On February 17, 2022 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the fourth quarter and full year ended December 31, 2021 and provided an update on the Company’s business and pipeline (Press release, Insmed, FEB 17, 2022, View Source [SID1234608212]).
"Insmed made tremendous progress across our four pillars throughout 2021, and we begin 2022 from a position of strength, with seven ongoing clinical trials, commercial operations in three major territories, a pathway to ARIKAYCE growth, and a highly innovative research engine to identify what we believe to be the most promising next set of candidates," commented Will Lewis, Chair and Chief Executive Officer of Insmed. "Importantly, our strong cash position will support advancement across our programs as we continue to lay the groundwork for what we believe will be a meaningful inflection point for the Company. I am enormously proud of our world-class team and what we have been able to achieve on behalf of patients with serious and rare diseases."
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Recent Corporate Developments & Program Highlights
ARIKAYCE
•Insmed launched ARIKAYCE in Japan in July of 2021, and early launch progress has been strong, with several positive trends.
•In Europe, ARIKAYCE has now been launched in Germany, the Netherlands, Wales, and Scotland. The Company is pursuing country-by-country reimbursement and launches throughout Europe, with a near-term focus on Italy, France, and England.
•Insmed continues to advance the post-marketing confirmatory frontline clinical trial program of ARIKAYCE in patients with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC). The ARISE trial, an interventional study designed to validate a patient-reported outcome (PRO) tool in MAC lung disease, is now 50% enrolled. The Company anticipates completing enrollment in the ARISE study in 2022 and having topline data in the first half of 2023. Insmed anticipates completing enrollment in ENCORE, a pivotal study evaluating the clinical benefits and safety of ARIKAYCE in patients with newly diagnosed MAC lung disease using the PRO tool, by the end of 2023.
1Brensocatib
•The Phase 3 ASPEN study, a global, randomized, double-blind, placebo-controlled trial to assess the efficacy, safety, and tolerability of brensocatib in patients with bronchiectasis, is now 50% enrolled. Insmed anticipates completing enrollment in this study in early 2023.
•A Phase 2 pharmacokinetic/pharmacodynamic study of brensocatib in patients with cystic fibrosis is underway and Insmed anticipates sharing data from this study by early 2023.
•Insmed plans to develop brensocatib in two additional neutrophil-mediated diseases – chronic rhinosinusitis without nasal polyps (CRS) and hidradenitis suppurativa (HS) – and to advance one indication into the clinic in 2022. Both CRS and HS are serious diseases in which patients today face significant unmet needs.
TPIP
•Insmed is advancing two Phase 2 studies of treprostinil palmitil inhalation powder (TPIP) in patients with pulmonary arterial hypertension (PAH). The Phase 2a study will measure the impact of TPIP on pulmonary vascular resistance (PVR) over a 24-hour period. The Company anticipates having preliminary data from a small number of patients in this study this year. The Phase 2b study will evaluate the effect of TPIP on PVR and 6-minute walk distance over a 16-week treatment period. As planned, site initiation for this study began in late 2021.
•Site initiation is underway for a Phase 2 study of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD). This study will assess the safety and tolerability of TPIP over a 16-week treatment period.
Translational Medicine
•Insmed is advancing a translational medicine portfolio consisting of several technology programs running in parallel. The Company anticipates filing an Investigational New Drug Application in a non-pulmonary indication for our first candidate from this portfolio by the end of 2022.
Fourth Quarter and Full-Year 2021 Financial Results
•Total revenue for the fourth quarter ended December 31, 2021 was $56.1 million, compared to total revenue of $41.4 million for the fourth quarter of 2020. Total revenue for the full year 2021 was $188.5 million, compared to total revenue of $164.4 million for the full year 2020.
•Total revenue for the full year 2021 comprised ARIKAYCE net sales of $159.5 million in the U.S., $16.0 million in Japan, and $12.9 million in Europe and rest of world. This compares to net sales of $157.5 million in the U.S. and $6.9 million in Europe for the full year 2020.
•Cost of product revenues (excluding amortization of intangible assets) was $13.3 million for the fourth quarter of 2021, compared to $10.9 million for the fourth quarter of 2020. For the full year 2021, cost of product revenues (excluding amortization of intangible assets) was $44.2 million compared to $39.9 million in 2020.
•Research and development (R&D) expenses were $76.4 million for the fourth quarter of 2021, compared to $67.8 million for the fourth quarter of 2020. For the full year 2021, R&D expenses were $272.7 million compared to $181.2 million in 2020.
•Selling, general and administrative (SG&A) expenses for the fourth quarter of 2021 were $65.3 million, compared to $56.0 million for the fourth quarter of 2020. For the full year 2021, SG&A expenses were $234.3 million, compared to $203.6 million in 2020.
•For the fourth quarter of 2021, Insmed reported a net loss of $113.0 million, or $0.95 per share, compared to a net loss of $102.2 million, or $1.00 per share, for the fourth quarter of 2020. For the full year 2021, Insmed reported a net loss of $434.7 million, or $3.88 per share, compared to a net loss of $294.1 million, or $3.01 per share, in 2020.
2Balance Sheet, Financial Guidance, and Planned Investments
As of December 31, 2021, Insmed had cash and cash equivalents and marketable securities of $766.8 million. The Company’s total operating expenses for the fourth quarter of 2021 were $155.2 million and for the full year 2021 were $563.6 million.
Insmed expects full-year 2022 revenues for ARIKAYCE to increase at least 30% year over year from 2021. The Company anticipates that its cash on hand will support its ongoing programs into 2024.
The Company plans to invest in the following key activities in 2022:
(i)commercialization and expansion of ARIKAYCE globally;
(ii)launch activities for ARIKAYCE in initial European countries and in Japan; and
(iii)clinical trial activities, including (a) advancement of the frontline clinical trial program for ARIKAYCE (ARISE and ENCORE), (b) advancement of brensocatib, including the Phase 3 ASPEN study in patients with bronchiectasis, (c) advancement of the Phase 2 clinical development programs of TPIP, and (d) advancement of our translational medicine efforts.
Conference Call
Insmed will host a conference call beginning today at 8:00 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing (844) 200-6205 (U.S. toll free), (646) 904-5544 (U.S. local), or +1-929-526-1599 (international) and referencing access code 359415. The call will also be webcast live on the company’s website at www.insmed.com.
A replay of the conference call will be accessible approximately 1 hour after its completion through March 17, 2022, by dialing (866) 813-9403 (U.S. toll free), (929) 458-6194 (U.S. local), or +44-204-525-0658 (international) and referencing access code 838814. 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.
About ARIKAYCE
ARIKAYCE is approved in the United States as ARIKAYCE (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion, and in Japan as ARIKAYCE inhalation 590 mg (amikacin sulfate inhalation drug product). 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 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).
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 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.
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About Brensocatib
Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis 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.
About TPIP
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, PH-ILD, 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.
WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS
ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases.
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.
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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.
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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 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 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 Use: ARIKAYCE 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.