RARE Daily

Glycomine Raises $35 Million to Advance Novel Treatment for Rare Glycosylation Disease

June 23, 2021

Glycomine, a company developing new therapies for orphan diseases, raised $35 million to close a $68 million series B financing.

Photo: Peter McWilliams, CEO of Glycomine

Proceeds from the financing will be used to advance the company’s lead drug candidate, GLM101, through initial clinical trials in patients. GLM101 is a novel substrate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG). The disease is a severe multisystem disorder with symptoms such as hypotonia, liver disease, coagulopathies, stroke-like episodes, as well as immune and nervous system disfunctions, and resulting mortality of 20 percent in the early years of life. There are no FDA-approved treatments specific to PMM2-CDG for the more than 1,000 patients currently diagnosed with the disorder.

Also known as CDG Type Ia, PMM2-CDG is the most prevalent of the more than 100 different congenital disorders of glycosylation (CDG). CDGs result in defective formation of the glycan chains essential for the structure and function of glycosylated proteins that represent as many as 50 percent of all proteins in the body. PMM2-CDG is caused by 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 United States and Europe and Rare Pediatric Disease designation in the United States.

The series B financing includes $35 million of new funds in addition to the $33 million announced in August 2019. New investors Abingworth and Sanofi Ventures led the current capital raise, with participation by RiverVest Venture Partners and Remiges Ventures. In addition, all previous Series B investors–Novo Holdings A/S, Asahi Kasei Pharma Ventures, Mission BioCapital, Sanderling Ventures, and Chiesi Ventures–participated.

“We have demonstrated in preclinical studies that GLM101 can restore the glycosylation pathways that are disrupted in PMM2-CDG,” said Peter McWilliams, CEO of Glycomine.

“This additional funding will enable us to confirm in the clinic the potential of GLM101 as a therapy for all PMM2-CDG patients, regardless of genotype, and we are looking forward to executing on our clinical program with this new infusion of capital.”

In connection with the financing, Bali Muralidhar, managing partner at Abingworth, Jim Trenkle, U.S. head of investments at Sanofi Ventures, and Niall O’Donnell, managing director of RiverVest, have been appointed to Glycomine’s Board of Directors.

Author: Rare Daily Staff

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