LPCN 2101 – Epilepsy
Novel mechanism of action
Sustain seizure freedom
Bioidentical, unlikely to cause birth defects
Product Candidate: LPCN 2101
Bioidentical oral neuroactive steroid (NAS) for epilepsy
Product Attributes:
LPCN 2101 is an oral formulation of eltanolone, a new chemical entity (NCE). Eltanolone is chemically similar to a naturally occurring neuroactive steroid (NAS), pregnanolone, a positive allosteric modulator of Ɣ-aminobutyric acid (GABA) receptor, representing a novel mechanism of action specifically targeted for patients with refractory epilepsy experiencing uncontrolled seizures.
While targeting the goal of seizure control, LPCN 2101 also has the potential for additional benefits in psychiatric disorders comorbidities (e.g., anxiety and/or depression) and sleep impairment. Moreover, these oral endogenous NASs could potentially address some of the fetal toxicity concerns related to unplanned or planned pregnancy.
About Indication:
Epilepsy is a symptom of a neurological problem that causes sudden, brief seizures. It can occur as a result of a neurological injury, a structural brain lesion, as a part of many systemic medical diseases or maybe genetic in origin. Epilepsy is a disorder of the brain that causes seizures, affecting the physical, mental, and social well-being of people, and is associated with a 2 to 3 times greater mortality rate compared with the general population.
Approximately, 2.9 million adults and 456,000 children with active epilepsy, meaning they are either taking medication or have had a seizure in the past year. About 38% of adults with epilepsy report having a disability.
Drug-Resistant Epilepsy (DRE) is defined by ILAE as the failure of two appropriate anti-seizure medications (ASMs) to achieve sustained seizure freedom. DRE affects 30-40% of epilepsy patients in the U.S.
It is estimated that approximately 900,000 childbearing (“CB”) aged women suffer from active epilepsy in the U.S. Women of CB age with epilepsy face many additional challenges due to hormonal influences on seizure activity and endocrine function throughout the different phases of their reproductive cycles.
While the lowest effective dose and monotherapy are preferred, management of patients with epilepsy is focused on controlling seizures, avoiding adverse events, and maintaining quality of life.
Drug-resistant epilepsy (DRE) affects approximately 30–40% of people living with epilepsy in the United States. Many of these patients cycle through multiple antiseizure medications (ASMs) with limited or no success, leaving them without adequate seizure control. Rescue treatments (primarily benzodiazepines) do not prevent future seizures, they only stop the current episode. Limitations include high risk of seizure recurrence within hours or days after a cluster.
There remains a significant need for new ASMs with novel mechanisms of action that can help these patients achieve seizure freedom. A novel ASM is also expected to prevent status epilepticus and prevent patients from using emergency room for seizure management. Ideal treatment options would also minimize cognitive, mood, and systemic side effects while addressing comorbid conditions such as depression, anxiety, and cognitive impairment.
Women with epilepsy (WWE) experience unique challenges throughout their lifespan due to the interplay of seizures, hormonal fluctuations, and ASM exposure.
The risks associated with ASMs are particularly concerning during early pregnancy, where fetal malformation and other adverse outcomes remain a significant worry. As a result, the level of planning required for women of childbearing potential—including contraception management, timing of pregnancy, and close monitoring—creates a substantial burden on patients, caregivers, and the healthcare system.
In addition, nonadherence to ASMs, anxiety about unplanned pregnancy, and delays in pregnancy recognition are common among WWE. These issues are intensified by the fact that several ASMs can reduce the effectiveness of hormonal contraceptives, increasing the risk of unplanned pregnancies.
Given these multifactorial challenges, there remains a clear unmet need for a treatment option specifically designed for women with active epilepsy, one that supports seizure control while addressing the unique reproductive, hormonal, and mental health considerations faced by this population.
There remains an unmet need to make available a treatment option specifically for women with active epilepsy.