Date: 2014-09-23
Type of information: Grant
Company: nLife Therapeutics (Spain)
Investors: The Michael J. Fox Foundation - MJFF (USA)
Amount: $350,000 (€ 271,000)
Funding type: grant
Planned used: nLife Therapeutics designed the NLF-PD-1233 molecule to target specific neurons in the brain to reduce expression of the alpha-synuclein protein, a major constituent of Lewy bodies (protein clumps that are the pathological hallmark of Parkinson’s disease). The funding will help characterize the mechanism of action of nLife’s lead compound NLF-PD-1233 in a Parkinson disease model and find a proper dose to engage clinical development. It will be used to characterize the pharmacokinetic and pharmacodynamic profiles of NLF-PD-1233 in an alpha-synuclein overexpression model of Parkinson’s disease (PD) vs. a healthy model. The primary endpoint of the study is to find a suitable treatment dose for intranasal and direct brain administration. nLife will compare the drug concentrations achieved when given through the nose to those when given directly to the brain or intravenously. The results are due in November 2015. The secondary endpoint will be to characterize the efficacy of NLF-PD-1233, analyzing the knockdown of alpha-synuclein mRNA expression and alpha-synuclein protein changes. Then, the company intends to characterize the concentration-response relationship for NLF-PD-1233 and alpha-synuclein mRNA knockdown. The resulting data will be used to model the projected dose-effect relationships for larger animal models and for humans. Clinical trials could start in December 2015.
Others: * On September 23, 2014, nLife Therapeutics, an early-stage biopharmaceutical company leading developments in nOligos (neuronal specific oligonucleotides) as therapeutic agents for CNS disorders, announced that The Michael J. Fox Foundation for Parkinson’s Research (MJFF) has awarded the company a grant of $350,000 (€ 271,000). nLife will conduct a research study entitled: “Pharmacokinetic and Pharmacodynamic characterization of a novel therapy to silence selectively alpha-synuclein in monoaminergic neurons of rat.” This is only the second grant from the MJFF to a Spanish company (as opposed to academia). Since its inception in 2000, the MJFF has awarded more than 1,750 grants worldwide, with 24 of them involving investigators from Spanish organizations.
Therapeutic area: Neurodegenerative diseases
