The Biotech Forum Free Report Oct 2016 | Page 4

Late Stage Therapies VB-111

What it Does: VB-111 kills the endothelial cells that create new blood vessel connections for the tumor. An engineered virus carries gene therapy which is activated by an excretion (TNFα) from the tumor itself, leading to apoptosis (effectively suicide) of the endothelial cells, ultimately starving the tumor, and inhibiting growth.

Key Differentiator(s):
First in class, novel therapy.

Received fast track designation in the U.S. for prolonging survival in patients with recurrent glioblastoma who have already received the standard treatment of chemotherapy and radiation.
Triggering the drug with TNFa (Tumor Necrosis Factor alpha) produced by the tumor is intended to directly target the therapy to the tumor, and spare normal tissues and blood vessels.
Results indicate it is safe, well tolerated, and combinable.

Easily administered using an IV infusion, once every 2 months.

Phase / Status / Expected Launch: VB-111 had very positive phase two results and is now in Phase III trials for rGBM. Interim trial results will be analyzed in the first half of 2017 and if the results of the interim data are positive, a BLA will be filed in 2018 and the FDA is likely to determine the status within 6 months due to SPA/Fast Track, and orphan status.
Expected Market: There are approximately10,000 new Glioblastoma multiforme (GBM) cases, 60,000 Thyroid cancer cases, and 22,000 ovarian cancer cases per year in the United States. According to Transparency Market Research in June of 2015, the global glioblastoma treatment market alone is estimated to reach USD 0.91 billion by 2022. Based on the Fast Track SPA Status, it is reasonable to assume that if approved, VB-111 will become part of the future standard of care to increase survival timeframes for many of these patients.

Other Considerations:
As per SPA Fast Track designation in the U.S. , incomplete Phase III trial data & analysis are acceptable for FDA approval and could result in the product getting into the market as soon as early 2018.

Received orphan drug designation in both the United States and Europe which translates to tax breaks, other financial incentives, and seven years of exclusivity for the treatment of rGBM if approved.

Current trials are for Glioblastoma multiforme, Thyroid cancer and Ovarian cancer specifically but the therapy has the potential for additional solid tumor indications.
Phase II results announced in the past quarter indicated double the response rate over the current standard of care.