Persephone Biosciences announced positive topline results from ARTEMIS, a randomized, double-blind, placebo-controlled clinical study of a synbiotic featuring two proprietary strains of Bifidobacterium infantis, a strain of Bifidobacterium longum and Bifidobacterium breve, along with human milk oligosaccharides (HMOs) and vitamin D, in a population of toddlers and infants. The findings, which await publication, will be presented at a future medical meeting, the company reported.

The synbiotic was created as a result of findings from the company’s previous clinical study, “My Baby Biome,” which was a metagenomic and metabolomic analysis of 412 infants’ microbiomes. The authors of the study found that approximately 25% of U.S. infants lack detectable Bifidobacterium, a strain which is associated with positive metabolic health outcomes and outcompetes other pathogenic strains for HMO utilization.

“Nothing exemplifies our commitment to science-led health solutions more than the fact that we designed, undertook, and have now completed the ARTEMIS study, the largest-ever clinical study in the U.S. for a proprietary synbiotic aimed at restoring healthy microbiomes in both infants and toddlers,” said Stephanie Culler, PhD, CEO and co-founder of Persephone Biosciences. “With today’s topline results the ARTEMIS study has demonstrated speedy and robust colonization after just 30 days of treatment, in stark contrast to Lactobacillus-based products already on the market, and provided efficacy data in the form of statistically significant improvement in treated toddlers’ quality of sleep.”

“Today’s data is just the beginning. In due course, we naturally plan to share more complete results from this trial at an appropriate scientific meeting or in a publication. As a next step in this scientific journey, we are looking at studying colonization of our synbiotic in children as young as 2-12 weeks. Because when it comes to creating a healthy microbiome for life, the earlier we can stage a course-correcting intervention, the better.”

The present study enrolled 58 infants between 2 and 12 months old, and 56 toddlers between one and two years old, with both the infant and toddler groups divided into placebo and treatment arms. The treatment arm was given a 30-day supply of Persephone’s infant or toddler synbiotic formulations, and stool samples were collected at baseline, at two points during treatment, and post-treatment period. The primary goal was to determine microbiome colonization and changes to the subject’s immune profile, and generate evidence of its safety and tolerability.

The principal investigator for the study was Charles Baum, MD, a gastroenterologist with experience in microbiome-focused clinical studies through his involvement in an ongoing Persephone study evaluating oncology treatment response and prevention. Richard Insel, MD, chief medical advisor at Persephone and research professor at the University of Rochester School of Medicine & Dentistry, was the co-investigator of the study.

Results

In the study, infants had a median age of 5.5 months, with an even gender distribution, with 76% born by vaginal birth and 98% receiving breast milk. In the toddler arms, the median age was 17 months, with an approximately even gender distribution, 68% born by vaginal birth and 48% receiving breast milk.

The tolerability was validated, with no statistical difference in adverse events during the study period between the two active groups relative to their placebo controls, and no increase in average GI-related distress relative to baseline.

Persephone’s strains were more abundant and prevalent in toddlers and infants who received the synbiotic, reflecting successful colonization not shown in placebo controls.

After 30 days of treatment, at least one strain could be detected in 72% of infants and 67% of toddlers.

Treatment induced a significant increase in abundance of B. infantis at the species level, as well as in genes associated with HMO utilization in both infants and toddlers treated with the synbiotic, but not in the respective placebo arms.

In many cases, the presence of the four Persephone strains persisted at two weeks after dosing stopped, demonstrating colonization of the strains in both infants and toddlers. Lactobacillus-based probiotics, in contrast, tend to have a lower propensity to colonize the gut, the company reported.

In the toddler cohort, the synbiotic resulted in a significant improvement in sleep relative to the placebo arm (p < 0.05), according to the company.

“With so many supplements of questionable design and efficacy being marketed to new parents, the ARTEMIS study differentiates and elevates Persephone’s synbiotic by providing parents with compelling tolerability, colonization, and sleep improvement data,” said Insel. “The link between the historical collapse of infant and toddler microbiomes and increasing prevalence of allergic disease including eczema, food allergy, and asthma is well-documented. This positive and encouraging initial data points to the long-term potential of a novel synbiotic like Persephone’s to reverse these worrying but growing trends.”

“It’s rare to conduct large-scale clinical studies in children outside of infant formula, so Persephone deserves full credit for breaking new ground in the name of child wellness. Additionally, I would be remiss in not noting that this study has been completed in record time, with less than a year from initiation to topline readout,” said Baum. “Lastly, while this study has focused on the tolerability and efficacy of Persephone’s synbiotic, we are already planning future studies to look at how the product can positively impact other areas of infant health.”