GVAX Cancer Vaccines

Cancer vaccines are an increasingly exciting option for cancer treatment, especially after the FDA approval of Provenge for the treatment of prostate cancer in 2010. BioSante Canadian Pharmaceuticals Inc. owns exclusive development and commercial rights in a pipeline of immunotherapies (cancer vaccines). BioSante's GVAX Cancer Vaccines use cell lines that are genetically modified to secrete granulocyte-macrophage colony-stimulating factor (GM-CSF), an immunostimulant. The cells then are irradiated to prevent further cell division; however the cells remain metabolically active.

Currently, GVAX Cancer Vaccines are in various Phase I and Phase II cancer clinical trials and may represent the widest portfolio (cancer types) of cancer vaccines in development. Studies are conducted primarily at Johns Hopkins Cancer Center and are sponsored/funded by The Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, various philanthropies and foundations. BioSante has entered various licenses including with Aduro BioTech and the Hussman Foundation for development and commercialization of certain of the vaccines (see Partners & Licenses).

BioSante GVAX Cancer Vaccine Presentation Video

Cancer Types Being Studied:

  • Pancreatic Cancer
  • Leukemia
  • Chronic Myeloid Leukemia (CML)
  • Acute Myeloid Leukemia (AML)
  • Multiple Myeloma
  • Breast Cancer
  • Prostate Cancer
  • Colorectal
  • Melanoma

Development / Regulatory Status

Pancreatic Cancer — Results of Phase Ib

Results from a Phase Ib clinical study that show BioSante’s GVAX Pancreas cancer vaccine increased the median survival of pancreatic cancer patients with previously treated, locally advanced or metastatic pancreatic adenocarcinoma (PDA), from 3.3 months when treated with ipilimumab (IPI; Yervoy; BMS), to 5.5 months on the combination of IPI plus GVAX Pancreas, an increase of more than 60 percent. The study was not powered for a direct comparison. In addition, the IPI/GVAX Pancreas combination demonstrated an increase in one year survival, from 7 percent to 27 percent.

A poster titled, “Phase 1b Study of Ipilimumab Alone or in Combination with Allogeneic Pancreatic Tumor Cells Transfected with a GM-CSF Gene in Pancreatic Cancer,” was presented at the 2012 Gastrointestinal Cancers Symposium by Dung T. Le, et al, lead investigator at Johns Hopkins University. The abstract of the poster is in the Journal of Clinical Oncology 30, 2012 (suppl 4; abstr 211). The primary endpoint was to determine the safety profile of IPI alone or in combination with GVAX Pancreas and the secondary endpoints were overall survival and toxicity, and the induction of mesothelin specific T cell responses. Toxicities were manageable. In addition, the postimmunotherapy induction of mesothelin-specific T cells in patients correlates with disease-free survival. The investigators concluded that, “Immunotherapy has potential even in advanced PDA.”

The 30 patient Phase Ib study was conducted by researchers at the Sidney Kimmel Cancer Center of Johns Hopkins University School of Medicine and the Bloomberg School of Public Health at Johns Hopkins, in Baltimore, Maryland. BioSante’s GVAX Pancreas cancer vaccine is made from allogeneic pancreatic cancer cells genetically modified to produce an immune system stimulator, the cytokine GM-CSF, and irradiated to prevent cell growth.

Pancreatic Cancer — Results of Phase II

Phase II clinical study that showed BioSante's GVAX Pancreas Cancer Vaccine increased the median survival of resected pancreatic cancer patients from 15 to 20 months, as reported in published data, to 24.8 months, an increase of more than 25 percent. In addition, the vaccine demonstrated a 35 percent increase in one year survival, from 63 percent to 85 percent. A new multicenter clinical study is planned to begin this year.

In the study, titled, "A Lethally Irradiated Allogeneic GM-CSF-Secreting Tumor Vaccine for Pancreatic Adenocarcinoma: A Phase II Trial of Safety, Efficacy, and Immune Activation," published in the February 2011 issue of Annals of Surgery, the primary endpoint was disease free survival and secondary endpoints were overall survival and toxicity, and the induction of mesothelin specific T cell responses. The median disease-free survival of patients was 17.3 months (95% CI, 14.6–22.8) with median survival of 24.8 months (95% CI, 21.2–31.6). The administration of immunotherapy was well tolerated. In addition, the postimmunotherapy induction of mesothelin-specific T cells in patients correlates with disease-free survival. The investigators concluded that "an immunotherapy approach integrated with chemoradiation is safe and effective for resected pancreas cancer."

The 60 patient Phase II study was conducted by researchers at the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center in Baltimore, Maryland. The research was funded by the National Institutes of Health and other foundations. BioSante's GVAX Pancreas Cancer Vaccine is made from allogeneic pancreatic cancer cells genetically altered to produce an immune system stimulator called GM-CSF, and irradiated to prevent cell growth.

Chronic Myeloid Leukemia (CML) — Results of a Phase II clinical trial

Of 19 patients treated with BioSante CML, 7 had complete remission.

  • Gleevec was taken for at least one year (range 13-53 months) pre-vaccine and then BioSante's GVAX CML was administered while the patients remained on a stable dose of Gleevec.
  • 19 patients enrolled (median of 72 months of follow-up)
  • 13 patients (8 of whom had increasing disease burden before vaccination): the number of remaining cancer cells declined
  • 12 patients reached their lowest levels of residual cancer cells to date following vaccination
  • 7 patients had complete remission

"We want to get rid of every last cancer cell in the body, and using cancer vaccines may be a good way to mop up residual disease," said lead investigator Hyam Levitsky, M.D., professor of oncology, medicine and urology at the Johns Hopkins Kimmel Cancer Center in Baltimore, Maryland.

Acute Myeloid Leukemia (AML) — Results of Phase II

Patients treated with BioSante's GVAX AML had both a relapse-free survival and overall survival rate improvement of approximately 30% compared with the non-BioSante-treated subjects.

  • GVAX AML was accompanied by immunotherapy-primed lymphocytes after autologous stem cell transplantation.
  • 54 subjects enrolled
  • 28 (52%) received a pre-transplantation GVAX AML dose
  • 46 (85%) subjects achieved complete remission during the treatment period. Of these patients, the 3-year relapse-free survival (RFS) rate was:
  • 47.4% in non-GVAX treated patients
  • 61.8% in the GVAX-treated group
  • Overall survival (OS) rate in all subjects was 57.4%
  • Overall survival (OS) rate was 73.4% in the BioSante-treated group
  • In summary, patients treated with GVAX AML had both a relapse-free survival and overall survival rate improvement of approximately 30% compared with the non-GVAX-treated subjects.

Prostate Cancer - Results of Phase I

Results from a Phase I dose escalation clinical study that show BioSante’s GVAX Prostate cancer vaccine in treating metastatic castration-resistant prostate cancer patients (mCRPC) in combination with ipilimumab (Ipi; Yervoy; BMS), resulted in 53 percent of patients achieving at least stable disease, with two patients showing clear regression of metastases. In addition, 23 percent of patients in the higher dose cohorts had confirmed partial PSA (prostate specific antigen) responses of greater than 50 percent from baseline.

The study, titled, “Combined Immunotherapy with Granulocyte-Macrophage Colony-Stimulating Factor-Transduced Allogeneic Prostate Cancer Cells and Ipilimumab in Patients with Metastatic Castration-Resistant Prostate Cancer: a Phase 1 Dose Escalation Trial” was published in February 2012 issue of The Lancet Oncology. The study combined fixed doses of GVAX Prostate in combination with escalating 0.1, 0.3, 1.0, 3.0 and 5.0 mg/kg doses of Ipi. The study enrolled 12 patients in three dose escalation cohorts and 16 patients in an expansion Ipi 3.0 mg/kg cohort. The reported duration of disease stabilization ranged from 3-27 months, and the median duration of PSA response was 12 months, (range 2-21 months). There were no serious adverse events in the lower dose Ipi cohorts and administration of immunotherapy was well tolerated and safe. The investigators concluded that, “Further research on the combined treatment of patients with mCRPC with vaccination and Ipilimumab is warranted.”

The 28 patient Phase I study was conducted by researchers at the VU University Medical Centre in Amsterdam, Netherlands. BioSante’s GVAX Prostate cancer vaccine is made from allogeneic prostate cancer cells genetically altered to produce an immune system stimulator called GM-CSF, and irradiated to prevent cell growth.

BioSante Orphan Drug Designation

BioSante has received FDA Orphan Drug designation for:

  • GVAX Pancreas vaccine to treat pancreatic cancer
  • GVAX AML vaccine to treat acute myeloid leukemia
  • GVAX CML vaccine to treat chronic myeloid leukemia
  • GVAX Melanoma vaccine to treat melanoma

The Orphan Drug Act (ODA) provides for granting special status to a product to treat a rare disease or condition upon request of a sponsor

  • The disease or condition for which the drug is intended affects fewer than 200,000 people in the United States or,
  • If the drug is a vaccine, the persons to whom the drug will be administered in the United States are fewer than 200,000 per year

Advantages of Orphan Drug Designation

  • Tax credits
  • Seven years of marketing exclusivity
  • Products approval submissions not subject to a prescription drug user fee
  • Products approved not subject to discounting under new healthcare law
  • Orphan Drug grants available

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