Targeting the brain sigma-1 receptor: A paradigm shift for the treatment of neuro-degenerative disorders

Informazioni relative al progetto

NeuroPa

ID dell’accordo di sovvenzione: 886169

DOI

10.3030/886169

Progetto chiuso

Data della firma CE 20 Novembre 2019

Data di avvio 1 Dicembre 2019

Data di completamento 31 Maggio 2020

Finanziato da

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Costo totale

€ 71 429,00

Contributo UE

€ 50 000,00

Coordinato da

SIGMATHERA SAS
France

Periodic Reporting for period 1 - NeuroPa (Targeting the brain sigma-1 receptor: A paradigm shift for the treatment of neuro-degenerative disorders)

Periodo di rendicontazione: 2019-12-01 al 2020-05-31

Sintesi del contesto e degli obiettivi generali del progetto

We are developing igmesine, a proprietary drug that holds the potential to provide treatment for almost all neurodegenerative diseases (NDD's: ALS, AD & PD are currently prioritized due to favorable development timelines), by successfully targeting multiple critical molecular processes involved in neurodegeneration. Igmesine activates, with high potency and specificity, recently characterized Sigma-1 receptors (Sigma-1R). The receptors are highly expressed in neurons and supporting cells (glia) of the central nervous system. Activation of Sigma-1R prevents cell-death, calcium excitotoxicity, accumulation of toxic misfolded proteins, limits oxidative stress and neuroinflammation. Intervention in these processes represents a novel, par excellence approach to slow down the progression of multiple neurogenerative disorders. SigmaThera designed the NeuroPa project to get a clear understanding of the commercial potential of igmesine, to strengthen their clinical development strategy and to define their optimal business strategy. The following report contains the market analysis, clinical & regulatory feasibility, and commercial feasibility.

Lavoro eseguito dall’inizio del progetto fino alla fine del periodo coperto dalla relazione e principali risultati finora ottenuti

The objectives of the Neuropa project were intended to maximize the chance of igmesine, and with it SigmaThera, becoming a success. This included a market analysis to assess the attractiveness and the dynamics of the target market of neurodegenerative diseases and define the most optimal commercialization strategy based on clinical development pathways and IP strategies.

There are significant pipeline competitors for igmesine in all target markets within the neurodegenerative market. This is driven by the large unmet needs caused by the lack of curative treatments. Although ALS currently holds the smallest market in terms of size and number of diagnosed patients, the commercial feasibility remains the highest due to the favourable clinical development pathway. Because of the rarity of the disease and high unmet need orphan drug designation status is expected to be granted. This would drastically shorten the clinical development timelines. In addition, due to the rapid disease progression, outcome measurements will be favourable for ALS compared to other NDD’s further adding to the commercial feasibility for igmesine.

Progressi oltre lo stato dell’arte e potenziale impatto previsto (incluso l’impatto socioeconomico e le implicazioni sociali più ampie del progetto fino ad ora)

Initially we will clinically validate Igmesine for the use against ALS. Although ALS currently holds the smallest market in terms of size and number of diagnosed patients, the commercial feasibility remains the highest due to the favourable clinical development pathway. Because of the rarity of the disease and high unmet need Orphan Drug Designation status is expected to be granted. This would drastically shorten the clinical development timelines. In addition, due to the rapid disease progression, outcome measurements will be favourable for ALS compared to other NDD’s further adding to the commercial feasibility for Igmesine. Upon successfully receiving market authorization for ALS, we will seek out a licensing deal with a large pharmaceutical industry partner to commercialize Igmesine. The generated funds from this deal will be invested in further development of our therapeutic pipeline by initiating clinical validation against AD followed by other neurodegenerative diseases alike. This strategy will ensure that the maximum number of patients with varying neurodegenerative diseases will ultimately benefit from igmesine.

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