Transcend Therapeutics Receives Breakthrough Therapy Designation for TSND-201 (methylone) for the Treatment of PTSD

Designation offers potential for expedited development of TSND-201 for the treatment of PTSD

Company planning to initiate Phase 3 program

NEW YORK, July 10, 2025 /PRNewswire/ -- Transcend Therapeutics, a clinical-stage biotechnology company that develops medicines to treat neuropsychiatric diseases, today announced that the U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy designation to TSND-201 (methylone) for the treatment of Post-Traumatic Stress Disorder (PTSD).

Breakthrough Therapy designation is granted if preliminary clinical evidence shows that it may demonstrate substantial improvement over available therapy. The Breakthrough Therapy pathway was designed to potentially expedite development and review timelines for promising investigational medicines for serious or life-threatening conditions.

The Breakthrough Therapy designation is based on the positive results from IMPACT-1, a randomized, placebo-controlled, Phase 2 clinical trial evaluating TSND-201, a rapid-acting neuroplastogen, for the treatment of PTSD. The IMPACT-1 study was a randomized, placebo-controlled Phase 2 trial of 65 patients with severe PTSD. TSND-201 achieved rapid, robust, and durable improvements in PTSD symptoms. Statistically significant and clinically meaningful symptom improvements occurred by Day 10 (-8.00 placebo-adjusted CAPS-5 improvement) with effects sustained through Day 64 (-9.64 placebo-adjusted CAPS-5 improvement). TSND-201 was well tolerated, with no hallucinations or discontinuations due to adverse events.

"This designation underscores the urgent need for new, more effective treatments for the 13 million Americans living with PTSD," said Blake Mandell, co-founder and CEO of Transcend Therapeutics. "Only two FDA-approved medications are available today—and no new PTSD treatments have been approved in over two decades. This Breakthrough Therapy designation enables us to work more closely with the FDA as we prepare to launch our Phase 3 program."

"A rapid-acting, durable treatment for PTSD would be a paradigm shift in psychiatry—and offer a potential lifeline for patients," said Murray B. Stein, M.D., M.P.H., Distinguished Professor of Psychiatry and Public Health at the University of California San Diego and a consultant to Transcend. "The current treatment standard, SSRIs, can take up to 12 weeks to show maximal effect and often cause side effects during that time. Even then, many patients on SSRIs don't get adequate benefit. This designation offers new hope that faster, more effective treatment may be within reach."

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.

About Breakthrough Therapy Designation
Breakthrough Therapy designation is granted by the FDA in order to expedite the development and review of drugs for serious or life-threatening conditions. In order to receive Breakthrough Therapy designation, a drug must demonstrate preliminary clinical evidence that the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. Breakthrough Therapy designation provides an organizational commitment involving senior managers from the FDA, more intensive FDA guidance on an efficient drug development program, and greater access to and more frequent communication with the FDA throughout the entire drug development and review process. It also provides the opportunity to submit sections of a New Drug Application (NDA) on a rolling basis, where the FDA may review portions of the NDA as they are received instead of waiting for the entire NDA submission. In addition, Breakthrough Therapy designated products are eligible for Priority Review, where the FDA has a goal to take action on an application within six months, as opposed to ten months under standard review. Breakthrough Therapy designation does not change the standards for approval.

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.

Contact: [email protected] 

SOURCE Transcend Therapeutics