MIRA PHARMACEUTICALS, INC.

Overview

MIRA Pharmaceuticals, Inc. (NASDAQ: MIRA, the “Company”, “we”, “us”) is a clinical-stage pharmaceutical development company advancing a pipeline of novel oral therapeutics targeting neurologic, neuropsychiatric, metabolic, and addiction-related disorders. The Company holds exclusive rights in the United States, Canada, and Mexico to Ketamir-2, MIRA-55, and SKNY-1, three drug candidates designed to address significant unmet medical needs across neuropathic and inflammatory pain, central nervous system disorders, and metabolic and behavioral conditions.

Ketamir-2 is a next-generation oral NMDA receptor modulator that has completed a Phase 1 clinical trial in healthy volunteers. The study included both single-ascending-dose (SAD) and multiple-ascending-dose (MAD) cohorts, and dosing has been completed across all cohorts. Based on preliminary safety data reviewed to date, no serious adverse events or dose-limiting toxicities have been reported; however, the database remains blinded and final audited safety and pharmacokinetic analyses are ongoing. The Company is preparing to initiate a Phase 2a clinical trial in chemotherapy-induced peripheral neuropathy (CIPN) in the first half of 2026, subject to regulatory feedback and site readiness.

MIRA-55 is a novel oral, non-psychoactive cannabinoid analog under preclinical development for inflammatory pain and central nervous system–related conditions, including anxiety and cognitive impairment. In validated preclinical models, MIRA-55 has demonstrated both analgesic and anti-inflammatory activity, including restoration of pain thresholds and reduction of inflammation in inflammatory pain models. In comparative studies, the compound produced analgesic effects comparable to morphine, while also demonstrating direct anti-inflammatory activity not observed with opioid treatment under the conditions evaluated.

In a series of behavioral and safety assessments, MIRA-55 did not demonstrate cannabinoid-like central nervous system adverse effects, including sedation, catalepsy, or anxiogenic responses, at tested doses in preclinical models. These findings support a differentiated pharmacological profile designed to minimize CB1-mediated effects while maintaining therapeutic activity.

The Company is advancing MIRA-55 through IND-enabling development activities and is targeting an Investigational New Drug (IND) submission for inflammatory pain, subject to completion of required preclinical studies, manufacturing readiness, regulatory interactions, and available capital resources.

The U.S. Drug Enforcement Administration (DEA) has completed its scientific review of Ketamir-2, MIRA-55, and SKNY-1 and concluded that each compound is not currently considered a controlled substance or listed chemical under the Controlled Substances Act (CSA) and applicable regulations.

On September 29, 2025, MIRA acquired SKNY Pharmaceuticals, Inc., a Delaware corporation (“SKNY”), a private company and related party, developing SKNY-1, a preclinical-stage oral therapeutic candidate designed to modulate CB1 and CB2 receptor signaling and selectively inhibit monoamine oxidase B (MAO-B). SKNY-1 is being developed to target pathways involved in energy balance, lipid metabolism, appetite regulation, reward, and craving-related behaviors.

SKNY-1 has been evaluated in preclinical behavioral and metabolic models. In validated animal models that simulate obesity and reward-driven behavior, oral administration of SKNY-1 was associated with reductions in body weight, food consumption, and nicotine-seeking behavior compared with controls. In these studies, weight reduction was not accompanied by measurable loss of muscle mass under the conditions evaluated. Additional findings demonstrated improvements in metabolic parameters and modulation of craving-related behaviors.

In behavioral assessments, SKNY-1 did not demonstrate anxiety-like or other adverse central nervous system–related behavioral effects in the models studied, and in certain conditions mitigated CB1-related behavioral responses. These observations support a differentiated pharmacological profile relative to prior CB1-targeting agents.

The Company is conducting additional preclinical studies to further characterize SKNY-1 in models of obesity and nicotine dependence and is advancing the program toward IND-enabling development activities. Subject to completion of required studies, manufacturing readiness, regulatory interactions, and available capital resources, the Company is targeting an Investigational New Drug (IND) submission for SKNY-1 in 2026.

Ketamir-2: Selective Oral NMDA Receptor Modulator

Ketamir-2 is a patent-pending, orally bioavailable NMDA receptor modulator being developed for neuropathic pain, with an initial focus on chemotherapy-induced peripheral neuropathy (CIPN). The Company has completed dosing in a Phase 1 clinical trial in healthy volunteers, including both single-ascending-dose (SAD) and multiple-ascending-dose (MAD) cohorts. Database lock, unblinding, and final safety and pharmacokinetic analyses are ongoing.

Ketamir-2 is designed to selectively modulate the NMDA receptor (PCP binding site) with low binding affinity and limited off-target receptor activity, with the goal of improving tolerability relative to ketamine. Subject to regulatory interactions and operational readiness, the Company plans to advance Ketamir-2 into a Phase 2a clinical trial in CIPN in the first half of 2026.

MIRA-55: Oral Cannabinoid Analog

MIRA-55 is a novel oral cannabinoid analog in preclinical development for inflammatory pain and central nervous system–related conditions, including anxiety and cognitive impairment. The compound has been designed to preferentially modulate CB2 receptor activity while minimizing CB1-mediated central nervous system effects.

In preclinical models, MIRA-55 has demonstrated analgesic and anti-inflammatory activity, including restoration of pain thresholds and reduction of inflammation in inflammatory pain models. In behavioral assessments, the compound did not demonstrate cannabinoid-like central nervous system adverse effects under the conditions evaluated. The Company is advancing MIRA-55 through IND-enabling development activities and is targeting an IND submission for inflammatory pain, subject to completion of required studies and regulatory interactions.

SKNY-1: Oral Therapeutic Candidate for Metabolic and Addiction-Related Indications

SKNY-1 is a preclinical-stage oral therapeutic candidate designed to modulate CB1 and CB2 receptor signaling and selectively inhibit monoamine oxidase B (MAO-B). The compound is being developed to target pathways involved in energy balance, appetite regulation, reward, and craving-related behaviors, with initial focus on weight management and nicotine dependence.

In preclinical metabolic and behavioral models, SKNY-1 has demonstrated reductions in body weight, food intake, and nicotine-seeking behavior. In these studies, weight reduction was not associated with measurable loss of muscle mass under the conditions evaluated. Behavioral assessments did not demonstrate adverse central nervous system–related effects in the models studied. The Company is conducting additional preclinical studies and is advancing SKNY-1 toward IND-enabling development activities, with a targeted IND submission in 2026, subject to completion of required studies and regulatory interactions.

Regulatory and DEA Classification

The U.S. Drug Enforcement Administration (DEA) has completed its scientific review of Ketamir-2, MIRA-55, and SKNY-1 and concluded that each compound is not currently considered a controlled substance or listed chemical under the Controlled Substances Act (CSA) and applicable regulations. This regulatory distinction may facilitate clinical development and commercialization by avoiding certain regulatory requirements applicable to controlled substances.

Preclinical Studies and Pharmacology of Ketamir-2

We have conducted preclinical studies to characterize the pharmacological profile, safety, and therapeutic potential of Ketamir-2, an investigational compound targeting neuropathic pain and related conditions. These studies include in vitro and in vivo assessments evaluating receptor binding, efficacy, selectivity, pharmacokinetics, metabolism, general pharmacology, and toxicology.

Mechanism of Action and Receptor Selectivity

Ketamir-2 has been identified as a low-affinity NMDA receptor antagonist that selectively binds to the phencyclidine (PCP) site, with an IC50 of approximately 100 µM. Its primary metabolite, nor-Ketamir-2, also interacts with the PCP site and does not exhibit meaningful affinity for other receptor systems evaluated. This targeted receptor interaction differentiates Ketamir-2 from ketamine, which exhibits broader receptor binding, including opioid and monoaminergic receptors, and binds to NMDA receptors with significantly higher affinity.

Efficacy in Animal Models

The pharmacological activity of Ketamir-2 has been evaluated in preclinical neuropathic pain and behavioral models. In neuropathic pain models, including the Chung model, treatment with Ketamir-2 was associated with improvements in pain thresholds at multiple dose levels. In behavioral assessments, including the open-field test, elevated plus maze, and forced swim test, Ketamir-2 demonstrated activity consistent with modulation of anxiety- and depression-related pathways.

These effects were observed without evidence of significant off-target receptor activity in the models evaluated. Preclinical findings may not be predictive of clinical outcomes.

Phase 1 Clinical Evaluation

Ketamir-2 has been evaluated in a randomized, double-blind, placebo-controlled Phase 1 clinical trial in healthy volunteers, including both single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. Dosing has been completed across all cohorts, and database lock and final analyses are ongoing.

Based on safety data reviewed to date, Ketamir-2 has been observed to be generally well tolerated at the dose levels evaluated, with no serious adverse events or dose-limiting toxicities reported. No clinically significant dissociative or psychotomimetic effects typically associated with ketamine were observed.

These observations are preliminary, and final safety, tolerability, and pharmacokinetic results remain subject to completion of data analysis.

Preclinical Studies and Pharmacology of MIRA-55

We have conducted preclinical studies to evaluate the pharmacological activity of MIRA-55, a cannabinoid analog under development for inflammatory pain and central nervous system–related conditions. These studies include receptor-binding assays, behavioral models, and inflammatory pain models.

In preclinical models, MIRA-55 demonstrated activity consistent with modulation of inflammatory pathways and pain responses, including reductions in inflammation and normalization of pain thresholds in validated inflammatory pain models. In comparative studies, MIRA-55 demonstrated analgesic activity in these models without evidence of opioid-like or cannabinoid-like central nervous system adverse effects under the conditions evaluated.

These findings are based on preclinical studies, and their relevance to human clinical outcomes has not been established.

Preclinical Studies and Pharmacology of SKNY-1

We have conducted preclinical studies to evaluate the pharmacological activity of SKNY-1, a therapeutic candidate targeting metabolic and addiction-related pathways, including appetite regulation and reward-driven behaviors.

In preclinical models, including zebrafish models designed to assess obesity and craving-related behaviors, SKNY-1 administration was associated with reductions in food intake, body weight, and nicotine-seeking behavior. In these studies, weight reduction was not associated with measurable loss of muscle mass under the conditions evaluated. Additional observations included changes in metabolic parameters and behavioral responses associated with reward and craving.

Preclinical behavioral assessments did not demonstrate adverse central nervous system–related effects in the models studied.

These findings are based on preclinical studies, and their relevance to human clinical outcomes has not been established.

Recent Developments

Completion of Phase 1 Clinical Trial of Ketamir-2

In March 2026, the Company announced the completion of dosing in its Phase 1 clinical trial evaluating Ketamir-2, a proprietary oral NMDA receptor modulator, in healthy volunteers. The randomized, double-blind, placebo-controlled study included both single ascending dose (SAD) and multiple ascending dose (MAD) cohorts.

A total of 56 participants were enrolled across all cohorts. Based on safety data reviewed to date, no serious adverse events or dose-limiting toxicities have been reported at any dose level tested. In addition, no clinically significant dissociative or psychotomimetic effects typically associated with ketamine were observed.

Database lock, unblinding, and final pharmacokinetic and safety analyses are ongoing.

Grant of Bonus Cash and RSUs

On March 29, 2026, the Board and the Compensation Committee of the Board determined the certain milestone in Company’s Phase I clinical trial had been achieved. As a result, on March 29, 2026, the grant date, the Company issued Erez Aminov $80,753 in cash and 83,500 vested restricted stock units, with the restricted stock units having an aggregate fair market value of approximately $86,000.

Resignation as Director Candidate

On March 30, 2026, Kelly Stackpole informed the Company that he will not be joining the Board in the future, as was originally disclosed in connection with the Company’s acquisition of SKNY.

Planned Phase 2a Study in Chemotherapy-Induced Peripheral Neuropathy (CIPN)

The Company intends to advance Ketamir-2 into a Phase 2a clinical study in patients with chemotherapy-induced peripheral neuropathy (CIPN) under its active Investigational New Drug (IND) application. Submission of the Phase 2a protocol and supporting documentation to the U.S. Food and Drug Administration (FDA) is expected in the first half of 2026, subject to regulatory review and site readiness.

Ketamir-2 Clinical Development Strategy

Ketamir-2 is being advanced through a staged clinical development program focused on evaluating safety, pharmacokinetics, and clinical efficacy in neuropathic pain indications.

The Company has completed a Phase 1 clinical trial of Ketamir-2 in healthy volunteers, including both single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. Final data analysis is ongoing.

Following completion of Phase 1, the Company is preparing to initiate a Phase 2a proof-of-concept study in patients with chemotherapy-induced peripheral neuropathy (CIPN) under its active Investigational New Drug (IND) application.

The planned Phase 2a study is expected to evaluate safety, tolerability, and preliminary efficacy using validated neuropathic pain endpoints. The Company’s objective is to generate data to support further clinical development and potential regulatory advancement.

Future development activities may include additional studies in neuropathic pain and other central nervous system–related indications, subject to regulatory feedback and available resources.

Compensation Award to Chief Executive Officer

On March 26, 2026, following the successful completion of the Phase 1 clinical trial for Ketamir-2, the Compensation Committee of the Board of Directors approved certain performance-based compensation awards to the Company’s Chief Executive Officer, Erez Aminov, in accordance with the Company’s 2023 Equity Incentive Plan and Short-Term Incentive program.

In connection with the achievement of this clinical milestone:

These awards were granted pursuant to the Company’s previously established compensation structure, which aligns executive incentives with key clinical and strategic milestones .

Regulatory Status and Development Progress

Ketamir-2 has completed a Phase 1 clinical trial in healthy volunteers under an active Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA). Final data analysis is ongoing. The Company is preparing to advance Ketamir-2 into a Phase 2a clinical study in patients with chemotherapy-induced peripheral neuropathy (CIPN), subject to regulatory review.

MIRA-55 and SKNY-1 are currently in preclinical development. The Company is preparing to initiate IND-enabling activities, including toxicology, safety pharmacology, and manufacturing development, in support of potential future IND submissions. The timing of such activities and submissions will depend on regulatory interactions, development progress, and available resources.

Clinical Development of Ketamir-2

Ketamir-2 has been evaluated in a randomized, double-blind, placebo-controlled Phase 1 clinical trial in healthy volunteers, including both single ascending dose (SAD) and multiple ascending dose (MAD) cohorts. Dosing has been completed across all cohorts, and final data analysis is ongoing.

Based on safety data reviewed to date, Ketamir-2 has been observed to be generally well tolerated at the dose levels evaluated, with no serious adverse events or dose-limiting toxicities reported. No clinically significant dissociative or psychotomimetic effects typically associated with ketamine were observed.

The Company is preparing to initiate a Phase 2a clinical study in patients with chemotherapy-induced peripheral neuropathy (CIPN) under its active Investigational New Drug (IND) application. The planned study is expected to evaluate safety, tolerability, and preliminary efficacy using validated neuropathic pain endpoints.

Clinical Manufacturing and Supply

Recipharm Israel Ltd., a contract development and manufacturing organization (CDMO), has completed development and GMP manufacturing of Ketamir-2 for use in preclinical and clinical studies.

Preclinical Development of Mira-55

The Company has conducted a series of in vitro and in vivo preclinical studies to characterize the pharmacological activity of MIRA-55.

Receptor Pharmacology

Radio-ligand binding and functional assays have demonstrated preferential CB2 receptor activity relative to CB1 receptors, consistent with the compound’s design to limit CB1-mediated psychoactive effects.

Pain and Inflammatory Models

MIRA-55 has been evaluated in validated models of inflammatory and nociceptive pain. In a formalin-induced inflammatory pain model, oral administration of MIRA-55 restored pain thresholds to baseline levels and significantly reduced inflammation as measured by paw edema. In these studies, MIRA-55 demonstrated greater normalization of pain responses compared to morphine, which primarily affects central pain perception without directly addressing inflammation.

Behavioral and Anxiety Models

MIRA-55 has been evaluated in the Elevated Plus Maze (EPM), a validated rodent model of anxiety-related behavior. In these studies, MIRA-55-treated animals demonstrated increased time spent in open arms compared to controls, consistent with reduced anxiety-like behavior, without evidence of sedation or locomotor impairment.

Central Nervous System Safety Profile

Across a range of validated behavioral assays, including hypothermia, catalepsy, open field, and EPM testing, MIRA-55 did not demonstrate cannabinoid-like central nervous system side effects typically associated with CB1 receptor activation. No evidence of sedation, motor impairment, or anxiogenic effects was observed in preclinical studies.

Preclinical results may not be predictive of clinical outcomes.

SKNY-1 Preclinical Development

The Company has conducted a series of preclinical studies to evaluate the pharmacological activity and therapeutic potential of SKNY-1 in models of metabolic regulation, appetite, and reward-driven behavior.

Metabolic and Weight Loss Models

SKNY-1 has been evaluated in validated zebrafish models of obesity and metabolic dysfunction. In these studies, oral administration of SKNY-1 resulted in significant reductions in body weight, with treated animals demonstrating up to approximately 30% weight loss over the study period. Importantly, weight reduction was achieved without evidence of muscle loss, suggesting preservation of lean body mass.

In addition, SKNY-1 treatment was associated with normalization of metabolic parameters, including reductions in liver fat accumulation and improvements in lipid profiles, supporting its potential role in metabolic regulation.

Appetite and Behavioral Models

In preclinical models assessing feeding behavior and compulsivity, SKNY-1 demonstrated dose-dependent reductions in high-calorie food consumption and decreased food-seeking behavior under stress conditions. Treated animals showed reduced compulsive feeding patterns and improved regulation of appetite-related behaviors.

Nicotine and Reward Models

SKNY-1 has also been evaluated in models of nicotine-seeking behavior. In these studies, treatment with SKNY-1 reduced nicotine preference and decreased reward-seeking behavior associated with nicotine exposure. Behavioral responses in treated animals approached those observed in non-dependent control groups, suggesting potential activity in addiction-related pathways.

Neurobehavioral and CNS Safety Profile

In validated behavioral models assessing anxiety-related responses, SKNY-1 demonstrated reversal of CB1 agonist-induced anxiety-like behavior and did not exhibit the adverse neuropsychiatric effects associated with earlier CB1-targeting compounds. These findings support a differentiated central nervous system profile relative to prior therapies in this class.

Hormonal and Neurochemical Effects

Preclinical studies have shown that SKNY-1 modulates key metabolic and neurohormonal markers associated with appetite and reward, including normalization of leptin and ghrelin levels and modulation of dopamine signaling pathways. These findings are consistent with the compound’s proposed mechanism targeting appetite regulation and reward processing.

Preclinical results may not be predictive of clinical outcomes.

Market Opportunity and Competitive Positioning

Ketamir-2 – Neuropathic Pain

Neuropathic pain is a significant and growing health concern in the United States, affecting an estimated 7–10% of adults based on published epidemiological data. According to third-party market research, the North American neuropathic pain market is estimated in the multi-billion-dollar range and is expected to grow significantly over the remainder of the decade, driven by an aging population, increasing prevalence of diabetes, chemotherapy-induced peripheral neuropathy (CIPN), and post-surgical nerve injuries.

There are currently no therapies approved by the U.S. Food and Drug Administration specifically for CIPN, and treatment is typically limited to off-label use of antidepressants, anticonvulsants, and opioids. These therapies often provide incomplete pain relief and may be associated with tolerability and safety concerns, including sedation, cognitive impairment, and risk of dependence.

Ketamir-2 is designed as an oral NMDA receptor modulator with a differentiated safety and tolerability profile relative to ketamine, including the absence of clinically significant dissociative effects observed to date. The Company believes Ketamir-2 may offer advantages including oral administration, reduced abuse potential, and improved tolerability, positioning it as a potential alternative to existing therapies.

MIRA-55 – Inflammatory Pain

Chronic inflammatory pain represents a large and underserved global market, historically dominated by opioids and nonsteroidal anti-inflammatory drugs (NSAIDs), both of which are associated with significant safety limitations, including risk of dependence, gastrointestinal toxicity, and cardiovascular adverse effects.

According to third-party market research, the global non-opioid pain treatment market represents a large and growing commercial opportunity, with continued demand for safer and more effective alternatives to existing therapies.

MIRA-55 is designed to address both inflammation and pain through preferential CB2 receptor activity, with preclinical data demonstrating dual anti-inflammatory and analgesic effects without evidence of cannabinoid-related central nervous system side effects. The Company believes this differentiated pharmacological profile may position MIRA-55 as a potential non-opioid alternative in the treatment of inflammatory pain.

SKNY-1 – Obesity and Nicotine Addiction

Obesity and nicotine addiction are among the leading causes of preventable death globally and represent large and rapidly growing therapeutic markets.

According to third-party market research and industry analyses, the global obesity therapeutics market is projected to exceed $100 billion over the next decade and may reach significantly higher levels as demand for effective and accessible treatments continues to increase. Current therapies, including GLP-1 receptor agonists, are associated with limitations such as injectable administration, gastrointestinal side effects, and loss of lean muscle mass.

The global smoking cessation market is also substantial and is expected to continue to grow, although existing therapies are associated with modest long-term success rates and, in some cases, neuropsychiatric safety concerns.

SKNY-1 is a differentiated oral therapeutic designed to modulate CB1, CB2, and MAO-B pathways involved in appetite, reward, and metabolic regulation. In preclinical studies, SKNY-1 demonstrated significant weight loss without evidence of muscle loss, reduction in high-calorie food consumption, and reversal of nicotine-seeking behavior, along with a favorable central nervous system safety profile relative to prior CB1-targeting agents.

The Company believes SKNY-1’s oral administration, multi-target mechanism, and dual indication potential may position it as a differentiated candidate across both obesity and addiction markets.

Development Strategy

Our development strategy is focused on advancing a diversified pipeline of differentiated small-molecule therapeutics targeting neurological, neuropsychiatric, inflammatory, and metabolic disorders. We aim to generate clinically meaningful data, pursue efficient regulatory pathways, and maximize long-term value through a combination of internal development and strategic collaborations.

Ketamir-2

For Ketamir-2, our strategy is to advance clinical development in neuropathic pain, with an initial focus on chemotherapy-induced peripheral neuropathy (CIPN), a condition with significant unmet medical need and no FDA-approved therapies.

We have completed Phase 1 clinical development, including both single ascending dose (SAD) and multiple ascending dose (MAD) cohorts, and are advancing toward a Phase 2a proof-of-concept study. Our objective is to generate clinical data demonstrating safety, tolerability, and preliminary efficacy in patients.

In parallel, we intend to evaluate additional indications where NMDA receptor modulation may have therapeutic relevance, including neuropsychiatric disorders. We also plan to explore strategic partnerships to support later-stage development and potential commercialization.

MIRA-55

For MIRA-55, our strategy is to advance a differentiated cannabinoid-based therapeutic targeting inflammatory pain and related conditions.

MIRA-55 is currently progressing through IND-enabling studies, including safety pharmacology, toxicology, and manufacturing scale-up activities. Our objective is to support a potential Investigational New Drug (IND) submission and initiate clinical evaluation, subject to completion of required studies and regulatory interactions.

We plan to focus initial development on inflammatory pain indications, based on preclinical data demonstrating dual anti-inflammatory and analgesic effects without evidence of cannabinoid-related central nervous system side effects. We may also evaluate additional indications aligned with the compound’s pharmacological profile.

SKNY-1

For SKNY-1, our strategy is to advance a novel oral therapeutic targeting obesity and nicotine addiction through a multi-pathway mechanism involving CB1, CB2, and MAO-B modulation.

SKNY-1 is currently in preclinical development, and we are preparing for IND-enabling studies, including additional pharmacology, safety, and toxicology assessments. We plan to conduct further studies in relevant animal models to support its therapeutic potential in metabolic and addiction-related indications.

Our objective is to position SKNY-1 as a differentiated oral therapy with potential dual-indication utility, and we intend to evaluate strategic development and partnering opportunities as the program advances.

Strategic Focus

Across our pipeline, we intend to:

Our goal is to build a portfolio of differentiated therapeutic candidates with the potential to address large and underserved markets while maximizing long-term shareholder value.

Preclinical Research and Pharmacology

Ketamir-2

Ketamir-2 has been evaluated in a series of in vitro and in vivo preclinical studies designed to characterize its pharmacological activity, receptor selectivity, and therapeutic potential.

Preclinical studies indicate that Ketamir-2 is a low-affinity NMDA receptor antagonist that selectively binds to the phencyclidine (PCP) site, with reduced off-target receptor interactions compared to ketamine. In animal models of neuropathic pain, including established rodent models, Ketamir-2 demonstrated improvements in pain thresholds relative to baseline. Behavioral assessments also indicated activity in models commonly used to evaluate anxiety- and depression-related endpoints.

Pharmacokinetic and metabolism studies suggest that Ketamir-2 is orally bioavailable, crosses the blood-brain barrier, and is metabolized primarily through N-demethylation pathways. Preclinical findings may not be predictive of clinical outcomes.

MIRA-55

MIRA-55 has been evaluated in preclinical studies assessing receptor pharmacology, behavioral effects, and activity in pain and inflammation models.

In vitro receptor-binding and functional assays indicate that MIRA-55 exhibits preferential activity at CB2 receptors relative to CB1 receptors. In comparative studies, MIRA-55 demonstrated greater potency at CB2 receptors than certain reference cannabinoids in GPCR-based assays.

In animal models, MIRA-55 demonstrated activity in behavioral assays, including the Elevated Plus Maze, suggesting anxiolytic-like effects without evidence of sedation or motor impairment. In addition, MIRA-55 showed activity in thermal and inflammatory pain models, including increased latency in hot plate testing and reduction of inflammatory responses in established models.

Additional studies have evaluated cognitive and behavioral endpoints to assess potential central nervous system effects. Across multiple assays, MIRA-55 did not demonstrate cannabinoid-like adverse behavioral effects observed with certain CB1-active compounds. Preclinical findings may not be predictive of clinical outcomes.

SKNY-1

SKNY-1 has been evaluated in preclinical metabolic and behavioral models relevant to obesity and addiction.

In zebrafish models of obesity and craving behavior, oral administration of SKNY-1 was associated with reductions in food consumption, body weight, and nicotine-seeking behavior compared to controls. Weight reduction observed in these studies was not associated with evidence of muscle loss.

Mechanistically, SKNY-1 is designed to modulate multiple pathways involved in appetite and reward, including CB1, CB2, and MAO-B. Preclinical data suggest that SKNY-1 may influence energy balance, metabolic activity, and craving-related behaviors.

These findings support continued preclinical development of SKNY-1. Preclinical findings may not be predictive of clinical outcomes.

The Company’s product candidates are being developed to address significant unmet needs across large and growing therapeutic markets, including neuropathic pain, inflammatory pain, obesity, and nicotine dependence. These markets are characterized by limitations in existing therapies, including safety, tolerability, and long-term efficacy. The Company believes its differentiated pipeline is positioned to address these gaps.

The Company operates in highly competitive therapeutic areas, with numerous pharmaceutical and biotechnology companies developing treatments targeting neuropathic pain, inflammatory pain, obesity, and nicotine dependence. Many of these competitors have substantially greater financial, technical, and commercial resources.

Regulation

The FDA and comparable regulatory authorities in state and local jurisdictions impose substantial and burdensome requirements upon companies involved in the clinical development, manufacture, marketing, and distribution of drugs. These agencies and other federal, state, and local entities regulate, among other things, the research and development, testing, manufacture, quality control, safety, effectiveness, labeling, storage, record keeping, approval, advertising and promotion, distribution, post-approval monitoring and reporting, sampling and export and import of our drug candidates.

U.S. Government Regulation

In the United States, the FDA regulates drugs under the Federal Food, Drug, and Cosmetic Act, or FDCA, and its implementing regulations. The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local and foreign statutes and regulations requires the expenditure of substantial time and financial resources. Failure to comply with the applicable U.S. requirements at any time during the product development process, approval process or after approval, may subject an applicant to a variety of administrative or judicial sanctions, such as the FDA’s refusal to approve pending New Drug Applications (or NDAs), withdrawal of an approval, imposition of a clinical hold, issuance of warning letters, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, refusals of government contracts, restitution, disgorgement or civil or criminal penalties.

The process required by the FDA before a drug may be marketed in the United States generally involves the following:

Pre-clinical studies

Before testing any drug or biological product candidate in humans, the product candidate must undergo rigorous pre-clinical testing. The pre-clinical developmental stage generally involves laboratory evaluations of drug chemistry, formulation, and stability, as well as studies to evaluate toxicity in animals, to assess the potential for adverse events (“AEs”) and, in some cases, to establish a rationale for therapeutic use. The conduct of pre-clinical studies is subject to federal regulations and requirements, including GLP regulations for safety/toxicology studies. An IND sponsor must submit the results of the pre-clinical studies, together with manufacturing information, analytical data, any available clinical data or literature and a proposed clinical protocol, to the FDA as part of the IND.

An IND is a request for authorization from the FDA to ship an investigation product and then administer it to humans and must be allowed to proceed by the FDA before human clinical trials may begin. Some long-term pre-clinical testing, such as animal tests of reproductive AEs and carcinogenicity, may continue after the IND is submitted. An IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA raises concerns or questions before that time related to one or more proposed clinical trials and places the trial on clinical hold. In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical trial can begin. As a result, submission of an IND may not result in the FDA allowing clinical trials to commence.

Clinical trials

The clinical stage of development involves the administration of the investigational product to healthy volunteers or patients under the supervision of qualified investigators, generally physicians not employed by, or under control of, the trial sponsor, in accordance with GCPs, which include the requirement that all research patients provide their informed consent for their participation in any clinical trial. Clinical trials are conducted under protocols detailing, among other things, the objectives of the clinical trial, dosing procedures, subject selection and exclusion criteria and the parameters to be used to monitor subject safety and assess efficacy. Each protocol, and any subsequent amendments to the protocol, must be submitted to the FDA as part of the IND. Furthermore, each clinical trial must be reviewed and approved by an IRB for each institution at which the clinical trial will be conducted to ensure that the risks to individuals participating in the clinical trials are minimized and are reasonable in relation to anticipated benefits. The IRB also approves the informed consent form that must be provided to each clinical trial subject or his or her legal representative and must monitor the clinical trial until completed. There also are requirements governing the reporting of ongoing clinical trials and completed clinical trial results to public registries. Information about most clinical trials must be submitted within specific timeframes for publication on the www.clinicaltrials.gov website. Information related to the product, patient population, phase of investigation, study sites and investigators and other aspects of the clinical trial is made public as part of the registration of the clinical trial. Sponsors are also obligated to disclose the results of their clinical trials after completion. Disclosure of the results of these trials can be delayed in some cases for up to two years after the date of completion of the trial. Competitors may use this publicly available information to gain knowledge regarding the progress of development programs.

Human clinical trials are typically conducted in three sequential phases, which may overlap or be combined:

Post-approval trials, sometimes referred to as Phase IV clinical trials, may be conducted after initial marketing approval. These trials are used to gain additional experience from the treatment of patients in the intended therapeutic indication, particularly for long-term safety follow up. In certain instances, the FDA may mandate the performance of Phase IV clinical trials as a condition of approval of an NDA or a Biologics License Application (“BLA”).

Progress reports detailing the results of the clinical trials must be submitted at least annually to the FDA and more frequently if significant adverse events (“SAEs”) occur. The FDA or the sponsor may suspend or terminate a clinical trial at any time, or the FDA may impose other sanctions on various grounds, including a finding that the research patients are being exposed to an unacceptable health risk. Similarly, an IRB can refuse, suspend, or terminate approval of a clinical trial at its institution if the clinical trial is not being conducted in accordance with the IRB’s requirements or if the drug has been associated with unexpected serious harm to patients.

Concurrently with clinical trials, companies usually complete additional pre-clinical studies and must also develop additional information about the physical characteristics of the drug or biological product as well as finalize a process for manufacturing the product in commercial quantities in accordance with cGMP requirements. The manufacturing process must be capable of consistently producing quality batches of the product candidate and, among other things, the sponsor must develop methods for testing the identity, strength, quality, potency, and purity of the final biological product. Additionally, appropriate packaging must be selected and tested, and stability studies must be conducted to demonstrate that the biological product candidate does not undergo unacceptable deterioration over its shelf life.

Marketing Approval

Assuming successful completion of the required clinical testing, the results of the pre-clinical studies and clinical trials, together with detailed information relating to the product’s chemistry, manufacture, controls, and proposed labeling, among other things, are submitted to the FDA as part of an NDA requesting approval to market the product for one or more indications. In most cases, the submission of an NDA is subject to a substantial application user fee.

The review process typically takes twelve months from the date the NDA is submitted to the FDA. The FDA conducts a preliminary review of all NDAs within the first 60 days after submission to determine whether they are sufficiently complete to permit substantive review before accepting them for “filing.” The FDA may request additional information rather than accept an NDA for filing. In this event, the application must be resubmitted with the additional information and may be subject to an additional application user fee. The resubmitted application is also subject to review before the FDA accepts it for filing. Once the submission is accepted for filing, the FDA begins an in-depth substantive review. The FDA reviews an NDA to determine, among other things, whether the drug is safe and effective and whether the facility in which it is manufactured, processed, packaged, or held meets standards designed to assure the product’s continued safety, quality and purity. Under the current guidelines in effect in the Prescription Drug User Fee Act (PDUFA), the FDA has a goal to review and act on the submission within ten months from the completion of the preliminary review of a standard NDA for a new molecular entity.

The FDA also may require submission of a REMS plan to ensure that the benefits of the drug outweigh its risks. The REMS plan could include medication guides, physician communication plans, assessment plans, and/or elements to assure safe use, such as restricted distribution methods, patient registries, or other risk minimization tools.

The FDA may refer an application for a novel drug to an advisory committee. An advisory committee is a panel of independent experts, including clinicians and other scientific experts, that reviews, evaluates and provides a recommendation as to whether the application should be approved and under what conditions. The FDA is not bound by the recommendations of an advisory committee, but it considers such recommendations carefully when making decisions.

Before approving an NDA, the FDA typically will inspect the facility or facilities where the product is manufactured. The FDA will not approve an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assure consistent production of the product within required specifications. Additionally, before approving an NDA, the FDA may inspect one or more clinical trial sites to assure compliance with GCP requirements.

After evaluating the NDA and all related information, including the advisory committee recommendation, if any, and inspection reports regarding the manufacturing facilities and clinical trial sites, the FDA may issue an approval letter, or, in some cases, a complete response letter. A complete response letter generally contains a statement of specific conditions that must be met in order to secure final approval of the NDA and may require additional clinical trials or pre-clinical studies in order for FDA to reconsider the application. Even with submission of this additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteria for approval. If and when those conditions have been met to the FDA’s satisfaction, the FDA will typically issue an approval letter. An approval letter authorizes commercial marketing of the drug with specific prescribing information for specific indications.

Post-approval requirements

Drugs manufactured or distributed pursuant to FDA approvals are subject to pervasive and continuing regulation by the FDA, including, among other things, requirements relating to recordkeeping, periodic reporting, product sampling and distribution, advertising and promotion and reporting of adverse experiences with the product. After approval, most changes to the approved product, such as adding new indications or other labeling claims are subject to prior FDA review and approval. There also are continuing annual user fee requirements for any marketed products and the establishments at which such products are manufactured, as well as new application fees for supplemental applications with clinical data.

Intellectual Property

KETAMIR-2

We license the U.S., Canadian, and Mexican patent rights for the use of KETAMIR-2 in human applications from MIRALOGX. MIRALOGX filed international application no. PCT/US2024/018594 under the Patent Cooperation Treaty (PCT) on March 6, 2024, titled, ANTIDEPRESSANT COMPOUNDS, PHARMACEUTICAL COMPOSITIONS, AND METHODS OF TREATING DEPRESSION AND OTHER DISORDERS, and in due course intends to enter the national phase in the United States, Canada and Mexico, among other countries. These applications, if granted and subject to payment of patent maintenance fees, would offer protection extending through at least March 6, 2044. The patent rights for KETAMIR-2 outside of the United States, Canada, and Mexico are not included in our current patent rights.

Our license from MIRALOGX is set forth in the Exclusive License Agreement, dated November 15, 2023, pursuant to which the licensed field of use includes therapeutic treatments and other medical or health uses in humans, and related preclinical studies and activities conducted in furtherance of obtaining regulatory approval for and commercialization of human therapeutic treatments and uses (the “MIRALOGX License Agreement”). “Licensed Product” is defined as a drug product containing as an active agent 2-(2-chlorophenyl)-2-(methylamino)cyclopentan-1-one or a pharmaceutically acceptable salt or ester thereof. We also have the right to grant corresponding sublicenses under the licensed patent rights. The MIRALOGX License Agreement provides for the payment to MIRALOGX of an 8% royalty (payable quarterly) on our net sales of Licensed Products by us or our sublicensees and on non-royalty bearing milestone revenue, with the royalty obligation ceasing upon the later of the expiration of the last-to-expire licensed patent. The agreement also provides for an up-front Cost Reimbursement of $100,000 payable to MIRALOGX to cover the already-incurred costs associated with the patent rights. The Cost Reimbursement is the only payment made to date under the agreement. MIRALOGX may terminate the agreement upon insolvency, an uncured breach including the failure to make any payment owed under the agreement or the failure to use commercially reasonable efforts to develop the licensed product, or upon a default of the November 15, 2023 Promissory Note and Loan Agreement. The MIRALOGX License Agreement provides that MIRALOGX will have sole control over the filing, prosecution, maintenance, and management of the licensed patent rights, provided that we will be responsible for the cost of prosecuting and maintaining the licensed patents. The agreement grants to us the primary right, but not the obligation, to enforce the licensed patent rights.

Besides relying on patents, we also rely on trade secrets, proprietary know-how and continuing innovation to develop and maintain our competitive position, especially when we do not believe that patent protection is appropriate or can be obtained. We seek protection of these trade secrets, proprietary know-how and any continuing innovation, in part, through confidentiality and proprietary information agreements. However, these agreements may not provide meaningful protection for, or adequate remedies to protect, our technology in the event of unauthorized use or disclosure of information. Furthermore, our trade secrets may otherwise become known to, or be independently developed by, our competitors. We intend to seek appropriate patent protection for technology in our research and development programs, where applicable, and their uses by filing patent applications in the United States and other selected countries. We intend for these patent applications to cover, where possible, claims for compositions of matter, medical uses, processes for preparation and formulations.

MIRA-55

We have a pending provisional patent application directed to MIRA-55, a structure that was synthesized and isolated during the research and development of MIRA1a titled “Synthetic Cannabinoid Analogs, Pharmaceutical Compositions and Methods of Treating Anxiety and Other Disorders”. The Company intends to pursue domestic and foreign filings based on the provisional application to seek global patent protection for MIRA-55.

MIRA1a

The U.S. Patent 10,787,675 B2, titled “Purified Synthetic Marijuana and Methods of Treatment by Administering Same,” which covers the MIRA1a compoundper se as a racemic mixture, an isolated R-enantiomer, or an isolated S-enantiomer, as well as pharmaceutical formulations of the compound, was assigned to our Company by SRQ Patent Holdings II, LLC (“SRQ”) in December 2021. This patent also covers MIRA1a in methods of treating Alzheimer’s disease, anxiety, depression, and addictions. Subject to payment of patent maintenance fees, the ‘675 patent offers protection extending through at least February 11, 2039. According to the assignment and royalty agreement, we owe 8% in royalty revenue on net sales price and royalty revenue and 8% of milestone payment revenue to SRQ.

The royalties shall continue, in each country on a product-by-product and country-by-country basis until the later of i) the date of expiration of the last to expire patent included within the Innovation, or ii) the date of expiration of the last strategic partnership/licensing agreement including the Innovation.

We currently have no plans to develop the MIRA1a compound for approval and commercialization in or outside of the United States. See “Risk Factors- Risks Related to Our Intellectual Property- We own the rights associated with our patents in the United States, but we do not own the rights to patents covering MIRA1a in foreign jurisdictions.”

SKNY-1

We hold a license in the U.S. and its territories, Mexico and Canada from MIRALOGX for the use of SKNY-1 in human applications. SKNY has filed international application no. PCT/US25/17127 under the Patent Cooperation Treaty (PCT) on February 25, 2025, titled “SYNTHETIC CANNABINOID ANALOGS, PHARMACEUTICAL COMPOSITIONS, AND METHODS OF TREATING ANXIETY AND OTHER DISORDERS,” and hold patent no. 63/653,326 on 5/30/24, titled “SYNTHETIC CANNABINOID ANALOGS, PHARMACEUTICAL COMPOSITIONS, AND METHODS OF TREATING BACTERIAL INFECTIONS AND VIRAL INFECTIONS,” and in due course intends to enter the national phase in the United States, among other countries. These applications, if granted and subject to payment of patent maintenance fees, would offer protection extending through at least February 25, 2045, and May 30, 2044, respectively. The patent rights for SKNY-1 outside of the United States are not included in the SKNY-1 licensing agreement.