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

by bloomscience | Aug 17, 2023 | Press Releases

• BL-001 demonstrated a favorable safety and tolerability profile in healthy volunteers that supports dosing up to 10-fold higher than the efficacious dose level in animal models
• Dose dependent strain kinetics of BL-001 observed
• The U.S. Food and Drug Administration (FDA) grants Rare Pediatric Disease Designation to BL-001 for Dravet syndrome

SAN DIEGO, August 17 2023 – Bloom Science, Inc., a clinical-stage, central nervous system (CNS) company focused on discovering and developing breakthrough therapeutics that target the Gut-Brain Axis for neurological diseases, today announced positive results from its Phase 1 clinical study in healthy volunteers that demonstrate a favorable safety, tolerability and strain kinetics profile of BL-001, an orally-delivered Live Biotherapeutic Product (LBP). Bloom is developing BL-001 for both Dravet syndrome and amyotrophic lateral sclerosis (ALS). Bloom also announced today that BL-001 received Rare Pediatric Disease Designation from FDA for Dravet syndrome.

“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,” said Paolo Baroldi, PhD, MD, Chief Medical Officer of Bloom Science. “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.”

Summary of Key BL-001 Phase 1 Study Results:

• BL-001 demonstrated a favorable safety profile and was well tolerated with no Serious Adverse Events (SAEs) across all four dose cohorts 
• Favorable strain kinetics were observed with BL-001 component strains increasing in a dose dependent manner
• All treatment-related Adverse Events (AEs) were mild with the exception of one subject in the highest dose who experienced moderate fatigue that resolved without intervention and they continued on treatment 
• In the highest dose cohort of BL-001, the most common treatment-related adverse event (AE) was decreased appetite (3/6 subjects) with no other treatment-related AEs occurring in more than a single individual
• All treatment-related AEs were transient, with the exception of decreased appetite which persisted until the follow-up period for two subjects, and all treatment-related AEs resolved without intervention
• No AEs led to study drug withdrawal and all participants completed the study
• There were no clinically significant ECG abnormalities reported in any individual across all treatment cohorts

“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.”

Based on the preclinical profile together with these Phase 1 results, Bloom intends to advance BL-001 into Phase 2 studies in both Dravet syndrome and ALS patients in 2024.  

About Phase 1 Clinical Trial of BL-001 in Healthy Volunteers
The Phase 1 study of BL-001 was a randomized, double-blind, placebo-controlled, single-center, multiple ascending dose study in healthy volunteers (NCT05818306). The primary objective of the study was to investigate the safety and tolerability of BL-001 in healthy volunteers for 28 consecutive days. In the study, BL-001 or placebo was administered to 32 healthy adult participants (BL-001 n=24; placebo n =8) across four dose cohorts.

About BL-001
BL-001 is a first-in-class, orally delivered, Live Biotherapeutic Product (LBP) being developed by Bloom Science for the treatment of multiple neurological diseases, including Dravet syndrome and ALS. 

BL-001 was first designed to replicate the antiepileptic effect of the Ketogenic Diet to modulate gamma aminobutyric acid (GABA) and other key bioenergetic pathways. BL-001 contains two rationally selected human gut microbes that have been shown in both cell-based assays and animal studies to eliminate hyper-excitatory activity, increase GABA in the hippocampus and significantly reduce or eliminate both seizure frequency and duration.

BL-001 also aims to address the underlying oxidative stress, a critical factor in the progression of ALS, to potentially slow or halt disease progression. BL-001 has been shown to attenuate motor-neuron loss, increase lifespan and motor coordination in preclinical ALS studies.

About Bloom Science
Bloom Science is a clinical-stage, central nervous system (CNS) company, trailblazing a path to transformative, novel therapeutics for patients with rare and complex neurological disorders. Bloom’s proprietary discovery platform, IrisRx™ enables a fundamentally different approach to developing medicines by harnessing our expanding insight into the Gut-Brain Axis to design and develop therapeutics that target novel biology with superior safety profiles. The IrisRx™ platform unlocks the potential for developing multi-functional therapeutics from the most common cell type in the human body, gut commensal microbes, while also utilizing an expanding proprietary knowledge base to advance therapeutic candidates based on synthetic biology. Bloom Science is advancing a deep pipeline of programs, including Dravet syndrome, other rare epilepsies, Amyotrophic Lateral Sclerosis (ALS), and other neurodegenerative and cognitive disorders. For more information, visit: https://bloomscience.com/

Forward-Looking Statement
This press release may contain “forward-looking statements,” including statements by Dr. Reyes or other persons on Bloom Science’s behalf and statements regarding Bloom Science’s expectations about any of the following: (i) the safety, efficacy and clinical progress of our various clinical programs, including our BL-001 program; (ii) the status of clinical trials (including activities at clinical trial sites); (iii) the data generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; and (iv) the therapeutic value, development, and commercial potential of Bloom Science Live Biotherapeutic Products. The words “believes,” “anticipates,” “plans,” “expects” and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although Bloom Science believes such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially.  You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. Bloom Science disclaims any obligation or undertaking to update or revise any forward-looking statements, other than as required by law.