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Saniona Receives U.S. FDA Orphan Drug Designation for Tesomet in Prader-Willi Syndrome

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Saniona (OMX: SANION), a clinical stage biopharmaceutical company focused on rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tesomet for the treatment of Prader-Willi syndrome (PWS).

Saniona is preparing to initiate a Phase 2b study of Tesomet in PWS in the first half of this year.

Orphan drug designation is a special status granted by the FDA to medicines and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. The number of patients with PWS is estimated to be between 11,000 and 34,000 in the U.S. and between 17,000 and 50,000 in Europe. Receiving orphan designation qualifies Saniona for certain development benefits, including tax credits, elimination of certain FDA license application fees, and seven years of market exclusivity in the U.S. following approval.

Saniona, Rudolf Baumgartner“There is currently no cure for Prader-Willi syndrome and no medicines approved to address the uncontrollable hunger, or hyperphagia, that characterizes this rare disease,” said Rudolf Baumgartner, M.D., Chief Medical Officer and Head of Clinical Development at Saniona. “Receiving orphan drug designation will help us advance Tesomet as expeditiously and efficiently as possible.”

Saniona previously evaluated Tesomet in a randomized, double-blind, placebo-controlled Phase 2a trial in adults and adolescents with PWS. Adult patients receiving Tesomet achieved a statistically significant reduction in hyperphagia, as well as a clinically meaningful reduction in body weight at a dose of 0.5 mg per day. A smaller study extension in an adolescent population showed that Tesomet appeared to be well tolerated at lower doses (0.125 mg/day and 0.25 mg/day) and suggested dose-dependent effects on weight and hyperphagia.

Saniona is currently planning to initiate a Phase 2b study of Tesomet in PWS in the first half of 2021. In preparation for this study, Saniona has opened an Investigational New Drug (IND) filing with the FDA, has selected the clinical research organization (CRO) that will support the clinical trial, and is in the process of assessing and selecting clinical trial sites in the U.S. and globally. Saniona has also selected the contract manufacturer to produce Tesomet for Phase 2b and Phase 3 clinical trials, and the contract manufacturer is actively working on clinical production. Additionally, Saniona has initiated multiple partnerships with the PWS advocacy community to ensure caregiver and patient feedback is incorporated into the clinical trial process and to provide the community with education on clinical trials.

Saniona is also evaluating Tesomet for the treatment of hypothalamic obesity (HO) and plans to begin a Phase 2b trial in this indication in the first half of this year.

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