MIRA Pharmaceuticals has submitted a Phase 2a protocol to the FDA for Ketamir-2, its oral NMDA receptor modulator, in chemotherapy-induced peripheral neuropathy a condition with no approved therapies.
Written By: Fariha Sameen, PharmD
Reviewed By: Pharmacally Editorial Team
MIRA Pharmaceuticals has submitted a Phase 2a clinical trial protocol to the U.S. Food and Drug Administration (FDA) for Ketamir-2, its investigational oral NMDA receptor modulator, in patients with chemotherapy-induced peripheral neuropathy (CIPN).
The randomized, double-blind, placebo-controlled study will evaluate the safety, tolerability, and efficacy of oral Ketamir-2 in patients with persistent neuropathic pain following chemotherapy, an area of significant unmet medical need.
CIPN Remains a Major Unmet Need
CIPN affects an estimated 30% to 40% of patients treated with neurotoxic chemotherapy. Symptoms such as pain, numbness, tingling, and sensory loss can persist long after treatment ends, significantly affecting daily function and quality of life.
Despite its prevalence, no FDA-approved therapies specifically target CIPN. Current treatment relies on medications such as duloxetine, gabapentin, pregabalin, and tricyclic antidepressants, which often provide limited symptom relief.
Ketamir-2 and the Phase 2a Study
Ketamir-2 is an oral NMDA receptor modulator being developed for chronic neuropathic pain. The NMDA pathway plays a central role in pain signaling and sensitization. Unlike ketamine, which requires intravenous administration and carries dissociative effects and controlled-substance restrictions, Ketamir-2 is administered orally once daily and has been classified by the U.S. Drug Enforcement Administration as a non-scheduled compound.
The Phase 2a study, known as Mira-002, will enroll patients with moderate-to-severe persistent CIPN at a leading U.S. cancer center. Using a three-period crossover design, participants will receive Ketamir-2 at 300 mg, Ketamir-2 at 600 mg, and placebo during separate seven-day treatment periods, each followed by a two-week washout.
The primary endpoint will assess changes in neuropathic pain intensity using the weekly mean 11-point Numeric Rating Scale (NRS). Secondary endpoints include the proportion of patients achieving at least 30% and 50% pain reduction, changes in CIPN symptoms measured by the EORTC QLQ-CIPN20 questionnaire, and assessment of central nervous system pharmacodynamic effects.
Eligible participants must have persistent CIPN for at least three months after chemotherapy, a baseline pain score of four or higher, and clinically stable cancer without active systemic treatment requirements.
Supported by Phase 1 Data
The protocol submission follows completion of the MIRA-001 Phase 1 study in 56 healthy volunteers. The study reported no serious adverse events or dose-limiting toxicities and supported the dose selection and once-daily dosing schedule being evaluated in Phase 2a.
Path Forward
According to company leadership, the crossover study design will help characterize both safety and dose-response activity while maintaining an efficient study size. Pending FDA review, the Phase 2a trial will represent the next key clinical milestone for Ketamir-2 and could support its broader development for neuropathic pain indications.
What This Means for Patients
Cancer survivors with persistent chemotherapy-related nerve pain currently have no FDA-approved treatment specifically for CIPN. If Ketamir-2 demonstrates meaningful pain relief and maintains the favorable safety profile seen in Phase 1 testing, it could offer a convenient oral treatment option for patients who continue to experience pain, numbness, and functional limitations months or years after completing chemotherapy.
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About the Writer
Fariha Sameen, PharmD (LinkedIn), is a clinical pharmacy professional with hands-on experience in patient counselling, medication review, therapeutic monitoring, and clinical documentation across multiple departments. She has experience identifying and assessing drug-related problems and supporting medication safety practices. Her interests include pharmacovigilance, ADR reporting, clinical research, and medical writing focused on clear, evidence-based communication.