MIRA Pharmaceuticals submits Ketamir-2 Phase 2a protocol for CIPN
MIRA Pharmaceuticals has submitted its Phase 2a protocol for Ketamir-2 to an institutional review board (IRB) at an unnamed leading cancer research institution, following incorporation of feedback received from the FDA. The NASDAQ-listed clinical-stage company is targeting site initiation in the first quarter of 2027, subject to IRB approval and routine site activation steps.
The study marks the first time Ketamir-2 will be evaluated in patients rather than healthy volunteers. A Phase 1 programme, now complete, established a favourable safety, tolerability and pharmacokinetic profile, with no serious adverse events or dose-limiting toxicities reported. The Phase 2a trial is designed as a randomised, double-blind, placebo-controlled crossover study enrolling adults with moderate-to-severe persistent CIPN. Participants will receive 300 mg and 600 mg doses of Ketamir-2, alongside placebo, across three seven-day treatment periods separated by 13-day washouts, allowing each participant to act as their own control.
The scientific rationale
Ketamir-2 is an oral modulator of the NMDA receptor PCP binding site, designed to replicate the analgesic mechanism of ketamine while avoiding several of its practical limitations. Ketamine has established clinical relevance in neuropathic pain through NMDA receptor inhibition, but its Schedule III controlled-substance status in the United States, intravenous route of administration and dissociative side-effect profile constrain its use in chronic settings. MIRA says Ketamir-2 is orally bioavailable, supports once-daily dosing based on Phase 1 pharmacokinetics, and has not been classified as a controlled substance by the DEA.
Chief Scientific Advisor Itzchak Angel said: "Ketamir-2 was designed as an oral NMDA receptor modulator with high selectivity and a differentiated pharmacologic profile, and we believe this Phase 2a study will provide an important first opportunity to determine whether this profile can translate into meaningful pain reduction in patients with persistent CIPN."
Market context and competitive landscape
CIPN represents a significant unmet need. There are currently no FDA-approved therapies specifically indicated for the condition, leaving clinicians to rely on off-label agents including duloxetine, gabapentin and, in some settings, intravenous ketamine infusions. The neuropathic pain market is broad: established players such as Pfizer and Eli Lilly hold dominant positions in adjacent indications, while a number of smaller biotech and specialty pharma companies are pursuing novel analgesic mechanisms, including sodium channel blockers, TRPV1 modulators and other NMDA-pathway compounds.
MIRA frames CIPN as a clinical entry point into the wider neuropathic pain market, suggesting that positive Phase 2a readouts could support label expansion into other pain indications driven by NMDA receptor dysregulation. That sequencing is logical: establishing proof of concept in a relatively well-characterised, mechanistically coherent patient population before pursuing broader indications is a conventional development strategy and should be familiar to investors evaluating the pipeline optionality.
The company holds exclusive worldwide rights to Ketamir-2 and is pursuing intellectual property protection across major markets. MIRA also plans to present Phase 1 pharmacokinetic and safety data at the International Conference on Addiction and Psychiatry in Tokyo on 5 to 6 October 2026, and will participate in BioJapan the same month as it continues licensing and partnership discussions.
Near-term catalysts for investors are the IRB approval, site initiation confirmation and, ultimately, top-line efficacy and dose-response data from the crossover study. Given the short treatment periods and crossover design, a readout could come relatively quickly once enrolment begins, though MIRA has not indicated a data timeline.