Epilepsy project

Panion is developing a novel gene therapy approach for the treatment of epilepsy in dogs and cats, which is founded on the basis of discoveries made at Lund University in Sweden and the University of Copenhagen in Denmark. The development for treatment of human epilepsy is performed by CombiGene AB.
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. Epilepsy is the most common neurological condition in dogs. Just as for human epilepsy there can be many different causes behind epilepsy, but the diagnostic criteria are similar to those for humans and the first line treatment are oral drugs, similar to those used for human epilepsy. However, due to differences in absorption, distribution, metabolism and excretion, the antiepileptic drugs used for humans are often not useful for dogs or they cause adverse reactions, and few drugs are authorized and marketed for dogs.
The prevalence of canine epilepsy varies widely by breed but is reported in the range between 1-5% of all dogs. As 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. Unfortunately, for many of these canine epileptic patients, euthanasia is the only alternative when medication does not work, – a process which can be emotionally trying for pet owners and their families.

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 and 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). A similar number of dogs lives in the EU and these are an equally interesting opportunity.
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 the gene therapy for drug resistant epilepsy represents a viable business opportunity.

Panion has held meetings with drug registration agencies in both the EU and the US. In the US, an application file (INAD) has been opened, Minor Use Minor Species (MUMS) status for dogs has been granted, and fee waivers given for our development product. In the EU, MUMS status for both dogs and cats has been granted and a registration as SME (Small and Medium sized company) at the European Medicines Agency gives access to reduced application fees and increased assistance from authorities.