Bloom Science Reports Positive Data from Phase 1 Trial of BL-001 Demonstrating Statistically Significant, Durable Weight Loss in Overweight Individuals

BL-001 achieved statistically significant placebo-adjusted mean weight loss at 28 days in overweight subjects

Overweight individuals in highest dose group experiencing weight loss at day 28 continued to lose weight as assessed 2 weeks after last dose

Dose-dependent metabolic changes mimicking the ketogenic diet observed

Clinical data on the potential ketogenic effect of BL-001 supports advancing into Phase 2 trials in both obesity and DEE (Dravet syndrome)

SAN DIEGO, March 4, 2025 /PRNewswire/ -- Bloom Science, Inc., a clinical-stage biotech company focused on the development of novel therapies for metabolic and neurological disorders, today announced that BL-001, an investigational oral therapy designed to replicate the positive effects of the ketogenic diet, demonstrated statistically significant placebo-adjusted weight loss at 28 days in a Phase 1 trial of 32 healthy adults ranging from healthy weight to overweight. The randomized, double-blind, placebo-controlled trial assessed safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of BL-001 when dosed once-daily (QD).

The overall treatment effect of BL-001 on weight loss (all treated BL-001 (n=24) vs Placebo (n=8)) was statistically significant (p=0.0424; MMRM, mixed model repeated measures) over the 28-day treatment period. Previously reported clinical trial results showed that BL-001 was well tolerated with minimal adverse events.

The percent weight change of the BL-001 treated overweight (BMI >= 25) subjects vs all subjects who received placebo (pooled) is statistically significant (p=0.0007, LS mean change -2.3% body weight change). The percent weight change between overweight and healthy weight individuals treated with BL-001 is also statistically significant (p=0.0003, LS mean change -2.2% body weight change). The highest recorded weight loss was 4.9%.

Additionally, 80% of the subjects who experienced weight loss during BL-001 treatment maintained a weight loss compared to their baseline body weight two weeks after their last dose. Of these, 100% of the overweight subjects receiving the highest dose continued to lose weight, resulting in 3.4% weight loss by the end of the 2-week follow-up period.

Ketogenic metabolic changes, including urinary ketones and reduced appetite, and weight loss were observed in the BL-001 treated subjects in a dose-dependent manner and not in placebo. Targeted biomarker analysis revealed statistically significant changes in key metabolic hormones involved in appetite control, metabolic regulation, and digestion. Additionally, a larger metabolomics analysis revealed statistically significant changes in circulating ketone bodies. These findings further support the potential ketogenic and weight loss effects, strengthening confidence in the biological activity of BL-001.

"We believe BL-001 represents a groundbreaking approach to obesity treatment, with the potential to address the limitations of existing therapies by offering a safer, naturally inspired, multi-pathway solution," said Louis Licamele, PhD, Chief Development Officer of Bloom Science. "We are excited by the current data and remain dedicated to developing a more patient-friendly alternative to current treatments that can provide sustainable weight loss while improving long-term compliance and outcomes."

BL-001 has previously been shown to replicate the effects of the ketogenic diet and prevent seizures in multiple animal models. Following the positive results from the Phase 1 trial, Bloom Science plans to study BL-001 for the treatment of Dravet syndrome and other Developmental and Epileptic Encephalopathies. The latest analysis also supports Bloom's plans to initiate a Phase 2 study in an obese population in 2026.

"While the ketogenic diet has long been known for its therapeutic effects, BL-001's potential to replicate its benefits in a more tolerable form of daily oral capsules opens exciting possibilities for patients," said Christopher Reyes, PhD, Chief Executive Officer of Bloom Science. "This validation supports our continued clinical efforts targeting both obesity and Dravet Syndrome."

About BL-001 and the Ketogenic Diet
BL-001 is a potential first-in-class, orally delivered, Live Biotherapeutic Product (LBP) candidate being developed by Bloom Science. BL-001 is designed to replicate the therapeutic effects of the ketogenic diet. Preclinical data has demonstrated replication of the ketogenic diet therapeutic effect in epilepsy models and further demonstrated modulation of gamma-aminobutyric acid (GABA) and other key bioenergetic pathways to suppress hyperexcitability, which is a key driver in seizures.

The ketogenic diet is a medical diet initially developed in 1921 to mimic fasting as a treatment for epilepsy. The ketogenic diet is very-low in carbohydrates and high in fat, and is used to treat epilepsy in children. There is accumulating clinical evidence that the diet may be effective for weight loss, reversing metabolic dysfunction and treating mental illness in certain patients. Despite its well-documented benefits for patients, the ketogenic diet can be an incredible challenge to maintain long term. A ketogenic diet mimetic like BL-001 has the potential to therefore offer substantial value to patients who benefit from such diet.

About Bloom Science
Bloom Science, Inc. is a clinical-stage biotech company focused on the development of novel first-in-class therapies for the treatment of metabolic and neurological disorders. Bloom's research and development activities leverage its expertise in gut-brain axis biology, metabolism and the microbiome to develop innovative therapeutics designed to improve patients' lives. Bloom's clinical programs include BL-001, a first-in-class, orally delivered, LBP designed to replicate the therapeutic effects of the Ketogenic Diet. 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. Bloom Science is advancing a deep pipeline of programs, focused on obesity, Dravet syndrome and Developmental and Epileptic Encephalopathies (DEEs), Amyotrophic Lateral Sclerosis (ALS), and other neurodegenerative and cognitive disorders.

For More Information: https://bloomscience.com/

Forward-Looking Statement
This press release may contain "forward-looking statements," including statements by Dr. Reyes, Dr. Licamele 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,"  "potential" 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.     

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SOURCE Bloom Science, Inc.