Insmed(US:INSM)2025-10-30 11:00Regulatory Approvals - ARIKAYCE was approved in the US in September 2018, in the EU in October 2020, and in Japan in March 2021[165]. - BRINSUPRI (brensocatib 25 mg and 10 mg tablets) was approved in the US in August 2025 and launched in Q3 2025[169]. - Regulatory submissions for brensocatib in the EU, UK, and Japan have been accepted, with a positive opinion from the EMA in October 2025[172]. - ARIKAYCE received accelerated approval in the US in September 2018 for the treatment of refractory MAC lung disease, with a total of 12 years of exclusivity granted by the FDA[175]. - In October 2020, ARIKAYCE received marketing authorization in the EU for treating NTM lung infections caused by MAC in adults with limited treatment options[181]. - BRINSUPRI (brensocatib) was approved by the FDA in August 2025 for treating NCFB in patients aged 12 and older[195]. Clinical Trials and Studies - The ENCORE trial for ARIKAYCE completed enrollment with 425 patients, and topline data is expected in H1 2026[170]. - The company anticipates initiating a Phase 3 study of TPIP in patients with PAH in early 2026 after positive results from the Phase 2b study[172]. - The company is advancing the ENCORE trial, which aims to support full approval for ARIKAYCE and potentially expand its label to include all MAC lung disease[180]. - The ENCORE trial enrolled 425 patients and aims to report topline data in the first half of 2026, focusing on respiratory symptom score and durable culture conversion for MAC lung disease[193]. - The WILLOW study demonstrated a 42% reduction in exacerbation risk for the 10 mg brensocatib group and a 38% reduction for the 25 mg group compared to placebo[200]. - The ASPEN trial included 1,680 adult patients and 41 adolescents, showing statistically significant reductions in the annualized rate of adjudicated pulmonary exacerbations for both brensocatib dosages[204]. - The primary endpoint of the ASPEN study showed a 21.1% reduction in annualized rate of pulmonary exacerbations for the 10 mg group and a 19.4% reduction for the 25 mg group compared to placebo[205]. - TPIP demonstrated a 35% placebo-adjusted reduction in pulmonary vascular resistance in the Phase 2b study for PAH, with a significant improvement in six-minute walk distance[213]. - In the TPIP PAH study, 84% of patients titrated to at least 480 µg once daily, and 90% of patients completed the study[214]. - The Phase 2a study of brensocatib in CF patients showed a clear dose-dependent inhibition of blood NSPs, consistent with its mechanism of action[206]. - The company anticipates reporting topline data from the BiRCh trial of brensocatib in CRSsNP by early January 2026[208]. - The company plans to initiate the PALM-ILD Phase 3 study for TPIP in the fourth quarter of 2025[212]. Financial Performance - Product revenues, net, increased by $48.9 million, or 52.4%, to $142.3 million for the three months ended September 30, 2025, compared to $93.4 million in the same period in 2024, driven by U.S. sales of BRINSUPRI and a 22.3% growth in global ARIKAYCE sales[232]. - Cost of product revenues (excluding amortization of intangible assets) rose by $8.2 million, or 38.7%, to $29.4 million for the three months ended September 30, 2025, primarily due to increased sales[234]. - R&D expenses increased by $35.6 million, or 23.6%, for the three months ended September 30, 2025, primarily due to higher compensation and stock-based compensation costs[232]. - SG&A expenses increased by $67.4 million, or 56.7%, for the three months ended September 30, 2025, mainly due to higher professional fees and external expenses[232]. - Amortization of intangible assets increased by $0.3 million following the FDA approval of BRINSUPRI in August 2025[232]. - Change in fair value of deferred and contingent consideration liabilities increased by $90.0 million, or 612.8%, due to a rise in the company's share price in 2025 compared to 2024[232]. - U.S. sales of ARIKAYCE reached $73.98 million, a 10.6% increase from $66.87 million in 2024, while international sales grew by 51.8% to $40.31 million[233]. - The total product revenues from BRINSUPRI in the U.S. amounted to $28.05 million following its FDA approval in August 2025[233]. - Cost of product revenues as a percentage of revenues decreased to 20.6% in 2025 from 22.7% in 2024, attributed to the lower manufacturing cost of BRINSUPRI compared to ARIKAYCE[234]. - R&D expenses for the three months ended September 30, 2025 were $186.4 million, an increase of $35.6 million, or 23.6%, compared to $150.8 million in the same period of 2024[237]. - Total external R&D expenses for the three months ended September 30, 2025 were $93.9 million, up $16.7 million, or 21.6%, from $77.2 million in 2024[238]. - SG&A expenses for the three months ended September 30, 2025 were $186.4 million, an increase of $67.4 million, or 56.7%, compared to $118.9 million in 2024[239]. - Product revenues, net, for the nine months ended September 30, 2025 were $342.6 million, an increase of $83.3 million, or 32.1%, compared to $259.3 million in 2024[246]. - Cost of product revenues (excluding amortization of intangible assets) for the nine months ended September 30, 2025 was $78.7 million, an increase of $19.1 million, or 32.1%, compared to $59.6 million in 2024[247]. - Total R&D expenses for the nine months ended September 30, 2025 were $516.2 million, an increase of $97.5 million, or 23.3%, compared to $418.6 million in 2024[249]. - Investment income for the three months ended September 30, 2025 was $18.3 million, an increase of $1.3 million, or 7.7%, compared to $17.0 million in 2024[242]. - Change in fair value of deferred and contingent consideration liabilities for the three months ended September 30, 2025 was $104.7 million, primarily due to an increase in share price[241]. - Amortization of intangible assets for the three months ended September 30, 2025 was $1.5 million, an increase of $0.3 million compared to $1.3 million in 2024[240]. - The company expects R&D expenses to increase in 2025 relative to 2024 due to ongoing clinical trial activities and related spending[250]. - SG&A expenses for the nine months ended September 30, 2025, were $488.7 million, an increase of $170.1 million, or 53.4%, compared to $318.6 million in 2024[251]. - Compensation and benefit-related expenses increased by $82.1 million, primarily due to an increase in headcount[251]. - Investment income rose to $45.4 million for the nine months ended September 30, 2025, up $9.4 million, or 26.0%, from $36.1 million in 2024[255]. - Net cash used in operating activities was $687.4 million for the nine months ended September 30, 2025, compared to $487.9 million in 2024[265]. - Cash and cash equivalents decreased to $334.8 million as of September 30, 2025, down from $555.0 million as of December 31, 2024[264]. - Marketable securities increased to $1,345.2 million as of September 30, 2025, compared to $878.8 million as of December 31, 2024[264]. - The company completed an underwritten offering of 8,984,375 shares at a public offering price of $96.00 per share in June 2025, resulting in net proceeds of $823.3 million[259]. - The change in fair value of deferred and contingent consideration liabilities was $182.0 million for the nine months ended September 30, 2025, primarily due to an increase in share price[254]. - Amortization of intangible assets increased to $4.1 million for the nine months ended September 30, 2025, from $3.8 million in 2024[253]. - Interest expense slightly decreased to $63.2 million for the nine months ended September 30, 2025, from $63.4 million in 2024, a decrease of $0.2 million, or 0.3%[256]. Product Development and Strategy - The company aims to commercialize additional clinical-stage products, including TPIP and INS1201, while advancing pre-clinical research programs[167]. - The company is actively evaluating in-licensing and acquisition opportunities for products addressing serious diseases with significant unmet needs[171]. - ARIKAYCE is included in international treatment guidelines for NTM lung disease, strongly recommended for patients with limited or no alternative treatment options[176]. - ARIKAYCE is administered once daily using the Lamira inhalation device, designed specifically for its delivery[174]. - The company is exploring additional research and lifecycle management programs for ARIKAYCE beyond its current indications[192]. - The company aims to ensure a successful US commercial launch of BRINSUPRI and expand its revenue stream from ARIKAYCE[178]. - The company plans to initiate a Phase 3 trial for TPIP in patients with PAH in early 2026, with additional studies anticipated for PPF and IPF in the second half of 2026[217].