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Insmed Reports First Quarter 2023 Financial Results and Provides Business Update -Enrollment Complete in Phase 3 ASPEN Trial of Brensocatib in Adult Patients with Bronchiectasis; Topline Data Readout on Track for Second

Key Takeaway: Insmed Incorporated reported its financial results for the first quarter of 2023, highlighting successful enrollment completion in the Phase 3 ASPEN trial for Brensocatib. The company also reported a 23% year-over-year increase in ARIKAYCE revenue, amounting to $65.2 million. Despite these positives, Insmed experienced a net loss of $159.8 million, an increase from the previous year, influenced by high operating expenses. Looking ahead, the company reaffirmed its revenue guidance for ARIKAYCE in 2023 and is set to provide updates on early-stage research efforts.

Market Sentiment Analysis

POSITIVE FACTORS

  • Enrollment completion in Phase 3 ASPEN trial for Brensocatib.
  • ARIKAYCE revenue increased by 23% compared to the previous year.
  • Company reaffirms full-year 2023 guidance for ARIKAYCE revenues.

CONCERNS & RISKS

  • Net loss increased to $159.8 million in Q1 2023 from $94.6 million in Q1 2022.
  • Significant operating expenses of $213.4 million, leading to operating losses.

Full Press Release Details

Insmed Reports First Quarter 2023 Financial Results and Provides Business Update
-Enrollment Complete in Phase 3 ASPEN Trial of Brensocatib in Adult Patients with Bronchiectasis; Topline Data Readout on Track for Second Quarter
-Company to Provide Detailed Update on Early-Stage Research Programs on May 8, 2023-
-Company on Track to Share Topline Data from Post-Marketing ARISE Study of ARIKAYCE (amikacin liposome inhalation suspension) in Third
-ARIKAYCE Total Revenue of $65.2 Million for the First Quarter of 2023, Reflecting 23% Annual Growth Over the First Quarter of 2022-
-Company Reaffirms Full-Year 2023 Guidance for Global ARIKAYCE Revenues of Between $285 Million and $300 Million-
BRIDGEWATER, N.J., May 4, 2023 /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 first quarter ended March 31, 2023 and provided a business update.
"Insmed began 2023 with solid momentum, demonstrated by a strong quarter of global revenues from our commercial business and continued execution of our clinical
development pipeline of mid-to-late-stage assets," commented Will Lewis, Chair and Chief Executive Officer of Insmed. "In the first quarter, we achieved our goal of enrollment completion of adult patients in the Phase 3 ASPEN trial. The pace of
enrollment and widespread geographic reach of this study speak to the global enthusiasm for this program as well as the tireless efforts of the Insmed teams that facilitated this significant accomplishment. In parallel, we also continue to advance
our early-stage research efforts. I am tremendously excited to debut several components of our early-stage platforms at our upcoming research day, including the indications, development candidates, proprietary technologies, and preclinical data to
support our efforts. Importantly, the event will also allow us to showcase some of the Insmed team leaders who are driving innovation in the treatment of serious and rare diseases."
Recent Pillar Highlights
Pillar 2: Brensocatib
Pillar 4: Early-Stage Research
First Quarter 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 (833) 470-1428 (U.S.) or (929) 526-1599 (international) and referencing access code 468531. 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 30 minutes after its
completion through June 3, 2023, by dialing (866) 813-9403 (U.S.) or (+44) 204-525-0658 (international) and referencing access code 281829. 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 March 31,
2023 2022
Product revenues, net $ 65,214 $ 53,107
Operating expenses:
Cost of product revenues (excluding amortization of intangible assets) 13,830 12,191
Research and development 127,865 84,356
Selling, general and administrative 79,914 56,748
Amortization of intangible assets 1,263 1,263
Change in fair value of deferred and contingent consideration liabilities (9,500 ) (11,618 )
Total operating expenses 213,372 142,940
Operating loss (148,158 ) (89,833 )
Investment income 10,524 137
Interest expense (20,003 ) (3,291 )
Change in fair value of interest rate swap (1,533 ) -
Other expense, net (111 ) (1,249 )
Loss before income taxes (159,281 ) (94,236 )
Provision for income taxes 483 385
Net loss $ (159,764 ) $ (94,621 )
Basic and diluted net loss per share $ (1.17 ) $ (0.80 )
Weighted average basic and diluted common shares outstanding 136,355 118,929
Consolidated Balance Sheets
(in thousands, except par value and share data)
As of March 31, 2023 As of December 31, 2022
(unaudited)
Assets
Current assets:
Cash and cash equivalents $ 968,907 $ 1,074,036
Marketable securities 29,774 74,244
Accounts receivable 30,523 29,713
Inventory 71,317 69,922
Prepaid expenses and other current assets 28,542 25,468
Total current assets 1,129,063 1,273,383
Fixed assets, net 59,556 56,491
Finance lease right-of-use assets 23,019 23,697
Operating lease right-of-use assets 21,154 21,894
Intangibles, net 67,493 68,756
Goodwill 136,110 136,110
Other assets 81,322 76,104
Total assets $ 1,517,717 $ 1,656,435
Liabilities and shareholders' equity
Current liabilities:
Accounts payable and accrued liabilities $ 179,005 $ 182,117
Finance lease liabilities 2,158 1,217
Operating lease liabilities 6,821 6,909
Total current liabilities 187,984 190,243
Debt, long-term 1,132,188 1,125,250
Royalty financing agreement 150,173 148,015
Contingent consideration 38,600 51,100
Finance lease liabilities, long-term 29,015 29,636
Operating lease liabilities, long-term 14,534 14,853
Other long-term liabilities 9,876 9,387
Total liabilities 1,562,370 1,568,484
Shareholders' equity:
Common stock, $0.01 par value; 500,000,000 authorized shares, 136,429,216 and 136,653,731 issued and outstanding shares at March 31, 2023 and December 31, 2022, respectively 1,364 1,357
Additional paid-in capital 2,809,242 2,782,416
Accumulated deficit (2,856,342 ) (2,696,578 )
Accumulated other comprehensive income 1,083 756
Total shareholders' (deficit) equity (44,653 ) 87,951
Total liabilities and shareholders' equity $ 1,517,717 $ 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
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.
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
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
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 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.
Please see Full Prescribing Information.
Insmed Incorporated is a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases. Insmed's first

Frequently Asked Questions

What is the ASPEN Trial about?

The ASPEN Trial is a Phase 3 study for Brensocatib in adult patients with bronchiectasis.

How much revenue did ARIKAYCE generate in Q1 2023?

ARIKAYCE generated $65.2 million in revenue for the first quarter of 2023.

What is Brensocatib designed to treat?

Brensocatib is developed for bronchiectasis and other neutrophil-mediated diseases.

When will topline data for ARIKAYCE be shared?

Topline data from the ARIKAYCE study is expected to be shared in the third quarter.

What is unique about the Lamira Nebulizer System?

The Lamira Nebulizer System efficiently aerosolizes ARIKAYCE using advanced technology.

Last updated: May 4, 2023