Roche Receives Rights to Ionis’ Antisense Kidney Disease Drug for $55 Million

After promising Phase 2 clinical data, Roche gave Ionis $55 million for the rights to IONIS-FB-LRx, an investigational antisense medicine designed to treat immunoglobulin A nephropathy (IgAN). Roche will push the development of the drug into a Phase 3 clinical study to evaluate the impact it could have on patients with rare kidney diseases.

The Promising Future of Antisense Medicines

Antisense oligonucleotides (ASOs) are synthetic single-stranded nucleic acids that bind to specific RNA targets, allowing for tremendous therapeutic benefit. Ionis designed IONIS-FB-LRx to reduce the production of complement factor B (FB), a regulating protein excessively produced in patients with IgAN. 

IgAN is not a kidney disease itself, but a cause of chronic kidney disease and renal failure. Typically surfacing in adults in their 20s or 30s, IgAN is rare, affecting about 1.29 out of 100,000 Americans but can lead to serious kidney inflammation, potentially leading to end-stage renal disease. Kidney transplantation is an option, but patients still risk IgAN recurrence because of the underlying pathophysiology associated with IgAN. 

In a Phase 2 study, IONIS-FB-LRx met its primary endpoint of change in 24-hour urinary protein at 29 weeks compared to baseline. The small study consisted of 10 participants but demonstrated convincing enough results for Roche to purchase the licensing rights from Ionis to push the drug’s development further. 

IONIS-FB-LRx is also in a different Phase 2 study, the GOLDEN trial, to evaluate whether it can halt or slow geographic atrophy progression in patients who have experienced age-related macular degradation. Ionis reached an undisclosed milestone in the GOLDEN trial, bolstering the potential of the drug. 

Related Article: Altering the Treatment Landscape of Neurodegenerative Diseases Using Antisense Oligonucleotides (ASOs)

Building on the Back of Strong, Lasting Partnerships

Ionis has long been an industry leader in antisense medicine technology, assisting the development of three FDA-approved drugs, including Biogen’s blockbuster spinal muscular atrophy treatment, Spinraza, which generated $441 million last year. Ionis continues to spearhead ASOs development with nearly 40 candidates in its pipeline, nine of which are in Phase 3 clinical trials. 

Carlsbad-based Ionis’ success is not only predicated on exciting technology but also key partnerships with several big pharma companies. Roche and Ionis initially partnered up in 2013, when Ionis went by the name Isis Pharmaceuticals. The two initially worked on a Huntington’s disease treatment before teaming up again to work on IONIS-FB-LRx in 2018 when Roche laid down $75 million upfront in the collaboration. 

Ionis developed its other FDA-approved therapy, Tegsedi, with Sobi and its European Medicines Agency-approved Waylivra alongside PTC Therapeutics. The ASOs-focused company is developing other candidates with other massive companies like Novartis, AstraZeneca, Bayer, and GSK. 

Though Ionis will have less influence on IONIS-FB-LRx as a result of its most recent deal, it set Roche up for success with the preliminary development of the candidate. Hopefully, Roche will find as much success in the upcoming Phase 3 trials as the two companies have seen with the drug so far.

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Author

Reed Slater