On May 5, 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 first quarter ended March 31, 2022 and provided a business update (Press release, Insmed, MAY 5, 2022, View Source [SID1234613617]).
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"I am proud of Insmed’s solid progress in the first quarter of 2022, with steady ARIKAYCE performance in three territories around the globe, advancement of seven clinical trials, including our ASPEN and ARISE/ENCORE trials, and continued development of our early-stage research programs," commented Will Lewis, Chair and Chief Executive Officer of Insmed. "We ended the quarter in a position of financial strength, with a strong balance sheet and steady revenue stream. The remainder of 2022 will be a critical execution period across the four pillars of our business, and I am confident we have the ambition, determination, and talent to achieve our objectives and deliver high-impact medicines for patients in need."
Recent Pillar Highlights
ARIKAYCE
In the first quarter of 2022, ARIKAYCE revenue grew 32% over the first quarter of 2021, reflecting steady U.S. performance and ongoing launch activities in Japan and Europe.
Enrollment remains on track in the post-marketing confirmatory, frontline clinical trial program of ARIKAYCE in patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex (MAC), consisting of the ARISE and ENCORE trials. Insmed anticipates completing enrollment in ARISE in 2022 and having topline data in the first half of 2023; the Company also anticipates completing enrollment in ENCORE by the end of 2023.
Insmed announced that it will present data at the American Thoracic Society (ATS) 2022 International Conference, taking place May 13-18, 2022, highlighting the reduction in hospitalizations after initiation of ARIKAYCE treatment in a retrospective cohort study of patients in real-world settings.
Brensocatib
Enrollment remains on track in 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. Insmed continues to anticipate completing enrollment in this trial in early 2023.
A Phase 2 pharmacokinetic/pharmacodynamic study of brensocatib in patients with cystic fibrosis is underway and Insmed continues to anticipate sharing data by early 2023.
As previously shared, Insmed plans to develop brensocatib in two new potential indications – chronic rhinosinusitis without nasal polyps and hidradenitis suppurativa.
Insmed will present data at the ATS 2022 International Conference evaluating the benefit-risk profile in a post-hoc analysis of the Phase 2 WILLOW study of brensocatib in patients with bronchiectasis.
TPIP
Insmed remains on track to share preliminary data from a small number of patients in a Phase 2a trial of treprostinil palmitil inhalation powder (TPIP) in patients with pulmonary arterial hypertension (PAH) this year. The Phase 2a study will measure the impact of TPIP on pulmonary vascular resistance (PVR) over a 24-hour period.
Insmed is also advancing a Phase 2b study to evaluate the effect of TPIP on PVR and 6-minute walk distance over a 16-week treatment period in patients with PAH, and a Phase 2 study to assess the safety and tolerability of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) over a 16-week treatment period.
Translational Medicine
Insmed is advancing a translational medicine portfolio encompassing a wide range of technologies and modalities, including gene therapy, gene editing, protein deimmunization, and manufacturing capabilities. The Company anticipates filing at least one Investigational New Drug Application per year from this portfolio.
First Quarter 2022 Financial Results
Total revenue for the first quarter ended March 31, 2022, was $53.1 million, compared to total revenue of $40.2 million for the first quarter of 2021. Total revenue for the first quarter of 2022 comprised ARIKAYCE net sales of $40.8 million in the U.S., $10.7 million in Japan, and $1.6 million in Europe and rest of world.
Cost of product revenues (excluding amortization of intangible assets) was $12.2 million for the first quarter of 2022, compared to $9.8 million for the first quarter of 2021.
Research and development (R&D) expenses were $84.4 million for the first quarter of 2022, compared to $61.4 million for the first quarter of 2021.
Selling, general and administrative (SG&A) expenses for the first quarter of 2022 were $56.7 million, compared to $51.6 million for the first quarter of 2021.
For the first quarter of 2022, Insmed reported a net loss of $94.6 million, or $0.80 per share, compared to a net loss of $91.6 million, or $0.89 per share, for the first quarter of 2021.
Balance Sheet, Financial Guidance, and Planned Investments
As of March 31, 2022, Insmed had cash and cash equivalents and marketable securities of $664.7 million. The Company’s total operating expenses for the first quarter of 2022 were $142.9 million.
Insmed continues to expect full-year 2022 global revenues for ARIKAYCE to increase at least 30% year over year from 2021. The Company also continues to anticipate that its cash on hand will support its ongoing business into 2024.
The Company plans to continue to invest in the following key activities in 2022:
(i)
commercialization and expansion of ARIKAYCE globally;
(ii)
launch activities for ARIKAYCE in Japan; and
(iii)
clinical trial activities, including (a) advancement of the confirmatory, 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 for TPIP, and (d) advancement of our translational medicine efforts.
Conference Call
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 (844) 200-6205 (U.S. toll free), (646) 904-5544 (U.S. local), or +1-929-526-1599 (international) and referencing access code 388457. 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 June 4, 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 252664. 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.
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.
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.
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.