Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| AstraZeneca PLC | AZN | 2 | PHASE2 | AZD0292 |
| GSK plc Sponsored ADR | GSK | 1 | PHASE2 | GSK3862995B |
| Merck & Co., Inc. | MRK | 1 | PHASE2 | Nebulized Ensifentrine |
| Baxter International Inc. | BAX | 1 | N/A | Undisclosed |
| Inogen, Inc. | INGN | 1 | N/A | Undisclosed |
Arikayce is an investigational small molecule being studied for respiratory conditions, specifically bronchiectasis and cystic fibrosis, in patients with chronic Pseudomonas aeruginosa infection. It is delivered via inhalation as a liposomal formulation of amikacin. As of the available data, Arikayce remains in clinical development and is not FDA approved.
Arikayce targets Pseudomonas aeruginosa, a bacterium that causes chronic lung infections in patients with bronchiectasis and cystic fibrosis. It is a liposomal formulation of amikacin, an aminoglycoside antibiotic, designed for inhalation to deliver the drug directly to the lungs.
Arikayce is developed by Insmed Incorporated, a biopharmaceutical company traded on NASDAQ under the ticker INSM. Insmed is responsible for the clinical development and potential commercialization of this investigational therapy.
Arikayce is in Phase 2 clinical development. The available data includes completed Phase 1 and Phase 2 trials, with no active trials currently listed. It is an investigational drug and has not received FDA approval.
Arikayce has been studied in three completed clinical trials: NCT00558844 (Phase 1 in cystic fibrosis patients), NCT00775138 (Phase 2 in bronchiectasis patients), and NCT03905642 (Phase 2 dose escalation study in cystic fibrosis). These trials evaluated safety and tolerability.
Yes, Arikayce is also known as liposomal amikacin for inhalation. This formulation delivers the antibiotic amikacin in a liposomal form for inhalation therapy, targeting chronic Pseudomonas aeruginosa infections in patients with bronchiectasis or cystic fibrosis.