Alnylam Pharmaceuticals has reported new positive interim results from a Phase l/ll trial of lumasiran for the treatment of Primary Hyperoxaluria Type 1 (PH1).

The results are from Part B of the trial and include findings until the data cut-off date of 29 March this year.

Part B is a randomised, single-blind, placebo-controlled analysis of lumasiran to treat patients with PH1.

During the trial, cohorts one and two received three monthly doses of lumasiran at 1mg/kg or 3mg/kg, respectively; while cohort three received two quarterly doses at 3mg/kg.

An additional eight patients were treated with open-label lumasiran in expansions of each of the first two cohorts, thereby bringing the number of patients enrolled in the trial to 20.

The patients randomised to the placebo group were given subsequent subcutaneous administration of lumasiran following administration of placebo.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

The new findings have reported that lumasiran showed a mean maximal reduction in urinary oxalate of 64% in patients enrolled in cohorts one-three.

“Furthermore, we believe these results validate our approach of targeting GO, a key liver enzyme involved in the excessive oxalate output in patients with PH1.”

All the patients treated with lumasiran experienced a reduction in urinary oxalate below 0.7mmol/24 hrs/1.73m², a threshold level associated with a reduced rate of progression to end-stage renal disease.

On day 85 of the trial, patients receiving lumasiran maintained a mean reduction in urinary oxalate of 63%.

Alnylam Pharmaceuticals Lumasiran programme vice-president and general manager Pritesh Gandhi said: “Given the encouraging results, we believe that lumasiran has the potential to alleviate the pathologic overproduction of oxalate, the metabolite that causes the severe, systemic manifestations of PH1.

“Furthermore, we believe these results validate our approach of targeting GO, a key liver enzyme involved in the excessive oxalate output in patients with PH1.

“Based upon our recent discussions with the FDA, we are on track to advance this programme into Phase lll development at mid-year, with the goal of bringing lumasiran to patients around the world as rapidly as possible.”

Alnylam is currently continuing dosing patients in Part B of the Phase l/ll trial and expects to transition the eligible patients into an open-label extension (OLE) study.

The company aims to present additional data from all cohorts, as well as from the OLE study later this year.