Looking to strengthen its neurology pipeline, Eli Lilly has secured an exclusive agreement with Massachusetts-based biotech QurAlis to develop and commercialize QRL-204, a potential first-in-class therapy aimed at treating amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
As part of the deal, Eli Lilly will pay $45 million upfront to QurAlis and make an additional equity investment. QurAlis could also receive up to $577 million in milestone payments and tiered royalties on net sales, depending on the success of QRL-204’s development and commercialization.
QRL-204 is a preclinical splice-switching antisense oligonucleotide designed to correct the mis-splicing of the UNC13A gene. This gene is crucial for neurotransmitter release and is mis-spliced in up to 63% of ALS cases and about one-third of FTD cases.
The partnership will also involve an R&D collaboration to find and develop more candidates targeting UNC13A. This will utilize QurAlis' FlexASO splice modulator platform, which creates splice-switching antisense oligonucleotides to correct RNA splicing errors. These ASOs target specific RNA sequences to restore normal gene function, which is essential for treating diseases like ALS and FTD where the UNC13A gene is often mis-spliced, leading to dysfunctional protein production. The platform focuses on increasing the potency and safety of ASOs to address genetic causes of these neurodegenerative diseases effectively.
ALS is a progressive disease that causes the loss of motor neurons, with TDP-43 protein abnormalities in 90% of cases. FTD, sharing similar features with ALS, also often involves TDP-43 issues as well.