CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Pinteon Therapeutics, a biotechnology company focused on protecting neuronal health by targeting neurotoxic forms of tau, today announced positive results from its Phase 1 study of PNT001, a novel tau antibody that uniquely targets a toxic epitope known to drive neurodegenerative disease. The results showed PNT001 was well-tolerated in healthy volunteers at doses that provide a potentially therapeutic cerebrospinal fluid (CSF) drug concentration. These doses will be evaluated in subsequent multiple ascending dose studies in patients with traumatic brain injury and other tauopathies.

“We are pleased by the positive results from our first Phase 1 study, which showed that PNT001 was well-tolerated at doses that may potentially provide a therapeutic effect,” said Martin Jefson, Ph.D., Pinteon’s chief executive officer. “We plan to initiate a multiple ascending dose study of PNT001 as we continue to advance our clinical program and work to develop a treatment that can stop the spread of pathologic tau and preserve brain function in patients living with tauopathies.”

PNT001 is a monoclonal antibody targeting an epitope on a highly neurotoxic conformation of the tau protein called cis-pT231. This conformation of tau is both acutely neurotoxic, but also contributes to the initiation and spread of tau pathology in neurodegenerative tauopathies.

The Phase 1 study was designed to evaluate the safety, tolerability, pharmacokinetics and immunogenicity of intravenously administered PNT001 in healthy adult volunteers. The study included 48 participants across six dose cohorts (33, 100, 300, 900, 2,700 or 4,000 mg). Participants were randomized to receive a single dose of PNT001 or placebo. At 16 weeks, three non-serious adverse Grade 1 events were determined to be related to study drug or placebo, and all resolved without sequelae. No maximum tolerated dose was identified, and there were no premature discontinuations, dose reductions or dose interruptions due to adverse events. Well-tolerated doses showed CSF concentrations of PNT001 that suggest a potentially therapeutic effect. More information about the study can be found on ClinicalTrials.gov (NCT04096287).

About Pinteon Therapeutics

Pinteon Therapeutics is advancing clinical studies of a novel antibody that aims to interrupt the spread of toxic tau and protect and preserve brain function in patients with neurodegenerative disease. Pinteon’s lead asset, PNT001, is the only antibody in development that targets cis-pT231 tau, a neurotoxic epitope that has been identified in multiple preclinical studies as a potent driver of neurodegenerative disease. Pinteon is led by an experienced management team with strong track records in CNS drug discovery and development and is funded by Morningside Ventures. For more information, visit pinteon.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210628005083/en/