Glycomine Secures $115M for Phase IIb Rare Disease Therapy

Glycomine has successfully secured $115 million in Series C funding to further the development of its primary investigational therapy, GLM101, by advancing it into a Phase IIb clinical trial for a rare genetic disorder. This disorder, known as phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), is a life-threatening and ultra-rare condition that currently has no approved treatments available. The latest funding aims to move GLM101, a mannose-1-phosphate replacement therapy, closer to becoming the first disease-modifying treatment for PMM2-CDG.

The disorder is caused by mutations in the PMM2 gene, leading to a deficiency in the synthesis of N-linked glycans. These glycans are crucial for the normal functioning of proteins and lipids in the body. As a result, individuals affected by PMM2-CDG experience a variety of severe symptoms, including ataxia, developmental delays, vision loss, and complications involving multiple organs. Currently, the only treatment options are supportive care measures such as physiotherapy, assistive devices, and interventions that target specific symptoms.

Glycomine is actively conducting an open-label Phase II clinical trial (NCT06657859) across various sites in Europe and the United States, with 20 patients enrolled so far. The trial recently expanded to include dosing in pediatric patients. Interim results from nine adult and adolescent participants revealed an average improvement of 11.9 points over 24 weeks on the international cooperative ataxia rating scale. These findings suggest a potential clinical benefit in addressing one of the disorder's most severe symptoms.

The new investment follows a previous funding round in June 2021, where Glycomine raised $35 million to support its Phase I program for GLM101. CEO Steve Axon expressed optimism about the progress, stating that the financing will allow Glycomine to initiate a randomised, placebo-controlled trial later this year. This trial represents a crucial step towards providing a groundbreaking treatment for patients with PMM2-CDG.

The funding round was co-led by CTI Life Sciences Fund and Advent Life Sciences, with significant contributions from Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures.

Concurrently, another initiative is exploring a repurposed therapy for PMM2-CDG. Maggie’s Pearl, a collaboration between Perlara, Maggie’s Cure, and the Mayo Clinic, is sponsoring a Phase III trial for the oral drug epalrestat (NCT04925960) in 40 pediatric patients. The US Food and Drug Administration (FDA) approved this Phase III study in December 2021, and the trial is now fully enrolled, with completion anticipated by the end of the year. Epalrestat, an aldose reductase inhibitor, is already approved in Japan for treating diabetic neuropathy.

This advancement in treatment options for PMM2-CDG comes amid growing concerns about the future of incentives for rare disease drug development in the United States. In particular, there are worries regarding the potential termination of the FDA’s pediatric priority review voucher (PRV) program, which could have significant implications for the sector.