Drug Development
Our commitment to quality and adherence to rigorous standards have led to breakthrough success, positioning Usona Institute at the forefront of psychedelic research and clinical development.
Psilocybin Program
In 2023, Usona's Psilocybin Program continued to make significant strides. Building upon the successful completion of our Phase 2 study (PSIL201) that culminated with strong data published in Journal of the American Medical Association (JAMA), we are preparing to launch a Phase 3 study (PSIL301) in early 2024. This brings us a considerable step closer to submitting our New Drug Application (NDA) and to effectively reach people in need with new therapeutic options for which current treatments fall short. Our focused attention on the rigorous requirements in quality, manufacturing, and regulatory affairs, are essential elements behind these advancements.
5 Key Takeaways from PSIL201 Study
The study’s patient population was—on average—severely depressed, but had low rates of treatment resistant depression, with most participants not on an antidepressant at study entry.
A single 25 mg dose of psilocybin, administered with psychosocial support to patients with Major Depressive Disorder (MDD) produced a rapid, and sustained antidepressant response compared to a niacin placebo (58% response at 6 weeks and 44% remission).
The induction of a mystical experience on dosing day strongly predicted long-term antidepressant response.
Psilocybin was well tolerated with most side effects occurring on the day of dosing. No serious treatment emergent adverse events occurred.
Findings suggest that psilocybin may produce a larger antidepressant effect in depressed patients without treatment resistance.
Read PSIL201 Data Summary in JAMA
Single-Dose Psilocybin Treatment for Major Depressive Disorder
“We are pleased about the results from PSIL201 which provide further support for psilocybin’s potential as a paradigm-changing treatment for people suffering from major depressive disorder.”
Mike Davis, MD – Usona Institute Chief Medical Officer
Key Accomplishments:
Completed End of Phase 2 (EOP2) meeting with the FDA
Published Phase 2 Clinical Study Results in JAMA
Mobilized team and designed the Phase 3 psilocybin study, PSIL301
Re-engaged with the European Medicines Agency (EMA) for scientific advice and to explore the potential for a global regulatory approval pathway
Initiated a focused initiative to include VA sites in Phase 3 to address the needs of the veteran population
Looking Ahead:
Completion of Phase 3 facilitator training and site mobilization
Successful launch of Phase 3 study, including VA sites
Engage with EMA and other global regulatory jurisdictions to evaluate potential for global regulatory approval
Clinical Development Progress to Date
Early Commercialization and Market Access
Early commercialization and market access planning will be essential to a timely and effective launch. Concurrent with our Phase 3 study, we are proactively preparing for market access to coincide with future NDA approval. Our focus is on developing patient-centric delivery models, especially to address demographics and areas with limited access to healthcare and reimbursement options.
Some of the key areas that we are focusing on include:
Stakeholder engagement, including payor and providers, patient advocacy groups, and KOLs
Infrastructure and Supply Chain Development
Training and Education
CMC Progress: Psilocybin
The Usona Chemistry, Manufacturing, and Controls (CMC) team plays a crucial role in our drug development efforts, concentrating on maintaining high-quality standards and reliability in psilocybin production. Throughout 2023, Usona CMC has accomplished significant achievements.
Key Accomplishments:
Developed a dissolution method to assess psilocybin quality
Ensured consistency and effectiveness by identifying key quality criteria for the Active Pharmaceutical Ingredient (API) before encapsulation
Achieved a record production amount during the Process Performance Qualification (PPQ) batches
Uncovered a consistent method for reliable and scalable psilocybin production for current and future needs
Looking Ahead:
Collaborate with manufacturing partners to solidify commercial-scale capabilities and establish a reliable and resilient global supply source
Support the timely launch of Phase 3 with commercial-ready drug product
5-MeO-DMT Program
The 5-MeO-DMT Program at Usona achieved major milestones in 2023. We successfully completed the first-in-human Phase 1 safety study, reinforcing our commitment to exploring new frontiers in psychedelic research.
Key Accomplishments:
Completed the Phase 1 safety study for 5-MeO-DMT
Trained and engaged closely with the clinical study teams, ensuring rigorous conduct of our research
Convened planning sessions with key subject matter experts to prepare for future Phase 2 efficacy studies
Clinical Development Progress to Date
Looking Ahead:
Complete Phase 1 data analysis and align with the FDA on plans for Phase 2 study design
Mobilize sites, educate facilitators, and launch of Phase 2 study
Prepare drug product suitable for Phase 2 studies
