Sepranolone

Sepranolone is a phase 2b-ready neurosteroid being developed for Tourette syndrome and other compulsion-related conditions.\n After sharing plans to shutter last summer, Asarina Pharma has managed to sell off its Tourette syndrome candidate to Relmada Therapeutics, another struggling biotech.Relmada, which has turned to strategic options after scrapping two phase 3 trials in major depressive disorder (MDD), is paying 2.75 million euros ($2.86 million) for full global rights to Asarina’s midstage candidate, dubbed sepranolone.The purchase includes a $250,000 credit from a previous payment Relmada made for an exclusivity agreement related to the asset, representing a total purchase price of 3 million euros ($3.1 million), according to a Feb. 6 Securities and Exchange Commission filing.Sepranolone is a phase 2b-ready neurosteroid—also known as a neuroactive steroid—being developed for Tourette syndrome and other compulsion-related conditions, such as obsessive-compulsive disorder. Swedish company Asarina revealed in April 2023 that the therapy reduced tic severity at 12 weeks by 28% according to a common rating scale of disease severity called the Yale Global Tic Severity Scale, compared to 12.6% in patients with Tourette’s who received standard of care. The open-label phase 2a study also hit key secondary endpoints, including improving quality of life, and there were no systemic side effects observed.  “While current treatments for Tourette syndrome provide only modest tic reduction and are often accompanied by significant side effects, the phase 2a data suggest that sepranolone has the potential to offer meaningful symptom relief with a more favorable safety profile,” Luca Pani, M.D., a professor of clinical psychiatry at the University of Miami’s Miller School of Medicine and chief advisor for Relmada, said in a Feb. 6 press release.Relmada plans to hold an investor event about its next steps for sepranolone later this year, according to the release. The Florida-based biotech will also continue exploring other strategic opportunities. The company began searching for options at the end of last year, coming when a data monitoring committee found that one of its late-stage trials in MDD would likely miss its main goal. The biotech then scrapped two trials and began considering a range of options, including a merger, buying or selling assets, or selling the company entirely. As for Asarina, the company worked for more than a year to get someone to snap up sepranolone. With no takers emerging by June 2024, the biotech asked shareholders to vote to liquidate, with the move approved in August. In October, the Swedish biotech delisted from the Nasdaq market in Stockholm.

The therapy has shown potential in improving the symptoms of Tourette’s syndrome in a Phase IIa study. Credit: Jack_the_sparow/Shutterstock. Relmada Therapeutics has acquired complete worldwide ownership rights to Sepranolone from Sweden-based Asarina Pharma. Sepranolone is a neurosteroid being developed as a potential treatment for Tourette’s syndrome (TS) and other compulsive disorders. TS is a complex neurological condition characterised by involuntary tics. The asset purchase agreement between the two companies is valued at €3m ($3.12m). The therapy has demonstrated a favourable safety profile in clinical neuro/hormonal trials with more than 335 subjects. Relmada Therapeutics CEO Sergio Traversa stated: “We are very pleased to announce this agreement with Asarina. Sepranolone aligns with our company’s mission to find solutions for difficult-to-treat central nervous system (CNS) disorders. There is a serious unmet need for improved TS therapies – not only [in] reducing tics but also improving quality of life without serious side effects. “We have been impressed by the encouraging Phase IIa efficacy signal with clinically meaningful tic reductions and quality-of-life improvements, combined with a robust safety data package showing no CNS off-target effects, that we believe reflect the compound’s selective binding properties.” As a first-in-class GABAA-modulating steroid antagonist (GAMSA), Sepranolone works by normalising the activity of the GABAA receptor by targeting alpha-2 and alpha-4, the receptor subtypes. This mechanism avoids direct interference with GABAA signalling, offering a selective treatment approach for TS and associated disorders. The therapy is under the protection of several issued patents extended until 2038. The therapy has shown potential in improving TS symptoms in an open-label Phase IIa study compared to standard of care alone. The changes were measured by the Yale Global Tic Severity Scale (YGTSS) scoring system, compared to the baseline. In August 2022, the US Food and Drug Administration granted fast-track status to Relmada’s REL-1017 as a single agent for treating major depressive disorder.