# EDGAR Filing Document

**Accession Number:** 0001347242
**File Stem:** 0001753926-25-001525
**Filing Date:** 2025-9
**Character Count:** 41501
**Document Hash:** e7a32d9ee22374705a905132ad9f3143
**Contains OCR:** False
**Source Format:** 

## Filing Content

## Filing Summary
**0001753926-25-001525.hdr.sgml**: 20250918

**ACCESSION NUMBER**: 0001753926-25-001525

**CONFORMED SUBMISSION TYPE**: 8-K

**PUBLIC DOCUMENT COUNT**: 43

**CONFORMED PERIOD OF REPORT**: 20250918

**ITEM INFORMATION**: Regulation FD Disclosure

**ITEM INFORMATION**: Other Events

**ITEM INFORMATION**: Financial Statements and Exhibits

**FILED AS OF DATE**: 20250918

**DATE AS OF CHANGE**: 20250918

**FILER**: 

**COMPANY DATA:**
- **COMPANY CONFORMED NAME:** LIPELLA PHARMACEUTICALS INC.
- **CENTRAL INDEX KEY:** 0001347242
- **STANDARD INDUSTRIAL CLASSIFICATION:** PHARMACEUTICAL PREPARATIONS [2834]
- **ORGANIZATION NAME:** 03 Life Sciences
- **EIN:** 000000000
- **STATE OF INCORPORATION:** DE
- **FISCAL YEAR END:** 1231

**FILING VALUES:**
- **FORM TYPE:** 8-K
- **SEC ACT:** 1934 Act
- **SEC FILE NUMBER:** 001-41575
- **FILM NUMBER:** 251321803

**BUSINESS ADDRESS:**
- **STREET 1:** 400 N LEXINGTON ST
- **STREET 2:** STE LL103
- **CITY:** PITTSBURGH
- **STATE:** PA
- **ZIP:** 15208
- **BUSINESS PHONE:** 412-901-0315

**MAIL ADDRESS:**
- **STREET 1:** 400 N LEXINGTON ST
- **STREET 2:** STE LL103
- **CITY:** PITTSBURGH
- **STATE:** PA
- **ZIP:** 15208

**FORMER COMPANY:**
- **FORMER CONFORMED NAME:** LIPELLA PHARMACEUTICALS INC
- **DATE OF NAME CHANGE:** 20051219

?xml version='1.0' encoding='ASCII'?

**UNITED STATES**

**SECURITIES AND EXCHANGE COMMISSION**

**Washington, D.C. 20549**

**FORM 8-K**

**CURRENT REPORT**

**Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934**

Date of Report (Date of earliest event reported): **September 18, 2025**

**Lipella Pharmaceuticals Inc.**

(Exact name of registrant as specified in its charter)

---

| | | |
|:---|:---|:---|
| **Delaware** | **001-41575** | **20-2388040** |
| (State or other jurisdiction <br> of incorporation) | (Commission File Number) | (IRS Employer <br> Identification No.) |

---

---

| | |
|:---|:---|
| **7800 Susquehanna St., Suite 505**<br>**Pittsburgh, PA** | **15208** |
| (Address of registrant's principal executive office) | (Zip code) |

---

Registrant's telephone number, including area code: **(412) 894-1853**

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

---

| | | |
|:---|:---|:---|
| **Title of each class** | **Trading Symbol(s)** | **Name of each exchange on which registered** |
| Common Stock, par value $0.0001 per share | LIPO | The Nasdaq Stock Market LLC \* |

---

\*Trading of the registrant's common stock on Nasdaq was suspended on June 20, 2025. The registrant's common stock is currently quoted on the OTCID Market operated by OTC Markets Group Inc. under the symbol "LIPO."

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company ☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

**Item 7.01 Regulation FD Disclosure**

*Press Release* 

On September 18, 2025, Lipella Pharmaceuticals Inc. (the "Company") issued a press release announcing positive final results from its completed Phase 2a multicenter, dose-ranging study evaluating LP-10, a proprietary liposomal tacrolimus oral rinse, in patients with symptomatic oral lichen planus ("OLP"). A copy of the press release is attached hereto as Exhibit 99.1.

*Corporate Presentation* 

The Company updated its presentation used by management to describe its business. A copy of the presentation is attached hereto as Exhibit 99.2.

The information in this Current Report on Form 8-K under Item 7.01, including the information contained in Exhibits 99.1 and 99.2, is being furnished to the Securities and Exchange Commission, and shall not be deemed to be "filed" for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities of that section, and shall not be deemed to be incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as shall be expressly set forth by a specific reference in such filing.

**Item 8.01 Other Events.**

On September 18, 2025, the Company announced positive final results from its completed Phase 2a multicenter, dose-ranging study evaluating LP-10, a proprietary liposomal tacrolimus oral rinse, in patients with symptomatic OLP. The study met its primary safety endpoint and achieved statistically significant improvements across all efficacy measures at the 4-week timepoint. These results support LP-10's potential to become the first FDA-approved therapy for OLP, a chronic inflammatory condition that affects an estimated 6 million Americans, with no approved treatment options.

The Phase 2a study was a multicenter, dose-ranging trial conducted at five leading U.S. clinical sites. Twenty-seven adults with biopsy-confirmed symptomatic OLP were sequentially enrolled into three dose cohorts. Patients used a 3-minute LP-10 oral rinse twice daily for 4 weeks, followed by a 2-week safety follow-up. The study population was representative of typical OLP patients: 81.5% were female, the median age was 62 years, and disease duration ranged from 1 to 28 years. All participants had previously failed standard therapies, including topical corticosteroids.

All 27 patients completed the full four-week treatment course, and no serious adverse events were reported. Systemic exposure was minimal, with 76% of tacrolimus blood measurements below detection limits (<1.0 ng/mL). Treatment-related adverse events were mild to moderate, with dry mouth being the most common, occurring in 18.5% of patients.

All three dose groups (0.25 mg, 0.5 mg, and 1.0 mg) demonstrated statistically significant improvements at Week 4 (all p<0.05) on secondary efficacy endpoints. Investigator Global Assessment scores showed clear reductions in ulceration and erythema, pain and sensitivity improved significantly with patients reporting meaningful reductions on numerical rating scales, and patient-reported symptoms, measured by the OLP Symptom Severity Measure (OLPSSM), showed meaningful improvement in overall symptom burden. Sustained efficacy was observed, with all patients maintaining clinical benefit and no evidence of worsening through the two-week follow-up.

Based on these results, the Company is advancing LP-10 into late-stage development, including preparation of a Phase 2b protocol incorporating FDA feedback, scaling manufacturing to support larger clinical trials, and exploring strategic partnerships and collaborations.

**Item 9.01 Financial Statements and Exhibits.**

---

| | |
|:---|:---|
| **Exhibit No.** | **Description** |
| [99.1](g084955_ex99-1.htm) | [Press Release, dated September 18, 2025](g084955_ex99-1.htm) |
| [99.2](g084955_ex99-2.htm) | [Investor Presentation](g084955_ex99-2.htm) |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) |

---

**SIGNATURES**

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

---

| | | |
|:---|:---|:---|
| Date: September 18, 2025 | **Lipella Pharmaceuticals Inc.** | **Lipella Pharmaceuticals Inc.** |
|  | By: | */s/ Jonathan Kaufman* |
|  |  | Name: Jonathan Kaufman <br> Title: Chief Executive Officer  |

---

## Exhibit 99.1

**Exhibit 99.1**

**Lipella Pharmaceuticals Announces Positive Final Results from Phase 2a Study of LP-10 in Oral Lichen Planus**

&nbsp;&nbsp;&nbsp;&nbsp;▪ *All 27 patients completed treatment with no serious adverse events, underscoring a favorable safety profile* 

&nbsp;&nbsp;&nbsp;&nbsp;▪ *Statistically significant improvements achieved across all efficacy endpoints at the 4-week timepoint* 

&nbsp;&nbsp;&nbsp;&nbsp;▪ *Preparing to initiate a pivotal Phase 2b study to advance LP-10 toward registration* 

PITTSBURGH, September 18, 2025 – Lipella Pharmaceuticals Inc. (Nasdaq : LIPO) ("Lipella," "we," "our," or the "Company"), a clinical-stage biotechnology company transforming care with innovative mucosal delivery solutions, today announced positive final results from its completed Phase 2a multicenter, dose-ranging study evaluating LP-10, a proprietary liposomal tacrolimus oral rinse, in patients with symptomatic oral lichen planus (OLP).

The study met its primary safety endpoint and achieved statistically significant improvements across all efficacy measures at the 4-week timepoint. These results support LP-10's potential to become the first FDA-approved therapy for OLP, a chronic inflammatory condition that affects an estimated 6 million Americans, with no approved treatment options.

"We believe these compelling results validate LP-10's potential to address a large, underserved patient population," said Jonathan Kaufman, CEO and co-founder of Lipella. "The combination of excellent safety, minimal systemic absorption, and robust efficacy across multiple validated endpoints positions LP-10 as a first-in-class therapy. Based on these data, we are preparing to advance LP-10 into a pivotal Phase 2b study."

**Key Study Results**

**Safety Profile**

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• All
27 patients completed the full 4-week treatment course

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• No
serious adverse events reported

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Minimal
systemic exposure: 76% of tacrolimus blood measurements were below detection limits (<1.0 ng/mL)

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Well-tolerated,
with only mild to moderate treatment-related adverse events; dry mouth being the most common, occurring in 18.5% of patients

**Efficacy Outcomes**

All three dose groups (0.25 mg, 0.5 mg, and 1.0 mg) demonstrated statistically significant improvements at Week 4 (all p<0.05) on the secondary efficacy endpoints:

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Investigator
Global Assessment (IGA) showed clear reductions in ulceration and erythema scores

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Pain
and Sensitivity improved significantly, with patients reporting meaningful reductions on numerical rating scales

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Patient
symptoms, as measured by the OLP Symptom Severity Measure (OLPSSM), demonstrated meaningful improvement in overall symptom burden

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Sustained
efficacy was observed, with all patients maintaining clinical benefit and no evidence of worsening through the 2-week follow-up
period

"Investigator examinations revealed significant healing, including reduction in inflammation and visible resolution of ulcerative lesions," said Dr. Michael Chancellor, Chief Medical Officer and Co-Founder of Lipella. "These observations were consistently supported by both investigator and patient-reported outcomes, demonstrating LP-10's meaningful impact on this debilitating condition. Importantly, the therapeutic benefits were generally maintained through the 2-week follow-up period."

**Study Design**

The Phase 2a study was a multicenter, dose-ranging trial conducted at five leading U.S. clinical sites. Twenty-seven adults with biopsy-confirmed symptomatic OLP were sequentially enrolled into three dose cohorts. Patients used a 3-minute LP-10 oral rinse twice daily for 4 weeks, followed by a 2-week safety follow-up.

The study population was representative of typical OLP patients: 81.5% were female, the median age was 62 years, and disease duration ranged from 1 to 28 years. All participants had previously failed standard therapies, including topical corticosteroids.

**Next Steps**

Based on these positive results, Lipella is advancing LP-10 into late-stage development:

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Preparation
of Phase 2b protocol incorporating FDA feedback

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Scaling
manufacturing capabilities to support larger clinical trials

&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;• Exploring
strategic partnerships and collaborations

"We believe LP-10 represents a paradigm shift in OLP treatment, if approved" added Kaufman. "By delivering tacrolimus through our proprietary liposomal formulation as an oral rinse, we've overcome the delivery challenges that have limited topical treatments while maintaining an exceptional safety profile. These results strengthen our conviction that LP-10 has the potential to transform the treatment landscape for millions of patients living with this chronic condition."

Full study results will be submitted for publication in a peer-reviewed journal and presented at upcoming medical conferences.

**About Oral Lichen Planus**

Oral lichen planus is a chronic inflammatory condition affecting the oral mucosa, characterized by white reticular lesions, erosions, and ulcerations that cause significant pain and functional impairment. Classified by the WHO as an oral potentially malignant disorder, OLP carries a malignant transformation risk of approximately 1.4%. Despite affecting an estimated 6 million Americans, there are currently no FDA-approved therapies, leaving patients to rely on off-label treatments with limited efficacy and concerning side effect profiles.

**About LP-10**

LP-10 is a proprietary liposomal formulation of tacrolimus designed as an oral rinse for the treatment of oral lichen planus. The sphingomyelin-based liposomal delivery system enables effective local administration while minimizing systemic absorption, addressing the key limitations of current topical treatments. LP-10 has previously demonstrated safety and preliminary efficacy in a Phase 2a study for hemorrhagic cystitis.

**About Lipella Pharmaceuticals Inc.**

Lipella Pharmaceuticals is a clinical-stage biotechnology company focused on reformulating and optimizing existing active pharmaceutical ingredients for new therapeutic applications in diseases with high unmet needs. The company completed its initial public offering in 2022. Learn more at <u>lipella.com</u> and follow us on <u>X</u> and <u>LinkedIn</u>.

**Forward-Looking Statements**

This press release includes certain "forward-looking statements." All statements, other than statements of historical fact, included in this press release regarding, among other things, our strategy, future operations, financial position, prospects, clinical trials, regulatory approvals, pipeline and opportunities, sources of growth, successful implementation of our proprietary technology, plans and objectives are forward-looking statements. Forward-looking statements can be identified by words such as "may," "will," "could," "continue," "would," "should," "potential," "target," "goal," "anticipates," "intends," "plans," "seeks," "believes," "estimates," "predicts," "expects," "projects" and similar references to future periods. Forward-looking statements are based on our current expectations and assumptions regarding future events and financial trends that we believe may affect among other things, market and other conditions, our financial condition, results of operations, business strategy, short- and long-term business operations and objectives, and financial needs. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. Our actual results may differ materially from those contemplated by the forward-looking statements. We caution you, therefore, against relying on any of these forward-looking statements. They are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, but are not limited to, risks related to clinical trial outcomes, regulatory approval processes, market acceptance, competitive products, intellectual property rights, availability of funding, and other factors described in our SEC filings. Any forward-looking statement made by us is based upon the reasonable judgment of our management at the time such statement is made and speaks only as of the date on which it is made. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

**CONTACT:**

Jonathan Kaufman<br> Chief Executive Officer<br> Lipella Pharmaceuticals Inc.<br> <u>Info@Lipella.com</u><br> 1-412-894-1853

## Exhibit 99.2

**Exhibit 99.2**

![](img001.jpg)

1 lipellapharmaceuticals.com A Clinical Stage Biotechnology Company Transforming Care with Innovative Mucosal Delivery Solutions Corporate Presentation September 2025

![](img002.jpg)

2 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 confidential and that you do not disclose any of the information to any other parties without the Company's prior express written permission . Although the Company believes the information contained herein is accurate in all material respects, the Company does not make any representation or warranty, either express or implied, as to the accuracy, completeness or reliability of the information contained in this presentation . Forward - Looking Statements The presentation includes certain "forward - looking statements . " All statements, other than statements of historical fact, included in this presentation regarding, among other things, our strategy, future operations, financial position, anticipated dividends, projected costs, prospects, pipeline and opportunities, sources of growth, successful implementation of our proprietary technology, plans and objectives are forward - looking statements . Forward - looking statements can be identified by words such as "may," "will," "could," "continue," "would," "should," "potential," "target," "goal," "anticipates," "intends," "plans," "seeks," "believes," "estimates," "predicts," "expects," "projects" and similar references to future periods . Forward - looking statements are based on our current expectations and assumptions regarding future events and financial trends that we believe may affect our financial condition, results of operations, business strategy, short - and long - term business operations and objectives, and financial needs . Because forward - looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict . Our actual results may differ materially from those contemplated by the forward - looking statements . We caution you, therefore, against relying on any of these forward - looking statements . They are neither statements of historical fact nor guarantees or assurances of future performance . There are risks, uncertainties and other factors, both known and unknown, that could cause actual results to differ materially from those in the forward - looking statements which include, but are not limited to, regional, national or global political, economic, business, competitive, market and regulatory conditions, and other factors . Any forward - looking statement made by us is based upon the reasonable judgment of our management at the time such statement is made and speaks only as of the date on which it is made . Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them . We undertake no obligation to update any forward - looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law . Nothing contained herein is, or shall be relied upon as, a promise or representation as to the past or future . The Company expressly disclaims any and all liability relating to or resulting from the use of this presentation . In addition, the information contained in this presentation is as of the date hereof, and the Company has no obligation to update such information, including in the event that such information becomes inaccurate . You should not construe the contents of this presentation or other information we provide at this meeting as legal, tax, accounting or investment advice or a recommendation . You should consult your own counsel and tax and financial advisors as to legal and related matters concerning the matters described herein .

![](img003.jpg)

3 Experienced Management Team Jonathan Kaufman, PhD Chief Executive Officer Michael Chancellor, MD Chief Medical Officer Doug Johnston, CPA Chief Financial Officer 23+ Years Experience Co - founded Lipella in 2005 Co - founder of Knopp Biosciences. CFO of Semprus Biosciences. CSO LaunchCyte LLC. PhD Penn. MBA Wharton. 30+ Years Experience Lipella co - founder. Joined Lipella in 2008 Co - founder of Cook - MyoSite ; Professor of Urology, William Beaumont Medical Center. MD University of Michigan. 15+ Years Experience Jointed Lipella in 2021 Experience in global and early - stage pharma finance operations Certified public accountant in Pennsylvania

![](img004.jpg)

4 Value of Liposomal Delivery Improved Mucosal Adhesion and Retention Liposomes adhere to oral mucosa and prolong drug residence time vs. standard rinses and ointments Enhanced local drug availability translates to more consistent symptom control Minimal Systemic Exposure Liposomal encapsulation slows diffusion, limiting systemic absorption and reducing the risk of systemic toxicity LP - 10 has shown a 98% reduction in systemic exposure vs. oral/IV dosing, with tacrolimus undetectable in 76% of blood samples Fewer Adverse Events LP - 10 has demonstrated a clean safety profile, with no serious adverse events or treatment - related discontinuations By prolonging local adhesion, LP - 10 minimizes systemic absorption and enhances tolerability, supporting improved patient compliance Liposomal delivery offers targeted, sustained local exposure with improved tolerability, positioning LP - 10 as a superior alternative to generic tacrolimus Liposomal Formulation Generic Tacrolimus LP - 10's liposomal formulation delivers enhanced efficacy and a superior safety profile over generic tacrolimus alternatives LP - 10 Delivery Differentiation

![](img005.jpg)

5 Lipella's Strategic Focus is on the Accelerated 505(b)(2) Pathway and Orphan Drug Indications Advantages of the 505(b)(2) pathway Accelerated Development : Enables Phase 2 initiation without Phase 1 by leveraging existing safety data Validated Compounds : Builds on well - characterized compounds with established safety and efficacy profiles CMC and Regulatory Advantage : Potentially reduces risk across manufacturing, safety, and clinical development FDA Flexibility : Enables a more streamlined and collaborative regulatory pathway Cost and Time Efficiency : Lowers development time and costs compared to traditional pathways Targeting Orphan Diseases with No Approved Therapies High Unmet Need : No FDA - approved treatments in target orphan indications Streamlined Development : Smaller, cost - effective trials with FDA flexibility Market Exclusivity : 7 years (U.S.); 10 years (EU & Japan) Reduced Competition : Limits market entry and boosts long - term value

![](img006.jpg)

6 Company Pipeline Proprietary Asset Pre - clinical IND - Enabling Phase 1 Phase 2 Phase 3 LP - 10 (oral rinse) (previously LP - 310) LP - 10 (intravesical) LP - 10 (oral rinse) (previously LP - 410) LP - 50 (intravesical) Oral Lichen Planus (OLP) Hemorrhagic Cystitis (HC) Bladder Cancer Oral GvHD

![](img007.jpg)

7 • Appearance : Lacy, white, raised patches; red, swollen, tender areas; open sores • Who it affects : Women are affected more often than men (2:1 female - to - male ratio), with onset typically occurring between ages 30 - 60 • Location : Found inside cheeks, gums, tongue, lips, and palate • Symptoms : Burning pain, sensitivity to spicy foods, gingivitis, discomfort when chewing or swallowing • Malignancy Potential : Malignant transformation (squamous cell carcinoma) between 0.4 - 5% (annual rate between 0.2 - 0.5%) OLP is a Painful, Autoimmune Disorder of the Oral Cavity Current OLP Treatments: Palliative with Significant Side Effects Recurrent lesions and potential for malignancy highlight a major unmet medical need

![](img008.jpg)

8 Current Treatments Disease Severity Examples Dosage form Use Case Key Limitation Topical Corticosteroids Mild to Moderate Clobetasol, Fluocinolide , dexamethasone rinse Creams, ointments, gels First line, localized symptomatic OLP Local Corticosteroid Use Limited by Safety & Tolerability Issues Oral candidiasis (higher risk with potency or prolonged use) Mucosal thinning/atrophy Delayed healing (impairs tissue regeneration) Tachyphylaxis (reduced effectiveness over time) Topical Immunomodulators Moderate to severe/Refractory Tacrolimus 0.1% ointment Creams, ointments Steroid - refractory cases or long term us Topical tacrolimus: Unpleasant, Systematically Risky Messy and Difficult to apply; Local irritation Unpleasant taste/texture Risk of systemic absorption despite topical use Systemic Corticosteroids For severe widespread OLP Prednisone (short term taper) Oral tabs/capsules, syrups, IV injection For acute flares or extensive erosive OLP Systemic steroids Carry Serious Long - Term Risks Short - term use only due to systemic side effects (e.g., weight gain, glucose intolerance, immunosuppression) Systemic Immunosuppressants Refractory/ Chronic Severe Cyclosporine; Azathioprine; methotrexate Oral tabs/capsules, syrups, IV injection, SQ injection Rarely used; reserved for unresponsive cases High Toxicity Burden Restricts Use of Systemic Immunosuppresants Mucosal thinning/atrophy Delayed healing (impairs tissue regeneration) Tachyphylaxis (reduced effectiveness over time LP - 10 (Oral Rinse) is A Next - Gen Targeted Tacrolimus for OLP Diagnosis and Treatment of Lichen Planus - : Am Fam Physician. 2011;84(1):53 - 60 An effective, easy - to - use therapy engineered to overcome the limitations of topical applications while minimizing systemic absorption

![](img009.jpg)

9 LP - 10 (Oral Rinse) (liposomal - tacrolimus) for Oral Lichen Planus

![](img010.jpg)

10 Tacrolimus reduces T - cell activation in the oral mucosa by inhibiting calcineurin LP - 10 LP - 10 Tacrolimus Has a Well - Established Track Record as a Potent Immunomodulator https:// www.researchgate.net /publication/301645225/figure/fig1/AS:355120102559744@1461678638185/Mechanism - of - action - of - tacrolimus - FK506 - In - the - cytoplasm - tac rolimus - FK506 - binds - to.png LP - 10 (oral rinse) delivers tacrolimus directly to affected tissue, reducing inflammation in OLP and oral GvHD LP - 10 (oral rinse) advancing in rare oral inflammatory diseases: • Ph2b - ready in OLP (T - cell mediated auto - immune) • Ph2 - ready in oral GvHD (Orphan Drug Designation)

![](img011.jpg)

11 LP - 10 (Oral Rinse) is Advancing the Standard of Care in OLP LP - 10 Unique Profile Liposomal tacrolimus formulation with enhanced mucosal penetration and targeted anti - inflammatory effects for oral lichen planus (OLP) and oral Graft - versus - Host Disease (GVHD) Safety Advantage Blood levels consistently below detection limit (<1.0ng/ml) or undetectable at most PK timepoints Mechanism Liposomal formulation enables high local concentration in oral mucosa while limiting systemic exposure Contact Time Avoids systemic toxicities (nephrotoxicity, hypertension) associated with other oral/IV tacrolimus Clinical Benefit Brief 3 - minute rinse (spit out) vs continuous absorption with other routes First Potential FDA - Approved Therapy, moving beyond symptomatic relief

![](img012.jpg)

12 Minimal systemic exposure: Tacrolimus was undetectable (<1.0 ng/mL) in 76% of blood samples (81/106), confirming localized drug delivery. Low measurable levels: Among 27 patients monitored at multiple timepoints, only 25 measurements exceeded 1.0 ng/mL; highest value was 4.5 ng/mL, well below the 15 ng/mL safety threshold. No dose - dependent accumulation : 0.25 mg:(7 instances); max 2.7 ng/mL 0.5 mg: (8 instances); max 2.2 ng/mL 1.0 mg: (10 instances); max 4.5 ng/mL Consistent safety profile: Most measurable levels clustered between 1.0 – 2.0 ng/mL across all dose groups. LP - 10 (Oral rinse) shows 98% Lower Systemic Exposure Compared To Oral or IV Adminstration IV tacrolimus Oral tacrolimus Topical ointment (0.1%) tacrolimus LP - 10 tacrolimus Favorable Systemic PK Profile for LP - 10 (Oral Rinse)

![](img013.jpg)

13 PHASE 2A RESULTS

![](img014.jpg)

14 Patient Demographics Objective: Evaluate safety, tolerability, and preliminary efficacy of LP - 10 oral rinse in patients with symptomatic OLP Design: Multicenter dose - escalation trial Population: Adults with biopsy - confirmed symptomatic OLP Regimen: Twice - daily oral rinse (10 mL) for 4 weeks Endpoints: • Primary: Safety and tolerability • Secondary: Symptom reduction, lesion severity, quality of life (QoL) LP - 10 (Oral Rinse) Ph2a Trial in Adults with Symptomatic OLP Group 1 (0.25mg); N=8 Group 2 (0.50mg); N=8 Group 3 (1.0mg); N=11 Overall (N = 27) Age years (median range) 61.5 (38 - 72) 61.5 (38 - 72) 64.0 (38 – 83) 62.0 (38 – 83) Female [n (%) ] 6 (75.0%) 7 (87.5%) 9 (81.8%) 22 (81.5%) Male [n (%) ] 2 (25.0%) 1 (12.5%) 2 (18.2%) 5 (18.5%) Duration of OLP (median years) 8 (2 – 20) 9.5 (1 – 28) 4.3 (5 – 12) 7 (1 - 28)

![](img015.jpg)

15 Study Overview M ulticenter 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. 27 subjects enrolled at 7 study sites in the U.S. LP - 10 reconstituted for at home oral rinse. The study consists of a Screening Phase, a Treatment Phase, and a Follow - up Phase. Throughout the Treatment Phase, patients will perform a 3 - minute oral rinse using 10 mL of LP - 10 twice daily for a duration of 4 weeks. The Follow - up Phase consists of one visit 2 weeks after the last dose of LP - 10

![](img016.jpg)

16 Validated Clinical / Lesion Severity Scales Validated Symptom / Pain Scales Comprehensive Oral Lichen Planus (OLP) Clinical Endpoints OLP Investigator Global Assessment (IGA)¹ • Investigator - rated assessment of OLP ulceration and erythema • Score range: 0 = clear, 4 = severe Reticulation, Erythema, and Ulceration (REU) Score² • Investigator - assessed evaluation of oral cavity lesions • Measures: Reticulation (white lines), Erythema (redness), Ulceration (ulcers) • Scored by appearance, location, and size • Score range: 0 – 115 1.(Brennan et al., 2023); 2. (Gobbo et al., 2017; Park et al.,2012); 3. (Breivik et al., 2008); 4 (Burke, Brennan, Ni Riordain , & Madsen, 2019) Pain & Sensitivity Numerical Rating Scale (NRS)³ • Patient - reported OLP pain and sensitivity over 24 hours • Rated separately on 0 – 10 scale (0 = none, 10 = worst imaginable) OLP Symptom Severity Measure (OLPSSM #1 – 7)⁴ • Validated, patient - reported OLP symptom questionnaire • Score range: 0=no symptoms at all; 28=max symptom severity Patient Global Response Assessment (GRA) • Single - question patient rating of symptom change vs. baseline • Score range: 0 = markedly worse, 6 = markedly improved Patient - Reported and Investigator - Rated Outcomes

![](img017.jpg)

17 Moderate OLP based on an OLP Investigator Global Assessment (IGA) score of ≥ 3 OLP Pain or Sensitivity Numerical Rating Scale (NRS) score of ≥ 3 Oral biopsy performed within the last 10 years before the Screening Visit demonstrating OLP and/or oral lichenoid mucositis i n t he absence of cancer or dysplasia Patients taking prescription oral steroid or rinse treatment(s) at the time of the Screening Visit agree to stop treatment fo r t he duration of the trial and to undergo a 4 - week washout period History of oral cavity or oropharyngeal cancers Patients who failed tacrolimus treatment for OLP in the past Patient is currently or has previously participated in another oral cavity therapeutic or device study within 3 months of scr een ing and has not returned to baseline Known allergy to liposomes and/or egg yolk and/or tacrolimus Inclusions and Exclusions Key Inclusion Criteria: Key Exclusion Criteria:

![](img018.jpg)

18 \* Not reported means not > 5% incidence LP - 10 (Oral Rinse): Well Tolerated Across All Dose Groups with No Serious AEs or Discontinuation - Related AE's Key Safety Findings Excellent safety profile with NO serious adverse events and minimal systemic absorption (76% undetectable blood tacrolimus level, all TEAE's mild - moderate)

![](img019.jpg)

19 \*p<0.0009, \*\*p<0.0001, significant decline in IGA score from baseline (week 0); Mixed - effects model by Tukey's test. Mean SEM 0 1 4 6 0 2 4 Week O L P I G A 0.25mg 0.5mg 1mg \*\* \*\* \* \* \* \* \*p<0.0001, significant decline in REU score from baseline (week 0); REU score range up to 40; baseline REU subtracted from REU post - treatment; Mean SEM Key Clinical Responses: IGA 1 & REU 2 in OLP 1) IGA: (Investigator Global Assessment); 2) REU (Reticulation, Erythema, and Ulceration Score Significant IGA Improvement with Meaningful Reduction in OLP Ulceration and Erythema Marked Reduction in REU Scores Demonstrates Strong Clinical Response

![](img020.jpg)

20 Patient Reported Pain Reduction (0 - 10 Scale) Patient Reported Sensitivity Reduction (0 - 10 Scale) 0 1 4 6 0 2 4 6 8 Week P a i n s c o r e 0.25mg 0.5mg 1mg \* \*p<0.0001,significant decline in pain score (0 - 10) from baseline (week 0); repeated measures ANOVA (Mixed - effects model) followed by Tukey's test. Mean SEM \* \* 0 1 4 6 0 2 4 6 8 Week S e n s i t i v i t y 0.25mg 0.5mg 1mg 20 \* \*\* \*\* \*p<0.001, \*\*p<0.0001, Significant decline in sensitivity score (0 - 10) from baseline (week 0); repeated measures ANOVA (Mixed - effects model) followed by Tukey's test. Mean SEM. LP - 10 (Rinse) in OLP: Statistically Significant Pain & Sensitivity Improvement

![](img021.jpg)

21 0 1 4 6 0 5 10 15 Week O L P S S M # 1 - 7 0.25mg 0.5mg 1mg \* \*\* \*

![](img022.jpg)

22 Very much better No Change Very much worse As compared to when you started the study, how would you rate your overall quality of life now? 6= Very much better 5= Moderately better 4= A little better 3=No change 2= A little worse 1= Moderately worse 0= Very much worse 0 . 2 5 m g 0 . 5 m g 1 m g 0 1 2 3 4 5 6 G R A All Patients Showed Clinicall y Meaningful Improvement in Global Response Assessment (GRA)

![](img023.jpg)

23 Clinician & Patient Voices Validate LP - 10's Impact Testimonials from Healthcare professionals and patients Clinician Voices Most significant advancement in OLP treatment in my career." - Alessandro Villa, DDS, PhD, MPH, Miami Cancer Institute "Liposomal delivery is a genuine breakthrough for oral mucosal disease." — Hervé Sroussi , DMD, PhD, Brigham & Women's / Harvard "LP - 10 offers superior efficacy with rapid onset and minimal systemic absorption." — Kamal Al - Eryani , DDS, PhD, UCSF School of Dentistry "Dramatic improvements in pain, function, and outcomes." — Michael Brennan, DDS, MHS, Wake Forest / Atrium Health "After years of suffering, LP - 10 gave me complete relief within 4 weeks." — Joan G. Pfeiffer, Clinical Trial Participant "LP - 10 was the only therapy that relieved my pain without intolerable side effects." — Clinical Trial Patient RN "As both a physician and patient, I can attest LP - 10 provided remarkable improvement in quality of life." — Clinical Trial Patient MD Patient Voices " "

![](img024.jpg)

24 LP - 10 (Oral Rinse) Investment Highlights

![](img025.jpg)

25 Patient Population Dynamics LP - 10 Addressable Patient Population Accounting for a modest diagnosis rate, symptomatic disease, and patients inadequately controlled on frontline corticosteroid s, the peak addressable population for LP - 10 is projected to be ~1.2 M Sources: LifeSci Consulting Primary Market Research (N=15 U.S. HCPs, 5 U.S. Payers); UpToDate; American Academy of Oral Medic ine ; Eisen et al., 2002; Raj et al., 2023 Total OLP Prevalence Diagnosed Prevalence Symptomatic Patients Poorly Controlled Patients ~6.2 M ~ 4.0 M ~ 3.2 M ~ 1.2 M LP - 10 Addressable Patients (2034) OLP Prevalence: ~6.2 M OLP is estimated to affect ~2% of U.S. adults; projected to grow modestly (~2% CAGR) through 2034 Diagnosed Prevalence: ~4.0 M While currently limited by access to care, low awareness in dentistry / primary care, and asymptomatic presentation, physicians anticipate diagnosis rates will grow significantly upon approval of a novel therapy Symptomatic Patients: ~3.2 M ~80% of OLP cases are symptomatic, driven by erythematous and erosive subtypes Poorly Controlled Patients: ~1.2 M Physicians report that ~90% of diagnosed symptomatic patients receive treatment; however, ~40% remain uncontrolled on first - line corticosteroids ~1.2 M LP - 10 Addressable Patients (2034)

![](img026.jpg)

26 Physician Reported Unmet Needs and LP - 10 Opportunity First FDA Approved Therapy Improved Awareness Safe Chronic Management • Topical steroids are limited to short courses; systemic use restricted to flares • Immunosuppressants control refractory disease but require monitoring, limiting routine use Lack of FDA - Approved Therapies • OLP is treated strictly off - label; high remaining need for approved therapies • Topical gels/ointments are unsuitable intraorally, raising tolerability concerns Oral - Specific Formulations • Patients often prefer rinses; gels/ointments are poorly tolerated, reducing adherence • Cost and access to compounded tacrolimus rinses drives home self - mixing Well Tolerated Minimal Systemic Absorption Easy to Use Rinse Greater Oral Cavity Coverage Sources: LifeSci Consulting Primary Market Research (N=15 U.S. HCPs, 5 U.S. Payers) Unmet Needs LP - 10 Value Drivers Physician - Reported Utilization High Low ~70% in 2L+ By addressing critical unmet needs in OLP care, LP - 10 is anticipated to achieve significant utilization in the 2L+ setting

![](img027.jpg)

27 LP - 10 Pricing and Access Dynamics Sources: LifeSci Consulting Primary Market Research (N=15 U.S. HCPs, 5 U.S. Payers) Payer Monthly WAC Price Expectations for LP - 10 $200 $800 $1,000 $1,500 $2,000 $3,000 $500 Payers cite ~$1 K as the most reasonable benchmark for LP - 10, though standard branded agent utilization management is expected at all price points due to broad availability of low - cost generics Minimal Active Management Discounting / Rebating BASE CASE: Payers report a monthly WAC of ~$1 K as the most reasonable benchmark for LP - 10 to achieve coverage, though prior authorization and generic step requirements are expected PREMIUM PRICING: A monthly WAC of $1.5 – 2 K was cited by payers as the maximum price LP - 10 could achieve without facing formulary exclusion although aggressive discounting and rebating will be required to maintain access COMPETITIVE PRICING: While rebating and discounting may be less extensive at lower price points, payers still expect utilization management given the availability of low - cost generic alternatives LP - 10 has limited pricing flexibility due to the entrenched use of inexpensive generics; coverage will depend on demonstrated clinical differentiation (e.g., durability, steroid - sparing effects) and competitive contracting terms 3 3 1 2 1 2

![](img028.jpg)

28 Experienced Team Strategic Manufacturing Capabilities Investment Highlights Focused on rare and orphan indications, leveraging the 505(b)(2) pathway to reduce costs and accelerate time to approval. Liposomal tacrolimus (LP - 10) • (LP - 10 Oral - rinse) for Oral Lichen Planus (OLP): Positive Ph2a across all doses • (LP - 10 Oral - rinse) for Oral GvHD: Phase 2 ready • (LP - 10 Intravesical) treatment for Hemorrhagic Cystitis (HC): Phase 2b ready Executive team, board of directors, and scientific advisory board all deeply entrenched in the biotech space Pittsburgh, PA facility supports liposomal R&D and clinical supply; commercial - scale manufacturing via Cook MyoSite partnership LP - 10 (Oral Rinse) advancing toward Phase 2b IND submission Pipeline strategy Advanced Pipeline Near Term Catalysts

![](img029.jpg)

29 Thank you!! lipellapharmaceuticals.com