NeoStem
420 Lexington Avenue
Suite 350
New York, New York 10170
Telephone: 1.212.584.4180

Target: Malignant Melanoma

About Melanoma

Melanoma, which originates in pigment-producing cells known as melanocytes, is the most lethal form of skin cancer. Melanoma is often caused by unrepaired DNA damage to skin cells from UV radiation. Patients who have progressed to stage IV melanoma have a cancer that has metastasized, or spread to distant sites in the body which may include the lymph nodes, lungs, liver, or brain. As a result, advanced melanoma is exceedingly difficult to treat, with a 5-year survival rate of approximately 15%1. There are an estimated 100,000 new cases per year in the U.S. of stage IV metastatic melanoma and melanoma kills approximately 10,000 in the U.S. annually2.

Treatments for stage IV melanoma are typically directed at slowing the growth of the cancer and prolonging survival. Current treatment options include radiation, chemotherapy, surgical resection, immunotherapy, or a combinatory approach. Unfortunately, most current approaches provide only temporary relief, with the metastatic cancer returning 3 to 6 months after treatment. Administering certain anti-melanoma drugs at higher doses appears to be more effective, but often results in more severe side effects.

In recent years immunotherapy and targeted drugs have emerged as promising approaches to treating advanced cancer. NeoStem’s lead product candidate, NBS20 (USAN: eltrapuldencel-T), targets malignant melanoma

NeoStem’s Phase 3 Intus Study of NBS20                  

NeoStem has received U.S. FDA approval to commence a Phase 3 trial assessing the efficacy of its patient specific cancer immunotherapy NBS20 (irradiated autologous in vitro proliferating melanoma cell line loaded onto an autologous dendritic cell combined with granulocyte macrophage colony-stimulating factor {GM-CSF}) in patients with late stage metastatic melanoma. The study has Special Protocol Assessment (SPA) and Fast Track designation, and the therapy has been awarded Orphan Drug designation. For additional details, please visit the trial’s listing on clinicaltrials.gov.

Patient enrollment for NeoStem’s Phase 3 Intus Study is now open. If you are interested in learning more about the study, please visit www.theintusstudy.com or the trial’s listing on clinicaltrials.gov.

About NBS20

NBS20 is an autologous initiating cell immune based therapy intended to eliminate the tumor cells capable of causing disease recurrence. NeoStem’s immunotherapeutic approach uses the patient’s isolated and purified tumor stem cells to train the immune system (Killer T-cells, CD8+) to identify and eliminate cancer stem cells, the root cause of tumor formation and the key drivers of tumor escape, tumor genesis, self-renewal and recurrence of cancer. We believe this platform technology is applicable for a broad spectrum of solid tumor cancers.

Phase 2 Results

In a Phase 2 randomized clinical trial of formulation comparable to NBS20, patients showed a significant improvement in two year overall survival from 31% in controls to 72% in treated patients (p=0.007) with advanced melanoma (Stage IV and recurrent Stage III). The toxicity profile was favorable with no grade IV and only one grade III (allergic reaction) event in the study. View trial results.

Facts about NBS20

  • NBS20 is intended to be given after surgery, radiation, chemotherapy, targeted therapies, or other immunotherapies that decrease the total cancer volume, but do not eliminate the cancer initiating (stem) cells or progenitor cells.  The treatment can also be given prior to other treatments.
  • Unlike approved and many experimental dendritic cell therapies which are simply a means of presenting to the patient’s immune system the broad array of antigens associated with the cancer stem cells, NeoStem’s therapy is designed to specifically expose the immune targets of the cancer initiating (stem) cells that make cancer lethal. This focuses the immune attack on the specific elements of the tumor that enable it to grow and metastasize, which is not the case with currently available approaches.
  • In the candidate’s randomized Phase 2 trial, survival was better in patients whose immune system was exposed to antigens presented by the exogenously expanded dendritic cells, rather than just by injecting the irradiated cancer cells themselves.
  • Patients may be enrolled in the study regardless of prior therapy, they are not restricted in terms of therapy at the time they would be receiving NBS20 and there is no subsequent restriction after they have completed that therapy. The key entry criterion is that the patient must have had a tumor resected from which a stem cell has been able to establish the self-renewing cell line.
Summary of Intus Study
Design Single randomized, double blind, placebo controlled trial for stage III recurrent or stage IV metastatic melanoma for registration
Primary Endpoint Overall Survival
Powering 80% power to detect 37.5% reduction in risk of death
Relation to standard therapies Adjunctive (clinical practice based trial)
Study size Planned 250 eligible patients across approximately 50 sites (US, Canada, Australia, New Zealand)
Treatment Autologous dendritic cells loaded with antigen from proliferating autologous tumor cells + GM-CSF
Control Peripheral blood mononuclear cells obtained from pheresis product + GM-CSF

 

  1. AJCC Cancer Staging 2010 (based on 17 academic centers)
  2. National Cancer Institute – 2014 SEER
NeoStem - Cell Therapy Development - Clinical