Full Press Release Details
Insmed Reports Fourth-Quarter and Full-Year 2023 Financial Results and Provides Business Update
-Topline Data from the Phase 3 ASPEN Trial of Brensocatib in Adult Patients with Bronchiectasis Remain on Track to Read Out in the Latter Part
of Second-Quarter 2024-
-Enrollment in the Phase 2 Study of TPIP in Patients with PH-ILD Completed in November 2023; Topline Data Expected in Second-Quarter 2024
Ahead of the ASPEN Readout-
-Company Ends 2023 With $780 Million of Cash, Cash Equivalents, and Marketable Securities, Providing Runway Beyond the Expected ASPEN Readout-
- ARIKAYCE (amikacin liposome inhalation suspension) Total Revenue of $83.7 Million for Fourth-Quarter and $305.2 Million for
Full-Year 2023, Reflecting 24% Annual Growth and Exceeding the Upper End of Full-Year 2023 Guidance Range-
-Company Reiterates Sales Guidance for 2024 Global ARIKAYCE Revenues in the Range of $340 Million to $360 Million, Reflecting Double-Digit Growth Compared to
BRIDGEWATER, N.J., February 22, 2024 /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, 2023 and provided a business update.
"Insmed continued to deliver strong performance in 2023, demonstrating commercial success evidenced by ARIKAYCE revenues that exceeded the upper end of our 2023
sales guidance range," said Will Lewis, Chair and Chief Executive Officer of Insmed. "The positive ARISE data announced in September 2023, followed by encouraging blinded TPIP data shortly thereafter, marked the beginning of a transformative
period of clinical catalysts for the Company. This series of meaningful data readouts from our mid- to late-stage pipeline is expected to continue uninterrupted in the months ahead, with topline results from the PH-ILD and ASPEN trials
expected in quick succession in the second quarter. We believe these near-term data readouts have the potential to fundamentally change the trajectory for our company and the patients we serve."
Recent Pillar Highlights
Pillar 2: Brensocatib
Pillar 4: Early-Stage Research
Fourth-Quarter and Full-Year 2023 Financial Results
Balance Sheet, Financial Guidance, and Planned Investments
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 (888) 210-2654 (U.S. and international) and referencing access code 7862189. 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 23, 2024, by dialing (800) 770-2030 (U.S. and
international) and referencing access code 7862189. 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.
Consolidated Statements of Net Loss
(in thousands, except per share data)
| Three Months Ended December 31, | Twelve Months Ended December 31, | |||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| Product revenues, net | $ | 83,693 | $ | 59,300 | $ | 305,208 | $ | 245,358 | ||||||||
| Operating expenses: | ||||||||||||||||
| Cost of product revenues (excluding amortization of intangible assets) | 18,443 | 13,069 | 65,573 | 55,126 | ||||||||||||
| Research and development | 137,029 | 124,763 | 571,011 | 397,518 | ||||||||||||
| Selling, general and administrative | 89,530 | 73,479 | 344,501 | 265,784 | ||||||||||||
| Amortization of intangible assets | 1,263 | 1,264 | 5,052 | 5,053 | ||||||||||||
| Change in fair value of deferred and contingent consideration liabilities | 15,700 | (1,800 | ) | 28,697 | (20,802 | ) | ||||||||||
| Total operating expenses | 261,965 | 210,775 | 1,014,834 | 702,679 | ||||||||||||
| Operating loss | (178,272 | ) | (151,475 | ) | (709,626 | ) | (457,321 | ) | ||||||||
| Investment income | 9,853 | 8,318 | 42,132 | 11,081 | ||||||||||||
| Interest expense | (20,784 | ) | (16,445 | ) | (81,694 | ) | (26,446 | ) | ||||||||
| Change in fair value of interest rate swap | 1,970 | (1,526 | ) | 320 | (1,526 | ) | ||||||||||
| Other income (expense), net | 2,170 | 1,130 | 1,856 | (5,939 | ) | |||||||||||
| Loss before income taxes | (185,063 | ) | (159,998 | ) | (747,012 | ) | (480,151 | ) | ||||||||
| Provision for income taxes | 998 | 125 | 2,555 | 1,383 | ||||||||||||
| Net loss | $ | (186,061 | ) | $ | (160,123 | ) | $ | (749,567 | ) | $ | (481,534 | ) | ||||
| Basic and diluted net loss per share | $ | (1.28 | ) | $ | (1.21 | ) | $ | (5.34 | ) | $ | (3.91 | ) | ||||
| Weighted average basic and diluted common shares outstanding | 144,806 | 132,694 | 140,433 | 123,035 |
Consolidated Balance Sheets
(in thousands, except par value and share data)
| As of | As of | |||||||
| December 31, 2023 | December 31, 2022 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 482,374 | $ | 1,074,036 | ||||
| Marketable securities | 298,073 | 74,244 | ||||||
| Accounts receivable | 41,189 | 29,713 | ||||||
| Inventory | 83,248 | 69,922 | ||||||
| Prepaid expenses and other current assets | 24,179 | 25,468 | ||||||
| Total current assets | 929,063 | 1,273,383 | ||||||
| Fixed assets, net | 65,384 | 56,491 | ||||||
| Finance lease right-of-use assets | 20,985 | 23,697 | ||||||
| Operating lease right-of-use assets | 18,017 | 21,894 | ||||||
| Intangibles, net | 63,704 | 68,756 | ||||||
| Goodwill | 136,110 | 136,110 | ||||||
| Other assets | 96,574 | 76,104 | ||||||
| Total assets | $ | 1,329,837 | $ | 1,656,435 | ||||
| Liabilities and shareholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable and accrued liabilities | $ | 214,987 | $ | 182,117 | ||||
| Finance lease liabilities | 2,610 | 1,217 | ||||||
| Operating lease liabilities | 8,032 | 6,909 | ||||||
| Total current liabilities | 225,629 | 190,243 | ||||||
| Debt, long-term | 1,155,313 | 1,125,250 | ||||||
| Royalty financing agreement | 155,034 | 148,015 | ||||||
| Contingent consideration | 84,600 | 51,100 | ||||||
| Finance lease liabilities, long-term | 27,026 | 29,636 | ||||||
| Operating lease liabilities, long-term | 11,013 | 14,853 | ||||||
| Other long-term liabilities | 3,145 | 9,387 | ||||||
| Total liabilities | 1,661,760 | 1,568,484 | ||||||
| Shareholders' equity: | ||||||||
| Common stock, $0.01 par value; 500,000,000 authorized shares, 147,977,960 and 135,653,731 issued and outstanding shares at December 31, 2023 and December 31, 2022, respectively | 1,480 | 1,357 | ||||||
| Additional paid-in capital | 3,113,487 | 2,782,416 | ||||||
| Accumulated deficit | (3,446,145 | ) | (2,696,578 | ) | ||||
| Accumulated other comprehensive (loss) income | (745 | ) | 756 | |||||
| Total shareholders' (deficit) equity | (331,923 | ) | 87,951 | |||||
| Total liabilities and shareholders' equity | $ | 1,329,837 | $ | 1,656,435 |
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.
Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with
bronchiectasis, CRSsNP, 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, 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 AND BOXED WARNING 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 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.