CurePSP Announces Enrollment of First Participant in the PSP Trial Platform

NEW YORK, Aug. 07, 2026 (GLOBE NEWSWIRE) -- The PSP Trial Platform (PTP), a landmark clinical trial testing multiple drugs simultaneously in the search for effective PSP treatments, has enrolled its first participant.

Progressive supranuclear palsy (PSP) is a rare brain disorder that can mimic Parkinson’s disease in loss of balance, movement disorders and stiffness. There is no cure and no approved treatment, in part due to the limited number of clinical trials for potential treatments.

Led by the University of California, San Francisco (UCSF) and funded by a five-year grant of up to $75.4 million from the National Institute on Aging (NIA), the PTP is being conducted at 50 sites nationwide. Modeled on the HEALY ALS Platform Trial, it tests multiple drugs concurrently — and new ones can be added as they become available — accelerating discovery without starting from scratch each time. The trial aims to enroll 440 participants over 2 years. Unlike traditional clinical trials, the PTP is designed so that 75% of participants receive an active drug during the first year, reducing the number of patients assigned to placebo. After that, all participants receive an active drug.

“We expect this trial to rapidly accelerate efforts to identify effective PSP therapies by increasing the number of promising drugs tested, while expanding access to potential reatments to more patients. It will also create a wealth of longitudinal data to enable researchers to better understand the causes of PSP and develop new diagnostic tests and therapies,” said Adam Boxer, MD, PhD, Endowed Professor in Memory and Aging at UCSF, and lead study Principal Investigator.  

“The enrollment of the first participant marks the beginning of an important new chapter for the PSP community,” said Kristophe Diaz, PhD, Chief Executive Officer of CurePSP. “The PTP is one of the most ambitious collaborative efforts ever undertaken in PSP research, bringing together leading researchers, trial sites, industry, government and nonprofit organizations to accelerate the development of better therapies.”

“This is good news for patients and their families who are struggling with this disease. We are hopeful that this enrollment is the first step towards finding an effective treatment for this illness,” said Anne-Marie Wills, MD, MPH, Director of the CurePSP Center of Care at Massachusetts General Hospital, and study co-Principal Investigator.

Two of the drugs being tested are LM11A-31, from PharmatrophiX, and AADvac1, from Axon Neuroscience. “Rather than targeting a single downstream consequence of disease, LM11A-31 is designed to protect neurons and their synaptic connections while addressing multiple mechanisms that drive neurodegeneration, including toxic tau biology,” said Dr. Frank M. Longo, Co-Founder of PharmatrophiX. “The first patient enrolled represents more than the start of a clinical trial, it is the beginning of a potential paradigm shift for these patients and families.”

“AADvac1's inclusion is an important step forward, built on more than two decades of our dedicated research into tau protein, a key driver of the disease pathology in PSP,” said Michal Fresser, CEO of Axon Neuroscience. “We remain committed to advancing science that may one day lead to meaningful therapy options.”

The PTP is open to patients with Richardson's syndrome who have experienced progressive symptoms for fewer than five years and are accompanied by a care partner.

For more information, visit our website at https://www.psp.org/ptp or view our ClinicalTrials.gov listing (NCT07173803) https://clinicaltrials.gov/study/NCT07173803 for a complete list of eligibility criteria. You can also complete the UCSF PSP Trials Prescreening survey to see if you may be eligible for any currently enrolling PSP trials, including PTP.

About UCSF
The University of California, San Francisco (UCSF) is exclusively focused on the health sciences and is dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. UCSF Health, which serves as UCSF’s primary academic medical center, includes top-ranked specialty hospitals and other clinical programs, and has affiliations throughout the Bay Area. UCSF School of Medicine also has a regional campus in Fresno. Learn more at https://ucsf.edu or see our Fact Sheet.

About CurePSP
CurePSP is the leading nonprofit organization dedicated to the awareness, care and cure for three neurodegenerative diseases: progressive supranuclear palsy (PSP), corticobasal degeneration (CBD) and multiple system atrophy (MSA). As a catalyst for new treatments and a cure, CurePSP establishes important partnerships and funds critical research internationally. Through its advocacy and support efforts, CurePSP enhances education, care delivery and quality of life for people living with PSP, CBD and MSA and their families. Science, community and hope are at the heart of CurePSP's mission and all its services. CurePSP is a registered 501(c)(3) charity within the United States (EIN: 52-1704978).

Contact:
Kristophe Diaz, PhD
Chief Executive Officer, CurePSP
646-725-1453
diaz@curepsp.org