FDA Grants Orphan Designation for Lipella's LP-310 Drug Candidate for Oral Graft-versus-Host Disease

Rhea-AI Summary

Lipella Pharmaceuticals Inc. (Nasdaq: LIPO) has been granted 'Orphan Drug Designation' from the U.S. FDA for its LP-310 drug candidate, aimed at treating oral Graft-versus-Host Disease (GvHD) and other inflammatory diseases of the oral cavity. The FDA's designation provides incentives and potential market exclusivity for the drug.

Positive

• LP-310 addresses a significant unmet need for the treatment of oral GvHD and other oral inflammatory diseases.
• The Orphan Drug Designation qualifies Lipella for incentives, including tax credits, exemption from user fees, and potential market exclusivity for seven years after approval.

Negative

• None.

Lipella Pharmaceuticals' LP-310 clinical stage asset is formulated for delivering active agents to the oral cavity for diseases of the mouth including oral lichen planus and oral graft-versus-host disease

PITTSBURGH, Nov. 10, 2023 /PRNewswire/ -- Lipella Pharmaceuticals Inc. (Nasdaq: LIPO), a clinical stage pharmaceutical company addressing serious diseases with significant unmet need based in Pittsburgh, PA, announced today that it has been granted "Orphan Drug Designation" from the U.S. Food and Drug Administration (FDA) for its drug candidate, LP-310, which has been designed for the treatment of oral Graft-versus-Host Disease (GvHD). 

FDA Grants Orphan Designation for Lipella's LP-310 Drug Candidate for Oral Graft-versus-Host Disease

LP-310 is Lipella's clinical stage pipeline asset intended to be indicated for inflammatory diseases of the oral cavity, including oral lichen planus and oral GvHD.

LP-310 liposomal tacrolimus is a proprietary oral rinse formulation of Lipella's lead drug candidate, LP-10. Lipella recently received FDA clearance for a Phase 2a clinical trial to evaluate LP-310's safety and efficacy in patients with symptomatic oral lichen planus, which currently has no FDA approved treatment, and can severely impact a patient's quality of life.

Dr. Jonathan Kaufman, Ph.D., Chief Executive Officer of Lipella, said, "We are very pleased to have received Orphan Drug Designation for LP-310 in oral GvHD. 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. We look forward to advancing this drug as a potential treatment for this painful complication of chronic GvHD."

Dr. Michael Chancellor, Chief Medical Officer at Lipella, said, "GvHD occurs when donor immune cells attack the recipient's body tissues after an allogeneic tissue or bone marrow transplant. GvHD affects approximately 30,000 Americans and oral GvHD contributes significantly to morbidity in cancer survivors. Morbidity of oral GvHD encompasses significant oral pain and discomfort, making it difficult for patients to eat, drink and speak. In addition, the risk of oral cavity infection, fibrosis and even oral cancer increases. Oral GvHD affects patients' quality of life and is a great unmet need in cancer survivors."

The FDA's "Orphan Drug Designation" program provides orphan status to drugs and biologics that are intended for the treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the United States. Orphan Drug Designation qualifies sponsors for incentives, including tax credits for qualified clinical trials, exemption from user fees and a potential seven years of market exclusivity after approval.

Lipella's LP-10 was previously granted Orphan Drug Designation by the FDA, making LP-310 the second of Lipella's leading product candidates to receive this designation.

About LP-310

LP-310 liposomal tacrolimus is a proprietary oral rinse formulation of Lipella's lead drug candidate, LP-10.

About Graft-versus-Host Disease

Graft-Versus-Host Disease (GvHD) occurs when  donor bone marrow or stem cells attack the recipient following a treatment for leukemia, lymphoma, and other hematological cancers. Acute GvHD most commonly affects the skin, liver, and the gastrointestinal tract, while chronic GvHD affects the skin, mouth, eyes, lungs, stomach, bowel and liver, as well as other organs. Oral lichen planus can occur as a single manifestation of chronic GvHD.

About Lipella Pharmaceuticals Inc.

Lipella Pharmaceuticals Inc. is a clinical stage pharmaceutical company with a focus on capital efficient therapeutic development opportunities, including the proprietary repositioning of generics for rare, morbid diseases that currently have no adequate treatments.  Additional information is available at www.lipella.com.

Forward-Looking Statements

This press release includes certain "forward-looking statements." All statements, other than statements of historical fact, included in this press release regarding, among other things, our strategy, future operations, financial position, prospects, pipeline and opportunities, sources of growth, successful implementation of our proprietary technology, plans and objectives are forward-looking statements. Forward-looking statements can be identified by words such as "may," "will," "could," "continue," "would," "should," "potential," "target," "goal," "anticipates," "intends," "plans," "seeks," "believes," "estimates," "predicts," "expects," "projects" and similar references to future periods. Forward-looking statements are based on our current expectations and assumptions regarding future events and financial trends that we believe may affect among other things, our financial condition, results of operations, business strategy, short- and long-term business operations and objectives, and financial needs. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. Our actual results may differ materially from those contemplated by the forward-looking statements. We caution you, therefore, against relying on any of these forward-looking statements. They are neither statements of historical fact nor guarantees or assurances of future performance. There are risks, uncertainties and other factors, both known and unknown, that could cause actual results to differ materially from those in the forward-looking statements which include, but are not limited to, regional, national or global political, economic, business, competitive, market and regulatory conditions, and other factors. Any forward-looking statement made by us is based upon the reasonable judgment of our management at the time such statement is made and speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law. Nothing contained herein is, or shall be relied upon as, a promise or representation as to the past or future. In addition, the information contained in this press release is as of the date hereof, and Lipella has no obligation to update such information, including in the event that such information becomes inaccurate. You should not construe the contents of this press release as legal, tax and financial advisors as to legal and related matters concerning the matters described herein.

Contacts
Dr. Jonathan Kaufman, CEO
Lipella Pharmaceuticals
Info@Lipella.com 
1-412-894-1853

Jeff Ramson
PCG Advisory
jramson@pcgadvisory.com
1-917-912-9130

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SOURCE Lipella Pharmaceuticals Inc.

FAQ

What is Lipella Pharmaceuticals Inc. (Nasdaq: LIPO) working on?

Lipella is working on a drug candidate, LP-310, which has been granted 'Orphan Drug Designation' by the FDA for the treatment of oral Graft-versus-Host Disease (GvHD) and other inflammatory diseases of the oral cavity.

What is the significance of the FDA's 'Orphan Drug Designation'?

The designation provides incentives, including tax credits, exemption from user fees, and potential market exclusivity for seven years after approval, for drugs intended to treat rare diseases affecting fewer than 200,000 people in the United States.

Why is LP-310 important?

LP-310 addresses a significant unmet need for the treatment of oral GvHD and other oral inflammatory diseases, providing potential relief for patients with these conditions.