Psychedelic biotechs near Phase 3
Several biotech companies focused on psychedelic therapeutics are advancing to Phase 3 trials for treating central nervous system disorders. The progress in developing treatments for conditions like resistant depression and anxiety signals growing regulatory and medical acceptance of these novel mental health modalities.
The U.S. Food and Drug Administration (FDA) has granted "Breakthrough Therapy" designation to several psychedelic compounds, a status designed to expedite the development of drugs that may offer substantial improvement over existing therapies. This includes psilocybin for treatment-resistant depression and major depressive disorder, an LSD formulation for generalized anxiety disorder, and MDMA for post-traumatic stress disorder. Compass Pathways is advancing its synthetic psilocybin candidate, COMP360, for treatment-resistant depression. In its larger Phase 3 trial with 581 participants, a 25 mg dose showed a statistically significant reduction in depression severity. The company plans to file for FDA approval by the end of 2026. Helus Pharma, formerly known as Cybin, is developing HLP003, a proprietary oral psilocybin compound for major depressive disorder. Phase 2 data for their lead asset showed a 71% remission rate at 12 months after two doses, and topline data from its pivotal Phase 3 study is anticipated in the fourth quarter of 2026. Definium Therapeutics, which rebranded from MindMed, is focusing on an LSD-derived tablet, DT120, for generalized anxiety disorder. The company has three Phase 3 trial results expected in 2026 for this candidate, which has also received FDA Breakthrough Therapy Designation. This new wave of psychedelic research follows pioneering studies in the mid-20th century that were largely halted by legal restrictions. Renewed interest from institutions like Johns Hopkins and Imperial College London helped re-establish the therapeutic potential of these compounds, leading to the current late-stage clinical trials. Investor interest in the sector has grown, with venture capital funding for psychedelic-focused startups increasing significantly. After a surge of over $2.5 billion between 2020 and 2022, investment strategies are now more focused on a select group of companies with late-stage clinical data. Despite the clinical progress, regulatory frameworks are still evolving. While jurisdictions like Oregon have legalized psilocybin for therapeutic use, and others have decriminalized it, these substances remain classified as Schedule I at the federal level in the United States.
