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)

+ [2]

Therapeutic Areas

Nervous System DiseasesCongenital DisordersOther Diseases+ [2]

Active Indication

Lennox Gastaut SyndromeEpilepsies, MyoclonicSeizures+ [5]

Inactive Indication

Drug Resistant Epilepsy

Originator Organization

Zogenix, Inc.

Active Organization

Zogenix, Inc.UCB Pharma SAZogenix International Ltd.

+ [4]

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

US (25 Jun 2020),

Epilepsies, MyoclonicSeizures

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

Structure/Sequence

Molecular FormulaC12H17ClF3N

InChIKeyZXKXJHAOUFHNAS-UHFFFAOYSA-N

CAS Registry404-82-0

View All Structures (2)

Clinical Trials associated with Fenfluramine Hydrochloride

NCT06679413

/ RecruitingPhase 1

A Phase 1, Single-Center, Repeat-Dose, Open-Label, Fixed-Sequence Drug-Drug Interaction Study of ZX008 in Healthy Male Or Female Study Participants 18 To 55 Years Of Age

The purpose of the study is to assess the single-dose pharmacokinetics (PK) of 3 probe drugs (midazolam, bupropion, and metformin) before and after repeat doses of ZX008

Start Date04 Dec 2024

Sponsor / Collaborator

UCB BioSciences, Inc.

NCT06598449

/ 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 Date22 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.

100 Clinical Results associated with Fenfluramine Hydrochloride

100 Translational Medicine associated with Fenfluramine Hydrochloride

100 Patents (Medical) associated with Fenfluramine Hydrochloride

3,363

Literatures (Medical) associated with Fenfluramine Hydrochloride

02 Jan 2025·Expert Review of Neurotherapeutics

Promising therapeutic strategies for Lennox-Gastaut syndrome: what’s new?

Review

Author: Brown, Richard J. ; Vasey, Michael J. ; Besag, Frank M. C.

INTRODUCTIONThe seizures in Lennox-Gastaut syndrome are typically resistant to treatment. Seven antiseizure medications (ASMs) in the US (six in the UK/EU) are licensed for the treatment of seizures in LGS: lamotrigine, topiramate, rufinamide, clobazam, felbamate (not licensed in the UK/EU), cannabidiol and fenfluramine. Other options include neurostimulation, corpus callosotomy and dietary therapies, principally the ketogenic diet and its variants. New treatments and therapeutic strategies are needed to improve management of both seizures and cognitive/behavioral comorbidities in LGS.AREAS COVEREDEmbase and Medline were searched for articles published between 1 January 2014 and 21 August 2024 reporting efficacy data for pharmacological, neurostimulation, surgical and dietary interventions in individuals with LGS focusing on recent advances. Ongoing and prospective studies were identified from the National Library of Medicine register of clinical trials.EXPERT OPINIONLGS remains a difficult-to-treat epilepsy. Although no major breakthroughs have been reported, several established and novel ASMs, some surgical strategies and other treatment approaches are of benefit or are showing promise. Progress remains incremental but any improvements in the management of this resistant epilepsy syndrome are worthwhile.

01 Jan 2025·Epilepsy & Behavior

A comprehensive review of evolving treatment strategies for Dravet syndrome: Insights from randomized trials, meta-analyses, real-world evidence, and emerging therapeutic approaches

Review

Author: Samanta, Debopam

Dravet syndrome (DS) is a severe genetic developmental and epileptic encephalopathy, primarily caused by SCN1A gene mutations. Historically, treatments like clobazam and valproate have been used without evidence from randomized controlled trials (RCTs). However, the therapeutic landscape of DS has evolved with multiple RCTs demonstrating the efficacy and safety of three antiseizure medications (ASMs): stiripentol, cannabidiol (CBD), and fenfluramine. In the absence of direct comparisons between these therapies, several network meta-analyses have been conducted to compare the ASMs, while expert consensus has independently been developed to formulate treatment guidelines. While these three ASMs show promise in reducing seizures, increasing awareness of non-seizure outcomes-such as cognitive development and quality of life-has shifted the focus of evaluation. Some recent real-world studies of these ASMs have reported improvements in these non-seizure outcomes, alongside sustained efficacy and safety. However, natural history studies continue to underscore persistent deficits in these areas and highlight suboptimal long-term seizure control despite the use of these therapies. This review addresses these gaps by first discussing network meta-analyses and treatment guidelines, along with the practical limitations of these approaches. It then examines the long-term efficacy, safety, non-seizure effects, and cost-effectiveness from real-world studies of these ASMs. Finally, emerging research on novel therapeutic approaches, including genetic and serotonergic modulation, is explored. By evaluating these developments, this review aims to guide clinical decision-making and propose future directions for optimizing DS care.

01 Jan 2025·Talanta

Identification of fenfluramine adulteration in slimming foods using a highly sensitive immunoassay

Article

Author: Yu, Xiaoqin ; Li, Zhaodong ; Wang, Yu ; Raza, Sayed Haidar Abbas ; Huang, Xin-an ; Huang, Xin-An ; Abbas Raza, Sayed Haidar ; Zhang, Shiwei ; Chen, Jiahong ; Qian, Zhenjie ; Lei, Hongtao ; Liu, Zhiwei ; Chen, Yanhong ; Zhenjie, Qian ; Pan, Kangliang

Fenfluramine, an appetite suppressant used for weight loss, can cause significant harm if overdosed. The unauthorized addition of fenfluramine to slimming foods has raised concerns. Currently, no rapid screening method is available for the quick detection of fenfluramine in the field. This study proposes six new haptens with varying spacer arm lengths and hydrophobicity, promising to elicit antibodies capable of being highly specific and strongly binding to fenfluramine. The study found that highly hydrophobic haptens with long spacer arms favored the generation of highly sensitive antibodies. Key interaction forces for antibody recognition of fenfluramine were revealed by intrinsic molecular mechanisms. Based on the above results, monoclonal antibody for fenfluramine was prepared and an ultrasensitive icELISA method with heterologous coating strategies was developed in slimming foods. The IC₅₀ of the method was 6.25 ng/mL, the linear detection range was 0.47-83.51 ng/mL and the detection limit was 0.10 ng/mL. Recovery rates in tea bags, tablets, capsules, coffee, and beverages ranged from 96.56 % to 108.90 %. The method was successfully applied to blind samples, showing strong correlation with HPLC-MS/MS results. Thus, the developed method is suitable for identifying fenfluramine adulteration in slimming foods.

News (Medical) associated with Fenfluramine Hydrochloride

06 Dec 2024

·www.prnewswire.com

UCB Presents New Data on Impact and Burden of Epilepsy at 2024 American Epilepsy Society (AES) Annual Meeting

Results of a social media listening study of women of childbearing age with epilepsy underscore the need for HCPs to provide relevant information at critical time points to support shared decision-making2 A quality-of-life survey administered to caregivers of individuals with developmental and epileptic encephalopathies (DEEs) suggests improved understanding of caregiver definitions of normal and disruptive experiences can help researchers prioritize areas of focus to improve outcomes3 Study shows prolonged seizures are a key driver in worsening quality of life for people with epilepsy and their caregivers4 Pharmacokinetic data from Phase 1 study shows alprazolam administration via the Staccato® hand-held device is an efficient delivery route currently being studied for stereotypical prolonged seizures5 New efficacy data on FINTEPLA® (fenfluramine) as a treatment for seizures associated with Lennox-Gastaut syndrome (LGS)6 ATLANTA, Dec. 6, 2024 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced data being presented at the American Epilepsy Society (AES) Annual Meeting, taking place December 6–10 in Los Angeles, California, that focus on the impact and burden of epilepsy on people living with epilepsy and their caregivers. FINTEPLA® (fenfluramine) is approved by the U.S. Food and Drug Administration (FDA) for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients two years of age and older. (PRNewsfoto/UCB) Key data being presented show women of childbearing age (WoCBA) with epilepsy face challenges in gaining access to relevant information related to their treatment at the right time and stage in their motherhood journey.2 Often, relevant information is conflicting, not provided or easy to obtain, and questions related to epilepsy and pregnancy remain unanswered, leading to confusion and heightened anxiety.2 "At UCB, we recognize that when we listen to people living with and caring for those with epilepsy and rare syndromes and give them the opportunity to share their stories, we are better able to understand and address their range of unique needs. The robust data presented at AES demonstrate our commitment to symptom management as well as holistic patient care," said Brad Chapman, Head of U.S. Epilepsy and Rare Syndromes, UCB. "Specifically for women of childbearing age, having the right information about epilepsy is not just about managing seizures—it's about working together to encourage open communication about epilepsy, providing access to trusted resources, and empowering the community with knowledge so they can make more confident decisions about their personal care journey." In other data presented, the impact and associated burden of prolonged seizures (PS) have been found to have a profound effect on the mental health of people living with epilepsy and their caregivers, including anxiety and/or depression, reinforcing the need to bring more holistic solutions to patients and their caregivers.4 "Prolonged seizures can significantly worsen both the physical and emotional well-being of people living with epilepsy and their caretakers, underscoring the critical need for timely, effective treatment. We are excited to present new data at AES on Staccato alprazolam, an investigational drug under study in a phase 3 trial for prolonged seizures," said Hugo Xi, Head of Medical Neurology, UCB. Presentation on WoCBA with Epilepsy Going Through Motherhood: Experiences of Women of Childbearing Age with Epilepsy Throughout their Motherhood Journey and Results from a Social Media Listening Study A social media listening study was conducted across six countries (U.S., UK, France, Germany, Spain and Italy) to characterize the issues and unmet needs of WoCBA (ages 15-49) with epilepsy related to their motherhood journey. Nearly 250,000 posts on social media accounts (X, forums, YouTube, blogs, Instagram and Facebook) were analyzed over a 12-month period.2 Study results, presented in a poster session, found that WoCBA with epilepsy want to be empowered through knowledge and feel they need support finding comprehensive information at the right time in their motherhood journey to increase their confidence in shared decision-making.2 However, relevant information often is not provided or easy to obtain, and these women experience confusion and heightened anxiety because they are given conflicting guidance, and their questions related to epilepsy, contraception, pregnancy, and parenthood remain unanswered.2 Presentation on Impact of Prolonged Seizures on Patients' and Caregivers' Quality of Life Interviews and focus groups were conducted with U.S.-based adults aged 18 or older with epilepsy and caregivers of people with epilepsy age 12 or older to identify unmet needs, determine the greatest areas of burden, and assess the impact on QoL of PS, which typically last 2 or more minutes and usually do not stop on their own or worsen over time.4 Study results, presented in a poster session, found that 71% of participants reported that they or their loved ones were currently experiencing PS. 4 Living with PS has a profound effect on the mental health of people with epilepsy and their caregivers.4 Among participants, 74% experienced anxiety and/or depression, and 60% reported receiving medication or seeking therapy to help manage their mental health.4 Given this negative impact on QoL, there is an opportunity to encourage discussion and build resources for social, emotional, and psychological support for individuals experiencing PS and their caregivers.4 Presentation on Inhalation as an Efficient Delivery Route of Alprazolam for the Treatment of Acute Seizures: Randomized Study of Staccato® Alprazolam Relative to Oral Alprazolam An analysis of data from a Phase 1 study of Staccato alprazolam compared the plasma concentration of alprazolam following administration via inhalation with the Staccato device with that of alprazolam administered orally.5 Staccato alprazolam, an investigational drug in Phase 3 clinical development, is designed to be used as a single-use epileptic seizure rescue therapy that combines the Staccato delivery technology with alprazolam, a benzodiazepine.5 Staccato® alprazolam is a hand-held device that can provide rapid systemic delivery of alprazolam via inhalation, with the potential to provide rapid and early seizure termination.5 Presentation On Disruptive Impacts Of Developmental And Epileptic Encephalopathies On Patient And Family Life: A Quality-Of-Life Survey An internet-based DEE survey to define "normal" and "disruptive" symptoms by understanding how 5 key domains (seizures, sleep, communication, behavior and activities of daily living [ADLs]) affect the individual and their family members, developed in consultation with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) patient communities via patient advocacy websites, social media, and patient community events, was presented as a late-breaking abstract.3 A majority of the 489 caregivers reported that the patients with DEE experienced disruptive seizures, sleep or behavior, with these disruptive symptoms leading to temporary loss of communication and/or at least one activity of daily living (ADL).3 Presentation on FINTEPLA® ( fenfluramine) Efficacy Trajectories in Placebo or Treatment Groups From Randomized Controlled Trial to Open-Label Extension A phase 3 randomized study of FINTEPLA (fenfluramine) for management of seizures associated with Lennox-Gastaut syndrome (LGS) was presented as a podium presentation.6 Results showed that patients previously randomized to placebo in the randomized controlled trial (RCT) exhibited improvements in efficacy outcomes following transition to FINTEPLA in the open-label extension (OLE).6 The change in frequency of seizures associated with a fall occurred during month 1 of the OLE indicating a rapid onset of efficacy.6 Improvements were similarly noted in responder rates for seizures associated with a fall and clinical global impression – improvement (CGI-I) scale.6 About Staccato® Alprazolam Staccato® alprazolam is an investigational treatment (Phase 3) currently being studied to assess its potential to rapidly terminate seizures at risk of becoming prolonged or progressing to a more severe seizure type. The ability to stop a seizure as it is starting is an unmet need for epilepsy management today in the outpatient setting.7 This portable treatment combines the Staccato® delivery technology with alprazolam, a benzodiazepine, in a single inhaled dose. *The safety and efficacy of Staccato® alprazolam have not been established, and it is not currently approved for use in this indication by any regulatory authority worldwide. About Epilepsy Epilepsy is a common neurological condition worldwide and affects approximately 50 million people. Epilepsy can develop in any person at any age and is usually diagnosed after a person has had at least two seizures (or after one seizure with a high risk for more) that were not caused by some known medical condition. About UCB in Epilepsy For three decades we've been committed to people living with epilepsy and their families, surrounding the patient and caregiver through every step of their care journey. At our core we have a responsibility to elevate the healthcare ecosystem. Because of this, we have established our legitimacy on the front lines of epilepsy research, development, and treatment innovation. But our past breakthroughs are only a prologue to our future. We will continue to reimagine how we care for patients, leveraging today's expertise for a better tomorrow. With so much experience behind us and so much potential ahead, we are more invested than ever in profoundly improving the lives of those living with or caring for those with epilepsy or a rare epilepsy syndrome – through our relentless pursuit of a seizure-free life. About FINTEPLA® (fenfluramine) Oral Solution1 FINTEPLA is indicated for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients 2 years of age and older. FINTEPLA is available only through a restricted distribution program called the FINTEPLA REMS. Further information is available at or by telephone at +1 877 964 3649. IMPORTANT SAFETY INFORMATION BOXED WARNING: VALVULAR HEART DISEASE and PULMONARY ARTERIAL HYPERTENSION There is an association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease and pulmonary arterial hypertension. Echocardiogram assessments are required before, during, and after treatment with FINTEPLA. FINTEPLA is available only through a restricted program called the FINTEPLA REMS. CONTRAINDICATIONS FINTEPLA is contraindicated in patients with hypersensitivity to fenfluramine or any of the excipients in FINTEPLA and with concomitant use, or within 14 days of the administration, of monoamine oxidase inhibitors because of an increased risk of serotonin syndrome. WARNINGS AND PRECAUTIONS Valvular Heart Disease and Pulmonary Arterial Hypertension (see Boxed Warning): Because of the association between serotonergic drugs with 5-HT2B receptor agonist activity, including fenfluramine (the active ingredient in FINTEPLA), and valvular heart disease (VHD) and pulmonary arterial hypertension (PAH), cardiac monitoring via echocardiogram is required prior to starting treatment, during treatment, and after treatment with FINTEPLA concludes. Cardiac monitoring via echocardiogram can aid in early detection of these conditions. In clinical trials for DS and LGS of up to 3 years in duration, no patient receiving FINTEPLA developed VHD or PAH. Monitoring: Prior to starting treatment, patients must undergo an echocardiogram to evaluate for VHD and PAH. Echocardiograms should be repeated every 6 months, and once at 3-6 months post treatment with FINTEPLA. The prescriber must consider the benefits versus the risks of initiating or continuing treatment with FINTEPLA if any of the following signs are observed via echocardiogram: valvular abnormality or new abnormality; VHD indicated by mild or greater aortic regurgitation or moderate or greater mitral regurgitation, with additional characteristics of VHD (eg, valve thickening or restrictive valve motion); PAH indicated by elevated right heart/pulmonary artery pressure (PASP >35 mmHg). FINTEPLA REMS Program (see Boxed Warning): FINTEPLA is available only through a restricted distribution program called the FINTEPLA Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified by enrolling in the FINTEPLA REMS. Prescribers must counsel patients receiving FINTEPLA about the risk of VHD and PAH, how to recognize signs and symptoms of VHD and PAH, the need for baseline (pretreatment) and periodic cardiac monitoring via echocardiogram during FINTEPLA treatment, and cardiac monitoring after FINTEPLA treatment. Patients must enroll in the FINTEPLA REMS and comply with ongoing monitoring requirements. The pharmacy must be certified by enrolling in the FINTEPLA REMS and must only dispense to patients who are authorized to receive FINTEPLA. Wholesalers and distributors must only distribute to certified pharmacies. Further information is available at or by telephone at 1-877-964-3649. Decreased Appetite and Decreased Weight: FINTEPLA can cause decreases in appetite and weight. Decreases in weight appear to be dose related. Approximately half of the patients with LGS and most patients with DS resumed the expected measured increases in weight during the open-label extension studies. Weight should be monitored regularly during treatment with FINTEPLA, and dose modifications should be considered if a decrease in weight is observed. Somnolence, Sedation, and Lethargy: FINTEPLA can cause somnolence, sedation, and lethargy. Other central nervous system (CNS) depressants, including alcohol, could potentiate these effects of FINTEPLA. Prescribers should monitor patients for somnolence and sedation and should advise patients not to drive or operate machinery until they have gained sufficient experience on FINTEPLA to gauge whether it adversely affects their ability to drive or operate machinery. Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including FINTEPLA, increase the risk of suicidal thoughts or behaviors in patients taking these drugs for any indication. Patients treated with an AED for any indication should be monitored for the emergence or worsening of depression, suicidal thoughts or behaviors, or any unusual changes in mood or behavior. Anyone considering prescribing FINTEPLA or any other AED must balance the risk of suicidal thoughts or behaviors with the risks of untreated illness. Epilepsy and many other illnesses for which AEDs are prescribed are themselves associated with morbidity and mortality and an increased risk of suicidal thoughts and behaviors. Should suicidal thoughts and behaviors emerge during treatment, consider whether the emergence of these symptoms in any given patient may be related to the illness being treated. Withdrawal of Antiepileptic Drugs: As with most AEDs, FINTEPLA should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse reaction, rapid discontinuation can be considered. Serotonin Syndrome: Serotonin syndrome, a potentially life-threatening condition, may occur with FINTEPLA, particularly during concomitant administration of FINTEPLA with other serotonergic drugs, including, but not limited to, selective serotonin-norepinephrine reuptake inhibitors (SNRIs), selective serotonin reuptake inhibitors (SSRIs), tricyclic antidepressants (TCAs), bupropion, triptans, dietary supplements (eg, St. John's Wort, tryptophan), drugs that impair metabolism of serotonin (including monoamine oxidase inhibitors [MAOIs], which are contraindicated with FINTEPLA), dextromethorphan, lithium, tramadol, and antipsychotics with serotonergic agonist activity. Patients should be monitored for the emergence of signs and symptoms of serotonin syndrome, which include mental status changes (eg, agitation, hallucinations, coma), autonomic instability (eg, tachycardia, labile blood pressure, hyperthermia), neuromuscular signs (eg, hyperreflexia, incoordination), and/or gastrointestinal symptoms (eg, nausea, vomiting, diarrhea). If serotonin syndrome is suspected, treatment with FINTEPLA should be stopped immediately and symptomatic treatment should be started. Increase in Blood Pressure: FINTEPLA can cause an increase in blood pressure. Rare cases of significant elevation in blood pressure, including hypertensive crisis, has been reported in adult patients treated with fenfluramine, including patients without a history of hypertension. In clinical trials for DS and LGS of up to 3 years in duration, no pediatric or adult patient receiving FINTEPLA developed hypertensive crisis. Monitor blood pressure in patients treated with FINTEPLA. Glaucoma: Fenfluramine can cause mydriasis and can precipitate angle closure glaucoma. Consider discontinuing treatment with FINTEPLA in patients with acute decreases in visual acuity or ocular pain. ADVERSE REACTIONS The most common adverse reactions observed in DS studies (incidence at least 10% and greater than placebo) were decreased appetite; somnolence, sedation, lethargy; diarrhea; constipation; abnormal echocardiogram; fatigue, malaise, asthenia; ataxia, balance disorder, gait disturbance; blood pressure increased; drooling, salivary hypersecretion; pyrexia; upper respiratory tract infection; vomiting; decreased weight; fall; status epilepticus. The most common adverse reactions observed in the LGS study (incidence at least 10% and greater than placebo) were diarrhea; decreased appetite; fatigue; somnolence; vomiting. DRUG INTERACTIONS Strong CYP1A2, CYP2B6, or CYP3A Inducers: Coadministration with strong CYP1A2, CYP2B6, or CYP3A inducers will decrease fenfluramine plasma concentrations. If coadministration of a strong CYP1A2, CYP2B6, or CYP3A inducer with FINTEPLA is necessary, monitor the patient for reduced efficacy and consider increasing the dosage of FINTEPLA as needed. If a strong CYP1A2, CYP2B6, or CYP3A inducer is discontinued during maintenance treatment with FINTEPLA, consider gradual reduction in the FINTEPLA dosage to the dose administered prior to initiating the inducer. Strong CYP1A2 or CYP2D6 Inhibitors: Coadministration with strong CYP1A2 or CYP2D6 inhibitors will increase fenfluramine plasma concentrations. If FINTEPLA is coadministered with strong CYP1A2 or CYP2D6 inhibitors, the maximum daily dosage of FINTEPLA is 20 mg. If a strong CYP1A2 or CYP2D6 inhibitor is discontinued during maintenance treatment with FINTEPLA, consider gradual increase in the FINTEPLA dosage to the dose recommended without CYP1A2 or CYP2D6 inhibitors. If FINTEPLA is coadministered with stiripentol and a strong CYP1A2 or CYP2D6 inhibitor, the maximum daily dosage of FINTEPLA is 17 mg. USE IN SPECIFIC POPULATIONS In patients with severe impairment of kidney function (estimated glomerular filtration rate [eGFR]) 15 to 29 mL/min/1.73m2, dosage adjustments are recommended. FINTEPLA has not been studied in patients with kidney failure (eGFR <15 mL/min/1.73m2). Combined molar exposures of fenfluramine and norfenfluramine were increased in subjects with various degrees of hepatic impairment (Child-Pugh Class A, B, and C), necessitating a dosage adjustment in these patients. To report SUSPECTED ADVERSE REACTIONS, contact UCB, Inc. at 1 844-599-2273 or FDA at 1-800-FDA-1088 or . Please see full Prescribing Information including Boxed Warning and Medication Guide , for additional Important Safety Information on FINTEPLA. About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of €5.3 billion in 2023. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCBUSA. For further information, contact UCB: Investor Relations Antje Witte T: +32.2.559.94.14 email [email protected] US Communications Becky Malone T +1.919.605.9600 Email [email protected] Forward looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future. UCB is providing this information, including forward-looking statements, only as of the date of this press release. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. REFERENCES FINTEPLA (fenfluramine) oral solution: U.S. prescribing information. Smyrna, GA: UCB, Inc. Baker GA, et al. 2024. AES. Poster number 2.284. Bailey L, et al. 2024. AES. Poster number 1.498. Kaye D, et al. 2024. AES. Poster number 1.100. Daniels T, et al. 2024. Poster number 3.404. Nabbout R, et al. 2024. AES. Poster number 1.401. Pina-Garza JE, Chez M, Cloyd J, et al. Outpatient management of prolonged seizures and seizure clusters to prevent progression to a higher-level emergency: Consensus recommendations of an expert working group. Epileptic Disord. 2024;26(4):484-497. US-DA-2401037 Date of Preparation: November 2024 FINTEPLA® is a registered trademark of the UCB Group of Companies. STACCATO® is a registered trademark of Alexza Pharmaceuticals, Inc., and is used by UCB under license. ©2024 UCB, Inc., Smyrna, GA 30080. All rights reserved. SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 3Phase 1

18 Nov 2024

·firstwordpharma.com

Eisai officially winds down epilepsy programme

Eisai has shuttered a pair of clinical trials evaluating lorcaserin to treat Dravet syndrome after struggling to recruit enough patients with the rare form of epilepsy. The discontinuations, first disclosed in August by the Dravet Syndrome Foundation, affect the Phase III MOMENTUM 1 study and the MOMENTUM 2 extended access programme. "As other FDA-approved therapies have become available to treat this complex condition, recruiting patients for an investigational therapy has been exceedingly challenging," an Eisai spokesperson told FirstWord.The trials may have suffered from unfortunate timing, as they launched around the same time that a new drug with a similar mechanism of action hit shelves. Zogenix won FDA approval in 2020 for Fintepla (fenfluramine), a serotonin releasing agent, to treat Dravet syndrome; the drugmaker was then bought by UCB for $1.9 billion in 2022. According to the disease foundation's August release, "Just as the studies for lorcaserin began, many patients were trying the newly available Fintepla for the first time, which may have been a factor in the difficulties enrolling enough participants in the study."Earlier this year, FirstWord spoke with a dozen key opinion leaders (KOLs) about the treatment landscape for epilepsy, and several physicians thought lorcaserin could offer a better safety profile than Fintepla given its more precise mode of action as a selective serotonin 2C receptor agonist (see – Spotlight On: Emerging epilepsy drugs to watch).It's the second major setback for lorcaserin across its decades-long development history. Approved by the FDA as Belviq in 2012 as a weight-loss aid, lorcaserin was withdrawn from markets in 2020 due to an increased occurrence of cancer.

Drug ApprovalAcquisitionPhase 3

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

100 Deals associated with Fenfluramine Hydrochloride

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KEGG	Wiki	ATC	Drug Bank
D04148	Fenfluramine Hydrochloride	A08AA02	DB00574

R&D Status

Approved

10 top approved records.

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

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Langer-Giedion Syndrome	Phase 3	DE	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	US	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	JP	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	NL	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	AU	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	BE	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	DK	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	MX	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	IT	Zogenix International Ltd.	27 Nov 2017
Langer-Giedion Syndrome	Phase 3	FR	Zogenix International Ltd.	27 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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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)	onhqrxafau(mseambuqsg) = xxommlkwew nbiztqdjcm (indtbsmzoo, kpcvaiflnw - gjewjobjnu) View more	-	13 Nov 2023
NCT03861871 (CTgov)	Phase 2	CDKL5 Deficiency Disorder \| Craniodiaphyseal Dysplasia	7	Fenfluramine Hydrochloride	mtwbigfkhh(tucbobrhto) = whjafxrzrc tkraruejje (xissvtpoxs, cvxtmwfdth - uyqdxtbnag) View more	-	04 Oct 2023
AAN2023	Phase 3	Lennox Gastaut Syndrome	-	Fenfluramine (0.2mg/kg/d)	(dzpztqhtec) = Adverse effects commonly seen were decreased appetite, somnolence, and fatigue zvgmgrsmfe (jdtsiarxgl )	Positive	25 Apr 2023
AAN2023	Phase 3	Lennox Gastaut Syndrome	-	Fenfluramine (0.7mg/kg/d)		Positive	25 Apr 2023
NCT03790137 (CTgov)	Phase 3	syndrome; salt-losing, adrenogenital syndrome \| Epilepsy, Reflex	20	Fenfluramine Hydrochloride	jancdnwxib(dqrsnkwksz) = xpfnigysuk ofxhkvtade (mybraknnea, qdghcjcktk - jdovwcfjdt) View more	-	08 Feb 2023
NCT02926898 (CTgov)	Phase 3	Epilepsies, Myoclonic	87	ZX008 (Fenfluramine Hydrochloride) (Cohort 2: ZX008 0.5 mg/kg/Day)	rnqywrrqyk(ipyzlelnas) = rhnuuodzyf nxbukkatwd (zytkkdvywk, aqlbicvljc - rzqtorooss) View more	-	19 Oct 2022
NCT02926898 (CTgov)	Phase 3	Epilepsies, Myoclonic	87	Matching Placebo (Cohort 2: Matching Placebo)	rnqywrrqyk(ipyzlelnas) = swrctgfwgf nxbukkatwd (zytkkdvywk, dqmcfjyqys - pthezcvjpa) View more	-	19 Oct 2022
NCT02682927 (CTgov)	Phase 3	Epilepsies, Myoclonic	262	placebo (Study 1: Placebo)	omgjrsgisu(xslnsgnite) = xilkofvrqj dgvbukchlb (giqijyinqn, qdasybbgoz - wazcfkneaf) View more	-	19 Oct 2022
NCT02682927 (CTgov)	Phase 3	Epilepsies, Myoclonic	262	ZX008 (Study 1: ZX008 0.8 mg/kg/Day)	omgjrsgisu(xslnsgnite) = zmylnomqkl dgvbukchlb (giqijyinqn, fvjtxucued - dzvqvritzx) View more	-	19 Oct 2022
NCT03355209 (Pubmed) Manual	Phase 3	Seizures \| Lennox Gastaut Syndrome	247	Fenfluramine	jbzgybfysg(sglaxyywoz) = amtquxllgm pbhlvwdmcy (rccenafgpn ) View more	Positive	05 Oct 2022
NCT03355209 (Pubmed \| JAMA Neurol)	Phase 3	Lennox Gastaut Syndrome	263	Fenfluramine 0.7-mg/kg/d	ifxdvbrwco(hgrbvyzxqd) = porbedgyud nqugseotjc (sueumdsckw, -31.0 to -8.7) View more	Positive	02 May 2022
NCT03355209 (Pubmed \| JAMA Neurol)	Phase 3	Lennox Gastaut Syndrome	263	Fenfluramine 0.2-mg/kg/d	ifxdvbrwco(hgrbvyzxqd) = dmbhjfxtme nqugseotjc (sueumdsckw )	Positive	02 May 2022
NCT02682927 \| NCT02926898 \| NCT02823145 \| NCT02826863 \| NCT03780127 (Pubmed \| Seizure)	Phase 3	Epilepsies, Myoclonic	732	Fenfluramine	nnhfwirsxe(mtyjckzitz) = rxmgogjhxu yohmstmxks (gwhpwztdoq, 0.4 - 6.7) View more	-	02 Nov 2021
NCT02823145 (4684, AAN2020)	Phase 3	Epilepsies, Myoclonic	330	Fenfluramine Hydrochloride Oral Solution	jufhkgxczr(ldbreqzubl) = There were no echocardiographic or clinical observations of valvular heart disease or pulmonary hypertension at any time during the OLE ypyzxjfley (oabchllspu )	Positive	14 Apr 2020

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