Results reported of the single ascending dose study in healthy volunteers
Enrollment completed of the multiple ascending dose study in healthy volunteers; results expected by end of Q1 2021
Dosing initiated in a Phase 1b trial in patients with Parkinson’s Disease; preliminary results expected by mid-year 2021
BOSTON, Feb. 10, 2021 (GLOBE NEWSWIRE) — Yumanity Therapeutics (NASDAQ: YMTX), a clinical-stage biopharmaceutical company focused on the discovery and development of innovative, disease-modifying therapies for neurodegenerative diseases, today provided several updates on its lead clinical-stage program, YTX-7739, in development for the treatment of Parkinson’s Disease.
“Recent advances in our understanding of the pathological processes underlying neurodegenerative diseases are providing the opportunities for innovative companies such as Yumanity to address this challenge with therapies that attack known disease pathways,” said Robert Scannevin, Ph.D., Head of Discovery at Yumanity Therapeutics. “Multiple lines of evidence indicate that misfolded alpha-synuclein is a strong risk factor for Parkinson’s disease. However, rather than targeting alpha-synuclein directly, we discovered that inhibiting the enzyme stearoyl-CoA desaturase (SCD) could overcome alpha-synuclein toxicity, suggesting its potential as a therapeutic target.”
Brigitte Robertson, M.D., Chief Medical Officer at Yumanity Therapeutics, added, “YTX-7739 is a novel small molecule SCD inhibitor, which can be administered orally and was shown to be brain penetrant in preclinical models. We have completed the single ascending dose (SAD) study in healthy volunteers, which provides early evidence of the drug candidate’s safety and tolerability profile. We look forward to reporting on the data from the ongoing Multiple Ascending dose (MAD) study in healthy volunteers and the preliminary results of our first clinical trial in patients with Parkinson’s disease, later this year. We wish to also thank our investigators, their staff, study volunteers and patients for their dedication in continuing this important research through the challenges of the COVID-19 pandemic.”
Results from the single ascending dose study in healthy volunteers: This was a Phase 1, single-ascending dose study of YTX-7739, a novel SCD inhibitor being developed for the treatment of Parkinson’s disease. Fifty-six healthy volunteers (aged 19-39 years of age; 22 males; 34 females) were administered single oral doses of YTX-7739, from 5 mg to 400 mg. Forty subjects participated in the placebo controlled, randomized, double blind part of the study which included 7 cohorts of 8 subjects each, randomized to treatment or placebo in a 6:2 ratio. Sixteen of these subjects also participated in 2 cohorts where YTX-7739 was administered with food. In addition, 2 cohorts of 8 subjects each (16 subjects) were conducted in an open label fashion to further inform dose selection for the MAD study. There were no safety concerns identified and YTX-7739 was found to be well tolerated with most adverse events being mild or moderate in severity. The half-life of YTX-7739 combined with a favorable dose-proportional pharmacokinetic (PK) profile, in the fed state, supports that low daily doses administered with food will sustain the target range of exposure. Drug plasma concentrations in the study exceeded levels of exposure estimated to be sufficient for target engagement based on pharmacodynamic modeling. Consistent with preclinical data, YTX-7739 also demonstrated clinically relevant drug concentrations in the cerebral spinal fluid (CSF). The results of this SAD study supported progression to the multiple ascending dose study.
Completion of enrollment in the multiple ascending dose study (MAD) in healthy volunteers: This is a Phase 1, placebo-controlled, randomized, double-blind study, investigating the safety, tolerability, and pharmacokinetics of once daily oral administration of 2 doses of YTX-7739 (15 mg and 25 mg) for 14 to 28 days in 16 healthy male and female volunteers. The study includes 2 cohorts of 8 subjects each, randomized to treatment or placebo in a 6:2 ratio. The study is also exploring plasma and CSF biomarker measures of pharmacodynamic activity. The company expects to report the results of this MAD study by the end of the current quarter. Detailed clinical data from these Phase 1 studies in healthy volunteers with YTX-7739 will be presented at a future medical conference.
Dosing initiated in a Phase 1b study in Parkinson’s disease patients: The company also announced the start of a placebo controlled, randomized double blind, MAD Phase 1b study of YTX-7739 in patients with Parkinson’s disease. This study is expected to enroll 30 subjects and will collect safety, tolerability, pharmacokinetic and pharmacodynamic parameters including potential biomarkers of SCD activity and target engagement in the CSF, plasma, and other fluids or tissues. Preliminary results are expected by mid-year 2021.
YTX-7739 is Yumanity Therapeutics’ proprietary lead small molecule investigational therapy designed to penetrate the blood-brain barrier and inhibit the activity of a novel target, stearoyl-CoA desaturase (SCD), that plays an important and previously unrecognized role in modulating neurotoxicity arising from the alpha-synuclein protein, a major driver of Parkinson’s disease and related neurodegenerative disorders. Misfolding and aggregation of alpha-synuclein triggers a cascade of events, ultimately resulting in neurotoxicity and the subsequent dysregulation of movement and cognition that afflicts patients living with these diseases. Through inhibition of SCD, YTX-7739 modulates an upstream process in the alpha-synuclein pathological cascade and has been shown to rescue or prevent toxicity in preclinical models. The company is assessing the potential utility of YTX-7739 in Parkinson’s disease.
SCD is an enzyme that catalyzes fatty acid desaturation, the products of which are incorporated into phospholipids, triglycerides, or cholesterol esters. These lipid-related molecules regulate multiple diverse cellular properties and processes, including membrane structure and function, vesicle trafficking, intracellular signaling and inflammation. SCD expression is regulated by a transcription factor known as SREBF1, which has been identified in human genome-wide association studies as a risk factor for Parkinson’s disease. In preclinical models, SCD inhibition appears to normalize the dynamic interaction of pathological alpha-synuclein with membranes, which improves neuronal function and reduces toxicity, leading to enhanced neuronal survival. Alpha-synuclein-dependent disruption of membrane-related biological pathways, such as vesicle trafficking, is closely linked to the formation of Lewy body protein/membrane aggregations, a hallmark pathological feature of Parkinson’s disease.
About Yumanity Therapeutics
Yumanity Therapeutics is a clinical-stage biopharmaceutical company dedicated to accelerating the revolution in the treatment of neurodegenerative diseases through its scientific foundation and drug discovery platform. The Company’s most advanced product candidate, YTX-7739, is currently in Phase 1 clinical development for Parkinson’s disease. Yumanity’s drug discovery platform is designed to enable the Company to rapidly screen for potential disease-modifying therapies by overcoming toxicity of misfolded proteins in neurogenerative diseases. Yumanity’s pipeline consists of additional programs focused on Lewy body dementia, multi- system atrophy, amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease), frontotemporal lobar dementia (FTLD), and Alzheimer’s disease. For more information, please visit www.yumanity.com.
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