Orviglance®

Orviglance* (ACE-MBCA, Ascelia Pharma manganese-based contrast agent with manganese chloride tetrahydrate) is a novel oral imaging agent for use in MR-imaging of the liver. Orviglance aims to improve the detection and visualization of focal liver lesions (including liver metastases and primary tumors) in patients with severe kidney impairment.

Orviglance is the first contrast agent in development for use in liver MR imaging in adults with severely impaired kidney function. This patient group is at risk of serious, and potentially fatal, side effects, including the serious and potentially fatal condition Nephrogenic Systemic Fibrosis (NSF), from using the currently available gadolinium-based contrast agents. These contrast agents, which are based on the heavy-metal gadolinium, carry Black Box warnings for patients with severely reduced kidney function.

Orviglance has been granted an Orphan Drug Designation by the US FDA. Clinical development of Orviglance has been completed with consistent positive efficacy and safety data from nine studies with 286 patients and healthy volunteers. In 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.

Submission of the New Drug Application (NDA) file to the FDA is expected by mid-2025.

SIGNIFICANT UNMET MEDICAL NEED IN DETECTING LIVER METASTASES

Early detection and localization of focal liver lesions is critical for optimal patient management.

The liver is the second most common organ for metastasis after the lymph nodes. Detecting liver metastases at an early stage is crucial for determining the right treatment method and the patient’s chances of survival. Studies show that the five-year survival rate can increase from 6 percent to 46 percent if liver metastases can be removed surgically. An accurate MRI scan using contrast agents is therefore critical to evaluate the possibility for surgical resection, but also for monitoring of treatment effect and surveillance for recurrence of the disease.

CONTRAST AGENTS IMPORTANT FOR LIVER MRI IMAGING

Contrast agents, or imaging drugs, are used to improve images taken inside the body by magnetic resonance imaging (MRI), x-rays, computed tomography (CT) and ultrasound.

Contrast agents enable the radiologist to distinguish normal from abnormal tissue, which in turn guides diagnosis, treatment decisions, monitoring and planning. This can ultimately improve a patient’s chances of survival.

MRI in combination with a contrast agent is regarded as the most sensitive diagnostic method for liver imaging.

GADOLINIUM CONTRAST AGENTS NOT SAFE FOR EVERYONE

The heavy metal gadolinium is the active ingredient in currently approved MRI contrast agents. However, patients with severe kidney impairment (defined as reduced kidney func­tion to the degree that the specific measure ‘estimated glomerular filtration rate’ (eGFR) is below 30 mL/min/1.73 m2) are at risk of developing Nephrogenic Systemic Fibrosis (NSF) if they are exposed to gadolinium.

NSF is a rare, but serious and life-threatening condition that causes extensive waxy thickening and hardening of the skin. It can lead to joint contractures and muscle and fascial fibrosis, which may lead to severe immobility. It can also affect the inner organs. NSF worsens over time and can cause death, which typically results from multi-system failure due to sclerotic transformation of organ systems.

Regulatory agencies, including the FDA and EMA, have released warnings about the use of GBCAs and clinical guidelines provide restrictions for the use in patients with severe renal impairment. Therefore, all gadolinium-based contrast agents (GBCAs) have black box warnings in their label for this patient population.

ORVIGLANCE AIMS TO BE THE STANDARD OF CARE

Today, the standard of care for a patient with severe kidney impairment who needs liver imaging is MRI without a contrast agent, also called unenhanced MRI. This significantly reduces the ability to find and treat liver metastases, and ultimately patients’ chances of survival. MRI in combination with a contrast agent is regarded as the most sensitive diagnostic method for liver imaging.

Orviglance aims to be the standard of care for liver imaging of patients with impaired kidney function as it offers high quality imaging compared to an unenhanced MRI.

ORVIGLANCE UPTAKE

Orviglance is an orally administrated contrast agent. It is based on the chemical element manganese, which is a natural trace element in the body. Orviglance also contains L-Alanine and Vitamin D3 to enhance the function of manganese as a contrast agent. After having been absorbed from the small intestine, the manganese is transported to the liver where it is taken up by and retained in the normal liver cells. The high manganese uptake causes the normal liver tissue, also known as hepatocytes, to appear bright on MR images. Metastases and tumor cells do not take up manganese to the same extent as normal liver tissue and therefore appear dark on MR images. Liver metastases are easier to identify due to this contrast effect by Orviglance.

Patient example from phase 2 study

Manganese is excreted from the liver via the bile. The route of administration, uptake and excretion of Orviglance means that only very small amounts of manganese reach the systemic blood circulation.

Watch this video to learn about Orviglance uptake in the liver

CLINICAL DEVELOPMENT SUCCESSFULLY COMPLETED

Clinical development successfully completed Clinical development of Orviglance has been completed with consistent positive efficacy and safety data from nine studies with 286 patients and healthy volunteers.

The pivotal Phase 3 study (SPARKLE) is a global multicenter study, which has been completed with 85 enrolled patients with suspected or known focal liver lesions and severely impaired kidney function.

The evaluation of the primary endpoint was independently carried out by three blinded, independent radiologists (readers), in accordance with regulatory guidance to the industry. The readers assessed changes of visualization of liver lesions with and without Orviglance, as well as other secondary efficacy endpoints.

The pivotal Phase 3 study for Orviglance, SPARKLE successfully met the primary endpoint and demonstrated that Orviglance significantly improved visualization of focal liver lesions compared to unenhanced MRI. The results had high statistical significance (P values <0.001) for all three readers and the reliability of the data was strong and conclusive for all three readers, including an acceptable level of variability.

Common adverse events in this vulnerable patient population were in line with previous studies with Orviglance, such as mild- to moderate nausea. No serious adverse drug reactions were observed.

The Phase 3 study was designed in accordance with industry standards, regulatory guidance for imaging agent development and based on discussions with regulatory agencies. The study aims to support a regulatory filing and approval for use of Orviglance for liver imaging in patients where the use of gadolinium may be medically inadvisable.

Submission of the New Drug Application (NDA) file to the FDA is expected by mid-2025.

Please note that Orviglance is an investigational medicinal product and is not yet approved for use by regulatory authorities in any jurisdiction.

*Trademark is registered in Europe, the US and several other markets.