Benzinga | Nov 2, 2020 08:11AM EST

Regulus Therapeutics Achieves Interim Enrollment In Phase 2 Study Of RG-012 For Alport Syndrome, Triggering $5M Payment From Sanofi Collaboration

Regulus to Receive Remaining $5 Million Payment for Enrollment Milestone

LA JOLLA, Calif., Nov. 2, 2020 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs, today announced that it has achieved the remaining $5 million milestone associated with interim enrollment under its Collaboration and License Agreement (the "Milestone") with Sanofi for its development of miR-21 programs. The Milestone was triggered upon achievement of an enrollment metric by Sanofi in its Phase 2 clinical study evaluating RG-012 for the treatment of patients with Alport Syndrome. The proceeds from this Milestone will be used to pay down the Company's term loan with Oxford LLC. The payment to Oxford will reduce the remaining principal due under the term loan to approximately $4.7 million and enable the Company to receive an extension of interest-only payments through 2021, an extension of seven months from the previous terms.

"We are very pleased with the commitment of our partner, Sanofi, to this important program," stated Jay Hagan, CEO of Regulus. "This is an important step in advancing a potential treatment for patients with Alport Syndrome, a rare genetic condition affecting the kidneys with no approved therapies."

Previously, the Company announced completion of the Phase 1b renal biopsy study, where patients with Alport Syndrome were dosed every other week for one year. Results from that study demonstrated kidney tissue concentrations that would be predictive of therapeutic benefit based on animal disease models as well as engagement of the target, miR-21. Over the course of the one year open-label study, promising trends in disease markers were observed and RG-012 was generally well tolerated with no serious adverse events. RG-012 has received orphan designation in both the U.S. and Europe.

Under the terms of the Collaboration and License Agreement, the Company is also eligible to receive an additional $25.0 million upon the successful achievement of a development milestone anticipated in 2023 related to the ongoing Phase 2 HERA study.