Vykat XR (diazoxide choline controlled release) / Soleno Therap, PANTHERx Rare - LARVOL DELTA

SI03-03. Introducing VYKAT™ XR (diazoxide choline) extended-release: The First and Only FDA-Approved Treatment for Hyperphagia in Adults and Children (≥4 Years) With Prader-Willi Syndrome (PWS) (ENDO 2025) - "Join us for a peer-led session covering: • The impact of hyperphagia in PWS • Clinical efficacy and safety data for VYKAT XR • Dosing, administration, and how to initiate treatment • A powerful firsthand account from a caregiver whose loved one is using VYKAT XR Don't miss this opportunity to learn from experts and gain a firsthand perspective on managing hyperphagia in PWS. Important Safety Information, full Prescribing Information, and Medication Guide for VYKAT XR will be available at the Science & Innovation Theater or online at VYKATXRHCP.com."

Clinical • Genetic Disorders • Pediatrics • Prader–Willi syndrome

The Glycemic Outcomes of Diazoxide Choline Extended-Release (DCCR) Tablets for Administered for Hyperphagia in Individuals with Prader-Willi Syndrome Over 4 Years (ENDO 2025) - "Administration of DCCR for the treatment of hyperphagia in individuals with PWS was generally well tolerated and associated with non-clinically significant increases in mean HbA1c over 3 years of use. Hyperglycemic adverse events were readily monitorable and generally manageable with standard of care concomitant medications allowing for continuation of DCCR therapy. Overall, the safety profile of DCCR in patients with PWS remains favorable."

Clinical • Diabetes • Metabolic Disorders • Prader–Willi syndrome • Type 2 Diabetes Mellitus

Characterization of Peripheral Edema in Individuals with Prader-Willi Syndrome During Long-term Administration of Diazoxide Choline Extended-Release Tablet (DCCR) Over 4.5 Years (ENDO 2025) - "Administration of DCCR to patients with PWS was associated with peripheral edema in a minority of C601 + C602-OLE participants. When present, peripheral edema tended to occur early in treatment, was usually Grade 1 or 2, and infrequently resulted in study medication interruption or discontinuation. Overall, DCCR was well tolerated in the intended population."

Clinical • Metabolic Disorders • Prader–Willi syndrome

Soleno Therapeutics, Inc. (SLNO) Secures EMA Validation for PWS Treatment VYKAT XR (Insider Monkey) - "Soleno Therapeutics, Inc...has achieved a major regulatory milestone as the European Medicines Agency (EMA) validated its Marketing Authorization Application for Diazoxide Choline Prolonged-Release Tablets (formerly DCCR) to treat hyperphagia in Prader-Willi syndrome (PWS) for patients aged four and older."

EMA filing • Prader–Willi syndrome

Expected vs. Observed Mortality Rates, Expressed As Number Needed to Treat, From a Phase 3 Clinical Trial Program of Patients With Hyperphagia and Prader-Willi Syndrome Treated With Diazoxide Choline Extended Release (DCCR) (ISPOR 2025) - "PWS is a rare condition with many unmet needs and associated with an elevated mortality rate. Patients treated with DCCR did not experience a mortality event through up to 4.5 years of continued exposure. Given the epidemiologically expected mortality rate, the resulting NNT suggests that treating patients with DCCR may also reduce mortality events."

Clinical • P3 data • Genetic Disorders • Prader–Willi syndrome

Soleno Therapeutics Announces Presentations Featuring VYKAT(TM) XR in Prader-Willi Syndrome at ISPOR 2025 (GlobeNewswire) - "Soleno Therapeutics, Inc...today announced that it will present data on the burden of Prader-Willi syndrome (PWS) and findings from its clinical development program of VYKAT XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia associated PWS at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Meeting 2025, which is being held May 13-16, in Montreal, Canada."

Clinical data • Prader–Willi syndrome

Long-Term Results for Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome: Developmental Behaviour Checklist 2 Response and Relationship to Hyperphagia Reductions (ESPE-ESE 2025) - "Behavioural improvements appeared to be independent of hyperphagia improvements. These hyperphagia and behavioural improvements should benefit patients with PWS and their families."

Clinical • Developmental Disorders • Metabolic Disorders • Mood Disorders • Prader–Willi syndrome • Psychiatry

LogiCare3PL Supports Commercial Launch of Soleno Therapeutics' VYKAT XR Following FDA Approval (PRNewswire) - "LogiCare3PL is proud to support the commercial launch of VYKAT XR, Soleno Therapeutics' newly FDA-approved therapy and the first and only treatment indicated for hyperphagia in patients with Prader-Willi syndrome (PWS). This milestone marks a significant advancement for the PWS community."

Launch US • Prader–Willi syndrome

Diazoxide choline (Vykat XR) for Prader-Willi syndrome-associated hyperphagia. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Prader–Willi syndrome

Soleno Therapeutics Announces VYKAT XR Launch (GlobeNewswire) - "Soleno Therapeutics, Inc...announced the U.S. commercial availability of VYKA XR (diazoxide choline) extended-release tablets, the company’s treatment for hyperphagia in patients four years of age and older with Prader-Willi syndrome (PWS), which was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2025. Prescriptions of VYKAT XR have now been delivered to the first individuals living with PWS who have been prescribed the medication."

Launch US • Prader–Willi syndrome

PANTHERx Rare Selected by Soleno Therapeutics for the Distribution of VYKAT XR (PRNewswire) - "PANTHERx Rare...is pleased to announce that it was selected by Soleno Therapeutics as the specialty pharmacy for the distribution of VYKAT XR (diazoxide choline extended-release tablets), a once daily oral tablet indicated for the treatment of hyperphagia in individuals four years of age and older with Prader-Willi syndrome (PWS). VYKAT XR is the first and only FDA-approved treatment for hyperphagia in individuals with PWS."

Commercial • Genetic Disorders • Prader–Willi syndrome

Soleno Therapeutics Announces U.S. FDA Approval of VYKAT XR to Treat Hyperphagia in Prader-Willi Syndrome (GlobeNewswire) - "Soleno Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has approved VYKAT XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome (PWS). Soleno expects VYKAT XR to be available in the U.S. beginning in April 2025....The FDA approval of VYKAT XR was based on an adequate and well-controlled study and safety data from the comprehensive clinical development program. Efficacy was established during the 16-week randomized withdrawal study period of Study 2-RWP (Study C602-RWP)....The primary safety analyses are based on Study 1 (Study C601) and the most common adverse reactions occurring in greater than or equal to 10% of individuals receiving VYKAT XR and at 2% greater than placebo included hypertrichosis, edema, hyperglycemia and rash."

FDA approval • Launch US • Genetic Disorders • Prader–Willi syndrome

Soleno Therapeutics Enters Into $200 Million Debt Financing with Oxford Finance LLC (GlobeNewswire) - "Soleno Therapeutics...announced it has entered into a loan and security agreement with Oxford Finance LLC and its affiliates (Oxford) for up to $200 million.....Under the terms of the agreement with Oxford, Soleno drew an initial $50 million at closing. $100 million will be available in three additional tranches, contingent upon U.S. Food and Drug Administration (FDA) approval of DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) and certain commercial milestones. The final $50 million may be made available upon the mutual consent of Soleno and Oxford."

Financing • Prader–Willi syndrome

Soleno Therapeutics Announces FDA Extension of Review Period for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome (GlobeNewswire) - "Soleno Therapeutics, Inc...announced that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. The new Prescription Drug User Fee Act (PDUFA) target action date is March 27, 2025."

PDUFA • Prader–Willi syndrome

Long-term Efficacy Results of Diazoxide Choline Extended-Release (DCCR) Tablets in Participants with Prader-Willi Syndrome from the Completed C601 (DESTINY PWS) and C602 Open Label Extension (OLE) Studies (ESPE 2024) - "Long-term administration of DCCR in participants with PWS was associated with statistically significant, clinically meaningful, and durable changes in hyperphagia, behavior measures, clinician/caregiver assessments, lean body mass, and metabolic markers."

Clinical • CNS Disorders • Depression • Metabolic Disorders • Mood Disorders • Prader–Willi syndrome • Psychiatry • LEP

Relaxation of Food Control Parameters Based on Improvements in the Food Safe Zone Questionnaire Occurs with Reduction of Hyperphagia in Clinical Trials of Diazoxide Choline Extended Release (DCCR) in Participants with Prader-Willi Syndrome (ESPE 2024) - "DCCR administration to participants with PWS resulted in durable, meaningful, and statistically significant reductions in hyperphagia in the Phase 3 studies even as food controls were relaxed, easing the burden of caring for patients with PWS."

Clinical • Infectious Disease • Metabolic Disorders • Novel Coronavirus Disease • Prader–Willi syndrome

Soleno Therapeutics Announces Regulatory Update on DCCR (diazoxide choline) Extended-Release Tablets to Treat Prader-Willi Syndrome (GlobeNewswire) - "Soleno Therapeutics, Inc...announced a regulatory update from the U.S. Food and Drug Administration (FDA). The FDA Review Division has determined that there does not appear to be a need for an advisory committee meeting at this time for the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS). However, the Division’s review team will continue to consider the potential need for an advisory committee meeting during their ongoing review."

FDA event • Prader–Willi syndrome

Soleno Therapeutics Announces U.S. FDA Acceptance for Filing and Priority Review of NDA for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome (GlobeNewswire) - "Soleno Therapeutics, Inc...announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for DCCR for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. The FDA granted Priority Review for the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 27, 2024."

FDA filing • PDUFA • Priority review • Genetic Disorders • Prader–Willi syndrome

Soleno Therapeutics Announces Submission of New Drug Application to the U.S. FDA for DCCR (Diazoxide Choline) Extended-Release Tablets for the Treatment of Prader-Willi Syndrome (GlobeNewswire) - "Soleno Therapeutics, Inc...announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia."

FDA filing • Genetic Disorders • Prader–Willi syndrome

Withdrawal of DCCR (Diazoxide Choline) Extended-Release Tablets Worsens Hyperphagia and Increases Weight and BMI in a 16-week Double-blind, Placebo-controlled, Randomized Withdrawal Period in Patients with Prader Willi Syndrome. (ENDO 2024) - "In this 16-week RWP, the Placebo group experienced significant worsening in hyperphagia and increases in body weight, BMI, and BMI z-scores, along with worsening trends for the CGI-S and CGI-I, compared to the DCCR group. DCCR was well tolerated, with a safety profile that was consistent with prior experience. These data suggest treatment with DCCR may lead to clinical benefits in people with PWS by reducing hyperphagia and improving body composition.Unless otherwise noted, all abstracts presented at ENDO must not be released to the press or the public until the date and time of presentation."

Clinical • Endocrine Disorders • Metabolic Disorders • Prader–Willi syndrome

Soleno Therapeutics Receives Breakthrough Therapy Designation from U.S. FDA for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome (PWS) (GlobeNewswire) - "Soleno Therapeutics, Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to diazoxide choline for the treatment of adults and children ages 4 years and older with genetically confirmed Prader-Willi syndrome (PWS) who have hyperphagia. The designation reflects the Agency’s determination that, based on an assessment of the preliminary data from the Phase 3 clinical development program, diazoxide choline may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies....'We remain focused on preparing our NDA submission for DCCR in PWS, which we continue to expect will occur in mid-2024.'"

Breakthrough therapy designation • NDA • Cardiovascular • Prader–Willi syndrome

Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. (PubMed, J Neurodev Disord) - P, P3 | "Long-term administration of DCCR to people with PWS resulted in changes in hyperphagia and other behavioral complications of PWS that are distinct from the natural history of the syndrome as exemplified by the cohort from PATH for PWS. The combined effects of administration of DCCR should reduce the burden of the syndrome on the patient, caregivers and their families, and thereby may benefit people with PWS and their families."

Journal • Endocrine Disorders • Genetic Disorders • Metabolic Disorders • Obesity • Prader–Willi syndrome

Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period (clinicaltrials.gov) - P3 | N=115 | Completed | Sponsor: Soleno Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Prader–Willi syndrome

Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome (clinicaltrials.gov) - P3 | N=83 | Active, not recruiting | Sponsor: Soleno Therapeutics, Inc. | Not yet recruiting ➔ Active, not recruiting

Enrollment closed • Prader–Willi syndrome

Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from long-term open-label study. (PubMed, Obesity (Silver Spring)) - "DCCR administration to people with PWS was well-tolerated and associated with broad-ranging improvements in the syndrome. Sustained administration of DCCR has the potential to reduce disease severity and the burden of care for families."

Journal • Dermatology • Diabetes • Mood Disorders • Prader–Willi syndrome • Psychiatry • LEP