A year and a few months since its founding, Mozart Therapeutics has secured $55 million to develop therapies for celiac disease and other immune-related conditions, the company announced Tuesday.
The Seattle-based biotech startup is developing compounds to quell overactive immune responses in celiac disease and other conditions.
“We have an opportunity to really do something different in autoimmune and inflammatory diseases,” said CEO Katie Fanning, an industry veteran who previously was CEO of Seattle biotech Nohla Therapeutics, in an interview with GeekWire.
The funding will help propel Mozart’s lead compound through preclinical studies to human testing, with trials for celiac disease expected to start in 2024.
The company’s program builds on research from the lab of co-founder Mark Davis, a professor at Stanford and head of the university’s Institute for Immunity, Transplantation and Infection.
Davis and his colleagues study cells called regulatory CD8 T cells. Such cells may fend off disease in multiple sclerosis and celiac disease, the researchers showed. The regulatory cells operate by suppressing disease-causing immune cells.
Additional work at Mozart suggests that regulatory CD8 T cells also may quell type 1 diabetes and inflammatory bowel disease, said Fanning.
“What Mozart is looking to do is leverage our understanding of that network that [Davis] described to build a portfolio of disease modifying therapeutics,” said Fanning. Davis co-founded the company in June 2020 with two other Stanford professors, K Christopher Garcia and Calvin Kuo.
The company’s lead compound bumps up the action of regulatory CD8 T cells with the aim of quelling overactive immune responses.
The Series A funding round was led by ARCH Venture Partners along with Sofinnova Partners. Other backers include MRL Ventures Fund from Merck, Leaps by Bayer, and Eli Lilly & Company.
Such drug company investment, “reflects the tremendous interest on the part of pharma for novel targets and novel ways to address the unmet needs that still exist in autoimmune disease,” said Fanning. Altitude Life Science Ventures and Alexandria Venture Investments were additional investors in the round, and the company received seed funding from ARCH.
Related: Sonoma Biotherapeutics raises $265M to advance therapies for autoimmune, inflammatory diseases
Mozart’s leadership team includes CSO Kristine Swiderek, previously senior VP of research at Alpine Immune Sciences and Courtney Crane, VP of discovery and translational science. Crane was previously an associate professor at the University of Washington and an investigator at Seattle Children’s Research Institute.
The 13-person company will build up its research arm and expand with hires in product development and other areas. Mozart is currently located in labs near Seattle’s waterfront with Sonoma Biotherapeutics and other biotech building tenants.
“Our lab space right now works and it supports our long-range plans,” said Fanning. But she’s already on the lookout for future space in Seattle’s crowded market for labs. “We’re going to get to a point of critical mass where we’re going to need more space,” she added.
Fanning was tapped to head the company after Nohla folded in 2019. Nohla’s lead investigational therapeutic, based on umbilical cord cells, did not meet clinical endpoints in phase 2 trials for blood cancers, said Fanning. Another company has since acquired the assets of the Fred Hutch spinout.
Regulatory T cells are a focus of a growing number of biotech companies, including Seattle and South San Francisco-based Sonoma, which raised $265 million this summer, and GentiBio, which is building operations in Boston and Seattle. Others include including Egle Therapeutics, TRex Bio and Avotres. Most companies are developing products based on a different type of cell, regulatory “CD4” T cells. Mozart’s focus on regulatory CD8 T cells stands out in the field, said Fanning.
Mozart may explore several compounds that harness these cells, said Fanning. The company is building “bispecific” compounds that bind to the cells and also inhibit a molecule on their surface, called KIR. KIR acts as a brake on the cells and inhibiting KIR gives the cells a nudge.
“We essentially release the brakes and allow the CD8 T regs to mobilize and be activated,” said Fanning, referring to regulatory CD8 T cells.
The company’s lead compound binds to the cells via one target on their surface, and another compound in development binds to a different target. Both compounds inhibit KIR.
Why the name Mozart? The company is “orchestrating a novel regulatory network to restore immune balance,” said Fanning.