Tacrolimus

BRISBANE, Calif.--(BUSINESS WIRE)--CareDx, Inc. (Nasdaq: CDNA) – The Transplant Company™ – a leading precision medicine company focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers – today announced that CareDx, together with study collaborators, presented new scientific evidence on the use of AlloSure® Lung in clinical practice at the International Society for Heart and Lung Transplantation (ISHLT) 45th Annual Meeting & Scientific Sessions, held from April 27-30 in Boston, Massachusetts. AlloSure Lung and AlloHome™ were featured in 8 abstracts including 2 oral presentations and key findings were: Surveillance monitoring using AlloSure Lung effectively identified rising dd-cfDNA levels signaling onset of acute cellular rejection and decline in levels after treatment for rejection ( Abstract 1627 ). AlloSure Lung was used to monitor safety and graft function of Tacrolimus (TAC) inhalation powder as a replacement to oral TAC in lung transplant patients with renal insufficiency ( Abstract 143, oral session). AlloSure Lung was used to investigate the impact of TAC drug levels on the outcomes of DSA-positive lung transplant recipients over a one-year follow-up ( Abstract 403 , mini oral). AlloHome remote patient monitoring system in lung transplant enables real-time treatment adjustments and improved triage, easing workload on transplant center staff ( Abstract 1204 ). “We are very pleased with the new data presented at ISHLT indicating that surveillance monitoring using AlloSure effectively detected rejection and could be used to monitor patient treatment responses,” said Robert Woodward, CareDx Chief Scientific Officer. Additionally, during a CareDx sponsored symposium, an interim analysis of the ALAMO (AlloSure Lung Assessment and Metagenomics Outcomes) prospective, multi-center study showed that AlloSure Lung may be prognostic of Chronic Allograft Lung Dysfunction (CLAD), a leading cause of death after lung transplantation. Elevated levels of AlloSure dd-cfDNA in patients early post-transplant were associated with future occurrence of restrictive allograft syndrome (RAS), a severe form of CLAD. "We have observed the advantages of using AlloSure Lung for monitoring patients for early signs of graft injury and rejection. The early findings from the ALAMO study are promising as it indicates that increased levels of AlloSure Lung dd-cfDNA may identify patients at higher risk of developing CLAD, which is a major cause of mortality among lung transplant patients," said Ciara Shaver, MD, PhD, Principal Investigator for ALAMO at Vanderbilt University Medical Center. "Furthermore, the ALAMO data show that AlloSure Lung may identify subclinical lung injury during CMV viremia. In these ways, AlloSure Lung may help clinicians to identify the patients with the greatest chance to respond to therapy to enhance long-term outcomes.” For a complete listing of abstracts, oral presentations and posters featuring CareDx’s heart and lung transplant portfolio, please follow this link. About CareDx – The Transplant Company CareDx, Inc., headquartered in Brisbane, California, is a leading precision medicine solutions company focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers. CareDx offers testing services, products, and digital healthcare solutions along the pre- and post-transplant patient journey and is the leading provider of genomics-based information for transplant patients. For more information, please visit www.caredx.com. Forward Looking Statements This press release includes forward-looking statements related to CareDx, Inc., including statements regarding the potential benefits and results that have been or may be achieved with AlloSure Lung, AlloHome, and other CareDx products, and statements regarding the interim study results of CareDx’s ALAMO registry at the 2025 International Society for Heart and Lung Transplantation Meeting (the “ISHLT Presentation”). These forward-looking statements are based upon information that is currently available to CareDx and its current expectations, speak only as of the date hereof, and are subject to risks and uncertainties that could cause actual results to differ materially from those projected, including risks that CareDx does not realize the expected benefits of AlloSure Lung, AlloHome, or other CareDx products or its ISHLT Presentation; risks that the ISHLT Presentation and the data to be presented may not follow the agenda as stated in this press release; risks that the findings in the studies supporting the data may be inaccurate, general economic and market factors, and other risks discussed in CareDx’s filings with the Securities and Exchange Commission (the “SEC”), including, but not limited to, the Annual Report on Form 10-K for the fiscal year ended December 31, 2024 filed by CareDx with the SEC on February 28, 2025, and other reports that CareDx has filed with the SEC. Any of these may cause CareDx’s actual results, performance, or achievements to differ materially and adversely from those anticipated or implied by CareDx’s forward-looking statements. You are cautioned not to place undue reliance on these forward-looking statements. CareDx expressly disclaims any obligation, except as required by law, or undertaking to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.

Second Cohort Results Highlight Efficacy Across All Key Measures and Reinforce Safety of Twice-Daily Oral Rinse Phase 2a Study Now Fully Enrolled Across All Three Dose Cohorts Advancing Toward Phase 2b IND Submission and Broader Regulatory Engagement PITTSBURGH, April 22, 2025 (GLOBE NEWSWIRE) -- Lipella Pharmaceuticals Inc. (Nasdaq: LIPO) (“Lipella,” “our,” “us,” or the “Company”), a clinical-stage biotechnology company addressing serious diseases with significant unmet needs, today announced positive topline results from the second cohort (0.50 mg) of its Phase 2a multicenter, dose-ranging trial evaluating LP-310, a liposomal-tacrolimus oral rinse formulation of LP-10, for the treatment of oral lichen planus (OLP). The Company also recently reported full enrollment across all three planned dose cohorts. Treatment with LP-310 at the 0.50 mg dose demonstrated statistically significant improvements in multiple patient-reported and investigator-measured efficacy endpoints, reinforcing LP-310’s potential as a non-steroidal, locally delivered therapy for OLP. The trial, currently active across seven U.S. study sites, has now progressed to the third and highest dose cohort (1.0 mg/10 mL), with topline results expected in the second quarter of 2025. “These results mark meaningful progress for LP-310 and further strengthen our confidence in its clinical and commercial potential,” said Jonathan Kaufman, Co-Founder and Chief Executive Officer of Lipella Pharmaceuticals. “With the Phase 2a trial now fully enrolled and final data expected this quarter, we are advancing toward key value-driving milestones, including a Phase 2b IND submission and broader regulatory engagement to support the program’s continued development.” Topline Findings from the 0.50 mg Cohort: Investigator Global Assessment (IGA): Improved from 3.42 ± 0.21 at baseline to 2.71 ± 0.30 at week 1 (p=0.029), 1.71 ± 0.43 at week 4 (p=0.007) and 2.75 ± 0.34 at week 6 (p=0.112).Reticulation, Erythema and Ulceration (REU) Score: Reduced from 26.91 ± 2.54 at baseline to 17.02 ± 2.36 at week 1 (p=0.003), 11.88 ± 2.91 at week 4 (p=0.003) and 18.47 ± 4.12 at week 6 (p=0.028).Oral Lichen Planus Symptom Severity Measure (OLPSSM): Decreased from 14.92 ± 2.10 at baseline to 9.87 ± 2.27 at week 1 (p=0.032), 4.88 ± 2.15 at week 4 (p=0.003) and 8.42 ± 3.98 at week 6 (p=0.028).Pain Numerical Rating Scale (NRS): Improved from 6.42 ± 0.75 at baseline to 4.25 ± 0.89 at week 1 (p=0.003), 2.25 ± 1.09 at week 4 (p=0.003) and 3.52 ± 1.51 at week 6 (p=0.028).Global Response Assessment (GRA): Significant improvement was observed at week 4 (p=0.028). Safety and Tolerability LP-310 continues to be well tolerated, with no treatment-related SAEs and no patient dropouts. All participants successfully adhered to the twice-daily 10-milliliter rinse regimen. Pharmacokinetic analysis confirmed that tacrolimus levels remained undetectable or minimal in all patients, reinforcing LP-310’s ability to deliver localized benefits without systemic toxicity. “We’re very encouraged by the positive data from the 0.50 mg cohort, which demonstrated statistically significant efficacy across multiple clinical measures while maintaining a strong safety profile,” said Dr. Michael Chancellor, Co-Founder and Chief Medical Officer of Lipella Pharmaceuticals. “Oral lichen planus is a painful, chronic condition with no FDA-approved treatment and limited therapeutic options. The symptom relief reported by patients, along with reductions in inflammation and ulceration, highlights LP-310’s potential to be a transformative, steroid-free, and easy-to-use therapy that addresses a longstanding clinical unmet need and improves quality of life.” Next Steps in DevelopmentWith enrollment now complete across all three dose cohorts, Lipella is positioned to deliver final topline data from the 1.0 mg cohort in Q2 2025. These data are expected to form the foundation for upcoming regulatory interactions and next-phase clinical advancement. Key development milestones include: Reporting final phase 2a topline results in Q2 2025Advancing discussions with regulatory agencies including submission of an Investigational New Drug (IND) application for a Phase 2b trial in late 2025Preparing to pursue FDA Breakthrough Therapy designation request About LP-310LP-310 is an innovative oral rinse formulation of LP-10 (tacrolimus), developed to address OLP. Designed to provide localized therapeutic effects while minimizing systemic exposure, LP-310 offers a promising new approach to managing this painful and often debilitating condition. A Phase 2a multicenter, dose-ranging clinical trial is currently underway to evaluate the safety, tolerability and efficacy of LP-310 in adult participants with symptomatic OLP. The trial includes three dose levels (0.25 mg, 0.5 mg and 1.0 mg of tacrolimus) and is being conducted across seven active U.S. sites, which are now recruiting participants. Lipella has reported topline data from the first two cohorts (0.25 mg and 0.50 mg), with the final cohort (1.0 mg) currently enrolling. Topline results from the 1.0 mg cohort are expected in the first half of 2025. For more information about the study or to participate, visit https://lipella.com/oral-lichen-planus-treatment/ or https://clinicaltrials.gov/study/NCT06233591. About Lipella Pharmaceuticals Inc. Lipella Pharmaceuticals is a clinical-stage biotechnology company focused on developing new drugs by reformulating active agents in existing generic drugs and optimizing these reformulations for new applications. Lipella targets diseases with significant unmet needs, where no approved drug therapies currently exist. The company completed its initial public offering in 2022. Learn more at lipella.com and follow us on X and LinkedIn. Forward-Looking StatementsThis press release includes certain "forward-looking statements" which are not historical facts, within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. 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, the current clinical trial results for LP-310 and our other products general capital market risks, our ability to regain and maintain compliance with the listing standards of The Nasdaq Stock Market LLC, regional, national or global political, economic, business, competitive, market and regulatory conditions, our current liquidity position and the need to obtain additional financing to support ongoing operations, and other risks as more fully described in our filings with the U.S. Securities and Exchange Commission. 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 or investment advice and should consult with your own advisors as to the matters described herein, as applicable. CONTACTJonathan KaufmanChief Executive OfficerLipella Pharmaceuticals Inc.Info@Lipella.com1-412-894-1853 PCG AdvisoryJeff Ramsonjramson@pcgadvisory.com