Leap Therapeutics Presents Positive Top-line Results of DKN-01 Combination Therapy for Cholangiocarcinoma at the European Society for Medical Oncology 2016 Congress

Cambridge, MA – October 8, 2016 – Leap Therapeutics, Inc. today announced the presentation of top-line data from its clinical trial of DKN-01 combination therapy in patients with cholangiocarcinoma at the European Society for Medical Oncology 2016 Congress (ESMO 2016) in Copenhagen, Denmark.  Lipika Goyal, MD, of Massachusetts General Hospital, an investigator on the study, presented a poster entitled “Phase I study of DKN-01, an anti-DKK1 monoclonal antibody, in combination with gemcitabine and cisplatin in patients with advanced biliary cancer.”  DKN-01 is a humanized monoclonal antibody targeting Dickkopf-1 (DKK1), a secreted protein that modulates cell signaling of the Wnt pathways and that promotes an immunosuppressive tumor microenvironment.  Published studies have indicated that DKK1 expression levels are elevated in patients with cholangiocarcinoma and associated with worse overall survival.

The two-part open-label, dose-escalating study enrolled 27 patients with cholangiocarcinoma who were treated with DKN-01 in combination with gemcitabine and cisplatin.  In Part A, patients received DKN-01 at either 150 (n=4) or 300 mg (n=3) with 1000 mg/m2 of gemcitabine and 25 mg/m2 of cisplatin on days 1 and 8 of each 21-day cycle.  The Part B expansion portion of the study evaluated the 300 mg dose of DKN-01 in combination with gemcitabine and cisplatin in 20 additional patients.  The primary objective of this study was to evaluate the safety, pharmacokinetics, and efficacy of DKN-01 in combination with gemcitabine and cisplatin in treatment-naïve and previously-treated patients.

Data from the study showed that DKN-01 in combination with gemcitabine and cisplatin was well tolerated and safe at each dose level.  There were no DKN-01 reported related serious adverse events or dose limiting toxicities.  At the selected 300 mg DKN-01 dose level, 7 of 21 evaluable patients (33%) experienced a partial response and 20 patients experienced a partial response or stable disease, representing a disease control rate of 95%.  The median progression-free survival and overall survival have not yet been reached, as many patients remain on study receiving therapy.

“Patients with advanced cholangiocarcinoma have no approved therapies beyond standard gemcitabine/cisplatin chemotherapy and desire better clinical outcomes.  These early but promising results from the ongoing single arm study of DKN-01 in combination with gemcitabine and cisplatin demonstrate the potential of DKN-01 in aggressive cancers, such as cholangiocarcinoma, and provide a strong rationale for further clinical development,” commented Andrew Zhu, MD, of Massachusetts General Hospital, an investigator in the study and co-author on the poster.

About Cholangiocarcinoma

Cholangiocarcinoma is a cancer that starts in the bile duct, a thin tube about 4 to 5 inches long that reaches from the liver to the small intestine.  The major function of the bile duct is to move a fluid called bile from the liver and gallbladder to the small intestine, where it helps digest the fats in food.  The Cholangiocarcinoma Foundation estimates that approximately 6,000 patients will be diagnosed with cholangiocarcinoma in the United States each year, with publications estimating that nearly 200,000 patients are diagnosed worldwide each year.  The majority of cholangiocarcinoma cases are diagnosed with advanced stage disease with a 5-year survival rate of less than 10%.  The standard treatment option for advanced patients is systemic chemotherapy and supportive care.

About DKN-01

DKN-01 is a humanized IgG4 monoclonal antibody with neutralizing activity against the Dickkopf-1 (DKK1) protein. High levels of DKK1 expression has been associated with poor prognosis in multiple cancers, and DKK1 has a critical role in mediating Wnt signaling pathways and maintaining an immunosuppressive tumor microenvironment.  DKN-01 is currently being studied in clinical trials in esophageal cancer and cholangiocarcinoma.  DKN-01 additionally demonstrated single agent activity in NSCLC in a Phase 1 dose escalation study.

About Leap Therapeutics

Leap Therapeutics’ most advanced clinical candidate, DKN-01, is a humanized monoclonal antibody targeting the Dickkopf-1 (DKK1) protein. DKN-01 is in clinical trials in patients with gastroesophageal cancer in combination with paclitaxel and in patients with biliary tract cancers in combination with gemcitabine and cisplatin. Leap’s second clinical candidate, TRX518, is a novel, humanized GITR agonist monoclonal antibody designed to enhance the immune system’s anti-tumor response. Leap has signed a Merger Agreement with Macrocure Ltd (Nasdaq:MCUR) that is expected to result in Leap becoming a public company that will trade on The Nasdaq Global Market under the symbol “LPTX.” For more information about Leap Therapeutics, visit http://www.leaptx.com or our public filings with the SEC that are available via EDGAR at http://www.sec.gov.


This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. These statements include statements relating to Leap’s expectations with respect to the development and advancement of DKN-01, TRX518, and other programs, including the initiation, timing and design of future studies, enrollment in future studies, business development, and other future expectations, plans and prospects. Leap has attempted to identify forward looking statements by such terminology as ‘‘believes,’’ ‘‘estimates,’’ ‘‘anticipates,’’ ‘‘expects,’’ ‘‘plans,’’ ‘‘projects,’’ ‘‘intends,’’ ‘‘may,’’ ‘‘could,’’ ‘‘might,’’ ‘‘will,’’ ‘‘should,’’ or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Although Leap believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from our expectations. These risks and uncertainties include, but are not limited to: the accuracy of our estimates regarding expenses, future revenues, capital requirements and needs for financing; the ability to complete a financing or form business development relationships to fund our expenses; the outcome, cost, and timing of our product development activities and clinical trials; the uncertain clinical development process, including the risk that clinical trials may not have an effective design or generate positive results; our ability to obtain and maintain regulatory approval of our drug product candidates; our plans to research, develop, and commercialize our drug product candidates; our ability to achieve market acceptance of our drug product candidates; unanticipated costs or delays in research, development, and commercialization efforts; the applicability of clinical study results to actual outcomes; the size and growth potential of the markets for our drug product candidates; our ability to continue obtaining and maintaining intellectual property protection for our drug product candidates; and other risks. Detailed information regarding factors that may cause actual results to differ materially will be included in Leap Therapeutics’ periodic filings with the Securities and Exchange Commission (the "SEC"), including Leap Therapeutics’ Form 10-K that Leap filed with the SEC on February 23, 2018. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors. Any forward looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

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