Ionis and Roche Advance First RNA-Targeted Therapy for Autoimmune Kidney Disease

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) and licensing partner Roche Holdings AG (OTC:RHHBY) on Wednesday shared prespecified interim outcomes from their ongoing Phase 3 IMAgINATION trial of investigational sefaxersen in adults diagnosed with primary IgA nephropathy (IgAN). "These interim phase 3 results show the clinical potential of sefaxersen to modify a key surrogate endpoint of kidney function in people with IgA nephropathy’’ said Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development. "Sefaxersen may therefore offer a new treatment option to help slow disease progression and potentially reduce the long-term need for dialysis or kidney transplantation," Garraway further added. Phase 3 Trial Meets Primary Endpoint The study met its primary endpoint at 37 weeks. Patients receiving once-monthly subcutaneous injections of sefaxersen demonstrated statistically significant and clinically meaningful reductions in proteinuria relative to placebo, as measured by the 24-hour urine protein-to-creatinine ratio (UPCR). Proteinuria represents a key indicator of renal damage, and lowering UPCR correlates with preserving long-term kidney function. Sefaxersen displayed a safety and tolerability profile consistent with previously reported data, with no new safety signals identified. Read Also: Ionis to Seek Expedited FDA Review for ALS Drug Sustained Evaluation And Disease Impact The study will continue for 105 weeks to evaluate changes in estimated glomerular filtration rate (eGFR) as a measure of long-term kidney function. IgAN, also known as Berger’s disease, is a serious, chronic, and progressive autoimmune kidney disease affecting at least 25 adults per million globally each year. Typically diagnosed before age 40, immune complex accumulation in the kidneys activates the alternative complement pathway, causing inflammation that leads to end-stage kidney disease in up to 50% of patients within 20 years. Novel RNA-Targeted Mechanism And Partnership Terms Sefaxersen is a highly specific, liver-directed antisense oligonucleotide that inhibits factor B production, representing the first RNA-targeted therapy for IgAN. Serum complement factor B levels are elevated in IgAN patients, and sefaxersen provides sustained control of the alternative complement pathway in a convenient self-administered monthly dose. The Phase 3 multicenter study enrolled 459 high-risk patients. Under the agreement, Roche licensed sefaxersen for complement-mediated diseases. Ionis received an upfront payment, license fee, and development milestones, and remains eligible for regulatory and sales milestones alongside tiered royalties on net sales. IONS Price Action: Ionis Pharmaceuticals shares were up 2.71% at $46.99 during premarket trading on Wednesday, according to Benzinga Pro data. Read Also: AI Trade Is ‘Dead Money’: Fidelity Strategist Warns $3.3 Trillion Buildout Is Inflationary With ‘Unknown Returns’ Photo: Shutterstock