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LAI plus multi-drug regimen

Phase 3

Mycobacterium Infections, Nontuberculous | Small molecule | Infectious Disease |Insmed Incorporated|Last Updated: May 7, 2020

Target and mechanism

ModalitySmall molecule

Also known as LAI

Success Probability

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Market & Valuation

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Trial Design

RandomizedNO_TREATMENT_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment366

FDA Designations

No designations recorded

Clinical trial landscape

LAI plus multi-drug regimen · 3 trials · 3 indications

Phase 3 2Phase 2 1
NCT02628600Open-label Safety Extension Study Assessing Safety and Tolerability of LAI in Patients Who Participated in Study INS-212NTM Lung Infection Due to MAC
COMPLETED163 Analytics
NCT02344004Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen AloneMycobacterium Infections, Nontuberculous
COMPLETED336 Analytics
PHASE3COMPLETED
Open-label Safety Extension Study Assessing Safety and Tolerability of LAI in Patients Who Participated in Study INS-212
NTM Lung Infection Due to MACUnlock trial analytics
PHASE3COMPLETED
Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone
Mycobacterium Infections, NontuberculousUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
From baseline to 28 days after end of treatment, up to 13 months

TEAEs are defined as those AEs that occurred on or after the date of first dose of study medication in INS-312 and within 28 days after the last dose. If it couldn't be determined whether the AE is treatment emergent due to a partial onset date, then it was classified as treatment emergent.

Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm
by Month 6

Sputum specimens were collected at Screening (Visit 1), Baseline (Visit 2), and at Visits 3 (Month 1) through 8 (Month 6). A negative culture result reflected a negative culture result for all sputum samples collected at each visit. Participants met the primary endpoint of culture conversion by Month 6 if they had 3 consecutive monthly MAC-negative sputum cultures during the first 6 months of the study. A participant needed to achieve the first of 3 consecutive negative sputum cultures (that defined culture conversion) by Month 4 in order to meet the primary endpoint by Month 6. Each participant in the intent to treat (ITT) population (ie, all randomized participants) was classified as either a converter or non-converter by Month 6.

Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone
up to Month 19

Sputum specimens were collected at screening, baseline (Day 1), during treatment, and at Months 1, 3, 6, and 12 months off treatment. Culture conversion with durability was defined as achieving culture conversion by Month 6 and then having no more than 2 consecutive broth positive cultures and no Agar positive culture up to 3 months off treatment. Converters with missing broth or Agar sputum culture result after Month 6 up to 3 months off treatment were considered as not achieving culture conversion with durability except those participants who are unable to produce sputum despite reasonable efforts, as reported by source documentation. Participants who had relapse/recurrence, had "rescue" medication and/or died before reaching 3 months off treatment were considered as not achieving culture conversion with durability.

Change from Baseline sputum culture at 12 months
Sputum examined for culture change from Baseline at 12 months

Secondary Endpoints

Number of Participants Achieving Culture Conversion by Month 6 and Month 12
by Month 6 and Month 12
Time to Culture Conversion
by Month 12
Change From Baseline (Day 1) to Month 6 and Month 12 in the 6MWT Distance
From baseline to Month 12 or end of treatment
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Prior LAI + Multidrug RegimenEXPERIMENTALParticipants in the prior Study INS-212 who received LAI+MDR. All participants in this safety extension study received LAI+MDR.
Prior Multidrug Regimen AloneEXPERIMENTALParticipants in the prior Study INS-212 who received MDR alone. All participants in this safety extension study received LAI+MDR.
Multi-drug RegimenNO_INTERVENTIONParticipants received their already prescribed anti-mycobacterial regimen (based on the 2007 American Thoracic Society/Infectious Diseases Society of America \[ATS/IDSA\] Guidelines)
LAI + Multi-drug RegimenEXPERIMENTALParticipants received LAI 590 mg QD in addition to their already prescribed anti-mycobacterial regimen (based on the 2007 ATS/IDSA Guidelines) LAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes
LAI plus multi-drug regimenEXPERIMENTALonce daily dosing of Liposomal-Amikacin for Inhalation (LAI) 590 mg for 12 months plus standard of care (SOC) mycobacterial multi-drug regimen in accordance with the 2007 ATS/ IDSA guidelines

Interventions

NameTypeDescription
LAI 590 mgDRUGLAI 590 mg QD: administered by inhaling drug product that had been aerosolized in an investigational eFlow nebulizer over approximately 14 minutes
Multi-drug regimenDRUGMultidrug antimycobacterial regimen from study INS-212
LAI (Liposomal Amikacin for Inhalation) 590 mgDRUGLAI 590 mg QD, administered by inhaling drug product that had been aerosolized in an eFlow nebulizer over approximately 14 minutes.
LAI plus multi-drug regimenDRUGLiposomal Amikacin for Inhalation (LAI) is the experimental treatment which, in this single arm will be taken in conjunction with standard of care multi-drug treatment regimen
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo

Key Inclusion Criteria: 1. had successfully completed the Month 6 and End of Treatment visits in Study INS-212 2. had not achieved the INS-212 protocol definition of culture conversion by Month 6 in Study INS-212 OR had experienced a relapse or recurrence by Month 6 in Study INS-212. Key Exclusion...

Countries:United States
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Frequently asked questions about LAI plus multi-drug regimen

What is LAI used for?

LAI is an investigational small molecule being developed for the treatment of nontuberculous mycobacterial infections, including NTM lung infection due to MAC. It is intended to be added to a multi-drug regimen for patients with these infections. The drug is currently in Phase 3 clinical development.

Who makes LAI?

LAI is being developed by Insmed Incorporated, a biopharmaceutical company traded on NASDAQ under the ticker symbol INSM. The company is conducting clinical trials to evaluate the safety and efficacy of LAI in patients with nontuberculous mycobacterial infections.

What phase is LAI in?

LAI is in Phase 3 clinical development. Two Phase 3 trials have been completed, including a study evaluating the efficacy of LAI when added to a multi-drug regimen compared to the regimen alone, and an open-label safety extension study. LAI remains investigational and is not yet approved.

What clinical trials is LAI in?

LAI has been studied in two completed Phase 3 clinical trials. The first, NCT02344004, enrolled 336 participants and evaluated LAI added to a multi-drug regimen versus the regimen alone in patients with nontuberculous mycobacterial infections. The second, NCT02628600, was an open-label safety extension study enrolling 163 patients with NTM lung infection due to MAC.

Is LAI the same as Arikayce?

LAI is not the same as Arikayce. LAI is a separate investigational drug being developed by Insmed Incorporated for nontuberculous mycobacterial infections. While both are associated with Insmed, they are distinct assets with different development programs and are not interchangeable.