BioWorld Today Contributing Writer

Asklepios Biopharmaceutical Inc. received $200,000 in grant funding from the Epilepsy Therapy Project (ETP) and the Epilepsy Foundation to advance its preclinical gene therapy for medically refractory epilepsy. The treatment could eventually offer an alternative to surgical brain resection, and the company expects the funding to support the program through an investigational new drug application filing.

With the new grant, the Middleburg, Va.-based company continues its strategy of pursuing nondilutive funding from disease foundations, which it used to fund its clinical program in Duchenne's muscular dystrophy.

"We very much like the fact that they have shown efficacy, low toxicity and good tolerability for the galanin molecule gene transfer, and have experience with the adenovirus vector that makes this all possible," ETP President Joyce Kramer told BioWorld Today.

The grant disbursal is contingent upon Asklepios receiving a phase II Small Business Innovation Research grant to complete the funding required for preclinical research. It has applied to the NIH via the National Institute of Neurologic Disorders and Stroke for that grant.

The company received a phase I SBIR grant in 2008 and successfully completed its milestones, according to Scott W. J. McPhee, Asklepios director of research and development.

The gene therapy is being developed for patients with mesial temporal lobe epilepsy (MTLE). Out of 2.5 million people with epilepsy in the U.S., between 250,000 and 375,000 have MTLE that is resistant to the currently available drug therapies. For those people, surgical resection of the brain tissue that is causing the seizures is the only alternative treatment.

Asklepios has completed proof-of-concept testing, and its next steps will be to model delivery in patients in preparation for clinical trials. In order to do this, the company has assembled a team of neurosurgeons, neurologists and epileptologists to work together to develop the best plan for delivering the therapy to the brain.

All of the patients in the trial will have failed three anticonvulsants and will be candidates for resective surgery.

"Because these patients have already been mapped for surgery, we'll have a clear idea of where the epileptic tissue is and where it's positioned . . . so we'll be targeting those sites," McPhee explained.

The goal is to insert a gene that will produce galanin at the tissue site that would normally be resected during surgery. Although it would be an early Phase I trial, if the patients show a therapeutic response, it could spare them the brain surgery. And, according to McPhee, because the therapy is a one-time treatment with a permanent effect, there is an additional opportunity to collect long-term efficacy data.

Some patients with MTLE are not candidates for surgery because the tissue causing the seizures is involved in language functions of the brain. McPhee hopes that if the gene therapy treatment is successful it can provide an alternative for those patients, as well.

The galanin gene will be delivered by Asklepios' gene delivery platform, a recombinant adeno-associated virus (rAAV)-based system called Biological Nano Particles (BNP). The BNPs are made out of pieces of different adeno-associated viruses and parvoviruses, engineered to target transducing muscle tissue and to evade the immune system.

Back in 2004, Asklepios was the first commercial biotech company to receive major grant funding from a nonprofit disease foundation, the Muscular Dystrophy Association. Since then, disease foundation funding has become more common as an alternative to angel or VC financing.

"We've kept going while some of the biotech zombies have gone under," McPhee said. "We keep a nondilutive approach. With the longer time frames involved in moving these technologies forward, it's less well suited to the VC, early exit strategies."

To date, Asklepios has had just one equity investor, a parent of a child with Duchenne's muscular dystrophy, who contributed $1 .5 million in series A financing in 2006 to boost the company's DMD therapy, biostrophin, now in clinical trials.

Asklepios has supplemented its portfolio of grants with licensing deals with partners including Merck & Co., Medtronic Inc., GlaxoSmithKline plc, Bayer Healthcare AG, and a number of smaller deals for its BNP technology. (See BioWorld Today, Sept. 29, 2006.)