BL-001 Designed to Replicate the Antiepileptic Effects of the Ketogenic Diet
Topline Results including Safety and Tolerability Expected in Third Quarter of 2023
SAN DIEGO, April 25, 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, today announced that the company has completed enrollment in all four cohorts of healthy adult participants as part of its Phase 1 clinical trial. Bloom is developing BL-001 for Dravet syndrome, a rare pediatric epilepsy that significantly impacts individuals throughout their lifetime.
“The initial dosing and completion of enrollment of our Phase 1 clinical trial for BL-001 represents a huge milestone for Bloom Science and for the epilepsy community as we advance our clinical development program for Dravet syndrome and other developmental epilepsies,” said Christopher Reyes, PhD, founder and CEO of Bloom Science. “We believe BL-001 has the potential to provide patients with the same neurological and seizure relief benefits of the Ketogenic Diet, but in the form of a simple daily oral therapeutic. Our preclinical studies have shown that BL-001 possesses strong potential to reduce neuronal hyperexcitability, control seizures and overcome drug-resistance with significantly fewer side effects compared to other drugs on the market. We are committed to developing a more holistic therapy for patients with Dravet syndrome that not only reduces seizure burden, but also treats significant comorbidities.”
Bloom’s BL-001 is an orally administered Live Biotherapeutic Product reverse translated from the positive clinical outcomes associated with the Ketogenic Diet in treating refractory epilepsy. The Phase 1 study of BL-001 is a randomized, double-blind, placebo-controlled, single-center, multiple ascending dose study in healthy volunteers. The primary objective of this study is to evaluate the safety and tolerability of BL-001. A total of 32 healthy adult volunteers have been enrolled, across four dose cohorts and randomized in a 3 to 1 ratio of BL-001 to placebo. The trial will also evaluate the effects of BL-001 on the gut microbiota and pharmacodynamic biomarkers.
The Phase 1 trial is being conducted in Europe. Following successful completion of the Phase 1 trial, Bloom intends to initiate a Phase 2 study in patients with Dravet syndrome.
More information about the Phase 1 trial is available at clinicaltrials.gov (NCT05818306).
About Dravet Syndrome Dravet syndrome, a rare and devastating treatment-resistant form of childhood epilepsy, presents significant unmet needs for patients. Patients typically try many different medications, sometimes experiencing significant side effects, yet seizures remain difficult to control and are not well managed with current medications. Dravet syndrome is characterized by intractable seizures and significant developmental delays. The onset of seizures is usually between five and eight months of age, after previous normal development. Dravet syndrome affects nearly every aspect of patients’ and their families’ lives. Ten to twenty percent of individuals with the condition do not survive beyond the age of ten. Beyond medication, the ketogenic diet is also used to treat Dravet syndrome.
About BL-001 BL-001 is a first-in-class, orally delivered, Live Biotherapeutic Product (LBP) being developed by Bloom Science for the potential treatment of seizures associated with Dravet syndrome and other rare developmental and epileptic encephalopathies (DEEs). BL-001 is designed to replicate the antiepileptic effect of the Ketogenic Diet to modulate gamma aminobutyric acid (GABA) and other key bioenergetic pathways to suppress hyperexcitability which is a key driver in seizures. 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.
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.
Bloom Science Forward-Looking Statement
This press release may contain a number of “forward-looking statements,” including statements made by Dr. Reyes in this press release, as well as statements regarding Bloom Science’s expectations about any or all 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, without limitation, activities at clinical trial sites) and expectations regarding data from clinical trials; (iii) the data that will be 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, including as compared to other therapies. Without limiting the foregoing, 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 that 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 they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for Bloom Science’s product candidates; uncertainties regarding the intellectual property protection for Bloom Science’s technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property. 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 contained in this press release, other than to the extent required by law.
Bloom is excited to welcome Roya Hakimzadeh as Director of Patient Advocacy. Roya joins Bloom with nearly a decade of experience working in rare disease at companies spanning development of small molecules to gene therapies. Her work in patient advocacy and patient services along with her dedication to patient-centric drug development is integral for the rare disease community Bloom is serving.
SAN DIEGO, Calif., January 4, 2023 — Dr. Stephen Wright joins Bloom Science, Inc., a clinical-stage biotechnology company developing novel medicines for neurological diseases and conditions, as a senior scientific and medical advisor alongside clinical advisors Jacqueline French, MD and Eric Kossoff, MD. The clinical advisory board provides guidance on Bloom’s discovery, development, and commercialization of new generation medicines – live biotherapeutic products (LBPs) – to treat neurological diseases.
Dr. Wright brings to Bloom over 30 years of biotech and pharmaceutical expertise developing medicines, including: Epidiolex, a prescription medicine that is used to treat seizures associated with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex in patients one year of age and older; and approval of the first second-generation 5 lipoxygenase inhibitor.
He served as chief medical officer and an executive member of the main board of directors of GW Pharmaceuticals from 2005 to 2018. He joined GW from Ispen, where he was senior vice president of clinical research and development and a member of the UK board of directors. Dr. Wright also served as venture head of neuroscience at Abbott Laboratories. He has numerous higher degrees and has authored over 100 publications. Dr. Wright is a visiting professor in the School of Chemistry, Food, and Pharmacy at The University of Reading.
“Bloom is thrilled to receive Dr. Stephen Wright’s innovative medical expertise as a member of our respected clinical advisory board as we advance our lead program to develop an orally-administered LBP to treat Dravet syndrome and other epileptic diseases,” said Christopher Reyes, PhD, CEO, founder, and director of Bloom Science.
Bloom clinical advisory board member Dr. French serves as chief scientific officer of the Epilepsy Foundation. She is also a professor of neurology in the Epilepsy Section at the New York University Langone Medical Center. Dr. French also serves as director of the Epilepsy Study Consortium, a nonprofit organization devoted to speeding new therapies through the development process and into the clinic.
Bloom medical advisor Dr. Kossoff is director of the Child Neurology Residency Program and a professor of neurology at Johns Hopkins Medicine. His expertise and research is in seizures and epilepsy, Ketogenic diet and Atkins diet for children and adults, infantile spasms, and Sturge-Weber syndrome.
ABOUT BLOOM SCIENCE
Bloom Science is a clinical-stage biotechnology company on a mission to bring transformative live biotherapeutic products (LBPs) to underserved patients with rare or complex neurological diseases who have not adequately responded to existing therapies. Bloom is taking a fundamentally different path to developing medicines by harnessing our expanding insight into how the gut microbiome modulates key processes in the immune system, nervous system and brain. Bloom has developed a platform to investigate how changes in the gut microbiome influence brain biochemistry and immune system activity that it can leverage to develop multi-functional therapeutics addressing multi-pathway driven neurological disorders. Learn more: https://bloomscience.com/
SAN DIEGO, Calif., January 4, 2023 — Bloom Science, Inc., a clinical-stage biotechnology company developing novel medicines for neurological diseases and conditions, and Yeda Research and Development Co Ltd, the Weizmann Institute of Science’s technology transfer arm to advance science for the benefit of humanity, have entered into an agreement in which Bloom obtained from Yeda an exclusive, worldwide license to use specific microbial strains in developing and commercializing live biotherapeutic products (LBPs) to ameliorate amyotrophic lateral sclerosis (ALS) symptoms.
There is a large unmet need for patients who have not responded to existing therapies for ALS, a disease that causes nerve cells to die, which breaks the neural pathways, and causes extreme mobility loss over time. Bloom’s systematic approach to developing a new generation of therapeutics starts with the gut microbiome.
“The gut microbiome’s influence on the brain offers incredible potential for healing,” said Bloom Science founder and CEO Christopher Reyes, PhD. “Bloom’s collaboration with Yeda enables us to advance our program to modulate the gut microbiome interaction with the brain and provide ALS patients with new therapeutic options that help restore brain function.”
Bloom’s final lead orally-administered LBP candidate selection for ALS, BL-002, started in 2022 with animal models and will advance towards human clinical trials in 2023. The company is testing multiple drug candidates for ALS.
Bloom’s BL-002 is built on evidence that oxidative stress drives ALS by causing the loss of motor neurons and the dysfunction of mitochondria. Nicotinamide, an important NAD+ pathway metabolite and a derivative of vitamin B3, can counter the effects of ALS.
BL-002 produces this vitamin and Bloom has shown it attenuates motor-neuron loss, and increases lifespan and motor coordination, in ALS model mice.
ABOUT BLOOM SCIENCE
Bloom Science is a clinical-stage biotechnology company on a mission to bring transformative live biotherapeutic products (LBPs) to underserved patients with rare or complex neurological diseases who have not adequately responded to existing therapies. Bloom is taking a fundamentally different path to developing medicines by harnessing our expanding insight into how the gut microbiome modulates key processes in the immune system, nervous system and brain. Bloom has developed a platform to investigate how changes in the gut microbiome influence brain biochemistry and immune system activity that it can leverage to develop multi-functional therapeutics addressing multi-pathway driven neurological disorders. Learn more: https://bloomscience.com/
SAN DIEGO, Calif., January 4, 2023 — Bloom Science, Inc., a clinical-stage biotechnology company developing novel medicines for neurological diseases and conditions, successfully completed a pre-IND (Investigational New Drug) meeting with the U.S. Food and Drug Administration (FDA) and gained alignment on the initial Phase 1 clinical trial of its oral, live biotherapeutic product (LBP), BL-001, for the treatment of patients with Dravet syndrome.
Dravet syndrome is a developmental and epileptic encephalopathy, a severe form of epilepsy with frequent and difficult to treat seizures that begin in infancy or early childhood and causes developmental challenges. Bloom’s BL-001 LBP is reverse translated from positive clinical outcomes associated with the ketogenic diet in treating refractory epilepsy.
Bloom presented to the FDA its planned Phase 1 study design, including the proposed patient population. The FDA aligned with the source and characterization of Bloom’s product strains, provided patient criteria guidance and agreed that no additional nonclinical studies are required to initiate the Phase 1 study in healthy volunteers.
BL-001 is set to enter the clinic, with a phase 1 clinical trial in early 2023.
ABOUT BLOOM SCIENCE
Bloom Science is a clinical-stage biotechnology company on a mission to bring transformative live biotherapeutic products (LBPs) to underserved patients with rare or complex neurological diseases who have not adequately responded to existing therapies. Bloom is taking a fundamentally different path to developing medicines by harnessing our expanding insight into how the gut microbiome modulates key processes in the immune system, nervous system and brain. Bloom has developed a platform to investigate how changes in the gut microbiome influence brain biochemistry and immune system activity that it can leverage to develop multi-functional therapeutics addressing multi-pathway driven neurological disorders. Learn more: https://bloomscience.com/
