Metagenomi is editing its pipeline, choosing to seek a partner for a program Moderna dropped and axing its amyotrophic lateral sclerosis (ALS) activities in response to setbacks at a competitor.
The biotech regained the rights to a preclinical primary hyperoxaluria type 1 program when its deal with Moderna fell apart earlier this year. At the time, Sarah Noonberg, M.D., Ph.D., chief medical officer at Metagenomi, said the company would assess the rare disease program to determine whether it met the criteria for further investment.
Metagenomi revealed its conclusion Wednesday. The company is deprioritizing the program but hoping a partner or licensee can be found for further development.
The biotech disclosed the action alongside news that it has opted against advancing its ALS program. The action was “based upon recent peer company clinical data regarding the lack of efficacy of Ataxin-2 as a therapeutic target for ALS,” Metagenomi said. Biogen and Ionis ended development of an ALS candidate designed to reduce expression of Ataxin-2 in May in response to lackluster phase 1/2 data.
Metagenomi framed the changes as part of its attempt to “exercise fiscal responsibility” over its internal programs and look for partnerships that can unlock other development opportunities. Internally, the biotech is focused on in vivo gene editing therapeutic approaches. Management is pursuing technology out-licensing for ex vivo cell therapies.
The changes reinforce Metagenomi’s focus on its hemophilia A program. Having declared a development candidate, the biotech is now trying to accelerate the release of 12-month data in nonhuman primates. Metagenomi previously said to expect data late this year. The readout could now arrive in September, giving Metagenomi an ahead-of-schedule look at the durability of its one-time treatment of the condition.
The biotech is on track to file to test the candidate in humans in 2026. Apart from that commitment, Metagenomi’s plans for 2026 have become a little hazier as the result of a rethink of its forecasting. The biotech is now only providing pipeline guidance through the end of 2025, by which point it aims to nominate one or two cardiometabolic candidates and continue to advance other programs toward IND filings.