5-HT1D receptor agonists(Serotonin 1d (5-HT1d) receptor agonists), 5-HT2A receptor agonists(Serotonin 2a (5-HT2a) receptor agonists), 5-HT2C receptor agonists(Serotonin 2c (5-HT2c) receptor agonists)

Therapeutic Areas

Nervous System DiseasesCongenital DisordersOther Diseases+ [2]

Active Indication

Lennox Gastaut SyndromeEpilepsies, MyoclonicSeizures+ [7]

Inactive Indication-

Originator Organization

Zogenix, Inc.

Active Organization

UCB Pharma SAZogenix International Ltd.

Zogenix, Inc.

+ [4]

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

US (25 Jun 2020),

Epilepsies, MyoclonicSeizures

RegulationOrphan Drug (US), Priority Review (US), Fast Track (US), Orphan Drug (EU)

Structure

Molecular FormulaC12H17ClF3N

InChIKeyZXKXJHAOUFHNAS-UHFFFAOYSA-N

CAS Registry404-82-0

Clinical Trials associated with Fenfluramine Hydrochloride

NCT06598449 / Not yet recruitingPhase 4IIT

Assessment of Safety of the Use of Fenfluramine in Children with Dravet Syndrome Under the Age of 24 Months

Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water. Many anti-seizure medications are sodium channel blockers and exacerbate seizures in this patient population. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed a treatment protocol to allow access to fenfluramine for children under 24 months of age.

Start Date01 Oct 2024

Sponsor / Collaborator

University of Colorado Denver

[+1]

NCT06118255 / RecruitingPhase 3

Open-Label, Single-Arm, Phase 3 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome

The primary purpose of this study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.

Start Date21 May 2024

Sponsor / Collaborator

UCB BioSciences, Inc.

NCT04289467 / RecruitingPhase 2IIT

A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms

This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Patients with infantile spasms that have not responded to treatment with vigabatrin and ACTH we will be invited to participate. Study participants will undergo baseline video-EEG, receive treatment with fenfluramine for 21 days, and then undergo repeat video-EEG to determine effectiveness. Patients with favorable response will have the opportunity to continue treatment for up to 6 months.

Start Date16 Jun 2023

Sponsor / Collaborator

Children's Hospital of Orange County

100 Clinical Results associated with Fenfluramine Hydrochloride

100 Translational Medicine associated with Fenfluramine Hydrochloride

100 Patents (Medical) associated with Fenfluramine Hydrochloride

3,264

Literatures (Medical) associated with Fenfluramine Hydrochloride

01 Oct 2024·Epilepsia Open

Practical considerations for the use of fenfluramine to manage patients with Dravet syndrome or Lennox–Gastaut syndrome in clinical practice

Review

Author: Cross, J. Helen ; Specchio, Nicola ; Strzelczyk, Adam ; Lagae, Lieven ; Scheffer, Ingrid E. ; Wirrell, Elaine C.

Abstract:

Fenfluramine (FFA), an antiseizure medication (ASM) with serotonergic and sigma‐1 receptor activity, is used to manage patients with developmental and epileptic encephalopathies (DEEs). It is approved in the US for treating seizures associated with Dravet syndrome (DS) and Lennox–Gastaut syndrome (LGS) in patients ≥2 years old and as add‐on therapy for seizures associated with DS and LGS in the EU, UK, and Japan in similarly aged patients. Consensus guidelines for treatment of DS have recommended FFA to be an early‐line ASM, and it has also shown efficacy in managing seizures associated with LGS. DS and LGS are DEEs associated with a range of seizure types, developmental impairments, and multiple comorbidities. Here we provide case vignettes describing 4 patients (3 DS and 1 LGS) aged 4–29 years old in whom up to 14 ASMs had previously failed, to illustrate real‐world practice issues encountered by neurologists. This review provides guidance on the use of FFA in the context of ASM polytherapy and drug–drug interactions (DDIs), behavioral issues, dose titration, and adverse events. Along with data from the clinical trial program, these case vignettes emphasize the low risk of DDIs, a generally well‐tolerated safety profile, and other seizure and nonseizure benefits (eg, improved cognition and sleep) associated with the use of FFA in DS or LGS.

Plain Language Summary:

Fenfluramine is used to treat seizures in individuals with Dravet syndrome and Lennox–Gastaut syndrome, but there are a range of issues that clinicians may face when treating patients. This review highlights four patients from the authors’ everyday clinical work and offers guidance and practical considerations by neurologists with expertise in managing these complex conditions related to drug interactions, dosing, and side effects associated with fenfluramine.

01 Oct 2024·Epilepsia Open

Fenfluramine treatment in pediatric patients with Dravet syndrome reduces seizure burden and overall healthcare costs: A retrospective and observational real‐world study

Article

Author: Møller, Rikke S. ; Olofsson, Kern ; Rubboli, Guido ; Gjerulfsen, Cathrine E. ; Johannessen Landmark, Cecilie ; Nikanorova, Marina

Abstract:

Objectives:

Dravet syndrome is a developmental and epileptic encephalopathy characterized by early onset epilepsy with multiple seizure types often intractable to treatment. Randomized clinical trials have demonstrated how treatment with fenfluramine significantly reduces seizure frequency in patients with Dravet syndrome. The study aims to (1) describe the efficacy and tolerability of fenfluramine in a Danish cohort of patients with Dravet syndrome; and (2) evaluate whether treatment with fenfluramine reduces epilepsy‐related hospital contacts administrated by pediatricians or epilepsy‐trained nurses.

Methods:

A retrospective registry‐based cohort study at the Danish Epilepsy Centre, Filadelfia, Dianalund, Denmark, enrolled 30 pediatric patients with Dravet syndrome treated with fenfluramine between 2017 and 2023.

Results:

Thirty patients with Dravet syndrome (aged 3–21 years, 12 females) with a verified pathogenic SCN1A variant were included. They were treated with fenfluramine at a mean duration of 29 months with a mean maintenance dose of 0.5 mg/kg/day. The number of patient‐years on treatment was 75 years. At last follow‐up, 6 patients had discontinued treatment due to lack of efficacy or adverse effects. In the remaining 24 patients, generalized tonic–clonic seizures were reduced by ≥30% in 83%, by ≥50% in 67%, and by 100% in 25%. Additionally, 71% of the patients were reduced in concomitant anti‐seizure medication, and 75% experienced a reduction (mean reduction at 52%, range 11%–94%) in epilepsy‐related hospital contacts from baseline to the end of the treatment period.

Significance:

Treatment with fenfluramine effectively reduced seizure frequency and concomitant antiseizure medication in patients with Dravet syndrome. Furthermore, a decrease in epilepsy‐related contacts by 80% was observed over 6 years of treatment, which may indicate cost‐effective benefits.

Plain Language Summary:

Patients with Dravet syndrome suffer from severe epileptic seizures that are difficult to treat with medication. Earlier, treatment with fenfluramine (an anti‐seizure medication) has been documented to decrease the total number of seizures in patients with Dravet syndrome. This publication summarizes the experiences with fenfluramine in children with Dravet syndrome at the Danish Epilepsy Centre, Filadelfia, Dianalund, Denmark. Our publication also illustrates that treatment with fenfluramine may reduce the patients' number of yearly contacts with doctors and nurses specialized in epilepsy treatment, which may indicate cost‐effectiveness.

01 Oct 2024·EPILEPSIA

Utilizing an acute hyperthermia‐induced seizure test and pharmacokinetic studies to establish optimal dosing regimens in a mouse model of Dravet syndrome

Article

Author: Reilly, Christopher A. ; Mensah, Jeffrey A. ; Metcalf, Cameron S. ; Wilcox, Karen S. ; Rower, Joseph E. ; Johnson, Kristina ; Freeman, Tia

Abstract:

Objective:

The current standard of care for Dravet syndrome (DS) includes polytherapy after inadequate seizure control with one or more monotherapy approaches. Treatment guidelines are often based on expert opinions, and finding an optimal balance between seizure control and adverse drug effects can be challenging. This study utilizes the efficacy and pharmacokinetic assessment of a second‐line treatment regimen that combines clobazam and sodium valproate with an add‐on drug as a proof‐of‐principle approach to establish an effective therapeutic regimen in a DS mouse model.

Methods:

We evaluated the efficacy of add‐on therapies stiripentol, cannabidiol, lorcaserin, or fenfluramine added to clobazam and sodium valproate against hyperthermia‐induced seizures in Scn1aA1783V/WT mice. Clobazam, N‐desmethyl clobazam (an active metabolite of clobazam), sodium valproate, stiripentol, and cannabidiol concentrations were quantified in plasma and brain using liquid chromatography–tandem mass spectrometry for the combinations deemed effective against hyperthermia‐induced seizures. The concentration data were used to calculate pharmacokinetic parameters via noncompartmental analysis in Phoenix WinNonLin.

Results:

Higher doses of stiripentol or cannabidiol, in combination with clobazam and sodium valproate, were effective against hyperthermia‐induced seizures in Scn1aA1783V/WT mice. In Scn1aWT/WT mice, brain clobazam and N‐desmethyl clobazam concentrations were higher in the triple‐drug combinations than in the clobazam monotherapy. Stiripentol and cannabidiol brain concentrations were greater in the triple‐drug therapy than when given alone.

Significance:

A polypharmacy strategy may be a practical preclinical approach to identifying efficacious compounds for DS. The drug–drug interactions between compounds used in this study may explain the potentiated efficacy of some polytherapies.

News (Medical) associated with Fenfluramine Hydrochloride

16 Oct 2024

·www.globenewswire.com

Firefly Neuroscience Collaborates with Bright Minds Biosciences to Analyze the Data from its Positive Phase 1 Study using its Artificial Intelligence, FDA-Cleared BNA™ Technology

During the Phase 1 study, subjects utilized EEG headsets provided by Firefly partner, Zeto, after which the data was analyzed using Firefly’s advanced AI BNA technology analysis platformTORONTO, Oct. 16, 2024 (GLOBE NEWSWIRE) -- Firefly Neuroscience, Inc. (“Firefly,” or the “Company”) (NASDAQ: AIFF), an Artificial Intelligence (“AI”) company developing innovative solutions that improve brain health outcomes for patients with neurological and mental disorders, today announced collaborating with Bright Minds Biosciences Inc. (CSE:DRUG) (NASDAQ:DRUG) (“Bright Minds” or the “Company”), a biotechnology company focused on developing novel drugs for the targeted treatment of neuropsychiatric disorders and refractory epilepsy, to analyze the data from its first-in-human Phase 1 study of its lead compound, BMB-101. The study demonstrated positive results of the qEEG (Quantitative Electroencephalogram) data. During the EEG recording, subjects were seated with a U.S. Food and Drug Administration (FDA)- approved 19 electrode EEG headset provided by Firefly strategic partner, Zeto™ Inc. Channels were sampled at 250 or 500 Hz and referenced to A1/A2 channels (linked-ears reference) during recording. The EEG recording time was 10 minutes (~5 minutes resting with eyes closed and ~5 minutes resting with eyes open). There were four EEG recording timepoints: day 1 pre-dose (immediately before dosing) and post-dose (1h after dosing), and day 7 pre-dose and post-dose. Data was analyzed using the FireFly Neuroscience advanced EEG analysis platform. “Our artificial intelligence platform has tremendous value for the advancement and insight of clinical studies,” said Jon Olsen, CEO of Firefly. “We are pleased to have successfully collaborated with Bright Minds to analyze the data of their phase 1 study and look forward to working closely with their team once again as well as other leading biotechnology companies.” “The positive topline findings from our recently completed Phase 1 study of BMB-101, together with the observations from the qEEG portion of the study, validate our approach, as we continue to evaluate this important product candidate. BMB-101 is clearly [getting into the brain/achieving brain penetration] and activating the target receptors as we had predicted, setting us up for potential success in a number of indications that have been validated with the 5-HT2C mechanism. With this study complete, BMB-101 is now a Phase 2 ready asset, and we intend to move forward with an investigative new drug submission immediately,” stated Ian McDonald, CEO of Bright Minds. About FireflyFirefly (NASDAQ: AIFF) is an Artificial Intelligence (“AI”) company developing innovative solutions that improve brain health outcomes for patients with neurological and mental disorders. Firefly’s FDA-510(k) cleared Brain Network Analytics (BNA™) technology revolutionizes diagnostic and treatment monitoring methods for conditions such as depression, dementia, anxiety disorders, concussions, and ADHD. Over the past 15 years, Firefly has built a comprehensive database of brain wave tests, securing patent protection, and achieving FDA clearance. The Company is now launching BNA™ commercially, targeting pharmaceutical companies engaged in drug research and clinical trials, as well as medical practitioners for clinical use. Brain Network Analytics was developed using artificial intelligence and machine learning on Firefly’s extensive proprietary database of standardized, high-definition longitudinal electroencephalograms (EEGs) of over 17,000 patients representing twelve disorders, as well as clinically normal patients. BNA™, in conjunction with an FDA-cleared EEG system, can provide clinicians with comprehensive insights into brain function. These insights can enhance a clinician’s ability to accurately diagnose mental and cognitive disorders and to evaluate what therapy and/or drug is best suited to optimize a patient’s outcome. Please visit https://fireflyneuro.com/ for more information. About BMB-101BMB-101, a highly selective 5-HT2C, Gq-protein biased agonist, has demonstrated compelling activity in a host of in vitro and in vivo nonclinical tests. Compared to Lorcaserin, BMB-101 exhibits strong Gq biased signaling, coupled with minimal beta-arrestin recruitment. Bright Minds believes that G-protein biased signaling translates to better tolerance profile for this second-generation 5-HT2C agonist, making BMB-101 a best-in-class 5-HT2C agonist. Mechanistically, Serotonin (5-Hydroxytryptamine, 5-HT) is a monoamine neurotransmitter widely expressed in the central nervous system, and drugs modulating 5-HT have made a major impact in mental health disorders. Central 5-HT systems have long been associated with the control of ingestive behaviors and the modulation of the behavioral effects of psychostimulants, opioids, alcohol, and nicotine. Results of clinical trials and animal studies indicate that 5-HT2C receptor agonists may have therapeutic potential in the treatment of addiction by decreasing the intake of opioids as well as impulsive behavior that can escalate compulsive drug use. BMB-101 is a new chemical entity (NCE) and constitutes a novel scaffold 5-HT2C agonist. 5-HT2C agonism is a well proven anticonvulsant mechanism. In translational animal models, BMB-101 demonstrated a significant reduction in both the number and intensity of epileptic seizures and is a promising candidate for the treatment of Dravet Syndrome and other epilepsies. The Phase 1 trial (NCT 05397041) has been completed and BMB-101 is now Phase 2 ready. About Bright MindsBright Minds is focused on developing novel transformative treatments for neuropsychiatric disorders, epilepsy, and pain. Bright Minds has a portfolio of next-generation serotonin agonists designed to target neurocircuit abnormalities that are responsible for difficult to treat disorders such as treatment resistant epilepsy, treatment resistant depression, PTSD, and pain. The Company leverages its world-class scientific and drug development expertise to bring forward the next generation of safe and efficacious drugs. Bright Minds’ drugs have been designed to potentially retain the powerful therapeutic aspects of psychedelic and other serotonergic compounds, while minimizing the side effects, thereby creating superior drugs to first-generation compounds, such as fenfluramine, psilocybin, LSD, and ibogaine. Forward-Looking StatementsCertain statements in this press release and the information incorporated herein by reference may constitute “forward-looking statements” for purposes of the federal securities laws concerning Firefly. These forward-looking statements include express or implied statements relating to Firefly’s management teams’ expectations, hopes, beliefs, intentions, or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements are based on current expectations and beliefs concerning future developments and their potential effects. There can be no assurance that future developments affecting Firefly will be those that have been anticipated. These forward-looking statements involve a number of risks, uncertainties (some of which are beyond Firefly’s control) or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to development and commercialization of BNA™ technology; risks related to Firefly’s ability to recognize the anticipated benefits of the merger (the “Merger”) with WaveDancer, Inc. (“WaveDancer”); risks related to Firefly’s ability to correctly estimate its operating expenses and expenses associated with the Merger and other events and unanticipated spending and costs that could reduce Firefly’s cash resources; the ability of Firefly to protect its intellectual property rights; competitive responses to the business combination; unexpected costs, charges or expenses resulting from the Merger; potential adverse reactions or changes to business relationships resulting from the completion of the Merger; legislative, regulatory, political and economic developments; and those factors described under the heading “Risk Factors” in the in the registration statement on Form S-4 filed by WaveDancer with the Securities and Exchange Commission on January 22, 2024, as amended, and declared effective on February 6, 2024. Should one or more of these risks or uncertainties materialize, or should any of Firefly’s assumptions prove incorrect, actual results may vary in material respects from those projected in these forward-looking statements. It is not possible to predict or identify all such risks. Forward-looking statements included in this press release only speak as of the date they are made, and Firefly does not undertake any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Firefly Neuroscience Investor ContactsKCSA Strategic CommunicationsValter Pinto / Jack Perkins(212) 896-1254Firefly@KCSA.com Firefly Neuroscience Media ContactKCSA Strategic CommunicationsRaquel Cona, Vice President(516) 779-2630Rcona@KCSA.com

Phase 1Phase 2Clinical Result

14 Oct 2024

·medcitynews.com

Lundbeck’s Neuro-Rare Pipeline Lands Cornerstone Drug via $2.6B Longboard Pharma Acquisition - MedCity News

Neuroscience drug developer Lundbeck is expanding its scope to epilepsy via the $2.6 billion acquisition of Longboard Pharmaceuticals, a company whose lead asset is in late-stage clinical development for a rare, inherited seizure disorder that has few approved treatments. According to deal terms announced Monday, Lundbeck will pay $60 in cash for each Longboard share, representing a 54.2% premium to the biotech’s closing stock price on Friday and a 77% premium to the stock’s average price for the month of September. When La Jolla, California-based Longboard went public in 2021, it priced shares at $16 apiece. Longboard’s lead drug candidate, bexicaserin, is in development for developmental and epileptic encephalopathies (DEEs), a group of disorders that include the rare epilepsies Dravet syndrome and Lennox-Gastaut syndrome. The oral small molecule is an agonist of the 5-HT2C receptor, whose roles include regulating seizure severity. This receptor is already targeted by Fintepla, a UCB drug approved to treat seizures associated with Dravet and Lennox-Gastaut. But Fintepla’s label carries a black box warning for cardiovascular complications. Bexicaserin is designed to selectively target 5-HT2C without also hitting other receptors that can cause adverse effects. presented by Health IT Accelerating Claim Processing: Strategies to Shorten the Life of a Claim These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes. By Greenway Health Jazz Pharmaceuticals’ cannabis-derived Epidiolex, approved for Dravet, Lennox-Gastaut, and tuberous sclerosis complex, has no black box warning. Some high-profile efforts in DEEs have encountered setbacks. In June, Takeda Pharmaceutical reported its drug candidate soticlestat failed separate placebo-controlled Phase 3 tests in Dravet and Lennox-Gastaut. Longboard had been making the case that bexicaserin can stand apart from other approaches to treat epilepsies. In January, the biotech reported Phase 1b/2a data showing a median 53.3% reduction in seizure frequency in patients with DEEs during the 75-day treatment period, results that had the company making claims of best-in-class potential. Shares of the biotech soared more than 300% after that data report. Bexicaserin’s efficacy results are holding up so far. According to a Lundbeck investor presentation, nine-month open-label data show a 57.7% reduction in countable motor seizures. In September, Longboard began a global Phase 3 test enrolling Dravet patients age 2 and older. This clinical trial is part of a broader program planned to evaluate the drug in about 480 patients who have a range of DEEs. The Longboard pipeline also includes LP659, a modulator of the S1P receptor that has potential applications in neuroinflammatory disorders. A Phase 1 single-ascending dose study has been completed but plans for a Phase 1 multiple-ascending dose study are subject to resolution of a partial clinical hold. Both bexicaserin and LP659 were licensed from Arena Pharmaceuticals. presented by Sponsored Post The Promise of Value-Based Care and MedTech Innovation Monica Vajani, Executive Director for mHUB’s MedTech Accelerator, discusses how mHUB is helping innovators transition healthcare towards value-based care. By Monica Vajani - mHUB Lundbeck focuses on neuroscience drugs. The Copenhagen-based company’s top-selling product is Rexulti, a schizophrenia and depression drug that last year expanded its approved uses to include agitation caused by Alzheimer’s disease. The product accounted for 4.5 billion Danish krone (about $661 million) in revenue last year, a 16% increase compared to the prior year, according to the company’s annual report. Within neuroscience, Lundbeck executives have identified rare neurological diseases as an opportunity for pipeline expansion. Lundbeck projects a fourth quarter 2028 launch for bexicaserin. The company estimates the drug could achieve peak sales of $1.5 billion to $2 billion. “This transformative transaction will become a cornerstone in Lundbeck’s neuro-rare franchise, with a potential to drive growth into the next decade,” Lundbeck President and CEO Charl van Zyl said in a prepared statement. “Bexicaserin addresses a critical unmet need for patients suffering from rare and severe epilepsies, for which there are very few good treatment options available.” The boards of directors of both Lundbeck and Longboard have approved the acquisition, which still needs the majority of Longboard’s outstanding voting shares to be tendered. The deal also must pass regulatory muster. The transaction is expected to close by the end of this year. Image: Getty Images, Iaremenko

Phase 3AcquisitionDrug ApprovalClinical ResultPhase 1

19 Sep 2024

·www.biospace.com

Bright Minds Biosciences to Host Investor & Analyst KOL Event on September 25th

Bright Minds Biosciences announced the initiation of Phase 2 clinical trial of BMB-101 in a group of drug-resistant epilepsies and will discuss the details of the BREAKTHROUGH clinical trial on September 25th. The event will feature Epilepsy KOLs to discuss the unmet needs for epilepsy treatment. BMB-101 is the first 5-HT2C agonist in clinical studies designed to exclusively target therapeutic pathways (via G-protein) without receptor desensitization. NEW YORK, Sept. 19, 2024 (GLOBE NEWSWIRE) -- Bright Minds Biosciences Inc. (NASDAQ / CSE: DRUG) announces the Key Opinion Leader (KOL) event with leading experts in epilepsy research and treatment. The event will include Dr. Dennis Dlugos, Dr. Joe Sullivan, and Dr. Jo Sourbron, who will provide critical insights into the evolving landscape of drug-resistant seizures and the unmet needs in epilepsy care. These thought leaders will explore the scientific innovations driving the Phase 2 BREAKTHROUGH clinical trial design announced last week and highlight the potential for novel treatments. Featured KOL speakers Dr. Dennis J. Dlugos , MD, MSCE, is a professor of neurology and pediatrics at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine. He is the Director of the Section of Clinical Neurophysiology and the Epilepsy/Clinical Neurophysiology Fellowship. He holds the Tristram C. Colket, Jr. Endowed Chair in Pediatric Neurology. Dr. Dlugos is a member of the American Academy of Pediatrics, the American Academy of Neurology, the American Epilepsy Society, the American Clinical Neurophysiology Society, and the Child Neurology Society. Dr. Dlugos is a frequent lecturer locally and at national medical meetings on topics related to pediatric neurology, epilepsy, and electroencephalograms. He has published papers in journals such as Neurology, Epilepsia, Archives of Neurology, Pediatric Neurology, and Journal of Child Neurology . Dr. Joseph Sullivan , a pediatric neurologist, serves as director of the UCSF Pediatric Epilepsy Center, where he specializes in evaluating and caring for children with epilepsy, particularly refractory epilepsy (in which medications fail to control seizures). He has special interests in Dravet syndrome and PCDH19-related epilepsy (two genetic forms of the condition) as well as evaluating children for epilepsy surgery. Dr. Sullivan is a member of the American Epilepsy Society and serves on boards for several organizations. His roles include chair of the PCDH19 Alliance’s scientific advisory board, member of the Epilepsy Foundation of Northern California’s board of directors, and member of the Dravet Syndrome Foundation’s medical advisory board and board of directors. Dr. Jo Sourbron , MD, PhD, MPharm, is a physician scientist with a clinical practice at the University Hospitals of Ghent (UZ Ghent, Belgium) and a postdoctoral researcher at the University of Leuven (UZ/KU Leuven, Belgium). Dr. Sourbron has a special interest in genetic forms of epilepsy and clinical trials for drug-resistant forms of epilepsy. He has been a preclinical researcher for over a decade, focused on finding novel serotonergic (5-HT) compounds for drug-resistant epilepsies. Dr. Sourbron has explored the efficacy and safety of a variety of anti epileptic drugs including, 5-HT2 agonists like fenfluramine and lorcaserin in various animal models. For the past five years, he has been particularly involved in research involving epilepsy with eyelid myoclonia (EEM) with prominent photic induction (Sunflower syndrome). Concomitantly, he was involved in the pilot clinical trial of fenfluramine in Sunflower syndrome patients. Finally, he participated in several other clinical trials for drug-resistant epilepsies, such as cannabidiol in the treatment of drug-resistant epileptic disorders. IMPORTANT INFORMATION: Registration Details: Advance registration is required to participate in the webcast. If you cannot join live, a replay will be available. Register at Livestorm . Q&A Instructions: To ask a question during the live Q&A session, please submit your request through the portal below the webcast player when you register or send an email to info@brightmindsbio.com . About BMB-101 BMB-101 is a novel scaffold 5-HT2C Gq-protein biased agonist developed using structure-based drug design. It was explicitly designed for chronic treatment of neurological disorders where tolerance and drug resistance are common issues. Biased agonism at the 5-HT2C receptor is one of its key features and adds another layer of functional selectivity within a well-validated target. BMB-101 works exclusively via the Gq-protein signaling pathway and avoids beta-arrestin activation, which is crucial to minimize the risk of receptor desensitization and tolerance development. This provides a novel mechanism, anti-epileptic drug designed to provide sustained seizure relief in hard-to-treat patient populations. In preclinical studies, BMB-101 has demonstrated efficacy in animal models of Dravet Syndrome and numerous models of generalized seizures. In Phase 1 clinical studies, BMB-101 was given to 64 healthy volunteers in a Single Ascending Dose (SAD), Multiple Ascending Dose (MAD) and food-effects study. BMB-101 was demonstrated to be safe and well tolerated at all doses. No Serious Adverse Events (SAEs) were observed, and Adverse Events (AEs) were mild in nature and in line with on-target effects for serotonergic drugs. An extensive target-engagement study was conducted using both fluid biomarkers (transient prolactin release) and physical biomarkers (Quantitative Electroencephalogram, qEEG). Both methods confirmed robust central target engagement. A qEEG signature typical for anti-epileptic drugs was observed, with a selective depression of EEG power at frequencies observed during epileptic seizures. Furthermore, a potentiation of frontal gamma-power was observed in this study which could indicate the potential for improved cognition. About Bright Minds Biosciences Bright Minds Biosciences is a biotechnology company developing innovative treatments for patients with neurological and psychiatric disorders. Our pipeline includes novel compounds targeting key receptors in the brain to address conditions with high unmet medical need, including epilepsy, depression, and other CNS disorders. Bright Minds is focused on delivering breakthrough therapies that can transform patients’ lives. Bright Minds Biosciences has developed a unique platform of highly selective serotonergic agonists exhibiting selectivity at different serotonergic receptors. This has provided a rich portfolio of NCE programs within neurology and psychiatry. Forward-Looking Statements This news release contains “forward-looking information”. Often, but not always, forward-looking statements can be identified by the use of words such as “plans”, “expects”, “is expected”, “budget”, “scheduled”, “estimates”, “forecasts”, “intends”, “anticipates”, or “believes” or variations (including negative variations) of such words and phrases, or state that certain actions, events or results “may”, “could”, “would”, “might” or “will” be taken, occur or be achieved. Forward-looking statements in this news release include design, progress, and completion of the BREAKTHROUGH Study, future clinical development of BMB-101, and future intended use or therapeutic benefit of BMB-101 to treat refractory epilepsy disorders. A variety of factors, including known and unknown risks, many of which are beyond our control, could cause actual results to differ materially from the forward-looking information in this news release. These factors include the company’s financial position and operational runway, regulatory risk to operating in the pharmaceutical industry, and inaccuracies related to the assumption made by management relating to general availability of resources required to operate the studies noted in this news release. Additional risk factors can also be found in the Company’s public filings under the Company’s SEDAR+ pro Forward-looking statements contained herein are made as of the date of this news release and the Company disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or results or otherwise. There can be no assurance that forward-looking statements will prove to be accurate, as actual results and future events could differ materially from those anticipated in such statements. The Company undertakes no obligation to update forward-looking statements if circumstances, management’s estimates or opinions should change, except as required by securities legislation. Accordingly, the reader is cautioned not to place undue reliance on forward-looking statements. The Canadian Securities Exchange has neither approved nor disapproved the information contained herein and does not accept responsibility for the adequacy or accuracy of this news release. Contact Information Alex Vasilkevich Chief Operating Officer Bright Minds Biosciences Inc. Phone: (414)7316422 Email: alex@brightmindsbio.com Website:

Phase 1Phase 2

100 Deals associated with Fenfluramine Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D04148	Fenfluramine Hydrochloride	A08AA02	DB00574

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Lennox Gastaut Syndrome	EU	UCB Pharma SA	20 Apr 2023
Lennox Gastaut Syndrome	IS	UCB Pharma SA	20 Apr 2023
Lennox Gastaut Syndrome	LI	UCB Pharma SA	20 Apr 2023
Lennox Gastaut Syndrome	NO	UCB Pharma SA	20 Apr 2023
Epilepsies, Myoclonic	US	UCB, Inc.	25 Jun 2020
Seizures	US	UCB, Inc.	25 Jun 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Drug Resistant Epilepsy	Phase 3	CA	Zogenix, Inc.	10 Dec 2022
Spinocerebellar Ataxias	Phase 3	CA	Zogenix, Inc.	10 Dec 2022
CDKL5 Deficiency Disorder	Phase 3	US	Zogenix, Inc.	08 Mar 2022
CDKL5 Deficiency Disorder	Phase 3	JP	Zogenix, Inc.	08 Mar 2022
CDKL5 Deficiency Disorder	Phase 3	AT	Zogenix, Inc.	08 Mar 2022
CDKL5 Deficiency Disorder	Phase 3	BE	Zogenix, Inc.	08 Mar 2022
CDKL5 Deficiency Disorder	Phase 3	GE	Zogenix, Inc.	08 Mar 2022
CDKL5 Deficiency Disorder	Phase 3	DE	Zogenix, Inc.	08 Mar 2022
CDKL5 Deficiency Disorder	Phase 3	IE	Zogenix, Inc.	08 Mar 2022
CDKL5 Deficiency Disorder	Phase 3	IL	Zogenix, Inc.	08 Mar 2022

Clinical Result

Indication

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03299842 (CTgov)	Phase 3	Seizures \| Epilepsies, Myoclonic	5	ZX008 (Fenfluramine Hydrochloride)	srijrakhhv(nrybodhdyh) = dnmcjrcecq zdotgnyfhp (fzlfwrqsyj, ofihxhrtak - bloybndqoy) View more	-	13 Nov 2023
NCT03861871 (CTgov)	Phase 2	CDKL5 Deficiency Disorder \| Craniodiaphyseal Dysplasia	7	Fenfluramine Hydrochloride	crvtapmuzw(ueyyortpat) = gtcmzokjyt nsbzavxtqq (gsvyumpzlm, zftniryzoy - lafljvhqqx) View more	-	04 Oct 2023
phase 3 clinical trial (Pubmed \| Epilepsia)	Phase 3	Epilepsies, Myoclonic	143	Placebo	ebjymyjqfd(iiglrgwdeo) = apyfpdqwvh mvynmmvalb (qnpzarvxjq ) View more	Positive	01 Oct 2023
				Fenfluramine .7 mg/kg/day	ebjymyjqfd(iiglrgwdeo) = kwcwhzqksb mvynmmvalb (qnpzarvxjq ) View more
IEC2023	Not Applicable	Lennox Gastaut Syndrome	-	Fenfluramine 0.7mg/kg/day	glcddurkoy(cqrzjrpwep) = aonuocnpog vnxtqnegyi (mzsqkgzdvk )	Positive	04 Sep 2023
				Fenfluramine 0.2mg/kg/day	glcddurkoy(cqrzjrpwep) = lrcbugpxga vnxtqnegyi (mzsqkgzdvk )
AAN2023	Not Applicable	Lennox Gastaut Syndrome	-	Fenfluramine 0.7mg/kg/day	scbavkdgfo(blggioodyh) = qpccocyesn jrodabrpan (ldwstfobno )	-	25 Apr 2023
				Fenfluramine 0.2mg/kg/day	scbavkdgfo(blggioodyh) = egqjgtzwns jrodabrpan (ldwstfobno )
AAN2023	Phase 3	Lennox Gastaut Syndrome	-	Fenfluramine (0.2mg/kg/d)	tixurkbelm(bmouhntamd) = Adverse effects commonly seen were decreased appetite, somnolence, and fatigue pjcipmiayc (ojyesxvnql )	Positive	25 Apr 2023
				Fenfluramine (0.7mg/kg/d)
NCT03790137 (CTgov)	Phase 3	syndrome; salt-losing, adrenogenital syndrome \| Epilepsy, Reflex	20	Fenfluramine Hydrochloride	tzmenfopke(pmyvbrlklk) = aoqqnkswwo mljliutokd (suowkxwson, hbqzfqhkwu - mzjpnmlqic) View more	-	08 Feb 2023
NCT02682927 (CTgov)	Phase 3	Epilepsies, Myoclonic	262	placebo (Study 1: Placebo)	hdmfwyqmqp(njoemzzxrp) = wslibjreqt btwiazvwnj (djihawiwpk, juvuroolqi - cfubevdndz) View more	-	19 Oct 2022
				ZX008 (Study 1: ZX008 0.8 mg/kg/Day)	hdmfwyqmqp(njoemzzxrp) = zpbuzxcdwb btwiazvwnj (djihawiwpk, iwglgwnbpw - tsyjgorvsc) View more
NCT02926898 (CTgov)	Phase 3	Epilepsies, Myoclonic	87	ZX008 (Fenfluramine Hydrochloride) (Cohort 2: ZX008 0.5 mg/kg/Day)	xqxxlmlrtk(gxfsrwlphu) = gcdcwsaoec jqczqbllwc (cwjfcibtxv, rkcxifbpqm - wftunwofjh) View more	-	19 Oct 2022
				Matching Placebo (Cohort 2: Matching Placebo)	xqxxlmlrtk(gxfsrwlphu) = iselzsablm jqczqbllwc (cwjfcibtxv, roaxxuwfzj - rqavhnvmmv) View more
NCT03355209 (Pubmed) Manual	Phase 3	Seizures \| Lennox Gastaut Syndrome	247	Fenfluramine	llaaztwqli(vhtpbzapmc) = rfuokfskra zlcnssbzwz (pmteehxgbz ) View more	Positive	05 Oct 2022

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