Lipocine Inc. · NASDAQ:LPCN
Lipocine — EASL Congress (June 2024)
24 slides from Lipocine's conference presentation, Jun 10, 2024
Slide 1: LPCN 1148 improves sarcopenia and hepatic encephalopathy in cirrhosis: 52-week phase 2 trial
S°EASL CONGRESS . PS Intervention with oral LPCN 1148 improves sarcopenia and hepatic encephalopathy (HE) in © ‘patients with cirrhosis: a 52-week phase 2 S randomized clinical trial a De — a,’ : i <i
Slide 2: Forward-Looking Statements
easicongress.eu #EASLCongress Forward-Looking Statements This presentation contains “forward-looking statements” that are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and include statements that are not historical facts regarding our product dev
Slide 3: Sarcopenia is common in cirrhosis and impacts clinically meaningful outcomes
Yi} Yi} | })\ , | Nfl oo Sarcopenia is common in cirrhosis and impacts clinically meaningful outcomes How patients feel: ¢ Health related quality of life How patients function: ¢ Frailty ¢ Activities of daily living How patients survive: ¢ Hepatic encephalopathy ¢ Mortality ¢ Peri-transplant outcome
Slide 4: Muscle mass pathways and low testosterone in cirrhosis
jongress Leucine" Exercise” y vers Linked to muscle mass Inhibits myostatin { Modulates mTOR L__yy Increases protein synthesis T Decreases turnover Y ‘ mTOR : - 90% of males with 4 cirrhosis have low free SATELLITE CELL PROLIFERATION testosterone AND DIFFERENTIATION Y MUSCLE PROTEIN SYNTHESIS 1. Sin
Slide 5: Product Candidate Attributes and Mechanism of Action
easicongress.eu #EASLCongress Product Candidate Attributes Oral androgen receptor agonist Dosage form comprising testosterone dodecanoate, a unique prodrug of the endogenous hormone Targeted Mechanism of Action Increase muscle mass and Via Improved liver function® and Induce hematopoiesis®; Improve
Slide 6: Central Hypothesis
easicongress.eu Central Hypothesis: LPCN 1148 will improve sarcopenia, sarcopenia related outcomes and clinically significant outcomes in patients with cirrhosis
Slide 7: Aims and objectives
eK i" di Ml Z Aims and objectives Specific Aims * To perform a phase 2 proof of » To generate proof of concept concept study to establish the —_ of overall benefit of LPCN short term safety and efficacy 1148 to support further ‘easicongress.eu #EASLCongress CO EE of LPCN 1148 in male development of
Slide 8: Study population
Mh i. th #EASLCongress ‘ Study population Inclusion criteria « Adult males ¢ Failure to obtain consent * Cirrhosis * Active severe encephalopathy * Sarcopenia — defined by BMI- + Active infection adjusted SMI" + Uncontrolled or recurrent Gl ¢ Listed for liver transplantation bleed in past 6 months *
Slide 9: Study design: two-arm, two-stage
#EASLCongress Two-arm, two stage (1:1 randomization, N=29) * Oral LPCN 1148, T dodecanoate, vs. Placebo * Standard of care treatments allowed, including HE therapies Stages: 1. 24-week placebo-controlled 2. 28-week open-label extension (OLE) + All subjects receive LPCN 1148 during OLE SCREENING TREA
Slide 10: Endpoints
‘easicongress.eu #EASLCongress t i Endpoints Primary endpoint * Baseline-adjusted change in “Week 24 and week 52: Skeletal Muscle Index (L3 * Feel and function: region, L3-SMl) at Week 24 in * patient reported outcomes (PGI-C) LPCN 1148 treated * Survives: ai * acute worsening of encephalopathy part
Slide 11: Baseline characteristics
Baseline ehareeiaies Age (years) BMI (kg/m?) L3-SMI (cm2/m?) MELD Score Medical History 2 1 Decompensation Event* 2 2 Decompensation Event Hepatic Encephalopathy (HE) Medical Therapy for HE* Ascites Esophageal Varices *Mecical therapy for HE includes lactulose an Sey date characte are mean + SD, mes
Slide 12: Participant disposition
easicongres #EASLCongress Participant disposition Study discontinuation: + Withdrawal of consent following liver transplant (n=2) -ompliane with protocol + Investigator recommendation to withdraw (n=2) + Death (n=1) Assessed for eligibility (N=48) Allocated LPCN 1148 (N=15) Completed Week 24 (n=15)
Slide 13: Analysis datasets
easicongress.eu #EASLCongress Analysis datasets LPCN 1148 switch LPCN 1148 Placebo nomplaceso (N=15) (N=14) (N=10) Safety set* Stage 1 15 14 — Stage 2 1 — 8 Modified intent-to-treat (mITT)* Stage 1 15 10 = Stage 2 15 — 6 * For LPCN 1148 switch from placebo, change from baseline during Stage 2 is cal
Slide 14: Primary endpoint met: LPCN 1148 improves L3-SMI at week 24
S2EASL CONGRES ila #EASLCongress Primary endpoint met : LPCN 1148 improves in L3-SMI at week 24 oe a LPCN 1148 Placebo Week 12 IL Week 24 Absolue Change from Baseline in L3-SMI (cm?/m?) Data are LS mean (SE). mITT set based on study drug compliance and having at least one post-baseline CT with LOCF.
Slide 15: LPCN 1148 improvement in L3-SMI observed during OLE
Tillan, tay easicongress.eu #EASLCongress LPCN 1148 improvement in L3-SMI observed during OLE L3-SMI Change from Baseline 12 =@=LPCN 1148 = =@—Placebo = @= Placebo to 1148 10 (n=15) (n=10) (n=6) * 7 J * Increased muscle area was maintained * REWeee through 52 weeks of treatment with 6 * LPCN 1148 ~
Slide 16: Substantial changes in muscle quality with LPCN 1148
Relative Change from Baseline in Muscle Composition Parameters (%) 15) -20 25 -30 LPCN 1148 12 n=10 IL Mar n study drug compliance and having at leas easicongress.eu Substantial Changes in Muscle Quality seen with LPCN 1148 Therapy * Significant increase in high quality muscle (HQM, +30 to +150 Houn
Slide 17: Overt hepatic encephalopathy events
Congress Recurrence Pe Overt HE was qesesesd with LPCN 1148 while on HE standard medication(s) Placebo LPCN 1148 REM TS LPCN 1148 switch from placebo Parameter (Through Week 24) | (Through Week 24) | (Week 24t0E0S) | Weoko4 19 EOS) N=14 N=15 N=11 ea History of HE prior to 5 5 5 mane eettan 11 (79% 1
Slide 18: LPCN 1148 improved hemoglobin and anemia in cirrhosis
LPCN 1148 improved hemoglobin and anemia in cirrhosis LPCN 1148 switch from placebo (Week 24 to EOS) N=38 Placebo LPCN 1148 (Through Week 24) | (Through Week 24) N=14 N=15 Baseline; mean (SE) 11.4 (0.8) Hemoglobin (g/dL) Week 24 CFB; mean (SE) 0.9 (0.3)*,* = Week 52 CFB; mean (SE) Ceo 1.4 (0.5)* 1.7
Slide 19: LPCN 1148 improved patient reported symptoms severity
i LPCN 1148 improved patient report ed symptoms severity Placebo LPCN 1148 LPCN 1148 switch (Through Week (Through Week from placebo 24) 52) (Week 24 to EOS) N=14 N=15 N=8 Patient Global Week 24 mean (SD) Impression of Change (PGI-C) Week 52 mean (SD) (SE), mITT set. compared to placebo + PGI-C —a s
Slide 20: Liver Frailty Index
easicongress.eu #EASLCongress LPCN 1148 LPCN 1148 switch (Through Week (Through Week from placebo 24) 52) (Week 24 to EOS) N=14 N=15 N=8 Baseline; mean (SE) 4.1 0.2) z Liver Frailty Ind BEN fe ndex Week 24 CFB; mean (SE) 0.17 (0.18) -0.17 (0.14) - Week 52 CFB; mean (SE) | - | -0.21 (0.15) -0.32 (0.1
Slide 21: Trends towards improvement in other secondary endpoints
Ml ny ae LPCN 1148: trends towards improvement in other secondary endpoints LPCN 1148 switch from placebo (Week 24 to EOS) N=8 Placebo LPCN 1148 LPCN 1148 (Through Week 24) | (Through Week 24) | (Week 24 to EOS) N=14 N=15 N=11 Deaths; n (%) 2 (14% 0 1 (9%) 0 11 ) Hospitalizations; n (%) 5 (33%) 5 (4
Slide 22: Safety and adverse events
easicongress.eu #EASLCongress LPCN 1148 switch from placebo (Week 24 to EOS) N=8 Placebo LPCN 1148 LPCN 1148 (Through Week 24) | (Through Week 24) | (Week 24 to EOS) N=14 N=15 N=11 Total AEs; n (%) 9 (64%) 9 (60%) 7 64%) 7 (88%) Serious AEs; n (%) 5 (33%) 5 (45%) 1 (13%) Severe AEs; n (%) 4 (27%) 3
Slide 23: Summary and conclusions
Summary and conclusions Oral androgen receptor agonist LPCN 1148 improved sarcopenia in adult male patients with cirrhosis + This improvement was maintained through 52 weeks of treatment LPCN 1148 was further associated with improvement in encephalopathy, anemia, total number of days hospitalized, a
Slide 24: Acknowledgements
Acknowledgements * We acknowledge the contribution of all of the investigators and staff for this trial and express our gratitude to the patients who participated in this study