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Insmed Provides Business Update

February 23, 2023

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, 2022 and provided a business update.

“Insmed had a strong finish to 2022 as we rounded out a year of commercial and clinical execution that set the stage for what I believe will be the most meaningful period in our company’s history,” commented Will Lewis, Chair and Chief Executive Officer of Insmed. “As we begin 2023, we are excited to share outputs from across our research and development programs that we hope will demonstrate clear benefit for patients with serious unmet medical needs. We expect that achieving this outcome will bolster the value of our pipeline and potentially enable us to grow from addressing tens of thousands of patients to more than a million. For example, with today’s announcement that screening is complete in adult patients in the Phase 3 ASPEN study of brensocatib in bronchiectasis, we are well on our way toward reporting topline results in the second quarter of 2024 and potentially bringing this first-in-disease therapy to patients. Importantly, we are fully capitalized to achieve these milestones and more.”

Recent Pillar Highlights

Pillar 1: ARIKAYCE

  • ARIKAYCE global revenue grew 30% in 2022 compared with 2021, reflecting strong U.S. performance, the ongoing launch in Japan, and contributions from European markets.
  • Insmed continues to advance the development of ARIKAYCE in a frontline setting of patients with Mycobacterium avium complex (MAC) lung disease, consisting of the post-marketing confirmatory ARISE and ENCORE trials. Insmed anticipates sharing topline efficacy and safety data from the ARISE study in the third quarter of 2023 and completing enrollment in the ENCORE study by the end of 2023.

Pillar 2: Brensocatib

  • Patient screening has been completed in adult patients in the Phase 3 ASPEN study, a global, randomized, double-blind, placebo-controlled trial to assess the efficacy, safety, and tolerability of brensocatib in bronchiectasis. Insmed continues to anticipate completing enrollment in this study in the first quarter of 2023 and sharing topline data in the second quarter of 2024.
  • As previously shared, Insmed plans to initiate a Phase 2 study of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) in mid-2023. Subject to U.S. Food and Drug Administration input, the Company anticipates that the trial will enroll approximately 270 patients randomized to either 10 mg brensocatib, 40 mg brensocatib, or placebo over a 24-week treatment period, with a primary endpoint of change in daily sinus total symptom score.

Pillar 3: TPIP

  • Insmed is currently enrolling two Phase 2 studies of treprostinil palmitil inhalation powder (TPIP), one in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) and the other in patients with pulmonary arterial hypertension (PAH).
  • The Company anticipates sharing interim, blinded dose titration and safety and tolerability data from both the PH-ILD and PAH studies in the second half of 2023, pending the rate of enrollment. Topline results from the PH-ILD study are on track to be shared in the first half of 2024.

Pillar 4: Early-Stage Research

  • Insmed plans to provide an update on its early-stage research portfolio, which encompasses gene therapy, artificial intelligence-driven protein engineering, and protein manufacturing, during an event on May 8, 2023. The event will take place in person in New York and will be webcast live. The Company will offer a deep dive into the various platforms comprising its research engine and an introduction to the experts leading these teams.
  • As previously shared, Insmed anticipates having at least six investigational new drug (IND) applications filed or Phase 1 studies underway from this portfolio by the end of 2025. The first IND is expected to be filed prior to the May 8 event.
  • Insmed anticipates sharing clinical data from a few of the earliest patients in a Phase 1/2 gene therapy study in a musculoskeletal disease in the first half of 2024.

Fourth Quarter and Full-Year 2022 Financial Results

  • Total revenue for the fourth quarter ended December 31, 2022, was $59.3 million, compared to total revenue of $56.1 million for the fourth quarter of 2021. Total revenue for the full year 2022 was $245.4 million, compared to total revenue of $188.5 million for the full year 2021.
  • Total revenue for the full year 2022 comprised ARIKAYCE net sales of $186.0 million in the U.S., $56.5 million in Japan, and $2.9 million in Europe and rest of world. In the fourth quarter of 2022, Insmed reached an agreement with the French authorities on a final reimbursement price for the temporary authorization for use (Autorisation Temporaire d’Utilisation) program, which resulted in a change in estimate that reduced revenue by approximately $7.5 million in the fourth quarter of 2022, of which $5.8 million related to periods prior to 2022.
  • Cost of product revenues (excluding amortization of intangibles) was $13.1 million for the fourth quarter of 2022, compared to $13.3 million for the fourth quarter of 2021. For the full year 2022, cost of product revenues (excluding amortization of intangibles) was $55.1 million compared to $44.2 million in 2021.
  • Research and development (R&D) expenses were $124.8 million for the fourth quarter of 2022, compared to $76.4 million for the fourth quarter of 2021. For the full year 2022, R&D expenses were $397.5 million compared to $272.7 million in 2021.
  • Selling, general and administrative (SG&A) expenses for the fourth quarter of 2022 were $73.5 million, compared to $65.3 million for the fourth quarter of 2021. For the full year 2022, SG&A expenses were $265.8 million, compared to $234.3 million in 2021.
  • For the fourth quarter of 2022, Insmed reported a net loss of $160.1 million, or $1.21 per share, compared to a net loss of $113.0 million, or $0.95 per share, for the fourth quarter of 2021. For the full year 2022, Insmed reported a net loss of $481.5 million, or $3.91 per share, compared to a net loss of $434.7 million, or $3.88 per share, in 2021.

Balance Sheet, Financial Guidance, and Planned Investments

  • As of December 31, 2022, Insmed had cash, cash equivalents, and marketable securities of $1.15 billion.
  • The Company’s total operating expenses for the fourth quarter of 2022 were $210.8 million and for the full year 2022 were $702.7 million.
  • Insmed continues to expect full-year 2023 global revenues for ARIKAYCE to be between $285 million and $300 million.
  • In 2023, Insmed anticipates that over 80% of total expenditures will be on its mid-to-late stage and commercial programs (ARIKAYCE, brensocatib, and TPIP), and that less than 20% of overall spend will be on its early-stage research programs, reflecting the Company’s historical approach to spending.
  • The Company plans to invest in the following key activities in 2023:
    1. commercialization and expansion of ARIKAYCE globally;
    2. advancement of brensocatib, including the Phase 3 ASPEN study in patients with bronchiectasis and commercial launch readiness activities, as well as development across additional neutrophil-mediated diseases;
    3. advancement of the confirmatory, frontline clinical trial program for ARIKAYCE (ARISE and ENCORE); and
    4. advancement of its earlier-stage pipeline, including the Phase 2 clinical development programs for TPIP and development of its early-stage research platforms.

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).