Lipocine Inc. · NASDAQ:LPCN
Lipocine — Investor Presentation (May 2025)
28 slides from Lipocine's investor presentation, May 2025
Slide 1: CNS Assets Presentation
CNS Assets Presentation May 2025 Lipocine │ January 2025 1
Slide 2: LPCN 2101 Endogenous Neuroactive Steroid for Epilepsy
LPCN 2101 Endogenous Neuroactive Steroid for Epilepsy
Slide 3: LPCN 2101: Positive Allosteric Modulator of the GABAA receptor
LPCN 2101: Positive Allosteric Modulator (PAM) of the GABAA receptor Lipocine Endogenous Neuroactive Steroid Epilepsy Targets Women With Focal Onset Epilepsy (WWE) Seizure (FOS) Acute Repetitive Seizure (ARS) Reddy and Estes, Trends Pharmacol Sci 2016 3
Slide 4: Anti-Seizures Activities of NAS and AEDs in Preclinical Models
Anti-Seizures Activities of NAS and AEDs in Preclinical Models Epilepsy Foundation Pipeline Conference 2018 Reddy DS Journal of Neuroendocrinology. 2021;00:e13028 https://doi.org/10.1111/jne.13028 • Neuroactive steroids (NAS) activities in protection of mice in most of the tonic and/or clonic and ge
Slide 5: Validated Proprietary Lip'ral Technology Platform
Validated Proprietary Lip’ral Technology Platform Oral Enablement of Neuroactive Steroid (NAS) Achieved Relevant Systemic Levels in Phase 1 Study Observed PK Profile Post Oral Administration 70 Mean Plasma Concentration of 60 50 NAS (ng/mL) 40 30 20 10 0 0 4 8 12 16 20 24 Time (hr) Study in post-men
Slide 6: LPCN 2101 Development Status
LPCN 2101 Development Status IND enabling pre- clinicals completed Phase 1 Next Step IND cleared for • Well tolerated epilepsy study • PK dose proportionality Phase 2 POC study* • PK dose completed proportionality *Photosensitive epilepsy for NAS and dose evaluation under consideration Corporate Pre
Slide 7: Women with Epilepsy (WWE)
Woman with Epilepsy (WWE)
Slide 8: Women with Epilepsy (WWE) of Childbearing Age
Women with Epilepsy (WWE) of Childbearing Age 900,000 of childbearing age women suffer from active epilepsy in US Treatment Challenges for WWE Seizure Control Pre-Pregnancy Pregnancy Comorbidities • Drug-drug interaction • Planned pregnancy • Fetal/neonatal toxicity • Anxiety - AED selection • Adher
Slide 9: Women with Epilepsy of Childbearing Age - Opportunity
Women with Epilepsy (WWE) of Childbearing Age - Opportunity Prevalence Standard of Care Limitations Unmet Need ~ 900,000 of women of childbearing Seizure control with low/no No drug approved specifically for (CB) suffer from active epilepsy in teratogenic risk WWE US1,2 Most approved ASMs have fetal
Slide 10: LPCN 2101: Novel Potential Alternative for WWE
LPCN 2101 Novel Potential Alternative for WWE Product Attributes Positive Allosteric Modulator (PAM) of the GABAA receptor Oral dosage form comprising a neuroactive steroid Product Candidate Differentiation Novel MOA specifically addressing WWE unmet needs Active molecule is endogenous to women Pote
Slide 11: Focal Onset Seizure (FOS)
Focal Onset Seizure (FOS)
Slide 12: Adult Focal Onset Seizure Landscape
Adult Focal Onset Seizure Landscape Estimated U.S. Diagnosed Adult FOS Patient Population (2020) 18M Patients (M) Adults with FOS Source: Xenon sponsored market research Easy to Control Difficult to Control Severe Refractory Treatment goal is to optimize efficacy while managing comorbidities and max
Slide 13: Typical POC & Pivotal Trial Design
Typical POC & Pivotal Trial Design Multicenter, double blind, randomized placebo-controlled adjunct therapy design for focal onset seizures Baseline for 8 weeks seizure evaluation 10 weeks of maintenance treatment phase Two weeks dose – ramp • Average of ≥ 4 seizures in 28 days • seizure frequency:
Slide 14: Acute Repetitive Seizure (ARS)
Acute Repetitive Seizure (ARS) Potential for Orphan Drug Designation
Slide 15: ARS Prevalence and Market Potential
ACUTE REPETITIVE SEIZURE (ARS) PREVALENCE AND MARKET POTENTIAL MARKET SIZE TO SURPASS AROUND US$ 4.3 BN BY 2027 Many names: Cluster seizures, serial seizures, crescendo seizures, seizure flurries, recurrent seizures, or cyclical seizures More than 2-3 seizures in 24 hours, or in some studies in
Slide 16: Candidate Attributes for ARS Indication
CANDIDATE Fast onset: Endogenous Bioavailable ATTRIBUTES Tmax: ≤ 90 NAS through oral min* administration* FOR ARS INDICATION Easy to Portability administer *Based on Lipocine Phase 1 studies results 16
Slide 17: Anti-Epileptic Drug (AED) Market Potential
ANTI-EPILEPTIC DRUG (AED) MARKET POTENTIAL HTTPS://WWW.FORTUNEBUSINESSINSIGHTS.COM/INDUSTRY-REPORTS/SEGMENTATION/ANTIEPILEPTIC-DRUG-MARKET-101189 17
Slide 18: LPCN 2203 Oral NAS for Essential Tremor
LPCN 2203 Oral NAS for Essential Tremor
Slide 19: Essential Tremor (ET)
Essential Tremor (ET) No new drug approved in 50+ years • Tremor highly disabling and stigmatizing • Stress can aggravate tremor in social setting • Major impact on activities of daily living leading to unemployment, anxiety and depression1 • Most common impacts on activities of daily living are pou
Slide 20: ET Patient Journey and Commonly Used Medications
ET Patient Journey and Commonly used medications • First line treatment of propranolol frequently started at PCP • 2nd and 3rd line treatments (e.g., primidone, benzodiazepine, gabapentin, topiramate) at general neurologist and movement disorder clinics • Patient survey indicates on-going management
Slide 21: Essential Tremor Management – Opportunity
Essential Tremor Management – Opportunity Daytime efficacy and improved tolerability remains an unmet need Prevalence Standard of Care Limitations Unmet Need • ~7 million patients in US1 Unfavorable benefit to risk profile3 Superior benefit to risk profile • Most of patients are intolerant or • Impr
Slide 22: Market Potential and Competitive Development Landscape
Market Potential and Competitive Development Landscape $3B+ blockbuster opportunity as 2nd line with 20% peak adoption Products in Development* Estimated ~7 M prevalence 2.2 % of SAGE-324: Adverse events - somnolence population1 68%, dizziness 38%, balance disorder 15%, diplopia 12%, dysarthria 12%,
Slide 23: LPCN 2203: Oral GABA Positive Allosteric Modulator
LPCN 2203: Oral GABA Positive Allosteric Modulator Achieved relevant target levels with good tolerability in Phase 1 studies Typical single dose PK profiles LPCN 2203 TEAEs Dose 1 Clinical Study Experience, N=28, 304 doses: Mean Plasma Concentration • LOC 0% Dose 2 • Somnolence, sedation 0% Dose 3 •
Slide 24: LPCN Product Target Differentiation
LPCN Product Target Differentiation Favorable benefit to Novel MOA risk profile Daytime efficacy Endogenous NAS Potential to address anxiety and depression comorbidities Corporate Presentation September 2022 Lipocine | 2024 24
Slide 25: Appendix
Appendix
Slide 26: LPCN 2101 Additional Potential Indications
LPCN 2101 Additional Potential Indications Other Convulsion Disorders Status epilepticus Catamenial Generalized onset (SE) epilepsy seizures Lennox-Gastaut Absence seizures Dravet Syndrome Syndrome 26
Slide 27: Mechanism of Action of AEDs Related to GABAA receptor
Mechanism of Action of AEDs Related to GABAA receptor Support LPCN 2101 MOA 27
Slide 28: Business Development Contacts
Business Development Contacts Kong Papangkorn, Ph.D. Business Development Manager kp@lipocine.com