation factor into pricing for brensocatib globally?

A: It's hard to speculate on the outcome, but Insmed is in a strong position to set prices in the U.S. first and then adjust for other regions, giving flexibility to respond to the environment.

Q: What trends are picked up from the bronchiectasis disease awareness website?

A: High interest and enthusiasm from patients and physicians, with over 60% of views on the New England Journal of Medicine publication coming from the public, indicating patient, family, and caregiver engagement.

Q: How is the TPIP trial measuring PVR and 6-minute walk distance?

A: PVR is the most important measure, with a 20% placebo-adjusted reduction from baseline as a clear win and 25% as a homerun. 6-minute walk distance is a less specific measure but a directional benefit of 15-20 meters is hoped for.

Q: Any surprises in the mid-cycle review meeting for brensocatib?

A: No surprises in the mid-cycle review meeting. For TPIP, divergence in measures is possible but not a major concern as the prostanoid class's vasodilatation is well-established.

Q: Thoughts on the HS trial's futility analysis?

A: The futility evaluation of the first 100 patients in the Phase 2 HS trial will look for a signal of efficacy, with an aim to see a 40% reduction in abscess and nodule count from baseline versus placebo to inform Phase 3 planning.

Q: Impact of Sarepta's DMD gene therapy patient death on Insmed's gene therapy strategy?

A: Insmed's gene therapy strategy uses intrathecal delivery, which has safety benefits, and they are titrating up slowly to ensure safety, with preclinical models showing good transduction and safety benefits.