Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
IONIS GHR-LRx · 2 trials · 1 indication
Incidence of adverse events (AEs), serious AEs, treatment related AEs, AEs leading to withdrawal
IGF-1 is a hormone that manages the effects of growth hormone (GH) in the body. Percent change from Baseline in IGF-1 levels was measured at Day 141. Baseline was defined as the last non-missing value prior to the first administration of Study Drug (ISIS 766720 or placebo). A negative percent change from Baseline indicated improvement. To perform a meaningful assessment of the pharmacodynamic (PD) activity of ISIS 766720, the lower dose groups (60 mg and 80 mg) and higher dose groups (120 mg and 160 mg) were combined to achieve group size of 7 or more for PD assessments and these were designated as low-dose and high-dose groups respectively.
A TEAE was defined as an adverse event that occurred after the initiation of study drug dosing and before the end of the follow-up period.
Vitals signs included blood pressure, heart rate, respiratory rate, and temperature recorded throughout the study. Clinical significance was determined by the investigator.
Physical examination included weight and body mass index (BMI) recorded throughout the study. Clinical significance was determined by the investigator.
Clinical laboratory assessments included clinical chemistry, hematology, and urinalysis. Clinically-significant abnormal laboratory values were reported as TEAEs if the results may, in the opinion of the Investigator, constitute or be associated with an AE.
ECG assessments included QT, QRS duration, PR interval, ventricular rate, QTcB, QTcF.
| Arm | Type | Description |
|---|---|---|
| IONIS GHR-LRx + Somatostatin Receptor Ligand (SRL) | EXPERIMENTAL | IONIS GHR-LRx (as per dose in previous study) will be administered subcutaneously once every 28 days for 53 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants received placebo by subcutaneous injection (SC) once every 4 weeks for 16 weeks. |
| Cohort A: IONIS GHR-LRx, 60 mg | EXPERIMENTAL | Participants received IONIS GHR-LRx, 60 milligrams (mg), SC, once every 4 weeks for 16 weeks. |
| Cohort B: IONIS GHR-LRx, 80 mg | EXPERIMENTAL | Participants received IONIS GHR-LRx, 80 mg, SC, once every 4 weeks for 16 weeks. |
| Cohort C: IONIS GHR-LRx, 120 mg | EXPERIMENTAL | Participants received IONIS GHR-LRx, 120 mg, SC, once every 4 weeks for 16 weeks. |
| Cohort D: IONIS GHR-LRx, 160 mg | EXPERIMENTAL | Participants received IONIS GHR-LRx, 160 mg, SC, once every 4 weeks for 16 weeks. |
| Name | Type | Description |
|---|---|---|
| IONIS GHR-LRx | DRUG | Participants will receive IONIS GHR-LRx by subcutaneous injection. |
| Somatostatin Receptor Ligand (SRL) | DRUG | Participants will receive Somatostatin Receptor Ligand (lanreotide, octreotide) once monthly. |
| IONIS-GHR-LRx | DRUG | IONIS GHR-LRx administered subcutaneously. |
| Placebo | DRUG | Placebo administered subcutaneously. |
Inclusion Criteria: * Randomized in index trial (CS2) and completed the entire study, or completion of the treatment period for CS2 and completed or plan to complete PTWK5 visit with an acceptable safety profile, per investigator judgment * Participants with confirmed stable monthly regimen of SRL ...
IONIS GHR-LRx is being developed for the treatment of acromegaly, an endocrine disorder. It is studied in adults with acromegaly who are already being treated with long-acting somatostatin receptor ligands. It is an investigational therapy and has not been approved for commercial use.
IONIS GHR-LRx is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker IONS. The company sponsored the clinical program evaluating the drug in acromegaly, including the Phase 2 safety and efficacy study and its extension study.
IONIS GHR-LRx is in Phase 2 development. Both of its clinical trials in acromegaly were Phase 2 studies, and both have been completed. The drug remains investigational and is not FDA approved. No active trials are currently listed for this program.
IONIS GHR-LRx has been evaluated in two completed Phase 2 trials in acromegaly. NCT03548415 tested safety, tolerability, and efficacy in 43 participants, and NCT03967249 was an extension study in 39 participants. Both enrolled adults on long-acting somatostatin receptor ligands across sites in the United States and Europe.
Yes, IONIS GHR-LRx and IONIS-GHR-LRx refer to the same investigational drug. The two spellings differ only by a hyphen and appear interchangeably in trial records and company materials for the acromegaly program developed by Ionis Pharmaceuticals.
Both Phase 2 trials of IONIS GHR-LRx were randomized, double-blind, and placebo-controlled, enrolling a combined 82 adults with acromegaly. Participants were at least 18 years old and were receiving long-acting somatostatin receptor ligands. The studies took place in the United States, Hungary, Lithuania, Poland, Romania, Russia, and Serbia.