BP-100-1.01

BP-100-1.01 is Bio-Path’s co-lead product, a liposomal delivered antisense cancer drug. The scientific name for BP-100-1.01 is L-Grb-2 (growth factor-bound protein-2), a liposome delivered antisense drug that targets an estimated $1 billion annual Chronic Myelogenous Leukemia (”CML”) market. There are approximately 40,000 patients in the U.S. with CML. Currently most patients are treated with Gleevec (Novartis), an inhibitor of the tyrosine kinase bcr-abl, the causative agent of CML. Gleevec is quite effective at treating CML but many patients develop resistance to Gleevec and, consequently, recurrence of their disease.

Grb-2 is an adaptor protein that links tyrosine kinases such as bcr-abl with their downstream signaling molecules, including RAS. RAS is a critical regulator of cellular proliferation. The Company’s collaborators have demonstrated that L-Grb-2 is effective in inhibiting the growth of human CML cells in animal models. All preclinical studies have been completed and the Company has filed an IND for this drug with the FDA. This project has been granted Orphan Drug status and the Company will be applying for an Orphan Drug grant of up to $600,000.

The initial clinical target for BP-100-1.01 is Gleevec-resistant CML. Another potential clinical target is breast cancer where it has been shown that down regulation of Grb-2 leads to inhibition of growth in breast cancer cells that express high levels of the EGFR or ErbB-2 (another member of the EGFR gene family) tyrosine kinases.

In addition to the significant benefit in treating CML patients, the BP-100-1.01 program represents an ideal system for proof of concept of the Company’s nucleic acid delivery technology. Purified populations of the human tumor cells are easily obtained from the blood and bone marrow of patients and can be used to evaluate the presence of the specific nucleic acid molecules and their effect on Grb-2 expression. Positive results from this work, (as predicted by the Company’s existing in-vivo and in-vitro pre-clinical data), will prove the clinical value of the Company’s novel drug delivery platform.