Insmed Announces Presentations at the American Tho

• Data support the benefit of treating patients with a combination of ALIS (Amikacin Liposome Inhalation Suspension) and guideline-based therapy (GBT) for the treatment of nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC)
• Improvements in Six Minute Walk Test (6MWT) observed for converters vs. non-converters
• Preliminary results of an investigator initiated ongoing Phase 2 trial studying ALIS in lung disease caused by M. Abscessus (MAB), demonstrating 30% of patients receiving ALIS met culture conversion criteria
• Regulatory review in the U.S. currently underway with PDUFA action date set for September 28, 2018

BRIDGEWATER, N.J., May 22, 2018 (GLOBE NEWSWIRE) -- Insmed Incorporated (Nasdaq: INSM ), a global biopharmaceutical company focused on the unmet needs of patients with rare diseases, today announced that detailed data from its ongoing Phase 3 CONVERT study of ALIS (amikacin liposome inhalation suspension) in adult patients with treatment refractory NTM lung disease caused by MAC were presented at the American Thoracic Society (ATS) 2018 International Conference.

Results from 336 randomized patients in the Phase 3 CONVERT study cumulatively demonstrated that ALIS, combined with GBT, improved culture conversion rates. The global CONVERT study met its primary endpoint of culture conversion by Month 6 with statistical significance (p<0.0001). In the study, the addition of ALIS to GBT eliminated evidence of NTM lung disease caused by MAC in sputum by Month 6 in 29% of patients, compared to 9% of patients on GBT alone. Additionally, results from an exploratory analysis using the 6MWT, a quality of life measure, demonstrated that converters from both groups (ALIS + GBT and GBT alone) saw a mean increase in distance walked at Month 6 of 16.8 meters as compared to baseline (p=0.011).

“The data presented at ATS underscore the positive impact that treatment with ALIS in combination with GBT can have in just six months treating patients with this rare, progressive and potentially fatal disease,” remarked Will Lewis, President and Chief Executive Officer of Insmed. “The improvement observed in the 6MWT suggest that eradication of NTM caused by MAC may provide a benefit in physical function for these patients.”

Investigators at Oregon Health & Science University and the University of Texas Health Science Center at Tyler also presented a poster of initial results from an ongoing Phase 2 investigator-sponsored study of ALIS in lung disease caused by M. Abscessus (MAB), the second most common NTM respiratory pathogen. The open-label study is investigating the safety, tolerability and efficacy of ALIS in both treatment-refractory and treatment-naïve patients with pulmonary disease due to MAB. To date, 30% of patients receiving ALIS for MAB lung disease met culture conversion criteria, with one-third of those patients reverting while on therapy.

The ATS abstracts are available online on Insmed’s website, www.insmed.com .

About NTM Lung Disease

NTM lung disease is a rare and serious disorder associated with increased rates of morbidity and mortality. There is an increasing prevalence of lung disease caused by NTM and Insmed believes it is an emerging public health concern worldwide. Patients with NTM lung disease may experience a multitude of symptoms such as fever, weight loss, cough, lack of appetite, night sweats, blood in the sputum, and fatigue. Patients with NTM lung disease frequently require lengthy hospital stays to manage their condition.  Insmed is not aware of any approved inhaled therapies specifically indicated for refractory NTM lung disease caused by MAC in North America, Japan or Europe.  Current guideline-based approaches involve use of multi-drug regimens not approved for the treatment of NTM lung disease, and treatment can be as long as two years or more.

The prevalence of human disease attributable to NTM has increased over the past two decades. In a decade long study (1997 to 2007), researchers found that the prevalence of NTM lung disease in the U.S. was increasing at approximately 8% per year and that NTM patients on Medicare over the age of 65 were 40% more likely to die over the period of the study than those who did not have the disease. In the U.S., Insmed estimates there will be between 75,000 and 105,000 patients with diagnosed NTM lung disease in 2018, of which the Company expects 40,000 to 50,000 will be treated for NTM lung disease caused by MAC.  Insmed expects that between 10,000 and 15,000 of these patients will be refractory to treatment.  In Japan, Insmed estimates there will be between 125,000 and 145,000 patients with diagnosed NTM lung disease in 2018, with approximately 60,000 to 70,000 of those patients being treated for NTM lung disease caused by MAC and 15,000 to 18,000 of these treated patients being refractory to treatment. Insmed also estimates there will be approximately 14,000 patients with diagnosed NTM lung disease in the EU5 (comprised of France, Germany, Italy, Spain and the United Kingdom) in 2018, of which the Company estimates approximately 4,400 will be treated for NTM lung disease caused by MAC and approximately 1,400 of these treated patients will be refractory to treatment.  

About ALIS

ALIS is a novel, inhaled, once-daily formulation of amikacin that is in late-stage clinical development for adult patients with treatment-refractory NTM lung disease caused by MAC. 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. Insmed's advanced pulmonary liposome technology uses charge neutral liposomes to deliver amikacin directly to the lung where it is taken up by the lung macrophages where the NTM infection resides. This prolongs the release of amikacin in the lungs while minimizing systemic exposure thereby offering the potential for decreased systemic toxicities. ALIS's ability to deliver high levels of amikacin directly to the lung distinguishes it from intravenous amikacin. ALIS is administered once daily using an optimized, investigational eFlow ^® Nebulizer System manufactured by PARI Pharma GmbH (PARI), a portable aerosol delivery system.

About CONVERT (INS-212) and INS-312

CONVERT is a randomized, open-label, global Phase 3 trial designed to confirm the culture conversion results seen in Insmed's Phase 2 clinical trial of ALIS in patients with refractory NTM lung disease caused by MAC. CONVERT is being conducted in 18 countries at more than 125 sites. The primary efficacy endpoint is the proportion of patients who achieved culture conversion at Month 6 in the ALIS plus GBT arm compared to the GBT-only arm. Patients who achieved culture conversion by Month 6 are continuing in the CONVERT study for an additional 12 months of treatment following the first monthly negative sputum culture. Patients who did not culture convert may have been eligible to enroll in our INS-312 study. INS-312 is a single-arm open-label extension study for patients who completed six months of treatment in the INS-212 study but did not demonstrate culture conversion by Month 6. Under the study protocol, non-converting patients in the ALIS plus GBT arm of the INS-212 study will receive an additional 12 months of ALIS plus GBT. Patients who crossed over from the GBT-only arm of the INS-212 study will receive 12 months of treatment of ALIS plus GBT.

About Insmed

Insmed Incorporated is a global biopharmaceutical company focused on the unmet needs of patients with rare diseases. The Company’s lead product candidate is ALIS, which is in late-state development for adult patients with treatment refractory NTM lung disease caused by MAC, which is a rare and often chronic infection that is capable of causing irreversible lung damage and can be fatal.  Insmed's earlier-stage clinical pipeline includes INS1007, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and INS1009, an inhaled nanoparticle formulation of a treprostinil prodrug that may offer a differentiated product profile for rare pulmonary disorders, including pulmonary arterial hypertension.  For more information, visit www.insmed.com.

Forward-looking Statements

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The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company’s forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company’s business, please see the factors discussed in Item 1A, "Risk Factors," in the Company’s Annual Report on Form 10-K for the year ended December 31, 2017 and any subsequent filings with the Securities and Exchange Commission.

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Contact:

Blaine Davis Insmed Incorporated (908) 947-2841 blaine.davis@insmed.com