FDA grants breakthrough designation to psilocybin analog for major depressive disorder

Key takeaways:

• The phase 2 trial of CYB003 in major depressive disorder demonstrated statistically significant improvement in depression symptoms at 4 months.
• CYB003 is expected to advance to a phase 3 trial in mid-2024.

The FDA has granted breakthrough therapy designation to Cybin’s CYB003, a proprietary deuterated psilocybin analog in development for treatment of major depressive disorder, according to a company press release.

The designation is supported by positive topline results from Cybin’s phase 2 trial of CYB003 in major depressive disorder (MDD), which demonstrated statistically significant improvement in depression symptoms at 4 months, with 75% of patients in remission from depression after receiving two 16 mg doses. In addition, there was an approximately 22-point reduction from baseline in Montgomery-Asberg Depression Rating Scale score at 4 months among participants receiving 12 mg or 16 doses.

CYB003 was well-tolerated, the release stated, with no drug-related serious adverse events; all adverse events were mild to moderate.

“The granting of breakthrough therapy designation by the FDA underscores the potential of CYB003 to fill a gap in the treatment landscape for MDD and serves to expedite and derisk our development program going forward,” Cybin CEO Doug Drysdale said in the release.

“We are grateful for the opportunity to accelerate the development and regulatory review process that this designation affords, as we prepare to advance CYB003 toward a phase 3 pivotal trial around midyear.”

If approved by the FDA, CYB003 would be the first known adjunctive psychedelic-based therapeutic for the treatment of MDD, the company noted.