MIRA Pharmaceuticals' Lead Drug Candidate Ketamir-2 First Manuscript Accepted for Publication in the Peer-Reviewed Journal Frontiers in Pharmacology

Rhea-AI Summary

MIRA Pharmaceuticals announced the acceptance of its first manuscript on Ketamir-2, their lead drug candidate, in Frontiers in Pharmacology. The publication validates Ketamir-2's differentiated pharmacology as a novel ketamine analog, highlighting its selective NMDA receptor antagonism, favorable safety profile, and oral bioavailability. Key findings show Ketamir-2's clean pharmacological profile with minimal off-target effects, no hyperlocomotion even at high doses, and demonstrated antidepressant and anxiolytic activity. The company's Phase 1 trial for neuropathic pain is progressing well, with plans to initiate Phase 2a trials by year-end 2025. Additionally, MIRA confirmed its acquisition of SKNY Pharmaceuticals, which includes SKNY-1, a CB1/CB2 inverse agonist for obesity and smoking cessation, is proceeding as planned.

Positive

• Publication in peer-reviewed journal provides external scientific validation of Ketamir-2's potential
• Ketamir-2 shows cleaner pharmacological profile with fewer side effects compared to ketamine
• Phase 1 trial progressing with no safety concerns reported
• Planned expansion into Phase 2a trials by year-end 2025
• Strategic acquisition of SKNY Pharmaceuticals expanding pipeline with SKNY-1 for obesity and smoking cessation

Negative

• Early-stage development with Phase 1 trials still ongoing
• Regulatory approval for Phase 2a trials still pending
• Merger with SKNY Pharmaceuticals still subject to regulatory approval

Insights

Pharmaceutical R&D Analyst

MIRA's Ketamir-2 publication provides scientific validation but remains early-stage with significant development hurdles ahead.

The peer-reviewed publication of MIRA's Ketamir-2 in Frontiers in Pharmacology represents a meaningful scientific validation milestone. This external credibility supports the company's claims about their lead compound's differentiated pharmacological profile compared to ketamine. The publication highlights several potentially significant advantages: highly selective NMDA receptor targeting with minimal off-target binding, absence of hyperlocomotion (a marker for dissociative/psychotic effects), demonstrated anxiolytic and antidepressant activity in preclinical models, and effective oral delivery with efficient brain penetration.

The oral bioavailability represents a substantial potential advantage over conventional ketamine, which typically requires IV or intranasal administration due to poor oral absorption. CNS drugs with convenient oral dosing typically achieve better patient compliance and can command premium pricing if approved.

The progression of the Phase 1 trial without reported safety concerns is encouraging but expected at this safety-focused stage. The planned advancement to Phase 2a in neuropathic pain by year-end 2025 establishes a reasonable development timeline, though investors should recognize that CNS drugs face historically challenging success rates in mid-to-late stage clinical trials.

While this publication validates MIRA's scientific approach and provides important mechanistic differentiation from ketamine, it's important to contextualize this as an early milestone. Many compounds with promising preclinical profiles fail to demonstrate efficacy in larger human trials. The company will likely require additional capital to advance both Ketamir-2 and the SKNY-1 asset (acquired through the SKNY Pharmaceuticals merger) through clinical development.

This scientific validation represents incremental progress but doesn't fundamentally alter the high-risk, early-stage profile of MIRA's development pipeline.

MIAMI, FL / ACCESS Newswire / June 18, 2025 / MIRA Pharmaceuticals, Inc. (Nasdaq:MIRA) ("MIRA" or the "Company"), a clinical-stage pharmaceutical company developing novel therapeutics for neurologic, neuropsychiatric, and metabolic disorders, today announced that the first manuscript describing its lead drug candidate Ketamir-2, currently being evaluated in an ongoing Phase 1 clinical trial for neuropathic pain, has been accepted for publication in the peer-reviewed journal Frontiers in Pharmacology.

The article, titled "KETAMIR-2, A NEW MOLECULAR ENTITY AND NOVEL KETAMINE ANALOG," authored by Itzchak Angel, Ph.D., MIRA's Chief Scientific Advisor, highlights Ketamir-2's pharmacological differentiation from ketamine and its potential as a next-generation CNS therapeutic.

Peer Review Validates Differentiated Pharmacology and Safety

Acceptance into Frontiers in Pharmacology provides external scientific validation by independent experts, underscoring the rigor and credibility of MIRA's research. The publication confirms that Ketamir-2 was specifically engineered to overcome limitations associated with ketamine-such as poor oral bioavailability, dissociative side effects, and non-specific receptor binding.

Key Highlights from the Publication:

• Highly Selective, Cleaner Mechanism: Ketamir-2 is a low-affinity NMDA receptor antagonist that selectively targets the NMDA PCP site. Unlike ketamine, Ketamir-2 showed no significant interaction with over 40 other receptors, transporters, or ion channel targets-including dopamine, opioid, serotonin, and monoaminergic systems-highlighting its clean pharmacological profile and reduced off-target effects.

• No Hyperlocomotion, Even at High Doses: In contrast to ketamine, Ketamir-2 did not induce hyperlocomotion in preclinical models-a behavior associated with agitation and schizophrenia-like symptoms-suggesting a favorable neurobehavioral safety profile.

• Demonstrated Antidepressant and Anxiolytic Activity: In validated behavioral models (Open Field Test, Elevated Plus Maze, Forced Swim Test), Ketamir-2 demonstrated clear anxiolytic and antidepressant-like effects. Ketamine, used as a control, either showed no benefit or limited effect in most tests.

• Oral Delivery with Efficient Brain Penetration: All studies were conducted via the oral route. Ketamir-2 was shown to cross the blood-brain barrier and is not a substrate for P-glycoprotein, which often limits oral drug delivery to the brain. This may explain Ketamir-2's ability to maintain CNS activity despite its lower NMDA receptor affinity.

"We are honored to see our foundational research on Ketamir-2 published in a high-impact scientific journal," said Erez Aminov, CEO of MIRA. "This milestone adds meaningful scientific credibility and supports our confidence in Ketamir-2's differentiated mechanism, favorable safety profile, and broad clinical potential."

"This peer-reviewed publication provides clear validation of the differentiated pharmacological profile of Ketamir-2," added Dr. Itzchak Angel, Chief Scientific Advisor. "Its clean pharmacological profile and safety make it a compelling next-generation alternative to ketamine."

Clinical and Corporate Updates

MIRA also announced that its Phase 1 trial of Ketamir-2 is progressing as planned, with no safety concerns reported to date and dose escalation advancing. The Company expects to initiate a Phase 2a clinical trial in neuropathic pain by year-end 2025, pending regulatory clearance.

In addition, the Company is preparing new scientific data submissions and presentations to further support Ketamir-2's clinical development and potential across CNS-related conditions.

MIRA also reaffirmed that the acquisition of SKNY Pharmaceuticals, which includes a first-in-class oral CB1/CB2 inverse agonist for obesity and smoking cessation (SKNY-1), is progressing on track. The Company has submitted the required regulatory filings for the merger to the U.S. Securities and Exchange Commission (SEC).

The publication will be available upon release at: www.frontiersin.org/journals/pharmacology

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. 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, including related to MIRA's potential merger with SKNY Pharmaceuticals, Inc. 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

View the original press release on ACCESS Newswire

FAQ

What are the key advantages of MIRA's Ketamir-2 over traditional ketamine?

Ketamir-2 offers selective NMDA receptor targeting, minimal off-target effects, better oral bioavailability, and no hyperlocomotion side effects, making it potentially safer and more effective than traditional ketamine.

What is the current development stage of MIRA's Ketamir-2?

Ketamir-2 is currently in Phase 1 clinical trials for neuropathic pain, with plans to advance to Phase 2a trials by the end of 2025, pending regulatory approval.

What conditions could MIRA's Ketamir-2 potentially treat?

Based on preclinical studies, Ketamir-2 shows potential for treating neuropathic pain, depression, and anxiety-related conditions due to its demonstrated antidepressant and anxiolytic effects.

What is the significance of MIRA's publication in Frontiers in Pharmacology?

The peer-reviewed publication provides external scientific validation of Ketamir-2's differentiated pharmacology and safety profile, adding credibility to MIRA's research and development efforts.

What is MIRA's acquisition of SKNY Pharmaceuticals about?

MIRA is acquiring SKNY Pharmaceuticals, which includes SKNY-1, a first-in-class oral CB1/CB2 inverse agonist being developed for obesity and smoking cessation treatments.