Lipella Pharmaceuticals Inc. (Pittsburgh, PA, USA) has announced promising topline results from the initial cohort of a phase IIa trial evaluating LP-310, a novel oral rinse formulation of liposomal tacrolimus (LP-10), for the treatment of oral lichen planus (OLP).¹

Oral lichen planus is a chronic immune-mediated inflammatory condition affecting approximately 1% of the global population.² The condition targets the oral mucosa, causing intermittent flares and lesions that can present in various forms, including reticular, papular, plaque-like, atrophic, erosive and bullous OLP.^3-6 While some individuals with OLP remain asymptomatic, others can experience a range of symptoms including chronic pain, ulcers, and difficulty eating and speaking.

Current treatment options for symptomatic oral lichen planus flares primarily include topical corticosteroids as the first-line therapy. Second- and third-line treatments range from topical calcineurin inhibitors to systemic corticosteroids, systemic retinoids and topical or systemic immunomodulators.⁵

LP-310 aims to provide a more localized therapeutic effect, with the hope that this will potentially reduce the systemic side effects often associated with existing treatments, offering a new alternative for patients with OLP.¹

Study design

The phase IIa study (A Multicenter, Dose-Ranging Trial Evaluating the Safety, Tolerability, and Efficacy of LP-10 in Subjects With Symptomatic Oral Lichen Planus; ClinicalTrials.gov identifier: NCT06233591) is a multicenter, dose-ranging trial designed to assess the safety, tolerability and efficacy of LP-310 in adults with symptomatic OLP.⁷ The trial includes three dose levels of tacrolimus: 0.25 mg, 0.5 mg and 1.0 mg.¹

In the initial cohort, eight patients received a 0.25 mg/10 mL LP-310 oral rinse twice daily, with follow-up assessments conducted at weeks 1 and 4, plus a two-week post-treatment follow-up. The efficacy of the treatment was measured using a range of assessment scores to evaluate pain, ulceration, inflammation and overall disease severity. The trial is being conducted across seven sites in the USA.¹

Key findings

These topline results from the first cohort showed significant improvements across multiple endpoints, with patients reporting marked reductions in pain, inflammation and overall disease severity.¹

• 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).

Safety and tolerability

The analysis also reported an encouraging safety profile for LP-310. In this small cohort, no product-related serious adverse events were reported and all patients adhered to the twice-daily 10 mL rinse regimen.¹ Pharmacokinetic analysis showed undetectable or minimal systemic absorption of tacrolimus, suggesting that LP-310 could provide a localized therapeutic action.¹

Next steps

“The statistically significant reductions in pain, ulceration and inflammation observed in this trial provide strong scientific validation of LP-310’s efficacy,” said Dr Michael Chancellor, Co-Founder and Chief Medical Officer of Lipella Pharmaceuticals. “These results, along with the treatment’s favourable safety profile, highlight LP-310’s potential to offer a highly targeted and well-tolerated therapy for OLP”.¹

The study is now progressing to higher doses of 0.5 mg/10 mL and 1.0 mg/10 mL, with full recruitment expected by mid-2025. Lipella plans to submit a phase IIb investigational new drug (IND) application and apply for FDA Breakthrough Therapy designation in late 2025.

References

1. GlobalNewswire. Lipella Pharmaceuticals Reports Positive Topline Phase 2a Results for LP-310 in the Treatment of Oral Lichen Planus. Press release. Available at: www.globenewswire.com/news-release/2025/02/11/3023967/0/en/Lipella-Pharmaceuticals-Reports-Positive-Topline-Phase-2a-Results-for-LP-310-in-the-Treatment-of-Oral-Lichen-Planus.html (accessed 17 February 2025).
2. González-Moles MÁ, Warnakulasuriya S, González-Ruiz I, et al. Worldwide prevalence of oral lichen planus: A systematic review and meta-analysis. Oral Dis. 2021;27:813–28. doi: 10.1111/odi.13323.
3. Nukaly HY, Halawani IR, et al. Oral lichen planus: A narrative review navigating etiologies, clinical manifestations, diagnostics, and therapeutic approaches. J Clin Med. 2024;13:5280. doi: 10.3390/jcm13175280.
4. Sugerman PB, Savage NW, Zhou X, et al. Oral lichen planus. Clin Dermatol. 2000;18:533–9. doi: 10.1016/s0738-081x(00)00142-5.
5. Louisy A, Humbert E, Samimi M. Oral lichen planus: An update on diagnosis and management. Am J Clin Dermatol. 2024;25:35–53. doi: 10.1007/s40257-023-00814-3.
6. Hamour AF, Klieb H, Eskander A. Oral lichen planus. CMAJ. 2020;192:E892. doi: 10.1503/cmaj.200309.
7. ClinicalTrials.gov. Evaluating LP-10 in Subjects With OLP. ClinicalTrials.gov identifier: NCT06233591. Available at: https://clinicaltrials.gov/study/NCT06233591 (accessed18 February 2025).

 

Disclosures: This article was created by the touchDERMATOLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.

Editors: Katey Gabrysch, Editorial Director and Gina Furnival, Senior Editorial Director

Support: No funding was received in the publication of this short article.

Cite: New Data in Oral Lichen Planus Shows LP-310 Oral Tacrolimus Reduces Pain, Ulceration, and Inflammation. touchDERMATOLOGY. February 18, 2025

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