In a recent press release, biopharmaceutical company Regulus Therapeutics, Inc. announced that their drug candidate, RGLS4326, received Orphan Drug Designation for the treatment of autosomal dominant polycystic kidney disease (ADPKD). This status is only granted to drugs and biologics designed to treat patients with rare diseases. For all intents and purposes, rare diseases are considered which affect 200,000 U.S. citizens or less.
There are two main forms of polycystic kidney disease: autosomal dominant and autosomal recessive. These refer to the way in which the disease is inherited from parents. Resulting from PKD1 and PKD2 genetic mutations, ADPKD causes cysts to develop in and on the kidneys. As a result, kidney function falls, preventing the organs from effectively filtering waste. Generally, cysts appear between the ages of 30 and 40. An estimated 500,000 Americans have polycystic kidney disease.
Symptoms of ADPKD include:
- High blood pressure
- Abdominal and back pain
- Kidney cysts
- Kidney stones
- Abdominal fullness
- Heart abnormalities
- Brain aneurysms
Learn more about ADPKD here.
Developed by Regulus Therapeutics, RGLS4326 is a novel oligonucleotide designed to regulate and inhibit miR-17. According to the NIH, this microRNA plays a role in hepatic (liver) lipid metabolism. However, RGLS4326 is designed to target and benefit kidney function.
Data from preclinical studies highlight the role of RGLS4326 in improving kidney function and slowing the growth of cysts. Additionally, Regulus Therapeutics tested RGLS4326 on healthy individuals. They found that the drug is well-tolerated and safe. Next, Regulus Therapeutics will explore RGLS4326 in a Phase 1b clinical trial. This will test the drug’s efficacy, safety, and tolerability directly for patients with ADPKD.
Since the drug received Orphan Drug Designation, Regulus Therapeutics also receives a variety of benefits. These include market exclusivity (if the drug gets approved), tax credits, and additional communication with the FDA.