Asterias Announces Publication of Positive Phase 2 Data on AST-VAC1 for the Treatment of Acute Myeloid Leukemia (AML) in 'Cancer'

FREMONT, Calif., April 26, 2017 /PRNewswire/ -- Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company focused on the emerging field of regenerative medicine, today announced that the results from its completed Phase 2 clinical trial of AST-VAC1 are now available online in Cancer,  a leading peer-reviewed journal of the American Cancer Society. The study publication is available at http://onlinelibrary.wiley.com/doi/10.1002/cncr.30696/full.

"This important publication in the journal Cancer shows why AST-VAC1 has the potential to become an important new therapy for AML patients by safely prolonging the duration of remission in patients with high-risk AML," said Steve Cartt, President and Chief Executive Officer of Asterias. "We believe these Phase 2 results also demonstrate the potential of our AST-VAC2 allogeneic (non-patient specific) dendritic cell cancer vaccine to achieve promising results in its upcoming Phase 1/2a study in non-small cell lung cancer and to potentially be developed for the treatment of other types of cancer, as well."

The publication, titled "Immune Responses and Long-Term Disease Recurrence Status After Telomerase-Based Dendritic Cell Immunotherapy in Patients With Acute Myeloid Leukemia," describes the previously reported results of the Phase 2 study of AST-VAC1, Asterias' patient-specific dendritic cell cancer vaccine, in which 57% of patients who received AST-VAC1 had prolonged relapse-free survival, including high-risk patients over 60 years old or in second remission.

The Phase 2 multicenter, open label trial was designed to evaluate the safety and tolerability of the AST-VAC1 vaccination regimen in patients with intermediate or high risk AML who were in complete clinical remission. Additional objectives of the study were to evaluate the immune responses to AST-VAC1 and to explore the effects of vaccination on relapse in this patient population. Long-term follow-up results showed that 11 out of 19 patients (58 percent) receiving AST-VAC1 during complete remission were relapse-free with a median follow-up of 52 months. In addition, four out of seven patients (57 percent) over the age of 60 remained in remission after a median 54 months of follow-up. Such prolonged relapse-free survival was favorable compared to that previously reported for these patient groups (20-40% overall group and 10-20% for subjects over 60 years old). AST-VAC1 was found to be safe and well-tolerated in this study over multiple vaccinations.

About Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia and has the potential for rapid progression, if untreated. In AML, the bone marrow produces an excess number of immature cells known as blasts. In AML, these blasts fail to mature into normal red and white blood cells. Instead, the blasts proliferate and accumulate in the bone marrow and peripheral blood, leading to deficiencies in normal mature cells. These deficiencies, often referred to as cytopenias, can induce several adverse effects including anemias and susceptibility to infections. Current treatment strategies for AML are associated with significant morbidities and in most instances, AML leads to death.

Approximately 20,500 new cases of AML are diagnosed in the U.S. annually. AML remains a high unmet clinical need, particularly in patients over the age of 60 years who face poor outcomes and have limited therapeutic options. Treatment and prognosis in AML is strongly influenced by a patient's age and tumor profile. Successful treatment and survival of advanced age patients or those with a high risk profile is very poor, with a four year relapse-free survival of 10% – 20% (Rolig et al, 2011). Detailed characterizations of genetic abnormalities associated with AML have elucidated their high number and relative complexity, making development of targeted therapeutics to these mutations very challenging. For this reason, broad immunotherapy approaches such as autologous cell vaccines are particularly promising.

About AST-VAC1

AST-VAC1 is a cancer immunotherapy, consisting of autologous mature antigen-presenting dendritic cells pulsed with a messenger RNA for the protein component of human telomerase (hTERT) and a portion of a lysosomal targeting signal (LAMP). hTERT is a common protein in tumor cells and is responsible for the increased proliferative lifespan of cancer cells. In AST-VAC1, the dendritic cells present telomerase to the immune system to induce T cells to target and kill hTERT-expressing tumor cells. The LAMP signal allows AST-VAC1 to stimulate both cytotoxic and helper T cell responses to telomerase, critical elements to induce and maintain immune responses that kill tumor cells. Because of the widespread expression of telomerase in the majority of cancers, AST-VAC1 is a platform immunotherapeutic that could be used alone or in conjunction with other therapeutics such as immune checkpoint inhibitors to target immune-based destruction of tumors.

About Asterias Biotherapeutics

Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.   

FORWARD-LOOKING STATEMENTS

Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

SOURCE Asterias Biotherapeutics, Inc.

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