Full Press Release Details
Insmed Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Business Update
-ARIKAYCE EU Launch Progresses, with Product Now Available and Reimbursed in Germany and the Netherlands-
-Frontline Clinical Trial Program of
ARIKAYCE(R) (amikacin liposome inhalation suspension) in Nontuberculous Mycobacterial (NTM) Lung Disease Caused by Mycobacterium avium
complex (MAC) Initiated in Q4 2020-
-Phase 3 ASPEN Study of Brensocatib in Bronchiectasis Initiated in Q4 2020-
-Topline Results from Phase 1 Study of Treprostinil Palmitil Inhalation Powder (TPIP) Support Continued Development with Once-Daily
-ARIKAYCE Total Revenue of $164.4 Million for the Full Year 2020-
BRIDGEWATER, N.J., February 25, 2021 /PRNewswire/ - Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives
of patients with serious and rare diseases, today reported financial results for the fourth quarter and full year ended December 31, 2020 and provided a business update.
"2020 was the most productive and significant year in Insmed's history, as we evolved from a single-product company with a successful U.S. launch to a truly
global organization advancing three distinct, value-creating programs. I am incredibly proud of our team's performance, which is all the more exemplary against the backdrop of COVID-19," commented Will Lewis, Chair and Chief Executive Officer of
Insmed. "In the fourth quarter, we initiated both the Phase 3 ASPEN study of brensocatib in patients with bronchiectasis and the ARIKAYCE frontline clinical trial program in patients with NTM lung disease caused by MAC; advanced our ARIKAYCE launch
in Europe and prepared for a potential approval and launch in Japan while maintaining steady performance in the U.S.; and advanced the development of TPIP, for which we announced positive Phase 1 data just last week. We begin 2021 with incredible
momentum and believe we have the capabilities and talent to achieve our ambitious vision."
Recent Corporate Developments & Program Highlights
Fourth Quarter and Full-Year 2020 Financial Results
Balance Sheet and Planned Investments
As of December 31, 2020, Insmed had cash and cash equivalents of $532.8 million. The Company's total operating expenses for the fourth quarter of 2020 were
$136.0 million and for the full year 2020 were $429.6 million. Adjusted R&D expenses for the fourth quarter of 2020 were $63.6 million and for the full year 2020 were $164.6 million. Adjusted SG&A expenses for the fourth quarter were $49.1
million and for the full year 2020 were $174.8 million. Adjusted R&D expenses and adjusted SG&A expenses are non-GAAP measures, which we describe further below.
The Company plans to invest in the following key activities in 2021:
Insmed will host a conference call beginning today at 8:30 AM Eastern Time. Shareholders
and other interested parties may participate in the conference call by dialing (833) 340-0284 (domestic) or (236) 712-2425 (international) and referencing conference ID number 4261618. The call will also be webcast live on the company's website at
A replay of the conference call will be accessible approximately two hours after its completion through March 27, 2021 by dialing (800) 585-8367 (domestic) or
(416) 621-4642 (international) and referencing conference ID number 4261618. A webcast of the call will also be archived for 90 days under the Investor Relations section of the company's website at www.insmed.com.
Non-GAAP Financial Measures
In addition to the U.S. generally accepted accounting principles (GAAP)
results, this earnings release includes non-GAAP financial measures: adjusted R&D expenses, which Insmed defines as R&D expenses less stock-based
compensation expense and depreciation; and adjusted SG&A expenses, which Insmed defines as SG&A expenses less stock-based compensation, depreciation, and certain milestones related to ARIKAYCE, which are payable under our amended agreements with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT). A reconciliation of these non-GAAP financial measures to their most directly comparable GAAP
financial measure is presented in the table attached to this press release.
Management believes that these non-GAAP financial measures are useful to both management and investors in analyzing our ongoing business and operating
performance. Management believes that providing this non-GAAP information to investors, in addition to the GAAP results, allows investors to view our financial results in the way that management views financial results. Management does not intend
the presentation of these non-GAAP financial measures to be considered in isolation or as a substitute for results prepared in accordance with GAAP. In addition, these non-GAAP financial measures may differ from similarly named measures used by
ARIKAYCE is approved in the United States as ARIKAYCE(R) (amikacin liposome inhalation suspension) and in the EU as ARIKAYCE(R) Liposomal
590 mg Nebuliser Dispersion. Current international treatment guidelines recommend the use of ARIKAYCE for appropriate patients. 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(R) 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(R) Nebulizer System manufactured by PARI Pharma GmbH (PARI).
About PARI Pharma and the Lamira(R) Nebulizer System
ARIKAYCE is delivered by a novel inhalation device, the Lamira(R) Nebulizer System, developed by PARI. Lamira(R) is a quiet, portable
nebulizer that enables efficient aerosolization of 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 to improve patient care.
Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with
non-cystic fibrosis bronchiectasis (NCFBE) and other neutrophil-mediated diseases. 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. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. Brensocatib is an investigational drug product that has not
been approved for any indication in any jurisdiction.
Treprostinil palmitil inhalation powder (TPIP) is a dry powder formulation of treprostinil palmitil, a treprostinil prodrug consisting of treprostinil linked
by an ester bond to a 16-carbon chain. Developed entirely in Insmed's laboratories, TPIP is a potentially highly differentiated prostanoid being evaluated for the treatment of patients with PAH and other rare and serious pulmonary disorders. TPIP is
administered in a capsule-based inhalation device. TPIP is an investigational drug product that has not been approved for any indication in any jurisdiction.
IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.
Hypersensitivity Pneumonitis has been reported with
the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis, pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated with ARIKAYCE
plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity
pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.
Hemoptysis has been reported with the use of
ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis occurs, manage patients as medically appropriate.
Bronchospasm has been reported with the use of
ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients treated with
ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7%). If bronchospasm occurs during the
use of ARIKAYCE, treat patients as medically appropriate.
Exacerbations of underlying pulmonary disease has
been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying pulmonary disease (reported as chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation of bronchiectasis) have
been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%) compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease occur during the use of ARIKAYCE, treat patients as medically appropriate.
Anaphylaxis and Hypersensitivity Reactions: Serious
and potentially life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients taking ARIKAYCE. Signs and symptoms include acute onset of skin and mucosal tissue hypersensitivity reactions (hives, itching,
flushing, swollen lips/tongue/uvula), respiratory difficulty (shortness of breath, wheezing, stridor, cough), gastrointestinal symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and cardiovascular signs and symptoms of anaphylaxis
(tachycardia, low blood pressure, syncope, incontinence, dizziness). Before therapy with ARIKAYCE is instituted, evaluate for previous hypersensitivity reactions to aminoglycosides. If anaphylaxis or a hypersensitivity reaction occurs, discontinue
ARIKAYCE and institute appropriate supportive measures.
Ototoxicity has been reported with the use of
ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus, and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17%) compared to patients treated with
background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background regimen vs 0.9% in the background
regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background regimen alone arm). Closely monitor
patients with known or suspected auditory or vestibular dysfunction during treatment with ARIKAYCE. If ototoxicity occurs, manage patients as medically appropriate, including potentially discontinuing ARIKAYCE.
Nephrotoxicity was observed during the clinical
trials of ARIKAYCE in patients with MAC lung disease but not at a higher frequency than background regimen alone. Nephrotoxicity has been associated with the aminoglycosides. Close monitoring of patients with known or suspected renal dysfunction
may be needed when prescribing ARIKAYCE.
Neuromuscular Blockade: Patients with neuromuscular
disorders were not enrolled in ARIKAYCE clinical trials. Patients with known or suspected neuromuscular disorders, such as myasthenia gravis, should be closely monitored since aminoglycosides may aggravate muscle weakness by blocking the release of
acetylcholine at neuromuscular junctions.
Embryo-Fetal Toxicity: Aminoglycosides can cause fetal harm when administered to a pregnant woman. Aminoglycosides, including ARIKAYCE, may be associated with total, irreversible, bilateral
congenital deafness in pediatric patients exposed in utero. Patients who use ARIKAYCE during pregnancy, or become pregnant while taking
ARIKAYCE should be apprised of the potential hazard to the fetus.
Contraindications: ARIKAYCE is contraindicated in
patients with known hypersensitivity to any aminoglycoside.
Most Common Adverse Reactions: The most common
adverse reactions in Trial 1 at an incidence 5% for patients using ARIKAYCE plus background regimen compared to patients treated with background regimen alone were dysphonia (47% vs 1%), cough (39% vs 17%), bronchospasm (29% vs 11%), hemoptysis
(18% vs 13%), ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%), musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs 10%), exacerbation of underlying pulmonary disease (15% vs 10%), diarrhea (13% vs 5%), nausea (12% vs 4%),
pneumonia (10% vs 8%), headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash (6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs 1%), and chest discomfort (5% vs 3%).
Drug Interactions: Avoid concomitant use of ARIKAYCE
with medications associated with neurotoxicity, nephrotoxicity, and ototoxicity. Some diuretics can enhance aminoglycoside toxicity by altering aminoglycoside concentrations in serum and tissue. Avoid concomitant use of ARIKAYCE with ethacrynic acid,
furosemide, urea, or intravenous mannitol.
Overdosage: Adverse reactions specifically associated
with overdose of ARIKAYCE have not been identified. Acute toxicity should be treated with immediate withdrawal of ARIKAYCE, and baseline tests of renal function should be
undertaken. Hemodialysis may be helpful in removing amikacin from the body. In all cases of suspected overdosage, physicians should contact the Regional Poison Control Center for information about effective treatment.
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 options. This 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