Clinical Trials Advance as Tesomet Granted FDA’s Orphan Drug Status

Tesomet, a potential treatment for Prader-Willi syndrome (PWS), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA).

Saniona, the therapy’s maker, now plans to initiate a Phase 2b study of Tesomet in the first half of this year.

“Receiving orphan drug designation will help us advance Tesomet as expeditiously and efficiently as possible,” Rudolf Baumgartner, MD, Saniona’s chief medical officer, said in a press release.

Orphan drug designation qualifies treatment developers for a number of benefits and incentives — such as certain tax credits and fee waivers — meant to help develop a potential therapy quickly. It also confers seven years of market exclusivity upon approval.

Therapies meant to treat disorders affecting fewer than 200,000 people in the U.S. may qualify for orphan drug status.

Saniona recently completed a Phase 2a trial (NCT03149445) of Tesomet’s safety and efficacy in adolescents and adults with PWS.

Patients receiving once-daily Tesomet in this study lost weight and experienced less hyperphagia (excessive hunger) compared to those receiving a placebo.

A 0.25 mg dose caused a 69% decrease in hyperphagia over three months, while a 0.5 mg daily dose resulted in a clinically meaningful reduction in body weight.

A smaller extension study among adolescents indicated that Tesomet was well-tolerated at lower daily doses of 0.125 mg and 0.25 mg and that it might have dose-dependent effects on both weight and hyperphagia.

As a requirement for the planned Phase 2b trial, Saniona has opened an investigational new drug application with the FDA. Also, the company has chosen the clinical research organization to conduct the study. It is also currently scouting potential trial sites in the U.S. and abroad.

In an effort to incorporate patient and caregiver feedback in clinical trials, and to help educate the PWS community on such studies, Saniona has forged several partnerships within the PWS advocacy community.

Because Tesomet is a new type of molecule, the FDA recently advised Saniona to conduct a Phase 3 study to confirm the compound’s safety and efficacy.

Tesomet’s active ingredients — the ones with therapeutic effect — are tesofensine and metoprolol.

Tesofensine, found to lower weight after first being developed as a treatment for neurodegenerative diseases, prevents nerve cells from reabsorbing the neurotransmitters (chemical messengers) dopamine, serotonin, and noradrenaline. Because this can induce an abnormal heart rate, developers combined it with metroprolol, which lowers blood pressure and improves circulation.

Saniona is also investigating Tesomet’s usefulness in treating hypothalamic obesity. The company is planning a Phase 2b trial for this condition, also to start in the first half of this year.