A Cambridge biotech startup based on work from a trio of Harvard researchers believes it has found a new approach to treating amyotrophic lateral sclerosis, or ALS, a debilitating neurodegenerative disease that robs people of their ability to move and is often fatal within a few years.
QurAlis said Thursday that it has raised $88 million from more than a dozen private investors, including EQT Life Sciences, which has taken a special interest in neurodegenerative disorders and dementias, as well as from the venture capital arms of pharmaceutical giants Amgen and Sanofi.
The company will use the money to test two drugs for ALS in early-stage clinical trials. If the results look promising, QurAlis plans to test the same compounds, or closely related ones, as treatments for frontotemporal dementia. Action-movie star Bruce Willis was recently diagnosed with that form of dementia, which sometimes shares genetic similarities to ALS.
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The new funding for QurAlis comes just over five months after another Cambridge firm, Amylyx Pharmaceuticals, won approval from the Food and Drug Administration for its own ALS drug, Relyvrio, which costs $158,000 a year. The FDA approved that therapy even though the company had only tested it in an intermediate-phase trial and scientists raised many questions about how well it worked.
Kasper Roet, cofounder and chief executive of QurAlis, said the approval of Relyvrio helped get investors excited in his company. “There’s definitely positive energy that came from that,” he said. More specifically, the FDA’s willingness to approve ALS drugs based on intermediate-phase studies suggests that these drugs could reach patients more quickly. “We consider that a very good sign,” he said.
The 45-person startup recently brought in $8 million more than Roet originally sought from investors, he said. “And there was a lot more interest around the table,” he added. The company has raised $143.5 million since it was founded in 2016.
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QurAlis is based on work from the former Harvard Stem Cell Institute researcher Kevin Eggan, who figured out how to transform skin cells from ALS patients into motor neurons, so that scientists could study the disease in a lab dish. Some scientists questioned whether the nerves-in-a-dish approach would accurately represent the disease.
“People didn’t think anything useful would come from it,” Roet said, who previously worked in Eggan’s lab. Eggan, Roet, and Harvard Stem Cell Institute scientist Clifford Woolf hoped to prove that assumption wrong. The group used their lab-grown nerve cells to pinpoint new genetic and molecular clues about what goes awry in ALS, and founded Quralis to develop drugs based on those findings.
QurAlis began testing one of its drugs late last year. That compound is designed to open up a particular subset of potassium channels that slow electrical conduction in nerves, which the startup’s founders discovered many ALS patients were lacking. “Without it, a cell becomes hyperactive, and that leads to neurodegeneration,” Roet said. “So we are putting the brakes back on the cell.”
QurAlis also recently began enrolling a clinical trial of its second drug designed to restore levels of a protein called stathmin-2, which is depleted in some patients with ALS. The protein plays an important role in repairing damaged motor neurons and protecting them from neurodegeneration, Roet said, and he hopes the drug will help keep the connection between nerve cells and muscles healthy. That drug is an RNA-based molecule that’s similar to Biogen’s therapy Spinraza, a treatment for spinal muscular atrophy.
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Ryan Cross can be reached at ryan.cross@globe.com. Follow him on Twitter @RLCscienceboss.