Drug Development/Existing Targets: Phase 1 trials

Initiate first in human, Phase 1 drug trials for therapeutic agents against at least 6 existing therapeutic targets. In addition to testing for safety, these trials will include assessment of target engagement.

2026 PAR-12-015: Alzheimer's Drug-Development Program (U01) PAR-15-174: Alzheimer's Drug-Development Program (U01) PAR-18-820: Alzheimer's Drug-Development Program (U01 Clinical Trial Optional) PAR-14-089: Early Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01) PAR-16-365: Early Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01) PAR-18-175: Early Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01 Clinical Trial Optional) PAR-18-877: Early Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01 Clinical Trial Optional) PAS-17-064: Advancing Research on Alzheimer's Disease (AD) and Alzheimer's-Disease-Related Dementias (ADRD) (R43/R44) PAS-18-187: Advancing Research on Alzheimer's Disease (AD) and Alzheimer's-Disease-Related Dementias (ADRD) (R43/R44 Clinical Trial Optional) PAS-17-065: Advancing Research on Alzheimer's Disease (AD) and Alzheimer's-Disease-Related Dementias (ADRD) (R41/R42) PAS-18-188: Advancing Research on Alzheimer's Disease (AD) and Alzheimer's-Disease-Related Dementias (ADRD) (R41/R42 Clinical Trial Optional) PAR-18-546: Blueprint Neurotherapeutics Network (BPN): Small Molecule Drug Discovery and Development of Disorders of the Nervous System (UG3/UH3) PAR-18-877: Early Stage Clinical Trials for the Spectrum of Alzheimer's Disease and Age-related Cognitive Decline (R01 Clinical Trial Optional) PAR-21-359: Early and Late Stage Clinical Trials for the Spectrum of Alzheimer’s Disease/Alzheimer’s Related Dementias and Age-Related Cognitive Decline (R01 Clinical Trial Optional) PAR-22-047: Alzheimer's Drug-Development Program (U01 Clinical Trial Optional) (Reissue of PAR-18-820) PAR-24-063: Blueprint Neurotherapeutics Network (BPN): Small Molecule Drug Discovery and Development for Disorders of the Nervous System (U44 Clinical Trial Optional) PAR-24-293: Blueprint Neurotherapeutics Network (BPN): Biologic-based Drug Discovery and Development for Disorders of the Nervous System (UG3/UH3 Clinical Trial Optional) PAR-24-294: Blueprint Neurotherapeutics Network (BPN): Biologic-based Drug Discovery and Development for Disorders of the Nervous System (U44 Clinical Trial Optional) PAR-25-051: Blueprint Neurotherapeutics Network (BPN): Small Molecule Drug Discovery and Development of Disorders of the Nervous System (UG3/UH3 Clinical Trial Optional) PAR-25-297: Alzheimer's Drug-Development Program (U01 Clinical Trial Optional) 5.A In Progress Initiate first in human, Phase 1 drug trials for therapeutic agents against at least 6 existing therapeutic targets… DN 2012 Summit: 3A, 3B, 3F, and 5E Drug Development - Existing Targets Phase 1a safety trial for CT1812, Small Molecule Targeting a Synaptic Receptor for Abeta Oligomers Phase 1b safety trial for CT1812, Small Molecule Targeting a Synaptic Receptor for Abeta Oligomers Gamma-Secretase Modulator, NGP 555 Multiple Ascending Dose clinical trial of PTI-125, a novel AD therapeutic candidate 2-Hydroxybenzylamine for the Prevention of Alzheimer's Disease: Initial Evaluation in Humans Active Immunotherapy (vaccine) for Cognitive decline in Adults with Down Syndrome A Pilot Study to Evaluate CT1812 Treatment on Aβ Oligomer Clearance in Alzheimer's Disease A Pilot PET Study to Evaluate CT1812 Treatment on Synaptic Density in Alzheimer’s Disease A Pilot Electroencephalography (EEG) Study to Evaluate the Effect of CT1812 Treatment on Synaptic Activity in Subjects with Mild to Moderate Alzheimerʼs Disease A Single Ascending Dose / Multiple Ascending Dose Phase I study of the GSM 776890 in healthy normal subjects A Phase 1b Multiple Ascending Dose Study of the Safety and Tolerability of BMS-984923 in Alzheimer's Disease First in Human Study of a Tau Self-Association Small Molecule Inhibitor in Healthy Volunteers Safety/Tolerability/Immunogenicity of first-in-human Aβ DNA vaccine, AV-1959D Phase 1 trials in early-stage AD subjects: based on IND18953 cleared by FDA Phase I clinical trial: Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Immunogenicity of AV-1959R in Healthy Participants Phase I clinical trial NCT05696483: Phase 1 Study of OLX-07010 in Healthy Adult and Elderly Participants 2017

  • Completion of 12 Phase 1 drug trials for agents against 6 existing therapeutic targets.

Summary of Key Accomplishments

Eleven Phase 1 trials have been supported testing new drug candidates for existing therapeutic targets (amyloid-beta related and acetylcholinesterase). Of these:

  • six Phase 1 trials have been completed, testing four new drug candidates against four amyloid beta-related therapeutic targets
  • three additional Phase 1 trials are ongoing
  • two trials that test two additional anti-amyloid drug candidates are expected to begin in 2022

The key accomplishments summary is current as of July 2022.