Yumanity Therapeutics has reeled in a $45 million Series A round as it uses nontraditional ways to find drugs that might treat neurodegenerative diseases such as Alzheimer’s and Parkinson’s.
The Cambridge, MA-based startup is based upon a drug discovery system created by its cofounder Sue Lindquist, a member and former director of the Whitehead Institute for Biomedical Research, which is affiliated with MIT. Lindquist and her cofounder Tony Coles, the former CEO of Onyx Pharmaceuticals, helped bootstrap Yumanity for some time, as my colleague Ben Fidler reported last year. (Both are pictured above.)
The firm’s work has not yet reached human testing, but it has progressed enough to convince a syndicate, led by Fidelity Management & Research, to invest. Other investors are Redmile Group, the venture arm of Alexandria Real Estate, and Dolby Family Ventures, which has a special focus on Alzheimer’s to honor the life of audio pioneer Ray Dolby, who died of the disease in 2013. (You can read more about the fund in a story I wrote last summer.)
Rounding out the syndicate are the drug firms Sanofi and Biogen, which do not gain any special rights to Yumanity’s work through their investment, according to Coles.
Yumanity is using yeast, modified to carry human genes, and human neurons to conduct research that delves into the serious problems caused by misfolded proteins, Lindquist’s expertise. The idea is to use the yeast and the neurons to find key genes and other so-called “markers” of diseases such as Alzheimer’s and Parkinson’s, then to unearth drugs that show promising activity in those diseases.
The neurons Yumanity uses come indirectly from patients themselves. Using a technique called induced pluripotency—its inventor Shinya Yamanaka won a Nobel Prize in 2012—Yumanity takes skin cells from a patient and in a series of steps turns them into neurons that it can experiment with outside the patient.
Induced pluripotency—a technology in its infancy—isn’t yet consistent enough to make cells that can be given to patients as a therapy, because of the fear of minute genetic changes that could spark cancer. But more researchers are turning to cells made this way to follow the effect of drugs in tests run outside the body. In the case of Yumanity, Coles and Lindquist wanted a better way to develop drugs to tackle neurodegeneration. (Lindquist is not an employee of the company but chairs the scientific advisory board.)
“Animal models are notoriously bad,” said Coles, referring to modified rodents and other animals that researchers use as stand-ins for humans in preclinical tests. “They’re not predictive at all.”
When asked if neurons created via induced pluripotency have a track record of helping make better predictions, Coles deferred to his chief scientific officer Ken Rhodes, a former neurology executive at Biogen. Via an e-mail forwarded by a company spokesman, Rhodes noted a clinical trial for people with amyotrophic lateral sclerosis, or ALS—also known as Lou Gehrig’s disease. The trial, not affiliated with Yumanity, features the epilepsy drug retigabine (Potiga) and was built upon research at the Harvard Stem Cell Institute that used induced pluripotency to create neurons from ALS patients.
“Yumanity is at the forefront of this application of iPSC technology in neurodegenerative disease, recognizing that it will take some time before there is full clinical validation of this approach,” Rhodes said.
About a year ago, Coles said he wanted to raise a big round of cash within a couple months. That didn’t happen. Coles brushed aside questions about fundraising difficulties. “It’s been an easy story to tell,” he said, and gave investors “credit” for understanding a sophisticated scientific story. Coles said the $45 million should take the company to its first human tests, but he declined to say how long it might take to get there. He also declined to predict which neurodegenerative indication Yumanity could address first in clinical trials: “We have a broad number of opportunities, and we’ll follow where the science takes us.”