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Pinteon Therapeutics Launches Phase 1 Trial of Novel Tau Antibody that Targets a Potent Driver of Neurodegenerative Disease

Company is backed by $17 million in Series A funding from Morningside Ventures

CAMBRIDGE, Mass., Oct. 31, 2019 – Pinteon Therapeutics today announced the launch of a Phase 1 trial of PNT001, a novel tau antibody that uniquely targets a toxic epitope known to drive neurodegenerative disease.

Pinteon is funded by a $17 million Series A from Morningside Ventures and led by an experienced team of executives with strong track records in CNS drug discovery and development.

The company’s lead asset, PNT001, targets a highly neurotoxic conformation of tau, known as cis-pT231 tau, that promotes the abnormal aggregation of tau proteins in neurons. This aggregation creates neurofibrillary tangles that cause neuronal death, block communication and impair memory and learning. PNT001 aims to disrupt the spread of this toxic form of tau by precisely targeting and neutralizing pathologic tau containing the cis-pT231 epitope.

“We believe that PNT001 can intervene at a critical point in disease progression to interrupt the transmission of pathological tau through neural circuits,” said Martin Jefson, Ph.D., Pinteon’s chief executive officer. “By halting the spread of pathologic tau, we hope to protect and preserve brain function in patients living with tauopathies. It’s a critically important goal, and I’m proud to have such an experienced team focused single-mindedly on this mission.”

PNT001 is the only antibody in development that targets cis-pT231 tau, which has been identified in multiple preclinical studies as a potent driver of neurodegenerative disease. Pinteon has initiated a Phase 1 trial of PNT001 in healthy volunteers that is designed to evaluate five single escalating dose levels. The primary endpoints will be safety and tolerability; Pinteon will also assess the pharmacokinetics of PNT001.

The company plans to move next into one or more Phase 1 trials of multiple ascending doses in patients with traumatic brain injury, progressive supranuclear palsy or Alzheimer’s disease. Tau pathology is a central component in each condition.

“PNT001 targets a potential pathogenic mechanism that other monoclonal antibodies in clinical development don’t target,” said Lawrence I. Golbe, M.D., emeritus professor of neurology at Rutgers University’s Robert Wood Johnson Medical School and a member of Pinteon’s scientific advisory board. “Of course, we hope that this mechanism is relevant in mTBI, PSP and Alzheimer’s, that PNT001 adequately engages its target in humans and above all, that patients benefit. The upcoming trials are designed to answer these important questions.”

Pinteon’s management team has decades of experience in drug discovery and development for neurologic disorders:

  • Chief Executive Officer Martin Jefson, Ph.D., led CNS research at Pfizer, where his teams discovered more than 40 development candidates, including many with novel mechanisms of action. He was the founding chief scientific officer at Rodin Therapeutics and Ataxion.
  • Chief Scientific Officer Michael Ahlijanian, Ph.D., led discovery teams focused on neurologic diseases as director of genetically defined disease biology and head of clinical biomarkers at Bristol-Myers Squibb. He has also held senior management roles at Pfizer, FORMA Therapeutics and EnVivo Pharmaceuticals.
  • Chief Medical Officer Larry Altstiel, M.D., Ph.D., is a neurologist who directed neuroscience clinical research at Pfizer, where he led selection, development and oversight of numerous preclinical and clinical studies of neurodegenerative disease treatments through Phase 3. He was also group leader for neurodegenerative diseases at Eli Lilly.

Pinteon’s scientific advisory board includes leading experts in neurodegenerative diseases and CNS drug discovery. In addition to Dr. Golbe, the scientific advisors are: Adam Boxer, M.D., Ph.D., an endowed professor of memory and aging at the University of California, San Francisco; Kurt Brunden, Ph.D., director of drug discovery at the University of Pennsylvania’s Center for Neurodegenerative Disease Research; Lee Goldstein, M.D., Ph.D., an associate professor of psychiatry at the Boston University School of Medicine; and Cristina Sampaio, M.D., Ph.D., chief medical officer at the CHDI Foundation.

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 

Media Contact:

Stephanie Simon
Ten Bridge Communications

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