Our therapeutic areas of focus
We are currently focused on developing new therapeutic options for epilepsy, depression, and pain—areas where recent innovation has been limited. Learn more about these conditions.
Epilepsy
Epilepsy is a neurological condition characterized by abnormal electrical activity in the brain that leads to spontaneous, recurrent, and unprovoked seizures. It is the fourth most common neurological condition and affects nearly 3 million adults in the US. The burden of disease is profound. People with epilepsy experience high rates of anxiety and cognitive impairment, have a lower quality of life, and have an increased risk of injury and premature death compared to the general population.
Most people with epilepsy experience focal seizures, which originate from one region of the brain. Others may experience generalized seizures, which affect both sides of the brain simultaneously.
Beyond seizures, up to 50% of people with epilepsy may develop depression symptoms, which may also increase seizure frequency, thereby compromising their ability to achieve seizure control goals. Research suggests there is a potential bidirectional relationship between epilepsy and depression.
Epilepsy treatment and unmet needs
Epilepsy is often managed with polytherapy—or concurrent use of multiple antiseizure medications (ASMs)—in an attempt to improve seizure control, and more than 30 ASMs are available today. However, despite treatment, about half of people with focal epilepsy still live with uncontrolled seizures. Therapeutic innovation has slowed, with most therapies relying on mechanisms discovered up to 70 years ago. With a dearth of new mechanisms, options to improve seizure control by layering distinct and complementary mechanisms are limited. Long-term seizure control also becomes harder to achieve with increasing lines of therapy.
Epilepsy treatment is further complicated by burdensome drug interactions, lengthy titration and dose-adjustment periods, and a failure to positively impact common comorbidities, like depression. Some available ASMs can also increase or cause new psychiatric adverse events. These challenges highlight the critical need for a new therapeutic approach.
What causes seizures?
In a healthy brain, voltage-gated potassium channels and sodium channels are essential for maintaining normal electrical signaling. When a neuron is sufficiently stimulated, sodium channels open, allowing sodium to enter the neuron and initiate an electrical signal. This then triggers the opening of potassium channels, which allows potassium to leave the neuron. The exit of potassium turns off the electrical signal and prepares the neuron to respond to the next stimulus. The coordination of the signal initiating and ending, and the timing of when this happens, is critical for normal nervous system function.
In people with focal or generalized seizures, the coordination of normal neuronal signaling breaks down. Neurons become overactive and send too many signals. The result is rapid, highly synchronized neuronal signaling that leads to seizures.
At Xenon, we are advancing research on the potential to target a specific group of potassium channels (Kv7), which would offer a different approach to decrease the neuronal excitability that leads to seizures.
Learn about our approach in epilepsy
Ask the Epileptologist: How do you describe epilepsy to your patients?
A conversation with Dr Toufic Fakhoury
Ask the Epileptologists: What are the Challenges of Living with Uncontrolled Seizures?
A conversation with Dr Danielle Becker and Dr Toufic Fakhoury
Learn about our approach in epilepsy
Depression
Major depressive disorder (MDD) is a leading cause of disability worldwide, affecting approximately 21 million adults in the US. A diagnosis requires at least five depressive symptoms over a two-week period, which must include either persistent depressed mood or anhedonia (a loss of pleasure). Effective treatments remain a major challenge, as one in three people experience inadequate responses to initial antidepressant therapy. Anhedonia is commonly associated with MDD, is correlated with poorer treatment outcomes, and there is a lack of effective antidepressants that specifically address it.
While the pathophysiology of MDD is only partially understood, increasing evidence suggests that dysregulation of certain brain circuits, including increased neuronal firing in key brain regions, may contribute to the symptoms of depression.
Depression treatment and unmet needs
Most available depression therapies have similar mechanisms, and innovation in recent years has been extremely limited. Standard-of-care agents also have liabilities, like weight gain and sexual dysfunction, and they can take several weeks to have an effect. There remains a high unmet need for new depression treatments.
Learn about our approach in depression
Pain
Acute and chronic pain affects more people than diabetes, heart disease, and cancer combined, yet effective, long-term treatment options are scarce.
Acute pain serves as the body's normal, short-term alarm signal in response to an injury or illness. In contrast, chronic pain is a widespread condition defined by the International Association for the Study of Pain (IASP) as pain that lasts or recurs for more than three months, persisting even after the initial cause has healed. It's more common than many realize; 2021 data from the US Centers for Disease Control and Prevention (CDC) show that it affects more than one in five American adults, often impacting daily life and well-being.