Transcend Therapeutics Announces Primary Endpoint Met in IMPACT-1 Phase 2 Study of TSND-201 in PTSD

• Primary endpoint met, with TSND-201 demonstrating statistically significant placebo-adjusted CAPS-5 improvement of -9.64 points on Day 64 (p = 0.011)
• TSND-201 demonstrated rapid improvements, with a statistically significant placebo-adjusted CAPS-5 improvement of -8.00 points on Day 10 (p = 0.012), with placebo-adjusted improvements remaining statistically significantly for the duration of the trial
• Robust and statistically significant improvements were noted across multiple secondary endpoints on Day 64
• TSND-201 was generally safe and well tolerated, with a safety profile consistent with its non-hallucinogenic pharmacology
• Company plans to align with FDA on Phase 3 program for PTSD

NEW YORK, March 31, 2025 /PRNewswire/ -- Transcend Therapeutics, a clinical-stage biotechnology company that develops medicines to treat neuropsychiatric diseases, today announced positive results from the IMPACT-1 study (Investigation of Methylone for Post-Traumatic Stress Disorder [PTSD]). IMPACT-1 was a randomized, double-blind, placebo-controlled, Phase 2 clinical trial evaluating TSND-201 (methylone), a rapid-acting neuroplastogen, for the treatment of PTSD. This study enrolled 65 patients with severe PTSD (Clinician-Administered PTSD Scale for DSM-5 [CAPS-5] ≥ 35), all of whom had previously tried a PTSD treatment, to receive 4 oral doses of TSND-201 or placebo, with each dose separated by one week. Patients were followed for a total of 64 days after the first dose.

On the primary endpoint, change from baseline to Day 64, TSND-201 demonstrated a statistically significant placebo-adjusted CAPS-5 improvement of -9.64 points on Day 64 (-23.28 points vs. -13.64 points; p = 0.011). TSND-201 also demonstrated rapid and statistically significant improvements from baseline compared to placebo on the CAPS-5 total severity score. Starting on Day 10, TSND-201 demonstrated a statistically significant placebo-adjusted CAPS-5 improvement of -8.00 points (-17.83 points vs. -9.83 points; p = 0.012), which was durable throughout the study until Day 64.

"We are encouraged by the positive Phase 2 results demonstrating TSND-201's potential as a rapid-acting and durable treatment for patients with PTSD," said Transcend Therapeutics Co-Founder and CEO Blake Mandell. "We look forward to working with the FDA to align on a Phase 3 program in PTSD."

TSND-201 demonstrated statistically significant improvements on multiple secondary endpoints, including response (≥ 50% improvement from baseline on CAPS-5; 57.1% vs. 19.2%; p = 0.002), remission (≤ 11 points on the CAPS-5; 32.1% vs. 11.5%; p = 0.036), and loss of PTSD diagnosis (60.7% vs. 30.8%; p = 0.014).

Treatment with TSND-201 was generally safe and well tolerated. The majority of adverse events were transient, occurring on the day of dosing and resolving the same day. The most commonly occurring adverse events in the TSND-201 group were headache, decreased appetite, nausea, dizziness, blood pressure increase, dry mouth, insomnia, muscle tightness, and feeling abnormal. One serious adverse event of seizure occurred in the TSND-201 group; the event occurred 7 days after the last dose in a patient with a history of seizure and was considered unrelated to study drug.

"It has been over two decades without a new treatment for PTSD. The results of this study suggest that TSND-201 may represent a significant clinical breakthrough — demonstrating clinically meaningful efficacy with a rapid onset," commented Murray B. Stein, M.D., M.P.H., Distinguished Professor of Psychiatry and Public Health at the University of California San Diego and Staff Psychiatrist at the Veteran Affairs San Diego Healthcare System. "Rapid-acting, episodic treatments could be a paradigm shift in the treatment of PTSD, providing hope for the many patients who have struggled to find effective relief."

About Transcend Therapeutics 
Transcend Therapeutics, founded in 2021, is a clinical-stage, neuroscience-focused company developing rapid-acting treatments for neuropsychiatric diseases. The company's mission is to develop new drugs for the millions of people for whom current psychiatric medicines have not worked. Transcend's leadership team has made pivotal contributions to 14 FDA approvals and $7B in M&A and public company value. Transcend has raised approximately $50M to date.

About TSND-201
TSND-201, a rapid-acting neuroplastogen, is a proprietary formulation of methylone developed by Transcend Therapeutics. TSND-201 has well-characterized primary pharmacology, with its primary site of action at the monoamine transporters, and no activity at 5HT-2a (i.e., not hallucinogenic). Transcend is developing TSND-201 as a rapid-acting and durable treatment for PTSD and other central nervous system (CNS) indications. TSND-201 is an investigational medication.

About PTSD
PTSD is a mental health condition that can occur after experiencing or witnessing a life-threatening or traumatic event. Symptoms may include flashbacks, nightmares, severe anxiety, and uncontrollable thoughts about the event. PTSD impacts approximately 13 million Americans each year and it is estimated that nearly 7% of U.S. adults will be diagnosed with PTSD during their lifetime. Only two medications are FDA-approved for the treatment of PTSD (sertraline and paroxetine), yet their efficacy is modest at best. New treatments for PTSD are urgently needed.

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SOURCE Transcend Therapeutics