New trial data show early biological signals for Parkinson’s therapy
Biomarker shifts suggest the treatment is working as designed
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- HER-096, a Parkinson's therapy, showed early biological signals in a Phase 1b trial.
- It affected biomarkers tied to proteostasis, mitochondrial function, and neuroinflammation.
- These findings support its expected mechanism and help guide Phase 2 trial planning.
Herantis Pharma has reported new Phase 1b biomarker data that support the expected mechanism of action of HER-096, its potential disease-modifying treatment for Parkinson’s disease.
The Phase 1b trial (NCT06659562) evaluated the safety of HER-096 and explored early signs of biological activity in both healthy volunteers and people with Parkinson’s disease. Results showed that HER-096 affected biomarkers linked to proteostasis — the cell’s system for making, folding, and clearing proteins — along with mitochondrial function, which supports cellular energy production, and neuroinflammation. These findings are consistent with earlier preclinical research.
The data were recently presented during a company webcast. According to the company, the findings will help guide dose selection and refine clinical endpoints for a planned Phase 2 trial designed to evaluate the treatment’s clinical efficacy.
“Biomarker evidence supporting the expected mechanism of action of HER-096 in people with Parkinson’s disease marks a key development milestone for Herantis,” Antti Vuolanto, CEO of Herantis Pharma, said in a company press release. “These data de-risk the program for potential partners and investors, support readiness for a planned Phase 2 trial to evaluate clinical efficacy and provide guidance for dose selection.”
Understanding what goes wrong in Parkinson’s disease
Parkinson’s disease develops as dopaminergic neurons — nerve cells that produce dopamine, a chemical messenger essential for movement — are gradually lost. A key feature of the disease is the buildup of toxic clumps of alpha-synuclein, a protein that disrupts normal nerve cell function and can ultimately lead to cell death.
As alpha-synuclein builds up, it is linked to inflammation in the brain and problems with proteostasis, the cell’s system for properly folding, managing, and clearing proteins. One part of this system, called the unfolded protein response (UPR), is activated when too many misfolded proteins accumulate. When the UPR remains overactive for long periods, it may add to nerve cell damage and death.
HER-096 is a lab-made version of cerebral dopamine neurotrophic factor (CDNF), a naturally occurring protein involved in supporting nerve cell health. The treatment is designed to be given as a subcutaneous, or under-the-skin, injection and to act on disease-related pathways in the brain. CDNF has been detected in blood and cerebrospinal fluid — the fluid that surrounds the brain and spinal cord — and is known to promote nerve cell survival and recovery.
In preclinical studies using mouse models of Parkinson’s disease, HER-096 was shown to protect and restore dopaminergic neurons. At the biological level, the treatment improved proteostasis and supported energy production by mitochondria, which are known to function poorly in Parkinson’s disease.
How the early trial tested the treatment
The Phase 1b trial evaluated HER-096’s safety, tolerability, and pharmacological properties in both healthy adults and people with Parkinson’s disease. In the first part of the study, which involved healthy volunteers, the treatment was found to be well-tolerated. A single 300 mg injection resulted in treatment levels in the CSF that exceeded the minimum target range, helping confirm a dosing schedule of two or three injections per week.
In the second part of the trial, people with Parkinson’s disease were randomly assigned to receive one of two doses of HER-096 — 200 or 300 mg— or a placebo, given twice weekly for four weeks. This phase of the study also examined how the treatment affected selected biomarkers and explored potential new markers of treatment response.
Results showed that treatment with HER-096 was associated with changes in biomarkers linked to proteostasis, mitochondrial function, and neuroinflammation. The therapy increased levels of proteins involved in protein synthesis, folding, and clearance, suggesting that HER-096 produces biologically meaningful effects in people with Parkinson’s disease.
“The coordinated shifts we observed across multiple disease-relevant pathways, including proteostasis, mitochondrial function and neuroinflammation, are consistent with a disease-modifying mechanism and align with our preclinical findings,” said Henri Huttunen, PhD, Herantis’ co-founder and chief scientific officer.
