Transcend Achieves Phase 2 Success in PTSD Treatment, Boosting Neuroplastogen Class

Transcend Therapeutics has achieved a significant milestone in the treatment of PTSD with its innovative non-hallucinogenic psychostimulant, methylone, also known as TSND-201. The small company, with fewer than 20 employees, has seen success in a mid-stage clinical study, moving it closer to a pivotal Phase 3 trial anticipated to start early next year.

This placebo-controlled study is the first to examine the efficacy of Transcend's TSND-201, which, while not a psychedelic, falls into the broader category of neuroplastogen treatments. These treatments aim to leverage the brain's ability to reorganize and form new neural connections, but without inducing hallucinations.

The study results revealed that TSND-201 outperformed the placebo in alleviating PTSD symptoms, as assessed by a clinical scale. Significant improvements in CAPS-5 scores were observed both shortly after treatment and in the longer term, with notable statistical enhancements at days 10 and 64. Specifically, patients receiving TSND-201 experienced an 8-point improvement over placebo at day 10 and a 9.64-point improvement at day 64. Impressively, nearly 61% of those treated with TSND-201 no longer met the criteria for a PTSD diagnosis, doubling the response rate seen with placebo.

This promising data has instilled a sense of optimism in CEO Blake Mandell, who underscores the scarcity of effective treatments available for PTSD sufferers. Reflecting on the study, Mandell noted the long-standing nature of PTSD symptoms among participants, many of whom had lived with the condition for an average of 20 years. Within just 10 days, a noticeable advantage over placebo was evident.

The trial enrolled 65 individuals with severe PTSD from the US, UK, and Ireland, all of whom had previously received treatment for the condition. Participants were administered four oral doses of either the active treatment or a placebo, with each dose spaced a week apart. The data collected on day 10 came after participants had completed their second treatment session.

Most adverse events were mild, resolving on the day of dosing. Notably, a patient with a history of seizures experienced one a week after the final dose, yet this was judged to be unrelated to the study drug.

Transcend is part of a growing group of biotech companies focused on developing psychedelic-inspired therapies without the associated hallucinogenic effects. Other firms, such as Delix Therapeutics and Gilgamesh, are exploring similar avenues. Despite the non-hallucinogenic nature of TSND-201, patients in the trial were monitored for at least eight hours, with varying experiences reported. Some participants were more talkative, others introspective, and a number experienced a positive side effect described as "non-sedated relaxation."

Mandell emphasized the importance of thorough side effect documentation, not only as a gesture of transparency but also as a learning opportunity from other companies' challenges. Transcend is keen to avoid pitfalls like those encountered by Lykos Therapeutics, which faced FDA rejection despite successful trials, due to issues with data integrity and adverse event monitoring.

To minimize bias, Transcend utilized central raters to assess CAPS-5 scores, ensuring assessments were independent of those conducting the dosing sessions. So far, the company has secured $50 million in funding, sufficient to reach the end of the year and prepare for the Phase 3 trial. Upcoming discussions with the FDA will address the study design, including possible adjustments to monitoring procedures. Transcend aims to attract further investment to support the next stages of its clinical program, with the ultimate goal of offering a new and effective treatment option for PTSD patients.