LIPO Lipella Pharmaceuticals Inc

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 DevelopmentWith 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-310LP-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.

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CONTACTJonathan KaufmanChief Executive OfficerLipella Pharmaceuticals Inc.Info@Lipella.com1-412-894-1853

PCG AdvisoryJeff Ramsonjramson@pcgadvisory.com