Herantis Pharma (HRNTS) Study update summary

Phase II study plans and design

• HER-096 will advance to a phase II-A proof-of-concept efficacy trial in about 100 de novo, early-stage Parkinson's patients in Europe, using a randomized, double-blind, placebo-controlled design for six months, followed by a six-month open-label extension.

• The primary endpoint is a sensitive digital motor score, with secondary endpoints including standard clinical rating scales (UPDRS), multimodal brain imaging, and selected fluid biomarkers.

• The trial will use twice-weekly subcutaneous injections, with a 1:1 randomization ratio and an overall participant duration of just over one year.

• Confirmed clinical sites are in Finland, Sweden, Luxembourg, and the Netherlands, with potential for expansion.

• Clinical trial application submission is expected by year-end, with first patient enrollment in the first half of next year and efficacy data readout anticipated in early 2029.

Rationale and regulatory feedback

• HER-096 targets core drivers of Parkinson's pathology, aiming for disease modification based on a unique mechanism derived from CDNF.

• Preclinical and phase I data show strong safety, blood-brain barrier penetration, and biomarker evidence supporting the mechanism of action.

• FDA found the planned Phase 2a study design appropriate, raised no concerns about CMC or preclinical data, and encouraged digital endpoints, noting further validation is needed for regulatory acceptance.

• The study design balances objectives, risks, and patient burden, with digital endpoints chosen for their sensitivity and objectivity over traditional clinical scales.

• The digital motor score is expected to provide a higher signal-to-noise ratio, potentially enabling smaller and shorter trials without loss of statistical power.

Digital health technology and partnership

• Collaboration with Indivi brings a smartphone-based digital motor score as the primary endpoint, enabling high-frequency, objective, real-world assessment of motor symptoms.

• Indivi's technology uses a multimodal deep phenotyping framework with 18 instruments to capture motor and cognitive functions, focusing on gait, balance, and upper limb bradykinesia.

• Digital endpoints are gaining traction in Parkinson's trials, with prior examples in Roche, NEULARK, and LUMA studies, but regulatory acceptance is still evolving.

• The partnership is a joint development effort, integrating drug and digital biomarker science from study design through regulatory engagement.

• Integration of a digital biomarker platform aims to provide objective, continuous disease progression data.