Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as liposomal amikacin for inhalation, Arikayce
ARIKACE · 4 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.
Changes in chemistry and hematology lab tests (clinically significant value of CTCAE grade ≥ 3).
| 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. |
| Cohort 1 - 280 mg ARIKACE™ | EXPERIMENTAL | Subjects in this cohort will receive 280 mg of ARIKACE™ |
| Cohort 2 - 560 mg ARIKACE™ | EXPERIMENTAL | Subjects in this cohort will receive 560 mg of ARIKACE™ |
| 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. |
| ARIKACE™ | DRUG | Study start date is before Jan 18, 2017. |
| Placebo | DRUG | Study start date is before Jan 18, 2017. |
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 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-581, VX-272 |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
ARIKACE is an investigational inhaled therapy being studied for cystic fibrosis, a genetic disease that affects the lungs. It was evaluated in a Phase 1 trial in patients with cystic fibrosis. It is not an approved treatment and remains in clinical development.
ARIKACE is being developed by Insmed Incorporated, which trades on the Nasdaq under the ticker INSM. Insmed is the sponsor of the ARIKACE clinical program in cystic fibrosis.
ARIKACE is in Phase 1 development. One Phase 1 trial has been completed, and no active trials are currently listed. The program remains investigational and has not been approved by the FDA for any indication.
ARIKACE was studied in one completed Phase 1 trial, NCT00777296, titled Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE). The trial enrolled 66 patients with cystic fibrosis across Belgium, Hungary, North Macedonia, Poland, Serbia, Slovakia, and Ukraine.
The ARIKACE Phase 1 trial NCT00777296 was a randomized, placebo-controlled, dose-escalation study of liposomal amikacin for inhalation. It enrolled 66 patients with cystic fibrosis, including patients as young as 6 years of age, and assessed multidose safety and tolerability. The study has been completed.
Yes. ARIKACE is the brand name for liposomal amikacin for inhalation, an investigational small molecule formulation developed by Insmed Incorporated. The Phase 1 trial NCT00777296 was conducted under the ARIKACE name and evaluated this inhaled liposomal amikacin product in cystic fibrosis.