Accentia Biopharmaceuticals, Inc. said its majority owned-subsidiary, Biovest International, Inc, reported achievement of a key milestone in Biovest's mission to gain approval for BiovaxID, its personalised anti-cancer vaccine, in a pivotal phase III Fast-Tracked clinical trial for the treatment of indolent follicular non-Hodgkin's lymphoma.
Biovest reported that the independent Data Monitoring Committee (DMC), which is vested with the responsibility of monitoring the safety and efficacy of the BiovaxID trial, has completed a closed session review of the unblinded results, including the primary and secondary endpoints analysis of the phase III study. Based on their analysis, the DMC has recommended that the results be unblinded, and has volunteered to participate, if needed, in Biovest's ongoing regulatory meetings with the US Food and Drug Administration (FDA) and other worldwide regulatory agencies (Health Authorities) in order to determine the most appropriate process for unblinding the results, and establishing a pathway to seek marketing approvals.
Accentia holds an approximate 76 per cent ownership stake in Biovest, owning more than 73 million shares. Accentia also maintains a 19.5 per cent royalty interest in global sales of BiovaxID.
"Our committee has completed a closed session unblinded review of the interim phase III safety and efficacy analyses regarding the BiovaxID registration study. Based on this review, the DMC recommends that the BiovaxID study be unblinded to the company for final analysis. We further suggest that the final clinical study report include data, as part of Biovest's overall regulatory strategy of submission to the regulatory authorities, through August 29, 2008, which is the date when the last randomised and vaccinated patient reaches 12-months follow-up from first injection," said Dr. Gerald Messerschmidt, chairman, DMC. "While the DMC cannot release information on our closed session discussions, our recommendation to unblind this pivotal registration study in late-August is based on the status of the study, the defined safety profile over 8-years of this phase III registration study and our review of the primary and secondary endpoint analyses. As part of the ongoing regulatory process, I as a representative of the DMC will relate our review of the closed session analysis of these interim data to Health Authorities as necessary."
As previously presented to the American Society of Haematology (ASH) and the American Society of Clinical Oncology (ASCO), the National Cancer Institute (NCI) demonstrated in a Phase 2 clinical trial that patients treated with BiovaxID achieved: a median disease-free survival of over eight years; a 95 per cent overall survival rate after 9.2 years of follow-up; and a 95 per cent cellular immune response. In this clinical trial, 73 per cent of evaluable patients were converted into molecular remission (bcl-2 negative), evidence that their blood has been cleared of residual cancerous cells. BiovaxID also demonstrated an outstanding safety profile.
Dr Steven Arikian, chairman and CEO, Biovest said, "The DMC's diligent review has resulted in an excellent outcome for BiovaxID. Not only has the Committee reconfirmed the favourable safety profile for BiovaxID, but as importantly, we believe their recommendation to unblind and their willingness to meet with the regulatory authorities to discuss their findings suggests a successful trial outcome. In light of the DMC's recommendation, we have already been in discussions with regulatory agencies, including the FDA, to seek consensus on the appropriate process for unblinding the results and establishing a pathway for potential accelerated and/or conditional approvals for BiovaxID.
"We also are coordinating with the EMEA regarding potential market access in Europe. If approved, Biovest will have distinguished itself as the first company to have its anti-cancer vaccine reach market in the US or EU, a Herculean achievement. As this study was initiated in 2000, we now have the largest longitudinal database of lymphoma patients receiving a personalized therapeutic vaccine. 234 patients were enrolled and 177 randomized in the Phase 3 trial of which, two-thirds received BiovaxID therapy, and one-third control treatment. We believe that these long-term and highly anticipated results will demonstrate that BiovaxID is capable of prolonging the period of disease-free survival for an incurable form of non-Hodgkin's lymphoma, and in some cases, perhaps even inducing indefinitely prolonged, durable remissions," he said.
The primary end-point of the trial is a comparison between treatment groups, those that receive BiovaxID and those that receive a control. The study is looking at duration of disease-free survival measured from the time of randomization (i.e. when a subject is randomly assigned to receive BiovaxID or control) to the time of confirmed relapse.
BiovaxID is a personalised, patient-specific therapeutic vaccine designed to stimulate the patient's own immune system to recognize and destroy cancerous B-cells that may remain in the body or may arise after the patient has been treated with chemotherapy. Unlike many other approaches to treating non-Hodgkin's lymphoma, BiovaxID is designed to kill only cancerous B-cells, with the initial indication of follicular Non-Hodgkin's lymphoma. Additionally, we anticipate that BiovaxID could potentially be used to treat other types of B-cell cancers, such as Mantle Cell Lymphoma, Chronic Lymphocytic Leukaemia and Multiple Myeloma.