Potential Treatment to Prevent Toxic Protein Clumping Enters Phase 1 Trial, Yumanity Announces

Potential Treatment to Prevent Toxic Protein Clumping Enters Phase 1 Trial, Yumanity Announces

Yumanity Therapeutics announced the start of a Phase 1 clinical study in healthy volunteers that will assess the safety and tolerability of YTX-7739, a potential disease-modifying therapy for Parkinson’s disease.

Trial results are expected to be announced in the first quarter of 2020.

YTX-7739 is designed to cross the blood-brain-barrier — a semipermeable membrane that protects the brain and spinal cord from the external environment — to inhibit the activity in the brain of an enzyme called stearoyl-CoA desaturase (SCD) .

This enzyme is known to play a key role in the production of certain fat molecules, called unsaturated fatty acids, that mediate the neurotoxic effects of alpha-synuclein protein accumulation — a key constituent of Lewy bodies, the toxic protein clumps that are a Parkinson’s hallmark.

In cell and animal disease models, the investigational medicine was shown to protect neurons against alpha-synuclein-derived toxicity and improve their survival.

“We advanced YTX-7739, an orally-active SCD inhibitor, into clinical development because of recent evidence established at Yumanity Therapeutics demonstrating its promise to protect cells from a-synuclein toxicity,” said Kenneth Rhodes, PhD, the company’s chief scientific officer, said in a press release.

“We look forward to fully characterizing the potential clinical use of YTX-7739, which is clearly differentiated from currently available Parkinson’s disease therapies that only address the symptoms, not the underlying causes.”

The Phase 1 trial is expected to enroll about 40 healthy volunteers, who will be randomly assigned to increasing doses of oral YTX-7739 or dose-matching oral placebo. Collected data will assess YTX-7739’s safety and tolerability, as well as its stability and metabolization (pharmacokinetics) inside the body: essentially, how the body affects a medicine.

If results are promising, Yumanity plans to advance YTX-7739 into a Phase 1b proof-of-concept clinical trial in patients, possibly in the second half of 2020.

“This Phase 1 trial will provide important validation for the broad application of our technology to help address arguably the most important therapeutic challenges of our time,” like that of “protect[ing] cells from neurodegeneration,” said Richard Peters, MD, PhD, Yumanity’s CEO.

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Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

One comment

  1. Michael Farrell MD says:

    Looking for treatment of the root cause and not just the symptoms. Recently had to stop working as an ER physician due to Parkinson’s.

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