Since May is Melanoma Awareness month, it seems fitting to take a look at new advancements taking place in the fight against skin cancer. The U.S. melanoma market is approximately $1 billion per year, which is money predominantly spent to treat metastatic melanoma.

A publicly traded company called NeoStem recently received an $18 million dollar grant from the California Institute for Regenerative Medicine for a new therapy being developed to help curb the damage done by melanoma. The injectable treatment, called NBS20, fights the disease by turning cancer cells against themselves. DOTmed News spoke to Dr. David J. Mazzo, the CEO of NeoStem, about the significance of the company's work.

DOTmed News: Can you tell us a bit about the development process for how this therapy came to be?

David J. Mazzo: NeoStem’s product candidate, NBS20, was developed initially by Dr. Robert Dillman, who was then with Hoag Cancer Center of Hoag Memorial Hospital Presbyterian in Newport Beach, California. Dr. Dillman is currently NeoStem’s Vice President of Oncology and the clinical lead on the NBS20 program.

In 2011, the technology platform that became NBS20 was acquired by California Stem Cell, Inc., which was subsequently acquired by NeoStem in 2014. In early 2015, NeoStem initiated the Phase 3 Intus Study (based on a Special Protocol Assessment from FDA) to investigate the therapy for the treatment of stage III recurrent and stage IV metastatic melanoma. That trial is now enrolling and randomizing patients.

The Intus trial is based on consistent, compelling results from two Phase 2 trials in identical patient populations evaluating the therapeutic vaccine that has become NBS20. The more recent of the two trials was a randomized trial comparing NBS20 to injections of autologous irradiated (inactivated) tumor cells in 42 patients. At two years, survival was 72 percent compared to 31 percent for control patients (p=0.007), which was consistent with the previous Phase 2 trial's findings in which NBS20 demonstrated 73 percent two-year survival in 54 patients, with a median survival of five years. Toxicity was minimal and consisted of mild to moderate local injection site reactions of the type normally associated with injections of GM-CSF (a protein secreted by immune cells that helps stimulate other immune cells to promote immune defenses against disease).

DOTmed News: What will the funding you've gotten allow you to do?

DJM: The funding from the California Institute for Regenerative Medicine serves two important purposes for NeoStem:

1) The grant directly supports a significant portion of the costs of our Phase 3 trial, and, in turn, supports our efforts to complete development, seek approval for the therapy from the regulatory authorities and make the therapy available to patients. That result could be both a great achievement for the company as well as have life-sustaining implications for people with metastatic melanoma.

2) The grant, given it size and the entity awarding it (the independent and esteemed California Institute for Regenerative Medicine), represents a scientific validation of the promise of the NBS20 therapy. We hope that this, along with the preferred status offered to the candidate (Fast Track and Orphan Drug designations) and our trial (Special Protocol Assessment) by FDA, provide a clear signal to the investment community of the potential for NBS20 to be paradigm-changing in the treatment of melanoma.

DOTmed News: How did you get the grant?

DJM: After learning of the announcement of the CIRM 2.0 initiative, our team moved very quickly to apply for this grant and answer the questions of the reviewing committee to the best of our ability. It is no small achievement for a relatively small biotech to pursue and achieve a grant of this size and it is a testament to our team, our technology and the previous trial results that we were awarded this grant.

DOTmed News: What are the hurdles standing between the drug and the U.S. market?

DJM: The Phase 3 trial must complete enrollment of 250 patients, expected by the fourth quarter of 2016, and we must complete the interim analysis, which is expected to occur in the fourth quarter of 2017. If the interim analysis is successful, we project a Biologics License Application submission to the FDA in the second quarter of 2018, leading to potential commercialization, if the FDA approves, as early as 2018 in the best-case scenario. The Fast Track designation of our candidate supports a priority review schedule by FDA.

DOTmed News: Do you have plans to get the therapy approved in other countries?

DJM: We have taken initial steps in the European Union, including achieving Advanced Therapeutic Medicinal Product classification from the European Medicines Agency. We plan to enroll patients in Canada, New Zealand, and Australia as well.

DOTmed News: What makes this therapy special compared to other therapies already on the market?

DJM: This product candidate offers the hope to improve the long-term survival of patients with metastatic melanoma. Recent therapies for metastatic melanoma have been approved that have yielded strong response rates without demonstrating a substantial impact on survival.

NBS20 is intended to help the immune system target cancer or tumor-initiating cells, which are thought to rapidly proliferate cancer cells, fuelling tumor growth, and ultimately spreading the disease throughout the body. NBS20 uses the patient’s own immune cells and tumor-initiating cells to create a therapeutic vaccine. NBS20 is unique in targeting these cancer or tumor-initiating cells; other therapies, which target different tumor cells, may treat existing cancer but may not be as likely to prevent tumor recurrence.