Lipella Reports Positive Phase 2a Results from Second LP-310 Cohort in Oral Lichen Planus; Final Data Expected Q2 2025

Rhea-AI Summary

Lipella Pharmaceuticals (NASDAQ: LIPO) announced positive topline results from the second cohort (0.50 mg) of its Phase 2a trial for LP-310, a liposomal-tacrolimus oral rinse for oral lichen planus (OLP) treatment. The trial demonstrated statistically significant improvements across multiple endpoints:

Key findings include:

• Investigator Global Assessment improved from 3.42 to 1.71 at week 4 (p=0.007)
• REU Score reduced from 26.91 to 11.88 at week 4 (p=0.003)
• Pain rating decreased from 6.42 to 2.25 at week 4 (p=0.003)

The treatment showed strong safety profile with no treatment-related SAEs and complete patient adherence. The trial is now fully enrolled across all three dose cohorts, with final topline data from the 1.0 mg cohort expected in Q2 2025. The company plans to submit an IND application for Phase 2b trial in late 2025.

Positive

• Statistically significant efficacy improvements across multiple clinical measures
• Strong safety profile with no treatment-related SAEs
• 100% patient adherence to treatment protocol
• Minimal systemic absorption of tacrolimus
• Full enrollment achieved across all three dose cohorts

Negative

• Efficacy measures showed some decline at week 6 compared to week 4
• Phase 2b trial IND submission not planned until late 2025

Insights

Biotech/Pharmaceutical Development Expert

Lipella's Phase 2a data shows statistically significant efficacy for LP-310 in oral lichen planus across multiple endpoints, with favorable safety profile and advancing regulatory pathway.

The positive Phase 2a results for Lipella's LP-310 represent a significant clinical milestone in the development of this novel oral lichen planus (OLP) therapy. The 0.50 mg cohort data demonstrated robust statistically significant improvements across all key efficacy measures, with particularly strong p-values (p=0.003) for multiple endpoints at week 4 of treatment.

The efficacy signal is compelling and consistent across both objective measurements and patient-reported outcomes. The demonstrated durability pattern - with maximal effect at week 4 followed by partial regression at week 6 (post-treatment) - confirms both drug activity and aligns with expectations for a chronic condition requiring maintenance therapy.

From a development perspective, LP-310's liposomal-tacrolimus formulation represents an elegant solution to the challenge of local drug delivery. The pharmacokinetic analysis confirming "undetectable or minimal" tacrolimus levels validates the targeted delivery mechanism, allowing therapeutic effect without systemic immunosuppression - a critical advantage for chronic use.

With the trial now fully enrolled across all three dose cohorts, Lipella has successfully navigated a key operational milestone. The company's regulatory strategy appears well-defined, with plans for Phase 2b IND submission and pursuit of Breakthrough Therapy designation, which could potentially expedite development for this indication with no FDA-approved treatments.

The final topline results from the highest dose cohort (1.0 mg) expected in Q2 2025 will be particularly informative for dose selection and could further strengthen the overall data package as Lipella advances toward late-stage development.

Medical Expert

LP-310 demonstrates clinically meaningful improvements in OLP symptoms and lesions with non-steroidal approach and minimal systemic absorption.

The Phase 2a results for LP-310 reveal clinically meaningful efficacy in treating oral lichen planus, a painful inflammatory condition with no FDA-approved treatments. The data shows substantial improvements across multiple validated assessment tools used to evaluate OLP severity.

The 56% reduction in REU scores (from 26.91 to 11.88, p=0.003) at week 4 represents significant improvement in the visible manifestations of disease - reticulation (white lacy patterns), erythema (redness), and ulceration. Simultaneously, the 65% decrease in pain scores (from 6.42 to 2.25, p=0.003) addresses the most debilitating symptom for patients with this condition.

Current OLP management relies heavily on topical corticosteroids, which have limitations including mucosal atrophy with prolonged use. LP-310's non-steroidal, liposomal tacrolimus formulation offers a mechanistically distinct approach that could provide safer long-term management for this chronic condition.

The regression of benefit observed at week 6 (post-treatment phase) confirms both the drug's activity and the expected need for maintenance therapy in this chronic inflammatory condition. The twice-daily oral rinse administration appears well-tolerated, with perfect adherence and no dropouts - critical factors for real-world effectiveness in a chronic condition.

Most importantly, the minimal systemic absorption demonstrated through pharmacokinetic analysis suggests LP-310 could deliver local immunomodulatory benefits while avoiding the serious systemic side effects associated with conventional tacrolimus - a considerable advantage for a therapy that may require long-term use.

Second Cohort Results Highlight Efficacy Across All Key Measures and Reinforce Safety of Twice-Daily Oral Rinse

Phase 2a Study Now Fully Enrolled Across All Three Dose Cohorts

Advancing Toward Phase 2b IND Submission and Broader Regulatory Engagement

PITTSBURGH, April 22, 2025 (GLOBE NEWSWIRE) -- Lipella Pharmaceuticals Inc. (Nasdaq: LIPO) (“Lipella,” “our,” “us,” or the “Company”), a clinical-stage biotechnology company addressing serious diseases with significant unmet needs, today announced positive topline results from the second cohort (0.50 mg) of its Phase 2a multicenter, dose-ranging trial evaluating LP-310, a liposomal-tacrolimus oral rinse formulation of LP-10, for the treatment of oral lichen planus (OLP). The Company also recently reported full enrollment across all three planned dose cohorts.

Treatment with LP-310 at the 0.50 mg dose demonstrated statistically significant improvements in multiple patient-reported and investigator-measured efficacy endpoints, reinforcing LP-310’s potential as a non-steroidal, locally delivered therapy for OLP. The trial, currently active across seven U.S. study sites, has now progressed to the third and highest dose cohort (1.0 mg/10 mL), with topline results expected in the second quarter of 2025.

“These results mark meaningful progress for LP-310 and further strengthen our confidence in its clinical and commercial potential,” said Jonathan Kaufman, Co-Founder and Chief Executive Officer of Lipella Pharmaceuticals. “With the Phase 2a trial now fully enrolled and final data expected this quarter, we are advancing toward key value-driving milestones, including a Phase 2b IND submission and broader regulatory engagement to support the program’s continued development.”

Topline Findings from the 0.50 mg Cohort:

• Investigator Global Assessment (IGA): Improved from 3.42 ± 0.21 at baseline to 2.71 ± 0.30 at week 1 (p=0.029), 1.71 ± 0.43 at week 4 (p=0.007) and 2.75 ± 0.34 at week 6 (p=0.112).
• Reticulation, Erythema and Ulceration (REU) Score: Reduced from 26.91 ± 2.54 at baseline to 17.02 ± 2.36 at week 1 (p=0.003), 11.88 ± 2.91 at week 4 (p=0.003) and 18.47 ± 4.12 at week 6 (p=0.028).
• Oral Lichen Planus Symptom Severity Measure (OLPSSM): Decreased from 14.92 ± 2.10 at baseline to 9.87 ± 2.27 at week 1 (p=0.032), 4.88 ± 2.15 at week 4 (p=0.003) and 8.42 ± 3.98 at week 6 (p=0.028).
• Pain Numerical Rating Scale (NRS): Improved from 6.42 ± 0.75 at baseline to 4.25 ± 0.89 at week 1 (p=0.003), 2.25 ± 1.09 at week 4 (p=0.003) and 3.52 ± 1.51 at week 6 (p=0.028).
• Global Response Assessment (GRA): Significant improvement was observed at week 4 (p=0.028).
  

Safety and Tolerability

LP-310 continues to be well tolerated, with no treatment-related SAEs and no patient dropouts. All participants successfully adhered to the twice-daily 10-milliliter rinse regimen. Pharmacokinetic analysis confirmed that tacrolimus levels remained undetectable or minimal in all patients, reinforcing LP-310’s ability to deliver localized benefits without systemic toxicity.

“We’re very encouraged by the positive data from the 0.50 mg cohort, which demonstrated statistically significant efficacy across multiple clinical measures while maintaining a strong safety profile,” said Dr. Michael Chancellor, Co-Founder and Chief Medical Officer of Lipella Pharmaceuticals. “Oral lichen planus is a painful, chronic condition with no FDA-approved treatment and limited therapeutic options. The symptom relief reported by patients, along with reductions in inflammation and ulceration, highlights LP-310’s potential to be a transformative, steroid-free, and easy-to-use therapy that addresses a longstanding clinical unmet need and improves quality of life.”

Next Steps in Development
With enrollment now complete across all three dose cohorts, Lipella is positioned to deliver final topline data from the 1.0 mg cohort in Q2 2025. These data are expected to form the foundation for upcoming regulatory interactions and next-phase clinical advancement. Key development milestones include:

• Reporting final phase 2a topline results in Q2 2025
• Advancing discussions with regulatory agencies including submission of an Investigational New Drug (IND) application for a Phase 2b trial in late 2025
• Preparing to pursue FDA Breakthrough Therapy designation request
  

About LP-310
LP-310 is an innovative oral rinse formulation of LP-10 (tacrolimus), developed to address OLP. Designed to provide localized therapeutic effects while minimizing systemic exposure, LP-310 offers a promising new approach to managing this painful and often debilitating condition.

A Phase 2a multicenter, dose-ranging clinical trial is currently underway to evaluate the safety, tolerability and efficacy of LP-310 in adult participants with symptomatic OLP. The trial includes three dose levels (0.25 mg, 0.5 mg and 1.0 mg of tacrolimus) and is being conducted across seven active U.S. sites, which are now recruiting participants. Lipella has reported topline data from the first two cohorts (0.25 mg and 0.50 mg), with the final cohort (1.0 mg) currently enrolling. Topline results from the 1.0 mg cohort are expected in the first half of 2025.

For more information about the study or to participate, visit https://lipella.com/oral-lichen-planus-treatment/ or https://clinicaltrials.gov/study/NCT06233591.

About Lipella Pharmaceuticals Inc.

Lipella Pharmaceuticals is a clinical-stage biotechnology company focused on developing new drugs by reformulating active agents in existing generic drugs and optimizing these reformulations for new applications. Lipella targets diseases with significant unmet needs, where no approved drug therapies currently exist. The company completed its initial public offering in 2022. Learn more at lipella.com and follow us on X and LinkedIn.

Forward-Looking Statements
This press release includes certain "forward-looking statements" which are not historical facts, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. 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. 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, the current clinical trial results for LP-310 and our other products general capital market risks, our ability to regain and maintain compliance with the listing standards of The Nasdaq Stock Market LLC, regional, national or global political, economic, business, competitive, market and regulatory conditions, our current liquidity position and the need to obtain additional financing to support ongoing operations, and other risks as more fully described in our filings with the U.S. Securities and Exchange Commission. 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. In addition, the information contained in this press release 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 press release as legal, tax or investment advice and should consult with your own advisors as to the matters described herein, as applicable.

CONTACT
Jonathan Kaufman
Chief Executive Officer
Lipella Pharmaceuticals Inc.
Info@Lipella.com
1-412-894-1853

PCG Advisory
Jeff Ramson
jramson@pcgadvisory.com

FAQ

What were the key efficacy results of LIPO's LP-310 Phase 2a trial second cohort?

The 0.50 mg dose showed significant improvements in multiple endpoints, including REU Score reduction from 26.91 to 11.88 at week 4, pain rating decrease from 6.42 to 2.25, and Investigator Global Assessment improvement from 3.42 to 1.71.

When will Lipella (LIPO) release final Phase 2a results for LP-310?

Lipella expects to release final topline data from the 1.0 mg cohort in Q2 2025.

What is the safety profile of LIPO's LP-310 in the Phase 2a trial?

LP-310 demonstrated strong safety with no treatment-related SAEs, no patient dropouts, and minimal to undetectable tacrolimus levels, confirming localized benefits without systemic toxicity.

What are LIPO's next development milestones for LP-310?

Lipella plans to report final Phase 2a results in Q2 2025, submit an IND application for Phase 2b trial in late 2025, and pursue FDA Breakthrough Therapy designation.