Regulus Raises $100 Million in PIPE Financing to Advance Rare Kidney Disease Pipeline

Rare Daily Staff 

Regulus Therapeutics, a company focused on innovative microRNA therapeutics for orphan kidney diseases, entered into a securities purchase agreement in connection with a private placement to certain institutional investors and other accredited investors for gross proceeds of approximately $100 million.

The oversubscribed financing includes participation from new and existing institutional investors, including Adage Capital Partners, Deep Track Capital, the Federated Hermes Kaufmann Funds, New Enterprise Associates, Octagon Capital, RA Capital Management, and Vivo Capital.

Regulus designs and develops therapies to target microRNA regulation of protein expression, which are non-coding RNAs that function as important regulators of gene expression of messenger RNA and play a role in multiple cellular processes. They do this by binding specific mRNA and blocking translation, leading to control of gene expression and direct degradation of target mRNA.

The company’s lead candidate RGLS8429 is a novel, next-generation oligonucleotide for the treatment of autosomal dominant polycystic kidney disease (ADPKD) and is designed to inhibit miR-17 and to preferentially target the kidney.

ADPKD is caused by mutations in the PKD1 or PKD2 genes and is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50 percent of ADPKD patients by age 60. Approximately 160,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million.

The company recently completed enrollment in the third cohort of patients in the phase 1b MAD study of RGLS8429 for the treatment of ADPKD, a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of RGLS8429 in adult patients with ADPKD. A fourth cohort of subjects will also be enrolled who will receive an open label fixed dose of RGLS8429 to compare biomarker and safety data to the weight-based dosing.