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Ionis Partner Roche Licenses Rare Kidney Disease Treatment

July 12, 2022

Ionis Pharmaceuticals said that its long-standing partner, Roche, will license and advance IONIS-FB-LRx, an investigational antisense medicine, into a phase 3 clinical study in patients with the rare and chronic kidney disease immunoglobulin A nephropathy.

Photo: Michael McCaleb, vice president, clinical development at Ionis

As a result, Ionis will receive $55 million from Roche for licensing IONIS-FB-LRx for IgAN and achieving a development milestone in the GOLDEN study, a phase 2 clinical study to determine whether the medicine can slow or halt the progression of geographic atrophy due to age-related macular degeneration, or AMD.

Roche will lead and be responsible for the phase 3 study of IONIS-FB-LRx in patients with IgAN and for future global development, regulatory and commercialization activities.

Immunoglobulin A nephropathy (IgAN) is an important cause of chronic kidney disease and renal failure. Also known as Berger’s disease, IgAN is characterized by deposition of IgA and Complement 3 (C3) activation products in the glomerular mesangium of the kidneys, resulting in inflammation and tissue damage. Although IgAN may occur at any age, it generally presents in the second or third decade of life. The clinical presentation, disease progression and histologic findings are highly variable among affected individuals. Current therapies are aimed at reduction of protein levels in the urine with administration of angiotensin inhibitors and control of blood pressure. Sometimes immunosuppressive therapies are given; however, this practice is not universally accepted.

IONIS-FB-LRx, an antisense drug using Ionis’ advanced LIgand Conjugated Antisense (LICA) technology, reduces the production of FB, a key protein in the complement innate immune system. FB is predominately produced in the liver and circulates throughout the vascular system, including vessels in the eye and kidney. This complement protein plays a pivotal role in an innate immunogenic cascade that, when overactivated, has been associated with the development of several complement-mediated diseases.

In the phase 2 study, IONIS-FB-LRx demonstrated a favorable safety and tolerability profile. The study data are consistent with the clinical profile seen across Ionis’ other LICA programs, further validating how advancements in the company’s LIgand-Conjugated Antisense technology platform position Ionis to deliver potentially transformative treatments for a range of unmet medical needs. Data from the Phase 2 study of IONIS-FB-LRx in patients with IgAN has been submitted for presentation at an upcoming medical meeting.

“Roche’s decision to advance the program reaffirms our shared confidence in the ability of Ionis’ antisense medicines to effectively target the root cause of difficult to treat diseases like immunoglobulin A nephropathy,” said Michael McCaleb, vice president, clinical development at Ionis. “The results of the phase 2 study provide initial clinical evidence that IONIS-FB-LRx reduces complement and protein levels in the urine of patients with IgAN.”

Author: Rare Daily Staff

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