The Phase IIa results demonstrated that Sepranolone could become the first-line pharma treatment for Tourette as it showed competitive tic reduction and improved quality of life while displaying no CNS off-target effects. Sepranolone not only reduced tic severity in its primary clinical endpoint as measured by YGTSS (the world-standard Yale Global Tic Severity Scale) – but also 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 side effects

Assoc Prof Bortolato will present at 9.30 PM CET on May 19 in TicCon’s Virtual Research Symposium. The presentation is entitled Putative novel pharmacological treatments for tic disorders. Register for the event here: https://touretteconference.org/en/registration

Asarina Pharma CEO Peter Nordkild: “we are excited that Professor Bortolato will be presenting these important results at such a key event 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.”