Pompe disease is caused by genetic mutations that make the body unable to break down glycogen, a complex sugar molecule. As a result, glycogen builds to toxic levels in cells, with muscle cells particularly affected.
Maze’s potential therapy works by blocking the activity of muscle glycogen synthase — the protein that makes glycogen in muscle cells. By blocking this protein’s activity, the treatment aims to decrease toxic glycogen levels in cells. This type of therapeutic strategy is called substrate reduction.
Maze is “rapidly progressing” toward applying for regulatory permission to start clinical trials of the experimental treatment, the company announced in a press release. If granted, the company expects trials to begin in the first half of 2022.
The new therapies were developed using Maze’s proprietary COMPASS platform. Simply put, this platform aims to leverage human genetic data to provide insights into treatment approaches for rare diseases.
According to Maze, the platform makes it easier for researchers to find a viable therapeutic target, and to design a medication for that target. The platform also provides insights relevant to drug development, such as information on potential tolerability profiles and biological markers of treatment effectiveness that could be used as measures in clinical trials.
“Since our founding, we have been leveraging insights from leading geneticists, combined with the growing availability of paired human genetic and clinical data, the evolution of functional genomic technologies and advances in computational power, to build our COMPASS platform in order to bring unique insights into efficient, genetics-based drug development,” said Jason Coloma, PhD, president and CEO of Maze.
“We are excited by the significant progress we have made with our platform and pipeline, bringing us an important step closer to our goal of delivering the right drug to the right patient at the right time,” Coloma added.
According to Maze, blocking the activity of muscle glycogen synthase has long been considered a potential way of treating Pompe disease. However, the structural complexity of the protein, as well as tolerability concerns with long-term reductions in muscle glycogen levels, limited its attractiveness as a therapeutic target. Insights gleaned from COMPASS have allowed Maze to overcome these issues, the company stated.
“New findings using COMPASS helped fill in fundamental data gaps, turning known but challenging targets into exciting, differentiated approaches to the genetic drivers of disease for our first three programs,” said Sarah Noonberg, MD, PhD, chief medical officer at Maze.
“We believe our COMPASS platform, integrated with our extensive drug discovery capabilities, will allow us to accelerate the pace of therapeutic development, as well as increase the likelihood of producing therapies that provide meaningful clinical benefit for patients,” Noonberg added.
The company’s other two lead candidates aim to treat chronic kidney disease and amyotrophic lateral sclerosis.
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