Cornerstone Pharmaceuticals Announces Published Data on CPI-613® (Devimistat) as an Effective Approach to Targeting Carcinoma Catabolism – UPDATE

Cranbury, N.J., June 16, 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 a publication on preclinical data supporting CPI-613^® (devimistat) targeting carcinoma catabolism.

The study published in PLOS ONE titled, “Evidence for a novel, effective approach to targeting carcinoma catabolism exploiting the first-in-class, anti-cancer mitochondrial drug, CPI-613,” addresses the multi-target, tumor-preferential inhibition of the mitochondrial tricarboxylic acid (TCA) cycle by the first-in-class drug, devimistat. By suppressing the TCA hub, indispensable to many metabolic pathways, devimistat substantially reduces the effective redundancy of tumor catabolism.

The authors of the study note that their preclinical results indicate a clearly defined, detailed, potentially general class of approaches to improved clinical targeting of carcinoma catabolism. Devimistat preferentially suppresses multiple essential components of the TCA cycle in carcinomas.

Moreover, they noted that devimistat has shown occasional strong single agent activity against otherwise largely untreatable relapsed, refractory, Myc-driven Burkitt lymphoma in ongoing clinical trials. Most recently, these responses include one complete remission in a set of eight Phase 1 patients.

“We’re proud to share the results from this recent study, as it demonstrates breakthroughs in understanding how to target carcinoma catabolism and devimistat’s potentially significant role,” said Sanjeev Luther, President, CEO and Chairman of Cornerstone Pharmaceuticals. “Devimistat has the potential to bring new hope for remission and survival for patients suffering from rare cancers and serve as a chance to fill the substantial gap in treatment.”

The authors of the study conclude: “Our results herein…indicate that continued investigation of the metabolic basis of CPI-613 resistance is likely to yield practical, promising approaches to substantially improved clinical attack on cancer metabolism.”

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 www.cornerstonepharma.com.

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