nLife Therapeutics, a young biopharmaceutical company leading the development of so-called nOligos (neuron-specific oligonucleotides) as therapeutic agents for CNS (Central Nervous System) disorders, announced today that the Michael J. Fox Foundation for Parkinson's Disease Research (MJFF) has awarded the company a grant of $350,000. Fox Foundation for Parkinson's Disease Research (MJFF) has awarded the company a $350,000 grant. nLife will conduct a research study entitled "Pharmacokinetic and pharmacodynamic characterization of a novel treatment to selectively silence α-synuclein in rat monoaminergic neurons."

Following the investment made by Healthequity, Caixa Capital Risc, the venture capital manager of "la Caixa" and Invercaria, this grant represents a new source of funding for the company and will be used to characterize the pharmacokinetic (distribution of the molecule) and pharmacodynamic (effect on the target protein) profiles of NLF-PD-1233, the lead compound of nLife.

This is the second funding that the MJFF has awarded to a Spanish company (and not to an academy). Since its creation in 2000, the MJFF has awarded more than 1,750 grants worldwide and only 24 of them went to organizations with Spanish researchers.

nLife Therapeutics designed the NLF-PD-1233 molecule to target specific neurons in the brain and thereby reduce the expression of the protein α-synuclein, which is one of the constituents of Lewy bodies (the protein accumulations that are the hallmark of Parkinson's disease). The goal of the MJFF-funded project is to characterize the dose and efficacy of the molecule in a model of Parkinson's disease (PD) with overexpression of α-synuclein compared to a healthy model. Results will be known in November 2015.

The funding will allow nLife to study the processes of absorption, distribution, elimination and efficacy of the compound. The primary endpoint of the study is to find an appropriate treatment dose for intranasal and direct brain administration. Thus, nLife will compare the drug concentrations achieved when the drug is administered through the nose with those obtained when the drug is administered directly into the brain or intravenously.

The primary endpoint is to characterize the efficacy of NLF-PD-1233 by analyzing the decrease in messenger RNA and α-synuclein protein expression. Next, the company intends to characterize the concentration-response relationship between the NLF-PD-1233 compound and the attenuation of α-synuclein messenger RNA. The resulting data will be used to model dose-response relationships anticipated for larger animal models and for humans. Clinical trials could begin in December 2015.

"Based on the data obtained in animal studies, we believe that NLF-PD-1233 may be able to halt the progression of Parkinson's Disease and alleviate motor and non-motor symptoms," said Andres Montefeltro, CEO of nLife Therapeutics. "If the results hold up in humans, it would be the first disease-modifying treatment for Parkinson's disease."

About Parkinson's disease

Parkinson's disease is a progressive degeneration of the central nervous system characterized by a loss of neurons and symptoms such as tremors, rigidity, cognitive impairment and autonomic dysfunction. There is currently no cure for Parkinson's disease, but drugs are available to help control the symptoms.

The motor symptoms of Parkinson's disease are caused by the loss of dopaminergic neurons. Thus, the drugs currently available are intended to supplement the missing dopamine. The most effective drug is levodopa (which is converted into dopamine once it reaches the brain); however, this drug loses its effectiveness after a few years of use and, at high doses, causes the appearance of involuntary movements, called: dyskinesia.

Another treatment is the direct administration of dopamine agonists. Although these drugs last longer, they are less effective than levodopa. On the other hand, more recently, alternative strategies have been marketed that focus on the regulation of enzymes directly involved in dopamine metabolism, but many side effects (such as increased dyskinesia, hallucinations and headaches) have been observed.

About α-synuclein and Parkinson's disease.

α-synuclein is a protein that is present in neurons. Its function has not been fully elucidated, but researchers believe that this protein plays an important role in the regulation of synaptic vesicles.

Sobre la tecnologia nOligos de nLife

nLife disposa d’una tecnologia d’administració d’oligonucleòtids neuronals específics (nOligos). Els oligonucleòtids són polímers dissenyats per fixar-se a cadenes complementàries específiques d’ARN missatge i, d’aquesta forma, promoure la degradació, el que produeix una disminució dels nivells de proteïnes específiques que es converteixen en tòxiques quan s’acumulen. La tecnologia d’nLife permet disminuir els nivells de proteïnes tòxiques únicament a les cèl·lules afectades, el que deixa intactes les sanes. Això s’aconsegueix adjuntant un lligant químic neuronal específic a l’oligonucleòtid. D’aquesta manera, la teràpia basada en la tecnologia nOligos té el potencial de convertir-se en un tractament modificador d’una malaltia i pot aplicar-se a proteïnes diana no modulables per petites molècules químiques convencionals. Això permetria el desenvolupament del tractament per a malalties del SNC que, d’altra forma, no tindrien cura.

Sobre nLife Therapeutics

nLife Therapeutics és una empresa biofarmacèutica que lidera el desenvolupament de nOligos (oligonucleòtids neuronals específics) com agents terapèutics per als trastorns del SNC. nLife disposa d’una plataforma única d’administració de nucleòtids per tractar diferents malalties neurodegeneratives que compta amb una sòlida protecció de la propietat intel·lectual.

El principal candidat (NLD-PD-1233) és un tractament innovador modificador de la malaltia dirigit a la malaltia de Parkinson (EP). L’empresa compta amb importants dades sobre estudis amb animals que demostren el mode d’acció de la tecnologia nOligos en els trastorns com la Depressió i la malaltia de Parkinson.

L’empresa també té en curs programes dirigits a la malaltia de Huntington i la malaltia d’Alzheimer.

Fundada en 2010 i amb seu a Granada (Espanya), nLife compta amb una inversió d’11 milions de dòlars americans aportats per tres empreses espanyoles de capital de risc: Crossroad Biotech, Caixa Capital Risc, la gestora de capital risc de “la Caixa” i HealthEquity.