NeoStem announces licensing agreement to begin Phase II hepatocellular carcinoma trial in China

The trial, to be conducted in China in accordance with international standards, will investigate the use of the Company’s developmental patient-specific cancer immunotherapy known as DC-TC, in patients with hepatocellular carcinoma (HCC).

Pursuant to the terms of the license agreement, the cost of the Phase II trial will be borne by CBMG, and CBMG will also be responsible for all clinical, marketing, and, should a product be approved, commercialization activities in China.

NeoStem will be responsible for all regulatory filings and will directly control and manage all manufacturing processes and procedures relating to the development of its DC-TC product. NeoStem could receive up to $30m in licensing milestone fees over the life of the agreement, with $1m payable upon enrollment of the first patient in the currently planned Phase II trial. Royalties and sublicense fees may also be payable.

The company’s DC-TC therapy involves growing cancer initiating (stem) cells that have been isolated from a patient’s resected tumor sample, and then enriching and inactivating them.

This newly created cancer initiating (stem) cell line is then combined with dendritic cells, a type of antigen-presenting immune cell that is derived from the same patient’s blood. The product is then reintroduced to the patient via a series of subcutaneous injections.

The planned study follows a Phase I trial which demonstrated safety in HCC patients. There were no serious adverse events related to the treatment in patients who co-presented with active hepatitis B and underlying cirrhosis, commonly associated with liver cancer in China, and patients were not charged for treatment.

NeoStem chairman and CEO Dr Robin Smith noted that the planned Phase II study in China is seeking to assess the efficacy and safety of our new DC-TC platform technology in treating hepatocellular carcinoma, and we look forward to working with CBMG to develop this indication in Asia.

The company has also recently reached agreement with the US Food and Drug Administration to begin a Phase III clinical trial for its lead product candidate, Melapuldencel- T, in metastatic melanoma, expected to begin in 2014.

Melapuldencel-T’s Phase 3 protocol has been granted Special Protocol Assessment and the product candidate has received Fast Track designation for metastatic melanoma, as well as Orphan Drug designation. Five year survival data from the Phase II clinical trial in metastatic melanoma has been accepted for a poster presentation at The American Society of Clinical Oncology’s 2014 annual meeting in June.