PharmatrophiX Selected for Landmark UCSF-led platform trial in rare neurodegenerative disease; Unveils New Data at Tau Global Conference

PharmatrophiX unveiled preclinical data at the Global Tau Conference further supporting evidence that LM11A-31 is as multi-action shield for the brain’s connections

MENLO PARK, Calif. — May 14, 2026 – PharmatrophiX, a privately-held, clinical-stage biopharmaceutical company pioneering the development of disease-modifying drugs targeting neurodegenerative diseases, today announced their lead candidate, LM11A-31, will be part of the NIH funded, University of California San Francisco (UCSF)-led, Progressive Supranuclear Palsy (PSP) Platform Trial (PTP) and will show new pre-clinical data at the Global Tau Conference in Washington, DC, May 15-16.

“PharmatrophiX has built an extremely compelling scientific foundation working closely with many of the leaders in the field, its lead asset LM11A-31 has demonstrated incredible promise with early-stage data showing significant disease-modifying effects as indicated by a wide range of imaging, CSF and plasma biomarkers. The company’s program provides a differentiated approach to treating neurodegenerative diseases,” said David Setboun, Pharm.D., MBA who was appointed to Chief Executive Officer of PharmatrophiX in January. “We are excited to take another step forward with the Progressive Supranuclear Palsy Trial Platform (PTP), furthering our understanding of tau-related diseases.”

PSP is a rare, fatal brain disease that causes balance, walking, eye movement and swallowing issues. It is believed to be caused by an excess of abnormal forms of the protein tau, which are also found in other neurodegenerative diseases such as Alzheimer’s. There is currently no effective treatment or cure for the disease.

“PSP remains one of the most devastating and under-recognized neurodegenerative diseases, with people and families facing a disease without approved therapies,” said Kristophe Diaz, PhD, Chief Executive Officer of CurePSP. “The inclusion of LM11A-31 in the PSP Trial Platform reflects the growing momentum and scientific maturity emerging across the PSP field. We are proud to collaborate with the PSP Trial Platform and the broader research community, including through convenings such as the Global Tau Conference, to help accelerate the development of urgently needed therapies for PSP. Approaches that combine strong mechanistic rationale with biomarker-driven development and translational science represent exactly the kind of progress needed to move the field forward for people living with PSP and related diseases.”

The PTP aims to accelerate the development of effective treatments for PSP, by testing potential therapies, including LM11A-31, with the hopes of developing new therapies to address this unmet need.

“We are highly enthusiastic to investigate LM11A-31’s potential to slow progression of clinical symptoms and disability associated with PSP as part of the NIH-funded PSP Trial Platform,” 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. “Dr. Longo’s pioneering work on modulation of p75 and its ability to promote synaptic integrity and prevent accumulation of toxic tau species in nonclinical models provide a compelling scientific rationale for including LM11A-31 in PTP. Moreover, the data acquired from Pharmatrophix’s previous Phase 2 clinical trial in AD have already demonstrated promising LM11A-31 induced biomarker changes supporting potentially beneficial effects in other neurodegenerative tauopathies such as PSP.”

At the Tau Global Conference, Frank Longo, MD, PhD will present new pre-clinical data that demonstrates LM11A-31’s ability to target tau acetylation – a chemical modification that increases tau toxicity and spread in the brain. Therapeutic research has largely focused on tau phosphorylation, another chemical change that when excessive can also drive tau toxicity and that is also prevented by LM11A-31. These new findings show that by targeting upstream signaling through the p75 neurotrophin receptor it may offer an important additional way to slow Alzheimer’s disease and other tau-based diseases such as PSP by preventing toxic tau acetylation and protecting synapses before damage spreads.

“Tau acetylation is an emerging driver of neurodegeneration, where chemical changes to the tau protein promote its accumulation, spread, and toxic effects on brain cells,” said Dr. Longo. “These exciting findings add to a growing body of evidence that LM11A-31 has broad therapeutic potential for neurodegenerative diseases.”

Poster Presentation:
Title: Modulation of the p75 neurotrophin receptor inhibits pathological tau acetylation in P301S mice
Session: Pre-Clinical Research
Presenter: Frank Longo, MD, PhD, Co-Founder and Chairman of the Board at PharmatrophiX

PharmatrophiX’s LM11A-31 targets the p75 neurotrophin receptor, which regulates the health of a neuron, and signals the receptor to protect neurons and their connections instead of eliminating them, while reducing abnormal tau build-up and decreasing neuro-inflammation. This multi-pronged approach has shown promise in a Phase 2a trial, which was published in Nature Medicine, where LM11A-31 was well-tolerated and showed early signs of improving the underlying biology of the disease in brain imaging and biomarkers.

About PharmatrophiX

PharmatrophiX is a clinical-stage biopharmaceutical company focused on the discovery and development of disease-modifying therapies for neurodegenerative diseases. PharmatrophiX was co-founded by Frank Longo, M.D., Ph.D., and Anne Chun Longo. The Company is advancing a pipeline of small-molecule drugs targeting p75 as well as the TrkB and TrkC receptors designed to prevent fundamental neurodegenerative mechanisms and to promote synaptic resilience with an emphasis on extensive collaboration with leading academic groups, disciplined clinical development and strategic collaboration.

About LM11A-31

PharmatrophiX’s lead asset is a first-in-class small molecule (LM11A-31) targeting the p75 neurotrophin receptor. In published pre-clinical studies across multiple neurodegenerative disease models, LM11A-31 has been found to render synapses resilient to pathological forms of amyloid and tau, reduce the accumulation and spread of a broad range of pathological tau species, and reduce disease-associated microglial and astrocyte dysfunction. These activities are consistent with the known extensive overlap between the intracellular degenerative signaling networks regulated by the p75 receptor and degenerative networks upregulated in Alzheimer’s and related neurodegenerative diseases.

About the PharmatrophiX LM11A-31 Alzheimer’s Disease Program

PharmatrophiX has completed a Phase 2a safety- and biomarker-focused RCT study assessing mild-moderate Alzheimer’s disease in 242 enrolled participants, LM11A-31 demonstrated significant beneficial effects in five biomarker categories: structural MRI, FDG-PET, CSF synaptic and glial biomarkers, CSF proteomic modules with high significant weighting for synaptic proteins and plasma p-tau217. ADAS-cog13 and MMSE scores progressed significantly in the placebo group, and a 50% reduction of progression was observed in the drug-treated group. A favorable safety profile was reported with the most frequent treatment emergent adverse events for LM11A-31 of nasopharyngitis (9.3%), diarrhea (7.5%), headache (6.2%), and asymptomatic eosinophilia (6.2%) which were transient and non-serious; and no treatment-related amyloid-related imaging abnormalities reported (NCT03069014; EudraCT 2018-001071-20).

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