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

Información del proyecto

AmInnovation

Identificador del acuerdo de subvención: 868484

DOI

10.3030/868484

Proyecto cerrado

Fecha de la firma de la CE 24 Mayo 2019

Fecha de inicio 1 Junio 2019

Fecha de finalización 30 Noviembre 2019

Financiado con arreglo a

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Coste total

€ 71 429,00

Aportación de la UE

€ 50 000,00

Coordinado por

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.)

Período documentado: 2019-06-01 hasta 2019-11-30

Resumen del contexto y de los objetivos generales del proyecto

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.

Trabajo realizado desde el comienzo del proyecto hasta el final del período abarcado por el informe y los principales resultados hasta la fecha

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.

Avances que van más allá del estado de la técnica e impacto potencial esperado (incluida la repercusión socioeconómica y las implicaciones sociales más amplias del proyecto hasta la fecha)

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