Rebiotix launches late-stage superbug trial for microbiome-based drug

Rebiotix isn’t a traditional drugmaker. Its products are alive.

The 32-person company, based in Roseville, Minn., is developing a microbiome-based therapy to prevent recurrent Clostridium difficile infection. Its Microbiota Restoration Therapy, which is delivered via enema or a lyophilized, non-frozen oral capsule, is designed to supply a patient’s intestinal tract with an array of healthy, human-derived microbes.

Founded in 2011, Rebiotix said this week that the first patient was treated in a Phase III trial of its lead drug, RBX2660. The late-stage trial is designed to assess the therapy’s ability to prevent recurrent C. diff. infection for eight weeks compared to a placebo.

In one Phase II trial, 87% of patients who received RBX2660 had no C. diff. recurrence after eight weeks.

The sometimes-fatal gastrointestinal disease comes along with a variety of severe symptoms, including fever and diarrhea. More than 500,000 people are infected annually with C. diff., according to Rebiotix, and up to 30% of C. diff. patients experience more than one episode and need repeat treatment.

“The key is that there’s an underlying condition generally that’s caused them to be susceptible to C. diff. in the first place,” Rebiotix founder & president Lee Jones told Drug Delivery Business News. “So what we’re doing with this disease is we’re dealing with the whole person.”

The company’s product is a one-time enema, working to restore the microbial environment found in a patient’s gut.

“It is like a flu shot; the patient goes in, gets the product in a 5-minute procedure and then they go home,” Jones said.

In the next year, Jones said Rebioitix hopes to fully enroll its Phase III trial and pursue regulatory approval with a Biologics License Application.

The Microbiota Restoration Therapy is regulated as a drug in the vaccines division, but arriving at that regulatory path wasn’t straightforward, according to Jones.

“The first question we had to answer was how do you characterize your product,” she said. “What’s the dose, what’s the potency and how do you determine that – because nobody had done this before.”

Rebiotix is among a growing number of companies looking to develop therapies aimed at healing the microbiome. But Jones sees her company as paving the path forward for an entirely new class of drugs.

“Every day I see new companies and new programs come out. We are, by far and away, the most clinically successful of any of the microbiome companies today,” Jones said.

“What we’re hoping to do is by leading the way in the industry, we can show others the way. I think there is such opportunity in microbiome space. It’s going to take more than just us, but if people continue to do good research, we should see the therapeutic benefit before too long.”

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