Herantis Pharma (HRNTS) Company presentation summary

Clinical development highlights

• Lead asset HER-096 is a synthetic peptide designed to mimic the active site of CDNF, targeting neurorestorative therapy for Parkinson's disease.

• Phase 1 studies in healthy volunteers and Parkinson's patients showed strong safety, tolerability, and efficient brain penetration.

• Biomarker data confirmed biological responses consistent with the drug's mechanism, supporting preclinical-to-clinical translatability.

• No dose-limiting toxicities or unexpected adverse events were observed; all participants completed the studies.

• Phase 2 proof-of-concept trial is planned, focusing on symptomatic improvement and disease modification in early-stage patients.

Mechanism of action and scientific rationale

• HER-096 modulates the Unfolded Protein Response (UPR) pathway via interaction with GRP78, aiming to restore proteostasis and reduce neurodegeneration.

• Demonstrates blood-brain barrier penetration and is administered subcutaneously 1–3 times per week.

• Preclinical studies show HER-096 supports mitochondrial health and function, addressing key Parkinson's disease pathology.

• Biomarker analyses reveal broad modulation of proteostasis, inflammation, and mitochondrial function, aligning with the proposed mechanism.

• Sustained biological effects are observed despite short plasma half-life, supporting intermittent dosing.

Market opportunity and strategic positioning

• Parkinson's disease affects 10 million people globally, with the pharmaceutical market projected to reach $11B by 2029 and $13B by 2034.

• No disease-modifying therapies are currently available, positioning HER-096 as a potentially first-in-class treatment.

• HER-096's differentiated mechanism and broad functionality offer potential in other CNS disorders.

• The program is de-risked with human data, clear patient population, and defined dosing, making it attractive for partnering.

• Intellectual property protection is established, with patents granted or pending in key markets.