Lipocine Inc. · NASDAQ:LPCN
Lipocine — Investor Presentation (March 2015)
40 slides from Lipocine's investor presentation, Mar 2, 2015
Slide 1: Corporate Presentation March 2015
Corporate Presentation March 2015 Exhibit 99.1
Slide 2: Forward-looking statements
2 This presentation contains and our discussions during this conference may include 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 forw
Slide 3: Lipocine Corporate Highlights
3 Lipocine Corporate Highlights ; Focused on innovative products for men’s and women’s health ; Lead asset NDA filing 2H 2015 ; Technology platform capable of “multiple hits” ; Several near term value drivers with pipeline products “Transformative“ Oral Testosterone Franchise First Oral Alternative
Slide 4: Product Pipeline
4 Product Pipeline LPCN 1021 - Oral Testosterone Replacement Therapy (TRT) Men’s Health LPCN 1111 - Next Generation Oral TRT Women’s Health LPCN 1107 - Pregnancy Support: Prevention of Preterm Birth Research/Preclinical Phase 1 Phase 2 Phase 3
Slide 5: Lip'ral Enables Optimal Dispersion in the Gastrointestinal Tract
5 Lip’ral Enables Optimal Dispersion in the Gastrointestinal Tract Lumen Aqueous Barrier Layer Membrane Systemic Circulation Improved absorption Effective transfer across aqueous barrier layer Optimal dispersed phase in GI tract Insoluble drug lipidic composition 5
Slide 6: Near Term Value Drivers
6 Near Term Value Drivers 4Q 2014 1 Q 2015 2 Q 2015 3 Q 2015 4Q 2015 LPCN 1021: 1 Year Safety Data LPCN 1021: File NDA LPCN 1107: FDA Development Guidance Meeting LPCN 1021: Pre - NDA Meeting with the FDA LPCN 1021: Last Patient Last Visit LPCN 1111: Initiate Phase 2b Study* * Subject to FDA clarity
Slide 7: Testosterone Deficiency is Prevalent in U.S.
7 Testosterone Deficiency is Prevalent in U.S. 1 1. Rev. Urol Vol 5. Suppl 1 2003 S3 - S10 - Figure 2 (2) Archive of Internal Medicine 2008 • Men in their 50s 1 in 10 • Men in their 60s 1 in 5 • Men in their 70s 1 in 4 Only 12% of Men with Low T Receive TRT 2
Slide 8: TRT Patient Demographics by Age
8 TRT Patient Demographics by Age ; The majority of TRT patients are ≤ 64 years old (82.6%)
Slide 9: Testosterone Replacement Therapy Market
9 ; US market > $2.3B in 2013 1 ; US market ~ 6.5M TRx in 2014 1 • 13% decrease in TRx from 2013 ; Current TRT “ class” label under review by FDA in US ; International markets for TRT growing ; Topicals dominate the market (~ 86% 1 of $ sales) • Inadvertent transference issue, black box warning • Po
Slide 10: Oral Expected to Improve Compliance and Patient Retention
10 ; Survey of 28 leading endocrinologists and urologists about oral testosterone compliance 1 • In your opinion, will oral testosterone improve patient compliance compared to existing options? x Oral T is expected to increase per patient revenue through better retention rates Oral Expected to Impro
Slide 11: If Approved, Oral T Will Likely Change the Landscape
11 If Approved, Oral T…. “ will likely dramatically change the landscape with regard to testosterone therapies, because the oral route of administration will be considerably easier to use than the more cumbersome routes of administration available with the commonly used marketed products 1 ” 1. Hylt
Slide 12: LPCN 1021: Oral Treatment Option for TRT
12 ; Novel product primarily directed at lymphatic delivery of Testosterone Undecanoate (TU) ; Enhanced profile over other forms of oral testosterone (T) • Native T has short half life • Methyl T is known to have liver safety issues ; Maintains effective T blood levels when dosed BID ; Target label
Slide 13: LPCN 1021: A Safer TRT Option
13 LPCN 1021: A “Safer” TRT Option ; Transfer potential to children, partner and pets ; No freedom to use around pregnant loved ones ; Skin irritation potential ; Lung impairment potential ; Scarring/injection site reactions ; Risk of infection ; Not flexible for dose reversals x No risk of transfer
Slide 14: LPCN 1021: Life Uninterrupted with Oral T
14 LPCN 1021: Life “Uninterrupted” with Oral T x Discrete x No waiting • Prior to dressing • In doctor’s office x Suitable for “on the go” use x No shower/swim restrictions x No mess/clean up x Non invasive Improved Compliance Convenience Enhanced Efficacy Higher Retention Rates
Slide 15: Current Regulatory Paradigm
15 Current Regulatory Paradigm 1 ; Meet primary end points • Responder analysis relating to average serum concentration 0 to 24 hours ( C avg ) and lower bound Confidence Interval (CI) ; Acceptable safety profile • Responder analysis relating to peak testosterone levels that is consistent with appro
Slide 16: SOAR Trial Phase 3 Study Design
16 ; Open label, randomized, active - controlled study of LPCN 1021 in men with low T Study of Oral Androgen Replacement (SOAR) Trial - Phase 3 Study Design Screening N = 315 0 Week 4 Week 8 Randomization LPCN 1021 225mg, TU, BID w/ meal (n = 210) Active Control (n = 105) PK /dose titration PK /dose
Slide 17: SOAR Trial - Dataset Definition
17 ; Primary Analysis Set • Efficacy Population (N=152): Subjects randomized into the study with at least one PK profile and no significant protocol deviations x Imputed missing data by Last Observation Carried Forward (LOCF) ; Additional Analysis Set • Safety Set (N=210): Subjects randomized into t
Slide 18: SOAR Trial - Primary Endpoint: Responder Analysis
18 SOAR Trial - Primary Endpoint: Responder Analysis and C avg Measure Efficacy Population Safety Set Full Analysis Set Number of subjects 152 210 192 % subjects with C avg w ithin normal range 88.2% 80.0% 87.5% 95 % CI lower bound 81.9% 73.8% 82.0% ; Primary endpoint target ≥ 75% subjects should ac
Slide 19: Primary Endpoint: Cavg and Distribution in Subjects Range
19 Primary Endpoint: C avg and Distribution in Subjects Range Efficacy Population C avg < 300 ng / dL 11.2 % C avg betwee n 300 and 1140 ng/ dL 88.2 % C avg > 1140 ng / dL 0.7 % Parameter Mean (CV) C avg ( ng / dL ) 447 (37%) ; % of subjects with C avg in various ranges x Less than 12% of the subjec
Slide 20: Converging Titration Regimen
20 Converging Titration Regimen Dataset: Efficacy population, N=152 Pre - titration Profile (All subjects at 225 mg) Post - titration P rofile 0 300 600 900 1200 0 6 12 18 24 Serum T Concentration (ng/dL) Time (Hr) Week 3 Subjects needing down titration Subjects needing no titration Subjects needing
Slide 21: SOAR Trial - Maintenance Dose Distribution
21 Final Dose (mg TU, BID) % of subjects 150 36% 225* 51% 300 13% SOAR Trial – Maintenance Dose Distribution of Subjects Dataset: Efficacy population, N=152 *starting dose x Majority of subjects with final dose of 225 mg BID
Slide 22: SOAR Trial - Titration Visits
22 SOAR Trial - Titration Visits Dataset: Efficacy population, N=152 Parameter % of subjects % subjects requiring no more than one dose change (either after week 3 or week 7) 85% % subjects requiring two dose changes (both after week 3 and 7) 15% x Vast majority of subjects reached final dose with n
Slide 23: SOAR Trial - Secondary Endpoints
23 SOAR Trial - Secondary Endpoints Measure FDA Threshold Efficacy Population Number of subjects 152 C max < 1500 ng/ dL ≥ 85 % 82.9% 1800 ≤ C max ≤ 2500 ng/ dL ≤ 5 % 4.6% C max > 2500 ng/ dL None 2.0% ; Proportion of subjects achieving maximum serum total T concentrations (C max ) in predefined C m
Slide 24: SOAR Trial - Excessive T Repletion Outliers
24 ; Excessive T repletion outliers were observed in three subjects ; These observed excessive T repletion outliers were transient, sporadic, isolated, and not clinically meaningful • Lack of dose or dosing time dependency • None of these subjects reported any adverse events SOAR Trial - LPCN 1021 E
Slide 25: SOAR Trial - LPCN 1021 Top Line Phase 3 Results Summary
25 ; Unequivocally met primary efficacy end point ; Patient friendly dosing regimen • No more than one titration required in 85% of the subjects • Starting dose was the right dose for majority of the subjects ; Variability consistent with or better than approved products ; Consistent intra/inter day
Slide 26: TRT Advisory Committee Meeting
26 TRT Advisory Committee Meeting ; US FDA Advisory committee meeting Topics for Discussion • Appropriate population for testosterone replacement therapy • Potential for adverse cardiovascular outcomes associated with TRT ; Recommendations • Voted in favor of revising the current label for testoster
Slide 27: FDA Confirmation to Lipocine
27 ; FDA confirmed that the design of the Phase 3 clinical study is currently acceptable for filing a NDA ; FDA noted that a sufficient number of subjects are required with at least one baseline and post - baseline efficacy response (Full Analysis Set) to provide adequate power to demonstrate effica
Slide 28: Competitive Environment for Oral Testosterone
28 Competitive Environment for Oral Testosterone Rextoro™: Clarus Therapeutics Current Status • Advisory Committee voted 18 - 3 that the overall benefit/risk profile of Rextoro® did not support approval • PDUFA date was November 3, 2014 - no announcement of approval LPCN 1021 Advantages • Unequivoca
Slide 29: LPCN 1111: Next Generation Oral Testosterone
29 ; Novel prodrug of testosterone for oral delivery through proprietary Lip’ral technology ; Maintains effective T blood levels when dosed once daily ; Status • Positive Phase 2a study results in hypogonadal men – Single daily oral dose provides T levels in the eugonadal range – No subject exceeded
Slide 30: LPCN 1111: Phase 2a Study in Hypogonadal Men
30 ; Five arm, sequential ascending dose study (N=12) • 3 single dose QD arms, (330mg, 550mg and 770mg TT) • 2 multiple dose QD arms for 28 days (550mg and 770mg TT) ; Successful outcomes • Demonstrated that once daily dosing is feasible • Good dose response • Steady state achieved by day 14 • No su
Slide 31: LPCN 1111 Phase 2a Study - Responder Analysis
31 LPCN 1111 Phase 2a Study - Responder Analysis At Day 28 Measure 550mg QD 770mg QD Typical FDA targets for approval of TRT % subjects with C avg w ithin normal range 67% 88% ≥ 75% % of subjects with C max ≤ 1500ng/ dL 100% 100% ≥ 85% % of subjects with C max between 1800ng/ dL and 2500mg/dl 0% 0%
Slide 32: Preterm Birth Represents a Significant Unmet Medical Need
32 Preterm Birth (PTB) Represents a Significant Unmet Medical Need One Preterm Infant per Minute in the US 1 □ 11.7% of all US pregnancies 2 result in PTB - a leading cause of mortality and morbidity 3 □ ~10x more first year medical costs are for PTB infants than for full term infants 4 □ ≥ $26 bill
Slide 33: LPCN 1107: Oral Candidate for Prevention of Preterm Birth
33 ; Potential to be the first oral standard - of care therapy • Same API as in the only approved injectable product • Elimination of 18 - 22 injections • Potential for orphan drug designation ; Status • Successful Phase 1a POC study in healthy non - pregnant women • Successful Phase 1b POC in pregn
Slide 34: LPCN 1107: Phase 1b Pregnant Women Study - Design and Outcomes
34 ; Open - label, three period, three treatment serial dosing study (N=8) • Period 1 : Two doses, 400 mg LPCN 1107 administered 12 hours apart • Period 2: Two doses, 800 mg LPCN 1107 administered 12 hours apart • Period 3: Single dose of 250 mg IM HPC ; Periodic blood sampling: Oral up to 24 hours,
Slide 35: LPCN 1107: Phase 1b Study in Pregnant Women - Results
35 LPCN 1107: Phase 1b Study in Pregnant Women - Results * one subject data analysis pending ; Single dose pharmacokinetic parameters Dose (BID) (N=7*) C max (ng/ml) [range] AUC 0 - 24 ( ng.h /ml) [range] 400mg BID 21.3 [11.5 – 36.2] 156 [81 – 234] 800mg BID 63.2 [37.8 - 144] 577 [323 – 1365] x Firs
Slide 36: LPCN 1107: Phase 1b Study in Pregnant Women - Results
36 LPCN 1107: Phase 1b Study in Pregnant Women - Results * one subject data analysis pending # AUCss calculated for post steady state 1 week duration ; Simulated steady state pharmacokinetic parameters Products / Dosing Regimen (N=7*) C ss,max (ng/ml) [range] AUC SS ( ng.h /ml ) # [range] LPCN 1107
Slide 37: LPCN 1107: Phase 1b Study in Pregnant Women - Results
37 LPCN 1107: Phase 1b Study in Pregnant Women - Results * one subject data analysis pending # AUCss calculated for post steady state 1 week duration ; Bioavailability in pregnant (study LPCN 1107 - 14 - 003) and non - pregnant women (study LPCN 1107 - 14 - 001) Study population (N) AUC SS ( ng.h /m
Slide 38: Competitive Environment for HPC for Prevention of Preterm Birth
38 Competitive Environment for HPC for Prevention or Preterm Birth Makena®: AMAG Pharmaceuticals, Inc. Current Status • Recently acquired from Lumara Health in November 2014 for $675M in upfront payments and potential additional contingent consideration of up to $350M • Net sales of Makena in 2014 w
Slide 39: Lipocine is a Compelling Value Proposition
39 Lipocine is a Compelling Value Proposition Stock Exchange NASDAQ Capital Markets Ticker Symbol LPCN Closing Stock Price (2/27/15) $7.24/share Market Capitalization (2/27/15) $92.7 million Fully Diluted Shares Outstanding (2/27/15) 14,356,586 Warrants Outstanding 0.14% Cash Balance (9/30/14) $31.7
Slide 40: Lipocine Corporate Highlights
40 Lipocine Corporate Highlights ; Focused on innovative products for men’s and women’s health ; Lead asset NDA filing 2H 2015 ; Technology platform capable of “multiple hits” ; Several near term value drivers with pipeline products “Transformative“ Oral Testosterone Franchise First Oral Alternative