DTRF Annual Patient Meeting Casey Cunningham, M.D. Chief Medical - - PowerPoint PPT Presentation

DTRF Annual Patient Meeting

Casey Cunningham, M.D. Chief Medical Officer September 21, 2019

• Tegavivint is a given as a once-weekly

intravenous infusion that has been shown to decrease beta-catenin protein levels.

• Desmoid tumors are known to solely be

caused by elevated beta-catenin protein.

• So, Tegavivint specifically targets the root

cause of desmoid tumors

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What is Tegavivint?

Desmoid Cell Line

Tegavivint

Phase 1/2a clinical study enrolling patients

• Open-label, multi-center, non-randomized study to evaluate safety of Tegavivint in patients with a

desmoid tumor that is unresectable and symptomatic or progressive.

• Once weekly intravenous infusion. Three weeks on drug followed by a week off.
• Enrolling at six centers in United States and one in Canada.
• Currently enrolling 6th cohort in Phase I dose escalation portion of study.
• All patients participating in Phase I will have the choice to automatically escalate to the recommended

phase 2 dose (RP2D) when we transition to the Phase 2a expansion phase of study, which will include 25 patients.

• For more information please visit

(https://clinicaltrials.gov/ct2/show/NCT03459469?term=tegavivint&rank=1)

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Phase I clinical trial update

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• Twelve patients have been treated by drug with 10

months being the longest treatment time

• No significant drug related side effect reported
• Patient receive MRI scan every three months
• All treated patients have stable disease with

decreasing tumor size in those that have received at least six cycles on drug

• Due to low toxicity profile and preliminary efficacy,

patients and physicians requested that we amend clinical protocol to allow for up to two years of treatment

3 6 9

• 50
• 40
• 30
• 20
• 10

10 20 30 40 50

Tumor Growth Time in treatment (Months)

%Change from baseline (WHO)

03-02, 2 mg/kg 3-01, 1 mg/kg 04-01, 2 mg/kg 05-01, 2 mg/kg 04-02, 3 mg/kg 02-01, 3 mg/kg 05-02, 3 mg/kg

(cohort 2) (cohort 3) (cohort 4) (cohort 5)

Currently enrolling clinical centers

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Institution Investigator Study Coordinator

Princess Margaret Cancer Centre (Toronto, ON) Albiruni Razak, MD Horace Wong, MSc, HonBSc horace.wong@uhn.ca Dana Farber Cancer Institute (Boston, MA) Suzanne George, MD Abigail Porcello aporcello@partners.org Massachusetts General Hospital (Boston, MA) Edwin Choy, MD Lia Tamburello lmtamburello@mgh.harvard.edu Seattle Cancer Care Alliance (Seattle WA) Lee Cranmer, MD Roxanne Moore romoore@seattlecca.org MD Anderson Cancer Center (Houston, TX) Vinod Ravi, MD Shreyaskumar Patel, MD Giberto Botello GBotello@mdanderson.org Memorial Sloan Kettering Cancer Center (New York, NY) Mrinal Gounder, MD Moriah Martindale martindm@mskcc.org The Ohio State University Comprehensive Cancer Center (Columbus, OH) David Liebner, MD Cassondra Faiella Cassondra.Faiella@osumc.edu