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Endocyte, Inc., is a biopharmaceutical company developing targeted therapies for the treatment of cancer and other serious diseases. We use our proprietary technology to create novel small molecule drug conjugates, or SMDCs, and companion imaging diagnostics. Our SMDCs actively target receptors that are over-expressed on diseased cells, relative to healthy cells. This targeted approach is designed to enable the treatment of patients with highly active drugs at greater doses, delivered more frequently, and over longer periods of time than would be possible with the untargeted drug alone. We are also developing companion imaging diagnostics for each of our SMDCs that are designed to identify the patients whose disease over-expresses the target receptors of the therapy and who are therefore more likely to benefit from treatment. This combination of an SMDC with its companion imaging diagnostic is designed to personalize the treatment of patients by delivering effective therapy, selectively to diseased cells, in the patients most likely to benefit.

Vintafolide (EC145) and the PRECEDENT Study

Our lead SMDC, vintafolide (EC145), targets the folate receptor, which is frequently over-expressed in some of the most prevalent, and difficult to treat, solid tumor cancer indications, including non-small cell lung, breast, colorectal, kidney, endometrial and other cancers including ovarian cancer.

Specifically, women with ovarian cancer may have disease that becomes resistant to the standard chemotherapy used to treat patients. No new drug has been registered for use in this area of high unmet medical need in over 10 years. Final data from our PRECEDENT study, a randomized Phase 2 trial of vintafolide (EC145) in combination with pegylated liposomal doxorubicin (PLD), versus PLD alone in women with platinum-resistant ovarian cancer has shown an increased median progression free survival (PFS) of 21.7 weeks vs. 11.7 weeks for the control arm (HR 0.626, p-value 0.031). The study also utilized etarfolatide (EC20), an investigational companion imaging diagnostic that is designed to identify patients with folate receptor positive tumors. As expected, in the folate receptor positive patients the improvement in PFS was even greater. In the subpopulation most positive for the folate receptor, PFS increased 17.4 weeks from 6.6 weeks to 24 weeks. The hazard ratio for PFS was 0.381 (p=0.018). Vintafolide (EC145) with PLD is the first combination to show a statistically significant increase in PFS for women with platinum resistant ovarian cancer.  A Phase 3 randomized study (PROCEED) further exploring the efficacy of vintafolide (EC145) + PLD in women with platinum resistant ovarian cancer has now begun accrual. Details of the study can be found at www.clinicaltrials.gov.

Small Molecule Drug Conjugates (SMDC) Technology

We believe our SMDC platform represents a novel approach, comparable to antibody drug conjugates, or ADC’s, in its ability to deliver highly active drug payloads in a targeted manner, but with a number of potential advantages:

1. Small size to better penetrate solid tumors. The targeting ligands for our SMDCs are approximately 300 times smaller in molecular weight than a typical antibody incorporated in ADCs. This may result in greater uptake and higher concentrations of these molecules within solid tumors.

2. Rapid clearance for reduced toxicity. The circulating half-life of ADCs currently in development generally range from several hours to several days. In contrast, our SMDCs are engineered to provide rapid uptake in targeted cells and rapid clearance from the bloodstream with a half-life of approximately 20 minutes. As a result of this shorter half-life, we believe there is a reduced risk that our SMDCs will release the unconjugated drug payload into the blood stream.

3. Companion imaging diagnostics for targeted therapy. A companion imaging diagnostic can be created for each of our SMDCs. The companion imaging diagnostic is intended to allow for real-time, full-body assessment of the receptor target without requiring an invasive tissue biopsy. Using full-body imaging, the receptor expression can be measured in every tumor and monitored throughout treatment.

4. Cost-effective and simple to manufacture. Our SMDCs are relatively simple to manufacture and do not have the complexity and expense of biological molecules, like antibodies and ADCs.

SMDC Pipeline

Endocyte is developing a number of SMDCs and companion imaging diagnostics which leverage our modular platform technology.  EC1456 targets a “super-potent” chemotherapy drug called tubulysin using our second-generation guidance system. With EC0652, we replaced the folate receptor targeting ligand with a ligand that binds to prostate specific membrane antigen, or PSMA.

Beyond Cancer

In addition to cancer, we have discovered that activated macrophages, a type of white blood cell found at sites of acute and chronic inflammation, also over-express the folate receptor. Activated macrophages release a variety of mediators of inflammation that contribute to a broad range of diseases, such as rheumatoid arthritis, osteoarthritis, inflammatory bowel disease and psoriasis. We have a number of SMDCs in preclinical development that are designed to inhibit the production of pro-inflammatory cytokines by activated macrophages.

Company History

Endocyte’s technology and therapeutic focus are derived from groundbreaking research conducted in the late-1980s and first published in 1991. Philip Low, PhD, a biochemist at Purdue University together with fellow Purdue biochemist Chris Leamon, PhD, now vice president of research at Endocyte, were the first to discover a mechanism to exploit the folate receptors as a means to deliver bio-active molecules inside cells. Using this strategy the vitamin would “carry” an attached drug directly to targeted cells and then into the targeted cells interior using a normal pathway for vitamin update. Recognizing the potential to advance the promising applications of receptor-targeted therapeutics, Dr. Low joined with Ron Ellis and investors to found Endocyte. The company currently has 80 employees.