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.
SAN DIEGO, May 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, today announced the appointment of Dr. Grace E. Colón as Chair of its Board of Directors, effectively immediately. A distinguished scientist and leading executive in biotechnology, Dr. Colón will lend her business, scientific, clinical and operational expertise towards helping Bloom Science in an exciting stage of research and development of new diagnostics and transformative medicines for patients with complex neurological disorders.
Dr. Colón has over 25 years of experience in biopharma, genomics, diagnostics, healthcare, industrial microbiology/biotechnology, synthetic biology, venture capital, management consulting, and entrepreneurship. Most recently, she was CEO and President of InCarda Therapeutics from 2013 to 2022, where she led the company from a seed stage start-up to one with a best-in-class inhaled cardiovascular disease therapy being evaluated in a pivotal Phase 3 trial and a growing pipeline. She serves on the boards of CareDx, Voyager Therapeutics, the MIT Corporation (MIT’s Board of Trustees) and the Biotechnology Innovation Organization (BIO), and is also Executive Chair (formerly CEO) of ProterixBio. Formerly, Dr. Colón held senior executive roles at Intrexon Corporation, Gilead Sciences, and Affymetrix. She was a partner at New Science Ventures, a New York based venture capital firm, and served on the boards of Paradigm Diagnostics (acquired by Exact Sciences), PerceptiMed and Cocoon Biotech and on the Advisory Board of the Miller Center for Social Entrepreneurship at Santa Clara University. Earlier in her career, Dr. Colón was a management consultant at McKinsey & Company and an engineer at Merck. She received her Ph.D. in chemical engineering from the Massachusetts Institute of Technology, where she was a National Science Foundation Fellow. She also holds an undergraduate degree in chemical engineering from the University of Pennsylvania.
“We are thrilled to welcome Dr. Colón as the Chair of our Board of Directors,” said Christopher Reyes, PhD, founder and CEO of Bloom Science. “Dr. Colón’s extensive technical and leadership expertise in biotech, including building strategic partnerships and fundraising, will help Bloom Science achieve our mission to deliver transformative, holistic therapeutics that have the potential to treat multiple comorbidities and that can result in fewer side effects compared to other drugs currently on the market for patients with neurological diseases.”
“Bloom Science is the leading microbiome company dedicated to developing innovative therapies for neurological diseases through the gut-brain connection,” said Dr. Colón. “Bloom’s uniquely rigorous and engineering-based approach to developing microbiome-based therapeutics, along with the company’s highly experienced team and exceptional board of directors, strongly positions the company to have a significant positive impact on patients who have not adequately responded to existing CNS and other therapies. I am honored to have the opportunity to support this vital and exciting work.”
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/
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/
