Alnylam Pharmaceuticals Phase III ILLUMINATE-A clinical trial of lumasiran has met the primary and secondary endpoints in patients suffering from primary hyperoxaluria type 1 (PH1).
Lumasiran is an investigational, subcutaneous gene silencing RNAi therapeutic that targets hydroxyacid oxidase 1 (HAO1), which is involved in the development of PH1.
The 30-patient ILLUMINATE-A trial compared a 3mg/kg monthly dose of lumasiran to placebo for three months at 16 sites across eight countries.
The primary endpoint was the percentage change in average 24-hour urinary oxalate excretion over three to six months compared to placebo.
Alnylam’s drug also demonstrated statistical significance for all tested secondary endpoints, including near-normal or normal urinary oxalate levels.
Lumasiran was generally well tolerated, with no serious or severe adverse events observed during the trial. The overall profile was generally consistent with that found in Phase I/II and open-label extension studies.
Alnylam Pharmaceuticals R&D president Akshay Vaishnaw said: “The results from ILLUMINATE-A demonstrate that lumasiran can significantly reduce the hepatic production of oxalate, which we believe can thereby address the underlying pathophysiology of PH1.
“Further, we are encouraged by the safety and tolerability profile of lumasiran and believe this investigational medicine has the potential to have a meaningful clinical impact on patients living with PH1.”
Based on the Phase III ILLUMINATE-A data, the company intends to submit applications early next year seeking approval for lumasiran from the US and European regulatory authorities.
The drug holds orphan drug designation in the US and EU, along with breakthrough therapy designation in the US and priority medicines (PRIME) designation in the EU.
Alnylam is also assessing the drug in the Phase III ILLUMINATE-B and ILLUMINATE-C trials to treat PH1.