Home Health Care PAQ Therapeutics scores $30M to unleash “Pac-Man” drugs on neuro disease

PAQ Therapeutics scores $30M to unleash “Pac-Man” drugs on neuro disease

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Few biotech executives explain their experimental therapies by comparing them to video games. Nan Ji, president and CEO of PAQ Therapeutics, does so enthusiastically, and by using a 40-year-old game at that. In the 1980s, Pac-Man was an arcade fixture and kids with pockets full of quarters lined up for a shot at navigating the namesake character through a maze, gobbling up pellets for points.

In a similar vein, PAQ Therapeutics is developing drugs that get a component of a cell to chomp on targets associated with disease. A growing number of biotechs are developing drugs that harness one of the cells’ disposal components to consume problem proteins. But Ji notes that proteins aren’t the only parts of a cell that can cause disease. Just as Pac-Man scores more by eating fruit, bells, and ghosts, PAQ’s drugs could address more diseases via consumption of a range of targets including lipids, mitochondria, and organelles.

“This is a very novel approach based on new biology,” Ji said. “We’re still early but we believe there is great potential.”

The Cambridge, Massachusetts-based startup is making steady progress with its research, enough to level up with $30 million in financing. The Series A funding round announced Thursday was led by Sherpa Healthcare Partners.

Using a cell’s built-in components to treat disease is catching on. A growing number of biotechs are jumping into targeted protein degradation, in which disease-causing proteins are directed to the part of a cell that disposes of old or damaged proteins. Ji knows the space well, having worked for three years as head of chemistry at Kymera Therapeutics, one of the companies that has reached clinical testing with this new approach to treating disease.

In 2019, while Ji was still at Kymera, he read a paper published in Nature that described using autophagy, a process of disposing of other cellular components, as a way of treating disease. That research did focus on a protein, specifically, the mutant huntingtin protein that cause the neurodegenerative disorder Huntington’s disease. But Ji said he was particularly interested in autophagy’s potential to address non-protein targets.

“Right away I realized this could offer an opportunity that is not there for targeted protein degradation,” he said.

Ji said he reached out to Boxun Lu, a professor at Fudan University in Shanghai and the last author on the Nature paper. Together, they started PAQ to develop new drugs that leverage autophagy. In the body, autophagy is carried out by the lysosome, a different cellular disposal system than the one that carries out protein degradation. Waste gets to the lysosome via the autophagosome, a sphere-shaped component of a cell. Ji said PAQ is developing small molecules that speed up the interaction between the autophagosome and the disease-associated parts of a cell. The molecules PAQ is developing are called autophagosome-tethering compounds, or ATTECs.

PAQ’s lead disease target is a neurodegenerative disorder that the company is not disclosing, other than to say that it’s a genetic disease that does not currently have any therapeutic options. Ji said that the company’s small molecule can cross the blood brain barrier to reach its target. So far, the company has done pharmacokinetic and pharmacodynamic studies in animals, tests that show how a drug is taken up by the body and the body’s biological response to the therapy. Ji said he is particularly interested in the latter, because in this undisclosed disease target, pharmacodynamics have a direct correlation with efficacy, and could be a harbinger of what to expect in humans.

PAQ isn’t the first biotech startup looking to autophagy as a way to address neurodegeneration. Libra Therapeutics is developing small molecules that increase autophagy. The San Diego-based biotech launched last year with $29 million in Series A cash and amyotrophic lateral sclerosis as its lead disease target. QurAlis, a Cambridge startup, raised $42 million in Series A financing last year for its research on several different approaches to ALS, including one in autophagy.

The other investors in PAQ’s new financing are Huagai Capital, MSA Capital, and MRL Ventures Fund. They are joined by seed investors, Nest.Bio Ventures and Matrix Partners China. With its fresh cash haul, Ji said that PAQ can continue to advance its lead program further in preclinical development. But he added that the technology is a platform with the potential to support drugs addressing targets beyond the central nervous system, and the new cash will also support the development of one or two compounds for other diseases. PAQ currently employs six and Ji said that the company will make additional hires as it expands its work.

Pac-Man is more than a convenient way to explain how PAQ’s drug’s work. The game was on the minds of the founders from the beginning. Scientific illustrations of the autophagosome resemble the letter C shape of the video game character, Ji explained. In fact, in the company’s earliest days, the scientists called their research “Project Pac-Man.”

The “Q” in the name references polyQ disorders, which are characterized by a portion of a protein that has repeated sequences. Huntington’s disease is a polyQ disease, and the letter Q in PAQ’s name is a nod to the research from scientific founder Lu that first caught Ji’s eye. When it came time to formally name the startup, Ji said the Pac-Man reference made sense.

“It was short and catchy,” he said. “It represents the history of company.”

Photo by Flickr user Gamescore Blog via a Creative Commons license

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