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Alnylam publish successful results from Phase III study of ultra-rare disease

Published on 06/04/21 at 11:47am

Alnylam Pharmaceuticals, a RNAi therapeutics company, have published successful results from their Phase III study of lumasiran, the first ever therapy approved for the treatment of primary hyperoxaluria type 1 (PH1) – an ultra-rare genetic disease characterised by oxalate overproduction.

Lumasiran, approved in the EU and US and marketed as Oxlumo, is the only available pharmacological treatment that can reduce production of oxalate, which is the toxic metabolite responsible for the severe symptoms of PH1, including the formation of painful and recurrent kidney stones and progression to kidney failure.

The trial results, published online in The New England Journal of Medicine (NEJM), showed that Oxlumo significantly reduced the levels of urinary oxalate relative to a placebo, with the majority of patients achieving normal or near-normal levels of urinary oxalate.

The Phase III trial, ILLUMINATE-A, was the largest controlled study ever conducted into PH1 with Oxlumo being the first treatment approved for PH1 and the first RNAi therapeutic evaluated in both children and adults.

A total of 38 patients completed the ILLUMINATE-A six-month primary analysis period, and all eligible patients transitioned to the study extension period. Results from the 12-month extension period were presented at the American Society of Nephrology (ASN) virtual congress in October 2020 and demonstrated sustained efficacy, with no adverse safety findings.

Prof. Yaacov Frishberg MD, Head of Division of Paediatric Nephrology, Shaare Zedek Medical Center, and lead co-author on the manuscript, said: “PH1 often presents in early life, with kidney stones, nephrocalcinosis, renal failure and, in advanced stages, systemic spread of oxalate throughout the body with life-threatening consequences. Oxalate drives disease manifestations and progression, and is the toxic mediator of end-organ damage in PH1.

“We believe the publication of the ILLUMINATE-A Phase III study results in the NEJM is a testament to lumasiran as an oxalate-lowering therapy which is expected to confer significant clinical benefit to children and adults living with this disease.”

Alnylam is currently evaluating Oxlumo across disease severity through the ILLUMINATE clinical program and in mid-2021, Alnylam is expecting to announce topline results from the ILLUMINATE-C Phase III study in PH1 patients with advanced renal disease, exploring the potential of Oxlumo in a broader range of PH1 patients.

Kat Jenkins

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