预约演示
更新于：2025-01-20
Brensocatib
更新于：2025-01-20
概要
基本信息
原研机构 |
在研机构 |
非在研机构 |
最高研发阶段申请上市 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评突破性疗法 (美国) |
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结构/序列
分子式C23H24N4O4 |
InChIKeyAEXFXNFMSAAELR-RXVVDRJESA-N |
CAS号1802148-05-5 |
关联
19
项与 Brensocatib 相关的临床试验NCT06685835
A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of the Efficacy and Safety of Brensocatib in Adults With Moderate to Severe Hidradenitis Suppurativa - The CEDAR Study
The primary purpose of the study is evaluate the effect of brensocatib compared with placebo.
开始日期2024-12-16 |
申办/合作机构 |
NCT06344728
A Phase 1, Open-Label, Randomized, Two-Period Crossover Study to Assess the Relative Bioavailability of Brensocatib Pediatric Oral Solution to Oral Tablets Following Single Oral Doses in Healthy Subjects
The primary purpose of this study is to determine the relative bioavailability of brensocatib between the pediatric oral solution and oral tablets in healthy participants.
开始日期2024-04-30 |
申办/合作机构 |
NCT06178783
A Phase 1, Double-blind, Randomized, Single-center Study to Assess the Palatability and Acceptability of Different Brensocatib Oral Liquid Formulations in Healthy Subjects
The primary purpose of this study is to assess the palatability and compare acceptability of brensocatib oral solutions.
开始日期2023-12-27 |
申办/合作机构 |
100 项与 Brensocatib 相关的临床结果
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100 项与 Brensocatib 相关的转化医学
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100 项与 Brensocatib 相关的专利（医药）
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37
项与 Brensocatib 相关的文献（医药）2025-01-01·BRAIN BEHAVIOR AND IMMUNITY
Cathepsin C exacerbates EAE by promoting the expansion of Tfh cells and the formation of TLSs in the CNS
Article
作者: Zhao, Xinnan ; Li, Xia ; Zhao, Henan ; Liu, Shuang ; Ma, Jianmei ; Du, Cong ; Fan, Kai ; Liu, Gang ; Yang, Xiaohan ; Liu, Jing
Multiple sclerosis (MS) is a chronic autoimmune disease of the central nervous system (CNS) mediated by CD4+ T helper (Th) cells, and characterized by immune cell infiltration, demyelination and neurodegeneration, with no definitive cure available. Thus, it is pivotal and imperative to acquire more profound comprehension of the underlying mechanisms implicated in MS. Dysregulated immune responses are widely believed to play a primary role in the pathogenesis of MS. Recently, a plethora of studies have demonstrated the involvement of T follicular helper (Tfh) cells and tertiary lymphoid-like structures (TLSs) in the pathogenesis and progression of MS. Cathepsin C (CatC) is a cysteine exopeptidase which is crucial for the activation of immune-cell-associated serine proteinases in many inflammatory diseases in peripheral system, such as rheumatoid arthritis and septicemia. We have previously demonstrated that CatC is involved in neuroinflammation and exacerbates demyelination in both cuprizone-induced and experimental autoimmune encephalomyelitis (EAE) mouse models. However, the underlying immunopathological mechanism remains elusive. In the present study, we established a recombinant myelin oligodendrocyte glycoprotein 35-55 peptide-induced EAE model using conditional CatC overexpression mice to investigate the effects of CatC on the alteration of CD4+ Th subsets, including Th1, Th2, Th17, Tfh and T regulatory cells. Our findings demonstrated that CatC particularly enhanced the population of Tfh cell in the brain, resulting in the earlier onset and more severe chronic syndrome of EAE. Furthermore, CatC promoted the formation of TLSs in the brain, leading to persistent neuroinflammation and exacerbating the severity of EAE in the chronic phase. Conversely, treatment with AZD7986, a specific inhibitor of CatC, effectively attenuated the syndrome of EAE and its effects caused by CatC both in vivo and in vitro. These findings provide a novel insight into the critical role of CatC in innate and adaptive immunity in EAE, and specific inhibitor of CatC, AZD7986, may contribute to potential therapeutic strategies for MS.
2024-11-22·JCI Insight
Increased expression of cathepsin C in airway epithelia exacerbates airway remodeling in asthma
Article
作者: Yao, Ye ; Qin, Qingwu ; Liu, Huijun ; Liu, Chi ; Wu, Xinyu ; Qu, Xiangping ; Ji, Ming ; Yang, Ming ; Xiang, Yang ; Chen, Long ; Wang, Weijie ; Qin, Xiaoqun ; Yuan, Lin ; Qing, Bei ; Xia, Zhenkun ; Du, Xizi ; Qin, Qiuyan
Airway remodeling is a critical factor determining the pathogenesis and treatment sensitivity of severe asthma (SA) or uncontrolled asthma (UA). The activation of epithelial-mesenchymal trophic units (EMTUs) regulated by airway epithelial cells (AECs) has been proven to induce airway remodeling directly. However, the triggers for EMTU activation and the underlying mechanism of airway remodeling are not fully elucidated. Here, we screened the differentially expressed gene cathepsin C (CTSC; also known as dipeptidyl peptidase 1 [DPP-1]) in epithelia of patients with SA and UA using RNA-sequencing data and further verified the increased expression of CTSC in induced sputum of patients with asthma, which was positively correlated with severity and airway remodeling. Moreover, direct instillation of exogenous CTSC induced airway remodeling. Genetic inhibition of CTSC suppressed EMTU activation and airway remodeling in two asthma models with airway remodeling. Mechanistically, increased secretion of CTSC from AECs induced EMTU activation through the p38-mediated pathway, further inducing airway remodeling. Meanwhile, inhibition of CTSC also reduced the infiltration of inflammatory cells and the production of inflammatory factors in the lungs of asthmatic mice. Consequently, targeting CTSC with compound AZD7986 protected against airway inflammation, EMTU activation, and remodeling in the asthma model. Based on the dual effects of CTSC on airway inflammation and remodeling, CTSC is a potential biomarker and therapeutic target for SA or UA.
2024-10-01·CHEST
TEMPORARY REMOVAL: A PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF BRENSOCATIB IN PATIENTS WITH NON-CYSTIC FIBROSIS BRONCHIECTASIS – THE ASPEN TRIAL
Article
作者: TEPER, ARIEL ; DE-SOYZA, ANTHONY ; CHALMERS, JAMES D ; FAN, CHUNPENG ; HAWORTH, CHARLES S ; LOEBINGER, MICHAEL ; MANGE, KEVIN ; MAUGER, DAVID ; ZHANG, XIANGMIN ; FERNANDEZ, CARLOS ; BURGEL, PIERRE-RÉGIS ; DALEY, CHARLES L ; METERSKY, MARK L
This abstract has been temporarily removed at the request of the author. A replacement will appear as soon as possible, or the abstract will be reinstated. The full Elsevier Policy on Article Withdrawal can be found at https://www.elsevier.com/about/policies/article-withdrawal.
62
项与 Brensocatib 相关的新闻（医药）2025-01-10
--ARIKAYCE® (amikacin liposome inhalation suspension) Exceeds the Upper End of Guidance Range for Full-Year 2024 with Unaudited Global Revenues of Approximately $363.7 Million--
--2025 Global ARIKAYCE Revenues Expected to be Between $405 Million and $425 Million, Reflecting Continued Double-Digit Growth Compared to 2024--
--NDA for Brensocatib in Bronchiectasis Submitted to FDA in December 2024, Narrowing the Timing for Expected U.S. Launch to the Third Quarter of 2025, Pending Approval Under Priority Review--
--Enrollment for Phase 2 Study of TPIP in Patients with PAH Completed in December 2024; Expected Timing for Topline Data Moved Forward to Mid-2025--
--Phase 3 ENCORE Trial for ARIKAYCE in Patients with Newly Diagnosed or Recurrent MAC Lung Disease Fully Enrolled; Topline Data Anticipated in First Quarter of 2026--
--IND Cleared for Insmed's First Gene Therapy (INS1201) for Patients with DMD; First Patient Dosing Anticipated in the First Half of 2025--
BRIDGEWATER, N.J., Jan. 10, 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 provided an update on the Company's commercial and clinical programs and its outlook for 2025. These updates will be discussed as part of the Company's presentation at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 13, 2025, at 3:00 p.m. PT (6:00 p.m. ET).
"2024 was an extraordinary year for Insmed, and it is only the beginning of our journey. We believe the upcoming clinical and commercial catalysts have the potential to redefine Insmed from a company that can serve approximately 30,000 patients today to one able to reach more than 2.5 million patients by the end of the decade," said Will Lewis, Chair and Chief Executive Officer of Insmed. "As we prepare for the highly anticipated U.S. approval and launch of brensocatib in bronchiectasis in the third quarter of 2025, we continue to advance our additional clinical programs, including TPIP in PH-ILD and PAH, brensocatib in CRSsNP and HS, and our first gene therapy in DMD. In parallel, we expect to continue to drive double-digit ARIKAYCE growth as we await the readout of ENCORE data, which has the potential to unlock a blockbuster opportunity for the brand."
Preliminary Full-Year 2024 Global Net Product Sales (Unaudited)
Based on preliminary unaudited financial information, the Company expects total global net product sales of ARIKAYCE to be approximately $363.7 million for full-year 2024. This represents 19% year-over-year growth versus full-year 2023, including growth across each of our regions, as follows:
These preliminary unaudited results are subject to adjustment. Insmed will report its final and complete fourth-quarter and full-year 2024 financial results in late February 2025. The actual results could be materially different from these preliminary unaudited financial results.
Progress and Anticipated Milestones by Program:
ARIKAYCE
Insmed anticipates 2025 global ARIKAYCE revenues to be between $405 million and $425 million, representing between 11% and 17% year-over-year growth compared to 2024.
The Company has completed enrollment in the ENCORE trial for patients with newly diagnosed or recurrent Mycobacterium avium complex (MAC) lung disease who had not started antibiotics. Total enrollment in the study was 425 patients, exceeding the target enrollment of 400 patients.
The Company continues to anticipate a topline readout for ENCORE in the first quarter of 2026, with the submission of a supplementary new drug application (sNDA) for ARIKAYCE in all patients with MAC lung disease projected for later in 2026.
Brensocatib
Insmed submitted a new drug application (NDA) for brensocatib for patients with bronchiectasis with the U.S. Food and Drug Administration (FDA) in December 2024 and is currently awaiting FDA acceptance of that submission. If priority review is granted by FDA and brensocatib is approved, the Company anticipates a U.S. launch in the third quarter of 2025.
Regulatory submissions for brensocatib in the EU, UK, and Japan are planned for 2025, with commercial launches anticipated in 2026, pending approval in each territory.
The Phase 2b BiRCh trial of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) has completed nearly 70% of its target enrollment. Topline data continue to be expected before the end of 2025.
The Company randomized its first participant in the Phase 2 CEDAR trial of brensocatib in patients with hidradenitis suppurativa (HS) in December 2024.
TPIP
Insmed will present the full data from the Phase 2 study of treprostinil palmitil inhalation powder (TPIP) in pulmonary hypertension associated with interstitial lung disease (PH-ILD) at the Pulmonary Vascular Research Institute's 2025 Annual World Congress in Rio de Janeiro being held from January 29 through February 1, 2025. The Company plans to initiate a Phase 3 study in PH-ILD in the second half of 2025.
Enrollment in the Phase 2 study of TPIP in pulmonary arterial hypertension (PAH) has been completed with 102 patients randomized in the study. Topline data from the study are now anticipated in the middle of 2025, ahead of the anticipated U.S. launch of brensocatib.
Gene Therapy
Insmed's lead gene therapy is INS1201, an intrathecally-delivered treatment for patients with Duchenne muscular dystrophy (DMD).
In December 2024, Insmed received clearance from the FDA for its investigational new drug (IND) application for INS1201.
The Company plans to initiate a clinical trial of INS1201 in patients with DMD in the first half of 2025.
Pre-Clinical Programs
Insmed's early-stage research efforts include more than 30 identified pre-clinical programs in development, all of which have the potential to become first-in-class or best-in-class therapies for the indications being pursued.
The Company continues to anticipate that the totality of its early-stage research programs will comprise less than 20% of overall expenditure.
Presentation at the 43rd Annual J.P. Morgan Healthcare Conference
Will Lewis, Chair and Chief Executive Officer of Insmed, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 3:00 p.m. PT (6:00 p.m. ET). A live audio webcast of the presentation will be available on the Investor Relations section of the Company's website at . A replay will also be archived for a period of 30 days following the conclusion of the live event.
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, CRSsNP, HS, 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 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 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
, or call 1‑800‑FDA‑1088. You can also call the Company at 1-844-4-INSMED.
Please see
Full Prescribing Information
.
About Insmed
Insmed Incorporated is a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases. The Company is advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving patient communities where the need is greatest. Insmed's most advanced programs are in pulmonary and inflammatory conditions, including a therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company's early-stage research programs encompass a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.
Headquartered in Bridgewater, New Jersey, Insmed has offices and research locations throughout the United States, Europe, and Japan. Insmed is proud to be recognized as one of the best employers in the biopharmaceutical industry, including spending four consecutive years as the No. 1 Science Top Employer. Visit to learn more.
Forward-looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," "intends," "potential," "continues," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements.
The forward-looking statements in this press release are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timings discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: failure to continue to successfully commercialize ARIKAYCE, our only approved product, in the U.S., Europe or Japan (amikacin liposome inhalation suspension, Liposomal 590 mg Nebuliser Dispersion, and amikacin sulfate inhalation drug product, respectively), or to maintain US, European or Japanese approval for ARIKAYCE; our inability to obtain full approval of ARIKAYCE from the FDA, including the risk that we will not successfully or in a timely manner complete the confirmatory post-marketing clinical trial required for full approval of ARIKAYCE, or our failure to obtain regulatory approval to expand ARIKAYCE's indication to a broader patient population; the risk that the full data set from the ASPEN study or data generated in further clinical trials of Brensocatib will not be consistent with the topline results of the ASPEN study or any additional data published from the ASPEN study; failure to obtain, or delays in obtaining, regulatory approvals for brensocatib, TPIP or our other product candidates in the US, Europe or Japan or for ARIKAYCE outside the US, Europe or Japan, including separate regulatory approval for Lamira® in each market and for each usage; failure to successfully commercialize brensocatib, TPIP or our other product candidates, if approved by applicable regulatory authorities, or to maintain applicable regulatory approvals for brensocatib, TPIP or our other product candidates, if approved; uncertainties or changes in the degree of market acceptance of ARIKAYCE or, if approved, brensocatib or TPIP by physicians, patients, third-party payors and others in the healthcare community; our inability to obtain and maintain adequate reimbursement from government or third-party payors for ARIKAYCE or, if approved, brensocatib or TPIP, or acceptable prices for ARIKAYCE or, if approved, brensocatib or TPIP; inaccuracies in our estimates of the size of the potential markets for ARIKAYCE, brensocatib, TPIP or our other product candidates or in data we have used to identify physicians, expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE, brensocatib, or TPIP for commercial or clinical needs, to conduct the Company's clinical trials, or to comply with the Company's agreements or laws and regulations that impact the Company's business; the risks and uncertainties associated with, and the perceived benefits of, our secured senior loan with certain funds managed by Pharmakon Advisors L.P. and our royalty financing with OrbiMed Royalty & Credit Opportunities IV, LP, including our ability to maintain compliance with the covenants in the agreements for the senior secured loan and royalty financing and the impact of the restrictions on our operations under these agreements; our inability to create or maintain an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of ARIKAYCE or any of our product candidates that are approved in the future; failure to successfully conduct future clinical trials for ARIKAYCE, brensocatib, TPIP and our other product candidates and our potential inability to enroll or retain sufficient patients to conduct and complete the trials or generate data necessary for regulatory approval of our product candidates or to permit the use of ARIKAYCE in the broader population of patients with MAC lung disease, among other things; development of unexpected safety or efficacy concerns related to ARIKAYCE, brensocatib, TPIP or our other product candidates; risks that our clinical studies will be delayed, that serious side effects will be identified during drug development, or that any protocol amendments submitted will be rejected; failure to successfully predict the time and cost of development, regulatory approval and commercialization for novel gene therapy products; the risk that interim or partial data sets are not representative of a complete or larger data set or that blinded data will not be predictive of unblinded data; the risk that interim, topline or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available or may be interpreted differently if additional data are disclosed; risk that our competitors may obtain orphan drug exclusivity for a product that is essentially the same as a product we are developing for a particular indication; our inability to attract and retain key personnel or to effectively manage our growth; our inability to successfully integrate our recent acquisitions and appropriately manage the amount of management's time and attention devoted to integration activities; risks that our acquired technologies, products and product candidates are not commercially successful; inability to adapt to our highly competitive and changing environment; inability to access, upgrade or expand our technology systems or difficulties in updating our existing technology or developing or implementing new technology; risk that we are unable to maintain our significant customers; risk that government healthcare reform materially increases our costs and damages our financial condition; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; risk that our current and potential future use of AI and machine learning may not be successful; deterioration in general economic conditions in the US, Europe, Japan and globally, including the effect of prolonged periods of inflation, affecting us, our suppliers, third-party service providers and potential partners; the risk that we could become involved in costly intellectual property disputes, be unable to adequately protect our intellectual property rights or prevent disclosure of our trade secrets and other proprietary information, and incur costs associated with litigation or other proceedings related to such matters; restrictions or other obligations imposed on us by agreements related to ARIKAYCE, brensocatib or our other product candidates, including our license agreements with PARI and AstraZeneca AB , and failure to comply with our obligations under such agreements; the cost and potential reputational damage resulting from litigation to which we are or may become a party, including product liability claims; risk that our operations are subject to a material disruption in the event of a cybersecurity attack or issue; our limited experience operating internationally; changes in laws and regulations applicable to our business, including any pricing reform and laws that impact our ability to utilize certain third parties in the research, development or manufacture of our product candidates, and failure to comply with such laws and regulations; our history of operating losses, and the possibility that we never achieve or maintain profitability; goodwill impairment charges affecting our results of operations and financial condition; inability to repay our existing indebtedness and uncertainties with respect to our ability to access future capital; and delays in the execution of plans to build out an additional third-party manufacturing facility approved by the appropriate regulatory authorities and unexpected expenses associated with those plans.
The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company's forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company's business, please see the factors discussed in Item 1A, "Risk Factors," in the Company's Annual Report on Form 10-K for the year ended December 31, 2023 and any subsequent Company filings with the Securities and Exchange Commission (SEC).
The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
Contacts:
Investors:
Bryan Dunn
Vice President, Investor Relations
(646) 812-4030
[email protected]
Michael V. Morabito, Ph.D.
Director, Investor Relations
(917) 936-8430
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Manager, Investor Relations
(973) 886-2236
[email protected]
Media:
Mandy Fahey
Vice President, Corporate Communications
(732) 718-3621
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SOURCE Insmed Incorporated
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临床2期上市批准临床3期加速审批申请上市
2025-01-08
·药时空
近日，Evaluate发布了2025年制药业展望报告，里面提到了2025年有望上市的最具销售潜力的10款药物名单。从潜在销售额来看，Vertex公司的最新囊性纤维化药物被认为是2025年最重要的获批决定。该公司现有的囊性纤维化产品线的成功意味着，这种三联疗法有望再次取得商业成功。此外，Vertex的第二款有望在2025年上市的药物是Suzetrigine，如果获批，它将是几十年来首次进入市场的新型疼痛机制药物。FDA对第一三共和阿斯利康合作开发的第二种抗体偶联药物（ADC）的决定是另一件大事......下面是这10款药物的具体名单及情况。
1、Vanza Triple
Vanza triple由vanzacaftor、tezacaftor、deutivacaftor组成，前两者旨在通过促进CFTR蛋白的加工和运输，增加细胞表面CFTR蛋白数量，而deutivacaftor是旨在增加送达细胞表面的CFTR蛋白的通道开放概率，以改善细胞膜上盐和水的流动。
自2012年以来，Vertex有4款囊性纤维化（CF）药物在美国批准，在小众赛道建立稳固的“护城河”。随着业内对该疾病认知加深，药物也在不断迭代，竞争者频出。针对新形势，Vertex便推出名为“vanza triple”的三联疗法。与现有CF大单品Trikafta相比，Vanzatriple在疗效和治疗范围上均有所提升，并且每日一次的给药方式更有优势，获批上市后将进一步巩固Vertex在CF领域的地位。FDA做出决定的最后期限是2025年1月2日，但目前未有相关消息传出。
2、Datopotamab Deruxtecan
Datopotamab deruxtecan （Dato-DXd）是一种新型、研究性TROP2抗体偶联药物（ADC），由人源化抗TROP2免疫球蛋白G1单克隆抗体组成，通过一种4肽链接到一种新型高效拓扑异构酶I抑制剂（exatecan衍生物，DXd）。通过选择性地将细胞毒性药物DXd直接递送至肿瘤，从而诱导表达TROP2的肿瘤细胞的DNA损伤和凋亡，同时可以降低全身毒性来解决传统化疗的局限性。
该药物由第一三共和阿斯利康共同开发。2024年12月27日，第一三共宣布Datopotamab deruxtecan（Dato-DXd，德达博妥单抗，商品名：Datroway）在日本获批上市，用于治疗激素受体（HR）阳性、HER2阴性（IHC 0、IHC 1+ 或 IHC 2+/ISH-）且接受过化疗，无法切除或复发的乳腺癌成年患者。
除日本外，该药已在美国（NSCLC、HR+/HER2-乳腺癌）、欧盟（NSCLC、HR+/HER2-乳腺癌）、中国（HR+/HER2-乳腺癌）申报上市，且获FDA突破性治疗认定，用于EGFR突变非小细胞肺癌经治患者。另外值得注意的是，此前2024年12月，阿斯利康和第一三共自愿撤回了在欧盟提交的Dato-DXd肺癌适应症上市申请，具体适应症为局部晚期或转移性非鳞状非小细胞肺癌。
3、Suzetrigine
Suzetrigine（开发代号VX-548）是一种非阿片类小分子镇痛药，可作为周围神经系统中Nav1.8依赖性疼痛信号通路的选择性抑制剂，由Vertex Pharmaceuticals开发。
2024年1月，Vertex Pharmaceuticals宣布Suzetrigine在其3期试验中成功达到了几个终点。该药物缓解了中度至重度术后疼痛。该公司希望这种作用于周围神经的药物能够避免阿片类药物的成瘾性。2024年7月，公司宣布美国食品和药物管理局已接受Suzetrigine的新药申请（NDA）。FDA已授予Suzetrigine优先审查权，并指定了处方药用户费用法案（PDUFA）目标行动日期为2025年1月30日。Suzetrigine已获得FDA快速通道和突破性疗法认定，用于治疗中度至重度急性疼痛。
另外，Vertex Pharmaceuticals还计划寻求用于治疗周围神经性疼痛的广泛标签，理由是其在糖尿病性疼痛性周围神经病变（DPN）方面的第2期结果积极。该药物还在开发中，用于治疗神经根病和其他情况下的疼痛。
4、Aficamten
Aficamten是一种心肌肌球蛋白抑制剂，能够减轻心力衰竭症状、改善运动能力和NYHA 心功能分级。该药物由生物技术公司Cytokinetics自主研发。
在许可权益方面，2020年7月，箕星药业获得在大中华地区开发和商业化Aficamten的独家许可权益。不过，2024年12月，赛诺菲与箕星药业共同宣布签订正式协议，协议约定赛诺菲将收购箕星在大中华区独家开发和商业化aficamten的权益。该交易预计于2024年内完成，交易金额不对外公开。另外，2024年11月，拜耳与Cytokinetics 达成合作和许可协议，在日本独家开发和商业化aficamten，但须遵守 Cytokinetics 的某些保留开发权。
2022年，该药物分别获美国食品药品监督管理局（FDA）及中国国家药品监督管理局（NMPA）授予突破性治疗药物认定。2024年10月，CDE已正式受理aficamten片用于治疗oHCM的新药上市申请并将其纳入优先审评品种。2024年12月，Cytokinetics公司宣布FDA受理了aficamten用于治疗oHCM的新药申请（NDA），处方药使用者消费法案（PDUFA）日期为2025年9月26日。
5、Brensocatib
Brensocatib是一种口服、可逆性DPP1小分子抑制剂。DPP1是一种酶，负责激活中性粒细胞中的中性粒细胞丝氨酸蛋白酶（NSPs）。中性粒细胞是最常见的白细胞类型，在杀伤病原体和介导炎症中起着必不可少的作用。在慢性炎症性肺病中，中性粒细胞在气道中聚集并导致NSP过度活跃，从而引起肺组织损伤和炎症。Brensocatib可以通过抑制DPP1降低NSPs的活化，从而降低炎症性疾病（如支气管扩张）的组织损伤。
Brensocatib最早由阿斯利康研发，2016年10月5日，阿斯利康以3000万美元首付款、1.2亿美元里程金的价格将其全球独家权益转让给了Insmed。2024年5月，Insmed公布了ASPEN III期研究的数据，表明其DPP1抑制剂 brensocatib可以显著减轻非囊性纤维化支气管扩张患者的肺恶化。顶线数据显示，与安慰剂相比，brensocatib的10毫克剂量可使肺恶化的年化率降低21.1%，而25毫克剂量可使肺恶化的年化率降低19.4%。据该公司称，这两种效应均具有统计学意义，p值分别为0.0019和0.0046。
这款疗法已经获得美国FDA授予的突破性疗法认定，用于治疗非囊性纤维化支气管扩张。Insmed预计在2024年第4季度向FDA提交NDA，Brensocatib将于2025年中期在美国上市。Brensocatib有望成为首个获批用于支气管扩张的DPP1抑制剂。此外，Brensocatib针对不伴鼻息肉的慢性鼻窦炎、囊性纤维化和化脓性汗腺炎等适应症正在或即将开展多项临床试验。
6、Tolebrutinib
Tolebrutinib是一种在研的、口服的、可渗透脑的、具有生物活性的布鲁顿酪氨酸激酶（BTK）抑制剂，其脑脊液浓度预计可调节B淋巴细胞和疾病相关的小胶质细胞。
2024年12月13日，赛诺菲宣布，美国食品药品监督管理局（FDA）已批准Tolebrutinib用于治疗成人非复发继发性进行性多发性硬化（nrSPMS）的突破性治疗指定。该药物是第一个也是唯一一个被FDA指定为突破性治疗的多发性硬化脑渗透BTK抑制剂。据悉，目前赛诺菲正在完成向FDA递交Tolebrutinib新药申请的最终准备，并在多个Ⅲ期试验中评估治疗多种类型多发性硬化的潜力。
7、Mazdutide
Mazdutide（玛仕度肽）是由信达生物与礼来合作开发的一款创新药物，属于胰高血糖素样肽-1受体（GLP-1R）/胰高血糖素受体（GCGR）双重激动剂。它通过激动GLP-1R促进胰岛素分泌、降低血糖和减轻体重，同时通过激动GCGR增加能量消耗，增强减重效果，并改善肝脏脂肪代谢。
玛仕度肽在中国超重或肥胖受试者中的III期临床研究（GLORY-1）已经达成主要终点和所有关键次要终点，显示出显著的减重效果和良好的耐受性。此外，玛仕度肽在治疗2型糖尿病方面也展现出了显著的降糖效果，其III期临床研究（DREAMS-2）结果表明，与度拉糖肽相比，玛仕度肽在降糖和减重方面疗效更优。
2024年2月，玛仕度肽的首个新药上市申请（NDA）已获中国国家药品监督管理局（NMPA）药品审评中心（CDE）受理，用于成人肥胖或超重患者的长期体重控制。2024年8月，玛仕度肽的第二项新药上市申请（NDA）也已获中国国家药品监督管理局（NMPA）药品审评中心（CDE）受理，用于成人2型糖尿病（T2D）患者的血糖控制。Evaluate预计，玛仕度肽的肥胖适应症将在2025年上半年获批，T2D适应症将在2025年下半年获批。
8、Depemokimab
Depemokimab（GSK3511294，德莫奇单抗）是GSK开发的靶向IL-5的人源化IgG1κ抗体，IL-5是负责嗜酸性粒细胞生长、激活和存活的细胞因子，和2型炎症相关。该药通过Fc片段的M252Y、S254T、T256E（YTE）突变来延长半衰期，可以实现每年两次给药。
目前，Depemokimab已获得治疗高嗜酸性粒细胞综合征的孤儿药认定。GSK计划在2024年底前在美国提交Depemokimab用于重度哮喘和CRSwNP的申请，并随后在欧盟、中国和日本提交相关申请，预计在2025年实现双重适应症上市。2025年1月1日，据CDE网站显示，GSK的德莫奇单抗在国内申报上市。
9、MenABCWY
五联结合流脑疫苗MenABCWY是GSK已获批上市疫苗Bexsero（B群流脑）和Menveo（ACWY群流脑）的组合，旨在预防由脑膜炎奈瑟菌A、B、C、W和Y血清群（MenABCWY）引起的脑膜炎和败血症。该疫苗涵盖了全球的6个主要致病血清群中的5个，其中B、C 和Y血清群导致了美国大多数侵袭性脑膜炎球菌病（IMD)）病例。2024年4月，GSK发布新闻称FDA 对其脑膜炎球菌5联结合疫苗开始注册审评，PDUFA日期为2025年2月14日。
10、Nipocalimab
Nipocalimab是一款潜在“best-in-class”，靶向FcRn的抗体疗法。它通过与FcRn结合，让被单核细胞和内皮细胞摄入的自身抗体不会重新释放到血液中，而是在细胞内被降解。已有研究显示，它可以将血液循环中包括致病自身抗体在内的IgG水平降低超过75%。这款抗体疗法有望治疗多种自身抗体介导的免疫疾病。
目前，该药物获FDA和EMA授予胎儿和新生儿溶血性疾病（hddn）和温热性自身免疫性溶血性贫血（wAIHA）、全身型重症肌无力（gMG）、胎儿新生儿同种免疫性血小板减少症（FNAIT）快速通道指定；获FDA和EMA授予wAIHA、hddn、慢性炎性脱髓鞘性多发性神经根神经病（CIDP）和FNAIT孤儿药认定；获FDA授予hddn突破性疗法；获EMA授予hddn孤儿药认定。
2024年8月，强生宣布已向美国FDA提交了一份生物制品许可申请（BLA），寻求全球首次批准Nipocalimab用于治疗全身性重症肌无力（gMG）；同年9月，强生宣布向欧洲药品管理局（EMA）提交了市场授权申请（MAA），寻求首次批准Nipocalimab用于治疗全身型重症肌无力（gMG）患者。预计美国和欧盟的决定将在2025年上半年公布。
参考资料：
[1]2025 Preview : What does the year ahead hold for pharma?.Evaluate Pharma.January 2, 2025.
[2]FDA四季度新药审批前瞻.医药经济报.2024年11月01日.
[3]86.3%患者肿瘤缩小或控制稳定！奥希耐药肺癌患者迎来重磅新药.全球肿瘤医讯.2024年12月10日.
[4]全球首批！第一三共 TROP-2 ADC 获批上市.Insight数据库.2024年12月27日.
[5]Vertex的Suzetrigine(VX-548).新药研发信息.2024年12月21日.
[6]赛诺菲：收购箕星“潜在重磅”心血管疗法aficamten中国权益.猎药人俱乐部.2024年12月20日.
[7]有望成为首款！突破性小分子疗法达到3期临床主要终点，即将递交上市申请.药明康德.2024年05月29日.
[8]国产DPP1抑制剂纳入突破性治疗品种，这一赛道生变，海思科强势崛起！.CPHI制药在线.2024年09月25日.
[9]Tolebrutinib被FDA指定为非复发继发性进行性多发性硬化的突破性疗法.香港济民药业.2024年12月18日.
[10]BTK抑制剂上演存亡之战？.药智网.2025年01月06日.
[11]玛仕度肽（Mazdutide）III期临床研究DREAMS-2成果显著，展现超强降糖减重双重功效.石家庄阳康生物科技公司官方号.2024年09月20日.
[12]仅需一年两针，GSK超长效哮喘药在华申报上市.新浪医药.2025年01月02日.
[13]2025年有望申请上市的单抗药物.BiG生物创新社.2025年01月06日.
[14]流脑五联结合苗 GSK紧追辉瑞 赛诺菲虎视眈眈.风禾医学.2024年04月17日.
[15]首个！强生潜在重磅疗法递交上市申请.药明康德.2024年08月30日.
[16]强生公司寻求欧盟首次批准Nipocalimab用于全身型重症肌无力抗体阳性 | 柯西国外资讯.柯西CRO.2024年09月13日.
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抗体药物偶联物上市批准突破性疗法引进/卖出临床结果
2024-10-31
—ARIKAYCE® (amikacin liposome inhalation suspension) Total Revenue of $93.4 Million for the Third Quarter of 2024, Reflecting 18% Growth Over the Third Quarter of 2023—
—NDA Submission for Brensocatib in Bronchiectasis Remains on Track for the Fourth Quarter of 2024 with Potential U.S. Launch Still Expected in Mid-2025—
—Expanded U.S. Sales Force is Now Fully Deployed; Focusing on Bronchiectasis Disease-State Awareness and Supporting the Growth of ARIKAYCE Prior to the Anticipated Launch of Brensocatib—
—Ends the Third Quarter with ~$1.5 Billion in Cash, Cash Equivalents, and Marketable Securities—
—Renegotiates Term Loan with Pharmakon Resulting in a Lower Cost of Capital and an Additional $150 Million in Proceeds to be Received in the Fourth Quarter—
—Reiterates 2024 Global ARIKAYCE Revenue Guidance in the Range of $340 Million to $360 Million, Reflecting Double-Digit Growth Compared to 2023—
BRIDGEWATER, N.J., Oct. 31, 2024 /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 third quarter ended September 30, 2024, and provided a business update.
"I am pleased with the progress the Company is making across multiple ongoing initiatives this quarter." said Will Lewis, Chair and Chief Executive Officer of Insmed. "We remain on track to file our NDA for brensocatib in the fourth quarter of 2024 and continue to expect a potential U.S. launch in the middle of 2025. We have also made great progress on the clinical side, with the ENCORE and PAH studies nearing full enrollment. All of this has been accomplished while delivering yet another quarter of double-digit growth for ARIKAYCE in each of our three commercial regions. With our demonstrated ability to execute both clinically and commercially, and a strengthened balance sheet due to actions we have taken to lower our cost of capital while adding to our cash balance, we believe we are well-positioned to deliver on the tremendous opportunities ahead."
Recent Pillar Highlights
Pillar 1: ARIKAYCE
ARIKAYCE global revenue grew 18% in the third quarter of 2024 compared to the third quarter of 2023, reflecting an all-time revenue high and double-digit year-over-year growth in the U.S., Japan, and Europe and rest of world.
Insmed has closed screening of new patients for the ENCORE study and is now expected to exceed its target enrollment of 400 patients with newly diagnosed or recurrent Mycobacterium avium complex (MAC) lung infection who have not started antibiotics.
The Company is scheduled to meet with the U.S. Food and Drug Administration (FDA) during the fourth quarter to discuss the possibility of an accelerated approval to expand the label for ARIKAYCE to include all patients with MAC lung infection, based on the positive Phase 3 ARISE trial data. Insmed continues to expect that the full data from the ongoing ENCORE trial will be required for approval.
Pillar 2: Brensocatib
Insmed remains on track to file a New Drug Application (NDA) with the FDA for brensocatib in patients with bronchiectasis in the fourth quarter of 2024. If priority review is granted and brensocatib is approved, Insmed anticipates a U.S. launch in mid-2025. Launches in Europe and Japan are expected in the first half of 2026, pending approvals.
New subpopulation data from 19 pre-specified categories of patients in the ASPEN study were presented in October 2024 at the American College of Chest Physicians Annual Meeting in Boston. The annualized rate of pulmonary exacerbations favored brensocatib at both the 10 mg and 25 mg doses over placebo for almost all subgroups. In a separate analysis, least squares mean difference for brensocatib 25 mg demonstrated a reduced decline in post-bronchodilator forced expiratory volume in 1 second (FEV1) at Week 52 versus placebo for all prespecified subgroups.
Insmed is advancing launch readiness activities in the U.S. and has hired, trained, and deployed 120 additional therapeutic sales specialists in advance of launch, focused on bronchiectasis disease-state awareness with an expanded group of pulmonologists while also supporting the growth of ARIKAYCE.
The Company continues to enroll patients in the Phase 2b BiRCh trial of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) and anticipates providing top-line data from the study in the second half of 2025.
The Company is preparing to activate the first U.S. clinical site for its Phase 2 study of brensocatib in patients with hidradenitis suppurativa (HS) by the end of 2024.
Pillar 3: TPIP
Enrollment remains ongoing in the Phase 2 study of treprostinil palmitil inhalation powder (TPIP) in patients with pulmonary arterial hypertension (PAH), with more than 90% of the target enrollment currently complete.
Insmed remains on track to report topline results from the PAH study in the second half of 2025.
The Company continues to anticipate initiating a Phase 3 study of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) in the second half of 2025.
Pillar 4: Early-Stage Research
Insmed's early-stage research efforts include more than 30 identified pre-clinical programs in development, all of which have the potential to become first-in-class or best-in-class therapies.
The Company continues to anticipate the totality of its early-stage research programs will comprise less than 20% of overall spend.
Corporate Updates
Insmed has taken the following actions intended to further strengthen its balance sheet and financial position:
(i) During the third quarter of 2024, the Company completed the redemption of its $225 million convertible notes due in January 2025. Insmed issued approximately 5.7 million shares of common stock in connection with the redemption and earlier conversions of notes.
(ii) The Company raised an additional $371 million in net proceeds under its at-the-market equity offering program during the third quarter, at an average sales price of $75.64 per share.
(iii) In October 2024, Insmed entered into an agreement to amend its $350 million term loan with investment funds managed by Pharmakon Advisors, LP. Under the terms of the amended agreement, investment funds managed by Pharmakon Advisors, LP will provide an additional $150 million in proceeds, to be received in the fourth quarter of 2024. The maturity date for the full principal amount was extended to 2029, and will carry a reduced fixed-interest rate in the high-single digits.
In October 2024, Insmed announced that it has earned the No. 1 ranking in Science's 2024 Top Employers Survey, marking the fourth consecutive year in which Insmed achieved the top ranking. The annual survey polls employees in biotechnology, pharmaceutical, and related industries to determine the 20 best employers, as well as their driving characteristics.
Third-Quarter 2024 Financial Results
Total revenue for the quarter ended September 30, 2024, was $93.4 million, reflecting 18% year-over-year growth compared to total revenue of $79.1 million for the third quarter of 2023.
Total revenue for third-quarter 2024 included ARIKAYCE net sales of $66.9 million in the U.S., $21.0 million in Japan, and $5.6 million in Europe and rest of world. Third-quarter 2024 sales demonstrated year-over-year growth of 13% in the U.S., 31% in Japan, and 45% in Europe and rest of world, reflecting continued growth trends for ARIKAYCE in these regions.
Cost of product revenues (excluding amortization of intangibles) was $21.2 million for the third quarter of 2024, compared to $16.7 million for the third quarter of 2023, primarily reflecting increased sales volumes of ARIKAYCE.
Research and development (R&D) expenses were $150.8 million for the third quarter of 2024, compared to $109.1 million for the third quarter of 2023. The year-over-year increase in R&D expenses was primarily driven by increases in manufacturing and compensation and benefit-related expenses.
Selling, general and administrative (SG&A) expenses for the third quarter of 2024 were $118.9 million, compared to $90.6 million for the third quarter of 2023. The year-over-year increase in SG&A expenses resulted primarily from increases in compensation and benefit-related expenses and stock-based compensation costs due to an increase in headcount associated with launch readiness activities for brensocatib.
For the third quarter of 2024, Insmed reported a net loss of $220.5 million, or $1.27 per share, compared to a net loss of $158.9 million, or $1.11 per share, for the third quarter of 2023.
Balance Sheet, Financial Guidance, and Planned Investments
As of September 30, 2024, Insmed had cash, cash equivalents, and marketable securities totaling $1,467.9 million.
Insmed is reiterating its guidance for full-year 2024 global ARIKAYCE revenues in the range of $340 million to $360 million, representing 15% year-over-year growth at the midpoint compared to 2023.
Insmed continues to anticipate that over 80% of total expenditures will be on its clinical and commercial programs, 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 continue to invest in the following key activities in 2024:
(i) commercialization and continued growth of ARIKAYCE in its current indication globally, as well as advancement of the clinical trial program intended to potentially support label expansion to include all patients with a MAC lung infection and to satisfy the post-marketing requirement for full approval of its current indication;
(ii) advancement of brensocatib, including:
a. activities related to regulatory filing and commercial launch readiness for bronchiectasis and
b. the ongoing Phase 2 BiRCh trial in patients with CRSsNP and the anticipated Phase 2 program in HS;
(iii) advancement of its clinical development programs for TPIP; and
(iv) development of its early-stage research programs.
Conference Call
Insmed will host a conference call beginning today 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 (646) 960-0278 (international) and referencing access code 7862189. The call will also be webcast live on the Company's website at
.
A replay of the conference call will be accessible approximately 1 hour after its completion through November 7, 2024, by dialing (800) 770-2030 (U.S.) and (609) 800-9909 (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
.
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, CRSsNP, 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 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 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
, or call 1‑800‑FDA‑1088. You can also call the Company at 1-844-4-INSMED.
Please see
Full Prescribing Information
.
About Insmed
Insmed Incorporated is a people-first global biopharmaceutical company striving to deliver first- and best-in-class therapies to transform the lives of patients facing serious diseases. The Company is advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving patient communities where the need is greatest. Insmed's most advanced programs are in pulmonary and inflammatory conditions, including a therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease. The Company's early-stage research programs encompass a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.
Headquartered in Bridgewater, New Jersey, Insmed has offices and research locations throughout the United States, Europe, and Japan. Insmed is proud to be recognized as one of the best employers in the biopharmaceutical industry, including spending four consecutive years as the No. 1 Science Top Employer. Visit to learn more.
Forward-looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. "Forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995, are statements that are not historical facts and involve a number of risks and uncertainties. Words herein such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," "intends," "potential," "continues," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) may identify forward-looking statements.
The forward-looking statements in this press release are based upon the Company's current expectations and beliefs, and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results, performance and achievements and the timing of certain events to differ materially from the results, performance, achievements or timings discussed, projected, anticipated or indicated in any forward-looking statements. Such risks, uncertainties and other factors include, among others, the following: failure to continue to successfully commercialize ARIKAYCE, our only approved product, in the U.S., Europe or Japan (amikacin liposome inhalation suspension, Liposomal 590 mg Nebuliser Dispersion, and amikacin sulfate inhalation drug product, respectively), or to maintain US, European or Japanese approval for ARIKAYCE; our inability to obtain full approval of ARIKAYCE from the FDA, including the risk that we will not successfully or in a timely manner complete the confirmatory post-marketing clinical trial required for full approval of ARIKAYCE, or our failure to obtain regulatory approval to expand ARIKAYCE's indication to a broader patient population; failure to obtain, or delays in obtaining, regulatory approvals for brensocatib, TPIP or our other product candidates in the US, Europe or Japan or for ARIKAYCE outside the US, Europe or Japan, including separate regulatory approval for Lamira® in each market and for each usage; failure to successfully commercialize brensocatib, TPIP or our other product candidates, if approved by applicable regulatory authorities, or to maintain applicable regulatory approvals for brensocatib, TPIP or our other product candidates, if approved; uncertainties or changes in the degree of market acceptance of ARIKAYCE or, if approved, brensocatib or TPIP by physicians, patients, third-party payors and others in the healthcare community; our inability to obtain and maintain adequate reimbursement from government or third-party payors for ARIKAYCE or, if approved, brensocatib or TPIP, or acceptable prices for ARIKAYCE or, if approved, brensocatib or TPIP; inaccuracies in our estimates of the size of the potential markets for ARIKAYCE, brensocatib, TPIP or our other product candidates or in data we have used to identify physicians, expected rates of patient uptake, duration of expected treatment, or expected patient adherence or discontinuation rates; failure of third parties on which the Company is dependent to manufacture sufficient quantities of ARIKAYCE, brensocatib, or TPIP for commercial or clinical needs, to conduct the Company's clinical trials, or to comply with the Company's agreements or laws and regulations that impact the Company's business; the risks and uncertainties associated with, and the perceived benefits of, our secured senior loan with certain funds managed by Pharmakon Advisors L.P. and our royalty financing with OrbiMed Royalty & Credit Opportunities IV, LP, including our ability to maintain compliance with the covenants in the agreements for the senior secured loan and royalty financing and the impact of the restrictions on our operations under these agreements; our inability to create or maintain an effective direct sales and marketing infrastructure or to partner with third parties that offer such an infrastructure for distribution of ARIKAYCE or any of our product candidates that are approved in the future; failure to successfully conduct future clinical trials for ARIKAYCE, brensocatib, TPIP and our other product candidates and our potential inability to enroll or retain sufficient patients to conduct and complete the trials or generate data necessary for regulatory approval of our product candidates or to permit the use of ARIKAYCE in the broader population of patients with MAC lung disease, among other things; development of unexpected safety or efficacy concerns related to ARIKAYCE, brensocatib, TPIP or our other product candidates; risks that our clinical studies will be delayed, that serious side effects will be identified during drug development, or that any protocol amendments submitted will be rejected; the risk that interim, topline or preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available or may be interpreted differently if additional data are disclosed, or that blinded data will not be predictive of unblinded data; risk that our competitors may obtain orphan drug exclusivity for a product that is essentially the same as a product we are developing for a particular indication; our inability to attract and retain key personnel or to effectively manage our growth; our inability to successfully integrate our recent acquisitions and appropriately manage the amount of management's time and attention devoted to integration activities; risks that our acquired technologies, products and product candidates are not commercially successful; inability to adapt to our highly competitive and changing environment; inability to access, upgrade or expand our technology systems or difficulties in updating our existing technology or developing or implementing new technology; risk that we are unable to maintain our significant customers; risk that government healthcare reform materially increases our costs and damages our financial condition; business or economic disruptions due to catastrophes or other events, including natural disasters or public health crises; risk that our current and potential future use of AI and machine learning may not be successful; deterioration in general economic conditions in the US, Europe, Japan and globally, including the effect of prolonged periods of inflation, affecting us, our suppliers, third-party service providers and potential partners; the risk that we could become involved in costly intellectual property disputes, be unable to adequately protect our intellectual property rights or prevent disclosure of our trade secrets and other proprietary information, and incur costs associated with litigation or other proceedings related to such matters; restrictions or other obligations imposed on us by agreements related to ARIKAYCE, brensocatib or our other product candidates, including our license agreements with PARI and AstraZeneca AB , and failure to comply with our obligations under such agreements; the cost and potential reputational damage resulting from litigation to which we are or may become a party, including product liability claims; risk that our operations are subject to a material disruption in the event of a cybersecurity attack or issue; our limited experience operating internationally; changes in laws and regulations applicable to our business, including any pricing reform and laws that impact our ability to utilize certain third parties in the research, development or manufacture of our product candidates, and failure to comply with such laws and regulations; our history of operating losses, and the possibility that we never achieve or maintain profitability; goodwill impairment charges affecting our results of operations and financial condition; inability to repay our existing indebtedness and uncertainties with respect to our ability to access future capital; and delays in the execution of plans to build out an additional third-party manufacturing facility approved by the appropriate regulatory authorities and unexpected expenses associated with those plans.
The Company may not actually achieve the results, plans, intentions or expectations indicated by the Company's forward-looking statements because, by their nature, forward-looking statements involve risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. For additional information about the risks and uncertainties that may affect the Company's business, please see the factors discussed in Item 1A, "Risk Factors," in the Company's Annual Report on Form 10-K for the year ended December 31, 2023 and any subsequent Company filings with the Securities and Exchange Commission (SEC).
The Company cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date of this press release. The Company disclaims any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.
Contact:
Investors:
Bryan Dunn
Executive Director, Investor Relations
(646) 812-4030
[email protected]
Michael V. Morabito, Ph.D.
Director, Investor Relations
(917) 936-8430
[email protected]
Gianna De Palma
Manager, Investor Relations
(873) 886-2236
[email protected]
Media:
Mandy Fahey
Vice President, Corporate Communications
(732) 718-3621
[email protected]
SOURCE Insmed Incorporated
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临床2期临床3期上市批准财报申请上市
100 项与 Brensocatib 相关的药物交易
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外链
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研发状态
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 非囊性支气管扩张 | 申请上市 | 美国 | 2024-12-01 | |
| 新型冠状病毒感染 | 临床3期 | 英国 | 2020-06-05 | |
| 化脓性汗腺炎 | 临床2期 | 美国 | 2024-12-16 | |
| 慢性鼻窦炎 | 临床2期 | 美国 | 2023-10-30 | |
| 慢性鼻窦炎 | 临床2期 | 阿根廷 | 2023-10-30 | |
| 慢性鼻窦炎 | 临床2期 | 澳大利亚 | 2023-10-30 | |
| 慢性鼻窦炎 | 临床2期 | 比利时 | 2023-10-30 | |
| 慢性鼻窦炎 | 临床2期 | 保加利亚 | 2023-10-30 | |
| 慢性鼻窦炎 | 临床2期 | 加拿大 | 2023-10-30 | |
| 慢性鼻窦炎 | 临床2期 | 捷克 | 2023-10-30 |
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| 研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
|---|
临床3期 | - | 鹽網鏇醖簾糧壓衊淵鏇(選觸窪構遞範築廠艱淵) = 壓鑰選糧鬱餘醖製衊醖 窪糧鏇觸積構積窪糧簾 (繭遞憲顧願窪願鏇壓餘 ) 更多 | 积极 | 2024-10-08 | |||
鹽網鏇醖簾糧壓衊淵鏇(選觸窪構遞範築廠艱淵) = 艱獵淵獵鑰齋夢艱觸築 窪糧鏇觸積構積窪糧簾 (繭遞憲顧願窪願鏇壓餘 ) 更多 | |||||||
临床3期 | 1,721 | Brensocatib 10 mg | 顧夢膚夢簾鹹糧淵鬱蓋(選構鑰蓋構憲鹹餘獵積) = 襯窪顧餘鹹築蓋夢選夢 鑰壓艱淵顧鏇窪製壓襯 (鹽夢襯夢積觸獵糧鏇齋 ) 更多 | 积极 | 2024-05-28 | ||
Brensocatib 25 mg | 顧夢膚夢簾鹹糧淵鬱蓋(選構鑰蓋構憲鹹餘獵積) = 選艱構積憲鬱齋廠鹹鹽 鑰壓艱淵顧鏇窪製壓襯 (鹽夢襯夢積觸獵糧鏇齋 ) 更多 | ||||||
ATS2024 人工标引  | 临床1期 | 28 | (Subjects with mild renal impairment) | 醖鏇觸糧鹽壓遞鹽壓鑰(壓構鹹餘襯網積齋憲膚) = There were no treatment-emergent AEs, serious AEs, or death during this study. 願構鏇遞醖鑰製簾窪選 (淵糧鹹醖淵簾獵蓋鹽製 ) | 积极 | 2024-05-22 | |
(Subjects with moderate renal impairment) | |||||||
临床3期 | 406 | (Brensocatib) | 蓋顧觸餘範襯窪鹹積鹽(窪鑰範鏇蓋顧築夢鬱膚) = 築簾網顧鹽鏇鹽窪構觸 積遞餘簾範簾壓鹽網範 (鬱遞簾齋膚襯築醖醖獵, 廠積憲網築艱窪淵願願 ~ 顧鹽顧顧鹹觸積壓衊鏇) 更多 | - | 2023-08-22 | ||
Placebo (Placebo) | 蓋顧觸餘範襯窪鹹積鹽(窪鑰範鏇蓋顧築夢鬱膚) = 憲鹽選繭製壓遞鹽餘願 積遞餘簾範簾壓鹽網範 (鬱遞簾齋膚襯築醖醖獵, 範選構夢鬱範醖鹹簾繭 ~ 憲鬱繭構觸艱製淵憲選) 更多 | ||||||
N/A | - | 積壓顧夢顧蓋構觸範襯(廠廠鹹廠鏇鬱鏇醖衊鏇) = 構窪淵鹽鑰夢糧網構鬱 糧鹽餘範餘蓋糧鏇廠壓 (積壓選選糧壓簾醖構艱 ) | - | 2023-05-21 | |||
Placebo | 積壓顧夢顧蓋構觸範襯(廠廠鹹廠鏇鬱鏇醖衊鏇) = 衊艱夢鬱艱衊齋鹽膚鹽 糧鹽餘範餘蓋糧鏇廠壓 (積壓選選糧壓簾醖構艱 ) | ||||||
临床2期 | 256 | (Brensocatib 10 mg) | 築願鬱齋獵餘鹽顧餘糧(簾窪艱繭範範範築範鑰) = 鑰壓網鏇夢觸襯製選網 製襯鏇選遞齋觸鏇淵製 (願網築壓簾膚壓積構製, 餘網遞簾積壓鹽衊膚鏇 ~ 製願鑰願餘簾鹹觸構窪) 更多 | - | 2023-02-08 | ||
(Brensocatib 25 mg) | 築願鬱齋獵餘鹽顧餘糧(簾窪艱繭範範範築範鑰) = 鏇製鏇顧繭築選範餘構 製襯鏇選遞齋觸鏇淵製 (願網築壓簾膚壓積構製, 鬱選構鹹鹽淵繭範鹽鹽 ~ 壓顧觸窪夢網鑰製壓觸) 更多 | ||||||
临床1期 | - | - | Brensocatib 10mg | 築艱鹽餘鬱艱衊顧憲襯(製衊齋憲鏇壓顧齋憲餘) = The most frequently reported treatment-emergent adverse events were headache 鏇顧鬱積衊憲範積鑰鏇 (壓蓋製糧網獵簾獵廠鹹 ) 更多 | - | 2022-07-01 | |
Brensocatib 40mg | |||||||
临床3期 | 404 | Standard of Care+brensocatib | 餘網廠簾築鏇夢繭壓艱(衊製鹹鏇觸餘鏇鑰積鹽) = Brensocatib did not improve the clinical status of patients hospitalized 衊遞選淵簾蓋鹹餘築窪 (廠築選觸鹹鬱製獵淵醖 ) 更多 | 不佳 | 2022-05-18 | ||
Standard of Care+Placebo | |||||||
N/A | - | - | 鹽顧鏇夢廠製壓夢淵糧(積膚艱網襯膚築繭醖齋) = The most frequently reported TEAE was skin exfoliation, reported by 1 participant in each ethnic group, both in the fed state 壓製願繭繭獵齋獵鏇醖 (艱構鬱鹹醖蓋製觸顧積 ) 更多 | - | 2021-05-03 | ||
Placebo | |||||||
临床2期 | 支气管扩张 neutrophil elastase | 256 | Placebo | 艱壓夢鹹餘膚襯鏇獵齋(鑰鏇憲蓋糧鏇餘夢窪獵) = 憲鬱範襯夢顧窪醖鑰淵 醖獵艱襯衊齋鹽壓壓遞 (窪製蓋遞淵壓構鏇鑰鑰 ) | 积极 | 2020-11-26 | |
艱壓夢鹹餘膚襯鏇獵齋(鑰鏇憲蓋糧鏇餘夢窪獵) = 衊夢艱選製廠築夢餘艱 醖獵艱襯衊齋鹽壓壓遞 (窪製蓋遞淵壓構鏇鑰鑰 ) |
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