Washington Editor

Neurogenetics Inc., a young privately held company, licensed exclusive worldwide rights to a first-in-class molecule owned by Eli Lilly and Co. that has demonstrated efficacy in several Phase II trials across pain models.

The compound is an AMPA/Kainate receptor antagonist that has been studied in patients with migraines, chronic lower back pain and pain associated with spinal cord trauma. Also, in animal models it showed efficacy in analgesia, migraine headache, anesthesia, epilepsy and neuroprotection.

Neil Kurtz, president and CEO of La Jolla, Calif.-based Neurogenetics, told BioWorld Today he feels privileged to gain access to a compound that has undergone five Phase II trials under the auspices of Indianapolis-based Lilly. "This represents a very compelling opportunity for a company such as Neurogenetics," said Kurtz, who believes the molecule has a worldwide annual market potential of $700 million.

Neurogenetics was founded in April 2000 on the work of Rudolph Tanzi, a professor of neurology and neuroscience at Harvard University and associate geneticist at Massachusetts General Hospital, and Steven Wagner, the former director of protein biochemistry at La Jolla-based Sibia Neurosciences Inc. (acquired by Merck & Co. Inc. in 1999). Neurogenetics has 50 employees and has raised $27 million in series A and B financings. Kurtz said the company has a manageable burn rate.

Much of Neurogenetics' work has been in Alzheimer's disease and related neurodegenerative disorders. As for the Lilly molecule, Kurtz said the company intends to explore it a little further in Phase II trials to determine its potential. "We don't know right now what the limitations are, but we are extremely excited and encouraged by the fact that the animal models have been so predictive of what we would see in humans.

"Basically what the animal models predicted, as far as pain goes, is that in situations where there are chronic pain syndromes, where there's been sensitization of the central nervous system due to chronic pain conditions, whether it is a migraine headache, chronic back pain or chronic neuropathic pain, under those conditions this molecule appears to be particularly effective and it works through a novel mechanism that, in my opinion, has clear safety advantages over what's currently in the clinic."

Kurtz would not discuss the financial arrangement with Lilly. Instead, he referred to a prepared statement that said Lilly will receive an up-front fee, success-based milestone payments and royalties on sales. Neurogenetics will assume responsibility for all clinical trials, regulatory approvals and marketing.

A Lilly spokeswoman said the company would not comment on the agreement.

The deal gives Neurogenetics the right to sublicense the compound, a move being considered for markets outside North America. "My guess is that there are going to be quite a few companies interested in this because it is first in class and it has proof of concept," Kurtz said.

Elsewhere in the pipeline, Neurogenetics expects to file an investigational new drug application for an Alzheimer's compound within 12 to 18 months.

The company also has two corporate agreements with Eisai & Co., of Tokyo, on the discovery of genes responsible for late-onset Alzheimer's disease to establish valid targets and for the development of new therapeutic products.