Overview

Genus Oncology has developed a new & proprietary technology platform with the potential to improve treatment of cancer.

Solid science. On track to success.

Genus Oncology was formed in 2007 with a mission of discovering, developing, and commercializing new anti-cancer agents that target the Mucin 1 (MUC1) oncoprotein. The incidence of overexpressed MUC1 in a wide array of carcinomas and hematologic malignancies has been known by researchers for many years. Until now, no viable approach existed to selectively target and block the function of MUC1 that leads to the formation of tumors.

Genus has now identified drug candidates that block the key intracellular functions of MUC1, and thereby, inhibit the ability of targeted tumor cells to survive and proliferate. Both in vitro and in vivo animal studies have demonstrated success in the complete regression of breast, prostate, lung, colon, ovarian, and pancreatic carcinomas. More importantly, our technology has demonstrated significant efficacy in treatment of refractory cancers, where current therapeutic options are extremely limited.

Phase I clinical trials are now underway in the U.S.

Key Points of Differentiation

Genus has developed a technology platform that include diagnostic tests that can be used to predictively screen candidates qualified for therapy and potentially offer drugs with high success rates across a large number of cancers. In brief:

  • MUC1 is significantly over expressed in 80-90% all solid tumors and in many hematologic malignancies (multiple myeloma, lymphoma, acute myelogenous leukemia, chronic myelogenous leukemia);
  • We offer a targeted approach to identify those patients who will respond to MUC1 therapy. Genus has two diagnostic tests: one that can qualify those patients whose cancers will likely respond to our peptide therapy; and a second to monitor the long term success of their treatment;
  • Genus’ lead drug candidate is a stable synthetic peptide that is designed to enter cancer cells and kill MUC1 function;
  • In vivo and in vitro studies have demonstrated that the lead drug candidate blocks MUC1 activity in targeted tumors, resulting in tumor cell death. Preclinical studies have demonstrated that the Genus peptides selectively target cells over expressing MUC1;
  • Genus has an extensive patent estate which covers both compositions of matter and use of our technologies for treating cancer;
  • Genus has an active small molecule pharmaceutical development program that has identified the key MUC1 binding sites, and identified key molecular scaffolds which block MUC1 function at the site. We are currently designing and testing drug candidates for activity with the goal of identifying a lead candidate in early 2012.

The Market

The market opportunity for a drug effectively targeting MUC1 is substantial. We estimate that MUC1 is a viable therapeutic target in approximately 900,000 cases of cancer in the US annually, and millions worldwide. For perspective, the cancer targets of Erbitux (EGFR), Herceptin (ErbB2), and Avastin (VEGF) together account for roughly 350,000 US cancer cases annually; Datamonitor projects these three agents will generate in excess of $6B in revenues in the US in 2009.

Given the safety and efficacy profile demonstrated in our preclinical studies, we expect our lead drug would be used as first line therapy alone and in combination with standard treatment for many cancers, and as primary treatment for those cancers which have become refractory to standard of care.

Our lead drug candidate is a first-in-class agent. Based on the activity seen to date across different tumor types, and the incidence and potential market sizes of those targets, we expect that the Genus peptide drug could achieve sales of over $5 billion annually and offer a significant benefit to patients in need of new cancer treatments.

Proprietary Technology

Genus has maintained a unique and leading position in the MUC1 field, driven by recent discoveries made at Dana-Farber Cancer Institute (DFCI), Harvard Medical School. While competitive programs have focused on peptide vaccines and antibodies targeting the MUC1 ectodomain (the segment of the protein outside the cell), we have focused on MUC1 protein segment inside the cell — the intracellular domain.

Genus intellectual capital includes over 20 years of research at DFCI and the proprietary discovery that the MUC1 receptor confers transformation and resistance of cancer cells to therapy. Our researchers have recently defined the growth and death pathways influenced by MUC1 and identified key binding sites with other proteins that influence the transformation from normal cells to tumor cells.

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