Regulus Receives Orphan Medicinal Product Designation from the European Commission for RG-012, a microRNA Therapeutic in Development for the Treatment of Alport Syndrome
LA JOLLA, Calif., March 25, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that the European Commission has granted orphan medicinal product designation for RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 (“miR-21″) for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy. In July 2014, the U.S. Food & Drug Administration granted orphan drug designation to RG-012 for the treatment of Alport syndrome.
“We are pleased to have received orphan medicinal product designation in the European Union for RG-012, a key microRNA therapeutic program under our ‘Clinical Map Initiative’,” said Paul Grint, M.D., Chief Medical Officer of Regulus. “Alport syndrome is a life threatening disease and patients have very limited treatment options because there is currently no approved therapy. We believe that RG-012 represents an opportunity to make a significant impact in the lives of patients with Alport syndrome and we look forward to advancing this program into the clinic.”
Regulus is currently enrolling patients in a natural history of disease study called ATHENA to gather information about the changes in renal function over time in patients with Alport syndrome. Data from [click to continue…]
Regulus Announces Key Goals Under its ‘Clinical Map Initiative’ for 2015
Accelerates RG-101 for HCV with Dual-Track Clinical Development Strategy; Top-Line, Single Dose, 4 mg/kg Results as Well as 2mg/kg
Extended Follow Up Results from Ongoing Study to be Reported in Early February 2015
LA JOLLA, Calif., Jan. 8, 2015 /PRNewswire/ – Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced key goals for 2015 under its ‘Clinical Map Initiative’ to advance its microRNA therapeutics portfolio and biomarkers platform.
“Regulus enters 2015 with the scientific and financial strength to realize the transformative potential of microRNAs. As such, we’ve set aggressive goals for the year focused on creating a clear path to value for what we believe to be our greatest opportunities,” said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of Regulus. “Under our ‘Clinical Map Initiative’, we are focusing our near term efforts on accelerating RG-101 for HCV with a Phase II dual-track clinical development strategy, while advancing our overall therapeutics pipeline and aligning our biomarker efforts to streamline our clinical development decisions.”
Key Goals Under Regulus’ ‘Clinical Map Initiative’ for 2015
- ‘Clinical Map’ of RG-101 for HCV Defined: Dual-Track Strategy Accelerates Phase II Development; Multiple Data Read-Outs in 2015. Following the favorable interim results reported inOctober 2014 from its ongoing clinical study, Regulus has accelerated development of RG-101, a wholly-owned, GalNAc-conjugated anti-miR targeting microRNA-122 (“miR-122″) for the treatment of HCV. Regulus is pursuing a Phase II dual-track development strategy (i) to investigate RG-101 in combination with oral agents to potentially shorten treatment durations, optimize clinical outcomes and potentially improve responses in certain underserved HCV patient populations; and (ii) to investigate RG-101 further as a single agent to determine [click to continue…]
-New study published in Science Translational Medicine demonstrates microRNA-21 contributes to fibrogenesis in the kidney
-Regulus, in partnership with Sanofi, developing novel anti-fibrotic therapies targeting microRNAs
B.N. Chau et al.
“MicroRNA-21 Promotes Fibrosis of the Kidney by Silencing Metabolic Pathways”
Sci Transl Med 15 February 2012:
Vol. 4, Issue 121, p. 121ra18
Sci. Transl. Med. DOI: 10.1126/scitranslmed.3003205
LA JOLLA, Calif., Feb. 16, 2012 /PRNewswire/ — Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, today announced that new preclinical data investigating the role of microRNA-21 (miR-21) in the treatment of kidney fibrosis has been published in the journal Science Translational Medicine. Regulus’ lead program for fibrosis targets miR-21, which is up-regulated in fibrotic tissues of humans. Previous preclinical studies by Regulus scientists and collaborators have shown that therapeutic oligonucleotides targeting miR-21 (anti-miR-21) can decrease fibrosis in preclinical models by reducing the expression of extracellular matrix proteins. Despite the current burden of fibrosis-related human disease, there are few therapies that can specifically treat this devastating disease.
“We are pleased with the published results demonstrating that targeting miR-21 with proprietary anti-miR oligonucleotides is effective at preventing kidney fibrosis in preclinical models,” said Neil W. Gibson, Ph.D., Regulus’ Chief Scientific Officer. ”We plan to select an anti-miR-21 development candidate [click to continue…]