Inidascamine (RL-007/FSV7-007): A Comprehensive Report on its Development for Cognitive Impairment Associated with Schizophrenia

1. Executive Summary

Inidascamine, also known by its developmental codes RL-007 and FSV7-007, is an orally administered, investigational neuromodulator currently in advanced clinical development for the treatment of Cognitive Impairment Associated with Schizophrenia (CIAS).[1] CIAS represents a significant unmet medical need, as there are currently no FDA-approved treatments for this debilitating aspect of schizophrenia.[3] Developed by Recognify Life Sciences, a subsidiary of atai Life Sciences, Inidascamine is distinguished by its multi-target mechanism of action, putatively modulating cholinergic, NMDA, and GABA-B receptor systems to enhance synaptic plasticity and cognition.[1]

The compound has a history of investigation, including earlier phase studies under the code FSV7-007, with over 500 unique subjects dosed, where pro-cognitive signals were observed.[2] This foundation has led to a focused development program for CIAS. A Phase 2a proof-of-mechanism study in CIAS patients demonstrated a clinically meaningful pro-cognitive profile and target engagement via qEEG biomarkers, alongside good tolerability.[7]

Currently, Inidascamine is being evaluated in a Phase 2b, randomized, double-blind, placebo-controlled, multi-arm proof-of-concept trial (NCT05686239) in approximately 234 CIAS patients.[3] This study, utilizing the FDA-supported MATRICS Consensus Cognitive Battery (MCCB) as its primary endpoint, is expected to yield topline results in mid-2025.[9] Preclinical data have consistently shown pro-cognitive effects in various animal models and a favorable safety profile, including good oral bioavailability and brain penetration in rats without typical sedative effects associated with GABAergic modulation.[6] The unique, potentially indirect, modulatory mechanism and the consistent pro-cognitive signals, coupled with a generally well-tolerated profile, position Inidascamine as a promising candidate for addressing the significant burden of CIAS.

2. Introduction to Inidascamine (FSV7-007/RL-007)

Inidascamine is an investigational small molecule being developed as a novel therapeutic agent. Its identity and the context of its primary therapeutic target are crucial for understanding its potential.

2.1. Nomenclature and Chemical Identity

The compound is known by several names reflecting its developmental journey. Its International Nonproprietary Name (INN) is Inidascamine.[1] The primary developmental code currently used by its developers, atai Life Sciences and its subsidiary Recognify Life Sciences, is RL-007.[1] An earlier developmental code, particularly during its time under Allergan, was FSV7-007.[2] These different identifiers are important to recognize when reviewing historical and current literature on the compound.

Chemically, Inidascamine is precisely defined by its CAS Number, 903884-71-9.[1] Its molecular formula is C12​H17​N3​O2​, corresponding to a molar mass of approximately 235.28 g/mol (reported as 235.287 g·mol⁻¹ or 235.28 g/mol depending on the source).[1] The systematic IUPAC name for Inidascamine is (2R,3S)-2-amino-3-hydroxy-3-(pyridin-4-yl)-1-(pyrrolidin-1-yl)propan-1-one, which describes its specific stereochemistry and functional groups.[1] This defined stereochemistry (absolute, with 2 defined stereocenters) is critical, as biological activity is often highly dependent on the spatial arrangement of atoms; indeed, its enantiomer, RL-007-03, is reported to be 100-fold less potent in certain preclinical assays.[6] Structurally, Inidascamine has been noted to share similarities with phenethylamines and amphetamines, although its primary pharmacological mechanism is distinct from typical stimulants.[1] This structural observation might warrant consideration for potential off-target activities, though current preclinical safety data appear favorable in this regard.[6]

Table 1: Key Identifiers of Inidascamine

Identifier Type	Value	Key Snippet(s)
INN	Inidascamine	1
Current Developmental Code	RL-007	1
Former Developmental Code	FSV7-007	2
CAS Number	903884-71-9	1
Molecular Formula	C12​H17​N3​O2​	1
Molar Mass	235.287 g·mol<sup>−1</sup> / 235.28 g/mol	1
IUPAC Name	(2R,3S)-2-amino-3-hydroxy-3-(pyridin-4-yl)-1-(pyrrolidin-1-yl)propan-1-one	1
Key Synonyms	RL007	1
UNII	3LW01V88B7	1
ChEMBL ID	ChEMBL5095258	1

2.2. Therapeutic Rationale: Cognitive Impairment Associated with Schizophrenia (CIAS)

The primary therapeutic indication for Inidascamine is Cognitive Impairment Associated with Schizophrenia (CIAS).[1] CIAS encompasses a range of deficits in attention, working memory, verbal learning, executive function, and processing speed, which are core features of schizophrenia.[14] These cognitive deficits are major determinants of functional disability, impacting patients' ability to engage in work, social interactions, and independent living.[7] Approximately 80% of individuals with schizophrenia experience CIAS, highlighting the widespread nature of this problem.[3]

A critical aspect of the therapeutic rationale for developing Inidascamine for CIAS is the profound unmet medical need. Currently, there are no pharmacological treatments approved by the U.S. Food and Drug Administration (FDA) or other major regulatory agencies specifically for CIAS.[3] Existing antipsychotic medications primarily target psychotic symptoms (e.g., hallucinations, delusions) but have limited to no efficacy in improving cognitive deficits. This therapeutic gap leaves a large patient population with persistent functional impairments, underscoring the potential impact of a novel, effective pro-cognitive agent like Inidascamine.

3. Development History and Corporate Affiliations

The development of Inidascamine has involved several corporate entities, reflecting a strategic evolution in its therapeutic focus and a common pathway for pharmaceutical asset progression.

3.1. Discovery and Initial Licensing

Inidascamine was originally discovered through phenotypic screening aimed at identifying compounds with effects on synaptic plasticity and cognition.[1] The compound, then known by the developmental code FSV7-007, was under the purview of Allergan, a major pharmaceutical company. Subsequently, Allergan exclusively licensed FSV7-007 to Recognify Life Sciences.[2] This transition from a large pharmaceutical entity to a more specialized biotechnology company often occurs when an asset shows promise but may not align perfectly with the larger company's immediate strategic priorities or requires a more focused development effort.

3.2. Role of Recognify Life Sciences

Recognify Life Sciences, notably co-founded by Dr. Thomas Südhof, a 2013 Nobel laureate in Physiology or Medicine for his work on vesicle trafficking (a key component of synaptic function), took on the development of FSV7-007, which was then referred to as RL-007.[2] Recognify's initial focus was on developing RL-007 for a range of neurodegenerative and neurocognitive diseases.[9] The involvement of a scientist of Dr. Südhof's caliber lent considerable scientific credibility to the compound and its underlying mechanism related to synaptic function. It was during this period, or from data generated under Allergan, that early human studies, including a Phase II trial for peripheral neuropathic pain, revealed pro-cognitive effects as exploratory endpoints, likely catalyzing the shift towards a primary cognitive indication.[2]

3.3. Acquisition and Current Development by atai Life Sciences

In a significant strategic move in January 2021, atai Life Sciences, a clinical-stage biopharmaceutical company specializing in mental health disorders, acquired a majority stake in Recognify Life Sciences.[2] This acquisition was specifically aimed at accelerating the development of RL-007 for the treatment of CIAS.[2] Atai Life Sciences provides substantial resources, including funding, enabling technologies, supportive infrastructure, and deep expertise in CNS drug development and regulatory pathways, to the RL-007 program.[2] Recognify Life Sciences now operates as a subsidiary of atai and is the entity formally conducting the ongoing clinical trials for RL-007, including the pivotal Phase 2b study.[3]

This development trajectory, from a large pharmaceutical company to a focused biotech and then into the portfolio of a specialized mental health platform company, is indicative of a strategic refinement process. The initial broad exploration of FSV7-007's potential likely narrowed as pro-cognitive signals emerged, particularly from the Phase II study in neuropathic pain where cognitive improvements were noted.[2] Such repurposing or refocusing based on emerging human data is a common and often fruitful strategy in drug development, allowing a compound to find its optimal therapeutic niche. The substantial prior human exposure of RL-007 (508 subjects across nine studies before atai's focused CIAS efforts [2]) provided a significant de-risking element, offering a foundational understanding of its safety and tolerability in humans, which is invaluable when embarking on development for a complex indication like CIAS.

4. Mechanism of Action and Pharmacological Profile

Inidascamine (RL-007) is characterized as a neuromodulator with a unique multi-target mechanism of action, aimed at enhancing cognitive functions by influencing key neurotransmitter systems and promoting synaptic plasticity.[1]

4.1. Multi-Target Neuromodulation

The primary proposed mechanism of Inidascamine involves the modulation of three critical neurotransmitter systems known to be central to learning, memory, and overall cognitive processes [1]:

• Cholinergic System: Inidascamine is reported to synergize with acetylcholine, potentially boosting cholinergic signaling.[5] The cholinergic system plays a well-established role in attention, learning, and memory.
• NMDA (Glutamatergic) System: The compound modulates the N-methyl-D-aspartate (NMDA) receptor system.[1] NMDA receptors are crucial for synaptic plasticity, including LTP, which is a cellular correlate of learning and memory.
• GABA-B Receptor System: Inidascamine influences the gamma-aminobutyric acid type B (GABA-B) receptor system.[1] GABA is the primary inhibitory neurotransmitter in the brain, and GABA-B receptors are involved in modulating neuronal excitability and synaptic transmission.

By acting on these systems, Inidascamine is believed to alter the excitatory/inhibitory balance in the brain, leading to its pro-cognitive effects.[3]

4.2. Enhancement of Synaptic Plasticity

A core element of Inidascamine's pharmacological profile is its ability to enhance synaptic plasticity, a fundamental process for learning and memory formation. Preclinical studies have shown that:

• RL-007 potently facilitates basal excitatory synaptic transmission in hippocampal slices.[6]
• It significantly enhances the induction of Long-Term Potentiation (LTP) in these ex vivo preparations.[2] LTP is a long-lasting enhancement in signal transmission between two neurons that results from stimulating them synchronously and is widely considered a key cellular mechanism underlying learning and memory.
• The effect of RL-007 on both basal neurotransmission and LTP exhibits an inverted U-shaped concentration-response curve, with maximal enhancement observed at concentrations between 10 nM and 100 nM, while lower (1 nM) and higher (1000 nM) concentrations were ineffective.[6] This non-linear dose-response is an important characteristic, suggesting that optimal therapeutic effects may occur within a specific exposure range.

4.3. Nuanced Interaction with GABA-B and Cholinergic Systems

Further investigation into RL-007's interaction with the GABA-B system has revealed a particularly interesting and potentially novel mechanism. While the pro-plasticity effects of RL-007 in hippocampal slices are occluded by GABA-B receptor antagonists (such as CGP-55845 and Saclofen), indicating that endogenous GABA-B receptor tone is necessary for RL-007's action, in vitro binding and functional assays showed that RL-007 does not directly interact with GABA-B receptors or GABA transporters.[6] This suggests that RL-007 does not act as a classical GABA-B agonist or antagonist but rather through an indirect mechanism that is dependent on a permissive GABA-B signaling environment. This indirect modulation may explain its favorable tolerability profile, particularly the lack of sedation typically associated with direct GABA-B agonists.[6]

Regarding the cholinergic system, while RL-007 is reported to synergize with acetylcholine to boost cholinergic signaling [5], its ability to potentiate LTP in hippocampal slices was found to be independent of cholinergic afferents (i.e., it worked even when cholinergic inputs were ablated).[6] This implies that its direct effects on synaptic plasticity are not solely reliant on enhancing cholinergic transmission, although such enhancement may contribute to its overall pro-cognitive profile in vivo.

4.4. Receptor Specificity and Stereoselectivity

In vitro screening against a broad panel of over 400 known CNS targets indicated that RL-007 does not directly interact with these common receptors or transporters.[6] This lack of promiscuous binding, coupled with its unique GABA-B-dependent but indirect action, suggests that RL-007 may act via a novel pharmacological target or pathway to exert its effects on synaptic plasticity.

The pharmacological activity of Inidascamine is also stereo-specific. Its enantiomer, RL-007-03, was found to be approximately 100-fold less potent in potentiating LTP in hippocampal slices.[6] This stereoselectivity is a strong indicator of a specific interaction with a biological target, rather than non-specific membrane effects.

The multi-modal mechanism of Inidascamine, particularly its sophisticated and potentially novel indirect interaction with the GABA-B system, differentiates it from many existing CNS therapeutics. This complex pharmacology, which aims to restore a more balanced excitatory/inhibitory tone and enhance synaptic plasticity across multiple relevant neurotransmitter systems, provides a compelling rationale for its investigation in CIAS, a disorder characterized by widespread cognitive deficits. The observed inverted U-shaped dose-response for its effects on LTP, also seen as a biphasic cognitive dose-response in early human studies [7], underscores the importance of careful dose selection in ongoing and future clinical trials to maximize therapeutic benefit.

5. Preclinical Research Findings

The preclinical development of Inidascamine (RL-007) has provided substantial evidence supporting its pro-cognitive potential and favorable safety profile. These studies have spanned in vitro, ex vivo, and in vivo models.

5.1. In Vitro and Ex Vivo Evidence of Synaptic Enhancement

As detailed in the mechanism of action, ex vivo studies using rat hippocampal slices have been instrumental in demonstrating RL-007's direct effects on synaptic function. The compound enhances Long-Term Potentiation (LTP), a key cellular correlate of learning and memory, and facilitates basal excitatory synaptic transmission.[2] These effects were observed within a specific concentration range (10-100 nM), highlighting an optimal window for its action.[6] Furthermore, the ability of RL-007 to potentiate LTP even in the absence of cholinergic inputs (in cholinergic ablated hippocampal slices) suggests a robust mechanism that is not solely dependent on intact cholinergic pathways, although it may synergize with them in vivo.[6]

5.2. In Vivo Efficacy in Animal Models of Cognition

Inidascamine has demonstrated pro-cognitive efficacy across a variety of animal models:

• General Pro-cognitive Effects: Early studies indicated pro-cognitive effects in healthy young animals, models of age-associated memory loss, and models involving cholinergic deficits.[2]
• Reversal of Age-Related Cognitive Decline: In aged rats, which naturally exhibit cognitive decline, daily oral administration of RL-007 (5.82 mg/kg) significantly improved spatial working memory performance in the Barnes maze. The treated aged rats made fewer errors, comparable to the performance of young, unimpaired rats, suggesting a reversal of age-related deficits.[6]
• Reversal of Pharmacologically-Induced Cognitive Deficits: RL-007 has consistently shown efficacy in reversing cognitive deficits induced by scopolamine, a muscarinic acetylcholine receptor antagonist.[5] This effect has been observed across multiple species, including rats and dogs, and is considered a highly relevant translational model for human cognitive impairment where cholinergic pathways are implicated, such as in schizophrenia.[19] An abstract and anticipated publication in Biological Psychiatry (May 2025) further details these findings in rats, dogs, and humans (likely referring to the human scopolamine challenge model).[19]

5.3. Anxiolytic Properties

In addition to its pro-cognitive effects, RL-007 has exhibited anxiolytic (anxiety-reducing) properties in animal models of unconditioned anxiety.[2] While not the primary focus of its current development, these anxiolytic effects could be beneficial, as anxiety is often comorbid with schizophrenia.

5.4. Preclinical Safety Pharmacology

The preclinical safety profile of RL-007 appears favorable:

• Lack of Sedation: Unlike many compounds that interact with the GABAergic system (e.g., benzodiazepines like diazepam), RL-007 did not induce sedation in mice even at a high intraperitoneal dose of 76 mg/kg.[6]
• CNS Safety: In a single-dose Good Laboratory Practice (GLP) CNS safety functional observation battery study in rats, oral doses of RL-007 up to 1527 mg/kg did not produce any acute neurobehavioral effects.[6]

This robust preclinical data package, demonstrating consistent pro-cognitive activity across various models and species, coupled with a promising safety profile (especially the lack of sedation), provided a strong rationale for advancing Inidascamine into clinical trials for CIAS. The ability to reverse scopolamine-induced deficits is a particularly compelling piece of translational evidence, given the cholinergic dysregulation often observed in schizophrenia.

5.5. Key Scientific Presentations of Preclinical Data

The preclinical findings for RL-007 have been disseminated through key scientific forums:

• ACNP 2023: An abstract by Donello JE, Walker GA, Schweighoffer F, and Pando MP titled "RL-007, a novel oral neuromodulator, enhances synaptic plasticity and cognition in non-clinical models" was presented at the American College of Neuropsychopharmacology Annual Meeting on December 5, 2023.[1] This presentation detailed the effects on LTP, spatial working memory in aged rats, and the unique GABA-B dependent mechanism.
• SOBP 2025: Recognify Life Sciences presented further RL-007 preclinical data at the Society of Biological Psychiatry annual meeting in 2025.[9]
• Biological Psychiatry Journal: An abstract/article titled "RL-007, a Novel Oral Neuromodulator, Reverses Cognitive Deficits Induced by Muscarinic Receptor Blockade in a Translational Model of Cognitive Impairment in Rats, Dogs and Humans" was anticipated for publication or was an abstract from a conference proceeding published in Biological Psychiatry around May 2025.[19]

6. Clinical Development Program for Cognitive Impairment Associated with Schizophrenia (CIAS)

The clinical development of Inidascamine (RL-007) for CIAS builds upon a foundation of earlier human studies and has progressed through targeted proof-of-mechanism and proof-of-concept trials.

6.1. Historical Clinical Context (Pre-CIAS Focus)

Prior to its acquisition and focused development for CIAS by atai Life Sciences and Recognify Life Sciences, Inidascamine (then known primarily as FSV7-007) had undergone substantial clinical investigation. A total of nine clinical studies, comprising seven Phase 1 trials and two Phase 2 trials, had been conducted, with 508 unique human subjects dosed.[2] This extensive early human exposure provided a valuable dataset on the compound's general safety, tolerability, and pharmacokinetics.

Crucially, pro-cognitive signals emerged from these earlier studies. Three of these trials demonstrated improvements in verbal learning and memory.[2] One notable study was a large Phase II trial where FSV7-007 was investigated for peripheral neuropathic pain; cognitive assessments included as exploratory endpoints revealed pro-cognitive effects.[2] This serendipitous or exploratory finding was likely a key factor in recognizing the compound's potential for treating primary cognitive disorders and redirecting its development towards CIAS.

6.2. Phase 1 Studies in Healthy Volunteers

While specific details of dedicated Phase 1 studies solely for the CIAS program are not extensively detailed in the provided information, the seven Phase 1 trials conducted historically [2] would have established the initial safety, tolerability, and pharmacokinetic profile of Inidascamine in healthy human volunteers. Such studies are standard in drug development and essential for determining appropriate dose ranges for subsequent patient trials. It is mentioned that qEEG changes observed in the later Phase 2a CIAS study were consistent with findings from a previous Phase 1 trial in healthy volunteers (likely one of these initial seven) [7], indicating that early human CNS effects were characterized.

6.3. Phase 2a Proof-of-Mechanism Biomarker Study (NCT04822883) in CIAS

To specifically investigate RL-007 in the target CIAS population, a Phase 2a study was conducted.

• Official Title: "A Single-arm, Single-blind, Multiple Dose Study to Evaluate Safety and the Effects of RL-007 on Electroencephalograms and Event-related Potentials in Subjects With Schizophrenia".[18]
• Status: This trial is reported as completed [18], with positive topline data announced in December 2021.[7]
• Study Design: The study was a single-arm, single-blind, multiple-dose investigation involving 32 subjects with schizophrenia, divided into four cohorts of 8 patients each.[7]
• Intervention: Patients received oral RL-007 three times daily (TID) at escalating doses across cohorts: 10 mg, 20 mg, 40 mg, and 80 mg.[7]
• Objectives: The primary objectives were to evaluate the safety and tolerability of RL-007 in CIAS patients and to assess its effects on electroencephalogram (qEEG) biomarkers and cognitive performance.[3]
• Key Reported Outcomes [7]:
• Safety and Tolerability: RL-007 was well tolerated by CIAS patients.
• Cognitive Effects: The study demonstrated a clinically meaningful pro-cognitive profile. Dose-related improvements were observed on exploratory cognitive endpoints, including the Brief Assessment of Cognition in Schizophrenia Symbol Coding Test (which assesses processing speed) and the Hopkins Verbal Learning Task (which assesses verbal learning and memory). A bi-phasic dose-response for cognitive effects, previously seen in other studies, was replicated.
• qEEG Biomarkers: Dose-dependent changes in qEEG were observed, with the most significant increases in alpha band amplitude (up to 17% increase) and the alpha-slow wave index (up to 21% increase) occurring at the 20 mg and 40 mg doses. These qEEG changes are considered markers of alertness and are believed to correlate with aspects of cognition. These findings were consistent with results from a prior study of RL-007 in a human model of cognitive impairment (scopolamine challenge in healthy volunteers).
• Sponsor: atai Life Sciences / Recognify Life Sciences.[7]

The positive outcomes from this Phase 2a study, particularly the objective qEEG biomarker evidence of target engagement and the pro-cognitive signals at the 20 mg and 40 mg doses, were crucial in supporting the progression to a larger, more definitive Phase 2b trial.[3]

6.4. Phase 2b Proof-of-Concept Study (NCT05686239) in CIAS

This ongoing trial is designed to provide robust evidence of RL-007's efficacy and safety in CIAS.

• Official Title: "An Adaptive, Randomized, Placebo-controlled, Double-blind Study to Evaluate the Safety and Efficacy of RL-007 in the Treatment of Cognitive Impairment Associated With Schizophrenia (CIAS)".[18]
• Status: The trial is currently recruiting participants.[18] Patient screening commenced in December 2022, with the first patient dosed anticipated by the end of that year.[3] The timeline for topline results has evolved, with the latest expectation being mid-2025 [9], revised from earlier projections of H1 2024 [3] and 2H 2024.[30]
• Study Design: This is a randomized, 3-arm, placebo-controlled, double-blind clinical trial.[3]
• Number of Participants: The study aims to enroll approximately 230 to 234 patients with CIAS.[3]
• Arms and Interventions [8]:
• Arm 1 (Experimental): RL-007 20 mg, administered orally, three times per day (TID).
• Arm 2 (Experimental): RL-007 40 mg, administered orally, TID.
• Arm 3 (Placebo Control): Placebo, administered orally, TID.
• Duration: The treatment period is 6 weeks, with a screening phase of approximately 1 month and a follow-up period of about 2 weeks.[3]
• Primary Outcome Measure: The primary endpoint is the change from baseline in the MATRICS Consensus Cognitive Battery (MCCB) neurocognitive composite score.[3] The MCCB is a standardized battery of tests assessing various cognitive domains and is an FDA-supported approvable endpoint for CIAS studies.[3]
• Key Secondary Outcome Measures [8]: These include:
• Clinical Global Impression - Severity (CGI-S).
• Symbol Coding Test (a sub-component of the MCCB).
• Attention/Vigilance domain of the MCCB.
• Social Cognition domain of the MCCB.
• Virtual Reality Functional Capacity Assessment Tool (VRFCAT), which assesses the ability to perform everyday tasks.
• Hopkins Verbal Learning Test - Revised (HVLT-R).
• Category Fluency Task.
• Key Eligibility Criteria [8]: Participants are adults aged 18-55 years with a diagnosis of schizophrenia for at least 6 months. They must be clinically stable on a single atypical antipsychotic medication (excluding clozapine) for at least 6 weeks prior to randomization, have a CGI-S score < 5 (indicating symptoms are not very severe), and a Body Mass Index (BMI) ≤ 40.0 kg/m². Major exclusions include recent hospitalization for medical or psychiatric reasons, other significant mental health diagnoses, history of significant brain injuries, and moderate to severe substance/drug abuse disorder (including alcohol) within the 6 months prior to informed consent.
• Sponsor: Recognify Life Sciences (a subsidiary of atai Life Sciences).[3]
• Locations: The trial is being conducted at sites in the United States and internationally, including locations in Bulgaria, Czechia, and Poland, as well as multiple U.S. states such as California, Florida, Georgia, Maryland, New Jersey, New York, North Carolina, Ohio, Texas, and Washington.[3]

The design of this Phase 2b study is robust, incorporating randomization, double-blinding, and a placebo control, which are essential for minimizing bias and generating high-quality evidence. The use of the MCCB as the primary endpoint aligns with regulatory expectations for CIAS trials. The inclusion of diverse secondary outcomes, including functional capacity measures like the VRFCAT, aims to capture a broader picture of RL-007's potential benefits. The evolving timeline for results is not uncommon in complex CNS trials and may reflect the challenges inherent in recruiting and managing studies in this patient population.

Table 2: Overview of Key Clinical Trials for Inidascamine (RL-007) in CIAS

Trial ID	Phase	Official Title	Status	No. of Patients	Study Design	Intervention Arms (Doses, Frequency, Duration)	Primary Outcome Measure(s) & Timeframe	Key Secondary Outcome Measures (Selected) & Timeframe	Summary of Reported Results/Current Status	Key Snippet(s)
NCT04822883	2a	A Single-arm, Single-blind, Multiple Dose Study to Evaluate Safety and the Effects of RL-007 on Electroencephalograms and Event-related Potentials in Subjects With Schizophrenia	Completed	32	Single-arm, single-blind, multiple-dose	RL-007: 10mg, 20mg, 40mg, 80mg, oral, TID	Safety, qEEG changes, Cognitive effects (exploratory)	Symbol Coding, HVLT	Well tolerated. Clinically meaningful pro-cognitive profile. Dose-related qEEG changes (alpha band, alpha-slow wave index) maximal at 20mg & 40mg.	7
NCT05686239	2b	An Adaptive, Randomized, Placebo-controlled, Double-blind Study to Evaluate the Safety and Efficacy of RL-007 in the Treatment of Cognitive Impairment Associated With Schizophrenia (CIAS)	Recruiting	~234	Randomized, 3-arm, placebo-controlled, double-blind	RL-007 20mg, oral, TID, 6 weeks; RL-007 40mg, oral, TID, 6 weeks; Placebo, oral, TID, 6 weeks	Change from baseline in MATRICS Consensus Cognitive Battery (MCCB) neurocognitive composite score at 6 weeks	CGI-S, Symbol Coding, Attention/Vigilance (MCCB), Social Cognition (MCCB), VRFCAT, HVLT-R, Category Fluency Task (all at 6 weeks)	Ongoing. Topline results expected mid-2025.	3

7. Pharmacokinetics and Metabolism (ADME)

Understanding the absorption, distribution, metabolism, and excretion (ADME) properties of Inidascamine (RL-007) is critical for interpreting its pharmacological effects and optimizing its clinical use. Current information is primarily derived from preclinical studies and inferences from early-phase human trials.

7.1. Absorption and Bioavailability

Inidascamine is formulated for oral administration.[1] Preclinical studies in rats have demonstrated good oral absorption and bioavailability. Specifically, the absolute oral bioavailability was reported to be 53% in male rats and 88% in female rats.[6] These figures suggest efficient absorption from the gastrointestinal tract in this species, which is a positive attribute for an orally administered drug. Human bioavailability data from Phase 1 studies have not been detailed in the provided snippets.

7.2. Distribution

A key pharmacokinetic property for a CNS-active drug is its ability to penetrate the blood-brain barrier (BBB). Preclinical data in rats indicate that RL-007 readily crosses the BBB. At equilibrium, the brain-to-blood ratio of unbound RL-007 concentration was approximately 31%.[6] This level of brain penetration suggests that therapeutically relevant concentrations of the drug can be achieved in the CNS following systemic administration.

7.3. Metabolism

Detailed information regarding the specific metabolic pathways and major human metabolites of Inidascamine is not available in the provided research snippets. The ACNP 2023 abstract by Donello et al. focused on the pharmacokinetics of the parent compound in rats.[6] Phase 1 studies in humans would typically investigate metabolism, but these results are not publicly detailed here. Understanding the metabolic profile is important for assessing potential drug-drug interactions and variability in patient response.

7.4. Excretion

The routes and rates of excretion of Inidascamine and its metabolites in humans or animal models are not specified in the available information.

7.5. Half-life

The elimination half-life of Inidascamine in humans is not explicitly stated in the provided snippets. This parameter is crucial for determining dosing frequency and predicting time to steady-state concentrations. The TID (three times a day) dosing regimen used in clinical trials [7] suggests a relatively moderate half-life that necessitates multiple daily doses to maintain therapeutic exposure, or it could be designed to manage Cmax-related effects or maintain more consistent trough concentrations.

7.6. Human Pharmacokinetic Data

While the seven Phase 1 studies involving 508 subjects conducted prior to the focused CIAS development would have extensively characterized human pharmacokinetics [2], specific parameters such as Cmax, Tmax, AUC, clearance, and volume of distribution in humans are not publicly available through the provided snippets. The consistency of qEEG effects between healthy volunteer studies and the Phase 2a CIAS patient study [7] implies that a predictable pharmacokinetic profile was established, allowing for consistent dosing and exposure. However, a full public disclosure of these parameters is pending.

The preclinical pharmacokinetic profile, particularly the good oral bioavailability and brain penetration in rats [6], is encouraging for a CNS drug. However, the current lack of detailed public information on human ADME parameters represents an information gap. This data will be critical for the later stages of development, for optimizing dosing regimens, understanding potential drug interactions, and assessing the need for dose adjustments in special populations.

Table 3: Summary of Key Pharmacokinetic Parameters for Inidascamine (RL-007)

Parameter	Species	Value/Observation	Snippet ID(s)
Route of Administration	Human	Oral	1
Oral Bioavailability	Rat (Male)	53% (absolute)	6
Oral Bioavailability	Rat (Female)	88% (absolute)	6
Brain Penetration (Unbound Brain/Blood Ratio at Equilibrium)	Rat	~31%	6
Human Pharmacokinetic Parameters (Cmax, Tmax, Half-life, Metabolites, Excretion)	Human	Assessed in Phase 1 studies; specific parameters not detailed in provided snippets.	2 (inferred)
Metabolism Notes	General	Not detailed for humans.
Excretion Notes	General	Not detailed for humans.

8. Safety and Tolerability Profile

The safety and tolerability of Inidascamine (RL-007) have been assessed in both preclinical models and multiple human clinical trials.

8.1. Preclinical Safety

Preclinical safety pharmacology studies have indicated a favorable profile:

• In a Good Laboratory Practice (GLP) compliant CNS safety functional observation battery study in rats, single oral doses of RL-007 up to 1527 mg/kg did not produce any acute neurobehavioral effects.[6] This suggests a wide safety margin in this species.
• RL-007 did not induce sedation in mice at a high intraperitoneal dose of 76 mg/kg. This is noteworthy as it contrasts with the sedative effects of diazepam, a benzodiazepine often used as a comparator in such assays, and is particularly relevant given the GABA-B system involvement in RL-007's mechanism.[6] The lack of sedation supports the hypothesis of an indirect modulatory effect on the GABA system rather than direct agonism.

8.2. Clinical Safety and Tolerability

Inidascamine has been administered to over 500 unique human participants across ten clinical studies, including seven Phase 1 trials, two earlier Phase 2 trials (primarily for other indications like neuropathic pain), and the more recent Phase 2a study in CIAS patients.[2]

• General Tolerability: Across these diverse studies and participant populations, RL-007 has been consistently reported as well tolerated at all doses tested.[3]
• Phase 2a CIAS Study (NCT04822883): In the Phase 2a proof-of-mechanism study involving 32 CIAS patients, RL-007 was specifically reported to be well tolerated.[7]
• Side Effect Profile: A key aspect of RL-007's tolerability is the reported absence of side effects typically associated with direct modulation of the cholinergic or GABA-B receptor systems.[2] For example, direct cholinergic agonists can cause significant gastrointestinal and autonomic side effects, while direct GABA-B agonists are often sedative. The ability of RL-007 to modulate these systems, likely indirectly, without inducing these characteristic adverse events is a significant potential advantage.
• Ongoing Monitoring: Current clinical trials, such as the Phase 2b study (NCT05686239), incorporate comprehensive safety monitoring protocols. These include regular assessment of vital signs (blood pressure, heart rate, breathing rate, body temperature), electrocardiograms (ECGs), mental health status (including suicidality risk, a standard precaution in schizophrenia trials), and systematic collection of all adverse events.[8]

The consistent and favorable tolerability profile observed for Inidascamine across a substantial number of human subjects is a critical asset for its development. For a CNS therapeutic intended for conditions like CIAS, where patients may already be on multiple medications and sensitive to side effects, a well-tolerated drug is more likely to achieve patient adherence and allow for chronic administration if needed. The differentiation from competitor pipeline options is also noted, with RL-007's "excellent tolerability profile" and "acute onset of action" being highlighted.[3]

Table 4: Summary of Safety and Tolerability Findings for Inidascamine (RL-007) from Clinical Trials

Study Phase/Type	Population	Key Safety/Tolerability Observations	Snippet ID(s)
Multiple Phase 1 & 2 Studies (pre-CIAS and CIAS focus)	>500 unique participants (including healthy volunteers and patients with conditions like neuropathic pain and CIAS)	Generally well tolerated across all doses tested.	2
Phase 2a (NCT04822883)	32 CIAS patients	RL-007 was well tolerated.	7
General Clinical Profile	Patients in various studies	Reported to have an "excellent tolerability profile." Appears to lack typical side effects associated with direct cholinergic or GABA-B modulation. No sedation observed preclinically at high doses.	2

9. Regulatory Status and Intellectual Property

The regulatory pathway and intellectual property landscape are critical components of Inidascamine's (RL-007) development and commercial potential.

9.1. Regulatory Status

• Investigational Drug: Inidascamine is currently an investigational drug and has not received marketing approval from the FDA, EMA, or any other regulatory agency for any indication.[1]
• Clinical Development Stage: The compound is in Phase 2b clinical development for CIAS in the United States and other international locations.[3]
• Regulatory Pathway for CIAS: The primary endpoint for the ongoing Phase 2b trial (NCT05686239) is the MATRICS Consensus Cognitive Battery (MCCB).[3] This is significant because the MCCB has been supported by the FDA as an approvable endpoint for CIAS.[3] This alignment with regulatory expectations provides a clearer pathway for demonstrating efficacy for this challenging indication.
• Special Designations: The provided snippets [1] do not mention any specific FDA or EMA special regulatory designations such as Fast Track, Breakthrough Therapy, or Orphan Drug Designation for Inidascamine (RL-007/FSV7-007) for the indication of CIAS.[32] While an orphan drug designation (EU/3/13/1173) was granted by the EMA on July 25, 2013, to Allergan Pharmaceuticals International Limited for "(2R,3S)-2-amino-3-hydroxy-3-(pyridin-4-yl)-1-(pyrrolidin-1-yl)propan-1-one" (the chemical name for Inidascamine) for the "treatment of Fragile X syndrome," this designation is for a different indication and a previous sponsor. Its relevance to the current CIAS program under atai/Recognify is indirect, primarily confirming the compound's identity and earlier exploration for neurodevelopmental disorders.

9.2. Intellectual Property

• Key Patent: Patent WO2008011478A2 is referenced in connection with Inidascamine.[12] This international patent application would likely cover the compound itself or its use. The specifics of its claims, territorial coverage, and expiration dates are crucial for determining the period of market exclusivity for Inidascamine.
• Other Patents: Additional patent numbers associated with Inidascamine include US patents 08288556, 08431599, 8288556, 8431599, 8927589, and US patent applications 20080312236, 20090036436, 20090318499, 20100048642, 20100093793, 20130005717, 20130202653, as well as WO2006081273A1.[13] These suggest a portfolio of intellectual property surrounding the molecule.
• atai Life Sciences' IP Portfolio: More broadly, atai Life Sciences reports having 23 patent applications and 12 granted patents that protect its drug development technologies in general, which would support its various programs, including RL-007.[34]

The established regulatory pathway for CIAS, with the MCCB as an accepted endpoint, provides some clarity for the ongoing Phase 2b trial. Strong intellectual property protection, anchored by patents like WO2008011478A2 and others, is fundamental for securing future market exclusivity and justifying the substantial investment required for late-stage clinical development and commercialization.

10. Discussion and Future Outlook

Inidascamine (RL-007) represents a novel therapeutic approach for Cognitive Impairment Associated with Schizophrenia (CIAS), a condition with a significant lack of effective treatments. Its development to date, characterized by a unique multi-modal mechanism of action, consistent pro-cognitive signals in preclinical and early clinical studies, and a favorable tolerability profile, positions it as a compound of considerable interest.

The core pharmacological hypothesis for Inidascamine centers on its ability to modulate cholinergic, NMDA, and GABA-B receptor systems, thereby enhancing synaptic plasticity and improving cognitive functions.[1] The preclinical evidence is compelling, with demonstrations of LTP enhancement, reversal of age-related cognitive decline in animal models, and efficacy in the scopolamine challenge model, which has translational relevance to human cognitive impairment.[2] Particularly intriguing is the indirect nature of its GABA-B modulation, which may contribute to its efficacy without the sedative side effects common to direct GABA-B agonists.[6] The Phase 2a study in CIAS patients provided early human validation, showing pro-cognitive effects and qEEG changes indicative of target engagement, especially at the 20 mg and 40 mg doses.[7] Furthermore, the compound has been well tolerated in over 500 individuals across various studies.[3]

The ongoing Phase 2b proof-of-concept trial (NCT05686239) is a critical next step. Its robust design, employing a randomized, double-blind, placebo-controlled methodology with the FDA-supported MCCB as the primary endpoint, is well-suited to provide a clearer picture of Inidascamine's efficacy in CIAS.[3] The inclusion of functional outcome measures like the VRFCAT will also be important for assessing real-world impact.[8] The results from this trial, now anticipated in mid-2025 [9], will be a major determinant of the future development pathway for Inidascamine.

The therapeutic landscape for CIAS is largely barren, with no FDA-approved pharmacological treatments.[3] This high unmet medical need means that a drug demonstrating clear and clinically meaningful cognitive improvement with a good safety profile could become a cornerstone of CIAS management. Inidascamine's potential for complementary use with existing antipsychotic medications, which primarily address positive symptoms, could offer a comprehensive treatment strategy for schizophrenia patients.[3]

However, the development of CNS drugs, particularly for cognitive disorders in schizophrenia, is fraught with challenges. Cognitive endpoints are complex to measure, patient populations can be heterogeneous, and the placebo response can be variable. The "valley of death" for CNS drug development is well-documented, with many promising early-stage compounds failing in later, more rigorous trials. The evolving timeline for the Phase 2b results may reflect some of these inherent difficulties in conducting such complex studies.

Should the Phase 2b trial yield positive results, Inidascamine would likely proceed to Phase 3 development, requiring even larger and longer studies to confirm efficacy and safety in a broader population. Long-term safety data will also be crucial, especially if the drug is intended for chronic administration.

11. Concluding Remarks and Expert Insights

Inidascamine (RL-007/FSV7-007) emerges from the available data as a scientifically intriguing and potentially valuable investigational neuromodulator for Cognitive Impairment Associated with Schizophrenia (CIAS). Its development is underpinned by a unique pharmacological profile, targeting multiple neurotransmitter systems implicated in cognition—cholinergic, NMDA, and notably, an indirect, GABA-B receptor tone-dependent mechanism that may confer efficacy without typical GABAergic side effects like sedation.[1] This multi-modal approach is a rational strategy for a complex, multifaceted condition like CIAS.

The strengths of the Inidascamine program lie in the consistent pro-cognitive signals observed from preclinical animal models through to early-phase human studies, including a Phase 2a trial in CIAS patients that showed positive effects on both cognitive measures and qEEG biomarkers.[2] The extensive prior human exposure (over 500 subjects) and the generally favorable safety and tolerability profile reported to date are significant assets, de-risking aspects of its continued development.[3] The clear regulatory pathway for CIAS, with the MATRICS Consensus Cognitive Battery (MCCB) being an FDA-supported endpoint, also provides a defined goal for the ongoing Phase 2b trial.[3]

Weaknesses or, more accurately, areas requiring further elucidation, include the current lack of detailed, publicly available human pharmacokinetic data (e.g., half-life, metabolism, excretion pathways). While preclinical PK in rats is promising (good oral bioavailability and brain penetration [6]), comprehensive human PK is essential for full characterization. The most significant pending factor is, of course, the outcome of the ongoing Phase 2b proof-of-concept study (NCT05686239). The success of this trial is pivotal for the future of Inidascamine. The shifting timelines for these results, now expected in mid-2025 [9], highlight the inherent challenges of CNS clinical trials.

The potential approval of Inidascamine would be a landmark achievement for patients suffering from CIAS, offering the first FDA-approved pharmacological treatment for this debilitating condition. It could significantly improve functional outcomes, quality of life, and societal participation for individuals with schizophrenia. For atai Life Sciences and its subsidiary Recognify, RL-007 represents a key pipeline asset [10]; its success would validate their strategic focus on innovative mental health treatments.

In conclusion, Inidascamine (RL-007) embodies a scientifically driven effort to address a critical unmet need in psychiatric pharmacotherapy. It balances innovation in its proposed mechanism with the inherent risks of CNS drug development. The preclinical and early clinical data are encouraging, supporting continued investigation. The forthcoming results from the Phase 2b trial will be a crucial inflection point, determining whether Inidascamine can progress towards potentially transforming the treatment landscape for cognitive impairment in schizophrenia.

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