Lipocine Inc. · NASDAQ:LPCN
Lipocine — Investor Presentation (November 2017)
45 slides from Lipocine's investor presentation, Nov 8, 2017
Slide 1: Corporate Presentation
Enabling oral drug delivery to improve patient compliance November 8, 2017 Corporate Presentation Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward - Looking Statements This presentation contains forward - looking statements about Lipocine Inc. (the “Company”). These forward - looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward - looking statement s re
Slide 3: Unique Specialty Pharmaceutical Company
Men's Health Franchise Testosterone Replacement Therapy (“TRT”) Women's Health Preterm Birth (“PTB”) Proprietary Drug Delivery Platform Significant Unmet Need In Both Therapeutic Areas 3 Unique Specialty Pharmaceutical Company Advanced Pipeline
Slide 4: Late-Stage Pipeline
Late - Stage Pipeline 4 Oral Product Candidates Targeting Significant Opportunities PRODUCT (Indication) PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NDA MEN’S HEALTH TLANDO™ (Oral Testosterone Replacement Therapy, TRT) NDA PDUFA Date Feb 8, 2018 LPCN 1111 (Next Gen. Oral TRT) FDA Meeting 1Q 2018 WOMEN’S HEA
Slide 5: Lipocine is Positioned for Success Because
Lipocine is Positioned for Success Because…… 5 Significant Advantages Over Existing Approved Products LPCN 1107 • Poised to be First oral to the market • Potentially better PTB results • Orphan drug designation – Major contribution to patient care TRT Franchise TLANDO™ • Patient preferred first oral
Slide 6: The Oral TRT Challenge
The Oral TRT Challenge • Native testosterone has poor oral bioavailability with a very short half life (~30 minutes) – Impractical frequent daily doses would be required to obtain effective levels – Inconsistent and unpredictable performance • Methyl testosterone – Liver toxicity – Unsafe for chroni
Slide 7: TLANDO and Lipr'al Technology
TLANDO™ TLANDO ® (LPCN 1021) Transported via Lymph Not prone to liver issues METHYL TESTOSTERONE (MT) Transported via Liver Prone to liver toxicity Systemic Circulation Thoracic Duct Lymph Duct Liver Liver GI Lumen GI Lumen Systemic Circulation Lipocine’s Innovative Lipr’al Technology • Primarily di
Slide 8: TLANDO
LIPOCIN= TLANDO™
Slide 9: TRT Market Approaching Heights of 2013
2017 2016 2014 2015 350 375 400 425 450 475 500 525 550 575 600 625 Sep 14 Oct 14 Nov 14 Dec 14 Jan 15 Feb 15 Mar 15 Apr 15 May 15 Jun 15 Jul 15 Aug 15 Sep 15 Oct 15 Nov 15 Dec 15 Jan 16 Feb 16 Mar 16 Apr 16 May 16 Jun 16 Jul 16 Aug 16 Sep 16 Oct 16 Nov 16 Dec 16 Jan 17 Feb 17 Mar 17 Apr 17 May 17 J
Slide 10: TRT $ Market Opportunity Is Large
TRT $ Market Opportunity Is Large $0 $500,000,000 $1,000,000,000 $1,500,000,000 $2,000,000,000 $2,500,000,000 Jul-15 Jul-16 Jul-17 Dollar Volume Is Steady With Minimal Promotional Investment In 2016, Branded Products accounted for 80+ % of total TRT $ market volume 10 Source: IMS July 2017
Slide 11: Hypogonadism Affects Up to 6MM American Men
Hypogonadism Affects Up to 20 MM American Men 1,2 TLANDO™ Has The Potential To Drive Significant Market Expansion 1.5MM 2.2MM 6MM 700,000 Available switch patients Men currently being treated 4 Men with diagnosed hypogonadism 3 Available naïve patients per year 5 3.8MM diagnosed but untreated patien
Slide 12: Issues with Current Non-oral TRT Options
Issues with Current Non - oral TRT Options • Black Box Warning – Secondary exposure to testosterone – Pulmonary oil micro embolism (POME) and anaphylaxis shock • Inconvenient application or painful injection • Poor persistence reflects need for oral – Average days on therapy is 100 days • More than
Slide 13: TRT Market Research Results
TRT Market Research Results Patients And Physicians Are Asking For A Novel Oral Option • Unprompted physicians mention the need for an oral • A product with no titration is seen as an advantage • Physicians have a strong interest for an oral BID product 8.5 out of 10 • Physician’s allocate 38% of pr
Slide 14: TLANDO: Potential First Oral Option
TLANDO™: Potential First Oral Option Profile Demonstrated Clinically with Target Label Regimen ▪ Met primary endpoint - 80% response rate in “worst - case analysis” vs. FDA requirement of 75% ▪ Met key secondary endpoint - No eligible subjects with T levels >2500 ng/ dL ▪ Other secondary endpoints g
Slide 15: Current Area of Interest by the FDA: Blood Pressure
Current Area of Interest by the FDA SOAR Phase 3 Clinical Trial Results: Mean Blood Pressure 50 70 90 110 130 150 0 13 26 39 52 Blood Pressure (mmHg) Time (Week) Mean Blood Pressure at Each Visit (SOAR Trial) Diastolic TLANDO Diastolic Androgel 1.62% Systolic TLANDO Systolic Androgel 1.62% ▪ Mean Bl
Slide 16: AEs Related to Increased Blood Pressure / Hypertension
Current Area of Interest by the FDA SOAR Phase 3 Clinical Trial Results: AE’s related to Increased Blood Pressure / Hypertension System Organ Classification / Preferred Term TLANDO™ (N=210) Androgel 1.62% (N=104) n (%) Events (n) n (%) Events (n) Investigations: Blood Pressure Increased 1 (0.5) 1 2
Slide 17: TLANDO: Addresses Previous FDA Concerns
TLANDO™: Addresses Previous FDA Concerns Salient 2014 Oral TU ADCOM Approvability Issues Primary Efficacy End point: Robust with only one subject dropout Consistent efficacy in multiple trials with target dosing regimen Fixed dose: No titration requirement Stable to varied dietary fat consumption Sa
Slide 18: Next Steps to Bring TLANDO to Patients
Next Steps to Bring TLANDO™ to Patients Regulatory Approval Focus • PDUFA goal date of February 8, 2018 • Advisory Committee meeting to be held on January 10, 2018 Partnering/licensing due diligence and discussions on - going Key required pre - commercial readiness activities progressing to ensure l
Slide 19: LPCN 1111
LIPOCIN= LPCN 1111 upen 1111 \ » => >
Slide 20: Higher Prescribing Preference for QD Product
Higher Prescribing Preference for QD Product 20 Potential For Increased TRT Market Share CE11. Now, please assume that Product X was dosed QD rather than BID as originally presented. Please re - estimate the percentage of patients for whom you would prescribe each of the following therapies 31% 4% 9
Slide 21: LPCN 1111: Next-Generation Oral TRT
LPCN 1111: Next - Generation Oral TRT • Novel bio - reversible prodrug of testosterone for oral delivery • Once - daily potential expected to sustain and improve market share of oral T franchise • Once - daily feasibility established in Phase 2a and 2b clinical trials – Single - daily oral dose prov
Slide 22: LPCN 1107
LIPOCIN= LPCN 1107
Slide 23: LPCN 1107: Prevention of Preterm Birth, An Unmet Medical Need
LPCN 1107: Prevention of Preterm Birth (PTB) 23 An Unmet Medical Need 1 Pediatric Research (2006) 60, 775 – 776 Preterm Birth 1 - 19 weeks 20 weeks 34 weeks 37 weeks 40 weeks O NE P RETERM B IRTH E VERY M INUTE 1
Slide 24: High PTB Medical Costs
High PTB Medical Costs • 12% of all US pregnancies 1 (475 - 500K) result in PTB (< 37 weeks) - a leading cause of neonatal mortality and morbidity • First year medical costs for PTB infants are ~ 10x higher than for full term infants 2 • 28% of preterm births are to women with histories of early del
Slide 25: LPCN 1107: First Oral PTB Candidate
LPCN 1107: First Oral PTB Candidate 25 Characteristics Of The Only Approved Product for PTB IM HPC, Makena ® : Current preterm birth standard of care ▪ $334 M in sales in 2016 ; on track to do ~$385M - $395M in 2017 ▪ 20 - 25% patients below reported better efficacy HPC level threshold ▪ Total of 18
Slide 26: LPCN 1107 - Oral HPC
LPCN 1107 - Oral HPC ▪ Potential for superior efficacy with Phase 3 target dose ▪ No patient discomfort upon administration ▪ Steady state achieved in 7 days ▪ Orphan drug designation – Major contribution to patient care LPCN 1107: First Oral PTB Candidate 26 Addresses Unmet Need
Slide 27: LPCN 1107: First Oral PTB Candidate, Commercial Outlook
LPCN 1107: First Oral PTB Candidate First Oral HPC for Prevention of Recurrent PTB • Preferred route - of - administration is oral Strong Exclusivity Position • Orphan Drug Designation • Technology/IP protection Potential for Superior Efficacy • Fewer PTB babies with significant healthcare cost savi
Slide 28: LPCN 1107: Advancing to Phase 3 Readiness
LPCN 1107: Advancing to Phase 3 Readiness 28 Key Design Elements for Phase 3 Study Proposed indication • To reduce the risk of preterm birth in women with a singleton pregnancy who have a history of singleton spontaneous preterm birth Design Elements • Open label, RCT, non - inferiority (“NI”) study
Slide 29: Upcoming Milestones Driving Value
Upcoming Milestones 29 Driving Value in 2017 Event Expected Timing TLANDO™ Advisory Committee meeting January 10, 2018 PDUFA goal date February 8, 2018 LPCN 1111 FDA meeting to discuss Phase 3 clinical trial 1Q 2018 LPCN 1107 CMC: process characterization & scale - up completed 4Q 2017
Slide 30: Key Financial Metrics
Key Financial Metrics 30 Stock Price, Market Cap, Cash Balance Ticker Symbol LPCN (Nasdaq Capital Market) Closing Stock Price (11/3/17) $3.50/share Market Capitalization (11/3/17) $74.2 million Fully Diluted Shares Outstanding (9/30/17) 23.5 million Cash Balance (9/30/17) $25.7 million Bank Debt (9/
Slide 31: Lipocine Investment Highlights
MEN’S HEALTH Testosterone Replacement Therapy (“TRT”) Franchise • TLANDO™ : Potential first oral TRT option - Differentiated product targeting ~$2.0 billion established US TRT market - Poised to meet and exceed unmet need • LPCN 1111 : Next generation potential once - daily oral TRT option - Positiv
Slide 32: Appendix
Appendix LPOCINE
Slide 33: TLANDO: DV Study Efficacy Results
TLANDO™: DV Study (Pivotal Efficacy) – Efficacy Results Primary Endpoints – Demonstrated Robust Efficacy Measure FDA Targets Safety Set* “Worst Case” Analysis # Number of subjects 95 95 % subjects with C avg w ithin normal range (300 - 1080 ng/ dL ) ≥75% 81% 80% 95% CI lower bound ≥ 65% 72% 72% * Su
Slide 34: TLANDO: DV Study Secondary Endpoints
TLANDO™: DV Study – Efficacy Results Secondary Endpoints Generally Met FDA Targets ▪ Proportion of subjects with T Cmax in predefined ranges against FDA targets (permissible excursions) that originally developed for transdermals - Key secondary endpoint (> 2500 ng/ dL ) • No trial eligible patient e
Slide 35: TLANDO: Long Term Safety in SOAR Study
TLANDO™: Long Term Safety Demonstrated Over 52 Weeks in SOAR (Pivotal Safety) Study AE’s greater than 5% TLANDO Active Control Upper Respiratory Tract Infection 5.2% 5.8% Fatigue 2.4% 6.7% ADRs greater than 2% TLANDO Active Control Headache 0.5% 2.9% Acne 2.4% 0.0% Safety Population: Subjects who re
Slide 36: TLANDO: Phase 3 Design Study (SOAR)
TLANDO™: Phase 3 Design Study of Androgen Replacement (SOAR) 36 Open - label, randomized, active - controlled study of LPCN 1021 in hypogonadal men Screening N=315 0 Week 4 Week 8 Randomization LPCN 1021 225 mg, TU, BID with Meal (n=210) Active Control (n=105) PK/Dose Titration PK/Dose Titration PK/
Slide 37: TLANDO: DV Study vs SOAR Trial
TLANDO™: DV Study vs SOAR Trial Key Design & Efficacy Result Differences DV Study – 225 mg BID without titration – Taken with meal – No BMI restriction – Mean Cavg (CV): 476 ng/ dL (37%)* SOAR Trial – 225 mg BID with titration – Taken with standard (20% – 35% fat) meal – Exclude BMI > 38 kg/m2 – Mea
Slide 38: Safety Relevance of TLANDO Cmax Excursions
LIPOCIN= Safety Relevance of TLANDO™ Cmax Excursions
Slide 39: DV Study vs SOAR Trial Cmax Outside Pre-defined Ranges
TLANDO™: DV Study vs SOAR Trial Cmax * Outside the Pre - defined Ranges are Transient 39 22.3 23.0 21.9 22.0 1.7 1.0 2.1 2.0 0 4 8 12 16 20 24 T Cmax > 1500 ng/dL T Cmax > 1800 ng/dL T Cmax > 1500 ng/dL T Cmax > 1800 ng/dL Time ( hr ) Time Spent in a day below (hr) Time Spent in a day above (hr) *Al
Slide 40: Safety Relevance of Cmax > 1500 ng/dL
AE’s greater than 5% in all patients Cmax (ng/dL) > 1500 ≤ 1500 Upper Respiratory Tract Infection 2.6% 5.9% Fatigue 0% 2.5% ADRs greater than 2% in all patients Cmax (ng/dL) > 1500 ≤ 1500 Headache 0% 0.9% Acne 2.6% 0.9% AEs Impact * Cmax on Efficacy day and AEs / ADRs following Efficacy day Safety R
Slide 41: HPC Concentration and PTB Rates
HPC Concentration and PTB Rates 41
Slide 42: LPCN 1107: HPC PK-PD Correlation
LPCN 1107: HPC PK - PD Correlation 42 HPC Concentration and PTB Rate with IM HPC, Makena 1 46.3 % 27.0 % 29.6 % 31.3 % Quartile 1: 3.7 - 8.1 ng/mL Quartile 2: 8.2 - 9.8 ng/mL Quartile 3: 9.9 - 12.4 ng/mL Quartile 4: 12.5 - 56 ng/mL 15 25 35 45 55 6 8 10 12 14 16 PTB rate (%, N=315) HPC trough plasma
Slide 43: LPCN 1107: Dose Finding Study Design
LPCN 1107: Dose Finding Study Design • Open - label, four - period, four - treatment study • 12 healthy pregnant women - Ages 18 - 35 years; 16 - 18 weeks gestation • All subjects received all four treatments 43 PK Study: Oral LPCN 1107 vs IM HPC, Makena Treatment A 400 mg BID Treatment B 600 mg BID
Slide 44: LPCN 1107: Dose-Finding PK Study Results
LPCN 1107: Dose - Finding PK Study Results 1 44 Oral LPCN 1107 vs IM HPC, Makena 1. PK results obtained post 8 days of BID dosing for LPCN 1107 and post 5 weeks for weekly IM HPC, Makena Lower reported PTB rate threshold 0 10 20 30 40 50 60 0 200 400 600 800 1000 Cavg(0 - 24/ 0 - 168) (ng/mL) Dose (
Slide 45: LPCN 1107: Economic Impact
LPCN 1107: Economic Impact Potential Lower PTB Rate – US and Resulting Savings Assuming 4.3% lower PTB rate relative to Makena® ~6000 fewer annual PTBs ‡ Estimated annual cost saving in ~$310M ‡‡ 45 ‡: Assuming 100% of 140,000 eligible US population treated ‡‡: Assuming ~$51,600 medical costs/PTB