Full Press Release Details
Insmed Reports Fourth-Quarter and Full-Year 2024 Financial Results and
Provides Business Update
-ARIKAYCE (amikacin liposome inhalation suspension) Total Revenue of
$104.4 Million for Fourth-Quarter and $363.7 Million for Full-Year 2024, Reflecting 19% Annual Growth and Exceeding the Upper End of Full-Year 2024 Guidance Range-
-Company Reiterates 2025 Global ARIKAYCE Revenue Guidance of $405 Million to $425 Million, Reflecting Double-Digit Growth Compared to 2024-
-NDA for Brensocatib in Patients with Bronchiectasis Accepted by FDA and Granted Priority Review with a PDUFA Target Action Date of August 12, 2025-
-Phase 2b Study of TPIP in Patients with PAH, Phase 2b BiRCh Study of Brensocatib in Patients with CRSsNP, and Phase 3 ENCORE Study of ARIKAYCE Remain
on Track for Topline Data in Mid-2025, End of 2025, and First Quarter of 2026, Respectively-
-Company Ends 2024 with Approximately $1.4 Billion of Cash, Cash Equivalents, and Marketable Securities-
BRIDGEWATER, N.J., February 20, 2025 /PRNewswire/ -- Insmed Incorporated (Nasdaq: INSM), a
people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases, today reported financial results for the fourth quarter and full year ended December 31,
2024 and provided a business update.
"As we report on the most important year in Insmed's history, we feel we are in a rare position in our industry, with extraordinary opportunities ahead. Our success across
all areas of the business in 2024-most notably the delivery of positive data from the landmark Phase 3 ASPEN study of brensocatib in bronchiectasis-has positioned us to potentially reach many more patients suffering from serious diseases, and
resulted in significant value creation," said Will Lewis, Chair and Chief Executive Officer of Insmed. "In 2025, we look forward to delivering the highly anticipated U.S. launch of brensocatib, pending FDA approval, and we are delighted to be one
step closer to achieving that goal with the FDA's acceptance of our NDA with Priority Review. Simultaneously, we expect to continue to advance our robust mid- to late-stage pipeline while generating double-digit worldwide revenue growth for ARIKAYCE. We believe that Insmed's strong balance sheet and unique company culture will enable us to deliver on behalf of patients in need."
Recent Progress and Anticipated Milestones by Program:
Pre-Clinical Programs
Fourth-Quarter and Full-Year 2024 Financial Results
| Three Months Ended December 31, | Twelve Months Ended December 31, | |||||||||||||||||||||||
| (in millions) | 2024 | 2023 | Growth | 2024 | 2023 | Growth | ||||||||||||||||||
| U.S. | $ | 67.8 | $ | 58.3 | 16.4 | % | $ | 254.8 | $ | 224.2 | 13.7 | % | ||||||||||||
| Japan | 30.7 | 21.0 | 46.6 | % | 87.7 | 65.7 | 33.4 | % | ||||||||||||||||
| Europe & Rest of World | 5.9 | 4.5 | 32.5 | % | 21.2 | 15.3 | 38.8 | % | ||||||||||||||||
| Total Revenues | $ | 104.4 | $ | 83.7 | 24.8 | % | $ | 363.7 | $ | 305.2 | 19.2 | % |
Balance Sheet, Financial Guidance, and Planned Investments
Insmed will host a conference call beginning today, February 20, 2025, at 8:00 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 February 27, 2025, 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, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Product revenues, net | $ | 104,442 | $ | 83,693 | $ | 363,707 | $ | 305,208 | ||||||||
| Operating expenses: | ||||||||||||||||
| Cost of product revenues (excluding amortization of intangible assets) | 26,151 | 18,443 | 85,742 | 65,573 | ||||||||||||
| Research and development | 179,727 | 137,029 | 598,367 | 571,011 | ||||||||||||
| Selling, general and administrative | 142,515 | 89,530 | 461,116 | 344,501 | ||||||||||||
| Amortization of intangible assets | 1,263 | 1,263 | 5,052 | 5,052 | ||||||||||||
| Change in fair value of deferred and contingent consideration liabilities | (14,800 | ) | 15,700 | 91,682 | 28,697 | |||||||||||
| Total operating expenses | 334,856 | 261,965 | 1,241,959 | 1,014,834 | ||||||||||||
| Operating loss | (230,414 | ) | (178,272 | ) | (878,252 | ) | (709,626 | ) | ||||||||
| Investment income | 17,257 | 9,853 | 53,307 | 42,132 | ||||||||||||
| Interest expense | (21,550 | ) | (20,784 | ) | (84,913 | ) | (81,694 | ) | ||||||||
| Change in fair value of interest rate swap | 870 | 1,970 | (236 | ) | 320 | |||||||||||
| Other (expense) income, net | (445 | ) | 2,170 | 29 | 1,856 | |||||||||||
| Loss before income taxes | (234,282 | ) | (185,063 | ) | (910,065 | ) | (747,012 | ) | ||||||||
| Provision for income taxes | 1,266 | 998 | 3,707 | 2,555 | ||||||||||||
| Net loss | $ | (235,548 | ) | $ | (186,061 | ) | $ | (913,772 | ) | $ | (749,567 | ) | ||||
| Basic and diluted net loss per share | $ | (1.32 | ) | $ | (1.28 | ) | $ | (5.57 | ) | $ | (5.34 | ) | ||||
| Weighted average basic and diluted common shares outstanding | 179,021 | 144,806 | 164,043 | 140,433 |
Consolidated Balance Sheets
(in thousands, except par value and share data)
| As of | As of | |||||||
| December 31, 2024 | December 31, 2023 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 555,030 | $ | 482,374 | ||||
| Marketable securities | 878,796 | 298,073 | ||||||
| Accounts receivable | 52,012 | 41,189 | ||||||
| Inventory | 98,578 | 83,248 | ||||||
| Prepaid expenses and other current assets | 37,245 | 24,179 | ||||||
| Total current assets | 1,621,661 | 929,063 | ||||||
| Fixed assets, net | 80,052 | 65,384 | ||||||
| Finance lease right-of-use assets | 18,273 | 20,985 | ||||||
| Operating lease right-of-use assets | 17,257 | 18,017 | ||||||
| Intangibles, net | 58,652 | 63,704 | ||||||
| Goodwill | 136,110 | 136,110 | ||||||
| Other assets | 93,226 | 96,574 | ||||||
| Total assets | $ | 2,025,231 | $ | 1,329,837 | ||||
| Liabilities and shareholders' equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable and accrued liabilities | $ | 285,209 | $ | 214,987 | ||||
| Finance lease liabilities | 2,961 | 2,610 | ||||||
| Operating lease liabilities | 9,358 | 8,032 | ||||||
| Total current liabilities | 297,528 | 225,629 | ||||||
| Debt, long-term | 1,103,382 | 1,155,313 | ||||||
| Royalty financing agreement | 161,067 | 155,034 | ||||||
| Contingent consideration | 144,200 | 84,600 | ||||||
| Finance lease liabilities, long-term | 24,064 | 27,026 | ||||||
| Operating lease liabilities, long-term | 9,112 | 11,013 | ||||||
| Other long-term liabilities | 499 | 3,145 | ||||||
| Total liabilities | 1,739,852 | 1,661,760 | ||||||
| Shareholders' equity: | ||||||||
| Common stock, $0.01 par value; 500,000,000 authorized shares, 179,382,635 and 147,977,960 issued and outstanding shares at December 31, 2024 and December 31, 2023, respectively | 1,794 | 1,480 | ||||||
| Additional paid-in capital | 4,645,791 | 3,113,487 | ||||||
| Accumulated deficit | (4,359,917 | ) | (3,446,145 | ) | ||||
| Accumulated other comprehensive loss | (2,289 | ) | (745 | ) | ||||
| Total shareholders' equity (deficit) | 285,379 | (331,923 | ) | |||||
| Total liabilities and shareholders' equity (deficit) | $ | 2,025,231 | $ | 1,329,837 |
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,
chronic rhinosinusitis without nasal polyps, hidradenitis suppurativa, 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.
INS1201 is an investigational micro-dystrophin adeno-associated virus gene replacement therapy that Insmed is developing as a potential treatment for patients with Duchenne
muscular dystrophy. Administered intrathecally, this approach has the potential to target both skeletal and cardiac muscles at lower doses. INS1201 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
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