ALS and Dravet Syndrome Agent BL-001 Demonstrates Safety, Tolerability in Early-Stage Study

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Over a 28-day period, no adverse events led to study drug withdrawal, with decreased appetite as the most common treatment-related AE observed in the highest dosed cohort.

Christopher Reyes, PhD, founder and chief executive officer, Bloom Science

Christopher Reyes, PhD

According to a recent announcement, Bloom Science’s investigational agent BL-001, a first-in-class therapeutic being developed for both Dravet syndrome and amyotrophic lateral sclerosis (ALS), demonstrated a positive safety and tolerable profile in a phase 1 study (NCT05818306) of healthy individuals. The company plans to advance the therapy into phase 2 studies of both patients with Dravet and ALS in 2024.1

The phase 1a, randomized, parallel-group, double-blind, placebo-controlled, single-center study assessed a cohort of 32 healthy participants who were randomly assigned 4:1 to 4 different doses of BL-001 or placebo for a 28-day period. At the conclusion of the treatment period, no serious adverse events (AEs) were observed across the dosed cohorts, no AEs led to study drug withdrawal, and all participants completing the trial. In addition, there were no clinically significant electrocardiogram abnormalities reported in any of the individuals across all treatment cohorts.

"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, MD, PhD, chief medical officer, Bloom Science, said in a statement.1 "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."

Other topline findings from the trial showed that all treatment-related AEs were mild with the exception of 1 individual treated in the high-dose cohort who experienced moderate fatigue that was resolved without intervention. The patient continued on treatment after this occurred. Furthermore, in the highest dose cohort of BL-001, the most common treatment-related AE was decreased appetite, occurring in 3 of the 6 individuals, with no other treatment-related AE occurring in more than a single patient.

READ MORE: Edaravone Reduces Risk of Milestone Events, Increases Survival in ALS

Decreased appetite, which persisted until the follow-up period for 2 treated individuals, was the only treatment-related AE that was not transient. All treatment-related AEs were resolved without intervention. Above all, favorable strain kinetics were observed with BL-001 component strains increasing in a dose-dependent manner.

BL-001, a live biotherapeutic, is designed to treat drug-resistant seizures by replicating the metabolic profile of validated ketogenic diets and thereby altering the metabolic processing of biochemicals. In doing so, BL-001 alters the bioenergetic infrastructures in neurons and astrocytes, driving increased biosynthesis of gamma aminobutyric acid (GABA), a neurotransmitter implicated in various seizure disorders including Dravet syndrome. Bloom’s second lead program, BL-002, targets motor-neuron cell death in ALS, and is built on evidence that oxidative stress drives ALS by causing the loss of motor neurons and the dysfunction of mitochondria.2

"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,” Christopher Reyes, PhD, founder and chief executive officer, Bloom Science, said in a statement.1 "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."

REFERENCES
1. Bloom Science announces positive topline data from a phase 1 clinical trial of BL-001, a potential first-in-class therapeutic being developed for both Dravet syndrome and ALS. News release. Bloom Science. August 17, 2023. Accessed August 21, 2023. https://www.prnewswire.com/news-releases/bloom-science-announces-positive-topline-data-from-a-phase-1-clinical-trial-of-bl-001-a-potential-first-in-class-therapeutic-being-developed-for-both-dravet-syndrome-and-als-301903352.html
2. Harnessing the gut-immune-brain axis to treat disease. Bloom Science. Biopharm Deal. https://www.nature.com/articles/d43747-022-00228-wAccessed August 21, 2023.
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