Biotech investors and drug companies are slowly warming up to a new drug delivery strategy that uses exosomes, lipid vesicles naturally secreted by cells, to get drugs into places they normally can’t go. Oxford, England-based Evox Therapeutics is the latest start-up to raise significant funds—$45.4 million in its series B financing round—to move its experimental exosome therapies closer to the clinic.
Neuroscientist Matthew J. A. Wood co-founded Evox in 2016 based on research at the University of Oxford in which he used exosomes in mice to deliver RNA drugs across the normally impassable blood-brain barrier.
Now Evox is disclosing the first details about its surprisingly diverse drug pipeline. Its most advanced program is for Niemann-Pick disease type C, a rare genetic condition in which a faulty protein causes an accumulation of cholesterol and lipids in the brain, liver, spleen, and other tissues. The buildup is particularly dangerous in the brain, where it leads to disability and early death.
Evox hopes to treat the disease by packaging functional copies of the protein in exosomes, which in animal studies helped shuttle the protein into many organs, including the brain, CEO Antonin de Fougerolles says. The program is just a starting point, he adds. Niemann-Pick disease is one of dozens of lysosomal storage disorders that potentially could be treated with a similar method.
One of the firm’s programs uses exosomes to deliver a protein replacement therapy for another lysosomal storage disorder. Evox is also developing exosome-based therapies to deliver proteins or messenger RNA, which would make proteins once inside cells, for undisclosed metabolic disorders.
“The many potential applications of exosomes have really gotten people very interested,” de Fougerolles says. Evox previously struck a deal with Boehringer Ingelheim to use exosomes to deliver an RNA drug to tumor cells. It also has a partnership with an undisclosed company to package a small-molecule drug in exosomes for delivery into the central nervous system.
“The number of discussions that we are having now with pharmaceutical companies has expanded exponentially over the last 12 months,” de Fougerolles says. He expects the new funding will help Evox move at least one program close to human tests within two years.