PVX-410, OncoPep’s lead program, is a novel therapeutic cancer vaccine that contains four proprietary peptides designed to specifically target antigens found on the surface of multiple myeloma (MM) cells and was granted orphan drug designation from the U.S. Food and Drug Administration in 2013. OncoPep is initially developing PVX-410 to target a precursor disease, called smoldering multiple myeloma (SMM). Pre-clinical research, conducted by Dana Farber Cancer Institute on MM cells in vitro, indicates that this unique combination of peptides may elicit an immune response to targeted antigens on MM cells.

Importantly, SMM has the potential to progress to full-blown MM. OncoPep believes that PVX-410 may potentially represent a future therapeutic option for patients with SMM. The safety and tolerability of PVX-410, alone and in combination with lenalidomide, are currently being evaluated for the treatment of patient with SMM in an open label, dose escalation, Phase 1/2a clinical trial. Lenalidomide, which is commercially available for the treatment of MM, is an immunomodulatory agent that may complement the mechanism of action of PVX-410. It has demonstrated clinical benefit when combined with dexamethasone in a Phase 3 clinical study treating patients with SMM.

While the primary endpoint of OncoPep’s clinical trial is evaluating safety, secondary endpoints are measuring immune and clinical response to the vaccine.

In a dose escalation arm of the study, patients received either a 0.4 mg or 0.8 mg dose of PVX-410 over 10 weeks in six bi-weekly subcutaneous injections along with the adjuvant poly ICLC. Patients receiving only PVX-410 completed active treatment and are currently in follow up.

Patients who currently enroll in the trial are assigned to a second arm, in which patients receive PVX-410, adjuvant and lenalidomide. These patients concurrently receive the 0.8 mg dose of PVX-410 over 10 weeks and three, 21-day courses of orally administered lenalidomide.

The treatment centers include the cancer centers at Massachusetts General Hospital (Boston), Dana Farber Cancer Institute (Boston), Beth Israel Deaconess Hospital (Boston), MD Anderson Cancer Center (Houston) and Winship Cancer Institute of Emory University (Atlanta). More information on this study can be found at www.clinicaltrials.gov (Identifier: NCT01718899).