Ascelia Pharma Hits Primary Endpoint in Orviglance Phase 3 Study

Ascelia Pharma AB (publ), a biotech firm dedicated to improving the lives of people with rare cancer conditions, has announced a significant breakthrough with its liver imaging drug candidate, Orviglance®. The pivotal Phase 3 study, named SPARKLE, successfully met its primary endpoint, demonstrating that Orviglance significantly enhanced the visualization of focal liver lesions compared to unenhanced MRI. The results achieved statistical significance across all three readers, showcasing a P value of less than 0.001.

Orviglance is being developed as a first-in-class contrast agent for liver MRI, specifically targeting patients with severely impaired kidney function. Recognizing its potential, the FDA has granted it Orphan Drug Designation. This designation underscores the urgent need for alternative imaging solutions for this vulnerable patient group, who are at high risk of developing Nephrogenic Systemic Fibrosis (NSF) from gadolinium-based contrast agents currently in use.

The clinical development of Orviglance has now been completed with nine studies involving 286 patients and healthy volunteers, culminating in the SPARKLE study. Despite initial challenges, including high intra-reader variability in image scoring, the company conducted a successful re-evaluation of the images with new readers, confirming the robust efficacy of Orviglance. Common adverse events observed were mild to moderate nausea, aligning with previous studies, and no serious adverse drug reactions were reported.

Magnus Corfitzen, CEO of Ascelia Pharma, expressed his excitement over the Phase 3 results, marking this as a significant milestone for the company. He emphasized the company's commitment to advancing Orviglance through the regulatory submission process and making it available to patients. The submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) is anticipated by mid-2025.

In parallel, Ascelia Pharma will continue its efforts in launch readiness and engage in discussions with potential commercialization partners to ensure that Orviglance reaches patients who require high-quality liver imaging without the safety risks associated with gadolinium.

The SPARKLE study included MRI data from 85 patients with known or suspected focal liver lesions and severely impaired kidney function. The enhancement of lesion visualization was evaluated by three independent radiologists as per regulatory standards, which validated the strong and conclusive efficacy data for Orviglance.

Ascelia Pharma has scheduled a virtual Investor Update titled “Bringing Orviglance to Patients” on Tuesday, 7 May 2024, at 14:00 CEST. This event will provide investors, analysts, and the media with an opportunity to understand the study's results in detail and learn about the next steps in regulatory and commercialization activities.

Orviglance holds significant promise for patients with impaired kidney function, who currently receive suboptimal unenhanced MRI. The product addresses an unmet medical need and represents an estimated global market potential of USD 800 million annually. By providing a safer alternative for liver imaging, Orviglance aims to improve the quality of life and outcomes for these patients.

Ascelia Pharma, headquartered in Malmö, Sweden, focuses on developing and commercializing novel drugs that address unmet medical needs in orphan oncology. Alongside Orviglance, the company is also developing Oncoral, an oral irinotecan chemotherapy tablet for gastric cancer. With its innovative pipeline, Ascelia Pharma continues to make strides in improving cancer care.