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Home/Science/Pipeline

Pipeline

The follow through to see it through

Whether we start from drug discovery or pick up an opportunity mid-pipeline, our team’s seamless coordination across functions and unwavering focus on transforming patients’ lives enable us to advance product candidates at any stage. 

Product Pipeline

Mycobacterium Avium Complex (MAC) Lung Disease

Preclinical Phase 1 Phase 2 Phase 3 Approved

ARIKAYCE® (Amikacin Liposome Inhalation Suspension)*

Refractory MAC Lung Disease

Approved

Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).1 People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.2,3,4

ARIKAYCE® (Amikacin Liposome Inhalation Suspension) Lifecycle Management

MAC Lung Disease

Phase 3

Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).1 People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.2,3,4

Neutrophil-driven Inflammatory Conditions

Preclinical Phase 1 Phase 2 Phase 3 Approved

Brensocatib: DPP1 Inhibitor

Non-Cystic Fibrosis Bronchiectasis

Phase 3

Non-cystic fibrosis bronchiectasis (NCFBE) is a chronic pulmonary disorder in which the bronchi become permanently dilated due to a vicious cycle of inflammation and complications from prior infections.5,6 The condition is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations.

Brensocatib: DPP1 Inhibitor

Cystic Fibrosis

Phase 2

Cystic fibrosis (CF) is a rare, progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.7 CF is caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene.8

Brensocatib: DPP1 Inhibitor

Chronic Rhinosinusitis without Nasal Polyps (CRSsNP)

Phase 1

CRS without nasal polyps is a chronic inflammatory process of the mucus membranes inside the sinuses. The most common symptoms include a decreased sense of smell and facial pain.9

Brensocatib: DPP1 Inhibitor

Hidradenitis Suppurativa (HS)

Phase 1

HS is a chronic relapsing inflammatory disorder, characterized by painful, inflamed, and swollen lesions affecting hair follicles, often in the armpits, groin, and skin folds.10

Additional Rare Pulmonary Diseases

Preclinical Phase 1 Phase 2 Phase 3 Approved

Treprostinil Palmitil Inhalation Powder

Pulmonary Hypertension associated with Interstitial Lung Diseases (PH-ILD)

Phase 2

Interstitial lung diseases (ILD) comprise a large group of diseases that cause fibrosis (scarring) of the lungs. Pulmonary hypertension (high blood pressure in the lungs) is a common and important complication of several ILDs that is associated with reduced exercise capacity and poor prognosis.12,13 PH-ILD is also known as WHO Group 3 Pulmonary Hypertension.

Treprostinil Palmitil Inhalation Powder

Pulmonary Arterial Hypertension (PAH)

Phase 2

Pulmonary arterial hypertension (PAH) is a serious, progressive, rare disease involving narrowing and constriction of the pulmonary arteries that carry blood from the right side of the heart to the lungs.11 PAH is also known as WHO Group 1 Pulmonary Hypertension.

Early-Stage Research

Preclinical Phase 1 Phase 2 Phase 3 Approved

INS1201: Gene Therapy

Duchenne Muscular Dystrophy (DMD)

Preclinical

DMD is caused by a genetic mutation that prevents the body from producing dystrophin, a protein that muscles need to work properly. Without it, muscle cells become damaged and weaken.14 DMD is the most common childhood onset form of muscular dystrophy and affects males almost exclusively.15

Gene Therapy

Stargardt Disease

Preclinical

Stargardt disease is a rare genetic eye disease caused by the buildup of fatty material on the macula, the small part of the retina needed for sharp, central vision.16 The disease leads to the loss of central vision in both eyes, typically beginning in childhood. Today, there are no treatments available for the disease, and management options focus on optimizing patients’ remaining sight.

Gene Therapy

Argininosuccinic Aciduria (ASA)

Preclinical

ASA is a rare genetic disorder characterized by a deficiency of the enzyme argininosuccinate lyase (ASL), which plays a role in the breakdown and removal of nitrogen in the body—a process known as the urea cycle. The lack of ASL results in excessive ammonia in the blood, which damages or inhibits the functions of neurons. The disease typically presents in infants and is marked by vomiting, refusal to eat, progressive lethargy, and coma.17

Gene Therapy

Multiple indications

Preclinical

Deimmunized Therapeutic Protein

Chronic Refractory Gout

Preclinical

Chronic refractory gout is a rare form of gout, which can cause chronic arthritis; swelling, redness, and intense pain in the joints; and lumps around the hands, elbows, and other body parts, known as tophi. It is caused by the buildup of uric acid in the body, either due to over-production of uric acid or the inability of the kidneys to remove it.18

Deimmunized Therapeutic Proteins

Multiple indications

Preclinical

Synthetic Rescue

Ataxia Telangiectasia (AT)

Preclinical

AT is an inherited, fatal childhood disorder primarily driven by mutations in the ataxia-telangiectasia mutated (ATM) gene, which is involved in DNA damage repair. Symptoms vary between individuals but typically involve degeneration across a range of neurological and neuromuscular functions, impaired metabolic and immune system functions, and an increased risk of cancer. People with AT often require significant support and face a significantly reduced life expectancy.19

Early-Stage Research

Multiple indications

Preclinical

* In the U.S., as a condition of accelerated approval, Insmed is conducting an additional clinical study to support full approval. Full approval has been granted in Europe and Japan.

Clinical Trials

Discover how we put passion to potential.

Learn more about our clinical trials

References

  1. Adjemian J, Prevots DR, Gallagher J, Heap K, Gupta R, Griffith D. Lack of adherence to evidence-based treatment guidelines for nontuberculous mycobacterial lung disease. Ann Am Thorac Soc. 2014;11(1):9-16.
  2. Mirsaeidi M, Hadid W, Ericsoussi B, Rodgers D, Sadikot RT. Non-tuberculous mycobacterial disease is common in patients with non-cystic fibrosis bronchiectasis. Int J Infect Dis.2013;17(11):e1000-e1004.
  3. Andréjak C, Nielsen R, Thomsen VØ, Duhaut P, Sørensen HT, Thomsen RW. Chronic respiratory disease, inhaled corticosteroids and risk of non-tuberculous mycobacteriosis. Thorax. 2013;68(3):256-262.
  4. Fritscher LG, Marras TK, Bradi AC, Fritscher CC, Balter MS, Chapman KR. Nontuberculous mycobacterial infection as a cause of difficult-to-control asthma: a case-control study. Chest. 2011;139(1):23-27.
  5. Prevalence and incidence of noncystic fibrosis bronchiectasis among US adults in 2013 (Weycker). https://www.ncbi.nlm.nih.gov/pubmed/28555504. Accessed December 2020.
  6. Advances in bronchiectasis: endotyping, genetics, microbiome, and disease heterogeneity (Flume). https://www.ncbi.nlm.nih.gov/pubmed/30215383. Accessed December 2020.
  7. Cystic Fibrosis Foundation. About Cystic Fibrosis. https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/. Accessed December 2020.
  8. National Institutes of Health. Genetic and Rare Disease Information Center. https://rarediseases.info.nih.gov/diseases/6233/cystic-fibrosis. Accessed October 2020
  9. Cho SH, Kim DW, Gevaert P. Chronic Rhinosinusitis without Nasal Polyps. J Allergy Clin Immunol Pract. 2016 ; 4(4): 575–582.
  10. Phan K, Charlton O, Smith SD. Global Prevalence of Hidradenitis Suppurative and Geographical Variation—Systematic Review and Meta-Analysis. Phan et al. Biomedical Dermatology (2020) 4:2.
  11. Pulmonary Hypertension Association. About Pulmonary Hypertension. https://phassociation.org/types-pulmonary-hypertension-groups/. Accessed January 2021.
  12. American Lung Association. Interstitial Lung Disease. https://www.lung.org/lung-health-diseases/lung-disease-lookup/interstitial-lung-disease. Accessed January 2021.
  13. Caminati A. Pulmonary hypertension in chronic interstitial lung diseases. European Respiratory Review 2013 22: 292-301 https://err.ersjournals.com/content/22/129/292. Accessed January 2021.
  14. Muscular Dystrophy Association. Duchenne Muscular Dystrophy (DMD). https://www.mda.org/disease/duchenne-muscular-dystrophy. Accessed April 2023.

  15. National Institute of Child Health and Human Development. What are the types and symptoms of muscular dystrophy (MD)? https://www.nichd.nih.gov/health/topics/musculardys/conditioninfo/types. Accessed April 2023.

  16. National Eye Institute. Stargardt Disease. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/stargardt-disease. Accessed March 2023.

  17. National Organization for Rare Disorders (NORD). Argininosuccinic Aciduria. https://rarediseases.org/rare-diseases/argininosuccinic-aciduria/. Accessed April 2023.

  18. National Kidney Foundation. Refractory Gout. https://kidney.org/atoz/content/refractory-gout. Accessed April 2023.

  19. National Institute of Neurological Disorders and Stroke. Ataxia Telangiectasia. https://www.ninds.nih.gov/health-information/disorders/ataxia-telangiectasia#:~:text=Ataxia%20Telangiectasia%20(AT)%E2%80%94also,movement%20of%20muscles)%20and%20speech. Accessed July 2023.

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