Ligand Pharmaceuticals Incorporated announced that data from a Phase I study with its selective androgen receptor modulator (SARM) LGD-4033 was featured today in a poster presentation at the 14th International Congress of Endocrinology in Kyoto, Japan. This Phase I clinical trial was the first study in humans of LGD-4033, and evaluated the safety, tolerability and pharmacokinetic profiles of the molecule in a single escalating dose, double blind, placebo-controlled study in 48 healthy volunteers.
The key findings include:
– LGD-4033 was well tolerated by healthy male volunteers after single oral doses up to 22 mg, the highest dose tested. No serious adverse events (SAE) or clinically significant dose-related adverse events were reported.
– Systemic exposure of LGD-4033 increased proportionally with the dose level after a single oral dose. Sustained systemic exposure was observed with appreciable plasma levels of LGD-4033 detectable a week post-dose. LGD-4033’s half-life was consistent with a regimen of once-daily oral dosing.
LGD-4033 is a non-steroidal selective androgen receptor modulator (SARM), expected to produce the therapeutic benefits of testosterone with improved safety, tolerability and patient acceptance due to tissue-selective mechanisms of action and oral routes of administration. Ligand has discovered several orally active, non-steroidal SARM compounds based on tissue-specific gene expression and other functional, cell-based technologies. LGD-4033 exhibited desirable in vivo efficacy on skeletal muscle and bone measurements in animal models of male hypogonadism and postmenopausal osteoporosis. The clinical applications for SARMs include the treatment of multiple muscle wasting disorders (e.g., sarcopenia, cachexia and frailty), the treatment of osteoporosis, male hypogonadism and female sexual dysfunction.