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Dicerna Receives Breakthrough Therapy Designation for DCR-PHXC for Treatment of Primary Hyperoxaluria Type 1.

M2 PHARMA-July 16, 2019-Dicerna Receives Breakthrough Therapy Designation for DCR-PHXC for Treatment of Primary Hyperoxaluria Type 1

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- The US Food and Drug Administration has granted a Breakthrough Therapy Designation to DCR-PHXC for the treatment of patients with primary hyperoxaluria type 1, US-based ribonucleic acid interference therapies developer Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA) said.

DCR-PHXC is the only RNAi investigational therapy in development for the treatment of all types of PH, a family of severe, rare, inherited disorders of the liver that often result in kidney failure.

In its communication to Dicerna, the FDA also conveyed its determination that PH type 2 and PH type 3 meet the criteria for a serious or life-threatening disease or condition, based on the Agency's standards.

The company will continue its ongoing dialogue with the FDA regarding endpoints for studies of DCR-PHXC in patients with PH2 and PH3, as part of the PHYOX clinical development programme.

The FDA's BTD is intended to expedite the development and review of a drug candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The BTD offers companies the opportunity for increased communication with FDA and an organizational commitment involving more intensive guidance from FDA senior managers.

Dicerna recently presented updated data from the PHYOX1 Phase 1 clinical trial of DCR-PHXC, which reported substantial post-dose reductions in 24-hour urinary oxalate levels in adult and adolescent study participants with PH1 and PH2.

The updated PHYOX1 data, presented at the Oxalosis and Hyperoxaluria Foundation's International Workshop on June 22, 2019, also showed that a single dose of DCR-PHXC led to normalisation or near-normalization of urinary oxalate levels in a majority of patients and was generally well-tolerated.

DCR-PHXC is the only RNAi investigational drug in development for the treatment of all types of primary hyperoxaluria, and the most advanced product candidate utilizing Dicerna's GalXC technology.

GalXC is a proprietary platform invented by Dicerna scientists to discover and develop next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. In animal models of PH, DCR-PHXC selectively silences lactate dehydrogenase A enzyme, or LDHA, in the liver, blocking the excess production of oxalate, a hallmark of the disease.

In preclinical studies of DCR-PHXC, the compound was well tolerated with no adverse effects in the liver. Studies have shown that people who are completely deficient in LDHA show no liver dysfunction and can lead normal lives. LDHA deficiency in the liver may be beneficial for patients with PH, as the LDHA enzyme is implicated in the abnormal production of oxalate in PH, which in turn is responsible for the severe damage to kidneys and other organs in patients with PH.

Dicerna is evaluating DCR-PHXC in the PHYOX clinical trial program. Interim results from the ongoing PHYOX1 Phase 1 study have demonstrated normalization or near-normalization of urinary oxalate levels in a majority of participants receiving DCR-PHXC, as well as a favourable tolerability profile.

Primary hyperoxaluria is a family of severe, rare, genetic liver disorders characterized by overproduction of oxalate, a natural chemical in the body that is normally eliminated as waste through the kidneys. In patients with PH, the kidneys are unable to eliminate the large amount of oxalate that is produced.

The accumulation of oxalate can result in severe damage to the kidneys and other organs.

There are three known genetic types of PH, each of which results from a mutation in a specific gene. In each type, the mutation decreases the activity of an enzyme in the liver, leading to an increase in the production of oxalate.

Dicerna Pharmaceuticals is a biopharmaceutical company using RNA interference, or RNAi, to create medicines that silence genes that cause disease.

The company's proprietary GalXC technology is intended to amplify its ability to create potent, selective and safe RNAi therapies to treat diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases and viral infectious diseases.

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Date:Jul 16, 2019
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