Psilocybin Analog for Treatment of Depression Receives FDA Breakthrough Therapy Designation


Cybin’s proprietary deuterated psilocybin analog, CYB003, provided robust and sustained improvements in depression symptoms in a phase 2 study.

Cybin has received breakthrough therapy designation from the FDA for its psilocybin analog (CYB003) therapy for the treatment of major depressive disorder (MDD), the biopharmaceutical company said in a news release.1 The company also announced positive 4-month durability data from a phase 2 trial of CYB003.

CYB003 is a proprietary deuterated psilocybin analog developed by Cybin / Aramyan -

CYB003 is a proprietary deuterated psilocybin analog developed by Cybin / Aramyan -

CYB003 is a proprietary deuterated psilocybin analog developed by Cybin. It is a new chemical entity and is part of a family of molecules called indolamines, which are related to psilocybin. The breakthrough therapy designation was granted based on phase 2 trial data of CYB003 that demonstrated a statistically significant improvement in depression symptoms at 4 months that were superior to approved antidepressants and data on other psychedelics.

Key Takeaways

  • Cybin's psilocybin analog therapy, CYB003, has received breakthrough therapy designation from the FDA for the treatment of major depressive disorder (MDD). This designation underscores the therapy's potential to address unmet needs in MDD treatment.
  • The phase 2 trial of CYB003 demonstrated promising results, with statistically significant improvements in depression symptoms sustained for up to 4 months.
  • CYB003 offers a novel approach to MDD treatment by providing robust and sustained symptom relief with intermittent dosing, without the need for daily medication.

“It is a testament to the hard work and dedication of the entire Cybin team that we have accomplished so much so quickly,” Doug Drysdale, CEO of Cybin said in a release.1 “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 de-risk our development program going forward. This designation provides for a streamlined review process and enhanced engagement with the FDA.”

READ MORE: FDA Grants Breakthrough Therapy Designation to LSD-Based Treatment for Generalized Anxiety Disorder

The phase 2 trial was a randomized, double-blind, placebo-controlled study to assess the safety and tolerability of multiple doses of CYB003 in healthy participants with and without MDD. The study found that 2 doses of either 12 mg or 16 mg of CYB003 provided robust and sustained improvements in depression symptoms. Of the participants, 75% responded after 2 doses of 16 mg.

Additionally, 60% of patients on 12 mg and 75% on 16 mg achieved remission from depression following 2 doses. CYB003 was also found to be well tolerated with no serious adverse events. All adverse events were reported to be either mild or moderate in intensity and there were no discontinuations due to adverse events.

"The sustained reduction in depression symptoms at the four-month mark after just two doses of CYB003 is a critical milestone, that demonstrates the durability of the response, following the rapid improvement in symptoms,” Amir Inamdar, chief medical officer of Cybin, said in a release.1 “It also paves the way for a change in the treatment paradigm for MDD. Unlike currently approved adjunctive treatments which require chronic, daily dosing, CYB003 allows for intermittent dosing without the challenges of withdrawing patients from their existing medications.”

MDD is one of the most common mental health disorders in the United States and impacts over 20 million Americans each year. The condition can severely limit an individuals ability to perform daily functions and major life activities. Around 61% of adults in the US received treatment for MDD in 2021, highlighting the need for more and novel therapies.2 If approved by the FDA, CYB003 would be the first known adjunctive psychedelic-based therapeutic for the treatment of MDD.

Cybin said that the new data supports progression to a pivotal phase 3 study of CYB003, which the company plans to begin later this year.

“Currently available standard treatments for MDD can be limited in efficacy, remission and response rates, presenting challenges for patients and mental health practitioners alike,” Maurizio Fava, MD, chair of the Department of Psychiatry and psychiatrist-in-chief at Massachusetts General Hospital, said in a release.1 “CYB003 may have potential to address these challenges, and with the FDA’s Breakthrough Therapy Designation, the regulatory path forward is accelerated.”

READ MORE: Mental and Behavioral Health Resource Center

1. Cybin Receives FDA Breakthrough Therapy Designation for its Novel Psychedelic Molecule CYB003 and Announces Positive Four-Month Durability Data in Major Depressive Disorder. News Release. Cybin. March 13, 2024. Accessed March 13, 2024.
2. Major Depression. Report. NIH. July 2023. Accessed March 13, 2024.
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