🔒21st century: An entrepreneur/scientist’s drug-induced dream

Marc F. Pelletier arrived in New Haven for a postdoctoral fellowship at Yale in 2002, knowing he wanted to form a biotech company “that would make the largest impact in medicine.”

Four years later, Pelletier and Walter Boron, a professor in Yale’s Department of Cellular & Molecular Physiology, co-founded Aeromics, to create a drug that would control edema (swelling) and improve patient outcomes by targeting molecular water channels.

They were inspired by Peter Agre, who won a Nobel Prize in chemistry for his discovery of these water channels, called aquaporins, which facilitate the movement of water across cell membranes, and by Norwegian neuroscientist Ole Petter Ottersen, whose research on aquaporin-4, a water channel situated at the blood-brain barrier, demonstrated its ability to reduce blood-brain water uptake.

“Knowing there was a path to solving the problem, it had to be done,” Pelletier, 50, says.

“They were not looking to crash out a new version of an old favorite,” explains Agre, Aeromics’ chief scientific officer emeritus. “They started at ground zero.”

Since completing a Phase I trial in March 2019 of its first drug candidate, AER-271, an intravenously administered aquaporin-4 inhibitor to control brain swelling after a major stroke, the company moved its office to New Haven, and is gearing up for a Phase II trial.

“We are not the sole innovators, but we are the sole developer of an aquaporin-4 inhibitor,” says Paul R. McGuirk, Aeromics’ chief technical officer.

Both Agre and McGuirk say Pelletier’s intellect and infectious enthusiasm piqued their interest in the company.

Growing up in Montreal, Pelletier learned technical problem-solving from his father, a mechanical engineer for a transit company. Pelletier earned a bachelor’s and master’s in biology at the University of Concordia in Montreal.

Pelletier developed transgenetic (genetically engineered) plants and focused on mammalian cells at McGill University, graduating in 2001 with a Ph.D. in biology.

He says his fascination with human biology evolved “during late PhD and early doctorate work.”

“Aeromics has spun out from the brain trust in New Haven, where there are people working on big problems and you can learn from them,” Pelletier says.

During the company’s early years, Pelletier spent hours screening hundreds of thousands of compounds, analyzing lab data, writing grants, figuring out company operations, setting up financial structures and trying to build a team.

A fan of Leo LaPorte’s seminal podcasts on technology, Pelletier contacted LaPorte about starting his own podcast, “Futures in Biotech.”

“I interviewed about 100 scientists, including five Nobel Laureates, to figure out how they did it and what drove them,” he says. One podcast, featuring Harvard genetics professor George Church, provided the springboard for the formation of Pelletier’s second company, Dodo Omnidata (now Iridia), which he co-founded in 2015 with Paul Predki and current CEO James P. Linton, to develop digital storage technology using DNA.

“Mark has done a phenomenal job,” Linton says. “His drive to make a difference sets him apart.”

McGuirk was introduced to Pelletier and Aeromics about a decade ago by the late Gregory Gardiner, then a partner at Elm City Ventures, and former director of Yale’s Office of Cooperative Research.

“They had a really neat idea, though I had a lot of doubts whether it was even possible to do this with a small molecule,” McGuirk says. “I thought I would help them out a little bit, and things fell into place.

McGuirk says Pelletier raised “well over $14 million,” which has been augmented by $4 million in venture capital funding.

Supporters include Connecticut Innovations, whose senior managing director of investments, Peter Longo, is a member of Aeromics’ board.

The company has received around at least a dozen patents.

Designed to test the safety of AER-271 in humans, Aeromics’s Phase I trial included 78 healthy volunteers at the Covance cGMP Phase 1 facility in Dallas.

The main goal of the Phase II trial is “to show is we actually can control edema and use imaging techniques to do that with CAT scans and MRIs,” McGuirk says. “We also want to show the drug is safe, and will measure neurological outcomes.”

In the U.S., 150,000 to 200,000 people each year suffer major strokes causing irreparable damage and death, according to McGuirk. “There are just about as many in Europe and double that in Asia.”

“We think the market is very large, and worth probably several billion in revenue,” he says.

“If this thing works,” quips Pelletier, “I might be out of a job.”