Lipella Pharmaceuticals, Inc. · OTC:LIPO
Lipella Pharmaceuticals — 2025 BIO CEO & Investor Conference
39 slides from Lipella Pharmaceuticals's conference presentation, Feb 11, 2025
Slide 1: A Clinical Stage Biotechnology Company Addressing Serious Diseases with Significant Unmet Need
A Clinical Stage Biotechnology Company Addressing Serious Diseases with Significant Unmet Need 2025 BIO CEO & Investor Conference Feb 11, 2025, New York, NY Jonathan Kaufman Michael Chancellor, MD Chief Executive Officer Chief Medical Officer NASDAQ:LIPO lipellapharmaceuticals.com
Slide 2: Disclaimers
NASDAQ:LIPO DISCLAIMERS The information in this presentation is being provided so you can familiarize yourself with Lipella Pharmaceuticals Inc. (“Lipella,” the “Company,” “we,” “us,” or “our”) during this informational meeting. We request that you keep any information we provide at this meeting con
Slide 3: Differentiated Technology Platform: Liposomal Formulation of Tacrolimus
NASDAQ:LIPO DIFFERENTIATED TECHNOLOGY PLATFORM: Liposomal Formulation of Tacrolimus Maximizing Therapeutic Responses with Directed Localized Administration Optimized Efficacy By encapsulating tacrolimus in liposomes, our technology ensures a more stable and controlled release, maximizing therapeutic
Slide 4: Company Pipeline
NASDAQ:LIPO COMPANY PIPELINE Proprietary Asset Pre-clinical IND-Enabling Phase 1 Phase 2 Phase 3 Approval LP-10 Hemorrhagic Cystitis LP-310 Oral Lichen Planus LP-410 Oral Graft-versus-Host Disease (GvHD) LP-50 Bladder Cancer A Clinical Stage Biotechnology Company Addressing Serious Diseases with Sig
Slide 5: De-risked Lead Programs with Robust Revenue Opportunity
NASDAQ:LIPO DE-RISKED LEAD PROGRAMS WITH ROBUST REVENUE OPPORTUNITY 505(b)(2) drug candidates Positioned For Market Exclusivity LP-10 Liposomal tacrolimus for the treatment of • Hemorrhagic cystitis affects ~60,000 annually Hemorrhagic cystitis. LP-10 has a well- • Expected annual revenue per patien
Slide 6: LP-310 for Oral Lichen Planus
NASDAQ:LIPO Lipella Lead Pipeline Product: LP-310 for Oral Lichen Planus Phase 2a multicenter dose escalation study completion anticipated in 2Q25 - Similar formulation of LP-10 for oral rinse - Increased local concentration in oral cavity while minimalizing systemic toxicity - 505b2 pathway expand
Slide 7: What Is Oral Lichen Planus (OLP)?
NASDAQ:LIPO WHAT IS ORAL LICHEN PLANUS (OLP)? • Chronic Disease: OLP is a T-cell-mediated autoimmune disorder affecting the oral mucosa. • Long-Term Condition: Unlike cutaneous lichen planus, OLP follows a chronic course, with most available therapies being palliative rather than curative. • Prevale
Slide 8: OLP Symptoms, Complications and Impact
NASDAQ:LIPO OLP SYMPTOMS, COMPLICATIONS AND IMPACT • Appearance: Lacy, white, raised patches; red, swollen, tender areas; open sores. • Location: Found inside cheeks, gums, tongue, lips, and Erosive type lichen planus- ulcerated palate. lesion in the buccal mucosa with erythematous borders • Symptom
Slide 9: Current Treatment Options for Oral Lichen Planus
NASDAQ:LIPO CURRENT TREATMENT OPTIONS FOR ORAL LICHEN PLANUS (OLP) Category Examples Key Advantage Key Limitation Topical Steroids Clobetasol, Betamethasone Reduces inflammation Risk of mucosal thinning Systemic Steroids Prednisone Controls severe cases Significant side effects Immunomodulators Tacr
Slide 10: Risks of Topical Steroid Use for OLP
NASDAQ:LIPO RISKS OF TOPICAL STEROID USE FOR ORAL LICHEN PLANUS (OLP) Risk/Problem Description Clinical Consequence Fungal Infection Immunosuppression in the oral cavity Oral thrush, requires antifungal treatment Masking Infections Steroids mask signs of bacterial/viral infections Delayed diagnosis
Slide 11: Positive Phase 2a Results for LP-310 in Oral Lichen Planus
NASDAQ:LIPO Positive Phase 2a Results for LP-310 in Oral Lichen Planus A Clinical Stage Biotechnology Company Addressing Serious Diseases with Significant Unmet Need 11
Slide 12: LP-310: Visible Treatment Impact
NASDAQ:LIPO LP-310: VISIBLE TREATMENT IMPACT Day 1 First Oral Rinse- Left mandibular gingiva Week 4 Last Oral Rinse- Left mandibular gingiva The decrease in inflammation, redness and healing of the white ulcers is remarkable and patients reported significant improvement. A Clinical Stage Biotechnolo
Slide 13: Study Overview
NASDAQ:LIPO STUDY OVERVIEW • Multicenter dose-ranging study in adult male and female subjects aged ≥18 years with symptomatic OLP designed to evaluate the safety, tolerability, and preliminary efficacy of LP-10 at dose levels of 0.25 mg, 0.5 mg, and 1.0 mg of tacrolimus. • Approximately 24 subjects
Slide 14: OLP Pain, Sensitivity and GRA
NASDAQ:LIPO OLP PAIN, SENSITIVITY AND GRA • Pain NRS: Improved OLP Pain OLP Sensitivity from 6.63+0.80 (baseline) to 10 10 4.38+0.96 at week 1 8 8 (p=0.004), 2.38+1.15 at week 4 (p=0.004), 6 6 and 3.60+1.63 at week 6 (p=0.031). 4 4 • GRA: Significant 2 2 improvement was 0 0 observed at week 4 (p=0.0
Slide 15: OLP Symptom Severity Measurement (OLPSSM)
NASDAQ:LIPO OLP SYMPTOM SEVERITY MEASUREMENT (OLPSSM) OLP Symptom Severity Measurement • OLPSSM (0-7): Reduced from 15.38+2.20 (baseline) to 10.13+2.34 at week 1 (p=0.035), 5.00+2.28 at week 4 (p=0.004), and 8.60+4.06 at 25 week 6 (p=0.031). 20 • Similar improvement seen in OLPSSM 8, 9 and 10 15 out
Slide 16: OLP REU Scoring System
NASDAQ:LIPO OLP Reu Scoring System 40 OLP REU SCORING SYSTEM 30 • REU: Improved from 27.75+2.71 20 (baseline) to 17.56+2.51 at week 1 (p=0.004), 12.69+3.06 at week 4 (p=0.004), and 19.60+4.31 at 10 week 6 (p=0.031). 0 Baseline Week 1 Week 4 Week 6 A Clinical Stage Biotechnology Company Addressing Se
Slide 17: OLP Investigator Global Assessment (IGA)
NASDAQ:LIPO OLP INVESTIGATOR OLP Investigator Global Assessment GLOBAL 4 ASSESSMENT (IGA) 3 • IGA: Decreased from 3.50+0.19 (baseline) to 2 2.75+0.31 at week 1 (p=0.031), 1.75+0.45 at week 1 4 (p=0.008), and 2.80+0.37 at week 6 (p=0.125). 0 Baseline Week 1 Week 4 Week 6 A Clinical Stage Biotechnolog
Slide 18: LP-310 Phase 2a Topline Results Summary
NASDAQ:LIPO LP-310 PHASE 2A TOPLINE RESULTS SUMMARY LP-310 data LP-310 Statistically significant Next Step shows safety pharmacokinetic improvement of efficacy ▪ Successful completion of ▪ No Product analysis demonstrated ▪ Investigator measurement of oral Phase 2a multicenter clinical related SAEs
Slide 19: LP-310 for Oral Lichen Planus: Blueprint for Success
NASDAQ:LIPO LP-310 FOR ORAL LICHEN PLANUS BLUEPRINT FOR SUCCESS A Parallel to Restasis: Developed by Allergan for dry eye disease, addressing an unmet need with no FDA-approved therapies. Market Impact: Despite early skepticism, its targeted, Steroid Limitations: Prolonged steroid use in the non-ste
Slide 20: Oral Lichen Planus Market Snapshot
NASDAQ:LIPO ORAL LICHEN PLANUS MARKET SNAPSHOT Target market: 6 to 7 million Americans Population of Severe Cases: 1.5 million Projected Annual Revenue per customer: $8,000 A Clinical Stage Biotechnology Company Addressing Serious Diseases with Significant Unmet Need 20
Slide 21: LP-310 Mechanism of Action
NASDAQ:LIPO LP-310 MECHANISM OF ACTION • Tacrolimus inhibits calcineurin, blocking T-cell activation and reducing inflammatory cytokines that are central to OLP pathology. • Advantages over steroid: Reduced risk of mucosal atrophy, suitability for patients unresponsive to steroids and lower systemic
Slide 22: Meta-Analysis Supports Tacrolimus in Oral Lichen Planus Treatment
NASDAQ:LIPO Meta-Analysis Supports Tacrolimus in Oral Lichen Planus Treatment: A Pathway for LP-310’s Success Clinical Meta-Analysis Highlights Tacrolimus’ Role in Oral Lichen Planus: LP-310's Pathway to Innovation Study % Study Events, Events, % ID OR (95% CI) Weight ID OR (95% CI) Treatment Contro
Slide 23: Clinical Pipeline to Treat Rare and Serious Diseases with Unmet Medical Need
NASDAQ:LIPO Clinical Pipeline to Treat Rare and Serious Diseases with Unmet Medical Need A Clinical Stage Biotechnology Company Addressing Serious Diseases with Significant Unmet Need 23
Slide 24: LP-10 for Hemorrhagic Cystitis (HC)
NASDAQ:LIPO LP-10 FOR HEMORRHAGIC CYSTITIS (HC) Liposomal tacrolimus treatment for hemorrhagic cystitis • Potent vasoconstrictor; reduces Urgent Need For Effective HC Treatment Options capillary blood flow to the bladder lumen • Potent anti-inflammatory; inhibits cytokine • HC is challenging to trea
Slide 25: LP-410 for Oral Graft-Versus-Host Disease
NASDAQ:LIPO LP-410 FOR ORAL GRAFT-VERSUS-HOST DISEASE Oral Graft-Versus-Host Disease (GVHD) is a clinical syndrome where donor-derived immunocompetent T cells react against patient tissues directly or through exaggerated inflammatory responses following HCT. • GVHD is a major cause of morbidity and
Slide 26: LP-50 for Bladder Cancer
NASDAQ:LIPO LP-50 FOR BLADDER CANCER Novel liposomal formulation of checkpoint inhibitors • Intravesical formulation for local, intravesical PD-1 (i.e. checkpoint) inhibition, intended for the treatment of non-muscle invasive bladder cancer (NMIBC). • Intravesical immunotherapy presents a promising
Slide 27: A Clinical Stage Biotechnology Company Addressing Serious Diseases
A Clinical Stage Biotechnology Company Addressing Serious Diseases with Significant Unmet Need Lipella Pharmaceuticals Inc. 7800 Susquehanna Street, Suite 505 Pittsburgh, PA 15208 412-894-1853 NASDAQ:LIPO lipellapharmaceuticals.com
Slide 28: Appendix I: LP-310 Clinical Design
2025 BIO CEO & Investor Conference Feb 11, 2025, New York, NY Presentation Appendix I: LP-310 Clinical Design Lipella Pharmaceuticals Inc. 7800 Susquehanna Street, Suite 505 Pittsburgh, PA 15208 412-894-1853 NASDAQ:LIPO lipellapharmaceuticals.com
Slide 29: LP-310 Clinical Snapshot
NASDAQ:LIPO LP-310 CLINICAL SNAPSHOT Design : Randomized, prospective multicenter dose escalation study with 24 patients. Efficacy : Anticipate a statistically significant decrease in oral lichen planus lesion score within 1 week of administration, maintained for at least 4 weeks. Quality of Life :
Slide 30: Trial Design Summary
NASDAQ:LIPO TRIAL DESIGN SUMMARY Recruitment sites and subject disposition Patient Disposition Number Comments Patient Demographics Mean Comment Age, years 61 Range 38-72 Screened, n 12 2 male, 10 female Race Enrolled, n 8 2 male, 6 female White 5 Tolerated in all Black 1 Number of instillations 448
Slide 31: Inclusions and Exclusions
NASDAQ:LIPO INCLUSIONS AND EXCLUSIONS Key Inclusion Criteria: Key Exclusion Criteria: ▪ Moderate OLP based on an OLP Investigator ▪ History of oral cavity or oropharyngeal cancers Global Assessment (IGA) score of ≥ 3 ▪ Patients who failed tacrolimus treatment for OLP in ▪ OLP Pain or Sensitivity Num
Slide 32: Key Takeaways LP-310
NASDAQ:LIPO KEY TAKEAWAYS LP-310 • Phase 2a multicenter trial on tract for completion • Targeting Large patient population with unmet needs in 2Q25 including: - 6 million patients living with OLP. • Tolerability: First in human Phase 2a LP-310 - Affects women 3x more often than men well tolerated wi
Slide 33: Appendix II: LP-10 Market Data
2025 BIO CEO & Investor Conference Feb 11, 2025, New York, NY Presentation Appendix II: LP-10 Market Data Lipella Pharmaceuticals Inc. 7800 Susquehanna Street, Suite 505 Pittsburgh, PA 15208 412-894-1853 NASDAQ:LIPO lipellapharmaceuticals.com
Slide 34: Cancer Survivorship & Hemorrhagic Cystitis
NASDAQ:LIPO CANCER Prostate Uterine Corpus Rectal & Other SURVIVORSHIP & Cancer Cancers Pelvic HEMORRHAGIC 700,000 200,000 300,000 Pelvic Radiation Survivors Pelvic Radiation Survivors Pelvic Radiation Survivors CYSTITIS 42,000 12,000 18,000 • Expected annual revenue per Severe HC Survivors Severe H
Slide 35: LP-10 Addresses a Potential $1.2 Billion Market
NASDAQ:LIPO LP-10 Addresses a potential $1.2 Billion Market Expected annual reimbursement is $20,000 per patient Market penetration of 60,000 patients yields $1.2 billion in recurring revenue Cancer Survivor Survivors of Pelvic Severe Original Incidence Prevalence Prevalence Original Malignancy Radi
Slide 36: Appendix III: Several LP-310 Endpoint Descriptions
2025 BIO CEO & Investor Conference Feb 11, 2025, New York, NY Presentation Appendix III: Several LP-310 Endpoint Descriptions Lipella Pharmaceuticals Inc. 7800 Susquehanna Street, Suite 505 Pittsburgh, PA 15208 412-894-1853 NASDAQ:LIPO lipellapharmaceuticals.com
Slide 37: Investigator Reported
NASDAQ:LIPO INVESTIGATOR REPORTED OLP Investigator Global Assessment (IGA) Definition version Definition version Score Grade Ulcer Ery thema Ulcer Erythema 0 Clear No ucler and None None and None 1 Almost clear A single ulcer <2 mm and/or Minor Barely perceptible and/or Light red Single ulcer 2-4.9m
Slide 38: Patient Reported
NASDAQ:LIPO PATIENT REPORTED OLP Pain and Sensitivity Rating Scale (NRS) Patient Global Oral Cavity Pain Response Please circle the number that best describes the Oral Lichen Planus pain Assessment (GRA) that you have experienced over the last 24 hours. As compared to when you started the study, how
Slide 39: A Clinical Stage Biotechnology Company Addressing Serious Diseases
A Clinical Stage Biotechnology Company Addressing Serious Diseases with Significant Unmet Need Lipella Pharmaceuticals Inc. 7800 Susquehanna Street, Suite 505 Pittsburgh, PA 15208 412-894-1853 NASDAQ:LIPO lipellapharmaceuticals.com
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