What have been the main milestones from Insmed in 2023, and what are the catalysts ahead after the successful completion of the Phase 3 ARISE and ASPEN studies?

2023 was an exciting year for Insmed, as we laid the foundation for what will be a transformational 2024. We made significant progress on both the commercial and clinical fronts, while simultaneously growing our business and team.

In 2023, we experienced the strongest year of commercial performance for our first approved therapy, five years after its initial US launch. Currently approved in the US, EU and Japan for a rare and serious lung disease, we are also investigating it in a broader patient population in two Phase 3 studies—ARISE and ENCORE. Last September, we reported positive topline data from ARISE. ENCORE remains ongoing, and we are working to finalize enrollment goals. We anticipate sharing topline data from ENCORE in 2025.

Last March, we announced enrollment completion of more than 1,700 adult patients in our Phase 3 ASPEN study assessing brensocatib, our lead pipeline candidate, in non-cystic fibrosis bronchiectasis. We plan to share topline data from ASPEN in the latter part of Q2 2024. If positive, this could mark a monumental advance for the more than one million diagnosed patients around the world who have no approved treatment specifically for bronchiectasis today. Beyond bronchiectasis, we are studying brensocatib in chronic rhinosinusitis without nasal polyps (CRSsNP) and hidradenitis suppurativa (HS). We initiated the Phase 2 BiRCh study in CRSsNP in late 2023 and plan to initiate a Phase 2 study in HS in the second half of 2024, pending positive results from the ASPEN study.

In October 2023, we announced encouraging blended, blinded data from our two ongoing Phase 2 studies of treprostinil palmitil inhalation powder (TPIP), the next candidate in our pipeline, in pulmonary hypertension associated with interstitial lung disease (PH-ILD) and pulmonary arterial hypertension (PAH). We plan to share topline data from the PH-ILD study in Q2 of this year, with topline data in PAH in 2025.

Lastly, in 2023, our research teams continued to advance pre-clinical studies across a range of devastating rare diseases by leveraging our novel gene therapy, AI-driven protein engineering, and proprietary manufacturing technologies. Additionally, last June, we acquired Adrestia Therapeutics out of Cambridge, UK, adding a world-class team and a leading synthetic rescue platform to our internal capabilities. Can you expand on the recent partnership with Google Cloud?

Last October, we announced a collaboration with Google Cloud to transform the drug discovery, development and commercialization landscape of the life sciences industry through the use of generative AI. Combining Google Cloud's AI technology and our own expertise in serious and rare diseases, our goal is to reduce the time and increase the efficiency of developing and delivering new medicines for patients. Potential applications include expediting the identification of potential therapeutic candidates for our early-stage pipeline, drafting clinical study reports and other labor-intensive regulatory documents, and helping to sift through large quantities of scientific literature, as well as internal enterprise data. What are the main challenges and opportunities you anticipate for pharmaceutical companies in the US in 2024?

Global financial markets, geopolitical events, regulatory and legislative changes, and evolving technologies will continue to influence our sector in the year ahead. I believe that companies in a strong financial position, the right talent and the ability to truly innovate for patients have enormous opportunities to succeed. Conversely, those in more precarious positions or that lack the ability to adapt to change may struggle to navigate these many complex external factors. What will be the main priorities for Insmed in 2024?

At the moment, we are laser focused on preparing to report data from our Phase 2 PH-ILD and Phase 3 ASPEN studies. If ASPEN is positive, we will immediately begin preparing regulatory submissions and expanding our team and infrastructures to support potential future launches in the US, EU and Japan. Simultaneously, we continue to progress a wide array of early- to mid-stage studies across our pipeline, and we remain committed to advancing each of these trials on behalf of patients in need. It goes without saying that underscoring all our great work is an incredible team of colleagues and a unique and very special culture. Preserving and strengthening this culture remains a top priority as we grow.