MIRA Pharmaceuticals Announces FDA Clearance of IND for Ketamir-2, Enabling U.S. Clinical Trials in Neuropathic Pain
MIRA Pharmaceuticals (NASDAQ:MIRA) announced FDA clearance of its IND application for Ketamir-2, a novel oral NMDA receptor antagonist for neuropathic pain treatment. The drug has shown superior efficacy to existing treatments in preclinical studies, demonstrating up to 112% greater pain relief than pregabalin and 70% more than gabapentin.
The company is completing the Single Ascending Dose (SAD) portion of its Phase 1 trial and preparing for Multiple Ascending Dose (MAD) studies. MIRA plans to initiate a U.S. Phase 2a clinical trial in Q4 2025. Additionally, MIRA's acquisition of SKNY Pharmaceuticals is expected to close in Q3 2025, bringing SKNY-1, a promising obesity and smoking cessation drug candidate, to its pipeline.
MIRA Pharmaceuticals (NASDAQ:MIRA) ha annunciato l'approvazione da parte della FDA della sua domanda IND per Ketamir-2, un nuovo antagonista orale del recettore NMDA per il trattamento del dolore neuropatico. Il farmaco ha dimostrato un'efficacia superiore rispetto ai trattamenti esistenti negli studi preclinici, offrendo fino al 112% di sollievo dal dolore in più rispetto al pregabalin e il 70% in più rispetto al gabapentin.
L'azienda sta completando la fase Single Ascending Dose (SAD) della sua sperimentazione di Fase 1 e si sta preparando per gli studi Multiple Ascending Dose (MAD). MIRA prevede di avviare una fase 2a clinica negli Stati Uniti nel quarto trimestre del 2025. Inoltre, l'acquisizione di SKNY Pharmaceuticals da parte di MIRA è prevista per il terzo trimestre del 2025, integrando nel suo portafoglio SKNY-1, un promettente candidato farmaco per l'obesità e la cessazione del fumo.
MIRA Pharmaceuticals (NASDAQ:MIRA) anunció la aprobación de la FDA para su solicitud IND de Ketamir-2, un nuevo antagonista oral del receptor NMDA para el tratamiento del dolor neuropático. El medicamento ha demostrado una eficacia superior a los tratamientos existentes en estudios preclínicos, proporcionando hasta un 112% más de alivio del dolor que pregabalina y un 70% más que gabapentina.
La compañía está finalizando la fase de Dosis Ascendentes Únicas (SAD) de su ensayo de Fase 1 y preparándose para los estudios de Dosis Ascendentes Múltiples (MAD). MIRA planea iniciar un ensayo clínico de Fase 2a en EE.UU. en el cuarto trimestre de 2025. Además, se espera que la adquisición de SKNY Pharmaceuticals por parte de MIRA se cierre en el tercer trimestre de 2025, incorporando SKNY-1, un prometedor candidato para el tratamiento de la obesidad y la cesación del tabaquismo, a su cartera.
MIRA Pharmaceuticals (NASDAQ:MIRA)는 신경병성 통증 치료를 위한 새로운 경구용 NMDA 수용체 길항제인 Ketamir-2의 IND 신청에 대해 FDA 승인을 받았다고 발표했습니다. 이 약물은 전임상 연구에서 기존 치료제에 비해 뛰어난 효능을 보였으며, 프레가발린 대비 최대 112% 더 큰 통증 완화와 가바펜틴 대비 70% 더 높은 효과를 입증했습니다.
회사는 현재 1상 임상시험의 단일 상승 용량(SAD) 단계를 완료 중이며, 다중 상승 용량(MAD) 연구를 준비하고 있습니다. MIRA는 2025년 4분기에 미국에서 2a상 임상시험을 시작할 계획입니다. 또한, MIRA가 SKNY Pharmaceuticals를 인수하는 건은 2025년 3분기에 완료될 예정이며, 이를 통해 비만 및 금연 치료 후보 약물인 SKNY-1이 파이프라인에 추가될 예정입니다.
MIRA Pharmaceuticals (NASDAQ:MIRA) a annoncé l'approbation par la FDA de sa demande IND pour Ketamir-2, un nouvel antagoniste oral du récepteur NMDA destiné au traitement de la douleur neuropathique. Ce médicament a démontré une efficacité supérieure aux traitements existants lors d'études précliniques, offrant jusqu'à 112 % de soulagement de la douleur en plus par rapport à la prégabaline et 70 % de plus que la gabapentine.
L'entreprise achève la phase Single Ascending Dose (SAD) de son essai de phase 1 et se prépare aux études Multiple Ascending Dose (MAD). MIRA prévoit de lancer un essai clinique de phase 2a aux États-Unis au quatrième trimestre 2025. Par ailleurs, l'acquisition de SKNY Pharmaceuticals par MIRA devrait être finalisée au troisième trimestre 2025, intégrant SKNY-1, un candidat prometteur pour le traitement de l'obésité et de l'arrêt du tabac, dans son portefeuille.
MIRA Pharmaceuticals (NASDAQ:MIRA) gab die FDA-Zulassung seines IND-Antrags für Ketamir-2 bekannt, einen neuartigen oralen NMDA-Rezeptor-Antagonisten zur Behandlung neuropathischer Schmerzen. Das Medikament zeigte in präklinischen Studien überlegene Wirksamkeit gegenüber bestehenden Behandlungen und bot bis zu 112 % stärkere Schmerzlinderung als Pregabalin sowie 70 % mehr als Gabapentin.
Das Unternehmen schließt derzeit den Abschnitt mit Einzeldosissteigerung (SAD) seiner Phase-1-Studie ab und bereitet sich auf Studien mit mehrfacher Dosissteigerung (MAD) vor. MIRA plant, im vierten Quartal 2025 eine Phase-2a-Studie in den USA zu starten. Zudem wird erwartet, dass die Übernahme von SKNY Pharmaceuticals im dritten Quartal 2025 abgeschlossen wird, wodurch SKNY-1, ein vielversprechender Kandidat gegen Fettleibigkeit und Rauchentwöhnung, in die Pipeline aufgenommen wird.
- FDA clearance of IND application for Ketamir-2, enabling U.S. clinical trials
- Superior efficacy demonstrated in preclinical studies - up to 112% greater pain relief than pregabalin
- Clean safety profile with no evidence of brain lesions or adverse CNS effects
- Planned acquisition of SKNY Pharmaceuticals on track for Q3 2025 closure
- Strong preclinical data showing good oral bioavailability and blood-brain barrier penetration
- Phase 1 trials still ongoing, with Phase 2a not starting until Q4 2025
- Multiple competing development programs may strain resources
- Clinical efficacy in humans yet to be demonstrated
Insights
MIRA's FDA IND clearance for Ketamir-2 represents significant regulatory progress for their novel non-opioid pain treatment showing strong preclinical efficacy.
The FDA's clearance of MIRA Pharmaceuticals' IND application for Ketamir-2 represents a critical regulatory milestone that validates their preclinical data package and enables U.S. clinical trials. This NMDA receptor antagonist has demonstrated several compelling differentiation factors from traditional ketamine, most notably the absence of Olney lesions in neurotoxicity studies – addressing a key safety concern with NMDA modulators.
The preclinical efficacy data is particularly impressive, with Ketamir-2 showing superior performance to gabapentin by 60% in chemotherapy-induced neuropathy models and outperforming pregabalin by 112% in nerve ligation models. These comparisons against current standard-of-care treatments suggest potential for meaningful clinical differentiation if results translate to humans.
MIRA is making efficient progress with their Phase 1 program, nearing completion of the Single Ascending Dose portion with plans to move to Multiple Ascending Dose studies. Their targeted timeline to initiate Phase 2a efficacy trials by Q4 2025 demonstrates disciplined execution of their development plan.
The compound's pharmacological profile – oral bioavailability, blood-brain barrier penetration, and lack of dissociative effects – addresses key limitations of IV ketamine while potentially maintaining efficacy. If successful, Ketamir-2 could fulfill the significant unmet need for effective, non-addictive, and accessible treatments for the growing neuropathic pain patient population that's currently underserved by existing therapies with modest efficacy and problematic side effects.
FDA IND clearance positions MIRA to advance Ketamir-2 into U.S. clinical trials with strong preclinical data suggesting competitive advantages.
This FDA clearance represents a significant de-risking event for MIRA Pharmaceuticals' lead program. The comprehensive preclinical package that supported the application – including pharmacology, safety, and manufacturing documentation – demonstrates the company's scientific rigor and regulatory execution capabilities.
MIRA is strategically expanding Ketamir-2's development across multiple high-value neuropathic pain indications. Their plans to pursue chemotherapy-induced peripheral neuropathy (CIPN) and diabetic peripheral neuropathy (DPN) target substantial market opportunities with growing prevalence and limited effective treatment options. The potential for a topical formulation further expands commercial possibilities.
The neuropathic pain market represents a substantial commercial opportunity with increasing prevalence driven by aging populations, rising diabetes rates, and cancer treatments. Current therapies provide only modest relief with problematic side effects, creating a clear market need for Ketamir-2's differentiated profile.
MIRA's broader pipeline strategy is taking shape with the planned acquisition of SKNY Pharmaceuticals, bringing in SKNY-1 for obesity and smoking cessation – two massive market opportunities. Meanwhile, MIRA-55, their cannabinoid analog showing morphine-comparable efficacy in pain models, adds further pipeline depth.
For a Nasdaq-listed clinical-stage company, this regulatory advancement, pipeline expansion, and strategic acquisition demonstrate a comprehensive growth strategy that could drive significant value creation if clinical development proves successful.
Oral, non-opioid drug candidate advancing through Phase 1 and nearing completion of the single ascending dose (SAD) portion as the Company prepares to launch U.S. Phase 2a by year-end
MIAMI, FLORIDA / ACCESS Newswire / July 29, 2025 / MIRA Pharmaceuticals, Inc. (NASDAQ:MIRA), a clinical-stage pharmaceutical company developing novel therapies for neurologic, neuropsychiatric, and metabolic disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for Ketamir-2, a novel oral NMDA receptor antagonist for the treatment of neuropathic pain.
The IND submission included a comprehensive data package encompassing preclinical pharmacology, safety, and toxicology studies, including a pivotal neurotoxicity study in Sprague-Dawley rats which showed no evidence of brain lesions or adverse CNS effects-a key distinction from traditional ketamine, which has been associated with NMDA-linked neurotoxicity and the formation of Olney lesions in similar preclinical models. These findings were reinforced by multiple validated neuropathic pain models-including chemotherapy-induced, diabetic, and nerve ligation-induced neuropathy-where Ketamir-2 consistently demonstrated potent analgesic effects and superior efficacy to gabapentin and pregabalin, with reversal of pain sensitivity observed in select models. The IND also incorporated the Company's Phase 1 readiness data and manufacturing documentation.
MIRA is nearing completion of the Single Ascending Dose (SAD) portion of its international Phase 1 trial at Hadassah Medical Center in Jerusalem, with dose escalation progressing smoothly. The Company is now preparing to initiate the Multiple Ascending Dose (MAD) portion of the study, which will assess the safety, tolerability, and pharmacokinetics of repeated dosing over several days. These data will help inform optimal dosing strategies as MIRA moves toward a Phase 2a study, with the goal of initiating its first U.S.-based efficacy trial in Q4 2025.
"This IND approval validates the strength of our preclinical data and the differentiated pharmacology of Ketamir-2," said Erez Aminov, Chairman and CEO of MIRA. "We are executing and advancing with precision and speed, and we believe Ketamir-2 may represent one of the most promising non-opioid, non-controlled neuropathic pain treatments in development today."
Building on Strong Preclinical Evidence
Ketamir-2 has consistently demonstrated robust efficacy and safety across a range of preclinical studies:
In a chemotherapy-induced neuropathy model, Ketamir-2 achieved near-complete normalization of pain sensitivity and outperformed gabapentin by
60% In a validated diabetic neuropathy model induced by a high-fat diet and low-dose streptozotocin (STZ) in rats, Ketamir-2 significantly reduced pain sensitivity, with some animals returning to pre-diabetic baseline sensitivity
In head-to-head studies using the chronic constriction injury (CCI) model-a widely accepted sciatic nerve ligation model of neuropathic pain-in rats, Ketamir-2 delivered up to
112% greater pain relief than pregabalin and70% more than gabapentinNo hyperlocomotion, sedation, or behavioral disturbances were observed, even at high doses
A comprehensive neurotoxicity study confirmed absence of Olney lesions, a key safety concern in NMDA modulators like ketamine
Ketamir-2 was shown to cross the blood-brain barrier efficiently, supported by its non-P-gp substrate status and evidence of CNS penetration in preclinical models, supporting oral CNS activity. Preclinical data indicate Ketamir-2 achieves good oral bioavailability-significantly higher than traditional ketamine, which has very low bioavailability when taken orally. This advantage, along with its selective receptor targeting and long-acting metabolite Nor-Ketamir-2, supports its potential as an at-home treatment option.
Demonstrated antidepressant and anxiolytic-like effects in validated behavioral models, with no dissociative or psychotomimetic side effects
Select findings were recently published in Frontiers in Pharmacology, confirming the molecule's clean pharmacological profile and positioning Ketamir-2 as a differentiated and scalable alternative to existing treatments. Additional preclinical publications are in progress.
Phase 2a Pathway and Strategic Expansion
With IND clearance secured, MIRA plans to initiate a U.S.-based Phase 2a clinical trial in neuropathic pain in Q4 2025. In parallel, the Company is:
In discussions with a leading U.S. institution to explore a dedicated trial in chemotherapy-induced peripheral neuropathy (CIPN)
Evaluating a Phase 2a study in diabetic peripheral neuropathy (DPN), a large, underserved indication with rising prevalence
Advancing formulation development for topical Ketamir-2 for localized pain applications
Neuropathic Pain: A High-Impact Opportunity Across North America
Neuropathic pain is a widespread and growing concern across North America, with prevalence expected to rise due to aging populations and increasing rates of diabetes and cancer-related treatments. Current therapies often provide only modest relief and are frequently associated with side effects such as sedation, dizziness, weight gain, and dependency risk, limiting their long-term use.
In chemotherapy-induced peripheral neuropathy (CIPN), a significant proportion of patients undergoing treatment experience chronic pain symptoms. Meanwhile, access to IV ketamine remains limited to specialized clinics with long wait times, high cost, and logistical burdens. Ketamir-2's oral, non-dissociative, and non-opioid profile may help overcome these barriers-offering a more accessible and scalable solution for patients in need.
"Ketamir-2 has demonstrated consistent, potent efficacy across preclinical pain models with a clean safety profile and broad therapeutic potential. The IND approval is a testament to our scientific rigor and may pave the way for meaningful clinical translation," said Dr. Itzchak Angel, Chief Scientific Advisor at MIRA.
Other Corporate Updates
MIRA also reports that the planned acquisition of SKNY Pharmaceuticals remains on track, with shareholder approval expected in Q3 2025. SKNY-1, the lead asset from SKNY Pharmaceuticals, is a next-generation oral therapy in development for obesity and smoking cessation. In a recent zebrafish model of obesity and craving, SKNY-1 demonstrated up to
"MIRA is executing at the highest level across all fronts," added Mr. Aminov. "Ketamir-2's clinical momentum, the SKNY-1 merger, and advancing MIRA-55 collectively reflect our commitment to delivering innovative, safe, and scalable therapies for patients with limited options. We're extremely proud of the team's performance, and we are just getting started."
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.
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
What is the significance of MIRA's FDA IND clearance for Ketamir-2?
How does Ketamir-2 compare to existing neuropathic pain treatments?
When will MIRA Pharmaceuticals complete its acquisition of SKNY Pharmaceuticals?
What are the key advantages of MIRA's Ketamir-2 over traditional ketamine?
What potential market opportunities is MIRA targeting with Ketamir-2?
