Lilly quietly drops three pipeline programs, including a gene therapy

Eli Lilly has discontinued three clinical-stage drugs, according to disclosures made alongside its earnings report ^[1]. The cuts come as the company awaits a Food and Drug Administration decision on orforglipron, its oral GLP-1 pill for obesity, and as it directs more resources toward cardiometabolic and immunology research ^[1].

The most notable drop is LY3884963, a gene therapy for a rare, inherited form of frontotemporal dementia linked to changes in the GRN gene, known as FTD-GRN ^[1]. The therapy came from Lilly's 2020 acquisition of Prevail Therapeutics, a deal worth more than $1 billion ^[1]. It had reached a phase 1/2 trial that began in 2020 and was expected to report main results in 2031 ^[1]. A Lilly spokesperson said the therapy was stopped because of "a lack of compelling efficacy in the studied patient population," not because of any safety concern ^[1]. Patients already enrolled will continue to be monitored during a safety follow-up period, and Lilly says it plans to share data from the program at a future scientific meeting ^[1].

Lilly is also ending LY3541860, an antibody that targets CD19 and was being tested in mid-stage trials for multiple sclerosis and rheumatoid arthritis ^[1]. The rheumatoid arthritis study, a phase 2a trial enrolling 35 patients, had been expected to report results last November ^[1]. The multiple sclerosis trial, aiming to enroll 200 patients over four years, was still listed as recruiting and had a 2027 readout planned ^[1]. Lilly said the drug "did not meet Lilly's high bar for continued development" and that the decision was not driven by safety ^[1].

The third drug being dropped is AC-225-PSMA-62, an experimental radioligand therapy for a specific type of prostate cancer, which was in a phase 1/2 trial expected to report in September 2027 ^[1]. Lilly said it did not meet the company's "bar for differentiated efficacy to advance to phase 2," and that the termination was unrelated to any new safety findings ^[1].

Lilly said the cuts are not tied to broader layoffs. A spokesperson said there are "no large-scale workforce reductions underway," describing the moves instead as routine adjustments to speed up and focus the company's drug development ^[1]. Even as it trims some programs, Lilly's overall research spending is rising: R&D costs increased 26% to $3.8 billion in the most recent quarter, about 20% of revenue ^[1]. The company still has other gene therapy work underway, including a mid-stage program from Prevail targeting the GBA1 gene for Parkinson's disease and Gaucher disease, and it recently signed a deal worth more than $1 billion for a genetic hearing-loss treatment ^[1].

Why it matters for patients

These cuts do not affect any approved GLP-1 medicine. Ozempic, Wegovy, Rybelsus, Mounjaro, and Zepbound are unaffected, and orforglipron remains under FDA review ^[1]. But the disclosure shows where Lilly is putting its money: obesity and metabolic disease, alongside immunology, rather than rare neurological disease, certain autoimmune targets, or this particular prostate cancer approach ^[1]. For patients enrolled in the discontinued trials, especially the dementia gene therapy study, this means no further dosing toward a new treatment answer, though those already in the study will still be followed for safety ^[1]. For people with FTD-GRN, a disease with few treatment options, it is not yet known from these sources what other therapies, if any, are in active development to replace this one. Lilly says it wants to apply what it learned from the discontinued programs to future research, but no specifics on how that would translate into new patient options have been disclosed ^[1].

What happens next

Lilly said it plans to present data from the discontinued dementia gene therapy program at a future scientific meeting, though no date has been set ^[1]. The company's other gene therapy programs, including the GBA1 candidate for Parkinson's disease and Gaucher disease, continue as planned ^[1]. Lilly's decision on orforglipron from the FDA is still pending, and the company holds a priority review voucher that could speed that review ^[1].