Skip to main content

Alzprotect announces the extension of Phase 2a clinical trial for the treatment of progressive supranuclear palsy (PSP)

Lille (France), 16 February 2023 – Alzprotect, the French biopharmaceutical company, announces the extension of its Phase 2a clinical trials of its drug candidate for Progressive Supranuclear Palsy (PSP), a neurodegenerative disease similar to Alzheimer’s disease, which severely impacts balance, eye movements, cognition and, in the more advanced stages of the disease, the ability to swallow.

The initial C04 study (a randomised, blinded study comparing two doses of AZP2006 to placebo) showed positive and promising results. These results have allowed Alzprotect to extend the trial for an additional six months for all eligible patients. Each of the primary objectives of the study were met, including high tolerability and safety for patients. In addition, there was evidence of interaction of the target protein, Progranulin, and promising signs of clinical efficacy.

The National Agency for the Safety of Medicines and Health Products (ANSM in French) and the Ethical Review Board (CPP in French) have given their approval for the extension, which should start in March and will be open-label (i.e. all patients will receive AZP2006 at a known dose).

Alzprotect will also conduct a larger pivotal clinical trial in early 2024, with the aim of accelerating the development of PSP treatment in Europe and the United States.

As Prof. Jean-Christophe Corvol, Professor of Neuropharmacology and Head of the Department of Neurology at the Pitié-Salpêtrière Hospital, points out: “The extension of this clinical phase 2a is good news for the patients who participated in it and will reinforce the promising results of the AZP2006 molecule developed by the Alzprotect teams. More generally, this decision represents additional hope for patients suffering from PSP, a rare and disabling neurodegenerative disease for which no treatment is currently capable of slowing down progression.”

Dr. Susanna Del Signore, Chief Medical Officer, added: “This agreement from the ANSM and the CPP underlines the sound clinical research methodology applied by the Alzprotect team, and will allow us to continue this research, building on the already consistent and informative initial results. This also reinforces the potential of our AZP2006 molecule as a potential treatment for PSP and other neurodegenerative diseases.”

Dr Philippe Verwaerde, Chief Executive Officer and President of Alzprotect, concluded: “The extension of the clinical Phase 2a represents a significant achievement and acknowledgement of the exceptional scientific and operational work of the Alzprotect teams. This milestone marks an important step towards discovering a potential treatment option for patients with PSP, while also presenting an opportunity to build upon the positive results achieved so far. The extension provides the necessary groundwork for the release of the Phase 2a study before advancing to the pivotal Phase II/III trials in Europe and the U.S. It will also support fundraising efforts in both markets to further advance the development of treatments for patients suffering from neurodegenerative diseases, who are in urgent need of treatment options.”

About Alzprotect

Alzprotect is a French biopharmaceutical company specialising in the research and development of innovative therapeutic solutions in the field of neurodegenerative diseases. It was founded in 2007 by Dr André Delacourte, one of the pioneers in Alzheimer’s disease research, and Prof Patricia Melnyk, an expert in medicinal chemistry, in collaboration with the University of Lille 2 and INSERM. Based in Lille, Alzprotect employs 8 people and is supported by BPI France, the French National Research Agency and Eurasanté. Alzprotect has 8 families of international patents covering the medicines it develops and their indications. Since December 2017, Alzprotect has been financially supported by Xerys funds, which has invested a total of €16.3 million to date. For more information:– LinkedIn page – video presentation


Alzprotect is developing AZP2006 (EZEPROGIND), an orally available small molecule that has been shown to increase the functional half-life of progranulin in the brain to help restore microglia homeostasis in neurodegenerative diseases. By modulating progranulin through its lysosome trafficking-related chaperone protein, prosaposin, AZP2006 functions through a novel progranulin-inducing mechanism of action in clinical development to date. Due to its nuanced control of progranulin, AZP2006 can be used to treat a variety of neurodegenerative diseases and is not limited to one/some indications. Following evaluation of the results of the PSP Phase 2a trial, the company will initiate an IND in the US for a randomised Phase 2B/3 trial in this indication. In addition, as part of its multi-indication drug development plan, additional trials in three new indications, Parkinson’s disease GBA-1, Alzheimer’s disease and amyotrophic lateral sclerosis, will be initiated by 2023.

EZEPROGIND is a drug candidate with a clear mode of action that differs from products developed by the pharmaceutical industry over the past 15 years. Unlike most products developed by competitors, EZEPROGIND targets all causes of neurodegeneration and is not only aiming at markers such as amyloid peptide or Tau protein. EZEPROGIND has been granted “orphan drug” status in Europe (European Medicines Agency) and in the United States (Food and Drug Administration) in the indication of Progressive Supranuclear Palsy (PSP). It has been tested in humans on 88 healthy subjects throughout three phase I clinical trials and has demonstrated excellent safety tolerance with no adverse effects. It has also been tested in phase 2a trials in patients with PSP, where it showed good tolerance and promising effects on disease-related symptoms.

About the neurodegenerative diseases PSP and Alzheimer’s 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. Alzheimer’s disease is the most common form of dementia with an estimated 47 million patients worldwide in 2017, a figure that should increase to 75 million by 2030 or 132 million by 2050, according to the 2017 World Alzheimer Report. The pharmacological targets are Abeta protein, Tau protein and neuroinflammation.

Press Contact:

FTI Consulting Strategic Communications
Dorine Rohou – T + 33 (0) 1 47 03 65 58
E-mail :

AlzProtect, Xerys


73 boulevard haussmann

75008 Paris - France

+33 (0)1 82 52 12 25