Therapy based on live gut microbes fares well in healthy adults in trial

Bloom Science plans Phase 2 trial of BL-001 in Dravet patients in 2024

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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BL-001, an investigational oral therapy for Dravet syndrome, was well-tolerated across multiple-ascending doses in healthy volunteers, top-line data from a Phase 1 trial show.

These results indicate that BL-001 can be dosed at concentrations up to 10-fold higher than the most effective dose in animal models, according to Bloom Science, which is developing the therapy.

The company expects to launch a Phase 2 study to test BL-001 in Dravet patients in 2024. A similar study in patients with amyotrophic lateral sclerosis, for whom BL-001 is also being developed, is also planned.

“Dravet syndrome is a rare and devastating form of childhood epilepsy in which there is an unmet need for patients whose seizures remain difficult to control and who experience significant side effects with current medications,” Paolo Baroldi, PhD, MD, chief medical officer of Bloom Science, said in a press release. “BL-001 shows promise as a novel treatment option that can change lives. With these results we plan to proceed into Phase 2 clinical development with doses we expect to be within the therapeutic window in both Dravet syndrome and ALS.”

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BL-001 granted rare pediatric disease designation for Dravet by FDA

Bloom Science also announced that the U.S. Food and Drug Administration granted BL-001 rare pediatric disease designation for Dravet syndrome, a status that provides incentives to develop treatments for serious or life-threatening disorders primarily affecting children and adolescents.

BL-001, developed using Bloom Science’s proprietary IrisRx platform, is an orally-administered medication composed of live microorganisms found in the human gut microbiome, which is the population of bacteria and other microbes naturally present in a person’s gut. Evidence is mounting on the influence of the gut microbiota on the levels of neurotransmitters in the brain via the gut-brain axis, a bidirectional communication system between the brain, the microbiota, and immune cells.

The compound was developed to mimic the antiepileptic effects of the ketogenic diet, which consists of high-fat, low-carbohydrate foods to induce a state of ketosis in the body. It works by modulating gamma aminobutyric acid (GABA), a neurotransmitter involved in different seizure disorders, as well as other important energy-related pathways. This helps decrease excessive brain activity, which is a primary causal factor for seizures.

The microbial species (strains) in BL-001 have been shown in cell-based and animal studies to increase the levels of GABA in a brain region called the hippocampus. As a result, seizure frequency and duration were both significantly reduced or eliminated.

“By taking a breakthrough approach to therapeutic development via the Gut-Brain Axis, we believe Bloom has the potential to develop completely novel, transformational treatments with superior safety and efficacy profiles that can have a significant impact on the lives of the patients suffering from rare diseases,” said Christopher Reyes, PhD, founder and CEO of Bloom Science.

“This milestone builds on many years of groundbreaking work that validate Bloom’s IrisRx platform and ability to select strains optimized for both safety and activity,” Reyes added.

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Phase 1 trial tested BL-001 versus placebo in 32 healthy volunteers

The Phase 1 trial (NCT05818306), which completed enrollment in April, tested BL-001 versus a placebo in 32 healthy volunteers administered across four escalating dose groups for 28 days.

The results showed BL-001 was safe and resulted in no serious adverse events registered in any of the four dose groups, with favorable outcomes observed in a dose-dependent manner.

All treatment-related adverse events were mild, expect for one participant who experienced moderate fatigue at the highest dose. This fatigue resolved without the need for medication, and treatment was maintained.

A decrease in appetite was the most common treatment-related adverse event seen with the highest dose, and the only one that persisted throughout the study. All other treatment-related adverse events resolved without the need for any medication.

No participant discontinued the treatment, and no heart abnormalities were reported.