MIRA Pharmaceuticals is set to advance its lead pain treatment, Ketamir-2, a novel oral ketamine analog, with the submission of an Investigational New Drug (IND) application to the FDA. This marks a crucial step towards initiating human trials for neuropathic pain, a market projected to reach $4.5 billion by 2030 in North America.
The company's IND application includes comprehensive data from in vitro and in vivo studies, detailing Ketamir-2’s pharmacology, pharmacokinetics, and toxicology. In parallel, MIRA is conducting a neurotoxicity study, as per FDA feedback, to support human dosing in the U.S.
Promising Preclinical Results
Preclinical studies have demonstrated Ketamir-2’s ability to achieve 100% pain reversal, fully normalizing pain thresholds in validated neuropathic pain models. Moreover, the drug has shown an encouraging safety profile, with no adverse effects observed in preclinical trials. Specifically, cardiovascular, CNS, and respiratory safety were confirmed in preclinical studies using dogs and rats at therapeutic doses. A 14-day toxicology study in dogs showed that Ketamir-2 was well-tolerated at daily doses up to 200 mg/kg, establishing the No Observed Adverse Effect Level (NOAEL).
Dr. Itzchak Angel, Chief Scientific Advisor at MIRA, stated, "The robust safety data we’ve generated for Ketamir-2 is nothing short of extraordinary. Ketamir-2 represents a groundbreaking step forward in addressing unmet medical needs, and I am confident that it has the potential to redefine treatment paradigms in this space."
Clinical Trial Plans
MIRA plans to begin Phase 1 trials outside the United States in Q1 2025 to evaluate safety, tolerability, and pharmacokinetics in healthy volunteers. Phase 2a trials are expected to follow in late 2025, assessing Ketamir-2’s efficacy in neuropathic pain patients. The Phase I/IIa trial will be conducted at The Centre for Human Drug Research (CHDR) in Leiden, The Netherlands, utilizing PainCart® technology for early efficacy assessment.
The Phase I/IIa trial is structured as a randomized, double-blind, placebo-controlled study conducted in two parts:
Phase I: Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) cohorts in healthy subjects will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics. PainCart will be used to measure pain response, providing robust early efficacy data, while psychoactivity will be assessed separately through dedicated testing.
Phase IIa: The Proof of Concept (POC) study, planned to begin in the last quarter of 2025, will focus on diabetic neuropathy patients, generating efficacy data in a patient population to validate the Phase I findings.
Addressing Unmet Needs in Neuropathic Pain
The neuropathic pain market in North America is valued at $3.1 billion and is expected to grow at a 6-7% compound annual growth rate, reaching $4.5 billion by 2030. MIRA aims to address the pressing need for safe, non-addictive treatments for neuropathic pain, offering an alternative to existing therapies that often cause intolerable side effects.
Beyond neuropathic pain, MIRA is exploring additional indications for Ketamir-2, including Major Depressive Disorder (MDD), MDD with Suicidal Ideation, and Post-Traumatic Stress Disorder. The company is also advancing MIRA-55, with preclinical trials targeting memory, cognition, and anxiety, and plans to submit an IND for MIRA-55 in 2025.
Erez Aminov, CEO and Chairman of MIRA, stated, "Our commitment to advancing Ketamir-2 into clinical trials as quickly as possible reflects our strategic focus on generating proof-of-concept data that positions MIRA for a potential M&A transaction or strategic partnership."
