Epilepsy project

Panion’s current parent company CombiGene AB is developing a novel gene therapy approach for the treatment of epilepsy and was founded on the basis of discoveries made at Lund University in Sweden and the University of Copenhagen in Denmark.

The treatment consists of an intracranial injection of an AAV vector designed to induce the overexpression of neuropeptide Y (NPY) and the Y2 receptor for NPY. This program is currently in preclinical development, and a Phase I safety study is planned for 2018.
As it turns out, epilepsy is the most common neurological condition also in dogs. Just as for human epilepsy there can be many different causes behind dog epilepsy, but the diagnostic criteria are similar to those for humans and the first line treatment are oral drugs which are also used for human epilepsy.

However, due to differences in absorption, distribution, metabolism and excretion (ADME), the antiepileptic drugs used for humans are usually not feasible for dogs. Unfortunately, for many of these canine epileptic patients, the only alternative if medication does not work is euthanasia, a process which can be emotionally trying for pet owners and their families.
The prevalence of canine epilepsy varies widely by breed, but is sometimes reported to be in the range between 1-5% of all dogs.

In humans about 30% of epilepsy patients are drug resistant, meaning that despite testing a number of drugs, the efficacy is unsatisfactory or totally absent. For canine epilepsy, too, it is estimated a drug resistance of about 30%.
Annual drug costs for treating canine epilepsy vary widely, depending on the complexity of the case. Simple drug regimens and monitoring may run up to $500 per year. But, as resistance is encountered, more complex drug regimens and monitoring can increase that annual drug cost to $5 000 or more.

The US is home to 78 million dogs an assuming a 1% epilepsy prevalence and a 30% drug resistant population the US addressable population is ~234 000 dogs. Pushing the assumptions further to ~2% prevalence, and the addressable population could approach 500,000 dogs in the US alone.

Some reports suggest the prevalence of canine epilepsy could be as high as 780 000 in the US (Anonymous, Veterinary Practice News 2015).

Regarding the price of advanced pet treatments, there are abundant examples of high priced treatments and products for companion animals:
• Treatment of canine lymphomas including diagnosis, chemotherapy, and follow-up consultations at costs varying from a few thousand up to $10 000 for a single treatment
• Diabetic dogs treated life-long with insulin replacement and subjected to special diet and exercise programs
• Bone marrow transplant (cancer), cat & dog ($15 000)
• Dogs getting pacemaker ($3 000) and cats suffering having kidney transplant ($8000).
Accordingly, Panion believes that the development and commercialization of the animal health application of CombiGene’s gene therapy for drug resistant epilepsy represents a viable business opportunity.