Ketamir-2 Demonstrates Strong Efficacy in Diabetic Neuropathy Model, with Some Subjects Achieving Complete Symptom Reversal

MIAMI, FLORIDA / ACCESS Newswire / April 16, 2025 / MIRA Pharmaceuticals, Inc. (NASDAQ: MIRA), or MIRA, a clinical-stage pharmaceutical company focused on developing novel therapeutics for neurologic and neuropsychiatric disorders, today announced compelling data demonstrating the efficacy of the oral ketamine analog, Ketamir-2, in a validated animal model of diabetic neuropathy.

In the study, Type 2 diabetes was induced in rats using a high-fat diet combined with a low dose of Streptozotocin (STZ). This resulted in hyperglycemia and neuropathic symptoms, including hyperalgesia and allodynia, mimicking human diabetic pathology. By week 8, most diabetic rats exhibited sensory hypersensitivity. Treatment with Ketamir-2 led to a significant reduction in neuropathic pain symptoms, with some animals returning completely to pre-diabetic baseline sensitivity. "Millions of patients with diabetic neuropathy are left with limited, often ineffective treatment options," said Erez Aminov, Chairman and CEO of MIRA.

"With Ketamir-2, we're aiming to offer a non-opioid alternative that's not only safer but potentially more effective. We're currently advancing our Phase I study and plan to initiate a Phase IIa trial in diabetic neuropathy patients by year-end. Given that the FDA has designated neuropathic pain as a high-priority area for Fast Track and Breakthrough Therapy pathways, we believe Ketamir-2 is uniquely positioned to meet this urgent need and unlock significant clinical and commercial value."

Translational Data Strengthens Clinical Momentum

These results build upon prior preclinical studies using other neuropathy animal models, where orally administered Ketamir-2 outperformed FDA-approved neuropathic pain medications such as pregabalin and gabapentin, while also demonstrating a favorable safety profile. Unlike traditional ketamine, Ketamir-2 does not induce dissociative or psychedelic effects and is not a substrate for P-glycoprotein (P-gp), allowing for more efficient penetration across the blood-brain barrier. MIRA has already begun dosing and recruitment in its Phase I clinical trial at Hadassah Medical Center in Jerusalem. The study is progressing smoothly and on schedule. The randomized, double-blind, placebo-controlled trial is assessing safety, tolerability, and pharmacokinetics in healthy volunteers. Completion is expected in Q4 2025, with a Phase IIa study in diabetic neuropathy patients planned to follow.

"The strong alignment between this model and human pathology, together with results obtained with other experimental models, gives us a high degree of scientific confidence as we move forward," said Dr. Itzchak Angel, Chief Scientific Advisor at MIRA. "Seeing such robust responses in a validated model of diabetic neuropathy reinforces the potential clinical impact of Ketamir-2 and helps guide the path ahead."

Addressing a Pressing Unmet Need

Diabetic neuropathy affects between 28% to 55% of patients with diabetes and remains one of the most prevalent and debilitating complications associated with the disease. As global diabetes rates continue to climb, the number of patients suffering from chronic neuropathic pain is expected to rise sharply, placing an increasing burden on healthcare systems worldwide.

Current treatment options are limited, with first-line therapies such as pregabalin, gabapentin, and duloxetine often delivering only modest symptom relief. These drugs are frequently associated with side effects such as dizziness, fatigue, cognitive impairment, and gastrointestinal discomfort. Up to 50% of patients fail to achieve meaningful pain reduction, underscoring the urgent need for safer and more effective alternatives. (Tesfaye et al., Diabetes Metab Res Rev. 2011)

At the same time, the demand for non-opioid therapies is growing rapidly, driven by the ongoing opioid crisis and heightened regulatory scrutiny. Ketamir-2's non-addictive profile and lack of dissociative side effects position it as a strong candidate in this emerging treatment landscape.

The economic burden of diabetic neuropathy is also substantial. A study published in Drugs & Aging estimated that diabetic peripheral neuropathy contributes more than $10 billion annually in direct medical costs in the U.S. alone. This includes costs related to increased hospitalizations, physician visits, disability, and lost productivity.
(Gordois et al., Drugs Aging. 2003)

Importantly, the FDA has previously identified neuropathic pain as a high-priority area for accelerated development, granting Fast Track and Breakthrough Therapy designations to compounds that demonstrate potential to improve outcomes over standard treatments.
(FDA Guidance for Industry - Expedited Programs for Serious Conditions)

MIRA believes that, with continued positive data, Ketamir-2 may be positioned to meet key criteria for future regulatory acceleration.

Looking Ahead

MIRA anticipates initiating its Phase IIa clinical trial in diabetic neuropathy by the end of 2025, with first human efficacy data expected in the first half of 2026. In parallel, development of a topical slow-release formulation of Ketamir-2 continues, aimed at providing localized pain relief with minimized systemic exposure.

In addition, MIRA is actively conducting ongoing studies assessing the efficacy of Ketamir-2 in models of post-traumatic stress disorder (PTSD), a serious and underserved neuropsychiatric condition. MIRA remains committed to unlocking the full therapeutic potential of Ketamir-2 across multiple indications.

MIRA is also pleased to report continued progress on the pending acquisition of SKNY Pharmaceuticals, a move that strengthens MIRA's strategic pipeline. MIRA is highly enthusiastic about the promise of SKNY-1, a next-generation oral therapeutic in development for the treatment of obesity and smoking cessation, two major public health challenges that lack scalable, well-tolerated solutions.

About MIRA Pharmaceuticals, Inc.

MIRA Pharmaceuticals, Inc. (NASDAQ: MIRA) is a clinical-stage pharmaceutical development company with two neuroscience programs targeting a broad range of neurologic and neuropsychiatric disorders. MIRA holds the exclusive U.S., Canadian, and Mexican rights for Ketamir-2, a novel, patent-pending oral ketamine analog under investigation to treat neuropathic pain (NP), treatment-resistant depression (TRD), major depressive disorder with suicidal ideation (MDD-SI), and post-traumatic stress disorder (PTSD).

MIRA's novel oral pharmaceutical marijuana analog, MIRA-55, is currently under investigation for treating adult patients suffering from anxiety and cognitive decline, often associated with early-stage dementia. If approved by the FDA, MIRA-55 could mark a significant advancement in addressing various neuropsychiatric, inflammatory, and neurologic diseases and disorders.

The U.S. Drug Enforcement Administration's scientific review concluded that both Ketamir-2 and MIRA-55 would not be considered controlled substances or listed chemicals under the Controlled Substances Act and its governing regulations.

Additional information about MIRA Pharmaceuticals is available at www.mirapharmaceuticals.com.

Cautionary Note Regarding Forward-Looking Statements

This press release and the statements of MIRA's management related thereto contain "forward-looking statements," which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements may be identified by words such as "aims," "anticipates," "believes," "could," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words or similar expressions that are intended to identify forward-looking statements. Any statements in this press release that are not historical facts may be deemed forward-looking. These forward-looking statements include, without limitation, statements regarding the anticipated benefits of the acquisition transaction and claims regarding SKNY-1. Any forward-looking statements in this press release are based on Mira's current expectations, estimates, and projections only as of the date of this release and are subject to a number of risks and uncertainties (many of which are beyond MIRA's control) that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These and other risks concerning MIRA's programs and operations are described in additional detail in the Annual Report on Form 10-K for the year ended December 31, 2024, and other SEC filings, which are on file with the SEC at www.sec.gov and MIRA's website at https://www.mirapharmaceuticals.com/investors/sec-filings. MIRA explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Contact Information

Helga Moya
info@mirapharma.com
(786) 432-9792

SOURCE: MIRA Pharmaceuticals