Herantis Pharma (HRNTS) R&D Day 2024 summary

Lead asset and development overview

• HER-096, a peptidomimetic based on CDNF, is in clinical development for Parkinson's disease and other CNS disorders, showing strong preclinical and Phase Ia safety and brain penetration data.

• HER-096 is currently in a Phase Ib study, with preparations underway for Phase II and active partnering discussions for commercialization.

• The molecule is synthetic, easy to manufacture, and has demonstrated the ability to cross the blood-brain barrier in humans.

• HER-096 aims for both symptomatic improvement and long-term disease modification, targeting a $10 billion global market opportunity.

• The company leverages a small core team and global partners, supported by an experienced advisory board.

Scientific rationale and mechanism of action

• HER-096 targets the unfolded protein response (UPR) pathway, modulating GRP78 to restore proteostasis and reduce neurodegeneration.

• Preclinical studies show HER-096 reduces alpha-synuclein aggregation, neuroinflammation, and protects dopamine neurons.

• HER-096 demonstrates a hit-and-run mechanism, with therapeutic effects outlasting its short plasma half-life.

• The UPR pathway is implicated in multiple diseases, suggesting broader therapeutic potential beyond Parkinson's.

• HER-096’s design is informed by extensive CDNF research, enabling efficient translation to clinical development.

Clinical progress and biomarker strategy

• Phase Ia study confirmed safety, tolerability, and blood-brain barrier penetration in healthy subjects.

• Phase Ib is ongoing, assessing safety and pharmacokinetics in Parkinson's patients, with biomarker collection to support future efficacy endpoints.

• Phase II preparations focus on optimal treatment duration, patient selection, and integration of clinical and biomarker endpoints.

• Biomarker strategy evolves from treatment response in early phases to disease progression and neuroimaging in Phase II.