Eli Lilly is splicing another asset onto its neurodegenerative disease pipeline, paying QurAlis $45 million for rights to a preclinical treatment for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
The deal gives Lilly global rights to QRL-204, a splice-switching antisense oligonucleotide (ASO) designed to restore UNC13A function. Researchers linked a variant in UNC13A to ALS and FTD years ago, but it took longer to find a causal link between the gene and diseases. A 2022 paper showed how UNC13A variants exacerbate reduced function of TDP-43, the RNA-binding protein implicated in many ALS and FTD cases.
QurAlis designed QRL-204 to modulate UNC13A splicing and restore normal synaptic activities in ALS and FTD. Having shared preclinical evidence that it is on to something in March, the biotech has persuaded Lilly to pay $45 million upfront and up to $577 million in milestones to add the ASO to its pipeline.
“Genetic precision medicines like QRL-204 that target specific causal components of disease pathology hold great promise for delivering meaningful advances against a range of neurodegenerative diseases like ALS and FTD,” Andrew Adams, Ph.D., senior vice president, neurodegeneration research at Lilly, said in a statement.
While hustling QRL-204 toward the clinic, Lilly will work with QurAlis to identify and develop additional therapies targeting UNC13A. The partners will use a QurAlis platform designed to create splice-switching ASOs with improved potency and increased therapeutic index.
UNC13A is implicated in roughly two-thirds of ALS cases and one-third of FTD cases, numbers that have caught the attention of a small band of biotechs. AcuraStem has an ASO against UNC13A in development for ALS and FTD. Maze Therapeutics also discovered an UNC13A ASO but has handed the rights to a new biotech that is yet to exit stealth, according to its pipeline.
While Lilly is a new challenger for the UNC13A niche, it is a familiar presence in the broader ALS and FTD spaces. The drugmaker expanded in ALS through its $135 million takeover of Disarm Therapeutics in 2020 and a smaller partnership with Verge Genomics the following year. Lilly added a FTD prospect to its pipeline when it bought Prevail Therapeutics for $880 million and has a stake in Arkuda Therapeutics.