Insmed Incorporated (Nasdaq: INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today provided a general business update.
Submission of New Drug Application for ARIKAYCE® (amikacin liposome inhalation suspension) in Japan
Insmed announced today that it has submitted a new drug application (JNDA) to Japan's Ministry of Health, Labour and Welfare (MHLW) for ARIKAYCE for the treatment of patients with non-tuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC) who did not sufficiently respond to prior treatment. Insmed subsequently plans to submit a separate Medical Device Notification (JMDN) to the MHLW for approval of the Lamira® Nebulizer System, the designated device for administration of ARIKAYCE.
"The submission of our JNDA filing for ARIKAYCE marks an important step in our journey to transform the way NTM lung disease caused by MAC is managed for patients in Japan. We look forward to continuing our work with the regulatory authorities during the review period," said Yuji Orihara, General Manager of Japan for Insmed.
AstraZeneca Exercises Option to Develop INS1007 for Treatment of COPD or Asthma
Insmed also announced today that AstraZeneca AB has exercised the first option pursuant to the companies' October 2016 license agreement under which AstraZeneca can advance clinical development of INS1007 in the indication of chronic obstructive pulmonary disease (COPD) or asthma. Under the terms of the agreement, upon exercise of this option, AstraZeneca is solely responsible for all aspects of the development of INS1007 up to and including Phase 2b clinical trials in COPD or asthma.
The agreement also includes a second and final option which, if exercised, would permit AstraZeneca to further develop INS1007 beyond Phase 2b clinical trials upon reaching agreement on commercial terms satisfactory to each party for the further development and commercialization of INS1007 in COPD or asthma. Insmed retains full development and commercialization rights for INS1007 in all other indications and geographies.
"We believe AstraZeneca's decision to exercise its option to develop INS1007 in COPD or asthma validates the potential of INS1007 to offer a promising new approach to modulating neutrophil activity, which could prove beneficial in a broad range of diseases beyond our initial focus in non-cystic fibrosis bronchiectasis," said Will Lewis, Chairman and CEO of Insmed.
In February 2020, Insmed reported positive top-line results from the global, randomized, double-blind placebo-controlled Phase 2 WILLOW study of INS1007 in adults with non-cystic fibrosis bronchiectasis. Insmed plans to advance INS1007 to Phase 3 development for the treatment of bronchiectasis. In addition, as previously communicated, Insmed is exploring several other indications for potential clinical development with INS1007 including, most recently, in vivo models of acute respiratory distress syndrome (ARDS) which may be brought on by any number of causes, including in the setting of respiratory failure due to COVID-19.
Business Decisions in Response to COVID-19 and Suspension of Revenue Guidance
Insmed recently implemented a number of corporate initiatives in response to the novel coronavirus (SARS-CoV-2) global pandemic which manifests as COVID-19. These include a remote working policy for all employees in order to aid the global containment effort and allow infectious disease specialists and pulmonologists to focus exclusively on treating patients and containing the virus. This policy was implemented with the protection of the Company's employees and patients in mind as its patients are particularly vulnerable to infection with potentially serious life-threatening consequences. The policy includes all of the field-based therapeutic specialists and other "customer-facing" employees who support ARIKAYCE prescribers. Insmed's Arikares Trainers will now offer remote training for patients who initiate treatment with ARIKAYCE rather than conducting in-person onboarding.
Importantly, Insmed has observed no disruptions to date in its supply chain for the production of ARIKAYCE. The Company believes it has adequate supply of finished product on hand to support its commercial efforts for at least the next 7 months, with production continuing, and sufficient active pharmaceutical ingredient used in ARIKAYCE to meet anticipated global requirements, including commercial, clinical, and compassionate use, through the end of 2022.
While Insmed has no current supply issues and continues to see use of ARIKAYCE, including new patient adds and continued prescription renewals, the general uncertainty regarding the impact of COVID-19 on the ARIKAYCE patient population and their physicians has led the Company to suspend its previously stated 2020 revenue guidance.
"Patients suffering from refractory NTM lung disease are typically older individuals with underlying lung conditions, and are often treated by pulmonologists and infectious disease specialists. These treating physicians are on the front lines in addressing this global pandemic and must now, understandably, focus their attention on COVID-19. We are active participants in the social distancing policy and recommend its adoption by others to try to help slow the spread of this virus and alleviate the demands on the US hospital system," said Mr. Lewis. "We are committed to the NTM community, and will continue to support them through these trying circumstances."
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. Current guideline-based treatment involves the use of multi-drug regimens that are not specifically approved for MAC lung disease. The course of treatment is often two years or more and is inadequate in treating the disease in many patients.
About ARIKAYCE® (amikacin liposome inhalation suspension)
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. 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. This approach prolongs the release of amikacin in the lungs 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® (amikacin liposome inhalation suspension) 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 INS1007
INS1007 is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase I (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, 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. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. INS1007 may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs.
