Consultant Neurologist Dr. Heidi Biernat, Head of the Tourette Clinic at Copenhagen’s Bispebjerg University Hospital and Principal Investigator in Asarina Pharma’s Phase IIa study in Sepranolone for Tourette syndrome, will present a Poster of the study’s positive results at ESSTS (15th European Conference on Tourette Syndrome and Tic Disorders), June 7-9 in Brussels, Belgium.
Dr Biernat was the Principal Investigator in Asarina’s Phase IIa study in Sepranolone for Tourette syndrome at Bispebjerg University Hospital’s Tourette Clinic, where she is responsible for the care of around 450 adult TS patients. Asarina Pharma’s endogenous compound Sepranolone demonstrated competitive tic reduction and improved quality of life while displaying no CNS off-target side effects in the study.
Sepranolone reduced tic severity with 28% in its primary clinical endpoint as measured by YGTSS (the world-standard Yale Global Tic Severity Scale), and achieved positive results in four key secondary endpoints compared with standard of care:
- 69% greater increase of Quality of Life (using the Gilles de la Tourette Syndrome Quality of Life total score (GTS-QOL)
- 50% greater reduction in impairment (YGTSS)
- 44% greater reduction of the premonitory urge to tic (PUTS – the Premonitory Urge to Tic scale)
- no off-target CNS effects or systemic side effects – a crucial metric for CNS drugs in an indication where legacy and new treatments in developments involve sometimes severe and debilitating extra-pyramidal side effects
The Phase IIa trial was an interventional, open-label, multi-center, randomized, parallel group study to explore the safety and efficacy of Sepranolone in patients with TS. Patients were randomized to either standard of care (SoC) or standard of care + injections of Sepranolone 10 mg twice weekly for 12 weeks. 3 adolescents and 25 adults with TS, 12-47 years, 18 males and 8 females, took part. The Poster, co-authored by Dr. Heidi Biernat, Jonathan Myrup Staalsø, Amalie Akulenok Berring-Uldum and Nanette Mol Debes, is titled Promising results on a clinical trial to explore the safety and efficacy of Sepranolone in pediatric and adult patients with Tourette syndrome.
On May 19 2023 the results were presented at the TicCon Virtual Research Symposium of the Tourette Association of America, by Assoc Prof Marco Bortolato in his presentation entitled Putative novel pharmacological treatments for tic disorders.
Dr Heidi Biernat: “The clear positive effect of treatment with Sepranolone is valuable and encouraging. The side effects of the antipsychotics still often being prescribed today are so heavy that very few patients keep taking them for long periods if they can be avoided. So the lack of side effects with Sepranolone was very motivating. I could easily see it becoming the new first-line treatment for Tourette patients who cannot manage their tics with CBIT and who require pharmaceutical treatment. When I see my colleagues try to prescribe for tics they still often have to go back to the old-fashioned antipsychotics, even though patients don’t like and can’t sustain those treatments. Sepranolone gives us a new option. I believe it has a strong future”.
Asarina Pharma CEO Peter Nordkild: “it is very fitting that Dr Biernat and Professor Bortolato present these important results at such key events in the Tourette calendar. We believe these results are an exciting step towards Sepranolone ultimately becoming the first-line pharmaceutical treatment for Tourette Syndrome – with no off-target effects and none of the severe side effects associated with today’s treatments”.
