Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Liposomal amikacin for inhalation (LAI), Amikacin Liposome Inhalation Suspension (ALIS), ARIKACE™, liposomal amikacin for inhalation
Arikayce · 3 trials · 2 indications
Number of Participants with indicated Adverse Events in subjects receiving 560 mg once daily dose of Arikayce™ administered for 6 cycles over 18 months.
Number of Subjects reporting TEAE in the Arikayce™ groups and the placebo groups during the study. The table shows the events incidents, not the number of participants.
Number of subjects reporting Incidence of clinically significant abnormalities in clinical values (Common Terminology Criteria for Adverse Events \[CTCAE\] grade \>= 3) in Arikayce™ and placebo groups.
Changes in PFT from pre-dose during the study were measured on Days 1, 14, and 28. Acute tolerability of the study treatment was assessed by examining the relative (rel.) changes in FEV1 from pre-dose assessments to 0-1 hour post-dose and 2-4 hours post-dose for each time point at which post-dose spirometry was conducted.
Number of Subjects with Decrease of \>= 15% in FEV1 (L) from Pre- to Post-dose by Study Day
Number of subjects with a SAE in the Arikace™ groups and the placebo group up to 28 days after study medication discontinuation. See SAE table in the safety section for details.
To evaluate the safety and tolerability of 28 days of daily dosing of nebulized Arikayce™, liposomal amikacin for inhalation.
| Arm | Type | Description |
|---|---|---|
| 560 mg Arikayce™ | EXPERIMENTAL | Subjects in this cohort will receive 560 mg of Arikayce™ |
| Cohort 1 - 280 mg Arikayce™ | EXPERIMENTAL | Subjects in this arm of the cohort 1 will receive 280 mg of Arikayce™ |
| Cohort 1 - Placebo | PLACEBO_COMPARATOR | Subjects in this arm of the cohort 1 will receive matching placebo. |
| Cohort 2 - 560 mg Arikayce™ | EXPERIMENTAL | Subjects in this arm of the cohort 2 will receive 560 mg of Arikayce™ |
| Cohort 2 - Placebo | PLACEBO_COMPARATOR | Subjects in this arm of the cohort 2 will receive matching placebo |
| A | ACTIVE_COMPARATOR | Arikayce™ at 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo. |
| B | PLACEBO_COMPARATOR | Matching placebo for 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo. |
| C | ACTIVE_COMPARATOR | Arikayce™ at 70 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo. |
| D | ACTIVE_COMPARATOR | Arikayce™ at 140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo. |
| E | PLACEBO_COMPARATOR | Matching placebo for 70 mg/140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo. |
| Name | Type | Description |
|---|---|---|
| Arikayce™ | DRUG | Amikacin (aminoglycoside) in a liposomal formulation. |
| 280 mg Arikayce™ | DRUG | Study subjects will receive Arikace™ 280 mg on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| Matching Placebo for Cohort 1 | DRUG | Study subjects will receive placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| 560 mg Arikayce™ | DRUG | Study subjects will receive Arikace™ 560 mg on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| Matching Placebo for Cohort 2 | DRUG | Study subjects will receive placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| Arikayce™ 560 mg | DRUG | Arikayce™ at 560 mg Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| Placebo for 560 mg | DRUG | Matching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| Arikayce™ 70 mg | DRUG | Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| Arikayce™ 140 mg | DRUG | Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
| Placebo for 70 mg / 140 mg | DRUG | Matching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer. |
Inclusion Criteria: 1. Written informed consent obtained from the patient or designated legal guardian prior to the performance of any study related procedures. 2. Male or female study subjects ≥ 6 years of age or older. 3. Confirmed diagnosis of CF defined as a positive sweat chloride \> 60 millie...
Top 4 of 6 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| AstraZeneca PLC | AZN | 3 | PHASE2 | AZD0292 |
| GSK plc Sponsored ADR | GSK | 1 | PHASE2 | GSK3862995B |
| Merck & Co., Inc. | MRK | 1 | PHASE2 | Ensifentrine |
| Genentech, Inc. | RHHBY | 1 | PHASE1 | GDC-6988 |
Arikayce is being studied in bronchiectasis and cystic fibrosis, two respiratory conditions. The clinical program has focused on patients with chronic infection due to Pseudomonas aeruginosa. Both indications are being evaluated in Phase 2 trials, and the studies enrolled adults and children as young as 6 years old.
Arikayce is being developed by Insmed Incorporated, which trades on the Nasdaq under the ticker INSM. Insmed is the sponsor of the clinical trials evaluating the drug in bronchiectasis and cystic fibrosis, including the dose escalation and safety studies listed on ClinicalTrials.gov.
Arikayce is in Phase 2 clinical development for bronchiectasis and cystic fibrosis. The program includes completed Phase 2 trials in both indications and an earlier Phase 1 study in cystic fibrosis. It remains investigational and has not been approved by the FDA for these uses.
Arikayce has been studied in completed trials including NCT03905642, a Phase 2 dose escalation extension study in cystic fibrosis, NCT00775138, a Phase 2 safety and tolerability study in bronchiectasis with chronic Pseudomonas aeruginosa infection, and NCT00558844, a Phase 1 cystic fibrosis study. All three trials have been completed.
Across the three completed trials, 154 patients have been enrolled in studies of Arikayce. The Phase 2 cystic fibrosis extension study enrolled 49 patients, the Phase 2 bronchiectasis study enrolled 64 patients, and the Phase 1 cystic fibrosis study enrolled 41 patients.