Core Viewpoint - Soleno Therapeutics, Inc. is actively participating in the 2025 United In Hope: International Prader-Willi Syndrome Conference, showcasing its commitment to advancing research and improving the lives of individuals affected by Prader-Willi syndrome (PWS) through multiple presentations [1][3]. Company Overview - Soleno Therapeutics focuses on developing novel therapeutics for rare diseases, with its first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, recently approved by the FDA on March 26, 2025 [11]. Conference Participation - The conference, anticipated to be the largest focused on PWS, is a collaboration between the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [2]. - Soleno will present one oral and six poster presentations, highlighting the significance of data and advocacy in addressing the needs of the PWS community [1][3]. Presentation Details - Key presentations include: - "Swallowability and Dosing Compliance of Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome" [4]. - "Diazoxide Choline Extended-Release (DCCR) Tablets Significantly Reduce Hyperphagia in Patients with PWS Who Are Managed with Strict Food Controls" [4]. - "Long-term Efficacy Results of Diazoxide Choline Extended-Release (DCCR) Tablets in Participants with Prader-Willi Syndrome" [7]. - Presenters include experts from Queen Mary University of London and the University of Florida, indicating a strong academic collaboration [4][7][8]. About Prader-Willi Syndrome - PWS is a rare genetic neurodevelopmental disorder affecting approximately one in every 15,000 live births, characterized by hyperphagia, which can lead to severe health complications [9][10].

Group 1: Conference Overview - The 2025 United In Hope: International Prader-Willi Syndrome Conference is the first-ever international meeting jointly hosted by the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [1][2] - The conference is expected to be the largest focused on Prader-Willi syndrome (PWS) ever held, taking place from June 24-28, 2025, in Phoenix, AZ [2] Group 2: Company Involvement - Soleno Therapeutics, Inc. announced one oral and six poster presentations at the conference, highlighting its commitment to advancing research and improving the lives of those impacted by PWS [1][3] - The company's first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, was approved by the U.S. FDA on March 26, 2025, for the treatment of hyperphagia in individuals with PWS [8] Group 3: Presentation Details - The oral presentation will cover long-term efficacy results of diazoxide choline extended-release tablets in participants with PWS from completed studies, presented by Evelien Gevers [4] - Multiple poster presentations will address various aspects of diazoxide choline extended-release tablets, including swallowability, dosing compliance, and the emotional impact of hyperphagia [4][5] Group 4: About Prader-Willi Syndrome - Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in gene expression on chromosome 15, occurring in approximately one in every 15,000 live births [6][7] - The defining symptom of PWS is hyperphagia, which can lead to severe health issues and significantly diminish the quality of life for affected individuals and their families [7]

Summary of Celeno Therapeutics Conference Call Company Overview - Celeno Therapeutics is based in Redwood Shores, San Francisco Bay Area, with approximately 140 employees and rapid growth [1] - The company has a single asset, DCCR (Dizoxidicholine Extended Release Tablets), now known as VICAT XR, approved by the FDA for treating hyperphagia in patients with Prader Willi syndrome (PWS) aged four years and older [1][6] Industry Context - Prader Willi syndrome (PWS) is a rare genetic neurobehavioral metabolic disorder occurring in about 1 in 15,000 births [3] - The hallmark symptom of PWS is hyperphagia, characterized by an insatiable desire to eat, leading to severe health risks and caregiver burdens [4][5][6] Clinical Development and Efficacy - VICAT XR is the first treatment approved for hyperphagia in PWS, with a clinical program demonstrating significant reduction in hyperphagia [7] - The clinical trials began in 2018, with the primary endpoint readout in June 2020 showing no statistical significance, attributed to COVID-19 impacts [8][9] - A subsequent randomized withdrawal phase in September 2023 yielded statistically significant results, indicating the drug's effectiveness [9][10] Safety Profile - The safety profile of DCCR is consistent with its parent molecule, diazoxide, with common adverse events including hypertrichosis, edema, and hyperglycemia, typically self-limiting [12] - Only two severe adverse events reported during the clinical program [12] Commercial Opportunity - The estimated market opportunity for VICAT XR in the US is over $2 billion, with approximately 12,000 individuals living with PWS in the US and an addressable market of about 10,000 patients [13][14] - A concentrated group of 300 healthcare providers (HCPs) primarily treats 2,100 PWS patients, providing a focused commercial strategy [15] Launch Strategy - The launch strategy for VICAT XR includes a robust clinical program, a strong commercial team, and deep engagement with payers and the PWS community [15][16] - The company has established a field force team with experience in rare disease launches and is working closely with payers to ensure coverage [16][20] Payer Engagement - Payers are receptive to the value proposition of VICAT XR, recognizing the urgent need for hyperphagia treatment and the drug's efficacy [20] - The company offers SILONO-one for end-to-end patient support, ensuring access to the drug and educational resources [21] European Market Potential - PWS is a global condition, with an estimated 9,500 individuals living with PWS in the EU, presenting a strong opportunity for VICAT XR [23] - The company has submitted a Marketing Authorization Application (MAA) to the EMA, with validation announced in May 2025 [24] Financial Highlights - Celeno Therapeutics reported a strong balance sheet with $290 million in cash at the end of Q1, allowing for the execution of its commercial strategy [2][25] - The company has 55 million shares outstanding and minimal debt [25]

Soleno Therapeutics (SLNO) FY 2025 Annual General Meeting June 05, 2025 11:00 AM ET Speaker0 Hello and welcome to the Soleno Therapeutics Inc. Twenty twenty five Annual Meeting of Stockholders. Please note that this meeting is being recorded. Questions may be submitted via the message icon at the top left of your screen by typing your message, then clicking the send icon to the right of the message box. The meeting is about to begin. Good morning, ladies and gentlemen. I am Jim MacInnes, Chief Financial Off ...

Summary of Soleno Therapeutics (SLNO) Conference Call Company Overview - **Company Name**: Soleno Therapeutics - **Location**: Redwood City, California, San Francisco Bay Area - **Employee Count**: Approximately 40, up from 20 in the past 18 months [1] Industry and Product Information - **Industry**: Biotechnology, specifically focused on rare genetic diseases - **Product**: VICAT XR - **Indication**: Treatment of hyperphagia in patients with Prader Willi Syndrome (PWS) aged four years and older - **Market Opportunity**: Estimated commercial opportunity greater than $2 billion in the U.S. and significant potential outside the U.S. [2] Disease Background - **Prader Willi Syndrome (PWS)**: - Genetic, neurobehavioral, and metabolic disorder occurring in 1 in 15,000 live births - Characterized by low muscle tone at birth, leading to severe hyperphagia and behavioral issues as patients grow older [3][4][6] - Patients often require significant family support, leading to high divorce rates and PTSD in unaffected siblings [10] Clinical Development and Approval - **Approval Process**: - Based on a long phase three program with breakthrough and fast track designations - First drug approved for PWS since the disease was described in 1956 [11][12] - **Clinical Trials**: - Initial study (c six zero one) did not meet primary endpoints but subsequent analysis excluding COVID-affected data showed significant efficacy [15][17] - Randomized withdrawal study demonstrated significant worsening in placebo patients, supporting FDA submission [17][33] Safety and Efficacy - **Efficacy**: - Hyperphagia scores improved from a median baseline of 23 to 9 after treatment [16][18] - Strong evidence of efficacy with a significant p-value of 0.002 in randomized withdrawal data [17] - **Safety Profile**: - Generally consistent with the parent molecule, with common adverse events being mild and self-limiting [20] - Notable adverse events include erythema multiforme and diabetic ketoacidosis [21] Market Dynamics - **Patient Population**: - Estimated 12,000 patients in the U.S. with a total addressable market (TAM) of about 10,000 [23][24] - High diagnosis rates (85-90%) in the Western world [25] - **Commercial Strategy**: - Strong analytics and field force teams with rare disease experience - Early engagement with payers and advocacy groups to facilitate understanding and acceptance of the drug [27][28] Competitive Landscape - **Competitors**: - ACADIA's carbitosin and Aardvark's bitter taste receptor drug are in late-stage development [40][42] - ACADIA's previous attempts faced regulatory challenges, while Aardvark's data is considered less interpretable [41][43] Financial Position - **Balance Sheet**: - Approximately $290 million in cash at the end of the last quarter, including $50 million in debt [3][39] Regulatory and Intellectual Property - **IP Protection**: - Significant intellectual property protection expected to last into the late 2030s [38] Conclusion - **Launch Strategy**: - Positioned to become the standard of care for PWS with no exclusions for severity of hyperphagia and no contraindications [35] - Initial launch metrics indicate a broad-based prescription paradigm, which is promising for future growth [36][37]

Core Insights - Soleno Therapeutics has received validation from the European Medicines Agency (EMA) for its Marketing Authorization Application (MAA) for Diazoxide Choline Prolonged-Release Tablets (DCCR) aimed at treating hyperphagia in patients with Prader-Willi syndrome (PWS) aged four years and older [1][2] - The company estimates approximately 9,500 patients with PWS in key European markets, including the UK, France, Germany, Italy, and Spain, and has been granted Orphan Drug Designation in the EU, which could provide up to 10 years of market exclusivity if approved [2] Company Overview - Soleno Therapeutics is focused on developing novel therapeutics for rare diseases, with its first commercial product being VYKAT XR (diazoxide choline) extended-release tablets, approved by the FDA on March 26, 2025 [3][12] - The company aims to expedite the availability of DCCR to patients in the EU following regulatory approval [2] Product Information - DCCR is indicated for the treatment of hyperphagia in adults and pediatric patients aged four years and older with PWS [6] - The defining symptom of PWS is hyperphagia, which can lead to severe health complications and significantly impact the quality of life for affected individuals and their families [5] Market Potential - The validation of the MAA represents a significant milestone for Soleno in its mission to deliver this therapy to the PWS community in Europe [2] - The potential market opportunity is underscored by the estimated prevalence of PWS, occurring in approximately 1 in every 15,000 live births [5]

Group 1 - Soleno Therapeutics, Inc. will participate in two upcoming investor conferences in June 2025, including the Jefferies Global Healthcare Conference and the Goldman Sachs 46 Healthcare Conference [1] - The Jefferies Global Healthcare Conference presentation is scheduled for June 4, 2025, at 10:30 AM ET, while the Goldman Sachs conference presentation will take place on June 10, 2025, at 8:40 AM ET [1] - Both presentations will be available for replay in the Investors section on the company's website [1] Group 2 - Soleno Therapeutics focuses on developing and commercializing novel therapeutics for rare diseases [2] - The company's first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, is designed for treating hyperphagia in individuals with Prader-Willi syndrome aged four years and older [2]

Core Insights - Soleno Therapeutics, Inc. announced poster presentations for its VYKAT XR clinical development program at the Pediatric Endocrine Society Meeting 2025, focusing on hyperphagia treatment in Prader-Willi syndrome (PWS) [1][2] Group 1: Company Overview - Soleno Therapeutics is a biopharmaceutical company dedicated to developing novel therapeutics for rare diseases [10] - The company's first commercial product, VYKAT XR, is a once-daily oral treatment for hyperphagia in individuals aged 4 years and older with PWS [10] Group 2: Product Information - VYKAT XR (diazoxide choline) was approved by the U.S. FDA on March 26, 2025, and is now available for U.S. patients [4] - The product is indicated for treating hyperphagia associated with PWS [4] Group 3: Clinical Research - Presentations at PES 2025 include studies on long-term administration of VYKAT XR and its effects on lean body mass and behavioral symptoms in PWS [2][3] - The studies highlight significant improvements in hyperphagia and behavioral symptoms upon resuming treatment after a randomized withdrawal period [2]

Core Insights - Soleno Therapeutics, Inc. is presenting data on Prader-Willi syndrome (PWS) and its clinical program for VYKAT™ XR at the ISPOR Meeting 2025 [1][2] - VYKAT XR is approved for treating hyperphagia in patients with PWS aged 4 years and older [4][9] Group 1: Presentation Details - The first presentation focuses on mortality rates from a Phase 3 clinical trial of patients with hyperphagia and PWS treated with VYKAT XR, scheduled for May 14 [2] - The second presentation examines the burden of PWS on patients and the healthcare system, highlighting emergency department visits and inpatient stays, scheduled for May 15 [2] Group 2: About Prader-Willi Syndrome - PWS occurs in approximately 1 in 15,000 live births, characterized by hyperphagia, which can severely impact quality of life [3] - Additional symptoms include behavioral issues, cognitive disabilities, low muscle tone, and increased risk of serious health complications [3] Group 3: About VYKAT XR - VYKAT XR was approved by the FDA on March 26, 2025, and is now available for U.S. patients [4] - It is a once-daily oral treatment specifically for hyperphagia in individuals with PWS [9]

Core Insights - Soleno Therapeutics, Inc. announced the presentation of data from its VYKAT XR clinical development program for treating hyperphagia associated with Prader-Willi syndrome (PWS) at the upcoming Joint Congress of ESPE and ESE Meeting 2025 [1][2] Company Overview - Soleno Therapeutics is focused on developing and commercializing novel therapeutics for rare diseases, with its first commercial product being VYKAT XR, an oral treatment for hyperphagia in patients aged 4 years and older with PWS [10] Product Information - VYKAT XR (diazoxide choline) was approved by the FDA on March 26, 2025, and is now available to U.S. patients [2] - The product is indicated for the treatment of hyperphagia in adults and pediatric patients with PWS [4] Clinical Presentation Details - The company will present a study titled "The Impact of Prader-Willi Syndrome (PWS) on Caregivers and the Healthcare System: A Burden of Illness Study Design" in a poster format on May 11 and May 12, 2025 [2] - An oral presentation titled "Developmental Behaviour Checklist 2 Response and Relationship to Hyperphagia Reductions" is scheduled for May 13, 2025 [6] Understanding Prader-Willi Syndrome - PWS occurs in approximately 1 in every 15,000 live births, characterized by hyperphagia, which can severely impact the quality of life for affected individuals and their families [3] - Additional symptoms include behavioral problems, cognitive disabilities, low muscle tone, and increased risk of co-morbidities such as diabetes and cardiovascular disease [3]