This week, biotechnology company Saniona was able to communicate positive topline results from the open label extension of the phase II study with Tesomet in hypothalamic obesity. This is encouraging news ahead of the upcoming supportive phase IIb study that the FDA has called for. Yesterday, the company held a webcast where the results – which show statistically significant and clinically meaningful reductions in body weight and waist circumference as well as improved glycemic control – were presented. A replay of the webcast is available here.
Saniona, a clinical phase company that focuses on the development of drugs for rare diseases, was yesterday able to strengthen its case for the wholly owned drug candidate Tesomet that is developed for the treatment of the rare condition hypothalamic obesity (HO).
Positive topline results
HO is characterised by a severe and debilitating obesity that is often accompanied by depression, impulse control disorders, as well as an increased risk of cardiovascular disease and type 2 diabetes. The topline results from the phase II study in HO showed that Tesomet was well tolerated by the patients during the 48-week study and that no clinically significant differences could be noted regarding heart rate or blood pressure. All patients completed the open-label extension study.
Specifically, the results showed statistically significant and clinically meaningful reductions in body weight and waist circumference from baseline to week 48, as well as improved glycemic control. The improvements observed during the first double-blind part of the study were maintained throughout the later open-label extension study.
The patients who received placebo during the double-blind part of the study and then Tesomet during the open-label extension study, also demonstrated reductions in body weight and waist circumference after the transition to Tesomet.
»The results of the open-label extension study reinforce the promising profile of Tesomet observed in the placebo-controlled portion of the Phase 2 study in patients with hypothalamic obesity. If approved, Tesomet could be the first treatment designed to address this rare disease. We look forward to continuing our discussions with the FDA and clarifying the path to bring Tesomet to the HO patients who desperately need treatment options« – Rudolf Baumgartner, CMO and Head of Clinical Development Saniona
A disease with a significant medical need
The results show Tesomet’s potential to manage several important symptoms of the disease, i.e. both the weight gain and the reduced metabolism that can lead to serious diabetes complications.
As there are currently no approved drugs for the treatment of HO, patients are referred to ineffective alternatives for weight loss such as surgery, medication and lifestyle counselling. This means that the medical need for new treatments is significant, and that Saniona – if Tesomet reaches the market – gets an exclusive position with a first-mover advantage.
In summary, this is an important milestone for Saniona. The company has proof-of-concept for Tesomet in two serious rare diseases, hypothalamic obesity and Prader Willi syndrome, which the candidate also is developed for. With each new central milestone, the total development risk is statistically reduced as they have several common denominators, such as a rapid and severe weight gain with increased risk of cardiovascular disease and metabolic disorders. This opens up a broader market opportunity for the company.
What happens next?
Yesterday’s positive topline results from the phase II study in HO were welcome after the pre-IND announcement in October from the FDA. As Tesomet is a new molecular entity, the FDA recommended Saniona to implement a supportive phase IIb study before initiating a phase III study to determine the safety and efficacy of the doses intended to be commercialised.
The authority also expressed concerns about an unintended use in the general obese population and suggested that this should be evaluated – for example, through a study of cardiovascular outcome – or that Saniona should present a plan to restrict use to the appropriate rare disease patient population. A plan for how these issues will be addressed is likely now being solidified in the Boston-based company.
In the press release, Saniona emphasises that it intends to present and/or publish additional data from the study in an appropriate peer-reviewed scientific forum. The company further intend to make this data available to the FDA in the ongoing communication on plans to ensure that Tesomet, if approved, is only given to patients for whom it is suitable. Pending this coordination with the FDA, Saniona intends to launch a phase IIb study in HO during the first half of 2021.
The results were presented in a webcast
For those who wish to obtain a deeper analysis of the company’s topline results, we suggest following the replay of Saniona’s webcast available via this link. The replay is also available through the company’s web site.
The presentation was given by Professor Ulla Feldt-Rasmussen, M.D., DMSc. from the Department of Medical Endocrinology and Metabolism at Rigshospitalet, Copenhagen University Hospital, who is the principal investigator on the phase II study.
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