SAN CARLOS, Calif.--(BUSINESS WIRE)--Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced that the company has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for GLM101 for the treatment of PMM2-CDG and has initiated dosing healthy volunteers in a Phase 1 clinical study. GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia. PMM2-CDG is the most prevalent congenital disease of glycosylation but has no FDA-approved treatments.
“The initiation of this clinical study is an important milestone for Glycomine and for PMM2-CDG patients and their families,” said Peter McWilliams, Ph.D., CEO of Glycomine. “We have demonstrated in preclinical studies that GLM101 can restore the glycosylation pathway that is disrupted in PMM2-CDG, and we look forward to advancing our clinical studies to confirm the potential of GLM101 as an important disease-modifying therapy for PMM2-CDG patients.”
“PMM2-CDG is a serious rare disease with no therapeutic options and represents a critical unmet medical need,” said Horacio Plotkin, M.D., FAAP, Chief Medical Officer of Glycomine. “Glycomine has developed a proprietary approach to delivering mannose-1-phosphate intracellularly to overcome the deficiency that leads to PMM2-CDG. The open-label Phase 1 study will evaluate safety and tolerability in healthy volunteers. With successful completion, we look forward to opening enrollment for patients, which is planned for the second half of 2022.”
About GLM101, a Potential Treatment for PMM2-CDG (CDG-Ia)
Glycomine’s GLM101 is a mannose-1-phosphate replacement therapy in development to treat PMM2-CDG. PMM2-CDG is a severe multisystem disorder with symptoms such as coagulopathies, endocrinopathies, hypotonia, stroke-like episodes, as well as immune and nervous system disfunctions, and resulting mortality of 20% in the early years of life. PMM2-CDG is caused by genetic mutations that lead to a deficiency of the enzyme phosphomannomutase 2 (encoded by the PMM2 gene). PMM2 converts mannose-6-phosphate to mannose-1-phosphate, which is an essential sugar molecule in the N-glycosylation pathway and is crucially important for proper glycoprotein structure and function. GLM101 is designed to deliver mannose-1-phosphate directly into cells and thereby bypass the PMM2 enzyme deficiency and address all disease-causing PMM2 mutations to restore pathway function. GLM101 has received Orphan Drug Designation in the U.S. and Europe and Rare Pediatric Disease Designation in the U.S.
About Glycomine, Inc.
Glycomine is developing orphan drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The company's approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments. The company is based in San Carlos, California and supported by leading international life sciences investors. For more info, visit www.glycomine.com.