Eli Lilly has axed another trial of a gene therapy acquired in its $1 billion Prevail Therapeutics buyout, further denting the drugmaker’s chances of delivering a return on its investment.
Prevail had two lead gene therapies when it accepted Lilly’s buyout bid in late 2020. Lilly dropped one of the gene therapies, the frontotemporal dementia prospect LY3884963, from its pipeline in February. The action made Prevail’s other lead program, the GBA1 gene therapy LY3884961, Lilly’s best shot at bringing one of the acquired assets to market.
Lilly narrowed LY3884961’s path to approval on Wednesday as part of its second-quarter results. The Big Pharma has removed (PDF) a phase 1/2 study testing the gene therapy in Gaucher disease type 1 (GD1) from its pipeline.
“The GBA1 gene therapy program for Gaucher disease type 1 did not meet our high internal bar for success and is being removed from the pipeline,” a Lilly spokesperson told Fierce. “There was no safety signal observed and Lilly is continuing the GBA1 gene therapy program in Parkinson’s disease with the Propel trial.”
The GD1 trial was enrolling an estimated 15 patients to evaluate the safety and tolerability of up to three dose levels of the gene therapy in adults with peripheral manifestations of the inherited lysosomal storage disorder. GBA1 mutations that drive deficient enzyme activity cause GD1, leading Lilly and other drug developers to study the potential for gene therapies to fix the problem at its source.
LY3884961 has suffered setbacks since Lilly acquired the asset. In the third quarter of 2022, Lilly reported a $206.5 million charge primarily related to changes in the estimated launch timing for the gene therapy. In the first quarter of 2024, Lilly ended (PDF) development of the asset in Gaucher disease type 2 (GD2).
Ending work in GD1 and GD2 leaves Lilly with a small gene therapy pipeline, which forms part of a broader set of genetic medicines that the company is developing. As well as the Parkinson’s program, Lilly is running phase 1/2 trials of gene therapies for otoferlin gene-mediated hearing loss and for vestibular schwannoma, a tumor that grows on the balance and hearing nerve. Lilly acquired both hearing-related gene therapies in its 2022 takeover of Akouos for $487 million upfront.
Lilly’s termination of the GD1 program removes a major player from the Gaucher gene therapy race. Spur Therapeutics, a biotech created through the merger of Freeline Therapeutics and SwanBio, is running a phase 3 trial of a GBA1 gene therapy in GD1. Lingyi Biotech is enrolling patients in a phase 1/2 trial of its asset, while Sanofi researchers published a paper on their own lead candidate, SS3-GBA1, late last year.