Stock to Watch: Lipella Pharmaceuticals Builds Value with Broad Pipeline of Promising Orphan Drugs

Clinical-stage biotechnology company, Lipella Pharmaceuticals (Nasdaq: LIPO) is building sustainable value for investors and setting itself apart through its strategic targeting of attractive addressable patient populations, a broad pipeline of product candidates, Orphan Drug endorsement from the FDA, and in-house manufacturing capabilities.

A PRISM MarketView Stock to Watch in 2024, Lipella has achieved a number of transformative milestones in the past six months, including beginning patient recruitment for a Phase 2a trial evaluating LP-310 in the treatment of Oral Lichen Planus (OLP). Dr. Jonathan Kaufman, CEO of Lipella, expressed his enthusiasm, stating, “his milestone brings us one step closer to providing a potential treatment for the estimated 6 million Americans affected by OLP, a condition that causes significant pain and has no approved therapies.”

Lipella’s near term catalysts are significant. The company recently completed a successful Type C meeting with the FDA to discuss its Phase 2b clinical trial design for a study of its lead candidate, LP-10, an intravesical liposomal formulation of tacrolimus, in hemorrhagic cystitis. Lipella’s Chief Medical Officer, Dr. Michael Chancellor, stated, “We can now proceed with the confidence that our Phase 2b study design can successfully meet its regulatory objectives. This clarity significantly advances our value proposition.”

2025 will also be a year of significant achievement for Lipella as it pursues a Phase 2a clinical trial in oral Graft-versus-Host disease (GvHD).

A Leader in Orphan Designations

In November 2023, Lipella was granted Orphan Drug Designation by the FDA for LP-310, its clinical-stage pipeline asset for inflammatory diseases of the oral cavity, including oral lichen planus  (OLP) and oral Graft-versus-Host disease (GvHD). LP-310 is a proprietary oral rinse formulation of Lipella’s lead drug candidate, LP-10. Lipella was previously granted Orphan Drug Designation for LP-10.

The FDA’s Orphan Drug Designation program grants special privileges to companies developing therapeutics intended to treat rare diseases. Companies qualify for incentives, including tax credits for clinical trials, exemption from user fees and a potential seven years of market exclusivity after approval. Drugs for rare diseases have a high rate of success in obtaining regulatory approval, with around 26% being approved, versus 11% for all other drugs.

Dr. Kaufman commented in a company announcement, saying, “This designation is an example of the way we build value, pursuing all available resources that can de-risk and accelerate our clinical research programs. Our collaborative practice with the FDA is critical to our ability to increase the value of all of our clinical assets.”

Industry-Leading Management Team

Lipella is led by an industry-leading management team and benefits from the significant contributions of two stellar scientific advisory boards. In April 2024, the company announced it would expand its Scientific Advisory Board to include oncology expertise, as it seeks to develop LP-50, an intravesical immunotherapeutic for the treatment of non-muscle invasive bladder cancer (NMIBC), a disease that affects nearly one million Americans. In 2023, the company announced it had created a Scientific Advisory Board of highly regarded experts in oral medicine to focus on the development of LP-310 in OLP, and assist with additional indications in oral liposomal drug delivery and treatment.

Additionally, Dr. Chancellor, the company’s Chief Medical Officer, is an internationally recognized expert in the treatment of urinary bladder dysfunction, and a key opinion leader in the field. He has conducted more than75 clinical trials and has consulted with numerous biotech companies developing urology products.

Manufacturing Advantage

In preparation for its upcoming clinical trials, Lipella announced it had entered a manufacturing partnership with Cook MyoSite, a company with two decades of Good Manufacturing Practice (GMP) manufacturing expertise, to facilitate Chemistry, Manufacturing, and Control (CMC) documentation for the company’s pipeline candidates, and for commercial grade manufacturing.

Dr. Chancellor said, “Cook MyoSite’s reputation for quality and safety standards, along with their extensive experience in GMP manufacturing, will be invaluable as we develop our IND application for LP-10. We are confident that this collaboration will expedite our efforts to produce clinical supplies and research products for LP-10, ultimately benefiting patients.”

In addition, Lipella maintains a sterile manufacturing facility in Pittsburgh, PA for the production of clinical supplies and research products. LP -10 has been produced at high quantities in this facility for Lipella’s clinical trials. The facility is also used for development of liposomal formulations intended for intravesical delivery. 

Lipella has emerged as a notable stock to watch in 2024. The company has demonstrated significant growth, with its first-quarter revenue increasing by 24% to $145.9k compared to the previous year. As Lipella pursues continued development of its broad clinical pipeline, and its strategic focus on enhancing its market position, the company presents a compelling opportunity for investors seeking sustainable, long-term value in the biotech sector.