Alice Melão,  —

Reata Pharmaceuticals’ investigational therapy bardoxolone methyl may effectively improve and preserve renal function in patients with chronic kidney disease caused by Alport syndrome, topline results from a Phase 3 clinical trial show. The pivotal trial, called CARDINAL (NCT03019185), met both its primary and secondary objectives. Namely, patients…

Reata Pharmaceuticals has reacquired development, manufacturing, and marketing rights for its proprietary Nrf2 activator products — including bardoxolone methyl (bardoxolone) for the treatment of Alport syndrome and omaveloxolone for Friedreich’s ataxia — the company announced. This follows the termination of a partnership with…

  The care of Alport syndrome patients at specific critical junctions — diagnosis, transition to adult care, and pregnancy — is among the topics of a recently published review study. The review, “The importance of clinician, patient and researcher collaborations in Alport syndrome,” was published in the journal…

A new genetic variant of the COL4A5 gene was identified in the family of a Chinese boy with atypical X-linked Alport syndrome. The case was reported in the study “Identification of a novel COL4A5 mutation in the proband initially diagnosed as IgAN from a Chinese family with…

Patients with X-linked or autosomal recessive Alport syndrome are at risk for developing hypertensive disease, which may also worsen their disease symptoms, a study shows. The study, “Increased microvascular disease in X-linked and autosomal recessive Alport syndrome: a case control cross sectional observational study,” was…

A new mice model carrying a COL4A5 gene mutation that mimics many of the clinical features of human X-linked Alport syndrome was developed by Japanese researchers. These animals can now be used to better understand the underlying metabolic and biochemical mechanisms involved in the development and progression of…

Abnormal activation of the immune system’s complement pathway — a common response to fight germs — may represent a mechanism shared among three distinct kidney conditions, a case report suggests. The rare case report of a patient who showed signs of thrombotic microangiopathy (TMA), Alport syndrome, and atypical…

Inhibition of the DDR1 enzyme with a newly identified small molecule compound can prevent renal tissue scarring and renal function loss in a mouse model of Alport syndrome, a study shows. The study, “DNA-encoded library-derived DDR1 inhibitor prevents fibrosis and renal function loss in a genetic mouse…

Under a restructured agreement with Regulus Therapeutics, Sanofi Genzyme now has sole responsibility for the development and marketing of the investigational therapy RG-012, currently under evaluation for the treatment of Alport syndrome. The amendment to the prior collaboration agreement between Sanofi and Regulus, RG-012’s initial developer, gives…

Topline results from the Phase 2 PHOENIX trial with Reata Pharmaceuticals’ bardoxolone methyl continue to demonstrate its potential to improve the outcome of patients with rare forms of chronic kidney diseases (CKD), including Alport syndrome and type 1 diabetic CKD. The PHOENIX study (NCT03366337) is assessing the safety…