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 placebo and two HER-096 dose groups, receiving treatment twice weekly for four weeks.

• 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 through exploratory assessments.

• Biomarker data are intended to de-risk and inform Phase 2 efficacy trials.

Key findings and data interpretation

• 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.

• Targeted assays showed improved glutathione redox balance and reduced mitochondrial DNA lesions, indicating enhanced oxidative stress defense and mitochondrial benefit.

• Concordant changes were observed in cerebrospinal fluid and nervous system-derived extracellular vesicles, supporting central and systemic effects.

• Consistent, multi-layered data analysis increased confidence in the observed biological shifts and supports the disease-modifying potential of HER-096.

Implications for clinical development

• Biomarker data provide proof of biology, bridging preclinical and clinical findings.

• Results support dose selection (notably 300 mg) and inform exposure-response relationships and clinical endpoints for Phase 2.

• Findings are expected to facilitate partnering and investor discussions for Phase 2 resourcing.

• Mitochondrial markers and proteostasis changes are particularly relevant to Parkinson's pathology.

• The data are seen as a major milestone, significantly reducing risk for further clinical development.