Ligand’s drug found effective against thrombocytopenia

LGD-4665 showed impressive activity following single and multiple doses with an increase in mean maximum platelet counts of 58% with a single dose administration of 120 mg and 83% with 10 mg dosed daily for 14 days. Results demonstrated that with a day 1 loading dose, the increase in mean maximum platelet counts was 27% with 2.5 mg, 43% with 5.0 mg and 79% with 7.5 mg daily for 13 days. A gradual decline in platelet levels was observed post-treatment. LGD-4665 was well-tolerated and demonstrated an encouraging safety profile at all dose levels and all dosing regimens.

Ligand’s Phase I clinical trial evaluated three dosing regimens of LGD-4665, including single doses, multiple daily doses for 14 days, and day 1 loading doses followed by daily doses for 13 days. The placebo-controlled, double-blind, dose-escalating Phase I clinical trial was conducted at a single center in 106 healthy male subjects. In the single dose portion of the trial, six subjects were randomized in the several dose cohorts to receive either a single dose of LGD-4665 or placebo (in a 2:1 ratio). Dose levels ranged from 1mg to 120mg, and were escalated sequentially based on the review of safety and activity (increase in platelet levels) according to predefined criteria.

Zofia Dziewanowska, vice president of clinical research, at Ligand, said: “Based on its strong potency, convenience and potential for dosing flexibility, we believe that LGD-4665 could be used as treatment for a wide variety of diseases associated with thrombocytopenia, including hepatitis C, chemotherapy-induced thrombocytopenia (CIT), myelodysplastic syndromes (MDS), idiopathic thrombocytopenic purpura (ITP) and several other cancers and liver diseases.”