NEW YORK, NY - 
(NewMediaWire) - June 13, 2023 - PCG Digital--Lipella Pharmaceuticals (Nasdaq: LIPO) is a clinical-stage biotechnology company addressing serious diseases with significant unmet medical need and potentially changing the lives of countless cancer survivors.
Lipella’s lead product candidate is a treatment targeting hemorrhagic cystitis, the serious, life-threatening bladder damage that can occur following pelvic radiation therapy or bladder-toxic chemotherapy for prostate, rectal, endometrial and cervical cancers. LP-10 has been granted Orphan Drug Designation by the FDA and may qualify for multiple accelerated pathways to regulatory approval.
Lipella’s Chief Executive Officer, Dr. Jonathan Kaufman, spoke to us about the company’s achievements to date and upcoming milestones to watch for in 2023.
Q: Which disease areas is Lipella targeting, and how do these relate to the cancer survivor community?
A: Our primary area of focus is hemorrhagic cystitis (HC), a serious rare disease that can result from pelvic radiation or chemotherapy for pelvic cancers. The effects of these treatments may not be felt until many years later when patients can acquire HC, which is characterized by uncontrolled blood loss from the bladder. HC is a potentially chronic, highly morbid condition with no approved drug therapy, and a four percent mortality rate.
Lipella is also targeting oral lichen planus (OLP), a chronic inflammatory disease that affects the mucous membrane of the oral cavity. OLP is painful and difficult to manage and can have serious complications including infections, scarring, and the potential for malignant transformation into oral squamous cell carcinoma. Most currently available therapies treat pain and symptoms, and there is no cure.
Q: Tell us about Lipella’s drug delivery technology?
A: Lipella has developed proprietary drug delivery technology that suspends a drug in a lipid, enhancing its solubility and bioavailability. Lipella’s metastable Liposome technology platform produces lipid particles optimized to deliver hydrophobic agents directly to the epithelium via partial membrane transfer. This formulation results in high local drug concentrations and low systemic exposure. Lipella has broad patent protection issued on this platform technology through 2034 (US patent #10,639,278).
Our technology is designed for use with other lipophilic drugs, bringing with it the potential for technology partnerships and future commercial licensing agreements. Liposomal tacrolimus has potential applications in the treatments for the bladder, urethra, oral cavity, esophagus and colon.
Q: What is the market opportunity for Lipella in hemorrhagic cystitis?
A: We estimate that peak annual revenues in HC exceed $1 billion. Our expected annual revenue is $20,000 per patient and Lipella is targeting an addressable market of approximately 60,000 potential new patients each year. Our mission is to provide supportive care to cancer survivors and we believe we have a strong opportunity to achieve this with our lead candidate, LP-10 for the treatment of HC.
Q: What is liposomal tacrolimus and why is the right choice for Lipella?
A: We believe that LP-10 is an outstanding drug candidate. Tacrolimus has a well-known mechanism of action and established safety and efficacy in a variety of uses. Liposomal tacrolimus is developed using our proprietary drug delivery and acts as a potent vasoconstrictor, reducing capillary blood flow to the bladder lumen, and a potent anti-inflammatory, inhibiting the cytokine cascade and reducing injury to the bladder tissue. Lipella has secured patent protection for LP-10 through 2034.
Q: Can you tell us about LP-10’s clinical progress and what you see as the pathway to regulatory approval?
A: We are pursuing the 505(b)(2) pathway for LP-10 and have successfully completed our Phase 2 clinical trial studying its safety and efficacy as a potential treatment for HC. Our clinical trial was a multi-center, dose-escalation study of 13 subjects with moderate to severe refractory HC. Subjects were treated with up to two courses of LP-10 intravesical bladder instillations, which were tolerated without report of serious adverse events. We were very pleased to note that the treatment response was fast and patients were able to tolerate LP-10. The study demonstrated decreased hematuria, decreased cystoscopic bleeding and ulceration sites, and improved urinary symptoms in patients.
We expect to meet with the FDA in the second half of 2023 for a Type B meeting to establish a path forward as we consider the design of our Phase 3 clinical trial.
Q: Can you talk more about Lipella’s pipeline of products?
A: Along with LP-10 for HC, Lipella is developing LP-310 as a potential treatment for OLP. LP-310 is our proprietary oral rinse formulation of LP-10 that allows for increased local concentration in the oral cavity while minimalizing systemic toxicity. Lipella has received Type B pre-IND guidance from the FDA for LP-310 and we expect to submit an IND application in late 2023. We are preparing to initiate a Phase 2a multicenter dose escalation study in 2024.
Q: Why is Lipella pursuing treatments for rare and orphan disease indications?
A: Our strategy is to develop treatments for serious rare and orphan diseases and to support the cancer survivor community while delivering long term value for shareholders. Orphan drug development can often be more profitable, entail lower risk and result in faster time to approval than non-orphan therapeutics. It also offers market exclusivity of seven years of exclusivity in the US and 10 years in the EU and Japan.
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