MIAMI, FLORIDA / ACCESS Newswire / April 1, 2025 / MIRA Pharmaceuticals, Inc. (NASDAQ:MIRA), a clinical-stage pharmaceutical company developing breakthrough therapeutics for neurologic and neuropsychiatric disorders, today announced the enrollment of the first subjects in its Phase 1 clinical trial of Ketamir-2, a novel oral ketamine analog in development for the treatment of neuropathic pain.
The Phase 1 trial is being conducted at the Clinical Pharmacology Unit of Hadassah Medical Center in Jerusalem, Israel. This randomized, double-blind, placebo-controlled study will assess the safety, tolerability, and pharmacokinetics of Ketamir-2 in healthy adult volunteers. A total of 56 subjects will be enrolled across both Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) cohorts.
This important clinical milestone follows compelling preclinical data, where Ketamir-2 demonstrated superior efficacy compared to FDA-approved neuropathic pain treatments, including pregabalin and gabapentin. It also showed a strong safety profile and oral bioavailability, without the dissociative or psychedelic effects commonly associated with ketamine. Additionally, Ketamir-2 is not a substrate for P-glycoprotein (P-gp), which supports its ability to efficiently cross the blood-brain barrier-a critical factor in treating central nervous system disorders.
"The Phase 1 trial is progressing smoothly and according to plan," said Dr. Itzchak Angel, Chief Scientific Advisor at MIRA. "Ketamir-2's unique mechanism and pharmacological profile make it a highly promising candidate for neuropathic pain. If successful, it could represent a safer, non-addictive alternative to opioids and a more effective option than current standard treatments like pregabalin and gabapentin."
Phase 1 Completion and Clinical Timeline
MIRA anticipates completing the Phase 1 study by Q4 2025. The Company plans to initiate a Phase 2a trial in patients with diabetic neuropathic pain by year-end, with first human efficacy data expected in the first half of 2026.
Expansion into Topical Ketamir-2 Formulation
MIRA has also successfully formulated a topical version of Ketamir-2, designed for slow-release, localized delivery in the treatment of inflammatory and neuropathic pain. This approach may offer targeted pain relief at the site of application while minimizing systemic exposure. Preclinical studies are ongoing to assess efficacy, with results expected to guide the next steps in clinical development.
Strategic Pipeline Growth Through SKNY Pharmaceuticals Acquisition
MIRA is also advancing the acquisition of SKNY Pharmaceuticals under a binding letter of intent. The transaction includes a $5 million capital infusion-comprised of cash or equivalent consideration-into MIRA, further strengthening the Company's financial position and supporting the advancement of SKNY-1, a next-generation oral therapeutic for weight loss and smoking cessation. The timing of this acquisition is especially significant given the widespread use and growing concern around GLP-1-based weight loss treatments such as Wegovy® (semaglutide) and Saxenda® (liraglutide). While these therapies have shown effectiveness, they are often associated with gastrointestinal side effects, gallbladder issues, muscle loss, high discontinuation rates, and both carry an FDA Black Box warning. Additionally, they require ongoing injections and come with substantial costs, creating barriers for long-term use and broad accessibility.
SKNY-1 is being developed as a novel, non-GLP-1 oral therapy that modulates cannabinoid receptor pathways to support metabolic regulation. If successful, SKNY-1 could provide a safer, more convenient, and cost-effective alternative in the growing global obesity market, projected to exceed $150 billion by the early 2030s.
"Enrolling our first subjects in this Phase 1 study marks a pivotal step forward for MIRA," said Erez Aminov, Chairman and CEO of MIRA. "It validates years of focused research and execution. With the addition of our topical formulation, the SKNY acquisition, and a growing pipeline of novel compounds, we are building a multi-dimensional platform that aims to transform the way we treat pain, addiction, and chronic disease."
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.
This press release and the statements of MIRA Pharmaceuticals' (or the "Company") 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 study results described herein as well as the timing for the Company's other preclinical studies and the filing of an IND for Ketamir-2. Any forward-looking statements in this press release are based on the Company'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 the Company'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 the Company'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 the Company's website at https://www.mirapharmaceuticals.com/investors/sec-filings. The Company explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.
