Ferrer Completes Recruitment of 220 Patients for the PROSPER Study, a Phase II Clinical Trial in Progressive Supranuclear Palsy (PSP), Two Months Ahead of Schedule

This is a randomized, double-blind, placebo-controlled Phase II clinical trial designed to evaluate the efficacy, safety, and pharmacokinetics of FNP-223 (oral formulation) in slowing the progression of progressive supranuclear palsy (PSP)The study is being conducted across 44 centers in the European Union, the United Kingdom, and the United StatesThe study reinforces Ferrer’s commitment to researching and developing transformative therapeutic solutions for people with rare neurological diseases

Spanish international pharmaceutical company Ferrer has announced the completion of patient recruitment for the PROSPER study, a Phase II clinical trial designed to assess the efficacy, safety, and pharmacokinetics of FNP-223, an orally available, potent and selective inhibitor of the OGA enzyme, in-licensed from Asceneuron, aimed at slowing disease progression in Progressive Supranuclear Palsy (PSP)¹.

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Ferrer's pharmaceutical production plant in Sant Cugat del Valles, Barcelona, Spain.

PSP is a rare, rapidly progressive and ultimately fatal neurodegenerative disease². To date, no disease-modifying therapies have been approved for PSP.

The PROSPER study is a randomized, double-blind, placebo-controlled trial³ that has successfully recruited the planned number of 220 participants with PSP in just 14 months, achieving this milestone on October 6^th, two months ahead of schedule. A total of 44 centers across the European Union, the United Kingdom, and the United States are participating. The study design includes a six-week screening period, followed by 52 weeks’ treatment with either FNP-223 or placebo and a subsequent four-week follow-up period after the completion of treatment³.

The study focuses on participants with early-stage progressive supranuclear palsy–Richardson syndrome (PSP-RS), a critical stage during which early intervention may have the greatest impact on slowing disease progression. Identifying the disease at this stage is a significant challenge due to its rapid progression and diagnostic complexity, making the achievement of this recruitment milestone ahead of schedule even more meaningful.

Prof. Dr. Med. Günter Höglinger, from the Ludwig-Maximilian University of Munich, and principal investigator and coordinator of the PROSPER study, stated: “I am deeply grateful to the patients, their caregivers, and the dedicated teams at our academic centers for their outstanding commitment, which has enabled us to reach this important milestone so swiftly. Together, we are making significant progress toward answering a critical scientific question: the role of OGA inhibition in PSP. Our shared efforts bring us closer to developing solutions that may truly improve the lives of patients.”

According to Kristophe Diaz, Chief Executive Officer of CurePSP, “Achieving full enrollment in a PSP clinical trial is no small feat, and Ferrer’s success with the PROSPER study is both a scientific and human milestone. Behind this progress are patients and families who choose to turn hope into action, and their participation accelerates the entire ecosystem toward meaningful therapies for PSP. For our community, each step like this brings us closer to lasting change.”

Oscar Pérez, Chief Scientific Officer at Ferrer, noted: “The early completion of recruitment marks a key step in Ferrer’s commitment to accelerating clinical research programs for complex and rare diseases such as PSP. In line with our purpose of using business to fight for social justice, we hope to deliver a potential solution that transforms the lives of people affected by this disease and those around them. We are deeply grateful to the patients, families, caregivers, investigators and patient associations for their trust and support, without which this milestone would not have been possible.”

Ferrer has also incorporated input from patients, caregivers, and healthcare professionals into the design of the PROSPER study, with the goal of tailoring clinical research to the real needs of participants, improving their experience, and ensuring more impactful clinical research.

Ferrer’s commitment to research

Ferrer is an international company based in Barcelona, strongly committed to the research and development of innovative therapies in areas of high unmet medical need, such as rare neurological diseases and pulmonary vascular and interstitial diseases. The company focuses on complex, global clinical developments, advancing projects across multiple stages of research.

With studies like PROSPER, Ferrer reinforces its mission to generate positive social impact by developing transformative solutions for serious and low-prevalence diseases that currently lack approved or available treatments.

About PSP

Progressive supranuclear palsy (PSP) is a rare neurodegenerative disease that primarily affects people over 60 years old and progresses rapidly, leading to severe disability.

PSP is a primary tauopathy caused by the abnormal accumulation of the tau protein in areas of the brain that control movement and cognition, particularly in subcortical regions and the brainstem^4,5. This buildup damages nerve cells and disrupts brain function, resulting in symptoms such as loss of balance, difficulty speaking or swallowing, abnormal eye movements, and cognitive or mood disturbances⁶.

Although PSP is rare, affecting between 5.8 and 6.5 people per 100,000⁷, its impact is profound: loss of independence typically occurs within a few years⁷, and average life expectancy after diagnosis ranges from 6 to 9 years. Genetic cases are uncommon⁸, though more than ten associated genes have been identified, with mutations in the MAPT gene being the main risk factor⁹. The potential role of environmental factors, such as exposure to toxins or infectious agents, is also under investigation⁷.

About Ferrer

At Ferrer we use business to fight for social justice. We have long been a company that looks to do things differently; instead of maximizing shareholder returns, we reinvest much of our profit in initiatives that give back to society. Back where it belongs. We go beyond compliance and are guided by the highest standards of sustainability, ethics and integrity. As such, we are the highest-rated B Corp pharma company in the world.

Founded in Barcelona in 1959, Ferrer offers transformative solutions for life-threatening diseases in more than one hundred countries. In line with our purpose, we have an increasing focus on pulmonary vascular and interstitial lung diseases and rare neurological diseases. Our 1,800-strong team is driven by a clear conviction: our business is not an end in itself, but a way to change lives.

We are Ferrer. Ferrer for good.
www.ferrer.com

References:

1. Permanne B, Sand A, Ousson S, Nény M, Hantson J, Schubert R, Wiessner C, Quattropani A, Beher D. O-GlcNAcase Inhibitor ASN90 is a Multimodal Drug Candidate for Tau and α-Synuclein Proteinopathies. ACS Chem Neurosci. 2022 Apr 20;13(8):1296-1314. doi: 10.1021/acschemneuro.2c00057. Epub 2022 Mar 31. PMID: 35357812; PMCID: PMC9026285.
2. Höglinger GU, et al. Mov Disord. 2017;32(6):853–864; 2. Agarwal S and Gilbert R. Progressive Supranuclear Palsy. [Updated 2023 Mar 27]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan. Available at: www.ncbi.nlm.nih.gov/books/NBK526098/ (Accessed: August 2025); 3. Boxer LB, et al. Lancet Neurol. 2017;16(7):552–563
3. ClinicalTrials.gov: A Randomized, Double-blind, Placebo-controlled, Phase 2 Study to Assess the Efficacy, Safety, and Pharmacokinetics of FNP-223 (Oral Formulation) to Slow the Disease Progression of Progressive Supranuclear Palsy (PSP) (PROSPER). ClinicalTrials.gov [Internet]. Available at: https://www.clinicaltrials.gov/study/NCT06355531. Accessed on 01/10/2025
4. Murray ME, Kouri N, Lin WL, Jack CR, Jr., Dickson DW, Vemuri P. Clinicopathologic assessment and imaging of tauopathies in neurodegenerative dementias. Alzheimers Res Ther. 2014;6(1):1. doi: 10.1186/alzrt231.
5. Planche V, Mansencal B, Manjon JV, Meissner WG, Tourdias T, Coupé P. Staging of progressive supranuclear palsy-Richardson syndrome using MRI brain charts for the human lifespan. Brain Commun. 2024;6(2):fcae055. doi: 10.1093/braincomms/fcae055.
6. National Institute of Neurological Disorders and Stroke (NINDS) [Internet]. Bethesda: National Institutes of Health; start date unknown [last updated date unknown; cited 23 Apr 2025]. Available at: https://www.ninds.nih.gov/health-information/disorders/progressive-supranuclear-palsy-psp.
7. Agarwal S, Gilbert R. Progressive Supranuclear Palsy. StatPearls. Treasure Island (FL): StatPearls Publishing
8. Ichikawa-Escamilla E, Velasco-Martínez RA, Adalid-Peralta L. Progressive Supranuclear Palsy syndrome: an overview. IBRO Neurosci Rep. 2024;16:598-608. doi: 10.1016/j.ibneur.2024.04.008.
9. Donker Kaat L, Boon AJ, Azmani A, Kamphorst W, Breteler MM, Anar B, et al. Familial aggregation of parkinsonism in progressive supranuclear palsy. Neurology. 2009;73(2):98-105. doi: 10.1212/WNL.0b013e3181a92bcc.

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