MIRA Pharmaceuticals Announces Completion of Phase 1 Single Ascending Dose for Oral Ketamir-2 with No Safety Concerns, Advances to Multiple Ascending Dose Stage
Rhea-AI Summary
MIRA Pharmaceuticals (NASDAQ:MIRA) has successfully completed the Single Ascending Dose (SAD) portion of its Phase 1 clinical trial for oral Ketamir-2, demonstrating favorable safety and tolerability with no severe adverse effects. The study, conducted at Hadassah Clinical Research Center in Israel, evaluated four cohorts with doses ranging from 50mg to 600mg across 32 participants.
The company is now advancing to the Multiple Ascending Dose (MAD) stage, which will test three cohorts with daily doses of 150mg, 300mg, and 600mg for five consecutive days in up to 24 participants. MIRA plans to initiate a Phase 2a trial for neuropathic pain in Q4 2025, targeting a market of 36-51 million people in North America.
Positive
- Successful completion of Phase 1 SAD trial with favorable safety profile
- No severe or clinically significant adverse effects observed in any dose level
- Preclinical studies showed superior efficacy versus ketamine, pregabalin, and gabapentin
- Large market opportunity targeting 36-51 million neuropathic pain patients in North America
Negative
- Phase 1 trial remains ongoing and blinded
- Approximately one-third of investigational drugs typically fail during Phase 1
- Multiple competing treatments already exist in the market
Insights
MIRA's Ketamir-2 successfully clears Phase 1 SAD trial with positive safety profile, advancing to MAD stage before Phase 2a for neuropathic pain.
MIRA Pharmaceuticals has achieved a critical milestone in its clinical development program for Ketamir-2, successfully completing the Single Ascending Dose (SAD) portion of its Phase 1 trial with no severe or clinically significant adverse effects observed across four cohorts (doses ranging from 50mg to 600mg). This safety profile is particularly noteworthy since approximately one-third of investigational drugs fail during Phase 1 due to safety concerns, according to the Tufts Center for the Study of Drug Development.
The trial employed rigorous CNS safety monitoring using validated assessment tools including the Columbia-Suicide Severity Rating Scale, Bowdle Visual Analogue Scale, and Ketamine Side Effect Tool—essential for detecting potential psychiatric or dissociative effects that might be associated with NMDA receptor-targeting compounds like Ketamir-2. This comprehensive safety monitoring strategy provides strong validation for the preliminary safety findings.
The company is now advancing to the Multiple Ascending Dose (MAD) stage, evaluating three cohorts receiving daily doses of 150mg, 300mg, or 600mg for five consecutive days in up to 24 participants. This progression keeps MIRA on track for its planned Phase 2a trial in neuropathic pain, scheduled to begin in Q4 2025 following their FDA IND clearance.
What's particularly promising is how Ketamir-2's clinical profile aligns with preclinical data showing superior efficacy in neuropathic pain models compared to existing treatments like pregabalin and gabapentin, without ketamine's dissociative side effects. As an orally bioavailable compound targeting the NMDA receptor with low affinity and minimal off-target activity, Ketamir-2 could potentially address the significant unmet need in the neuropathic pain market affecting 36-51 million people in North America alone. The company is strategically positioned to expand its clinical program to other indications including depression, anxiety, and PTSD, contingent upon continued positive results.
Following FDA IND clearance for neuropathic pain, the Company is preparing to initiate its U.S. Phase 2a trial in Q4 2025
MIAMI, FL / ACCESS Newswire / August 19, 2025 / MIRA Pharmaceuticals, Inc. (NASDAQ:MIRA) ("MIRA" or the "Company"), a clinical-stage pharmaceutical company developing novel oral therapeutics for neurologic, neuropsychiatric, and metabolic disorders, today announced the successful completion of the Single Ascending Dose (SAD) portion of its ongoing Phase 1 clinical trial evaluating oral Ketamir-2. The study, conducted at the Hadassah Clinical Research Center in Israel under the direction of Principal Investigator Prof. Yoseph Caraco, demonstrated a favorable safety and tolerability profile, with no severe or clinically significant adverse effects observed to date.
"Completion of the SAD portion with a favorable safety and tolerability profile is an important milestone in the clinical development of Ketamir-2," said Prof. Yoseph Caraco, Principal Investigator of the Phase 1 study. "Importantly, no severe or clinically significant adverse effects have been observed to date, which supports continued progression into the Multiple Ascending Dose stage of the trial."
Phase 1 Study Overview
The study- "A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single-Centre Study of Single and Repeated Dosing of Ascending Doses, to Evaluate the Safety, Tolerability and Pharmacokinetics of Oral Ketamir-2 in Healthy Adult Subjects"-is designed to establish the safety, tolerability, and pharmacokinetics of Ketamir-2 in healthy adult volunteers.
Design: Single-center, randomized, double-blind, placebo-controlled SAD/MAD with sentinel dosing and Safety Steering Committee (SSC) reviews between cohorts.
SAD completed: Four cohorts (single doses 50 mg to 600 mg); 32 participants treated (male and female).
Safety monitoring: Extensive central nervous system (CNS) safety assessments were performed using well-validated clinical research tools:
Columbia-Suicide Severity Rating Scale (C-SSRS) - screens for suicidal ideation or behavior, supporting early detection of potential mood or psychiatric changes.
Bowdle Visual Analogue Scale (VAS) - measures possible psychedelic or dissociative effects sometimes seen with ketamine and related compounds.
Ketamine Side Effect Tool (KSET) - tracks a broad range of known ketamine-related side effects over time, including sensory, cognitive, and mood changes.
These tools provided multiple, complementary layers of safety evaluation to help detect even subtle CNS effects throughout the trial.
Status: To date, no severe or clinically significant adverse effects have been observed at any dose level in the SAD portion of the study.
While the study remains ongoing and blinded, it is worth noting that, across the pharmaceutical industry as a whole, approximately one-third of investigational drugs fail during Phase 1 due to safety concerns (Tufts Center for the Study of Drug Development). These interim observations provide encouraging context as MIRA advances Ketamir-2 through the next stage of clinical evaluation, consistent with the Company's mission to prioritize safety in every step of development.
The Company is advancing to the Multiple Ascending Dose (MAD) portion, which will evaluate three cohorts receiving daily oral doses of 150 mg, 300 mg, or 600 mg for five consecutive days in up to 24 participants.
"The emerging human safety profile complements the preclinical data we've generated, which show Ketamir-2's superior efficacy in multiple neuropathic pain models without triggering the hallmark CNS side effects of ketamine," said Dr. Itzchak Angel, Chief Scientific Advisor of MIRA. "This combination of efficacy, safety, and oral delivery positions Ketamir-2 as a promising next-generation treatment for neuropathic pain and potentially other CNS disorders."
Strategic & Commercial Potential
Ketamir-2 is a proprietary, orally bioavailable new molecular entity that selectively targets the NMDA receptor (PCP site) with low affinity and shows no significant off-target activity across a broad receptor panel. Preclinical studies have demonstrated superior performance versus ketamine, pregabalin, or gabapentin (depending on comparator and model) in gold-standard neuropathic pain models-without the dissociative effects associated with ketamine.
Neuropathic pain affects an estimated 36-51 million people in North America and represents a multi-billion-dollar market today, with long-term growth driven by diabetes prevalence, cancer survivorship, and aging-related nerve damage. MIRA intends to submit a Phase 2a clinical protocol in neuropathic pain by year-end 2025 and continue evaluating potential applications in depression, anxiety, PTSD, and localized pain, subject to ongoing results and regulatory feedback.
"We are pleased to see our Phase 1 program progress as planned," said Erez Aminov, CEO of MIRA. "Advancing into the MAD stage is an important operational step as we continue to build a rigorous safety and PK foundation for Ketamir-2 and work to deliver a differentiated, non-opioid option for patients with neuropathic pain."
About MIRA Pharmaceuticals, Inc.
MIRA Pharmaceuticals, Inc. (NASDAQ:MIRA) is a clinical-stage pharmaceutical company focused on the development and commercialization of novel therapeutics for neurologic, neuropsychiatric, and metabolic disorders. The Company's pipeline includes oral drug candidates designed to address significant unmet medical needs in areas such as neuropathic pain, inflammatory pain, obesity, addiction, anxiety, and cognitive decline.
For more information, please visit 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. 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 the Form 14A filed by MIRA on June 18, 2025, and other SEC filings, which are on file with the SEC at www.sec.gov and on 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:
Helga Moya
info@mirapharma.com
(786) 432-9792
SOURCE: MIRA Pharmaceuticals
View the original press release on ACCESS Newswire
FAQ
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