The company is looking forward to enrolling patients with desmoid tumors to confirm the clinical benefits of nirogacestat

SpringWorks Therapeutics, a clinical-stage biopharmaceutical company, has reportedly announced the beginning of Phase 3 “DeFi” (Desmoid/Fibromatosis) study for evaluating nirogacestat, a selective, oral and small molecule gamma-secretase inhibitor, to treat adult patients having progressing desmoid tumors.

Seemingly, desmoid tumors are sporadic and are often disfiguring and debilitating soft-tissue tumors which can aggressively attack surrounding healthy tissues, including blood vessels joints, nerves, internal organs, and muscles. Based on their location and size, desmoid tumors can cause substantial morbidities, including internal bleeding, severe pain, debilitating loss of range of motion, disfigurement, and in rare cases desmoid tumor scan be lethal.

Sources familiar with the matter mentioned that an estimated 1,000 to 1,500 novel cases of desmoid tumors are diagnosed every year in the United States. For the treatment of desmoid tumors, there are presently no therapies approved by the U.S. FDA.

Chief Executive Officer of SpringWorks Therapeutics, Saqib Islam stated that those patients who are living with these debilitating tumors, the DeFi trial represents a significant step forward and could result in the first approved treatment for this underserved patient community.

Apparently, the company is looking forward to enrolling patients with desmoid tumors to confirm the clinical benefits of nirogacestat.

In June 2018, the FDA approved Orphan Drug designation for nirogacestat for the treatment of patients who were suffering from desmoid tumors, and in November 2018, the FDA granted Fast Track designation for nirogacestat in the treatment of adult patients with, unresectable, progressive, refractory or recurrent desmoid tumors or deep fibromatosis.

For the uninitiated, the DeFi trial is a double-blind, randomized, placebo-controlled, global Phase 3 trial for evaluating the efficacy, tolerability, and safety of nirogacestat in adult patients with progressing desmoid tumors. The study will enrol around 100 adult patients, who will receive placebo or 150 mg of nirogacestat twice daily.

Source Credit: https://www.springworkstx.com/wp-content/uploads/2019/05/DeFi-Study-Initiation-Press-Release.pdf