December 28, 2021
Saniona (OMX: SANION), a clinic–leading biopharmaceutical company focused on rare diseases, announced today initiation of a phase 2b clinical trial of Tesomet in patients with Prader-Willi syndrome (PWS). Tesomet is an experimental fixed-dose therapeutic combination of tesofensin, a triple monoamine reuptake inhibitor, and metoprolol, a selective beta-1 blocker. Data from the trial is expected in the first half of 2023.
“The launch of this Phase 2b clinical trial of Tesomet in Prader-Willi syndrome follows our recent launch of a Phase 2b clinical trial of Tesomet in hypothalamic obesity – two serious conditions with high unmet need “said Rudolf Baumgartner, MD, medical director. and head of clinical development at Saniona. “In addition to these two trials, we are also conducting a Phase 1 clinical trial of SAN711, which means Saniona ends 2021 with three ongoing clinical trials. This is a significant achievement that represents the hard work and expertise of our clinical, regulatory, technical and quality teams.
The Phase 2b clinical trial in PWS consists of a 16-week, randomized, double-blind, placebo-controlled treatment period followed by a 36-week open label extension period. The trial is expected to enroll approximately 120 patients with genetically confirmed PWS. Initially, the trial will recruit adults (18 to 65 years) then, after confirmation by the data oversight committee and the FDA, it is planned to extend the trial to adolescents (13 to 17 years) . During the 16-week double-blind period, participants will be randomized to receive a daily dose of Tesomet at one of three dose levels or a placebo. During the 36-week open-label extension period, participants who wish to continue treatment, including those who initially received a placebo, will receive the highest tolerated dose of Tesomet as established during the period. double blind. The primary focus of the study will be the change in binge eating at week 16, as measured by the Binge Eating Questionnaire for Clinical Trials (HQ-CT), a caregiver-reported survey that assesses behavior foraging, such as how often you eat or how much time you spend talking about food, which has been used as the primary endpoint for most clinical trials in PWS. Secondary endpoints include change in body weight, change in caregiver’s perception of binge eating, change in clinician’s perception of overall disease severity, and change in patient’s impression of overeating. clinician of the overall clinical condition.
The clinical trial is being conducted at multiple sites around the world, including the United States, New Zealand, Australia, and several European countries including the United Kingdom, Sweden, Italy, Spain and others. More information is available at www.praderwillisyndromestudy.com or www.clinicaltrials.gov.
“Currently, there is no approved treatment for binge eating, the uncontrollable hunger that is one of the most debilitating symptoms of PWS,” said Paige Rivard, CEO of Prader-Willi Syndrome Association USA. “We are encouraged to see Saniona treat binge eating, and we look forward to working with them to support this clinical trial through education and awareness within our community. “
“As a medical scientist with over 40 years of experience in clinical practice, I have conducted extensive research on PWS and cared for many patients and families with this disorder,” said Merlin G. Butler, MD, Ph.D., Professor of Psychiatry, Behavioral Sciences, and Pediatrics, and Director of the Research and Genetics Division and the KUMC Genetics Clinic at the University of Kansas Medical Center. “The complex presentation of PWS has made it a difficult target for drug developers, but I am encouraged by the initial data from Tesomet and look forward to the data from this clinical trial. “
Saniona previously evaluated Tesomet in an initial phase 2, randomized, double-blind, placebo-controlled trial in adults and adolescents with PWS. Adult patients receiving Tesomet achieved a statistically significant reduction in binge eating, as well as a reduction in body weight. Adolescent patients demonstrated dose-dependent reductions in binge eating and body weight in the open extensions of the study. Tesomet received orphan drug designation in the PWS from the FDA, which also confirmed that Tesomet can be advanced via the 505 (b) (2) regulatory pathway. Saniona works in partnership with the Foundation for Prader-Willi Research (FPWR), the Prader-Willi Syndrome Association USA (PWSA-USA) and the International Prader-Willi Syndrome Organization (IPSWO) to inform clinical trial design and processes development, and to raise awareness in the PWS community.
Saniona is also evaluating Tesomet in a Phase 2b clinical trial for hypothalamic obesity (HO). The FDA granted orphan drug designation to Tesomet as HO in July 2021.
For more information please contact
Trista Morrison, Director of Communications, Saniona. Office: +1 (781) 810-9227. Email: [email protected]
Informationhas been submitted for publication, through the contact person indicated above, to 17:00 HEC on 28 December 2021.
Saniona is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies for patients with rare diseases for which there is a lack of available treatment options. The company’s flagship product candidate, Tesomet, is in mid-stage clinical trials for hypothalamic obesity and Prader-Willi syndrome, rare serious disorders characterized by severe weight gain, impaired metabolic function and severe disease. uncontrollable hunger. Saniona has developed a proprietary ion channel drug discovery engine anchored in IONBASE ™, a database of over 130,000 compounds, of which over 20,000 are proprietary ion channel modulators from Saniona. With its expertise in ion channels, Saniona advances two wholly owned ion channel modulators, SAN711 and SAN903. SAN711 is in a Phase 1 clinical trial and may be applicable in the treatment of rare neuropathic disorders, and SAN903 is in preclinical development for rare inflammatory, fibrotic and hematologic disorders. Led by an experienced scientific and operational team, Saniona has an established research organization in the Copenhagen area, Denmark, and headquarters in the Boston area, Massachusetts, United States. The company’s shares are listed on the Nasdaq Stockholm Small Cap (OMX: SANION). Learn more at http://www.saniona.com.
Tesomet is an experimental fixed-dose combination of tesofensin (a triple monoamine reuptake inhibitor) and
metoprolol (a selective beta blocker). Saniona Advances Tesomet for Hypothalamic Obesity and Prader-Willi
syndrome, two rare serious disorders characterized by severe weight gain, impaired metabolic functions and uncontrollable hunger. The programs are currently in clinical development. Saniona owns the global rights to Tesomet and is actively evaluating opportunities to advance this treatment globally.
About Prader-Willi Syndrome (PWS)
Prader-Willi syndrome (PWS) is a rare genetic disorder that causes a variety of symptoms. Patients often suffer from binge eating, uncontrollable hunger, and may display abnormal foraging behavior, such as stealing food. Additional symptoms include abnormal growth and body composition; low muscle tone or hypotonia; and social, emotional or cognitive deficits. Binge eating is reported by caregivers as one of the most worrying aspects of PWS, as this insatiable hunger persists regardless of how much food patients consume and often forces caregivers to put locks on refrigerators and cabinets. food storage areas. Many people with PWS become obese and suffer from shortened life expectancy and significant mortality. Common causes of death in PWS include respiratory and heart failure, infection, suffocation, gastric rupture, and pulmonary embolism. There are no drugs approved specifically for the binge eating disorder associated with PWS, and there is no cure for this condition. Current treatment is symptom-dependent and often includes hormone replacement therapy, including growth hormone. It is estimated that PWS will affect between 11,000 and 34,000 patients in the United States and between 17,000 and 50,000 patients in Europe.
Tesomet Ph2b trial launch press release PWS_12.28.21