MADRID – A Spanish biopharmaceutical company is pioneering the development of an oral medicine based on faeces. The company develops biopharmaceuticals based on human microbiota and aims to be the first in the world to develop this type of drug.
It doesn’t sound very appealing, a drug based on the faecal microbiota, the bacteria found in human faeces. Still, it is an emerging field of research in full swing. Juan Basterra is CEO of the Basque biopharmaceutical company Mikrobiomik, which focuses on the development of these biopharmaceuticals. Furthermore, he talks about it in NIUS Diario.
Medicines based on intestinal microbiota
Five years ago, his company was founded with the aim of “researching, developing and producing medicines based on the intestinal microbiota”, that is, on a stool. These drugs are marketed through licenses to third parties and talks are already underway with several pharmaceutical companies. One of the studies is very advanced and is in phase III of the study.
According to Basterra, this biopharmaceutical will be groundbreaking because it will be administered orally in capsules. The plan is to complete the tests at the end of this year so that the drug can reach the market in late 2024 or early 2025.
“It will be the first of its kind, in Europe and perhaps in the world,” says Basterra. And it will mean an important qualitative leap. Because the only drug that currently exists based on faecal microbiota, ‘Rebyota’, is administered rectally.
It all started three years ago when Mikrobiomik reached an agreement with Gregorio Marañón Hospital in Madrid. It was already testing a prototype of microbiota capsules. The purpose of this was to avoid invasive routes such as colonoscopy that are currently used.
They tested a dose of four capsules for recurrent Clostridioides difficile infection (CDI), a bacteria that is the leading cause of hospital infections and, like many others, have become resistant to antibiotics.
The Gregorio Marañón was “the first European hospital to try faecal microbiota transplantation,” explains Basterra. And they found that the effectiveness of oral administration was the same as that of invasive routes.
Delivery to clinical trials
Based on this prototype, Mikrobiomik received approval from the Spanish Agency for Medicines and Health Products (AEMPS) to manufacture the drug with the aim of supplying it to clinical trials, both the company’s own and those of third parties. And they got to work, “focusing on a phase III that would demonstrate its safety and efficacy.” Furthermore, a trial is currently underway in 21 hospitals in Spain, with 72 patients. “We will have the results at the end of this year,” Basterra announces.
Antibiotics as a comparator
But there’s more. The comparator in his test is not a placebo, but an antibiotic: fidaxomicin. It’s “the world’s first trial” that puts it that way. Because this antibiotic works best against this bacteria, it is the first choice in the treatment of this infection: the reference treatment. It’s about whether Mikrobiomik’s drug would work better. And based on the results so far, it appears to be.
Basterra is very optimistic. “The criterion for efficacy in these studies is that the patient has had no recurrence after 8 weeks.” And he assures that the results they see are “very good, both in terms of efficacy and safety.”
His goal, he insists, is to “become the first company in the world to market a gut flora drug in oral capsules.” It is administered in a single dose of four capsules.
Against the main cause of hospital infections
Whom would this medicine be for? They are testing it with “elderly patients and with many chronic conditions, that is, very delicate.” People who have an imbalance in their microbiota and who are usually hospitalised and eventually become infected with CDI,” explains Basterra.
This bacteria “is the most common cause of hospital-acquired infectious diarrhea.” One of those hospital infections for which antibiotics are less and less effective. In fact, the C. difficile bacteria is considered “one of the greatest microbial threats to human health” by the US CDC.
We are talking about one of those hospital bacteria that has become resistant to antibiotics. What experts call a “silent pandemic” because “there are many deaths per year about whom little is said, says Bruno González Zorn, Professor of Microbiology at UCM and WHO Advisor on Antibiotic Resistance and One Health. It is estimated that 4,000 people die every year in Spain for this reason.
In this case, we are talking about a bacteria that causes severe and potentially deadly diarrhea. And it is resistant to most antibiotics. The infection recurs in many cases and the incidence is increasing. In the US alone, it is estimated that there are between 15,000 and 30,000 deaths per year.
Is the future in the stool?
Is the faecal microbiota the future? “This is just the beginning, but it’s going very fast,” admits Basterra. And yes, it may be new for humans, he admits, because they are medicines in which excrement is the raw material, while the active substance is microbiota. There is not even harmonised regulation in Europe yet. “That has to be arranged per country and that delays the start of the projects enormously.”