BP1003 (Liposomal Stat3) for Pancreatic Cancer

Pancreatic adenocarcinoma (PDAC) is a cancer of the exocrine cells of the pancreas. Approximately 50,000 people are diagnosed with PDAC each year while 40,000 die from the disease, placing it 4th for cancer deaths despite being the 12th most common cancer. It is estimated that by 2030, PDAC will be the second most lethal cancer after lung cancer. This is due to rapid advancements that have been made in the treatment of other cancers as well as the rise of type II diabetes, a risk factor for development of PDAC.

Because most people are diagnosed with PDAC late in the disease’s progression, survival rates are very low. Typical survival for a metastatic or advanced patient is only 6-9 months from diagnosis. Treatment of this disease is hampered by the location of the pancreas, which is difficult to reach with conventional therapies and the fibrotic nature of the tumors, which protects them from penetration by chemotherapeutics. A novel and unconventional therapeutic is needed to overcome these barriers to treatment.

BP1003 is an RNAi nanoparticle containing antisense DNA targeting Stat3, a protein associated with increased PDAC severity and poor survival in the majority (80-100%) of pancreatic tumors. Bio-Path intends to initiate IND-enabling studies of BP1003 in 2018.

In addition, expression of Stat3 has been associated with higher disease severity in numerous other cancers including non-small cell lung (NSCLC), prostate, cervical, breast, colon and stage III and IV ovarian cancers. Further, dysregulation of the Jak-Stat pathway is also involved in both autoimmunity and cancer, suggesting that BP1003 may have potential to treat systemic diseases and syndromes for which immunomodulation is needed. Preclinical discovery for BP1003 in additional solid tumors is underway.

A drug that inhibits Stat3 will have significant clinical impact for all solid tumors that express Stat3.


Prexigebersen (Liposomal Grb2 antisense) is Bio-Path’s lead product candidate, a neutral-charge, liposome-incorporated antisense drug designed to inhibit protein synthesis of Grb2 (growth factor receptor bound protein 2).

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