Essential Tremor – LPCN 2203
Product Candidate: LPCN 2203
Improve daytime daily functioning without CNS depressant effects.
Product Attributes:
LPCN 2203 is an oral formulation of a bioidentical GABAA receptor modulating neuroactive steroid. It is targeted for the treatment of essential tremor and has the potential to reduce tremor enabling daily activities by a mechanism novel to currently available but unapproved treatments. Moreoover, LPCN 2203 with it unique GABA PAM activity is expected to address the psychiatric comorbidities associated with ET.
About Indication:
Essential Tremor (“ET”) is one of the most common movement disorders, affecting an estimated 7 million adults in the United States.1 For those with ET, uncontrollable shaking of the hands, head, and voice creates difficulty eating, dressing, writing, and completing other day-to-day tasks.2 In addition to physical symptoms, anxiety and depressive symptoms often manifest and have a substantial social impact.1
The etiology of ET is largely unknown, but gamma aminobutyric acid (GABA)ergic dysfunction has been postulated as a key contributor.3 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.
While ET is often associated with aging populations, ET can begin much earlier in life, with a progressive disease course that can eventually necessitate a care partner. Social anxiety and depressive symptoms can manifest in patients with ET as tremor severity increases, and may negatively impact a patient’s ability to work and engage in hobbies. In an interview study of ET patients and care partners, the most common impacts on activities of daily living are pouring liquids and writing/typing (100%) and grooming/hygiene, drinking, dressing, eating, and reading (80-85%). Overall, 90% of participants noted the emotional impact of ET, with 75% reporting tremor-related worry or anxiety.
1. Vetterick C, Lyons KE, Matthews LG. Adv Ther 2022;39:5546-5567.
2. American Academy of Neurology Practice Guideline 2011 Patient Summary
3. Gironell A. Tremor Other Hyperkinet Mov 2016;4:254.
The only FDA-approved pharmacological treatment for ET was approved more than 50 years ago, with very limited clinical evidence of efficacy.
Currently, there is only one drug (propanolol) FDA-approved for ET, and many second-line therapies have limited evidence of efficacy.
Approximately 30-50% of people do not respond to treatment, and discontinuation rates are as high as 63% within 5 years of treatment initiation. Therefore, a significant unmet need exists for ET pharmacotherapy, including daytime efficacy and improved tolerability, a PRN (pro re nata) or “as needed” option, as well as a need to address anxiety/depression comorbidity associated with ET.