Revolutionary therapeutic treatment for stopping progression of Parkinson's disease

Projektinformationen

ReTreatPD

ID Finanzhilfevereinbarung: 190132320

DOI

10.3030/190132320

Projekt abgeschlossen

EK-Unterschriftsdatum 7 April 2023

Startdatum 1 Mai 2023

Enddatum 30 April 2025

Finanziert unter

• The European Innovation Council (EIC)

Gesamtkosten

€ 4 083 500,00

EU-Beitrag

€ 2 500 000,00

Koordiniert durch

HERANTIS PHARMA OYJ
Finland

Periodic Reporting for period 2 - ReTreatPD (Revolutionary therapeutic treatment for stopping progression of Parkinson's disease)

Berichtszeitraum: 2024-05-01 bis 2025-04-30

Zusammenfassung vom Kontext und den Gesamtzielen des Projekts

Parkinson's disease (PD) is a neurodegenerative disorder affecting over 10 million worldwide, and 1.2 million in the EU. This chronic disabling disease affects the central nervous system with loss of dopamine neurons in the brain causing motor symptoms such as tremor and balance disturbances, as well as non-motor features such as dementia. The current dopamine substitution treatments alleviate some of the symptoms, however, there are no disease-modifying therapies. In EU the annual cost related to PD is 14 B€ currently and it is expected to increase with an ageing population.

Herantis has developed HER-096 for stopping the progression of PD. HER-096 is a peptidomimetic which reduces cellular stress, prevents formation of pathological protein aggregates, and alleviates neuroinflammation thereby modifying disease progression. HER-096 combines this compelling mechanism of action with the ability to penetrate to the brain after patient friendly subcutaneous injection. Thus, HER-096 is on its way to become a first-in-class disease-modifying drug to slow or even stop the progression of PD.

The ReTreatPD project aims to advance the development of HER-096 to achieve clinical proof of concept for HER-096 in PD, which would enable out-licensing HER-096 to a global industry partner for commercialization. The specific aim of the Grant project is to advance HER-096 project towards Phase 2 clinical trials related to biomarker selection, conduct of preclinical safety data, improve the product formulation and advance partnering discussions. If the project is successful, HER-096 can revolutionize the treatment of Parkinson’s disease, thus, alleviating the huge burden it now causes patients, their caregivers and healthcare.

More about Herantis Pharma plc: www.herantis.com

Arbeit, die ab Beginn des Projekts bis zum Ende des durch den Bericht erfassten Berichtszeitraums geleistet wurde, und die wichtigsten bis dahin erzielten Ergebnisse

We have identified novel target engagement biomarker candidates that will be tested in the ongoing phase 1b. Potential disease progression biomarkers related to modulation of disease relevant pathologies such as alpha-synuclein aggregation and preservation of dopaminergic neurons and dopamine levels have been further validated preclinically. These can be potentially used as clinical biomarker readouts in the coming clinical trials.

We have manufactured HER-096 for chronic tox preclinical studies. The preclinical tox studies are currently ongoing with in life phase ending in Aug 2025.

The formulation development aims at increasing the concentration of HER-096 in the subcutaneous injection to lower the injection volume and to increase the pH of the solution to mitigate the potential risk of injection site reaction. We have produced proof-of-concept demonstrating that the injection volume can be decreased to 1/3 of the original volume and at the same time increase the pH.

We have also very successfully advanced the partnering discussions with pharmaceutical companies. We have currently around 50 companies waiting for the data of the ongoing Phase 1b clinical trial.

The Phase 1b trial where HER-096 is for the first time administered into patients with Parkinson's disease is financed outside of the ReTreatPD project by The Michael J Fox foundation and Parkinson's UK. During the ReTreatPD we have also completed the Phase 1a trial demonstrating favourable safety profile and efficient brain penetration of subcutaneously administered HER-096.

Fortschritte, die über den aktuellen Stand der Technik hinausgehen und voraussichtliche potenzielle Auswirkungen (einschließlich der bis dato erzielten sozioökonomischen Auswirkungen und weiter gefassten gesellschaftlichen Auswirkungen des Projekts)

Novel target engagement biomarkers for HER-096 and its unique mode of action will greatly support dose finding and biological validation of the drug in upcoming clinical trials, candidate biomarkers will be tested already in the ongoing phase 1b clinical trial.

During the ReTreatPD project and partly based on the results achieved in the project, Herantis has collected the financing and conducted Phase 1b trial where HER-096 is for the first time administered into patients with Parkinson's disease - the study will have toppling data readout in September 2025 and final reporting by end of 2025. During the ReTreatPD we have also completed the Phase 1a trial demonstrating favourable safety profile and efficient brain penetration of subcutaneously administered HER-096.

During the ReTreatPD project Herantis has raised EUR 9.7 million equity financing and 3.6 million of R&D financing from The Michael J Fox Foundation and Parkinson's UK.

HER-096 has a multimodal mechanism of action addressin the kay pathologies of Parkinson's disease

Summary of HER-096 development in Parkinson's disease