New CDI Therapy Builds Tailored, Standardized Whole Microbiome Therapeutics

A potential new therapy for recurrent and severe cases of Clostridioides difficile (CDI) has been successfully evaluated in a phase I trial and its developers say they will continue to develop the product.

MyBiotics Pharma Ltd., a microbiome-based therapeutics developer, said they completed the trial for MBX-SD-202, a whole microbiome treatment, last month. The product is based on an company’s proprietary SuperDonor platform, which allows the company to duplicate and produce live microbial populations derived from donor fecal samples.

“We are excited to have completed the first human test of our scalable whole microbiome SuperDonor technology,” David Daboush, the company’s CEO, said in a press release. “The trial was successful and enables us to continue with our clinical program as planned, advancing our pipeline in C diff treatment and potentially additional indications already in development.”

CDI has become a major public health problem around the world. Though it can successfully be treated with antibiotics, the infection recurs in about 1 in 4 cases following antibiotic treatment. About 30,000 people die from CDI in the United States each year.

In recent years, fecal microbiota transplantation (FMT) has emerged as a promising new treatment avenue for patients with recurrent CDI. The infection tends to thrive in patients with microbiota that are altered by antibiotics and/or other causes. The stool-transplantation procedure works by using a healthy donor’s sample to restore a patient’s microbiota to a normal, healthy state.

However, MyBiotics said FMT comes with significant limitations. The procedure can be costly and relies upon finding a sufficient number of donors who can pass screening procedures. Even with screening, the procedure is not without risk, such as accidentally transplanting harmful bacteria.

The goal of the SuperDonor computational platform is to identify, design, and create therapeutics that have a pre-selected target microbial profile. Like FMT, the therapeutics are based on biological samples, such as fecal samples. Unlike FMT, samples can be scaled and standardized in a controlled manner.

The company said the platform can be used to develop a range of therapies, from single-strain products, to complex microbial consortia, to whole microbiome products like MBX-SD-202. The platform uses artificial intelligence and machine learning to design the products, the company said.

The phase 1 trial was designed to confirm the safety of MBX-SD-202. In the trial, healthy volunteers were divided into 3 groups, each of which received a single capsule of MBX-SD-202 with a different dosing level for each group. Subjects were then monitored for 30 days using lab tests, stool samples, and in-person visits. The company said all 3 doses in the trial appeared to be safe. No serious adverse events were reported, and none of the patients had significant shifts in general health or gastrointestinal symptoms.

“Now that its safety has been confirmed in healthy subjects, we look forward to applying the SuperDonor technology to multiple indications, such as oncology, in the company’s pipeline,” Daboush said.

In addition to recurrent CDI and immuno-oncology, the company’s pipeline also includes investigational treatments for bacterial vaginosis and non-alcoholic steatohepatitis, though MBX-SD-202 is the furthest along in development. The company said development of MBX-SD-202, is expected to continue through 2023.