Delivering aGal (RPV-001) to the right place via the native receptor

In Fabry disease, patients are at risk of acute or chronic pain, significant damage to the heart, kidney, and blood vessels that can be fatal. The condition is caused by a lack or an impaired function of the enzyme a-galactosidase (aGal), which leads to an abundance of sphingolipids accumulating in cells, more precisely the cell’s storage compartment called lysosomes, and tissues. Fabry disease is a rare disease, but its prevalence is considered to be strongly underestimated and improvements in diagnostics may lead to more patients receiving a correct diagnosis earlier.

Currently available treatments for Fabry disease, a well-established therapeutic segment for enzyme replacement strategies, generate sales well in excess of US-$ 1 billion per year. Being a chronic condition, the unmet medical need increasingly shifts towards improved safety and tolerability. Products with improved pharmacodynamics unlocking more convenient dosing schedules, ways of administration and ultimately achieving better cost effectiveness have significant market potential.

Eleva has generated preclinical and clinical results from a Phase 1b trial for its recombinant version of alpha-galactosidase aGal (RPV-001) indicating a lasting therapeutic effect measured by a reduction in sphingolipid levels over the course of 28 days following a single injection. We were also able to show that cell uptake of aGal (RPV-001) is faster compared to mammalian cell-based products, and is more directed to the relevant organs, namely the kidneys. The glycosylation pattern our moss-based expression process provides likely drives uptake of aGal (RPV-001) via the mannose receptor.

Eleva is currently evaluating strategic alliances to accelerate the development of the aGal (RPV-001) program towards market approval.