July 18, 2008

Biovest/Accentia’s BiovaxID Revives Hope for Therapeutic Cancer Vaccines

Biovest, a subsidiary of Accentia, has announced positive data from a pivotal trial of BiovaxID in lymphoma, and the vaccine now stands a good chance of becoming the first therapeutic cancer vaccine to gain approval in the seven major markets. However, despite this breakthrough, a number of economical and logistical issues could prevent the vaccine from achieving significant market penetration.

Biovest's BiovaxID prolonged disease-free survival in patients with follicular lymphoma, meeting the primary endpoint of a pivotal Phase III trial. In the randomized, double-blinded placebo-controlled trial, 629 follicular lymphoma patients in first remission received either injection with BiovaxID plus granulocyte macrophage colony-stimulating factor (GM-CSF), or injection with keyhole limpet hemocyanin (KLH), which acted as the placebo, plus GM-CSF.

The median disease-free survival in patients who received BiovaxID was 33.8 months, an increase of one year compared to patients in the control arm, whose disease-free survival was 21.2 months. Three years after injection, the rate of disease-free survival was approximately 100% higher in the BiovaxID arm. Both of these results reached statistical significance.

BiovaxID is a personalized therapeutic vaccine that combines a tumor-specific idiotype protein, derived from the patient's lymph node biopsy tissue, and KLH, a protein carrier. Biovest, a majority-owned subsidiary of Accentia, is developing the drug, and now intends to seek accelerated and/or conditional approvals in the US and EU for follicular lymphoma.

Follicular lymphoma is one of the most common types of non-Hodgkin's lymphoma (NHL), with a forecast incidence of approximately 30,000 in the seven major pharmaceutical markets in 2008. Current treatment options for the disease are rarely curative, and nearly all patients eventually suffer relapse following first-line therapy. Median overall survival is typically between eight and 10 years.

The positive data reported for BiovaxID are surprising, following the failures of several therapeutic vaccines in recent years. Notably, two other personalized therapeutic vaccines in Phase III development for NHL, Favrille's Specifid and Genitope's MyVax, failed to meet primary endpoints this year. This clears the way for BiovaxID to become the first therapeutic vaccine to reach the market for NHL, assuming it gains approval. Moreover, the vaccine could be the first of its type to gain approval in the seven major pharmaceutical markets for any type of cancer.

However, several hurdles stand between BiovaxID and commercial success, even assuming it gains approval in either the US or EU. The manufacturing process for the vaccine lasts approximately six months, which is likely to increase its cost and put it out of reach of more cost-conservative healthcare budgets. Additionally, the need to prepare the vaccine individually from each patient's biopsy tissue and to ship it frozen once prepared could present significant distribution problems, preventing mass-scale production.

The commonplace use of Rituxan (rituximab; Biogen Idec/Genentech/Roche) as a maintenance therapy for follicular lymphoma could further limit uptake of BiovaxID. It is questionable whether Biovest or Accentia have the capabilities to overcome such difficulties, and the vaccine could struggle to penetrate the NHL market without the help of a larger player.