Metagenomi abandons ALS program, seeks new kidney disease partner

Metagenomi, a biotech company, has decided to halt its amyotrophic lateral sclerosis (ALS) program after analyzing recent clinical data from peer companies. The decision was influenced by the failure of Biogen and Ionis Pharmaceuticals' ATXN2-targeting ALS candidate. This candidate, called BIIB105, did not show efficacy in reducing neurodegeneration biomarkers or improving functional measures during the Phase I/II ALSpire study, leading to its discontinuation in May.

Metagenomi announced this strategic shift in its second-quarter earnings report. The company stated the need to streamline its early-stage programs to better identify near-term pipeline priorities. Consequently, Metagenomi will now focus its internal development efforts on in vivo gene editing therapeutics and liver indications, while planning to out-license its ex vivo cell therapy technologies. Additionally, the company aims to find a partner or licensee for its newly regained primary hyperoxaluria type 1 (PH1) program.

The PH1 program came back under Metagenomi's control after the termination of a nearly three-year collaboration with Moderna in May. Metagenomi possesses preclinical proof-of-concept data for this candidate and is actively seeking a development partner to advance the asset. This move will allow the company to concentrate on its haemophilia A program. Metagenomi disclosed that it has named MGX-001 as its lead development candidate, anticipating a filing in 2026 to begin in-human testing.

Financially, Metagenomi has a runway into 2027, with approximately $300 million in cash and marketable securities. However, the company has faced significant challenges since its public debut in February. As the first preclinical biotech to go public this year, Metagenomi priced its offering at $15 per share, the lower end of its proposed range. Since then, the share price has dropped to around $3, resulting in a nearly 80% decrease in the company's market value.

In summary, Metagenomi is realigning its focus by discontinuing its ALS program and prioritizing gene editing and liver disease therapeutics. The company is also seeking strategic partnerships for its ex vivo cell therapy technologies and the PH1 program. With a lead candidate for haemophilia A set for regulatory filing in 2026, Metagenomi aims to advance its pipeline while managing its financial resources amidst a challenging market environment.