Proof of Concept Data Released for Potential Primary Hyperoxaluria Treatment

According to a story from BioPortfolio, the drug development company Dicerna Pharmaceuticals recently announced the release of proof of concept data in its Phase I trial testing the company’s experimental product DCR-PHXC. This drug is in development for the treatment of primary hyperoxaluria. Dicerna is a leader in the development of ribonucleic acid interference based therapeutics.

About Primary Hyperoxaluria

Primary hyperoxaluria is a rare medical condition which is most characterized by the chronic appearance of kidney and bladder stones. These stones appear as the result of the excessive production of a substance called oxalate. The disease is often identified by the excessive concentration of oxalate in the urine; oxalate combines with calcium in order to form bladder and kidney stones. There are three types of primary hyperoxaluria which are caused by mutations affecting the AGXT, GRHPR, and HOGA1 genes respectively. Symptoms of this condition include bloody urine, injury to the kidneys and other organs, urinary tract infections, and end stage kidney disease. When concentrations of oxalate are especially high, the substance can make bones more likely to break. Treatment is mostly symptomatic; the only disease modifying approach available is a transplant of the kidney and liver. To learn more about primary hyperoxaluria, click here.

Study Results

In the proof of concept assessment, DCR-PHXC was able to bring oxalate levels down to normal in that majority of eight patients that participated in the study. These patients had either type 1 or type 2 variant of primary hyperoxaluria. The investigational therapy also appeared to be safe and well tolerated in the short term period of this initial trial. The most significant side effect were injection site reactions, but they did not occur in all patients.

The results of this early trial was an encouraging step for the company as the drug was able to display its potential in treating potentially all forms of primary hyperoxaluria. DCR-PHXC acts on an enzyme called lactase dehydrogenase, which plays a role in the production of oxalate, which occurs in the liver.

Overall, the data from the proof of concept trial looks promising. Hopefully, this means that DCR-PHXC will be an effective treatment for primary hyperoxaluria in the future.


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