Skip to main content
Premium Trial:

Request an Annual Quote

Eli Lilly Exclusively Licenses ASO Candidate for ALS From QurAlis

NEW YORK – QurAlis on Monday said it has entered into an exclusive license agreement granting Eli Lilly global rights to develop and commercialize its antisense oligonucleotide (ASO) candidate QRL-204.

Under the terms of the agreement, Lilly will pay Cambridge, Massachusetts-based QurAlis $45 million upfront and make an additional equity investment. QurAlis will also be eligible for milestone payments of up to $577 million and tiered royalties on net sales should QRL-204 reach the market.

QRL-204 is a splice-switching ASO designed to correct mis-splicing and restore production of UNC13A proteins, which play a role in regulating neurotransmitter release at synapses. It's being developed as a potential treatment for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases.

Mis-splicing related to UNC13A occurs in up to 63 percent of ALS patients and in up to one-third of FTD patients, according to QurAlis. UNC13A is one of several pre-mRNAs that can become mis-spliced due to certain UNC13A mutations or a loss of nuclear TAR DNA binding protein-43 (TDP-43), both of which are associated with many ALS and FTD cases.

"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, senior VP of neurodegeneration research and director of the Lilly Institute for Genetic Medicine, said in a statement.

QurAlis and Indianapolis-based Lilly will collaborate to advance QRL-204 and identify other UNC13A-targeting compounds using QurAlis' proprietary FlexASO splice modulator platform, which generates splice-switching ASOs.

Preclinical data suggest that QurAlis' splice-switching ASOs modulate UNC13A splicing, increase UNC13A protein levels, and restore normal synaptic activities in ALS and FTD.