2020 Annual Report
As Insmed enters a new phase of growth in 2021, we carry forward the strong momentum of a successful 2020, marked by outstanding accomplishments across our three pillars: ARIKAYCE, brensocatib, and TPIP. We also continue to progress a robust pipeline of earlier-stage research that we hope will enable us to transform the lives of patients for years to come.
ARIKAYCE is the cornerstone of our business and a product we developed entirely in-house, from laboratory to commercialization. It is the first and only approved treatment for its indication, and is now being studied in a clinical trial program for an additional indication.
In 2020, while the COVID-19 pandemic posed unprecedented challenges to the ways we typically engage with healthcare professionals and patients, our U.S. business remained steady. Our field-based teams quickly pivoted to an entirely remote and, later, a hybrid model to continue to provide critical support and information to our customers—uninterrupted.
We also saw substantial progress in our global expansion plans for ARIKAYCE. In October of 2020, Insmed received marketing authorization from the European Commission for ARIKAYCE. We have since secured reimbursement and launched ARIKAYCE in Germany and the Netherlands, and we expect reimbursement decisions across Europe to continue throughout 2021 and into 2022.
Most recently, in March of 2021, ARIKAYCE was approved by Japan’s Ministry of Health, Labour and Welfare. Insmed anticipates launching ARIKAYCE in Japan in mid-2021.
2020 Financial Highlights
Brensocatib, our DPP1 inhibitor and most advanced pipeline candidate, represents a potential new pathway for addressing a broad array of neutrophil-mediated diseases. After reporting positive data from the Phase 2 WILLOW study in patients with bronchiectasis in early 2020, we quickly advanced to Phase 3 development, initiating the ASPEN study in December of last year. We are targeting enrollment of more than 1,600 patients across 480 sites in 40 countries for this double-blind, placebo-controlled trial. The unmet needs in this therapeutic area are significant, with more than 1 million patients currently diagnosed with bronchiectasis around the world and no treatments specifically approved for this condition.
Beyond bronchiectasis, we are working to expand our focus for brensocatib to additional potential indications. Initially, we plan to commence a Phase 2 pharmacokinetic multiple-dose study of brensocatib in patients with cystic fibrosis in mid-2021.
We were also proud to contribute to the fight against the ongoing pandemic by supporting the investigator-initiated STOP-COVID19 trial, a UK-based study of brensocatib in hospitalized patients with COVID-19.
Treprostinil Palmitil Inhalation Powder (TPIP)
TPIP, our next pipeline candidate, is a novel, dry powder formulation of treprostinil palmitil that we believe represents an opportunity to harness the full potential of the prostanoid pathway. In February of 2021, we announced topline data from a Phase 1 study in healthy volunteers, which showed that TPIP was generally well tolerated, with a pharmacokinetic profile that supports once-daily dosing. Today, we are advancing TPIP into the next stage of development for the treatment of pulmonary arterial hypertension (PAH), while also exploring its potential in other rare and serious pulmonary disorders, including pulmonary hypertension associated with interstitial lung disease (PH-ILD) and idiopathic pulmonary fibrosis (IPF).
Though my role is not directly related to helping patients, it’s my part in how this organization runs that feeds into that. I think I speak for everyone at Insmed when I say that doing our part, whatever that may be, is what makes Insmed the company it is.”
Chief Information Officer