Date: 2014-11-15
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the American Society of Nephrology's (ASN) Kidney Week 2014 meeting being held November 11-16, 2014 in Philadelphia, PA
Company: Sanofi (France) Regulus Therapeutics (USA - CA)
Product: RG-012 (single stranded, chemically modified oligonucleotide that binds to and inhibits the function of micro RNA-21)
Action
mechanism:
- microRNA. Regulus has discovered that miR-21 is highly overexpressed in mouse models of Alport syndrome. Regulus is developing RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of miR-21, which has demonstrated potent inhibition of miR-21 in vitro and in vivo, a decrease in the rate of progression of renal fibrosis, an increase in the lifespan of mice by up to fifty percent, and a favorable pharmacokinetic profile that supports the potential for a once per week dosing regimen.
Disease: Alport syndrome
Therapeutic
area: Rare diseases - Genetic diseases - Kidney diseases
Country:
Trial
details:
Latest
news:
- • On November 15, 2014, Regulus Therapeutics announced that positive new preclinical data was presented on RG-012 for the treatment of renal dysfunction in Alport syndrome patients at the American Society of Nephrology's (ASN) Kidney Week 2014 meeting. Alport syndrome is a life-threatening, genetic kidney disease with no approved therapy, and currently, ACE (angiotensin-converting enzyme) inhibitors are emerging as standard of care to treat proteinuria, an indicator of chronic kidney disease in these patients.
- Studies have shown that miR-21 plays a role in the progression of Alport syndrome and is up-regulated in mouse disease models, other renal fibrosis models and human CKD patients. These findings, and the new preclinical data presented at the ASN meeting, provide additional evidence that RG-012 may provide therapeutic benefit in the treatment of Alport syndrome.
- In an oral presentation titled "Anti-miR-21 as a Potential Novel Therapy for Both Early and Late Stages of Alport Syndrome", Regulus' strategic alliance partner, Genzyme presented new data demonstrating that treatment with RG-012 in a mouse model of disease improves renal function, protects against kidney damage and extents the lifespan of the mice when treatment begins at both early and late stages of the disease by reversing multiple dysregulated pathways. Further, combination studies were performed with RG-012 and ramipril, Sanofi's ACE inhibitor, a kidney protectant, to assess the therapeutic utility of adding a microRNA therapeutic to this emerging standard of care. In these preclinical studies, treatment with RG-012 was shown to protect kidney function better than ramipril alone and an additive therapeutic effect was observed in combination with ramipril.
Is
general: Yes
