Spanish Ferrer SA pays €122.5m for ALS candidate

Ferrer inked a €112.5m deal with the US-headquartered AI drug discovery specialist Verge Genomics to commercialise the PIKfyve (1 phosphatidylinositol 3 phosphate 5 kinase) blocker VRG50635 in Central and South America, Southeast Asia and Japan that includes upfront and milestone payments plus royalities. Verge’s clinical-stage amyotrophic lateral sclerosis (ALS) drug VRG50635 was developed using the  AI platform Converge. VRG50635, which is currently being tested in a Phase Ib trial, is believed to improve the survival of neurons in patients affected by ALS.

In January, Ferrer’s Phase III oral ALS formulation of edaravone, a compound from Mitsubishi Tanabe Pharma which got approval in another indication with another formulation, failed in a phase III trial. Currently, in Europe there is a single approved treatment option in the form of riluzole, marketed by Sanofi as Rilutek, and as Teglutik that is marketed by Italfarmaco SA (France, Germany, Italy, Spain) and by Martindale Pharmaceuticals (UK). According to data from the ADORE trail, edaravone (100mg, once daily) did not demonstrate a significant benefit over placebo in slowing ALS progression, measured by change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) score after 48 weeks of daily oral edaravone dosing.

Verge Genomics’ platform intergrates human omics data, in vitro testing data  with clinical data from ALS patients and controls to screen the best drug candidates.