MIRA Pharmaceuticals Advances Ketamir-2 to Phase 2a in Chemotherapy-Induced Peripheral Neuropathy (CIPN) Following FDA Review
Ketamir-2 moves from healthy-volunteer testing into its first efficacy-focused CIPN trial, supporting MIRA’s broader neuropathic pain strategy.
Rhea-AI Summary
MIRA Pharmaceuticals (MIRA) has advanced Ketamir-2 toward a Phase 2a study in chemotherapy-induced peripheral neuropathy (CIPN) by submitting a protocol for Institutional Review Board review after incorporating U.S. Food and Drug Administration feedback.
The randomized, double-blind, placebo-controlled, three-period crossover trial at a leading cancer research institution will enroll adults with moderate-to-severe persistent CIPN and test oral Ketamir-2 at 300 mg and 600 mg versus placebo across three seven-day treatment periods separated by 13-day washouts. Objectives include evaluating safety, tolerability, efficacy, dose-response, and effects on neuropathic pain intensity, CIPN-related symptoms, functional status, and central nervous system adverse effects. The study, planned for site initiation in the first quarter of 2027 subject to IRB and routine site-activation completion, is Ketamir-2’s first patient trial under an active IND and builds on a Phase 1 program that reported a favorable safety, tolerability, and pharmacokinetic profile with no serious adverse events or dose-limiting toxicities.
Positive
- Phase 2a protocol for Ketamir-2 in CIPN submitted for IRB review after FDA feedback
- Company targets Q1 2027 for site initiation, subject to IRB and site-activation completion
- Phase 1 program showed no serious adverse events or dose-limiting toxicities and a favorable pharmacokinetic profile
- Ketamir-2 is an oral NMDA receptor modulator with once-daily dosing supported by Phase 1 pharmacokinetics
- Ketamir-2, MIRA-55, and SKNY-1 are not classified as controlled substances by the DEA
- MIRA holds exclusive worldwide rights to Ketamir-2 and is pursuing intellectual property protection
Negative
- None.
Details
Market reaction after Phase 2a protocol submission: MIRA +5.48%
Following this news, MIRA has gained 5.48%, reflecting a notable positive market reaction. Our momentum scanner has triggered 2 alerts so far, indicating moderate trading interest and price volatility. The stock is currently trading at $0.76. Trading volume is elevated at 2.6x the average, suggesting notable buying interest.
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Key Figures
- Site initiation target
- Q1 2027
- Phase 2a CIPN study, subject to IRB and site activation
- Ketamir-2 doses
- 300 mg and 600 mg
- Phase 2a CIPN study
- Treatment periods
- Three seven-day periods
- Randomized, double-blind, placebo-controlled crossover study
- Washout periods
- 13 days
- Between treatment periods in the Phase 2a study
- Serious adverse events
- No serious adverse events
- Completed Phase 1 study in healthy volunteers
- Dose-limiting toxicities
- None reported
- Completed Phase 1 study in healthy volunteers
Historical Context
-
FDA acknowledged the Phase 2a CIPN protocol for Ketamir-2
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Key Terms
chemotherapy-induced peripheral neuropathy medical
institutional review board regulatory
pharmacokinetics medical
dose-limiting toxicities medical
investigational new drug regulatory
AI-generated analysis. How Rhea-AI works. Not financial advice.
Protocol Submitted for IRB Review at a Leading Cancer Research Institution, Advancing the Study Toward Site Initiation
MIAMI, Sept. 17, 2026 (GLOBE NEWSWIRE) -- MIRA Pharmaceuticals, Inc. (NASDAQ: MIRA) ("MIRA" or the "Company"), a clinical-stage pharmaceutical company developing novel oral small-molecule therapeutics, today announced that, following review and comments from the U.S. Food and Drug Administration (FDA), the Phase 2a protocol evaluating Ketamir-2 in chemotherapy-induced peripheral neuropathy (CIPN) has been submitted for Institutional Review Board (IRB) review at a leading cancer research institution.
MIRA incorporated the FDA's feedback into the protocol and is targeting site initiation in the first quarter of 2027, subject to completion of IRB review and other routine site activation requirements. The study represents Ketamir-2's first clinical evaluation in patients with CIPN and is designed to generate initial efficacy and dose-response data while further characterizing safety and tolerability.
"We are entering an important stage for Ketamir-2 as we prepare to evaluate the drug for the first time in patients with persistent CIPN," said Erez Aminov, CEO of MIRA. "This marks the transition from establishing safety and pharmacokinetics in healthy volunteers to evaluating Ketamir-2's potential therapeutic effect in patients."
About the Phase 2a Study
The randomized, double-blind, placebo-controlled, three-period crossover study will enroll adults with moderate-to-severe persistent CIPN. Participants will receive Ketamir-2 at 300 mg and 600 mg, as well as placebo, in a defined treatment sequence across three seven-day treatment periods separated by 13-day washout periods, allowing each participant to serve as their own control.
The study will evaluate the safety, tolerability, efficacy, and dose-response relationship of oral Ketamir-2, including its effect on post-chemotherapy neuropathic pain intensity. Additional assessments will evaluate CIPN-related symptoms and functional status, as well as potential central nervous system adverse effects.
The study builds on MIRA's completed Phase 1 clinical program in healthy volunteers, in which Ketamir-2 demonstrated a favorable safety, tolerability, and pharmacokinetic profile, with no serious adverse events or dose-limiting toxicities reported. The Phase 2a study will be conducted under MIRA's active Investigational New Drug (IND) application.
Addressing a Significant Unmet Need in Neuropathic Pain
CIPN is a painful and potentially persistent complication of certain commonly used chemotherapies that can continue long after cancer treatment has ended. Treatment options remain limited, and there are currently no FDA-approved therapies specifically indicated for CIPN. Existing treatment approaches offer limited options for managing painful CIPN, underscoring the need for new therapies specifically developed for this condition.
Ketamine has demonstrated analgesic activity in neuropathic pain through modulation of the NMDA receptor pathway, supporting the therapeutic relevance of this mechanism. However, its broader use as a chronic treatment can be constrained by intravenous administration, dissociative and other neuropsychiatric effects, monitoring requirements, and its Schedule III controlled-substance status.
Ketamir-2 is being developed as an oral NMDA receptor modulator designed to engage this therapeutically relevant pathway while potentially overcoming key limitations associated with ketamine. Ketamir-2 is orally administered; its Phase 1 pharmacokinetic profile supports once-daily dosing, and it is not classified as a controlled substance by the U.S. Drug Enforcement Administration (DEA).
"There is a strong scientific rationale for targeting NMDA receptor signaling in neuropathic pain," said Itzchak Angel, Ph.D., Chief Scientific Advisor of MIRA. "Ketamine has provided important clinical validation of this pathway, but its properties can limit its practicality for chronic use. Ketamir-2 was designed as an oral NMDA receptor modulator with high selectivity and a differentiated pharmacologic profile, and we believe this Phase 2a study will provide an important first opportunity to determine whether this profile can translate into meaningful pain reduction in patients with persistent CIPN."
While CIPN is the initial patient population being evaluated in Phase 2a, MIRA believes that positive clinical evidence in CIPN could support the evaluation of Ketamir-2 across additional neuropathic pain conditions where NMDA receptor signaling may play a role. The Company views CIPN as a potential clinical entry point into the broader neuropathic pain market.
October Scientific Presentation and BioJapan Participation
MIRA expects to present Ketamir-2 at the International Conference on Addiction and Psychiatry in Tokyo, Japan, on October 5–6, 2026, where an abstract titled "An Oral Selective NMDA Modulator for Chemotherapy-Induced Peripheral Neuropathy—Phase 1 Safety, Pharmacokinetics, and Clinical Development Update" has been accepted for oral presentation by Dr. Itzchak Angel, MIRA's Chief Scientific Advisor.
MIRA also expects to participate in BioJapan in October as the Company continues licensing and strategic partnership discussions around Ketamir-2 and its broader pipeline.
About Ketamir-2
Ketamir-2 is MIRA Pharmaceuticals' proprietary oral NMDA receptor modulator in clinical development for neuropathic pain. The compound is designed to selectively modulate the NMDA receptor PCP binding site with low binding affinity and limited off-target receptor activity.
MIRA holds exclusive worldwide rights to Ketamir-2 and is pursuing intellectual property protection across major global pharmaceutical markets.
About MIRA Pharmaceuticals, Inc.
MIRA Pharmaceuticals, Inc. (NASDAQ: MIRA) is a clinical-stage pharmaceutical company developing novel oral small-molecule therapeutics. The Company's pipeline includes Ketamir-2 for chemotherapy-induced peripheral neuropathy (CIPN), which has completed Phase 1 and is advancing toward Phase 2a under an active IND; MIRA-55, an investigational oral drug candidate for inflammatory pain; and SKNY-1, an investigational oral drug candidate for obesity. Following scientific review, the U.S. Drug Enforcement Administration has determined that Ketamir-2, MIRA-55, and SKNY-1 are not classified as controlled substances.
Cautionary Note Regarding Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements generally may be identified by the use of words such as "anticipate," "expect," "plan," "can," "could," "would," "may," "will," "believe," "estimate," "forecast," "goal," "project," "guidance," "potential," "intend," "seek," "target" and other words of similar meaning, although not all forward-looking statements include these words. Forward-looking statements may include, but are not limited to, statements regarding the therapeutic potential, mechanism of action, development plans, regulatory pathway, safety profile, efficacy, anticipated clinical development, commercialization prospects, market opportunity, and future development of MIRA-55, SKNY-1, Ketamir-2, and the Company's other product candidates.
These forward-looking statements are based on current expectations, estimates, forecasts, and projections, as well as management's current beliefs and assumptions, and are subject to significant risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, among others, risks related to preclinical and clinical development, the ability to obtain regulatory approvals, the outcome of future studies, reliance on third parties, intellectual property protection, financing needs, market conditions, and the other risks identified under the heading "Risk Factors" contained in the Company's Annual Report on Form 10-K and the Company's subsequent filings with the U.S. Securities and Exchange Commission (“SEC”).
Forward-looking statements contained in this press release speak only as of the date of this press release, and the Company undertakes no obligation to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by applicable law.
Investors are cautioned not to place undue reliance on these forward-looking statements. Additional information regarding these and other risks and uncertainties is contained in the Company's filings with the SEC, including its Annual Report on Form 10-K and subsequent filings, available at www.sec.gov and in the Investors section of the Company's website at www.mirapharmaceuticals.com. Forward-looking statements should be considered in light of these risks and uncertainties.
Investor and Media Contact
Krystina Quintana
MIRA Pharmaceuticals
info@mirapharma.com
(786) 432-9792
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What are the key design features of the Ketamir-2 Phase 2a CIPN study?
The Phase 2a trial is a randomized, double-blind, placebo-controlled, three-period crossover study in adults with moderate-to-severe persistent CIPN. Participants receive oral Ketamir-2 at 300 mg and 600 mg and placebo in a defined treatment sequence across three seven-day treatment periods, each separated by 13-day washout periods so that each participant serves as their own control. Endpoints include safety, tolerability, efficacy, dose-response, neuropathic pain intensity, CIPN-related symptoms, functional status, and potential central nervous system adverse effects.
When is MIRA planning to initiate clinical sites for the Ketamir-2 Phase 2a trial?
MIRA is targeting site initiation for the Phase 2a Ketamir-2 CIPN study in the first quarter of 2027, subject to completion of Institutional Review Board review and other routine site activation requirements.
What prior clinical data on Ketamir-2 support moving into patients with CIPN?
The Phase 2a CIPN study builds on a completed Phase 1 clinical program in healthy volunteers in which Ketamir-2 demonstrated a favorable safety, tolerability, and pharmacokinetic profile, with no serious adverse events or dose-limiting toxicities reported.
Why is Ketamir-2 being developed for chemotherapy-induced peripheral neuropathy?
CIPN is a painful, potentially persistent complication of certain commonly used chemotherapies, and there are currently no FDA-approved therapies specifically indicated for CIPN. Existing treatments offer limited options for managing painful CIPN. Ketamine has shown analgesic activity in neuropathic pain via NMDA receptor modulation, and Ketamir-2 is designed as an oral NMDA receptor modulator to engage this pathway while potentially overcoming limitations associated with ketamine such as intravenous administration, dissociative effects, monitoring requirements, and controlled-substance status.
How does Ketamir-2 differ from ketamine in terms of pharmacology and regulation?
Ketamir-2 is an oral NMDA receptor modulator designed to selectively modulate the NMDA receptor PCP binding site with low binding affinity and limited off-target receptor activity, aiming for a differentiated pharmacologic profile. It is orally administered, its Phase 1 pharmacokinetic profile supports once-daily dosing, and, following scientific review, the U.S. Drug Enforcement Administration has determined that Ketamir-2 is not classified as a controlled substance.
What broader development plans does MIRA have for Ketamir-2 beyond CIPN?
While CIPN is the initial patient population being evaluated in Phase 2a, MIRA believes that positive clinical evidence in CIPN could support evaluation of Ketamir-2 across additional neuropathic pain conditions where NMDA receptor signaling may play a role, viewing CIPN as a potential clinical entry point into the broader neuropathic pain market.
What upcoming scientific and business development activities involve Ketamir-2?
MIRA expects Ketamir-2 to be presented at the International Conference on Addiction and Psychiatry in Tokyo, Japan, on October 5–6, 2026, through an oral presentation titled “An Oral Selective NMDA Modulator for Chemotherapy-Induced Peripheral Neuropathy—Phase 1 Safety, Pharmacokinetics, and Clinical Development Update” by Dr. Itzchak Angel, the company’s Chief Scientific Advisor. MIRA also expects to participate in BioJapan in October while continuing licensing and strategic partnership discussions related to Ketamir-2 and its broader pipeline.