Psychedelic Biotechs Approach Key Milestone

The companies at the forefront include Compass Pathways with its psilocybin-based therapy, COMP360, for treatment-resistant depression (TRD), and Definium Therapeutics (formerly MindMed), which is targeting generalized anxiety disorder with an LSD-derived compound. Other key players are GH Research, developing an inhaled 5-MeO-DMT treatment for TRD, and Helus Pharma, which is advancing a proprietary oral compound for major depressive disorder. These efforts have attracted significant capital; AtaiBeckley is backed by billionaire investor Peter Thiel, and GH Research raised $160 million in its 2021 IPO. Many of these biotechs have rebranded, like Helus Pharma (formerly Cybin) and Definium Therapeutics (formerly MindMed), to shift from a research focus to a commercial-ready pharmaceutical operation. The compounds under investigation, such as psilocybin and LSD, are classified as Schedule I substances under the federal Controlled Substances Act, which designates them as having no accepted medical use and a high potential for abuse. This classification creates significant regulatory hurdles for researchers and developers. Despite their Schedule I status, the FDA has granted "Breakthrough Therapy Designation" to several of these treatments, including Definium's DT120 for generalized anxiety disorder. This designation is intended to expedite the development and review of drugs that show substantial improvement over available therapies. The treatment model for these compounds is not a simple take-home pill. It typically involves psychedelic-assisted therapy, where a single dose is administered in a clinical setting with psychological support from trained therapists before, during, and after the experience. Following the successful completion of two large-scale Phase 3 trials, a company can submit a New Drug Application to the FDA. The agency's standard review time for such an application is 10 months, though a priority review can shorten this to 6 months.