Sofetabart mipitecan for gynecological cancers earns FDA breakthrough status
Developer Eli Lilly has also a launched a Phase 3 clinical trial of potential treatment
Written by |
The U.S. Food and Drug Administration (FDA) has granted its breakthrough therapy designation to sofetabart mipitecan as a potential treatment for people with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received prior bevacizumab (sold as Avastin, among others) and Elahere (mirvetuximab soravtansine), if eligible.
The FDA gives this designation to experimental therapies that have the potential to fill unmet needs in the treatment of serious diseases, with the goal of speeding the development of potentially important new medicines.
“Platinum-resistant ovarian cancer remains one of the most challenging settings in gynecologic oncology, with limited treatment options and poor outcomes for patients,” Bhavana Pothuri, MD, a professor of obstetrics/gynecology and medicine at New York University Grossman School of Medicine, said in a press release from Lilly. “The breakthrough therapy designation and preliminary clinical data for sofetabart mipitecan across all levels of [folate receptor alpha] expression are encouraging and point to its potential as a meaningful treatment option for patients.”
Sofetabart mipitecan targets key protein
Sofetabart mipitecan, also known as LY4170156, contains an antibody that targets folate receptor alpha, a protein that’s often expressed by gynecological cancer cells. The cancer-targeting antibody is conjugated to a cell-killing molecule, and the therapy is designed to deliver the toxin specifically to cancer cells.
Eli Lilly, the drug’s developer, has also launched a Phase 3 clinical trial to test its experimental anticancer therapy sofetabart mipitecan in people with certain forms of gynecological cancer.
The study, dubbed FRAmework-01 (NCT07213804), aims to enroll more than 1,000 people with high-grade serous or high-grade endometrioid ovarian, primary peritoneal, or fallopian tube cancer that has failed to respond or has relapsed after current standard treatments. The study is being run at more than 200 sites worldwide, with some already recruiting participants.
The study is specifically enrolling two patient groups. One group will include patients whose tumor is resistant to platinum-based chemotherapy and who have already received treatment with the approved therapy bevacizumab. These patients will be randomly assigned to treatment with sofetabart mipitecan or their doctor’s choice of approved treatments, including chemotherapy or Elahere, a type of targeted therapy.
We are pleased the FDA has granted breakthrough therapy designation for sofetabart mipitecan, reflecting the significant unmet need in platinum-resistant ovarian cancer and the promising initial results shown in our Phase 1 study.
The second group will include patients whose cancer is sensitive to platinum-based chemotherapy. These patients will be randomly assigned to sofetabart mipitecan in combination with bevacizumab, or to an approved chemotherapy regimen combined with bevacizumab.
In both groups of patients, the main goal of the study is to determine whether treatment with sofetabart mipitecan can prolong progression-free survival, or the time patients remain alive without signs that their cancer has worsened.
The new FDA designation was based in part on data from an ongoing Phase 1 study (NCT06400472) testing sofetabart mipitecan in people with a variety of solid tumor types. Data from more than 100 people with platinum-resistant ovarian cancer showed that nearly all had a reduction in tumor size following treatment with sofetabart mipitecan, irrespective of how much folate receptor alpha protein was detectable on their tumor cells. The therapy was overall well-tolerated, with low incidences of alopecia (hair loss), peripheral neuropathy (nerve pain), and interstitial lung disease. The most common adverse events included nausea, fatigue, and anemia.
“We are pleased the FDA has granted breakthrough therapy designation for sofetabart mipitecan, reflecting the significant unmet need in platinum-resistant ovarian cancer and the promising initial results shown in our Phase 1 study,” said Jacob Van Naarden, executive vice president and president of Lilly Oncology and head of corporate business development. “Building on compelling results generated to date, we’ve initiated our Phase 3 FRAmework-01 trial with the goal of bringing a potential therapeutic option to patients with advanced ovarian cancer, across all levels of folate receptor expression.”
