Glycomine grabs $115M series C to fund phase 2 journey for rare genetic disorder drug

Glycomine has bagged a $115 million series C round to continue the progress of its lead candidate through phase 2 development for a rare genetic disorder.

The asset in question, dubbed GLM101, is a mannose-1-phosphate replacement therapy. Glycomine is touting the drug as the first disease-modifying therapeutic in development for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), a rare, life-threatening condition caused by a genetic mutation that results in the PMM2 protein having reduced activity.

Glycomine has already taken GLM101 into a phase 2 study that has enrolled more than 20 patients in Europe and the U.S. So far, data from nine adult and adolescent patients have shown an 11.9-point improvement in a score of ataxia—a lack of muscle control that is a hallmark of PMM2-CDG—over 24 weeks.

The San Carlos, California-based biotech is now looking to use its series C cash to fund a phase 2b study to further demonstrate GLM101’s efficacy. Today’s fundraise was led by CTI Life Sciences Fund as well as funds managed by abrdn and Advent Life Sciences alongside previous backers Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures and Asahi Kasei Ventures.

As part of the financing, CTI Ventures Managing Partner Youssef Bennani, Ph.D., and Advent Life Sciences General Partner Dominic Schmidt, Ph.D., are joining Glycomine’s board.

“We are excited to partner with our new investors who have strong track records in rare diseases and for the continued support from our existing investors,” Glycomine’s CEO Steve Axon said in the April 16 release. “This financing will enable us to advance GLM101 into a randomized, placebo-controlled trial later this year—an important step toward bringing the first disease-modifying therapeutic to patients with PMM2-CDG.”

Glycomine's latest fundraise follows a $12 million series A in 2016 and a $68 million series B spread across 2019 and 2021.

Other players in the PMM2-CDG space are assessing whether aldose reductase inhibitors can treat the disorder—namely Applied Therapeutics’ govorestat and Perlara’s attempt to repurpose the diabetic neuropathy drug epalrestat.