Lipella Pharmaceuticals has announced positive topline results from its Phase 2a multicenter dose-ranging trial of LP-310, a liposomal-tacrolimus oral rinse being developed for the treatment of oral lichen planus (OLP). The trial demonstrated statistically significant improvements across multiple efficacy endpoints and a strong safety profile, marking a potential advancement in OLP therapy.
Efficacy and Safety Results
The Phase 2a trial evaluated LP-310 in adult participants with symptomatic OLP. Key findings include:
- Investigator Global Assessment (IGA): Improved from 3.50 ± 0.19 at baseline to 2.75 ± 0.31 at week 1 (p=0.031), 1.75 ± 0.45 at week 4 (p=0.008), and 2.80 ± 0.37 at week 6 (p=0.125).
- Reticulation, Erythema, and Ulceration (REU) Score: Reduced from 27.75 ± 2.71 at 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 week 6 (p=0.031).
- Oral Lichen Planus Symptom Severity Measure (OLPSSM): Decreased from 15.38 ± 2.20 at 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 week 6 (p=0.031).
- Pain Numerical Rating Scale (NRS): Improved from 6.63 ± 0.80 at baseline to 4.38 ± 0.96 at week 1 (p=0.004), 2.38 ± 1.15 at week 4 (p=0.004), and 3.60 ± 1.63 at week 6 (p=0.031).
- Global Response Assessment (GRA): Significant improvement was observed at week 4 (p=0.031).
Notably, the trial reported no product-related serious adverse events, and all patients adhered to the twice-daily 10 mL rinse regimen without any dropouts. Pharmacokinetic analysis showed undetectable or minimal tacrolimus blood levels, suggesting localized benefits without systemic toxicity. Eight participants received a twice-daily dose of 0.25 mg of LP-310 in the first cohort.
Expert Commentary
"The statistically significant reductions in pain, ulceration, and inflammation observed in this trial provide strong grounds for the scientific validation of LP-310’s efficacy," said Dr. Michael Chancellor, Co-Founder and Chief Medical Officer of Lipella Pharmaceuticals. He added, "These results, combined with the treatment’s favorable safety profile, highlight the potential of LP-310 to deliver a highly targeted and tolerable therapy for OLP."
The Need for New OLP Therapies
OLP is a chronic inflammatory condition affecting the mucous membranes inside the mouth, causing pain and discomfort during eating, drinking, and speaking. Characterized by symptoms like burning pain, white patches, swollen tissue, and open sores, OLP affects approximately 6 million Americans. Currently, there are no FDA-approved therapies specifically for OLP, highlighting a significant unmet medical need.
Future Development Plans
Lipella is actively recruiting for the trial, with plans to activate additional sites and complete the study by mid-2025. The company is preparing to submit a Phase 2b clinical trial investigational new drug application and request Breakthrough Therapy designation from the FDA in the second half of 2025. The second cohort of the Phase 2a study is currently dosing, with topline data expected in the first half of 2025.
About LP-310
LP-310 is an oral rinse formulation of LP-10 (tacrolimus), designed to provide localized therapeutic effects while minimizing systemic exposure. It represents a promising new approach to managing OLP, offering potential relief for patients suffering from this debilitating condition.
