Parkinson’s therapy is tolerated well, clearing path for Phase 2 trial
HER-096 is a derivative of CDNF, which promotes nerve cell survival, recovery
An experimental therapy being developed by Herantis Pharma for Parkinson’s disease was well tolerated and able to penetrate the blood-brain barrier (BBB), a semipermeable membrane that prevents certain substances, including medications, from entering the brain.
That’s according to top-line data from a Phase 1b trial (NCT06659562), which is testing the safety and pharmacological properties of HER-096 in healthy volunteers and people with mild to moderate Parkinson’s disease.
The full trial data, including biomarkers, are expected to become available before the end of the year. The safety and pharmacological trial data support a Phase 2 trial to assess HER-096’s efficacy in people with Parkinson’s, according to the company.
“We are thrilled to achieve this important milestone, successfully meeting the trial’s primary and secondary endpoints,” Antti Vuolanto, CEO of Herantis Pharma, said in a company press release. “We are now excited to advance this program to Phase 2 as we explore HER-096’s potential to become the first disease-modifying therapy for Parkinson’s disease.”
What is HER-096?
Administered by an injection under the skin, or subcutaneously, HER-096 is a lab-made derivative of cerebral dopamine neurotrophic factor (CDNF) that mimics the activity of this naturally occurring protein, which promotes nerve cell survival and recovery.
CDNF is present in the blood and cerebrospinal fluid (CSF), the liquid that bathes the brain and spinal cord. A previous Phase 1/2 trial (NCT03295786) showed delivering CDNF using a brain-implanted delivery device was safe and well tolerated in people with advanced Parkinson’s. It also reduced slowness of movement and disease severity, according to the company. HER-096 is designed to enter the brain after subcutaneous injection.
Previous data from the first part of the Phase 1b trial, which enrolled 12 healthy volunteers who received a single 300 mg HER-096 injection, showed the treatment levels in the CSF exceeded the minimum target range of 50-100 nanogram per milliliter (ng/mL) that was set based on preclinical data. It also confirmed a dose interval of two or three administrations per week.
In the second part, 24 people with Parkinson’s were randomly assigned to either 200 or 300 mg of treatment, or a placebo, twice weekly for four weeks. The results demonstrate HER-096 was safe and well tolerated at the tested regimens. Adverse events were generally mild to moderate in severity and mainly included injection site reactions. The results suggest that twice-weekly dosing regimen with a 300 mg dose is suitable for the future Phase 2 trial.
“The Phase 1b results combined with the previously reported clinical and preclinical data provide a good rationale for advancing HER-096 into a Phase 2 trial focused on efficacy,” Anders Gersel Pedersen, MD, chairman of Herantis scientific advisory board, said. “This is an important step forward for HER-096 as a very promising clinical-stage disease-modifying drug candidate addressing the unmet clinical need in Parkinson’s disease.”
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