Neurologix posts positive Ph2 results, looks for Ph3 funding

After another round of good news for Neurologix--in the form of positive six-month Phase II Parkinson's trial results--Xconomy's Arlene Weintraub focused on the company's next big concern: funding. And as the company's co-founder Michael Kaplitt told her, "The biggest concern...is fear of the unknown" for investors.

"The gene we're putting in restores the balance between GABA and glutamate," co-founder Matthew During explains to Xconomy. "That enables the network that controls movement to restore that movement." And unlike other post-dopamine drug treatment alternatives, like deep brain stimulation, Neurologix's procedure is a one-time treatment. "The take-home message is that our therapy is effective to a degree that is sustainable," Kaplitt said.

Neurologix is penny stock, despite its innovative gene therapy for Parkinson's sufferers. Kaplitt and fellow co-founder Matthew During focused on finding the right vector to deliver the functioning genes. That factor, they say, determines the safety of the gene therapy field, which was questioned after the death of 18-year-old Jesse Gelsinger in 1999, sparking a free-fall in funding. The gene therapy field has since enjoyed a resurgence. Neurologix gained $12 million in 2006 and $20 million in 2007 from preferred stock sales, and the company has a partnership with Medtronic for the gene therapy procedure.

Before the company's Phase III plans are set in motion, Neurologix will perform a crossover study, in which control patients from the previous trial (who received a "sham surgery" without gene therapy) are given the treatment. If the crossover goes well, the company could start its one-year Phase III trial by the end of 2012.

- here's the Neurologix release
- read the Xconomy piece

Editor's Note: The story previously stated that the FDA had requested the Neurologix crossover study before a Phase III trial commenced, but this plan was developed by the company, not the FDA.

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