Saniona Cleared to Continue Phase II Prader-Willi Syndrome Treatment Program

Shares of Denmark-based Saniona are up more than 14 percent on the Stockholm Exchange after the company announced it has been cleared to initiate the second part of a mid-stage trial to develop a treatment for adolescents with Prader-Willi Syndrome.

Saniona is investigating its Tesomet medication for the genetic disease characterized by an extreme and insatiable appetite in patients diagnosed with the disease. Saniona is developing Tesomet as a potential treatment to spur significant weight-loss in PWS patients and reduce hyperphagia, which is the excessive hunger that is a hallmark of the disease.

Prader-Willi Syndrome is a genetic disease that leads to dysfunctional signaling in the brain’s appetite centers, the hypothalamus. Patients with the experience a ravenous insatiable appetite which persists no matter how much the patients eat. Many PWS patients become morbidly obese and suffer significant mortality. Patients with PWS have a shortened life expectancy unless the disease can be well-controlled. Common causes of death in PWS include respiratory disease, cardiac disease, infection, choking, gastric rupture, and pulmonary embolism.

In October 2017 Saniona opted to conduct an interim analysis of its Phase II study before it elected to continue. When it announced its intentions last fall Saniona said nine of the 10 patients in its trial had either completed the dosing or discontinued dosing. The company said, "some patients showed indication of weight loss and reduced food cravings," which were key efficacy endpoints. However, Saniona noted that some patients were forced to discontinue the study due to adverse effects which could have been related to the medication.

Safety concerns forced Boston-based Zafgen, Inc. to shutter its experimental Prader-Willi Syndrome treatment beloranib.

After months of examining the data Saniona its analysis showed Tesomet does provide clinically meaningful weight loss and a significant reduction in hyperphagia. However, the company revealed that patients PWS should be given lower doses of Tesomet compared to other patient groups, the company said.

"We believe that low dose Tesomet could prove to be a compelling treatment for adolescents with this currently untreatable disease. Patients suffering from Prader Willi syndrome currently face debilitating hyperphagia, which has severe consequences that also affect their families and carry a very high cost to payors and the society," Jørgen Drejer, chief executive officer of Saniona said in a statement.

Roman Dvorak, Saniona’s chief medical officer, said that the data from the first part of the study has been "impressive." Not only did Tesomet help PWS patients reduce weight, it also generated a "remarkable reduction in cravings."

As the company looks to initiate the second half of the trial, Saniona said the next phase could potentially include up to 10 adolescents with PWS. After coming to an understanding about dosing, Saniona said PWS patients will receive either 25 mg of Tesomet or placebo. The primary endpoint of the second phase will be a change in body weight over a 12-week period. Secondary endpoints will be examining excessive food cravings, body composition, lipids and other metabolic parameters.

Earlier this month Boston-based Rhythm Pharmaceuticals acquired a pre-clinical program from Takeda Pharmaceuticals for PWS. The company recently saw disappointment as its lead candidate, setmelanotide, failed to help patients reduce their weight during a Phase II study. Rhythm said it will continue to assess opportunities for setmelanotide in PWS and plans to pursue these in parallel with the development of RM-853, the drug it licensed from Takeda. The company said it could see if the two products work well as a combination therapy for PWS patients.

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