The start of a potentially big biomedical breakthrough is blooming within all of us. Drug research aiming to turn bacteria into microbiome therapies now spans programs aiming to address gut disorders, autism, cancer, and more.
Given the number of late-stage and mid-stage studies expected to report data in 2020—assuming they stay on track amid the COVID-19 pandemic—this could be a pivotal year for the field, said Isabelle de Cremoux, CEO and managing partner of Seventure Partners. Positive results could help win over large pharmaceutical companies and investors who have doubted microbiome therapies.
“In every new (drug) class there’s always been people that were very skeptic, and thought that it would just be something very short,” de Cremoux said. “When they see good data, then they change their minds. I’m sure that will happen in the microbiome field as well.”
Clinical trials are expected to produce good safety data because many of these experimental therapies are produced from bacteria already found in the human body, de Cremoux said. She added that there is already real-world evidence for some therapies as hospitals around the world use them to treat treating patients on a compassionate-use basis. Those results give her good reason to believe that large clinical trials will ultimately lead to positive results.
De Cremoux’s comments came during a panel at the recent Bio-Europe Spring 2020 conference, which was held digitally this year due to the coronavirus pandemic. She was joined by Hervé Affagard, CEO of Lyon, France-based MaaT Pharma, and Thomas Mitchell, vice president of program and alliance management for Cambridge, UK-based Microbiotica.
In many cases, microbiome therapies will complement other treatments for patients, said Affagard. His company is developing products that restore the microbiome in patients who have received stem cell transplants, antibiotics, or chemotherapy. MaaT013, an experimental enema-based treatment, is being evaluated in two separate mid-stage tests, one for patients with acute graft-versus-host disease and the other in patients with acute myeloid leukemia or myelodysplastic syndromes.
“You are tackling the disease in a totally different way, using the bacteria,” Affagard said. “That should not replace some existing drugs, unless they are not working, of course.”
Microbiome drug development poses challenges, some of them not unlike those faced by other classes of therapies when they first emerged. Among the obstacles is manufacturing. Affagard recalled MaaT’s initial conversations with contract manufacturers four years ago were “like a dead end for us. We had no choice but to do our own facility, our own GMP facility. Today, the situation is very different.”
Mitchell noted that many microbiome companies do their own manufacturing, pointing to two Cambridge, MA-based companies, Seres Therapeutics (NASDAQ: MCRB) and Vedanta Biosciences, as examples. Microbiotica’s research includes programs for inflammatory bowel disease (IBD) and Clostridium difficile infection. Mitchell says his company takes its investigational treatments as far as possible internally, then turns the work over to a contract manufacturer. The vendor choices are improving as the field matures, he added.
The maturing field is also seeing a boost in dealmaking. Vedanta signed one of the earliest microbiome partnerships with a large pharmaceutical company, inking a 2015 alliance with Johnson & Johnson (NYSE: JNJ) focused on Crohn’s disease and ulcerative colitis. In 2017, Finch Therapeutics began a partnership with Takeda Pharmaceutical (NYSE: TAK) aiming to develop IBD treatments. Microbiotica struck up its own IBD partnership with Roche’s Genentech the following year. Mitchell described that deal as validation for Microbiotica, as well as the microbiome therapies field. Big pharma companies “are realizing it quite early on, the power of the microbiome, not only to support their existing products, but also to look at it as a completely new modality,” he said.
To date, much of the microbiome research has focused on bacteria. But de Cremoux noted that the microbiome also includes yeast, viruses, and fungi. Those microorganisms also represent opportunities for new research and new alliances. The partnering landscape has moved in step with the progress made in microbiome research, she said. Six or seven years ago, the one or two microbiome deals made each year netted a couple of million dollars up front and modest royalty and milestone terms. That’s changed in the past two years. Microbiome companies are now landing $50 million or even $100 million up front, with milestones potentially bringing as much as $800 million more. De Cremoux predicts the sector’s first billion-dollar deal will close this year, or perhaps in 2021.