LPCN 2203 – Essential Tremor

LPCN 2203 – Essential Tremor

Improve daily activity performance 
Fewer CNS adverse effects
Less psychosocial burden

Product Candidate: LPCN 2203

Improve daytime functioning without inducing significant CNS-depressant effects

Product Attributes: 

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 tremor 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 creates 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 will gradually worsen over time.

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.
  • Tremor significantly impairs 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.
  • Disease impact is not always predicted by tremor amplitude alone; age, occupation, and lifestyle demand often drive treatment decisions.
  • ET is not only physically impairing but 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 essential tremor (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.

LPCN 2203 Oral NAS for Essential Tremor

Innovating beyond
boundaries for patients