2013 ANNUAL REPORT
To our Stockholders,
Endocyte achieved significant milestones in 2013 and is further establishing its position as a world-leader in the development of small molecule drug conjugates (SMDC) and companion imaging agents. We remain committed to advancing precision medicines to provide patients with cancer and other difficult-to-treat diseases with improved therapeutic options. I am enthusiastic about Endocyte’s future success and building on our accomplishments in 2013.
Our proprietary technology and continuous learning has enabled us to further advance additional investigational SMDC therapeutic candidates and each respective companion imaging agent. Our world-class team of researchers and partners in academia begin with their deep understanding of disease biology to design SMDCs to target receptors that are preferentially expressed on diseased cells. Our SMDC pipeline consists of highly targeted and potent investigational therapeutics that are delivered directly to the diseased cells while avoiding healthy cells, minimizing side effects. A key differentiator in our development paradigm is that each of our SMDCs is co-developed with a companion imaging agent which is used to non-invasively identify patients’ target receptor status at the time therapy would be administered—a truly unique and advanced approach to precision medicine. The receptor status information obtained from using the companion imaging agent is extremely useful in designing SMDCs and clinical trials. This information is used to differentiate which patients’ disease expresses the target receptor and are most likely to benefit from SMDC therapy and those patients without the target receptor who are less likely to benefit from SMDC therapy. Our novel paradigm for the development of precision medicine will help to advance the interests of investigators, regulatory agencies, payors, and patients.
Entering the Commercialization Era and Clinical Advancement of Vintafolide and Etarfolatide
On March 21, 2014 the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued positive opinions for the Conditional Marketing Authorization of etarfolatide and vintafolide for the selection and treatment of adult patients with folate receptor-positive platinum-resistant ovarian cancer (PROC). We expect the European Commission to issue the approval for the Conditional Marketing Authorizations in the second quarter. Together with our partner, Merck, we will launch the products in the European Union following the approval. We have a very strong and experienced team in place in the EU that has been preparing the market for the launch of etarfolatide and vintafolide.
When approved, vintafolide and etarfolatide will be the first SMDCs marketed, the first that use the folate-receptor as a biomarker, and the first targeted therapeutic that uses a companion imaging agent for PROC. This is a transformative event for Endocyte and will usher in a new era of commercializing our discoveries.
Advancing the clinical development of vintafolide and etarfolatide is also progressing into multiple indications including ovarian, non-small cell lung cancer, triple-negative breast cancer and with different drug combinations for cancer. The Phase 3 randomized PROCEED registration trial for ovarian cancer continues on plan to enroll patients globally and we expect to finish enrollment by early 2015. The randomized Phase 2b TARGET study for non-small cell lung cancer met the primary endpoint of progression free survival and overall response rate. Interim overall survival data was also positive with final survival results expected at the end of this year. The third investigational indication for vintafolide is triple-negative breast cancer; our partner Merck plans to initiate a randomized Phase 2 trial in the second quarter. A phase 1 safety study combining vintafolide with the commonly used drugs platinum and paclitaxel, is nearing completion with plans to test the triplet therapy in endometrial cancer. Additionally, vintafolide and etarfolatide received orphan drug designation by the FDA for the treatment of ovarian cancer. This designation had previously been received in the EU. The full data set from the Phase 2b PRECEDENT study was published in the Journal of Clinical Oncology. Finally, we were pleased to enter into an exclusive license agreement with Nihon Medi-Physics (NMP) to develop and commercialize our folate-targeted companion imaging agent, etarfolatide, for cancer in Japan.
Expanding the Proprietary Pipeline
In 2013, we advanced the development of our proprietary pipeline of SMDCs and companion imaging agents. Notably, in the fourth quarter we launched a Phase 1 trial of folate-targeted tubulysin (EC1456) for cancer patients with advanced solid tumors, using etarfolatide as the companion imaging agent. This represents the second SMDC in Endocyte’s pipeline to advance into clinical development, and we look forward to its continued clinical development in 2014. We also made significant progress with the clinical strategy of our prostate-specific membrane antigen (PSMA)-targeted tubulysin SMDC, EC1719, co-developed with the companion imaging agent EC0652, and plan further advancements in 2014 with the filing of the IND and subsequent Phase 1 trial. We have a number of exciting new SMDCs in discovery with plans to further expand our oncology pipeline.
Strengthening our Financial Position
We ended 2013 with a cash balance of approximately $149 million, and our cash position remains strong as we continue to invest in the development of our proprietary pipeline going forward. Merck will be financially responsible for the development of vintafolide in new indications and funds manufacturing costs for vintafolide, along with a portion of the PROCEED trial and all of the TARGET trial costs.
We look forward to 2014 as a transformational year for Endocyte with upcoming milestones that include a potential EU launch and broader development of vintafolide and etarfolatide along with the continued expansion of our proprietary pipeline. We believe that Endocyte is well positioned for great success over the coming years. I am confident that the continued dedication of the Endocyte team to excellence, integrity and the development of precision medicines for patients with debilitating diseases will drive growth of the organization and its value to stockholders over the coming year. On behalf of Endocyte and its Directors, I would like to thank the investigators and patients who have been and are participating in our clinical programs, the stockholders and everyone who has supported us in our journey to advance precision medicine. We look forward to your continued involvement and will be updating you in the coming year.
Ron Ellis President and Chief Executive Officer UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549
Form 10-K