Insmed Receives Positive CHMP Opinion for ARIKAYCE Liposomal 590 mg Nebuliser Dispersion for the Treatment of NTM Lung Infections Caused by MAC in Non-CF Patients with Limited Treatment Options

- If Approved, ARIKAYCE Will Be First and Only Therapy in the European Union for This Difficult-to-Treat Condition -

- Decision from European Commission Expected Second Half of 2020 -

BRIDGEWATER, NJ, USA I July 24, 2020 I Insmed Incorporated (Nasdaq: INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending ARIKAYCE Liposomal 590 mg Nebuliser Dispersion for the treatment of non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium complex (MAC) in adults with limited treatment options who do not have cystic fibrosis. Consideration should be given to official guidance on the appropriate use of antibacterial agents.

The European Commission (EC) will review the CHMP opinion, with a final decision anticipated in the second half of 2020.

"Today's positive opinion from the CHMP marks an important step in our journey to transform the way MAC lung disease is managed for patients around the world. If approved by the EC, ARIKAYCE would be the first therapy in both the European Union and the United States for patients with this chronic, debilitating condition," said Will Lewis, Chairman and Chief Executive Officer of Insmed. "We look forward to potentially bringing ARIKAYCE to appropriate patients in Europe as quickly as possible."

The CHMP opinion is based on results from the Phase 3 CONVERT study, which demonstrated that once-daily ARIKAYCE, when combined with multi-drug regimen (MDR), improved sputum culture conversion rates in patients with refractory NTM lung disease caused by MAC compared to MDR therapy alone.

"The positive CHMP opinion underscores the potential benefit of ARIKAYCE in the management of MAC lung disease for patients who are particularly difficult to treat and continue to suffer in the absence of an approved therapy in Europe," said Prof. Dr. Christoph Lange, Medical Director of Clinical Infectious Diseases at the Medical Clinic of the Research Center Borstel, Leibniz Lung Center, Germany. "As demonstrated in the Phase 3 CONVERT study, ARIKAYCE has the potential to improve culture-conversion rates in patients with MAC lung disease who were not responsive to standard of care—a critical milestone in the treatment landscape of this disease."

In the United States, ARIKAYCE (under the generic name amikacin liposome inhalation suspension), is the first and only approved treatment for MAC lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. Insmed has also submitted a new drug application for ARIKAYCE in Japan for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment. New international treatment guidelines issued in July 2020 recommend the use of ARIKAYCE in combination with a multidrug regimen in patients with MAC lung disease who have failed standard therapy after at least six months of treatment.

ARIKAYCE previously received Orphan Drug Designation in the European Union for the treatment of NTM lung infections.

About MAC Lung Disease

Mycobacterium avium complex (MAC) lung disease is a rare and serious disorder that can significantly increase morbidity and mortality. Patients with MAC lung disease can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, MAC lung disease can cause severe, even permanent damage to the lungs, and can be fatal. MAC lung disease is an emerging public health concern worldwide with significant unmet need.

About ARIKAYCE

In the United States, ARIKAYCE is the first and only FDA-approved therapy indicated for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. Current international treatment guidelines recommend the use of ARIKAYCE in combination with a multidrug regimen in patients with MAC lung disease who have failed standard therapy after at least six months of treatment. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed's proprietary PULMOVANCE™ liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides, while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira® Nebulizer System manufactured by PARI Pharma GmbH (PARI).

ARIKAYCE is not approved in any country other than the United States.

About PARI Pharma and the Lamira® Nebulizer System

ARIKAYCE is delivered by a novel inhalation device, the Lamira® Nebulizer System, developed by PARI. Lamira® is a quiet, portable nebulizer that enables efficient aerosolization of liquid medications, including liposomal formulations such as ARIKAYCE, via a vibrating, perforated membrane. Based on PARI's 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms and new pharmaceutical formulations that work together to improve patient care.

About CONVERT (INS-212)

CONVERT was a randomized, open-label, global Phase 3 study designed to confirm the sputum culture conversion results seen in Insmed's Phase 2 clinical study of ARIKAYCE in patients with refractory NTM lung disease caused by MAC. CONVERT was conducted in 18 countries at more than 125 sites. In the U.S., the primary efficacy endpoint was the proportion of patients who achieved sputum culture conversion at Month 6 in the ARIKAYCE plus MDR arm compared to the MDR-only arm. 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. The CONVERT study also evaluated the proportion of patients who maintained sputum culture conversion 3 months off all MAC treatment, which was the primary efficacy endpoint in the EU.

U.S. INDICATION

LIMITED POPULATION: ARIKAYCE® is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment optionsThis drug is indicated for use in a limited and specific population of patients.

This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Limitation of UseARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1–800–FDA–1088. You can also call the Company at 1-844-4-INSMED.

Please see Full Prescribing Information.

About Insmed

Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first commercial product, ARIKAYCE® (amikacin liposome inhalation suspension), is the first and only therapy approved in the United States for the treatment of refractory Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. MAC lung disease is a chronic, debilitating condition that can cause severe and permanent lung damage. Insmed's earlier-stage clinical pipeline includes brensocatib, a novel oral reversible inhibitor of dipeptidyl peptidase 1 with therapeutic potential in non-cystic fibrosis bronchiectasis and other inflammatory diseases, and treprostinil palmitil, an inhaled 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.

SOURCE: Insmed

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