Herantis Pharma (HRNTS) Study Result summary

Study background and objectives

• HER-096 is a small peptide designed to halt Parkinson's progression by mimicking CDNF and protecting dopamine neurons.

• The study included 24 Parkinson's patients, with dosing over four weeks at two dose levels and placebo, administered twice weekly.

• Phase 1b focused on demonstrating biological response and target pathway engagement in humans.

• Over 2.5 million biomarker data points were analyzed to assess biological effects and support future clinical development.

• Biomarker data are intended to de-risk and inform Phase 2 efficacy trials, dose selection, and partnering discussions.

Key findings and biomarker results

• HER-096 induced broad, dose-dependent proteomic changes in plasma, especially at 300 mg.

• Biomarker shifts were observed in proteostasis, mitochondrial function, and neuroinflammation, consistent with the expected mechanism of action.

• Significant improvements were observed in mitochondrial function and oxidative stress defense, including glutathione balance and reduced mitochondrial DNA damage.

• Concordant changes were seen across plasma, cerebrospinal fluid, and extracellular vesicles, supporting multi-layered biological engagement.

• Consistent results across analyses reinforce confidence in advancing to later-stage clinical trials.

Interpretation and implications

• The biomarker profile confirms HER-096's biological activity in humans and supports its disease-modifying potential, aligning with preclinical findings.

• Findings reduce risk for Phase 2 and support dose selection, exposure-response analysis, and patient stratification.

• Multi-omics and orthogonal data layers increase confidence in the observed biological shifts.

• Mitochondrial changes are particularly relevant to Parkinson's pathophysiology.

• HER-096 was previously shown to be generally safe and well tolerated in Phase 1b.