European Medicines Agency Grants Orphan Drug Designation to Cornerstone Pharmaceuticals’ CPI-613® (Devimistat) for Treatment of Patients with Advanced Unresectable Biliary Tract Cancer

Cranbury, NJ, May 24, 2022 (GLOBE NEWSWIRE) -- Cornerstone Pharmaceuticals, Inc. (“Cornerstone” or the “Company”), a company focused on rare cancer therapeutics and formerly known as Rafael Pharmaceuticals, today announced that the European Medicines Agency (EMA) has granted orphan drug designation to CPI-613® (devimistat) for the treatment of advanced unresectable biliary tract cancer.

Also known as cholangiocarcinoma, biliary tract cancer is an extremely rare and hard to treat cancer, affecting just over 2,000 people in the UK annually with incidents steadily increasing every year.

“We are on a mission to develop cancer treatments for patients who have significant unmet clinical needs. Biliary tract cancer is considered rare and aggressive, with a large gap in effective treatment options,” said Sanjeev Luther, President and CEO of Cornerstone Pharmaceuticals. “Our goal is that this milestone will provide hope for patients and families in a setting where the current prognosis is devastating.”

This orphan drug designation is the fourth granted for devimistat by the EMA, following existing designations for Burkitt’s lymphoma, pancreatic cancer and acute myeloid leukemia (AML). The Food and Drug Administration (FDA) has granted orphan drug designation for devimistat in seven indications in the United States, including in biliary tract cancer.

“With strong and continued Phase 2 enrollment rates and additional trial sites opening, the research community remains engaged towards identifying the capabilities devimistat has in biliary cancer,” said Dr. Vaibhav Sahai, MBBS, M.S., principal investigator with Cornerstone’s Phase 2 trial for patients with biliary tract cancer in combination with gemcitabine and cisplatin. “Devimistat is a potentially life-saving therapy, bringing hope to patients and loved ones affected by biliary tract cancer.” 

About CPI-613® (Devimistat)
CPI-613® (devimistat) is a first-in-class clinical lead compound of Cornerstone, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases cellular stress and the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients’ benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, soft tissue sarcoma, Burkitt’s lymphoma and biliary tract cancer. The European Medicines Agency (EMA) has granted orphan drug designation to devimistat for pancreatic cancer, acute myeloid leukemia and Burkitt’s lymphoma.

About Cornerstone Pharmaceuticals, Inc.
Cornerstone Pharmaceuticals (“Cornerstone”) is committed to the development of therapies for rare cancers that have few to no treatment options available. Cornerstone’s lead compound, CPI-613® (devimistat), is designed to target the mitochondria of cancer cells in order to disrupt their energy production, cutting off the fuel for disease growth. Devimistat is undergoing multiple clinical trials and has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the following indications: Burkitt’s lymphoma, biliary tract cancer, soft tissue sarcoma, myelodysplastic syndrome (MDS) and peripheral T-cell lymphoma. In addition, the European Medicines Agency (EMA) has granted orphan drug designation to devimistat for Burkitt’s lymphoma.

With science stemming from the world’s most renowned institutions and compassion that runs deep, Cornerstone strives to improve the lives of people with rare cancers, inaugurating a new era of hope. For more information, please visit

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