CurePSP's PSP Trial Platform enrols first participant
CurePSP and the University of California, San Francisco have announced the enrolment of the first participant in the PSP Trial Platform (PTP), a multi-arm adaptive trial designed to evaluate several candidate therapies for progressive supranuclear palsy simultaneously. The trial is funded by a five-year grant of up to $75.4 million from the National Institute on Aging and will run across 50 sites in the United States.
Progressive supranuclear palsy is a rare, rapidly progressing neurodegenerative disorder that shares features with Parkinson's disease, including balance impairment, movement difficulties and stiffness. There is no approved treatment, and the condition's rarity has historically made it difficult to recruit sufficient numbers of patients for conventional randomised trials.
Trial design and initial drug arms
The PTP is modelled on the HEALY ALS Platform Trial, which demonstrated that platform designs can meaningfully compress development timelines in rare neurological disease. The PSP platform is structured so that 75% of participants receive an active treatment in the first year, with all participants receiving an active drug thereafter. The trial targets enrolment of 440 people with Richardson's syndrome who have experienced progressive symptoms for fewer than five years. New drug arms can be added as additional candidates become available, without restarting the platform from scratch.
Two therapies are already included. LM11A-31, developed by PharmatrophiX, is described by co-founder Frank M. Longo as a neuroprotective agent targeting multiple mechanisms of neurodegeneration, including toxic tau biology. AADvac1, from Axon Neuroscience, is an active vaccine directed at tau protein; the Slovak company says the candidate is built on more than two decades of tau research. Both drugs target tau pathology, which is the defining pathological hallmark of PSP, though they do so through distinct mechanisms.
Adam Boxer, the endowed professor in memory and ageing at UCSF leading the study, said the trial would "create a wealth of longitudinal data to enable researchers to better understand the causes of PSP and develop new diagnostic tests and therapies." Anne-Marie Wills, director of the CurePSP Centre of Care at Massachusetts General Hospital and co-principal investigator, described first enrolment as "the first step towards finding an effective treatment."
Market and competitive context
The wider tauopathy landscape has attracted sustained interest from larger pharmaceutical companies, though a series of high-profile failures in Alzheimer's disease-related tau programmes has tempered expectations. PSP-specific development has been comparatively sparse, with no approved therapy and only a handful of late-stage studies completed to date. Platform trials are increasingly regarded by regulators and funders as the preferred approach for rare neurodegenerative conditions because they allow shared infrastructure, control arms and biomarker collection to be used across multiple investigational drugs, substantially reducing per-drug costs.
The NIA grant, administered through UCSF, is among the larger single investments in PSP research recorded publicly. CurePSP, a registered non-profit, serves as the patient advocacy and partnership co-ordinator for the effort, which also involves industry sponsors and academic trial sites. The inclusion of an industry partner such as Axon Neuroscience alongside an earlier-stage asset from PharmatrophiX illustrates the coalition model the platform is designed to facilitate.
Near-term milestones include completion of planned enrolment within two years and interim readouts on individual drug arms as data accumulate. The platform's adaptive structure means that arms showing insufficient signal could be dropped and new ones added, making it a living study rather than a fixed protocol.