Core Insights - Bright Minds Biosciences Inc. has announced the addition of four global experts in Prader-Willi Syndrome (PWS) to its Scientific Advisory Board to support its PWS program, which includes the nomination of BMB-105 as a new clinical candidate [1][2] - The company aims to address the unmet medical needs associated with PWS, a complex neurodevelopmental disorder affecting approximately one in every 15,000 live births, particularly focusing on hyperphagia and behavioral symptoms [2][8] Company Developments - The company held a Key Opinion Leader (KOL) event on November 6, 2025, to announce the initiation of its PWS program and the nomination of BMB-105 [1] - The first proof-of-pharmacology clinical study will evaluate BMB-101's utility in addressing hyperphagia and behavioral/neuropsychiatric symptoms of PWS, which will inform the design of subsequent studies with BMB-105 [2] Scientific Advisory Board - The newly appointed experts include Tania Markovic, Jennifer L. Miller, Elizabeth Roof, and Theresa V. Strong, each bringing extensive experience in managing and researching PWS [2][3][4][6] - Their expertise will guide the company in clinical studies and address the quality-of-life challenges faced by patients and families [2] Clinical Study Overview - The NOVA clinical study is a double-blind, randomized, Phase 2a study lasting up to 16 weeks, with a 4-week screening period followed by a 1:1 randomization to either BMB-101 or placebo [7] - The study aims to assess the effect of BMB-101 on hyperphagia-related behaviors and associated behavioral disorders in PWS, with primary and secondary endpoints focused on various severity scores [10][11] About Prader-Willi Syndrome - PWS is characterized by hyperphagia, which poses significant health risks and affects daily life, with no current drugs adequately addressing these issues [8][9] - The disorder also includes neurobehavioral challenges such as emotional dysregulation and cognitive impairment, further impacting quality of life [9] About BMB-101 - BMB-101 is a novel 5-HT2C Gq-protein biased agonist designed for chronic treatment of neurological disorders, showing promise in preclinical studies for various conditions including PWS [10][11][12] - Phase 1 clinical studies demonstrated that BMB-101 was safe and well-tolerated, with no serious adverse events reported [12] Company Background - Bright Minds is focused on developing innovative treatments for neurological and psychiatric disorders, with a pipeline that includes compounds targeting conditions with high unmet medical needs [15][16]

Core Insights - Bright Minds Biosciences Inc. has announced the addition of four global experts in Prader-Willi Syndrome (PWS) to its Scientific Advisory Board to support its PWS program, which includes the nomination of BMB-105 as a new clinical candidate [1][2] - The company aims to address the unmet medical needs associated with PWS, a complex neurodevelopmental disorder affecting approximately one in every 15,000 live births, particularly focusing on hyperphagia and behavioral symptoms [2][9] Company Developments - The company held a Key Opinion Leader (KOL) event on November 6, 2025, to announce the initiation of its PWS program and the nomination of BMB-105 [1] - The first proof-of-pharmacology clinical study will evaluate BMB-101's utility in addressing hyperphagia and behavioral/neuropsychiatric symptoms of PWS, which will inform the design of subsequent studies with BMB-105 [2] Scientific Advisory Board Members - Tania Markovic, a leading expert in obesity management and Prader-Willi Syndrome, brings over 20 years of clinical experience [2] - Dr. Jennifer Miller specializes in the treatment of individuals with PWS and has been involved in clinical trials targeting hyperphagia for over 12 years [3] - Elizabeth Roof has nearly 30 years of experience working with children and teens with PWS and has managed multiple clinical trials in this area [4][6] - Dr. Theresa V. Strong has a background in medical genetics and has directed research programs focused on PWS [7] Clinical Study Overview - The NOVA clinical study is a double-blind, randomized, Phase 2a study lasting up to 16 weeks, with a 4-week screening period to establish the presence and degree of PWS symptoms [8] - Participants will be randomized in a 1:1 ratio to receive either BMB-101 or a placebo, followed by a Maximum Tolerated Dose (MTD) titration phase and a maintenance phase [8] Drug Information - BMB-101 is a novel 5-HT2C Gq-protein biased agonist designed for chronic treatment of neurological disorders, demonstrating efficacy in preclinical studies for various conditions including PWS [12][13] - In Phase 1 clinical studies, BMB-101 was shown to be safe and well-tolerated among 64 healthy volunteers, with no serious adverse events reported [13] Company Background - Bright Minds is focused on developing innovative treatments for neurological and psychiatric disorders, with a pipeline that includes compounds targeting conditions with high unmet medical needs [16][17]

Core Insights - Tonix Pharmaceuticals is advancing its TNX-2900 program into a Phase 2 clinical trial for the treatment of Prader-Willi Syndrome (PWS), a rare genetic disorder with significant unmet medical needs [1][2] - TNX-2900 is a magnesium-potentiated intranasal oxytocin formulation aimed at improving receptor binding and reducing dose-related inconsistencies [1][4] - The FDA has granted TNX-2900 Orphan Drug and Rare Pediatric Disease designations, which may provide a Priority Review Voucher upon approval [1][2] Group 1: Clinical Trial Details - The Phase 2 trial will be randomized, double-blind, placebo-controlled, and will include participants aged 8 to 17.5 years [2] - Participants will receive 12 weeks of treatment with TNX-2900 at one of three dose levels or placebo in a 1:1:1:1 ratio [2] - The primary efficacy endpoint is the change from baseline in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [2] Group 2: Prader-Willi Syndrome Overview - PWS affects approximately 1 in 10,000 to 1 in 30,000 births and is characterized by severe hyperphagia, behavioral challenges, and obesity [3][5] - The average life expectancy for individuals with PWS is less than 30 years, highlighting the urgent need for effective treatments [2][6] - Current interventions for PWS are often inadequate and difficult to sustain [3] Group 3: Mechanism of Action - Research indicates that PWS is associated with a functional deficiency of oxytocin, which regulates feeding behaviors [4][6] - TNX-2900 aims to enhance oxytocin receptor binding and signaling while minimizing off-target effects related to vasopressin [4][7] - Previous studies have shown that intranasal oxytocin therapy can improve behaviors associated with PWS [6] Group 4: Company Background - Tonix Pharmaceuticals is a fully-integrated biotechnology company with a focus on central nervous system disorders, immunology, and infectious diseases [8] - The company has recently received FDA approval for Tonmya, a non-opioid analgesic for fibromyalgia, marking a significant milestone in its product development [8] - Tonix is also developing other candidates targeting various conditions, including TNX-801 for mpox and smallpox [8]

Core Insights - Soleno Therapeutics has recently received FDA approval for its first drug, VYKAT, targeting Prader-Willi Syndrome, and has successfully launched the product [4]. Company Overview - Soleno Therapeutics operates in the rare disease sector, focusing on Prader-Willi Syndrome with a single approved asset [4]. - The company reported a strong launch performance, achieving 646 start forms and engaging 295 prescribers within three months post-launch, exceeding initial expectations [4].

Company Overview - Aardvark Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel small-molecule therapeutics aimed at activating innate homeostatic pathways for treating metabolic diseases, particularly Prader-Willi Syndrome (PWS) [1][4] - The lead compound, ARD-101, is currently in Phase 3 clinical development for hyperphagia associated with PWS, a rare disease characterized by insatiable hunger [4] Conference Participation - Aardvark will participate in the 2025 United in Hope Conference, engaging with the PWS community and presenting data from its Phase 2 study of ARD-101 [1][2] - The presentations will include a poster and a five-minute oral presentation titled "Reduction in Hyperphagia in the ARD-101 Phase 2 Clinical Trial Informs Phase 3 HERO Trial in PWS," highlighting ARD-101's favorable safety profile and early evidence for reduced hyperphagia [2][3] Ongoing Studies - The company is currently enrolling patients in the ongoing Phase 3 HERO study, which is informed by the data from the Phase 2 study [2][3] - Aardvark is also developing ARD-201, a fixed-dose combination of ARD-101 with a DPP-4 inhibitor, aimed at addressing limitations of currently marketed GLP-1 therapies for obesity and related conditions [4]