Lipella Pharmaceuticals has successfully completed subject enrollment in its multicenter Phase IIa clinical trial evaluating LP-310, a liposomal tacrolimus oral rinse, for the treatment of oral lichen planus (OLP). The dose-ranging study has enrolled patients across three tacrolimus dose cohorts (0.25mg, 0.50mg, and 1.0mg) at seven clinical trial sites throughout the United States.
The trial is designed to assess the tolerability, preliminary efficacy, and safety profile of LP-310 in adult patients with symptomatic OLP. Study participants receive the treatment as a 10ml oral rinse administered twice daily over a four-week period.
Dr. Michael Chancellor, Chief Medical Officer and co-founder of Lipella Pharmaceuticals, highlighted the significance of this milestone: "The completion of enrollment in our Phase IIa trial marks an important step in advancing a non-steroidal, locally delivered treatment for oral lichen planus, an underserved condition with no FDA-approved therapies."
Promising Early Results
According to Lipella, preliminary data from the first two dose cohorts has already demonstrated encouraging safety and efficacy signals. Dr. Chancellor noted, "We have already observed encouraging safety and efficacy signals in the first two dose cohorts, including clinically meaningful reductions in pain, ulceration, and inflammation."
The trial's primary objectives focus on evaluating the pharmacokinetics and safety profile of LP-310. Secondary endpoints assess the treatment's impact on inflammation, oral ulceration, and pain through both patient-reported outcomes and investigator-measured scoring systems.
Topline results from the final cohort, which is receiving the highest dose of 1.0mg tacrolimus, are expected in the second quarter of 2024. This data will be crucial for determining the next steps in the development program and broader clinical strategy for LP-310.
Development Timeline and Regulatory Strategy
With enrollment now complete, Lipella is planning to submit an Investigational New Drug (IND) application for a Phase IIb trial in late 2025. The company is also exploring potential regulatory designations, including breakthrough therapy status, which could accelerate the development and review process.
Understanding Oral Lichen Planus
Oral lichen planus is a T-cell-mediated autoimmune condition that causes painful erosions and ulcerative lesions in the oral mucosa. The condition significantly impacts patients' quality of life, affecting their ability to eat, drink, and speak comfortably.
Currently, there are no FDA-approved treatments specifically for OLP, highlighting the significant unmet medical need that Lipella's LP-310 aims to address. Standard treatments often include topical corticosteroids, which can have limitations in terms of efficacy and side effects with prolonged use.
LP-310: A Novel Approach
LP-310 represents a non-steroidal, locally delivered approach to treating OLP. The liposomal formulation of tacrolimus is designed to enhance drug delivery to the affected oral tissues while potentially minimizing systemic absorption and associated side effects.
Tacrolimus, the active ingredient in LP-310, is an established immunosuppressive agent that inhibits T-cell activation and proliferation, which are key pathological processes in OLP. The oral rinse formulation allows for direct application to affected areas, potentially improving efficacy while reducing systemic exposure.
If successful in later-stage trials, LP-310 could become the first FDA-approved treatment specifically for oral lichen planus, offering new hope to patients suffering from this challenging condition.
