EC5026 (STEP Study) Phase 1 Parkinson's Disease Trial (NCT07142044)

Overview

EC5026 is a fatty acid amide hydrolase (FAAH) inhibitor being developed by EicOsis for the treatment of Parkinson's disease. The STEP Study (NCT07142044) is a Phase 1 clinical trial evaluating the safety, tolerability, and pharmacokinetics of EC5026 in patients with Parkinson's disease["eicosis2024"].

FAAH inhibition represents a novel approach to Parkinson's disease therapy by modulating the endocannabinoid system, which plays important roles in neuroprotection, neuroinflammation, and motor control["faahpd"].

Trial Details

Key Information

Overview

EC5026 is a fatty acid amide hydrolase (FAAH) inhibitor being developed by EicOsis for the treatment of Parkinson's disease. The STEP Study (NCT07142044) is a Phase 1 clinical trial evaluating the safety, tolerability, and pharmacokinetics of EC5026 in patients with Parkinson's disease["eicosis2024"].

FAAH inhibition represents a novel approach to Parkinson's disease therapy by modulating the endocannabinoid system, which plays important roles in neuroprotection, neuroinflammation, and motor control["faahpd"].

Trial Details

Key Information

| Parameter | Value |
|-----------|-------|
| NCT Number | NCT07142044 |
| Trial Name | STEP Study (EC5026) |
| Phase | Phase 1 |
| Status | Recruiting |
| Sponsor | EicOsis, Inc. |
| Indication | Parkinson's Disease |
| Intervention | EC5026 (oral) |
| Comparator | Placebo |
| Study Start | 2025 |
| Estimated Completion | 2026 |
| Enrollment | ~40-60 patients |

Study Design

The trial employs a randomized, double-blind, placebo-controlled design:

• Allocation: Randomized (escalating dose cohorts)
• Intervention: EC5026 oral tablets
• Control: Matching placebo tablets
• Duration: Multiple ascending dose (MAD) cohorts
• Follow-up: 4-week safety follow-up

Mechanism of Action

FAAH Inhibition

Fatty acid amide hydrolase (FAAH) is the primary enzyme responsible for degrading endogenous cannabinoids (endocannabinoids) such as anandamide. By inhibiting FAAH, EC5026 increases anandamide levels in the brain, which:

Therapeutic Rationale

Parkinson's disease involves progressive degeneration of dopaminergic neurons in the substantia nigra, leading to motor symptoms. FAAH inhibition may address multiple aspects of PD pathophysiology:

• Neuroprotection: Enhanced endocannabinoid signaling may protect remaining dopaminergic neurons from degeneration
• Anti-inflammatory Effects: Reduced neuroinflammation could slow disease progression
• Motor Symptom Modulation: CB1 receptor modulation may improve motor function

Inclusion Criteria

• Age 40-75 years
• Diagnosis of idiopathic Parkinson's disease (UK Brain Bank criteria)
• Disease duration 1-15 years
• Hoehn & Yahr stage 1-3
• On stable PD medication regimen for ≥4 weeks (if receiving treatment)
• MDS-UPDRS Part 3 score ≥10
• MMSE score ≥24
• Able to swallow tablets
• Willing to comply with study procedures
• Written informed consent

Exclusion Criteria

• Atypical parkinsonism (PSP, MSA, CBS)
• Significant cognitive impairment (MMSE <24)
• History of stroke or significant neurological disease
• Active malignancy
• Severe medical conditions (cardiovascular, hepatic, renal)
• Prior FAAH inhibitor therapy
• Known hypersensitivity to EC5026
• Pregnancy or breastfeeding
• Current participation in other clinical trials
• Substance abuse disorder

Endpoints

Primary Endpoints

Secondary Endpoints

Exploratory Endpoints

• Biomarker analysis (inflammatory markers)
• CSF sampling (in selected cohorts)
• Neuroimaging substudy

Study Population

Target Population

Early to mid-stage Parkinson's disease patients who:

• Meet clinical diagnostic criteria for idiopathic PD
• Have no significant comorbidities
• Are able to complete all study assessments

Sample Size

The Phase 1 trial will enroll approximately 40-60 healthy volunteers and PD patients across multiple dose escalation cohorts.

Safety Profile

Expected Profile

Based on preclinical data and known FAAH inhibitor class effects:

• Common: Gastrointestinal symptoms (mild nausea, abdominal discomfort)
• Potential: Dizziness, fatigue
• Class Effects: Some FAAH inhibitors have shown hepatotoxicity in long-term use — close monitoring required

Monitoring

• Vital signs
• Laboratory tests (CBC, chemistry, liver function)
• ECG monitoring
• Adverse event assessment

Clinical Significance

Novel Mechanism

EC5026 represents a disease-modifying approach through FAAH inhibition:

Comparison to Other PD Therapies

| Therapy | Type | Route | Phase | Target |
|---------|------|-------|-------|--------|
| EC5026 | Small molecule | Oral | Phase 1 | FAAH (endocannabinoid) |
| Buntanetap | Small molecule | Oral | Phase 3 | Alpha-synuclein aggregation |
| Exenatide | GLP-1 agonist | Injection | Phase 3 | Neuroprotection |
| LRRK2 inhibitors | Small molecule | Oral | Phase 2 | LRRK2 kinase |

Challenges and Considerations

• Phase 1 Stage: Early development — safety profile still being established
• Mechanism Validation: FAAH inhibition efficacy in PD requires clinical confirmation
• Long-term Effects: Unknown if chronic FAAH inhibition is safe
• Therapeutic Window: Optimal dosing needs to be determined

Current Status

As of the latest available information:

• Status: Recruiting
• Locations: Multiple clinical trial sites (check ClinicalTrials.gov for details)
• Updates: Check ClinicalTrials.gov NCT07142044 for current enrollment status

Related Pages

• [EicOsis company](/diseases/mpan)
• [FAAH Inhibitor Therapy](/genes/th)
• [Endocannabinoid System](/mechanisms/endocannabinoid-system)
• [Parkinson's Disease](/diseases/parkinsons-disease)

External Links

• [ClinicalTrials.gov: NCT07142044](/diseases/amyotrophic-lateral-sclerosis)
• EicOsis Official Website
• [Michael J. Fox Foundation](/institutions/michael-j-fox-foundation)
• [Parkinson's Foundation](/institutions/parkinsons-foundation)

References