Jennerex Biotherapeutics, Inc., a private, clinical-stage biotechnology company, has received the orphan drug designation from the United States Food and Drug Administration (FDA) for Pexa-Vec (JX-594, pexastimogene devacirepvec) for the treatment of hepatocellular carcinoma (HCC, commonly referred to as liver cancer). Pexa-Vec, Jennerex's lead product candidate, is an oncolytic immunotherapy designed to rapidly de-bulk tumours via tumour cell lysis, induce a systemic anti-tumour immune response, and selectively target tumour vasculature resulting in a rapid reduction in tumour blood flow.

The Pexa-Vec clinical programme in liver cancer includes TRAVERSE, a global phase IIb trial in advanced HCC patients who have failed sorafenib therapy.

"We are pleased to receive FDA orphan drug designation for Pexa-Vec. This designation is an important step in the development of Pexa-Vec as a potential treatment for this lethal disease," said Laurent Fischer, MD, president and chief executive officer of Jennerex. "We look forward to announcing top-line data from our TRAVERSE trial by the end of this year and to bring Pexa-Vec to the market as soon as possible."

The FDA provides orphan drug designation to drugs that seek to treat rare diseases or conditions for which there may be few adequate therapies. Orphan drug designation is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States. The designation will provide Jennerex with the opportunity of seven years of marketing exclusivity, grant funding to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential waiver of the FDA's application user fee. Jennerex received orphan drug designation on Pexa-Vec for the treatment of HCC from the European Medicines Agency (EMA) in 2009.

Hepatocellular carcinoma is the fifth most common cancer worldwide and the third leading cause of cancer death. Currently, there are few approved treatment options for advanced HCC patients.


Pexa-Vec was engineered from vaccinia vaccine, which has been used for decades as a vaccine in healthy individuals. Pexa-Vec was also engineered to express GM-CSF, a white blood cell growth factor, which activates a systemic immune response to kill tumour cells throughout the body. Pexa-Vec exploits the unique characteristics of vaccinia, including its stealth extracellular envelope form, which allows the virus to survive in the bloodstream in the presence of neutralizing antibodies, leading to its ability to be administered both intravenously (IV) and intratumorally (IT). Unlike many targeted therapies that rely on a single target, Pexa-Vec is applicable to multiple solid tumour targets.

Pexa-Vec is currently being evaluated in an international, randomized phase IIb clinical trial (TRAVERSE) in patients with advanced HCC who have failed sorafenib therapy. It is also being tested as monotherapy in sorafenib-naïve HCC patients and in combination with sorafenib. In addition, Pexa-Vec is being evaluated in a phase I-II clinical trial in patients with treatment-refractory colorectal cancer as monotherapy and in combination with irinotecan, and in a phase IIa clinical trial in treatment-refractory kidney cancer patients.

Phase I and phase II clinical trials in multiple cancer types to date have shown that Pexa-Vec, delivered either directly into tumours or intravenously, induces tumour shrinkage and/or necrosis and is well-tolerated (over 250 patients treated to date). Objective tumour responses have been demonstrated in a variety of cancers including liver, colon, kidney, lung cancer and melanoma. Pexa-Vec has had a predictable and manageable safety profile to date which includes flu-like symptoms that typically resolve in 24 hours.

Pexa-Vec is the lead product candidate from Jennerex' SOLVE platform, a groundbreaking approach offering new therapeutic options for patients with life-threatening cancers that can be injected directly into tumor tissue or administered systemically by infusion. Pexa-Vec is partnered in Europe with Transgene, a member of the Institute Merieux group, In South Korea with Green Cross and in China with Lee's Pharmaceuticals.

Jennerex Biotherapeutics, Inc. is focused on the development and commercialization of first-in-class, breakthrough targeted oncolytic immunotherapy products for solid tumours.