DefinedHealth

Aileen Nicoletti, PhD

Therapeutic vaccines are in the spotlight largely due to the maturing data for products in development for oncology, the first testing ground for this approach. The high profile approvable verdict by the FDA on Provenge (after a 13-4 vote of the Cellular, Tissue and Gene Therapies Advisory Committee affirmed that “substantial evidence of efficacy” in asymptomatic, metastatic, androgen-independent prostate cancer patients had been demonstrated) highlights some of the challenges associated with developing products using this novel, complex immunological approach. Therapeutic vaccines in the clinic reach across virtually all major therapeutic categories. In addition to oncology, the more advanced programs are aimed at hypertension, Alzheimer’s disease, smoking cessation and diabetes. Proof-of-concept already has been established for hypertension, where Cytos has shown reductions in blood pressure comparable to oral therapy in patients receiving its angiotensin II vaccine, with even greater benefit in early morning blood pressure control.

With both the potential to replace or augment oral standards of care in some indications (e.g., hypertension) and to provide unique benefits in areas of great unmet need (e.g., Alzheimer’s), therapeutic vaccine approaches represent a hot area for deal making, and big pharma (Roche, Novartis, Merck, Wyeth, and Pfizer) is making serious investments in the space. A recent Pfizer analyst presentation refers to therapeutic vaccines as the “follow on to the monoclonal antibody segment in oncology, Alzheimer’s disease and asthma,” a tall order for a platform that is highly variable in its approach (DNA, protein, ex vivo manipulation and even RNA), dosing and administration regimens, and desired outcomes (tolerizing vs. immunostimulating). That said, the potential for blockbuster success clearly exits: imagine controlling dyslipidemia with a once-per-year vaccination against LDL rather than with a daily statin.

Aside from the technical challenges, abundant commercial and regulatory conundrums have to be addressed: what price will these support; are issues with compliance for an injection versus a daily oral better or worse; how to address logistical hurdles for cell-based vaccine approaches that involve a processing side (e.g., many of the cancer vaccines); and importantly, where is the safety bar for diseases that can be managed with orals and where a vaccine might obviate the ability to take patients off the therapy if necessary? We invite you to join us as we examine therapeutic vaccine approaches now in the clinic and to brainstorm with us and your senior level industry peers regarding the challenges associated with developing these products and evaluating their potential to impact standards of care and change treatment paradigms in a variety of therapeutic areas.