FDA accepts Adaptimmune’s NDA for affinity enhanced T-Cell therapy targeting AFP in liver cancer

The IND is now active.

This will be Adaptimmune’s second wholly-owned therapeutic candidate to enter clinical trials. The company initiated a study in December 2015 to evaluate its unpartnered T-cell therapy targeting the MAGE-A10 cancer antigen in patients with non-small cell lung cancer.

The acceptance of this IND allows Adaptimmune to initiate an open label Phase I study designed to evaluate the safety and anti-tumor activity of its AFP therapeutic candidate in hepatocellular carcinoma (HCC). Site selection activities are under way, and the company anticipates that enrollment will commence in the second half of 2016.

"Hepatocellular carcinoma is one of the most common and deadly types of cancer in the world and it represents a significant unmet medical need, as there is a dearth of effective therapies for advanced disease," said Rafael Amado, Adaptimmune’s Chief Medical Officer.

"We are pleased to initiate clinical evaluation of our AFP T-cell therapeutic candidate in this patient population."

AFP is believed to be highly expressed in approximately 30 percent of hepatocellular carcinomas. Expression has been shown to be absent or very low in most adult non-reproductive tissues and will be evaluated prior to enrollment.

Adaptimmune’s proprietary technology enables the company to routinely generate TCRs which address intracellular targets, such as AFP, that are not accessible to other therapies.

This will be a Phase I open label clinical trial evaluating the safety and anti-tumor activity of autologous T-cells expressing enhanced TCRs specific for AFP in HLA-A2 positive subjects with advanced HCC.

The study will enroll up to 30 subjects with measurable, histologically confirmed HCC, not amenable to resection or loco-regional therapy, and progressive disease following (or intolerant of or refuses) sorafenib treatment. The primary objective of the study is to evaluate the safety and tolerability of this therapy in subjects with AFP-positive HCC.

Additional objectives include anti-tumor activity, persistence of genetically modified cells in the body, and evaluation of the phenotype and functionality of genetically modified cells isolated from peripheral blood or tumor post infusion.

About Adaptimmune’s TCR Technology

Adaptimmune’s proprietary T-cell receptor (TCR) technology enables the company to genetically optimize TCRs in an effort to equip them to recognize and bind cancer antigens that are presented in small quantities on the surface of a cancer cell, whether of intracellular or extracellular origin, thus initiating cell death.

The company’s differentiated, proprietary technology allows it to reliably generate parental TCRs to naturally presented targets, affinity optimize its TCRs to bind cancer proteins from solid and hematologic cancers that are generally unavailable to naturally occurring TCRs, and to significantly reduce the risk of side effects resulting from off-target binding of healthy tissues.

About Hepatocellular (Liver) Cancer

A cancer that starts in the liver is called primary liver cancer. Hepatocellular cancer is the most common form of liver cancer in adults. More than 700,000 people are diagnosed with this cancer each year throughout the world. Liver cancer is also a leading cause of cancer deaths worldwide, accounting for more than 600,000 deaths each year.

In the United States, it is estimated that about 39,000 new cases will be diagnosed in 2016, and about 27,000 people will die from it. Liver cancer death rates have generally been increasing since 1980; from 2003 to 2012, rates increased by 2.7 percent per year. It is often hard to detect liver cancer early because signs and symptoms often do not appear until the disease is in its later stages.