Disruptive Innovation to Treat Alzheimer’s and Parkinson’s diseases: new target, new patented drug, new therapeutic strategy.

Project Information

AmInnovation

Grant agreement ID: 868484

DOI

10.3030/868484

Project closed

EC signature date 24 May 2019

Start date 1 June 2019

End date 30 November 2019

Funded under

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Total cost

€ 71 429,00

EU contribution

€ 50 000,00

Coordinated by

AMYPORE
France

Periodic Reporting for period 1 - AmInnovation (Disruptive Innovation to Treat Alzheimer’s and Parkinson’s diseases: new target, new patented drug, new therapeutic strategy.)

Reporting period: 2019-06-01 to 2019-11-30

Summary of the context and overall objectives of the project

AmyP53 is a disruptive and innovative therapeutic drug candidate for the treatment of neurodegenerative diseases, Parkinson’s and Alzheimer’s. The aim of the proposal was to de-risk AmyP53 by evaluating its safety in animals. The goals of the program were to determine the Maximum Tolerated Dose (MTD) and the Dose Range-Finding Study (DRF). This study was critical to assess the feasibility of our innovation and to proceed quickly towards a clinical evaluation in humans for treatment of AD and PD. In particular we planned to assess the safety of the administration of AmyP53 via the intranasal route, which although challenging, is noninvasive, compatible with long term dosing, perfectly adapted for disabled patients and the scale-up challenges.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

1. Work performed
- The candidate therapeutic agent AmyP53 has been synthesized under strictly controlled conditions and fully characterized for chemical and biological properties.
- It has been administered at very high doses in male and female rats via the intravenous and intranasal routes.

2. Main results achieved
- Pure synthetic AmyP53 is highly soluble in water and can be formulated as a nasal spray compatible with therapeutic use.
- All animals treated with AmyP53 were healthy and did not display any sign of toxicity in regular preclinical studies, whatever the dose of AmyP53 tested.
- In particular, all AmyP53-treated animals were found similar to control untreated animals for body weight, food consumption, behaviour, blood markers, major organs function and histology including brain, liver, and kidney.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

On the basis of the fully completed AmInnovation program, we concluded that the intranasal administration of AmyP53 is safe for both male and female animals below the dose of 5 mg/kg, which corresponds to 5000x the anticipated therapeutic dose in humans; i.e. 1 µg/kg.

Therefore, all the objectives of the approved AmInnovation program were successfully achieved. In particular, the intranasal administration of AmyP53 was totally de-risked, which was a key prerequisite for developing our solution against Alzheimer’s & Parkinson’s diseases with a nasal spray treatment.

The follow-up of this study will thus to investigate the safety and pharmacokinetics of Amy53 administered through the nasal route and implement a first in-man (clinical trial Phase 1/2 with patients). By the meantime, the data obtained during the AmInnovation SME Phase 1 H2020 grant will be published in a scientific journal in 2020 and the European Commission will be acknowledged.

Therapeutic mechanism of AmyP53