Herantis Pharma has announced the dosing of the first patients in the final cohort of its Phase 1b clinical trial evaluating HER-096, a novel therapeutic candidate for Parkinson's disease. The trial aims to assess the safety, tolerability, and pharmacokinetics of repeated subcutaneous doses of HER-096 in patients with Parkinson's disease.
HER-096 is being developed as a disease-modifying therapy rather than a symptomatic treatment, potentially addressing a critical unmet need for the approximately 10 million people worldwide suffering from this progressive neurological condition.
"Parkinson's disease is a life altering condition, and there is an urgent need for new treatments that can halt or slow its progression," said Antti Vuolanto, CEO of Herantis Pharma. "We are highly encouraged by the potential of HER-096. Its disease-modifying properties combined with its ability to cross the blood-brain barrier makes it a compelling candidate to become a transformative therapy for Parkinson's that addresses the underlying disease, not just the symptoms."
Novel Mechanism of Action
HER-096 represents a significant innovation in the Parkinson's treatment landscape. It is a first-in-class synthetic peptidomimetic designed to mimic the activity of cerebral dopamine neurotropic factor (CDNF) protein. The compound's ability to effectively cross the blood-brain barrier allows for convenient subcutaneous administration, potentially improving patient compliance and quality of life compared to more invasive treatment options.
The drug employs a multi-modal mechanism of action, primarily modulating the Unfolded Protein Response (UPR) pathway. This pathway plays a critical role in restoring cellular protein balance and preventing the processes that lead to cytotoxic protein aggregation and neuronal cell death in the brain—key pathological features of Parkinson's disease. Additionally, HER-096 reduces inflammation in affected brain areas, potentially addressing multiple aspects of disease pathology.
Phase 1b Trial Design and Progress
The Phase 1b clinical study consists of two parts. Part 1, which has been completed, involved eight healthy volunteers who received a single 300 mg subcutaneous dose of HER-096 to assess safety and pharmacokinetic properties. Encouraging pharmacokinetic data from this phase were announced in January 2025.
Part 2 is a randomized, double-blind, placebo-controlled portion of the trial involving patients with Parkinson's disease, divided into two cohorts:
- In the first cohort, 12 patients were dosed twice weekly over a four-week period. Eight patients received 200 mg of HER-096 and four received placebo.
- Following a positive review by the Data and Safety Monitoring Board (DSMB), the trial has now progressed to the final cohort, where patients will receive 300 mg doses of HER-096 or placebo twice weekly over a four-week period.
Beyond safety and pharmacokinetics, the trial will evaluate selected biomarkers and aims to identify novel treatment-response biomarkers in Parkinson's disease patients. Symptom progression will be monitored using both the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and a wearable recording device, providing comprehensive assessment of potential clinical benefits.
The trial is being conducted in Turku and Helsinki, Finland by Clinical Research Services Turku – CRST Oy, with funding provided by The Michael J. Fox Foundation for Parkinson's Research (MJFF) and Parkinson's UK.
Building on Previous Success
This Phase 1b trial builds upon positive results from the earlier Phase 1a trial, which demonstrated a good safety and tolerability profile for HER-096, along with effective blood-brain barrier penetration following subcutaneous administration in healthy volunteers.
"We remain focused on completing the final cohort of this Phase 1b trial and expect to report topline data in September 2025," added Vuolanto.
Addressing a Critical Unmet Need
Parkinson's disease remains a devastating neurological condition with no available cure. Current treatments primarily address symptoms rather than the underlying disease processes and are often associated with significant side effects.
If successful, HER-096 could represent a paradigm shift in Parkinson's disease treatment by potentially halting or slowing disease progression rather than merely managing symptoms. The convenient subcutaneous administration route would also offer practical advantages over more invasive therapeutic approaches.
The development of HER-096 aligns with growing industry focus on disease-modifying therapies for neurodegenerative conditions, an area of significant unmet medical need and substantial market potential.
Herantis Pharma, listed on the Nasdaq First North Growth Market Finland, continues to advance its pipeline of disease-modifying therapies for Parkinson's disease, with HER-096 as its lead candidate.
