Alzprotect Completes Phase 2a Clinical Trial Enrollment for AZP2006 in Progressive Supranuclear Palsy (PSP)
Lille (France), January 20th, 2022 – ALZPROTECT, a biopharmaceutical company developing treatments for neurodegenerative diseases, today announced it has completed patient enrollment for the Company’s phase 2a clinical trial evaluating its platform asset, AZP2006 (“EZEPROGIND”) in Progressive Supranuclear Palsy (PSP), a rare neurodegenerative disorder which seriously impacts walking, balance, eye movement, and in the latter stage of the disease, the ability to swallow. This PSP trial, which is part of a multi-indication development plan for AZP2006, has recruited thirty-six patients and is being conducted at the Pitié-Salpêtrière University Hospital of Paris, the University Hospital of Lille, and the Hospital Foundation Adolphe de Rothschild. For PSP, AZP2006 has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). It was tested on 102 healthy human subjects throughout three phase I clinical trials and reported excellent tolerability, with no adverse effects.
“This is an important milestone for the company and of course, the PSP patient community who are suffering from this debilitating disease where there are no treatment options currently available, “commented Philippe Verwaerde, CEO of Alzprotect. “All of us involved with the development of AZP2006 are excited by its potential to be used as standalone therapy in early disease, and for its being able to be combined easily with other agents as neurodegenerative diseases progress fast. At the time when novel neurology agents are coming under acute reimbursement scrutiny, a simple modality like AZP2006 makes good sense as an addition to the treatment armamentarium in neurodegenerative diseases. As such this Phase 2A in PSP will serve as an excellent proxy for patient tolerability and early efficacy signals for this drug candidate.”
Alzprotect is developing AZP2006 (EZEPROGIND), an orally available small molecule which has demonstrated that it can augment the functional half-life of Progranulin in the brain to help restore microglia homeostasis in neurodegenerative diseases. By modulating Progranulin through its cognate lysosome-trafficking chaperone protein, Prosaposin, AZP2006 works through a novel mechanism of action from other Progranulin-inducing agents in clinical development so far. Due to its nuanced control of Progranulin, AZP2006 can be used to treat a variety of neurodegenerative diseases and does not have to limited to one/few indications. Upon evaluation of the results from the Phase 2A PSP trial, the Company will launch a US IND for a randomized Phase 2B/3 trial in this indication. Also, as part of its multi-indication drug development plan additional trials in three new indications, GBA-1 Parkinson’s disease, Alzheimer’s disease and Amyotrophic lateral sclerosis will be launched by 2023.
About the Phase 2a study with EZEPROGIND in PSP
The primary objective of this clinical trial is to evaluate the tolerability of the product in PSP patients; to strengthen the pharmacokinetic (PK) and pharmacodynamic (PD) data of the product after 3 month-treatment; and to evaluate the impact of the treatment on the disease’s markers (more than 20 of them, targeting inflammation and neurodegeneration). This Phase 2a study is expected to be completed by July 2022 with top-line results published in Q3 2022. As part of the trial, 36 PSP patients receive a placebo or one of two EZEPROGIND doses which are administered orally for 3 months, followed by a 3-month observation period.
Efficacy endpoints include PK/PD evaluation of AZP2006 in patients, biomarker data across a panel of chosen biomarkers, and evaluating four-different PSP rating scales to be used in subsequent and registrational trials.
About Progressive Supranuclear Palsy (PSP)
PSP is a tauopathy with predominant accumulation of Tau isoforms with four repeat motifs (4R). It is characterized by neurofibrillary degeneration and neuronal loss in the brainstem, basal ganglia, frontal motor and associative cortex. The disease causes brainstem damage that progressively affects balance, vision, mobility, swallowing and speech. The number of PSP cases in Europe and the United States is estimated at 30,000 and 25,000, respectively. The average life expectancy of PSP patients ranges from 5 to 7 years.
Alzprotect imagines and develops therapeutic solutions to slow down or stop neurodegenerative diseases and restore patients’ brain capacity. Founded in 2007, Alzprotect is a French Lille-based company created by Dr. André Delacourte, one of the pioneers of research on Alzheimer's disease, and Dr. Patricia Melnyk, expert in medicinal chemistry, in collaboration with Lille 2 University and INSERM. The company employs 8 people and is supported by BPI France, the National Research Agency and Eurasanté. Alzprotect is committed to the development of innovative therapeutic solutions in the field of neurodegenerative diseases. Alzprotect has 4 international patent families covering the medicines it develops and their indications worldwide.
For more information: http://www.alzprotect.com/en – page LinkedIn – corporate video - Ezeprogind MOA video
Dr Philippe Verwaerde, PhD