Bound Therapeutics LLC executed an exclusive license from Thomas Jefferson University to use our novel platform technology (Compositions and Methods of Using MicroRNA Inhibitors, PCT/US2015/015681) to develop microRNA therapeutics and diagnostics.
Preliminary data showed that our proprietary lead compound designed using our platform reversed off-target effects from conventionally designed microRNA-blocking agents in a triple negative breast cancer cell model.
Bound Therapeutics agents achieve cancer cell-specific delivery of the lead compound through receptor-mediated endocytosis by conjugation with a small peptide (Wickstrom and Basu, Peptide Nucleic Acid Conjugates. U.S. 6,180,767, 2001).
Our platform yields single molecule agents that circulate and enter cells with no need for liposome or nanoparticle formulations. We will optimize agents for the needs of each target and customer.
Triple negative breast cancer (TNBC) is an aggressive form of breast cancer at a later stage. TNBC accounts for 20% of all breast cancers, striking and killing about 40,000 US women each year. There is a tremendous gap in treating TNBC due to the lack of effective targeted agents. The only available supplemental option for chemotherapy, Avastin, was invalidated by FDA for the breast cancer indication in 2011 as a result of poor clinical outcome and safety issues. Drugs in the late stage pipeline include poly(ADP-ribose) polymerase (PARP) inhibitors and immune checkpoint blockers. These are only modestly effective in treating a small subset of TNBC patients.
Only routine chemotherapy and radiation are the standard of care, with significant side effects that aggravate the suffering of patients. Therefore, a new molecular therapeutic agent that works in conjunction with chemotherapy with greater efficacy, specificity, and safety will bridge the gap in TNBC therapy.
Bound Therapeutics designs novel molecular targeting agents that comprise an attractive option for patients. We minimize side effects through a platform technology that enables cancer cell-specific delivery of all compounds. Faster response, decreased dosage, and decreased side effects will reduce the longterm cost of disease management.
Assuming the cost of our lead compound for TNBC is comparable to that of Avastin, the projected annual market potential will reach $2.8 billion, provided that the cost for the new treatment is $100,000/patient/year.
MicroRNA therapeutics is a growing field with a market size of $70.5 million as of 2013 and a compound annual growth rate (CAGR) of 14.9%. Bound Therapeutics LLC will focus on commercializing our lead compound in triple negative breast cancer. The initial developmental phase from lead optimization to phase I clinical safety trial will be supported by non-dilutive federal research grants in parallel with venture capital placements. Our exit strategy lies in acquisition by a pharmaceutical firm with a broad breast cancer therapeutics portfolio in order to complete phase II/III trials and the commercialization of the lead compound within the next 10 years.
In the long term, Bound Therapeutics will increase our revenue stream by branching out to other therapeutic markets including lung cancer, cardiovascular disease, and diabetes. These chronic diseases display high incidence in the U.S., with 224,000 new cases for lung cancer, 720,000 for heart disease, and 1.7 million for diabetes every year. In the short term, we will develop individual compounds applicable to each therapeutic indication in alliances with pharmaceutical companies that are determined to broaden their pipeline. We will generate additional profit from joint ventures and royalty payments with each compound.
The orphan status of triple negative breast cancer enables us to expedite FDA approval. Bound Therapeutics will complete the initial stages of development from proof-of-concept to lead optimization with non-dilutive capital by obtaining federal Small Business Innovative Research funding. In parallel, we will secure venture capital to take our lead compound all the way through a phase I clinical safety trial.
As a primary focus, we will commercialize our lead compound for triple negative breast cancer as a proof-of-principle for our platform. As a secondary focus, Bound Therapeutics will expand our pipeline of microRNA-blocking or supplementation agents to other therapeutic markets, such as lung cancer, cardiovascular disease, and diabetes.
In August 2016, we filed national stage patent applications in countries that invest the most in microRNA research, including the US, European Union, and Japan.
On 27 April, the USPTO allowed our application for "Bound Therapeutics" as a registered trademark. We will use the "BND" prefix on each of our candidate leads and tool compounds. For example, BND4037 blocks the miR-17-5p guide strand, without imitating the activity of the miR-17-3p passenger strand.