Herantis Pharma announced today that the first subject with Parkinson's disease has been dosed in Part 2 of its Phase 1b clinical trial evaluating HER-096, a potential disease-modifying therapy. This milestone follows encouraging pharmacokinetic data from Part 1 of the study, providing critical insights for future dosing strategies.
The pharmacokinetic analysis from Part 1, which involved eight healthy subjects receiving a single 300 mg subcutaneous dose of HER-096, demonstrated that the drug concentration in cerebrospinal fluid (CSF) significantly exceeded the minimum target range of 50-100 ng/ml established in preclinical studies. Importantly, the data revealed extended CSF exposure compared to plasma, supporting the planned dosing interval of two or three subcutaneous administrations per week in future studies.
"These pharmacokinetic findings are particularly encouraging as they confirm that HER-096 is effectively crossing the blood-brain barrier and reaching therapeutically relevant concentrations in the CSF," said a representative from Herantis Pharma, though specific attribution was not provided in the announcement.
Phase 1b Trial Design and Objectives
The ongoing Phase 1b trial has now advanced to Part 2, which is designed as a randomized, double-blind, placebo-controlled study involving patients diagnosed with Parkinson's disease. The trial includes two dose cohorts (200 mg and 300 mg of HER-096), with 12 subjects in each cohort randomized in a 2:1 ratio to receive either HER-096 or placebo.
The study protocol consists of:
- A screening period
- A four-week dosing period with two subcutaneous doses administered weekly
- A four-week follow-up period
Primary objectives focus on evaluating the safety, tolerability, and pharmacokinetics of repeated subcutaneous doses of HER-096 in Parkinson's patients. Secondary aims include assessment of selected biomarkers and identification of novel treatment response biomarkers.
The trial will monitor Parkinson's symptoms using both the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and wearable recording devices, providing comprehensive data on potential clinical effects. Topline results are expected in the third quarter of 2025.
The study is being conducted at sites in Turku and Helsinki, Finland, by the contract research organization Clinical Research Services Turku (CRST Oy).
HER-096: Mechanism of Action and Therapeutic Potential
HER-096 represents an innovative approach to treating Parkinson's disease. It is an engineered peptidomimetic molecule designed to mimic the activity of Cerebral Dopamine Neurotrophic Factor (CDNF), a protein known to promote neuronal survival and functional recovery.
The drug's mechanism of action centers on modulating the Unfolded Protein Response (UPR) pathway, which plays a crucial role in maintaining cellular protein balance (proteostasis). By regulating this pathway, HER-096 aims to prevent processes that lead to cytotoxic protein aggregation and neuronal cell death in the brain—hallmarks of Parkinson's disease pathology.
Additionally, HER-096 has demonstrated anti-inflammatory properties in the affected brain regions. This multimodal mechanism of action suggests potential not only to alleviate symptoms but possibly to halt disease progression, addressing a significant unmet need in Parkinson's treatment.
Clinical Development Background
The current Phase 1b trial builds on positive results from a Phase 1a study completed in late 2023, which demonstrated a favorable safety and tolerability profile for HER-096 in healthy volunteers. Importantly, that initial study also confirmed efficient blood-brain barrier penetration following subcutaneous administration—a critical factor for central nervous system therapeutics.
Parkinson's disease affects approximately 10 million people worldwide and is characterized by progressive neurodegeneration, particularly of dopamine-producing neurons in the substantia nigra. Current treatments primarily focus on symptom management rather than addressing the underlying disease mechanisms, highlighting the significant need for disease-modifying therapies like HER-096.
Pharmaceutical Innovation and Delivery Approach
A notable aspect of HER-096 is its convenient subcutaneous administration route, which could offer significant advantages over more invasive delivery methods often required for neurological treatments. This approach combines the mechanistic benefits of CDNF with practical clinical application.
The subcutaneous delivery system allows for self-administration, potentially improving treatment adherence and reducing healthcare system burden compared to treatments requiring clinical visits for administration.
Looking Forward
As Herantis Pharma advances this clinical program, the biotech community will be watching closely to see if HER-096 can deliver on its promise as a disease-modifying therapy for Parkinson's disease. The company, listed on the Nasdaq First North Growth Market Finland, continues to focus its pipeline on innovative approaches to neurodegenerative diseases.
The Phase 1b trial is registered on ClinicalTrials.gov (NCT06659562), and additional information about participation is available on the company's dedicated research website.
