Ascelia Pharma AB (publ) (ticker: ACE), a biotech focused on improving the life of people living with rare cancer conditions, today announced the successful completion of patient enrollment in the company’s pivotal phase 3 clinical study, SPARKLE, with the lead candidate drug Orviglance®.
“We are very pleased to have reached our enrollment target of 80 patients. This is a major milestone in the history of Ascelia Pharma and a key step on the journey to making Orviglance available to patients around the world. The focus will now switch to the evaluation of MRI images by independent radiologists, as required by regulatory standards, and the headline results from this pivotal study are expected in mid-2023”, said Magnus Corfitzen, CEO of Ascelia Pharma.
Orviglance, which has been granted an Orphan Drug Designation by the US Food and Drug Administration (FDA), is the first contrast agent in development for use in liver magnetic resonance imaging (MRI) in patients with severely impaired kidney function.
Cancer can be difficult to diagnose and treat because of its ability to metastasize - spread beyond the location of the primary tumor. The liver is the second most likely organ for metastases to develop, playing a significant role in the demise of patients diagnosed with breast or colorectal cancer. MRI is usually the preferred imaging modality for both initial cancer disease staging and monitoring of liver metastases and relies on contrast agents to improve the quality of the obtained scan. However, the standard MRI contrast agents currently available contain the heavy metal gadolinium, which when used in patients with impaired kidney function can increase the risk of the serious and potentially fatal condition, Nephrogenic Systemic Fibrosis (NSF). For this reason, regulatory bodies have issued warnings for their use in this patient population and patients with severe kidney impairment typically have an MRI without contrast enhancement, which reduces image quality and ability to detect and localize cancer tumors or metastases.
Ascelia Pharma’s SPARKLE study aims to demonstrate that Orviglance improves the detection and visualization of focal liver lesions, including liver metastases and primary tumors, in patients with severe kidney impairment.
The SPARKLE study is the last of nine studies in the extensive clinical development program for Orviglance, which will enable Ascelia Pharma to complete a New Drug Application (NDA) submission to the FDA.
Orviglance aims to give cancer patients with poor kidney function access to safe and effective liver imaging to live longer and healthier lives. The unmet need for these patients represents an addressable market potential of USD 500-600 million in the US, Europe and Japan.
“On behalf of the entire Ascelia Pharma team, I would like to thank patients, investigators, and other collaborators who have been involved in SPARKLE. We have succeeded in enrolling 80 patients in a rare disease clinical study under highly challenging circumstances, including a global pandemic and the invasion of Ukraine. Based on strong results in earlier clinical studies, we are optimistic of a positive outcome of SPARKLE and look forward to the next steps in bringing Orviglance through the regulatory process and making it available to patients for whom the use of gadolinium-based products may be medically inadvisable”, said Magnus Corfitzen.
Ascelia Pharma is a biotech company focused on orphan oncology treatments. We develop and commercialize novel drugs that address unmet medical needs and have a clear development and market pathway. The company has two drug candidates – Orviglance (previously referred to as Mangoral) and Oncoral – in clinical development. Ascelia Pharma has global headquarters in Malmö, Sweden, and is listed on Nasdaq Stockholm (ticker: ACE). For more information, please visit http://www.ascelia.com.
About Orviglance (previously referred to as Mangoral)
Orviglance (manganese chloride tetrahydrate) is a novel oral contrast agent for MR-imaging developed to improve the detection and visualization of focal liver lesions (including liver metastases and primary tumors) in patients with reduced kidney function. These patients are at risk of serious side effects from the currently available class of gadolinium-based contrast agents. Orviglance has been granted an Orphan Drug Designation by the US Food and Drug Administration (FDA). A clinical program of nine studies, including the global Phase 3 study SPARKLE, have been completed.