News
London, United Kingdom – April 25, 2025 –
The Progressive Supranuclear Palsy (PSP) Trial Platform (PTP), led by Drs. Adam Boxer (University of California, San Francisco [UCSF]), Irene Litvan (University of California, San Diego), Julio Rojas (UCSF) and Anne-Marie Wills (Massachusetts General Hospital), has selected two promising drug candidates—Axon Neuroscience’s AADvac1 and Alzprotect’s AZP2006—for inclusion in the platform trial as the first two compounds to be evaluated. The trial aims to accelerate the development of effective treatments for PSP, a rare and fatal neurodegenerative disease. Additional compounds to be evaluated in the trial are expected to be announced later this year.
Funded by the National Institute on Aging (NIA), part of the U.S. National Institutes of Health (NIH), the platform trial is supported by a five-year grant. Its design enables multiple therapies to be tested concurrently, establishing a perpetual, efficient and flexible path to evaluate promising drug candidates.
“This public-private partnership represents an unprecedented opportunity to accelerate the development of treatments for PSP,” said Professor Adam Boxer, endowed professor in memory and aging in the UCSF Department of Neurology, as well as a principal investigator for the trial. “By bringing together innovative therapeutic approaches like AADvac1 and AZP2006 in an efficient clinical trial design, we aim to address the urgent needs of patients and their families in less time, at a lower cost and with fewer patients on placebo than traditional clinical trials.”
Scientific Innovations: AADvac1 and AZP2006
AADvac1, developed by Axon Neuroscience (Bratislava, Slovakia), is an active immunotherapy targeting pathological Tau proteins, which induce and drive PSP and Alzheimer’s disease (AD) pathology. The active immunotherapy elicits production of antibodies that bind to abnormal pathological Tau, preventing its aggregation and spread, and facilitating its clearance by microglia. In a completed 24-month Phase 2 study in Alzheimer’s Disease, AADvac1 demonstrated a favorable safety profile, with therapeutic effects on plasma and cerebrospinal fluid biomarkers that could signal a slowing of disease progression.
“We are proud and grateful that AADvac1 has been selected by the expert committee for this innovative platform trial,” said Michal Fresser, CEO of Axon Neuroscience. “We believe the Tau-targeting active immunotherapy approach of AADvac1 holds strong potential in the treatment of human tauopathies, and we look forward to partnering with the PSP Trial Platform and the PSP research community to bring forward new treatment options for patients facing a significant unmet medical need.”
AZP2006, developed by Alzprotect (Lille, France), is a synthetic small molecule designed to restore lysosomal homeostasis and modulate progranulin (PGRN), while also reducing Tau aggregation and neuroinflammation. Preclinical studies demonstrated its ability to correct lysosomal dysfunction in neurodegeneration models. In a 3-month Phase 2a study in PSP patients, AZP2006 demonstrated encouraging clinical and biomarker signals of efficacy. These positive outcomes were further supported by results from a 6-month open-label extension study, reinforcing its therapeutic potential. The compound has received Orphan Drug Designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
“We are honored that AZP2006 has been selected for inclusion in this landmark PSP platform trial,” said Philippe Verwaerde, CEO of Alzprotect. “We are excited to collaborate with the PSP Trial Platform and the broader PSP community to advance innovative therapies for patients with our scientific approach targeting lysosomal dysfunction.”
About PSP Trial Platform – Collaborative Effort
The trial's steering committee includes researchers: Dr. Adam Boxer (UCSF), Dr. Julio Rojas-Martinez (UCSF), Dr. Anne-Marie Wills (Massachusetts General Hospital), and Dr. Irene Litvan (University of California, San Diego). CurePSP—the leading nonprofit advancing research, patient services, and advocacy for PSP and other underserved neurodegenerative diseases—is collaborating with UCSF and approximately 50 trial sites to recruit participants and ensure patient perspectives are integrated throughout the trial. “I am thrilled to see AADvac1 and AZP2006 enter this landmark platform trial—a powerful, multi‑stakeholder collaboration that unites academia, industry, and our community, which we are proud to support,” said Kristophe Diaz, PhD, Executive Director and Chief Science Officer of CurePSP. “This collaborative platform design can compress years of drug development into real, near‑term hope for people living with PSP.”
Enrollment for the trial is expected to begin at the end of 2025, focusing on patients with Richardson’s syndrome, the most common form of PSP. The trial aims to enroll a population that is fully representative of the US population by providing language support and covering transportation and accommodation costs.
This platform trial represents a collaborative effort among academia, industry, and patient advocacy groups to accelerate the discovery of effective treatments for PSP and improve the lives of those affected by this devastating disease. To learn more about the trial, visit https://www.psp.org/ptp.
About Tau Global Conference
Tau Global Conference 2025 brings together three major Tau-focused conferences (Global Tau, EuroTau and CurePSP Neuro), and is hosted by the Alzheimer’s Association, CurePSP and the Rainwater Charitable Foundation. This conference plays a critical role in bringing together interdisciplinary researchers and perspectives to move Tau research forward.
About Axon Neuroscience
Axon Neuroscience was founded in 1999 by immunologist Professor Michal Novak. In 1988 Professor Novak discovered Tau protein as the major component of neurofibrillary pathology in Alzheimer’s disease while working in Laboratory of Molecular Biology, MRC in Cambridge, UK. Axon has been developing active immunotherapy, monoclonal antibody and small molecules for treatment of neurodegenerative diseases and tauopathies.
About Alzprotect
Founded in 2007, Alzprotect is a French Lille-based company committed to the development of innovative therapeutic solutions in the field of neurodegenerative diseases. Including tauopathies, amyloidopathies and synucleinopathies Alzprotect is advancing in the development of AZP2006 (EZEPROGIND), an innovative synthetic molecule administered orally, designed to optimize lysosome homeostasis by regulating both Progranulin and its chaperone protein, Prosaposin.
Lille, France – December 5th, 2024 – Alzprotect, a pioneering biopharmaceutical company developing innovative treatments for neurodegenerative diseases, is pleased to announce the appointment of Professor Christian Bailly to its Board of Directors.
Professor Christian Bailly brings to Alzprotect substantial expertise in pharmaceutical research and life sciences, built over more than 30 years of experience. Currently an Associate Professor at the University of Lille and an independent consultant for the pharmaceutical industry, Professor Bailly has also held leadership roles in the pharmaceutical sector, notably as Director of Research and Development at Pierre Fabre Médicament.
Renowned for his skills in drug discovery and fostering collaborations between academia and industry, Professor Bailly has led multiple research centers and overseen innovative programs that have resulted in the development of new therapies. His extensive expertise in chemistry, biology, and pharmacology, combined with his commitment to bridging academic and industrial research, will be invaluable in supporting Alzprotect’s strategy for developing treatments for neurodegenerative diseases.
“We are delighted to welcome Professor Christian Bailly to our Board of Directors,” said Dr. Philippe Verwaerde, President of Alzprotect. “His exceptional career and scientific expertise will strengthen our ability to successfully advance our research projects and achieve our strategic goals, particularly in the development of our drug candidate AZP2006.”
Professor Bailly will contribute to shaping Alzprotect’s scientific and strategic vision, emphasizing innovation and the development of treatments that address the needs of patients suffering from conditions such as progressive supranuclear palsy, Alzheimer’s disease, and Parkinson’s disease.
Comment from Professor Bailly, new member of the Board of Directors: "I am thrilled to join the board of one of France’s most innovative companies in the field of neurodegenerative disease treatment, at a pivotal moment as the company transitions to Phase 2/3 trials and pursues ambitious growth. I look forward to contributing to their R&D activities and to their much-anticipated success in serving patients."
Loos March 13Th 2024
ALZPROTECT is thrilled to announce that it has received favorable feedback from both the American (FDA) and European (EMA) authorities regarding the regulatory path for advancing the clinical development of Ezeprogind/AZP2006 for patients with PSP. This significant milestone follows the already very promising results of the phase 2a study in PSP and marks a decisive advancement. It will provide a clear path for the further development of Ezeprogind/AZP2006 within both scientific and regulatory expectations.
Regulatory authorities have provided crucial insights into various design elements, with a particular focus on selecting study endpoints pertinent to the PSP indication and ensuring that the demographic and characteristic profile of the study sample closely mirrors the global PSP population. This feedback has armed Alzprotect with a valuable roadmap, enabling the refinement of the intended Phase 2/3 study protocol to ensure seamless alignment with scientific standards and regulatory requirements. With well-defined regulatory pathways for the PSP indication, Alzprotect's forthcoming Proof of concept randomized controlled PROMISE-PSP trial, scheduled to commence in late 2024, is set to deliver outcomes that align with scientific and regulatory expectations across the United States and the European Union.
Dr. Artin Karapet, our Chief Medical Officer, expressed his heartfelt joy stating, "I am truly delighted to witness this alignment between agencies from different shores on critical program elements. Their guidance not only paves a feasible path forward but also brings AZP2006 one step closer to our patient community."
Phil Verwaerde, Chief Executive Officer, added, "The alignment with regulatory authorities marks a pivotal moment for ALZPROTECT. It validates our vision and the potential of Ezeprogind/AZP2006 not only for PSP but also for a broader neurodegenerative disease spectrum. We are dedicated to accelerating our efforts to meet the urgent needs of patients."
The molecule, AZP2006, proposes a fundamentally distinct mechanism of action compared to existing treatments: Targeting the root causes of neurodegeneration by stimulating the action of a protein essential to the functioning of the brain, Progranulin.
AZP2006 has shown excellent safety and promising efficacy in human trials, including 102 healthy subjects across three Phase I trials and 36 PSP patients in a Phase 2a trial. Alzprotect plans to initiate a randomized Phase 2b/3 proof of concept for PSP and expand its drug development to include Parkinson's disease, Alzheimer's disease, and amyotrophic lateral sclerosis in the coming years.
Loos, september 21st 2023
World Alzheimer’s day 2023
ALZPROTECT DEVELOPS AN INNOVATIVE TREATMENT AND EXPANDS ITS CLINICAL TRIALS, FOLLOWING VERY POSITIVE RESULT
Alzprotect, a biopharmaceutical company based in Lille, is currently testing an innovative drug against a neurodegenerative disease closely related to Alzheimer's disease. The very good results obtained have allowed Alzprotect to extend the clinical trial of this treatment for 6 months, and to prepare a larger scale study. The molecule being tested, AZP2006, offers a mode of action radically different from existing treatments, and brings considerable hope to all the patients concerned. Alzprotect is financed by the Xerys Invest funds (Paris)
Alzprotect, a French biopharmaceutical company, is testing an innovative drug against Progressive Supranuclear Palsy (PSP), a neurodegenerative disease closely related to Alzheimer's disease.
The molecule being tested, AZP2006, proposes a fundamentally distinct mechanism of action compared to existing treatments: it is the first to target all the causes of neurodegeneration, by stimulating the action of a protein essential to the functioning of the brain, Progranulin. This mode of action makes the molecule capable of acting on several neurodegenerative diseases.
The Lille University Hospital, the Pitié-Salpêtrière Hospital, and the Adolphe de Rothschild Foundation Hospital are participating in the clinical trials.
The clinical trials, currently in phase 2a, were conducted in three centers: the Lille University Hospital under the direction of Professor Defebvre, the Pitié-Salpêtrière Hospital with Professor Corvol, and the Adolphe de Rothschild Foundation Hospital with Dr. Obadia. The study revealed promising results: the treatment was perfectly tolerated, And the first signs of efficacy have been observed and will need to be confirmed in the upcoming phase 2/3 study.
These good results have allowed the extension of the trial: patients will be able to benefit from the treatment for an additional 6 months, thanks to the approval granted by the National Agency for the Safety of Medicines (ANSM) and the Committee for the Protection of Persons (CPP). Furthermore, Alzprotect is preparing a larger phase 2/3 trial, in Europe and the United States, to test the efficacy of the molecule over a 12-month treatment period.
A molecule capable of targeting several neurodegenerative diseases.
« The extension of this phase 2a clinical trial is good news for the patients who participated in it and will reinforce the promising results for the AZP2006 molecule, says Pr. Jean-Christophe Corvol, Professor of Neuropharmacology and head of the Neurology Department at Pitié-Salpêtrière. More generally, this decision represents additional hope for patients suffering from PSP, a debilitating and rare neurodegenerative disease, for which no treatment has been able to halt the progression to date. ».
« This progress concerns not only the patients involved, but also potentially people suffering from other neurodegenerative diseases," adds Philippe Verwaerde, CEO of Alzprotect. "We plan to launch trials with patients suffering from Alzheimer's disease, or even Parkinson's disease, very soon».
Lille (France), on 6th March 2023, Alzprotect, a biotechnology company focused on developing treatments for neurodegenerative diseases, announced the appointment of Dr. Olivier Defert as Chief Operating Officer (COO). Dr. Defert brings over 20 years of experience in the biotech and pharmaceutical industries, having held leadership positions in several companies. He is a highly qualified medicinal chemist who has held leadership positions at BioVersys SAS and Tarsier Pharma, among others. He founded Olea MedChem Consulting, where he served as CEO from 2009 to 2021. In 2010, he co-founded the company Amakem NV, where he managed CMC, IND-enabling studies, and medicinal chemistry as Director of Preclinical Development. He has managed several research programs and collaborations with academic institutions, pharmaceutical companies, and investors. His expertise in medicinal chemistry, CMC, preclinical development, and project management will be essential in advancing Alzprotect's drug development programs. The appointment of Dr. Defert as COO underscores Alzprotect's commitment to building a world-class team and advancing its mission to develop innovative therapies for neurodegenerative diseases. Alzprotect is funded by Xerys Invest.
Dr. Philippe Verwaerde, CEO and President of Alzprotect, stated, "We are thrilled to welcome Dr. Olivier Defert as Alzprotect's new Chief Operating Officer. His extensive experience and exceptional leadership skills in the biotech and pharmaceutical industries make him the ideal candidate to help drive our growth and innovation in developing drugs for Alzheimer's disease. Dr. Defert's expertise in medicinal chemistry, CMC, preclinical development, and project management will be a major asset to our team as we work to develop new therapies for neurodegenerative diseases. We are confident that his appointment will accelerate our mission to offer innovative and effective treatments to patients and families affected by PSP and Alzheimer's disease. We are privileged to have Dr. Defert on our team and look forward to progressing together."
Dr. Noelle Callizot, Chief Science Officer, added: "Olivier's arrival is an opportunity for Alzprotect and will accelerate the development of AZP2006 in PSP and in new therapeutic indications. His experience also allows us to quickly approach the development of AZP2006 at an international level."
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 validation of the extension of the clinical Phase 2a represents a recognition of the outstanding scientific and operational work of the Alzprotect teams. This milestone is a further step towards the discovery of a potential treatment option for patients with PSP, as well as an opportunity to build on the positive results obtained to date. The extension will better prepare for the release of the Phase 2a study prior to the pivotal Phase II/III in Europe and the U.S., while supporting fundraising efforts in both markets to further develop treatments for patients with neurodegenerative diseases requiring treatment options.”
Lille (France), 20 September 2022 – On the eve of world Alzheimer’s day, Alzprotect, a biopharmaceutical company developing treatments for neurodegenerative diseases, announces the completion of the phase 2a clinical trial evaluating its platform asset, AZP2006 (“EZEPROGIND”) in Progressive Supranuclear Palsy (PSP), a rare neurodegenerative disease, sharing key features with Alzheimer’s disease, and which seriously impacts balance, eye movement, cognition, and in the latter stage of the disease, the ability to swallow.
The clinical trial, which is part of a multi-indication development plan for AZP2006, was conducted at the Pitié-Salpêtrière University Hospital of Paris, the University Hospital of Lille, and the Hospital Foundation Adolphe de Rothschild on 36 patients whose recruitment was successfully completed in January.
The primary objectives of the study were met, namely the investigational product good safety and tolerability in PSP patients. Clinicians were satisfied with symptom changes. The results of CSF and plasma biomarkers and clinical exploratory endpoints will be reported later this year.
This success allows Alzprotect's team to envision the development of the PSP treatment in Europe and the United States as well as an acceleration of the development of treatments for other indications, in particular Alzheimer's disease, which could thus be the target indication of clinical trials by the end of 2023.
AZP2006 has been granted Orphan Drug status by the U.S. Food and Drug Administration (FDA) and the 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.
Dr. Susanna Del Signore, Chief Medical Officer, commented: “This is a real success for Alzprotect to have completed in time a methodologically complex study which will allow for, once its bio-analytical and pharmacokinetic results are integrated with the excellent clinical safety data, confirmed by the Data and Safety Monitoring Board on several occasions, the proposal of a confirmatory study plan to the European Agency and the FDA early next year. This represents real hope for the PSP patient community.”
As Dr. Jean-Christophe Corvol, Professor of Neuropharmacology and Head of the Department of Neurology at the Pitié-Salpêtrière Hospital, reminds us: "PSP is a rare but disabling neurodegenerative disease for which no treatment is currently capable of slowing down the progression. He explains: "AZP2006 would reduce neuronal loss and preserve the function of neurons in the areas of the central nervous system affected by PSP. The completion of this phase 2a clinical trial with the support of the Alzprotect teams is very encouraging.”
"We are very pleased with these clinical results which are excellent indicators of patient tolerance and promising signals for our drug candidate. We strongly believe in the potential of AZP2006 as a potential treatment for neurodegenerative diseases", said Dr Philippe Verwaerde, CEO of Alzprotect. He concludes: "Now, the next structuring steps are the publication of our phase 2a study, the preparation of the pivotal phase II/III in both Europe and the United States, and a fundraising process to pursue our projects and support people suffering from neurodegenerative diseases in therapeutic impasse," .
Lille, France – June 1st 2022 – Alzprotect, a French biopharmaceutical company involved in the development of new therapeutic solutions in the neurodegenerative diseases area, today announced it has been awarded a USD $338,000 grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to develop the first-in-class brain penetrant small molecule targeting Progranulin (PGRN).
This grant will further expand Alzprotect’s research efforts to develop AZP2006 (INN Ezeprogind) into a new therapeutic area: Parkinson's disease (PD), with a focus on the GBA1 mutation-associated with Parkinson's disease (PD/GBA1). The grant will fund an early-stage program at Alzprotect to study the effects of AZP2006 in models of PD based on loss of function from preliminary results and a strong rationale. The objectives are to investigate the target engagement of AZP2006 and to perform a proof-of-concept (POC) study in an animal model of PD/GBA1.
Dr. Noelle Callizot, CSO declared: “We are pleased and very proud to have the scientific and financial support of the MJFF. The novel mode of action of AZP2006 involving the lysosome and progranulin allows us to consider the development of AZP2006 in PD/GBA1 patients. The excellent safety profile of AZP2006 makes it a promising candidate drug for these patients. We thank MJFF for its financial assistance in contributing to the achievement of our research goals.”
Dr. Philippe Verwaerde, President and CEO of Alzprotect, said, " We are very proud of the recognition of our preliminary research advances in Parkinson’s and of the potential of our lead compound AZP2006 based on progranulin modulation. This research progress is materialized today by this grant from the Foundation.”
France, Lille - 5th April 2022 - Alzprotect, a French biopharmaceutical company specialised in the development of new therapeutic solutions for the treatment of neurodegenerative diseases, in particular Alzheimer's disease, appoints Dr Susanna Del Signore as Chief Medical Officer (CMO).
Dr Susanna Del Signore has over 20 years of experience in research and development (R&D) in the pharmaceutical industry, particularly in the field of neurology. She has been a project leader, attaining the position of Chief Medical Officer (CMO), for the development of drugs across phases I to III. She has held increasing responsibility in large pharmaceutical (Servier and then Sanofi, between 1993 and 2015) and biotechnology companies (Biophytis between 2016 and 2018). From 2005 to 2009 she worked at the European Medicines Agency (EMA), in the evaluation of new pharmacological treatments for neurodegenerative diseases, liaising with European national agencies, the Food and Drug Administration (FDA), and clinical experts. Susanna Del Signore founded BLUECOMPANION in 2015, a start-up focused on the design and implementation of e-health projects for public-private partnerships and on innovative methodologies for the generation of regulatory quality clinical data. She has built meaningful expertise in the design and conduct of first-in-man and phase 2 clinical trials, clinical development planning, and regulatory strategy, both in France and internationally (Europe, USA, Japan). For instance, she contributed to a global regulatory policy project within the MIT-led think tank on adaptive licensing. In 2013, she coordinated on behalf of Sanofi and in collaboration with Eli Lilly, Novartis and GSK, an IMI (Innovative Medicines Initiatives) call for projects in geriatrics, targeting age-related sarcopenia as a new indication.
Dr Philippe Verwaerde, founder and CEO of Alzprotect, says: "I am delighted to welcome Susanna to our team; she has significant experience as a Chief Medical Officer and has a solid understanding not only of the issues related to the development of new generation therapies, but also of protocols and clinical trials as they evolve, arbitration, and reallocation of resources based on results and strategy. She has mastered, in all its ethical nuances, the imponderable need to discuss with the wider scientific community, KOLs, and patient associations according to the prerogatives set out by the regulatory authorities. We are confident that her experience combined with her managerial skills will be of great value to Alzprotect as we prepare to further develop AZP2006 in Phase 2/3 clinical trials for the treatment of progressive supranuclear palsy (PSP). Dr. Susanna Del Signore is a great asset to complement our team and and we are honored to have her support in the implementation of our clinical plan.”
"I am particularly proud be joining Alzprotect, a persevering French biotech company that is wholeheartedly and relentlessly dedicated to the development of ground-breaking therapies for neurodegenerative diseases, with products based on a novel and promising mechanism of action. I am thrilled to have been offered the opportunity to work on Progressive Supranuclear Palsy, an orphan neurodegenerative disease that primarily affects older persons, usually after the age of seventy . Such a disease should be diagnosed as early as possible and treated with a view to slowing motor and cognitive decline. I will be listening to and working with patients, their families, and the medical community to implement in our planned clinical trials the best methodologies," commented Dr Susanna Del Signore.
Olivier Ossipoff, Chairman of Xerys Invest, added: "We are very pleased to see Dr Susanna Del Signore appointed as Chief Medical Officer and join the Alzprotect team. Her arrival marks an important step in the evolution of this exceptional biotech company that we have been supporting since 2017 and underlines its desire to accelerate the implementation of its clinical development plan."