Dicerna Sells Oxlumo Royalty Interest to Royalty Pharma for up to $240 Million

Rare Daily Staff

Dicerna Pharmaceuticals said that Royalty Pharma has acquired Dicerna’s royalty interest in Oxlumo (lumasiran) for an upfront cash payment of $180 million and up to $60 million in contingent sales-based milestone payments.

Oxlumo, which the U.S. Food and Drug Administration and European Medicines Agency approved for the treatment of primary hyperoxaluria (PH) type 1, is marketed by Alnylam Pharmaceuticals.

“This agreement with Royalty Pharma reflects our mutually held recognition of the importance of this market and the high unmet need for patients with PH—a population that Dicerna is also seeking to address with our lead product candidate, nedosiran, which we are developing for PH types 1, 2 and 3,” said Douglas Fambrough, president and CEO of Dicerna.

Primary hyperoxaluria (PH) is a family of ultra-rare, life-threatening genetic disorders that initially manifest with complications in the kidneys. There are three known types of PH (PH1, PH2 and PH3), each resulting from a mutation in one of three different genes. These genetic mutations cause enzyme deficiencies that result in the overproduction of a substrate called oxalate. Abnormal production and accumulation of oxalate leads to recurrent kidney stones, nephrocalcinosis and chronic kidney disease that may progress to end-stage renal disease requiring intensive dialysis. Compromised renal function eventually results in the accumulation of oxalate in a wide range of organs including the skin, bones, eyes and heart.

In the most severe cases, symptoms start in the first year of life. A combined liver-kidney transplant may be undertaken to resolve PH1 or PH2, but it is an invasive solution with limited availability and high morbidity that requires lifelong immune suppression to prevent organ rejection. Genetic studies suggest approximately 8,500 people in the U.S. are affected by PH, and researchers estimate that more than 80 percent of patients remain undiagnosed. There is currently only one approved therapy available specifically for PH that is for the treatment of patients with PH1.

Dicerna became entitled to royalties on worldwide net product sales of Oxlumo as part of a 2020 non-exclusive intellectual property cross-license agreement between Dicerna and Alnylam related to the companies’ PH programs. Dicerna is entitled to royalties in the mid to high single digits based on Oxlumo global net sales.

Photo: Douglas Fambrough, president and CEO of Dicerna