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.