Imugene (ASX: IMU) has landed a prized United States Food and Drug Administration (FDA) orphan drug approval for its bile tract cancer therapy following positive results from its recent metastatic advanced solid tumour (MAST) trials to treat the rare and insidious disease known as cholangiocarcinoma.
The FDA has granted orphan drug designation (ODD) to the company’s novel oncolytic virotherapy, “VAXINIA”. The ODD provides a range of incentives for Imugene, including market exclusivity for seven years, tax credits, potential grant funding and a waiver on various administration fees.
Management says its application was supported by pre-clinical and clinical data from the company’s phase-one MAST trial. It has now also initiated a bile tract cancer expansion trial, with 10 patients to be enrolled in the program.
The trials are investigating the safety and efficacy of VAXINIA, which has already produced a stunning complete response in one patient and a stabilising of the disease in another.
A complete response is when all signs of the disease have disappeared in response to the treatment undertaken. However, it does not infer that the patient is completely cured of the cancer.
The company says the aggressive bile tract disease is a solid cancer with a high unmet clinical need. Its MAST trial delivers VAXINIA as either a stand-alone monotherapy, or in conjunction with another inhibitor.
It added that the trial is currently in a dose-escalation phase.
Receiving Orphan Drug Designation from the FDA is a major milestone for us. It reflects the potential of VAXINIA to address the urgent need for new treatments for cholangiocarcinoma, a disease with limited therapeutic options. We are excited to continue advancing this program, which has already shown meaningful clinical responses in patients.
Imugene Managing Director and CEO Leslie Chong
The FDA grants ODD status to promote the development of drugs aimed at treating rare diseases that affect fewer than 200,000 people in the US. The range of incentives provided by the FDA assists companies to pursue the types of diseases found in fewer numbers, rather than simply chase those with huge returns on offer for successful therapies.
Cholangiocarcinoma is a rare malignancy in bile ducts and is often resistant to normal treatments, including chemotherapy and immunotherapy. VAXINIA has been designed to target and destroy cancer cells and generate an immune response against the tumour.
Bile ducts, or tracts, are tiny canals that connect some of the organs in a person’s digestive system. Their purpose is to carry bile between the organs.
A tube carries bile from the liver and gallbladder, through the pancreas, and into the small intestine. The common bile duct starts where the ducts from the liver and gallbladder join and ends at the small intestine.
Imugene’s goal is to develop a range of immunotherapies that activate a cancer patient’s own immune system to shape up to the tumours and deliver a knockout blow. And now in many instances, immunotherapy has helped a patient’s immune system take the fight to the cancer and win the contest.
Imugene is continuing along that path and looking to prove that VAXINIA can punch on with the toughest of bile tract cancers and emerge victorious.
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