MIRA Pharmaceuticals Announces Favorable Topline Results from Phase 1 SAD Study of Oral Ketamir-2, a Next-Generation Non-Scheduled Ketamine Analog
MIRA Pharmaceuticals (NASDAQ:MIRA) announced positive topline results from its Phase 1 single ascending dose (SAD) study of Ketamir-2, its novel oral ketamine analog for neurologic disorders. The study, involving 32 healthy adults across four dose cohorts (50-600mg), demonstrated favorable safety and pharmacokinetic profiles.
Key findings include dose-proportional exposure, rapid absorption within 1-2 hours, and a terminal half-life of 2-5 hours for Ketamir-2, with its active metabolite showing 6.5-8.5 hours half-life. The drug was well-tolerated with no serious adverse events or typical ketamine-associated CNS side effects, supporting once-daily dosing potential.
MIRA is proceeding with the multiple ascending dose (MAD) portion of the Phase 1 study, followed by a planned Phase 2a trial in neuropathic pain patients.
MIRA Pharmaceuticals (NASDAQ:MIRA) ha annunciato risultati positivi di topline dal suo studio di fase 1 con dosi singole ascendenti (SAD) di Ketamir-2, il suo nuovo analogo orale della ketamina per disturbi neurologici. Lo studio, che ha coinvolto 32 adulti sani in quattro coorti di dose (50-600 mg), ha mostrato profili di sicurezza e farmacocinetica favorevoli.
Tra i principali risultati vi sono esposizione proporzionale alla dose, assorbimento rapido entro 1-2 ore e un'emivita terminale di 2-5 ore per Ketamir-2, con il suo metabolita attivo che mostra un'emivita di 6,5-8,5 ore. Il farmaco è stato ben tollerato con nessun evento avverso grave o tipici effetti collaterali a carico del sistema nervoso centrale associati alla ketamina, supportando un potenziale dosaggio una volta al giorno.
MIRA sta proseguendo con la porzione di dosi ascendenti multiple (MAD) dello studio di fase 1, seguita da uno studio di fase 2a pianificato in pazienti affetti da dolore neuropatico.
MIRA Pharmaceuticals (NASDAQ:MIRA) anunció resultados positivos de topline de su estudio de fase 1 de dosis única ascendente (SAD) de Ketamir-2, su nuevo análogo oral de ketamina para trastornos neurológicos. El estudio, que involucró a 32 adultos sanos en cuatro cohortes de dosis (50-600 mg), mostró perfiles de seguridad y farmacocinética favorables.
Entre los hallazgos clave se encuentran exposición proporcional a la dosis, rápida absorción en 1-2 horas y una vida media terminal de 2-5 horas para Ketamir-2, con su metabolito activo mostrando una vida media de 6,5-8,5 horas. El fármaco se toleró bien sin eventos adversos graves ni efectos secundarios típicos del sistema nervioso central asociados a la ketamina, respaldando un potencial de dosificación una vez al día.
MIRA continúa con la parte de dosis múltiples ascendentes (MAD) del estudio de fase 1, seguida de un ensayo planificado de fase 2a en pacientes con dolor neuropático.
MIRA Pharmaceuticals(NASDAQ:MIRA)가 신경계 질환 için 개발 중인 새로운 경구용 케타민 유사체 Ketamir-2의 1상 SAD(단일 상승 용량) 연구에서 긍정적인 topline 결과를 발표했습니다. 4개의 용량 코호트(50-600mg)에서 32명의 건강한 성인이 참여한 이 연구는 안전성 및 약물동역학(PK) 프로파일이 우수함을 보였습니다.
주요 결과로는 용량 의존적 노출, 1-2시간 내 빠른 흡수, Ketamir-2의 말기 반감기가 2-5시간인 반면 활성 대사체의 반감기는 6.5-8.5시간으로 나타났습니다. 이 약은 두통성 CNS 부작용 등 일반적인 케타민 관련 부작용 없이 양호하게 내약성이 입증되었고, 하루 한 번 투여 가능성을 시사합니다.
MIRA는 1상 연구의 MAD(다회투여 상승) 부분을 진행하고 있으며, 신경병성 통증 환자를 대상으로 한 2a상 시험이 예정되어 있습니다.
MIRA Pharmaceuticals (NASDAQ:MIRA) a annoncé des résultats positifs de topline de son étude de phase 1 en dose unique croissante (SAD) de Ketamir-2, son nouvel analogue oral de la kétamine destiné aux troubles neurologiques. L’étude, impliquant 32 adultes sains répartis sur quatre cohorts de dose (50-600 mg), a démontré des profils de sécurité et de pharmacocinétique favorables.
Les résultats clés incluent une exposition proportionnelle à la dose, une absorption rapide en 1-2 heures et une demi-vie terminale de 2-5 heures pour Ketamir-2, tandis que son métabolite actif présente une demi-vie de 6,5-8,5 heures. Le médicament a été bien toléré avec AUC et sans événements indésirables graves ni d’effets indésirables centraux typiques de la kétamine, soutenant un potentiel de posologie une fois par jour.
MIRA poursuit la partie MAD (doses ascendantes multiples) de l’étude de phase 1, suivie d’un essai de phase 2a prévu chez des patients souffrant de douleur neuropathique.
MIRA Pharmaceuticals (NASDAQ:MIRA) gab positive Topline-Ergebnisse aus ihrer Phase-1-Studie mit aufsteigender Einzeldosis (SAD) von Ketamir-2, ihrem neuen oralen Ketamin-Analogon für neurologische Erkrankungen, bekannt. Die Studie umfasste 32 gesunde Erwachsene in vier Dosis-Cohorten (50-600 mg) und zeigte ein vorteilhaftes Sicherheits- und pharmakokinetisches Profil.
Zu den wichtigsten Erkenntnissen gehören dosisproportionale Exposition, rasche Absorption innerhalb von 1-2 Stunden und eine terminale Halbwertszeit von 2-5 Stunden für Ketamir-2, während der aktive Metabolit eine Halbwertszeit von 6,5-8,5 Stunden aufweist. Das Medikament wurde gut vertragen, mit keinen schweren unerwünschten Ereignissen oder typischen kognitiv-zentrumsbedingten Nebenwirkungen der Ketamin, was ein Potenzial für eine einmal tägliche Dosierung unterstützen könnte.
MIRA setzt die MAD-Teil des Phase-1-Studiums fort, gefolgt von einer geplanten Phase-2a-Studie bei Patienten mit neuropathischen Schmerzen.
MIRA Pharmaceuticals (NASDAQ:MIRA) أعلنت نتائج موجزة إيجابية من دراسة المرحلة 1 من جرعات وحيدة متزايدة (SAD) لـ Ketamir-2، نظير Ketamine الفموي الجديد لعلاج الاضطرابات العصبية. شملت الدراسة 32 بالغاً سليماً عبر أربع مجموعات جرعات (50-600 ملغ)، وأظهرت ملفات أمان وفارماكوكينيتك مواتية.
من أبرز النتائج وجود تعرض جرعاتي نسبياً للجرعة، وامتصاص سريع خلال 1-2 ساعة، ونصف عمر حاسم لـ Ketamir-2 يتراوح بين 2-5 ساعات، بينما يظهر المستقلب النشط نصف عمر 6.5-8.5 ساعات. تم تحمل الدواء جيداً دون أحداث سلبية خطيرة أو آثار جانبية مركزية مرتبطة بالكينتامينه، مما يدعم إمكانية الجرعة مرة واحدة يومياً.
تستمر MIRA في الجزء MAD من دراسة المرحلة 1، تليها تجربة المرحلة 2a مخطط لها في مرضى الألم العصبي المحيّز.
MIRA Pharmaceuticals (NASDAQ:MIRA) 宣布其用于神经系统疾病的新型口服型氯胺类似物 Ketamir-2 的第一阶段单次剂量递增（SAD）研究的 topline 积极结果。该研究涉及 32名健康成年志愿者，分4组剂量（50-600 mg），显示了有利的安全性和药代动力学特征。
关键发现包括 剂量依赖性暴露、在1-2小时内快速吸收，以及 Ketamir-2 的终末半衰期为 2-5 小时，其活性代谢物的半衰期为 6.5-8.5 小时。该药物耐受性良好，未出现 严重不良事件 或典型的与氯胺相关的中枢神经系统副作用，支持每日一次给药的潜力。
公司正在推进该研究的第一阶段 MAD（多剂量递增）部分，随后计划在神经痛患者中开展第二阶段 2a 研究。
- Drug demonstrated favorable safety profile with no serious adverse events across all dose levels
- Successful achievement of predictable pharmacokinetics supporting once-daily dosing
- No CNS side effects typically associated with ketamine were observed
- Clear pathway to Phase 2a trials established
- Company actively exploring strategic partnering opportunities
- Early-stage clinical development with significant future trials required
- Potential competition from existing ketamine treatments
- Strategic partnerships not yet secured
Insights
MIRA's Ketamir-2 shows promising Phase 1 safety profile and advantageous PK characteristics for potential neuropathic pain treatment.
The Phase 1 SAD study results for MIRA Pharmaceuticals' Ketamir-2 represent a significant early clinical milestone. The trial demonstrated dose-proportional pharmacokinetics across all tested levels (50mg-600mg), with rapid absorption (Tmax 1-2 hours) and a favorable half-life profile. Most notably, the primary active metabolite, nor-Ketamir, showed a 6.5-8.5 hour half-life, supporting once-daily dosing — a substantial advantage over traditional oral ketamine which suffers from poor bioavailability and short duration.
The clean safety profile is particularly compelling. Across all dose cohorts, Ketamir-2 exhibited no serious adverse events or dose-limiting toxicities. Critically, specialized CNS assessments (using validated tools like C-SSRS, Bowdle VAS, and KSET) confirmed the absence of ketamine's characteristic dissociative and psychotomimetic effects — a differentiating feature that addresses a major limitation of conventional ketamine therapy.
The progression to multiple ascending dose (MAD) studies is appropriate given these results. For chronic conditions like neuropathic pain, demonstrating tolerability with repeated dosing will be essential. The absence of addictive potential and severe CNS effects positions Ketamir-2 favorably in the analgesic landscape, where non-opioid alternatives for neuropathic pain remain a significant unmet need. The company's mentioned interest in strategic partnerships suggests recognition that larger resources may accelerate later-phase development in this competitive therapeutic area.
Study demonstrated Ketamir-2 was safe and well tolerated at all dose levels, with a favorable safety and tolerability profile, with no severe or clinically significant adverse effects observed. The drug showed rapid and predictable absorption, a favorable duration of action supporting once-daily dosing, and no CNS side effects typically seen with ketamine.
MIAMI, FLORIDA / ACCESS Newswire / September 22, 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 topline results from the single ascending dose (SAD) portion of its ongoing Phase 1 clinical trial evaluating the safety, tolerability, and pharmacokinetics (PK) of its lead oral candidate, Ketamir-2, in healthy volunteers.
The randomized, placebo-controlled study enrolled 32 healthy adult participants across four escalating oral dose cohorts (50 mg to 600 mg). The primary endpoints were safety, tolerability, and PK characterization.
Key Pharmacokinetic Findings
Dose-proportional increases in exposure (Cmax and AUC) were observed across all dose levels tested.
Median time to maximum plasma concentration (Tmax) reached within 1-2 hours, consistent across cohorts, confirming rapid and predictable absorption.
Terminal half-life (t½) of Ketamir-2 ranged from 2-5 hours, while its primary active metabolite, nor-Ketamir, demonstrated a longer half-life of 6.5-8.5 hours. This favorable duration of action supports convenient once-daily dosing and may contribute to sustained therapeutic benefit.
This contrasts with oral ketamine, which is characterized by erratic absorption and a much shorter half-life, limiting its clinical use in chronic treatment. The predictable PK profile of Ketamir-2 supports convenient once-daily dosing for patients with neuropathic pain and potentially other CNS conditions.
Safety and Tolerability
Ketamir-2 was generally safe and well tolerated across all four cohorts
No dose-limiting toxicities or serious adverse events were observed
Treatment-emergent adverse events were transient and resolved without intervention
In addition to routine safety assessments, CNS effects were carefully monitored using validated tools (C-SSRS, Bowdle VAS, KSET). Across all SAD cohorts, Ketamir-2no clinically significant adverse effects observed.
Next Steps
Based on the data, MIRA is initiating the multiple ascending dose (MAD) portion of the Phase 1 study in healthy volunteers, to be followed by a Phase 2a trial in patients with neuropathic pain.
Management Commentary
"These first-in-human data clearly demonstrate a favorable safety profile and predictable pharmacokinetics across a wide dosing range," said Erez Aminov, CEO of MIRA. "Importantly, the absence of the hallmark CNS side effects typically associated with ketamine, which we first observed preclinically, has now been confirmed clinically - reinforcing Ketamir-2's differentiated profile. Given the significant unmet need for safe, effective, and non-addictive treatments for neuropathic pain, we believe Ketamir-2 is well positioned as a highly attractive development candidate, and the Company is actively exploring strategic partnering opportunities to accelerate its advancement."
Dr. Itzchak Angel, CSA of MIRA, added: "The Phase 1 pharmacokinetic data demonstrate that Ketamir-2 is rapidly absorbed, predictably metabolized, and suitable for once-daily dosing - a major advantage compared to oral ketamine, which suffers from poor bioavailability and short half-life. Importantly, we were able to confirm in a human clinical study that Ketamir-2 continues to differentiate itself from ketamine by showing a robust safety and pharmacokinetics profile well adapted for its intended use. These findings underscore its potential as a next-generation therapeutic."
About Ketamir-2
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:
Neuropathic pain: Superior efficacy upon oral administration versus ketamine, pregabalin, and gabapentin in gold-standard models, without the dissociative side effects associated with ketamine.
PTSD: In the validated Single Prolonged Stress (SPS) model, Ketamir-2 restored normalized behavior in stressed animals, reversing hallmark PTSD-like behaviors consistent with non-stressed controls. A larger follow-on PTSD study is ongoing.
Depression: Activity in established preclinical models supports the potential of Ketamir-2 as a differentiated treatment for major depressive disorders, including treatment-resistant depression (TRD).
Inflammatory pain (topical formulation): In animal models, a topical formulation of Ketamir-2 demonstrated pain relief equal to injected morphine, underscoring its versatility and non-opioid potential across pain indications.
The U.S. Drug Enforcement Administration's scientific review of Ketamir-2 concluded that it would not be considered a controlled substance or listed chemical under the Controlled Substances Act and its governing regulations.
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 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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