Breakthrough in oral lichen planus? Lipella Pharmaceuticals’ LP-310 delivers promising results

Lipella Pharmaceuticals has announced encouraging topline results from its Phase 2a dose-ranging trial for LP-310, a liposomal-tacrolimus oral rinse formulation designed to treat oral lichen planus. Following the news, the company’s shares surged by 37% to $4.18, reflecting investor confidence in the potential of this novel therapy. The trial’s outcomes not only underscore LP-310’s promising efficacy but also its strong safety profile, marking a significant milestone for Lipella Pharmaceuticals as it progresses through clinical development.

What is oral lichen planus, and why is LP-310 a potential breakthrough?

Oral lichen planus is a chronic inflammatory condition affecting the mucous membranes of the mouth, including the tongue, cheeks, gums, and palate. The disease manifests through painful symptoms such as burning sensations, white patches, ulcerations, and swollen tissues. This can significantly impact a patient’s quality of life, making simple activities like eating, drinking, and speaking difficult. Despite affecting around six million Americans, there are currently no FDA-approved treatments for oral lichen planus, leaving patients with limited options.

This gap in therapeutic solutions highlights the potential importance of LP-310 treatment results. As an innovative oral rinse formulation of LP-10 (tacrolimus), LP-310 is designed to deliver localized treatment while minimizing systemic exposure. Its mechanism of action targets inflammation directly at the site of the disease, offering a novel approach in a field where effective therapies are scarce.

How did LP-310 perform in clinical trials?

Lipella Pharmaceuticals’ Phase 2a trial evaluated LP-310’s safety, tolerability, and efficacy in adult patients with symptomatic oral lichen planus. Conducted across seven sites in the United States, the trial enrolled participants who received a twice-daily oral rinse at a dose of 0.25 mg/10 mL. The study’s findings were significant, showcasing improvements across multiple patient-reported and investigator-measured efficacy endpoints.

The LP-310 treatment results demonstrated statistically significant reductions in key symptoms such as pain, ulceration, and inflammation. By week four, patients reported noticeable improvements in their condition, with sustained benefits observed through week six. Importantly, no product-related serious adverse events were reported, and no patients withdrew from the study, reinforcing LP-310’s favorable safety profile.

Dr. Michael Chancellor, Co-Founder and Chief Medical Officer of Lipella Pharmaceuticals, emphasized the significance of these findings, stating that the trial provides strong evidence of LP-310’s therapeutic potential. “The reductions in pain and inflammation observed in this study validate LP-310’s efficacy while highlighting its excellent safety profile. This is an important step forward in addressing an unmet medical need for patients with oral lichen planus,” Dr. Chancellor said.

What do the trial results mean for the future of LP-310?

The Phase 2a trial’s success paves the way for further development. Lipella Pharmaceuticals has already advanced the study to the next dose cohort, increasing the concentration to 0.5 mg/10 mL. Recruitment remains ongoing, with the company planning to activate additional clinical sites to support trial expansion. The company aims to complete the trial by mid-2025, with data from higher dose cohorts expected in the first half of that year.

Jonathan Kaufman, Co-Founder and CEO of Lipella Pharmaceuticals, highlighted the broader implications of the trial. “Oral lichen planus represents a significant unmet need within the healthcare system. Our goal is to deliver an effective, targeted therapy that addresses the challenges faced by patients living with this chronic condition. The positive Phase 2a results are highly encouraging and underscore LP-310’s potential to transform the treatment landscape,” Kaufman stated.

In addition to continuing patient recruitment, Lipella Pharmaceuticals is preparing key regulatory submissions. The company plans to file an investigational new drug application for a Phase 2b clinical trial in the second half of 2025. Additionally, Lipella will seek Breakthrough Therapy Designation from the U.S. Food and Drug Administration, which could expedite the development and review process, given the significant unmet medical need associated with oral lichen planus.

What makes LP-310 different from other treatments?

Unlike conventional therapies that often rely on systemic immunosuppressants or corticosteroids with potential side effects, LP-310 offers a localized treatment approach. Its liposomal-tacrolimus formulation is designed to act directly on affected oral tissues, minimizing systemic absorption and reducing the risk of adverse effects commonly associated with systemic drugs.

Pharmacokinetic analysis from the trial confirmed that tacrolimus blood levels were undetectable or minimal in all participants, further supporting LP-310’s localized activity. This feature is particularly valuable for chronic conditions like oral lichen planus, where long-term treatment may be necessary.

Lipella Pharmaceuticals’ strategy of reformulating active agents from existing generic drugs, such as tacrolimus, represents a cost-effective and scientifically robust approach. By optimizing these reformulations for new applications, the company aims to address diseases with significant unmet medical needs, including conditions like oral lichen planus, where treatment options are limited.

What’s next for Lipella Pharmaceuticals and LP-310?

Lipella Pharmaceuticals is scheduled to present the LP-310 treatment results at the BIO CEO & Investor Conference on February 11, 2025, in New York. This presentation will provide additional insights into the company’s development strategy and clinical progress. It also offers an opportunity to engage with potential investors and healthcare professionals interested in the future of oral lichen planus treatment.

Looking ahead, Lipella plans to continue expanding its clinical program for LP-310, with a focus on completing the Phase 2a trial, initiating Phase 2b studies, and pursuing regulatory milestones. The company’s efforts could lead to the first FDA-approved therapy specifically for oral lichen planus, potentially transforming the standard of care for millions of affected individuals.

As the trial progresses, the medical community will closely watch how LP-310 continues to perform. If subsequent studies confirm the promising results observed so far, Lipella Pharmaceuticals may be on the brink of delivering a much-needed breakthrough for patients suffering from this debilitating condition.