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 an investigational small molecule being developed for the treatment of acromegaly, a condition caused by excess growth hormone. It is intended for patients with acromegaly who are already being treated with long-acting somatostatin receptor ligands. The drug is currently in Phase 2 clinical development and is not yet approved.
IONIS GHR-LRx targets the growth hormone receptor, reducing the activity of growth hormone in the body. By blocking this receptor, the drug aims to lower the elevated hormone levels that drive acromegaly. This mechanism is designed to complement existing treatments such as somatostatin receptor ligands, which are commonly used in acromegaly management.
IONIS GHR-LRx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical trials to evaluate the safety and efficacy of this drug in patients with acromegaly who are on stable treatment with long-acting somatostatin receptor ligands.
IONIS GHR-LRx is in Phase 2 clinical development. Two Phase 2 trials have been completed, with a total of 82 participants enrolled. Both trials were randomized, double-blind, and placebo-controlled, and they evaluated the drug in patients with acromegaly being treated with long-acting somatostatin receptor ligands. The drug is investigational and not yet approved.
IONIS GHR-LRx has been studied in two completed Phase 2 trials. The first, NCT03548415, enrolled 43 participants with acromegaly on long-acting somatostatin receptor ligands. The second, NCT03967249, was an extension study with 39 participants. Both trials were conducted in the United States and several European countries, including Hungary, Lithuania, Poland, Romania, Russia, and Serbia.
IONIS GHR-LRx is a distinct investigational drug, not the same as existing acromegaly treatments. It is designed to target the growth hormone receptor, whereas standard therapies like somatostatin receptor ligands work through different mechanisms. It is being studied specifically as an add-on to these ligands, rather than as a replacement.