New spinoff launched at The Jackson Laboratory

Courtesy of The Jackson Laboratory Kevin Mills, a researcher at The Jackson Laboratory, is leading a new spinoff from the Bar Harbor lab focused on treatments for cancer and autoimmune diseases.

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By Lori Valigra

The Jackson Laboratory has spun off the second business in its 80-year history, led by cancer researcher Kevin Mills.

Mills told Mainebiz that his new company, Cyteir Therapeutics Inc., is set on developing cancer therapies and autoimmune disease treatments. At the heart of the venture is a specific drug to treat chronic lymphocytic leukemia.

Cyteir Therapeutics Inc. is still a virtual company run out of his space at Jackson Lab, but Mills says he has around $100,000 in capital for the new venture and one Chicago-based partner he is not ready to disclose.

To get the company's first drug into a Phase 1 clinical trial within two years, Mills said he estimates needing $5 million in investments. He estimates needing $7 to $10 million to bring two drug candidates into Phase 1 trials within two-and-a-half years.

"In my lab, we've discovered the drug candidate and the pathway that could be a real 'Achilles heel' for leukemia and autoimmune diseases," says Mills, adding that it might be possible to develop a companion diagnostic for the small molecule drug. "And our therapeutics are targeted, so they may decrease the burden of side effects."

For funding, Mills said he's looking to the Maine Technology Institute, federal Small Business Innovation Research and Small Business Technology Transfer awards, venture capitalists and partnerships with pharmaceutical companies that could help with financing, resources and infrastructure. It was a seed grant from MTI that helped Mills develop a system to screen for potential new drugs that resulted in the new spinoff's first drug candidate.

Mills hopes to have some of the initial funding by the beginning of 2013. By the end of 2013 he hopes to move the company to its own quarters. He also plans to add one senior person experienced in science and industry and two scientists who will report to that person.

Currently, Mills is working with Jackson Lab to complete an intellectual property agreement that will likely bring royalties from Cyteir's potential product sales back to the Bar Harbor lab, which saw its first spinoff form just 10 years ago when Bar Harbor BioTechnology Inc. of Trenton licensed proprietary gene analysis methods from the laboratory.

Privately held, Bar Harbor BioTechnology has attracted nine individual and institutional investors, including New Hampshire-based Borealis Ventures, the company's lead investor.

The company is the first startup for Mills, a career-long academic who joined the laboratory's research staff in 2005 following a five-year post-doctoral fellowship at Children's Hospital and Harvard Medical School, both in Boston. He has a doctorate degree in biology from the Massachusetts Institute of Technology.

The drug candidate from Cyteir, which is named for the cell reference "cyt" and the Norse deity of healing "eir," doesn't actually kill cancer cells directly, Mills explains.

Instead, it acts on the genetic program of the cancer cells, causing them essentially to commit suicide. Those cancer cells originate when a genetic pathway instrumental in the immune system goes awry to overactivate the cells, which then drive the leukemia. The technique from Mills' lab targets only these cells — not others in the body — so they are killed. That targeted technique reduces side effects, he says.

Currently, the 20,000 or so new patients with chronic lymphocytic leukemia in the United States receive various treatments each year, which range from monitoring the condition to chemotherapy. The latter patients might relapse and become resistant to treatment, he says.

"Our treatment should work for patients who were never treated before and likely also for patients who have relapsed after standard chemotherapy," he says.

The drug will not restore damaged tissue, Mills said, but it will reduce or eliminate cancer cells.

Mills admits that he brings only an academic background to the new venture, but he was eager to get a treatment to patients.

"This is a vehicle to take our science to clinical applications," he says. "It allows me to pursue a different intellectual problem as well, an applied-science problem and not just basic science."

Editor's note: This story was corrected from an earlier version. Mills estimated needing $7 to $10 million to move two drugs into Phase 1 clinical trials. A previous version said he would need that amount to move one drug into Phase 2 trials.