LPCN 2203 – Essential Tremor
Improve daily activity performance
Fewer CNS adverse effects
Less psychosocial burden
Product Candidate: LPCN 2203
Bioidentical oral neuroactive steroid (NAS) for essential tremor (ET)
Product Attributes:
LPCN 2203 is being advanced for essential tremor (ET), with the potential to improve daytime functioning in patients with essential tremor, without significant CNS-depressant effects.
LPCN 2203 is an oral formulation of a bioidentical GABAA receptor modulating neuroactive steroid for essential tremor (ET). Synaptic GABAA receptors are the most abundant form of GABAA receptors in the brain with the α1β2γ2 receptor subtype alone accounting for ~43% of all GABAA receptors. The NAS in LPCN 2203 is highly potent PAM targeting synaptic GABAA receptor subunit α1β2γ2. LPCN 2203 has the potential to reduce tremors, enabling daily activities through this novel mechanism. Additionally, LPCN 2203 is expected to address comorbidities associated with ET.
About Indication:
Essential Tremor (ET) is one of the most common movement disorders, affecting an estimated seven million adults in the United States.
Patients suffering with ET may have symptoms of uncontrollable shaking of the hands, head, and voice, causing difficulty eating, dressing, writing, and completing other day-to-day tasks. In addition to physical symptoms, anxiety and depressive symptoms often manifest and have a substantial social impact. ET onset can occur at any age, but the risk is greatest in older populations. The disease course is progressive and typically worsens over time.
Reportedly, the functional and psychosocial burden of ET is profound:
- 90% of patients experience difficulty with writing, eating, drinking, and performing self-care.
- 79% of employed individuals reduce their work hours or change jobs because of tremor severity.
- 56% of patients rely on caregivers—family, friends, or professionals—to manage daily activities.
- Tremors significantly impair the ability to perform essential tasks such as eating, drinking, and typing, often resulting in loss of independence, unemployment, social withdrawal, and mood disorders such as anxiety and depression.
- The impact of the disease is not always predicted by tremor amplitude alone; age, occupation, and lifestyle often drive treatment decisions.
- ET not only causes physical impairment, but is also highly stigmatizing, contributing to low quality of life.
- Current medications provide limited symptom relief, and patients who remain refractory frequently resort to invasive surgical interventions.
Currently available pharmacological options for ET are inadequate. The only FDA-approved medication for ET was introduced more than 50 years ago, and its clinical efficacy remains limited. Despite widespread use, 30–50% of patients do not respond to existing therapies, and up to 63% discontinue treatment within five years due to insufficient benefit or poor tolerability.
Given these limitations, there is an urgent need for innovative, well-tolerated, and effective therapies that can improve daily functioning, reduce psychosocial burden, and offer flexible treatment options for people living with essential tremor.