View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. News
December 18, 2019

Alnylam’s late-stage trial of lumasiran in PH1 meets endpoints

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).

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.

Related Companies

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Clinical Trials Arena