Ascelia Pharma AB (publ) announced that liver imaging drug candidate, Orviglance®?, significantly improved visualization of focal liver lesions, successfully meeting the primary endpoint in the pivotal Phase 3 study SPARKLE. The Phase 3 study demonstrated strong superiority in visualization of focal liver lesions with Orviglance (CMRI) vs. unenhanced MRI with statistical significance for all three readers (<0.001) Orviglance is in development as a first-in-class contrast agent for use in liver MRI in patients with severely impaired kidney function and has been granted FDA Orphan Drug Designation.

Orviglance addresses a global USD 800 million market annually. The Phase 3 data marks the completion of Orviglance clinical development with nine studies in 286 patients and healthy volunteers. Submission of the New Drug Application (NDA) file to the US Food and Drug Administration (FDA) is expected by mid-2025.

The pivotal Phase 3 study, SPARKLE, successfully met the primary endpoint and demonstrated that the company's magnetic resonance imaging (MRI) contrast agent, Orviglance significantly improved the visualization of focal liver lesions compared to unenhanced MRI. The Company expects to submit the New Drug Application (ND) file to the US Food & Drug Administration (FDA) by mid-2025 to obtain regulatory approval. Clinical development of Orviglance has now been completed with consistent positive efficacy and safety data from 286 patients and healthy volunteers in nine studies, of which the SPARKLE is the last and pivotal study.

Patient enrollment in the global multi-center SPARKLE study was completed early 2023, with MRI data from 85 patients with known or suspected focal liver lesions and severely impaired kidney function. In accordance with regulatory requirements, the improvement of visualization of lesions was evaluated by three independent radiologist readers. In mid-2023, the unexpected discovery of high intra- reader variability in the study image scoring by the readers prevented the Company from evaluating the efficacy data from SPARKLE.

Therefore, a new evaluation of the images with new readers was required. The company successfully completed the re-evaluation according to the planned timeline with the announcement today. Orviglance has been granted an Orphan Drug Designation by the US Food and Drug Administration ("FDA") for liver MRI in patients with severely impairment kidney function.

These patients have the highest risk of developing the serious and potentially fatal condition Nephrogenic Systemic Fibrosis (NSF) after exposure to the currently available class of gadolinium-based contrast agents. These patients are at risk of serious side effects from the currently available class of gadolium-based contrast agents. Orviglance, has been granted an Orphan drug Designation by the US Food & Drug Administration ("FDA").

A clinical program of nine studies, including the pivotal global Phase 3 study SPARKLE, has successfully been completed with strong and consistent efficacy and safety results. Oncoral. Oncoral, the Phase 3 study SPARKLE has successfully been completed with strong & consistent efficacy and safety results.