San Francisco-area biotech gets new cash to launch clinical program for congenital sugar processing disease
A rare disease biotech from the suburbs of San Francisco has returned to the venture well for a Series B extension, aiming to get its lead program through its first clinical trials.
Glycomine pulled in a new $35 million haul for the studies, the biotech announced Wednesday morning, and plans to launch a Phase I study for the lead compound by the end of the year. Wednesday’s funds pushed Glycomine’s Series B total to $68 million after an earlier raise back in August 2019.
Abingworth and Sanofi Ventures co-led the fundraise.
The biotech is on a mission to bring treatments for diseases related to glycosylation, or a process in which the body processes sugar on the surfaces of proteins. If this function is out of whack, it can lead to a host of problems throughout vital organ systems, CEO Peter McWilliams told Endpoints News, including the liver and heart.
Glycomine’s lead candidate aims to treat the most prevalent congenital glycosylation disease, whose full scientific name — phosphomannomutase 2-congenital disorder of glycosylation — is often shorthanded to PMM2-CDG. Patients with the disease contain a genetic mutation within the PMM2 protein that limits proper sugar processing activity.
To try to return the function to normal, Glycomine has developed a phosphate replacement therapy dubbed GLM101 to supply the missing sugar component, McWilliams said. Combined with a lipid nanoparticle to survive circulation, the candidate can attack the disease right at the source of the problem.
“We’re really getting to the crux of disease, getting to that sugar component,” McWilliams said. “The body has the glycosylation machinery, it can make glycan, we’re just providing the ingredients to take it up and make properly glycosylated proteins.”
There aren’t any FDA approved treatments for PMM2-CDG, he added, with 20% of children dying before they reach the age of six.
Glycomine is aiming to get the program started with a Phase I healthy volunteer study by the end of 2021, with the hope of dosing patients in the second half of next year. The biotech has laid out a “very classic” clinical development program, McWilliams said, starting with adolescent patients and working its way down to infants.
With the new cash in hand, McWilliams believes Glycomine will be able to reach a broader set of milestones in developing the drug. They’ve spent the last couple years setting up and enrolling a natural history study to try to further understand how some biomarkers might play a role in the disease.
Now, though, McWilliams is ready to take the next step.
In addition to Abingworth and Sanofi Ventures, other new investors included RiverVest Venture Partners and Remiges Ventures. In addition, all previous Series B investors participated: Novo Holdings A/S, Asahi Kasei Pharma Ventures, Mission BioCapital, Sanderling Ventures and Chiesi Ventures.