PAN-301-1 is a cancer vaccine being developed by Panacea Pharmaceuticals to treat prostate cancer that is resistant to other treatments. It targets HAAH, a protein that is not normally found in healthy cells but is found in many different types of cancer cells.

How PAN-301-1 works

HAAH or human aspartyl (asparaginyl) B hydroxylase, is an enzyme that plays a role in the development of the fetus but is not usually found in cells after birth. However, a wide variety of solid and blood cancer cells have HAAH, and its presence is associated with a worse prognosis. HAAH helps cancer cells grow, move, and invade other areas of the body.

PAN-301-1 is a vaccine that exposes the immune system to HAAH, with the aim of stimulating an immune response that targets HAAH. It uses a neutralized bacteriophage, a type of virus that normally infects bacteria but is harmless to humans. The bacteriophage presents hundreds of copies of HAAH protein fragments on its surface, producing a HAAH-specific antibody response and provoking the immune system to attack and destroy HAAH targets on cancer cells.

Preclinical studies have shown that the PAN-301-1 vaccine can slow the growth and spread of cancer, and increase survival in treated animals compared to untreated animals.

PAN-301-1 in clinical trials

In January 2017, Panacea announced that it had enrolled and dosed a first patient in an open-label, multi-center Phase 1 study (NCT03120832) to test the safety of PAN-301-1 in men with persistent prostate cancer.

Patients in this study have cancer that has returned after successful treatment, determined by the levels of a protein called prostate-specific antigen (PSA). PSA levels are used to monitor patients who have had prostate cancer, and raised levels can be a sign of the cancer returning.

Dose escalation will be used to determine the highest dose that can be safely given to patients, and to establish an appropriate dose for future Phase 2 trials. The trial will also assess whether and how much PAN-301-1 is needed to trigger an immune response specifically against HAAH, a measure called immunogenicity.

The trial is still recruiting about 18 prostate cancer patients at sites in Alabama, California, Nebraska, and South Carolina. It expects to conclude in December 2017.

Additional information

PAN-301-1 is given as an intradermal injection, which means it is injected into the skin and does not require time-consuming infusions.

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