Lichen Planus, Oral

PITTSBURGH, April 03, 2024 (GLOBE NEWSWIRE) -- Lipella Pharmaceuticals Inc. (Nasdaq: “LIPO”) (“Lipella,” “our,” “us” or the “Company”), a clinical-stage biotechnology company addressing serious diseases with significant unmet need, today announced that the U.S. Food and Drug Administration (“FDA”) has granted a Type C meeting request to discuss the company’s proposed Phase-2b clinical trial design for the evaluation of LP-10, an intravesical liposomal formulation of tacrolimus, as a potential treatment for moderate to severe hemorrhagic cystitis (HC). Lipella expects to meet with the FDA on May 21, 2024. HC is a serious, life-threatening form of gross hematuria (i.e. visible blood in urine) caused by cancer treatments including pelvic radiation therapy, and certain chemotherapies including cyclophosphamide and ifosfamide, which are most commonly used for the treatment of breast cancer. Dr. Michael Chancellor, Lipella’s Chief Medical Officer, expressed anticipation for advancing the LP-10 program, stating, “We are eager to advance our LP-10 program efficiently, and our scheduled FDA meeting in the second quarter of 2024 will play a pivotal role in enhancing the regulatory prospects of LP-10.” Lipella successfully completed a Phase-2a open-label, dose-escalation clinical trial in early 2023 demonstrating the safety, and the potential efficacy of LP-10 in 13 patients with a history of HC. The study demonstrated preliminary efficacy in multiple measures, including urine microscopy, urinalysis, urinary urgency, as well as a potential dose response in the reduction of bleeding sites observed during bladder cystoscopy. Lipella published these results in the peer-reviewed journal, International Urology and Nephrology, in September 2023, and received preliminary guidance from the FDA regarding potential registration endpoints. The company’s anticipated Type C meeting with the FDA will focus on reaching an agreement for the proposed Phase-2b trial design, which Lipella expects to be a 36-subject, prospective, double-blind, placebo-controlled study with a primary efficacy assessment derived from the reduction in patient-reported frequency of gross hematuria episodes over a seven-day period. About LP-10, LP-310 and LP-410 LP-10 is a liposomal tacrolimus formulation for intravesical administration to treat hemorrhagic cystitis (HC). LP-10 has been evaluated in a multicenter Phase 2a dose escalation trial of 13 subjects with moderate to severe refractory HC, with positive top line results demonstrating safety and efficacy, short duration of systemic uptake of LP-10, and a dose response including decreased hematuria, decreased cystoscopic bleeding and ulceration sites, and improved urinary symptoms in patients. Lipella has been granted Orphan Disease Designation by the FDA for LP-10 in the treatment of moderate to severe hemorrhagic cystitis. LP-310/LP-410 liposomal tacrolimus is a proprietary oral rinse formulation of Lipella’s lead drug candidate, LP-10, for the indications of oral lichen planus (OLP) and oral Graft-versus-Host Disease (GvHD). Lipella has been granted orphan drug designation by the FDA for LP-310 in the treatment of GvHD. About Lipella Pharmaceuticals Inc. Lipella Pharmaceuticals is a clinical-stage biotechnology company focused on developing new drugs by reformulating the active agents in existing generic drugs and optimizing these reformulations for new applications. Additionally, Lipella maintains a therapeutic focus on diseases with significant, unaddressed morbidity and mortality where no approved drug therapy currently exists. Lipella completed its initial public offering in December 2022. For more information, please visit or LinkedIn. 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, clinical trials, regulatory approvals, 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, market and other conditions, 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, risks related to the effective application of the use of proceeds from the private placement, general capital market risks, 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 the Company 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. CONTACT Dr. Jonathan Kaufman, CEO Lipella Pharmaceuticals Info@Lipella.com 1-412-894-1853 PCG Advisory Jeff Ramson jramson@pcgadvisory.com

Lipella Granted Two Orphan Drug Designations by FDAPITTSBURGH, Dec. 20, 2023 (GLOBE NEWSWIRE) -- Lipella Pharmaceuticals Inc., (Nasdaq: LIPO) ("Lipella," "our," "us" or the "Company"), a pioneering, clinical-stage pharmaceutical company, celebrates its first anniversary as a publicly traded entity. Based in Pittsburgh, PA, the Company has made substantial strides over the past year, achieving critical milestones and advancing its pipeline with three clinical indications and two orphan drug designations granted. A highlight of the year includes the successful completion of a Type C meeting with the U.S. Food and Drug Administration (FDA) focused on the clinical program for LP-10, Lipella's leading drug candidate for the treatment of hemorrhagic cystitis (HC). Jonathan Kaufman, CEO of Lipella, expressed his satisfaction with the meeting's outcome, noting, “The FDA's feedback from our recent Type C meeting gives us a clear path forward into 2024. Our planned Phase-2b trial for LP-10 will involve 36 subjects in a double-blind, placebo-controlled study, focusing on the treatment's impact on gross hematuria." Further bolstering its portfolio, Lipella has secured FDA approval for its Investigational New Drug (IND) application for a Phase 2a multicenter trial of an oral rinse formulation of LP-310, targeting Oral Lichen Planus (OLP). With central Institutional Review Board (IRB) approval in hand, the Company is in the process of selecting study sites for this trial. Additionally, Lipella's LP-310 has been granted orphan drug designation for the treatment of oral graft-versus-host disease (GVHD) with tacrolimus. The Company is poised to submit a Phase 2a IND for this indication in the first quarter of 2024. Reflecting on the Company's journey, Dr. Michael Chancellor, Lipella’s Chief Medical Officer, remarked, “Our first year as a public company, especially during challenging market conditions, has been remarkable. Heading into 2024, we are enthusiastic about advancing our assets through clinical trials, bolstered by two orphan designations.” As Lipella Pharmaceuticals steps into another year of innovation and growth, it remains committed to developing groundbreaking treatments that address critical healthcare needs. About LP-10 and LP-310 LP-10 is a liposomal tacrolimus formulation for intravesical administration to treat hemorrhagic cystitis (HC). LP-10 has been evaluated in a multi-center Phase 2a dose escalation trial of 13 subjects with moderate to severe refractory HC. Positive top line results demonstrated safety and efficacy, short duration of systemic uptake of LP-10, and a dose response including decreased hematuria, decreased cystoscopic bleeding and ulceration sites, and improved urinary symptoms in patients. Lipella has been granted Orphan Disease Designation by the FDA for LP-10 in the treatment of moderate to severe hemorrhagic cystitis. LP-310 liposomal tacrolimus is a proprietary oral rinse formulation of Lipella’s lead drug candidate, LP-10, for the indications of OLP and oral GVHD. Lipella has been granted orphan drug designation by the FDA for LP-310 in the treatment of GVHD. About Hemorrhagic Cystitis Hemorrhagic cystitis (HC), a severe yet rare condition often triggered by pelvic radiation therapy and specific chemotherapies, is characterized by gross hematuria and currently lacks approved drug treatment. Lipella's LP-10 stands at the forefront of addressing this unmet medical need. About Oral Lichen Planus Oral Lichen Planus (OLP) is a chronic, inflammatory, T-cell-mediated, autoimmune oral mucosal disease. It is a painful condition with malignant potential, and complications include infections, scarring, stress and depression. OLP affects approximately 6-7 million Americans. There is no approved pharmacotherapy and most currently available treatments are palliative. 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. Oral manifestation of GVHD is frequently involved and is a rare and serious disease. Lipella has been granted orphan disease designation for LP-310 treatment of oral GVHD. About Lipella Pharmaceuticals Inc. Lipella Pharmaceuticals is a clinical-stage biotechnology company focused on developing new drugs by reformulating the active agents in existing generic drugs and optimizing these reformulations for new applications. Additionally, Lipella maintains a therapeutic focus on diseases with significant, unaddressed morbidity and mortality where no approved drug therapy currently exists. Lipella completed its initial public offering in December 2022. For more information, please visit www.lipella.com or LinkedIn. Forward-Looking StatementsThis 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, clinical trials, regulatory approvals, 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, market and other conditions, 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, risks related to the effective application of the use of proceeds from the private placement, general capital market risks, 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 the Company 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. CONTACTDr. Jonathan Kaufman, CEOLipella PharmaceuticalsInfo@Lipella.com 1-412-894-1853 Jeff RamsonPCG Advisoryjramson@pcgadvisory.com

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). Continue Reading 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. FDA Grants Orphan Designation for Lipella's LP-310 Drug Candidate for Oral Graft-versus-Host Disease Post this 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 . 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 [email protected] 1-412-894-1853 Jeff Ramson PCG Advisory [email protected] 1-917-912-9130 SOURCE Lipella Pharmaceuticals Inc.