Celsius Therapeutics inks first biopharma deal—an IBD study with Janssen

Celsius is working with single-cell sequencing because tissues are heterogeneous—they contain many different kinds of cells. (Celsius Therapeutics)

Celsius Therapeutics launched in May last year with $65 million and a goal to create precision drugs for cancer and autoimmune disease. But first, it has to amass the data it needs to figure out what, exactly, drives disease in different patients. Celsius’ new partnership with Janssen Biotech will help it get there—and help Janssen work on a treatment for inflammatory bowel disease (IBD).

Under the deal, Celsius will use its single-cell genomics and machine learning technology to identify biomarkers that predict patient response in a phase 2a study testing Janssen’s plaque psoriasis med Tremfya and its immunosuppressive drug Simponi in patients with ulcerative colitis. Cambridge, Massachusetts-based Celsius will pick up undisclosed payments, as well as milestones, based on the use of biomarkers it pinpoints.

It’s a win-win situation for both, Celsius CEO Tariq Kassum, M.D., told FierceBiotech. Janssen can feed the “cellular insights” from the collaboration into its clinical work, and Celsius will use them to “seed” its platform, adding the data to its own database and “interrogating” the information in its own drug discovery efforts.

Celsius is working with single-cell sequencing because tissues are heterogeneous—they contain many different kinds of cells. Like fruits in a smoothie, some types of cells may overpower others in a mixture. Looking cell by cell allows Celsius to look at individual “fruit” to discover which genes determine what cells do and how they interact with each other, along with what goes wrong to cause disease, Celsius co-founder and Chief Scientific Officer Christoph Lengauer told FierceBiotech in a previous interview.

RELATED: Celsius bags $65M to develop precision drugs for cancer, autoimmune disease

What’s more, Celsius’ approach can shed light on why some people with “completely normal genetics” get IBD.

“It’s because of some kind of programming being expressed in an aberrant fashion in those patients’ colonic tissues,” Kassum said.

“Celsius has the ability to look at disease on a cell-by-cell level at an extremely high resolution [and] the ability to look at RNA expression. We are actually seeing the behavior of cells, rather than their somatic DNA programming,” Kassum said. “This makes single-cell RNA sequencing a uniquely beneficial tool if you’re trying to understand the driver of a disease in otherwise genetically normal tissue.

Though the Janssen collab is Celsius’ first biopharma industry deal, the company is already working with academic partners to get its hands on patient samples and their associated clinical and biological data. These academic deals can turn up anywhere between 50 and 500 samples, said Celsius Chief Business Officer Michael Boretti. What’s exciting about the Janssen deal though, is the scale.

“We are likely to get hundreds of samples from the collaboration. It’s a much greater sample size to be able to understand different disease etiologies … Looking at ulcerative colitis, maybe there are differences that can help us understand why some patients respond to therapies and why some don’t,” Kassum said.

Celsius is funded by Third Rock Ventures, Alphabet's venture arm GV, Heritage Provider Network, Casdin Capital and Alexandria Venture Investments. Since launch, it has built what Kassum calls an industrialized version of the RNA sequencing platform.

“When the company was launched, single-cell RNA sequencing was the provenance of academic researchers … The throughput is a sample here, a sample there. The way you get it is you have a postdoc in the room who gets a fresh piece of tissue and then sprints to the lab, be it across town or down the hall. That doesn’t scale well,” Kassum said.

“We have changed the processes and protocols and built capabilities such that we can handle hundreds of samples,” he said. Celsius can also deal with frozen tissue, which lets it collect samples the world over.

“We have several partnerships across all areas. Using a campaign approach, we have the ability to identify things by combining data in a campaign that we wouldn’t see in one individual study,” Boretti said. These campaigns include autoimmune disease, oncology and immuno-oncology.

Celsius is in talks with “a number of companies” about collaborations ranging from “little tiny things” to “potentially very big things,” Kassum said. None of them are finalized at the moment, so the company is keeping them under wraps for now.

That’s not to say Celsius won’t be kept busy over the coming year.

“I think the next six to 12 months, we are going to be generating a lot of data from the samples we’ve been getting … It’s going to be a lot of data to process, a lot of trees where we then have to start looking to see if we can see the forest. What are these data telling us?” Kassum said.

At launch, Celsius had about 15 staffers by Lengauer’s count, and it has nearly tripled since then to 40. The company is looking to bolster its leadership, as well as its broader team; in particular, it is on the hunt for computational biologists. If all goes well, the company should be well into the 50-employee range by the end of the year.

Editor's note: This story was updated to correct Christoph Lengauer's title. He is the co-founder, chief scientific officer and former president of Celsius, not the president. The story also originally quoted Daniel Leon, who is, in fact, the founder of a different company called Celsius Network.