DDL-920's Clinical Trials

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SUMMARY

DDL-920 is a novel molecule developed by UCLA researchers, including Varghese John, Istvan Mody, and S. Thomas Carmichael, with research beginning around May 2019. It has demonstrated promising preclinical results in mouse models for both Alzheimer's disease and stroke recovery, showing restoration of cognitive function and improved movement control. As of July 2026, DDL-920 remains in preclinical development, with researchers actively preparing for human clinical trials, though no trials have officially commenced.

Timeline

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Timeline of developments

July 2026 1 developments

  1. DDL-920 Researchers Actively Preparing for Human Clinical Trials

    As of July 2026, UCLA researchers, including Varghese John, Istvan Mody, and S. Thomas Carmichael, are actively engaged in the final preparations for human clinical trials for DDL-920. While the Investigational New Drug (IND) application is being finalized or is under review, no trials have officially commenced. The team is focused on securing regulatory approval and initiating Phase 1 studies for this promising molecule.

March 2026 1 developments

  1. Internal Review and Quality Control of IND Application

    The drafted Investigational New Drug (IND) application underwent rigorous internal review and quality control checks by the UCLA team and their partners. This process ensured accuracy, completeness, and compliance with all regulatory guidelines. A thorough internal review is critical to minimize potential delays or rejections from the FDA upon submission.

November 2025 1 developments

  1. Preparation and Drafting of the Investigational New Drug (IND) Application

    Following the Pre-IND meeting, the UCLA team, in collaboration with regulatory experts, began the intensive process of preparing and drafting the full Investigational New Drug (IND) application. This extensive document includes all preclinical data, manufacturing information, and the detailed protocol for proposed Phase 1 human clinical trials. Its submission is required before human testing can begin.

August 2025 1 developments

  1. Feedback Received from Regulatory Authorities on Pre-IND Meeting

    The research team received comprehensive feedback from the FDA following their Pre-IND meeting. This feedback included guidance on specific data requirements, potential concerns regarding preclinical studies, and recommendations for the design of initial human clinical trials. Addressing these points was essential for preparing a robust and approvable IND application.

May 2025 1 developments

  1. Pre-Investigational New Drug (IND) Meeting Request Submitted to FDA

    UCLA researchers formally submitted a request for a Pre-Investigational New Drug (IND) meeting with the U.S. Food and Drug Administration (FDA). This meeting is a crucial step for drug developers to receive early feedback from the FDA on their preclinical data, proposed clinical trial design, and overall development plan. It helps streamline the IND application process.

February 2025 1 developments

  1. Long-term Preclinical Efficacy and Safety Data Compilation

    All accumulated preclinical data, including long-term efficacy in disease models and comprehensive safety assessments, were compiled and rigorously analyzed. This extensive data package summarized DDL-920's pharmacological profile, mechanism of action, efficacy in two distinct neurological conditions, and its safety characteristics. This compilation was a critical step towards regulatory submission.

November 2024 1 developments

  1. Dose-Range Finding Studies for Efficacy and Safety

    Researchers conducted detailed dose-range finding studies to identify optimal therapeutic doses of DDL-920 that maximized efficacy while minimizing side effects. These studies involved testing various concentrations of the drug in animal models for both Alzheimer's and stroke. The aim was to establish a clear therapeutic window for potential human trials.

August 2024 1 developments

  1. Sub-chronic Toxicology Studies and Safety Profile Assessment

    Following acute toxicology, sub-chronic studies were conducted to evaluate DDL-920's safety profile over an extended period, typically weeks to months. These studies monitored for any cumulative toxicity, organ damage, or other adverse effects from repeated dosing. The data gathered was crucial for establishing a preliminary safety margin and informing potential human dosing.

May 2024 1 developments

  1. Comprehensive Acute Toxicology Studies Initiated

    With strong efficacy data in hand, comprehensive acute toxicology studies for DDL-920 were initiated. These studies assessed the immediate adverse effects of high doses of the drug in animal models over a short period. The goal was to determine the maximum tolerated dose and identify any acute safety concerns before proceeding to longer-term studies.

February 2024 1 developments

  1. DDL-920 Mimics Rehabilitation Effects in Stroke Recovery

    Further analysis revealed that DDL-920's effects in stroke models successfully reproduced the benefits typically associated with physical rehabilitation. The drug appeared to facilitate neural plasticity and reorganization, leading to functional gains similar to those achieved through intensive therapy. This suggested DDL-920 could serve as a pharmacological adjunct or alternative to traditional rehabilitation.

November 2023 1 developments

  1. Improved Motor Control and Functional Recovery in Stroke Models

    Beyond reducing initial damage, DDL-920 treatment in stroke mouse models led to significant improvements in motor control and overall functional recovery. Behavioral assessments demonstrated enhanced limb coordination, balance, and general mobility in treated animals compared to controls. These findings suggested that DDL-920 could actively promote brain repair and rehabilitation processes post-stroke.

August 2023 1 developments

  1. DDL-920 Reduces Brain Damage Volume in Stroke Models

    Initial efficacy studies in ischemic stroke mouse models showed that DDL-920 significantly reduced the volume of brain damage (infarct size) following a stroke. This indicated a neuroprotective effect of the drug, potentially limiting the immediate impact of the ischemic event. Reducing infarct volume is a critical outcome for improving long-term recovery in stroke patients.

May 2023 1 developments

  1. Development of Ischemic Stroke Mouse Models

    Following promising results in Alzheimer's models, the research expanded to investigate DDL-920's potential in stroke recovery. Specialized ischemic stroke mouse models were developed and validated to mimic human stroke conditions. These models allowed researchers to assess the drug's ability to mitigate brain damage and promote functional recovery after a stroke event.

February 2023 1 developments

  1. Confirmation of Gamma Oscillation Enhancement in AD Models

    Researchers confirmed that the observed cognitive and memory improvements in AD mouse models were directly linked to DDL-920's ability to enhance gamma oscillations in the brain. Electrophysiological recordings showed a significant increase in gamma wave activity in treated mice. This validated the drug's hypothesized mechanism of action in a living organism and its relevance to cognitive restoration.

August 2022 1 developments

  1. DDL-920 Restores Cognitive Function in AD Mouse Models

    Preclinical studies in Alzheimer's disease mouse models demonstrated that DDL-920 was effective in restoring cognitive function. Treated mice showed significant improvements in various cognitive tasks, indicating a reversal of some AD-related impairments. This finding provided strong evidence for DDL-920's therapeutic potential in addressing the cognitive deficits associated with Alzheimer's.

May 2022 1 developments

  1. Development and Utilization of Alzheimer's Disease Mouse Models

    To test DDL-920's efficacy for Alzheimer's disease, researchers meticulously developed and utilized specialized mouse models that exhibited symptoms of the condition. These models were crucial for evaluating the drug's potential to mitigate cognitive decline and other AD-related pathologies in a living system. This step marked the transition to in vivo efficacy testing.

January 2022 1 developments

  1. Initial Brain Penetration Studies Confirm CNS Access

    Complementing the pharmacokinetic studies, initial investigations confirmed DDL-920's ability to cross the blood-brain barrier and achieve therapeutic concentrations within the central nervous system. This was a critical finding, as it indicated the drug could reach its target sites in the brain. Effective CNS penetration is a prerequisite for any neurological disorder treatment.

October 2021 1 developments

  1. Pharmacokinetic Studies Reveal Oral Administration Potential

    Pharmacokinetic studies were conducted on DDL-920, revealing favorable properties for oral administration. The drug demonstrated good oral bioavailability and the ability to distribute effectively into various regions of the brain. This characteristic was highlighted as a significant advantage, as it suggested a non-invasive and accessible treatment method for future patients.

July 2021 1 developments

  1. Dose-Dependent Inhibition of Tonic Currents Confirmed In Vitro

    Further in vitro studies were conducted to establish the dose-response relationship of DDL-920's effect on tonic currents in parvalbumin interneurons. These experiments confirmed that the compound's inhibitory action on GABAARs was dose-dependent, providing a clearer understanding of its potency. This data was vital for guiding subsequent in vivo dosing strategies and confirming the drug's pharmacological profile.

April 2021 1 developments

  1. Initial In Vitro Confirmation of DDL-920's Mechanism

    DDL-920 underwent initial in vitro testing, which confirmed its ability to reduce large tonic currents in parvalbumin-expressing interneurons at very low concentrations, as low as 1 nM. This demonstrated that the compound effectively modulated the targeted GABAARs. These early laboratory results provided crucial validation for DDL-920's intended biological action.

September 2020 1 developments

  1. DDL-920 Discovered as a Promising Candidate Molecule

    After an extensive search, including reviewing older scientific literature and conducting refined screenings, the research team identified DDL-920 as a molecule with the potential to antagonize specific GABAARs. This mechanism was hypothesized to block the inhibition of parvalbumin neurons, thereby enhancing gamma oscillations. The identification of DDL-920 marked a significant step forward in their drug discovery process.

June 2020 1 developments

  1. Refined Screening Focuses on Alpha1-Beta2-Delta GABAAR Subunits

    Building on the identification of parvalbumin interneurons and their specific GABAAR subunits, the research team refined their screening process. They focused on compounds that selectively targeted the α1β2δ subunits of GABAARs, aiming for a more precise modulation of gamma oscillations. This targeted approach was crucial for developing a drug with high specificity and reduced off-target effects.

March 2020 1 developments

  1. Identification of Parvalbumin Interneurons as Key Targets

    Early in their research, the UCLA team identified parvalbumin-expressing interneurons (PV+INs) and their associated GABA type A receptors (GABAARs), particularly those containing α1β2δ subunits, as critical targets. These neurons play a vital role in generating gamma oscillations, and modulating them was deemed essential for restoring brain rhythm function. This discovery provided a specific biological pathway for their drug development efforts.