Insmed Incorporated (Insmed) operates as a global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases.
The company’s first commercial product, ARIKAYCE, is approved in the U.S. as ARIKAYCE (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion and in Japan as ARIKAYCE inhalation 590mg (amikacin sulfate inhalation drug product). ARIKAYCE received accelerated app...
Insmed Incorporated (Insmed) operates as a global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases.
The company’s first commercial product, ARIKAYCE, is approved in the U.S. as ARIKAYCE (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion and in Japan as ARIKAYCE inhalation 590mg (amikacin sulfate inhalation drug product). ARIKAYCE received accelerated approval in the U.S. in September 2018 for the treatment of MAC lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options in a refractory setting. In October 2020, the European Commission (EC) approved ARIKAYCE for the treatment of nontuberculous mycobacterial (NTM) lung infections caused by MAC in adults with limited treatment options who do not have cystic fibrosis (CF). In March 2021, Japan's Ministry of Health, Labour and Welfare (MHLW) approved ARIKAYCE for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment with a multidrug regimen. NTM lung disease caused by MAC (which it refers to as MAC lung disease) is a rare and often chronic infection that can cause irreversible lung damage and can be fatal.
The company’s pipeline includes clinical-stage programs, brensocatib, TPIP, and INS1201, as well as pre-clinical research programs. Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1), which it is developing for the treatment of patients with bronchiectasis and other neutrophil-mediated diseases, including chronic rhinosinusitis without nasal polyps (CRSsNP) and hidradenitis suppurativa (HS). TPIP is an inhaled formulation of the treprostinil prodrug treprostinil palmitil which may offer a differentiated product profile for pulmonary hypertension associated with interstitial lung disease (PH-ILD) and pulmonary arterial hypertension (PAH). INS1201 is an intrathecally delivered gene therapy for patients with Duchenne muscular dystrophy (DMD). The company’s pre-clinical research programs encompass a wide range of technologies and modalities, including gene therapy, artificial intelligence-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.
INSMED, PULMOVANCE, and ARIKAYCE are trademarks of Insmed.
ARIKAYCE
Following the announcement of positive topline results from the ARISE trial, in June 2024, the company met and aligned with the FDA on the primary endpoint for the ENCORE trial. If the data are positive, ENCORE may support a label expansion to include all MAC lung patients, as well as support full approval for the current refractory indication.
The company completed enrollment in the ENCORE trial with 425 patients in the fourth quarter of 2024.
The company anticipates reporting topline data from the ENCORE trial in the first quarter of 2026, with the submission of a U.S. supplementary new drug application for ARIKAYCE in all patients with MAC lung disease projected for late 2026.
Brensocatib
The company announced positive topline results from the ASPEN trial in May 2024. The study met its primary endpoint, with both dosage strengths of brensocatib demonstrating statistically significant reductions in the annualized rate of adjudicated pulmonary exacerbations (PEs) versus placebo.
The company’s new drug application (NDA) for brensocatib in patients with bronchiectasis was accepted and granted priority review by the FDA in February 2025. Under the Prescription Drug User Fee Act (PDUFA), the FDA set a target action date of August 12, 2025. It is advancing commercial readiness activities in preparation for a launch of brensocatib for patients with bronchiectasis and, if approved, the company anticipates a U.S. launch in the third quarter of 2025. Launches in Europe and Japan are expected in 2026, pending approvals.
The company initiated a Phase 2b study of brensocatib in patients with CRSsNP, which it refers to as the BiRCh trial, in the fourth quarter of 2023. It anticipates reporting topline data from the BiRCh trial before the end of 2025.
The company initiated a Phase 2b study of brensocatib in patients with HS, which it refers to as the CEDAR trial, in December 2024.
TPIP
In May 2024, the company reported topline safety data and certain exploratory efficacy endpoints from the Phase 2a study of TPIP in patients with PH-ILD. It anticipates initiating a Phase 3 study of TPIP in patients with PH-ILD in the second half of 2025.
Enrollment of the Phase 2b study of TPIP in patients with PAH was completed in the fourth quarter of 2024, with 102 patients enrolled in the study. The company anticipates topline results for this study in mid-2025.
Gene Therapy
In the fourth quarter of 2024, the company received clearance from the FDA for its investigational new drug (IND) application for INS1201, an intrathecally delivered gene therapy for patients with DMD. It anticipates initiating a clinical trial in patients with DMD in the first half of 2025.
Pre-Clinical Programs
The company continues to progress its pre-clinical research programs across a wide range of technologies and modalities, including gene therapy, artificial intelligence-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.
To complement the company’s internal research and development, it also actively evaluates in-licensing and acquisition opportunities for products, product candidates and technologies, including those that address serious diseases with significant unmet need.
Strategy
The company strives to develop and commercialize first- and best-in-class therapies that serve patient communities where the need is greatest. The company’s first product, ARIKAYCE, is approved in the U.S. as ARIKAYCE (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion and in Japan as ARIKAYCE inhalation 590mg (amikacin sulfate inhalation drug product). It is not aware of any other approved inhaled therapies specifically indicated to treat MAC lung disease in North America, Europe or Japan. ARIKAYCE has the potential to prove beneficial in other patients with refractory MAC. The company’s product candidates are brensocatib, its Phase 3 product candidate that it is developing for patients with bronchiectasis and other neutrophil-mediated diseases, TPIP, its Phase 2 product candidate that may offer a differentiated product profile for patients with PH-ILD and PAH, and INS1201, its intrathecally delivered gene therapy product candidate for patients with DMD. The company announced positive topline results from its Phase 3 ASPEN trial of brensocatib in May 2024 and the acceptance by the FDA of its NDA, with priority review granted, for brensocatib in patients with bronchiectasis in February 2025, which it anticipates will be followed by filings with the European and Japanese regulatory authorities. The company is also advancing its pre-clinical research programs encompassing a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.
The key elements of the company’s strategy are to continue to provide ARIKAYCE to appropriate patients and expand its reliable revenue stream; advance commercial readiness activities to serve significantly more patients facing serious diseases; produce topline clinical data readouts in the near and long term; and control spending, prudently deploying capital to support the best return-generating opportunities.
ARIKAYCE for Patients with MAC Lung Disease
ARIKAYCE is the company’s first approved product. ARIKAYCE received accelerated approval in the U.S. in September 2018 for the treatment of refractory MAC lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. In October 2020, ARIKAYCE received approval in Europe for the treatment of NTM lung infections caused by MAC in adults with limited treatment options who do not have CF. In March 2021, ARIKAYCE received approval in Japan for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment with a multidrug regimen. MAC lung disease is a rare and often chronic infection that can cause irreversible lung damage and can be fatal. Amikacin solution for parenteral administration is an established drug that has activity against a variety of NTM; however, its use is limited by the need to administer it intravenously and by toxicity to hearing, balance, and kidney function. Unlike amikacin solution for intravenous administration, its proprietary Pulmovance technology uses charge-neutral liposomes to deliver amikacin directly to the lungs where liposomal amikacin is taken up by the lung macrophages where the MAC infection resides. This technology also prolongs the release of amikacin in the lungs, while minimizing systemic exposure, thereby offering the potential for decreased systemic toxicities. ARIKAYCE's ability to deliver high levels of amikacin directly to the lung and sites of MAC infection via the use of its Pulmovance technology distinguishes it from intravenous amikacin. ARIKAYCE is administered once-daily using Lamira, an inhalation device developed and manufactured by PARI. Lamira is a portable nebulizer that enables aerosolization of liquid medications via a vibrating, perforated membrane, and was designed specifically for ARIKAYCE delivery.
ARIKAYCE also has been included in the international treatment guidelines for NTM lung disease treatment. The evidence-based guidelines, issued by the American Thoracic Society (ATS), European Respiratory Society (ERS), European Society of Clinical Microbiology and Infectious Diseases (ESCMID), and Infectious Diseases Society of America (IDSA), strongly recommend the use of ARIKAYCE for MAC lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options who have failed to convert to a negative sputum culture after at least six months of treatment.
In October 2020, the FDA approved a supplemental new drug application for ARIKAYCE, adding important efficacy data regarding the durability and sustainability of culture conversion to the ARIKAYCE label. The data, which are from the Phase 3 CONVERT study of ARIKAYCE, demonstrate that the addition of ARIKAYCE to guideline-based therapy (GBT) was associated with sustained culture conversion through the end of treatment, as well as durable culture conversion three months post-treatment compared with GBT alone.
In March 2018, the company submitted an NDA for ARIKAYCE to the FDA to request accelerated approval. Accelerated approval allows drugs that are being developed to treat a serious or life-threatening disease or condition and provide a meaningful therapeutic benefit over existing treatments to be approved substantially based on an intermediate endpoint or a surrogate endpoint that is reasonably likely to predict clinical benefit, rather than a clinical endpoint, such as survival or irreversible morbidity. In September 2018, the FDA granted accelerated approval for ARIKAYCE under the Limited Population Pathway for Antibacterial and Antifungal Drugs (LPAD) for the treatment of refractory MAC lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. LPAD, which was enacted as part of the 21st Century Cures Act, serves to advance the development of new antibacterial drugs to treat serious or life-threatening infections in limited populations of patients with unmet needs. As required for drugs approved under the LPAD pathway, labeling for ARIKAYCE includes certain statements to convey that the drug has been shown to be safe and effective only for use in a limited population.
As a condition of accelerated approval, the company must conduct a post-marketing confirmatory clinical trial. In December 2020, it commenced the post-marketing confirmatory clinical trial program for ARIKAYCE in patients with MAC lung disease consisting of the ARISE trial, an interventional study designed to validate cross-sectional and longitudinal characteristics of a PRO tool in MAC lung disease, and the ENCORE trial, designed to establish the clinical benefits and evaluate the safety of ARIKAYCE in patients with newly diagnosed or recurrent MAC lung infection who have not started antibiotics using the PRO tool validated in the ARISE trial. In September 2023, the company announced positive topline results from the ARISE trial. The study met its primary objective of demonstrating that the QOL-B respiratory domain works effectively as a PRO tool in patients with MAC lung disease. In June 2024, it met and aligned with the FDA on the primary endpoint for the ENCORE study. If the data are positive, ENCORE may support a label expansion to include all MAC lung patients as well as support full approval for the refractory indication. Based on feedback and in alignment with the FDA, the company has determined that the primary endpoint for the ENCORE study will include eight questions from the QOL-B respiratory domain PRO. It completed enrollment of the ENCORE study in the fourth quarter of 2024, with 425 patients enrolled. The company anticipates reporting topline data in the first quarter of 2026.
In October 2020, the EC granted marketing authorization for ARIKAYCE for the treatment of NTM lung infections caused by MAC in adults with limited treatment options who do not have CF. ARIKAYCE can be prescribed for appropriate patients across the European Union (EU) countries, as well as in the United Kingdom (U.K.). ARIKAYCE is reimbursed nationally in France, Belgium, the Netherlands, the U.K. and Ireland. The company has worked with the German National Association of Statutory Health Insurance Funds towards an agreement on the reimbursement price of ARIKAYCE that would allow it to better serve the needs of patients in Germany; however, since it has been unable to reach an agreement, patient supply of ARIKAYCE in Germany was enabled by import from other EU countries in September 2022. To date, the company has been unable to reach an acceptable agreement of a nationally reimbursed price with the Italian Medicines Agency; however, ARIKAYCE remains commercially available for physicians to prescribe in Italy under Class C, where it sets the price and funding is agreed locally.
In March 2021, Japan's MHLW approved ARIKAYCE for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment with a multidrug regimen. In July 2021, the company launched ARIKAYCE in Japan.
The CONVERT Study and 312 Study
Accelerated approval of ARIKAYCE was supported by preliminary data from the CONVERT study, a global Phase 3 study evaluating the safety and efficacy of ARIKAYCE in adult patients with refractory MAC lung disease, using achievement of sputum culture conversion (defined as three consecutive negative monthly sputum cultures) by Month 6 as the primary endpoint. Patients who achieved sputum culture conversion by Month 6 continued in the CONVERT study for an additional 12 months of treatment following the first monthly negative sputum culture in order to assess the durability of culture conversion, as defined by patients that have completed treatment and continued in the CONVERT study off all therapy for three months. In May 2019, the company presented at the American Thoracic Society meeting that 41/65 (63.1%) of patients on ARIKAYCE plus GBT who had achieved culture conversion by Month 6 had maintained durable culture conversion for three months off all therapy compared to 0/10 (0%) on GBT only (p<0.0002). Safety data for these patients were consistent with safety data previously reported for patients by Month 6 of the CONVERT study.
The ARISE Study
The ARISE trial was a global, randomized, double-blind, placebo-controlled Phase 3b study in adult patients with newly diagnosed or recurrent MAC infections that aimed to generate evidence demonstrating the domain specification, reliability, validity, and responsiveness of PRO-based scores, including a respiratory symptom score. The ARISE study met its primary objective of demonstrating that the QOL-B respiratory domain works effectively as a PRO tool in patients with MAC lung disease. Based on feedback and in alignment with the FDA, the company has determined that the primary endpoint for the ENCORE study will include eight questions from the QOL-B respiratory domain PRO.
Further Research and Lifecycle Management
The company is exploring and supporting research and lifecycle management programs for ARIKAYCE beyond treatment of refractory MAC lung disease as part of a combination antibacterial regimen for adult patients who have limited or no treatment options. As noted above, the company will continue to advance the post-marketing confirmatory MAC lung disease clinical trial program for ARIKAYCE, through the completed ARISE and ongoing ENCORE trials, which are intended to fulfill the FDA's post-marketing requirement to allow for the full approval of ARIKAYCE in the U.S., as well as to support the use of ARIKAYCE as a treatment for patients with MAC lung disease.
The ENCORE trial is a randomized, double-blind, placebo-controlled Phase 3b study to evaluate the efficacy and safety of an ARIKAYCE-based regimen in patients with newly diagnosed or recurrent MAC infection who have not started antibiotics. Patients are randomized 1:1 to receive ARIKAYCE plus background regimen or placebo plus background regimen once daily for 12 months. Patients will then discontinue all study treatments and remain in the trial for three months for the assessment of durability of culture conversion. The primary endpoint is change from baseline to Month 13 in respiratory symptom score. The key secondary endpoint is the proportion of subjects achieving durable culture conversion at Month 15. In June 2024, the company met and aligned with the FDA on the primary endpoint for the ENCORE study. If the data are positive, ENCORE may support a label expansion to include all MAC lung patients, as well as support full approval for the refractory indication. Based on feedback and in alignment with the FDA, it has determined that the primary endpoint for the ENCORE study will include eight questions from the QOL-B respiratory domain PRO. The company completed enrollment of the ENCORE study in the fourth quarter of 2024, with 425 patients enrolled. The company anticipates reporting topline data in the first quarter of 2026.
Product Pipeline
Brensocatib
Brensocatib is a small molecule, oral, reversible inhibitor of DPP1, which the company licensed from AstraZeneca in October 2016. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs) in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. Neutrophils contain the NSPs (including neutrophil elastase, proteinase 3, and cathepsin G) that have been implicated in a variety of inflammatory diseases. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases, such as bronchiectasis by inhibiting DPP1 and its activation of NSPs.
In June 2020, the FDA granted breakthrough therapy designation for brensocatib for the treatment of adult patients with non-cystic fibrosis bronchiectasis (NCFBE) for reducing exacerbations. The FDA's breakthrough therapy designation is designed to expedite the development and review of therapies that are intended to treat serious or life-threatening diseases and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy. The benefits of breakthrough therapy designation include more frequent communication and meetings with the FDA, eligibility for rolling and priority review, intensive guidance on an efficient drug development program, and organizational commitment from the FDA involving senior managers. In November 2020, brensocatib was granted access to the PRIME scheme from the European Medicines Agency (EMA) for patients with NCFBE.
In October 2021, the EMA’s Paediatric Committee approved the brensocatib Pediatric Investigational Plan for the treatment of patients with NCFBE. Subsequently, the ASPEN trial included 41 adolescent patients between ages 12 to 17, which will fulfill the pediatric study requirements to support marketing applications in this patient population in the U.S., Europe and Japan.
The WILLOW Study
The WILLOW study was a randomized, double-blind, placebo-controlled, parallel-group, multi-center, multi-national, Phase 2b study to assess the efficacy, safety and tolerability, and pharmacokinetics of brensocatib administered once daily for 24 weeks in patients with NCFBE. The WILLOW study was conducted at 116 sites and enrolled 256 adult patients diagnosed with NCFBE who had at least two documented pulmonary exacerbations in the 12 months prior to screening. Patients were randomized 1:1:1 to receive either 10 mg or 25 mg of brensocatib or matching placebo. The primary efficacy endpoint was the time to first pulmonary exacerbation over the 24-week treatment period in the brensocatib arms compared to the placebo arm.
WILLOW Efficacy Data
The company announced topline data for the WILLOW study in February 2020 and full data for the WILLOW study in June 2020. In September 2020, final results from the WILLOW study were published online in the New England Journal of Medicine.
The ASPEN Study
Based on the positive results of the WILLOW study, in December 2020 the company commenced the ASPEN study, a global, randomized, double-blind, placebo-controlled Phase 3 study to assess the efficacy, safety, and tolerability of brensocatib in adult patients with bronchiectasis. Patients with bronchiectasis due to CF were not enrolled in the study. The primary endpoint was the rate of adjudicated PEs over the 52-week treatment period. Secondary endpoints included the time to first adjudicated PE, the proportion of subjects free of adjudicated PE by 52 weeks, the absolute change from baseline in post-bronchodilator FEV1, the reduction in annualized rate of severe adjudicated PE, and the change from baseline in the Bronchiectasis QOL-B Respiratory Symptoms Domain Score.
The company announced positive topline results from the ASPEN trial in May 2024. The primary efficacy analysis included data from 1,680 adult patients and 41 adolescent patients. Brensocatib was well-tolerated in the study. In addition, the study met its primary endpoint, with both dosage strengths of brensocatib demonstrating statistically significant reductions in the annualized rate of adjudicated PEs versus placebo. The study also met several of its prespecified secondary endpoints with statistical significance. In February 2025, the FDA accepted the company’s NDA, with priority review granted, for brensocatib in patients with bronchiectasis.
Further Research and Development
In August 2019, the company received notice from the FDA that it was awarded a development grant for specific work to be performed on a PRO tool. The grant funding was for the development of a novel PRO tool for use in clinical trials to measure symptoms in patients with NCFBE with and without NTM lung infection. The grant has come to the end of the funding period and met the objectives with grant deliverables submitted to the FDA’s Drug Development Tool Qualification Program for evaluation by the FDA.
In January 2023, the company reported topline data from the Phase 2a, multiple-dose, pharmacokinetic/pharmacodynamic study of brensocatib in patients with CF. This Phase 2a study included both patients who were on background CFTR modulator drugs and patients who were not on CFTR modulator drugs. The study duration was approximately one month and dosed CF patients to placebo, 10 mg, 25 mg, and 40 mg of brensocatib. A clear dose-dependent and exposure-dependent inhibition of blood NSPs was observed in patients treated with brensocatib across all doses in this study, consistent with the mechanism of action of brensocatib. Safety and tolerability were consistent with what was observed during the Phase 2b WILLOW study, with no significant drug-related findings. The company concluded that an additional cohort evaluating a 65 mg dose of brensocatib is not needed in this patient population.
The company is conducting further studies to explore the potential of brensocatib, including neutrophil-mediated diseases, including CRSsNP and HS. CRSsNP has one approved pharmacological therapy (corticosteriod nasal spray); however, many patients do not respond to corticosteroids or endoscopic sinus surgery. The Phase 2b BiRCh trial of brensocatib in patients with CRSsNP is underway. In the fourth quarter of 2024, the company initiated a Phase 2b study of brensocatib in patients with HS.
Treprostinil Palmitil Inhalation Powder
TPIP is an investigational inhaled formulation of a treprostinil prodrug that has the potential to address certain of the current limitations of existing prostanoid therapies. TPIP prolongs duration of effect and may provide patients with greater consistency in pulmonary arterial pressure reduction over time. Current inhaled prostanoid therapies must be dosed four to nine times per day. Reducing dose frequency has the potential to ease treatment burden for patients and improve compliance. Additionally, TPIP may be associated with fewer side effects, including severity and/or frequency of cough, headache, throat irritation, nausea, flushing and dizziness that are associated with high initial drug levels and local upper airway exposure when using current inhaled prostanoid therapies. TPIP may offer a differentiated product profile for PH-ILD and PAH.
In February 2021, the company announced topline results from the Phase 1 study of TPIP in healthy volunteers. The objective of this first-in-human single ascending dose and multiple ascending dose study was to assess the pharmacokinetics and tolerability profile of TPIP. Data from the study demonstrated that TPIP was generally well tolerated, with a pharmacokinetic profile that supports continued development with once-daily dosing. The most common AEs across all cohorts in the study were cough, dizziness, headache, and nausea. Most AEs were mild in severity and consistent in nature with those typically seen with other inhaled prostanoid therapies.
In May 2024, the company reported topline safety data and certain exploratory efficacy endpoints from the Phase 2a study of TPIP in patients with PH-ILD. The company anticipates initiating a Phase 3 registration program in PH-ILD in the second half of 2025. It also has an ongoing Phase 2b study designed to investigate the effect of TPIP in patients with PAH. Enrollment in the Phase 2b study of TPIP in PAH completed in the fourth quarter of 2024 and the company anticipates topline results in mid-2025.
Gene Therapy
In the fourth quarter of 2024, the company received clearance from the FDA for its IND application for INS1201, a microdystrophin adeno-associated virus gene replacement therapy for patients with DMD. Administered intrathecally, this approach has the potential to target both skeletal and cardiac muscles at lower doses. The company anticipates initiating a clinical trial in patients with DMD in the first half of 2025.
Pre-Clinical Development
The company’s early-stage research efforts are consisted of its preclinical programs, advanced through internal research and development and augmented through business development activities. In March 2021, the company acquired a proprietary protein deimmunization platform, called Deimmunized by Design, focused on the reengineering of therapeutic proteins to evade immune recognition and reaction. In August 2021, the company acquired Motus Biosciences, Inc. (Motus) and AlgaeneX, Inc. (AlgaeneX), preclinical stage companies engaged in the research, development and manufacturing of gene therapies for rare genetic disorders. In January 2023, the company acquired Vertuis Bio, Inc. (Vertuis), a privately held, preclinical stage company engaged in the research and development of gene therapies for rare genetic disorders. In June 2023, the company acquired Adrestia Therapeutics Ltd. (Adrestia), a privately held, preclinical stage company using precision genetic models to search for therapeutic targets, precision diagnostics, novel drug compounds and new applications for existing drugs.
The company continues to progress its pre-clinical research programs across a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.
Manufacturing
ARIKAYCE is manufactured by Resilience Biotechnologies Inc. (Resilience) (formerly Therapure Biopharma Inc.) in Canada at a 200 kilogram (kg) scale.
In October 2017, the company entered into certain agreements with Patheon UK Limited (Patheon), a wholly-owned subsidiary of Thermo Fisher Scientific, Inc. (Thermo Fisher), related to increasing its long-term production capacity for ARIKAYCE commercial inventory. The agreements provide for Patheon to manufacture and supply ARIKAYCE for its anticipated long-term commercial needs. Under these agreements, the company is required to deliver to Patheon the required raw materials, including active pharmaceutical ingredients, and certain fixed assets needed to manufacture ARIKAYCE.
In January 2024, the company entered into certain agreements with Patheon Inc., a wholly-owned subsidiary of Thermo Fisher, related to the manufacture and supply of brensocatib by Patheon Inc. for its anticipated long-term commercial needs. In addition, in September 2024, the company entered into a commercial manufacturing and supply agreement with Esteve Química, S.A. (Esteve) for the manufacture and supply of brensocatib's active pharmaceutical ingredient. It is required to deliver to Patheon Inc. the active pharmaceutical ingredient needed to manufacture brensocatib.
Intellectual Property
The company owns or licenses rights to more than 850 issued patents and pending patent applications in the U.S. and in foreign countries, including more than 300 issued patents and pending patent applications related to ARIKAYCE. The company actively seeks patent protection by filing patent applications, including on inventions that are important to the development of its business in the U.S., Europe, Japan, Canada, and selected other foreign markets that it considers key for its product candidates. These international markets generally include Australia, China, India, Israel and Mexico.
ARIKAYCE Patents
Of the patents and applications related to ARIKAYCE, there are 13 in force issued U.S. patents that cover the ARIKAYCE composition and its use in treating NTM that are listed in the FDA Orange Book. These patents and their expiration dates, based on filing dates, are as follows:
US Patent No. 7,718,189 (expires June 6, 2025).
US Patent No. 8,226,975 (expires August 15, 2028).
US Patent No. 8,632,804 (expires December 5, 2026).
US Patent No. 8,679,532 (expires December 5, 2026).
US Patent No. 8,642,075 (expires December 5, 2026).
US Patent No. 9,566,234 (expires January 18, 2034).
US Patent No. 9,895,385 (expires May 15, 2035).
US Patent No. 10,251,900 (expires May 15, 2035).
US Patent No. 10,751,355 (expires May 15, 2035).
US Patent No. 11,446,318 (expires May 15, 2035).
US Patent No. 12,016,873 (expires May 15, 2035).
US Patent No. 12,168,021 (expires May 15, 2035).
US Patent No. 12,168,022 (expires May 15, 2035).
In addition, the company owns four pending U.S. patent applications that cover the ARIKAYCE composition and/or its use in treating NTM lung disease, including those caused by MAC infections. One or more of the patent applications, if issued as patents in their current form, may be eligible for listing in the FDA Orange Book for ARIKAYCE. It also owns a pending U.S. application that covers methods for making ARIKAYCE. The company anticipates that in the U.S., it will have patent coverage for ARIKAYCE and its use in treating NTM lung disease, including NTM lung disease caused by MAC, through at least May 15, 2035.
Ten patents have been granted by the European Patent Office (EPO) (European Patent Nos. 1909759, 1962805, 2823820, 2852391, 3067046, 3142643, 3466432, 3766501, 4005576 and 4122470) that relate to ARIKAYCE and its use in treating NTM lung disease, including those caused by MAC infections. In addition, the company has additional patent applications pending before the EPO that relate to ARIKAYCE and its use in treating NTM lung disease. European Patent No. 1909759 (the ’759 patent), owned by it, was previously opposed by Generics (UK) Ltd. A hearing was held on October 19, 2015, during which it submitted amended claims. The European Patent Office Opposition Division (EPOOD) maintained the patent as amended and Generics (UK) Ltd appealed the decision. The EPO Technical Board of Appeals heard arguments related to the appeal on January 8, 2019, and the product claims of the patent were held invalid. The method of manufacture claims was remitted to the EPOOD for further consideration, and the EPO has since maintained the validity of these claims. European Patent Nos. 1962805 and 3067046, both of which expire approximately five months after the ‘759 patent (December 5, 2026, vs. July 19, 2026), also include claims related to ARIKAYCE and its use in treating NTM lung disease. European Patent Nos. 2852391 and 4005576 each expires May 21, 2033, and include claims related ARIKAYCE together with a vibrating mesh nebulizer having certain properties. European Patent Nos. 3142643, 3466432, 3766501 and 4122470 each expire May 15, 2035, and include claims related to ARIKAYCE and its use for treating MAC lung infections.
More than 50 patents have also been issued and are in force in other major foreign markets, e.g., Japan, China, Korea, Australia, and India, that relate to ARIKAYCE and/or methods of using ARIKAYCE for treating various pulmonary disorders, including NTM lung disease. More than 30 foreign patent applications are pending that relate to the ARIKAYCE composition and/or its use in treating various pulmonary disorders, including NTM lung disease.
Through the company’s agreements with PARI, it has license rights to the U.S. and foreign patents and applications that cover the Lamira medical device through January 18, 2034. The company has entered into a commercial supply agreement with PARI, and it also has rights to use the nebulizers in expanded access programs and clinical trials.
Brensocatib Patents
Through the company’s agreement with AstraZeneca, it has licensed US Patent Nos. 9,522,894, 9,815,805, 10,287,258, 10,669,245, 11,655,221, 11,655,222, 11,655,223, 11,655,224, 11,673,871, 11,773,069, 11,814,359, and 12,054,465, which have claims related to brensocatib and methods for using brensocatib in certain treatment methods, including the treatment of obstructive diseases of the airway, such as bronchiectasis. US Patent No. 9,522,894 expires March 12, 2035, while the remaining U.S. patents expire January 21, 2035 (not taking into account any potential patent term extension). Counterpart patents of the aforementioned U.S. patents have issued in Australia, Canada, Europe, China, Japan, South Korea, India, Israel, and Mexico and expire January 21, 2035, not accounting for any potential patent term extension. The company has also licensed US Patent No. 12,059,424 from AstraZeneca, and this patent expires February 21, 2040. The claims in US Patent No. 12,059,424 relate to certain components of the brensocatib oral tablet. Counterpart patents have issued in China, Europe and Japan; and expire March 1, 2039. In addition, patent applications related to brensocatib and methods of using the same to treat indications of interest, such as bronchiectasis, HS and chronic rhinosinusitis are pending in the U.S. and throughout the world, including in Europe, China, and Japan.
TPIP Patents
The company owns US Patent Nos. 9,255,064, 9,469,600, 10,010,518, 10,526,274, 10,995,055 and 11,795,135, each expiring October 24, 2034 (not taking into account any potential patent term extensions or adjustments), each with claims covering treprostinil palmitil, the treprostinil prodrug component of TPIP, compositions comprising the same, and/or its use. US Patent No. 9,255,064 has claims reciting hexadecyl-treprostinil, and other treprostinil prodrugs. US Patent No. 9,469,600 has claims related to TPIP and other treprostinil prodrug formulations. US Patent No. 10,010,518 has claims directed to methods of treating pulmonary hypertension, including PAH, using compositions related to TPIP, such as treprostinil prodrug formulations. US Patent No. 10,526,274 has claims directed to methods for treating pulmonary fibrosis with treprostinil palmitil. US Patent No. 10,995,055 has claims directed to compositions comprising treprostinil palmitil in the form of a dry powder, and methods for treating pulmonary hypertension with the same. US Patent No. 11,795,135 has claims directed to methods for treating PH-ILD, with treprostinil palmitil. Counterpart patent applications to these U.S. Patents have issued in Europe, Japan and other foreign jurisdictions. Counterpart patent applications to these U.S. Patents are also pending in select jurisdictions, including the U.S., Europe, and Japan.
The company owns pending patent applications that relate to methods for using treprostinil prodrugs and formulations comprising the same, including TPIP in treating patients with PAH and other diseases, as well as methods for manufacturing such treprostinil prodrugs and formulations. Should the patent applications related to TPIP formulations and methods of using TPIP in pulmonary hypertension treatment methods issue, these patents would expire in October 2041.
Trademarks
In addition to the company’s patents and trade secrets, it has filed applications to register certain trademarks in the U.S. and/or abroad, including INSMED and ARIKAYCE. The company received two registrations for the INSMED mark and one registration for the ARIKAYCE mark from the U.S. Patent and Trademark Office (USPTO). It has also received notices of allowance or registrations in a number of countries abroad for the INSMED and ARIKAYCE marks, among others. The EMA has authorized the use of the name ARIKAYCE liposomal, and the U.S. Food and Drug Administration (FDA) has approved its use of the name ARIKAYCE, as the trade name for amikacin liposome inhalation suspension.
License and Other Agreements
Multi-program Agreements
PPD Development, L.P. (a wholly-owned subsidiary of Thermo Fisher)
In April 2020, the company entered into a master services agreement with PPD Development, L.P. (PPD) pursuant to which it retained PPD to perform clinical development services in connection with certain of its clinical research programs. The company has entered into project addenda with PPD to perform clinical development services over several years for its ARISE, ENCORE and ASPEN studies and other trials involving brensocatib and TPIP.
ARIKAYCE-related Agreements
The company relies on agreements with a number of third parties in connection with the development and manufacture of ARIKAYCE.
PARI
The company has a licensing agreement with PARI for use of the optimized Lamira Nebulizer System for delivery of ARIKAYCE in treating patients with NTM lung infections, CF and bronchiectasis. Under the licensing agreement, it has rights under several U.S. and foreign issued patents and patent applications involving improvements to the optimized Lamira Nebulizer System, to exploit the system with ARIKAYCE for the treatment of such indications, but it cannot manufacture the nebulizers except as permitted under its Commercialization Agreement with PARI, which is described in further detail below. Lamira has been approved for use in the U.S. (in combination with ARIKAYCE) and EU and is authorized for use in Japan. The company also has rights to use the nebulizers in expanded access programs and clinical trials. Lamira must receive regulatory approval before it can market ARIKAYCE outside the U.S., EU and Japan, and it is labeled as investigational for use in its clinical trials outside of these regions.
The company has certain obligations under this licensing agreement in relation to specified licensed indications. With respect to NTM, it met all obligations to achieve certain commercial, developmental and regulatory milestones by the required deadlines. With respect to bronchiectasis, the company has satisfied its obligation to use commercially reasonable efforts to initiate a Phase 3 trial for bronchiectasis. With respect to CF, it is obligated to use commercially reasonable efforts to develop, obtain regulatory and reimbursement approval, market and sell ARIKAYCE in two or more major European countries, as well as to achieve certain milestones specified in the licensing agreement.
In July 2014, the company entered into a Commercialization Agreement with PARI for the manufacture and supply of the Lamira Nebulizer Systems and related accessories (the Device), which is an e-Flow nebulizer modified and optimized for use with ARIKAYCE.
Resilience
In February 2014, the company entered into a contract manufacturing agreement with Therapure Biopharma Inc., which has been assumed by Resilience, for the manufacture of ARIKAYCE, on a non-exclusive basis, at a 200 kg scale.
Patheon (a wholly-owned subsidiary of Thermo Fisher) and related agreements
In October 2017, the company entered into certain agreements with Patheon related to the increase of its long-term production capacity for ARIKAYCE. The agreements provide for Patheon to manufacture and supply ARIKAYCE for its anticipated commercial needs. Under these agreements, the company is required to deliver to Patheon the required raw materials, including active pharmaceutical ingredients, and certain fixed assets needed to manufacture ARIKAYCE. Patheon's supply obligations will commence once certain technology transfer and construction services are completed.
Cystic Fibrosis Foundation Therapeutics, Inc.
In 2004 and 2009, the company entered into research funding agreements with Cystic Fibrosis Foundation Therapeutics, Inc.
Brensocatib-related Agreements
AstraZeneca
In October 2016, the company entered into a license agreement with AstraZeneca (the AZ License Agreement), pursuant to which AstraZeneca granted it exclusive global rights for the purpose of developing and commercializing AZD7986 (renamed brensocatib).
Patheon Inc. (a wholly-owned subsidiary of Thermo Fisher) and Related Agreements
In January 2024, the company entered into certain agreements with Patheon Inc. related to the manufacture and supply of brensocatib by Patheon Inc. for its anticipated long-term commercial needs. Under these agreements, the company is required to deliver to Patheon Inc. the active pharmaceutical ingredients needed to manufacture brensocatib.
Esteve Química, S.A.
In September 2024, the company entered into a commercial manufacturing and supply agreement with Esteve for the manufacture and supply of brensocatib's active pharmaceutical ingredient.
Research and Development (R&D)
The company’s R&D expenses were $598.4 million during the year ended December 31, 2024.
History
Insmed Incorporated was founded in 1988. The company was incorporated in the Commonwealth of Virginia in 1999.
