A small California biotech hopes to get its rare disease drug candidate to the FDA’s doorstep next year with the help of a $115 million Series C.
The funding for Glycomine, disclosed Wednesday morning, comes four years after a
Series B extension
. The new money will take the startup into a Phase 2b trial and help it collect primary data from the test around the middle of next year, CEO Steven Axon told
Endpoints News
.
Glycomine is developing a mannose-1-phosphate replacement therapy called GLM101 for patients with phosphomannomutase-2 congenital disorder of glycosylation. It estimates there are about 10,000 to 15,000 people in the US and Europe with the genetic disorder, also called PMM2-CDG, for which there are no approved therapies.
Despite many investors and large pharma companies gravitating toward drug development opportunities in broader patient populations, like immunology and obesity, Glycomine was able to drum up interest in its Series C over the course of about six months, Axon said.
“Rare diseases are probably not the hottest area of investment,” Axon said. “But when you have a later-stage drug, when you have relatively compelling clinical data, a pretty simple story, this is a 15-person company, people that looked at the program felt that this really could be a drug.”
Investors in the decade-old startup include CTI Life Sciences Fund, Aberdeen, Advent Life Sciences, Novo Holdings, Sanofi Ventures, Abingworth and others.
PMM2-CDG is one of several hundred disorders of glycosylation, which involves the processing of sugar on protein surfaces. PMM2-CDG represents about 60% of all genetic glycosylation disorder patients, Axon said.
The disease can impact any one of roughly 10,000 glycosylated proteins, Axon said. To make its mannose-1-phosphate replacement therapy effective, Glycomine encapsulates the phosphate in a lipid nanoparticle that extends its half-life from about five minutes to 80 hours, the CEO said. That LNP delivery also helps get the substrate directly into the cell, he said, noting mannose-1 “a charged molecule and has no transporter access into cells.”
Glycomine is investigating GLM101 in adults and children as young as 2 years old in a Phase 2a study. It expects to start the
Phase 2b trial
in the middle of this year, and plans to include about 40 to 50 people in the placebo-controlled trial, Axon said.
The trial will test whether a high dose of GLM101, at 30 mg/kg, is better than placebo on an evaluation known as the International Cooperative Ataxia Rating Scale (ICARS) at six months. After the initial six months, patients on placebo will switch over to Glycomine’s drug and continue to be followed.
“It could be a pivotal study but it will require regulatory discussions to determine if there’s any additional data that’s required before we file,” Axon said.
Glycomine is using about 10 to 14 sites in the US and Europe for the trial. Axon said those sites helped Glycomine do a 140-patient natural history study.
The Series C gets Glycomine “well beyond the Phase 2b readout” but not enough to launch the drug, should it get approval, Axon said. The company could consider pharma partnerships, an IPO or other routes to become a commercial drugmaker.
“We’re cognizant of the financial markets and the ability to go public, which would be a reasonable next step in good markets,” Axon said. “Fingers crossed on the markets turning a little bit in the right direction for us.”
The biotech’s board has pharma dealmaking know-how. Glycomine added Joshua Grass as its chair in the fall of 2024. Grass was CEO of Escient Pharmaceuticals when Incyte bought the biotech startup for
$750 million
last year, and before that was CEO of Modis Therapeutics when Zogenix bought the early-stage drug developer for
$250 million upfront
.