Positive results from early trial of drug that could protect brain cells
HER-096, a potential new Parkinson's treatment, has shown promising phase 1 trial results, proving it’s safe, well-tolerated, and effective in reaching the brain in people with Parkinson's. This is a crucial step towards a drug that could slow, stop, or even repair Parkinson's damage.
The trial, funded by Parkinson’s UK through the Parkinson’s Virtual Biotech, in partnership with the Parkinson’s Foundation, as well as funding from the Michael J. Fox Foundation, provides a strong foundation for moving HER-096 into larger phase 2 trials. The hope is that this will take place in 2026.
What is HER-096?
HER-096 is a small molecule designed to act like the growth factor called cerebral dopamine neurotrophic factor (CDNF), naturally found in the brain and known to support brain cell survival and health. It is also thought to reduce the clumping of alpha-synuclein, a protein associated with Parkinson’s. These mechanisms suggest HER-096 could slow or even stop the progression of Parkinson’s. A company based in Finland called Herantis Pharma Plc (Herantis) is leading the development of this drug.
Key findings from the phase 1 trial
The trial involved two stages. Initially, 12 healthy participants received a single injection of HER-096 to see whether it was safe and had any unexpected side effects. This is a crucial stage as this was the first time the drug had been used in people.
This was followed by a trial involving 24 people with Parkinson’s, who received twice-weekly injections of HER-096 or a placebo injection over four weeks. The participants were all around 5 years post diagnosis and were able to continue taking their usual medication.
The results were positive. HER-096 was found to be safe and participants in the trial generally tolerated the injections well. Importantly, the drug performed as expected in the body, successfully reaching the brain at levels that have the potential to protect and maybe even repair brain cells. It’s likely that 2 injections a week will be the method trialled in future larger trials.
While movement symptoms remained stable during the 4 week trial, it’s important to note that the study was not designed to evaluate the drug’s impact on symptoms or progression of Parkinson's. This will be the focus of the next phase of research.
A more detailed analysis of the results, expected to be released by the end of 2025, will provide additional insights. For example, it will include findings from the use of a device called the Personal KinetiGraph (PKG), which all participants wore to track and measure movement.
What’s next?
The positive data from this phase 1 trial provides strong evidence for HER-096 to move into phase 2 trials. These larger and longer studies will investigate whether the drug has the potential to reduce Parkinson’s symptoms and progression.
Read our glossary of terms which explains more about phase 1 and phase 2 trials.
Dr. Lynsey Bilsland, Managing Director of the Parkinson’s Virtual Biotech, said:
“It is great to see the positive news from the initial trial of HER-096 in people with Parkinson’s. It adds another glimmer of hope that we are on the right track towards much needed treatments that can slow or stop Parkinson’s, something that we currently don’t have. It is further testament that the Parkinson’s Virtual Biotech and charity funding is an important mechanism to support early-stage trials into new drugs for people living with the condition. We look forward to seeing this progress into larger and longer studies.”
Dr. Antti Vuolanto, CEO of Herantis Pharma, added:
“We are thrilled to achieve this important milestone, successfully meeting the trial’s primary and secondary endpoints. These results are a testament to our team’s expertise and dedication, demonstrating our ability to drive the development programme forward efficiently. We sincerely thank the Parkinson’s patients participating in this trial, other contributors, and patient organisations, the Michael J. Fox Foundation and Parkinson’s UK, for their support and engagement in this study. We are now excited to advance this programme to phase 2 as we explore HER-096’s potential to become the first disease-modifying therapy for Parkinson’s.”
HER-096 is based on the promise of growth factors as a way to slow or stop Parkinson's. But what are growth factors and what trials are underway to explore their potential to stop, slow or reverse Parkinson’s?
While the HER-096 clinical trial is not yet ready to recruit participants, there are still plenty of research projects that you can take part in and make a difference today. Be the first to know about opportunities in your area by joining our free Research Support Network.
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